{"nct_id": "NCT04654689", "title": "Impact of the Combined Treatment of Liposomed Polyphenols With G04CB02 on the ALS Patients", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fundaci\u00f3n Universidad Cat\u00f3lica de Valencia San Vicente M\u00e1rtir", "summary": "Amyotrophic lateral sclerosis (ALS) is a disease of an inflammatory nature, which causes progressive muscle weakness associated with cognitive and behavioural disorders. Pathogenically, it is characterised by loss of oxidative control, excitotoxicity due to excess glutamate and intestinal dysbiosis. In the absence of curative treatment, the aim of the study is to assess the impact at a clinical level of the combination of liposomed polyphenols to improve their effectiveness, with the drug G04CB02 which shows great anti-ALS properties by Molecular Topology methodology. A prospective, longitudinal, mixed, analytical, experimental and double-blind study is proposed, with a population sample of 60 patients distributed randomly in 30 patients in the intervention group who will receive treatment for 2 months, and 30 patients in the control group who will receive a placebo for the same period. The assessment will be at time 0, and at 2 months and 4 months after treatment, with functional, cognitive and behavioural tests, and of the state of inflammation and oxidation; and at time 0 and 2 months, of the intestinal microbiota.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Liposomed polyphenols resveratrol and curcumin"}, {"type": "OTHER", "name": "Placebo for liposomed resveratrol and curcumin"}, {"type": "DIETARY_SUPPLEMENT", "name": "Isocaloric Diet"}, {"type": "DRUG", "name": "G04CB02"}, {"type": "OTHER", "name": "Placebo microcrystalline methylcellulose"}], "start_date": "2021-11-20", "url": "https://clinicaltrials.gov/study/NCT04654689", "target_entities": ["oxidative_stress", "glutamate_excitotoxicity", "neuroinflammation"], "locations": [{"facility": "Jos\u00e9 Enrique de la Rubia Ort\u00ed", "city": "Valencia", "state": "Valencia", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* All ALS patients, over 18 years of age and with a clear diagnosis and symptomatology of ALS since at least 6 months.\n\nExclusion Criteria:\n\n* Women under 50 years of age and childbearing age.\n* Tracheotomy patients.\n* Patients with invasive or non-invasive ventilation with positive ventilatory pressure\n* Patients with evidence of dementia.\n* Patients with alcohol or drug abuse dependency.\n* Patients infected with B or C hepatitis, or HIV positive\n* Renal patients with creatinine levels twice as high as normal markers.\n* Liver patients with liver markers (ALT, AST) elevated 3 times above normal levels.\n* Patients included in other research with drugs or therapies in the experimental phase.\n* Patients treated with anticoagulants or with haemostatic problems", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Liposomed polyphenols (resveratrol and curcumin) with G04CB02", "targeting_mechanism": "Combination of antioxidant polyphenols and an undefined agent (G04CB02) targeting oxidative stress and inflammation in ALS pathogenesis.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07423494", "title": "Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)", "phase": "PHASE1, PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "n-Lorem Foundation", "summary": "This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10", "interventions": [{"type": "DRUG", "name": "nL-CHCHD-001"}], "start_date": "2026-03", "url": "https://clinicaltrials.gov/study/NCT07423494", "target_entities": ["CHCHD10"], "locations": [{"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s)\n* Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records\n* Genetically confirmed neurological disorder\n\nExclusion Criteria:\n\n* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures\n* Use of an investigational medication within less than 5 half-lives of the drug at enrollment", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "nL-CHCHD-001", "targeting_mechanism": "Personalized antisense oligonucleotide (ASO) designed to target pathogenic CHCHD10 variant in a single ALS patient.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04516096", "title": "A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Amylyx Pharmaceuticals Inc.", "summary": "The protocol is intended to provide extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for ALS.", "interventions": [{"type": "DRUG", "name": "AMX0035"}], "start_date": "2020-11-22", "url": "https://clinicaltrials.gov/study/NCT04516096", "target_entities": ["TARDBP"], "locations": [{"facility": "Forbes Norris MDA/ALS Research Center - California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Memorial Medical Center", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "University of Michigan Medical Center", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Wake Forest Baptist Medical Center", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Any patient who has completed follow-up in an Amylyx sponsored trial of AMX0035 for the treatment of ALS will be eligible to enroll into this Protocol\n* Capable of providing informed consent\n* Capable and willing to follow trial procedures\n* Capable and willing of travelling to the site at regular intervals for interim site visits and to return and collect study drug or able to attend telemedicine remote site visits if such are currently in use at the site\n* Participants who have established care with a neurologist at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the protocol.\n* Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug.\n* Women must not be nursing, be pregnant or planning to become pregnant for the duration of the study and 3 months after last dose of study drug\n* Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide sperm for donation for the duration of the study and 3 months after last dose of study drug\n\nExclusion Criteria:\n\n* Ongoing severe adverse events that in the opinion of the Site Investigator are contraindication to the study drug, including severe renal or liver insufficiency or Class III/IV heart failure (per New York Heart Association)\n* Ongoing serious adverse event that was assessed as related to study drug per the Site Investigator\n* Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the patient to provide informed consent, according to Site Investigator judgment;\n* Current severe biliary disease which may result in the investigator medical judgement in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder;\n* Clinically significant unstable medical condition (other than ALS) that would pose a risk to the patient if they were to participate in the study, according to Site Investigator judgment;\n* Treatment, current or within 90 days from screening with any cell therapies or gene therapies;\n* Implantation of Diaphragm Pacing System (DPS);\n* Current or planned exposure to any prohibited medications listed in Section", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AMX0035", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06177431", "title": "An Open Label Extension Study of Monepantel in Individuals With Motor Neurone Disease", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neurizon Therapeutics Limited", "summary": "This study is a multicenter, 12-month open label extension study, following Phase 1 Study MON-2021-001, with a single dose of monepantel (MPL) once daily (QD) for the treatment of individuals with MND.", "interventions": [{"type": "DRUG", "name": "Monepantel"}], "start_date": "2024-02-13", "url": "https://clinicaltrials.gov/study/NCT06177431", "target_entities": ["pyruvate_dehydrogenase_kinase_inhibition"], "locations": [{"facility": "Macquarie University", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}, {"facility": "Calvary Health Care Bethlehem", "city": "Melbourne", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.814, "lon": 144.96332}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Signed informed consent obtained prior to initiation of any study specific procedures and treatment.\n2. Individuals who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.\n3. Able to swallow study drug tablets.\n4. Individuals must have completed Study MON-2021-001 and, in the opinion of the investigator, have been compliant with the study procedures and study treatment.\n5. Diagnosis of ALS/MND defined as clinically possible, probable, or definite according to Awaji-shima Consensus Recommendations.\n6. Not taking riluzole or on a stable dose of riluzole for at least 4 weeks prior to the screening visit; subjects are not allowed to start taking riluzole during the study.\n7. Individual has a competent caregiver/support person who can and will be able to support the individual's participation in the study, including assisting with the administration of study drug.\n8. Adequate bone marrow reserve, renal and liver function:\n\n   * absolute neutrophil count \u2265 1500/\u00b5l.\n   * platelet count \u2265 120,000/\u00b5l.\n   * hemoglobin \u2265 11 g/dL.\n   * creatinine clearance \u2265 60 mL/min (Cockroft \\& Gault formula).\n   * alanine aminotransferase and/or aspartate aminotransferase \u2264 3 x upper limit of normal.\n   * total bilirubin \u2264 2.0 x ULN.\n   * serum albumin \u2265 2.8 g/dL.\n9. Women and men with partners of childbearing potential must use effective contraception while on study treatment and women of childbearing potential must be non-lactating.\n\nExclusion Criteria:\n\n1. Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g., malabsorption) deemed to jeopardize intestinal absorption of study drug.\n2. Participated in another investigational drug research study within 4 weeks (28 days) of the Baseline Visit or five half-lives of the drug, whichever is longer.\n3. Any other significant illness or condition that in the opinion of the study investigator would interfere with the study conduct.\n4. Dementia that may affect either outcome measures or subject understanding and/or compliance with study requirements and procedures.\n5. Women and men of childbearing potential not using effective contraception while on study treatment.\n6. Women who are breast feeding.\n7. Individuals at risk of or are known to carry a SOD1 mutation or VCP mutation.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Monepantel", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00420719", "title": "Motor-Point Stimulation for Conditioning the Diaphragm of Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Synapse Biomedical", "summary": "The overall goal of this research is to delay the respiratory decline of patients with Amyotrophic Lateral Sclerosis (ALS) thereby increasing their lifespan by conditioning the diaphragm with laparoscopically placed electrodes.\n\nThis device currently holds an Investigational Device Exemption No. G040142 in the United States and is currently undergoing clinical trials at University Hospitals (Cleveland), Johns Hopkins, Mayo Clinic Jacksonville, California Pacific Medical Center (CPMC), Henry Ford Health System, The Methodist Hospital, and Stanford University.", "interventions": [{"type": "DEVICE", "name": "Intramuscular diaphragm electrodes"}], "start_date": "2004-10", "url": "https://clinicaltrials.gov/study/NCT00420719", "target_entities": [], "locations": [{"facility": "Forbes Norris - California Pacific Medical Center (CPMC)", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Stanford University Medical Center", "city": "Stanford", "state": "California", "country": "United States", "status": "", "lat": 37.42411, "lon": -122.16608}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Henry Ford Health System", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "University Hospitals Of Cleveland", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "The Methodist Hospital", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Groupe Hospitalier Pitie-Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age 18 or older\n* Participants with familial or sporadic ALS diagnosed as laboratory-supported probable, probable, or definite according to the World Federation of Neurology El Escorial criteria will be eligible\n* Bilateral phrenic nerve function clinically acceptable as demonstrated by bilateral diaphragm movement with fluoroscopic sniff test or with EMG recordings and nerve conduction times\n* Forced Vital Capacity (FVC) between 50 - 85% of predicted values to begin screening procedures.\n* FVC greater than 45% of predicted value at time of surgery.\n* No underlying cardiac or pulmonary diseases that would increase the risk of general anesthesia greater than the expected risk of the patient with ALS\n* Negative pregnancy test in females of child-bearing potential\n* Informed consent from patient or designated representative\n\nExclusion Criteria:\n\n* Preexisting implanted electrical device such as pacemaker or cardiac defibrillator.\n* Underlying pulmonary diseases that were present prior to ALS that would effect pulmonary tests independent of ALS\n* Active cardiovascular disease that would increase the risk of general anesthesia\n* Current pregnancy or breastfeeding\n* Hospitalization for a treated active infection within the last 2 months\n* Significant decision making incapacity preventing informed consent by the subject due to a major mental disorder such as major depression or schizophrenia, or dementia such as having Alzheimer's disease.\n* Marked obesity", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05095571", "title": "A Trial of Nicotinamide/Pterostilbene Supplement in ALS: The NO-ALS Extension Study", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Haukeland University Hospital", "summary": "Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The mean survival from the time of diagnosis is 2.5 years. Apart from Riluzole, there is no effective treatment. Care of advanced ALS will have a cost of 4-8 million NOK (Norwegian kroner) per year.\n\nResearch i.a. from the investigators department has shown that increased activity in histone deacetylation enzymes (sirtuins) together with increased access to Nicotinamide Adenine Dinucleotide (NAD) can delay disease progression. Nicotinamide riboside (NR) can increase cells' access to NAD and Pterostilbene will stimulate sirtuins.\n\nThe investigators want to study whether combination therapy with NR and Pterostilbene can inhibit neurodegeneration in ALS and thereby delay disease development, increase survival and improve quality of life in ALS.\n\nIn the NO-ALS extension study the investigators will follow the patients who completed the original NO-ALS study. Objectives are to evaluate adverse events and give patients possibility of compassionate use, and secondarily to see if the combination of NR and pterostilbene (EH301) will decrease progression of motor symptoms and loss of vital capacity, and increase survival time in patients with ALS.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "EH301 (Nicotinamide Riboside/Pterostilbene)"}], "start_date": "2021-10-07", "url": "https://clinicaltrials.gov/study/NCT05095571", "target_entities": ["nad_metabolism", "histone_deacetylase_inhibition"], "locations": [{"facility": "Haukeland University Hospital", "city": "Bergen", "state": "", "country": "Norway", "status": "RECRUITING", "lat": 60.39299, "lon": 5.32415}, {"facility": "Nordlandssykehuset HF", "city": "Bod\u00f8", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 67.28267, "lon": 14.37513}, {"facility": "Vestre Viken HF", "city": "Drammen", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 59.74389, "lon": 10.20449}, {"facility": "Helse F\u00f8rde HF", "city": "F\u00f8rde", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 61.45217, "lon": 5.85717}, {"facility": "Helse Fonna HF", "city": "Haugesund", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 59.41378, "lon": 5.268}, {"facility": "S\u00f8rlandet sykehus", "city": "Kristiansand", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 58.14671, "lon": 7.9956}, {"facility": "Sykehuset Innlandet HF", "city": "Lillehammer", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 61.11514, "lon": 10.46628}, {"facility": "Akershus University Hospital", "city": "L\u00f8renskog", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": null, "lon": null}, {"facility": "Helse M\u00f8re og Romsdal", "city": "Molde", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 62.73752, "lon": 7.15912}, {"facility": "Helse Nord-Tr\u00f8ndelag HF", "city": "Namsos", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 64.46624, "lon": 11.49572}, {"facility": "Oslo Univerity Hospital", "city": "Oslo", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 59.91273, "lon": 10.74609}, {"facility": "Sykehuset \u00d8stfold HF", "city": "Sarpsborg", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 59.28391, "lon": 11.10962}, {"facility": "Sykehuset i Telemark HF", "city": "Skien", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 59.20962, "lon": 9.60897}, {"facility": "Stavanger University Hospital", "city": "Stavanger", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 58.97005, "lon": 5.73332}, {"facility": "Universitetssykehuset Nord-Norge", "city": "Troms\u00f8", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 69.6489, "lon": 18.95508}, {"facility": "St. Olavs Hospital HF", "city": "Trondheim", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 63.43049, "lon": 10.39506}, {"facility": "Sykehuset i Vestfold HF", "city": "T\u00f8nsberg", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 59.26754, "lon": 10.40762}], "contact_phone": "+4755975063", "contact_email": "obty@haukeland.no", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients who fulfilled the criteria for the NO-ALS study and have completed the study will be proposed inclusion in the NO-ALS extension study protocol. Patients from both arm 1 and arm 2 in the NO-ALS study will be allowed inclusion in the prolongation study\n\nExclusion Criteria:\n\nIndividuals will be excluded if any of the following exclusion criteria apply:\n\n* Dementia, fronto temporal dementia (FTD) or other neurodegenerative disorder interfering with compliance.\n* Metabolic, neoplastic, or other physically or mentally debilitating disorder.\n* Patients who become tracheostomized as part of the treatment of ALS.\n* Patients with short expected survival at the discretion of the investigator. Such cases cannot be expected to follow protocol procedures.\n* Use of Vit B3 or blue berry extracts outside the study", "sex": "ALL", "min_age": "35 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "EH301 (Nicotinamide Riboside/Pterostilbene)", "targeting_mechanism": "Increases NAD+ levels and histone deacetylation enzyme (sirtuin) activity to enhance mitochondrial proteostasis and neurogenesis in motor neurons.", "targeting_mechanism_pmid": "31929756", "animal_results": "Nicotinamide Riboside enhances mitochondrial proteostasis and adult neurogenesis through activation of mitochondrial unfolded protein response signaling in the brain of ALS SOD1(G93A) mice.", "animal_results_pmid": "31929756", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00415519", "title": "Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS) Who Met Severity Classification III", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "The primary objective of this study is to evaluate the efficacy of 60mg of MCI-186 via intravenous drip once a day in patients with ALS whose severity is classified as grade III, based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24 weeks administration in double-blind, placebo-controlled manner. And in addition, this study will be performed to examine the safety of MCI-186 to ALS patients who met severity classification III.", "interventions": [{"type": "DRUG", "name": "MCI-186"}, {"type": "DRUG", "name": "Placebo of MCI-186"}], "start_date": "2006-12-31", "url": "https://clinicaltrials.gov/study/NCT00415519", "target_entities": ["oxidative_stress"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients who are defined as \"definite ALS,\" \"probable ALS\" or \"probable-laboratory-supported ALS,\" met diagnostic criteria revised EL Escorial for Airlie House.\n* Patients who cannot take at least one action of eating a meal, excreting, or moving with oneself alone, and need assistance in everyday life.\n* Patients whose progress of the condition during 12 weeks before administration meet other requirements.\n\nExclusion Criteria:\n\n* Patients judged to be inadequate to participate in this study by their physician, because those patients' general condition deteriorated to the point that they need to be hospitalized for severe hepatic disease, sever heart disease, sever renal disease and so on, or they need to be administered antibiotics to infection.\n* Patients who complain the difficulty in breathing caused by deteriorating the respiratory function.\n* Patients with such complications as Parkinson's disease, schizophrenia, dementia, renal failure, or other severe complication, and patients who have the anamnesis of hypersensitivity to edaravone.\n* Pregnant, lactating, and probably pregnant patients, and patients who want to become pregnant, and patients who can not agree to contraception.\n* Patients who have been administered other investigational products within 12 weeks before consent, or who are participating in other clinical trials at present.\n* In addition to the above exclusion criteria, patients judged to be inadequate to participate in this study by their physician.", "sex": "ALL", "min_age": "20 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MCI-186", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00353665", "title": "Memantine for Disability in Amyotrophic Lateral Sclerosis (MEDALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Lisbon", "summary": "The purpose of this trial is to study the effect of Memantine (uncompetitive, moderate affinity, NMDA receptor antagonist that binds to the NMDA receptor channel, and regulates the calcium influx into the neurons), a drug used to treat Alzheimer\u00b4s disease, on the progression of Amyotrophic Lateral Sclerosis (ALS). Memantine is added to riluzole (the single drug approved to treat ALS).", "interventions": [{"type": "DRUG", "name": "Memantine (Ebixa)"}, {"type": "DRUG", "name": "riluzole"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2005-07", "url": "https://clinicaltrials.gov/study/NCT00353665", "target_entities": ["GRIN1"], "locations": [{"facility": "Department of Neurology - Hospital de Santa Maria", "city": "Lisbon", "state": "", "country": "Portugal", "status": "", "lat": 38.72509, "lon": -9.1498}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Definite or probable disease - revise El Escorial criteria\n* Normal blood tests\n* Riluzole treatment during 1 month or more\n* EMG in accordance with El Escorial criteria\n\nExclusion Criteria:\n\n* Other diseases (such as PNP)\n* Both ADM muscles \\< 3 on MRC scale\n* Conduction block on nerve conduction tests\n* Disease duration \\> 3 years\n* ALS-FRS \\< 25\n* Forced vital capacity - \\<60%", "sex": "ALL", "min_age": "20 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Memantine", "targeting_mechanism": "Uncompetitive, moderate affinity NMDA receptor antagonist that binds to the NMDA receptor channel and regulates calcium influx into neurons.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00355576", "title": "Combination Therapy Selection Trial in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Columbia University", "summary": "The objective of this study is to compare two combinations of drugs, minocycline and creatine or celecoxib and creatine, in a phase II trial designed to determine which combination is more effective for ALS.", "interventions": [{"type": "DRUG", "name": "Celecoxib"}, {"type": "DRUG", "name": "Creatine"}, {"type": "DRUG", "name": "Minocycline"}], "start_date": "2006-07", "url": "https://clinicaltrials.gov/study/NCT00355576", "target_entities": ["neuroinflammation", "oxidative_stress"], "locations": [{"facility": "Phoenix Neurological Associates", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "UCLA", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Medical College of Georgia", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "University of Illinois", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Mayo Clinic Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "UMDNJ", "city": "New Brunswick", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.48622, "lon": -74.45182}, {"facility": "University of New Mexico", "city": "Albuquerque", "state": "New Mexico", "country": "United States", "status": "", "lat": 35.08449, "lon": -106.65114}, {"facility": "Beth Israel", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Oregon Health and Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "UT Southwestern Medical Center", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Vermont", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* A clinical diagnosis of possible, laboratory-supported probable, probable or definite ALS, according to modified EL Escorial criteria\n* FVC greater or equal to 60% at the screening visit\n* Symptom onset within 5 years\n* 21 to 85 years of age\n* If patients are taking riluzole, they must be on a stable dose for at least the past thirty days\n* A woman of childbearing age, must be nonlactating and surgically sterile or using an effective method of birth control (barrier method) and have a negative pregnancy test\n* Able to maintain adequate hydration levels defined as 6-8 cups (8ounces/cup) of water or a non-caffeinated beverage per day\n* Willing and able to give signed informed consent that has been approved by an Institutional Review Board (IRB)\n\nExclusion Criteria:\n\n* Tracheotomy and mechanical ventilation\n* Diagnosis of other neurodegenerative diseases (Parkinson's disease, Alzheimer's disease, etc)\n* Unstable medical illness (coronary artery disease, advanced cancer, active esophageal or gastroduodenal ulcers, etc) in the last one year\n* Systemic Lupus Erythematosis\n* FVC \\< 60%\n* Pregnancy or lactation\n* Allergy to minocycline, tetracyclines, celecoxib, sulfonamides, NSAIDS, or creatine\n* History of congestive heart failure\n* Renal disease \\[baseline Cr \\> 1.5 (men) or 1.2 (women)\\]\n* History of significant hepatic disease (baseline AST/ALT or bilirubin \\> 1.5x normal)\n* Use of an investigational agent within thirty days of enrollment\n* First degree relative with ALS or gene identified familial ALS\n* Inability or unwillingness to maintain adequate daily hydration (defined above)\n* Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.\n* History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols.", "sex": "ALL", "min_age": "21 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Celecoxib", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00397423", "title": "G-CSF Treatment for Amyotrophic Lateral Sclerosis: A RCT Study Assessing Clinical Response", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University", "summary": "This study will examine the effectiveness of G-CSF in treating patients with amyotrophic lateral sclerosis (ALS) - a fatal neurological degenerative disease that causes adult-onset, progressive motor neurons loss in the spinal cord, brain stem and motor cortex. Patients develop progressive wasting and weakness of both upper and lower limbs, bulbar and respiratory muscles. Usually death from respiratory failure typically is within 3-5 years of diagnosis. Although there are various treatments for ALS, riluzole is the only approved treatment to delay the disease progression. G-CSF is an approved drug that is used to increase white blood cell counts. Moreover, G-CSF and its receptor are expressed by neurons. It acts anti-apoptosis by activating several protective pathways, stimulates neuronal differentiation of adult neural stem cells in the brain, and improves long-term recovery. G-CSF is a novel neurotrophic factor, and a highly attractive candidate for the treatment of neurodegenerative conditions such as ALS.\n\nPatients 18 to 65 years of age who have had mild to moderately severe ALS for 0.5 to 2 years of duration may be eligible for this study. Candidates will be screened with a medical history and possible review of medical records, physical examination, blood test, urine and stool analyses, electrocardiogram, electrophysiological examination, neurological imaging and, for women, a pregnancy test.\n\nParticipants will have drug therapy according to randomized number. One group receives G-CSF while other group receives placebo. All of the participants receive riluzole treatment. For the procedure, patients are given a medication to lessen anxiety and any discomfort. Patients receive drug injections every 3 months for 5 days. The G-CSF dosage is 5\u03bcg/kg/day. Physical examination and interview, Appel ALS scale and ALS-Functional Rating Scale will be done in 14, 28 days and 3, 6, 9, 12 months. Electrophysiological examination will be tested per 3 months. Blood samples will be collected on treat 5, 14, 28 days and 3, 6, 9, 12 months.", "interventions": [{"type": "DRUG", "name": "Granulocyte Colony Stimulating Factor"}, {"type": "DRUG", "name": "NS"}], "start_date": "2006-12", "url": "https://clinicaltrials.gov/study/NCT00397423", "target_entities": ["neuroprotection"], "locations": [{"facility": "Dongsheng Fan", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* All subjects must have a verifiable diagnosis of ALS of 0.5 to 2 years' duration. The diagnosis must be supported by the Revised Criteria of the World Federation of Neurology. The grades of diagnosis must be clinically definite ALS or clinically probable ALS.\n* All subjects must be over age 18 and below 65.\n* The ALS is mildly to moderate based on ALS Health State Scale.\n* Electrophysiological features show CMAP amplitude of motor nerve normal or mild declining.\n* Serum creatine kinase is normal or mild upper, less than 500U/L.\n\nExclusion Criteria:\n\n* If anyone of the above eligibility requirements is not met\n* Use of any other investigational agent within 30 days beginning the treatment phase of this study\n* Severe cardiac, pulmonary, hepatic or/and hematic disease\n* HIV positivity or signs and symptoms consistent with HIV infection\n* Pregnant or nursing women\n* History of cancer with less than 5 years documentation of a disease-free state\n* History of anaphylactic reaction or hypersensitivity to G-CSF or proteins derived from E.coli\n* Alcohol or drug abuse in recent 1 year\n* Can't understand or obey the rules of treatment\n* Blood donor in recent 30 days", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Granulocyte Colony Stimulating Factor (G-CSF)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03427086", "title": "Safety and Tolerability of High Dose Biotin in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "American University of Beirut Medical Center", "summary": "This is a randomized double blinded randomized 2:1 study. The duration of the study is 6 month. The safety and tolerability of high doses of biotin (300 mg/ day) will be compared to placebo in patients with amyotrophic lateral sclerosis. Patients will be evaluated at baseline, 3, and 6 month. The primary outcome will be any adverse effects recorded. The secondary outcomes will be motor disability measured by ALS-FRS, change in Pulmonary function test parameters (FEV1- FVC), change in subject weight (in kg).", "interventions": [{"type": "DRUG", "name": "Biotin"}, {"type": "DRUG", "name": "Placebo Oral Tablet"}], "start_date": "2018-01-29", "url": "https://clinicaltrials.gov/study/NCT03427086", "target_entities": ["biotin"], "locations": [{"facility": "American univeristy of Beirut medical center", "city": "Beirut", "state": "", "country": "Lebanon", "status": "", "lat": 33.89332, "lon": 35.50157}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Amyotrophic Lateral Sclerosis (ALS) volunteers must be diagnosed within 3 years prior to participation as having possible, probable, or definite ALS, either sporadic or familial according to modified El Escorial criteria\n* Age 18-80, able to provide informed consent, and comply with study procedures\n* Participants must not have started Riluzole and/or Nuedexta for at least 30 days, or be on a stable dose of Riluzole and/or Nuedexta for at least 30 days, prior to screening (Riluzole and/or Nuedexta -na\u00efve participants are permitted in the study)\n\nExclusion Criteria:\n\n* The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent, according to PI judgment.\n* Exposure to any experimental agent within 30 days of entry or at any time during the trial or enrollment in another research study within 30 days of or during this trial.\n* Slow Vital Capacity test less than 50% of the predicted value Patients who had already undergone tracheostomy", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Biotin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01938495", "title": "Diaphragm Pacing System (DPS) In Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Barrow Neurological Institute", "summary": "The study is being conducted to determine if DPS treatment for people with ALS and hypoventilation is associated with improved survival or diaphragm function.\n\nThe primary objective of the study is to conduct a multi-center, randomized controlled clinical trial comparing standard of care (control) to diaphragm stimulator treatment with the NeuRx\u00ae Diaphragm Pacing System\u2122 (DPS) with respect to survival.\n\nThe secondary objective of the study is to conduct a multi-center, randomized controlled clinical trial to compare standard of care treatment (control) to DPS in ALS subjects with hypoventilation.", "interventions": [{"type": "DEVICE", "name": "NeuRx\u00ae Diaphragm Pacing System\u2122 (DPS)"}], "start_date": "2013-08", "url": "https://clinicaltrials.gov/study/NCT01938495", "target_entities": ["neuromuscular_transmission"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Medical Center", "city": "San Diego", "state": "California", "country": "United States", "status": "", "lat": 32.71571, "lon": -117.16472}, {"facility": "Stanford University", "city": "Stanford", "state": "California", "country": "United States", "status": "", "lat": 37.42411, "lon": -122.16608}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Florida, Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Iowa", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Henry Ford Health Systems", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "St Louis University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates P.C.", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Health Care", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Wake Forest University", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Drexel University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburgh", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "University of Texas Southwestern", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Texas", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Vermont", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "Virginia Mason Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 21 years or older.\n2. Sporadic or familial ALS diagnosed as definite, probable or possible ALS as defined by revised El Escorial criteria.\n3. Evidence of hypoventilation at Screening with at least one of the following:\n\n   1. Maximal static inspiratory pressure (MIP) \\<60 cm H20.\n   2. Upright or supine forced vital capacity (FVC) \\<50% predicted for gender, age, and height.\n4. A phrenic nerve potential should be recordable bilaterally.\n5. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to randomization (riluzole-na\u00efve subjects are permitted in the study).\n6. Capable of providing informed consent and following trial procedures.\n7. Geographically accessible to the site.\n8. Negative urine pregnancy test at Screening in women of child bearing potential (WOCBP). (Women who are post-menopausal or who have had a hysterectomy are deemed not of child bearing potential).\n9. Women of child bearing potential must use an adequate form of contraception: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for \u2265 3 months, barrier method in conjunction with spermicide, or another adequate method.\n\nExclusion Criteria:\n\n1. Upright forced vital capacity (FVC) \u2264 45% of predicted for gender, age, and height.\n2. Any prior use of non-invasive ventilation (NIV) outside of sleep (nighttime or naps).\n3. Any pulmonary or cardiac disorder or other medical disorder that would be a contraindication for general anesthesia or DPS hardware implantation in the chest.\n4. Implanted electrical device such as a pacemaker or cardiac defibrillator.\n5. Known diaphragm abnormality such as hiatal hernia or para-esophageal hernia of abdominal contents into the thoracic cavity.\n6. Participation in another treatment research study for people with ALS.\n7. Exposure to any other agent currently under investigation for the treatment of people with ALS (off-label use or investigational) within 30 days of the Screening Visit.\n8. Clinically significant history of unstable or severe cardiac, oncologic, hepatic, psychiatric, renal disease, or other medically significant illness.\n9. Pregnant women or women currently breastfeeding.", "sex": "ALL", "min_age": "21 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NeuRx Diaphragm Pacing System (DPS)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00876772", "title": "Olanzapine for the Treatment of Appetite Loss in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Charite University, Berlin, Germany", "summary": "Amyotrophic Lateral Sclerosis (ALS) is an adult neurodegenerative disease that is caused by a selective degeneration of the motor nerve cells in the cortex and myelon. As a result of motor neurodegeneration, a progredient paralysis of the extremities and of the speaking, swallowing, and breathing musculature develops. ALS leads to death by respiratory insufficiency in a mean course of 3-5 years. More than 80% of ALS patients present with a clinically significant and undesirable weight loss. The cause of weight loss is heterogeneous. Fundamentally, the investigators must distinguish malnutrition, cachexia and loss of appetite. Loss of weight is an independent prognosis factor in ALS. Effective treatment of undesirable weight loss is an important therapy goal for ALS.\n\nThe researchers propose an investigational therapy of ALS with oral administration of Olanzapine. The rationale for this study is based on the weight-increasing effect of OLN. The clinical trial aims to employ OLN-induced weight gain or weight stabilization as a symptomatic therapy for the loss of appetite. An undesired weight loss of at least 10% of the body weight should be reduced through the weight-increasing effect of OLN. The hypothesis states that the undesired weight loss in ALS patients during treatment with OLN 10mg in combination with Riluzole (RIL) 100mg is at least 20 percentage points less than for treatment with placebo in combination with 100 mg RIL.", "interventions": [{"type": "DRUG", "name": "Olanzapine"}], "start_date": "2011-03", "url": "https://clinicaltrials.gov/study/NCT00876772", "target_entities": ["olanzapine"], "locations": [{"facility": "Charit\u00e9 - Universit\u00e4tsmedizin, Berlin, Germany", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients between the ages of 18 and 80 years old\n* Clinical diagnosis of definitive, probable, and possible ALS (revised El Escorial Criteria) or diagnosis of the clinical ALS-variants of Progressive Muscle Atrophy (PMA)\n* Sporadic and familial ALS\n* Beginning of symptoms of paralysis at least 6 months prior\n* Treatment with a steady dose of RIL 100 mg/day for at least 1 month\n* A score of \u2264 28 in the symptom-oriented Council on Nutrition appetite questionnaire (CNAQ) by which appetite is evaluated\n* Patient consent\n\nExclusion Criteria:\n\n* Patients with known hypersensitivity to OLN, RIL, or one of the active ingredients\n* Percutaneous Endoscopic Gastronomy (PEG)\n* Clinically significant eating disorder\n* Deliberate weight loss\n* Underlying consumptive disease with undesired weight loss\n* Overweight with BMI \u2265 25 kg/m2\n* Clinically significant hypotonia and history of recurrent syncopes (\\> 1 syncope)\n* Clinically severe concomitant illnesses, including psychiatric illnesses\n* Pregnant or nursing women\n* Severe neutropenia (\\< 750/mm3)\n* Open angle glaucoma\n* Diabetes mellitus\n* Prostatic hyperplasia\n* Extrapyramidal movement disorders including from late dyskinesia\n* Dementia and incompetence to grant informed consent\n* Clinically significant EKG changes\n* EKG proof of a QT time corrected according to Fridericia (QTcF) \\> 500 ms\n* Treatment with substances that are metabolized by the Cytochrom-P450-System CYP1A2 (e. g. Carbamazepine, Fluvoxamin, and Ciprofloxacin)\n* Treatment with Mirtazapine within the past 3 months\n* Treatment with steroids or appetite-stimulating substances including anabolics within the past 3 months\n* Treatment with Valproat within the past 3 months\n* Treatment with hepatotoxic medicines\n* Treatment with tetrahydrocannabinol within the past 3 months\n* Treatment with another atypical or typical neuroleptic within the past 3 months\n* Treatment with any other study medication \\< 1 month before the beginning of the study\n* Destructive use of psychotropic substances within the past 3 months\n* Destructive use of alcohol\n* Laboratory parameters outside the normal range that are associated with a clinically significant cardiovascular, pulmonologic, hematologic, hepatological, metabolic, or renal disease or that interfere with interpretation of the clinical study or that require medications that are not permitted in the study protocol\n* Elevation of the serum transaminase levels (ALT/AST) to more than 3-times of the upper normal value\n* Elevation of the bilirubin and gamma glutamyl transferase levels (GGT) to beyond the maximum normal value\n* History of a cardiopulmonary reanimation und prevention of sudden cardiac death\n* History of clinically significant EKG changes\n* History of thrombotic events including deep leg vein thrombosis and pulmonary artery embolism\n* History of a paralytic ileus\n* History of epilepsy or an episodic seizure", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Olanzapine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00004771", "title": "Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Center for Research Resources (NCRR)", "summary": "OBJECTIVES:\n\nI. Evaluate the effects of androgen suppression with leuprolide and androgen replacement with testosterone enanthate on muscle strength in men with Kennedy's disease or other motor neuron disease.", "interventions": [{"type": "DRUG", "name": "leuprolide"}, {"type": "DRUG", "name": "testosterone"}], "start_date": "1992-10", "url": "https://clinicaltrials.gov/study/NCT00004771", "target_entities": ["Androgen receptor"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "PROTOCOL ENTRY CRITERIA:\n\n--Disease Characteristics--\n\nMen aged 18 and over with motor neuron disease, i.e.:\n\n* X-linked spinal and bulbar muscular atrophy (Kennedy's disease)\n* Confirmed by androgen receptor, exon-1 mutation genotype\n* Amyotrophic lateral sclerosis\n* Spinal muscular atrophy\n\nSignificant muscle weakness on manual muscle testing\n\nNo prisoners\n\nNo mental disability", "sex": "MALE", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "leuprolide and testosterone", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05860244", "title": "Effect of Salbutamol on Walking Capacity in Ambulatory ALS Patients", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "Preclinical and clinical data strongly suggest that administration of salbutamol in ALS patients may improve walking capacity related to motor fatigue by enhancing neuromuscular transmission. Salbutamol may exert a neuroprotective effect and slow down the progression of clinical signs and symptoms. The main objective of the study is to test the efficacy of salbutamol on walking capacity in ALS patients and the secondary objective is to measure the target engagement of salbutamol on the neuromuscular junction (NMJ) at EMG (decrement of repetitive nerve stimulation in three nerves/muscle couples), as well as safety and tolerability. The exploratory objectives are to study the effect of salbutamol on fatigue scales, muscle strength, respiratory function, motor unit count, muscle and spinal MRI parameters and blood biomarkers", "interventions": [{"type": "DRUG", "name": "Salbutamol"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2024-09", "url": "https://clinicaltrials.gov/study/NCT05860244", "target_entities": ["neuromuscular_transmission"], "locations": [{"facility": "H\u00f4pital Piti\u00e9 Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "01 42 16 58 70", "contact_email": "g.querin@institut-myologie.org", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subjects who meet the revised El Escorial criteria for probable or definite sporadic ALS\n* Adult patients between 18 and 75 years of age\n* Patients who are ambulatory and able to perform the 6MWT and quantitative muscle testing at screening (ALSFRS-R-walking = 3)\n* Patients able and willing to travel to the site, and, in the investigator's opinion, who are likely to attend visits for at least 6 months\n* Patients who signed written informed consent\n* Stable dose of riluzole for a minimum of 4 weeks prior to baseline or has not taken it for 4 weeks prior to baseline\n* For child-bearing aged women, efficient contraception (cf protocol p32)\n* Forced vital capacity (fVC) in a sitting position \\> 70 %\n\nExclusion Criteria:\n\n* Patients with significant spasticity of the lower limbs interfering with walking capacity (Ashworth scale score \\> 2)\n* Patients with fronto-temporal dementia associated with ALS\n* Patients presenting respiratory insufficiency causing dyspnea during walking\n* Patients taking drugs that could interfere with NMJ function (anticholinesterase \u2026) or muscle function (steroids, statins\u2026)\n* Patients taking any forbidden drugs (see list in annex)\n* Hypersensitivity to salbutamol or to excipients of the drug and placebo\n* Known contraindication for the studied drug such as ischemic cardiomyopathy or risk of ischemic cardiomyopathy: history of ischemic heart disease or coronaropathy or/and significant ischemic ECG alterations at screening visit\n* Any clinically significant alterations in the following biological parameters glycemia, kalemia, creatinemia and hematology in the month prior to inclusion according to local laboratory threshold (cf protocol page 33)\n* Vulnerable persons defined in Articles L1121-5 to L 1121-8-1 and L1122-1-2 of the Code de la Sant\u00e9 Publique\\* (\\*CSP)\n* Participation in another interventional trial up to 3 months before inclusion\n* Patients having any relevant concomitant disease considered at risk of interfering with study procedures in the opinion of the investigator", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Salbutamol", "targeting_mechanism": "\u03b22-adrenergic agonist that enhances neuromuscular transmission and may exert neuroprotective effects.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04248465", "title": "An Efficacy and Safety Study of Ravulizumab in ALS Participants", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Alexion Pharmaceuticals, Inc.", "summary": "The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.", "interventions": [{"type": "DRUG", "name": "Placebo"}, {"type": "BIOLOGICAL", "name": "Ravulizumab"}], "start_date": "2020-03-30", "url": "https://clinicaltrials.gov/study/NCT04248465", "target_entities": ["complement_pathway"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Neuromuscular Research Center and Clinic", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "HonorHealth Research Institute", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "Loma Linda University Medical Center", "city": "Loma Linda", "state": "California", "country": "United States", "status": "", "lat": 34.04835, "lon": -117.26115}, {"facility": "University of Southern California", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California-Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Stanford University Medical Center", "city": "Palo Alto", "state": "California", "country": "United States", "status": "", "lat": 37.44188, "lon": -122.14302}, {"facility": "University of California San Diego Medical Center", "city": "San Diego", "state": "California", "country": "United States", "status": "", "lat": 32.71571, "lon": -117.16472}, {"facility": "Norris MDA/ALS Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of California San Francisco Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Anschutz Medical Campus School of Medicine", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "University of Florida at Shands Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "University of Chicago Medical Center", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Indiana University Medical Center", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Kansas Medical Center Research Institute, Inc.", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Kentucky", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Johns Hopkins University School Of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Las Vegas Clinic", "city": "Las Vegas", "state": "Nevada", "country": "United States", "status": "", "lat": 36.17497, "lon": -115.13722}, {"facility": "Beth Israel Medical Center - PRIME", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Atrium Health Neuroscience Institute", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "The Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "The Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Allegheny Neurological Associates", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Vanderbilt University Medical Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Houston Methodist Neurological Institute-Movement Disorders Clinic", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Nerve & Muscle Center of Texas", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Vermont Medical Center", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "Virginia Commonwealth University, Neurology Clinical and Translational Research Office", "city": "Richmond", "state": "Virginia", "country": "United States", "status": "", "lat": 37.55376, "lon": -77.46026}, {"facility": "Sentara Neurology Specialists", "city": "Virginia Beach", "state": "Virginia", "country": "United States", "status": "", "lat": 36.85293, "lon": -75.97799}, {"facility": "Swedish Neuroscience Institute", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Brain and Mind Centre", "city": "Camperdown", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.88965, "lon": 151.17642}, {"facility": "Westmead Hospital", "city": "Westmead", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.80383, "lon": 150.98768}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Perron Institute for Neurological and Translational Science", "city": "Nedlands", "state": "Western Australia", "country": "Australia", "status": "", "lat": -31.98184, "lon": 115.8073}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Heritage Medical Research Centre (HMRC)", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Stan Cassidy Center for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "LHSC - University Hospital", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Toronto Sunnybrook Hospital", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "University Hospital of Quebec-Universite Laval", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Royal University Hospital", "city": "Saskatoon", "state": "Saskatchewan", "country": "Canada", "status": "", "lat": 52.13238, "lon": -106.66892}, {"facility": "\u00c5lborg Universitets Hospital", "city": "Aalborg", "state": "", "country": "Denmark", "status": "", "lat": 57.048, "lon": 9.9187}, {"facility": "Aarhus University Hospital Department of Neurology", "city": "Aarhus", "state": "", "country": "Denmark", "status": "", "lat": 56.15674, "lon": 10.21076}, {"facility": "Bispebjerg Hospital", "city": "Copenhagen", "state": "", "country": "Denmark", "status": "", "lat": 55.67594, "lon": 12.56553}, {"facility": "CHU de Nice H\u00f4pital Pasteur 2", "city": "Nice", "state": "Alpes Maritimes", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "CHU de Limoges - H\u00f4pital Dupuytren", "city": "Limoges", "state": "Haute Vienne", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Hopital Gui de Chauliac", "city": "Montpellier", "state": "Herault", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU Tours - H\u00f4pital Bretonneau", "city": "Tours", "state": "Indre Et Loire", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Hopital Neurologique Pierre Wertheimer", "city": "Bron", "state": "Rhone", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "Hopital Roger Salengro - CHU Lille", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "H\u00f4pital de la Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Groupe Hospitalier Pitie-Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Universitaetsklinikum Ulm", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Klinikum rechts der Isar der TU Muenchen", "city": "Munich", "state": "Bavaria", "country": "Germany", "status": "", "lat": 48.13743, "lon": 11.57549}, {"facility": "Universitaetsmedizin Goettingen", "city": "Goettigen", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": null, "lon": null}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universitaetsklinikum Jena", "city": "Jena", "state": "Thuringia", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Rambam Health Care Center", "city": "Haifa", "state": "", "country": "Israel", "status": "", "lat": 32.81303, "lon": 34.99928}, {"facility": "Hadassah University Hospital - Ein Kerem", "city": "Jerusalem", "state": "", "country": "Israel", "status": "", "lat": 31.76904, "lon": 35.21633}, {"facility": "Tel Aviv Sourasky Medical Center", "city": "Tel Aviv", "state": "", "country": "Israel", "status": "", "lat": 32.08088, "lon": 34.78057}, {"facility": "Ospedale San Raffaele", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "ICS Maugeri IRCCS", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Istituto Auxologico Italiano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Azienda Ospedaliero-Universitaria di Modena - Ospedale Civile di Baggiovara", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone", "city": "Palermo", "state": "", "country": "Italy", "status": "", "lat": 38.1166, "lon": 13.3636}, {"facility": "Azienda Ospedaliero Universitaria Pisana", "city": "Pisa", "state": "", "country": "Italy", "status": "", "lat": 43.70853, "lon": 10.4036}, {"facility": "University of Turin", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Nagoya University Hospital", "city": "Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "Chiba University Hospital", "city": "Chiba", "state": "Chiba", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "Yoshino Neurology Clinic", "city": "Ichikawa-shi", "state": "Chiba", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Tohoku University Hospital", "city": "Sendai", "state": "Miyagi", "country": "Japan", "status": "", "lat": 38.26667, "lon": 140.86667}, {"facility": "Niigata University Medical & Dental Hospital", "city": "Niigata", "state": "Niigata", "country": "Japan", "status": "", "lat": 37.92259, "lon": 139.04125}, {"facility": "Shiga University of Medical Science Hospital", "city": "\u014ctsu", "state": "Shiga", "country": "Japan", "status": "", "lat": 35.0, "lon": 135.86667}, {"facility": "Tokushima University Hospital", "city": "Tokushima", "state": "Tokushima", "country": "Japan", "status": "", "lat": 34.06667, "lon": 134.56667}, {"facility": "Medical Hospital, Tokyo Medical and Dental University", "city": "Bunky\u014d City", "state": "Tokyo-To", "country": "Japan", "status": "", "lat": 35.5331, "lon": 139.4217}, {"facility": "Toho University Omori Medical Center", "city": "\u014cta-ku", "state": "Tokyo-To", "country": "Japan", "status": "", "lat": 35.56126, "lon": 139.71605}, {"facility": "Keio University Hospital", "city": "Shinjuku-Ku", "state": "Tokyo-To", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "University Medical Centre Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "CityClinic", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Hospital Universitari de Bellvitge", "city": "L'Hospitalet de Llobregat", "state": "Barcelona", "country": "Spain", "status": "", "lat": 41.35967, "lon": 2.10028}, {"facility": "Hospital de la Santa Creu i Sant Pau", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitari Vall d'Hebron", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Universitari i Politecnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Karolinska Trial Alliance (KTA)", "city": "Huddinge", "state": "", "country": "Sweden", "status": "", "lat": 59.23705, "lon": 17.98192}, {"facility": "Norrlands universitetssjukhus", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "Kantonsspital St. Gallen", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "The National Hospital for Neurology & Neurosurgery", "city": "London", "state": "Greater London", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Royal Hallamshire Hospital", "city": "Sheffield", "state": "West Midlands", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n1. A diagnosis of sporadic or familial ALS, defined by the El Escorial criteria (possible, laboratory-supported probable, probable, or definite ALS).\n2. ALS onset \u2264 36 months from Screening.\n3. Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.\n4. Upright slow vital capacity \u2265 65% predicted at Screening.\n5. If on riluzole, participant must be on a stable dose for 30 days; if on edaravone, participant must be on a stable dose for 60 days (2 cycles).\n6. Body weight \u2265 40 kilograms at Screening.\n7. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n\nKey Exclusion Criteria:\n\n1. History of Neisseria meningitidis infection.\n2. Human immunodeficiency virus (HIV) infection (evidenced by HIV 1 or HIV 2 antibody titer).\n3. Dependence on invasive or non-invasive mechanical ventilation.\n4. Previously or currently treated with a complement inhibitor.\n5. Exposure to an investigational drug or device within 30 days of Screening or 5 half lives of the study drug, whichever is greater.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Ravulizumab", "targeting_mechanism": "Complement cascade inhibitor targeting the complement system's role in motor neuron degeneration.", "targeting_mechanism_pmid": "27056040", "animal_results": "Complement activation has been found in animal models of ALS, including the SOD1G93A mouse model, with evidence suggesting an early role for the complement system in disease progression.", "animal_results_pmid": "27056040", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03690791", "title": "Efficacy of Cannabinoids in Amyotrophic Lateral Sclerosis or Motor Neurone Disease", "phase": "PHASE3", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Gold Coast Hospital and Health Service", "summary": "This is a randomised, double-blind, placebo controlled study on a cannabis-based medicine extract (MediCabilis CBD Oil), in patients with Amyotrophic Lateral Sclerosis or Motor Neurone Disease. Participants will be randomised in a 1:1 ratio to receive MediCabilis CBD Oil or placebo oil. The treatment duration is 6 months with one-month safety follow up. Participants will be checked every month either face to face or via telephone and will be assessed to collect data for study objectives such as ALSFRS-R, Forced Vital Capacity, pain and spasticity score, and quality of life. Thirty (30) participants will be randomised.", "interventions": [{"type": "DRUG", "name": "MediCabilis CBD Oil"}, {"type": "DRUG", "name": "Placebo Oil"}], "start_date": "2019-01-09", "url": "https://clinicaltrials.gov/study/NCT03690791", "target_entities": ["Cannabinoid receptor"], "locations": [{"facility": "Gold Coast Hospital and Health Service", "city": "Gold Coast", "state": "Queensland", "country": "Australia", "status": "", "lat": -28.00029, "lon": 153.43088}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Affected by ALS/MND, either of definite or probable according to the El Escorial revised criteria\n2. Can provide written informed consent\n3. Able and willing to comply with all study requirement\n4. Male or female, ages 25-80 years old\n5. Onset of first symptom within the last 2 years\n6. Forced Vital Capacity (FVC) of at least 60% on baseline\n\nExclusion Criteria:\n\n1. Participants who are bedridden\n2. Have used or taken cannabis or cannabinoid-based medications within 30 days of study entry\n3. History of any psychiatric disorder other than depression associated with their underlying condition including immediate family history of schizophrenia\n4. Heavy consumption of alcohol or use of illicit drug\n5. Hypersensitivity to cannabinoids or any of the excipients\n6. Any of the following: eGFR \\<30 mL/min/1.73m2, ejection fraction \\<35%, or ASL and ALT \\>5 X ULN\n7. Unwillingness of a female participant of child bearing potential, or their partner, to use effective contraception during the study and 30 days thereafter\n8. Pregnant, lactating mother or female participant planning pregnancy during the course of the study and for 30 days thereafter\n9. Received any investigational drug or medical device within 30 days prior randomisation\n10. Any other significant disease or disorder which, in the opinion of the investigator, may either put the participant at risk because of participation in the study, or may influence the result of the study, or the participant's ability to participate in the study\n11. Inability to cooperate with the study procedures\n12. Unwilling to stop driving vehicle or operating dangerous machinery whilst on study drug.\n13. Close affiliation with the study team, e.g. close relative of the investigator", "sex": "ALL", "min_age": "25 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MediCabilis CBD Oil", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06230562", "title": "DIAGALS: Relation Between Tar DNA Binding Protein(TDP)-43 et Nrf-2 in ALS: a Track to Improve Diagnosis and Prognosis of the Disease", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Tours", "summary": "In response to oxidative stress, cells activate the Nrf-2 pathway, which induces translation of its target genes and corresponding proteins involved in the antioxidant response. This explains the interest in the Nrf-2 pathway in the pathophysiology of Amyotrophic lateral sclerosis (ALS), supported by the results of several studies and the modulatory effect of TDP-43 on the Nrf-2 pathway. Since both TDP-43 and Nrf-2 proteins are present in the peripheral blood mononuclear cells (PBMC) of ALS patients and may be correlated with disease progression, the investigators wish to explore their relationship and their application in the clinic as potential blood biomarkers for ALS.", "interventions": [{"type": "BIOLOGICAL", "name": "Blood sample"}, {"type": "BIOLOGICAL", "name": "Blood sample"}], "start_date": "2024-02", "url": "https://clinicaltrials.gov/study/NCT06230562", "target_entities": ["TARDBP", "NFE2L2", "oxidative_stress"], "locations": [], "contact_phone": "+33247474665", "contact_email": "e.mousset@chu-tours.fr", "eligibility": {"criteria": "Patients group :\n\nInclusion Criteria:\n\n* Men and women \u2265 18 years old\n* Person affiliated to a French social security scheme or equivalent\n* ALS diagnosed according to El Escorial criteria\n* Diagnosis of ALS \\< 6 months\n* Onset of symptoms \\< 2 years\n* Signed informed consent\n\nNon-inclusion criteria :\n\n* Pregnant or breast-feeding\n* Treatment with oral or injectable anticoagulants, antiplatelet agents (EXCEPT aspirin at the maximum authorized dosage of 160 mg per day)\n* Unbalanced diabetes\n* Long-term corticosteroid therapy\n* Persons deprived of their liberty by judicial or administrative decision; Persons under legal protection: guardianship or curators\n* Genetic mutations associated with ALS\n\nControl group :\n\nInclusion criteria:\n\n* Male or female volunteer aged 18 or over\n* Person affiliated to a French social security scheme or equivalent\n* Signed informed consent\n\nNon-inclusion criteria :\n\n* Pregnant or breast-feeding women\n* Treatment with oral or injectable anticoagulants, antiplatelet agents (except aspirin at the maximum authorized dosage of 160 mg per day)\n* Unbalanced diabetes\n* Long-term corticosteroid therapy\n* Neurological diseases\n* Patient under legal protection (safeguard of justice, curators and guardianship), or in a situation of deprivation of liberty\n* Genetic mutations associated with ALS", "sex": "ALL", "min_age": "18 Years", "max_age": "100 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "The Nrf-2 pathway in response to oxidative stress, modulated by TDP-43, to induce antioxidant proteins.", "targeting_mechanism_pmid": "34663413", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02469896", "title": "A Trial of Tocilizumab in ALS Subjects", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Barrow Neurological Institute", "summary": "This research study is being done to find out if tocilizumab, also known as Actemra\u2122, can help with Amyotrophic Lateral Sclerosis (ALS). The investigators also want to find out if tocilizumab is safe to take without causing too many side effects.\n\nCurrently ALS has no cure and 2 modestly effective treatment to slow the progression of the disease. Although not the initial cause of ALS, the immune system plays a role in the death of motor neurons. The immune cells that participate in this process are stimulated by a substance called interleukin-6 (IL-6) whose effect is blocked by tocilizumab and thus, may slow the death of motor neurons and slow the disease.", "interventions": [{"type": "DRUG", "name": "Tocilizumab"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2015-11", "url": "https://clinicaltrials.gov/study/NCT02469896", "target_entities": ["IL-6"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Wake Forest University School of Medicine", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Penn State College of Medicine Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Participants with ALS (El Escorial criteria: possible, laboratory-supported probable, probable or definite)\n* Capable of providing informed consent and complying with trial procedures.\n* High inflammatory profile of PBMC gene expression\n* Upright SVC \u226540% of predicted value for gender, height and age at Screening and in the opinion of the investigator is able to comply with and complete the trial.\n* Women must not be able to become pregnant for the duration of the study.\n* Negative tuberculosis blood or skin test at Screening\n* Not taking riluzole, or on a stable dosage for at least 30 days prior to Screening.\n* Subjects medically able to undergo lumbar puncture (LP)\n* Subjects must agree not to take live attenuated vaccines 30 days before Screening, throughout the duration of the trial and for 60 days following the subject's last dose of study drug\n* Geographic accessibility to the study site\n\nAdditional MRI-PET Inclusion Criteria (MGH only):\n\n* High or mixed affinity to bind TSPO protein (Ala/Ala or Ala/Thr) (see section 7.1)\n* Upper Motor Neuron Burden (UMNB) Scale Score \u226525 (out of 45) at the Screening Visit.\n* Able to safely undergo MRI-PET scans based on the opinion of the site investigator.\n\nExclusion Criteria:\n\n* Prior use of Tocilizumab, cell-depleting therapies, alkylating agents, total lymphoid irradiation\n* Stem cell therapies\n* Dependence on mechanical ventilation as defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use\n* Presence of tracheostomy at Screening\n* Exposure to any anti-inflammatory agent currently under investigation for the treatment of patients with ALS (off label use or investigational) within 30 days prior to the Screening Visit (examples include NP001 and Lunasin). Medications that do not have an anti-inflammatory mechanism, such as mexiletine or retigabine are allowed if on stable dose for 30 days prior to Screening visit\n* Treatment with a prohibited medication within 30 days of the Screening Visit\n* Treatment with intravenous gamma globulin, plasmapheresis or Prosorba column within 6 months of Screening\n* Presence of diaphragm pacing system at Screening.\n* Primary or secondary immunodeficiency (history of or currently active) unless related to primary disease under investigation\n* History of or active diverticulitis, diverticulosis requiring antibiotic treatment, peptic ulcer disease, or gastrointestinal (GI) tract perforation, or chronic ulcerative lower GI disease such as Crohn's disease, ulcerative colitis or other symptomatic lower GI conditions that might predispose to perforations\n* Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other opportunistic infections.\n* History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies\n* Presence of any of the following clinical conditions: bleeding diathesis, or any other clinical condition that would, in the opinion of the investigator, place the patient at increased risk during LP. Drug abuse or alcoholism within the past 12 months. Unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active infectious disease, including current or prior malignancy. Rheumatic autoimmune disease, mixed connective tissue disease, scleroderma, polymyositis, or significant systemic involvement secondary to rheumatoid arthritis. Evidence of active malignant disease, malignancies diagnosed within the previous 5 years, or breast cancer diagnosed within the previous 5 years. Human immunodeficiency virus infection or other immunodeficient state.Uncontrolled hypertension defined as systolic blood pressure \\> 170 or diastolic blood pressure \\> 110. Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days of the Screening Visit\n* Any major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks of screening\n* Screening alanine aminotransferase (ALT), aspartate aminotransferase (AST), or total bilirubin \\> than 1.5 times the upper limit of normal (ULN), serum creatinine \\> 1.6 mg/dL in female patients and \\> 1.9 mg/dL in male patients (patients with serum creatinine values exceeding limits may be eligible for the study if their estimated glomerular filtration rate (GFR) are \\>30), hemoglobin \\< 85 g/L, white blood cells \\< 3.0 x 109/L, absolute neutrophil count of \\<2000/mm3, absolute lymphocyte count \\< 0.5 x 109/L, platelet concentration of \\<100,000/mm3, positive Hepatitis B surface antigen (HBsAg)\n* Pregnant women or women currently breastfeeding\n* No history of chicken pox infection or no history of varicella zoster vaccination\n* Any reason in the opinion of the investigator that the patient may not be able to comply with study procedures, complete the study or is unsuitable for immunosuppressive therapy.\n\nAdditional MR-PET Exclusion Criteria (MGH only):\n\n* Any contraindication to undergo MRI studies such as\n\n  * History of a cardiac pacemaker or pacemaker wires\n  * Metallic particles in the body\n  * Vascular clips in the head\n  * Prosthetic heart valves\n  * Claustrophobia\n* Radiation exposure that exceeds the site's current guidelines\n* Current use of tobacco products including cigarettes, e-cigarettes, cigars, snuff and chewing tobacco, or nicotine replacement products such as gum, or patch\n* Taking any other anti-inflammatory or immune modulating medications except for over the counter NSAIDs\n* Unwilling or unable to discontinue benzodiazepine usage (other than Lorazepam, Clonazepam, or Zolpidem) for one day prior to scanning", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tocilizumab", "targeting_mechanism": "IL-6 receptor antagonist that inhibits the immune-mediated death of motor neurons.", "targeting_mechanism_pmid": "28612258", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05542576", "title": "Study of AMDX-2011P as a Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Amydis Inc.", "summary": "The purpose of this research study is to assess safety and tolerability of a single intravenous (given through a vein) dose of the investigational retinal tracer AMDX-2011P in patients with neurodegenerative diseases (Parkinson's disease and ALS).", "interventions": [{"type": "DRUG", "name": "AMDX2011P"}], "start_date": "2022-08-24", "url": "https://clinicaltrials.gov/study/NCT05542576", "target_entities": ["amyloidogenic_proteinopathy"], "locations": [{"facility": "Eye Research Foundation", "city": "Newport Beach", "state": "California", "country": "United States", "status": "", "lat": 33.61891, "lon": -117.92895}, {"facility": "Brittany NIcholl", "city": "Pasadena", "state": "California", "country": "United States", "status": "", "lat": 34.14778, "lon": -118.14452}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nFor Subjects with Parkinson's Disease\n\n1. Clinically established Parkinson's disease based on Movement Disorder Society (MDS) Clinical Diagnostic Criteria for Parkinson's disease (Table 8) and a modified Hoehn \\& Yahr scale of 1-3 (Table 9).\n2. No suspected atypical parkinsonian syndromes due to drugs, metabolic disorders, encephalitis, or degenerative diseases.\n\n   For Subjects with ALS\n3. Confirmed diagnosis of ALS with both upper and lower motor neuron involvement.\n\n   For All Subjects\n4. Ability to undergo retinal imaging.\n5. Subject or legally authorized representative must provide signed informed consent (or signed assent form) prior to study entry and have the ability and willingness to attend and comply with the necessary study procedures and visits at the study site. For subjects unable to physically sign the informed consent, a guardian or trusted care giver can sign on their behalf in presence of an independent witness.\n6. Contraception use by study subjects of childbearing potential (male and female) and female partners of childrearing potential male subjects should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n\nExclusion Criteria:\n\n1. Presence of any underlying physical or psychological medical condition that would make it unlikely that the subject will complete the study per protocol.\n2. Clinically significant laboratory abnormalities assessed by the investigator.\n3. Active malignancy and/or history of malignancy in the past 5 years, with the exception of completely excised non-melanoma skin cancer or low-grade cervical intraepithelial neoplasia.\n4. Prolonged QTcF (\\>450 ms for males and \\>470 ms for females), cardiac arrhythmia, or any clinically significant abnormality in the resting ECG, as judged by the investigator.\n5. Presence of any ocular condition that would significantly hinder the ability to detect and quantify hyper-fluorescent puncta (e.g., eyes with significant hyper-autofluorescence that would mask the ability to detect, quantify, and discern post-injection hyper-fluorescent signal from pre-injection hyper-autofluorescence signal).\n6. Use of any new prescription therapies or vaccines within 7 days prior to the study drug administration.\n7. Drugs with potential phototoxicity per Package Insert are prohibited within 48 hours or 5 half-lives, whichever is longer, prior to first study drug until End-of-study (EOS) visit, except for those required for treatment of underlying disease.\n8. Administration of investigational product in another study within 30 days prior to the first study drug administration, or five half-lives, whichever is longer.\n9. Females who are pregnant or breastfeeding.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AMDX-2011P", "targeting_mechanism": "Retinal tracer for imaging amyloidogenic proteinopathy in neurodegenerative diseases.", "targeting_mechanism_pmid": "36834792", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02548663", "title": "Sport Therapy and Osteopathy Manipulative Treatment in ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Milano Bicocca", "summary": "This project assessed muscle oxidative metabolism and fatigue in patients affected by amyotrophic lateral sclerosis (ALS) undergoing to three months of individualized cardiovascular and strength training. Muscle oxidative metabolism and strength will be assessed by non-invasive methods, such as near-infrared spectroscopy (NIRS) and mechanomyography (MMG). NIRS is a technique giving indications on the capacity of oxygen extraction of muscles during exercise. MMG allows analyzing the pattern of motor unit recruitment and related fatigue. The investigators will also assess the effects of training on pain tolerance and quality of life (QoL) by the Brief Pain Inventory and the McGill Quality of Life questionnaires, using the validated Italian versions. Patients will be assessed longitudinally before (time T0) and after three months of individualized training (time T1). After one month of de-training (time T2) the investigators will assess the hypothetic persistence of any treatment-related effect. The effect of three months-osteopathic treatment (osteo) on pain and QoL will be assessed as well.", "interventions": [{"type": "OTHER", "name": "Sport therapy"}, {"type": "OTHER", "name": "Osteopathic treatment"}], "start_date": "2014-06", "url": "https://clinicaltrials.gov/study/NCT02548663", "target_entities": ["oxidative_stress"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of ALS\n* early stages of disease\n* able to perform exercise with major muscle groups.\n\nExclusion Criteria:\n\n* non-invasive ventilation (NIV)\n* tracheostomy\n* coronaropathy\n* ongoing infectious diseases\n* cognitive deficits.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00140452", "title": "Phase II Study Using Thalidomide for the Treatment of ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Dartmouth-Hitchcock Medical Center", "summary": "The use of Thalidomide in patients with ALS who have disease progression.", "interventions": [{"type": "DRUG", "name": "Thalidomide"}], "start_date": "2005-02", "url": "https://clinicaltrials.gov/study/NCT00140452", "target_entities": ["TNF-alpha", "angiogenesis"], "locations": [{"facility": "Dartmouth Hichcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinically proven ALS\n* Disease duration less than or equal to 5 years\n* ALSFRS-R score equal to or greater then 30\n\nExclusion Criteria:\n\n* Patients with known deep venous thrombosis or hyper coagulable state will be excluded\n* Patients with FVC less than 80%", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Thalidomide", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03127514", "title": "AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Amylyx Pharmaceuticals Inc.", "summary": "The CENTAUR trial was a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.", "interventions": [{"type": "DRUG", "name": "AMX0035"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2017-06-22", "url": "https://clinicaltrials.gov/study/NCT03127514", "target_entities": ["TARDBP", "FUS"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "UC Irvine Medical Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Forbes Norris MDA/ALS Research Center - California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Florida Medical Center", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Carol and Frank Morsini Center for Advanced Health Care - University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University Hospital", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kentucky Medical Center", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Ochsner Neuroscience Institute", "city": "New Orleans", "state": "Louisiana", "country": "United States", "status": "", "lat": 29.95465, "lon": -90.07507}, {"facility": "Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Memorial Medical Center", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "University of Michigan Medical Center", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Washington University Medical Center", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates P.C.", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Mount Sinai Beth Israel", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Wake Forest Baptist Medical Center", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Ohio State University Wexner Medical Center", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Oregon Health & Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "The Penn Comprehensive ALS Center", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Temple University Hospital", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Texas Neurology, P.A.", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Texas Health Science Center at San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "ALS Center at the Swedish Neuroscience Institute", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n1. Male or female, aged 18-80 years of age\n2. Sporadic or familial ALS diagnosed as definite as defined by the World Federation of Neurology revised El Escorial criteria\n3. Less than or equal to 18 months since ALS symptom onset\n4. Capable of providing informed consent and following trial procedures\n5. Slow Vital Capacity (SVC) \\>60% of predicted value for gender, height, and age at the Screening Visit\n6. Subjects must either not take riluzole or be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole-na\u00efve subjects are permitted in the study.\n7. Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug. Women must not be planning to become pregnant for the duration of the study and 3 months after last dose of study drug\n8. Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide for sperm donation for the duration of the study and 3 months after last dose of study drug\n\nKey Exclusion Criteria:\n\n1. Presence of tracheostomy\n2. Exposure to PB, Taurursodiol or UDCA within 3 months prior to the Screening Visit or planning to use these medications during the course of the study\n3. History of known allergy to PB or bile salts\n4. Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \\> 3 times the upper limit of the normal\n5. Renal insufficiency as defined by a serum creatinine \\> 1.5 times the upper limit of normal\n6. Poorly controlled arterial hypertension (systolic blood pressure (SBP)\\>160mmHg or diastolic blood pressure (DBP)\\>100mmHg) at the Screening Visit\n7. Pregnant women or women currently breastfeeding\n8. History of cholecystectomy\n9. Biliary disease which impedes biliary flow including active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder.\n10. History of Class III/IV heart failure (per New York Heart Association - NYHA)\n11. Severe pancreatic or intestinal disorders that may alter the enterohepatic circulation and absorption of TUDCA including biliary infections, pancreatitis and ileal resection\n12. The presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the subject to provide informed consent, according to Site Investigator judgment\n13. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study\n14. Active participation in an ALS clinical trial evaluating a small molecule within 30 days of the Screening Visit\n15. Exposure at any time to any biologic under investigation for the treatment of subjects with ALS (off-label use or investigational) including cell therapies, gene therapies, and monoclonal antibodies.\n16. Implantation of Diaphragm Pacing System (DPS)", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AMX0035", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02152449", "title": "Oral Nutritional Supplementation in Amyotrophic Lateral Sclerosis (ALS) Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Limoges", "summary": "The purpose of this study is to determine whether an early oral nutritional supplementation (ONS) in amyotrophic lateral sclerosis (ALS) patients is effective on the treatment of this rapidly progressive disease.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Oral nutritional supplementation"}], "start_date": "2014-07", "url": "https://clinicaltrials.gov/study/NCT02152449", "target_entities": ["nutritional_status"], "locations": [{"facility": "Service de Neurologie", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients \u226518 years of age, diagnosed with ALS (\\<2 months before inclusion) according to Airlie House criteria : definite, probable, or probable laboratory supported;\n* Time between first symptoms and diagnosis less than 18 months\n* Sporadic or familial cases\n* Patient agreement to be followed in a given ALS centre during the duration of the study\n* Patients with a loss of at least 1 point in 3 items of the ALSFRS-R rating scale or with a loss of at least 2 points in 2 items of the ALSFRS-R rating scale\n* Patients who signed the informed consent form\n\nExclusion Criteria:\n\n* Associated dementia or inability to understand the requirements of the protocol.\n* No helper\n* ONS already begun\n* Artificial nutrition: enteral or parenteral nutrition\n* Known hypersensitivity to components of ONS\n* Absence of treatment with Riluzole (RILUTEK\u00ae)\n* Patient under guardianship or curatorship\n* Participation in another research protocol.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Oral nutritional supplementation", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00386464", "title": "Noninvasive Ventilation in ALS Patients With Mild Respiratory Involvement", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "Non-invasive ventilation or BiPAP\u00ae, which is a form of breathing support delivered through a facemask, is a successful treatment for the respiratory complications of amyotrophic lateral sclerosis (ALS). It has been shown to prolong survival, improve quality of life, and improve cognitive function. It is widely used among patients with ALS who have advanced breathing difficulties. It is not known whether there is benefit to using non-invasive ventilation earlier in the disease course.\n\nThere is evidence that non-invasive ventilation may slow down the decline in breathing function. If this were true then it would make sense to start non-invasive ventilation use earlier than the current clinically accepted practices.\n\nThe purpose of this study is to determine whether using non-invasive ventilation early in the course of disease can slow the decline in breathing function.\n\nPatients remain in the study for 6 months and are asked to make 7 clinic visits during which time they will undergo pulmonary function tests and complete questionnaires.", "interventions": [{"type": "DEVICE", "name": "noninvasive positive pressure ventilation"}], "start_date": "2002-04", "url": "https://clinicaltrials.gov/study/NCT00386464", "target_entities": [], "locations": [{"facility": "Johns Hopkins University School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Probable or definite ALS by El Escorial criteria\n* age \\>17 years\n* FVC \\>60\n* minimal respiratory symptoms (no orthopnea or dyspnea at rest)\n* ability to provide informed consent\n\nExclusion Criteria:\n\n* Presence of another neurodegenerative disease\n* arterial CO2 above 45 mmHg\n* O2 below 60 mmHg\n* coexisting chronic lung disease unrelated to ALS\n* presence of an unstable medical condition such as coronary artery disease, liver failure, renal failure or cancer in the 30 days preceding enrollment", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05797701", "title": "A Study in Healthy Adult Male & Female Participants to Assess the Amount of the Study Medicine (SAR443820) Absorbed by the Body, When Given Orally in Fasted Condition as a Tablet Versus as a Capsule (Part 1) and When Given Orally as a Tablet in Fasted Condition Versus as a Tablet After Food (Part 2)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sanofi", "summary": "Part 1:\n\nThis is an open label, balanced randomized, single dose, 2-sequence, 2-period (period 1 and period 2), 2-treatment crossover (between Treatment A and Treatment B for Part 1), study part to determine the relative bioavailability of SAR443820 in tablet formulation versus capsule formulation under fasted conditions.\n\nTwo treatments are as follows:\n\n* Treatment A: SAR443820 - tablet formulation in fasted condition\n* Treatment B: SAR443820 - capsule formulation in fasted condition Each administration will be a single dose of SAR443820 separated by a wash out of at least 5 days.\n\nPart 2:\n\nThis is an open-label, balanced randomized, single dose, 2-sequence, 2-period (period 1 and period 2), 2-treatment crossover (between Treatment C and Treatment D for Part 2) study part to perform a preliminary assessment of the effect of a high-fat meal on pharmacokinetic parameters of single dose of SAR443820 in tablet formulation.\n\nTwo treatments are as follows:\n\n* Treatment C: SAR443820 - tablet formulation in fasted condition\n* Treatment D: SAR443820 - tablet formulation in fed condition Each administration will be a single dose of SAR443820 separated by a wash out of at least 5 days.\n\nParticipants are not allowed to participate in more than one part of the study. In both Parts 1 and 2, the assessment of pharmacokinetic, safety and tolerability are performed at each treatment period at baseline (prior single dosing) up to 48-hour postdosing in healthy adult male and female participants.", "interventions": [{"type": "DRUG", "name": "SAR443820"}, {"type": "DRUG", "name": "SAR443820"}], "start_date": "2021-07-28", "url": "https://clinicaltrials.gov/study/NCT05797701", "target_entities": [], "locations": [{"facility": "Prism Research-Site Number:8400001", "city": "Saint Paul", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.94441, "lon": -93.09327}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female participant must be 18 to 55 years of age inclusive, at the time of signing the informed consent\n* Participants who are overtly healthy as determined by medical evaluation including medical history, physical examination, laboratory tests, and cardiac monitoring\n* Body weight between 50.0 and 100.0 kg, inclusive, if male, and between 40.0 and 90.0 kg, inclusive, if female, body mass index between 18.0 and 30.0 kg/m\\^2, inclusive\n* All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies\n* Having given written informed consent prior to undertaking any study-related procedure\n\nExclusion Criteria:\n\n* Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, or infectious disease, or signs of acute illness\n* Any medication (including St John's Wort) within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy; any non-live Covid-19 vaccine within the last 2 weeks before randomization, any live attenuated vaccine within the last 28 days before randomization and any other non-vaccine biological drugs given within 4 months before randomization\n* Positive result for hepatitis B, C or human immunodeficiency virus (HIV)\n* Positive result on urine drug screen\n* Positive urine alcohol test\n* Positive severe acute respiratory syndrom coronavirus 2 (SARS-CoV-2) test\n* Any consumption of citrus fruits (grapefruit, orange, etc) or their juices within 5 days before inclusion\n\nThe above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "55 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "SAR443820", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06829212", "title": "Research on Wireless Brain Implant System for General Control of External Devices", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shanghai StairMed Technology Co., Ltd.", "summary": "The clinical trial aims to evaluate the safety and efficacy of the minimally invasive, wireless brain-machine interface system (WRS) in enabling general brain control of external devices, such as a cursor and other assistive technologies, for paralyzed and amputee patients.\n\nWRS integrates a high-throughput, ultra-flexible neural electrode with an extremely small cross-sectional size-approximately one-hundredth the diameter of a human hair. Moreover, the implantable component is fully embedded within the body, leaving no visible external traces.", "interventions": [{"type": "DEVICE", "name": "WRS"}], "start_date": "2025-03", "url": "https://clinicaltrials.gov/study/NCT06829212", "target_entities": [], "locations": [{"facility": "Huashan Hospital Affiliated to Fudan University", "city": "Shanghai", "state": "Shanghai Municipality", "country": "China", "status": "RECRUITING", "lat": 31.22222, "lon": 121.45806}], "contact_phone": "021-80510178", "contact_email": "kongcen@stairmed.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Aged 18-80 years (inclusive), any gender.\n* Diagnosed with spinal cord injury, brainstem stroke, amyotrophic lateral sclerosis, or other motor neuron diseases causing partial or complete paralysis, or bilateral upper limb amputation.\n* Diagnosis stable for at least 6 months before screening, with the condition present for at least 1 year.\n* Willing to follow the study protocol and attend all visits, with or without caregiver assistance.\n* Informed consent signed by participant and/or caregiver, with full understanding of the trial's purpose.\n\nExclusion Criteria:\n\n* Previous implantation of metal objects or devices (except dental implants or non-impacting implants).\n* Long-term use of anticoagulants/antiplatelets with insufficient cessation, or abnormal coagulation test results.\n* Unable to tolerate anesthesia or surgery.\n* Severe neurological disorders or brain injury leading to significant dysfunction.\n* Scalp conditions that may impair wound healing.\n* Acute or severe infections.\n* Cognitive impairment or psychiatric disorders.\n* Severe dysfunction of vital organs, malignancies, or autoimmune diseases.\n* Life expectancy under 1 year.\n* Drug or alcohol abuse.\n* Pregnant, breastfeeding, or planning pregnancy during the study.\n* Other conditions deemed unsuitable by the investigator.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04744532", "title": "iPSC-based Drug Repurposing for ALS Medicine (iDReAM) Study", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Kyoto University", "summary": "This study consists of a phase 1 part and a phase 2 part.\n\nPhase 1 part:\n\nThis is a phase 1, open-label, multicenter, dose escalation study to evaluate the safety and tolerability of bosutinib to determine the maximum tolerated dose(MTD) and a recommended phase 2 dose (RP2D) of bosutinib for treatment of ALS patients. Also, efficacy will be evaluated exploratory.\n\nPhase 2 part:\n\nThis is an open label, multicenter, phase 2 part whose purpose is to evaluate the efficacy exploratorily and the long-term (for 24 weeks) safety of bosutinib for the treatment of ALS patients.", "interventions": [{"type": "DRUG", "name": "Bosutinib (Phase 1 part)"}, {"type": "DRUG", "name": "Bosutinib (Phase 2 part)"}], "start_date": "2019-03-19", "url": "https://clinicaltrials.gov/study/NCT04744532", "target_entities": ["SRC"], "locations": [{"facility": "Hiroshima University", "city": "Hiroshima", "state": "", "country": "Japan", "status": "", "lat": 34.4, "lon": 132.45}, {"facility": "Nara Medical University", "city": "Kashihara", "state": "", "country": "Japan", "status": "", "lat": 34.58333, "lon": 135.61667}, {"facility": "Kyoto University", "city": "Kyoto", "state": "", "country": "Japan", "status": "", "lat": 35.02107, "lon": 135.75385}, {"facility": "Kitasato University", "city": "Sagamihara", "state": "", "country": "Japan", "status": "", "lat": 35.56707, "lon": 139.24167}, {"facility": "Tokushima university", "city": "Tokushima", "state": "", "country": "Japan", "status": "", "lat": 34.06667, "lon": 134.56667}, {"facility": "Toho University", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "Tottori University", "city": "Yonago", "state": "", "country": "Japan", "status": "", "lat": 35.43333, "lon": 133.33333}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "\\[Phase 1 part\\]\n\nInclusion criteria:\n\n1. Evidence of a personally signed and dated informed consent document indicating that the patient has been informed of all pertinent aspects of the study. To be additionaly signed by a delegate signer if the subject is unable to handwrite.\n2. Patients aged \u226520 years and \\<80 years at the time of informed consent\n3. Patients with positive already-reported SOD1 gene mutation and progressive muscle weakness; sporadic ALS patients who are categorized as either \"Definite ALS\" or \"Probable ALS\" or \"Probable-laboratory supported ALS\" in the Updated Awaji Criteria for the diagnosis of ALS\n4. Patients at Grade 1 or 2 in the Japan ALS Severity Scale of the grant-in-aid program for chronic diseases from the Japanese Ministry of Health, Labour and Welfare; patients with positive SOD1 mutation of Grade 1, 2 or 3\n5. Patients with ALS that occurred within 2 years at the time of the first registration; patients with positive SOD1 mutation within 5 years after disease onset\n6. Patients who can visit hospital regularly as outpatients\n7. Patients with change in total ALSFRS-R score during the observation period are -1 to -3 points\n8. Urine pregnancy test (for females of childbearing potential) negative at screening\n\n   Female patients of nonchildbearing potential must meet at least 1 of the following criteria:\n   1. Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed with a serum follicle stimulating hormone (FSH) level confirming the postmenopausal state;\n   2. Have undergone a documented hysterectomy and/or bilateral oophorectomy;\n   3. Have medically confirmed ovarian failure. All other female patients (including female patients with tubal ligations) are considered to be of childbearing potential.\n\n   Male and female patients of childbearing potential must agree to use one highly effective method of contraception as outlined in this protocol, throughout the study and for at least 28 days after the last dose of investigational product.\n9. Patients with appropriate renal function as defined as follows at the time of the first and second registrations\n\n   a. Serum creatinine \u22641.5 \u00d7 upper limit of normal (ULN) or estimated creatinine clearance \u226560 mL/min as calculated using the method standard for the institution.\n10. Patients with appropriate hepatic function as defined as follows at the time of the first and second registrations b. Total serum bilirubin \u22641.5 \u00d7 ULN unless the patient has documented Gilbert syndrome; c. AST and ALT \u22642.5 \u00d7 ULN\n11. Able to take oral tablets\n12. Patients whose acute effect of previous treatment has recovered to the baseline or CTCAE v.4.03 \u2264 Grade 1 at the time of the first and second registrations\n13. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures\n\nExclusion criteria:\n\n1. Patients with tracheostomy\n2. Patients who have used non-invasive ventilation due to ALS symptoms\n3. Patients whose %FVCs are less than 70% at the time of first and second registrations\n4. Patients who have nerve conduction study findings of demyelination such as conduction block\n5. Patients who are taking edaravone; patients who started riluzole or edaravone after start of the observation period; patients who changed the dosage of riluzole after start of the observation period\n6. Patients with bulbar type ALS with dysphagia and dysarthria\n7. Patients with cognitive impairment\n8. Pregnant female patients; breastfeeding female patients; fertile male and female patients of childbearing potential who are unwilling or unable to use 1 highly effective methods of contraception as outlined in this protocol for the duration of the study and for at least 28 days after the last dose of investigational product\n9. History of clinically significant or uncontrolled cardiac disease including:\n\n   * History of, or active, congestive heart failure;\n   * Uncontrolled angina or hypertension within 3 months prior to registration;\n   * Myocardial infarction within 12 months prior to registration;\n   * Clinically significant ventricular arrhythmia (such as ventricular tachycardia, ventricular fibrillation, or Torsades de pointes);\n   * Diagnosed or suspected congenital or acquired prolonged QT interval history or prolonged QTc (QTcF should not exceed 500 msec);\n   * Unexplained syncope\n10. Uncontrolled hypomagnesemia or uncorrected hypokalemia due to potential effects on the QT interval\n11. Patient who is taking the following medicines during study drugs administration.\n\n    a Combination of warfarin or other anticoagulation. Combination of therapeutic anticoagulant therapy with low molecular weight heparin is acceptable b Src or c-Abl inhibitors c Other treatments for cancer d Drugs known to prolong the QT interval or predispose to Torsades de Pointe e Current or anticipated use of a strong or moderate CYP3A inhibitor and inducer f Drugs affecting gastric pH such as Proton pump inhibitors (e.g., lansoprazole)\n12. History of malignancy within 5 years prior to registration with the exception of basal cell carcinoma or cervical carcinoma in situ or Stage 1 or 2 cancer that is considered adequately treated and currently in complete remission for at least 12 months\n13. Patients who were enrolled in other clinical study within 12 weeks before the first registration, or are expected to be enrolled in other clinical study using a study drug during this study\n14. Known prior or suspected severe hypersensitivity to study drugs or any component in their formulations\n15. Patients with active, uncontrolled bacterial, fungal, or viral infection, including hepatitis B virus (HBV), hepatitis C virus (HCV), known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS) related illness\n16. Recent or ongoing clinically significant GI disorder (eg, Crohn's disease, ulcerative colitis, or prior total or partial gastrectomy).\n17. Patients with chronic obstructive pulmonary disease\n18. Major surgery or radiotherapy within 14 days prior to registration at the time of the first registration\n19. Patient who fulfills the conditions:\n\n    1. Neutrophil count (ANC) \\<1,500/mm3 or white blood cell \\<3,000/mm3 at the time of the first and second registration\n    2. Hemoglobin \\<9.0 g/dL at the time of the first and second registrations\n    3. Platelet count \\<100,000/L at the time of the first and second registrations\n20. Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study\n21. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or Pfizer employees, including their family members, directly involved in the conduct of the study\n\n\\[Phase 2 part\\]\n\nInclusion criteria:\n\n1. Evidence of a personally signed and dated informed consent document indicating that the patient has been informed of all pertinent aspects of the study. To be additionally signed by a delegate signer if the subject is unable to handwrite.\n2. Patients aged \u226520 years and \u226475 years at the time of informed consent\n3. ALS patients who are categorized as either \"Definite ALS\" or \"Probable ALS in the El Escorial and revised Airlie House criteria for the diagnosis of ALS\n4. Patients at Grade 1 or 2 in the Japan ALS Severity Scale of the grant-in- aid program for chronic diseases from the Japanese Ministry of Health, Labour and Welfare\n5. Patients with ALS within 2 years of symptom onset at the time of the first registration\n6. Patients with change in total ALSFRS-R score during the observation period from -1 to -4 points\n7. Patients with score of at least 2 on all items of ALSFRS-R; 4.Writing, 5.Feeding behavior (1) must have at least 2 points on each side.\n8. Urine pregnancy test (for females of childbearing potential) negative at screening\n\n   Female patients of nonchildbearing potential must meet at least 1 of the following criteria:\n   1. Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed with a serum follicle stimulating hormone (FSH) level confirming the postmenopausal state;\n   2. Have undergone a documented hysterectomy and/or bilateral oophorectomy;\n   3. Have medically confirmed ovarian failure. All other female patients (including female patients with tubal ligations) are considered to be of childbearing potential.\n9. Patients with appropriate renal function as defined as follows at the time of the first and second registrations\n\n   a. Estimated creatinine clearance or eGFR \u226560 mL/min (mild renal impairment) as calculated using the method standard for the institution (the CKD-EPI equation is recommended, other methods such as Cockcroft-Gault or MDRD may be used. The same method should be applied throughout the study period.).\n10. Patients with appropriate hepatic function as defined as follows at the time of the first and second registrations\n\n    1. Total serum bilirubin 1.5 \u00d7 ULN unless the patient has documented Gilbert syndrome;\n    2. AST and ALT 2.5 \u00d7 ULN\n11. Patients who can consistently take the investigational drug and other oral tablets with water throughout the study period.\n12. Patients whose adverse event during previous treatment has recovered to the baseline (Visit 5: before the start of study drug administration) or CTCAE v.4.03 \u2264 Grade 1 at the time of the first and second registrations. Excluding the case where the investigator (sub-investigator) judges that the event is not a safety risk.\n13. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures\n\nExclusion criteria:\n\n1. Patients with tracheostomy\n2. Patients who had decreased respiratory function and complained of dyspnea at the time of enrollment (One of the three items on the ALSFRS-R related to respiratory (10) dyspnea, (11) orthopnea, or (12) respiratory failure is less than 3 points).\n3. Patients whose %FVCs are at least 80 % at the time of first and second registrations\n4. Patients who have nerve conduction study findings of demyelination such as conduction block\n5. Patients using edaravone within 4 weeks prior to enrollment in the observation period; patients using edaravone at the time of enrollment in the observation period; patients who started edaravone after start of the observation period\n6. Patients who started riluzole after start of the observation period; patients who changed the dosage of riluzole after start of the observation period\n7. Patients with bulbar-onset type ALS with dysphagia and dysarthria\n8. Patients with Parkinson's disease and syndromes, schizophrenia, cognitive impairment, and other comorbidities that may have a significant impact on the evaluation of drug efficacy\n9. Patients with a history of spinal surgery such as cervical spondylosis or disc herniation after the onset of ALS, or patients who were scheduled to undergo surgery during the study period\n10. Patients whose symptoms could not be ruled out as symptoms of a disease that requires differential diagnosis, such as cervical spondylosis or multifocal motor neuropathy.\n11. Pregnant female patients; breastfeeding female patients\n12. History of clinically significant or uncontrolled cardiac disease including:\n\n    * History of, or active, congestive heart failure;\n    * Uncontrolled angina or hypertension within 3 months prior to registration;\n    * Myocardial infarction within 12 months prior to registration;\n    * Clinically significant ventricular arrhythmia (such as ventricular tachycardia, ventricular fibrillation, or Torsades de pointes);\n    * Diagnosed or suspected congenital or acquired prolonged QT interval history or prolonged QTc (QTcF should not exceed 500 msec);\n    * Unexplained syncope\n13. Uncontrolled hypomagnesemia or uncorrected hypokalemia due to potential effects on the QT interval\n14. Patient who is taking the following medicines during study drugs administration. Refer to Prohibited Medications.\n\n    a Combination of warfarin or other anticoagulation. Combination of low molecular weight heparin is acceptable b Src or c-Abl inhibitors c Drugs known to prolong the QT interval or predispose to Torsades de Pointe d Current or anticipated use of a strong or moderate CYP3A inhibitor and inducer e Drugs affecting gastric pH such as Proton pump inhibitors (e.g., lansoprazole)\n15. History of malignancy within 5 years prior to the first registration with the exception of basal cell carcinoma or cervical carcinoma in situ or Stage 1 or 2 cancer that is considered adequately treated and currently in complete remission for at least 12 months\n16. Patients who were enrolled in other clinical study within 12 weeks before the first registration, or are expected to be enrolled in other clinical study using a study drug during this study\n17. Known prior or suspected severe hypersensitivity to study drugs or any component in their formulations\n18. Patients with active, uncontrolled bacterial, fungal, or viral infection, including hepatitis B virus (HBV), hepatitis C virus (HCV), known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS) related illness\n19. Recent or ongoing clinically significant GI disorder (eg, Crohn's disease, ulcerative colitis, or prior total or partial gastrectomy).\n20. Patients with chronic obstructive pulmonary disease\n21. Major surgery within 14 days prior to registration at the time of the first registration\n22. Patient who fulfills the conditions:\n\n    1. Neutrophil count (ANC) \\<1,500/mm3 or white blood cell \\<3,000/ mm3 at the time of the first and second registration\n    2. Hemoglobin \\<9.0 g/dL at the time of the first and second registrations\n    3. Platelet count \\<100,000/\u03bcL at the time of the first and second registrations\n23. Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study\n24. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or Pfizer employees, including their family members, directly involved in the conduct of the study", "sex": "ALL", "min_age": "20 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Bosutinib", "targeting_mechanism": "inhibition of the Src/c-Abl pathway", "targeting_mechanism_pmid": "28539470", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07006571", "title": "At-home Treatment With Cortico-spinal tDCS for Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Trieste", "summary": "Amyotrophic lateral sclerosis (ALS) is a progressive neurological disease that causes gradual muscle weakness and loss of muscle mass. It affects all muscles that control movement, speech, swallowing, and breathing. Unfortunately, ALS is currently incurable, and treatments are limited. Only two medications, riluzole and edaravone, have been approved and can slightly extend survival, typically between 20 and 48 months from diagnosis.\n\nRecent research has identified a useful biomarker known as neurofilament light chain (NfL), which increases in the blood as nerve cells become damaged. Measuring NfL levels can help track the progression of ALS.\n\nA promising non-invasive treatment called transcranial direct current stimulation (tDCS) has shown potential benefits for patients with ALS. tDCS involves safely applying mild electrical currents to specific areas of the brain and spinal cord. This approach aims to stimulate nerve cells, potentially improving their function and slowing disease progression. Initial studies have reported temporary improvements in muscle strength and survival when tDCS was used over a short period.\n\nBased on these encouraging results, our study proposes a new home-based tDCS treatment program specifically designed for ALS patients. Participants will use an easy-to-operate, safe, and portable device at home. The treatment involves placing electrodes on the scalp and the neck area to stimulate both the motor areas of the brain and the spinal cord. Therapy sessions will occur five days per week over 16 weeks.\n\nThis home-based approach allows patients to comfortably receive therapy without daily trips to the hospital, making treatment more accessible and convenient. By providing this therapy at home, the investigators aim to improve the quality of life for ALS patients and explore new possibilities in treating and managing ALS and other neurodegenerative diseases.", "interventions": [{"type": "DEVICE", "name": "Real tDCS"}, {"type": "DEVICE", "name": "Sham tDCS"}], "start_date": "2025-05-19", "url": "https://clinicaltrials.gov/study/NCT07006571", "target_entities": ["corticospinal_tract"], "locations": [{"facility": "Clinica Neurologica, Azienda Sanitaria Universitaria Giuliano Isontina", "city": "Trieste", "state": "Trieste", "country": "Italy", "status": "RECRUITING", "lat": 45.64953, "lon": 13.77678}], "contact_phone": "+39 0403994282", "contact_email": "benussialberto@gmail.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female patients with a probable, laboratory-supported diagnosis of ALS, or defined ALS according to current clinical criteria\n* Age greater than 18 years\n* Onset of disease \u2264 24 months\n* Disease progression in the last 3 months\n* A score \u2265 2 on the \"respiratory failure\" item on the ALS Functional Rating Scale Revised (ALSFRS-R)\n* Treatment with riluzole or edaravone is permitted, provided it has been stable for at least 1 month prior to enrollment in the study, or no ALS-specific treatment\n* Presence of a caregiver who can assist the patient and who has successfully completed the necessary training in the use of the device\n* Signature of informed consent\n\nExclusion Criteria:\n\n* People with fixed electrical stimulators (e.g. cardiac pacemakers, nerve stimulators, hearing implants) that would not work or would be damaged by the electric field;\n* People with particular intracranial metal foreign bodies (e.g. splinters, some prostheses, screws and nails) that could interact with the electric field\n* People with a history of epilepsy;\n* As the effects of tDCS on the developing fetus are not known, pregnant women will be excluded from the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03076632", "title": "Interactions Between Neurostimulation and Physical Exercise", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Bronx VA Medical Center", "summary": "* People with cervical spinal cord injury (SCI) and amyotrophic lateral sclerosis (ALS) have reduced connections in the nerve circuits between the brain and the hands. Activating spared nerve circuits is one potential way to improve recovery.\n* The investigators are testing different combinations of physical wrist and hand movements paired with magnetic brain stimulation and electrical spinal cord or nerve stimulation to see the effects on nerve transmission to hand muscles.\n* This is a preliminary study. This study is testing for temporary changes in nerve transmission to hand muscles. There is no expectation of long-term benefit from this study. If temporary changes are seen in this study, then future studies would focus on how to prolong that effect.", "interventions": [{"type": "DEVICE", "name": "Cervical plus transcranial stimulation"}, {"type": "DEVICE", "name": "Cervical stimulation plus hand/wrist exercise"}, {"type": "DEVICE", "name": "Electromyographic (EMG)-triggered (closed-loop) stimulation"}], "start_date": "2017-04-01", "url": "https://clinicaltrials.gov/study/NCT03076632", "target_entities": ["neural_circuit_activation"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age between 21 and 65 years;\n2. Chronic (more than 12 months since injury) incomplete SCI between levels C2-C8 or diagnosis of definite or probable ALS;\n3. Incomplete weakness of left or right hand muscles: score of 2, 3, or 4 (out of 5) on manual muscle testing of finger extension, finger flexion, or finger abduction;\n4. Detectable F-wave responses of the left or right abductor pollicis brevis muscle to median nerve stimulation.\n\nExclusion Criteria:\n\n1. Multiple spinal cord lesions;\n2. History of seizures;\n3. Ventilator dependence or patent tracheostomy site;\n4. Use of medications that significantly lower seizure threshold, such as tricyclic antidepressants, amphetamines, neuroleptics, dalfampridine, and bupropion;\n5. History of stroke, brain tumor, brain abscess, or multiple sclerosis;\n6. History of moderate or severe head trauma (loss of consciousness for greater than one hour or evidence of brain contusion or hemorrhage or depressed skull fracture on prior imaging);\n7. History of implanted brain/spine/nerve stimulators, aneurysm clips, ferromagnetic metallic implants, or cardiac pacemaker/defibrillator;\n8. Significant coronary artery or cardiac conduction disease;\n9. Recent history (within past 6 months) of recurrent autonomic dysreflexia, defined as a syndrome of sudden rise in systolic pressure greater than 20 mm Hg or diastolic pressure greater than 10 mm Hg, without rise in heart rate, accompanied by symptoms such as headache, facial flushing, sweating, nasal congestion, and blurry vision (this will be closely monitored during all screening and testing procedures);\n10. History of bipolar disorder;\n11. History of suicide attempt;\n12. Active psychosis;\n13. Heavy alcohol consumption (greater than equivalent of 5 oz of liquor) within previous 48 hours;\n14. Open skin lesions over the face, neck, shoulders, or arms;\n15. Pregnancy\n16. Unsuitable for study participation as determined by study physician.", "sex": "ALL", "min_age": "21 Years", "max_age": "65 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03321487", "title": "Blood-Brain Barrier Opening Using MR-Guided Focused Ultrasound in Patients With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "InSightec", "summary": "The purpose of this study is to evaluate the safety, tolerability, and feasibility of Blood-Brain Barrier (BBB) opening using transcranial MRI-guided focused ultrasound in conjunction with an intravenous ultrasound contrast agent in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DEVICE", "name": "Blood-Brain Barrier opening with MRgFUS"}], "start_date": "2018-04-13", "url": "https://clinicaltrials.gov/study/NCT03321487", "target_entities": ["blood_brain_barrier_permeability"], "locations": [{"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Diagnosed with laboratory supported probable, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria (Brooks et al. 2000).\n2. Right-hand dominant male or female aged 18 years or older.\n3. Capable of providing informed consent and complying with study procedures, including tolerability in the supine position and MRI examination without significant claustrophobia.\n4. If taking riluzole, on a stable dose for at least 30 days prior to Screening Visit.\n5. Slow Vital Capacity equal to or more than 50% predicted value for gender, height and age in the 30 days prior to the Screening Visit and able to lie supine without BiPAP.\n6. Severe left arm weakness and functional impairment, defined as Medical Research Council muscle strength score equals 3 or less in the index finger abduction and thumb abduction on the left side; OR severe left leg weakness and functional impairment, defined as Medical Research Council muscle strength score equals 3 or less at the hip flexors and ankle dorsiflexors on the left side.\n7. Able to communicate during the ExAblate\u00ae MRI-guided FUS procedure.\n\nExclusion Criteria:\n\n1. Unable to complete high-density CT and MRI studies of the head at the Screening Visit or any other MRI contraindication, such as:\n\n   * Large body habitus and not fitting comfortably into the scanner\n   * Difficulty lying supine and still for up to 3 hours in the MRI unit or significant claustrophobia\n2. MRI findings:\n\n   * Active infection/inflammation\n   * Acute or chronic brain hemorrhages, specifically lobar or subcortical microbleeds, siderosis or macrohemorrhages\n   * Tumor/space occupying lesion\n   * Meningeal enhancement\n3. More than 30% of the skull area traversed by the sonication pathway is covered by scars, scalp disorders (e.g., eczema), or atrophy of the scalp.\n4. Clips or other metallic implanted objects in the skull or the brain, except shunts.\n5. Significant cardiac disease or unstable hemodynamic status including:\n\n   * Documented myocardial infarction within six months of enrollment\n   * Unstable angina on medication\n   * Unstable or worsening congestive heart failure\n   * Left ventricular ejection fraction below the lower limit of normal\n   * History of a hemodynamically unstable cardiac arrhythmia\n   * Cardiac or phrenic pacemaker\n   * Known right-to-left, bidirectional, or transient right-to-left cardiac shunt\n   * Patients with relative contraindications to perflutren including subjects with a family or personal history of QT prolongation or taking concomitant medications known to cause QTc prolongation,\n   * QT prolongation observed on screening ECG (QTc \\> 450 for men and \\>470 for women)\n6. Uncontrolled hypertension (systolic \\> 150 or diastolic BP \\> 100 on medication).\n7. On medications that increase the bleeding risk, specifically: a) aspirin or another antiplatelet medication (clopidogrel, prasugrel, ticlopidine, abciximab) for the last 7 days prior to treatment; b) oral, subcutaneous or intravenous anticoagulant medications, such as oral vitamin K inhibitors for the last 7 days, non-vitamin K inhibitor oral anticoagulant (dabigatran, apixaban, rivaroxaban) for the last 72 hours, and intravenous or subcutaneous heparin-derived compounds for the last 48 hours.\n8. History of a bleeding disorder, coagulopathy or a history of spontaneous hemorrhage.\n9. Known frontotemporal dementia.\n10. Abnormal coagulation profile, specifically: platelet \\<100,000/\u03bcl, Prothrombin Time \\>14 seconds, activated partial thromboplastin time (aPTT) \\>36 seconds, and INR \\> 1.3.\n11. Known cerebral or systemic vasculopathy, specifically cerebral amyloid angiopathy or systemic or central nervous system vasculitis.\n12. Known auto-immune condition with or without neurological manifestations (e.g., multiple sclerosis (MS), systemic lupus erythematous (SLE), Rheumatoid arthritis).\n13. Current or planned use of oral, intramuscular or intravenous steroid drugs (such as prednisone, prednisolone, dexamethasone, triamcinolone, methylprednisolone, oxandrolone, and others) or immunosuppressant drugs (azathioprine, mycophenolate, tacrolimus, sirolimus, cyclophosphamide, and others) for more than 7 days.\n14. Known sensitivity/allergy to gadolinium (an alternative product may be used), DEFINITY\u00ae contrast or any of its components.\n15. Untreated, uncontrolled sleep apnea.\n16. Impaired renal function with cystatin C-based estimated glomerular filtration rate \\<30 mL/min/1.73m2 and acute renal injury.\n17. Currently in a clinical trial involving an investigational product or non-approved use of a drug or device.\n18. Known respiratory diseases, specifically: chronic pulmonary disorders e.g., severe/uncontrolled COPD, pulmonary vasculitis, or other causes of reduced pulmonary vascular cross-sectional area, asthma or hay fever.\n19. Patients with a history of drug allergies or multiple allergies where the benefit/risk of administering DEFINITY\u00ae is considered unfavorable by the study physicians in relation to the product monograph for DEFINITY\u00ae.\n20. Unqualified fit for the anesthesia by an anesthesiologist assessment, ASA I-III.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MR-guided focused ultrasound (MRgFUS) with ultrasound contrast agent", "targeting_mechanism": "MR-guided focused ultrasound opens the blood-brain barrier to enable delivery of therapeutic agents to the central nervous system.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06069934", "title": "A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2)", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Prilenia", "summary": "Protocol PL101-ALS501: This EAP will provide access to pridopidine for up to 200 patients with ALS who are ineligible for clinical trials.", "interventions": [{"type": "DRUG", "name": "Pridopidine"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT06069934", "target_entities": ["D2 dopamine receptor"], "locations": [{"facility": "University of Alabama at Birmingham", "city": "Birmingham", "state": "Alabama", "country": "United States", "status": "", "lat": 33.52066, "lon": -86.80249}, {"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California, San Diego Health", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "UC Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center - Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Anschutz Medical Campus", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "Nova Southeastern University", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "Mayo Clinic Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "The University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Lahey Hospital Medical Center", "city": "Burlington", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.50482, "lon": -71.19561}, {"facility": "University of Massachusetts Chan Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Essentia Health", "city": "Duluth", "state": "Minnesota", "country": "United States", "status": "", "lat": 46.78327, "lon": -92.10658}, {"facility": "University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Mayo Clinic Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Stony Brook University Hospital", "city": "Stony Brook", "state": "New York", "country": "United States", "status": "", "lat": 40.92565, "lon": -73.14094}, {"facility": "SUNY Upstate", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "OhioHealth", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Thomas Jefferson University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Lewis Katz School of Medicine at Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "UT Southwestern Medical Center", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Virginia Commonwealth University", "city": "Richmond", "state": "Virginia", "country": "United States", "status": "", "lat": 37.55376, "lon": -77.46026}, {"facility": "Medical College of Wisconsin", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "CHALS-CCT Program, UPR-MSC", "city": "San Juan", "state": "", "country": "Puerto Rico", "status": "", "lat": 18.46633, "lon": -66.10572}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria Amyotrophic Lateral Sclerosis (ALS):\n\n* Sporadic or familial ALS.\n* Patient does not qualify for clinical trials of pridopidine or as per site investigator's opinion, and is not medically or geographically suitable for other clinical trials.\n* Capable of providing informed consent and complying with study procedures, in the site investigator's opinion.\n* Patient has established care with a physician at a specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP.\n* Pridopidine naive patients must have a life expectancy of at least 6 months in the site investigator's opinion.\n\nExclusion Criteria ALS:\n\n* Confirmed prolonged Fridericia-corrected QT (QTcF) interval (\\>450 ms for men; \\>470 ms for women).\n* Clinically significant heart disease, clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, asymptomatic sustained ventricular tachycardia, or left bundle branch block.\n* Known history of long QT syndrome or a first degree relative with long QT syndrome.\n* Use of prohibited medications within the 4 weeks prior to baseline.\n* Use of Nuedexta (\\>20 mg dextromethorphan and \\>10 mg quinidine twice daily); citalopram \\>20 mg/day; escitalopram \\>10 mg/day.\n* Known allergy to pridopidine or any of the exipients (silicified microcrystalline cellulose, magnesium stearate).\n* History of any clinically significant or unstable medical condition or laboratory abnormality that, based on site investigator's judgment, may interfere with assessment of the study objectives.\n* Female who is pregnant or nursing or who plans to get pregnant during the course of the EAP.\n* Female of child-bearing potential or male unwilling or unable to use accepted methods of birth control.\n* Use of investigational treatments for ALS (as part of participation in a clinical trial or another EAP) within 5 half-lives (if known) or 30 days (whichever is longer) prior to screening (other than pridopidine).\n* Patient receives or has received any gene or cell-based therapy.\n* Active cancer or history of cancer, except for basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.\n* Patients who chose to take experimental medications and/or supplements, and for whom this is the only reason they are not eligible for trials.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Pridopidine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02874209", "title": "Noninvasive Assessment of Neuronal Damage by MRI Sodium ( 23Na ) in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique Hopitaux De Marseille", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting the central and peripheral motor neurons, characterized by the rapidity of its evolution (median survival of 3 years). The pathophysiology of the disease is still poorly understood. Neuronal death results from several cellular mechanisms entangled, including mitochondrial dysfunction. The absence of diagnostic marker causes a significant delay in diagnosis, on average a year. On the other hand, the wish biomarker is important for therapeutic trials. Recently, MRI sodium (23Na) demonstrated its importance to detect noninvasively sodium accumulations associated with neuronal suffering. This neuronal pain can be caused by mitochondrial dysfunction causing the accumulation in the sodium and calcium cell causing neuronal death. These studies were conducted in multiple sclerosis, Alzheimer's disease, Huntington's disease, stroke and brain tumors. They demonstrated that sodium MRI could be an effective and sensitive biomarker for detecting and quantifying neuronal degeneration. The goal of this study is to assess neuronal damage noninvasively by MRI sodium in amyotrophic lateral sclerosis.", "interventions": [{"type": "OTHER", "name": "sodium MRI"}], "start_date": "2015-09", "url": "https://clinicaltrials.gov/study/NCT02874209", "target_entities": ["neuronal_sodium_homeostasis"], "locations": [{"facility": "Assistance Publique Hopitaux de Marseille", "city": "Masreille", "state": "", "country": "France", "status": "RECRUITING", "lat": null, "lon": null}], "contact_phone": "", "contact_email": "aude.grapperon@ap-hm.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Amyotrophic lateral sclerosis patient according to the El Escorial criteria revised Brooks et al. 2000 bulbar or spinal beginning\n\nExclusion Criteria:\n\n* patient or healthy volonteer presenting MRI contre indications to this exam.\n* patient or healthy volonteer presenting severe high blood pressure undergoing medication to treat it or not.\n* patient or healthy volonteer having chronic psychiatric illness, dementia", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Sodium MRI measures intracellular sodium accumulation as a biomarker of neuronal dysfunction and neuronal death in motor neurons.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05959850", "title": "A Double-blind Randomised, Placebo-controlled Clinical Trial to Test Ambroxol Treatment in ALS", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The Florey Institute of Neuroscience and Mental Health", "summary": "Ambroxol is a simple cough medicine that is predicted to slow ALS disease progression. This study aims to investigate if ambroxol in high doses is effective in treating ALS. This study will be carried out across 5 research sites in Australia (2 NSW, 1 VIC, 1 SA and 1 TAS), where newly diagnosed ALS patients will be asked to participate. Participation will be over a 32-week period, where they will come in for a 4-week screening, 24-week treatment, and 4-week end of study safety follow-up period. The participants will receive either the placebo or drug solution that they will take three times a day, up-dosing each week until they reach the maximum dose or highest dose they can tolerate. Throughout the study their disease progression will be assessed using tests, questionnaires, and blood biomarkers.", "interventions": [{"type": "DRUG", "name": "Ambroxol"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2023-06-13", "url": "https://clinicaltrials.gov/study/NCT05959850", "target_entities": ["Glucocerebrosidase"], "locations": [{"facility": "Brain and Mind Centre", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": -33.86785, "lon": 151.20732}, {"facility": "Concord Repatriation General Hospital", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": -33.86785, "lon": 151.20732}, {"facility": "Flinders Medical Centre", "city": "Adelaide", "state": "South Australia", "country": "Australia", "status": "RECRUITING", "lat": -34.92866, "lon": 138.59863}, {"facility": "Launceston General Hospital", "city": "Launceston", "state": "Tasmania", "country": "Australia", "status": "RECRUITING", "lat": -41.43876, "lon": 147.13467}, {"facility": "Calvary Health Care Bethlehem", "city": "Melbourne", "state": "Victoria", "country": "Australia", "status": "RECRUITING", "lat": -37.814, "lon": 144.96332}], "contact_phone": "+61 3 9035 6521", "contact_email": "bradley.turner@florey.edu.au", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Must have given written informed consent before any study related assessments are performed and must be able to understand purpose of the study, including any possible risks and adverse events.\n2. ALS as diagnosed according to the recently proposed Gold Coast diagnostic criteria.\n3. First symptom of ALS less than or equal to 18 months prior to screening. The qualifying first symptoms of ALS are limited to manifestations of weakness in extremity, bulbar, or respiratory muscles. Cramps, fasciculations, or fatigue should not be taken in isolation as a first symptom of ALS.\n4. Forced vital capacity (FVC) greater than or equal to 60% of predicted value as adjusted for gender, height and age at the Screening Visit.\n5. Male or female patients aged 18 years or greater (inclusive) and less than 85 years at the time of ALS diagnosis.\n6. Able to swallow liquid.\n7. Able to perform reproducible pulmonary function tests\n8. Female patients must be post-menopausal or sterilized or must not be breastfeeding, have no intention to become pregnant during the study, and use acceptable methods of contraception or abstain from intercourse.\n9. Male patients who have not had a vasectomy and confirmed zero sperm count must agree after receiving the first dose of study drug either to use acceptable methods of contraception or abstain from intercourse.\n10. If on riluzole, stable dosing for 30-days prior to screening.\n11. Pre-study ALSFRS-R progression between disease onset and screening of greater than or equal to 0.5 points/month (calculated by ALSFRS-R total score decline from 48 divided by the months since onset of ALS symptoms).\n\nExclusion Criteria:\n\n1. Use of non-invasive ventilation (NIV) support for ALS only or gastrostomy tube at time of screening.\n2. Exposure to investigational drug within 12-weeks prior to screening.\n3. At screening of any medically significant cardiac, pulmonary, GI, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or data.\n4. Patient with a history of significant other major medical conditions based on the Investigator's judgment.\n5. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.\n6. Any person who is an employee or an Investigator or Sponsor, or an immediate relative of an Investigator.", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Ambroxol", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05409508", "title": "Psychological Management by Meditation of Full COnscience in Virtual REality of People With Amyotrophic Lateral Sclerosis: Effects on Cognition, Behavior, Quality of Life and Psychological Well-being", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Angers", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that results in progressive paralysis of the muscles involved in voluntary motor skills, speech, swallowing and breathing. It also causes non-motor symptoms including psychological, cognitive and behavioral difficulties that have a negative impact on patients' quality of life, well-being and long-term development. There is no curative therapy for ALS and drug treatments have little effect on non-motor symptoms. Interventions based on mindfulness meditation, defined as a state of consciousness that arises when one decides to focus attention in the present moment without judgment on the real experience, seem to be a promising tool for the reduction of non-motor symptoms in a number of progressive neurological conditions (Alzheimer's disease, multiple sclerosis, etc.), suggesting that mindfulness significantly helps in the management of these symptoms. Our project therefore aims to implement a mindfulness meditation program adapted to the management of non-motor symptoms in ALS based on virtual reality (VR).", "interventions": [{"type": "OTHER", "name": "mindfulness meditation care"}, {"type": "OTHER", "name": "no mindfulness meditation care"}], "start_date": "2022-05", "url": "https://clinicaltrials.gov/study/NCT05409508", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Majors to inclusion\n* Mother tongue: French\n* Patients for whom ALS of bulbar or spinal form defined according to El Escorial criteria is possible, probable or certain\n* Able to carry out the investigations and interventions provided for in the protocol\n* Signature of informed consent to participate in the study\n\nExclusion Criteria:\n\n* Participation in intervention research modifying management\n* History likely to disrupt cognition (constituted stroke, sequelae of traumatic brain injury, active epilepsy, learning disabilities, alcohol dependence syndrome, drug use, psychiatric disorders), severe cognitive impairment (MMSE \\<24)\n* People who meet the diagnostic criteria for Frontotemporal Dementia\n* Pregnant or lactating women\n* Persons deprived of their liberty by administrative or judicial decision\n* Persons undergoing psychiatric care under duress\n* Persons subject to a legal protection measure\n* Persons unable to express their consent\n* Persons not affiliated or not beneficiaries of a social security scheme", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Mindfulness meditation delivered in virtual reality targets psychological, cognitive, and behavioral non-motor symptoms of ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03241784", "title": "Ph1 T-Regulatory Cells in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The Methodist Hospital Research Institute", "summary": "Open-label pilot study to determine the safety and tolerability of autologous CD4+ CD25+ regulatory T cells infusions with concomitant subcutaneous IL-2 injections taken 3 times per week in 3 participants with ALS.", "interventions": [{"type": "BIOLOGICAL", "name": "Autologous T-regulatory lymphocytes"}, {"type": "BIOLOGICAL", "name": "Interleukin-2"}], "start_date": "2016-05-16", "url": "https://clinicaltrials.gov/study/NCT03241784", "target_entities": ["immune_regulation", "IL2"], "locations": [{"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 18 years or older.\n2. Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria (Appendix 1).\n3. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days (riluzole-na\u00efve subjects are permitted in the study).\n4. Capable of providing informed consent and following trial procedures.\n5. Geographically accessible to the site.\n6. Women must not be able to become pregnant (e.g. post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for \u2265 3 months, barrier method in conjunction with spermicide, or another adequate method.\n7. Subjects must agree not to take live attenuated vaccines (including seasonal flu vaccine) 30 days before blood collection.\n8. Available autologous Tregs product with greater than or equal to 50% expression of CD4, CD25 and FoxP3 determined by flow-cytometry.\n9. Subjects must have been previously evaluated and followed clinically by a neuromuscular specialist at Houston Methodist Neurological Institute\n10. Normal Alanine aminotransferase level (ALT)\n11. Normal Serum creatinine level\n\nExclusion Criteria:\n\n1. Prior use of cells therapies\n2. Concurrent use of other experimental ALS therapies\n3. Pregnant or breastfeeding or planning to become pregnant or planning a partner's pregnancy.\n4. Other unstable medical or psychiatric illness\n5. Known immune deficiency or history of lymphoma or leukemia\n6. History of lymphopenia.\n7. History of acquired or inherited immune deficiency syndrome, including leukopenia.\n8. History of severe untreated chronic obstructive sleep apnea.\n9. FVC less than 50% predicted at screening.\n10. Exposure to any other agent currently under investigation for the treatment of subjects with ALS (off-label use or investigational) within 30 days of the Baseline Visit.\n11. The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to the PI's judgment, or a history of active substance abuse within the prior year.\n12. Clinically significant history of cardiac, oncologic, hepatic, or renal dysfunction, or other medically significant illness.\n13. The presence of any immunologic or autoimmune disease\n14. Severe cardiac dysfunction defined clinically, or as a left ventricular ejection fraction less than 40% of predicted or abnormal EKG findings.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous T-regulatory lymphocytes and Interleukin-2", "targeting_mechanism": "Regulatory T cells exert immunomodulatory and neuroprotective effects to attenuate neuroinflammation in ALS.", "targeting_mechanism_pmid": "32484719", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04165850", "title": "Open Label Study to Evaluate Ciprofloxacin/Celecoxib Combination in Patients With ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "NeuroSense Therapeutics Ltd.", "summary": "This is an open label, off label study, to provide interested ALS patients with Ciprofloxacin/Celecoxib fixed dose combination, while assessing safety and tolerability and routine disease progression measures (ALSFRS-R and Vital Capacity).", "interventions": [{"type": "DRUG", "name": "Fixed dose combination Ciprofloxacin/Celecoxib"}], "start_date": "2019-11-25", "url": "https://clinicaltrials.gov/study/NCT04165850", "target_entities": ["PTGS2", "neuroinflammation"], "locations": [{"facility": "Sourasky Medical Center", "city": "Tel Aviv", "state": "", "country": "Israel", "status": "", "lat": 32.08088, "lon": 34.78057}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to comprehend and willing to sign an Informed Consent Form (ICF)\n2. Males or females between the ages of 18 and 75 years of age, inclusive\n3. Diagnosis of familial or sporadic ALS (defined as meeting the laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria)\n4. Disease duration after first symptom less than 3 years prior to baseline\n5. Patients may be treated in parallel with Riluzole and/or Edaravone; 30 days of stable use prior to enrollment is required\n6. Upright forced vital capacity (FVC) \u2265 50% of predicted for age, height, weight and sex at screening\n7. Patient is able to swallow tablets/ capsules\n8. A caregiver (if one is needed)\n9. Female patients must be post-menopausal (\u2265 1 year) OR sterilized, OR if of childbearing potential (i.e., females who have had their first period unless they are anatomically or physiologically incapable to become pregnant), must have a negative pregnancy test, and agree to use contraceptive drugs or devices (e.g., diaphragm plus spermicide, or oral contraceptives) for the duration of the study and 10 weeks after the last treatment dose AND require male partners to use a condom during sexual intercourse\n\nExclusion Criteria:\n\n1. A past history of adverse reaction/hypersensitivity to either NSAIDs, celecoxib or fluoroquinolones, ciprofloxacin\n2. Any known clinically significant abnormal gastric mucosal erosion, ulcer or tumor or/and GI disorder\n3. Known history of clinically significant impairment of renal function (creatinine \u2265 1.5)\n4. Known or suspected congestive heart and/or coronary heart disease, previous history of myocardial infarction, uncontrolled arterial hypertension, or rhythm abnormalities requiring permanent treatment\n5. Known history of QT/QTc prolongation, Torsade de pointes (TdP) (e.g. heart failure, hypokalemia, family history of Long QT syndrome) and the use of concomitant medications that prolong the QT/QTc interval\n6. Known or suspected diagnosis or family history of epilepsy\n7. Presence at screening of any medically significant cardiac, pulmonary, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety data, including, but not limited to:\n\n   1. Mean systolic blood pressure \\>180 mm Hg; mean diastolic blood pressure \\>100 mm Hg (measurements taken after few min rest) that persist on 3 successive measurements taken at least 2 minutes apart\n   2. NYHA Class II or greater congestive heart failure\n   3. Chronic obstructive pulmonary disease or asthma requiring daily use of bronchodilator medications\n   4. Poorly controlled or brittle diabetes mellitus\n   5. Cognitive impairment, related to ALS or otherwise, sufficient to impair patient's ability to understand and/or comply with study procedures and provide informed consent\n8. Patient who is treated with chronic aspirin or NSAIDs, and is at risk if stopped. Clopidogrel is allowed and can replace Aspirin.\n9. Female who is pregnant or breastfeeding or with intention of becoming pregnant during the course of the study\n10. Any impairment or social circumstance that, in the opinion of the Investigator, would render the patient not suitable to participate in the study\n11. Patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study\n12. Patient is participating in (or plans to participate in) any other investigational drug trial, or plans to be exposed to any other investigational agent, device and/or procedure, from 30 days prior to Screening through study completion", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Ciprofloxacin/Celecoxib fixed dose combination", "targeting_mechanism": "Combination targeting neuroinflammation through antibiotic and anti-inflammatory mechanisms to address persistent CNS inflammation in ALS.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03103815", "title": "Trial of Amivita in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Wujin People's Hospital", "summary": "The primary objectives of this study are to determine the safety and efficacy of Amivita, a compound of amino acids and vitamines in patients with Amyotrophic lateral sclerosis (ALS)ALS. The secondary objectives are to measure quality of life before and during intervention. This is a self-controlled clinical trial. Twenty patients in our ALS center who are already receiving riluzole or other treatments but the condition is worsening will receive treatment for 1o months. The evaluating investigators will be blinded to treatment assignment. Primary outcome measures will be adverse events, the ALS Functional Rating Scale-Revised (ALSFRS-R), and survival. Subjects will also be assessed at enrollment and at study end for weight loss, forced vital capacity (FVC), quality of life and grip strength.", "interventions": [{"type": "DRUG", "name": "Amivita"}], "start_date": "2017-04-24", "url": "https://clinicaltrials.gov/study/NCT03103815", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Wujing People's Hospital", "city": "Changzhou", "state": "Jiangsu", "country": "China", "status": "RECRUITING", "lat": 31.77359, "lon": 119.95401}], "contact_phone": "86-519-85579128", "contact_email": "513325835@qq.com", "eligibility": {"criteria": "Inclusion Criteria\n\n* Patients must be men or women between the ages of 18 and 70 years\n* Patient is clinical definite or probable ALS by the hospitals listed in the protocol\n* Women who are of child bearing potential must have a negative pregnancy test\n* Willing to comply with the study visits\n* Will not take riluzole during the study period\n* Be able to sign informed consent document\n\nExclusion Criteria\n\n* Myotonic dystrophy\n* Myasthenia gravis\n* Post-poliomyelitis syndrome\n* Multifocal motor neuropathy with or without conduction block\n* Hirayama disease\n* Kennedy disease\n* Hereditary spastic paraplegia\n* Syringomyelia\n* Spinal cord and brain stem tumors\n* Paraneoplastic syndromes\n* Severe liver or kidney disease disease\n* Infection, severe diarrhea or vomiting\n* Serious heart or lung diseases or malignant tumor history\n* HIV infection\n* Pregnancy or breastfeeding\n* Have no ability to communicate\n* Have participated in other clinical trials within 4 weeks\n* Any form of substance abuse, psychiatric disorder, or other condition that, in opinion of the investigator, may interfere with the study", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Amivita", "targeting_mechanism": "Amino acids and vitamins combination addressing oxidative stress and antioxidant system dysfunction in ALS.", "targeting_mechanism_pmid": "34663413", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03843710", "title": "31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Amyotrophic Lateral Sclerosis (REPAIR-ALS)", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Clene Nanomedicine", "summary": "REPAIR-ALS is a single-center open label pilot, sequential group, investigator and patient blinded study to assess the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been diagnosed with Amyotrophic Lateral Sclerosis (ALS) within twelve (12) months of Screening. The primary endpoint is the ratio of the oxidized to reduced form of nicotinamide adenine dinucleotide (NAD+:NADH) measured non-invasively by 31phosphorous magnetic resonance spectroscopy (31P-MRS).", "interventions": [{"type": "DRUG", "name": "Gold Nanocrystals"}], "start_date": "2020-03", "url": "https://clinicaltrials.gov/study/NCT03843710", "target_entities": ["oxidative_stress", "neuronal_redox_state"], "locations": [{"facility": "UT Southwestern", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to understand and give written informed consent.\n2. Male or female patients aged 35 years or greater (inclusive) and less than 75 years of age at the time of ALS diagnosis.\n3. Patients with a confirmed ALS diagnosis: \"definite ALS\" or \"probable ALS\" or \"possible\" diagnostic criteria per the revised El Escorial Criteria as determined by a neurologist subspecializing in ALS (e.g., the Principal Investigator by study site).\n4. Stable background therapy (e.g., stable dosing of riluzole within the prior 6-weeks) per Investigator discretion.\n5. At the time of Screening disease duration less than or equal to 24-months from symptom onset OR within 12-moths of a confirmed ALS diagnosis.\n6. Forced vital capacity (FVC) \\>/= 60% of predicted value as adjusted for gender, height, and age at the Screening Visit.\n7. Patients who are ambulatory (e.g., normal ambulation, early ambulation difficulties, or walks with assistance) on the ALSFRS-R scale.\n\nExclusion Criteria:\n\n1. At Screening patients who utilize, or in the Investigator's judgment will be imminently dependent upon during the course of this study:\n\n   1. Non-invasive ventilation\n   2. Gastrostomy (e.g., use of percutaneous endoscopic gastrostomy tube)\n   3. Use of wheel chair\n2. Patient who have previously undergone tracheostomy.\n3. Patient with a history of significant other major medical condition based on the Investigator's judgment.\n4. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or study procedures.\n5. Patient with clinically significant abnormalities in hematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with study participation.\n6. Patient participating in any other investigational drug trial or using investigational drug (within 12 weeks prior to screening and thereafter)\n7. Females who are pregnant or nursing or who plan to get pregnant during the course of this clinical trial or within 6 months of the end of this trial.\n8. Positive screen for drugs of abuse or known alcohol abuse.\n9. Women of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control during the study or for 6 months following completion of study participation.\n10. Women with a positive pregnancy test, are lactating, or are planning to become pregnant during the study.\n11. Patients with implanted metal objects in their body that may be affected by an MRI procedure.\n12. Patients who are claustrophobic or otherwise unlikely to be able to complete the MRI scanning procedures.\n13. Patients with a history of gold allergy.", "sex": "ALL", "min_age": "35 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Gold Nanocrystals (CNM-Au8)", "targeting_mechanism": "Gold nanocrystals restore impaired neuronal redox state by targeting NAD+:NADH ratio imbalance and oxidative stress in ALS.", "targeting_mechanism_pmid": "34198557", "animal_results": "ASC-Exosomes delayed disease progression in SOD1(G93A) mice, demonstrating the feasibility of targeting oxidative stress in ALS models.", "animal_results_pmid": "32455791", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05039268", "title": "3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Macquarie University, Australia", "summary": "Phase 2 open label trial to investigate the safety and potentially efficacy of 3K3A-APC in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "3K3A-APC Protein"}], "start_date": "2021-11-25", "url": "https://clinicaltrials.gov/study/NCT05039268", "target_entities": ["PAR1", "neuroprotection"], "locations": [{"facility": "Macquarie University", "city": "Macquarie Park", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.78105, "lon": 151.12757}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patients must have clinically definite ALS (Awaji Criteria)\n2. Male or female age 18 years and less than 75 years at time of ALS study\n3. Symptom onset less than 36 months before screening\n4. Diagnosis of ALS less than 24 months before screening\n5. Clinically definite Upper Motor Neuron signs\n\nExclusion Criteria:\n\n1. Current treatment with anticoagulants (e.g., warfarin, novel oral anticoagulants, heparin) that might preclude safe completion of the lumbar puncture\n2. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia\n3. Use of investigational drugs or devices within 60 days prior to Baseline (dietary supplements taken outside of a clinical trial are not exclusionary, e.g., coenzyme Q10)\n4. Prolonged prothrombin time or activated partial thromboplastin time \\>2xULN\n5. Severe hypertension or hypotension\n6. Glomerular filtration rate (GFR) \\<35 mL/min\n7. Forced vital capacity (FVC) at screening of \\<50% of predicted\n8. Prior exposure to any exogenous form of APC\n9. Inability to lie flat for procedures (MRI, PET, LP)\n10. Pregnant or lactating during the study period", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "3K3A-APC Protein", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04569435", "title": "Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Annexon, Inc.", "summary": "This study is a multi-center, open-label study of intravenous (IV) ANX005 in participants with ALS.", "interventions": [{"type": "DRUG", "name": "ANX005"}], "start_date": "2021-01-15", "url": "https://clinicaltrials.gov/study/NCT04569435", "target_entities": ["C3"], "locations": [{"facility": "Annexon Investigational Site 04", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Annexon Investigational Site 01", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Annexon Investigational Site 02", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Annexon Investigational Site 03", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Annexon Investigational Site 10", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Annexon Investigational Site 09", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Annexon Investigational Site 07", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Annexon Investigational Site 08", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Diagnosis of ALS according to the World Federation of Neurology revised EI Escorial criteria.\n* Onset of weakness within 3 years prior to Day 1 visit.\n* Slow Vital Capacity \u2265 50% of predicted normal adjusted for sex, age, and height (from the sitting position).\n* ALS Functional Rating Scale-Revised (ALSFRS-R) \u2265 30 at the Screening visit (Week -2).\n* If female, must be postmenopausal, surgically sterilized, or childbearing potential must agree to use highly effective methods of contraception from Screening until 3 months after the last infusion with study medication.\n* Males with a woman partner of childbearing potential must agree to use highly effective methods of contraception from Screening until Week until 3 months after the last infusion with study medication.\n* Documented history of vaccinations within 5 years prior to Screening visit against encapsulated bacterial pathogens or willing to undergo vaccinations.\n\nKey Exclusion Criteria:\n\n* Clinically significant intercurrent illness, medical condition, or medical history that would jeopardize the safety of the participant, limit participation, or compromise the interpretation of the data derived from the participant.\n* Participants with body weight \\> 150 kilograms.\n* Antinuclear antibodies (ANA) titer \u2265 1:160 (for either of the 2 ANA results a minimum of 2 weeks apart) during the Screening Period.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ANX005", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00349622", "title": "Clinical Trial Ceftriaxone in Subjects With ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "The purpose of the study is to evaluate the safety and efficacy of ceftriaxone treatment in amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "ceftriaxone"}, {"type": "OTHER", "name": "placebo"}], "start_date": "2006-07", "url": "https://clinicaltrials.gov/study/NCT00349622", "target_entities": ["glutamate_excitotoxicity"], "locations": [{"facility": "Phoenix Neurological Associates", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California, Davis", "city": "Davis", "state": "California", "country": "United States", "status": "", "lat": 38.54491, "lon": -121.74052}, {"facility": "University of California, San Francisco- Fresno", "city": "Fresno", "state": "California", "country": "United States", "status": "", "lat": 36.74773, "lon": -119.77237}, {"facility": "Loma Linda University School of Medicine (CA)", "city": "Loma Linda", "state": "California", "country": "United States", "status": "", "lat": 34.04835, "lon": -117.26115}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California, Los Angeles", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California, Irvine - MDA ALS Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of California, San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Health Sciences Center", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "George Washington University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Mayo Clinic Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "ALS Center at Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Medical College of Georgia", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Northwestern University Medical School", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Indiana University (Regenstrief Health Center)", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Kentucky Medical Center", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Lahey Clinic", "city": "Burlington", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.50482, "lon": -71.19561}, {"facility": "Henry Ford Health System", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Saint Mary's Healthcare", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "Hennepin County Medical Center (Berman Center)", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "St. Louis University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Bryan LGH Medical Center (University of Nebraska)", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "UMDNJ- Robert Wood Johnson School of Medicine", "city": "New Brunswick", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.48622, "lon": -74.45182}, {"facility": "Albany Medical Center", "city": "Albany", "state": "New York", "country": "United States", "status": "", "lat": 42.65258, "lon": -73.75623}, {"facility": "Beth Israel Medical Center (NY)", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Cornell Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Wake Forest University School of Medicine", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Oregon Clinic (Providence Clinic)", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Pennsylvania State University, Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University College of Medicine (Hahnemann Campus)", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Allegheny Hospital", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "University of Pittsburgh", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Medical University of South Carolina", "city": "Charleston", "state": "South Carolina", "country": "United States", "status": "", "lat": 32.77632, "lon": -79.93275}, {"facility": "Vanderbilt University", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Utah Health Sciences Center", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Virginia", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Calgary", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Univeristy of Alberta ALS Clinic", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Dalhousie University", "city": "Halifax", "state": "Nova Scotia", "country": "Canada", "status": "", "lat": 44.64269, "lon": -63.57688}, {"facility": "London Health Sciences Center, University Campus", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "University of Toronto", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "CHUM (Centre Hospitalier de l'Universit\u00e9 de Montr\u00e9al), Notre-Dame Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute (McGill University)", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Laval University", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Participants will be people with ALS, at least 18 years of age.\n* Participants must be medically able to undergo the study procedures and have a caregiver or other individual who will be available to help with daily study medication administration.\n* Participants should live within a reasonable distance of the study site, due to frequent study visits.\n\nExclusion Criteria:\n\n* Participants cannot be taking any other experimental medications for ALS, or have a history of sensitivity to cephalosporin antibiotics (such as Ancef, Keflex, Ceclor, Ceftin, Lorabid, Suprax, or Fortaz).", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ceftriaxone", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04579666", "title": "MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Apellis Pharmaceuticals, Inc.", "summary": "This is a 24-month, Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS)", "interventions": [{"type": "DRUG", "name": "Pegcetacoplan (APL-2)"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2020-09-30", "url": "https://clinicaltrials.gov/study/NCT04579666", "target_entities": ["complement_pathway"], "locations": [{"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of Colorado", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Augusta University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Indiana University", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "Johns Hopkins", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "The Berman Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "University of Vermont Medical Center", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "Brain and Mind Centre", "city": "Camperdown", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.88965, "lon": 151.17642}, {"facility": "Central Coast Neurosciences Research", "city": "Erina", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.43218, "lon": 151.38972}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Gold Coast University Hospital", "city": "Southport", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.96724, "lon": 153.39796}, {"facility": "Nueor-Immunology Clinical Researh Education and Support Service (N-CRESS), Austin Health", "city": "Heidelberg", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.75, "lon": 145.06667}, {"facility": "AZ Sint-Lucas & Volkskliniek", "city": "Ghent", "state": "", "country": "Belgium", "status": "", "lat": 51.05, "lon": 3.71667}, {"facility": "Universitaire Ziekenhuizen Leuven (UZ Leuven)", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Vseobecna fakultni nemocnice v Praze", "city": "Prague", "state": "", "country": "Czechia", "status": "", "lat": 50.08804, "lon": 14.42076}, {"facility": "FORBELI s.r.o.", "city": "Prague", "state": "", "country": "Czechia", "status": "", "lat": 50.08804, "lon": 14.42076}, {"facility": "Hopital Pellegrin", "city": "Bordeaux", "state": "", "country": "France", "status": "", "lat": 44.84124, "lon": -0.58046}, {"facility": "H\u00f4pital Neurologique Pierre Wertheimer", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "CHU Gabriel Montpied", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "", "lat": 45.77969, "lon": 3.08682}, {"facility": "H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges Dupuytren 1", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "CHU de Nice H\u00f4pital Pasteur", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Charit\u00e9 - Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Medizinische Hochschule Hannover Klinik f\u00fcr Neurologie", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4tsklinikum Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Universit\u00e4tsmedizin Rostock, Klinik und Poliklinik f\u00fcr Neurologie", "city": "Rostock", "state": "", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "University of Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Ospedale Niguarda - Nemo Clinical Center - Fondazione Serena Onlus", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Ospedale Civile S. Agostino Estense di Modena, Azienda Ospedaliero Universitaria di Modena", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "AOUP \"P. Giaccone\"", "city": "Palermo", "state": "", "country": "Italy", "status": "", "lat": 38.1166, "lon": 13.3636}, {"facility": "Azienda Ospedaliera Universitaria di Torino - Citt\u00e0 della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "National Hospital Organization Higashinagoya National Hospital", "city": "Aichi", "state": "", "country": "Japan", "status": "", "lat": 32.51879, "lon": 130.62158}, {"facility": "National Hospital Organization Omuta National Hospital", "city": "Fukuoka", "state": "", "country": "Japan", "status": "", "lat": 33.6, "lon": 130.41667}, {"facility": "National Hospital Organization Asahikawa Medical Center", "city": "Hokkaido", "state": "", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "National Hospital Organization Hyogo-Chuo National Hospital", "city": "Hy\u014dgo", "state": "", "country": "Japan", "status": "", "lat": 43.36667, "lon": 144.43333}, {"facility": "National Hospital Organization Iou National Hospital", "city": "Ishikawa", "state": "", "country": "Japan", "status": "", "lat": 26.42333, "lon": 127.82139}, {"facility": "National Hospital Organization Matsumoto Medical Center", "city": "Matsumoto", "state": "", "country": "Japan", "status": "", "lat": 36.23333, "lon": 137.96667}, {"facility": "Niigata National Hospital National Hospital Organization", "city": "Niigata", "state": "", "country": "Japan", "status": "", "lat": 37.92259, "lon": 139.04125}, {"facility": "National Hospital Organization Okinawa National Hospital", "city": "Okinawa", "state": "", "country": "Japan", "status": "", "lat": 26.33583, "lon": 127.80139}, {"facility": "National Hospital Organization Higashisaitama National Hospital", "city": "Saitama", "state": "", "country": "Japan", "status": "", "lat": 35.90807, "lon": 139.65657}, {"facility": "Shizuoka Institute of Epilepsy and Neurological Disorders", "city": "Shizuoka", "state": "", "country": "Japan", "status": "", "lat": 34.98333, "lon": 138.38333}, {"facility": "Juntendo University Hospital", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "Tokyo Medical University Hospital", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Uniwersytecki Szpital Kliniczny w Olsztynie Klinika Neurologii", "city": "Olsztyn", "state": "", "country": "Poland", "status": "", "lat": 53.78376, "lon": 20.49272}, {"facility": "Centrum Medyczne NeuroProtect", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "City Clinic Sp. z o.o.", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Hospital Universitari Vall d'Hebron", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Bellvitge University Hospital", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitari I Politecnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "SI Institute of Neurology, Psychiatry and Narcology of NAMSU", "city": "Kharkiv", "state": "", "country": "Ukraine", "status": "", "lat": 49.98177, "lon": 36.25475}, {"facility": "Centre of Reconstructive and Restorative Medicine (University Clinic) Odessa National Medical University", "city": "Odesa", "state": "", "country": "Ukraine", "status": "", "lat": 46.48572, "lon": 30.74383}, {"facility": "Zaporizhzhya Regional Clinical Hospital", "city": "Zaporizhzhya", "state": "", "country": "Ukraine", "status": "", "lat": 47.85167, "lon": 35.11714}, {"facility": "University Hospitals Sussex NHS Foundation Trust", "city": "Brighton", "state": "", "country": "United Kingdom", "status": "", "lat": 50.82838, "lon": -0.13947}, {"facility": "Maurice Wohl Clinical Neuroscience Institute, King's College London", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "St George's University Hospitals NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* At least 18 years of age\n* Sporadic ALS diagnosed as definite, probable, or laboratory-supported probable as defined by the revised El Escorial criteria\n* Slow vital capacity (SVC) \u226560% of the predicted value at screening\n* Onset of ALS symptoms within 72 weeks (18 months) prior to screening\n* Total ALSFRS-R score of \u226530 at screening\n* Have vaccination within 5 years against Streptococcus pneumoniae, Neisseria meningitidis (types A, C, W, Y, and B), and Haemophilus influenzae (type B) or agree to receive vaccination\n\nExclusion Criteria:\n\n* Confirmed or suspected other causes of neuromuscular weakness\n* Diagnosed with another neurodegenerative disease (examples include Parkinson's disease and Huntington's disease)\n* Significant pulmonary disorder not attributed to ALS (eg, chronic obstructive pulmonary disease, pulmonary fibrosis, cystic fibrosis, pulmonary arterial hypertension)\n* If taking riluzole, participant must be on a stable dose for 30 days prior to the start of the screening period. Use of riluzole is not required for participation.\n* If taking edaravone, participant must be on a stable dose for 60 days prior to the start of the screening period. Use of edaravone is not required for participation.\n* Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or within 5-half lives of the treatment (whichever is longer) prior to the start of the screening period or during study participation\n* Use of any other complement inhibitor within 30 days or within 5-half lives of the treatment (whichever is longer) prior to the start of the screening period or during study participation", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Pegcetacoplan (APL-2)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03851302", "title": "Effects of Remote Ischemic Conditioning on Hand Use in Individuals With SCI and ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Bronx VA Medical Center", "summary": "Rehabilitation interventions such as physical training and neural stimulation after spinal cord injury (SCI) have been shown to increase neural plasticity. However, both physical training and neural stimulation require a large number of repetitions, and the retention of the intervention effects may be fleeting. In this proposal the investigators will test Remote ischemic conditioning (RIC), which has been shown to promote neural plasticity and has practical and theoretical advantages. RIC consists of transiently restricting blood flow to any 'remote' limb using a blood pressure cuff. This induces several of the body's systemic defensive reactions. RIC has been shown to improve motor learning. The investigators propose that RIC alters motor pathway excitability through a combination of systemic increases in plasticity-promoting factors and inhibition of inflammatory factors. The investigators have designed a clinical trial to test this hypothesis in 8 persons with SCI and 8 able-bodied controls. All participants will receive active/sham RIC plus a hand exercise. The investigators will measure effects on blood pressure, motor neuron excitability, and systemic inflammatory markers before and after RIC as well as after hand exercise. Starting July 2021, we will also enroll 5 individuals with Amyotrophic lateral sclerosis (ALS) in this study.", "interventions": [{"type": "OTHER", "name": "Active Remote Ischemic Conditioning"}, {"type": "OTHER", "name": "Sham Remote Ischemic Conditioning"}, {"type": "OTHER", "name": "Isometric hand exercise"}], "start_date": "2019-10-28", "url": "https://clinicaltrials.gov/study/NCT03851302", "target_entities": [], "locations": [{"facility": "James J. Peters VA Medical Center", "city": "The Bronx", "state": "New York", "country": "United States", "status": "", "lat": 40.84985, "lon": -73.86641}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Able-bodied participants\n\n1. Age between 18 and 75 years;\n2. No known central or peripheral neurological disease or injury.\n\nSCI participants\n\nInclusion Criteria:\n\n1. Age between 18 and 75 years;\n2. Chronic (more than 12 months since injury) motor-incomplete SCI between neurological levels C2-C8\n3. Detectable F-wave responses of the left or right abductor pollicis brevis (APB) to median nerve stimulation;\n4. Detectable motor evoked potentials in left or right APB muscles to transcranial magnetic stimulation;\n5. Able to perform thumb-middle finger opposition pinch task with detectable APB EMG muscle activity.\n\nALS participants\n\n1. Age between 21 and 75 years;\n2. Diagnosis of probable or definite ALS.\n3. Incomplete weakness of left or right wrist or hand muscles: score of 2, 3, or 4 (out of 5) on manual muscle testing of finger extension, finger flexion, or finger abduction.\n4. Detectable motor evoked potentials in left or right APB muscles to transcranial magnetic stimulation;\n5. Able to perform thumb-middle finger opposition pinch task with detectable APB electromyography (EMG) muscle activity.\n\nExclusion Criteria:\n\n1. Multiple spinal cord lesions;\n2. History of seizures;\n3. Use of medications that significantly lower seizure threshold, such as amphetamines and bupropion;\n4. History of implanted brain/spine/nerve stimulators, aneurysm clips, or cardiac pacemaker/defibrillator;\n5. Any extremity soft tissue, orthopedic, or vascular condition or injury that may contraindicate remote limb ischemic conditioning (RLIC) (uncontrolled hypertension, peripheral vascular disease, hematological disease, severe hepatic or renal dysfunction);\n6. Any other contraindication to undergoing magnetic resonance imaging (except for claustrophobia);\n7. Clinically significant infection of any kind (urinary tract, pulmonary, skin or other)\n8. Significant coronary artery or cardiac conduction disease;\n9. Open skin lesions over the neck, shoulders, or arms;\n10. Pregnancy\n11. Unsuitable for study participation as determined by study physician. In addition, a medical record review will be conducted to identify any other medical concerns that might increase the risks associated with participation.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Remote Ischemic Conditioning (RIC)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07606235", "title": "Transcutaneous Superior Laryngeal Nerve Stimulation to Upregulate Swallowing Frequency and Urge to Swallow in Patients Living With ALS", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Nova Southeastern University", "summary": "Prospective, single-arm pilot study evaluating transcutaneous electrical nerve stimulation of the superior laryngeal nerve (TENS-SLN) in individuals with ALS.\n\nUp to 5 participants with confirmed ALS and bulbar involvement will be enrolled.\n\nPrimary outcomes: swallowing frequency and perceived urge to swallow. Participants complete a baseline visit, followed by two supervised treatment sessions within one week, and a final post-treatment evaluation.\n\nOptional visits (up to 2) may be used to individualize stimulation parameters prior to treatment.\n\nSwallowing function will be assessed using physiological monitoring, fiberoptic endoscopy, and videofluoroscopic swallow study (VFSS).\n\nTENS-SLN is a non-invasive neuromodulation approach targeting sensory pathways to facilitate swallowing without inducing muscle contraction.\n\nThis pilot study is designed to assess feasibility, safety, and preliminary effect sizes to inform future randomized trials, and is not powered to determine efficacy.", "interventions": [{"type": "OTHER", "name": "Transcutaneous Superior Laryngeal Nerve Stimulation to Upregulate Swallowing Frequency and Urge to Swallow"}], "start_date": "2026-06", "url": "https://clinicaltrials.gov/study/NCT07606235", "target_entities": [], "locations": [], "contact_phone": "(954) 262-1271", "contact_email": "rw602@nova.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Confirmed diagnosis of ALS (El-Escorial Criteria)\n* ALS FRS Bulbar Subscore of \u2264 10 and \u2265 3.\n* Functional Oral Intake scale score \u2265 2\n\nExclusion Criteria:\n\n* A diagnosis of significant cognitive impairment or frontotemporal dementia per the treating neurologist or neuropsychologist,\n* Current nasogastric tube placement\n* Current head and neck carcinoma\n* Pacemaker or implanted defibrillator or history of diagnosed arrhythmia, bradycardia, or repeated attacks of hypotension,\n* Implanted vagal nerve stimulator\n* Current pregnancy\n* History of epilepsy\n* Infected, broken or inflamed skin on the neck, or impaired sensation at the site of sEMG or TENS placement\n* Living greater than 50 miles from the NSU clinic round trip\n* Any clinical reason that this stimulation may not be a suitable treatment according to the principal investigator.", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06765564", "title": "Clinical Study of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell Therapy for Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shanghai East Hospital", "summary": "Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disease in the human motor system characterized by the selective involvement of spinal cord anterior horn cells, brainstem motor nuclei, and the corticospinal tract. It predominantly presents as concurrent damage to upper and lower motor neurons.\n\nInduced pluripotent stem cells (iPSCs) are a type of induced pluripotent stem cell derived from autologous or allogeneic cell sources. They can differentiate into various functional cell types, including specific motor neuron cells. iPSCs are used for stem cell replacement therapy. iPSCs hold significant clinical potential for ALS treatment. The iPSC database with human leukocyte antigen characteristics may represent a promising technology. This technology has the potential to obtain high-quality cell products and reduce the risk of graft rejection. Moreover, human iPSCs have demonstrated a certain degree of efficacy in the transplantation of neural stem/progenitor cells derived from ALS rodent models.\n\nThe potential mechanisms of iPSC therapy for ALS include: the differentiated motor neuron precursor cells can replace damaged motor neurons, and restore motor conduction function; by secreting neurotrophic factors, they protect neurons; through immune regulation, they inhibit inflammatory reactions, and slow the progression of ALS.\n\nXellsmart Biomedical (Suzhou) Co., Ltd. is developing an injectable solution for ALS treatment using human iPSC-derived motor neuron precursor cells to address the pressing need for ALS therapy.", "interventions": [{"type": "DRUG", "name": "iPSC-MNP"}], "start_date": "2024-03-13", "url": "https://clinicaltrials.gov/study/NCT06765564", "target_entities": ["motor_neuron_regeneration"], "locations": [{"facility": "Shang hai East Hospital", "city": "Shanghai", "state": "Shanghai Municipality", "country": "China", "status": "RECRUITING", "lat": 31.22222, "lon": 121.45806}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patients themselves or their legal guardians must consent to undergo this treatment protocol and sign the Informed Consent Form (ICF).\n2. Age between 18 and 60 years, inclusive, with no gender restrictions.\n3. Diagnosed with ALS according to the World Federation of Neurology criteria, and the initial diagnosis date is between 6 to 24 months before the screening date.\n4. Patients who have received standard treatment in the past with poor efficacy or disease progression.\n5. Forced Vital Capacity (FVC) should be \u226550%.\n6. During any night of the screening period, the total time with peripheral blood oxygen saturation \\<90% should not exceed 2%.\n7. Patients should be deemed by the investigator to be in good nutritional status, with a Body Mass Index (BMI) \u226518.5.\n8. Male patients and their spouses, as well as women of childbearing age, should agree to implement effective contraceptive measures from the time of signing the ICF until one year after the start of treatment.\n9. Patients should be able to cooperate in the collection and preservation of medical history data and the visit process.\n\nExclusion Criteria:\n\n1. Patients with symptoms of neuromuscular weakness but cannot be conclusively determined to have ALS.\n2. Patients diagnosed with severe cognitive impairment, clinical dementia, or major psychiatric disorders, including but not limited to schizophrenia, bipolar disorder, or severe depression, according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V).\n3. Patients with any disease that impairs nerve or muscle function, such as peripheral neuropathy or metabolic myopathy.\n4. Patients with a history of malignant tumors or a previous diagnosis of malignancy.\n5. Within the two weeks preceding the screening period, patients who experienced acute active infections requiring treatment with antibiotics, antiviral drugs, or antifungal medications.\n6. ALS patients with concomitant respiratory failure.\n7. Patients who have previously undergone any allogeneic cell therapy or organ transplantation.\n8. Patients who have Participated in other clinical trials within the three months prior to screening.\n9. Patients with a history of tracheostomy or those using mechanical ventilatory support.\n10. Patients with a documented history of severe allergic reactions to general anesthesia drugs or previous severe allergic reactions for other reasons.\n11. Patients with intracranial organic diseases causing increased intracranial pressure.\n12. Patients with elevated liver function test results during the screening period, such as total bilirubin \\>1.5 times the upper limit of normal (ULN), or aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \\>3 times ULN.\n13. Patients who have abnormal kidney function test results during the screening period, such as serum creatinine \\>1.5 mg/dL or an estimated creatinine clearance rate \\<60 mL/min calculated by the Cockcroft and Gault formula.\n14. Other clinically significant laboratory abnormalities during the screening period.\n15. Patients with hepatitis A, active hepatitis B (HBsAg positive and HBV DNA \u2265500 IU/ml, excluding drug- or other-caused hepatitis), active hepatitis C (anti-HCV antibody positive and HCV RNA positive), hepatitis E, human immunodeficiency virus (HIV) antibody positive, or syphilis treponemal antibody positive.\n16. Patients with impaired consciousness.\n17. Coagulation abnormalities (prothrombin time \\[PT\\] or international normalized ratio \\[INR\\] \\>1.5 times ULN; activated partial thromboplastin time \\[APTT\\] \\>1.5 times ULN) or those currently receiving anticoagulation therapy.\n18. Poorly controlled hypertension, with systolic blood pressure \\>160 mmHg and/or diastolic blood pressure \\>100 mmHg after treatment.\n19. Severe diabetes with late complications; patients with other diseases affecting limb mobility (e.g., limping, osteoarthritis, rheumatoid arthritis, gout, etc.).\n20. Patients who have undergone surgery or experienced trauma (including fractures) in the past month.\n21. Pregnant or breastfeeding women.\n22. Patients who, in the opinion of the investigator, have poorly controlled systemic diseases or other conditions that make them unsuitable for participation in this clinical study.", "sex": "ALL", "min_age": "18 Years", "max_age": "60 Years", "healthy_volunteers": false, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "iPSC-MNP (induced pluripotent stem cell-derived motor neuron precursor cells)", "targeting_mechanism": "Differentiation of induced pluripotent stem cells into motor neuron precursor cells to replace degenerating motor neurons in ALS.", "targeting_mechanism_pmid": "29789581", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01759784", "title": "Intraventricular Transplantation of Mesenchymal Stem Cell in Patients With ALS", "phase": "PHASE1", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Royan Institute", "summary": "ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). ALS is the most common of the five motor neuron diseases.Riluzole (Rilutek) is the only treatment that has been found to improve survival but only to a modest extent. It lengthens survival by several months, and may have a greater survival benefit for those with a bulbar onset. It also extends the time before a person needs ventilation support.Stem cell transplantation is a new hopeful way to improve the patients conditions and reduce the period of disabilities.", "interventions": [{"type": "BIOLOGICAL", "name": "Intraventricular injection"}], "start_date": "2014-03", "url": "https://clinicaltrials.gov/study/NCT01759784", "target_entities": ["motor_neuron_regeneration"], "locations": [{"facility": "Royan Institute", "city": "Tehran", "state": "", "country": "Iran", "status": "", "lat": 35.69439, "lon": 51.42151}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age:18-65\n* both gender\n* duration of disease\\<2 years\n* FVC\\>40% ALS-FRS\\>26\n\nExclusion Criteria:\n\n* neurological and psychiatric concomitant disease\n* concomitant systemic disease\n* treatment with corticosteroid,Ig,immunosuppressive during 12 months.", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Mesenchymal stem cells (MSC)", "targeting_mechanism": "Intraventricular transplantation of mesenchymal stem cells to provide neuroprotective support and slow motor neuron degeneration in ALS.", "targeting_mechanism_pmid": "", "animal_results": "Intravenous injection of bone marrow-derived mesenchymal stem cells expressing neurogenin-1 (Ngn1) in SOD1G93A mice increased lifespan by 3 days, delayed disease onset by 5 days, and reduced motor neuron loss, though cells were poorly distributed to the CNS.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00925847", "title": "Effect of Lithium Carbonate in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "The purpose of the study is to determine whether lithium is safe and effective in the treatment of ALS", "interventions": [{"type": "DRUG", "name": "lithium"}], "start_date": "2009-06", "url": "https://clinicaltrials.gov/study/NCT00925847", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Piti\u00e9-Salp\u00eatri\u00e8re Hospital", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of clinically possible, clinically probable laboratory-supported, clinically probable or clinically definite ALS (according to WNF EL Escorial diagnostic criteria, revised according to the AIRLIE House Conference 1998)\n* Concomitant standard Riluzole therapy (50mg twice daily)\n* patients included in ALS reference center\n* women of childbearing age be non-lactating and surgically sterile or using a highly effective method of birth control and have a negative pregnancy test\n* capable of thoroughly understanding all information given and giving full informed consent according to GCP\n* Patients with gastrostomy\n\nExclusion Criteria:\n\n* evidence of major psychiatric disorder or clinically evident dementia precluding evaluation of symptoms\n* any medical condition known to contre-indicate lithium treatment (dysthyroid, cardiopathy, renal insufficiency)\n* presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment\n* known hypersensitivity to any component of the study drugs", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "lithium carbonate", "targeting_mechanism": "Lithium modulates glycogen synthase kinase-3 (GSK-3) and other signaling pathways to reduce neuroinflammation and support motor neuron survival.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06344260", "title": "Neural Stem Cell Treatment for Amyotrophic Lateral Sclerosis (STEMALS)", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Casa Sollievo della Sofferenza IRCCS", "summary": "A Not for Profit Phase II Study to Evaluate Safety, Efficacy and Biomarkers secondary endpoints of Human Neural Stem cell intracerebroventricular transplantation in amyotrophic lateral sclerosis patients: a randomized, placebo controlled, triple blind study.\n\nThis is an approximate 24-months study (PHASE B) consisting, per patient, of a 30-day screening period, 12-month enrollment and follow up period. A preliminary 3+3 dose-escalation open-label phase (PHASE A) will be performed in order to test the toxicity of the two proposed cell doses. The study will be stopped when all the subjects included in the treatment period complete the study visits. The study uses an ATMP, for that reason all the patients follow up will be prosecuted long life.", "interventions": [{"type": "PROCEDURE", "name": "human Neural Stem Cells (hNSC)"}, {"type": "PROCEDURE", "name": "Saline (Placebo)"}], "start_date": "2024-01-25", "url": "https://clinicaltrials.gov/study/NCT06344260", "target_entities": ["motor_neuron_regeneration"], "locations": [{"facility": "Casa Sollievo Della Sofferenza IRCCS", "city": "San Giovanni Rotondo", "state": "Foggia", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 41.70643, "lon": 15.7277}, {"facility": "Centro SLA Azienda Ospedaliera Universit\u00e0 Maggiore della Carit\u00e0", "city": "Novara", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.44694, "lon": 8.62118}, {"facility": "Azienda Ospedaliera di Padova", "city": "Padua", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 45.40797, "lon": 11.88586}, {"facility": "Azienza Ospedaliera Universitaria - Policlinico \"P. Giaccone\" Universit\u00e0 degli Studi di Palermo", "city": "Palermo", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 38.1166, "lon": 13.3636}], "contact_phone": "+390882835928", "contact_email": "m.carella@operapadrepio.it", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patient provides written informed consent, informed consent signature collection prior to any study procedure (patient has good acceptance and understanding of the informed consent);\n2. Definite, probable diagnosis according to the revised El Escorial criteria;\n3. Age: 18-65 years;\n4. FVC \\>70%;\n5. Onset \u2264 24 months;\n6. Patients with an ALSFRS-R score of at least 26; overall, including a score of at least 2 on each of the 1-9 ALSFRS-R individual component items and of at least 3 of the 10-12 individual components items;\n7. Evidence of fast progression of the disease. We exclude slow progressors at the time of screening defined as Patient with an ALSFRS-R total score progression between onset of the disease and screening of \\< 0.3 per month. We document the fast progression of the disease defined as ALSFRS-R total score decrease of \u2265 1 point per month during a 12 week run-in period between screening and randomization;\n8. Patient should be on a stable dose of Riluzole for \\> 30 days from pre-screening visit or not taking riluzole at all, nor plan to begin riluzole during the study period;\n9. Patient is medically able to tolerate transient immunosuppression regimen;\n10. Presence of a willing and able caregiver who understands the need to attend all follow-up visits, even if mobility declines.\n\nExclusion Criteria:\n\n1. Psychiatric disease or other neurological diseases different from ALS;\n2. Evidence of any concurrent illness or treatments limiting the safety to participate or any condition that the neurosurgeon feels may pose complications for the surgery;\n3. Cancer within the previous 10 years;\n4. Immunosuppressive therapy within 12 weeks of screening; active autoimmune disease or infection (including hepatitis B, hepatitis C, or HIV);\n5. Cognitive impairment;\n6. Contraindications to perform MRI scans, CSF withdrawal and Skin biopsy;\n7. Patient unable to understand informed consent form;\n8. Pregnancy and breast feeding;\n9. Patient has been treated previously with any stem cell or somatic cells therapy;\n10. Patient has participated in another clinical treatment trial or received other experimental medications outside of a clinical trial within 1 month prior to start of this study.", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "human Neural Stem Cells (hNSC)", "targeting_mechanism": "Intracerebroventricular transplantation of human neural stem cells to provide neuroprotective factors and differentiate into supportive glial and neuronal cell types.", "targeting_mechanism_pmid": "32043626", "animal_results": "Preclinical stem cell studies in mouse and rat ALS models (primarily SOD1 mutant models) demonstrated potential benefit of stem cell therapy, though comprehensive preclinical analysis is outside the scope of available evidence.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03793868", "title": "Perampanel Single Ascending Dose Transcranial Magnetic Stimulation Biomarker Study in Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mayo Clinic", "summary": "To evaluate if transcranial magnetic stimulation can be used as a biomarker in Amyotrophic Lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Perampanel"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2018-12-04", "url": "https://clinicaltrials.gov/study/NCT03793868", "target_entities": ["GRIA2"], "locations": [{"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. A probable laboratory supported, probable or definitive ALS diagnosis by revised El Escorial criteria.\n2. Sporadic or familial ALS.\n3. Ages of 18-70.\n4. Agree to use reliable contraception\n5. Randomization will occur after a baseline MT has been established; any subject in whom a MT cannot be established will be excluded.\n6. Caregiver willing to report adverse behavioral events. -\n\nExclusion Criteria:\n\n1. History of epilepsy.\n2. Significant laboratory abnormality (AST or alanine aminotransferase \\>3x upper limit of normal, or glomerular filtration rate \\<60)\n3. History of aggressive behavior.\n4. Subject unwilling to abstain from alcohol for 2 weeks after each dosing.\n5. History of drug abuse in the last 5 years\n6. Other severe medical conditions, including psychiatric conditions, which would cause an increased risk in the opinion of the investigator, including but not limited to renal failure and liver failure.\n7. Skull defect or other physical contraindication for TMS\n8. Pacemaker or implanted defibrillator\n9. Inability to take study capsule by mouth\n\nFemales only: Subject is pregnant \\[as confirmed by a positive serum human chorionic gonadotropin (hCG) test for females of reproductive potential (FRP) only\\], subject is breastfeeding, or subject is of reproductive potential and does not agree to follow use of reliable contraception.\n\n\\-", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Perampanel", "targeting_mechanism": "AMPA receptor antagonist that blocks glutamate-mediated excitotoxicity in motor neurons.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02943850", "title": "CNS10-NPC-GDNF for the Treatment of ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cedars-Sinai Medical Center", "summary": "The investigator is examining the safety of transplanting cells that have been engineered to produce a growth factor into the spinal cord of patients with Amyotrophic Lateral Sclerosis (ALS). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. These cells have been derived to specifically become astrocytes, which is a type of neuronal cell. The growth factor is called glial cell line-derived neurotrophic factor, or GDNF. GDNF is a protein that promotes the survival of many types of neuronal cells. Therefore, the cells are called \"CNS10-NPC-GDNF.\" The investigational treatment has been tested in animals, but it has not yet been tested in people. In this study, we want to learn if CNS10-NPC-GDNF cells are safe to transplant into the spinal cords of people.", "interventions": [{"type": "BIOLOGICAL", "name": "Stem cell (HPC) implantation"}, {"type": "DEVICE", "name": "Stereotactic surgical device"}], "start_date": "2017-04-01", "url": "https://clinicaltrials.gov/study/NCT02943850", "target_entities": ["GDNF"], "locations": [{"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Confirmed diagnosis of ALS (Lab-supported Probable, Probable or Definite EI Escorial Criteria)\n2. Duration of symptoms \u2264 36 months\n3. Progressive weakness in lower extremities, with EMG supported evidence of denervation in both lower extremities.\n4. Forced Vital Capacity \\>60% of predicted normal in supine.\n5. Male/Female; Age: 18 and older\n6. Able to provide Informed Consent\n7. Be geographically accessible to the study site and able to travel to study site for required visits\n8. Have caregiver to assist in the transportation and care required by participation in the study\n9. Not taking riluzole or on a stable dose for \u2265 30 days\n10. For women of child bearing capacity, negative pregnancy test prior to surgery\n11. Medically able to undergo thoracolumbar laminectomy or laminoplasty as determined by the site PI and Neurosurgeon\n12. Medically able to tolerate the immunosuppression regimen as determined by the site PI\n\nExclusion Criteria:\n\n1. Using invasive ventilatory assistance\n2. Diagnosis of another active or unstable medical illness that may interfere with study participation at discretion of PI\n3. Presence of any of the following conditions:\n\n   1. Current drug or alcohol abuse\n   2. Any known immunodeficiency syndrome\n   3. Unstable medical condition\n   4. Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening\n4. Persons of child bearing capacity not willing to practice birth control\n5. Receiving any investigational device/biologic/drug in past 30 days or any previous exposure to stem cell therapy\n6. Any condition in the lower extremities which precludes serial strength testing\n7. Any condition that the Neurosurgeon feels may pose complications for the surgery\n8. Any condition or ALS disease phenotype that the site PI feels may interfere with participation in the study or in the interpretation of study endpoints", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CNS10-NPC-GDNF (neural progenitor cells secreting GDNF)", "targeting_mechanism": "Neural progenitor cells engineered to secrete glial cell line-derived neurotrophic factor (GDNF), a growth factor that provides trophic support to degenerating motor neurons.", "targeting_mechanism_pmid": "36064599", "animal_results": "Transplantation of clinical-grade human neural stem cells reduced neuroinflammation, prolonged survival and delayed disease progression in SOD1 rats.", "animal_results_pmid": "31024007", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01897818", "title": "Communication by Brain - Computer Interface in Amyotrophic Lateral Sclerosis:Feasibility Study", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Centre Hospitalier Universitaire de Nice", "summary": "ALS is a severe progressive neurodegenerative disease characterized by degeneration motor neurons leading to death in 3 to 5 years. Gradually in time, the patient deprived of all motor skills as well as the possibility of communication written and oral developing a state close Locked In Syndrome (LIS). The main objective is to establish the feasibility of brain-computer interface using the pathological condition, with dependent disabled subjects as a means of communication.", "interventions": [{"type": "DEVICE", "name": "communication system P300 Speller"}], "start_date": "2013-07", "url": "https://clinicaltrials.gov/study/NCT01897818", "target_entities": [], "locations": [{"facility": "H\u00f4pital de l'Archet I", "city": "Nice", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.70313, "lon": 7.26608}], "contact_phone": "", "contact_email": "desnuelle.c@chu-nice.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* age \\>= 18\n* have a diagnosis of ALS suspected, possible, probable with EMG\n* be able to follow the study process and to comply with the schedule of visits upon entry into the study\n* understand the purpose of the study\n* expressing P300 wave in the conditions of the study\n\nExclusion Criteria:\n\n* have a mental illness or clinical dementia defined clinically significant may hinder the patient's ability to follow the procedures of the study\n* have a significant history of photosensitive epilepsy\n* have a history of allergy to the gel used for the electrodes\n* Major protected by law (guardianship, curators)\n* have uncorrectable visual disorders\n* not being able to maintain a sitting position and focus on a computer screen for more than 30 min.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Communication system P300 Speller", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07067229", "title": "Non-invasive Brain Stimulation and Exercise Intervention for Patients With Motor Neuron Disease", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Chulalongkorn University", "summary": "Motor neuron disease (MND) is a progressive neurological disorder involving degeneration of motor neurons, leading to muscle weakness, speech and swallowing difficulties, and respiratory failure. This study aims to develop a novel treatment approach combining personalized repetitive transcranial magnetic stimulation (rTMS) with mixed reality (MR) exercise-based games (exergames) to slow disease progression and improve quality of life. In this randomised controlled trial study will compare three groups: (1) rTMS with MR exercise (personalized intervention), (2) rTMS with MR exercise (standard intervention), and (3) sham rTMS with MR exercise. Outcomes will be assessed at baseline, 3 months, and 6 months post intervention. The long-term goal is to implement this approach in clinical settings to enhance care for people with MND.", "interventions": [{"type": "DEVICE", "name": "Personalized rTMS"}, {"type": "DEVICE", "name": "Standard rTMS"}, {"type": "DEVICE", "name": "Sham rTMS"}], "start_date": "2025-08-01", "url": "https://clinicaltrials.gov/study/NCT07067229", "target_entities": [], "locations": [{"facility": "King Chulalongkorn Memorial hospital, The Thai Red Cross Society", "city": "Bangkok", "state": "Pathumwan", "country": "Thailand", "status": "RECRUITING", "lat": 13.75398, "lon": 100.50144}], "contact_phone": "Thailand: 88-951-9195", "contact_email": "jakkrit.a@chula.ac.th", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Participants aged between 18 and 80 years\n* Diagnosed with any type of motor neuron disease (MND)\n* Have mild to moderate severity, as assessed by the Sinaki-Mulder scale, with a severity level between 1 and 3\n\nExclusion Criteria:\n\n* History of other neurological disorders, such as stroke\n* Use of ventilatory support\n* Severe dementia", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Personalized repetitive transcranial magnetic stimulation (rTMS) combined with mixed reality exercise-based games", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00800501", "title": "A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Newron Sweden AB", "summary": "This study is conducted to evaluate the safety and tolerability of the drug product sNN0029, containing the growth factor VEGF165, when administered directly into one of the fluid filled cavities in the brain using an implanted catheter and an implanted SynchroMed\u00ae II pump. Patients with Amyotrophic Lateral Sclerosis will be enrolled.", "interventions": [{"type": "DRUG", "name": "sNN0029"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2008-12", "url": "https://clinicaltrials.gov/study/NCT00800501", "target_entities": ["VEGF"], "locations": [{"facility": "University Hospital Leuven, Department of Neurology", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria.\n2. Age 18 to 75 years inclusive.\n3. If patients are being treated with riluzole, they must have been on a stable dose for at least 30 days.\n4. Ophthalmological examination at screening with normal findings regarding vascular structure and function.\n5. MRI/magnetic resonance angiography (MRA) examination of the brain and cervical spinal cord at screening with no findings of tumors or potential sources of pathological bleedings, or abnormality that may interfere with the assessments of safety or efficacy or that would, in the judgement of the investigator, represent a surgical risk to the subject.\n6. Values of coagulation parameters including platelet count, normalized prothrombin complex (PK-INR), activated partial thromboplastin time (APTT) within normal ranges.\n7. Patient is medically able to undergo the surgery required for stereotactic implantation of the catheter and infusion pump.\n8. Patient has been given written and verbal information, has had the opportunity to ask questions about the study, and understands the time and procedural commitments.\n9. Patient has given signed consent (written) to participate in the study. In the event that a patient who gives oral informed consent is not physically able to sign the informed consent form (ICF) due to disease progression, a witness may sign the ICF on the patient's behalf.\n\nExclusion Criteria:\n\n1. Impaired respiratory function judged to pose a risk to the patient during anaesthesia for the device implantation.\n2. Hypertension defined as blood pressure \\>160 mmHg systolic or \\>90 mmHg diastolic.\n3. Diagnosis of diabetes mellitus.\n4. Proliferative retinopathy.\n5. Non-proliferative retinopathy of moderate severity or higher.\n6. Concurrent clinically significant dementia as determined by the investigator.\n7. Concurrent clinically significant depression as determined by the investigator.\n8. History of structural brain disease other than ALS, including tumours and hyperplasia.\n9. Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection), alters wound healing (e.g., including bleeding disorders), or renders chronic ICV delivery or device implants medically unsuitable.\n10. Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally. Physicians should specifically investigate anatomical factors at or near the implant site (e.g., vascular abnormalities, neoplasms, or other abnormalities), underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver disease, or other medical conditions), and the administration of any antiplatelet or anticoagulant medication (e.g., aspirin, Plavix, NSAIDs) in the pre- or perioperative period. Any of those conditions or drugs could place a patient at an increased risk for intraoperative or postoperative bleeding.\n11. Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter.\n12. Presence of cardiac pacemakers, spinal cord stimulators, implantable programmable intraspinal drug pumps, or any other device that may interfere or interact with the programmer, without prior approval by Medtronic.\n13. Clinically significant abnormalities in hematology or clinical chemistry parameters as assessed by the investigator.\n14. Ongoing medical condition that according to the investigator would interfere with the conduct and assessments in the study. Examples are medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of safety and efficacy of investigational product or device performance, or would compromise the ability of the subject to undergo study procedures (e.g., MRI), or to give informed consent.\n15. Participation in another clinical trial with an investigational drug or device within 3 months prior to Screening visit.\n16. For female subjects, ongoing pregnancy or planned pregnancy during the period of treatment with study drug.\n17. Breast feeding during the period of treatment with study drug.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "sNN0029 (containing vascular endothelial growth factor VEGF165)", "targeting_mechanism": "Vascular endothelial growth factor (VEGF165) delivered intracerebroventricularly to promote neuroprotection and angiogenesis in motor neurons.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06307301", "title": "Study in ALS With Abatacept & IL-2", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The Methodist Hospital Research Institute", "summary": "In Amyotrophic Lateral Sclerosis (ALS), the reduction of regulatory T-lymphocyte (Treg) numbers and suppressive function correlates with rapid disease progression. The investigator completed a phase 1 study of infusions of expanded autologous Tregs in combination with subcutaneous IL-2 injections in ALS patients, which showed enhancement of Treg numbers and suppressive function in vivo. The enhanced Treg suppressive function correlated strongly with slowing and stabilization of disease progression. Drugs that enhance endogenous Treg numbers and suppressive function may also stabilize disease in ALS. This phase 1 study aims to determine whether the combination therapy of subcutaneous IL-2 and abatacept (Orencia\u00ae) is safe and well-tolerated in 6 patients with ALS, and whether the therapy enhances Treg numbers and suppressive function in vivo.", "interventions": [{"type": "DRUG", "name": "Abatacept Injection [Orencia] and Proleukin (aldesleukin)"}], "start_date": "2021-10-28", "url": "https://clinicaltrials.gov/study/NCT06307301", "target_entities": ["regulatory_t_lymphocytes"], "locations": [{"facility": "Houston Methodist Research Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nPatients will be eligible for initial enrollment on this study if they meet the following criteria at the time of screening:\n\n1. Provided informed consent and authorized use of protected health information (PHI) in accordance with national and local patient privacy regulations.\n2. ALS meeting El Escorial criteria for possible, probable, lab-supported probable, or definite ALS.\n3. At least 18 years old.\n4. Total bilirubin less than or equal to 1.5 mg/dL\n5. Alanine aminotransferase level (ALT) less than or equal to five times normal, albumin greater than or equal to 3.0 gm/dL\n6. Serum creatinine less than 1.5 mg/dL\n7. Capable of complying with all study procedures, including the study drug delivery procedure, in the Investigator's opinion.\n8. A family member or caretaker who is expected to be consistently available to administer both study drugs of abatacept and IL-2 if the participant is unable to do so.\n9. On a stable regimen of riluzole for at least 30 days at the time of screening. If not on riluzole at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial.\n10. Patients on edaravone willing to refrain from taking edaravone on the same day as they will receive the abatacept injection for the duration of the trial. If not on edaravone at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial.\n11. Forced vital capacity (FVC) \u226550% of predicted capacity for age, height, and sex at screening, or receiving treatment with noninvasive ventilation if FVC \\< 50% of predicted for age, height, and sex at screening.\n\nExclusion Criteria:\n\nPatients will be ineligible to participate if any of the following are true at the time of screening:\n\n1. Serious, active bacterial, fungal, or viral infection, active or latent tuberculosis.\n2. Tracheostomy.\n3. Severe cardiac dysfunction defined as left ventricular ejection fraction \\<40% if an echocardiogram is medically indicated to clarify ongoing symptoms or EKG findings.; a history of non-controlled cardiac arrhythmias; history of cardiac tamponade; Unstable angina or MI in the last 3 months.\n4. Hypersensitivity or allergy to IL-2 or abatacept.\n5. History of bowel ischemia/perforation, or GI bleeding requiring surgery.\n6. History of resistant seizures, history of coma or toxic psychosis lasting \\>48 hours.\n7. Platelets \\<100,000/mm3; hematocrit \\<30%.\n8. History of cancer in the past 5 years (except cutaneous Basal cell carcinoma or squamous cell carcinoma).\n9. Hx of immunomodulation therapy including IL-2 or abatacept administration in the past 90 days.\n10. Treatment with another investigational drug, biological agent, or device within 30 days or 5 half-lives of screening, whichever is longer.\n11. If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or unwilling to use effective contraception for the duration of the trial and for 90 days after treatment.\n12. If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 90 days after treatment", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Abatacept (Orencia) and Proleukin (aldesleukin/IL-2)", "targeting_mechanism": "Abatacept blocks T-cell costimulation (CD80/CD86-CD28 interaction) while aldesleukin (recombinant IL-2) expands and enhances regulatory T-lymphocyte (Treg) suppressive function to reduce neuroinflammation.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06665165", "title": "AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Amylyx Pharmaceuticals Inc.", "summary": "This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "AMX0114"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2025-04-07", "url": "https://clinicaltrials.gov/study/NCT06665165", "target_entities": ["TARDBP"], "locations": [{"facility": "University of California, San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 32.84727, "lon": -117.2742}, {"facility": "Georgetown University Hospital Pasquerilla Healthcare Center", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "RECRUITING", "lat": 38.89511, "lon": -77.03637}, {"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 29.65163, "lon": -82.32483}, {"facility": "Mayo Clinic in Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 30.33218, "lon": -81.65565}, {"facility": "Orlando Regional Medical Center, Orlando Health Neuroscience Institute", "city": "Orlando", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 28.53834, "lon": -81.37924}, {"facility": "Massachusetts General Hospital, Healey & AMG Center for ALS", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "Mayo Clinic in Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "RECRUITING", "lat": 44.02163, "lon": -92.4699}, {"facility": "Temple University of the Commonwealth System of Higher Education", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 39.95238, "lon": -75.16362}, {"facility": "Alliance for Multispecialty Research, LLC", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "RECRUITING", "lat": 35.96064, "lon": -83.92074}, {"facility": "Houston Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Calgary", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "RECRUITING", "lat": 51.05011, "lon": -114.08529}, {"facility": "McMaster University", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 43.25011, "lon": -79.84963}, {"facility": "London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 42.98339, "lon": -81.23304}, {"facility": "McGill University Health Centre - Centre for Innovative Medicine", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "857-320-6200", "contact_email": "clinicaltrials@amylyx.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Ability to understand the purpose and risks of this study, willingness to comply with the study and to provide informed consent in accordance with local laws and regulations.\n2. Male or female, at least 18 years of age.\n3. Diagnosis of clinically definite or clinically probable ALS, made by a physician who is experienced with management of ALS.\n4. Time since onset of first symptom of ALS should be \\<24 months prior to beginning the study. Date of ALS symptom onset is defined as the onset of weakness (in the limbs, bulbar region, or trunk).\n5. If the participant is to be treated with riluzole and/or edaravone before or during the trial, then treatment must be previously started and maintained at a stable regimen for at least 30 days prior to starting the study and through the end of the study.\n6. Women of childbearing potential (e.g., not post-menopausal for at least one year or surgically sterile) must agree to use an acceptable birth control method for the duration of the trial and 60 days after the last dose of Study Drug or be of non-childbearing potential.\n7. Female participants or female partners of male participants must not be pregnant or plan to become pregnant for the duration of the trial and for up to 90 days after the last dose of Study Drug.\n8. Male participants must agree to abstain from sperm donation for the duration of the trial and practice contraception with a female partner, for at least 90 days after last dose of Study Drug.\n\nExclusion Criteria:\n\n1. Presence of tracheostomy or permanent assisted ventilation.\n2. SVC less than 65%.\n3. Abnormal liver function defined as aspartate aminotransferase and/or alanine aminotransferase \\> 3 times the upper limit of normal (ULN) and/or total bilirubin \\> 1.5 times the ULN (obtained within 4 weeks of first dose) except when a result of Gilbert syndrome.\n4. Abnormal renal function defined as estimated glomerular filtration rate (eGFR) \\< 60 mL/min/1.73m2.\n5. Other laboratory abnormalities, including abnormalities in platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time.\n6. Pregnant women (confirmed by a pregnancy test within 7 days prior to first dose) or women currently breastfeeding.\n7. Current or previous clinically significant, unstable medical condition (other than ALS), that in the opinion of the Investigator could affect a participant's safety or ability to comply with the study.\n8. Significant abnormalities in physical/neurological examination, vital signs, or electrocardiogram (ECG), which in the opinion of the Investigator could affect the safety of the participant.\n9. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that could affect the participant's ability to provide informed consent or comply with study procedures.\n10. Current or previous enrollment in another trial involving use of an investigational therapy, in most cases within 30 days after the last dose of the study drug, prior to starting this study.\n11. Current or previous treatment with small interfering ribonucleic acid, stem cell therapy, any ASO or gene therapy.\n12. Any contraindications for lumbar puncture or repeated intrathecal injection and/or underlying disorders that could be affected by intrathecal injections.\n13. Prior severe reaction or known hypersensitivity to any part of the Study Drug.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AMX0114", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06408727", "title": "Intermediate Expanded Access Protocol CNMAu8.EAP04", "phase": "Expanded Access", "status": "TEMPORARILY_NOT_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Clene Nanomedicine", "summary": "An Intermediate Expanded Access Protocol (EAP) with CNM-Au8 for Amyotrophic Lateral Sclerosis for NIH Grant RFA-NS-23-012", "interventions": [{"type": "DRUG", "name": "CNM-Au8"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT06408727", "target_entities": [], "locations": [{"facility": "Nova Southeastern University", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Synapticure", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Iowa", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "Columbia Unniversity", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Pennsylvania State University", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to understand and give written informed consent.\n2. Male or female participants aged 18 years or greater (inclusive) at the time of informed consent completion.\n3. Participants with a confirmed diagnosis of ALS per Gold Coast criteria as determined by a neurologist specializing in ALS (e.g., the site principal investigator or sub-investigator for this study).\n4. Participant is able to daily consume up to 60 mL of the investigational drug suspension without substantial dysphagia, OR can intake the investigational product through a feeding tube.\n5. Participant must have completed standard clinical safety labs within the prior 90 days from the Baseline visit, including a chemistry panel (e.g., CMP) and a hematology panel (e.g., CBC).\n6. Participant has a baseline score by the TRICALS risk calculator that is less than -2 (i.e., participant is not at increased risk of early death; https://tricals.shinyapps.io/risk-profile/).\n7. Participant meets the following criteria:\n\n   1. Baseline Vital Capacity \\>15% predicted,\n   2. Baseline ALSFRS-R Score \\>8, and,\n   3. Baseline BMI \\>17.5 kg/m2\n8. Participants have established care with a neurologist at the specialized ALS center or remotely enrolling site involved in the study and will maintain this clinical care throughout the duration of the EAP within the United States.\n\nExclusion Criteria:\n\n1. Participant is eligible for participation in any double-blind placebo-controlled study the treatment of ALS at the same research site.\n2. Participant has a history of any clinically significant or unstable medical condition (other than ALS) that may interfere with assessment of safety or compromise the study objectives.\n3. Based on the investigator's judgment, participants who may have difficulty complying with the protocol and/or any study procedures.\n4. Within the prior 90-days the participant has had clinically significant findings on standard hepatic, hematologic, or renal safety assays, including but not limited to: (i) ALT or AST \u2265 3 times upper limits of normal, (ii) direct (conjugated) with bilirubin \u22652 times upper limits of normal, (iii) low platelet counts (\\< 150 x 109 per liter) or eosinophilia (absolute eosinophil count of \u2265 500 eosinophils per microliter), (iv) serum creatinine \\>1.2 mg/dL, or (v) eGFR \\< 45 ml/min per 1.73 m2.\n5. Participant is currently involved in another placebo-controlled clinical trial (note: concomitant therapy with other investigational products is permitted with certain restrictions-see concomitant medications below).\n6. Females who are pregnant or nursing or who plan to get pregnant during the EAP, or within 6 months of the end of this trial.\n7. Females of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control.\n8. History of gold allergy.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CNM-Au8", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07223723", "title": "A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE4", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogen", "summary": "In this study, researchers will learn more about the safety of tofersen, also known as Qalsody\u00ae. This is a drug available for doctors to prescribe for people with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in people who have a mutation in the superoxide dismutase 1 gene, also known as SOD-1.\n\nThis is known as a \"postmarketing\" study. In this kind of the study, the goal is to learn more about how a drug works after it has been approved for use in the general public. Tofersen was approved in China in September 2024. The main goal of this study is to collect long-term safety information in Chinese participants with SOD-1 ALS.\n\nThe main question researchers want to answer in this study is:\n\n\u2022 How many participants have adverse events (AEs) and serious adverse events (SAEs)?\n\nAn AE is a health problem that may or may not be caused by a drug during the study. An AE is considered serious when it results in death, is life-threatening, causes lasting problems, or requires hospital care.\n\nResearchers will also learn more about :\n\n* How the body processes tofersen.\n* How much tofersen is found in the cerebrospinal fluid (CSF), or the fluid that surrounds the brain and the spinal cord.\n\nThis study will be done as follows:\n\n* Participants will be screened to check if they can join the study. The screening period will be up to 4 weeks.\n* After joining the study, participants will receive the first 3 doses of 100 milligrams (mg) of tofersen about 14 days apart. This will be given through an intrathecal (IT) injection. This means it will be given into the fluid surrounding the spine.\n* After that, participants will receive 10 more doses every 28 days through IT injections. Participants will have up to 13 total doses of tofersen in this study.\n* Participants will have up to 15 visits to their study research center. Each participant will be in the study for up to 52 weeks (1 year).", "interventions": [{"type": "DRUG", "name": "Tofersen"}], "start_date": "2025-12-11", "url": "https://clinicaltrials.gov/study/NCT07223723", "target_entities": ["SOD1"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Age \u2265 18 years at the time of informed consent.\n* Must have diagnosis of SOD1-ALS.\n* If taking riluzole, participant must be on a stable dose for \u2265 30 days prior to Day 1 and expected to remain at that dose until the final study visit.\n* If taking edaravone, participant must have initiated edaravone \u2265 60 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study.\n* All women of childbearing potential must practice effective contraception during the study.\n\nKey Exclusion Criteria:\n\n* Hypersensitivity to the active substance or any of the excipients of tofersen injection.\n* Current or past administration of tofersen injection, in either a commercial or a clinical study setting.\n* Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.\n* Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer, prior the Baseline Visit.\n* Participants who are pregnant or currently breastfeeding, and those intending to become pregnant during the study.\n\nNote: Other protocol-defined Inclusion/Exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tofersen", "targeting_mechanism": "Antisense oligonucleotide that reduces SOD1 mRNA and protein levels.", "targeting_mechanism_pmid": "37975798", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00983983", "title": "High Fat/High Calorie Trial in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "The purpose of this study is to determine the safety, tolerability, and preliminary efficacy of long-term use of high fat/high calorie and high calorie diets in people with amyotrophic lateral sclerosis (ALS) (Lou Gehrig's disease).", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Oxepa"}, {"type": "DIETARY_SUPPLEMENT", "name": "Jevity 1.5"}, {"type": "DIETARY_SUPPLEMENT", "name": "Jevity 1.0"}], "start_date": "2009-10", "url": "https://clinicaltrials.gov/study/NCT00983983", "target_entities": [], "locations": [{"facility": "Barrow Neurological Institute/St. Joseph's Hospital and Medical Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California at Irvine", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "California Pacific Medical Center, University of California at San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Sarasota Memorial Hospital", "city": "Sarasota", "state": "Florida", "country": "United States", "status": "", "lat": 27.33643, "lon": -82.53065}, {"facility": "Emory University School of Medicine", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Neurology Clinical Trials Unit, Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Saint Mary's Health Care", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "Columbia Presbyterian Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Carolinas Medical Center Neuromuscular/ALS-MDA Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Oregan Health and Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Drexel University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Vermont", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Clinical diagnosis of ALS\n2. Male or female subjects aged 18 years or older\n3. Must already be tolerating tube feedings through either a gastrostomy tube (G-tube or PEG) or jejunostomy tube (J-tube)\n4. Must require non-invasive ventilation (BIPAP) for less than 10 hours/day\n5. Women of childbearing potential must have a negative pregnancy test at screening and be non-lactating.\n\nExclusion Criteria:\n\n1. History of hepatitis including non-alcoholic steatohepatitis (NASH), cholecystectomy, prior biliary disease such as gallstones\n2. History of diabetes\n3. History of prior myocardial infarction or stroke\n4. Laboratory values: Screening alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 2.0 times the upper limit of normal or total bilirubin greater than 1.5 times the upper limit of normal\n5. Allergy to soy, fish, or milk products", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04953286", "title": "Ocular Surface Metabolo-lipidomics in Lateral Amyotrophic Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Tours", "summary": "Amyotrophic Lateral Sclerosis (ALS) is the most common neurodegenerative disease affecting the motor neuron. Currently, there is no diagnostic test and no examination that can predict the evolution of this pathology. The search for diagnostic and prognostic biomarkers is therefore essential for a better understanding of the pathophysiology of ALS, which remains poorly understood, and also for better clinical management. The ocular surface, made up of liquid elements, tears, and cells, is an accessible anatomical-physiological entity that has demonstrated its usefulness in the identification of biomarkers in neurodegenerative diseases such as Parkinson's or Alzheimer's. To date, no study has explored the ocular surface as a biomarker in ALS", "interventions": [{"type": "OTHER", "name": "Measure of visual acuity"}, {"type": "OTHER", "name": "Interferometry"}, {"type": "OTHER", "name": "Samples of basal tears"}, {"type": "OTHER", "name": "Central corneal sensitivity"}, {"type": "OTHER", "name": "Slit lamp examination and undilated fundus"}, {"type": "OTHER", "name": "Conjunctival impression"}, {"type": "OTHER", "name": "Evaluation of the corneal innervation"}], "start_date": "2021-09-17", "url": "https://clinicaltrials.gov/study/NCT04953286", "target_entities": [], "locations": [{"facility": "Ophthalmology Department, University Hospital of Tours, France", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Neurology Department, University Hospital of Tours, France", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Centre d'Investigation Clinique_CIC 1415", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Case group selection criteria :\n\nInclusion Criteria:\n\n* Patient with clinically defined or probable primary ALS according to Airlie House criteria(1)\n* Familial or sporadic form\n* \u226518 years of age\n* Patient affiliated with a social security plan\n* Informed consent signed by the patient\n\nExclusion Criteria:\n\n* Motor neuron disease mimicking ALS\n* Pregnant or breastfeeding woman\n* Treatment that may have a neuroprotective effect\n* Any eye drops or treatments that may interfere with tear production\n* Lens wearer\n* Eye surgery \u22643 months\n* Any ocular pathology other than ametropia, oculomotor disorder, amblyopia\n* Any general pathology other than ALS with ocular repercussions\n* Protective measure of guardianship or curators\n\nControl group selection criteria:\n\nInclusion Criteria:\n\n* No diagnosed neurological pathology\n* \u226518 years of age\n* Patient affiliated with a social security plan\n* Informed consent signed by the participant\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding woman\n* Treatment likely to have a neuroprotective effect\n* Any eye drops or treatments that may interfere with tear production\n* Lens wearer\n* Eye surgery \u22643 months\n* Any ocular pathology except ametropia, oculomotor disorder, amblyopia\n* Any general pathology with ocular repercussions\n* Protective measure of guardianship or curator", "sex": "ALL", "min_age": "18 Years", "max_age": "100 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06977451", "title": "Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS", "phase": "PHASE1, PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "n-Lorem Foundation", "summary": "This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10", "interventions": [{"type": "DRUG", "name": "nL-CHCHD-001"}], "start_date": "2024-06-24", "url": "https://clinicaltrials.gov/study/NCT06977451", "target_entities": ["CHCHD10"], "locations": [{"facility": "Columbia University, Irving Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s).\n* Ability to travel to the study stie and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.\n* Genetically confirmed neurological disorder.\n\nExclusion Criteria:\n\n* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.\n* Use of an investigational medication within less than 5 half-lives of the drug at enrollment", "sex": "ALL", "min_age": "63 Years", "max_age": "63 Years", "healthy_volunteers": false, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "nL-CHCHD-001", "targeting_mechanism": "Personalized antisense oligonucleotide targeting CHCHD10 to suppress pathogenic variants in this gene associated with ALS", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01583088", "title": "Early Stage Amyotrophic Lateral Sclerosis Phrenic Stimulation", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "ALS is is characterized by a progressive degeneration of motor neurons, leading to progressive weakness of muscles, including respiratory muscles, the diaphragm. Although specific therapy is lacking, correct respiratory therapy improves quality of life and increases survival. Substituting the failing respiratory muscles by non invasive mechanical ventilatory assistance (NIV) is the current standard of care. Intradiaphragmatic phrenic nerve stimulation is a new treatment and has been the object of a preliminary international proof-of-concept multicenter trial. This trial suggests that the intradiaphragmatic phrenic nerve stimulation slows down the rate of decline of the diaphragm. Our new hypothesis is that phrenic stimulation induces diaphragm conditioning and can delay the need for mechanical ventilation in ALS patients. We will study, during 24 months, 2 groups of 37 patients at the beginning of the respiratory dysfunction, using a intradiaphragmatic phrenic nerve stimulation in one group and a sham stimulation in the other group. Although, all the patients will be implanted, thus, at the end of the study, all the patients will receive effective stimulation.", "interventions": [{"type": "DEVICE", "name": "phenique nerve stimulation NeurX\u2122 (Synapse Biomedical)"}, {"type": "DEVICE", "name": "sham phrenic nerve stimulation"}], "start_date": "2012-09", "url": "https://clinicaltrials.gov/study/NCT01583088", "target_entities": ["respiratory_function"], "locations": [{"facility": "APHP, GH Piti\u00e9 Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis is laboratory-supported probable, probable, or definite according to the World Federation of Neurology El Escorial criteria\n* Forced Vital Capacity (FVC) from 80 - 60% of predicted values at enrollment\n* Bilateral phrenic nerve function acceptable as demonstrated by bilateral diaphragm EMG recordings and nerve conduction times without axonal lesion\n\nExclusion Criteria:\n\n* Active cardiovascular disease that would increase the risk of general anesthesia. (FEVG\\<60%)\n* Underlying pulmonary diseases that were present prior to ALS that would effect pulmonary tests independent of ALS, in particular COPD with FEV1\\<30%\n* Pre-existing implanted electrical device such as a pacemaker or cardiac defibrillator\n* respiratory infection or decompensation in the last 30 days\n* Marked obesity affecting suitability for surgery\n* Significant scoliosis or chest deformity affecting suitability for surgery\n* Pre-existing diaphragm abnormality such as a hiatal hernia or paraesophageal hernia\n* Patient on NIV, CPAP or Oxygen for a reason other than ALS\n* Criteria for NIV (VC\\<50% of predicted values and/or Pi max and SNIP\\<60% of predicted values; and/or nocturnal desaturations without SAOS and/or PaCO2\\>45 mm d'Hg)\n* Supine VC\\<50% of predicted values", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NeurX\u2122 phrenic nerve stimulator", "targeting_mechanism": "Intradiaphragmatic phrenic nerve stimulation to directly activate the diaphragm and maintain respiratory function", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03659422", "title": "Dietary Approach to Improving Quality of Life in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Terry L. Wahls", "summary": "We have had reports of an individual who utilized a modified Paleolithic diet and vitamin/ supplement program as part of his approach to managing ALS related symptoms. This individual has experienced stability in his ALS functional rating score and stable to improving strength over an 18 month period. There are also anecdotal reports of ALS patients who have utilized a dietary approach based on a Paleolithic eating plan of improved function. This is a safety study. We will be assessing if patients can implement the proposed modified Paleolithic diet (Wahls Elimination), if lean muscle mass is maintained on the study diet, and what changes occur in the ALS functional symptoms and quality of life.", "interventions": [{"type": "OTHER", "name": "Modified Paleolithic diet"}], "start_date": "2019-07-01", "url": "https://clinicaltrials.gov/study/NCT03659422", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)\n2. Ability to prepare, or have prepared for them, home-cooked meals\n3. Age between 18 and 80 years\n4. Followed by ALS clinic at the University of Iowa\n5. Willingness to adopt the study diet\n\nExclusion Criteria:\n\n1. Clinically significant liver, kidney, or heart disease\n2. Taking insulin or Coumadin\n3. Ventilator dependence\n4. Psychiatric disorder making dietary compliance difficult (e.g. schizophrenia)\n5. Unwillingness to have blood specimens collected\n6. Dysphagia present\n7. Greater than two years since onset of ALS symptoms", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Modified Paleolithic diet", "targeting_mechanism": "Dietary intervention aimed at addressing metabolic abnormalities and nutritional deficits in ALS", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05508074", "title": "Treatment Combining Riluzole and IFB-088 in Bulbar Amyotrophic Lateral Sclerosis (TRIALS Protocol)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "InFlectis BioScience", "summary": "Prospective, international, randomised, double-blind, placebo controlled, multicentre, parallel group study. Patients will be randomised in a 2:1 allocation ratio to receive either IFB-088 + riluzole 100 mg or placebo + riluzole 100 mg. This clinical trial is an exploratory study, designed to show a signal of efficacy of IFB-088 through ALSFRS-R, MITOS and King's College. Respiratory function will be followed through SVC. Biomarkers and quality of life will also be evaluated throughout the study.\n\nPatients will be treated over a 6-month period. After a screening/consent visit, patients will undergo clinic visits at randomisation (V0), at 2 weeks (V1), and at months 1 (V2), 3 (V3) and 6 (V4). One week after V0, the patient will undergo urine analysis (dipstick)and blood sampling for measurement of creatinine\n\n, as well as blood sampling for measurement of creatinine and calculation of eGFR at months 2, 4 and 5. At the V2 visit, in addition to other assessments, patients will undergo blood sampling for PK measurements and urine sampling for crystalluria examination. Blood and urine chemistry, as well as physical examination and vital signs assessment to assess safety will be performed at each visit for safety purpose and crystalluria examination will be repeated at the follow-up visit, performed one month \u00b1 one week after V4.", "interventions": [{"type": "DRUG", "name": "IFB-088 50mg/day"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Riluzole 100mg/day"}], "start_date": "2022-12-02", "url": "https://clinicaltrials.gov/study/NCT05508074", "target_entities": ["riluzole", "ifb_088"], "locations": [{"facility": "H\u00f4pital Neurologique Pierre Wertheimer", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "APHM H\u00f4pital La Timone Adultes SCE Maladies Neuromusculaires / SLA", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHU de Nantes - H\u00f4pital Laennec", "city": "Nantes", "state": "", "country": "France", "status": "", "lat": 47.21725, "lon": -1.55336}, {"facility": "CHU de Toulouse - H\u00f4pital Pierre-Paul Riquet", "city": "Toulouse", "state": "", "country": "France", "status": "", "lat": 43.60426, "lon": 1.44367}, {"facility": "CHU Bretonneau", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Ospedale Civile Sant'Agostino Estense", "city": "Baggiovara", "state": "", "country": "Italy", "status": "", "lat": 44.60416, "lon": 10.86256}, {"facility": "Centro Clinico NeMO per le Malattie Neuromuscolari", "city": "Gussago", "state": "", "country": "Italy", "status": "", "lat": 45.58358, "lon": 10.15717}, {"facility": "IRCSS Istituto Neurologico Carlo Besta", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Sant'Andrea Hospital Unit of Neuromuscular Disorders", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Diagnosis of probable or definite ALS according to the revised El Escorial criteria \\[29\\], with bulbar onset of disease, familial or sporadic form,\n2. Onset of symptoms \u2264 18 months prior to screening, as reported by the patient,\n3. Adult males or females, aged at least 18 years old,\n4. SVC \\> 60% of predicted value for age and sex,\n5. ALSFRS-R score \u2265 36,\n6. Treatment with riluzole 100 mg/day, at stable dose since at least one month and well tolerated,\n7. Male or female patient of childbearing potential10 who agrees to use highly effective mechanical contraception methods (sexual abstinence, intrauterine device, bilateral tubal occlusion, vasectomised partner) throughout the study, and for 3 months after the end of the treatment,\n8. Patient who read, understood and signed the ICF,\n9. Patient who is willing to adhere to the study visit schedule and is capable to understand and comply with protocol requirements.\n\nExclusion Criteria:\n\n1. Known other significant neurological disease(s),\n2. Serious illness(es) or medical condition(s) (e.g. unstable cardiac disease, cancer, hematologic disease, hepatitis or liver failure, renal failure) that is not stabilised or that could require hospitalisation and may jeopardise the participation in the study,\n3. Abnormal renal function at screening defined as estimated glomerular filtration rate (eGFR) \\< 60 mL/min/1.73m2,\n4. Abnormal liver function at screening defined as total bilirubin levels \\>1.5 ULN, and/or AST and/or ALT \\>3 ULN,\n5. Neutropenia (ANC \\<1.5 x 109/L) at screening,\n6. Other causes of neuromuscular weakness,\n7. Non progressive or very rapidly progressing ALS (ALSFRS-R decline from disease onset to randomisation \u2264 0.1 / month or \u2265 1.2 / month)11,\n8. Non-invasive ventilation,\n9. Tracheotomy,\n10. Weight loss \u2265 10% compared to weight at symptoms onset as declared by the patient or BMI \\<18 kg/m2 at screening,\n11. Dementia or other severe active psychiatric illness, including suicidal ideation assessed using the Columbia-Suicide Severity Rating Scale (C-SSRS),\n12. Patient with a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect of ALS on respiratory function,\n13. Patient treated by edaravone for ALS,\n14. Patient using unauthorised concomitant treatments, namely moderate or strong inhibitors or inducers of CYP1A2, strong inhibitors or inducers of CYP2D6 or 2C19 and strong inhibitors of OCT2, as listed in Section 6.2. Combined oral contraceptives containing ethinylestradiol are forbidden concomitant medications,\n15. Smoker of \\> 10 cigarettes per day (e-cigarettes and nicotine patches are permitted),\n16. Known hypersensitivity to any of the ingredients or excipients of the IMPs,\n17. Pregnant, lactating women,\n18. Patient who participated in another trial of investigational drug(s) within 30 days prior to randomisation, or 5 half-lives of the previous investigational product, whichever is longer,\n19. Patient who has forfeited their freedom by administrative or legal award, or who is under guardianship or under limited judicial protection.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "IFB-088", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07571174", "title": "A Substudy of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Eli Lilly and Company", "summary": "The main purpose of this study is to assess the long-term safety and tolerability of LY4256984 in participants with Amyotrophic Lateral Sclerosis (ALS). This study is a long-term extension of study J6I-MC-OWAA (NCT07100119) and is part of the OLMP (NCT07571200) master protocol that will last approximately 96 weeks.", "interventions": [{"type": "DRUG", "name": "LY4256984"}], "start_date": "2026-05-14", "url": "https://clinicaltrials.gov/study/NCT07571174", "target_entities": ["ly4256984"], "locations": [{"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "NOT_YET_RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "University Hospital - London Health Sciences Centre", "city": "London", "state": "", "country": "Canada", "status": "NOT_YET_RECRUITING", "lat": 42.98339, "lon": -81.23304}, {"facility": "McGill University Health Centre", "city": "Montreal", "state": "", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}, {"facility": "Sunnybrook Research Institute", "city": "Toronto", "state": "", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}, {"facility": "Universit\u00e4tsklinikum Schleswig-Holstein", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 53.86893, "lon": 10.68729}, {"facility": "Universit\u00e4tsmedizin Rostock", "city": "Rostock", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 54.0887, "lon": 12.14049}, {"facility": "Universitaetsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "NOT_YET_RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Universitari de Bellvitge", "city": "L'Hospitalet de Llobregat", "state": "", "country": "Spain", "status": "NOT_YET_RECRUITING", "lat": 41.35967, "lon": 2.10028}, {"facility": "Hospital Universitari i Politecnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "NOT_YET_RECRUITING", "lat": 39.47391, "lon": -0.37966}], "contact_phone": "1-317-615-4559", "contact_email": "LillyTrials@Lilly.com", "eligibility": {"criteria": "Participants must meet eligibility criteria in the \\[L0U-MC-OLMP\\] screening protocol before entry into the treatment study.\n\nInclusion Criteria:\n\n* Have completed the main treatment period/phase as well as any off-treatment period/phase of Study OWAA, the parent study for this ISA.\n\nExclusion Criteria:\n\n* The participant has conditions that preclude a lumbar puncture (LP), such as:\n\n  * A history of clinically significant back pain, back pathology, and/or back injury (for example, degenerative disease, spinal deformity, or spinal surgery) that may predispose to complications or technical difficulty with LP.\n  * Allergy to local anesthetics, such as lidocaine or its derivatives.\n  * A local infection at the intended site of the LP.\n  * Less than 100 giga per liter \\[(\\<100 GI/L) is equivalent to 100,000 per cubic millimeter (100,000/mm\u00b3)\\] platelets or clinically significant coagulation abnormality or significant active bleeding, or\n  * Currently receiving treatment with an anticoagulant, antiplatelet agent, or other drug that affects coagulation or platelet function. Low dose (according to local medical guidelines) aspirin is permitted.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "LY4256984", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01959373", "title": "Dysfunctions and Plasticity Mechanisms of Motor System Assessed by Cortico-cortical and Cortico-muscular Coherence Analysis in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "SUSPENDED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique Hopitaux De Marseille", "summary": "Amyotrophic lateral sclerosis (ALS) is characterized clinically by abnormalities of both upper motor neurons (UMN) and lower motor neurons (LMN). The presence of UMN signs is not always easy to establish. The only technique used in routine to assess the corticospinal dysfunctions is based on transcranial magnetic stimulation (TMS). However, this technique is largely dependent on LMN state and is based on artificial motor cortex activation.\n\nThe main objective of our study project is to evaluate a new method assessing functional changes in motor system in ALS patients. By using cortico-muscular and cortico-cortical coherences, it could be possible to show modifications in both cortico-muscular relationship and in cortical activity coordination which could be related to clinical state in ALS patients. We notably expect a decrease in cortico-muscular coherence in ALS patients. Furthermore, these analyses could provide new insights in motor system plasticity phenomena. We expect a partial covering of voluntary motor command by cortical areas adjacent to primary motor cortex. Lastly, the hypothesis that an increased proportion of voluntary motor control may be assumed by ipsilateral corticospinal tract could be tested by coherence analyses.\n\nCoherence analysis might be a useful method to detect corticospinal tract dysfunctions. This method has the advantage to be painless and not to use artificial stimulations as it is used in TMS.", "interventions": [{"type": "PROCEDURE", "name": "electroencephalograms (EEG)"}, {"type": "PROCEDURE", "name": "electro-myography (EMG)"}], "start_date": "2013-10", "url": "https://clinicaltrials.gov/study/NCT01959373", "target_entities": [], "locations": [{"facility": "Assistance Publique Hopitaux de Marseille", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patient female or male, more than 18 years,\n* Patien with a diagnosis of amyotrophic lateral sclerosis according to the El Escorial criteria of Brooks et al. 1994\n* Patient , the beginning of the SLA date less than 12 months,\n* Patient not having a familial form of ALS,\n* Patient not having cancer, autoimmune disease, liver failure, severe hypertension or untreated, severe conduction disorders or uncontrolled arrhythmia\n* Patient not having a chronic psychiatric disease, dementia.\n* Patient with normal visual function\n* Patients receiving social coverage\n* Patient have read, understood and signed an informed consent after information\n\nExclusion Criteria:\n\n* Patient minor\n* Patient with a familial form of ALS,\n* Patient associated with severe progressive disease (cancer, autoimmune disease , liver failure )\n* Patient (s) with chronic mental illness, dementia ,\n* Presence of atypical clinical signs such as cerebellar ataxia , extrapyramidal signs, sensory disorders , autonomic dysfunction .\n* Clinical signs of chronic respiratory failure or slow and / or forced less than 70% of the theoretical value or chronic hypercapnia than 45 mmHg vital capacity .\n* Patient private freedom following a judicial or administrative decision\n* Patient major Trust\n* Pateinte pregnant, parturient , lactating\n* Patient major in legal protection ( guardianship )\n* Pateinte pregnant , parturient , lactating\n* Patient hospital without consent\n* Patient admitted in a health or social establishment for purposes other than research", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00833820", "title": "Repetitive Transcranial Magnetic Stimulation (rTMS) in Amyotrophic Lateral Sclerosis", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Catholic University of the Sacred Heart", "summary": "The investigators' preliminary studies demonstrated that repetitive transcranial magnetic stimulation (rTMS) of the brain may determine a slight slowing in the rate of disease progression in ALS patients (Di Lazzaro et al 2004, 2006). The aim of this study is to investigate whether rTMS of the motor cortex performed over a long period of time (12 months) in a group of patients with ALS, can have a more pronounced beneficial effect. The investigators will compare the disease progression in two groups of patients: the first group of patients will be treated with real rTMS (one week daily treatment every month) and the second group of patients will be treated with sham (placebo) rTMS.", "interventions": [{"type": "PROCEDURE", "name": "Real rTMS of the brain"}, {"type": "PROCEDURE", "name": "Sham rTMS of the brain"}], "start_date": "2007-05", "url": "https://clinicaltrials.gov/study/NCT00833820", "target_entities": ["motor_cortex_plasticity"], "locations": [{"facility": "Institute of neurology Universit\u00e0 Cattolica", "city": "Rome", "state": "", "country": "Italy", "status": "", "lat": 41.89193, "lon": 12.51133}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Eligible patients should have a diagnosis of definite ALS according to the El Escorial revised criteria with clear clinical upper and lower motor neuron signs.\n\nExclusion Criteria:\n\n* Contraindications to transcranial magnetic stimulation:\n\n  * Tracheostomy\n  * Pace-maker\n  * Implanted metallic devices\n  * Epilepsy", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "repetitive transcranial magnetic stimulation (rTMS)", "targeting_mechanism": "Non-invasive neuromodulation of motor cortex to slow disease progression", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03452618", "title": "Predictive Factors for the Diagnosis of Early Noninvasive Ventilation Equipment", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Toulouse", "summary": "To compensate for insufficiency of diagnostic tools, the present study propose to look for the predictive factors of an early fitting by noninvasive ventilation.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "diagnosis variables"}], "start_date": "2017-12-14", "url": "https://clinicaltrials.gov/study/NCT03452618", "target_entities": [], "locations": [{"facility": "University Hospital Toulouse", "city": "Toulouse", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.60426, "lon": 1.44367}], "contact_phone": "5 67 77 16 91", "contact_email": "dupuis.m@chu-toulouse.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of amyotrophic lateral sclerosis just performed,\n* Patient who do not have the criteria for fitting by NIV and who have a Vital capacity \u2265 70% of theoretical values\n\nExclusion Criteria:\n\n* Patient under court bail/ guardianship\n* Lack of consent for participation in the study\n* Presence of criteria for setting up the NIV for diagnosis according to the HAS (HealthCare Analytics Summit) 2006 recommendations\n* Vital capacity \\<70% of the theoretical values\n* Patient under Continuous Positive Pressure (CPP) or NIV for a respiratory pathology other", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06819124", "title": "Examining Interactions Between PALS and Caregivers", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Penn State University", "summary": "The goal of this clinical trial is to learn about the effect of communicative interaction on verbal communication in people with amyotrophic lateral sclerosis (ALS) and their caregivers.\n\nThe question is, What are the effects of communicative interaction on verbal communication in people with ALS when they interact with their caregivers and does this change over time?\n\nParticipants will read words and sentences while they are interacting with their caregivers.", "interventions": [{"type": "BEHAVIORAL", "name": "Structured Communicative Interaction"}, {"type": "BEHAVIORAL", "name": "Unstructured communicative interaction"}], "start_date": "2025-01-22", "url": "https://clinicaltrials.gov/study/NCT06819124", "target_entities": [], "locations": [{"facility": "Speech Core, Pennsylvania State University", "city": "University Park", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 40.80201, "lon": -77.85639}], "contact_phone": "814-867-3373", "contact_email": "ajo150@psu.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\nSpeakers with amyotrophic lateral sclerosis (ALS) (PALS-people with ALS)\n\n* diagnosis of ALS following the revised EL Escorial criteria\n* no history of other neurological conditions (e.g., stroke)\n* no cognitive impairment assessed by Telephone Montreal Cognitive Assessment (mini MoCA)\n* detectable speech disturbance according to the ALS Functional Rating Scale-Revised (ALSFRS-R)\n* the ability to produce single words\n* being a native speaker of American English (AE).\n\nCaregivers\n\n* being a caregiver of a participant with ALS\n* being a native speaker of American English (AE).\n\nExclusion Criteria:\n\n\\- None - if volunteer meets the inclusion criteria, then they will be enrolled", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04494256", "title": "A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation", "phase": "PHASE1, PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogen", "summary": "The ALSpire Study is a clinical trial evaluating the investigational drug BIIB105 in adults living with amyotrophic lateral sclerosis (ALS).\n\nThe ALSpire Study consists of two parts:\n\n* Part 1: 6-month placebo-controlled study. During Part 1, participants are randomly assigned to receive either BIIB105 or placebo in a 3:1 or 2:1 ratio (depending on the participant's assigned Cohort).\n* Part 2: up to 3-year long-term open-label extension. During Part 2, all participants receive BIIB105.\n\nThe objectives of the study are to evaluate:\n\n* The safety and tolerability of BIIB105 in people with ALS\n* What the body does to BIIB105 (also called \"pharmacokinetics\")\n* What BIIB105 does to the body (also called \"pharmacodynamics\")\n* Whether BIIB105 can slow the worsening of clinical function", "interventions": [{"type": "DRUG", "name": "BIIB105"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2020-09-28", "url": "https://clinicaltrials.gov/study/NCT04494256", "target_entities": ["ATXN2", "biib105"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California San Diego Medical Center", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Stanford Neuromuscular Research Center", "city": "Palo Alto", "state": "California", "country": "United States", "status": "", "lat": 37.44188, "lon": -122.14302}, {"facility": "University of Colorado Hospital - Neuroscience Center -Anschutz Medical Campus", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Georgetown University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Orlando Health", "city": "Orlando", "state": "Florida", "country": "United States", "status": "", "lat": 28.53834, "lon": -81.37924}, {"facility": "The Emory Clinic", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "ALS Clinic - Department of Neurology, Neuromuscular Division, Johns Hopkins University, School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Washington University, School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Houston Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "Montreal Neurological Institute-Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "A.O.U. Citt\u00e0 della salute e della scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\nPart 1:\n\n* Ability of the participant to understand the purpose and risks of the study and indicate informed consent, and the ability of the participant or the participant's legally authorized representative, to provide signed and dated informed consent and authorization to use protected health information in accordance with national and local privacy regulations.\n* No known presence or family history of mutations in the superoxide dismutase 1 (SOD1) or fused in sarcoma (FUS) genes.\n* Participants in Cohorts A, B, C1 and D1, must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria (revised according to the Airlie House Conference 1998 \\[Brooks 2000\\]). Participants in Cohort C2 and D2, must meet any of the prior conditions, but may also only meet clinically possible criteria for diagnosing ALS, or exhibit weakness attributable to ALS in the presence of ataxin-2 protein (ATXN2) intermediate repeats.\n* In participants in Cohorts C2 and D2, confirmed intermediate cytosine-adenine-guanine/cytosine-adenine-adenine (CAG/CAA) repeat expansion in the ataxin-2 (ATXN2) gene as defined by at least 1 allele carrying 30 to 33 CAG/CAA repeats.\n* Slow vital capacity (SVC) criteria:\n* In participants in Cohorts A, B, C1, and D1, SVC \u226560% of predicted value as adjusted for sex, age, and height (from the sitting position).\n* In participants in Cohort C2 and D2, SVC \u226550% of predicted value as adjusted for sex, age, and height (from the sitting position).\n* If taking riluzole, participant must be on a stable dose for \u226530 days prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that it should be discontinued for medical reasons, in which case it may not be restarted during the study.\n* Participants taking concomitant edaravone at study entry must be on a stable dose for \u226560 days prior to the first dose of study treatment (Day 1). Participants taking concomitant edaravone must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this study.\n* Screening values of coagulation parameters including platelet count, international normalized ratio (INR), prothrombin time (PT), and activated partial thromboplastin time (aPTT) should be within normal ranges.\n* Has an informant/caregiver who, in the Investigator's judgment, has frequent and sufficient contact with the participant as to be able to provide accurate information about the participant's cognitive and functional abilities at screening.\n\nPart 2:\n\n* Ability of the participant to understand the purpose and risks of the study and indicate informed consent, and the ability of the participant or the participant's legally authorized representative to provide signed and dated informed consent and authorization to use protected health information in accordance with national and local privacy regulations\n* Participants must have completed Study NCT04494256 Part 1 through Week 25 (Day 175 Visit for Cohorts A, B, C1, C2; Day 176 Visit for Cohorts D1, D2). This inclusion criterion does not apply to a participant if Part 1 was terminated by the Sponsor before the participant reached Week 25.\n* Participants from Cohorts A, B, C1, and C2 must have a washout of \u226516 weeks between the last dose of study treatment received in Study NCT04494256 Part 1 and the first dose of BIIB105 received in Study NCT04494256 Part 2. Participants from Cohorts D1 and D2 do not require a washout period.\n* If taking riluzole, participant must be on a stable dose for \u226530 days prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that it should be discontinued for medical reasons, in which case it may not be restarted during the study.\n* Participants taking concomitant edaravone at study entry must be on a stable dose for \u226560 days prior to the first dose of study treatment (Day 1). Participants taking concomitant edaravone must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this study.\n* Screening values of coagulation parameters including platelet count, INR, PT, and aPTT should be within normal ranges.\n\nKey Exclusion Criteria\n\nPart 1:\n\n* History or positive test result at Screening for human immunodeficiency virus (HIV).\n* Current hepatitis C infection.\n* Current hepatitis B infection.\n* History of alcohol or substance abuse \u22646 months of Screening that would limit participation in the study, as determined by the Investigator.\n* Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.\n* Presence of tracheostomy.\n* In participants from Cohorts A, B, C1, and D1, history of myocardial infarction, as determined by the Investigator.\n* In participants from Cohorts A, B, C1, and D1, poorly controlled type 1 or 2 diabetes mellitus defined as hemoglobin A1c (HbA1c) \u22658% during Screening.\n* In participants in Cohorts A, B, and C1, prescreening ALSFRS-R slope \\>-0.4 points/month, where prescreening ALSFRS-R slope is defined as: (ALSFRS-R score at Screening - 48) / (months from date of symptom onset to date of Screening). This criterion is not applicable for Cohorts C2, D1, and D2.\n* Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs) or biological agent within 1 month or 5 half-lives of study agent, whichever is longer, before Screening.\n* Treatment with an approved disease-modifying therapy for ALS other than riluzole or edaravone within 1 month or 5 half-lives of therapy, whichever is longer, before completion of screening.\n* Treatment with an antiplatelet or anticoagulant therapy that cannot safely be interrupted for lumbar puncture (LP) according to local standard of care and/or institutional guidelines, in the opinion of the Investigator or Prescriber.\n* Female participants who are pregnant or currently breastfeeding and those intending to become pregnant during the study.\n\nPart 2:\n\n* History or positive test result at Screening for HIV. If participants from Cohorts D1 and D2 who would seamlessly roll from Part 1 into Part 2 test positive for HIV during screening for Part 2 but are clinically asymptomatic, they may enroll in Part 2 at the discretion of the Investigator.\n* Current hepatitis C infection. If participants from Cohorts D1 and D2 who would seamlessly roll from Part 1 into Part 2 test positive for hepatitis C during screening for Part 2 but are clinically asymptomatic, they may enroll in Part 2 at the discretion of the Investigator.\n* Current hepatitis B infection. If participants from Cohorts D1 and D2 who would seamlessly roll from Part 1 into Part 2 test positive for hepatitis B during screening for Part 2 but are clinically asymptomatic, they may enroll in Part 2 at the discretion of the Investigator.\n* History of alcohol or substance abuse \u2264 6 months of Screening that would limit participation in the study, as determined by the Investigator.\n* Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system during the study period.\n* In participants from Cohorts A, B, C1, and D1, history of myocardial infarction, as determined by the Investigator.\n* In participants from Cohorts A, B, C1, and D1, poorly controlled type 1 or 2 diabetes mellitus defined as HbA1c \u22658% during Screening.\n* Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs; excluding BIIB105) or biological agent within 1 month or 5 half-lives of study agent, whichever is longer, before Screening.\n* Treatment with an antiplatelet or anticoagulant therapy that cannot safely be interrupted for LP according to local standard of care and/or institutional guidelines, in the opinion of the Investigator or Prescriber.\n* Female participants who are pregnant or currently breastfeeding and those intending to become pregnant during the study.\n\nNOTE: Other protocol defined Inclusion/Exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "BIIB105", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06527222", "title": "A Study of Ranolazine in ALS", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Swathy Chandrashekhar, MBBS", "summary": "The purpose of this study is to evaluate safety, effect on cramps, function and quality of life of ranolazine versus placebo for the treatment of ALS.", "interventions": [{"type": "DRUG", "name": "Ranolazine"}, {"type": "DRUG", "name": "Ranolazine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2025-04-29", "url": "https://clinicaltrials.gov/study/NCT06527222", "target_entities": ["muscle_cramps"], "locations": [{"facility": "University of California, San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Kansas Medical Center", "city": "Fairway", "state": "Kansas", "country": "United States", "status": "RECRUITING", "lat": 39.02223, "lon": -94.6319}, {"facility": "University of Kansas Medical Center: Wichita", "city": "Wichita", "state": "Kansas", "country": "United States", "status": "RECRUITING", "lat": 37.69224, "lon": -97.33754}, {"facility": "University of Missouri Health Care", "city": "Columbia", "state": "Missouri", "country": "United States", "status": "RECRUITING", "lat": 38.95171, "lon": -92.33407}, {"facility": "The Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 39.96118, "lon": -82.99879}], "contact_phone": "913-945-9932", "contact_email": "Kjennens2@kumc.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* 18 years or older\n* Diagnosed with clinically definite, possible, probably, or lab-supported probable ALS per revised El Escorial criteria\n* Breathing assessment called forced vital capacity (FVC) greater than or equal to 50%.\n* Able to swallow pills at the start of the study and expected to for the length of the study.\n* If on ALS modifying medications must be on a stable dose at least 30 days.\n* Experiencing 4 or more cramps per week during a 2-week screening period.\n\nExclusion Criteria:\n\n* Disease duration \\< 5 years\n* Tracheostomy invasive ventilation, or noninvasive ventilation of more than 12 hours/day\n* Pregnant or lactating, adults unable to consent, and prisoners\n* Taking ranolazine or investigational drug or has received an investigational drug within 30 days (or 5 half-lives for drugs, whichever is longer) prior to screening\n* Medically uncontrolled comorbidities (heart, liver, kidney disease)\n* Baseline QTc interval prolongation \\>450 ms for men/ \\>470 ms for women, history of long QT syndrome, or medications which prolong the QT interval\n* Participation in an experimental drug trial less than 30 days before screening\n* Patients have to be on a stable dosage of any medications used to treat muscle cramps for \u226530 days or have been off these medications \u226530 days prior to randomization.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Ranolazine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05983211", "title": "Quantitative and Repetitive TMS in ALS - Recruiting for Stage 2", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sunnybrook Health Sciences Centre", "summary": "Stage 1 \\[Enrolment closed\\]:\n\nThe goal of this open-label pilot clinical trial is to evaluate the safety and feasibility of accelerated, repetitive transcranial magnetic stimulation (rTMS) using continuous theta-burst stimulation (cTBS) in patients with ALS.\n\nStage 2 \\[CURRENTLY ENROLLING\\]:\n\nThe goal of this open-label pilot clinical trial is to evaluate the safety, tolerability and target engagement of accelerated, high dose cTBS using TMS in patients with ALS.", "interventions": [{"type": "DEVICE", "name": "Repetitive Transcranial Magnetic Stimulation"}], "start_date": "2024-05-01", "url": "https://clinicaltrials.gov/study/NCT05983211", "target_entities": ["motor_cortex_excitability"], "locations": [{"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}], "contact_phone": "(416) 480-6100", "contact_email": "alsresearch@sunnybrook.ca", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis with ALS as per the 2020 Gold Coast Criteria;\n* Age \u2265 18 years;\n* Able to provide informed consent to study procedures and treatments;\n* Patients are allowed to start or continue the standard of care treatments for ALS. Presently these include oral riluzole, and oral or intravenous edaravone;\n* Able to lie supine without BiPAP or breathing discomfort for at least 1 hour;\n* No contraindications to TMS as follow;\n* Metal implants in the head or neck (such as aneurysm clips, vessel stents), implanted medication pump, implanted brain stimulators, pacemaker, cochlear implants, or history of epilepsy. Dental fillings are permitted;\n* Current use of medications or medical conditions that, at the discretion of the Principal Investigator, could potentially increase the risk of seizures or interfere with stage outcomes;\n* On medications that affect TMS measures in a PRN regimen (as needed). Continuous use of these medications on a fixed dose of 30 days prior to first Baseline Visit or after a wash-out period of 2 weeks is accepted. These medications include, but are not limited to: benzodiazepines, muscle relaxants, tricyclic antidepressants, selective and non-selective serotonin reuptake inhibitors, hypnotics (including anti-histamine drugs) and anticholinergics drugs;\n* History of seizure, convulsion, or epilepsy;\n\nExclusion Criteria:\n\n* Known diagnosis of dementia;\n* Definitely or possibly pregnant (if applicable);\n* History of allergy to Ag-AgCl electrode gel (standard neurophysiology electrodes);\n* Unable to tolerate TMS procedures;\n* Lack of MRI brain performed prior to the stage, inability to perform an MRI at baseline due to orthopnea, or:\n* Large body habitus and not fitting comfortably into the scanner;\n* Difficulty laying still for up to 1 hour in the MRI unit or significant claustrophobia;\n* Metallic implants;\n* Any contraindications for receiving rTMS treatment as follow:\n* have received rTMS for any previous indication due to the potential compromise of subject blinding;\n* have increased intracranial pressure, a space occupying brain lesion, any history of seizure except those therapeutically induced by ECT or a febrile seizure of infancy, significant head trauma with clear radiological evidence of cerebrovascular injury on imaging;\n* have an intracranial implant or any other metal object within or near the head, excluding the mouth, that cannot be safely removed;\n* have clinically significant laboratory abnormality, in the opinion of the one of the principal investigators or study physicians;\n* are currently taking more than lorazepam 2 mg daily (or equivalent) or any dose of an anticonvulsant due to the potential to limit rTMS efficacy;\n* Any other clinical condition that, in the opinion of the Site Investigator, would place the subject at increased risk or preclude the subject's full compliance with completion of the stage.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Repetitive transcranial magnetic stimulation using continuous theta-burst stimulation (cTBS) to modulate motor cortex activity in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02058732", "title": "Therapeutic Imaging Biomarkers for Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "Study the cervical spinal cord and brain over time to assess changes and differences in subjects with amyotrophic lateral sclerosis.", "interventions": [{"type": "OTHER", "name": "MRI"}], "start_date": "2013-11", "url": "https://clinicaltrials.gov/study/NCT02058732", "target_entities": [], "locations": [{"facility": "University of Michigan Hospital", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. 18 years of age or older\n2. Subjects with amyotrophic lateral sclerosis -\n\nExclusion Criteria:\n\n1. Do not have opportunistic central nervous system infection\n2. Do not have a history of head injury\n3. Do not have cerebrovascular disease\n4. Do not have a contraindication for MRI(e.g. cardiac pacemaker, ferromagnetic or metallic implants).\n5. Must not be pregnant -", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04220021", "title": "Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Florida", "summary": "The primary objective is to assess the safety and tolerability of Metformin in subjects with C9orf72 amyotrophic lateral sclerosis administered for 24 weeks. The overall objective is to determine if Metformin is safe in C9orf72 ALS patients and is a potentially viable therapeutic treatment for C9-ALS that reduces repeat-associated non-canonical start codon - in DNA (non-ATG) (RAN) proteins that are produced by the C9orf72 repeat expansion mutation.", "interventions": [{"type": "DRUG", "name": "Metformin"}], "start_date": "2020-01-10", "url": "https://clinicaltrials.gov/study/NCT04220021", "target_entities": ["C9orf72"], "locations": [{"facility": "UF Health at the University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subjects have a diagnosis of probable or definite ALS in accordance with the Revisited El-Escorial Criteria.\n* Subjects have a likely diagnosis of chromosome 9 open reading frame 72 (C9orf72) positive ALS/FTD.\n* Subjects must be currently on an oral diet and able to take foods, pills and liquids by mouth equivalent to a score of 4 or above on the Functional Oral Intake Scale\n* Subjects must have no known allergy to barium sulfate or Metformin.\n* Subjects or subject's legally authorized representative must be willing and able to complete informed consent/assent and HIPAA authorization.\n* Ability to comprehend and be informed of the nature of the study, as assessed by the PI or Co-Investigators.\n* Subjects prescribed to take Metformin at or before the time of first dosing. (The study is open to subjects currently taking Metformin or subjects who have taken Metformin in the past).\n* Availability to participate for the entire study duration.\n* Female subjects of childbearing potential must have a negative urine pregnancy test prior to Videofluoroscopic Swallow Study (VFSS) exam during Visit 1, 3, and 4.\n\nExclusion Criteria:\n\n* Subjects who score 3 or below on the Functional Oral Intake Scale\n* Subjects who do not carry the C9ORF72 hexanucleotide repeat expansion as determined by laboratory analysis.\n* Subjects with a history of clinically significant liver disease, renal disease, or any other medical condition judged to be exclusionary by the investigator.\n* Subjects who are unwilling to sign informed consent or subjects who for any other reason in the judgment of investigator are unable to complete the study.\n* Female subjects who have a positive urine pregnancy test (\u03b2hCG) at screening or visit 1, are trying to become pregnant or are breastfeeding.\n* Subjects with active cancer within the previous 2 years, except treated basal cell carcinoma of the skin.\n* Subjects who have taken any experimental drug within 30 days prior to enrollment or within 5 half-lives of the investigational drug -whichever is the longer period.\n* Subjects with known history or presence of moderate or severe renal impairment as defined by an estimated glomerular filtration rate (eGFR) value below 30 mL/min/1.73 m2.\n* Subjects with hepatic impairment as defined by baseline elevations of serum aminotransferases greater than 5 times upper limit of normal or evidence of liver dysfunction (e.g., elevated bilirubin).\n* Use of potentially hepatotoxic drugs: (e.g., allopurinol, methyldopa, sulfasalazine).\n* Subjects with clinically significant abnormal laboratory values in the judgment of the investigator.\n* Subject with implanted electrical device (i.e. cardiac pacemaker or a neurostimulator), metal or metallic clip(s) in their body (i.e. an aneurysm clip in the brain) that will be damaged by participation in the MRI portion of the study.\n* Anything else that, in the opinion of the investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Metformin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02794857", "title": "Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuraltus Pharmaceuticals, Inc.", "summary": "This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo.", "interventions": [{"type": "DRUG", "name": "NP001"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2016-08-29", "url": "https://clinicaltrials.gov/study/NCT02794857", "target_entities": ["neuroinflammation"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Mayo Clinic Arizona", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California, Irvine, Department of Neurology", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Forbes Norris MDA/ALS Research Center, CPMC", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami Miller School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Emory University, Department of Neurology", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Kentucky, Albert B. Chandler Medical Center", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Clinical & Translational Science Institute, University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Carolinas Medical Center, Neurosciences Instutite-Neurology", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke Neurological Disorders Clinic at Morreene Road", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Cleveland Clinic Foundation-Cleveland Clinic Hospital", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "The Ohio State University Wexner Medical Center", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence Brain & Spine Institute, ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Houston Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Texas Health Sciences Center San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "Montreal Neurological Institute", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)\n* Forced vital capacity greater than or equal to 65% of that predicted for age and height\n* Onset of ALS-related weakness less than 3 years prior to first dose of study drug\n* Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening\n* Stable dose (greater than 30 days) of riluzole if undergoing treatment with this agent\n* For females: Not be of childbearing potential or agree to use adequate birth control during the study\n\nKey Exclusion Criteria:\n\n* Life expectancy of less than 6 months\n* Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)\n* Active pulmonary disease\n* Gastrostomy\n* Stem cell therapy\n* Immune modulator therapy or participation in studies of other agents within 12 weeks of pre-screening/screening\n* Unstable medical condition other than ALS", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NP001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03019419", "title": "Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Tokyo Medical University", "summary": "To investigate the safety and the efficacy of perampanel in patients with sporadic amyotrophic lateral sclerosis", "interventions": [{"type": "DRUG", "name": "Perampanel"}, {"type": "DRUG", "name": "placebo"}], "start_date": "2017-04-24", "url": "https://clinicaltrials.gov/study/NCT03019419", "target_entities": ["glutamate_excitotoxicity"], "locations": [{"facility": "Kitasato University East Hospital", "city": "Kanagawa", "state": "", "country": "Japan", "status": "", "lat": 37.58333, "lon": 139.91667}, {"facility": "Kumamoto Saishunso National Hospital", "city": "Kumamoto", "state": "", "country": "Japan", "status": "", "lat": 32.80589, "lon": 130.69181}, {"facility": "Nagoya University Hospital", "city": "Nagoya", "state": "", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "Okayama University Hospital", "city": "Okayama", "state": "", "country": "Japan", "status": "", "lat": 34.65, "lon": 133.93333}, {"facility": "Hokkaido University Hospital", "city": "Sapporo", "state": "", "country": "Japan", "status": "", "lat": 43.06667, "lon": 141.35}, {"facility": "Tohoku University Hospital", "city": "Sendai", "state": "", "country": "Japan", "status": "", "lat": 38.26667, "lon": 140.86667}, {"facility": "Shiga University of Medical Science Hospital", "city": "Shiga", "state": "", "country": "Japan", "status": "", "lat": 36.02247, "lon": 138.13005}, {"facility": "Tokyo Medical University", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "The University of Tokyo Hospital", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "Tokyo Metropolitan Neurological Hospital", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "University of Tsukuba Hospital", "city": "Tsukuba", "state": "", "country": "Japan", "status": "", "lat": 36.08333, "lon": 140.11667}, {"facility": "Yamaguchi University Hospital", "city": "Yamaguchi", "state": "", "country": "Japan", "status": "", "lat": 34.18333, "lon": 131.46667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "\\[Eligibility Criteria for Interim Registration\\]\n\n* Patients who are able to submit written informed consent. If patients are duly capable of study consent but are unable to sign by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation.\n* Patients who are male or female aged 40 years to 78 years at the time of obtaining informed consent\n* Patients who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised El Escorial Airlie House diagnostic criteria.\n* The sum of the 3 respiratory items of the ALSFRS-R must total 12 points or more\n* Patients within 2-year elapsed time period from disease onset at the time of obtaining informed consent\n* Patients who can visit study site for out-patient treatment\n\n\\[Eligibility Criteria for Registration\\]\n\nSubjects who meet the following criteria in addition to the inclusion criteria for the interim registration\n\n* The progression on score of ALSFRS-R during 12 weeks of observation period must be between -2 and -5\n* Patients who has not initiated newly introduced riluzole therapy after starting the observation period. Or those who has not received dose escalation or resumed administration of riluzole therapy after previous down titration or discontinuation\n* Patients who has not initiated newly introduced edaravone therapy after starting the observation period\n* Patients who are judged to be eligible for continuation of the study by the investigators\n\n\\[Exclusion Criteria\\]\n\n* Patients who underwent tracheostomy.\n* Patients who experienced non-invasive positive pressure ventilation.\n* Patients whose percent-predicted forced vital capacity (%FVC) is \u226480%.\n* Patients with progressive bulbar palsy type.\n* Patients with cognitive impairment, severe disease in the renal, cardiovascular, or hematological system.\n* Patients with hepatic disease.\n* Patients with malignant tumor.\n* Pregnant women or women with a possibility of becoming pregnant.\n* Patients who participated in another clinical study within 12 weeks before starting the observation period.\n* Patients who has initiated perampanel therapy in the past or at present.\n* Patients who are judged to be ineligible for study entry by the investigators.", "sex": "ALL", "min_age": "40 Years", "max_age": "78 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Perampanel", "targeting_mechanism": "AMPA receptor antagonist that blocks glutamate excitotoxicity.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02306109", "title": "Effect of Motor Rehabilitation Treatment on Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Azienda Unita' Sanitaria Locale Di Modena", "summary": "ErmoSLA is a multicentric, randomized, controlled trial to compare effects of an \"intensive\" or \"standard\" motor rehabilitation treatment on motor disability in people with ALS", "interventions": [{"type": "PROCEDURE", "name": "Standard motor rehabilitation treatment"}, {"type": "PROCEDURE", "name": "Intensive motor rehabilitation treatment"}], "start_date": "2015-01", "url": "https://clinicaltrials.gov/study/NCT02306109", "target_entities": [], "locations": [{"facility": "Department of Neuroscience, S. Anna Hospital", "city": "Ferrara", "state": "", "country": "Italy", "status": "", "lat": 44.83804, "lon": 11.62057}, {"facility": "Department of Neuroscience, S.Agostino-Estense Hospital", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Department of Neuroscience, IRCCS Arcispedale Santa Maria Nuova", "city": "Reggio Emilia", "state": "", "country": "Italy", "status": "", "lat": 44.69825, "lon": 10.63125}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of possible, probable or definite ALS according to the Revised El Escorial Criteria\n* Time from diagnosis \\<18 months at screening.\n* Forced vital capacity (FVC)\\> 50% at screening\n* Written informed consent\n\nPatients will be required to take the full dose of Riluzole, but not assuming Riluzole do not constitute a criterion for exclusion.\n\nExclusion Criteria:\n\n* Enrolment in any other clinical trial in the three months prior to screening\n* Tracheostomy or NIV for\\> 23h/day for 14 consecutive days at screening.\n* Diagnosis of severe neurodegenerative diseases in addition to the ALS\n* Diagnosis of severe heart disease, current neoplasia, any unstable medical condition that contraindicates an intensive rehabilitation treatment\n* State of pregnancy or breastfeeding\n* Residency outside Emilia-Romagna Region\n* Lack of multidisciplinary follow-up", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02525471", "title": "A Pilot Study of RNS60 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sabrina Paganoni, M.D.", "summary": "The purpose of this study is to determine the safety and tolerability of RNS60 in patients with Amyotrophic lateral sclerosis (ALS). Investigators will also measure the impact of RNS60 on several markers of neuro-inflammation, measured by blood biomarkers and positron emission tomography (PET) imaging.", "interventions": [{"type": "DRUG", "name": "RNS60"}], "start_date": "2015-10", "url": "https://clinicaltrials.gov/study/NCT02525471", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Amyotrophic Lateral Sclerosis (ALS) volunteers must be diagnosed as having possible, probable, probable-laboratory supported, or definite ALS, either sporadic or familial according to modified El Escorial criteria.\n* Age 18-80, able to provide informed consent, and comply with study procedures.\n* Participants must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to screening (riluzole-na\u00efve participants are permitted in the study).\n* Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control) for the duration of the study and 3 months after study completion.\n* Males should practice contraception for the duration of the study and 3 months after completion.\n* Ability to safely lie flat for 90 min for Positron Emission Tomography (PET) procedures in the opinion of the study physician.\n* High or mixed affinity to bind translocator (TSPO) protein (Ala/Ala or Ala/Thr)\n\nExclusion Criteria:\n\n* Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine transaminase (ALT) \\> 3 times the upper limit of the normal.\n* Renal insufficiency as defined by a serum creatinine \\> 1.5 times the upper limit of normal.\n* The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent, according to PI judgment.\n* Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if they were to participate in the study.\n* History of HIV, clinically significant chronic hepatitis, or other active infection.\n* Females must not be lactating or pregnant.\n* Active participation in another ALS clinical trial within 30 days of the Screening Visit\n* Exposure to immunomodulatory medications within 30 days of the Screening Visit.\n* Any contraindication to undergo MRI studies such as\n\n  * History of a cardiac pacemaker or pacemaker wires\n  * Metallic particles in the body\n  * Vascular clips in the head\n  * Prosthetic heart valves\n  * Claustrophobia\n* Radiation exposure that exceeds the site's current guidelines\n* Current use of tobacco products including cigarettes, cigars, snuff and chewing tobacco, or nicotine replacement products such as gum or patch", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RNS60", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01884571", "title": "Immunosuppression in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Emory University", "summary": "This is a multicenter, 15-month study evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale (Revised) (ALSFRS-R) score in up to 33 subjects with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Basiliximab"}, {"type": "DRUG", "name": "Methylprednisolone"}, {"type": "DRUG", "name": "Prednisone"}, {"type": "DRUG", "name": "Tacrolimus"}, {"type": "DRUG", "name": "Mycophenolate mofetil"}], "start_date": "2013-10", "url": "https://clinicaltrials.gov/study/NCT01884571", "target_entities": ["immune_system"], "locations": [{"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria for participants with symptom onset within the past 24 months:\n\n* Male or female patients 18-65 years of age.\n* ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial Criteria.\n* Symptom onset \u2264 24 months from screening visit.\n* A score of \u226538 on the Revised ALS Functional Rating Scale.\n* Slow vital capacity (SVC) measure \\>80% of predicted for gender, height and age at screening.\n* Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to the screening visit (riluzole-na\u00efve subjects are permitted in the study).\n* Negative tuberculosis (TB) test within 3 months of Screening Visit.\n* Subjects medically able to undergo lumbar puncture (LP) as determined by the investigator (i.e., no bleeding disorder, allergy to local anesthetics, or a skin infection at or near the LP site).\n* Capable of providing informed consent and following study procedures.\n* Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study.\n* Women of childbearing potential must have a negative pregnancy test at screening and be non-lactating.\n* Geographic accessibility to the study site.\n\nInclusion Criteria for participants with symptom onset greater than 24 months before screening:\n\n* Male or female patients age 18 or older.\n* ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial Criteria.\n* Symptom onset \\>24 months from screening visit.\n* Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to the screening visit (riluzole-na\u00efve subjects are permitted in the study).\n* Negative tuberculosis (TB) test within 3 months of Screening Visit.\n* Subjects medically able to undergo lumbar puncture (LP) as determined by the investigator (i.e., no bleeding disorder, allergy to local anesthetics, or a skin infection at or near the LP site).\n* Capable of providing informed consent and following study procedures.\n* Geographic accessibility to the study site.\n* Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study.\n* Women of childbearing potential must have a negative pregnancy test at screening and be non-lactating.\n\nExclusion Criteria\n\n* Prior use of basiliximab, solumedrol, prednisone, tacrolimus or mycophenolate mofetil within 30 days of the Screening Visit.\n* Known allergy or sensitivity to basiliximab, solumedrol, prednisone, tacrolimus or mycophenolate mofetil or a formulation of one of these drugs.\n* Treatment with an immunosuppressant medication within 30 days of the Screening Visit.\n* Active peptic ulcer disease.\n* Any medical disorder that would make immunosuppression contraindicated including, but not limited to, human immunodeficiency virus (HIV), tuberculosis, or evidence of active cytomegalovirus (CMV) or infection.\n* Subjects who have a diaphragm pacing system (DPS).\n* Women who are pregnant, breastfeeding, or planning to become pregnant in the next 12 months.\n* Active drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements.\n* Use of invasive or non-invasive mechanical ventilation (including Continuous Positive Airway Pressure (CPAP) or Bi-level Positive Airway Pressure (BiPAP)) for any part of the day or night prior to the Screening Visit (participants with symptom onset within past 24 months only).\n* Exposure to any other agent currently under investigation for the treatment of patients with ALS (off-label use or investigational) within 30 days of the Screening Visit.\n* Inability to safely complete study activities based on the discretion of the site investigator.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Basiliximab, Methylprednisolone, Prednisone, Tacrolimus, Mycophenolate mofetil", "targeting_mechanism": "Immunosuppression therapy targeting immune-mediated motor neuron degeneration.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07047209", "title": "Trial of Oral Digoxin in Individuals With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "This clinical trial is being conducted to learn about safety and tolerability of digoxin in ALS individuals. Additionally, this trial aims to better understand if digoxin has an effect on slowing neurodegeneration in ALS.", "interventions": [{"type": "DRUG", "name": "Digoxin"}], "start_date": "2025-05-27", "url": "https://clinicaltrials.gov/study/NCT07047209", "target_entities": ["Na/K-ATPase"], "locations": [{"facility": "Nova Southeastern University", "city": "Davie", "state": "Florida", "country": "United States", "status": "", "lat": 26.06287, "lon": -80.2331}, {"facility": "Northwestern Universsity", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "MGH", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Ability to provide written informed consent.\n2. Adults \\>18 years of age with a diagnosis of symptomatic ALS as determined by an ALS neurologist, and meets either the revised El Escorial Criteria (clinically possible, probable, probable lab-supported, or definite) or the Gold Coast Criteria.\n3. Available or pending CLIA certified ALS genetic panel report.\n4. Less than or equal to 24-months since onset of weakness attributed to ALS.\n5. Vital capacity (VC) of \\> 65% predicted value for gender, height and age at screening.\n6. Clinically unremarkable Complete Blood Counts, including but not limited to Hemoglobin \u2265 9 g/dL, Platelets \u2265 150 x 109 cells/L.\n7. No clinically significant abnormalities in the Comprehensive Metabolic Panel per site/sub-investigator's judgment, including but not limited to:\n\n   1. Serum alanine aminotransferase or aspartate aminotransferase \\< 3\u00d7 upper limit of normal, or serum total bilirubin \\<1.5\u00d7 upper limit of normal\n   2. Estimated GFR (eGFR) of \\> 30 mL/min/1.73m2\n   3. Other clinically significant electrolyte and metabolic abnormalities\n8. Ability and willingness to complete all study procedures per the Site Investigator's clinical assessment.\n9. Negative pregnancy test within 7 days prior to first dose for women of child-bearing potential (WOCB), defined as a sexually mature woman who has not undergone a hysterectomy or who has not been naturally postmenopausal for at least 24 consecutive months (i.e., who has had menses any time in the preceding 24 consecutive months).\n10. Individuals enrolling in the C9orf72 cohort of the trial must have CLIA certified ALS gene panel demonstrating \\>31 repeats of C9orf72 hexanucleotide repeat expansion, deemed pathologic.\n\nExclusion Criteria:\n\n* 1\\. Clinically significant unstable medical or surgical condition that would pose a risk to the participant's trial procedural participation or interfere with data collection, according to the Site Investigator's judgment (e.g., active infection requiring antibiotics).\n\n  2\\. Presence of cognitive or mental health disorders impairing ability to provide informed consent for the study per Site investigator assessment.\n\n  3\\. Active cancer or history of cancer, unless it was successfully treated for durable remission or cure more than 3 years ago. (Note that basal cell carcinoma, squamous cell carcinoma in situ, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies that have been curatively excised at any time previously and with no evidence of disease recurrence for at least 3 years are not exclusionary.) 4. Prior solid organ transplantation. 5. Concomitant use of investigational treatments for ALS within 5 half-lives or 30 days, whichever longer.\n\n  6\\. Screening 12-lead ECG showing QT interval corrected for rate (QTcF) \\> 470 msec for women and \\> 450 msec for men, absence of second degree or higher AV block or other clinically significant cardiac arrythmias.\n\n  7\\. If female, breastfeeding, pregnant, or of child-bearing potential and unwilling to use effective contraception for duration of the trial and after discontinuing treatment.\n\n  8\\. Serious cardiac condition within the last 6 months, such as uncontrolled arrhythmia, myocardial infarction, unstable angina, or heart disease defined by the New York Heart Association (NYHA) Class III or Class IV or hereditary long QT syndrome.\n\n  9\\. Exclusion criteria for Lumbar Punctures: active bleeding tendencies OR inability to withhold antiplatelets or anticoagulants safely as per institutional guidelines around LPs OR anatomical spine considerations making it unsafe or challenging for serial LPs OR allergy to local anesthesia used for procedure.\n\n  10\\. Clinically active cardiac disorders including sinus bradycardia (HR \\<40 bpm) or sinus tachycardia (HR\\>140 bpm), cardiac arrythmias \\[first-degree, second-degree (Wenckebach), or third-degree heart block; atrial tachycardia with block; AV dissociation; accelerated junctional (nodal) rhythm; unifocal or multiform ventricular premature contractions (especially bigeminy or trigeminy); ventricular tachycardia; and ventricular arrythmias\\]; Persistent or chronic atrial fibrillation that is not controlled using standard medications 11. Concomitant treatment with amiodarone at any dose or quinidine at a dose greater than 20 mg/day", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Digoxin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07726563", "title": "Plasmapheresis in Amyotrophic Lateral Sclerosis With Autoantibody Against NRIP 2 (PALADIN2)", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Taiwan University Hospital", "summary": "10-20% of patients with ALS have anti-NRIP autoantibody and the titer of anti-NRIP autoantibody is correlated with motor functional decline and mortality in ALS. The PALADIN2 clinical trial is a single arm study, which intends to enroll 20 ALS patients having anti-NRIP autoantibody in plasma. Patients will receive 3 courses of plasmapheresis per 3 months in order to maintain low concentration of anti-NRIP autoantibody in plasma. The study will follow up these patients for another 6 months after plasmapheresis. This project will potentially confirm the efficacy and safety of plasmapheresis for ALS patients having anti-NRIP autoantibody.", "interventions": [{"type": "DEVICE", "name": "plasmapheresis"}], "start_date": "2026-05-19", "url": "https://clinicaltrials.gov/study/NCT07726563", "target_entities": ["NRIP1"], "locations": [{"facility": "National Taiwan University Hospital", "city": "Taipei City", "state": "Taipei", "country": "Taiwan", "status": "RECRUITING", "lat": null, "lon": null}], "contact_phone": "+886972651560", "contact_email": "milikai@ntuh.gov.tw", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS patients above 20-year-old who have anti-NRIP autoantibody in plasma\n* Agree to receive plasmapheresis treatment\n* Agree to participate in the study and receive serial examinations\n\nExclusion Criteria:\n\n* Under permanent ventilator support\n* Cannot receive plasmapheresis treatment or serial examinations\n* Under pregnancy\n* Blood fibrinogen level below 50 mg/dl\n* Belong to special subtype of ALS, such as primary lateral sclerosis, progressive muscular atrophy, flail arm syndrome, flail leg syndrome.", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "plasmapheresis", "targeting_mechanism": "Removal of anti-NRIP autoantibodies from plasma to reduce their circulating concentration and associated motor neuron degeneration.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02851914", "title": "SSRIs vs. TCAs for Depression in ALS Patients", "phase": "EARLY_PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "St. Louis University", "summary": "Depression is seen in 9-11% of ALS patients and adequate and proper treatment is needed. In this study, ALS patients will be screened for depression using self-reported multiple choice questionnaire. Patients who fulfill the criteria for depression based on this screening tool will be evaluated by psychiatrist before inclusion in the study. The investigators will also measure quality of life and functional status by simple questionnaires. The patients will be allocated into two treatment groups to receive either TCA or SSRI for 12 weeks. Patients will be evaluated every 4 weeks and phone calls will be made in between the visits if needed to assess about efficacy and any side effects. If any patient reports having suicidal thoughts on any of these phone calls or clinic visits, he/she will be immediately sent to the ER for appropriate management. The investigators will repeat the questionnaires in the clinic visits, and use them in the data analysis to look for any improvement and to compare the two medication classes used in this study. This data may be used later on to do larger studies and help to make standard recommendations in treating depression in ALS patients.", "interventions": [{"type": "DRUG", "name": "Tricyclic Antidepressants (\"TCA\")"}, {"type": "DRUG", "name": "Selective Serotonin Uptake Inhibitors (\"SSRI\")"}], "start_date": "2015-07-21", "url": "https://clinicaltrials.gov/study/NCT02851914", "target_entities": ["serotonin_reuptake"], "locations": [{"facility": "Monteleone Hall, Saint Louis University, 1438 South Grand Blvd.", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Documented diagnosis of definite or probable ALS\n* Informed and written consent for enrollment in study\n* Gender: both male and female\n* Age: 25-80 years\n* BDI score 19 or above\n* Depression diagnosis by mental health provider\n\nExclusion Criteria:\n\n* History of psychotic disorder, premorbid bipolar depression\n* ALS-FRS score \\< 26\n* Cognitive impairment\n* Currently on SSRIs or TCAs. However if for some reason they are off their treatment, then they can be enrolled in the study after a washout period of 30 days.\n* Currently on other antidepressants such as monoamine oxidase inhibitors (MAOIs), selective norepinephrine re-uptake inhibitors (SNRIs) etc.", "sex": "ALL", "min_age": "25 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tricyclic Antidepressants and Selective Serotonin Uptake Inhibitors", "targeting_mechanism": "Modulation of serotonin and norepinephrine reuptake to treat depression in ALS patients.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01424163", "title": "Dexpramipexole Japanese PK Study", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "This is a single and multiple dose, open-label study to evaluate the pharmacokinetics (PK), safety, and tolerability of dexpramipexole administered orally to adult Japanese and Caucasian healthy subjects.", "interventions": [{"type": "DRUG", "name": "Single dose reduced"}, {"type": "DRUG", "name": "Single dose standard"}, {"type": "DRUG", "name": "Multiple Dose"}, {"type": "DRUG", "name": "Multiple Dose"}], "start_date": "2011-08", "url": "https://clinicaltrials.gov/study/NCT01424163", "target_entities": ["dexpramipexole"], "locations": [{"facility": "Research Site", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subjects who are able and willing to give written informed consent.\n* Adult Japanese and Caucasian males/females aged 18 to 60 years inclusive and between 18 and 30 kg/m2 body mass index (BMI), inclusive.\n* Male and female subjects will be enrolled on the study. Male subjects and female subjects of childbearing potential, must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment\n* Japanese subjects must be born in Japan and have both parents and four grandparents of Japanese descent.\n* Japanese subjects must have lived outside of Japan for no more than 5 years.\n* Japanese subjects must not have significant changes with regard to diet; i.e., their diet must not have significantly changed since leaving Japan.\n* Caucasian subjects will be matched individually (on a 1:1 basis) to Japanese subjects with respect to gender and age, and if possible BMI.\n\nExclusion Criteria:\n\n* Subjects who do not conform to the above inclusion criteria.\n* Female subjects who are pregnant, trying to become pregnant or lactating.\n* Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, hematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders.\n* Subjects who have a clinically relevant surgical history.\n* Subjects who have previously received dexpramipexole or pramipexole.", "sex": "ALL", "min_age": "18 Years", "max_age": "60 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "dexpramipexole", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05357950", "title": "A Phase IIb, Multi-Center, Multinational, Double-Blind, Placebo-Controlled Study, With an Open Label Extension, to Evaluate Safety, Tolerability and Efficacy of PrimeC in Subjects With ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "NeuroSense Therapeutics Ltd.", "summary": "69 subjects with ALS will be enrolled in the study and randomized at a 2:1 ratio to receive the study drug or placebo tablets. Randomization sequences will be in random block sizes and stratified for ENCALS risk category \\[high risk \u2265 -4.5 vs. low risk \\< -4.5\\], and for background ALS treatment (riluzole and/or edaravone and/or sodium phenylbutyrate and/or taurursodiol) vs. no background ALS treatment.\n\nAll subjects will be administered the drug/placebo twice daily (BID), two tablets each time, for 6 months. Subjects will be allowed to receive standard of care (SOC) treatment of approved products (i.e., riluzole and edaravone). Additionally, subjects will be allowed to receive treatment with off-label sodium phenylbutyrate and taurursodiol, which are accepted for ALS treatment.\n\nSubjects will be evaluated every 2 months for safety, tolerability (adverse events, safety laboratory, vital signs, ECG, withdrawal rates and reasons) and efficacy (e.g. biomarkers, clinical outcomes (ALSFRS-R and SVC, quality of life and survival).\n\nAll subjects who complete the 6 months dosing will be switched to the active arm for a 12-month open label extension (OLE).", "interventions": [{"type": "DRUG", "name": "PrimeC"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2022-05-31", "url": "https://clinicaltrials.gov/study/NCT05357950", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Lawson Health Research Institute", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Tel Aviv Sourasky Medical Center", "city": "Tel Aviv", "state": "", "country": "Israel", "status": "", "lat": 32.08088, "lon": 34.78057}, {"facility": "IRCCS Istituti clinici Maugeri", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "A.O.U. Citta della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to comprehend and willing to sign an informed consent form (ICF)\n2. Males or females between the ages of 18 and 75 years of age, inclusive\n3. Diagnosis of familial or sporadic ALS (defined as meeting the laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the Gold Coast criteria)\n4. Disease duration after first symptom (muscle weakness) less than 30 months prior to screening\n5. Pre-enrollment ALSFRS-R slope from disease onset \u2265 0.3 points per month\n6. ALSFRS-R at screening \u2265 25\n7. Item 3 (swallowing) in ALSFRS-R \u2265 3\n8. Subjects may be treated in parallel with riluzole and/or edaravone and/or sodium phenylbutyrate and/or taurursodiol; 60 days of stable use prior to enrollment is required\n9. Upright slow vital capacity (SVC) \u2265 60% of predicted for age, height, weight and sex at screening according to the GLI-2012\n10. 18 \\< BMI \\< 30\n11. A caregiver (if one is needed)\n12. Female subjects must be post-menopausal (\u2265 1 year) OR sterilized, OR if of childbearing potential (i.e., females who have had their first period unless they are anatomically or physiologically incapable to become pregnant), must have a negative pregnancy test, and agree to use contraceptive drugs or devices (e.g., diaphragm plus spermicide, or oral contraceptives) for the duration of the study and 10 weeks after the last treatment dose AND require male partners to use a condom during sexual intercourse\n\nExclusion Criteria:\n\n1. A past history of adverse reaction/hypersensitivity to either NSAIDs, celecoxib or fluoroquinolones, ciprofloxacin\n2. Any known clinically significant abnormal gastric mucosal erosion, ulcer or tumor or/and GI disorder and/or bariatric surgery\n3. Known history of clinically significant impairment of renal function (creatinine \u2265 1.5)\n4. Known or suspected symptomatic congestive heart and/or coronary heart disease, previous history of myocardial infarction, uncontrolled arterial hypertension, or rhythm abnormalities requiring permanent treatment\n5. Known history of QT/QTc prolongation, Torsade de pointes (TdP) (e.g. heart failure, hypokalemia, family history of Long QT syndrome) and the use of concomitant medications that prolong the QT/QTc interval\n6. Known or suspected diagnosis or family history of epilepsy in first degree relatives\n7. Known predisposition to tendinitis\n8. Known or suspected to be a poor CYP2C9 metabolizers who also uses pharmacologic agents (prescription or over-the-counter) or herbal products known or suspected to induce or inhibit CYP2C9 within 30 days before enrollment\n9. Tracheostomy or percutaneous gastrostomy use\n10. Presence at screening of any medically significant cardiac, pulmonary, musculoskeletal, or psychiatric illness that might interfere with the subject's ability to comply with study procedures or that might confound the interpretation of clinical safety data, including, but not limited to:\n\n    1. Mean systolic blood pressure \\>160 mm Hg and/or mean diastolic blood pressure \\>100 mm Hg (measurements taken after a few minutes rest) that persist on 3 successive measurements taken at least 2 minutes apart\n    2. NYHA Class II or greater congestive heart failure\n    3. Chronic obstructive pulmonary disease or asthma requiring daily use of bronchodilator medications\n    4. Poorly controlled or brittle diabetes mellitus\n    5. Cognitive impairment, related to ALS or otherwise, sufficient to impair subject's ability to understand and/or comply with study procedures and provide informed consent\n11. Subject who is treated with chronic aspirin or NSAIDs and is at risk if stopped. Clopidogrel is allowed and can replace Aspirin.\n12. Any contraindication for ciprofloxacin and celecoxib according to the current prescribing information.\n13. Female who is pregnant or breastfeeding or with intention of becoming pregnant during the course of the study.\n14. Any impairment or social circumstance that, in the opinion of the Investigator, would render the subject not suitable to participate in the study.\n15. Subject, or subject's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.\n16. Subject is participating in (or plans to participate in) any other investigational drug trial, or plans to be exposed to any other investigational agent, device and/or procedure, from 30 days prior to Screening through study completion.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "PrimeC", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05724173", "title": "Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Leigh R. Hochberg, MD, PhD.", "summary": "The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tetraplegia to control a computer cursor and other assistive devices with their thoughts.", "interventions": [{"type": "DEVICE", "name": "BrainGate Neural Interface System"}], "start_date": "2023-10-18", "url": "https://clinicaltrials.gov/study/NCT05724173", "target_entities": [], "locations": [{"facility": "Stanford University School of Medicine", "city": "Stanford", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.42411, "lon": -122.16608}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "617-724-9247", "contact_email": "lhochberg@mgh.harvard.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Between 18 and 80 years of age.\n* Participants must have a diagnosis of amyotrophic lateral sclerosis (ALS) as verified by a clinical expert in neurologic diseases.\n* Participants with a diagnosis of ALS with anarthria, or severe dysarthria with decline in the preceding four months.\n* Must be within a three-hour drive of the Study site and geographically stable for at least 15 months after enrollment.\n\nExclusion Criteria:\n\n* Visual impairment such that extended viewing of a computer monitor would be difficult even with ordinary corrective lenses\n* Chronic oral or intravenous steroids or immunosuppressive therapy\n* Other serious disease or disorder that could seriously affect ability to participate in the study\n\n(There are additional exclusion criteria)", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "BrainGate Neural Interface System", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05949294", "title": "Study of AROSOD-1 in Adult Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Arrowhead Pharmaceuticals", "summary": "In this Phase 1 adult symptomatic patients with amyotrophic lateral sclerosis (ALS) carrying a superoxide dismutase 1 (SOD1) gene mutation thought to be causative of ALS, will receive single ascending doses of ARO-SOD1 administered by intrathecal (IT) infusion. The study is primarily intended to evaluate safety, but will also evaluate the effect of ARO-SOD1 on SOD1 cerebrospinal fluid (CSF) levels as a biomarker of pharmacodynamic (PD) effect, therefore lumbar punctures will be required at timepoints throughout the study. After each participant has completed their individual final visit, participants whose SOD1 CSF levels have recovered to a satisfactory level may rescreen and enroll into higher dose cohorts; or if unable or unwilling to rescreen may enroll into an open-label study to be added by amendment when supported by nonclinical data for multidose administration.", "interventions": [{"type": "DRUG", "name": "ARO-SOD1 Injection"}], "start_date": "2024-01", "url": "https://clinicaltrials.gov/study/NCT05949294", "target_entities": ["SOD1"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of ALS based on source-verifiable medical record and meets the Gold Coast Criteria\n* Pathogenic or likely pathogenic SOD1 mutation based on source-verifiable medical records or genetic testing during Screening\n* Slow Vital Capacity (SVC) \u2265 50% of predicted value adjusted for sex, age, and height (from a sitting position) at Screening\n* Body Mass Index (BMI) \u2265 18.0 kg/m2 at Screening\n* Able to complete at least 6 months of follow-up\n* If taking any medication or supplement to treat ALS or ALS symptoms, must be on stable dose for \u2265 4 weeks prior to Day 1 and expected to remain at that dose until final study visit and not expected to start these medications after the first dose of ARO-SOD1\n* Able and willing to provide written informed consent and to comply with all study assessments\n* Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Males must not donate sperm and females must not donate eggs during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later.\n\nExclusion Criteria:\n\n* Current or anticipated need of a diaphragm pacing system (DPS) during the study\n* Participants using tofersen can be enrolled only after a washout period of approximately 20 weeks from the last tofersen dose to the first planned dose of ARO-SOD1\n* History of having received stem cell therapy for ALS treatment\n* Any current or anticipated contraindications to lumbar puncture (LP)\n* The presence of an implanted shunt for drainage of CSF or an implanted central nervous system (CNS) catheter\n* Human immunodeficiency virus (HIV), seropositive for hepatitis B virus (HBV), seropositive for hepatitis C virus (HCV)\n* Uncontrolled hypertension\n* Severe cardiovascular disease\n* History of drug abuse or alcoholism within 6 months of study enrollment\n* Inability to comply with study requirements\n\nNote: additional inclusion/exclusion criteria may apply per protocol", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ARO-SOD1", "targeting_mechanism": "Reduces SOD1 mRNA and protein levels in patients with SOD1-mediated ALS by delivering a microRNA targeting SOD1 via intrathecal infusion.", "targeting_mechanism_pmid": "32640133", "animal_results": "Spinal cord SOD1 levels were lower in treated patients compared to untreated SOD1-mediated ALS patients; AAV9-delivered shRNA-SOD1 silencing vectors transduced motor neurons and glial cells throughout the spinal cord and delayed paralysis in SOD1G37R ALS mice; AAV-delivered artificial microRNA extended survival and delayed paralysis in ALS mouse models.", "animal_results_pmid": "32640133", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05193994", "title": "Triumeq in Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Macquarie University, Australia", "summary": "To determine if Triumeq improves survival in Amyotrophic Lateral Sclerosis (ALS) compared with placebo", "interventions": [{"type": "DRUG", "name": "Dolutegravir, Abacavir and Lamivudine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2022-02-24", "url": "https://clinicaltrials.gov/study/NCT05193994", "target_entities": ["Integrase"], "locations": [{"facility": "MQ Health Neurology", "city": "North Ryde", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.79677, "lon": 151.12436}, {"facility": "Neuroscience Research Australia (NeuRA)", "city": "Randwick", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.91439, "lon": 151.24895}, {"facility": "Sunshine Coast University Hospital", "city": "Birtinya", "state": "Queensland", "country": "Australia", "status": "", "lat": -26.74322, "lon": 153.11913}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Flinders Medical Centre", "city": "Bedford Park", "state": "South Australia", "country": "Australia", "status": "", "lat": -35.02204, "lon": 138.56815}, {"facility": "Launceston General Hospital", "city": "Launceston", "state": "Tasmania", "country": "Australia", "status": "", "lat": -41.43876, "lon": 147.13467}, {"facility": "Calvary Health Care Bethlehem", "city": "Caulfield South", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.89562, "lon": 145.02597}, {"facility": "The Perron Institute", "city": "Nedlands", "state": "Western Australia", "country": "Australia", "status": "", "lat": -31.98184, "lon": 115.8073}, {"facility": "Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Christchurch Hospital", "city": "Christchurch", "state": "", "country": "New Zealand", "status": "", "lat": -43.53333, "lon": 172.63333}, {"facility": "Dunedin Hospital", "city": "Dunedin", "state": "", "country": "New Zealand", "status": "", "lat": -45.87416, "lon": 170.50361}, {"facility": "Clinical Trials Unit, Tauranga Hospital", "city": "Tauranga", "state": "", "country": "New Zealand", "status": "", "lat": -37.68611, "lon": 176.16667}, {"facility": "Wellington Regional Hospital", "city": "Wellington", "state": "", "country": "New Zealand", "status": "", "lat": -41.28664, "lon": 174.77557}, {"facility": "Univerzitetni klini\u010dni center Ljubljana", "city": "Ljubljana", "state": "", "country": "Slovenia", "status": "", "lat": 46.05108, "lon": 14.50513}, {"facility": "Hospital del Mar", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario y Politecnico la Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Studiecenheten at Akademiskt specialistcentrum", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "University of Edinburgh, Anne Rowling Regenerative Nuerology Clinic", "city": "Edinburgh", "state": "", "country": "United Kingdom", "status": "", "lat": 55.95206, "lon": -3.19648}, {"facility": "The Walton Centre", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "University College London Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "King's College Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "St George's Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Oxford University Hospital", "city": "Oxford", "state": "", "country": "United Kingdom", "status": "", "lat": 51.75222, "lon": -1.25596}, {"facility": "Plymouth University Hospital", "city": "Plymouth", "state": "", "country": "United Kingdom", "status": "", "lat": 50.37153, "lon": -4.14305}, {"facility": "Royal Preston Hospital", "city": "Preston", "state": "", "country": "United Kingdom", "status": "", "lat": 53.76282, "lon": -2.70452}, {"facility": "Sheffield Teaching Hospital", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}, {"facility": "Royal Stoke Hotel", "city": "Stoke", "state": "", "country": "United Kingdom", "status": "", "lat": 53.25, "lon": -2.86667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age \u2265 18 years at the time of screening\n2. Diagnosis of ALS according to the Gold Coast Criteria\n3. Capable of providing informed consent and complying with trial procedures\n4. TRICALS risk profile \\> -6.0 and \\< -2.0\n5. Those taking Riluzole must be on a stable dose for at least 30 days prior to the baseline visit or must have stopped taking Riluzole at least 30 days prior to the baseline visit\n6. Women must not become pregnant (e.g., post-menopausal, surgically sterile, using highly effective birth control methods or not having potentially reproductive sex) for the duration of the study plus five days. Highly effective methods of birth control are those with a failure rate of \\< 1% per year when employed consistently and correctly, e.g. Combined (oestrogen and progestogen containing) hormonal contraception or progestogen-only hormonal contraception. For more information, please refer to the HMA CTFG Guidelines: https://www.hma.eu/fileadmin/dateien/Human\\_Medicines/01-About\\_HMA/Working\\_Groups/CTFG/2014\\_09\\_HMA\\_CTFG\\_Contraception.pdf?fbclid=IwAR3AY5Ha0ESDyqIBeUaYI9VTFWmx9bbt8NZ-80N-5ME6pkBb1UHvFsTwqlQ\n7. Women of childbearing potential must have a negative serum pregnancy test at screening and be non-lactating. Patients will be advised regarding appropriate contraception. A menstruation history will be taken at each visit. Women of childbearing potential are defined as females who are fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy (https://www.hma.eu/fileadmin/dateien/Human\\_Medicines/01-About\\_HMA/Working\\_Groups/CTFG/2014\\_09\\_HMA\\_CTFG\\_Contraception.pdf?fbclid=IwAR3AY5Ha0ESDyqIBeUaYI9VTFWmx9bbt8NZ-80N-5ME6pkBb1UHvFsTwqlQ)\n8. For participants taking antacids (regularly or as required), participant is willing and able to avoid taking antacids for at least 6 hours before and 2 hours after Triumeq\n9. Participant taking taurursodiol supplements (TUDCA) can participate in this trial if the supplement does not contain sodium phenylbutyrate.\n10. Participants taking taurursodiol supplements (TUDCA) that also contain sodium phenylbutyrate must be willing to stop supplementation 30 days prior randomisation.\n\nExclusion Criteria:\n\n1. People who are HLA-B\\*5701 positive\n2. Known hypersensitivity to Dolutegravir, Abacavir or Lamivudine, or to any of the excipients\n3. Safety Laboratory Criteria at screening:\n\n   * ALT \u2265 5 times upper limit of normal (ULN)\n   * AST \u2265 3 times ULN\n   * Bilirubin \u2265 1.5 times ULN with clinical indicators of liver disease\n   * Creatinine clearance \\< 30 mL / min\n   * Platelet concentration of \\< 100 x109 per L\n   * Absolute neutrophil count of \\< 1x109 per L\n   * Haemoglobin \\< 100 g/L\n   * Amylase \u2265 2 times ULN\n   * Lactate \u2265 2 times ULN\n4. Moderate to severe hepatic impairment, as defined by local clinical guidelines\n5. Presence of HIV antibodies at screening\n6. Presence of Hepatitis C antibodies at screening unless participants have had effective treatment for Hepatitis C\n7. Presence of Hepatitis B core or surface antigen at screening\n8. Participation in any other investigational drug trial or using investigational drug within 30 days prior to screening\n9. Use of NIV \u226522 h per day or having a tracheostomy\n10. Edaravone dose within 30 days prior to screening. Edaravone is approved by the FDA and in Japan, but remains an investigational product in Europe and Australia\n11. Clinically significant history of unstable or severe cardiac, oncological, psychiatric, hepatic, or renal disease or other medically significant illness\n12. Taking medication contraindicated with Triumeq: Dofetilideor Fampridine (dalfampridine)\n13. Taking Tofersen within 3 months prior to screening.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Triumeq (dolutegravir, abacavir, and lamivudine)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07088159", "title": "Intermediate-size Patient Population Expanded Access Protocol", "phase": "Expanded Access", "status": "AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Spinogenix", "summary": "The purpose of this Expanded Access Program is to provide tazbentetol to ALS patients who are not eligible to enroll in an ALS clinical trial. This Expanded Access Program will assess safety and tolerability, and clinical efficacy of tazbentelol.", "interventions": [{"type": "DRUG", "name": "Tazbentetol"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT07088159", "target_entities": ["tazbentetol"], "locations": [{"facility": "University of Alabama Birmingham", "city": "Birmingham", "state": "Alabama", "country": "United States", "status": "AVAILABLE", "lat": 33.52066, "lon": -86.80249}, {"facility": "Mayo Scottsdale", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "AVAILABLE", "lat": 33.50921, "lon": -111.89903}, {"facility": "Kaiser Permanente Los Angeles", "city": "Los Angeles", "state": "California", "country": "United States", "status": "AVAILABLE", "lat": 34.05223, "lon": -118.24368}, {"facility": "Cedar-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "AVAILABLE", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "AVAILABLE", "lat": 37.77493, "lon": -122.41942}, {"facility": "Nova Southeastern University", "city": "Davie", "state": "Florida", "country": "United States", "status": "AVAILABLE", "lat": 26.06287, "lon": -80.2331}, {"facility": "Synapticure", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "AVAILABLE", "lat": 41.85003, "lon": -87.65005}, {"facility": "Dartmouth-Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "AVAILABLE", "lat": 43.64229, "lon": -72.25176}, {"facility": "Atlantic Health", "city": "Summit", "state": "New Jersey", "country": "United States", "status": "AVAILABLE", "lat": 40.71562, "lon": -74.36468}, {"facility": "New Mexico VA Health Care System", "city": "Albuquerque", "state": "New Mexico", "country": "United States", "status": "AVAILABLE", "lat": 35.08449, "lon": -106.65114}, {"facility": "NYU Langone Health", "city": "New York", "state": "New York", "country": "United States", "status": "AVAILABLE", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "AVAILABLE", "lat": 40.71427, "lon": -74.00597}, {"facility": "Thomas Jefferson University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "AVAILABLE", "lat": 39.95238, "lon": -75.16362}, {"facility": "Baylor College of Medicine", "city": "Houston", "state": "Texas", "country": "United States", "status": "AVAILABLE", "lat": 29.76328, "lon": -95.36327}, {"facility": "VCU ALS Research Group", "city": "Henrico", "state": "Virginia", "country": "United States", "status": "AVAILABLE", "lat": 36.59264, "lon": -78.61611}], "contact_phone": "1 (661) 862-7122", "contact_email": "als302eap@cbcc.global", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS diagnosis\n* Age 18 -85 years at time of signing informed consent form\n* Ineligible for other interventional ALS clinical research participation\n* Vital Capacity greater than 35% of predicted capacity for age, height, and sex\n* If currently taking standard of care treatment for ALS, must be on stable dose for at least 30 days prior to taking tazbentetol.\n* Life expectancy of at least 6 months, according to Investigator's judgement\n\nExclusion Criteria:\n\n* Clinically significant and/or unstable medical condition (other than ALS) that would pose a risk to the patient\n* Known ongoing or clinically uncontrolled cardiac disease\n* Clinically significant liver disease\n* Clinical significant cognitive impairment or neurological disorder, as determined by Investigator judgement\n* Concomitant use of another investigational medical product for treatment of ALS\n* Unable to reliably and regularly swallow whole oral medications on a daily basis", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tazbentetol", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01776970", "title": "Safety and Efficacy on Spasticity Symptoms of a Cannabis Sativa Extract in Motor Neuron Disease", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ospedale San Raffaele", "summary": "The clinical primary hypothesis is that there will be a difference between a Cannabis Sativa extract and placebo in their effect on spasticity in Motor Neuron Disease (MND) patients with signs of involvement of the upper motor neuron (UMN) resulting in disabling spasticity.\n\nSecondary goals of the study are to evidence of improvement in other symptoms (in particular pain), and to show favourable trends on functionality measures. Finally, cannabis based drug safety and tolerability will be studied through vital parameters (including weight and pulmonary function) measurement, and analyzing ALS function rating scale progression slope hopefully, showing a slowing of the functional values decrease, owing to cannabis neuroprotective effects)", "interventions": [{"type": "DRUG", "name": "Cannabis Sativa extract Oromucosal spray"}], "start_date": "2013-01", "url": "https://clinicaltrials.gov/study/NCT01776970", "target_entities": ["spasticity"], "locations": [{"facility": "San Raffaele Scientific Institute", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Fondazione Salvatore Maugeri IRCCS, Istituto Scientifico", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "NEuroMuscular Omnicentre (NEMO), Fondazione Serena - H C\u00e0 granda", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Universita' Degli Studi Di Padova, Azienda Ospedaliera Di Padova, Neurologic Department;", "city": "Padova", "state": "", "country": "Italy", "status": "", "lat": 44.38225, "lon": 11.14261}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion criteria:\n\nSubjects must fulfil ALL of the following criteria:\n\n* Written informed consent\n* Subject able and willing to comply with all study requirements\n* Affected by ALS, either of definite, probable or possible category according to the El Escorial revised criteria or by primary lateral sclerosis (Pringle's criteria)\n* Affected of spasticity, equal or above 1 in the Ashworth Scale for spasticity in 2 or more muscle groups\n* Who will judge spasticity a relevant cause of movements impairment\n* Subject has spasticity due to MND of at least three months duration, which is not wholly relieved with current anti-spasticity therapy\n* Subject fulfils at least one of the two criteria below. Subject must be either:\n\n  1. Currently established on a regular dose of anti-spasticity therapy, or\n  2. Previously tried and failed, or could not tolerate suitable anti-spasticity therapy\n* Stabilization of factors affecting spasticity: any physiotherapy regimen or medication likely to affect spasticity will be optimised before the study and not altered in the 3 weeks before start of treatment\n* Subject is willing for his or her name to be notified to the responsible authorities for participation in this study, as applicable.\n\nAdditional inclusion Criteria to be met at baseline\n\n\u2022 Subjects have registered spasticity NRS scores via the personal clinical diary over the 6 days (day 2 to day 7) before randomization\n\nExclusion criteria:\n\n* Any concomitant disease or disorder that has spasticity-like symptoms or that may influence the subject's level of spasticity\n* Subjects receiving Botulinum Toxin during the preceding 6 months\n* Bedridden and tracheotomised patients\n* Fixed-tendon contractures\n* Severe cognitive impairment\n* Currently using or has used cannabis, cannabinoid-based medications or Acomplia (Rimonabant) within 30 days of study entry and unwilling to abstain for the duration of the study\n* Any history or immediate family history of schizophrenia, other psychotic illness, severe personality disorder or other significant psychiatric disorder other than depression associated with their underlying condition\n* Any known or suspected history of a diagnosed dependence disorder, current heavy alcohol consumption, current use of an illicit drug or current non-prescribed use of any prescription drug\n* Subjects with poorly controlled epilepsy or recurrent seizures (Subjects who have had one or more fits in the year prior to Visit 1 will be excluded)\n* Any known or suspected hypersensitivity to cannabinoids or any of the excipients\n* Subject has experienced myocardial infarction or clinically relevant cardiac dysfunction within the last 12 months or has a cardiac disorder that, in the opinion of the investigator would put the subject at risk of a clinically relevant arrhythmia or myocardial infarction\n* Subject has a diastolic blood pressure of \\<50 mmHg or \\>105 mmHg (when measured in a sitting position at rest for five minutes) or a postural drop in the systolic blood pressure of greater than 20 mmHg\n* Personal history suggestive of relevant impaired renal or hepatic function\n* Female subjects of child bearing potential, unless willing to ensure that they or their partner use effective contraception during the study and for three months thereafter\n* Female subject who is pregnant, lactating or planning pregnancy during the course of the study and for three months thereafter\n* Subjects who have received any IMP within the 8 weeks before Visit 1\n* Any other significant disease or disorder which, in the opinion of the investigator, may either put the subject at risk because of participation in the study, or may influence the result of the study, or the subject's ability to participate in the study\n* Unwilling to abstain from donation of blood during the study\n* Patients will be asked not to drive while they will be receiving medication", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Cannabis Sativa extract (oromucosal spray)", "targeting_mechanism": "Activates cannabinoid receptors, particularly CB2 receptors in reactive astrocytes, to modulate neuroinflammation in motor neuron disease.", "targeting_mechanism_pmid": "28069688", "animal_results": "CB2 receptors are upregulated in reactive astrocytes in canine degenerative myelopathy, a disease model of ALS.", "animal_results_pmid": "28069688", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05306457", "title": "CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cedars-Sinai Medical Center", "summary": "The investigator is examining the safety of transplanting cells, that have been engineered to produce a growth factor, into the motor cortex (brain) of patients with Amyotrophic Lateral Sclerosis (ALS). The cells are called neural progenitor cells, which are a type of stem cell that can become several different types of cells in the nervous system. These cells have been derived to specifically become astrocytes, which is a type of neural cell. The growth factor is called glial cell line-derived neurotrophic factor, or GDNF. GDNF is a protein that promotes the survival of many types of neural cells. Therefore, the cells are called \"CNS10-NPC-GDNF.\" The investigational treatment has been tested in people by delivering it to the spinal cord. However, it has only been delivered to the motor cortex of animals. In this study, we want to learn if CNS10-NPC-GDNF cells are safe to transplant into the motor cortex (brain) of people.", "interventions": [{"type": "BIOLOGICAL", "name": "CNS10-NPC-GDNF"}], "start_date": "2022-05-08", "url": "https://clinicaltrials.gov/study/NCT05306457", "target_entities": ["GDNF"], "locations": [{"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion:\n\n1. Confirmed diagnosis of ALS (Possible, Lab-supported Probable, Probable or Definite El Escorial Criteria)\n2. Duration of ALS symptoms \u2264 36 months\n3. Progressive weakness in upper extremities, with EMG supported evidence of denervation in both upper extremities\n4. Forced Vital Capacity \u226550% of predicted normal in supine\n5. Age: 18 years or older\n6. Able to provide Informed Consent\n7. Be geographically accessible to the study site and able to travel to study site for required visits\n8. Have caregiver to assist in the transportation and care required by participation in the study\n9. Not taking riluzole and/or edaravone or on a stable dose for \u2265 30 day\n10. For women of child bearing capacity, negative pregnancy test prior to surgery and willingness to use birth control for the duration of the trial.\n11. Medically able to undergo craniotomy as determined by the site PI and/or investigators\n12. Medically able to tolerate the immunosuppression regimen as determined by the site PI\n\nExclusion:\n\n1. Using invasive ventilatory assistance\n2. Diagnosis of another active or unstable medical illness that may interfere with study participation at discretion of PI\n3. Presence of any of the following conditions:\n\n   1. Current drug or alcohol abuse\n   2. Any known immunodeficiency syndrome\n   3. Unstable medical condition\n   4. Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening\n4. Persons of child bearing capacity not willing to practice birth control\n5. Receiving any investigational device/biologic/drug in the past 30 days or any previous exposure to stem cell therapy\n6. Any condition in the upper extremities that precludes serial strength or coordination testing\n7. Any condition that the investigators feel may pose complications for the surgery\n8. Any condition or ALS disease phenotype that the site PI feels may interfere with participation in the study or in the interpretation of study endpoints\n9. Allergy to Beta-Lactam antibiotics\n10. Donor Specific Antibodies (DSA) \u2265 2500MFI or CPRA \u2265 20%\n11. Contraindications to MRI", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CNS10-NPC-GDNF (neural progenitor cells engineered to secrete glial cell line-derived neurotrophic factor)", "targeting_mechanism": "Transplanted neural progenitor cells differentiate into astrocytes and secrete GDNF to provide neuroprotection and support motor neuron survival.", "targeting_mechanism_pmid": "36064599", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07521930", "title": "Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "The goal of this clinical trial is to evaluate the safety and preliminary efficacy of an implantable device that records and stimulates different areas of the brain to allow adults affected by disabling paralysis (see Eligibility for more details) to control and receive feedback from assistive devices.", "interventions": [{"type": "DEVICE", "name": "INTENT Neural Interface System"}], "start_date": "2026-09", "url": "https://clinicaltrials.gov/study/NCT07521930", "target_entities": [], "locations": [{"facility": "Johns Hopkins Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "4109559441", "contact_email": "ncrone@jhmi.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Complete or incomplete tetraplegia (quadriplegia), tetraparesis (quadriparesis), severe ataxia, or disabling motor impairments in both upper limbs, based on neurological exam. In addition, these motor impairments may be combined with severe motor-related speech impairment (dysarthria or anarthria), as in Locked In Syndrome (LIS) and amyotrophic lateral sclerosis (ALS), including the bulbar variant of ALS.\n* Clinical diagnosis must be established for the etiology of motor impairments, including brainstem stroke\\*, traumatic spinal cord injury (SCI), or progressive and irreversible neuromuscular disease, including muscular dystrophy and motor neuron disease, including amyotrophic lateral sclerosis (ALS). \\*Brain stem stroke is defined as an acute onset of neurological deficit with clinical features of brain stem or cerebellar dysfunction lasting more than 24 hours together with computed tomography (CT) or magnetic resonance imaging (MRI) evidence of ischemic infarction or parenchymal hemorrhage.\n* Candidates with traumatic spinal cord injury must have a complete or incomplete tetraplegia or tetraparesis (ASIA Impairment Scale A, B, C) with an injury level of C6 or higher.\n* Candidates with tetraplegia or tetraparesis from traumatic SCI and other non-progressive neurological disorders must have an upper extremity motor score (UEMS, ISNCSCI) of 7 or less in each of the upper extremities. Candidate must also have less than antigravity strength (\\< 3) throughout the lower limbs.\n* Candidates with progressive conditions with shortened life expectancy, such as ALS, must have less than antigravity strength (\\<3) throughout the upper limbs.\n* Persistence of motor impairments at least 12 months prior to enrollment if due to a non-progressive neurological cause such as stroke or spinal cord injury\n* Meeting surgical safety criteria, including surgical clearance by the participant's primary healthcare provider, study physicians, and any necessary consultants\n* Ability to communicate reliably, such as through speech or eye movement\n* Stable psychosocial support system with caregiver capable of monitoring participant throughout the study\n* Ability and willingness to travel to study location up to five days per week for the duration of the study\n* Ability to understand and comply with study session instructions\n* Corrected visual acuity sufficient for use of computer monitor\n\nExclusion Criteria:\n\n* Psychiatric conditions or cognitive impairments that would interfere with obtaining informed consent or fully participating in study activities.\n* Individuals with active implanted devices, including devices that are incompatible with magnetic resonance imaging (MRI).\n* Contraindications to MRI or anticipated need for an MRI during the study period\n* Medical conditions contraindicating device implantation surgery (for example significant pulmonary, cardiovascular, metabolic, or renal impairments making the surgical procedure unsafe)\n* Chronic anti-coagulation and medical contraindication to temporary suspension for surgery\n* Medical conditions contraindicating chronic device implantation (e.g. osteomyelitis, chronic infection, poorly controlled diabetes, cancer, severe autoimmune disorder, epilepsy, poor wound healing)\n* Participants with dental caries and a significant risk of dental or periodontal infection\n* Chronic oral or intravenous use of steroids or immunosuppressive therapy\n* Active cancer within the past year or ongoing chemotherapy\n* Uncontrolled autonomic dysreflexia within the past 3 months\n* Hydrocephalus with or without an implanted ventricular shunt\n* Other chronic, unstable medical conditions that could interfere with subject participation.\n* Persistent suicidal ideation within the past 12 months.\n* History of substance use disorder within the past year\n* Pregnancy (confirmation through blood test)\n* Nursing an infant, planning to become pregnant, or not using adequate birth control", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02588807", "title": "Food Supplement for the Treatment of Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Herb Spirit", "summary": "The purpose of the study is to evaluate the safety of combining phospholipids with medicinal plants for treatment of patients with amyotrophic lateral sclerosis (ALS)", "interventions": [{"type": "DRUG", "name": "Spirit1"}], "start_date": "2021-01-01", "url": "https://clinicaltrials.gov/study/NCT02588807", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Hacarmel Hospital", "city": "Haifa", "state": "", "country": "Israel", "status": "", "lat": 32.81303, "lon": 34.99928}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Males aged 18 to 75 years, Females\\>50 years\n2. Diagnosis of \"probable\" or \"definite\" ALS according to the El Escorial revised criteria\n3. A documented history of ALS symptoms for more than 6 month prior to study enrolment, and no more than 40 month.\n4. Patients capable of understanding and signing Informed Consent.\n\nExclusion Criteria:\n\n1. Patients allergic to seafood\n2. Patients with forced vital capacity \\< 75%\n3. Patients who are respiratory dependent, underwent tracheostomy, or cannot swallow.\n4. Patients with cardiovascular diseases\n5. Patients with diabetes\n6. Patients with active peptic ulcers\n7. Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc).\n8. Patients suffering from other chronic significant disease, malignant diseases or any other disease that may risk the patient or interfere with the ability to interpret the results.\n9. Patients that can not sign/understand the Informed Consent Form.\n10. Female patients who are pregnant or lactating\n11. Patients who have received and experimental drug or have participated in a clinical trial within 1 month prior to screening", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Spirit1", "targeting_mechanism": "Reduces neuroinflammation in the CNS to slow ALS progression.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07589764", "title": "Pilot Trial Utilizing \u03b2-hydroxy-\u03b2-methylbutyrate to Lower IGFBP7 Levels in People With ALS", "phase": "PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Duke University", "summary": "This is an open label trial of a supplement called HMB in patients with ALS. The researchers are evaluating its safety and tolerability, as well as its ability to lower insulin-like growth-factor binding protein 7 (IGFBP7) and Neurofilament light chain levels (NFL) and to slow ALS Functional Rating Scale, Revised (ALSFRS-R) progression.", "interventions": [{"type": "DRUG", "name": "\u03b2-hydroxy-\u03b2-methylbutyrate (HMB)"}], "start_date": "2026-10-01", "url": "https://clinicaltrials.gov/study/NCT07589764", "target_entities": ["IGFBP7"], "locations": [{"facility": "Duke University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}], "contact_phone": "919-613-2681", "contact_email": "alsresearch@duke.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Male or female aged at least 18 years.\n2. Sporadic or familial ALS diagnosed as per Gold Coast Criteria (37).\n3. Patient is able to understand and express informed consent (in the opinion of the site investigator).\n4. Patient is able to read and write English.\n5. Patient is expected to survive for the duration of the trial.\n6. Women must not be pregnant (will have evidence of a negative pregnancy test obtained by study team at baseline, or by local physician within past 7 days or be post-menopausal)\n7. Women must not be able to become pregnant (e.g., post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception, or other hormonal contraception, for example patch or contraceptive ring), intrauterine device (IUD) in place for \u2265 3 months, barrier method in conjunction with spermicide, or another adequate method.\n\nExclusion Criteria:\n\n1. Actively or recently (within past 30 days) participating in another intervention trial.\n2. Currently or recently (within 30 days) taking HMB\n3. Prior side effects from HMB deemed to be significant by the investigator\n4. Patient has a medical or psychiatric illness that could in the investigator's opinion interfere with the patient's ability to participate in this study.\n5. Pregnant women or women currently breastfeeding.\n6. Elevated serum calcium or vitamin D levels.\n7. Life expectancy shorter than the duration of the trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "\u03b2-hydroxy-\u03b2-methylbutyrate (HMB)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07187388", "title": "Investigating the Impact of Electrical Stimulation on Facial Pain, Jaw Movement and Oral Health in People With Motor Neuron Disease.", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Nova Southeastern University", "summary": "The goal of this clinical trial is to evaluate the impact of non-invasive electrical stimulation, when placed on the facial muscles can reduce facial pain and improve jaw mobility, and chewing in individuals with Amyotrophic Lateral Sclerosis (ALS) and Primary Lateral Sclerosis (PLS). The secondary goal is to evaluate the impact of non-invasive electrical stimulation on patient reported difficulty performing oral hygiene tasks in individuals with ALS and PLS. Participants will attend one in-person clinic visit and participate in one telephone interview 24 hours after the treatment. The clinic visit will include pre-intervention assessments, a single 30-minute treatment of electrical stimulation followed by post-intervention assessments.\n\nThe assessments will include a self-rating of jaw and facial pain, a range of motion test where participants will be asked to open their jaw as wide and as far to the side as possible, and a chewing efficiency test using a saltine cracker. Twenty-four hours later, participants will receive a follow-up phone call to self-rate their facial pain and report any difficulty performing oral hygiene tasks.\n\nThe treatment consists of a single 30-minute electrical stimulation session. Electrode pads will be placed on the participant's facial region, specifically over the masseter muscle belly and the TMJ area, while the participant is seated comfortably. The pads will be connected to an FDA-approved electrical stimulator, and the current will be adjusted to the participant's comfort level. Once set, the participant will remain seated for 30 minutes. At the end of the session, the stimulator will be turned off and the electrode pads removed.", "interventions": [{"type": "DEVICE", "name": "Transcutaneous Electrical Nerve Stimulation to reduce facial pain and improve jaw range of motion in ALS and PLS"}], "start_date": "2026-04-02", "url": "https://clinicaltrials.gov/study/NCT07187388", "target_entities": [], "locations": [{"facility": "Nova Southeastern University, David and Cathy Husman Neuroscience Institute", "city": "Davie", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.06287, "lon": -80.2331}], "contact_phone": "954-817-5730", "contact_email": "kayla.chomko@nova.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of possible, probable, or definite ALS (El-Escorial Revisited) OR diagnosis of definite PLS by the treating neurologist and based on the consensus diagnostic criteria for PLS\n* Subjective report of jaw pain, indicated by a NRS score of \\> or = 3/10.\n\nExclusion Criteria:\n\n* history of head or neck cancer\n* history of CVA\n* history of past facial surgery with hardware placement\n* history of pacemaker\n* diagnosis of significant cognitive impairment by the treating physician\n* history of seizures or diagnosis of epilepsy\n* open wound at the area of electrode placement\n* complete loss of sensation at the area of electrode placement", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05218668", "title": "Rho Kinase Inhibitor in Amyotrophic Lateral Sclerosis (REAL)", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Woolsey Pharmaceuticals", "summary": "A Phase 2a Open-Label Preliminary Safety, Efficacy, and Biomarker Study of WP-0512 in Patients with Amyotrophic Lateral Sclerosis (ALS)", "interventions": [{"type": "DRUG", "name": "Fasudil (WP-0512)"}, {"type": "DRUG", "name": "Fasudil (WP-0512)"}], "start_date": "2021-12-22", "url": "https://clinicaltrials.gov/study/NCT05218668", "target_entities": ["ROCK"], "locations": [{"facility": "Neuromuscular Research Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of Colorado", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "National Jewish Health", "city": "Denver", "state": "Colorado", "country": "United States", "status": "", "lat": 39.73915, "lon": -104.9847}, {"facility": "Lakes Research", "city": "Miami Lakes", "state": "Florida", "country": "United States", "status": "", "lat": 25.90871, "lon": -80.30866}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kentucky", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Cox Medical Center", "city": "Springfield", "state": "Missouri", "country": "United States", "status": "", "lat": 37.21533, "lon": -93.29824}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Macquarie University Hospital", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Brisbane", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.46794, "lon": 153.02809}, {"facility": "Calvary Health Bethlehem Hospital", "city": "Melbourne", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.814, "lon": 144.96332}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Between 18 and 75 years of age (inclusive) at Screening 1.\n2. Subject has had a diagnosis of probable laboratory-supported, probable, or definite ALS (as defined by El Escorial Revised ALS diagnostic criteria) by Screening 1, and no other cause of the neurological impairment has been identified.\n3. Average decrease in ALSFRS-R of 0.5 to 3 (inclusive) points per month, calculated using: Cohort 1 - the most recent historical ALSFRS-R score from at least 3 months prior to Screening 1. If there is no qualifying previous score, an estimated rate will be calculated using the historical date of ALS symptom onset (weakness and/or dysarthria and/or dysphagia). Cohort 2 - the historical date of ALS symptoms onset.\n4. Percent predicted SVC \u2265 50% at Screening 1.\n5. ALS symptom onset (weakness and/or dysarthria, and/or dysphagia) within 48 months of Screening 1.\n6. Subjects taking riluzole, edaravone, or phenylbutyrate (PB) and/or tauroursodeoxycholic acid (TUDCA) may be included if the following criteria are met at Screening 1, and there is no change in treatment between Screening 1 and Enrollment:\n\n   * Stable dose of riluzole for at least 30 days;\n   * Stable dose of edaravone for at least 3 cycles; and/or\n   * Stable dose of PB and/or TUDCA for at least 90 days\n\n   Subjects taking any of these drugs prior to screening who intend to discontinue them before starting the study must have discontinued the drug(s) at least 28 days before Screening 1.\n7. Women of childbearing potential (WCBP) must agree to abstain from sex or use an adequate method of contraception for the duration of the screening period, the study drug treatment period, and for 28 days after the last dose of study drug.\n8. Males must agree to abstain from sex with WCBP or use an adequate method of contraception for the duration of the study drug treatment period and for 75 days after.\n9. Capable of providing informed consent and following trial procedures (where subject consents but is unable to sign the informed consent a legally authorized representative (LAR)/surrogate must sign on their behalf).\n\nExclusion Criteria:\n\n1. ALSFRS-R \\< 24 at Screening 1.\n2. Expected change in dosing of riluzole, edaravone, or PB and/or TUDCA between Screening 1 and the end of the study.\n3. Presence of other causes of neuromuscular weakness or other neurodegenerative diseases that could interfere with the objectives of the study or the safety of the subject, in the opinion of the Investigator.\n4. Mechanical ventilation via tracheostomy. (Use of non-invasive ventilation e.g., continuous positive airway pressure, non-invasive bi-level positive airway pressure or non-invasive volume ventilation is not an exclusion).\n5. Any medical condition (including cardiovascular, hematologic, renal, hepatic, or psychiatric diseases) that in the opinion of the Investigator would disallow safe participation in the trial or interpretation of the study results.\n6. Suicidal ideation per the Columbia-Suicide Severity Rating Scale (C-SSRS) that in the opinion of the Investigator would pose a safety risk.\n7. ALT \u2265 3 x upper limit of normal (ULN) or aspartate aminotransferase (AST) \u2265 3 x ULN at Screening.\n8. Estimated glomerular filtration rate (eGFR) \\< 45 mL/min/1.73m2 at Screening.\n9. Participants who, in the opinion of the Investigator, are unable or unlikely to comply with the dosing schedule or study evaluations.\n10. Treatment in a clinical trial with another investigational drug within 28 days or 5 half-lives of drug before Screening 1, whichever is longer.\n11. Exposure at any time to any gene therapies under investigation for the treatment of ALS.\n12. Treatment with clenbuterol within 28 days of Screening 1, or any time between Screening 1 and enrollment.\n13. On more than one of the following drug classes: long-acting nitrates, beta-blockers, or calcium channel blockers. (Note: subjects may be on one of the drug classes.)\n14. Systolic blood pressure \\< 90 mmHg and/or diastolic blood pressure \\< 60 mmHg at Screening. (Note: in the case of a systolic blood pressure \\< 90 and/or diastolic blood pressure \\< 60, BP measurements should be repeated after 10 minutes, and the higher reading used for Inclusion/Exclusion.)\n15. Known hypersensitivity to the active (fasudil) or inactive ingredients in the study drug.\n16. Known to be pregnant or lactating; or positive pregnancy test for WCBP.\n17. For Cohort 1 only: At Screening 2, neutrophil count \\< 1,500/mm3, platelets \\< 100,000/mm3, international normalized ratio (INR) \\> 1.5 or any contraindication to or unable to tolerate lumbar puncture, including use of anticoagulant medications that cannot be withheld. For example, if a subject is taking warfarin and it cannot be withheld for lumbar puncture, this would exclude the subject from study entry.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Fasudil (WP-0512)", "targeting_mechanism": "Rho kinase inhibitor with neuroprotective and anti-inflammatory effects.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02464852", "title": "Assessment of the Effects of Sheffield Support Snood in MND Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sheffield Teaching Hospitals NHS Foundation Trust", "summary": "The aim of this study is to evaluate the effects on MND patients of a new collar specifically designed for people affected by neck weakness: the Sheffield Support Snood.", "interventions": [{"type": "BEHAVIORAL", "name": "Head movements"}, {"type": "BEHAVIORAL", "name": "Activities of daily living (drinking, washing hand and eating)"}, {"type": "DEVICE", "name": "Sheffield Support Snood"}, {"type": "DEVICE", "name": "sEMG"}], "start_date": "2015-04", "url": "https://clinicaltrials.gov/study/NCT02464852", "target_entities": [], "locations": [{"facility": "Royal Hallamshire Hospital", "city": "Sheffield", "state": "South Yorkshire", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinical status consistent with MND\n* Capability to understand instructions\n* Capability to perform testing procedures\n* Ability to give informed consent\n\nExclusion Criteria:\n\n* Presence of any inflammatory or other disease involving joint or muscle pathology that might affects testing results\n* Unable to give informed consent", "sex": "ALL", "min_age": "16 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01569958", "title": "Transcranial Direct Current Stimulation as a Novel Therapeutic Approach in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Universit\u00e0 degli Studi 'G. d'Annunzio' Chieti e Pescara", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease characterized by progressive weakness and muscular atrophy due to the degeneration and loss of motor neurons, the nerve cells that, in the central nervous system (motor cortex, brainstem and spinal chord), control voluntary movement. Riluzole, the only drug approved for ALS treatment, modestly slow disease progression.\n\nTranscranial direct current stimulation (tDCS) is a noninvasive technique of neuromodulation that is currently studied as a possible therapeutic tool for several neurological and psychiatric diseases and has been found safe and well tolerated. Based on experimental evidence in animals and human subjects, tDCS is expected to reduce motor cortex excitability and excitotoxicity, that is neuronal injury induced by excessive glutamatergic stimulation, one of postulated pathophysiological mechanisms in ALS.\n\nThis study will investigate if transcranial direct current stimulation of motor cortex is useful in delaying disease progression and is well tolerated in ALS patients.", "interventions": [{"type": "OTHER", "name": "transcranial direct current stimulation"}, {"type": "OTHER", "name": "Sham stimulation"}], "start_date": "2012-07", "url": "https://clinicaltrials.gov/study/NCT01569958", "target_entities": [], "locations": [{"facility": "Centro Regionale Malattie Neuromuscolari, Ospedale Clinicizzato \"SS. Annunziata\"", "city": "Chieti", "state": "Chieti", "country": "Italy", "status": "RECRUITING", "lat": 42.34827, "lon": 14.16494}, {"facility": "Azienda Policlinico Universit\u00e0 Federico II", "city": "Naples", "state": "Napoli", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 40.85216, "lon": 14.26811}, {"facility": "Policlinico Universitario Agostino Gemelli", "city": "Rome", "state": "Rome", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 41.89193, "lon": 12.51133}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of probable, laboratory-supported probable, or definite amyotrophic lateral sclerosis according to the El Escorial revised criteria\n* spinal onset\n* aged 18 to 85 years inclusive\n* disease duration \u2264 24 months\n* disease progression in the past 3 months\n* FVC \u2265 70% of predicted\n* score \u2265 2 at the item \"swallowing\"of the ALS Functional Rating Scale Revised\n* score \u2265 2 at the item \"walking\"of the ALS Functional Rating Scale Revised\n* in treatment with steady regimen of riluzole for a minimum of 1 month before study entry, and desiring its continuation\n* able to give informed consent\n* written informed consent\n\nExclusion Criteria:\n\n* bulbar onset\n* previous poliomyelitis\n* motor neuron diseases other than ALS\n* clinical involvement of other neurological systems\n* pregnancy, lactation,or unwillingness to contraception if required\n* possible contraindications to tDCS: metals in the head (excluding the mouth); electromedical devices; seizures; drugs or neurological conditions lowering seizure threshold; alcoholism; severe heart diseases\n* any severe disease other than ALS\n* experimental drugs within 1 month prior to enrollment\n* drugs potentially modifying the response to tDCS", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "transcranial direct current stimulation (tDCS)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01530438", "title": "Study of Cognitive and Emotional Disorders in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Caen", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that involves not only motor structures, as was previously thought, but also brain areas dealing with cognition as well as parts of the limbic system. Clinical, imaging and pathological evidence suggests that ALS and fronto-temporal dementia (FTD) have several features in common, and that these two diseases could be the two ends of a pathological continuum.", "interventions": [{"type": "OTHER", "name": "MRI + 18FDG-PET + neuropsychological assessments ; 18FDG performed especially for the research"}], "start_date": "2009-04", "url": "https://clinicaltrials.gov/study/NCT01530438", "target_entities": [], "locations": [{"facility": "University Hospital Center", "city": "Caen", "state": "", "country": "France", "status": "RECRUITING", "lat": 49.18585, "lon": -0.35912}, {"facility": "University Hospital Center", "city": "Rouen", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 49.44313, "lon": 1.09932}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* All participants :\n\n  * study level \\> 7 years\n  * mother tongue : french\n  * signature of the informed consent of the protocol in accord with the Comit\u00e9 de Protection des Personnes\n  * medical, neurological, neuroradiological and neuropsychological approfondis in accord with the specific inclusion and non inclusion criteria sp\u00e9cifiques at each population\n* Patients ALS :\n\n  * 18 to 80 years old\n  * Diagnostic defined or probable in according to the reviewed criteria of El Escorial.\n* Patients ALS / FTD :\n\n  * 18 to 8O years old\n  * Diagnostic defined or probable in according to El Escorial reviewed criteria and diagnostic of frontal-temporal dementia in according to Lund et Manchester criteria.\n* Control Subjects :\n\n  * 45 to 75 years old\n  * DRS \u2265 130\n  * BECK \\< 8\n\nExclusion Criteria:\n\n* All particpants :\n\n  * Major past history (chronic pulmonary disease, cardiac disease, metabolic, haematological, endocrinological or severe immunological, cancer) ;\n  * Chronic use of alcohol or drugs ;\n  * IRM contraindications\n\nProtected adults, and persons non affiliated to social protection system won't be able to participate at this study. The inclusion of the participant in another biomedical research protocol(during the study or into 12 months before the inclusion) is too a non inclusion criterion.\n\n* Patients SLA and patients SLA / FTD\n\n  * Severe bulbar disorders\n  * Severe restrictive respiratory insufficiency (VC\\<50%) with orthopny\n  * Communication disorders with motor origin (non assessable tests)\n* Control Subjects :\n\n  * Pregnant or nursing women\n  * Unability to submit at the study medical follow-up for geographic or psychiatric reasons(previous or ongoing).\n  * DRS score \\< 130\n  * Depressive syndrome (BECK) \u2265 8", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03766321", "title": "Fecal Microbiota Transplantation Effect on Amyotrophic Lateral Sclerosis Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Azienda Ospedaliero-Universitaria di Modena", "summary": "Given the role of adaptive immunity in ALS, the pathogenicity of some clostridial strains on motorneurons, the putative role of cyanobacteria in ALS development, and the increasing interest for microbiota in neurodegenerative disorders, the modification of intestinal microbiota might affect ALS at its core.\n\nThis interventional study aims at evaluating the biological and disease-modifying effects of Fecal Microbiota Transplant (FMT) in patients affected by Amyotrophic Lateral Sclerosis. As a primary aim of the study, the investigators postulate ALS patients treated with FMT compared to the control arm will display increased Tregs number, which is a favourable biomarker of disease activity and progression. Clinical outcomes as disease progression measured by ALS Functional Rating Scale Revised (ALSFRS-R) score, survival, respiratory function and quality of life will be assessed during the whole treatment and follow-up period.\n\nMoreover, biological activity of FMT will be evaluated in different biomatrices, together with FMT safety and tolerability in a cohort of ALS patients.", "interventions": [{"type": "BIOLOGICAL", "name": "Fecal microbiota transplantation"}, {"type": "BIOLOGICAL", "name": "Placebo"}], "start_date": "2020-07-01", "url": "https://clinicaltrials.gov/study/NCT03766321", "target_entities": ["microbiota"], "locations": [{"facility": "Catholic University of Sacred Heart - Fondazione Policlinico \"A. Gemelli\"", "city": "Roma", "state": "Italy", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}, {"facility": "Clinica Neurologica, Ospedale Clinicizzato \"SS Annunziata\"", "city": "Chieti", "state": "", "country": "Italy", "status": "", "lat": 42.34827, "lon": 14.16494}, {"facility": "Azienda Ospedaliero Universitaria di Modena", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "UO Neurofisiopatologia, Azienda Ospedaliera d\u00ec Perugia", "city": "Perugia", "state": "", "country": "Italy", "status": "", "lat": 43.1122, "lon": 12.38878}, {"facility": "NEuroMuscular Omnicentre Centre (NeMO), Fondazione Serena Onlus-Fondazione Policlinico A. Gemelli", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients diagnosed with a laboratory supported, clinically \"probable\" or \"definite\" amyotrophic lateral sclerosis according to the Revised El Escorial criteria (Brooks, 2000)\n* Sporadic or familial ALS\n* Female or male patients aged between 18 and 70 years old\n* Disease duration from symptoms onset no longer than 18 months at the screening visit\n* Patients treated with a stable dose of Riluzole (100 mg/day) for at least 30 days prior to screening\n* Patients with a weight \\> 50 kg and a BMI \u226518\n* Patients with a FVC (Forced Vital Capacity) equal or more than 70% predicted normal value for gender, height, and age at the screening visit\n* Patients able and willing to comply with study procedures as per protocol\n* Patients able to understand, and capable of providing informed consent at screening visit prior to any protocol-specific procedures\n* Use of effective contraception both for males and females\n\nExclusion Criteria:\n\n* Known organic gastrointestinal disease\n* History of gastrointestinal malignancy; ongoing malignancies\n* Use of immunosuppressive or chemotherapy within the past 2 years\n* Celiac disease and/or food (e.g.lactose) intolerance\n* Previous gastrointestinal surgery\n* Any condition that would make endoscopic procedures contraindicated\n* Acute infections requiring antibiotics\n* Antimicrobial treatment or probiotics 4 weeks prior to screening\n* Severe comorbidities (heart, renal, liver failure); severe renal (eGFR\\< 30ml/min/1.73m2), or liver failure or liver aminotransferase (ALT/AST \\> 2x Upper limit of normal),\n* Autoimmune diseases, inflammatory disorders (SLE, Rheumatoid arthritis, connective tissue disorder) or chronic infections (HIV, hepatitis B or C infection)\n* Abuse of alcohol or drugs\n* HIV, tuberculosis, hepatitis\n* Participation in clinical trials \\<30 days before screening\n* Existing blood dyscrasia (e.g., myelodysplasia)\n* White blood cells\\<4,000/mm\u00b3, platelets count\\<100,000/mm\u00b3, hematocrit\\<30%\n* Patients who underwent non-invasive ventilation, tracheotomy and /or gastrostomy\n* Women who are pregnant or breastfeeding", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Fecal Microbiota Transplantation (FMT)", "targeting_mechanism": "Modification of intestinal microbiota composition to target dysbiosis and reduce pro-inflammatory mediators implicated in ALS pathogenesis.", "targeting_mechanism_pmid": "28947596", "animal_results": "In the SOD1(G93A) ALS mouse model, dysbiosis and increased intestinal permeability were demonstrated, and neurodegenerative disorders with gut dysbiosis affect the central nervous system via pro-inflammatory mediators, impacting gut-brain communications.", "animal_results_pmid": "28947596", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01257581", "title": "Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Nazem Atassi", "summary": "The purpose of the study is to evaluate the safety and efficacy of high dose creatine and two dosages of tamoxifen treatment in amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "creatine"}, {"type": "DRUG", "name": "tamoxifen"}], "start_date": "2011-03", "url": "https://clinicaltrials.gov/study/NCT01257581", "target_entities": ["mitochondrial_function", "Estrogen receptor"], "locations": [{"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Medical Center", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Washington University at St. Louis", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Pennsylvania State University, Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Medical College of Wisconsin", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Familial or sporadic ALS.\n* Disease duration from diagnosis no greater than 36 months at Screening Visit.\n* Aged 18 years or older.\n* Capable of providing informed consent and complying with trial procedures.\n* Vital capacity (VC) equal to or more than 50% predicted normal value for gender, height and age at the Screening Visit.\n* Not taking, or on a stable dose of riluzole (50mg bid) for at least 30 days prior to the Screening Visit.\n* Women must not be able to become pregnant for the duration of the study (e.g., post menopausal for at least one year, surgically sterile, or practicing adequate birth control methods) for the duration of the study. Women of childbearing potential must have a negative serum pregnancy test at the Screening Visit and be non-lactating.\n\nExclusion Criteria:\n\n* History of known sensitivity or intolerability to creatine monohydrate or tamoxifen citrate or to any other related compound.\n* Prior exposure to creatine or tamoxifen within 30 days of the Screening Visit.\n* Exposure to any investigational agent within 30 days of the Screening Visit.\n* Use of coumarin anticoagulants (warfarin sodium), rifampin, aminoglutethimide, medroxyprogesterone, letrozole, or bromocriptine.\n* Presence of any of the following clinical conditions: Clinical evidence of unstable medical or psychiatric illness at the Screening Visit; Screening aspartate aminotransferase (AST) \\> 3 times the upper limit of normal or serum creatinine \\> 1.5 mg/dl (133 umol/L); Permanent assisted ventilation or mechanical ventilation; or Lactating or have a positive serum pregnancy test at the Screening Visit.\n* History of any of the following: blood clots including deep vein thrombosis, pulmonary embolism, and stroke, cataracts, renal problems, endometrial cancer, uterine sarcoma, or diabetes mellitus.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "creatine and tamoxifen", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02242071", "title": "Cell Therapy for Motor Neuron Disease/Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neurogen Brain and Spine Institute", "summary": "The effect of autologous bone marrow mononuclear cells on Motor Neuron Disease/Amyotrophic Lateral Sclerosis patients.", "interventions": [{"type": "BIOLOGICAL", "name": "Stem Cell"}], "start_date": "2008-12", "url": "https://clinicaltrials.gov/study/NCT02242071", "target_entities": [], "locations": [{"facility": "Neurogen brain and spine institute", "city": "Navi Mumbai", "state": "Maharashtra", "country": "India", "status": "", "lat": 19.03681, "lon": 73.01582}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with the diagnosis of definite Motor Neuron Disease/amyotrophic lateral sclerosis.\n* Age 18-80 years\n\nExclusion Criteria:\n\n* HIV/HBV/HCV\n* Malignancies\n* Bleeding tendencies\n* Pneumonia\n* Renal failure\n* Severe liver dysfunction\n* Severe anemia \\[hb \\< 8\\]\n* Any bone marrow disorder\n* Space occupying lesion in brain\n* Pregnancy and lactation\n* Other acute medical conditions/infections such as respiratory infection and pyrexia\n* left ventricular ejection fraction \\< 25%\n* Patient on artificial ventilatory support", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "autologous bone marrow mononuclear cells", "targeting_mechanism": "stem cells that differentiate into support cells such as astrocytes, oligodendrocytes or microglia to provide trophic support, growth factors, anti-inflammatory cytokines, and glutamate buffering to degenerating motor neurons", "targeting_mechanism_pmid": "32043626", "animal_results": "preclinical stem cell studies in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1) demonstrated potential benefit of stem cell therapy for ALS", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06598202", "title": "Exploring Nasal Drop Therapy With Small Extracellular Vesicles for ALS", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Xuanwu Hospital, Beijing", "summary": "This is a multicenter, randomized, double-blind, placebo-controlled, dose-escalation trial. The goal of this clinical trial is to evaluate the safety and preliminary efficacy of nasal drop exosomes derived from human umbilical cord blood mesenchymal stem cells (hUC-MSC-sEV-001) in amyotrophic lateral sclerosis.", "interventions": [{"type": "DRUG", "name": "exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop"}, {"type": "DRUG", "name": "a placebo of exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop"}], "start_date": "2024-12-01", "url": "https://clinicaltrials.gov/study/NCT06598202", "target_entities": [], "locations": [{"facility": "Xuanwu Hospital ,Capital Medical University", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "RECRUITING", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "010 8319 8277", "contact_email": "haojunwei@vip.163.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age: 18-80 years, inclusion of both genders;\n* Disease duration: \u22656 months and \u22642 years (counted from the onset of any ALS symptoms);\n* Subjects must meet the El Escorial revised criteria (2000) for the diagnosis of ALS, with a diagnosis of Definite ALS, Probable ALS, Probable laboratory-supported ALS, or Possible ALS;\n* A score of \u22652 on each item of the revised ALS Functional Rating Scale (ALSFRS-R), with a score of 4 for items related to dyspnea, orthopnea, and respiratory insufficiency;\n* BMI: Between 18 and 30 kg/m\u00b2;\n* Subjects must have a baseline forced vital capacity percentage (%FVC) \u226570%;\n* Allowed concomitant treatments: Oral administration of riluzole/edaravone at standard doses for \u226530 days; regular intravenous edaravone with planned sequential oral treatment. During the trial and follow-up period, the dosage and type of concomitant medications must remain unchanged;\n* Subjects of childbearing potential must use appropriate and effective contraception from 2 weeks prior to trial enrollment until the end of the follow-up period;\n* The subject or legal representative must be able to sign an informed consent form and comply with the study requirements for medication administration and follow-up.\n\nExclusion Criteria:\n\n* Diagnosed as non-ALS based on clinical presentation and available clinical examinations (e.g., neurophysiological tests, MRI, or other imaging, laboratory tests);\n* Abnormal nasal anatomy, nasal cavity damage, severe rhinitis, or nasal disease affecting the administration of the study drug;\n* Requires nasal insertion of a gastric tube;\n* Peripheral venous hemoglobin (HGB) \\< 100 g/L, absolute neutrophil count (NEUT) \\< 1.5\u00d710\\^9/L, platelet count (PLT) \\< 100\u00d710\\^9/L, white blood cell count (WBC) \\< 4.0\u00d710\\^9/L or \u2265 12\u00d710\\^9/L, serum albumin \\< 30 g/L; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \u2265 3\u00d7 the upper limit of normal (ULN);\n* Severe renal insufficiency: Glomerular Filtration Rate (GFR) \\< 30 mL/min (Cockcroft-Gault formula), or other known severe renal diseases;\n* Positive for hepatitis B surface antigen, e antigen, e antibody, or core antibody combined with positive hepatitis B virus DNA; positive for hepatitis C virus antibody; positive syphilis serum antibody; or positive for HIV antibody;\n* History of acute myocardial infarction or interventional treatment within the last 6 months, or heart failure (classified as NYHA III-IV);\n* Presence of severe localized or systemic infection, immunodeficiency, or currently taking immunosuppressants;\n* Concurrent severe systemic diseases such as immunodeficiency diseases, coagulation disorders, or malignancies;\n* Vaccination within 1 month prior to the first administration or during the study until the end of follow-up;\n* Known allergy to the drugs used in this study or similar drugs;\n* Participation in another study and administration of an investigational product within the last 3 months;\n* Contraindications to MRI (e.g., presence of metal implants) or inability to tolerate MRI (e.g., claustrophobia);\n* Pregnant or breastfeeding women, or women of childbearing potential who cannot or are unwilling to use appropriate contraception;\n* Unwillingness or inability to comply with the procedures required by the protocol;\n* Any other conditions deemed unsuitable for inclusion by the investigators.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "exosomes derived from human umbilical cord blood mesenchymal stem cells (hUC-MSC-sEV-001)", "targeting_mechanism": "small extracellular vesicles derived from mesenchymal stem cells that modulate neuroinflammation and provide neuroprotective effects through delivery of bioactive molecules including miRNAs", "targeting_mechanism_pmid": "33462263", "animal_results": "ASC-exosomes ameliorated disease progression in SOD1(G93A) murine ALS model", "animal_results_pmid": "32455791", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06658977", "title": "RolloverTreatment With Triumeq for People With ALS Following the Lighthouse II Trial", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Macquarie University, Australia", "summary": "Triumeq is an approved medicine for HIV. The effectiveness of Triumeq in Amyotrophic Lateral Sclerosis (ALS) is being investigated in the Lighthouse II trial. This study aims to assess whether Triumeq is safe and effective at delaying ALS disease progression when given long term. It is available for participants who have completed the Lighthouse II study. The main measurements are safety, tolerability and survival.\n\nThe study will go for approximately 2 years.", "interventions": [{"type": "DRUG", "name": "Abacavir 600mg, Lamivudine 300mg and Dolutegravir 50mg (Triumeq)"}], "start_date": "2024-11-15", "url": "https://clinicaltrials.gov/study/NCT06658977", "target_entities": ["Reverse transcriptase"], "locations": [{"facility": "Macquarie University, Neurology", "city": "North Ryde", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.79677, "lon": 151.12436}, {"facility": "Neuroscience Research Australia (NeuRA)", "city": "Randwick", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.91439, "lon": 151.24895}, {"facility": "Royal Brisbane and Womens Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Flinders Medical Centre", "city": "Bedford Park", "state": "S.A.", "country": "Australia", "status": "", "lat": -35.02204, "lon": 138.56815}, {"facility": "Launceston General Hospital", "city": "Launceston", "state": "Tasmania", "country": "Australia", "status": "", "lat": -41.43876, "lon": 147.13467}, {"facility": "Calvary Health Care Bethlehem", "city": "Caulfield South", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.89562, "lon": 145.02597}, {"facility": "The Perron Institute", "city": "Nedlands", "state": "W.A.", "country": "Australia", "status": "", "lat": -31.98184, "lon": 115.8073}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Participants diagnosed with ALS according to the Lighthouse II protocol who completed the Lighthouse II trial.\n* Participants taking Riluzole must be on a stable dose.\n* Participants taking taurursodiol supplements (TUDCA) can participate in this trial if the supplement does not contain sodium phenylbutyrate.\n* Women must not become pregnant (e.g., post-menopausal, surgically sterile, using highly effective birth control methods or not having potentially reproductive sex) for the duration of the study plus five days; and women of childbearing potential must have a negative urine pregnancy test at baseline and be non-lactating.\n* For participants taking antacids (regularly or as required), participant is willing and able to avoid taking antacids for at least 6 hours before and 2 hours after the Triumeq dose.\n* Capable of providing informed consent and complying with the trial procedures.\n\nExclusion Criteria:\n\n* In the Principal Investigator's opinion, the participant is unlikely to be compliant with the study drug dosing.\n* People who are HLA-B\\*5701 positive.\n* Presence of HIV antibodies at screening\n* Presence of Hepatitis C antibodies at screening unless participants have had effective treatment for Hepatitis C\n* Presence of Hepatitis B core or surface antigen at screening\n* Known hypersensitivity to Dolutegravir, Abacavir or Lamivudine, or to any of the excipients.\n* Moderate to severe hepatic impairment, as defined by local clinical guidelines.\n* Participation in any other investigational drug trial or using another investigational drug within 5 half-lives of that drug.\n* Use of NIV \u226522 h per day or having a tracheostomy\n* Clinically significant history of unstable or severe cardiac, oncological, psychiatric, hepatic, or renal disease or other medically significant illness\n* Taking medication contraindicated with Triumeq: Dofetilide or Fampridine (dalfampridine)", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Triumeq (abacavir 600mg, lamivudine 300mg, dolutegravir 50mg)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07257302", "title": "Lung Insufflation Capacity Training and Respiratory Function in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Center of Neurology and Psychiatry, Japan", "summary": "The goal of this clinical trial is to determine whether lung insufflation capacity (LIC) training can help maintain respiratory function and prolong tracheostomy-free survival in people with amyotrophic lateral sclerosis (ALS).\n\nThe main questions are:\n\nDoes early and continuous LIC training slow the decline in forced vital capacity (FVC)? Does LIC training prolong the time to tracheostomy or death?\n\nThis single-center study at the National Center of Neurology and Psychiatry (NCNP) in Japan will enroll 25 adults with ALS diagnosed according to the El Escorial or Awaji criteria. This is a single-arm study with no concurrently enrolled control group. Comparative analyses will use matched external controls. PRO-ACT will be used primarily to evaluate short-term respiratory-function trajectories, while JACALS, if data access is approved, will be used primarily to evaluate long-term time-to-event outcomes.\n\nParticipants will:\n\n* Use the LIC Trainer device to perform lung insufflation training twice daily at home\n* Visit the clinic every 3 months for respiratory and functional assessments\n* Undergo respiratory tests, including FVC, LIC, maximum insufflation capacity (MIC), and cough peak flow (CPF)\n* Complete the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)", "interventions": [{"type": "DEVICE", "name": "LIC Trainer (Lung Insufflation Capacity Trainer)"}], "start_date": "2025-11-01", "url": "https://clinicaltrials.gov/study/NCT07257302", "target_entities": [], "locations": [{"facility": "National Center of Neurology and Psychiatry (NCNP)", "city": "Kodaira", "state": "Tokyo", "country": "Japan", "status": "RECRUITING", "lat": 35.72603, "lon": 139.48508}], "contact_phone": "+81-42-341-2712", "contact_email": "rinri-jimu@ncnp.go.jp", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age 20 years or older\n* Diagnosis of amyotrophic lateral sclerosis (ALS) according to the El Escorial or Awaji criteria\n* Not using noninvasive ventilation (NIV) and without tracheostomy at the start of LIC training\n* Able to perform study assessments and provide written informed consent (or assisted signature with communication aid)\n\nExclusion Criteria:\n\n* Chronic pulmonary disease other than ALS (e.g., COPD, interstitial lung disease)\n* Severe cognitive or communication impairment preventing study participation\n* Uncontrolled cardiovascular disease, including unstable angina, recent myocardial infarction, decompensated heart failure, serious arrhythmia, severe aortic stenosis, or active myocarditis/endocarditis\n* Uncontrolled hypertension\n* Acute systemic illness or fever\n* Recent pulmonary embolism, acute cor pulmonale, or severe pulmonary hypertension\n* Severe hepatic or renal dysfunction\n* Any condition judged inappropriate by the investigator", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "LIC Trainer (Lung Insufflation Capacity Trainer)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03491462", "title": "Arimoclomol in Amyotropic Lateral Sclerosis", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "ZevraDenmark", "summary": "A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)", "interventions": [{"type": "DRUG", "name": "Arimoclomol"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2018-07-31", "url": "https://clinicaltrials.gov/study/NCT03491462", "target_entities": ["heat_shock_protein"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "HonorHealth Neurology", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "UC Irvine Health ALS and Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "University of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of Kansas Medical Center (KUMC) - Landon Center on Aging", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Providence Brain & Spine Institute", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Texas Southwestern Medical Center", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "Catholic University Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universitaetsklinikum Ulm - Klinik fuer Neurologie", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Instituti Clinica Scientifici Maugeri - IRCCS", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Centrum Medyczne NeuroProtect", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Citi Clinic", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Carlos III - Hospital Universitario La Paz, ALS Unit", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Ume\u00e5 University Hospital", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "Kantonsspital St.Gallen, Muskelzentrum/ALS Clinic", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "Leonard Wolfson Experimental Neurology Centre", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subject meets revised El Escorial criteria for clinically possible, clinically probable / clinically probable ALS laboratory-supported, clinically definite ALS or clinically definite familial ALS laboratory-supported\n* 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath)\n* ALS Functional Rating Scale-Revised (ALSFRS-R) equal to or above 35 and erect (seated) Slow Vital Capacity (SVC) % predicted equal to or above 70% at Screening\n\nExclusion Criteria:\n\n* Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of Screening and Baseline\n* Pregnant or breast-feeding\n* Current or anticipated use of diaphragmatic pacing\n* Any other relevant medically significant condition which could present risk to the subject or interfere with the assessment of safety or has an increased risk of causing death during the trial", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "arimoclomol", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03237741", "title": "Bioavailability of GDC-0134 and the Effect of Food and Proton Pump Inhibitor on Pharmacokinetics of GDC-0134 in Healthy Female Participants", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Genentech, Inc.", "summary": "This study will evaluate the pharmacokinetics and safety of GDC-0134 in healthy female volunteers of non-childbearing potential. The first part of the study will compare the bioavailability of a prototype capsule of GDC-0134 relative to an existing GDC-0134 reference capsule (Periods 1 and 2). The second part of the study will assess the effect of GDC-0134-in-applesauce preparation under fasting conditions, the effect of low and high fat foods as well as the effect of elevated stomach pH via pre-treatment with rabeprazole, a proton pump inhibitor (PPI), under fasted and high-fat meal conditions (Periods 3 and 4).", "interventions": [{"type": "DRUG", "name": "Reference capsule GDC-0134"}, {"type": "DRUG", "name": "Prototype capsule GDC-0134"}, {"type": "DRUG", "name": "rabeprazole"}], "start_date": "2017-08-07", "url": "https://clinicaltrials.gov/study/NCT03237741", "target_entities": [], "locations": [{"facility": "Quotient Clinical Ltd, Clinical Research Unit", "city": "Nottingham", "state": "", "country": "United Kingdom", "status": "", "lat": 52.9536, "lon": -1.15047}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Healthy female participants between 30 and 65 years of age, inclusive;\n* Within body mass index range 18.0 to 35.0 kilograms per square meter (kg/m\\^2), inclusive;\n* Female participants will be of non-childbearing potential;\n* In good health, determined by no clinically significant findings from medical history, 12-lead echocardiogram (ECG), and vital signs;\n* Clinical laboratory evaluations within the reference range for the test laboratory, unless deemed not clinically significant by the investigator;\n* Normal ophthalmology assessment.\n\nExclusion Criteria:\n\n* Males and females of childbearing potential;\n* Significant history or clinical manifestation of any significant metabolic, allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal (GI), neurological, or psychiatric disorder (as determined by the investigator;\n* History of significant hypersensitivity, intolerance, or allergy to any drug compound, food, or other substance, unless approved by the investigator;\n* History of stomach or intestinal surgery or resection that would potentially alter absorption and/or excretion of orally administered drugs;\n* History of GI bleeding or GI ulcers;\n* Any personal or family history of bleeding disorders, and any personal use of drugs known to affect blood clotting within 30 days of dosing;\n* Any acute or chronic medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the subject's safe participation in and completion of the study.", "sex": "FEMALE", "min_age": "30 Years", "max_age": "65 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "GDC-0134", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01154283", "title": "Study of Standard Noninvasive Positive Pressure Ventilation (NIPPV) and Low Expiratory Pressure NIPPV in ALS Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Kirsten Gruis", "summary": "The purpose of the study is to test whether noninvasive positive pressure ventilation (NIPPV) without expiratory positive airway pressure (EPAP) (inspiratory positive airway pressure (IPAP)-only) will result in an increase in patient usage of NIPPV compared with standard, Bi-level NIPPV. Secondarily, the investigators will assess measures of dyspnea, quality of life, patient satisfaction, and side effects.", "interventions": [{"type": "DEVICE", "name": "Viasys\u00ae Healthcare, Pulmonetic Systems, lap-top ventilator (LTV machine)"}], "start_date": "2008-01", "url": "https://clinicaltrials.gov/study/NCT01154283", "target_entities": [], "locations": [{"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age greater than 18 years\n2. Definite or probable ALS by El Escorial criteria\n3. Maximal inspiratory force \\< 60 cm/H2O or FVC \\<50% predicted (American College of Chest Physicians' criteria for initiating NIPPV)\n4. Patients must have used NIPPV (typically administered as BiPAP) for at least 2 months\n5. Currently using NIPPV \u22655 days a week with an EPAP = 4 cm H2O.\n\nExclusion Criteria:\n\n1. Any medical condition that will interfere with participation\n2. Inability to consent for him/herself\n3. Known obstructive sleep apnea or obstructive pulmonary disease.", "sex": "ALL", "min_age": "19 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04172792", "title": "Safety and Tolerability Ultra-high-caloric Food Supplements in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Albert Christian Ludolph, Prof.", "summary": "The LIPCAL-ALS study (NCT02306590) has provided preliminary evidence that a high-caloric nutrition might prolong survival in fast-progressing ALS patients. Since increasing the amount of calories of the intervention might possibly increase the beneficial effect, the investigators seek to investigate whether an ultra-high caloric diet (UHCD), featuring the double amount of calories compared to LIPCAL-ALS, will be well tolerated by ALS patients and may serve as an intervention for a potential LIPCALII study. For this purpose, the investigators will compare two different UHCDs (one fat-rich and one carbohydrate-rich) with regard to safety and tolerability over a time frame of 4 weeks. A third group will receive the original diet from LIPCAL, and a fourth group will receive no intervention (control group).", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "high-caloric fatty diet"}, {"type": "DIETARY_SUPPLEMENT", "name": "ultra-high-caloric fatty diet"}, {"type": "DIETARY_SUPPLEMENT", "name": "ultra-high-caloric carbohydrate-rich diet"}], "start_date": "2019-11-26", "url": "https://clinicaltrials.gov/study/NCT04172792", "target_entities": [], "locations": [{"facility": "University of Ulm, Department of Neurology", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of Amyotrophic Lateral Sclerosis (ALS) according to the revised version of the El Escorial criteria (Ludolph et al. 2015)\n* Slope of ALS Functional Rating Scale Revised (ALSFRS-R) of \\>0.25 points per month at baseline visit based on the formula (48 - score at baseline visit) / (time between date of first symptom and baseline visit)\n* stable on standard therapy riluzole (100 mg/day) for at least 4 weeks\n* capable of thoroughly understanding all information given and giving full informed consent according to good clinical practice (GCP)\n\nExclusion Criteria:\n\n* already taking any dietary supplements\n* participation in another clinical trial within the preceding 8 weeks\n* tracheostomy or assisted ventilation of any type which exceeds 23 hours per day\n* pregnancy or breast-feeding females", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03127267", "title": "Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients", "phase": "PHASE3", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AB Science", "summary": "The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Masitinib (6.0)"}, {"type": "DRUG", "name": "Riluzole"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Masitinib (4.5)"}], "start_date": "2021-02-02", "url": "https://clinicaltrials.gov/study/NCT03127267", "target_entities": ["C-KIT", "PDGFRA"], "locations": [{"facility": "University of Alabama at Birmingham", "city": "Birmingham", "state": "Alabama", "country": "United States", "status": "RECRUITING", "lat": 33.52066, "lon": -86.80249}, {"facility": "University of Southern California", "city": "Los Angeles", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of Kentucky", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "RECRUITING", "lat": 37.98869, "lon": -84.47772}, {"facility": "Johns Hopkins Medicine Brain Science Institute", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.29038, "lon": -76.61219}, {"facility": "Lahey Hospital and Medical Center", "city": "Burlington", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.50482, "lon": -71.19561}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "RECRUITING", "lat": 38.02931, "lon": -78.47668}, {"facility": "University Hospital Leuven (UZ Leuven)", "city": "Leuven", "state": "", "country": "Belgium", "status": "RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "Bispebjerg Hospital", "city": "Copenhagen", "state": "", "country": "Denmark", "status": "RECRUITING", "lat": 55.67594, "lon": 12.56553}, {"facility": "CHU de Angers", "city": "Angers", "state": "", "country": "France", "status": "RECRUITING", "lat": 47.47156, "lon": -0.55202}, {"facility": "Groupe Hospitalier Pellegrin Tripode", "city": "Bordeaux", "state": "", "country": "France", "status": "RECRUITING", "lat": 44.84124, "lon": -0.58046}, {"facility": "H\u00f4pital neurologique Pierre Wertheimer", "city": "Bron", "state": "", "country": "France", "status": "RECRUITING", "lat": 45.73865, "lon": 4.91303}, {"facility": "CHU Gabriel Montpied", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "RECRUITING", "lat": 45.77969, "lon": 3.08682}, {"facility": "CHU de Lille - Hopital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "RECRUITING", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges - H\u00f4pital Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "RECRUITING", "lat": 45.83362, "lon": 1.24759}, {"facility": "CHU de Marseille - H\u00f4pital de la Timone", "city": "Marseille", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHRU de Montpellier - Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nancy - Hopital Central", "city": "Nancy", "state": "", "country": "France", "status": "RECRUITING", "lat": 48.68439, "lon": 6.18496}, {"facility": "CHU H\u00f4pital Pasteur Nice", "city": "Nice", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.70313, "lon": 7.26608}, {"facility": "CHRU de Tours - Hopital Bretonneau", "city": "Tours", "state": "", "country": "France", "status": "RECRUITING", "lat": 47.39484, "lon": 0.70398}, {"facility": "Department of Neurology, University of Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "Athens Naval Hospital", "city": "Athens", "state": "", "country": "Greece", "status": "RECRUITING", "lat": 37.98376, "lon": 23.72784}, {"facility": "Eginition Hospital", "city": "Athens", "state": "", "country": "Greece", "status": "RECRUITING", "lat": 37.98376, "lon": 23.72784}, {"facility": "University General Hospital of Larissa", "city": "Larissa", "state": "", "country": "Greece", "status": "RECRUITING", "lat": 39.62847, "lon": 22.42112}, {"facility": "General University Hospital of Patras", "city": "Rio", "state": "", "country": "Greece", "status": "RECRUITING", "lat": 38.29558, "lon": 21.78504}, {"facility": "Hadassah University Hospital", "city": "Jerusalem", "state": "", "country": "Israel", "status": "RECRUITING", "lat": 31.76904, "lon": 35.21633}, {"facility": "Tel-Aviv Medical Center H\u00f4pital Sourasky (ICHILOV)", "city": "Tel Aviv", "state": "", "country": "Israel", "status": "RECRUITING", "lat": 32.08088, "lon": 34.78057}, {"facility": "Ospedale Civile Sant'Agostino - Estense", "city": "Baggiovara", "state": "Modena", "country": "Italy", "status": "RECRUITING", "lat": 44.60416, "lon": 10.86256}, {"facility": "ASST degli Spedali Civili di Brescia", "city": "Brescia", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.53558, "lon": 10.21472}, {"facility": "Centro Clinico NeMO Fondazione Serena Onlus", "city": "Gussago", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.58358, "lon": 10.15717}, {"facility": "Clinico Nemo Center (Centro Clinico NeMO Milano)", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "IRCCS Istituto Auxologico Italiano", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "Istituti Clinici Scientifici Maugeri IRCCS", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "San Raffaele Hospital (Ospedale San Raffaele)", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "University Hospital Maggiore della Carita", "city": "Novara", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.44694, "lon": 8.62118}, {"facility": "Azienda Ospedale-Universit\u00e0 Padova", "city": "Padova", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 44.38225, "lon": 11.14261}, {"facility": "IRCCS Mondino Foundation", "city": "Pavia", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.19205, "lon": 9.15917}, {"facility": "University Hospital in Turin (Azienda Ospedaliero-Universitaria Citt\u00e0 della Salute e della Scienza di Torino)", "city": "Torino", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 44.88856, "lon": 11.99138}, {"facility": "Oslo University Hospital HF Ullev\u00e5l", "city": "Oslo", "state": "", "country": "Norway", "status": "RECRUITING", "lat": 59.91273, "lon": 10.74609}, {"facility": "Centrum Medyczne Neuromed", "city": "Bydgoszcz", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 53.1235, "lon": 18.00762}, {"facility": "Hospital de Santa Maria", "city": "Lisbon", "state": "", "country": "Portugal", "status": "RECRUITING", "lat": 38.72509, "lon": -9.1498}, {"facility": "Moscow city clinical Hospital after V.M. Buyanov", "city": "Moscow", "state": "", "country": "Russia", "status": "RECRUITING", "lat": 55.75204, "lon": 37.61781}, {"facility": "Scientific Practical Medical Center \"Innovation and Health\"", "city": "Novosibirsk", "state": "", "country": "Russia", "status": "RECRUITING", "lat": 55.02259, "lon": 82.93175}, {"facility": "Clinical Centre of Serbia", "city": "Belgrade", "state": "", "country": "Serbia", "status": "RECRUITING", "lat": 44.80401, "lon": 20.46513}, {"facility": "Klinicni center Ljubljana", "city": "Ljubljana", "state": "", "country": "Slovenia", "status": "RECRUITING", "lat": 46.05108, "lon": 14.50513}, {"facility": "Hospital General Universitario de Alicante", "city": "Alicante", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 38.34517, "lon": -0.48149}, {"facility": "Hospital Universitari de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Carlos III", "city": "Madrid", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 40.4165, "lon": -3.70256}, {"facility": "Clinical Hospital Santiago de Compostela", "city": "Santiago de Compostela", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 42.88052, "lon": -8.54569}, {"facility": "Hospital Universitario y Politecnico La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 39.47391, "lon": -0.37966}, {"facility": "Centralsjukhuset Karlstad (Central Hospital Karlstad)", "city": "Karlstad", "state": "", "country": "Sweden", "status": "RECRUITING", "lat": 59.3793, "lon": 13.50357}, {"facility": "Sk\u00e5ne University Hospital", "city": "Malm\u00f6", "state": "", "country": "Sweden", "status": "RECRUITING", "lat": 55.60587, "lon": 13.00073}, {"facility": "Norrlands universitetssjukhus", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "RECRUITING", "lat": 63.82842, "lon": 20.25972}, {"facility": "The State Institution of Neurology, Psychiatry and Narcology of NAMS of Ukraine", "city": "Kharkiv", "state": "", "country": "Ukraine", "status": "RECRUITING", "lat": 49.98177, "lon": 36.25475}, {"facility": "Medical Center of LLC Medical Center Dopomoga Plus", "city": "Kyiv", "state": "", "country": "Ukraine", "status": "RECRUITING", "lat": 50.45466, "lon": 30.5238}, {"facility": "Communal Non-Profit Enterprise of Lviv Regional Council, Lviv Regional Clinical Hospital, Neurological Department", "city": "Lviv", "state": "", "country": "Ukraine", "status": "RECRUITING", "lat": 49.83826, "lon": 24.02324}], "contact_phone": "+33(0)147200014", "contact_email": "clinical@ab-science.com", "eligibility": {"criteria": "Main inclusion criteria include:\n\n* Patients diagnosed with laboratory supported probable, clinically probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria\n* Patient with a familial or sporadic ALS\n* ALS disease duration from diagnosis no longer than 24 months at the screening visit\n* Patient treated with a stable dose of riluzole (100 mg/day) for at least 12 weeks days prior to the baseline visit\n* Patient with an ALSFRS-R score progression between onset of the disease and screening of \\> 0.3 per month, confirmed with an ALSFRS-R score progression of \u2265 1 point during a 12-week run-in period between screening and randomization.\n* Patient with a score, at screening, of at least 26 overall, including a score of at least 3 on item #3 and at least 2 on each of the 12 ALSFRS-R individual component items and with a score, at randomization, of at least 2 on each of the 12 ALSFRS-R individual component items\n\nMain exclusion criteria include:\n\n* Patient with dementia or significant neurological, psychiatric, systemic or organic disease, uncontrolled or that may interfere with the conduct of the trial or its results\n* Patient with a FVC \\< 60% predicted normal value for gender, height, and age at screening and baseline\n* Pregnant, or nursing female patient", "sex": "ALL", "min_age": "18 Years", "max_age": "81 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Masitinib", "targeting_mechanism": "Tyrosine kinase inhibition that abrogates neuroinflammation and controls microgliosis in ALS.", "targeting_mechanism_pmid": "27400786", "animal_results": "Masitinib significantly prolonged survival and controlled microgliosis, neuroinflammation, and emergence of aberrant glial cells in SOD1G93A rats, with efficacy even when delivered after paralysis onset.", "animal_results_pmid": "27400786", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05003167", "title": "Effectiveness of Expiratory Muscle Strength Training for Improving Communication in ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Purdue University", "summary": "A tele-health treatment study for individuals with early stage ALS with the aim to improve communication, cough response, and respiratory strength. All participants complete a respiratory strength training program using an Expiratory Muscle Strength Training (EMST 150) device from the comfort of their homes for 6 weeks.", "interventions": [{"type": "DEVICE", "name": "Expiratory Muscle Strength Training (EMST-150)"}], "start_date": "2021-01-01", "url": "https://clinicaltrials.gov/study/NCT05003167", "target_entities": [], "locations": [{"facility": "Purdue University", "city": "West Lafayette", "state": "Indiana", "country": "United States", "status": "", "lat": 40.42587, "lon": -86.90807}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of ALS\n* Within the early stages of disease progression (i.e., able to talk, breathe, and eat independently)\n* Speaker of English\n* Have a family member/caregiver willing to assist as needed\n* Have access to an electronic device and internet for tele-health\n\nExclusion Criteria:\n\n* A history of neurological disease (besides ALS)\n* A history of asthma or respiratory problems (e.g., COPD, emphysema)\n* A history of head, neck, or chest surgery (except mastectomy)\n* A history of smoking within the last 5 years\n* Reliance on mechanical ventilation (including CPAP)", "sex": "ALL", "min_age": "40 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05349721", "title": "Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "PTC Therapeutics", "summary": "This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.", "interventions": [{"type": "DRUG", "name": "PTC857"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2022-05-15", "url": "https://clinicaltrials.gov/study/NCT05349721", "target_entities": ["HSPA4"], "locations": [{"facility": "UC Irvine Health ALS and Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Forbes Norris MDA/ALS Research Center at California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Holy Cross Hospital, Phil Smith Neuroscience Institute", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Augusta University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "University of Kansas Medical Center (KUMC) - Landon Center on Aging", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Henry Ford Health System Department of Neurology", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Neurology Associates, P.C. / Somnos Clinical Research", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "University of Nebraska Medical Center", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "", "lat": 41.25626, "lon": -95.94043}, {"facility": "Providence Brain and Spine Institute", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Lewis Katz School of Medicine at Temple Universtiy", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "National Neuromuscular Research Institute", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Nerve and Muscle Center of Texas", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Medical College of Wisconsin", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "STAT Research S.A.", "city": "Ciudad Aut\u00f3noma de Buenos Aires", "state": "Buenos Aires", "country": "Argentina", "status": "", "lat": null, "lon": null}, {"facility": "Iadin Srl.", "city": "Buenos Aires", "state": "", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "Hospital Ramos Mej\u00eda", "city": "Buenos Aires", "state": "", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Brisbane", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.46794, "lon": 153.02809}, {"facility": "Gold Coast Hospital", "city": "Southport", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.96724, "lon": 153.39796}, {"facility": "Calvary Health Care Bethlehem", "city": "Caulfield South", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.89562, "lon": 145.02597}, {"facility": "Austin Health", "city": "Heidelberg", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.75, "lon": 145.06667}, {"facility": "AZ Sint-Lucas Gent", "city": "Ghent", "state": "", "country": "Belgium", "status": "", "lat": 51.05, "lon": 3.71667}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "University hospital Brno, Department of Neurology", "city": "Brno", "state": "", "country": "Czechia", "status": "", "lat": 49.19522, "lon": 16.60796}, {"facility": "FORBELI s.r.o.", "city": "Prague", "state": "", "country": "Czechia", "status": "", "lat": 50.08804, "lon": 14.42076}, {"facility": "CHU de Bordeaux", "city": "Bordeaux", "state": "", "country": "France", "status": "", "lat": 44.84124, "lon": -0.58046}, {"facility": "H\u00f4pital Neurologique Pierre Wertheimer", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "CHU Gabriel Montpied", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "", "lat": 45.77969, "lon": 3.08682}, {"facility": "CHRU Lille - H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU Dupuytren 1 Limoges", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "CHU Gui de Chauliac (Pharmacie Saint-Eloi & Gui de Chauliac, Hopital Saint-Eloi)", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CRMR SLA - MNM du CHU de Nice", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Charite - Universitatsmedizin - Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Hannover Medical School", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "University Hospital Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Universitaetsklinikum Schleswig-Holstein (UKSH) Campus Luebeck, Klinik fuer Neurologie, Praezisionsneurologie", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "", "lat": 53.86893, "lon": 10.68729}, {"facility": "University of Ulm, Dept. of Neurology", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Centro Clinico Nemo Brescia", "city": "Brescia", "state": "", "country": "Italy", "status": "", "lat": 45.53558, "lon": 10.21472}, {"facility": "Istituti Clinici Scientifici Maugeri IRCCS", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Istituto Auxolgoico Italiano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Azienda Ospedaliero Universitaria di Modena", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Maggiore della Carita University Hospital, Neurology department, ALS center", "city": "Novara", "state": "", "country": "Italy", "status": "", "lat": 45.44694, "lon": 8.62118}, {"facility": "ALS Clinical Research Center, University Hospital Policlinico \"P Giaccone\"", "city": "Palermo", "state": "", "country": "Italy", "status": "", "lat": 38.1166, "lon": 13.3636}, {"facility": "IRCCS Fondazione Mondino - Reparto Neuroncologia/Neuroinfiammazione", "city": "Pavia", "state": "", "country": "Italy", "status": "", "lat": 45.19205, "lon": 9.15917}, {"facility": "Policlinico Umberto I", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}, {"facility": "AOU Citta Della Salute e Scienza", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "PTC Clinical Site", "city": "Japanese City", "state": "", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Centrum Medyczne Neuro Protect", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "City Clinic Research Sp. Z o.o", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Unidad Neuromuscular. Servicio de Neurologia Hospital Universitari Vall d'Hebron", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "H. St Pau", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario y Politecnico La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Skanes universitetssjukhus, VE Neurologi", "city": "Malm\u00f6", "state": "", "country": "Sweden", "status": "", "lat": 55.60587, "lon": 13.00073}, {"facility": "Norrlands universitetssjukhus Neurologens Forskningsavdelning", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* ALS with preserved function, defined as:\n\n  1. Onset of the first symptom leading to the diagnosis of ALS \u226424 months at the time of the initial Screening Visit\n  2. Revised EL Escorial criteria of either:\n\n  (i) Clinically definite ALS (ii) Clinically probable ALS\n* A total ALSFRS-R score of at least 34 at the start of the Screening Period\n* No significant respiratory compromise as evidenced by slow vital capacity \u226560% at the start of the Screening Period\n* All chronic concomitant medications (both prescription and over the counter), and non-pharmacologic therapy regimens, excluding standard-of-care therapy riluzole, edaravone, or sodium phenylbutyrate/taurursodiol, should be stable and unchanged from 14 days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study\n* Female participants must have a negative breast cancer imaging screening status (not considered clinically abnormal and/or requiring further evaluation/treatment) within 6 months prior to the Screening Visit, or during the Screening Period.\n* Standard-of-care therapy for the treatment of ALS (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol) should be stable and unchanged from 30 (-3) days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study.\n\nKey Exclusion Criteria:\n\n* Females who are pregnant or nursing or plan to become pregnant during the study\n* Participants with clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular/ischemic disease or any other condition that, in the opinion of the investigator would jeopardize the safety of the participant or impact the validity of the study results\n* Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the investigator or the medical monitor, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant\n* Current participation in any other investigational study with an investigational product or participation within 30 days prior to the start of the Screening Period or 5 half-lives of the previously taken investigational drug, whichever is longer\n* Participant has previously received PTC857\n* Participant is receiving a combination of edaravone and sodium phenylbutyrate/taurursodiol treatment, where applicable, within 30 days prior to the start of the Screening Period\n* For female participants, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "PTC857", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01811355", "title": "Mexiletine for the Treatment of Muscle Cramps in ALS", "phase": "PHASE4", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Bjorn Oskarsson, MD", "summary": "The purpose of this study is to determine if mexiletine is effective for the treatment of muscle cramps in Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Mexiletine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2013-05", "url": "https://clinicaltrials.gov/study/NCT01811355", "target_entities": ["Voltage-gated sodium channel"], "locations": [{"facility": "UCD Telehealth Network - Lake Almanor Clinic", "city": "Chester", "state": "California", "country": "United States", "status": "", "lat": 40.30627, "lon": -121.23191}, {"facility": "UCSD Department of Neurosciences ALS Clinical Trials (ACT) Program", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "UCLA Neuromuscular Research Program", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "UCD Telehealth Network", "city": "Multiple Locations", "state": "California", "country": "United States", "status": "", "lat": null, "lon": null}, {"facility": "UC Irvine Health ALS & Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "UC, Davis Medical Center ALS Clinic", "city": "Sacramento", "state": "California", "country": "United States", "status": "", "lat": 38.58157, "lon": -121.4944}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS diagnosed according to El Escorial criteria (Awaji version) as: Possible, Probable, or Definite.\n* Experiencing cramps as a moderate or severe symptom as defined by willingness to take a medication for the symptom\n* \u22652 cramps per week during run in week\n* Life expectancy \\> 6 months, estimated by clinician\n* Able to take drug capsule by mouth\n* No significant EKG abnormality on screening\n* aspartate aminotransferase / alanine aminotransferase \\<2x upper limit of normal measured at screening\n* Having successfully filled out the cramp diary and cramp and fasciculation scales on six out of the last seven days of run in period\n\nExclusion Criteria:\n\n* Inability to communicate by telephone or email\n* Allergy/ known sensitivity to mexiletine\n* Prior use of mexiletine\n* AV block unless subject has pacemaker\n* Cardiac arrhythmia\n* Prior myocardial infarction\n* Other significant EKG abnormality\n* Liver disease\n* History of leucopenia (WBC \\<3,500/mm3)\n* Epilepsy\n* Other serious and unstable medical condition\n* Pregnant woman\n* Breastfeeding woman\n* Active drug or alcohol use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements\n* Use of quinidine (alone or as a component of Nuedexta\u00ae) during the study\n* Inability or unwillingness of subject to give written informed consent\n* Woman of childbearing potential, not willing to use at least two approved methods of contraception\n* Use of a prohibited medication during study", "sex": "ALL", "min_age": "21 Years", "max_age": "89 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Mexiletine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02682030", "title": "The Use of Airway Clearance Devices in ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cedars-Sinai Medical Center", "summary": "The investigator is examining the use of one airway clearance medical device compared to the use of two airway clearance medical devices together in patients with amyotrophic lateral sclerosis (ALS). More specifically, the investigator wants to know how effective the use of either a mechanical High Frequency Chest Compression (HFCC) device is on its own or the use of both a mechanical High Frequency Chest Compression (HFCC) device and Cough Assist together to maintain a healthy airway and clear secretions.\n\nThe first device is a passive form of mechanical High Frequency Chest Compression (HFCC), which was designed to help clear the airway of mucus and other secretions through mechanical knocking of the chest area. The second device, called a Cough Assist, aids patients to clear mucus and secretions that they would otherwise be unable to clear with coughing. This study will enroll up to 20 people in total at CSMC.", "interventions": [{"type": "DEVICE", "name": "High Frequency Chest Compression Device (HFCC)"}, {"type": "DEVICE", "name": "Cough Assist"}], "start_date": "2016-03", "url": "https://clinicaltrials.gov/study/NCT02682030", "target_entities": [], "locations": [{"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Suspected, possible, probable, Probable (Lab-Supported), or Definite ALS according to El Escorial Criteria\n2. Males and females age 18 and above\n3. Novel to airway clearance device use\n4. Forced vital capacity \u2264 75% of predicted\n\nExclusion Criteria:\n\n1. Any contraindication for pulmonary ventilation scan including allergy to radioisotopes\n2. Any contraindication for use of a pulmonary clearance device\n\n   * Susceptibility to pneumothorax\n   * Recent (within 30 days) barotrauma\n   * Unstable head or neck injury\n   * Active hemorrhage with hemodynamic instability", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01277315", "title": "Safety and Tolerability of Anakinra in Combination With Riluzol in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Charite University, Berlin, Germany", "summary": "Amyotrophic Lateral Sclerosis (ALS) is an adult neurodegenerative disease that is caused by a selective degeneration of the motor nerve cells in the cortex and myelon. As a result of motor neurodegeneration, a progredient paralysis of the extremities and of the speaking, swallowing, and breathing musculature develops. ALS leads to death by respiratory insufficiency in a mean course of 3-5 years. So far, Riluzole is the only approved neuroprotective medication which effects a slight lifespan prolongation of 1.5 - 2.5 months. Riluzole inhibits the presynaptic glutamate release and lowers the level of glutamate liberated by activated microglia.\n\nThe researchers propose an investigational therapy of ALS with subcutaneous administration of 100 mg of Anakinra. The neuronal inflammation is a crucial pathogenetic factor of the motor neuron degeneration. Inflammatory processes are detectable in sporadic ALS, in the autosomal-dominant form of ALS and in transgenic mouse model. The rationale of this clinical trial is based on the anti-inflammatory effect of Anakinra. One of the key mediators of inflammatory response is Interleukin-1. Anakinra is a recombinant produced Interleukin-1 receptor antagonist. This gives Anakinra anti-inflammatory attributes that presumably reduce motor neuron degeneration and disease progression.", "interventions": [{"type": "DRUG", "name": "Anakinra"}], "start_date": "2011-02", "url": "https://clinicaltrials.gov/study/NCT01277315", "target_entities": ["IL-1", "neuroinflammation"], "locations": [{"facility": "Charit\u00e9 University Hospital", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients between 18 and 80 years of age\n* Clinical diagnosis of amyotrophic lateral sclerosis with predominant affection of the lower motor neuron or the clinical ALS variant of progressive muscular atrophy (PMA)\n* Clinical signs of lower motor neuron degeneration in at least one anatomic region beyond the brain stem\n* Sporadic and familial ALS\n* Onset of paresis six months to four years before study inclusion\n* Treatment with riluzol 100mg/d at least 1 month before study inclusion\n\nExclusion Criteria:\n\n* Diagnosis of amyotrophic lateral sclerosis with predominant affection or the upper motor neuron without clinical signs of a concurrent affection of the lower motor neuron in at least one anatomic region beyond the brain stem (spastic ALS) - Diagnosis of primary lateral sclerosis (PLS)\n* Patients with known intolerance to anakinra, riluzol or one of the additives\n* Clinically severe hypoventilation syndrome with vital capacity \\< 50%\n* Pregnancy or breastfeeding\n* Continuous non-invasive ventilation with ventilator-free time \\< 2 hours - Tracheotomy and mechanical ventilation\n* Laboratory parameters outside the normal range that correspond to a clinically severe cardiovascular, pulmological, hematological, hepatological, metabolic or renal disease\n* Malignancies\n* Severe renal insufficiency (creatinine clearance \\< 30 ml/min)\n* History of recurrent infections or a disease that may predispose to infections\n* Severe neutropenia (absolute neutrophil count \\< 1.5 x 109/l)\n* Monoclonal gammopathy of unknown significance\n* Infections including infections with HIV and hepatitis B and C\n* Dementia and unable to give informed consent\n* History of epilepsy and epileptic seizures\n* Contraindication to E coli-derived proteins, anakinra or any components of the product\n* Concurrent therapy of anakinra and etanercept or other TNF blocking agents", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Anakinra", "targeting_mechanism": "IL-1 receptor antagonist that reduces neuroinflammation", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03367650", "title": "Epidemiology and Genetics of the Amyotrophic Lateral Sclerosis in the French West Indies", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Centre Hospitalier Universitaire de la Guadeloupe", "summary": "The diagnosis and the follow-up of the patients reached of SLA is centralized, since a few years, at the the Caribbean Reference center of the rare neurological diseases (CERCA lab\u00e9lis\u00e9 in 2006) in Martinique and at the Unity of coverage of the neuromuscular Diseases, SLA and the rare neurological diseases (create in 2010) in Guadeloupe. Several phenotypic characteristics seemed to us to take out again data collected during the follow-up of the patients (26 in Guadeloupe, since the creation of the unity) in particular patients' high proportion of exceptionally long evolution (more than 10 years). Besides, we diagnosed several cases (10 cases in Guadeloupe since 2000) of association SLA- Parkinsonien Syndrome.\n\nThis association, considered as exceptional could establish a particular phenotypic entity which we would like to describe. We are interested also originally geographical of the patients, with the hypothesis that he could exist in the Antilles one or several geographical isolates of the disease allowing to lead a \u00e9tiologique investigation in search of a possible genetic or environmental cause.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Blood sample and environmental survey"}], "start_date": "2014-05-13", "url": "https://clinicaltrials.gov/study/NCT03367650", "target_entities": [], "locations": [{"facility": "Hospital University Center of Pointe-\u00e0-Pitre", "city": "Pointe-\u00e0-Pitre", "state": "", "country": "Guadeloupe", "status": "RECRUITING", "lat": 16.23638, "lon": -61.53459}, {"facility": "Hospital University Center of Martinique", "city": "Fort-de-France", "state": "", "country": "Martinique", "status": "RECRUITING", "lat": 14.60365, "lon": -61.07418}], "contact_phone": "0590 93 46 86", "contact_email": "chantal.lerus@chu-guadeloupe.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patient or third-party responsible for receiving information on the study and who signed informed consent ;\n* Patient age over 18 years;\n* Patient living in the Antilles;\n* Patient with ALS or SLP (primary lateral sclerosis, pure central form of ALS).\n\nExclusion Criteria:\n\n* Patient non-affiliated to the social security scheme ;\n* in case of difficulty of monitoring patient, exclusion of the longitudinal study.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04889248", "title": "Inspiratory Muscle Training With Powerbreath Device in Patients With ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Universidad Francisco de Vitoria", "summary": "Abstract:\n\nContext/background: people affected by Amyotrophic Lateral Sclerosis (ALS) see their own life totally disturbed after the diagnosis. This disease also courses, apart from the functional and depressing worsening, with internal damage manifested by a cardio respiratory deterioration. There are not many clinical studies publications about this disease given that is considered a weird illness with short prognosis.\n\nObjectives: to examine the effects of the inspiratory muscle training (IMT) on respiratory muscle strength, heart rate variability (HRV), quality of life and mood in patients with ALS.\n\nMethods: 20 volunteer patients, male and female, with ALS, bulbar or spinal will take part of the cuasi-experimental study and they will be divided into two groups: an experimental group (n = 10) and a control group (n = 10). The Maximum Inspiratory Pressure (PIM), the HRV, the quality of life and mood will be measured. The participants of experimental group will conduct 30 inspirations per day, 15 in the morning and 15 in the evening, 5 days per week, through 8 weeks. The resistance of the training in the experimental group will be increase acording to the PIM measured at the first visit. During the first week, the resistance will be at 30% of PImax, weeks 2 and 3 at 40%, weeks 4 and 5 at 50% and the last 3 weeks at 60%. After 8 weeks, all participants will fill up again all scales and post training measurements will be taken.", "interventions": [{"type": "OTHER", "name": "Inspiratory Muscle Training with Powerbreath IMT device."}], "start_date": "2021-05-17", "url": "https://clinicaltrials.gov/study/NCT04889248", "target_entities": [], "locations": [{"facility": "Universidad Francisco de Vitoria", "city": "Pozuelo de Alarc\u00f3n", "state": "Madrid", "country": "Spain", "status": "", "lat": 40.43293, "lon": -3.81338}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n\\- Subjects with ALS\n\nExclusion Criteria:\n\n* PImax more than 30mmH2O\n* Score higher than 2 in GDS Reisberg scale\n* Using already respiratory devices during the day (except night CPAPs support) more than 14h/day.\n* Unstable medical disease for the last 3 years.\n* Use of IMT contraindicated for medical reasons.", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Inspiratory muscle training to strengthen respiratory muscles and counteract cardiorespiratory deterioration in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06765499", "title": "The Study Evaluating the Improvement of Nutritional Status and Frailty With Silkworm Pupa Powder Among Patients With Motor Neuron Disease", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "First People's Hospital of Hangzhou", "summary": "Motor Neuron Disease (MND) is the result of dysfunction of the upper motor neurons in the precentral gyrus of the frontal lobe or the lower motor neurons in the ventral horn of the spinal cord. Amyotrophic lateral sclerosis (ALS) is the most common, disabling, and fatal motor neuron disease in adults. Sarcopenia is a syndrome characterized by progressive loss of skeletal muscle mass, accompanied by a reduction in muscle strength and (or) function, and it is an important feature of MND. Aging is an objective and inevitable process that involves the gradual degeneration and loss of physiological functions in various tissues, organs, and cells. With the continuous accumulation of various injuries, the body eventually exhibits signs of frailty such as fatigue, reduced muscle strength, and weight loss. Data from adult ALS patients indicate that 58% of patients are at risk of frailty. Silkworm pupa contains high-quality animal protein and has a wide range of activities in antioxidant, antitumor, antibacterial, and immune enhancement, making it highly nutritious and medicinally valuable. Silkworm pupa extracts can enhance grip strength in older adults with relatively low skeletal muscle mass. As a natural food ingredient with high safety, the value of silkworm pupa in ALS patients lacks corresponding research, which limits its further application in clinical practice. This study aims to select ALS patients as the research subjects and use a randomized, controlled, double-blind prospective study design to evaluate the effectiveness of silkworm pupa tablets in improving sarcopenia, frailty, and quality of life in ALS patients. The study strives to improve the frailty condition of ALS patients and enhance their quality of life by supplementing nutrition, thereby providing new strategies for comprehensive intervention and management of ALS patients.", "interventions": [{"type": "OTHER", "name": "Chinese medicine - Silkworm pupa powder"}, {"type": "OTHER", "name": "Ultra-low dose silkworm pupa powder"}], "start_date": "2025-03-04", "url": "https://clinicaltrials.gov/study/NCT06765499", "target_entities": ["sarcopenia"], "locations": [{"facility": "Hangzhou First People's Hospital", "city": "Hangzhou", "state": "Zhejiang", "country": "China", "status": "RECRUITING", "lat": 30.29365, "lon": 120.16142}], "contact_phone": "0571-56007429", "contact_email": "757318708@qq.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Voluntarily participate in the clinical study, fully understand and be informed about the study, and sign the Informed Consent Form (ICF); willing to follow and capable of completing all trial procedures.\n* Gender is not limited, age at the time of signing ICF: \u226518 years old, \u226480 years old; if the ALS patient is at least 18 years old, then the weight must be over 40 kilograms.\n* Diagnosis conforms to the confirmed and probable ALS in the ALS2020 diagnostic criteria (Gold Coast Criteria).\n* Clinical, neurophysiological, or pathological examination confirms evidence of lower motor neuron involvement.\n* Frailty Phenotype scale (FP) \u22651.\n* Exclude other diseases.\n* Agree to provide peripheral blood, fecal, and urine samples for biomarker analysis during the study period.\n\nExclusion Criteria:\n\n* Patients with other neurological diseases similar to ALS symptoms or affecting drug efficacy evaluation, such as cervical spondylosis, lumbar disease, dementia, etc.\n* Patients with other autoimmune diseases, such as Multiple Sclerosis (MS), Polymyositis, Myasthenia Gravis, Guillain-Barr\u00e9 Syndrome, Ankylosing Spondylitis, Rheumatoid Arthritis, Systemic Lupus Erythematosus, Vitiligo, etc.\n* Severe renal insufficiency: Creatinine clearance \\< 30 mL/min (Cockcroft-Gault formula), or other known severe renal insufficiency diseases.\n* Severe liver damage: ALT, AST \\> 3 times the upper limit of normal, or other known liver diseases such as acute and chronic active hepatitis, cirrhosis, etc.\n* Patients with severe pulmonary function insufficiency such as Chronic Obstructive Pulmonary Disease (COPD), pulmonary fibrosis, etc.\n* During the screening period, patients with acute myocardial infarction or interventional treatment within the last 6 months, heart failure patients (classified as NYHA class III-IV patients).\n* Patients with other severe primary diseases of the nervous system, heart, lungs, hematopoietic system, or endocrine system, and mental illness patients.\n* Those suspected or confirmed to have a history of alcohol or drug abuse.\n* Expected survival \u2264 3 months.\n* Pregnant or breastfeeding women, subjects of reproductive age (including male subjects who have had heterosexual intercourse and their female partners with childbearing potential) who plan to become pregnant or are unwilling to take effective contraceptive measures from the start of screening to 3 months after discontinuing medication.\n* Those who are allergic to known ingredients of the trial products; or have a history of drug allergies or severe allergic diseases (anaphylactic shock, etc.).\n* During the study, individuals assessed by researchers as potential drug abusers who may affect the efficacy of therapeutic drugs.\n* Presence of other severe physical or mental diseases or abnormal laboratory tests that may increase the risk of participating in the study and patients deemed unsuitable for participation by the researcher.\n* Patients with malignant tumors.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Silkworm pupa powder", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06165172", "title": "Early Feasibility Study of the MyoRegulator\u00ae for Treatment of ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "PathMaker Neurosystems Inc.", "summary": "This is a single-center, single-arm, open-label study aiming to assess the safety and feasibility of the MyoRegulator\u00ae device when used to treat individuals with amyotrophic lateral sclerosis (ALS). This study is the first use of the MyoRegulator\u00ae device to treat individuals with ALS. The main objective of this study is to confirm that individuals with ALS can tolerate the study treatment regimen without any evidence of serious adverse events related to the use of the device.\n\nThe MyoRegulator\u00ae device is a non-significant risk (NSR) investigational non-invasive neuromodulation device that uses multi-site direct current (multi-site DCS) stimulation. It has been used in two completed clinical trials evaluating its efficacy to treat post-stroke muscle spasticity and is currently being evaluated in a third trial in this post-stroke population.", "interventions": [{"type": "DEVICE", "name": "MyoRegulator\u00ae"}], "start_date": "2023-06-21", "url": "https://clinicaltrials.gov/study/NCT06165172", "target_entities": [], "locations": [{"facility": "Spaulding Rehabilitation Hospital", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* 18 years of age or older\n* A clinical diagnosis of ALS as confirmed by medical history\n* Willing to forgo botulinum toxin, phenol or alcohol injections, intrathecal baclofen, digitalis, and morphine for the study duration\n* Willing to refrain from participation in any other clinical trial for the duration of this study\n* Willing to forgo pregnancy for the duration of the study\n* Willing and able to give informed consent or have informed consent provided for them by their legal guardian\n* Cognitive function sufficient to understand the study and follow instructions (per interview with appropriate clinician)\n\nExclusion Criteria:\n\n* Study participants who are on permanent assisted ventilation (PAV) defined as \\>22h of noninvasive or invasive ventilation a day for \\> 7 consecutive days.\n* Implanted intrathecal pump\n* Prior botulinum toxin injection(s) within 12 weeks of study enrollment\n* Prior phenol or alcohol injections within 6 months of study enrollment\n* Presence of potential risk factors for trans-spinal direct current stimulation:\n* Damaged skin at the stimulation sites (i.e., skin with ingrown hairs, acne, razor nicks, wounds that have not healed, recent scar tissue, broken skin, etc.)\n* Lack of sensory perception at the stimulation sites\n* Presence of an electrically, magnetically, or mechanically activated implant (including cardiac pacemaker) or any other electrically sensitive support system with the exception of loop recorders\n* Ferrous metal in the path of the current flow (jewelry must be removed during stimulation)\n* Past history of epileptic seizures or unexplained spells of loss of consciousness during the previous 36 months\n* Any medical condition that would prevent the participant from being able to participate in the clinical outcome measures\n* Pregnant females, as determined by a pregnancy test at enrollment (in females of child-bearing potential)", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MyoRegulator\u00ae", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03548311", "title": "Clinical Trial of Ultra-high Dose Methylcobalamin for ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Eisai Co., Ltd.", "summary": "To examine the clinical efficacy and safety of ultra-high dose (50mg, im, twice a week) methylcobalamin in retarding the progression of symptoms in amyotrophic lateral sclerosis (ALS) patients, we enroll ALS patients diagnosed by Updated Awaji Criteria within 12 months after the clinical onset. First they are followed for 12 weeks with Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) scores, and only those who exhibit drops of 1-2 points are allowed to enter into the test period. A total of 128 patients are randomized and the half having placebo. They are blindly evaluated for drops of ALSFRS-R in 16 weeks, as the primary outcome. After this, all subjects receive methylcobalamin.", "interventions": [{"type": "DRUG", "name": "methylcobalamin"}, {"type": "DRUG", "name": "saline solution"}], "start_date": "2017-11-01", "url": "https://clinicaltrials.gov/study/NCT03548311", "target_entities": ["mitochondrial_function"], "locations": [{"facility": "Nagoya University Hospital", "city": "Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "Miyoshi Neurological Clinic", "city": "Miyoshi", "state": "Hiroshima", "country": "Japan", "status": "", "lat": 34.8, "lon": 132.85}, {"facility": "Sapporo Medical University Hospital", "city": "Sapporo", "state": "Hokkaido", "country": "Japan", "status": "", "lat": 43.06667, "lon": 141.35}, {"facility": "Kobe Central Munincipal Medical center", "city": "Kobe", "state": "Hy\u014dgo", "country": "Japan", "status": "", "lat": 34.6913, "lon": 135.183}, {"facility": "Ioh National Hospital", "city": "Kanazawa", "state": "Ishikawa-ken", "country": "Japan", "status": "", "lat": 36.6, "lon": 136.61667}, {"facility": "Kitasato University East Hospital", "city": "Sagamihara", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.56707, "lon": 139.24167}, {"facility": "Shiga Medical University Hospital", "city": "\u014ctsu", "state": "Shiga", "country": "Japan", "status": "", "lat": 35.0, "lon": 135.86667}, {"facility": "Chiba University Hospital", "city": "Chiba", "state": "", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "Murakami Karindo Hospital", "city": "Fukuoka", "state": "", "country": "Japan", "status": "", "lat": 33.6, "lon": 130.41667}, {"facility": "Okayama University Hospital", "city": "Okayama", "state": "", "country": "Japan", "status": "", "lat": 34.65, "lon": 133.93333}, {"facility": "Tokushima University Hospital", "city": "Tokushima", "state": "", "country": "Japan", "status": "", "lat": 34.06667, "lon": 134.56667}, {"facility": "Juntendo University Hospital", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "Toho University Hospital", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "Teikyo University Hospital", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "Tokyo Metropolitan Neurological Hospital", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "Wakayama Medical University Hospital", "city": "Wakayama", "state": "", "country": "Japan", "status": "", "lat": 34.23333, "lon": 135.16667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nTreatment and OLE Phase:\n\n* ALS patients within 12 months after clinical onset at the entry\n* Diagnosed based on updated Awaji criteria: definite, probably or laboratory supported probable\n* Decline of ALSFRSR being 1 or 2 during observation period of 12 weeks\n* Japanese Clinical Severity Scale 1 or 2\n* Those who can visit the participating medical centers\n\nTsunagi Phase:\n\n\\- The subjects for the Tsunagi phase are patients with ALS who are continuing the administration of this drug at the time of approval, and have obtained consent for the transition to the Tsunagi phase.\n\nExclusion Criteria:\n\nTreatment and OLE Phase:\n\n* Those who have tracheostomy\n* Those who had NIPPV\n* %FVC\\<60%\n* Those who have Chronic Obstructive Pulmonary Disease (COPD)\n* Those who have symptoms and signs of B12 deficiency\n* Those who had edaravone less than 4 weeks prior to entry\n* Those who changed the schedule and dosing of riluzole\n* Those who have dementia\n* Those who have the possibility of pregnancy\n* Those who have serious respiratory or cardiac diseases\n* Those who have malignancies\n* Those who participated other clinical trials within 12 weeks\n* Those who have allergies to B12 and related compounds\n\nTsunagi Phase:\n\nNot applicable.", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "methylcobalamin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05837507", "title": "Amyotrophic Lateral Sclerosis Non-invasive Ventilation Exchange", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Montpellier", "summary": "The median survival of patients with amyotrophic lateral sclerosis (ALS) is 3 to 5 years and mortality is mainly related to respiratory failure. Non-invasive ventilation (NIV) and multidisciplinary management improve the quality of life and survival of patients. However, patients have mobility difficulties related to the progressive worsening of functional disabilities. The research team hypothesize that the use of a multimodal digital platform, including in particular telemonitoring of NIV and teleconsultation, will slow down the evolution of the disabilities of patients with ALS and improve their quality of life.", "interventions": [{"type": "OTHER", "name": "Telerehabilitation solution (m-Rehab)"}, {"type": "OTHER", "name": "Usual management including NIV."}], "start_date": "2023-07-05", "url": "https://clinicaltrials.gov/study/NCT05837507", "target_entities": [], "locations": [{"facility": "Uh Montpellier", "city": "Montpellier", "state": "Montpellier", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Clinique du Mill\u00e9naire", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients between 30 and 85 years old\n* Diagnosis of ALS\n* Indication and acceptance of non-invasive ventilation\n* Patient equipped with suitable equipment (tablet, computer, telephone, etc.) with an internet connection at home\n* Patient able to read and understand the procedure, and able to express consent for the study protocol\n\nExclusion Criteria:\n\n* Treatment with non-invasive ventilation in the previous three months\n* Refusal/inability to use a smart phone or digital device\n* Patient currently participating or having participated in the month preceding inclusion in another clinical interventional research that could impact the study, this impact is left to the discretion of the investigator.\n* Subject under guardianship or curators\n* Subject not affiliated to a social security scheme, or not a beneficiary of such a scheme.\n* Subject deprived of liberty by judicial or administrative decision\n* Pregnant, parturient, breastfeeding women\n* Refusal to give consent", "sex": "ALL", "min_age": "30 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Telerehabilitation platform with telemonitoring to support non-invasive ventilation management and multidisciplinary care in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06671236", "title": "Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Novabio Therapeutics", "summary": "An open, multi- center phase \u2160 clinical study evaluating the safety and efficacy of autologous human polyclonal regulatory T cell injection (NP001 cell injection) in patients with Neurodegenerative diseases (ALS).", "interventions": [{"type": "BIOLOGICAL", "name": "Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)"}], "start_date": "2024-11-21", "url": "https://clinicaltrials.gov/study/NCT06671236", "target_entities": ["neuroinflammation"], "locations": [{"facility": "The First Affiliated Hospital of Zhengzhou University", "city": "Zhengzhou", "state": "Henan", "country": "China", "status": "RECRUITING", "lat": 34.75778, "lon": 113.64861}], "contact_phone": "609-716-7786; +86 15800843507", "contact_email": "lumingqi@novabiotx.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n\\-\n\nPatients must meet all of the following criteria to be eligible for enrollment in this study:\n\n1. Male or female patients aged 18 to 70 years;\n2. According to current international diagnostic criteria:\n\n   ALS: defined by the Gold Coast Diagnostic Criteria (Shefner, 2020) as having a diagnosis of sporadic or familial amyotrophic lateral sclerosis (ALS), diagnosed as a probable, probable, or definite patient with laboratory support according to the World Federation of Neurology El Escorial criteria;\n3. If there is a stable dose for more than one month prior to study entry. For example, patients with ALS can continue treatment with riluzole (Rilutek\u00ae) and/or edaravone (Radicava\u00ae);\n4. Patients must have \\> two weeks after the end of major surgery and after the completion of participation in other research trials;\n5. Patients must have recovered from clinical toxicity (CTCAE \\[5th Edition\\] toxicity values have resolved to \\< 2);\n6. Serum creatinine less than or equal to 2.0 mg/dL;\n7. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \\< 3 x upper limit of normal;\n8. Bilirubin \\< 1.5 (except Gilbert's disease);\n9. Lung slow vital capacity (SVC) \\> 70% of predicted normal;\n10. No history of abnormal bleeding tendency;\n11. Informed consent must be obtained prior to performing any study-related procedures that are not part of standard medical care, with the understanding that the participant may withdraw from the study without influence for the future medical care.\n\nExclusion Criteria:\n\n\\-\n\nSubjects with any of the following cannot be enrolled in this study:\n\n1. uncontrolled infection;\n2. \\< 3 drugs do not adequately control hypertension;\n3. Documented history of pulmonary embolism within 6 months of enrollment;\n4. Clinically significant cardiology, defined as: myocardial infarction, NYHA-graded class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmia, or ECG evidence of acute ischemia or abnormal conduction system within 6 months prior to enrollment;\n5. Patients with a history of coronary artery bypass grafting or angioplasty will be evaluated by cardiology and considered on a case-by-case basis;\n6. Seropositive for HIV, hepatitis B or hepatitis C;\n7. Pregnant or lactating patients;\n8. Patients of childbearing potential or males with partners of childbearing potential who are unwilling to use contraception;\n9. Participation in any other interventional study;\n10. Treatment with another investigational drug, biologic, or device within 30 days or 5 half-lives (whichever is longer) of the screening period. Patient participation in observational/non-interventional clinical studies will be discussed with the Medical Monitor;\n11. Prior treatment with ALS gene or cell therapy;\n12. History of clinically significant tumor, liver or kidney disease, or other uncontrolled disease;\n13. presence of a feeding tube;\n14. Current use of antipsychotics, antiepileptic drugs (except benzodiazepines, gabapentin, pre-Bahrain) or class 1 (e.g., flecainide) or class 3 (e.g., amiodarone) antiarrhythmic drugs;\n15. Subjects who, in the opinion of the investigator, are at significant risk of suicide;\n16. Other conditions that the investigator considers unsuitable for enrollment.", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous Human Polyclonal Regulatory T Cells (NP001 Cell Injection)", "targeting_mechanism": "Modulation of neuroinflammation through adoptive transfer of regulatory T cells to suppress CNS inflammation in ALS.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02290886", "title": "A Multicenter Phase I/II Clinical Trial to Evaluate Safety of Mesenchymal Stem Cell in Patients With Amyotrophic Sclerosis Lateral", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Andalusian Initiative for Advanced Therapies - Fundaci\u00f3n P\u00fablica Andaluza Progreso y Salud", "summary": "A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "OTHER", "name": "Intravenous administration of placebo"}, {"type": "DRUG", "name": "Intravenous administration of 1 million of MSC"}, {"type": "DRUG", "name": "Intravenous administration of 2 million of MSC"}, {"type": "DRUG", "name": "Intravenous administration of 4 million of MSC"}], "start_date": "2014-07", "url": "https://clinicaltrials.gov/study/NCT02290886", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Hospital Regional Universitario Reina Sof\u00eda", "city": "C\u00f3rdoba", "state": "", "country": "Spain", "status": "", "lat": 37.89155, "lon": -4.77275}, {"facility": "Hospital Regional Universitario de M\u00e1laga", "city": "M\u00e1laga", "state": "", "country": "Spain", "status": "", "lat": 36.72016, "lon": -4.42034}, {"facility": "Hospital Universitario Virgen Macarena, Servicio de Neurolog\u00eda", "city": "Seville", "state": "", "country": "Spain", "status": "", "lat": 37.38283, "lon": -5.97317}, {"facility": "Hospital Universitario Virgen del Roc\u00edo", "city": "Seville", "state": "", "country": "Spain", "status": "", "lat": 37.38283, "lon": -5.97317}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Women and males over 18-year-old.\n2. Good understanding of the protocol and aptitude to grant the informed assent.\n3. Diagnosis of sporadic ALS, with diagnosis of certainty, that is to say, definite or probable, in agreement with the criteria of \"El Escorial\", of the World Federation of Neurology.\n4. Forced vital capacity of at least 50 % of the one that would correspond to them for sex, height and age.\n5. More than 6 and less than 36 months of evolution of the disease (from the beginning of the symptoms).\n6. Possibility of obtaining, at least, 50gr of adipose tissue.\n7. Treatment with riluzole, for at least, a month before the inclusion.\n\nExclusion Criteria:\n\n1. Any concomitant disease that under investigator's criteria could concern the measures of the clinical variables of the trial (hepatic, renal or cardiac insufficiency, diabetes mellitus, etc).\n2. Previous therapy with stem cells.\n3. Participation in another clinical trial during 3 months previous to the entry in this trial.\n4. Any disease lymphoproliferative\n5. Tracheostomy and /or gastrostomy.\n6. Haemophilia, diathesis hemorrhagic or anticoagulative current therapy.\n7. Hypersensitivity known to the bovine foetal whey or the gentamicin.\n8. Medical precedents of infection of the HIV or any serious condition of immunocompromised.\n9. Positive HBV or HCV serology\n10. Levels of creatinine in whey \\> 3.0 in subjects not submitted to haemodialysis.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous Mesenchymal Stem Cells (MSC) from adipose tissue", "targeting_mechanism": "Reduction of neuroinflammation through cell therapy to address inflammation-driven neurodegeneration in ALS.", "targeting_mechanism_pmid": "37433768", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06992596", "title": "UAE-PRIME: A Feasibility Study of a Precise Robotically Implanted Brain-Computer Interface for the Control of External Devices", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuralink Corp", "summary": "The UAE-PRIME Study is a feasibility study designed to assess the initial clinical safety and functionality of the Neuralink N1 Implant and R1 Robot. This study involves participants who have tetraparesis, tetraplegia, or a diagnosis that may lead to these conditions.\n\nThe N1 Implant is a wireless, rechargeable device mounted on the skull, connected to electrode threads that are inserted into the brain by the R1 Robot, which is a robotic device specifically designed for this procedure.", "interventions": [{"type": "DEVICE", "name": "N1 Implant"}, {"type": "DEVICE", "name": "R1 Robot"}], "start_date": "2025-05-09", "url": "https://clinicaltrials.gov/study/NCT06992596", "target_entities": [], "locations": [{"facility": "Cleveland Clinic Abu Dhabi (CCAD)", "city": "Abu Dhabi", "state": "", "country": "United Arab Emirates", "status": "RECRUITING", "lat": 24.45118, "lon": 54.39696}], "contact_phone": "(877) 398-4465", "contact_email": "clinical-team-ct@neuralink.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* (a) A diagnosis of a spinal cord injury (\\>12 months), stroke (\\>12 months), or other neurological condition causing the participant to experience bilateral upper limb motor impairment, with no expectation of recovery.\n\nOR (b) A diagnosis of Amyotrophic Lateral Sclerosis (ALS) or other progressive neurological condition where the natural history of the disease is well understood and where there is tetraparesis and the expectation, in the view of the participant's treating neurologist, that the disease will progress such that the participant will meet criteria 1a within 1 year of recruitment.\n\n* Life expectancy \u2265 12 months.\n* Ability to communicate verbally or with the use of a computer or communication aid, and working proficiency in English.\n* Presence of a stable caregiver\n\nExclusion Criteria:\n\n* Moderate to high risk for serious perioperative adverse events\n* Morbid obesity (Body Mass Index \\> 40)\n* History of poorly controlled seizures or epilepsy\n* History of poorly controlled diabetes\n* Requires magnetic resonance imaging (MRI) for any ongoing medical conditions\n* Acquired or hereditary immunosuppression\n* Smoking tobacco or use of other tobacco products \\> once per month within the last year.\n* Psychiatric or psychological disorder\n* Pre-existing damage to the cortical region of interest (as determined by MRI)\n* Any condition which, in the opinion of the Investigator, would compromise your ability to safely participate in the study or undergo the implantation procedure", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "N1 Implant and R1 Robot (Brain-Computer Interface)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03567213", "title": "Investigation on the Cortical Communication (CortiCom) System", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "The CortiCom system consists of 510(k)-cleared components: platinum PMT subdural cortical electrode grids, a Blackrock Microsystems patient pedestal, and an external NeuroPort Neural Signal Processor. Up to two grids will be implanted in the brain, for a total channel count of up to 128 channels, for six months. In each participant, the grid(s) will be implanted over areas of cortex that encode speech and upper extremity movement.", "interventions": [{"type": "DEVICE", "name": "Surgical implantation of CortiCom system"}], "start_date": "2021-12-14", "url": "https://clinicaltrials.gov/study/NCT03567213", "target_entities": [], "locations": [{"facility": "Johns Hopkins Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "410-955-6772", "contact_email": "ncrone@jhmi.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinical diagnosis of tetraplegia (quadriplegia), brainstem stroke , amyotrophic lateral sclerosis (ALS) or Locked-in Syndrome (LIS)\n* Tetraplegia diagnosis, ALS diagnosis, stroke, or LIS etiology onset occurred at least one year prior to enrollment\n* Complete or incomplete tetraplegia (quadriplegia), tetraparesis (quadriparesis), or severe ataxia. In addition, these motor impairments may be combined with severe motor-related speech impairment (dysarthria or anarthria), as in LIS.\n* 22-70 years\n* Meeting surgical safety criteria, including surgical clearance by the participant's primary healthcare provider, study physicians, and any necessary consultants\n* Ability to communicate reliably, such as through eye movement\n* Willingness and ability to provide informed consent\n* Screened by rehabilitation psychologist with a result showing that the participant has a stable psychosocial support system with caregiver capable of monitoring participant throughout the study\n* Ability and willingness to travel up to 100 miles to study location up to three days per week for the duration of the study\n* Ability to understand and comply with study session instructions\n* Participant consents to the study and still wishes to participate at the time of the study\n\nExclusion Criteria:\n\n* Performance on formal neuropsychological testing that indicates significant psychiatric conditions or cognitive impairments that would interfere with obtaining informed consent or fully participating in study activities.\n* Suicide attempt or persistent suicidal ideation within the past 12 months.\n* Implanted devices that are incompatible with MRI, which may include pacemakers, cardiac defibrillators, spinal cord or vagal nerve stimulators, deep brain stimulators, and cochlear implants.\n* History of substance abuse, narcotic dependence, or alcohol dependence in past 24 months\n* Medical conditions contraindicating surgery of a chronically implanted device (e.g. osteomyelitis, diabetes, hepatitis, any autoimmune disease/disorder, epilepsy, skin disorders causing excessive skin sloughing or poor wound healing, blood or cardiac disorder requiring chronic anti-coagulation)\n* Other chronic, unstable medical conditions that could interfere with subject participation.\n* Presence of pre-surgical findings in anatomical, functional, and/or vascular neuroimaging that makes achieving implant locations within desired risk levels too challenging (to be decided by neurological and neurosurgical team)\n* Prior cranioplasty\n* Inability to undergo MRI or anticipated need for an MRI during the study period\n* Participants with active infections or unexplained fever\n* Participants with other morbid conditions making the implantation of the recording elements unsafe; not limited to: significant pulmonary, cardiovascular, metabolic, or renal impairments making the surgical procedure unsafe\n* Pregnancy (confirmation through blood test)\n* Nursing an infant, planning to become pregnant, or not using adequate birth control\n* Corrected vision poorer than 20/100\n* HIV or AIDS infection\n* Existing scalp lesions or skin breakdown\n* Chronic oral or intravenous use of steroids or immunosuppressive therapy\n* Active cancer within the past year or requires chemotherapy\n* Uncontrolled autonomic dysreflexia within the past 3 months\n* Hydrocephalus with or without an implanted ventricular shunt\n* Participants in whom it is medically contraindicated to stop anti-coagulant medications during surgery", "sex": "ALL", "min_age": "22 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CortiCom system (platinum PMT subdural cortical electrode grids)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03070119", "title": "Long-Term Evaluation of BIIB067 (Tofersen)", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogen", "summary": "The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 (tofersen) in participants with amyotrophic lateral sclerosis (ALS) and confirmed superoxide dismutase 1 (SOD1) mutation. The secondary objectives are to evaluate the pharmacokinetic (PK), pharmacodynamic (PD), biomarker effects, and efficacy of BIIB067 administered to participants with ALS and a confirmed SOD1 mutation.", "interventions": [{"type": "DRUG", "name": "Tofersen"}], "start_date": "2017-03-08", "url": "https://clinicaltrials.gov/study/NCT03070119", "target_entities": ["SOD1"], "locations": [{"facility": "Research Site", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Research Site", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Research Site", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Research Site", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Research Site", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Research Site", "city": "Orlando", "state": "Florida", "country": "United States", "status": "", "lat": 28.53834, "lon": -81.37924}, {"facility": "Research Site", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Research Site", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Research Site", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Research Site", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Research Site", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Research Site", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Research Site", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Research Site", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Research Site", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Research Site", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}, {"facility": "Research Site", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Research Site", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Research Site", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Research Site", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Research Site", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Bispebjerg Hospital", "city": "Copenhagen", "state": "", "country": "Denmark", "status": "", "lat": 55.67594, "lon": 12.56553}, {"facility": "Research Site", "city": "Clermont-Ferrand", "state": "Puy De Dome", "country": "France", "status": "", "lat": 45.77969, "lon": 3.08682}, {"facility": "Research Site", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Research Site", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Research Site", "city": "Bunky\u014d City", "state": "", "country": "Japan", "status": "", "lat": 35.5331, "lon": 139.4217}, {"facility": "Research Site", "city": "Kagoshima", "state": "", "country": "Japan", "status": "", "lat": 31.56667, "lon": 130.55}, {"facility": "Research Site", "city": "Shinjuku-ku", "state": "", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Research Site", "city": "Suita-shi", "state": "", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Research Site", "city": "Sheffield", "state": "South Yorkshire", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Must have diagnosis of superoxide dismutase 1-amyotrophic lateral sclerosis (SOD1-ALS), and must have completed the End of Study Visit for either Parts A, B, or C of Study 233AS101 (NCT02623699) (i.e., were not withdrawn).\n* If taking riluzole, participant must be receiving a stable dose for \u226530 days prior to Day 1.\n* If taking edaravone, participant must have initiated edaravone \u226560 days (2 treatment cycles) prior to Day 1. Edaravone may not be administered on dosing days during this study.\n* Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.\n* For female participants of childbearing potential must agree to practice effective contraception during the study and be willing and able to continue contraception for 5 months after their last dose of study treatment.\n* Participants from Study 233AS101 Parts A and B must have a washout \u226516 weeks between the last dose of study treatment received in Study 233AS101 and the first dose of BIIB067 received in the current Study 233AS102.\n\nKey Exclusion Criteria:\n\n* History of allergies to a broad range of anesthetics.\n* Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for bleeding during or after a Lumbar Puncture (LP) procedure. These risks could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).\n* Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter.\n* Prior or current treatment with small interfering ribonucleic acid (RNA), stem cell therapy, or gene therapy.\n* Treatment with another investigational drug, biological agent (excluding BIIB067), or device within 1 month or 5 half-lives of study agent, whichever is longer.\n* Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.\n* Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis(N4-methylthiosemicarbazone)) or pyrimethamine.\n* Female participants who are pregnant or currently breastfeeding.\n* Current enrollment in any other interventional study.\n\nNOTE: Other protocol defined Inclusion/Exclusion criteria may apply", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tofersen (BIIB067)", "targeting_mechanism": "Antisense oligonucleotide that reduces mutant SOD1 expression to slow motor neuron degeneration.", "targeting_mechanism_pmid": "37975798", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04882904", "title": "Continuous Measurement of Activity in Patients With Muscle Pathology and in Control Subjects. ActiSLA Part.", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Centre Hospitalier Universitaire de Liege", "summary": "ActiSLA is a monocentric academic study. Patients with amyotrophic lateral sclerosis may be included on a voluntary basis. The investigators plan to include a group of approximately 20 patients with ALS.\n\nThe investigators have planned to assess patient every three months for a year. On each visit, participants will undergo a clinical examination with MRC sum score and Ashworth scores.\n\nThey will perform few tests ( 6-minutes walk test (6MWT), dynamometric measure, electromyography, Edinburgh Cognitive and Behavioural ALS Screen ) and will answer to some questionaires (dysphagia handicap scale, ALS-SFR-r).\n\nAfter each visit, participants will wear Actimyo for one month daily.", "interventions": [{"type": "DEVICE", "name": "Actimyo\u00b0"}], "start_date": "2020-09-25", "url": "https://clinicaltrials.gov/study/NCT04882904", "target_entities": [], "locations": [{"facility": "CHR Citadelle", "city": "Li\u00e8ge", "state": "Li\u00e8ge", "country": "Belgium", "status": "RECRUITING", "lat": 50.63373, "lon": 5.56749}], "contact_phone": "43218222", "contact_email": "Laurie.Medard@citadelle.be", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinically defined or probable diagnosis of ALS according to El Escorial criteria revised with Awaji's electro-diagnostic algorithm.\n* Over 18 years old.\n* Signed informed consent\n* If patient on Riluzole, the dosage should be stable for 1 month and continued throughout the study period.\n\nExclusion Criteria:\n\n* Patients with excessive cognitive disorders, limiting the understanding of task or with apparent communication difficulties hindering data collection.\n* Any other previous or present pathology having an impact on motor function.\n* Recent surgery or trauma (less than 6 months) in the upper or lower limbs.\n* Prior neurological, endocrine, infectious, allergic, or chronic or acute inflammatory pathology in the three weeks preceding inclusion.\n* Patients participating in an interventional clinical trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00931944", "title": "Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "This is an open-label, multi-center study designed to extend the evaluation of the safety, tolerability, and clinical effects of oral administration of KNS-760704 in patients with ALS.", "interventions": [{"type": "DRUG", "name": "KNS-760704"}], "start_date": "2009-07", "url": "https://clinicaltrials.gov/study/NCT00931944", "target_entities": ["potassium_channels"], "locations": [{"facility": "University of Arkansas for Medical Sciences", "city": "Little Rock", "state": "Arkansas", "country": "United States", "status": "", "lat": 34.74648, "lon": -92.28959}, {"facility": "UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "The Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Miami Miller School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusettes General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Bryan LGH Medical Center East", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Columbia University, Lou Gehrig MDA/ALS Research Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University College Of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburgh School of Medicine", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Vanderbilt University Medical Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "University of Texas Health Sciences Center of San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patient has provided signed informed consent for this trial before the commencement of any study-related procedure\n2. Patient is actively participating in Knopp Protocol KNS-760704-CL201 and has completed the Part 2 Week 28 visit in that study or patient is actively receiving RTPB under Research IND #60,948\n\nExclusion Criteria:\n\n1. Patient did not participate in Knopp Protocol KNS-760704-CL201 or patient did not receive RTPB under Research IND #60,948.\n2. Patient discontinued from KNS-760704-CL201 for any reason other than enrollment into this study (applies to patients enrolled in KNS-760704-CL201 only)\n3. Patient was not taking RTPB under Research IND #60,948 prior to April 30, 2009 (applies to patients enrolled under Research IND #60,948 only)", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "KNS-760704", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02492516", "title": "Intravenous Injection of Adipose Derived Mesenchymal Stem Cell for ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Royan Institute", "summary": "Amyotrophic lateral sclerosis (ALS) is a lethal degenerative disorder that upper motor and lower motor neurons are destroyed in brain stem and spinal cord. Riluzole is the only therapeutic option now. Recently several studies have shown that stem cell transplantation is safe and can be effective in reduction of disease progression and increase of quality of life.", "interventions": [{"type": "BIOLOGICAL", "name": "mesenchymal stem cells"}], "start_date": "2014-09", "url": "https://clinicaltrials.gov/study/NCT02492516", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Royan Institute", "city": "Tehran", "state": "", "country": "Iran", "status": "", "lat": 35.69439, "lon": 51.42151}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Both gender\n* Age: 18-55\n* Sporadic form of disease\n* ALS-FRS\\> = 24\n* FVC \\>= 40%\n\nExclusion Criteria:\n\n* Familial form of ALS\n* Malignancy\n* Autoimmune disease\n* Diagnosis of other motor neuron diseases", "sex": "ALL", "min_age": "18 Years", "max_age": "55 Years", "healthy_volunteers": false, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "mesenchymal stem cells", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02917681", "title": "Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Sao Paulo General Hospital", "summary": "The study aims to evaluate primarily safety of two injections of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis patients. Secondary outcomes of efficacy will also be evaluated", "interventions": [{"type": "OTHER", "name": "Two intrathecal MSC injections"}], "start_date": "2016-09", "url": "https://clinicaltrials.gov/study/NCT02917681", "target_entities": ["neuroinflammation"], "locations": [{"facility": "University of Sao Paulo School of Medicine Clinics Hospital", "city": "S\u00e3o Paulo", "state": "S\u00e3o Paulo", "country": "Brazil", "status": "RECRUITING", "lat": -23.5475, "lon": -46.63611}], "contact_phone": "551130617460", "contact_email": "contato@projetoelabrasil.com.br", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age between 18 and 70\n2. Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000)\n3. ALSFRS-r \u226530 at enrollment\n4. Forced Vital Capacity \u226565% of the height and weight standard\n5. No-pregnancy agreement\n6. Regional accessibility to the study site\n7. Capability to give away agreed consensus\n8. Patients will be followed at Academic Institutions at their hometown\n\nExclusion Criteria:\n\n1. Previous cellular therapy\n2. Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections\n3. Personal history of auto-Immune, myeloproliferative or myelodysplastic diseases, leukemia, lymphoma, whole-body irradiation, hip fracture, severe scoliosis or incapacity to undergo any of the study's proposed procedures\n4. Any other disease that may interfere with the study\n5. Any other neurological diseases\n6. Aspartate or alanine aminotransferases elevated \\>3x normality upper limit\n7. Serum creatinine \\>2x normality upper limit\n8. Hepatitis B and C, HIV, HTLV I and II and syphilis\n9. Immunosuppressant drug use within 6 weeks from the study's screening\n10. Pregnancy or breast-feeding\n11. Acquired or inherited Immunodeficiency\n12. Participation in other clinical trials\n13. Non-invasive ventilation, tracheostomy or diaphragm pacing\n14. Substance abuse within one year and other unstable mental health diseases according to researcher's judgement\n15. Gastrostomy or any alternative feeding means\n16. Inappropriate in-vitro MSC expansion", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "autologous mesenchymal stem cells", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "Bone marrow-derived mesenchymal stem cells have shown potential in several ALS rodent models with varying delivery methods, cell amounts, timing of intervention, and differentiation states.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03645031", "title": "Acute Intermittent Hypoxia and Breathing in Neuromuscular Disease", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Florida", "summary": "This project seeks to investigate the effects of a single acute intermittent hypoxia (AIH) session on respiratory and non-respiratory motor function and EMG (electromyography) activity on patients with ALS (amyotrophic lateral sclerosis) and healthy controls.", "interventions": [{"type": "OTHER", "name": "Acute Intermittent Hypoxia"}, {"type": "OTHER", "name": "Sham Acute Intermittent Hypoxia"}], "start_date": "2018-10-01", "url": "https://clinicaltrials.gov/study/NCT03645031", "target_entities": ["hypoxia_signaling"], "locations": [{"facility": "UF Clinical Research Center", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* a healthy adult\n* clinical diagnosis of ALS\n* baseline FVC \\>60% predicted for age, sex and height.\n\nExclusion Criteria:\n\n* pregnant\n* diagnosed cardiovascular disease\n* a BMI \\>35 kg/m2\n* currently take selective serotonin reuptake inhibitors (SSRI)\n* history of seizures\n* history of hospitalization for sepsis\n* respiratory infection or took antibiotic medications within the past 4 weeks\n* use external respiratory support during any waking hours\n* participate in a pharmaceutical trial to treat ALS\n* have any other medical condition the PI or medical director identify would make it unsuitable to participate.", "sex": "ALL", "min_age": "21 Years", "max_age": "75 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Acute Intermittent Hypoxia", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07100119", "title": "A Study of LY4256984 in Participants With Sporadic Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Eli Lilly and Company", "summary": "The purpose of this study is to evaluate how well LY4256984 is tolerated and what side effects may occur in participants with sporadic amyotrophic lateral sclerosis (ALS). The study drug will be administered intrathecally (IT) into the spine. Blood tests will be performed to check how much LY4256984 gets into the bloodstream and how long it takes the body to eliminate it.", "interventions": [{"type": "DRUG", "name": "LY4256984"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2025-08-05", "url": "https://clinicaltrials.gov/study/NCT07100119", "target_entities": ["C9orf72"], "locations": [{"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "Heritage Medical Research Clinic", "city": "Calgary", "state": "", "country": "Canada", "status": "NOT_YET_RECRUITING", "lat": 51.05011, "lon": -114.08529}, {"facility": "Walter Mackenzie Health Sciences Centre", "city": "Edmonton", "state": "", "country": "Canada", "status": "NOT_YET_RECRUITING", "lat": 53.55014, "lon": -113.46871}, {"facility": "London Health Sciences Centre", "city": "London", "state": "", "country": "Canada", "status": "NOT_YET_RECRUITING", "lat": 42.98339, "lon": -81.23304}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}, {"facility": "Sunnybrook Research Institute", "city": "Toronto", "state": "", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}, {"facility": "Universit\u00e4tsklinikum Schleswig-Holstein", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 53.86893, "lon": 10.68729}, {"facility": "Universit\u00e4tsmedizin Rostock Sektion f\u00fcr Translationale Neurodegeneration \"Albrecht Kossel\" Klinik und Poliklinik f\u00fcr Neurologie", "city": "Rostock", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 54.0887, "lon": 12.14049}, {"facility": "Universit\u00e4tsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Universitario de Bellvitge", "city": "L'Hospitalet de Llobregat", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 41.35967, "lon": 2.10028}, {"facility": "Hospital Universitari i Politecnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 39.47391, "lon": -0.37966}], "contact_phone": "1-317-615-4559", "contact_email": "LillyTrials@Lilly.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Have a definite, possible, or probable diagnosis of sporadic amyotrophic lateral sclerosis (ALS) made by a physician experienced with the management of ALS\n* ALS symptom onset as determined by the Investigator within 24 months of Screening\n* Have a body mass index (BMI) within the range of greater than or equal to 18.0 and less than or equal to 35.0 kilogram per square meter (kg/m\u00b2) (inclusive)\n\nExclusion Criteria:\n\n* Have a history or presence of medical illness including, but not limited to, any cardiovascular, renal, hepatic, gastrointestinal, respiratory, hematological, endocrine, psychiatric, or neurological disease, convulsions, or any clinically significant laboratory abnormality\n* Have a history of another neurodegenerative disease or significant dementia/severe cognitive problems\n* Have serum alanine aminotransferase (ALT), aspartate transferase (AST), or total bilirubin levels greater than 2 x upper limit of normal.\n* Have a significant renal impairment (estimated glomerular filtration rate \\<60 milliliters per minute \\[mL/min\\]/1.73 m\u00b2).\n* Have a 12-lead electrocardiogram (ECG) abnormality at screening, in the opinion of the investigator, that increases the risks associated with participating in the study\n* Show clinically significant abnormalities in lumbar spine previously known or determined by screening lumbar X-ray or fluoroscopy (if performed)", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "LY4256984", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00868166", "title": "Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients Suffering From ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hoffmann-La Roche", "summary": "The purpose of the assay is to assess the safety and the efficacy of TRO19622 330 mg QD as add-on therapy to riluzole 50 mg bid in the treatment of patients suffering from ALS, as compared to placebo, assessed by the 18-month survival rate.", "interventions": [{"type": "DRUG", "name": "Olesoxime"}, {"type": "DRUG", "name": "Placebo Comparator"}, {"type": "DRUG", "name": "Riluzole"}], "start_date": "2009-04-30", "url": "https://clinicaltrials.gov/study/NCT00868166", "target_entities": ["mitochondrial_dysfunction"], "locations": [{"facility": "University Hospital Gasthuisberg - Dept Neurology - Herestraat 49", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "HCL H\u00f4pital Neurologique et Neurochirurgical Pierre Wertheimer - Neurologie C et Laboratoire d'\u00e9lectromyographie - 59, boulevard Pinel", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "CHRU de LILLE - H\u00f4pital Roger Salengro - Centre SLA-MMN - Sce de Neurologie et Pathologie du Mouvement", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Centre SLA Limoges - Service de Neurologie", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "H\u00f4pital La Timone - Service Neurologie et Maladies Neuromusculaires", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Clinique du Motoneurone - Sce d'Explorations Neurologiques - H\u00f4pital Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nice - H\u00f4pital de l'Archet 1 - Centre de R\u00e9f\u00e9rence pour les Maladies Neuromusculaires et la SLA", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Groupe Hospitalier PITIE-SALPETRIERE - F\u00e9d\u00e9ration des Maladies du Syst\u00e8me Nerveux", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Charit\u00e9 Universit\u00e4tsmedizin Berlin, Campus Virchow-Klinikum, Neurologische Poliklinik Ambulanz f\u00fcr ALS und andere Motoeneuronenerkrankungen", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Universit\u00e4tsklinik und Poliklinik f\u00fcr Neurologie - Martin-Luther-Universit\u00e4t Halle-Wittenberg", "city": "Halle", "state": "", "country": "Germany", "status": "", "lat": 51.48158, "lon": 11.97947}, {"facility": "Neurologische Klinik Medizinische Hochschule", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4ts- und Rehabilitationskliniken Ulm (RKU) - Neurologische Universit\u00e4tsklinik", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Hospital Carlos III - Unidad de ELA - Sinesio Delgado, 10", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "King's MND Care and Research Center - Academic Neurosciences Building PO Box 41 Institute of Psychiatry", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Academic Neurology Unit - University of Sheffield - Section of Neuroscience - Division of Genomic Medicine - School of Medicine and Biomedical Sciences", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with sporadic or familial Amyotrophic Lateral Sclerosis\n* Patients with a clinical diagnosis of laboratory-supported probable, probable, or definite ALS according to the modified El Escorial criteria8.\n* Have signed an Informed Consent to participate to the trial before any study related procedure has taken place.\n* Be of age \\>18 (exclusive) and \\< 80 years (inclusive).\n* If a female, not lactating, has a negative pregnancy test and agrees to use an effective method of birth control.\n* Onset of ALS Symptoms (weakness) for more than 6 months (inclusive) and less than 36 months(inclusive).\n* Slow vital capacity (SVC), measured three times, one of the measure being \\>/= 70% of that predicted.\n* Treated with riluzole at the stable dose of 50 mg bid for at least 30 days before enrolment.\n\nExclusion Criteria:\n\n* Tracheostomy, invasive ventilation, or non invasive positive pressure ventilation (NIPPV).\n* Gastrostomy.\n* Evidence of major psychiatric disorder or clinically evident dementia.\n* Diagnosis of a neurodegenerative disease in addition to ALS.\n* Have a current medication that could interfere with TRO19622 pharmacokinetics: tamoxifene.\n* Have current medications that could interfere with TRO19622 absorption such as ezetimibe, bile salts chelators (cholesteramine), fibrates, phytosterols, niacin (vitamin B3),fish oils. Have a current medication of lipid lowering agents other than statins.\n* Known hypersensitivity to any component of the study drug.\n* Patients with known intolerance or contra-indication to riluzole.\n* Have a recent history (within the previous 6 months) or current evidence of alcohol or drug abuse.\n* Have concurrent unstable disease involving any system eg, carcinoma other than basal cell carcinoma, any cardiac dysrhythmia, myocardial infarction, clinical or ECG signs of myocardial ischemia, cardiac insufficiency, angina symptoms, current symptoms of Coronary Artery Disease, or any other condition that in the opinion of the Investigator would make the patient unsuitable for study participation.\n\n  . In Germany: Have any cardiac dysrhythmia, myocardial infarction, clinical or ECG signs of myocardial ischemia, cardiac insufficiency, angina symptoms, current symptoms of Coronary Artery Disease or any cardiovascular illness known or identified at the screening or inclusion visits, or have concurrent unstable disease involving any system eg, carcinoma other than basal cell carcinoma or any other condition that in the opinion of the Investigator would make the patient unsuitable for study participation.\n* Having a baseline QTc (Bazett) \\> 450 msec for males and \\> 470 msec for females.\n* Patients with known hepatitis B/C or HIV positive serology.\n* Be pregnant female or lactating.\n* Have renal impairment defined as blood creatinine \\> 1:5 X upper limit of normal.\n* Have hepatic impairment and/or liver enzymes (ALAT or ASAT) \\> 3 X ULN.\n* Hemostasis disorders or current treatment with oral anticoagulants.\n* Be possibly dependent on the Investigator or the Sponsor (eg, including, but not limited to, affiliated employee).\n* Participated in any other investigational drug or therapy study with a non approved medication, within the previous 3 months.\n* Patients without Social Security Insurance (France).", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Olesoxime", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05744310", "title": "Effects of Long Term Ventilation Support on the Quality of Life of ALS Patients and Their Families", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Haukeland University Hospital", "summary": "Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is two to three years. The patient physical and psychological sufferings in ALS are immense, and apart from Riluzole, there is no effective treatment. Care of advanced ALS have an estimated cost of 4-8 million NOK per year. Perhaps the most challenging topic of ALS care is the decision to extend ventilation support into the stages of disease that require treatment both during day and night. In these cases, treatment is clearly life-sustaining and although quality of life may be maintained, the burden of caregiving imposed upon family or health care workers is huge, regardless of tracheostomy (TIV) or non-invasive (NIV) modality.\n\nThe present study is a longitudinal questionnaire study in Norway measuring overall quality of life, health-related quality of life, and disease-specific quality of life in ALS patients, partners and children before and after the introduction of life sustaining ventilation support. The investigators aim to increase the knowledge on how life-sustaining ventilation support with NIV or TIV affects the quality of life in ALS patients, life partners and children. The results from the study may provide crucial information for clinicians and patients on one of the most difficult ethical issues of ALS treatment. The investigators anticipate that this information will facilitate a shared decision making processes, weighing benefits and disadvantages in a wider perspective.", "interventions": [{"type": "DEVICE", "name": "Long term mechanical ventilation support"}, {"type": "DEVICE", "name": "No long term mechanical ventilation support"}], "start_date": "2023-04-21", "url": "https://clinicaltrials.gov/study/NCT05744310", "target_entities": [], "locations": [{"facility": "Nordland Hospital Bod\u00f8", "city": "Bod\u00f8", "state": "Nordland", "country": "Norway", "status": "RECRUITING", "lat": 67.28267, "lon": 14.37513}, {"facility": "S\u00f8rlandet Hospital Trust", "city": "Kristiansand", "state": "Vest Agder", "country": "Norway", "status": "RECRUITING", "lat": 58.14671, "lon": 7.9956}, {"facility": "Haukeland University Hospital", "city": "Bergen", "state": "", "country": "Norway", "status": "RECRUITING", "lat": 60.39299, "lon": 5.32415}, {"facility": "Akershus University Hospital", "city": "L\u00f8renskog", "state": "", "country": "Norway", "status": "RECRUITING", "lat": null, "lon": null}, {"facility": "Oslo University Hospital", "city": "Oslo", "state": "", "country": "Norway", "status": "RECRUITING", "lat": 59.91273, "lon": 10.74609}, {"facility": "\u00d8stfold Hospital Kalnes", "city": "Sarpsborg", "state": "", "country": "Norway", "status": "NOT_YET_RECRUITING", "lat": 59.28391, "lon": 11.10962}, {"facility": "Stavanger University Hospital", "city": "Stavanger", "state": "", "country": "Norway", "status": "RECRUITING", "lat": 58.97005, "lon": 5.73332}, {"facility": "Universitetssykehuset Nord-Norge", "city": "Troms\u00f8", "state": "", "country": "Norway", "status": "RECRUITING", "lat": 69.6489, "lon": 18.95508}, {"facility": "St. Olavs Hospital", "city": "Trondheim", "state": "", "country": "Norway", "status": "RECRUITING", "lat": 63.43049, "lon": 10.39506}], "contact_phone": "+ 47 55975063", "contact_email": "ole-bjorn.tysnes@helse-bergen.no", "eligibility": {"criteria": "Inclusion criteria for patients:\n\n1. A clinical diagnosis of probable ALS according to the revised El Escorial criteria\n2. Progression of the illness leading the consulting physician to offer treatment with LTMV\n3. Can communicate in Norwegian\n\nInclusion criteria for partners of ALS patients:\n\n1. Partner of a patient with ALS with progression of the illness leading the consulting physician to offer treatment with LTMV\n2. Can communicate in Norwegian\n\nInclusion criteria for children:\n\n1. Children from 8 years and older having a parent who suffers from ALS with progression of the illness leading the consulting physician to offer treatment with LTMV\n2. Can communicate in Norwegian\n\nExclusion criteria for patients, partners and children of ALS patients:\n\n1\\. Potential participants with cognitive impairment or dementia.", "sex": "ALL", "min_age": "8 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02710162", "title": "Accurate Screening Tools for Dysphagia in Amyotrophic Lateral Sclerosis (ALS)", "phase": "EARLY_PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Florida", "summary": "Individuals with th Amyotrophic Lateral Sclerosis are at high risk for swallowing impairment (dysphagia) which leads to malnutrition, decreased pulmonary health, aspiration and aspiration pneumonia. These sequelae necessitate timely identification of at risk individuals to ensure optimal management of oral intake and pulmonary function. The purpose of this study is to evaluate the discriminant ability of several non-invasive screening tools at detecting swallowing impairment in individuals with ALS.", "interventions": [{"type": "DEVICE", "name": "Micro Mouth Pressure Meter"}, {"type": "DEVICE", "name": "Iowa Oral Performance Instrument"}, {"type": "DEVICE", "name": "Electrical Impedance Myography"}, {"type": "DRUG", "name": "Capsaicin"}, {"type": "PROCEDURE", "name": "Videofluoroscopic Swallowing Study"}, {"type": "PROCEDURE", "name": "Pulmonary Function Testing"}, {"type": "OTHER", "name": "Swallowing Related Quality of Life Questionnaire"}, {"type": "OTHER", "name": "Functional Oral Intake Scale"}, {"type": "OTHER", "name": "Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised"}, {"type": "OTHER", "name": "Eating Assessment Tool-10"}, {"type": "OTHER", "name": "Communicative Effectiveness Survey"}, {"type": "OTHER", "name": "The Center for Neurologic Studies Bulbar Function Scale"}], "start_date": "2016-04", "url": "https://clinicaltrials.gov/study/NCT02710162", "target_entities": [], "locations": [{"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of probable or definite ALS\n\nExclusion Criteria:\n\n* allergies to barium or capsaicin\n* tracheotomy or mechanical ventilation\n* absence of diaphragmatic pacer\n* respiratory disease (COPD).", "sex": "ALL", "min_age": "21 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01285583", "title": "Safety Extension Study of TRO19622 in ALS", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hoffmann-La Roche", "summary": "The purpose of the assay is to assess the safety of TRO19622 330 mg QD as add-on therapy to riluzole 50 mg bid in the treatment of patients suffering from ALS, after completion of the preceding clinical trial (TRO19622 CL E Q 1015-1) in an open label extension.", "interventions": [{"type": "DRUG", "name": "TRO19622"}], "start_date": "2010-10", "url": "https://clinicaltrials.gov/study/NCT01285583", "target_entities": ["mitochondrial_dysfunction"], "locations": [{"facility": "University Hospital Gasthuisberg - Dept Neurology - Herestraat 49", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "HCL H\u00f4pital Neurologique et Neurochirurgical Pierre Wertheimer - Neurologie C et Laboratoire d'\u00e9lectromyographie - 59, boulevard Pinel", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "CHRU de LILLE - H\u00f4pital Roger Salengro - Centre SLA-MMN - Sce de Neurologie et Pathologie du Mouvement", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Centre SLA Limoges - Service de Neurologie", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "H\u00f4pital La Timone - Service Neurologie et Maladies Neuromusculaires", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Clinique du Motoneurone - Sce d'Explorations Neurologiques - H\u00f4pital Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nice - H\u00f4pital de l'Archet 1 - Centre de R\u00e9f\u00e9rence pour les Maladies Neuromusculaires et la SLA", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Groupe Hospitalier PITIE-SALPETRIERE - F\u00e9d\u00e9ration des Maladies du Syst\u00e8me Nerveux", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Charit\u00e9 Universit\u00e4tsmedizin Berlin, Campus Virchow-Klinikum, Neurologische Poliklinik Ambulanz f\u00fcr ALS und andere Motoeneuronenerkrankungen", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Universit\u00e4tsklinik und Poliklinik f\u00fcr Neurologie - Martin-Luther-Universit\u00e4t Halle-Wittenberg", "city": "Halle", "state": "", "country": "Germany", "status": "", "lat": 51.48158, "lon": 11.97947}, {"facility": "Neurologische Klinik Medizinische Hochschule", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4ts- und Rehabilitationskliniken Ulm (RKU) - Neurologische Universit\u00e4tsklinik", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Hospital Carlos III - Unidad de ELA - Sinesio Delgado, 10", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "King's MND Care and Research Center - Academic Neurosciences Building PO Box 41 Institute of Psychiatry", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Academic Neurology Unit - University of Sheffield - Section of Neuroscience - Division of Genomic Medicine - School of Medicine and Biomedical Sciences", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients must have completed the 18-month safety and efficacy study of TRO19622 in Amyotrophic Lateral Sclerosis (ALS) patients treated with riluzole (Protocol TRO19622 CL E Q 1015-1).\n* Both the investigator and the patient will decide based on previous good tolerance and other clinical grounds whether or not to participate to the open-label extension.\n* If patients were on anti-vitamin K during the double-blind period, when entering the open-label extension, coagulation tests should be monitored in exactly the same conditions as if a new anticoagulant treatment was initiated and the dose of anti-vitamin K should be adjusted accordingly.\n* Patients enrolling from this prior safety and efficacy study must:\n\n  * If female of childbearing age of potential, continue to use adequate birth control methods and have a negative serum pregnancy test at the preceding double-blind protocol termination visit. Male and female partners must agree to use an effective method of birth control during their participation in the trial and for at least 15 days after the last IMP dose. Both partners must use reliable methods of contraception with 2 independent methods. The following measures are acceptable: Hormone contraceptives (e.g. oral contraceptives or comparable methods), intrauterine device, condoms with spermicidal coating or in combination with spermicidal creams, total abstinence or sterilisation performed in the past.\n  * Be able to follow the investigator's instructions and be able to comply with the visit schedule and visit requirements; and\n  * Sign a written informed consent.\n\nExclusion Criteria:\n\nPatients may not participate in this study if they have an ongoing, unresolved, clinically significant medical problem (including patients having experienced serious adverse events or non-serious, but medically significant adverse events during the preceding safety and efficacy study that was assessed to be related to the study medication by the investigator) that in the judgment of the investigator would make it unsafe for the patient to participate in the trial.", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "TRO19622", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07625605", "title": "The Meals Study for Amyotrophic Lateral Sclerosis and Parkinson's Disease", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The University of Tennessee, Knoxville", "summary": "Brief summary template\n\nThe goal of this trial is to test the feasibility and efficacy of the Mediterranean-DASH Intervention for Neurodegenerative Delay (the MIND diet) in people living with amyotrophic lateral sclerosis (ALS) and Parkinson's disease (PD) and healthy controls. The main question\\[s\\] it aims to answer are:\n\n* How feasibile is our educational dietary intervention, which teaches participants with ALS, PD, and healthy controls to eat by the neuroprotective MIND dietary pattern?\n* How does the MIND dietary pattern affect the gut microbiome, metabolome, and lipidome in people with a neurodegenerative disease (ALS or PD) and healthy controls?\n* Does the MIND diet affect clinical measures of ALS and PD?\n* Does the MIND diet affect human biomarkers of systemic inflammation, metabolism, and neurodegeneration?\n\nParticipants will:\n\n* Receive a MIND diet cookbook, a folder with educational handouts, and weekly emails with links to educational videos about the MIND diet.\n* Complete a food diary, a MIND diet tracker, and a weekly questionnaire about their experience of eating by the MIND diet.\n* Collect stool specimens at the beginning, middle, and end of the study.\n* Undergo venipuncture at the beginning and end of the study.", "interventions": [{"type": "BEHAVIORAL", "name": "The MIND diet"}], "start_date": "2023-11-20", "url": "https://clinicaltrials.gov/study/NCT07625605", "target_entities": [], "locations": [{"facility": "Gary E Shealy Memorial ALS Clinic", "city": "Elizabethton", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.34872, "lon": -82.21069}, {"facility": "University Neurology", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}, {"facility": "The Cole Center for Parkinson's & Movement Disorders", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria for Participants with ALS:\n\n* Meet the El Escorial Criteria for definite, probable, or possible ALS:\n\n  * Definite ALS is defined as presence of upper motor neuron (UMN) and lower motor neuron (LMN) signs in the bulbar region and at 2 of the other spinal regions\n  * Probable ALS is defined as presence of UMN and LMN in at least 2 regions with UMN signs rostral to LMN signs; probable ALS supported by laboratory results is defined as presence of UMN in 1 region with electromyographic evidence of LMN involvement in another region\n  * Possible ALS is defined as presence of UMN and LMN in 1 region or UMN in 2 or 3 regions (monomelic ALS, progressive bulbar palsy, and primary lateral sclerosis)\n* Voluntarily participate and able to consent\n* Have internet access\n* Willing to communicate by telephone or teleconferencing, and email\n* Minimum age of 30 years old\n* Able to masticate (chew) and swallow at the time of the study\n* Willing to return to the clinic eight weeks after enrolling for a regularly scheduled visit and post intervention data collection, or if they are not patients of the clinical sites, then they are willing to come to the clinic twice for study activities (once at enrollment and once upon exiting).\n* Patient (and their caregiver, if needed) must be willing and able (in the Investigator's opinion) to comply with all study requirements\n\nExclusion Criteria for Participants with ALS:\n\n* Patients that require a special diet (celiac/gluten free, vegetarian, or vegan)\n* Antibiotic use in past 6 months\n* History of bowel diseases or cancer (e.g., irritable bowel syndrome, celiac sprue, Crohn's\n* Younger than age 30\n* Unable to provide consent\n* Feeding by tube feed or primarily liquid diets\n* Unable to masticate or swallow.\n\nInclusion Criteria for Participants with PD:\n\n* Meet clinical criteria for PD per the MDS clinical criteria (Postuma et al, Movement Disorders,\n* 2015\\) as determined by the treating Movement Disorder specialist\n* Hoehn Yar \\< 2.5, as determined by the treating Movement Disorder Specialist.\n* Be 50 to 80 years of age upon enrollment.\n* Levodopa daily equivalents of no more than 500 a day\n* Subjects are within five years of the onset of symptoms at time of study enrollment.\n* Participants agree not to change PD medications during the 7-week dietary intervention.\n* Voluntarily participate and able to consent\n* Able to masticate (chew) and swallow at the time of the study\n* Have internet access\n* Willing to communicate by telephone or teleconferencing, and email\n\nExclusion Criteria for Participants with PD:\n\n* May not be taking medications concerning for a drug induced parkinsonism two years prior to\n* study entry such as antipsychotics, metoclopramide (Reglan), prochlorperazine (Compazine),\n* promethazine (Phenergan) or amiodarone\n* The movement disorder specialist must find no red flags for concern for atypical or Parkinson's Plus per the MDS diagnostic criteria (Postuma et al)\n* Patients that require a special diet (celiac/gluten free, vegetarian, or vegan)\n* Antibiotic use in past 6 months\n* History of bowel diseases or cancer (e.g., irritable bowel syndrome, celiac sprue, Crohn's Disease, colorectal cancer)\n* Unable to provide consent or struggling with major cognitive concerns as determined by the\n* treating Movement Disorder specialist\n* Feeding by tube feed or primarily liquid diets\n* Unable to masticate or swallow.\n\nInclusion Criteria for Healthy Controls:\n\n* Voluntarily participate and able to consent\n* Able to speak, read, and write English\n* Internet access,\n* Willing to communicate by telephone or teleconferencing, and email\n* Minimum age of 30 years\n* Able to masticate (chew) and swallow at the time of the study\n* Willing to visit the clinic two times to complete study activities\n* Patient (and their caregiver, if needed) must be willing and able (in the Investigator's opinion) to\n* comply with all study requirements\n\nExclusion Criteria for Healthy Controls:\n\n* Healthy people who require a special diet (celiac/gluten free, vegetarian, or vegan)\n* Antibiotic use in past 6 months\n* History of bowel diseases or cancer (e.g., irritable bowel syndrome, celiac sprue, Crohn's Disease, colorectal cancer)\n* Younger than age 30\n* Unable to consent\n* Feeding by tube feed or primarily liquid diets\n* Diagnosis with any neurodegenerative disease (e.g., Alzheimer's disease, Parkinson's disease, multiple sclerosis, peripheral neuropathies).", "sex": "ALL", "min_age": "30 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "The MIND diet promotes neuroprotection through dietary pattern modification.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02872142", "title": "Efficacy and Safety of Plasma Exchange With Albutein\u00ae 5% in Participants With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Grifols Therapeutics LLC", "summary": "This is a pilot, phase 2, prospective, open-label, single-arm study to evaluate disease progression, forced vital capacity, and the safety and tolerability of plasma exchange (PE) using Albutein\u00ae 5% in participants with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "BIOLOGICAL", "name": "Albutein 5%"}, {"type": "PROCEDURE", "name": "Plasma Exchange"}], "start_date": "2016-08-29", "url": "https://clinicaltrials.gov/study/NCT02872142", "target_entities": [], "locations": [{"facility": "Dartmouth-Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Signed informed consent\n* Subjects over 18 years of age and less than 70 years old\n* Subjects with a possible, probable-lab supported, probable, or definite diagnosis of Amyotrophic Lateral Sclerosis (ALS), according to the revised El Escorial criteria\n* Subjects having experienced their first ALS symptoms within 18 months prior to recruitment/consent\n* Forced Vital Capacity \\> 70%\n* Subjects must be medically suitable for study participation and willing to comply with all planned aspects of the protocol, including blood sampling, at the time of inclusion in the study.\n\nExclusion Criteria:\n\n* Subjects with pre-existing clinically significant lung disease not attributable to ALS\n* Subjects diagnosed with other neurodegenerative diseases or diseases associated with other motor neuron dysfunction\n* Participation in another investigational product study within one month prior to screening\n* Females who are pregnant, breastfeeding, or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device or intrauterine system, condom or occlusive cap with spermicidal foam/ gel/ film/ cream/ suppository, male sterilization, or true abstinence) throughout the study\n* Difficult or problematic peripheral vein access and inability to implant a central catheter which would make continuous Plasma exchange (PE) not feasible as per the visit protocol\n* Contraindication to undergo PE or subject has abnormal coagulation parameters at the discretion of the Outpatient Apheresis Unit team, including but not limited to:\n\n  1. Thrombocytopenia (platelets \\<100,000/ microliter \\[\u03bcL\\])\n  2. Fibrinogen \\<1.5 gram per liter (g/L)\n  3. International Normalized Ratio \\>1.5\n  4. Beta-blocker treatment and bradycardia \\<50 beats/min\n  5. Treatment with angiotensin-converting enzyme inhibitors which may increase the risk of allergic reactions, unless a preventive change in hypotensive treatment occurs prior to enrollment\n* History of anaphylaxis or severe systemic response to any plasma-derived albumin preparation, component of Albutein\u00ae 5%, or other blood product(s)\n* Subjects unable to interrupt treatment with acetylsalicylic acid, other oral antiplatelet, or anticoagulant\n* Renal dysfunction by elevated creatinine concentration \\>2 milligram per deciliter (mg/dL)\n* Presence of heart disease that contraindicates PE treatment, including ischemic cardiopathy and congestive heart failure\n* Presence of prior behavioural disorders requiring pharmacological intervention with less than 3 months of stable treatment\n* Mentally challenged subject who cannot give independent informed consent\n* Any condition that would complicate compliance with the study protocol (i.e., illness with the expectation of less than one year survival, abuse of drugs or alcohol, etc.)", "sex": "ALL", "min_age": "19 Years", "max_age": "69 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Albutein 5% (human albumin)", "targeting_mechanism": "Plasma exchange with albumin replacement aims to remove circulating pathogenic factors in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02987413", "title": "Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hospital e Maternidade Dr. Christ\u00f3v\u00e3o da Gama", "summary": "Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that selectively affects motor neurons in the brain and spinal cord, leading to bulbar, respiratory, and limb weakness. There is no effective treatment, and the disease usually progresses to death within 2 to 4 years. The therapeutic plasticity of mesenchymal stem cells (MSCs) may be an attractive therapy to this complex disease, turning MSCs strong candidates for cellular therapy in ALS.\n\nDesign-A phase 1 open-safety clinical trial. 4 patients will be selected according to a restricted inclusion and exclusion criteria and after 2 escalated infusions of MSCs, there will be a follow up period of one year Methods - Primary endpoint: safety of mesenchymal autologous stem cells infusions escalated in two intrathecal administrations in patients with ALS defined as severe adverse events (SAe). Secondary endpoints: clinical response, laboratorial and magnetic resonance imaging of patients submitted to cellular escalating doses applied in the study. Quality of life, according to El Escorial criteria, ALSFR scale and functional scales.\n\nConclusion: This study is a primary step before a large randomized double-blind clinical trial for ALS. It is expected to confirm the safety of escalated MSCs therapy in ALS patients, initial data of efficacy in addition to improved quality of life.", "interventions": [{"type": "BIOLOGICAL", "name": "Autologous Mesenchymal stem cells (MSCs)"}], "start_date": "2015-04-28", "url": "https://clinicaltrials.gov/study/NCT02987413", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Hospital e Maternidade Dr Christovao da Gama", "city": "Santo Andr\u00e9", "state": "S\u00e3o Paulo", "country": "Brazil", "status": "", "lat": -23.66389, "lon": -46.53833}, {"facility": "Instituto de Ensino e Pesquisas - IEP-S\u00e3o Lucas", "city": "S\u00e3o Paulo", "state": "S\u00e3o Paulo", "country": "Brazil", "status": "", "lat": -23.5475, "lon": -46.63611}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Women and males over 18-year-old.\n* Diagnosis of ALS in agreement with the criteria of \"EL SCORE\"\n* Less than 24 months of evolution of the disease (from the beginning of the symptoms).\n* Good understanding of the protocol and aptitude to grant the informed consent\n* Infertile women (post-menopause or hysterectomized)\n* Brazilian citizen and permanent resident.\n\nExclusion Criteria:\n\n* Any significant medical condition (congestive heart failure, angina, respiratory failure, and others)\n* Any auto-immune disease\n* Any malignant diseases\n* Systemic infection\n* Mental illness\n* Depressive state", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous Mesenchymal stem cells (MSCs)", "targeting_mechanism": "Mesenchymal stem cells differentiate into supportive glial cells and secrete growth factors and anti-inflammatory cytokines to support degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Bone marrow-derived MSCs demonstrated potential therapeutic benefit in mouse and rat ALS models.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07213440", "title": "Identification of Early Markers for ALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Institut National de la Sant\u00e9 Et de la Recherche M\u00e9dicale, France", "summary": "Although several molecules have been proposed as biomarker candidates, a clinically established signature for an early or even premotor diagnosis of ALS is not available. Due to the already advanced, disease stage at the time of diagnosis as well as rapid disease progression, an early diagnosis is mandatory for efficacious disease-modifying therapies.\n\nIn this project, the investigators will develop a clinical molecular fingerprint of PGMC that will provide insight into the molecular pathogenesis of ALS and allow earlier diagnosis.", "interventions": [{"type": "PROCEDURE", "name": "lumbar puncture"}], "start_date": "2024-09-30", "url": "https://clinicaltrials.gov/study/NCT07213440", "target_entities": [], "locations": [{"facility": "CHU Tours", "city": "Tours", "state": "France", "country": "France", "status": "RECRUITING", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "+33247473724", "contact_email": "philippe.corcia@univ-tours.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\nFIRST GROUP: Premotor gene mutation carriers (PGMC):\n\n* 18-90 years of age\n* Provision of a written informed consent\n* Affiliation with a social security scheme or beneficiary of such a scheme\n* Diagnosed by a clinically certified laboratory with a disease- causing mutation in a known ALS gene by predictive genetic testing\n* No symptoms of motor neuron disease explainable otherwise than by mutation in a known ALS gene\n\nSECOND GROUP: Control subjects to premotor gene mutation carriers (CTR):\n\n* 18-90 years of age\n* Provision of a written informed consent\n* Affiliation with a social security scheme or beneficiary of such a scheme\n* No known genetic mutation and no known ALS disease in close family\n* No diagnosed motor-neuron disease\n\nTHIRD GROUP: ALS (EALS) / ALS mimics (MIM)\n\n* 18-90 years of age\n* provision of a written informed consent\n* affiliation with a social security scheme or beneficiary of such a scheme\n* Patients with pure motor symptom or early ALS (EALS) or ALS mimics (MIM)\n\nEALS are patients with pure motor symptom / early motor symptoms of ALS, including those, where the diagnosis of ALS can already be made. These may be patients who meet the following criteria:\n\nAccording to El Escorial criteria : patients who can be classified as possible ALS or those who show upper motor neuron (UMN) signs only or lower motor neuron (LMN) signs only, so that classification as possible ALS is also not possible. Symptoms should not persist for more than 12 months.\n\nAccording to Gold Coast criteria: Patients who do not fulfill the criterion of temporal progression or patients who only show UMN signs or only LMN signs in one region and thus do not fulfill the diagnostic criteria of ALS.\n\nExclusion Criteria:\n\n* Inability to express consent to the study\n* Persons subject to a judicial safeguard measure, under guardianship or curatorship.\n* Linguistic incapacity or psychic refusal to read the information.\n* Pregnant women\n* Foreseen inability to attend scheduled visits\n* Persons refusing to take one of the following samples: Acquisition of blood samples, Acquisition of tear fluid samples, Acquisition of urine sample", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00372879", "title": "Clinical Trial of Vitamin E to Treat Muscular Cramps in Patients With ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's", "summary": "Muscular cramps are a common and uncomfortable symptom of amyotrophic lateral sclerosis (ALS). This clinical trial will compare the response of high dose vitamin E supplementation to placebo for treatment of muscular cramps in patients with ALS. We hypothesize that vitamin E will be more effective than placebo in treating cramps.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Vitamin E"}], "start_date": "2006-12", "url": "https://clinicaltrials.gov/study/NCT00372879", "target_entities": ["oxidative_stress"], "locations": [{"facility": "London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Adults (\\> age 18 years)\n* Probable or definite ALS by El Escorial Revised criteria\n* At least 2 painful muscle cramps in one or more of the limbs per week.\n* May have tried other medications for cramping in the past.\n* If participants are currently on treatment for cramps and are continuing to have at least 2 cramps per week, they can be included in the trial. In this situation, the individual's previous cramp medication can be continued during the trial.\n* Ideally, patients should not have any medication alterations during the duration of the trial.\n* Willing to discontinue supplementary vitamin E and multivitamins containing \\> 400 IU of vitamin E during the trial.\n\nExclusion Criteria:\n\n* Patients who are unable to safely consume the trial capsules. Individuals with significant dysphagia can be included into the study if they have a functioning PEG tube or GJ tube, through which the medication can be given.\n* Patients who are unable to fill out the daily diary, either personally or via a proxy.\n* Patients who have had medication changes within the last 4 weeks prior to the onset of the trial will be excluded.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Vitamin E", "targeting_mechanism": "Vitamin E functions as an antioxidant to reduce oxidative stress in ALS.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01378676", "title": "A Study to Evaluate the Effects of Multiple Doses of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "The study will generate data on safety, tolerability and pharmacokinetics after multiple daily doses of CK-2017357 in patients with ALS. Patients will be randomized into one of four different treatment groups, receiving daily oral doses of either placebo, 125 mg, 250 mg, or 375 mg of CK-2017357 for 14 days.", "interventions": [{"type": "DRUG", "name": "Placebo (Part A)"}, {"type": "DRUG", "name": "CK-2017357 (Part A)"}, {"type": "DRUG", "name": "CK-2017357 (Part A)"}, {"type": "DRUG", "name": "CK-2017357 (Part A)"}, {"type": "DRUG", "name": "Riluzole 50 MG (Part B)"}, {"type": "DRUG", "name": "Placebo (Part B)"}, {"type": "DRUG", "name": "CK-2017357 (Part B)"}, {"type": "DRUG", "name": "CK-2017357 (Part B)"}, {"type": "DRUG", "name": "CK-2017357 (Part B)"}], "start_date": "2011-06", "url": "https://clinicaltrials.gov/study/NCT01378676", "target_entities": ["muscle_contractility"], "locations": [{"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Kansas", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical Center", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Neuromuscular ALS-MND Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University College of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n1. Able to comprehend and willing to sign an Informed Consent Form (ICF)\n2. Males or females 18 years of age or older\n3. A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria)\n4. Maximum voluntary grip strength in at least one hand between 10 \\& 40 pounds (females) and 10 \\& 60 pounds (males)\n5. Upright Slow Vital Capacity (SVC) \\>50% of predicted for age, height, and sex\n6. Able to swallow tablets with water\n7. Willing and able to remain off riluzole for 4 weeks (Part A only)\n8. Currently taking and tolerating a stable dose of 50 mg BID riluzole (Part B only)\n9. Willing and able to reduce daily dose of riluzole to 50 mg for 4 weeks (Part B only)\n10. Willing and able to refrain from caffeine-containing products during study participation\n11. Willing and able to remain off warfarin and theophylline-containing medications during study participation\n12. Has a caregiver who is capable of observing and reporting patient status, and also assisting in the proper use of nocturnal oximetry equipment\n13. Able to perform pulmonary function tests\n\nKey Exclusion Criteria:\n\n1. Life expectancy \\<3 months\n2. Participation in any trial in which receipt of investigational study drug occurred within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing\n3. Any prior treatment with CK-2017357\n4. Use of non-invasive positive pressure ventilation (NIPPV) for any part of the day or night\n\nOther protocol-defined inclusion/exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CK-2017357", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06828523", "title": "Assessing Perceptual Effects of Interactive Tasks", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Penn State University", "summary": "In this study, we will evaluate how solo, naive listeners perceive the speech of people with amyotrophic lateral sclerosis (ALS) and age-matched speakers produced across interactive and non-interactive contexts with an unfamiliar, naive interlocutor.", "interventions": [{"type": "BEHAVIORAL", "name": "Listener judgements of speech produced in different tasks"}], "start_date": "2025-10-17", "url": "https://clinicaltrials.gov/study/NCT06828523", "target_entities": [], "locations": [{"facility": "Speech Core, Pennsylvania State University", "city": "University Park", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 40.80201, "lon": -77.85639}], "contact_phone": "814-867-3373", "contact_email": "ajo150@psu.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* passing the remote hearing screening\n* having no known speech, language or neurological disorders per self-report\n* being a native monolingual speaker of American English\n* having no experience communicating with people with dysarthria\n* being between the ages of 18 and 65.\n\nExclusion Criteria:\n\n* None - if volunteer meets the inclusion criteria, then they will be enrolled", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04615923", "title": "HEALEY ALS Platform Trial - Regimen D Pridopidine", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.\n\nRegimen D will evaluate the safety and efficacy of a single study drug, pridopidine, in participants with ALS.", "interventions": [{"type": "DRUG", "name": "Pridopidine"}, {"type": "DRUG", "name": "Matching Placebo"}], "start_date": "2020-12-18", "url": "https://clinicaltrials.gov/study/NCT04615923", "target_entities": ["dopamine_d2_receptor_signaling"], "locations": [{"facility": "Healey Center for ALS at Mass General", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\nExclusion Criteria:\n\n* The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\n  1. Participants with a confirmed prolonged Fridericia-corrected QT (QTcF) interval (defined as a QTcF interval of \\>450 ms for men and \\>470 ms for women).\n  2. Participants with clinically significant heart disease, clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, or asymptomatic sustained ventricular tachycardia, or presence of left bundle branch block.\n  3. Participants with known history of long QT syndrome or a first degree relative with this condition.\n  4. Participants using prohibited medications within the 4 weeks prior to the Regimen Specific Screening Visit, as detailed in section 5.9.\n  5. Participants using the following medications at the time of the Regimen Specific Screening Visit:\n\n     1. Nuedexta - at a dosage higher than 20 mg dextromethorphan + 10 mg quinidine BID\n     2. Citalopram - at a dosage higher than 20 mg/day\n     3. Escitalopram - at a dosage higher than 10 mg/day\n  6. Participants with a known allergy to any ingredient of the study intervention (pridopidine, silicified microcrystalline cellulose, and magnesium stearate).", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Pridopidine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05178810", "title": "Study to Investigate the Efficacy and Safety of FAB122 (Daily Oral Edaravone) in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ferrer Internacional S.A.", "summary": "Multicenter, multinational, double-blind, randomized (2:1), placebo-controlled Phase III study to investigate the efficacy and safety of 100 mg FAB122 once daily as oral formulation in ALS patients.", "interventions": [{"type": "DRUG", "name": "FAB122"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-10-18", "url": "https://clinicaltrials.gov/study/NCT05178810", "target_entities": ["oxidative_stress"], "locations": [{"facility": "University Hospitals Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "CHRU de Lille - H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges - H\u00f4pital Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Centre Hospitalo-Universitaire La Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHU de Montpellier", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU Nice", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "H\u00f4pital de la Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "CHRU de Tours", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Universit\u00e4tsklinikum Carl Gustav Carus", "city": "Dresden", "state": "", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Hannover Medical School", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4tsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Trinity College Dublin/Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Azienda Ospedaliera Universitaria Cagliari", "city": "Cagliari", "state": "", "country": "Italy", "status": "", "lat": 39.23054, "lon": 9.11917}, {"facility": "Centro Clinico NEMO", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "University of Milan Medical School", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "University of Torino - Rita Levi Montalcini Department of Neuroscience", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Azienda Ospedaliero Universitaria Di Modena", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Azienda Ospedaliera Universitaria ( A O U ) dell'Universit\u00e0 degli studi della Campania \"Luigi Vanvitelli\"", "city": "Naples", "state": "", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}, {"facility": "University of Padua - Azienda Ospedaliera di Padova", "city": "Padua", "state": "", "country": "Italy", "status": "", "lat": 45.40797, "lon": 11.88586}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Centrum Medyczne Neuromed", "city": "Bydgoszcz", "state": "", "country": "Poland", "status": "", "lat": 53.1235, "lon": 18.00762}, {"facility": "Linden Medical Centre", "city": "Krakow", "state": "", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}, {"facility": "City Clinic SP. z o. o.", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Centro Hospitalar Universit\u00e1rio Lisboa-Norte", "city": "Lisbon", "state": "", "country": "Portugal", "status": "", "lat": 38.72509, "lon": -9.1498}, {"facility": "Hospital Universitari de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario de Basurto", "city": "Bilbao", "state": "", "country": "Spain", "status": "", "lat": 43.26271, "lon": -2.92528}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Universitario La Paz-Carlos III", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Regional Universitario M\u00e1laga", "city": "M\u00e1laga", "state": "", "country": "Spain", "status": "", "lat": 36.72016, "lon": -4.42034}, {"facility": "Hospital Cl\u00ednico Universitario de Santiago de Compostela", "city": "Santiago de Compostela", "state": "", "country": "Spain", "status": "", "lat": 42.88052, "lon": -8.54569}, {"facility": "Hospital Virgen del Rocio", "city": "Seville", "state": "", "country": "Spain", "status": "", "lat": 37.38283, "lon": -5.97317}, {"facility": "Hospital Universitario y Polit\u00e9cnico La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Karolinska Institutet", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "King's College London", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Manchester MND care centre", "city": "Manchester", "state": "", "country": "United Kingdom", "status": "", "lat": 53.48095, "lon": -2.23743}, {"facility": "John Radcliffe Hospital", "city": "Oxford", "state": "", "country": "United Kingdom", "status": "", "lat": 51.75222, "lon": -1.25596}, {"facility": "Sheffield Teaching Hospitals NHS Foundation Trust", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Main Inclusion Criteria:\n\n* Age 18 - 80 years (both inclusive), male or female;\n* Diagnosis of definite, probable, probable laboratory supported or possible ALS as based on the El Escorial and the revised Airlie House diagnostic criteria for ALS;\n* Onset of first symptoms\\* no longer than 24 months prior to randomization;\n\n  \\*Date of onset is the date the patient reported one or more of the following symptoms:\n* Muscle weakness in limbs\n* Speech/swallowing difficulties\n* Respiratory symptoms: dyspnea was noticed\n* SVC equal to or more than 70% of the predicted normal value for gender, height and age at screening visit;\n* Change in ALSFRS-R score between 0.35 points and 1.5 points per month (both inclusive) in the period from onset of first symptoms to the Screening visit;\n* Capable of providing informed consent and complying with trial procedures.\n\nMain Exclusion Criteria:\n\n* Diagnosis of Primary Lateral Sclerosis;\n* Diagnosis of Frontotemporal Dementia;\n* Diagnosis of other neurodegenerative diseases (e.g. Parkinson disease, Alzheimer disease);\n* Diagnosis of polyneuropathy;\n* Other causes of neuromuscular weakness;\n* Have a significant pulmonary disorder not attributed to ALS and/or require treatment interfering with the evaluation of ALS on respiratory function;\n* Use of intravenous (IV) edaravone within 6 months of the screening visit;\n* Depend on mechanical ventilation (invasive or non-invasive) or require tracheostomy at Screening;\n* Renal impairment as indicated by a creatinine clearance of less than 50 mL/min as calculated by the Cockcroft Gault equation;\n* Subject has a history of clinically significant hepatic disease, hepatitis or biliary tract disease, or subject has a positive screening test for HIV, hepatitis B or C;", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "FAB122 (edaravone)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00444613", "title": "A Study in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Eisai Co., Ltd.", "summary": "The purpose of this study is to investigate the efficacy and confirm the safety of E0302 in patients with Amyotrophic Lateral Sclerosis (ALS) by assessing changes in scores of survival rate and functional rating scale.", "interventions": [{"type": "DRUG", "name": "E0302 (mecobalamin)"}, {"type": "DRUG", "name": "E0302 (mecobalamin)"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2007-04", "url": "https://clinicaltrials.gov/study/NCT00444613", "target_entities": ["neuronal_function_and_energy_metabolism"], "locations": [{"facility": "", "city": "Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "", "city": "Akita", "state": "Akita", "country": "Japan", "status": "", "lat": 39.71667, "lon": 140.11667}, {"facility": "", "city": "Aomori", "state": "Aomori", "country": "Japan", "status": "", "lat": 40.81667, "lon": 140.73333}, {"facility": "", "city": "Chiba", "state": "Chiba", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "", "city": "Touon-shi", "state": "Ehime", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Fukuoka", "state": "Fukuoka", "country": "Japan", "status": "", "lat": 33.6, "lon": 130.41667}, {"facility": "", "city": "Kitakyusyu-shi", "state": "Fukuoka", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Fukushima", "state": "Fukushima", "country": "Japan", "status": "", "lat": 37.75, "lon": 140.46667}, {"facility": "", "city": "Maebashi", "state": "Gunma", "country": "Japan", "status": "", "lat": 36.4, "lon": 139.08333}, {"facility": "", "city": "Higashihiroshima-shi", "state": "Hiroshima", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Miyoshi-shi", "state": "Hiroshima", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Otake-shi", "state": "Hiroshima", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Sapporo", "state": "Hokkaido", "country": "Japan", "status": "", "lat": 43.06667, "lon": 141.35}, {"facility": "", "city": "Kanazawa", "state": "Ishikawa-ken", "country": "Japan", "status": "", "lat": 36.6, "lon": 136.61667}, {"facility": "", "city": "Ichinoseki-shi", "state": "Iwate", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Sagamihara-shi", "state": "Kanagawa", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Yokohama", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.43333, "lon": 139.65}, {"facility": "", "city": "Nangoku-shi", "state": "Kochi", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Kyoto", "state": "Kyoto", "country": "Japan", "status": "", "lat": 35.02107, "lon": 135.75385}, {"facility": "", "city": "Tsu", "state": "Mie-ken", "country": "Japan", "status": "", "lat": 34.73333, "lon": 136.51667}, {"facility": "", "city": "Sendai", "state": "Miyagi", "country": "Japan", "status": "", "lat": 38.26667, "lon": 140.86667}, {"facility": "", "city": "Watari-gun", "state": "Miyagi", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Nagano", "state": "Nagano", "country": "Japan", "status": "", "lat": 36.65, "lon": 138.18333}, {"facility": "", "city": "Higashisonogi-gun", "state": "Nagasaki", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Kashiwazaki-shi", "state": "Niigata", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Niigata", "state": "Niigata", "country": "Japan", "status": "", "lat": 37.92259, "lon": 139.04125}, {"facility": "", "city": "Tsukubo-gun", "state": "Okayama-ken", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Ginowan-shi", "state": "Okinawa", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Toyonaka-shi", "state": "Osaka", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Hasuda-shi", "state": "Saitama", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Saitama-shi", "state": "Saitama", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "\u014ctsu", "state": "Shiga", "country": "Japan", "status": "", "lat": 35.0, "lon": 135.86667}, {"facility": "", "city": "Hamamatsu", "state": "Shizuoka", "country": "Japan", "status": "", "lat": 34.7, "lon": 137.73333}, {"facility": "", "city": "Shizuoka", "state": "Shizuoka", "country": "Japan", "status": "", "lat": 34.98333, "lon": 138.38333}, {"facility": "", "city": "Shimotsuke-shi", "state": "Tochigi", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Tokushima", "state": "Tokushima", "country": "Japan", "status": "", "lat": 34.06667, "lon": 134.56667}, {"facility": "", "city": "Yoshinogawa-shi", "state": "Tokushima", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Bunkyo-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Kodaira-shi", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "\u014cta-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.56126, "lon": 139.71605}, {"facility": "", "city": "Wakayama", "state": "Wakayama", "country": "Japan", "status": "", "lat": 34.23333, "lon": 135.16667}, {"facility": "", "city": "Yonezawa-shi", "state": "Yamagata", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Shimonoseki-shi", "state": "Yamaguchi", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Yanai-shi", "state": "Yamaguchi", "country": "Japan", "status": "", "lat": null, "lon": null}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patients who are able to submit written informed consent. If patients are duly capable of study consent but are unable to sign (or affix a seal) by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation.\n2. Patients who are aged 20 years or older at the time of obtaining informed consent.\n3. Patients who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised El Escorial Airlie House diagnostic criteria.\n4. Patients who are at stage 1 or 2 of the severity criteria for ALS.\n5. Patients within 3-year elapsed time period from disease onset at the start of observation period.\n6. Patients who can visit study site for out-patient treatment.\n\nExclusion Criteria:\n\n1. Patients who underwent tracheostomy.\n2. Patients who experienced non-invasive positive pressure ventilation.\n3. Patients whose percent-predicted forced vital capacity (%FVC) is \\>=60%.\n4. Patients with multiple disturbances of conduction detected by nerve conduction test.\n5. Patients with neurological symptom(s) due to vitamin B12 deficiency.\n6. Patients who initiated newly introduced riluzole therapy after starting the observation period. Or those who received dose escalation or resumed administration of riluzole therapy after previous down titration or discontinuation.\n7. Patients with cognitive impairment.\n8. Pregnant women or women with a possibility of becoming pregnant.\n9. Patients or their partners who are not willing to use reliable contraception.\n10. Patients with severe disease in the renal, cardiovascular, hematological, or hepatic system (severe disease will be judged referring to \"Ministry of Health, Labor and Welfare\" (MHLW) Drug Safety Dept. Notification No. 80, Drug Safety Classification Criteria for Severity of Adverse Drug Reaction by Medicinal Products, Grade 3).\n11. Patients with malignant tumor.\n12. Patients who participated in another clinical study within 12 weeks before starting the observation period.\n13. Patients with present illness or history of drug allergy or severe allergic disease (anaphylactic shock).\n14. Patients who are judged to be ineligible for study entry by the investigator or subinvestigator.", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "E0302 (mecobalamin)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03488524", "title": "Open Label Extension Study of AMX0035 in Patients With ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Amylyx Pharmaceuticals Inc.", "summary": "This extension study, in which all participants received active treatment (AMX0035), was designed to assess the longer-term safety and therapeutic potential of AMX0035 for participants who have completed the Main Study (AMX3500, also known as CENTAUR).", "interventions": [{"type": "DRUG", "name": "AMX0035"}], "start_date": "2018-03-29", "url": "https://clinicaltrials.gov/study/NCT03488524", "target_entities": ["TARDBP", "PFN1"], "locations": [{"facility": "Barrow Neurological Institute-Dignity Health, St Joseph's Hospital and Medical Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Florida College of Medicine", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "University of South Florida College of Medicine", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University Hospital", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "University of Iowa, Carver College of Medicine", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kentucky Medical Center", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Ochsner Neuroscience Institute", "city": "New Orleans", "state": "Louisiana", "country": "United States", "status": "", "lat": 29.95465, "lon": -90.07507}, {"facility": "Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Memorial Medical Center", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "University of Michigan, Michigan Medicine", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Washington University Medical Center", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates, PC", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Wake Forest School of Medicine", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Ohio State University, Wexner Medical Center", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Oregon Health & Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "The Penn Comprehensive Neuroscience Center", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Temple University Hospital", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Texas Neurology, PA", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Texas Health Science Center, San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "Swedish Neuroscience Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Completion of all visits in the randomized, double blind AMX3500 study. Participants who received tracheostomy or permanent assisted ventilation (PAV) during the course of the main study could enroll in the OLE if they completed all visits in the main study.\n2. Must enroll in the OLE within 28 days of the Week 24 visit of the main study.\n3. Signed informed consent to enter the OLE phase.\n\nExclusion Criteria:\n\n1. Discontinued study drug prematurely in the double-blind phase of the study for reasons other than tracheostomy or PAV.\n2. Exposure to or anticipated requirement for any disallowed medication listed in the protocol.\n3. Any ongoing adverse events that in the opinion of the Site Investigator are clear contraindications to the study drug.\n4. Unstable cardiac or other life-threatening disease emergent during the randomized, double blind study\n5. Any major medical condition that in the opinion of the Site Investigator would interfere with the study and place the subject at increased risk.", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AMX0035", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03202017", "title": "Lung Volume Recruitment Combined With Expiratory Muscle Strength Training in ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Minnesota", "summary": "The purpose of this study is to investigate the effects of two treatment techniques called Expiratory Muscle Strength Training (EMST) and Lung Volume Recruitment (LVR) on breathing, swallowing, speech, and cough function in persons with mild to moderate ALS. Half of the participants will do EMST alone, and the other half of the participants will do EMST and LVR.", "interventions": [{"type": "PROCEDURE", "name": "Expiratory Muscle Strength Training (EMST)"}, {"type": "PROCEDURE", "name": "EMST + Lung Volume Recruitment (LVR)"}], "start_date": "2018-03-01", "url": "https://clinicaltrials.gov/study/NCT03202017", "target_entities": [], "locations": [{"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS defined as possible, laboratory-supported probable, probable, or definite by El Escorial criteria\n* Reduced Maximal Expiratory Pressure (MEP) compared to norms for age and sex\n* Forced Vital Capacity (FVC) \\> 65% predicted\n\nExclusion Criteria:\n\n* Inability to provide informed consent\n* Relative contraindications to LVR, including known pneumothorax, active internal bleeding, unstable hypertension, unstable angina, emphysema, recent barotrauma, or FEV1 (Forced Expiratory Volume, trial 1)/FVC ratio \\< 0.7.\n* Use of EMST or breath stacking \\> 3 days/week within 12 weeks of screening\n* Amyotrophic Lateral Sclerosis-Cognitive Behavioral Scale (ALS-CBS) score predictive of dementia (\u2264 10)\n* Participation in another clinical trial of an intervention in ALS within 30 days of study enrollment or during study enrollment", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03154450", "title": "EncoreAnywhere Use in Motor Neurone Disease", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sheffield Teaching Hospitals NHS Foundation Trust", "summary": "Motor neurone disease is a progressive incurable disease causing weakness and paralysis of muscles. Respiratory failure is the most common cause of death in motor neurone disease. Patients with respiratory failure use a machine that supports breathing using a mask and ventilator (non-invasive ventilation: NIV) and using it for more than five hours per night has been shown to prolong life and improve symptoms such as poor sleep and breathlessness. NIV is however, challenging to use and some patients are unable to adhere to the required regime meaning they fail to gain benefit. Timely support is important to help individuals overcome early hurdles and barriers to using becoming regular NIV users.\n\nThe Philips EncoreAnywhere is a system that allows continuous monitoring of the use and effectiveness of ventilation and allows instant adjustment of ventilator settings. The aim of this project is to explore if \"real time\" feedback and support, as well as remote changes to NIV settings using the EncoreAnywhere system could increase the number of individuals successfully using NIV. This project also aims to explore the impact of using EncoreAnywhere on the process of initiation of NIV, on both patients and staff.\n\nPatients starting NIV at the Sheffield MND care centre will be provided with the standard ventilator plus a Philips modem for the first three months of use. In half the patients clinicians will be able to use the EncoreAnywhere system to review patients' use of NIV, make adjustments and give feedback. In the other half, the data will be collected but not available to the clinical team. Clinical data will be collected as part of usual care: adherence, clinical encounters and resource use and patients will be asked to complete questionnaires at baseline, one month and three months. This will allow the care team to predict the potential impact on the service and on clinical care. This is a small pilot, feasibility study, and if the study is deemed feasible, a further larger randomized controlled trial is planned. The study will last for a maximum of 12 months, recruiting up to 40 patients.", "interventions": [{"type": "DEVICE", "name": "EncoreAnywhere with data available to review"}, {"type": "DEVICE", "name": "EncoreAnywhere with no data available to review"}], "start_date": "2016-01-21", "url": "https://clinicaltrials.gov/study/NCT03154450", "target_entities": [], "locations": [{"facility": "Royal Hallamshire Hospital", "city": "Sheffield", "state": "South Yorkshire", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patients with motor neuron disease diagnosed by a consultant neurologist.\n2. Receiving care from the Sheffield Teaching Hospitals NHS Trust MND clinic\n3. Respiratory failure diagnosed by the clinical team needing to start NIV\n4. Determined to be suitable for and willing to commence non-invasive ventilation including the EncoreAnywhere features as part of their usual care at the Royal Hallamshire Hospital MND clinic.\n\nExclusion Criteria:\n\n1. Patients already established on non-invasive ventilation e.g. in obstructive sleep apnoea\n2. Those with no mobile internet reception in their homes (required to use EncoreAnywhere", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01753076", "title": "Study of Ozanezumab (GSK1223249) Versus Placebo in the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "GlaxoSmithKline", "summary": "This is a 48-week, randomised, multi-centre, double-blind, placebo-controlled, parallel group investigation of the efficacy and safety of intravenous (IV) ozanezumab (GSK1223249) compared to placebo in subjects with Amyotrophic Lateral Sclerosis (ALS). Following a screening period of up to four weeks, eligible subjects will be randomised (1:1) to receive IV placebo or 15 milligram (mg)/ kilogram (kg) IV ozanezumab every 2 weeks for a period of 48 weeks with a follow-up visit around 14 weeks after the last infusion. A total of approximately 294 eligible subjects will be randomised from approximately 37 centers worldwide. The primary objective is to assess the effect of ozanezumab on the physical function and survival of ALS subjects over a treatment period of 48 weeks. Function will be measured using the ALS Functional Rating Scale - Revised (ALSFRS-R). Secondary objectives include the evaluation of other clinical outcomes associated with ALS (respiratory function, muscle strength, progression free survival and overall survival) in support of the primary objective. Quality of life, safety, tolerability, immunogenicity and pharmacokinetics (ozanezumab and riluzole) will also be assessed.", "interventions": [{"type": "DRUG", "name": "Ozanezumab"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2012-12-20", "url": "https://clinicaltrials.gov/study/NCT01753076", "target_entities": ["Nogo-A"], "locations": [{"facility": "GSK Investigational Site", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "GSK Investigational Site", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "GSK Investigational Site", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "GSK Investigational Site", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "GSK Investigational Site", "city": "Randwick", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.91439, "lon": 151.24895}, {"facility": "GSK Investigational Site", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "GSK Investigational Site", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "GSK Investigational Site", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "GSK Investigational Site", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "GSK Investigational Site", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "GSK Investigational Site", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "GSK Investigational Site", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "GSK Investigational Site", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "GSK Investigational Site", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "GSK Investigational Site", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "GSK Investigational Site", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "GSK Investigational Site", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "GSK Investigational Site", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "GSK Investigational Site", "city": "Munich", "state": "Bavaria", "country": "Germany", "status": "", "lat": 48.13743, "lon": 11.57549}, {"facility": "GSK Investigational Site", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "GSK Investigational Site", "city": "Bochum", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "GSK Investigational Site", "city": "Jena", "state": "Thuringia", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "GSK Investigational Site", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "GSK Investigational Site", "city": "Turin", "state": "Piedmont", "country": "Italy", "status": "", "lat": 45.07049, "lon": 7.68682}, {"facility": "GSK Investigational Site", "city": "Verona", "state": "Veneto", "country": "Italy", "status": "", "lat": 45.43854, "lon": 10.9938}, {"facility": "GSK Investigational Site", "city": "Kanagawa", "state": "", "country": "Japan", "status": "", "lat": 37.58333, "lon": 139.91667}, {"facility": "GSK Investigational Site", "city": "Miyagi", "state": "", "country": "Japan", "status": "", "lat": 26.62566, "lon": 128.18236}, {"facility": "GSK Investigational Site", "city": "Osaka", "state": "", "country": "Japan", "status": "", "lat": 34.69379, "lon": 135.50107}, {"facility": "GSK Investigational Site", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "GSK Investigational Site", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "GSK Investigational Site", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "GSK Investigational Site", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "GSK Investigational Site", "city": "Preston", "state": "Lancashire", "country": "United Kingdom", "status": "", "lat": 53.76282, "lon": -2.70452}, {"facility": "GSK Investigational Site", "city": "Brighton", "state": "", "country": "United Kingdom", "status": "", "lat": 50.82838, "lon": -0.13947}, {"facility": "GSK Investigational Site", "city": "Edgbaston", "state": "", "country": "United Kingdom", "status": "", "lat": 52.4623, "lon": -1.92115}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with diagnosis of familial or sporadic ALS\n* Onset of muscle weakness no more than 30 months before screening visit.\n* Slow Vital Capacity (SVC) of at least 65% predicted for gender, age, ethnicity and height at Screening.\n* If on riluzole, the dose must have been stable for at least 28 days prior to Baseline visit.\n* Age 18 - 80 years inclusive.\n* Female subjects may participate if they are of non-child-bearing potential or if they are of child-bearing potential they must agree to use the approved contraceptive methods\n* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \\<=2x upper limit of normal (ULN); alkaline phosphatase and bilirubin \\<=1.5xULN.\n* QTc (both QTcB and QTcF) \\<450 milliseconds (msec) or \\<480 msec for subjects with Bundle Branch Block at Screening and Baseline (average from triplicate ECGs).\n\nExclusion Criteria:\n\n* Patients with other neuromuscular disorders (including a history of polio) which in the opinion of the investigator could have contributed to the muscular atrophy or weakness caused by ALS\n* Patients with primary lateral sclerosis, monomelic ALS, ALS Parkinsonism dementia complex.\n* Patients requiring non-invasive or mechanical ventilation (non-invasive ventilation for sleep apnoea is allowed subject to discussion with Medical Monitor)\n* Patients on diaphragmatic pacing.\n* Presence of any of the following clinical conditions: Drug abuse or alcoholism, uncontrolled hypertension, active major infectious disease, unstable psychiatric illness within 90 days of the Screening visit\n* Subjects, who in the investigator's judgement, pose a significant suicide risk. - Current or chronic history of liver disease, known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones), positive Hepatitis B surface antigen or Hepatitis C antibody test.\n* Subjects who have participated in a clinical trial involving receipt of a biopharmaceutical product within 6 months prior to the first dosing day.\n* Exposure to non-biological experimental agents 1 month or 5 half-lives prior to Baseline visit (whichever is longer).\n* History of sensitivity to ozanezumab, or components thereof, or a history of other allergies that, in the opinion of the investigator, contraindicates participation in the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Ozanezumab", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06681610", "title": "Testing Pulse Stimulation to Improve Motor Function in People With ALS: A Pilot Study", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Parc de Salut Mar", "summary": "The goal of this clinical trial is to assess the efficacy of TPS of the motor cortex on biomarkers and clinical endpoints in patients with ALS. The main questions it aims to answer are:\n\n* Stage 1: Is there a change in the short intracortical inhibition (SICI) of the motor cortex from baseline to week 8?\n* Stage 2: Is there a change from baseline to month 6 in the ALS functional rating scale-revised (ALSFRS-R) total score?\n\nIn stage 2, researchers will compare the group receiving the stimulation vs the group receiving a sham stimulation to see if there is a difference in motor cortex activity and in the ALSFRS-R score\n\nParticipants will receive either:\n\n* the TPS treatment\n* a sham TPS treatment", "interventions": [{"type": "DEVICE", "name": "Transcranial Pulse Stimulation"}], "start_date": "2024-10-24", "url": "https://clinicaltrials.gov/study/NCT06681610", "target_entities": ["motor_cortex_excitability"], "locations": [{"facility": "Hospital del Mar", "city": "Barcelona", "state": "Catalonia", "country": "Spain", "status": "RECRUITING", "lat": 41.38879, "lon": 2.15899}], "contact_phone": "+34932 48 30 00", "contact_email": "aleonjorba@psmar.cat", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with diagnosis of sporadic ALS (definite or clinically probable) as defined by the World Federation of Neurology revised El Escorial criteria\n* SVC of 50% or greater of estimated measure and presence of measurable motor evoked potential\n* 21 to 80 years old and male or female\n\nExclusion Criteria:\n\n* patients with fALS (based on medical history) that are unable to tolerate TMS and MRI studies or have a contraindication as described below", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02710110", "title": "Respiratory Strength Training in Persons With Amyotrophic Lateral Sclerosis (ALS)", "phase": "EARLY_PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Florida", "summary": "Dysphagia (swallow impairment), dystussia (cough impairment) and respiratory impairment are hallmark features of amyotrophic lateral sclerosis (ALS). These symptoms are the cause of fatal aspiration, malnutrition and respiratory insufficiency that together account for 91.4% of ALS mortality. Unfortunately, treatments to prolong and maintain these vital functions are currently lacking. Although the use of exercise in ALS is controversial, recent evidence suggests that mild to moderate intensity exercise applied early in the disease slows disease progression, improves motor function, preserves motor neuron number, reduces muscle hypoplasia, atrophy astrogliosis, and prolongs survival in animal models of ALS and human clinical trials.\n\nThis research study is designed to determine the impact of respiratory strength training on breathing, airway protection and swallowing in persons with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DEVICE", "name": "PowerLung trainer"}, {"type": "DEVICE", "name": "Micro Mouth Pressure Meter"}, {"type": "PROCEDURE", "name": "Pulmonary Function Testing"}, {"type": "PROCEDURE", "name": "Videofluoroscopic swallowing study"}, {"type": "OTHER", "name": "Swallowing Quality of Life Questionnaire"}, {"type": "DEVICE", "name": "Iowa Oral Pressure Instrument"}, {"type": "DRUG", "name": "Capsaicin"}], "start_date": "2016-04", "url": "https://clinicaltrials.gov/study/NCT02710110", "target_entities": ["respiratory_function", "swallowing_dysphagia"], "locations": [{"facility": "UF Health Shands", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS),\n* Amyotrophic Lateral Sclerosis Rating Scale Revised score greater than 34,\n* forced vital capacity greater than 70%,\n* cognition within normal limits as determined by Montreal assessment of cognition score \\>25\n\nExclusion Criteria:\n\n* allergies to barium,\n* tracheotomy or mechanical ventilation,\n* diaphragmatic pacer,\n* concurrent respiratory disease (e.g. COPD),\n* pregnant at the time of the study due to radiation exposure", "sex": "ALL", "min_age": "21 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Capsaicin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07287397", "title": "Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Vector Y Therapeutics", "summary": "PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS).\n\nTwo escalating dose (low dose and high dose) cohorts are planned. The duration of the study will be a maximum of 5 years and 5 weeks (265 weeks) for each participant. The screening period may last up to 5 weeks to complete screening procedures.", "interventions": [{"type": "GENETIC", "name": "VTx-002"}, {"type": "DRUG", "name": "Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone"}], "start_date": "2025-12-19", "url": "https://clinicaltrials.gov/study/NCT07287397", "target_entities": ["vtx_002"], "locations": [{"facility": "St Joseph's Hospital and medical Center - Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "NOT_YET_RECRUITING", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California San Diego Medical Center", "city": "San Diego", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 32.71571, "lon": -117.16472}, {"facility": "Mayo Clinic in Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami School of Science", "city": "Miami", "state": "Florida", "country": "United States", "status": "NOT_YET_RECRUITING", "lat": 25.77427, "lon": -80.19366}, {"facility": "Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "Herbert Irving Comprehensive Cancer Center", "city": "New York", "state": "New York", "country": "United States", "status": "NOT_YET_RECRUITING", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 39.95238, "lon": -75.16362}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "Kings College Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "NOT_YET_RECRUITING", "lat": 51.50853, "lon": -0.12574}, {"facility": "Royal Hallamshire Hospital", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "patients@vectorytx.com", "contact_email": "patients@vectorytx.com", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n1. Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit requirements\n2. Male or female \u2265 18 years of age\n3. Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 2000) (probable, laboratory results supported; clinically probable, clinically definite)\n4. Confirmed absence of ALS caused by FUS and SOD1 gene mutations confirmed by laboratory tests.\n5. A maximum of 18 months since first appearance of weakness (e.g., limb weakness, dysarthria, dysphagia, shortness of breath)\n6. Erect (seated) SVC % predicted \u2265 80% at Screening\n7. Treatment Research Initiative to Cure ALS (TRICALS) risk score between -2 and -6 at Screening\n8. Has a reliable caregiver/partner/legal representative willing and able to support the participant in participation in the study and to give informed consent on behalf of the participant in the case that disease progression prevents the participant of giving consent (local legal rules will apply).\n9. Treatment with riluzole and/or edaravone is allowed if treatment was started and has remained at a stable dose for at least 2 weeks (riluzole) or one treatment cycle (edaravone) before the Screening visit\n10. Women of childbearing potential (WOCBP) and male participants with female partners who are WOCBP must agree to use highly effective contraception during and after the study. WOCBP cannot be pregnant or breastfeeding\n11. Women of nonchildbearing potential must be post-menopausal or surgically sterile (e.g. hysterectomy, bilateral tubal ligation, ovaries removed)\n\nKey Exclusion Criteria:\n\n1. Diagnosis of a significant CNS or peripheral nervous system disease other than ALS that may be a cause for the participant's ALS symptoms or may confound study objectives\n2. Spinal, cervical, or brain MRI/MRA indicating clinically significant abnormality\n3. Presence of tracheostomy and feeding tube at Screening\n4. Contraindications to corticosteroid use (e.g. due to osteoporosis, uncontrolled blood pressure, diabetes or cholesterol).\n\n5\\. Significant concomitant disease or condition within 6 months of Screening that could pose an unacceptable safety risk to the participant or interfere with the participant's ability to comply with study procedures, e.g. heart disease, uncontrolled diabetes, liver disease, autoimmune diseases needing strong immune-suppressing drugs, cancer, etc or a current psychiatric diagnosis.\n\n6\\. Clinically significant abnormalities in laboratory test results at Screening for example poor liver or kidney function, abnormal clotting or infections such as Hepatitis or HIV\n\n7\\. Use of blood thinners (e.g., warfarin, heparin, and novel oral anticoagulants) and being unable to safely stop them before certain study procedures.\n\n8\\. Contraindications to imaging methods MRI, MRA, CT due to claustrophobia and/or intolerance to contrast agents.\n\n9\\. Contraindications to general anaesthesia (GA) or deep sedation\n\n10 Positive test for illegal drugs (except prescribed medications or permitted medicinal/recreational marijuana if used responsibly)\n\n11\\. Generally frail or if the Investigator deems participation in the study would not be in the best interest of the participant or is likely to prohibit further participation during the study\n\nOther protocol-defined inclusion/exclusion criteria may apply\n\n\\-", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "VTx-002", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07325591", "title": "Efficacy and Safety of Tazbentetol in ALS Participants", "phase": "PHASE2, PHASE3", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Spinogenix", "summary": "The objectives of this study are to examine the effects of tazbentetol on clinical measures of ALS, patient reported outcomes (PROs), long-term safety and tolerability.", "interventions": [{"type": "DRUG", "name": "Tazbentetol"}, {"type": "DRUG", "name": "placebo"}], "start_date": "2026-10-01", "url": "https://clinicaltrials.gov/study/NCT07325591", "target_entities": ["tazbentetol"], "locations": [], "contact_phone": "503 915 1400", "contact_email": "contact@spinogenix.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age 18-80\n* ALS TRICALS risk score\n* Stable dose of standard of care treatment\n* Contraception use by men or women consistent with local regulations\n* Able and willing to provide written informed consent\n\nExclusion Criteria:\n\n* Underlying physical or psychological condition prohibiting study completion\n* Clinically significant cardiac disease\n* Active or history of malignancy in the past 5 years\n* Serious infection within 1 month of screening\n* Acute illness within 30 days of Day 1\n* History of suicidal behavior or suicidal ideation\n* Active cigarette smokers and users of nicotine-containing products\n* Neurodegenerative disease\n* External respiratory support or supplemental oxygen requirement\n* HIV, hepatitis B and hepatitis C positive\n* Vaccines within 14 days\n* Other investigational products within 30 days\n* Blood donation within 30 days\n* Plasma donation within 7 days\n* Pregnant or breastfeeding", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tazbentetol", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03707795", "title": "Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study", "phase": "EARLY_PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Edward Kasaraskis", "summary": "By doing this study the investigator hopes to learn more about a potential cause of amyotrophic lateral sclerosis (ALS) called \"oxidative stress\". Oxidative stress is essentially an imbalance between the production of certain chemicals in the body called \"free radicals\" and the ability of the body to counteract or detoxify their harmful effects through neutralization by antioxidants. It is thought that factors such as environmental exposure (chemicals and lead), diet, smoking,alcohol consumption, physical activity and psychological stress cause oxidative stress to occur inside the body.\n\nBy doing this study, the investigator hopes to learn whether the FDA-approved steroid medication called Betamethasone will restore overall antioxidant activity fALS patients with mutations in the Fused in Sarcoma gene (FUS gene).\n\nParticipants who agree to take part in this research study, agree to the following responsibilities:\n\n* Attend all scheduled visits\n* Notify the study doctor of any illnesses, unexpected or troublesome side effects, or any other medical problems that occur during the study\n* Be completely honest with their answers to all questions\n* Check with the study doctor before taking any new medications, whether prescribed or \"over the counter,\" even vitamins and herbal supplements.", "interventions": [{"type": "DRUG", "name": "Betamethasone sodium phosphate/betamethasone acetate (Celestone\u00ae Soluspan\u00ae), 30 mg IM once a day for four days"}], "start_date": "2017-08-21", "url": "https://clinicaltrials.gov/study/NCT03707795", "target_entities": ["FUS", "oxidative_stress"], "locations": [{"facility": "University of Kentucky Medical Center", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of familial ALS (fALS)\n* Relative of a fALS person and carry the FUS gene\n\nExclusion Criteria:\n\n* Under 20 years or over 80 years of age\n* Cannot tolerate steroids, including betamethasone\n* Are unwilling or unable to attend all scheduled research visits\n* Currently participating in another clinical drug trial\n* Major neurological disease, other than ALS\n* Pregnant", "sex": "ALL", "min_age": "20 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Betamethasone sodium phosphate/betamethasone acetate", "targeting_mechanism": "Corticosteroid with anti-inflammatory and antioxidant properties targeting oxidative stress implicated in FUS-related ALS pathogenesis.", "targeting_mechanism_pmid": "34663413", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00445172", "title": "A Long-Term Study in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Eisai Co., Ltd.", "summary": "The purpose of this study is to investigate the safety and efficacy of long-term E0302 administration in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "E0302 (mecobalamin)"}], "start_date": "2008-02", "url": "https://clinicaltrials.gov/study/NCT00445172", "target_entities": ["mecobalamin"], "locations": [{"facility": "", "city": "Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "", "city": "Aomori", "state": "Aomori", "country": "Japan", "status": "", "lat": 40.81667, "lon": 140.73333}, {"facility": "", "city": "Chiba", "state": "Chiba", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "", "city": "Touon-shi", "state": "Ehime", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Fukuoka", "state": "Fukuoka", "country": "Japan", "status": "", "lat": 33.6, "lon": 130.41667}, {"facility": "", "city": "Kitakyushi-shi", "state": "Fukuoka", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Fukushima", "state": "Fukushima", "country": "Japan", "status": "", "lat": 37.75, "lon": 140.46667}, {"facility": "", "city": "Maebashi", "state": "Gunma", "country": "Japan", "status": "", "lat": 36.4, "lon": 139.08333}, {"facility": "", "city": "Higashihiroshima-shi", "state": "Hiroshima", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Miyoshi-shi", "state": "Hiroshima", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Otake-shi", "state": "Hiroshima", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Sapporo", "state": "Hokkaido", "country": "Japan", "status": "", "lat": 43.06667, "lon": 141.35}, {"facility": "", "city": "Kanazawa", "state": "Ishikawa-ken", "country": "Japan", "status": "", "lat": 36.6, "lon": 136.61667}, {"facility": "", "city": "Ichinoseki-shi", "state": "Iwate", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Sagamihara-shi", "state": "Kanagawa", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Yokohama", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.43333, "lon": 139.65}, {"facility": "", "city": "Nangoku-shi", "state": "Kochi", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Kyoto", "state": "Kyoto", "country": "Japan", "status": "", "lat": 35.02107, "lon": 135.75385}, {"facility": "", "city": "Tsu", "state": "Mie-ken", "country": "Japan", "status": "", "lat": 34.73333, "lon": 136.51667}, {"facility": "", "city": "Sendai", "state": "Miyagi", "country": "Japan", "status": "", "lat": 38.26667, "lon": 140.86667}, {"facility": "", "city": "Watari-gun", "state": "Miyagi", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Nagano", "state": "Nagano", "country": "Japan", "status": "", "lat": 36.65, "lon": 138.18333}, {"facility": "", "city": "Higashisonogi-gun", "state": "Nagasaki", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Kashiwazaki-shi", "state": "Niigata", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Niigata", "state": "Niigata", "country": "Japan", "status": "", "lat": 37.92259, "lon": 139.04125}, {"facility": "", "city": "Tsukubo-gun", "state": "Okayama-ken", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Ginowan-shi", "state": "Okinawa", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Toyonaka-shi", "state": "Osaka", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Hasuda-shi", "state": "Saitama", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Saitama-shi", "state": "Saitama", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Hamamatsu", "state": "Shizuoka", "country": "Japan", "status": "", "lat": 34.7, "lon": 137.73333}, {"facility": "", "city": "Shizuoka", "state": "Shizuoka", "country": "Japan", "status": "", "lat": 34.98333, "lon": 138.38333}, {"facility": "", "city": "Shimotsuke-shi", "state": "Tochigi", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Tokushima", "state": "Tokushima", "country": "Japan", "status": "", "lat": 34.06667, "lon": 134.56667}, {"facility": "", "city": "Yoshinogawa-shi", "state": "Tokushima", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Bunkyo-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Kodaira-shi", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "\u014cta-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.56126, "lon": 139.71605}, {"facility": "", "city": "Wakayama", "state": "Wakayama", "country": "Japan", "status": "", "lat": 34.23333, "lon": 135.16667}, {"facility": "", "city": "Yonezawa-shi", "state": "Yamagata", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Shimonoseki-shi", "state": "Yamaguchi", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "", "city": "Yanai-shi", "state": "Yamaguchi", "country": "Japan", "status": "", "lat": null, "lon": null}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patients who have completed Phase II/III study of E0302 (E0302-J081-761, hereafter referred to as Study 761) except for those patients who discontinued the treatment of Study 761. Completed patients are defined as those who completed the treatment period of Study 761, those on 24-hour use of non-invasive positive pressure ventilation (NIPPV), or those who eventually resulted in the use of NIPPV.\n2. Patients who are able to submit written informed consent. If patients are duly capable of study consent but are unable to sign (or affix a seal) by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation.\n\nExclusion Criteria:\n\n1. Patients with cognitive impairment.\n2. Pregnant women or women who may have a possibility of becoming pregnant.\n3. Patients or their partners who are not willing to use reliable contraception.\n4. Patients with severe disease in the renal, cardiovascular, hematological, or hepatic system (severe disease will be judged referring to \"Ministry of Health, Labor and Welfare (MHLW) Drug Safety Dept. Notification No. 80, Drug Safety Classification Criteria for Severity of Adverse Drug Reaction by Medicinal Products, Grade 3.\" However, an event due to the primary disease will be precluded).\n5. Patients with malignant tumor.\n6. Patients who participated in another clinical study after the completion of Study 761.\n7. Patients with present illness or history of drug allergy or severe allergic disease (anaphylactic shock).\n8. Patients who are judged to be ineligible for study entry by the investigator or subinvestigator.", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "E0302 (mecobalamin)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03296501", "title": "Intraspinal Transplantation of Autologous ADRC in ALS Patients", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mossakowski Medical Research Centre Polish Academy of Sciences", "summary": "The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis (ALS). All enrolled patients will have a documented at least 3-months clinical and electrophisiological observation of ALS disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).", "interventions": [{"type": "BIOLOGICAL", "name": "Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients"}], "start_date": "2015-10-13", "url": "https://clinicaltrials.gov/study/NCT03296501", "target_entities": ["neurotrophic_factors", "motor_neuron_degeneration"], "locations": [{"facility": "Medical University of Warsaw", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* clinically definite or probable ALS according to El Escorial criteria\n* life expectancy of more than 1 year\n* INR \u22642 before liposuction\n* lack of treatment with immune-suppressants and/or corticosteroids within min. 20 days prior to recruitment\n* constant riluzole treatment (50 mg/bid) throughout the study period\n* compliance with treatment regimen e.g. will and possibility to attend check-up visits\n* Polish citizens\n\nExclusion Criteria:\n\n* primary haematological disease, including hypercoagulable states\n* Presence of comorbidity that would stand in the way of neurosurgical treatment-\n* previous history of a spinal-cord surgery at the clinically affected level\n* previous/current history of neoplasm or comorbidity that could impact upon patient's survival\n* PEG\n* pregnancy /lactation\n* noninvasive/invasive mechanical ventilation at time of recruitment\n* alcohol abuse, cocaine amphetamine, etc.", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "autologous adipose derived regenerative cells (ADRC)", "targeting_mechanism": "Stem cells differentiate into support cells such as astrocytes, oligodendrocytes or microglia to produce growth factors, anti-inflammatory cytokines, and provide neuroprotection to degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Stem cell therapies have demonstrated potential benefit in preclinical studies using mouse and rat ALS models expressing mutant superoxide dismutase 1.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02017912", "title": "Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study of Autologous MSC-NTF Cells in Patients With ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Brainstorm-Cell Therapeutics", "summary": "This is a multi-center, randomized, double blind, placebo controlled study to evaluate the safety and efficacy of autologous (self) transplantation of Neurotrophic factors-secreting Mesenchymal Stromal Cells (MSC-NTF, NurOwn\u2122) in patients with ALS .\n\nMSC-NTF cells are a novel cell-therapeutic approach which is expected to effectively deliver Neurotrophic factors, which are potent survival factors for neurons, directly to the site of damage.", "interventions": [{"type": "BIOLOGICAL", "name": "Nurown MSC-NTF cells"}, {"type": "BIOLOGICAL", "name": "Placebo"}], "start_date": "2014-05", "url": "https://clinicaltrials.gov/study/NCT02017912", "target_entities": ["neurotrophic_factors", "motor_neuron_survival"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "UMass Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria\n\n1. Males and females ages 18 to 75 years old, inclusive.\n2. ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.\n3. Disease onset, as defined by first reported occurrence of symptomatic weakness, spasticity, or bulbar symptoms, of more than 12 months and less than or equal to 24 months.\n4. Current disease symptoms must include limb weakness.\n5. ALSFRS-R \u226530 at the Screening Visit.\n6. Upright slow vital capacity (SVC) measure \u226565% of predicted for gender, height, and age at the Screening Visit.\n7. Subjects must be taking a stable dose of riluzole for at least 30 days prior to enrolment or not be on riluzole, and not have been on it for at least 30 days prior to enrolment (riluzole-na\u00efve subjects are permitted in the study).\n8. Capable of providing informed consent and willing and able to follow study procedures, including willingness to undergo lumbar puncture.\n9. Geographic accessibility to the study site and willingness and ability to comply with follow-up.\n10. Women of child-bearing potential must agree not to become pregnant for the duration of the study. Women must be willing to consistently use two forms of contraceptive therapy throughout the course of the trial, and undergo a pregnancy test one week before bone marrow aspiration. Men must be willing to consistently use two forms of contraceptive if their partners are of child-bearing age.\n11. Citizen or permanent resident of the United States.\n\nExclusion Criteria:\n\n1. Prior stem cell therapy of any kind.\n2. Inability to lie flat for the duration of intrathecal cell transplantation and/or bone marrow biopsy, or inability to tolerate study procedures for any other reason.\n3. History of autoimmune disease (excluding thyroid disease) myelodysplastic or myeloproliferative disorder, leukemia or lymphoma, whole body irradiation, hip fracture, or severe scoliosis.\n4. Any unstable clinically significant medical condition other than ALS (e.g., within six months of baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure), treatment with anticoagulants that, in the opinion of the investigator, would compromise the safety of patients.\n5. Any history of malignancy including any malignancy affecting the central nervous system and melanoma, within the previous 5 years, with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline).\n6. Serum AST or ALT value \\>3.0 times the upper normal limit.\n7. Serum creatinine value \\>2.0 times the upper normal limit.\n8. Positive test for Hepatitis B, Hepatitis C, HIV.\n9. Current use of immunosuppressant medication or use of such medication within 4 weeks of Screening visit (Visit 1).\n10. Any history of acquired or inherited immune deficiency syndrome.\n11. Exposure to any other experimental agent (off-label use or investigational) or participation in a clinical trial within 30 days prior to Screening Visit (Visit 1).\n12. Use of non-invasive ventilation (NIV), diaphragm pacing system or invasive ventilation (tracheostomy).\n13. Any history of either substance abuse within the past year, or unstable psychiatric disease according to PI judgment.\n14. Placement or usage of feeding tube.\n15. Pregnant women or women currently breastfeeding.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MSC-NTF cells (autologous neurotrophic factor-secreting mesenchymal stromal cells)", "targeting_mechanism": "Mesenchymal stromal cells engineered to secrete neurotrophic factors that provide survival signals and neuroprotection to motor neurons.", "targeting_mechanism_pmid": "", "animal_results": "In SOD1G93A transgenic mice, hematopoietic bone marrow-derived mesenchymal stem cells expressing Ngn1 increased lifespan by 3 days, delayed disease onset by 5 days, and reduced motor neuron loss.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02781454", "title": "Mexiletine in Sporadic Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Washington", "summary": "The purpose of this research study is to find out whether the drug mexiletine will be effective in lowering motor neuron electrical activity in the brains and nerves in the arms of people with ALS. The investigators will also determine if there are any signs that the drug may slow down the progression of ALS and reduce muscle cramps and muscle twitching. This will be determined through transcranial magnetic stimulation (TMS) and threshold tracking nerve conduction studies (TTNCS). In this trial, the participants will be taking either 300mg/day of mexiletine, 600mg/day of mexiletine, or placebo (non-active study drug).", "interventions": [{"type": "DRUG", "name": "Mexiletine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2016-10", "url": "https://clinicaltrials.gov/study/NCT02781454", "target_entities": ["Sodium channel", "motor_neuron_electrical_activity"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Augusta University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Beth Israel Deaconess Medical Center", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Columbia Universtiy Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Pennsylvania State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "University of Pittsburgh", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Medical University of South Carolina", "city": "Charleston", "state": "South Carolina", "country": "United States", "status": "", "lat": 32.77632, "lon": -79.93275}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Sporadic ALS diagnosed as possible, laboratory-supported probable, probable, or definite ALS as defined by revised El Escorial criteria.\n2. Age 18 years or older.\n3. Symptom onset of weakness or spasticity due to ALS \u2264 60 months prior to Screening Visit.\n4. Slow vital capacity (SVC) measure \u226550% of predicted for gender, height, and age at the screening visit.\n5. Must be able to swallow capsules throughout the course of the study, according to Site Investigator judgment.\n6. Capable of providing informed consent and following trial procedures.\n7. For TMS: a resting motor threshold defined as 50% of pulses eliciting a motor evoked potential (MEP) of amplitude \u2265 50 \u00b5V.\n8. For TTNCS: median Compound Muscle Action Potential (CMAP) \u2265 1.5 mV.\n9. Subjects must not have taken riluzole for at least 30 days or be on a stable dose of riluzole for at least 30 days prior to the Screening Visit and continue on the stable dose throughout the course of the study (riluzole-na\u00efve subjects are permitted in the study).\n10. Subjects must not have taken medication for muscle cramping such as cyclobenzaprine, baclofen, carisoprodol, or methocarbamol, for at least 30 days prior to screening or be on a stable dose for at least 60 days prior to screening.\n11. Geographic accessibility to the site.\n12. Women must not become pregnant for the duration of the study and must be willing to use two contraceptive therapies and have a negative pregnancy test throughout the course of the study.\n13. Use of medications known to affect the neurophysiology measures in the study must be scheduled, not as needed (pro re nata, PRN). A subject must have been on a fixed dose for 30 days prior to the Screening Visit, and there must be no reason to believe that a subsequent change would be necessary during the course of the study. These medications include: benzodiazepines, muscle relaxants, tricyclic antidepressants, selective serotonin reuptake inhibitors, non-selective serotonin reuptake inhibitors, hypnotics (including anti-histamines) and anti-cholinergics.\n\nExclusion Criteria:\n\n1. Invasive ventilator dependence, such as tracheostomy.\n2. Creatinine level greater than 1.5 mg/dL at screening.\n3. Serum Glutamic-Oxaloacetic (SGOT/AST) / Serum Glutamic-Pyruvic (SGPT/ALT) greater than 3 times the upper limit of normal at screening.\n4. History of known sensitivity or intolerability to mexiletine or lidocaine.\n5. Any history of either substance abuse within the past year, unstable psychiatric disease, cognitive impairment, or dementia.\n6. Clinically significant conduction abnormalities on electrocardiogram or a known history of cardiac arrhythmia.\n7. Known history of epilepsy.\n8. Known history of congestive heart failure (CHF) or history of myocardial infarction within the past 24 months.\n9. Use of mexiletine for 30 days prior to Screening Visit.\n10. Exposure to any other experimental agent (off-label use or investigational) including high dose creatine (\\>10 grams a day) within 30 days prior to Screening Visit.\n11. Metal in the head and neck region, cardiac pacemaker or brain stimulator, cochlear implants, implanted infusion device or personal history of epilepsy.\n12. Use of amiodarone, flecainide, duloxetine, tizanidine, or clozapine.\n13. Pregnant women or women currently breastfeeding.\n14. Placement of Diaphragm Pacing System (DPS) device \\< 60 days prior to Screening Visit.\n15. Planned DPS device implantation during study participation", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Mexiletine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06286475", "title": "A Study of VRG50635 in Healthy Volunteers", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Verge Genomics", "summary": "The primary objectives of this study are to investigate the safety and tolerability of VRG50635 and to determine how VRG50635 is absorbed by the body.", "interventions": [{"type": "DRUG", "name": "VRG50635"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2024-02-29", "url": "https://clinicaltrials.gov/study/NCT06286475", "target_entities": [], "locations": [{"facility": "Lincoln Celerion Inc.,", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Must be \u2265 19 and \u2264 55 years of age at Screening.\n2. Must be willing and able to voluntarily give written informed consent by signing an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form prior to any study-related procedures being performed.\n3. Must have a body mass index \u2265 18.5 and \u2264 32 kilogram per square meter (kg/m2) and weigh \u2265 50 kg.\n4. Participants of childbearing potential are eligible to participate if they are not pregnant or breastfeeding and agree to use one highly effective and one barrier method of contraception, if sexually active, for the duration of the study through 90 days after the last study drug administration. Participants must not donate eggs for the duration of study through 90 days after the last dose of study drug.\n5. Participants capable of producing sperm must agree that they will use one barrier method of contraception and that their partners of childbearing potential will use one highly effective method of contraception for the duration of the study through 90 days after the last study drug administration. Participants must not donate sperm for the duration of study through 90 days after the last dose of study drug.\n\nExclusion Criteria:\n\n1. Have a history of clinically significant hematologic, renal, neurologic, pancreatic, gastrointestinal, hepatic, cardiovascular, psychological, pulmonary, metabolic, endocrine, immunological, allergic disease, or other major disorders, as determined by the PI.\n2. Have any surgical or medical condition that could possibly affect drug absorption (including inflammatory bowel disease, history of gastrectomy, cholecystectomy, or other gastrointestinal tract surgery except appendectomy).\n3. Have a current significant medical or psychiatric condition, as determined by the PI.\n4. Have a history of serious adverse reaction or serious hypersensitivity to any drug.\n5. Have, in the opinion of the PI, evidence of clinically significant hepatic or renal impairment including alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> the upper limit of normal (ULN) or bilirubin \\> 1.5 \u00d7 ULN. Note: Participants with Gilbert syndrome without evidence of hepatic impairment may be enrolled.\n6. Have a history or presence of an abnormal electrocardiogram (ECG), including, but not limited to, complete left bundle branch block, second- or third-degree heart block, evidence of prior myocardial infarction, or any other abnormality that is clinically significant in the PI's opinion or precludes accurate interpretation and calculations of cardiac intervals (e.g., QT, QRS).\n7. Have clinically significant abnormalities, as assessed by the PI, in laboratory tests at the screening and admission visits. Note: Participants may be rescreened at the discretion of the PI with Sponsor approval.\n8. Have abnormal blood pressure: supine systolic blood pressure \\< 90 or \\> 140 mmHg, supine diastolic blood pressure \\< 50 or \\> 90 mmHg, pulse rate \\< 40 or \\> 100 bpm, and body temperature \\< 35.4 or \\> 37.8 \u00b0C (\\< 96 or \\> 100.3 \u00b0F) at the screening and admission visits.\n9. Have a prolonged corrected QT interval using Fridericia's formula (QTcF) during the 12- lead ECG at Screening or pre-dose on Day 1 \\> 450 ms for participants assigned male at birth and \\> 470 ms for participants assigned female at birth.\n10. Have a hemoglobin level \\< 12 g/dL (participants assigned male at birth) or \\< 10.5 g/dL (participants assigned female at birth).\n11. Have participated in any other investigational drug study within 30 days of dosing or within 7 half-lives of the investigational product, whichever is longer, or have previously participated in the current study.\n12. Use any prescription medication within 7 days or 5 half-lives (whichever is longer) of the first dose administration and/or anticipated use through the follow-up visit.\n13. Use any over-the-counter medication (including vitamin/mineral supplements and herbal medicines, such as St. John's wort and non-standard herbal teas) within 7 days of the first dose administration and/or anticipate using through study participation and the follow-up visit.\n14. Receive a positive result in a SARS-CoV-2 test or a COVID-19 vaccine within 30 days prior to Screening.\n15. Have poor peripheral venous access.\n16. Have donated blood or plasma or otherwise lost \u2265 500 mL of blood within 8 weeks of Screening.\n17. Have consumed alcohol, caffeine, or cytochrome P450 (CYP)-altering foods within 48 hours prior to dosing.\n18. Have an average daily caffeine intake \\> 800 mg/day (equivalent to approximately 8 cups of coffee per day).\n19. Have a documented medical history of alcoholism or current use of more than 21 units of alcohol per week, of drug abuse, or of drug addiction in the last 2 years.\n20. Have a positive drug or alcohol test at Screening.\n21. Use tobacco or nicotine products (including tobacco- or nicotine-containing products in any form, including e-cigarettes and vaping) within 90 days before Screening or have a positive nicotine test at the screening or admission visit.\n22. Have a history of suicidal behavior or suicidal ideation within the past 60 months, as determined by a positive response (\"Yes\") to either question 4 or question 5 of the Columbia-Suicide Severity Rating Scale (C-SSRS) at Screening.\n23. Have one or more of the following laboratory test abnormalities at Screening: (a) Positive hepatitis C virus (HCV) antibodies; (b) Positive hepatitis B surface antigen (HbsAg) or hepatitis B core antibody (HbcAb); (c) Positive HIV 1/2 antibodies.\n24. Engage in strenuous activity (including exercise) for \u2265 1 hour within 48 hours of any study day.\n25. Are part of the clinical staff personnel or have family members of the clinical site staff or the Sponsor.\n26. Have any other issue which, in the opinion of the PI, will make the participant ineligible for study participation.\n27. Multiple Dose Part only: Are not eligible for lumbar puncture (have anticoagulation, antiaggregation, or blood coagulation pathologies, history of lumbar spine surgery, acquired or congenital spine malformation, clinical signs of intracranial hypertension, cutaneous infection at the puncture site).", "sex": "ALL", "min_age": "19 Years", "max_age": "55 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "VRG50635", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07292545", "title": "Video-Based Proprioceptive Exercise Program in ALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Tarsus University", "summary": "In the project, it was aimed to evaluate the proprioceptive system in the disease, which is still mysterious and therefore has no curative treatment under current conditions, and to examine the effects of the video-based proprioceptive home exercise programme on trunk and limb control and daily life activity as well as trunk and limb control. The study was planned to include 20 patients with a definite diagnosis of ALS. Proprioceptive sensory examination will be performed again in these patients, who are currently being followed up with ALS diagnosis and whose physical examination including neurological examination has been performed in detail, and the \"Revised Amyotrophic Lateral Sclerosis Functional Rating Scale\" (R-ALSFRS) will be applied to the patients. Subsequently, the patients will be followed up by applying a video-based proprioceptive home exercise programme 3 days a week for 8 weeks. At the end of the 8th week, a detailed neurological examination including proprioceptive sensation will be performed and the R-ALSFRS scale will be applied. In addition, ALS quality of life scale will be applied to the patients before and after the home programme. The data obtained after the treatment programme will be analysed and interpreted.", "interventions": [{"type": "OTHER", "name": "video based exercise"}], "start_date": "2025-07-14", "url": "https://clinicaltrials.gov/study/NCT07292545", "target_entities": ["proprioceptive_system"], "locations": [{"facility": "Tarsus University", "city": "Mersin", "state": "TARSUS", "country": "Turkey (T\u00fcrkiye)", "status": "RECRUITING", "lat": 36.81196, "lon": 34.63886}], "contact_phone": "03246000033", "contact_email": "fzt_evrim@hotmail.com", "eligibility": {"criteria": "Inclusion Criteria:Inclusion Criteria:\n\n* ALS patients aged 18-70\n* Those with a definite ALS diagnosis according to the Gold Coast criteria\n* Those who can sit independently\n* Those who have no cognitive problems\n* Those who agree to participate in the study will be included in the study.\n\nExclusion Criteria:\n\n* ALS patients with neurological, orthopedic or visual dysfunction mimicking ALS,\n* Those who cannot complete active joint movements in the upper and lower extremities,\n* ALS patients who do not agree to participate in the study will not be included in the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05797753", "title": "A Study to Assess the Effect of Erythromycin on the Test Medicine (SAR443820) When Given Orally as Tablets to Healthy Adult Male and Female Participants (Part A); and the Effect of Itraconazole on the Test Medicine (SAR443820) When Given Orally as Capsules to Healthy Adult Male Participants (Part B)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sanofi", "summary": "This is a Phase 1, cross-over, 2-part study for pharmacokinetic (PK) assessment of SAR443820 when co-administered with cytochrome P450 3A4 (CYP3A4) inhibitors (erythromycin ethyl succinate (EES) in Part A and possibly itraconazole in Part B).\n\nIn Part A, the objective is to assess the effects of repeated administration of EES as CYP3A4 inhibitor, on the PK profile of a single oral dose of SAR443820 tablet in healthy male and female participants.\n\nIn Part B, the objective is to assess the effects of repeated administration of itraconazole on the PK profile of a single oral dose of SAR443820 capsule in healthy male participants.\n\nPart A includes a screening period, Period 1 (SAR443820), a wash-out period and Period 2 (SAR443820 + EES). Part B includes a screening period, Period 1 (SAR443820), a wash-out period and Period 2 (SAR443820 + itraconazole). The washout period between single SAR443820 administration in Period 1 and the start of dosing with EES (Part A) or itraconazole (Part B) in Period 2 is at least 4 days.\n\nThe study duration is approximately 7 weeks for each Part A and Part B.\n\nThe treatment duration is:\n\n* For SAR443820 (both Part A and Part B): 1 day in each Period; single dose of SAR443830 on Period 1 (P1)-Day 1 and on Period 2 (P2)-Day 6 for each Part.\n* For EES (Part A): 9 days of treatment in Period 2 with P2-Day 1 starting at least 4 days after P1-Day 1.\n* For itraconazole (Part B): the treatment duration lasts 11 days in Period 2 and it is fixed once the results of Part A are issued, P2-Day 1 starting at least 4 days after P1-Day 1.", "interventions": [{"type": "DRUG", "name": "SAR443820"}, {"type": "DRUG", "name": "SAR443820"}, {"type": "DRUG", "name": "Erythromycin ethyl succinate"}, {"type": "DRUG", "name": "Itraconazole"}], "start_date": "2022-02-18", "url": "https://clinicaltrials.gov/study/NCT05797753", "target_entities": [], "locations": [{"facility": "Nucleus Network Site Number : 8400001", "city": "Saint Paul", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.94441, "lon": -93.09327}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Part A: male or female participant must be 18 to 55 years of age inclusive, at the time of signing the informed consent\n* Part B: male participant only must be 18 to 55 years of age inclusive, at the time of signing the informed consent\n* Body weight between 50.0 and 100.0 kg, inclusive, if male, and between 40.0 and 90.0 kg, inclusive, if female, body mass index between 18.0 and 30.0 kg/m\\^2, inclusive\n* Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies\n\nExclusion Criteria:\n\n* Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, gynecologic (if female), or infectious disease, or signs of acute illness - Any medication (including St John's Wort) within 14 days before inclusion or within 5 times the elimination half-life or pharmacodynamic half-life of the medication, with the exception of hormonal contraception or menopausal hormone replacement therapy; any non-live Covid-19 vaccine within the last 2 weeks before inclusion, any live attenuated vaccine within the last 28 days before inclusion and any other non-vaccine biological drugs given within 4 months before inclusion\n* Current enrollment in Part A (applicable for Part B) or past participation in previous clinical study on SAR443820\n* Positive result for hepatitis B, C or human immunodeficiency virus (HIV)\n* Positive result on urine drug screen\n* Positive urine alcohol test\n* Any consumption of citrus fruits (grapefruit, orange, etc) or their juices within 5 days before inclusion The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial", "sex": "ALL", "min_age": "18 Years", "max_age": "55 Years", "healthy_volunteers": false, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "SAR443820", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03457753", "title": "Riluzole Oral Soluble Film Safety and Tolerability in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Aquestive Therapeutics", "summary": "The primary objective of this study is to assess the safety and tolerability, with emphasis on the oral cavity, of ROSF (containing riluzole 50mg) in subjects with amyotrophic lateral sclerosis (ALS) administered twice daily for 12 weeks. Secondary objectives include (1) to record the subject's assessment of any difficulty taking riluzole administered as ROSF and any difficulty taking riluzole in the tablet formulation and (2) to record the relative preference, if any, of subjects and caretakers, for riluzole administered as ROSF vs. the riluzole tablet.", "interventions": [{"type": "DRUG", "name": "Riluzole Oral Soluble Film"}], "start_date": "2018-03-01", "url": "https://clinicaltrials.gov/study/NCT03457753", "target_entities": ["glutamate_excitotoxicity"], "locations": [{"facility": "Texas Neurology, P.A.", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Male or female subjects, between 18-80 years of age, inclusive.\n2. Subjects having a diagnosis of probable or definite ALS in accordance with the Revisited El-Escorial Criteria.\n3. Subjects must have no known allergy to riluzole or inactive ingredients\\* in ROSF.\n4. Subjects or subject's legally authorized representative must be willing and able to complete informed consent/assent and HIPAA authorization.\n5. Ability to comprehend and be informed of the nature of the study, as assessed by the Primary or Sub-Investigator.\n6. Subjects prescribed to take riluzole at or before the time of first dose. (The study is open to subjects currently taking riluzole at screening, subjects who are not currently taking riluzole at screening but who have taken riluzole in the past, and subjects to be newly started on riluzole (given as ROSF in the course of this study).\n7. Availability to volunteer for the entire study duration and willing to adhere to all protocol requirements.\n8. Female subjects of childbearing potential must have a negative urine pregnancy test at Screening and Visit 1-3. Female subjects of childbearing potential (i.e. not surgically sterile, not 2 years postmenopausal, or not with a sterile partner) must have a negative pregnancy test at screening and Visit 1-3, agree to abstinence, practicing double barrier contraception or using an FDA approved barrier method contraceptive (e.g., licensed hormonal or barrier methods) for greater than 2 months prior to screening visit and commit to an acceptable form of birth control for the duration of the study and for 30 days after participation in the study.\n9. Subjects, in the judgment of the investigator, must be suitable candidates for administration of ROSF (riluzole oral soluble film).\n\nExclusion Criteria:\n\n1. Subjects with a history of clinically significant liver disease, renal disease, or any other medical condition judged to be exclusionary by the investigator.\n2. Subjects who are unwilling to sign informed consent or subjects who for any other reason in the judgment of investigator are unable to complete the study.\n3. Female subjects who have a positive urine pregnancy test (\u03b2hCG) at screening or visit 1, are trying to become pregnant or are breastfeeding.\n4. Subjects with active cancer within the previous 2 years, except treated basal cell carcinoma of the skin.\n5. Subjects who have taken any experimental drug within 30 days prior to enrollment or within 5 half-lives of the investigational drug -whichever is the longer period. However, subjects who have previously completed other Aquestive sponsored ROSF clinical studies within the last 30 days prior to enrollment may be eligible for consideration for entry into this study.\n6. Subjects with known history or presence of moderate or severe renal impairment as defined by a calculated creatinine clearance of \u226450 mL/minute.\n7. Subjects currently taking riluzole with alanine aminotransferase (ALT) levels greater than 5 times upper limit of normal or with evidence of clinical jaundice. (Riluzole should be discontinued in these patients.)\n8. Subjects who will be receiving riluzole for the first time who exhibit baseline elevations of several liver function tests (especially elevated bilirubin). (These findings at baseline should preclude the use of riluzole including ROSF.)\n9. Use of potentially hepatotoxic drugs: (e.g., allopurinol, methyldopa, sulfasalazine).\n10. Subjects with clinically significant abnormal laboratory values in the judgment of the investigator.\n11. Use of strong or moderate CYP1A2 inhibitors (e.g., ciprofloxacin, enoxacin, fluvoxamine, methoxsalen, mexiletine, thiabendazole, vemurafenib, zileuton) and CYP1A2 inducers (e.g. rifampin and barbiturates) in the previous 30 days before first drug administration.\n12. Employee or immediate relative of an employee of the investigator, MonoSol Rx LLC, any of its affiliates or partners, or inVentiv Health.\n13. Anything else that, in the opinion of the investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Riluzole Oral Soluble Film", "targeting_mechanism": "Riluzole blocks glutamatergic neurotransmission by acting as a sodium channel blocker on presynaptic neurons to decrease glutamate release and increase glutamate reuptake, reducing glutamate excitotoxicity.", "targeting_mechanism_pmid": "37296644", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07204977", "title": "Acamprosate in C9orf72 Hexanucleotide Repeat Expansion Amyotrophic Lateral Sclerosis (ACALS)", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)", "summary": "Background:\n\nAmyotrophic lateral sclerosis (ALS) is a disorder that damages nerve cells in the brain and spinal cord. It can cause muscle weakness, paralysis, and loss of movement. The symptoms grow worse over time. Half of all people with ALS live only 3 to 5 years after diagnosis. Current drug treatments can slow the progress of the disease, but they cannot stop or reverse it.\n\nObjective:\n\nTo test a study drug (acamprosate) in people with ALS with a mutation in the C9orf72 gene.\n\nEligibility:\n\nPeople aged 18 years and older with ALS. They must have a mutation in the C9orf72 gene.\n\nDesign:\n\nParticipants will have 13 visits over 32 weeks. Five visits will be at the clinic, and 8 visits will be by phone.\n\nParticipants will have a baseline visit of up to 3 days. They will have a physical exam with blood tests. They will have imaging scans and tests of their breathing ability. Their memory, thinking, and behavior will be assessed. They will have a neurologic exam to check their reflexes, strength, balance, eyes, and coordination. They will complete questionnaires about their daily life. They will have a lumbar puncture to collect fluid from the area around the spinal cord.\n\nAcamprosate is a pill taken by mouth. Participants will take 2 pills by mouth 3 times a day with meals for 24 weeks. They will record their doses and any missed doses in a diary.\n\nBaseline tests will be repeated during follow-up clinic visits. These tests may be spread out over 3 days.\n\nDuring phone visits, participants will talk about how they are doing. They will review their diary with researchers.", "interventions": [{"type": "DRUG", "name": "Acamprosate calcium"}], "start_date": "2026-02-02", "url": "https://clinicaltrials.gov/study/NCT07204977", "target_entities": ["C9orf72"], "locations": [{"facility": "National Institutes of Health Clinical Center", "city": "Bethesda", "state": "Maryland", "country": "United States", "status": "", "lat": 38.98067, "lon": -77.10026}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "* INCLUSION CRITERIA:\n\nIn order to be eligible to participate in this study, an individual must meet all of the following inclusion criteria:\n\n1. Aged 18 years or older at the time of the screening visit.\n2. Able to provide informed consent and comply with study procedures and availability for the duration of the study. If a participant lacks capacity to give consent, a Legally Authorized Representative (LAR) will be authorized to give consent on behalf of the individual.\n3. Diagnosis of ALS (possible, probable, or definite according to the World Federation of Neurology El Escorial revised criteria or the Gold Coast Criteria) with or without mild cognitive impairment, mild behavioral impairment, or frontotemporal dementia (FTD).\n4. CLIA-certified genetic testing showing a pathogenic hexanucleotide repeat expansion in the C9orf72 gene.\n5. Symptom duration less than 2 years, or if greater than 2 years, disease progression at a rate that, in the judgement of the investigator, would allow for completion of the study.\n6. If taking riluzole, edaravone, or phenylbutyrate/TUDCA, the participant must be on a stable dose for at least 30 days prior to the screening visit, or have stopped taking riluzole, edaravone, or phenylbutyrate/TUDCA at least 30 days prior to the baseline visit.\n7. Participant must be competent to self-administer the medication as deemed by study team. Alternatively, participant must have a competent caregiver who can and will be responsible for administering the study drug. If there is no caregiver, another qualified individual must be available to administer the study drug.\n8. Participant has established care with a neurologist and will maintain this clinical care throughout the study.\n9. For females of reproductive potential: use of effective or highly effective contraception for at least one month prior to screening and agreement to use such a method during study participation and for an additional 8 weeks after the end of acamprosate administration. Participants of childbearing potential must have a negative pregnancy test at screening.\n10. For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner.\n\nEXCLUSION CRITERIA:\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n1. Dependence on daytime mechanical ventilation (invasive or non-invasive, including Continuous Positive Airway Pressure (CPAP) or Bilevel Positive Airway Pressure (BiPAP) at the time of the screening visit.\n2. Participation in any other investigational drug trial or using investigational drug(s) (within 4 weeks prior to the Day 0 visit and thereafter).\n3. Participants must not become pregnant or breastfeed for the duration of the study. Participants of childbearing potential must have a negative pregnancy test at screening and be non-lactating.\n4. Men who are trying to become fathers or donate sperm.\n5. History of positive test or positive result at screening for HIV.\n6. History of severe sulfite allergy (i.e., anaphylaxis).\n7. Presence of any of the following clinical conditions at the time of screening:\n\n   1. Active drug abuse or alcoholism.\n   2. Unstable medical condition that, in the opinion of the investigators, makes participation unsafe.\n   3. Renal impairment estimated glomerular filtration rate \\<=60mL/min/1.73m\\^2.\n   4. Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days of the screening visit.\n8. Safety Laboratory Criteria at the screening visit:\n\n   1. Estimated glomerular filtration rate \\<=60mL/min/1.73m\\^2.\n   2. Platelet concentration of \\<100,000/microl.\n   3. PT and PTT \\>1.2 times the upper limit of normal.\n   4. Hemoglobin \\<10mg/dL.\n   5. Positive Hepatitis B Surface Antigen and Hepatitis C Virus Antibody\n9. Participants who are unable to swallow tablets whole, as required by the administration guidelines. This includes individuals with any medical condition or physical limitation that impedes their ability to swallow solid dosage forms.\n10. Participants who are dependent on a gastrostomy tube for medication administration. Since acamprosate is in tablet form that cannot be crushed or altered, it is unsuitable for administration through a gastrostomy tube.", "sex": "ALL", "min_age": "18 Years", "max_age": "99 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Acamprosate calcium", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03196375", "title": "A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Flex Pharma, Inc.", "summary": "The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease \\[including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy (PMA)\\] experiencing muscle cramps. Participants will be asked to take two study products during the course of the study. One of these study products will be a placebo.\n\nApproximately 120 participants in approximately 30 study centers across the United States are expected to take part. Participants will be in the study for approximately 3 months and visit the study clinic 3 times.", "interventions": [{"type": "DRUG", "name": "FLX-787-ODT (orally disintegrating tablet)"}, {"type": "DRUG", "name": "Placebo ODT"}], "start_date": "2017-07-28", "url": "https://clinicaltrials.gov/study/NCT03196375", "target_entities": [], "locations": [{"facility": "Honor Health Research Institute", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "Mayo Clinic", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "California Pacific Medical Center", "city": "Sacramento", "state": "California", "country": "United States", "status": "", "lat": 38.58157, "lon": -121.4944}, {"facility": "University of California - Davis", "city": "Sacramento", "state": "California", "country": "United States", "status": "", "lat": 38.58157, "lon": -121.4944}, {"facility": "University of California San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "GW Medical Faculty Associates Inc.", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida Health", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Indiana University Neuroscience Center", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Lahey Hospital and Medical Center", "city": "Burlington", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.50482, "lon": -71.19561}, {"facility": "University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Saint Louis University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Guilford Neurologic Associates", "city": "Greensboro", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.07264, "lon": -79.79198}, {"facility": "Wake Forest University", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Providence Brain and Spine Institute", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Oregon Health Sciences University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburgh", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Medical University of South Carolina", "city": "Charleston", "state": "South Carolina", "country": "United States", "status": "", "lat": 32.77632, "lon": -79.93275}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Baylor Scott and White Health", "city": "Round Rock", "state": "Texas", "country": "United States", "status": "", "lat": 30.50826, "lon": -97.6789}, {"facility": "UT Health San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "The University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Vermont Medical Center", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "Virginia Commonwealth University", "city": "Richmond", "state": "Virginia", "country": "United States", "status": "", "lat": 37.55376, "lon": -77.46026}, {"facility": "Saint Luke's Rehabilitation Institute", "city": "Spokane", "state": "Washington", "country": "United States", "status": "", "lat": 47.65966, "lon": -117.42908}, {"facility": "Medical College of Wisconsin", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Documented diagnosis of Motor Neuron Disease (MND) \\[including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy (PMA)\\]\n* Expected survival \\> 6 months\n* Weekly muscle cramping (defined as: a sustained muscle contraction that's most often painful and lasts seconds to minutes)\n\nExclusion Criteria:\n\n* Presence of major gastrointestinal disorders, such as inflammatory bowel disease, diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or esophageal lesions/ulcers\n* Presence of laryngospasm or significant swallowing problems\n* Presence of percutaneous endoscopic gastrostomy, esophagogastroduodenoscopy, or G-tube\n* Unable or unwilling to discontinue medications for cramps and/or opiates\n* Inability to tolerate a spicy sensation in the mouth or stomach\n* Actively using illicit drugs or history of chronic substance abuse within the past year prior to screening, including abuse of alcohol\n* Intention to change the current level of tobacco use or use of nicotine-containing products (i.e., new smokers or those actively trying to quit may not enrolled)\n* Participated in a clinical study (except natural history studies without administration of an investigational product) within 30 days prior to screening", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "FLX-787-ODT", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07656714", "title": "HFCWO Vest Plus Mechanical Cough Assist Versus Cough Assist Alone on Respiratory Infections in ALS: a Pilot Trial", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hospital Universitario Virgen Macarena", "summary": "This pilot randomised single-blind trial evaluated whether adding high-frequency chest wall oscillation (HFCWO) via chest vest (ComfortCough II) to mechanical cough assist improves respiratory outcomes and reduces respiratory infections and hospital admissions compared to mechanical cough assist alone in patients with amyotrophic lateral sclerosis (ALS) receiving non-invasive ventilation.", "interventions": [{"type": "DEVICE", "name": "High-frequency chest wall oscillation (HFCWO) vest"}, {"type": "DEVICE", "name": "Mechanical cough assist"}], "start_date": "2021-04-15", "url": "https://clinicaltrials.gov/study/NCT07656714", "target_entities": [], "locations": [{"facility": "Hospital Universitario Virgen Macarena", "city": "Seville", "state": "Andalusia", "country": "Spain", "status": "", "lat": 37.38283, "lon": -5.97317}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Confirmed diagnosis of ALS according to the Revised El Escorial criteria\n2. Under follow-up at the Multidisciplinary ALS Unit of Hospital Universitario Virgen Macarena\n3. Age between 18 and 90 years\n4. Peak cough flow less than 270 L/min or forced vital capacity (FVC) less than 80% of predicted\n5. Ability to travel to the Neurolab centre\n\nexclusion criteria:\n\n1. Need for invasive mechanical ventilation\n2. Total dependence on non-invasive ventilation (NIV)\n3. Severe bulbar involvement\n4. Cognitive impairment\n5. Comorbidities incompatible with the intervention: pregnancy, rib fractures, known risk of pneumothorax or pneumomediastinum, cerebrospinal fluid fistula, pneumocephalus, or recent barotrauma", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05407324", "title": "Dazucorilant in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Corcept Therapeutics", "summary": "The purpose of this 2-part study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Dazucorilant 300 mg"}, {"type": "DRUG", "name": "Dazucorilant 150 mg"}, {"type": "OTHER", "name": "Placebo"}, {"type": "DRUG", "name": "Dazucorilant"}], "start_date": "2022-11-15", "url": "https://clinicaltrials.gov/study/NCT05407324", "target_entities": ["Glucocorticoid receptor"], "locations": [{"facility": "062", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "RECRUITING", "lat": 33.44838, "lon": -112.07404}, {"facility": "278", "city": "San Francisco", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.77493, "lon": -122.41942}, {"facility": "287", "city": "Neptune City", "state": "New Jersey", "country": "United States", "status": "RECRUITING", "lat": 40.20011, "lon": -74.02792}, {"facility": "353", "city": "New York", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 40.71427, "lon": -74.00597}, {"facility": "108", "city": "Leuven", "state": "", "country": "Belgium", "status": "ACTIVE_NOT_RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "425", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "ACTIVE_NOT_RECRUITING", "lat": 43.25011, "lon": -79.84963}, {"facility": "273", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "ACTIVE_NOT_RECRUITING", "lat": 45.50884, "lon": -73.58781}, {"facility": "422", "city": "Bron", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 45.73865, "lon": 4.91303}, {"facility": "258", "city": "Lille", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 50.63391, "lon": 3.05512}, {"facility": "257", "city": "Limoges", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 45.83362, "lon": 1.24759}, {"facility": "261", "city": "Marseille", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 43.29695, "lon": 5.38107}, {"facility": "423", "city": "Montpellier", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 43.61093, "lon": 3.87635}, {"facility": "259", "city": "Nice", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 43.70313, "lon": 7.26608}, {"facility": "262", "city": "Paris", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 48.85341, "lon": 2.3488}, {"facility": "256", "city": "Tours", "state": "", "country": "France", "status": "ACTIVE_NOT_RECRUITING", "lat": 47.39484, "lon": 0.70398}, {"facility": "255", "city": "Berlin", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 52.52437, "lon": 13.41053}, {"facility": "270", "city": "Bonn", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 50.73438, "lon": 7.09549}, {"facility": "268", "city": "Dresden", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 51.05089, "lon": 13.73832}, {"facility": "260", "city": "Hanover", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 52.37052, "lon": 9.73322}, {"facility": "265", "city": "Jena", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 50.92878, "lon": 11.5899}, {"facility": "386", "city": "M\u00fcnchen", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 51.60698, "lon": 13.31243}, {"facility": "267", "city": "Rostock", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 54.0887, "lon": 12.14049}, {"facility": "269", "city": "Ulm", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "253", "city": "Dublin", "state": "", "country": "Ireland", "status": "ACTIVE_NOT_RECRUITING", "lat": 53.33306, "lon": -6.24889}, {"facility": "264", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "283", "city": "Bydgoszcz", "state": "", "country": "Poland", "status": "ACTIVE_NOT_RECRUITING", "lat": 53.1235, "lon": 18.00762}, {"facility": "385", "city": "Krakow", "state": "", "country": "Poland", "status": "ACTIVE_NOT_RECRUITING", "lat": 50.06143, "lon": 19.93658}, {"facility": "254", "city": "Warsaw", "state": "", "country": "Poland", "status": "ACTIVE_NOT_RECRUITING", "lat": 52.22977, "lon": 21.01178}, {"facility": "274", "city": "Warsaw", "state": "", "country": "Poland", "status": "ACTIVE_NOT_RECRUITING", "lat": 52.22977, "lon": 21.01178}, {"facility": "302", "city": "Barcelona", "state": "", "country": "Spain", "status": "ACTIVE_NOT_RECRUITING", "lat": 41.38879, "lon": 2.15899}, {"facility": "115", "city": "Barcelona", "state": "", "country": "Spain", "status": "ACTIVE_NOT_RECRUITING", "lat": 41.38879, "lon": 2.15899}, {"facility": "303", "city": "Madrid", "state": "", "country": "Spain", "status": "ACTIVE_NOT_RECRUITING", "lat": 40.4165, "lon": -3.70256}, {"facility": "282", "city": "M\u00e1laga", "state": "", "country": "Spain", "status": "ACTIVE_NOT_RECRUITING", "lat": 36.72016, "lon": -4.42034}, {"facility": "194", "city": "Valencia", "state": "", "country": "Spain", "status": "ACTIVE_NOT_RECRUITING", "lat": 39.47391, "lon": -0.37966}, {"facility": "263", "city": "Stoke-on-Trent", "state": "", "country": "United Kingdom", "status": "ACTIVE_NOT_RECRUITING", "lat": 53.00415, "lon": -2.18538}], "contact_phone": "(650) 249-9965", "contact_email": "study652@corcept.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male and female patients \u226518 years of age with sporadic or familial ALS. In Part 1, patients must have a risk of ALS progression characterized by a European Network for the Cure of ALS (ENCALS) risk profile score \u2265 -6 and \u2264 -3. In Part 2 patients must have a risk of ALS progression characterized by an Treatment Research Initiative to Cure ALS (TRICALS) risk profile score \u2265 -7 and \u2264 -3.\n* If taking riluzole, edaravone, and/or sodium phenylbutyrate and taurursodiol, must be on a stable dose prior to Screening. Sodium phenylbutyrate and taurursodiol are not permitted for patients enrolled in Part 2 of the study.\n* Part 2 only: Patients with a pathogenic mutation in superoxide dismutase 1 gene (SOD1) must not be receiving treatment with tofersen or eligible for treatment with tofersen if available. Patients who have received prior treatment with tofersen and discontinued due to safety and/or efficacy reasons prior to Screening are eligible.\n* Part 2 only: Use of ultra high-dose methylcobalamin for the treatment of ALS is permitted provided the patient has been on a stable dose for \u226511 weeks prior to the Day 1 visit.\n\nExclusion Criteria:\n\n* History of a clinically significant non-ALS neurologic disorder\n* Inability to swallow capsules.\n* Blood platelet count \\<150,000/mm\\^3.\n* Renal impairment indicated by Estimated Glomerular Filtration Rate (eGFR) \u226430 mL/min/1.73 m\\^2. Part 2 only: Patients with a recent history of acute kidney injury should have returned to their baseline renal function (i.e, eGFR prior to acute kidney injury) prior to enrollment.\n* Human immunodeficiency virus (HIV) or current chronic/active infection with hepatitis C virus or hepatitis B virus. Part 2 only: Known history of HIV or chronic/active infection with hepatitis C or hepatitis B virus; testing does not need to be performed if infection status is unknown.\n* Women who are pregnant, planning to become pregnant, or are breastfeeding.\n* Use of non-invasive ventilation (NIV) or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation.\n* Cancer that is currently being treated (except adequately controlled basal cell carcinoma or squamous cell carcinoma of the skin, stage I endometrial cancer or carcinoma in situ of the cervix or breast) or a history of cancer with an expected survival \\< 2 years.\n* Current or anticipated need of a diaphragm pacing system (DPS).\n* Previous exposure or treatment with glucocorticoid receptor modulators or antagonists.\n* Taking, or have taken, any systemic, inhaled, or potent dermatologic topical corticosteroids (Class I to III) within a period equivalent to 5 half-lives of the corticosteroid used prior to first dose of study drug. Patients who have stopped glucocorticoid use should have an alternative option if their condition deteriorates during the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Dazucorilant", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02881476", "title": "Therapeutic Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Warmia and Mazury", "summary": "The goal of this study is to investigate the safety and tolerability of allogeneic Wharton's jelly-derived mesenchymal stem cells administration in the individuals with diagnosed amyotrophic lateral sclerosis.", "interventions": [{"type": "OTHER", "name": "Biological: Cell-based therapy"}], "start_date": "2015-11", "url": "https://clinicaltrials.gov/study/NCT02881476", "target_entities": ["neuroprotection"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)\n* good understanding of the protocol and willingness to consent\n* signed informed consent\n* disease duration: up to 2 years\n* FVC \\> 50% / pulmonologist certificate about respiratory function of the patient\n\nExclusion Criteria:\n\n* cancer,\n* autoimmune diseases\n* renal failure,\n* subject is a respiratory dependent.\n* subject unwilling or unable to comply with the requirements of the protocol\n* pregnancy, breastfeeding", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "allogeneic Wharton's jelly-derived mesenchymal stem cells", "targeting_mechanism": "Cell-based therapy that targets multiple disease pathways by supporting motor neuron survival through production of growth factors and anti-inflammatory cytokines.", "targeting_mechanism_pmid": "34890069", "animal_results": "Preclinical stem cell studies have been performed in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1) and demonstrated potential benefit of stem cell therapy for ALS.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03150290", "title": "Brown Adipose Tissue in ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Institut National de la Sant\u00e9 Et de la Recherche M\u00e9dicale, France", "summary": "Weight loss is a common phenomenon in ALS. During the course of the disease, difficulty in swallowing and mastication can be responsible for a decrease in caloric intake and thus for weight loss. However, significant weight loss can also be observed in patients with no feeding difficulties. About half of ALS patients have an increase in their resting energy consumption, but the origin of this \"hypermetabolism\" remains unknown. \"Brown\" fat is specialized in the production of heat. Unlike \"white\" fat that stores excess caloric intakes, brown fat consumes energy. In humans, brown fat has long been considered as absent in adults. However, recent imaging techniques have been able to detect brown fat deposits in some adult subjects. The aim of this study is thus to determine the role of brown fat on energy consumption in Amyotrophic Lateral Sclerosis.", "interventions": [{"type": "OTHER", "name": "Indirect Calorimetry."}, {"type": "DEVICE", "name": "18-FDG-PET"}], "start_date": "2017-10-26", "url": "https://clinicaltrials.gov/study/NCT03150290", "target_entities": [], "locations": [{"facility": "Paris Als Center, Salpetriere Hospital", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "10 ALS patients, fulfilling the following inclusion criteria:\n\n* Aged 18 to 85 (inclusive)\n* Possible, probable (clinically or laboratory) or definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria\n* With unexplained significant loss of weight of 5 percent (or more) of normal body weight in the last 6 months (n=5 subjects) Or - without weight loss (n= 5 subjects)", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03792490", "title": "Inhibition of Rho Kinase (ROCK) With Fasudil as Disease-modifying Treatment for ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Medical Center Goettingen", "summary": "Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder and therapeutic options are limited. The rho kinase (ROCK) inhibitor Fasudil was shown to be neuroprotective, induced axonal regeneration and improved survival and behavioral outcome in models of ALS and other neurodegenerative diseases. The aim of this phase IIa, multi-center and double-blind study is to analyze the safety, tolerability and efficacy of fasudil in two different doses compared to placebo in approximately 16 trial sites in Germany, France and Switzerland. Intravenous application of fasudil will be performed in 80 patients and placebo in 40 patients two times daily for 20 treatment days. The hypothesis is that fasudil is safe and well-tolerated and its application will significantly improve the clinical outcome in patients with ALS.", "interventions": [{"type": "DRUG", "name": "Fasudil"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2019-02-20", "url": "https://clinicaltrials.gov/study/NCT03792490", "target_entities": ["ROCK"], "locations": [{"facility": "Centre Hospitalier Universitaire Marseille", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Centre Hospitalier Universitaire Montpellier", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Centre Hospitalier Universitaire Nice", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Centre Hospitalier Universitaire Tours", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Charit\u00e9 Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Universit\u00e4tsklinikum Carl Gustav Carus Dresden", "city": "Dresden", "state": "", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "University Medical Center G\u00f6ttingen", "city": "G\u00f6ttingen", "state": "", "country": "Germany", "status": "", "lat": 51.53443, "lon": 9.93228}, {"facility": "Universit\u00e4tsklinikum Halle (Saale)", "city": "Halle", "state": "", "country": "Germany", "status": "", "lat": 51.48158, "lon": 11.97947}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4tsklinikum Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Universit\u00e4tsklinikum Leipzig", "city": "Leipzig", "state": "", "country": "Germany", "status": "", "lat": 51.33962, "lon": 12.37129}, {"facility": "Klinikum rechts der Isar der Technischen Universit\u00e4t M\u00fcnchen", "city": "M\u00fcnchen", "state": "", "country": "Germany", "status": "", "lat": 51.60698, "lon": 13.31243}, {"facility": "Universit\u00e4tsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "University of W\u00fcrzburg", "city": "W\u00fcrzburg", "state": "", "country": "Germany", "status": "", "lat": 49.79391, "lon": 9.95121}, {"facility": "Kantonsspital St. Gallen", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Probable (clinically or laboratory) or definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria\n* Disease duration more than 6 months and less than 24 months (inclusive). Disease onset defined as date of first muscle weakness, excluding fasciculations and cramps\n* Vital capacity more than 65% of normal (slow vital capacity; best of three measurements)\n* Age: \u2265 18 years\n* Patients have to be treated with Riluzole (2 x 50mg/d), must be stable for at least four weeks before randomization\n* Patients who have started on Edaravone therapy shall continue Edaravone treatment. Edaravone treatment must not be discontinued for reasons of trial participation.\n* Women of childbearing age must be non-lactating and surgically sterile or using a highly effective method of birth control and have a negative pregnancy test. Acceptable methods of birth control with a low failure rate i.e. less than 1% per year) when used consistently and correct are such as implants, injectables, combined oral contraceptives, hormonal intrauterine devices (IUDs), sexual abstinence or vasectomized partner\n* Capable of thoroughly understanding all information given and giving full informed consent according to good clinical practice (GCP)\n* Patients have to have a valid health insurance, when recruited in a center in France\n\nExclusion Criteria:\n\n* Previous participation in another clinical study involving trial medication within the preceding 12 weeks or five terminal half times of the longest to be eliminated trial medications (whichever is longer) or previous participation in this trial\n* Tracheostomy or continuous assisted ventilation of any type during the preceding three months before randomization or a significant pulmonary disorder not attributed to ALS, which may complicate the evaluation of respiratory function, intermittent non-invasive ventilation is permitted,\n* Patients with a history of intracranial bleeding, known intracerebral aneurysms or Moyamoya disease, or positive family history for the above. If only family history positive, magnetic resonance (MR)- or x-ray-based cranial imaging not older than 24 months must confirm absence of bleeding, aneurysms or Moyamoya.\n* Gastrostomy\n* Any medical condition known to have an association with motor neuron dysfunction or involving neuromuscular weakness or another neurodegenerative disease, e.g. Parkinson's disease (PD) or Alzheimer's disease (AD), which might confound or obscure the diagnosis of ALS\n* Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment\n* Patients with known arterial hypotension (resting blood pressure \\<90/60 mmHg) or previous hypotensive episodes or requiring treatment for increasing of blood pressure, such as fludrocortisone, midodrine, etilefrine, cafedrine or theodrenaline\n* Patients with an uncontrollable or unstable arterial hypertensive disease (resting blood pressure \\>180 mmHg systolic and/or \\>120 mmHg diastolic under current antihypertensive medication)\n* Known pulmonary hypertension and any medication prescribed for treatment of pulmonary hypertension\n* Confirmed hepatic insufficiency or abnormal liver function (stable aspartate transaminase (ASAT) and/or alanine aminotransferase (ALAT) greater than 3 times the upper limit of the normal range) and determined to be non-transient through repeat testing\n* Renal insufficiency with a glomerular filtration rate (GFR) \\<60 ml/min/1,73m\u00b2 (calculated by Modification of Diet in Renal Disease (MDRD) equation) and determined to be non-transient through repeat testing\n* Major psychiatric disorder, significant cognitive impairment or clinically evident dementia precluding evaluation of symptoms\n* Hypersensitivity to any component of the study drug\n* Liable to be not cooperative or comply with the trial requirements (as assessed by the investigator), or unable to be reached in the case of emergency\n* Pregnant or breast-feeding females or females with childbearing potential, if no adequate contraceptive measures are used\n* Prisoners or subjects who are involuntary incarcerated\n* Patients subject to legal protection measures", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Fasudil", "targeting_mechanism": "Rho kinase (ROCK) inhibitor that induces neuroprotection and axonal regeneration.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04569084", "title": "Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS) based on the change in ALS Functional Rating Scale- Revised (ALSFRS-R) score from baseline up to Week 48:", "interventions": [{"type": "DRUG", "name": "MT-1186"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2020-11-13", "url": "https://clinicaltrials.gov/study/NCT04569084", "target_entities": ["oxidative_stress"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center (SJHMC)", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "HonorHealth Neurology", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "Woodland Research Northwest", "city": "Rogers", "state": "Arkansas", "country": "United States", "status": "", "lat": 36.33202, "lon": -94.11854}, {"facility": "UCSD Medical Center", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Loma Linda University Health Care - Department of Neurology", "city": "Loma Linda", "state": "California", "country": "United States", "status": "", "lat": 34.04835, "lon": -117.26115}, {"facility": "University California Los Angeles Medical Center (UCLA)", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Irvine (UCI) Health - Women's Healthcare Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "University of Colorado Anschutz Medical Campus", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "UF Health Cancer Center", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida (USF) - Carol and Frank Morsani Center for Advanced Health Care (CAHC)", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University - School of Medicine", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern University Feinberg School of Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Ochsner Center for Primary Care and Wellness", "city": "Jefferson", "state": "Louisiana", "country": "United States", "status": "", "lat": 29.96604, "lon": -90.15313}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Lahey Hospital", "city": "Burlington", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.50482, "lon": -71.19561}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Neurology Associates, P.C. - Lincoln", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Las Vegas Clinic", "city": "Las Vegas", "state": "Nevada", "country": "United States", "status": "", "lat": 36.17497, "lon": -115.13722}, {"facility": "Dent Neurologic Institute", "city": "Amherst", "state": "New York", "country": "United States", "status": "", "lat": 42.97839, "lon": -78.79976}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Thomas Jefferson University, Jefferson Weinberg ALS Center", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Lewis Katz School of Medicine at Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University Of Pittsburgh Medical Center", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Wesley Neurology Clinic, P.C.", "city": "Cordova", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.15565, "lon": -89.7762}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Nerve And Muscle Center Of Texas", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "The University of Vermont (UVM) and UVM Medical Center National ALS Center of Excellence", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "Sentara Neurology Specialists", "city": "Virginia Beach", "state": "Virginia", "country": "United States", "status": "", "lat": 36.85293, "lon": -75.97799}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "St. Luke's Rehabilitation Institute", "city": "Spokane", "state": "Washington", "country": "United States", "status": "", "lat": 47.65966, "lon": -117.42908}, {"facility": "West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic", "city": "Morgantown", "state": "West Virginia", "country": "United States", "status": "", "lat": 39.62953, "lon": -79.9559}, {"facility": "Medical College of Wisconsin", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Regional Health Authority B", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "Health Science Center Mcmaster University", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "London Health Sciences Centre - University Hospital", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Odette Cancer Center-Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Recherche Sepmus, Inc", "city": "Greenfield Park", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.48649, "lon": -73.46223}, {"facility": "Centre Hospitalier De L'Universite De Montreal (Chum) Notre-Dame Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute And Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "CHU de Quebec-Hopital-Enfant-Jesus", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Saskatoon City Hospital", "city": "Saskatoon", "state": "Saskatchewan", "country": "Canada", "status": "", "lat": 52.13238, "lon": -106.66892}, {"facility": "Universitaetsklinikum Wuerzburg", "city": "Wuezburg", "state": "Germany", "country": "Germany", "status": "", "lat": null, "lon": null}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "UKRUB - Berufsgenossenschaftliches Universitatsklinikum Bergmannsheil GmbH - Medizinische Klinik III", "city": "Bochum", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Charite Campus Virchow", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Universit\u00e4tsklinik Bonn-Motoneuronambulanz, Klinik und Poliklinik f\u00fcr Neurodegenerative Erkrankungen und Gerontopsychiatrie", "city": "Bonn", "state": "", "country": "Germany", "status": "", "lat": 50.73438, "lon": 7.09549}, {"facility": "Georg-August-Universitaet Goettingen - Universitaetsmedizin Goettingen (UMG)", "city": "G\u00f6ttingen", "state": "", "country": "Germany", "status": "", "lat": 51.53443, "lon": 9.93228}, {"facility": "Universitaetsklinikum Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Klinikum Rechts der Isar der Technischen Universitaet Muenchen", "city": "M\u00fcnchen", "state": "", "country": "Germany", "status": "", "lat": 51.60698, "lon": 13.31243}, {"facility": "University Medical Center Rostock", "city": "Rostock", "state": "", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Universitaets- und Rehabilitationskliniken Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Deutsche Klinik fuer Diagnostik", "city": "Wiesbaden", "state": "", "country": "Germany", "status": "", "lat": 50.08601, "lon": 8.24435}, {"facility": "Universita degli Studi di Torino - Centro Regionale Esperto Per La Sclerosi Laterale Amiotrofica (CRESLA)", "city": "Turin", "state": "Piedmont", "country": "Italy", "status": "", "lat": 45.07049, "lon": 7.68682}, {"facility": "Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca Granda - Centro Clinico Nemo (Neuro Muscular Omnicentre)", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Istituto Nazionale Neurologico Carlo Besta", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Istituto Auxologico Italiano - Istituto Di Ricovero e Cura a Carattere Scientifico - Istituto Scientifico Ospedale San Luca", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Centro SLA di Palermo", "city": "Palermo", "state": "", "country": "Italy", "status": "", "lat": 38.1166, "lon": 13.3636}, {"facility": "Policlinico A. Gemelli", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}, {"facility": "National Hospital Organization Higashinagoya National Hospital", "city": "Meito-ku, Nagoya-shi", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "Nagoya University Hospital", "city": "Showa-ku, Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "National Hospital Organization Chibahigashi National Hospital", "city": "Chuo-ku, Chiba-shi", "state": "Chiba", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "Murakami Karindoh Hospital", "city": "Nishi-ku, Fukuoka-shi", "state": "Fukuoka", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Fukushima Medical University Hospital", "city": "Fukushima", "state": "Fukushima", "country": "Japan", "status": "", "lat": 37.75, "lon": 140.46667}, {"facility": "Hiroshima University Hospital", "city": "Minami-ku, Hiroshima-shi", "state": "Hiroshima", "country": "Japan", "status": "", "lat": 34.4, "lon": 132.45}, {"facility": "National Hospital Organization Hokkaido Medical Center", "city": "Sapporo", "state": "Hokkaido", "country": "Japan", "status": "", "lat": 43.06667, "lon": 141.35}, {"facility": "National Hospital Organization Iou National Hospital", "city": "Kanazawa", "state": "Ishikawa-ken", "country": "Japan", "status": "", "lat": 36.6, "lon": 136.61667}, {"facility": "Kagawa University Hospital", "city": "Miki-cho, Kita-gun", "state": "Kagawa-ken", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Yokohama City University Hospital", "city": "Kanazawa-ku, Yokohama-shi", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.43333, "lon": 139.65}, {"facility": "Kitasato University Hospital", "city": "Minami-ku, Sagamihara-city", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.56707, "lon": 139.24167}, {"facility": "National Hospital Organization Kumamoto Saishun Medical Center", "city": "Koshi-shi", "state": "Kumamoto", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "National Hospital Organization Utano National Hospital", "city": "Ukyo-ku, Kyoto City", "state": "Kyoto", "country": "Japan", "status": "", "lat": 35.02107, "lon": 135.75385}, {"facility": "Tohoku University Hospital", "city": "Sendai", "state": "Miyagi", "country": "Japan", "status": "", "lat": 38.26667, "lon": 140.86667}, {"facility": "Niigata University Medical & Dental Hospital", "city": "Asahimachidori, Chuo-ku, Niigata-shi", "state": "Niigata", "country": "Japan", "status": "", "lat": 37.92259, "lon": 139.04125}, {"facility": "Kansai Electric Power Hospital", "city": "Fukushima-ku, Osaka-shi", "state": "Osaka", "country": "Japan", "status": "", "lat": 34.69379, "lon": 135.50107}, {"facility": "National Hospital Organization Osaka Toneyama Medical Center", "city": "Toyonaka-shi", "state": "Osaka", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Saitama Neuropsychiatric Institute", "city": "Chuo-ku, Saitama-shi", "state": "Saitama", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Shiga University of Medical Science Hospital", "city": "\u014ctsu", "state": "Shiga", "country": "Japan", "status": "", "lat": 35.0, "lon": 135.86667}, {"facility": "National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders", "city": "Aoi-ku, Shizuoka-shi", "state": "Shizuoka", "country": "Japan", "status": "", "lat": 34.98333, "lon": 138.38333}, {"facility": "Juntendo University Hospital", "city": "Bunkyo-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Tokyo Metropolitan Neurological Hospital", "city": "Fuch\u016b", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.67452, "lon": 139.48216}, {"facility": "Teikyo University Hospital", "city": "Itabashi-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Toho University Omori Medical Center", "city": "\u014cta-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.56126, "lon": 139.71605}, {"facility": "Keio University Hospital", "city": "Shinjuku-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Chiba University Hospital", "city": "Chiba", "state": "", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "Seoul National University Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Hanyang University Medical Center", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Samsung Medical Center", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "University hospital Bern (Inselspital)", "city": "Bern", "state": "Canton of Bern", "country": "Switzerland", "status": "", "lat": 46.94809, "lon": 7.44744}, {"facility": "Hopitaux Universitaires de Geneve (HUG) (Hopital Cantonal)", "city": "Geneva", "state": "", "country": "Switzerland", "status": "", "lat": 46.20222, "lon": 6.14569}, {"facility": "Neurocenter of Southern Switzerland", "city": "Lugano", "state": "", "country": "Switzerland", "status": "", "lat": 46.01008, "lon": 8.96004}, {"facility": "Zentrumsleiter Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Subjects must provide a signed and dated informed consent form (ICF) to participate in the study. Subjects must be able (in the judgment of the Investigator) to understand the nature of the study and all risks involved with participation in the study. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.\n2. Subjects will be male or female, \u2265 18 to 75 years of age at the time the ICF is signed.\n3. Subjects will be diagnosed with Definite ALS or Probable ALS according to the El Escorial revised criteria for the diagnosis of ALS.\n4. Subjects with a baseline score \u2265 2 points on each individual item of the ALSFRS- R at screening and baseline visits.\n5. Subjects have a screening and baseline %forced vital capacity (FVC) \u2265 70%.\n6. Subjects with 1- to 4-point decline for 8 weeks (\u00b17 days) in ALSFRS-R total score between screening and baseline visits.\n7. Subjects whose first symptom of ALS has occurred within 2 years of providing written informed consent.\n\nExclusion Criteria:\n\nExclusions Related to Primary Diagnosis\n\n1. Subjects with a history of spinal surgery after the onset of ALS, such as surgery for cervical spondylosis or a herniated disc, or plans for such surgery during the study period.\n\n   Exclusions Related to Other Neurological Disorders (including, but not limited to the following)\n2. Subjects with the possibility that the current symptoms may be symptoms of a disease requiring differential diagnosis, such as cervical spondylosis and multifocal motor neuropathy, cannot be ruled out.\n\n   Exclusions Related to General Health or Concomitant Conditions\n3. Subjects undergoing treatment for a malignancy.\n4. Subjects with a complication that could have a significant effect on efficacy evaluations, such as Parkinson's disease or syndrome, schizophrenia, bipolar disorder, and dementia.\n5. Subjects who have the presence or history of any clinically significant (CS) disease (except ALS) that could interfere with the objectives of the study (the assessment of safety and efficacy) or the safety of the subject, as judged by the Investigator.\n6. Subjects who are female, of childbearing potential, and pregnant (a positive pregnancy test) or lactating at the screening visit (Visit 1).\n7. Subjects of childbearing potential unwilling to use acceptable method of contraception from the screening visit until 3 months after the last dose of study medication. Subjects who are sexually active who do not agree to use contraception during the study period.\n8. Subjects who have a significant risk of suicidality. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a specific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the Columbia-Suicide Severity Rating Scale (C-SSRS) within the 3 months before the screening visit.\n9. Subjects who have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) elevations greater than 2 times the upper limit of normal (ULN) at screening.\n10. Subjects with a Glomerular Filtration Rate (GFR) \\< 30 mL/Min Per 1.73 m2 at screening, using the Larsson Equation.\n\n    Exclusions Related to Medications\n11. Subjects with history of hypersensitivity to edaravone, any of the additives or inactive ingredients of edaravone, or sulfites.\n12. Subjects with hereditary problems of fructose intolerance (eg, fructose, sucrose, invert sugar, and sorbitol).\n13. Subjects who participated in another study and were administered an investigational product within 1 month or 5 half-lives of the investigational agent, whichever is longer, before providing informed consent for the present study.\n14. Subjects who have received any previous treatment with edaravone.\n15. Subjects who have received stem cell therapy.\n16. Subjects who are unable to take their medications orally at baseline (Visit 2).", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MT-1186 (oral edaravone)", "targeting_mechanism": "Antioxidant that reduces oxidative stress and reactive oxygen species.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05370079", "title": "Control Cohort CTRL COH", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hospices Civils de Lyon", "summary": "Autoimmune encephalitis (AE) and paraneoplastic neurological syndromes (PNS) are rare disease that could be difficult to diagnose. So it necessary to obtain numerous sample from different disease to develop more specific diagnosis kit It could be possible through the characterisation of new genetic biomarkers.", "interventions": [{"type": "BIOLOGICAL", "name": "Collection of biological sample (blood and/or CSF)"}], "start_date": "2023-08-08", "url": "https://clinicaltrials.gov/study/NCT05370079", "target_entities": [], "locations": [{"facility": "Hospices Civils de Lyon", "city": "Bron", "state": "", "country": "France", "status": "RECRUITING", "lat": 45.73865, "lon": 4.91303}], "contact_phone": "(33) 4 72 35 78 06", "contact_email": "jerome.honnorat@chu-lyon.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* patient with consent\n* patient with following disease: Parkinson's disease, Amyotrophic lateral sclerosis, Glioblastoma, cancer without neurological disease (pulmonary cancer, breast cancer, ovarian cancer, melanoma, thymoma), rheumatoid arthritis.\n\nExclusion Criteria:\n\n* refusal consent\n* patient with neurological disorder compatible with paraneoplastic neurological syndrome or auto-immune encephalitis\n* patient under guardianship", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00403104", "title": "Placebo Controlled Study of ONO2506PO in the Presence of Riluzole in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ono Pharmaceutical Co., Ltd.", "summary": "The purpose of this study is to determine if oral treatment with ONO-2506PO in patients diagnosed with ALS, who have had onset of muscle weakness within 14 months of randomization, could lead to the slowing of decline in respiratory function, functional status, muscle strength, quality of life and survival compared with placebo group.", "interventions": [{"type": "DRUG", "name": "ONO-2506PO"}, {"type": "DRUG", "name": "ONO-2506PO"}], "start_date": "2006-11", "url": "https://clinicaltrials.gov/study/NCT00403104", "target_entities": ["astrocyte_activation"], "locations": [{"facility": "L. Boltzmann Forschungsinstitut, Neurologische Abteilung, Kaiser Franz Josef Hospital, Wien", "city": "Vienna", "state": "", "country": "Austria", "status": "", "lat": 48.20849, "lon": 16.37208}, {"facility": "UCL Saint-Luc", "city": "Brussels", "state": "", "country": "Belgium", "status": "", "lat": 50.85045, "lon": 4.34878}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Hopital Roger Salengro - Clinique Neurologique, Neurologie A", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Hopital Duruytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Hopital de la Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Hopital de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Hopital l-Archet 1", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Hopital LaPitie Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Charite Campus Virchow, ALS Ambulanz", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Neurologische Universitatsklinik Bergmannsheil", "city": "Bochum", "state": "", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Poliklinik der Universitat Erlangen-Nurnberg, Neurologische Klinik", "city": "Erlangen", "state": "", "country": "Germany", "status": "", "lat": 49.59099, "lon": 11.00783}, {"facility": "Martin-Luther-Universitat Halle-Wittenberg, Klinikum der Medizinischen Fakultat, Universitatsklinik und Poliklinik fur Neurologie", "city": "Halle", "state": "", "country": "Germany", "status": "", "lat": 51.48158, "lon": 11.97947}, {"facility": "Medizinische Hochschule Hannover, Neurologische Klinik", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Interdisziplinares Zentrum fur Palliativmedizin", "city": "M\u00fcnchen", "state": "", "country": "Germany", "status": "", "lat": 51.60698, "lon": 13.31243}, {"facility": "Klinik und Poliklinik fur Neurologie der Universitat Ulm-Universitatsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Deutsche Klinik fur Diagnostik, Fachbereich Neurologie", "city": "Wiesbaden", "state": "", "country": "Germany", "status": "", "lat": 50.08601, "lon": 8.24435}, {"facility": "Dipartimento di Neurologia e Laboratorio di Neuroscienze - Universita di Milano - IRCCS - Istituto Auxologico Italiano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Divisione di Neuroriabilitazione II - Fondazione Salvatore Maugeri - IRCCS", "city": "Pavia", "state": "", "country": "Italy", "status": "", "lat": 45.19205, "lon": 9.15917}, {"facility": "Dipartimento di Neuroscienze - Divisione di Neurologia II - Azienda Ospedaliera S. Giovanni Battista - Molinette", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Academic Medical Centre (AMC) Amsterdam - Dept of Neurology", "city": "Amsterdam", "state": "", "country": "Netherlands", "status": "", "lat": 52.37403, "lon": 4.88969}, {"facility": "University Medial Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Kantonsspital St. Gallen, Muskelzentrum/ALS Clinic", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "Academic Neuroscience Centre", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Royal Preston Hospital", "city": "Preston", "state": "", "country": "United Kingdom", "status": "", "lat": 53.76282, "lon": -2.70452}, {"facility": "University of Sheffield - Academic Neurology Unit", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Diagnosis of clinically possible, clinically probable laboratory-supported, clinically probable or clinically definite ALS (according to WNF EL Escorial diagnostic criteria, revised according to the Airlie House Conference 1998)\n2. Onset of muscle weakness within 14 months randomization\n3. Concomitant standard Riluzole therapy (50mg twice daily)\n\nExclusion Criteria:\n\n1. Presence of a tracheotomy, mechanical ventilation or non-invasive ventilation\n2. Requirement for prescription drugs used for potential neuroprotective benefit -", "sex": "ALL", "min_age": "18 Years", "max_age": "74 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ONO-2506PO", "targeting_mechanism": "ONO-2506PO targets astrocyte activation and dysfunction, modulating glial support for neuronal function in ALS.", "targeting_mechanism_pmid": "28641533", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06169176", "title": "RAPA-501 Therapy of ALS Expanded Access Protocol", "phase": "Expanded Access", "status": "TEMPORARILY_NOT_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Rapa Therapeutics LLC", "summary": "RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem cells in patients living with amyotrophic lateral sclerosis (pwALS).", "interventions": [{"type": "OTHER", "name": "RAPA-501 Autologous T stem cells"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT06169176", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Mayo Clinic Hospital Phoenix", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "University of California Irvine Health", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "University of California, San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "University of Iowa Health Care", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Hackensack University Medical Center", "city": "Hackensack", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.88593, "lon": -74.04347}, {"facility": "Providence Portland Medical Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Male or female patients \u2265 18 years of age.\n2. Patients with sporadic or familial ALS diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria.\n3. Pulmonary slow vital capacity (SVC) \\< 50% of predicted normal (as measured within three months prior to screening or at the time of screening; inability to measure an SVC value at the time of screening that is due to severe reduction in respiratory function will also fulfill this eligibility criterion #3). However, SVC values \u226550% are acceptable if an EAP recipient of RAPA-501 is re-enrolled to the study.\n4. Must have a source of autologous T cells potentially sufficient to manufacture RAPA-501 cells, as defined by a peripheral CD3+ T cell count \u2265 500 cells per \u03bcl.\n5. Patients who are taking riluzole (Rilutek\u00ae), edaravone (Radicava\u00ae), and/or sodium phenylbutyrate/taurursodial (Relyvrio\u2122) are eligible if taking the drug for at least 30 days prior to the screening visit.\n6. Patients must be \u2265 two (2) weeks removed from major surgery, or investigational therapy.\n7. Patients must have no ongoing, unstable serious illness other than ALS, as determined by the Site Investigator.\n8. Serum creatinine less than or equal to 2.0 mg/dL.\n9. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \u2264 3 x upper limit of normal (ULN).\n10. Bilirubin \u2264 1.5 (except if due to Gilbert's disease).\n11. No history of abnormal bleeding tendency.\n12. Voluntary written consent must be given before performance of any study related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to future care.\n13. Not enrolled in another interventional clinical trial or expanded access protocol and must have stopped taking other experimental drug(s) at least 2 weeks prior to screening.\n\nExclusion Criteria:\n\n1. Active uncontrolled infection.\n2. Hypertension not adequately controlled by \u2264 3 medications.\n3. History of documented pulmonary embolus within 6 months of enrollment.\n4. Clinically significant cardiac pathology, as defined by: myocardial infarction within 6 months prior to enrollment, Class III or IV heart failure according to NYHA, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.\n5. HIV, hepatitis B, or hepatitis C seropositive.\n6. Pregnant or breastfeeding subjects.\n7. Subjects of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception.\n8. Subjects may be excluded at the Principal Investigator discretion or if it is deemed that allowing participation would represent an unacceptable medical or psychiatric risk.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RAPA-501 autologous hybrid TREG/Th2 T stem cells", "targeting_mechanism": "RAPA-501 modulates neuroinflammation through immunotherapy targeting regulatory T cells and Th2 responses to mitigate motor neuron degeneration in ALS.", "targeting_mechanism_pmid": "35303907", "animal_results": "ASC-Exosomes ameliorated disease progression in SOD1(G93A) murine ALS model with increased lifespan of 17 days, delayed decline in motor performance and weight loss.", "animal_results_pmid": "32455791", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02450552", "title": "Clinical Trial of Ezogabine (Retigabine) in ALS Subjects", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Brian Wainger", "summary": "This study evaluates the effect of retigabine (600 mg/day, 900 mg/day, or placebo) on motor neuron activity in people with Amyotrophic Lateral Sclerosis (ALS). The total study duration is approximately 14 weeks. ALS subjects will take study drug for approximately 10 weeks.", "interventions": [{"type": "DRUG", "name": "Ezogabine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2015-06", "url": "https://clinicaltrials.gov/study/NCT02450552", "target_entities": ["KCNQ channels"], "locations": [{"facility": "Barrow Neuological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Cedars-Sinai", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "UC Irvine Medical Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Mayo Clinic in Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Augusta University (Georgia Regents Medical Center)", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Beth Israel Deaconess Medical Center", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Duke University Hospital", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Penn State College of Medicine Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "ALS Subject Inclusion Criteria:\n\n* Male or female, aged 18 to 80.\n* Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.\n* Slow vital capacity (SVC) measure \u2265 50% of predicted for gender, height and age at the Screening Visit,OR in the opinion of the SI, ability to perform and safely complete all study visit procedures.\n* Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days prior to the Screening Visit and continue on the stable dose throughout the course of the study (riluzole-na\u00efve subjects are permitted in the study).\n* Subjects must be able to swallow oral medication at the Screening Visit and expected to be able to swallow tablets throughout the course of the study.\n* Capable of providing informed consent and following trial procedures.\n* Geographically accessible to the site.\n* Women must not be able to become pregnant (e.g., post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal contraception, for example patch or contraceptive ring), intrauterine device (IUD) in place for \u2265 3 months, barrier method in conjunction with spermicide, or another adequate method.\n* Use of medications known to affect the neurophysiology measures in the study must be scheduled, not as needed (pro re nata, PRN). A subject must have been on a fixed dose for 30 days prior to the Screening Visit, and there must be no reason to believe that a subsequent change would be necessary during the course of the study. These medications include: benzodiazepines, muscle relaxants, tricyclic antidepressants, selective serotonin reuptake inhibitors, non-selective serotonin reuptake inhibitors, hypnotics (including anti-histamines) and anti-cholinergics.\n* TMS shows sufficient MEP amplitude and/or NCS studies show sufficient CMAP amplitude.\n\nALS Subject Exclusion Criteria:\n\n* Medical condition, laboratory finding, or physical exam finding that precludes participation.\n* Serum AST and ALT value \\>2.0 times the upper normal limit\n* Clinically significant conduction abnormalities on electrocardiogram or a known history of cardiac arrhythmia, myocardial infarction within the past 24 months, or congestive heart failure.\n* Estimated glomerular filtration rate \\< 50 mL/min at Screening Visit.\n* Concomitant digoxin treatment.\n* Known allergic reactions to components of the study product(s).\n* Exposure to any other agent currently under investigation for the treatment of patients with ALS (off-label use or investigational) within 30 days of the Screening Visit including ezogabine, exposure to cell replacement therapy within six months of the Screening Visit or any prior intraparenchymal cell replacement injection within the spinal cord or brain at anytime in the past.\n* Presence of tracheostomy at the Screening Visit.\n* History of clinically significant urinary retention, , or current use of medications to treat urinary retention.\n* History of drug and or alcohol abuse within 12 months of the Screening Visit.\n* The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to SI judgment.\n* Clinically significant history of unstable or severe cardiac, oncologic, hepatic, or renal disease, or other uncontrolled medical condition.\n* Presence of feeding tube.\n* Current use of antipsychotic, antiepileptic (except benzodiazepines, gabapentin, pregabalin) or class 1 (e.g. flecainide) or class 3 (e.g. amiodarone) antiarrhythmic medications. Quinidine or a quinidine-containing drug is allowed if the quinidine dose is not greater than 20 mg/day (for a full list of medications, please reference the study MOP).\n* Inability to perform either TMS or NCS studies due to insufficient MEP or CMAP amplitude.\n* Pregnant women or women currently breastfeeding.\n* Contraindication to TMS studies including ferromagnetic metal in the head or neck (potentially found in aneurysm clips, implanted medication pumps, implanted brain stimulators, pacemakers, cochlear implants), or history of epilepsy. Dental fillings are permitted.\n* Anything else that, in the opinion of the SI, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.\n\nHealthy Control Subject Inclusion Criteria:\n\n* Male or female, aged 18 to 80.\n* Absence of a known neurological disorder.\n* Capable of providing informed consent and following trial procedures.\n* Geographically accessible to the site.\n* Age (+/- 10 years and site-matched to a ALS participant within 6 months of their Baseline visit).\\[Matched controls only\\]\n* TMS shows sufficient MEP amplitude and/or NCS studies show sufficient CMAP amplitude (amplitudes defined in MOP).\n* Use of medications known to affect the neurophysiology measures in the study must be scheduled, not as needed (pro re nata, PRN). A subject must have been on a fixed dose for 30 days prior to the Screening Visit, and there must be no reason to believe that a subsequent change would be necessary during the course of the study. These medications include: benzodiazepines, muscle relaxants, tricyclic antidepressants, selective serotonin reuptake inhibitors, non-selective serotonin reuptake inhibitors, hypnotics (including anti-histamines) and anti-cholinergics.\n\nHealthy Control Subject Exclusion Criteria:\n\n* History of ALS or other neurodegenerative disease.\n* Presence of positive family history of ALS.\n* Current use of an antipsychotic or antiarrhythmic medication\n* Definitely or possibly pregnant.\n* Contraindication to TMS studies including ferromagnetic metal in the head or neck ( potentially found in aneurysm clips, implanted medication pumps, implanted brain stimulators, pacemakers, cochlear implants), or history of epilepsy. Dental fillings are permitted.\n* Anything that, in the opinion of the SI, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Ezogabine (Retigabine)", "targeting_mechanism": "Ezogabine is a potassium channel opener that enhances motor neuron excitability and activity in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01984814", "title": "Stem Cell Therapy for Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neurogen Brain and Spine Institute", "summary": "The effect of autologous bone marrow mononuclear cells on duration of survival in Amyotrophic Lateral Sclerosis patients.", "interventions": [{"type": "BIOLOGICAL", "name": "Stem cell"}], "start_date": "2008-12", "url": "https://clinicaltrials.gov/study/NCT01984814", "target_entities": ["neuroprotection"], "locations": [{"facility": "Neurogen brain and spine institute", "city": "Mumbai", "state": "Maharashtra", "country": "India", "status": "", "lat": 19.07283, "lon": 72.88261}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with the diagnoses of definite amyotrophic lateral sclerosis.\n* Clear information about date of onset of symptoms, documented contact information and follow up information\n\nExclusion Criteria:\n\n* Patients with diagnoses of Progressive Lateral sclerosis, Progressive bulbar plasy, Progressive spinal muscular atrophy, Progressive muscular atrophy, monomelicamyotrophy or madras motor neuron disease.\n* patients with co-morbidities like presence of acute infections such as Human Immunodeficiency Virus/Hepatitis B Virus/Hepatitis C Virus, malignancies, bleeding tendencies, renal failure, severe liver dysfunction and other acute medical conditions such as respiratory infection and pyrexia.", "sex": "ALL", "min_age": "26 Years", "max_age": "76 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous bone marrow mononuclear cells", "targeting_mechanism": "Stem cell transplantation provides neuroprotection and motor neuron support through secretion of trophic factors and modulation of inflammatory responses in ALS.", "targeting_mechanism_pmid": "36064599", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02383654", "title": "Compassionate Treatment : An Exploratory Clinical Trial to Assess Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "China Medical University Hospital", "summary": "The purpose of the study is to determine the safety and possible effectiveness of Autologous Adipose-Tissue Derived Stem Cells treatment in a Amyotrophic Lateral Sclerosis(ALS) patient.", "interventions": [{"type": "PROCEDURE", "name": "Autologous Adipose-Tissue Derived Stem Cells"}], "start_date": "2015-01", "url": "https://clinicaltrials.gov/study/NCT02383654", "target_entities": ["neuroprotection"], "locations": [{"facility": "China Medical University Beigang Hospital", "city": "Beigang", "state": "Yunlin", "country": "Taiwan", "status": "", "lat": 23.5701, "lon": 120.30162}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Compassionate Treatment\n\nThe number of participants for a person.\n\nThe patients (Guo XX) of 28% of lung function assessment (normal lung function was greater than 80%), the daily life of motor function score was 17 points (normal function score of 40 points).", "sex": "MALE", "min_age": "35 Years", "max_age": "50 Years", "healthy_volunteers": false, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "Autologous Adipose-Tissue Derived Stem Cells", "targeting_mechanism": "Adipose-derived stem cells differentiate into supportive cells (astrocytes, oligodendrocytes, microglia) that produce growth factors and anti-inflammatory cytokines to protect degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Stem cell therapy has demonstrated potential benefit in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1).", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06556394", "title": "A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ractigen Therapeutics.", "summary": "This is a Phase 1, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects with Amyotrophic Lateral Sclerosis (ALS) with Superoxide Dismutase Type 1 (SOD1) Gene Mutation", "interventions": [{"type": "DRUG", "name": "RAG-17"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2024-12-24", "url": "https://clinicaltrials.gov/study/NCT06556394", "target_entities": ["SOD1"], "locations": [{"facility": "Beijing Tiantan Hospital", "city": "Beijing", "state": "", "country": "China", "status": "RECRUITING", "lat": 39.9075, "lon": 116.39723}, {"facility": "West China Hospital of Sichuan University", "city": "Chengdu", "state": "", "country": "China", "status": "RECRUITING", "lat": 30.66667, "lon": 104.06667}, {"facility": "The Second Affiliated Hospital Zhejiang University School of Medicine", "city": "Hangzhou", "state": "", "country": "China", "status": "RECRUITING", "lat": 30.29365, "lon": 120.16142}], "contact_phone": "+86 18051622388", "contact_email": "lilc@ractigen.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Voluntarily consents to participate in this study and provides written informed consent prior to the start of any study specific procedures.\n2. \u2265 18 years of age at the time of informed consent.\n3. Diagnosis of possible, laboratory supported probable, probable, or definite ALS according to the World Federation of Neurology El Escorial.\n4. Documented SOD1 mutation.\n5. Forced vital capacity (FVC) \u226550% of predicted value as adjusted for sex, age, and height (measured seated).\n6. If taking riluzole or edaravone, subject must be on a stable dose or \u226530 days prior to Day 1 and expected to remain at that dose until the final study visit.\n\nExclusion Criteria:\n\n1. Documented p.F21C SOD1 mutation.\n2. Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.\n3. Current enrollment in any other interventional study.\n4. History of or positive test result for human immunodeficiency virus, hepatitis C virus antibody or hepatitis B virus.\n5. Pregnant or currently breastfeeding.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RAG-17", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01492686", "title": "Phase 3 Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "The primary objective of the study is to confirm the efficacy of 60 mg of MCI-186 via intravenous drip infusion once a day in the patients with ALS based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24 weeks administration in double-blind, placebo-controlled manner. The study is also to examine the safety of MCI-186 to the ALS patients.", "interventions": [{"type": "DRUG", "name": "MCI-186"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "MCI-186 in open label phase"}], "start_date": "2011-12-31", "url": "https://clinicaltrials.gov/study/NCT01492686", "target_entities": ["oxidative_stress"], "locations": [{"facility": "", "city": "Osaka", "state": "", "country": "Japan", "status": "", "lat": 34.69379, "lon": 135.50107}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients whose conditions are defined as \"definite ALS\"or \"probable ALS\"diagnostic criteria El Escorial and revised Airlie House.\n* Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life.\n* Patients of less than 2 years after the onset of ALS.\n* Patients whose progress of the condition during 12 weeks before administration meet other requirements.\n\nExclusion Criteria:\n\n* Patients with such complications as Parkinson's disease, schizophrenia, dementia, renal failure, or other severe complication, and patients who have the anamnesis of hypersensitivity to edaravone.\n* Pregnant, lactating, and probably pregnant patients, and patients who want to become pregnant, and patients who can not agree to contraception.\n* Patients who have participated in other trials within 12 weeks before consent, or who are participating in other clinical trials at present.\n* In addition to the above exclusion criteria, patients judged to be inadequate to participate in this study by their physician.", "sex": "ALL", "min_age": "20 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MCI-186", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02880033", "title": "Oxidative Stress and Apoptosis of Energy Metabolism by Deferiprone From the Circulating Lymphocytes", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Lille", "summary": "Peripheral blood mononuclear cells (PBMC) and platelets could be interesting ex vivo models to study brain diseases. Indeed, there is no access to neurons from patients. However, PBMC can exhibit different physiopathological mechanisms that are ubiquitous (i.e. oxidative stress, mitochondriopathy with energy metabolism, inflammation, protein folding, iron metabolism and programmed cell death ...). The platelets are pivotal in the healing system with large range of growth factors. A new therapeutic concept of conservative iron chelation with deferiprone for neuroprotection is under development.\n\nThe action of deferiprone on the different mechanisms and notably the oxidative stress are to obtain from a collection of PBMC and platelets from patient having Parkinson's disease and Amyotrophic lateral sclerosis and healthy controls to study ex vivo.\n\nPBMC and platelets will be stored for future analyses.", "interventions": [{"type": "DRUG", "name": "deferiprone"}, {"type": "DRUG", "name": "placebo"}], "start_date": "2011-02", "url": "https://clinicaltrials.gov/study/NCT02880033", "target_entities": ["oxidative_stress", "iron_metabolism"], "locations": [{"facility": "H\u00f4pital Roger Salengro, CHRU de Lille", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Parkinson's disease according to Movement Disorders Society criteria\n* Amyotrophic Lateral Sclerosis according to El escorial criteria\n* Age and sex matched healthy controls\n\nExclusion Criteria:\n\n* Severe comorbidities (cancer, other degenerative diseases, hemopathy, inflammatory diseases)", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "deferiprone", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06551649", "title": "Human Amniotic Mesenchymal Cell Secretome for Neurodegeneration and Neuroinflammation", "phase": "NA", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fondazione Policlinico Universitario Agostino Gemelli IRCCS", "summary": "Neurodegenerative diseases are debilitating conditions characterized by chronic inflammation, leading to dysfunction of both the non-neuronal cellular components of the central nervous system and peripheral blood immune cells. Thus, it is crucial to develop an innovative therapeutic strategy that not only effectively contrast neurodegeneration but also aims to reduce inflammation.\n\nThe overall aim of the study is to provide a preclinical in vitro demonstration of the immunomodulatory and pro-regenerative potential of the human amniotic mesenchymal stromal cell (hAMSC) secretome in counteracting neurodegeneration.\n\nThis potential will be evaluated in three-dimensional in vitro models of neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS) and chronic demyelinating disease (multiple sclerosis - MS). To this end, the study includes sample collection from patients without pharmacological treatment and without medical devices. Patients diagnosed with ALS, patients diagnosed with MS, and healthy volunteers will be recruited to collect blood samples and skin biopsies. Patient-specific and control organoid platforms, mimicking cellular heterogeneity and tridimensional interactions within the central nervous system including the inflammatory compartment, will be developed to be used as a valuable tool to investigate the in vitro efficacy of the hAMSC secretome.", "interventions": [{"type": "OTHER", "name": "Venous blood draw and skin biopsy"}], "start_date": "2025-01-17", "url": "https://clinicaltrials.gov/study/NCT06551649", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Fondazione Policlinico Universitario A. Gemelli IRCCS", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}, {"facility": "Fondazione Policlinico Universitario Agostino Gemelli IRCCS", "city": "Rome", "state": "", "country": "Italy", "status": "", "lat": 41.89193, "lon": 12.51133}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* For patients with amyotrophic lateral sclerosis (ALS): aged between 50 and 60 years and similar age of onset and duration of the disease.\n* For patients with multiple sclerosis (MS): with recently confirmed diagnosis of MS, aged between 20 and 50 years and considering the male-to-female ratio in MS of approximately 2:1.\n* For healthy volunteers: spouses of patients unaffected by any neurological disease and matching their age and gender ratio for both conditions.\n\nExclusion Criteria:\n\n* Patients who do not consent to participate in the study.\n* MS patients who have received treatment with immunomodulators or corticosteroids and are in an acute phase of the disease.", "sex": "ALL", "min_age": "20 Years", "max_age": "60 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Reducing neuroinflammation and modulating immune responses to protect against neurodegeneration.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01536249", "title": "Dexpramipexole and Cimetidine Drug Drug Interaction (DDI)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "This study will assess the effect of cimetidine on the pharmacokinetics (PK) of dexpramipexole in healthy volunteer and evaluate the safety and tolerability of dexpramipexole when given with or without cimetidine. Additionally, this study will explore the influence of genetic variation on the PK of dexpramipexole when given with or without cimetidine.", "interventions": [{"type": "DRUG", "name": "Dexpramipexole"}, {"type": "DRUG", "name": "Cimetidine plus Dexpramipexole"}], "start_date": "2012-03", "url": "https://clinicaltrials.gov/study/NCT01536249", "target_entities": ["mitochondrial_complex_i"], "locations": [{"facility": "Research Site", "city": "Overland Park", "state": "Kansas", "country": "United States", "status": "", "lat": 38.98223, "lon": -94.67079}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subjects who, in the opinion of the Investigator, are healthy as determined by medical history, physical examination, and 12 lead ECG\n* Adult males/females aged 18 to 55 years inclusive\n* Male and female subjects of childbearing potential must practice effective contraception during the study and up to 90 days after their last dose.\n\nExclusion Criteria:\n\n* History of malignant disease, including solid tumors and hematologic malignancies.\n* History of clinically significant endocrine, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, and renal, or other major diseases, as determined by the Investigator.\n* Treatment with prescription medication and/or over-the-counter products and herbal-containing and/or alternative health preparations and procedures.\n* Surgery within 90 days prior to check-in.", "sex": "ALL", "min_age": "18 Years", "max_age": "55 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "Dexpramipexole", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04745299", "title": "Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R\u00ae Inj.) Treatment in Patients With ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Corestemchemon, Inc.", "summary": "ALSUMMIT is a double-blind, randomized, placebo-controlled, multi-center, parallel, phase III clinical trial to evaluate and confirm the efficacy and long-term safety of repeated Lenzumestrocel (Neuronata-R\u00ae inj.) treatment.", "interventions": [{"type": "BIOLOGICAL", "name": "Lenzumestrocel"}, {"type": "DRUG", "name": "Riluzole"}, {"type": "DRUG", "name": "Placebo Comparator"}], "start_date": "2021-02-24", "url": "https://clinicaltrials.gov/study/NCT04745299", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Pusan National University Yangsan Hospital", "city": "Yangsan", "state": "Kyungsangnam-do", "country": "South Korea", "status": "", "lat": 35.34199, "lon": 129.03358}, {"facility": "Hanyang university hospital", "city": "Seoul", "state": "Seoul", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Korea University Anam Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Samsung Medical Center", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Seoul National University Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "\\[Inclusion Criteria\\]\n\n1. Subjects who show both upper motor neuron signs and lower motor neuron signs at the same time in neurological tests.\n2. Among subjects diagnosed with familial or sporadic ALS, subjects falling into clinically definite ALS, probable ALS and probable ALS-lab supported according to The revised World Federation of Neurology El Escorial Criteria\\[Rix Brooks, 2000\\], during 17-weeks period prior to the administration and ALSFRS-R score (progression rate) of 1.03 \u00b1 50%/month (meaning mean value in that total period).\n3. For subjects who are under Riluzole treatment, those who have received stable dose of Riluzole for more than 28 days before screening visit.\n4. Subjects with duration of disease of no more than 2 years from the first diagnosis date.\n5. Subjects whose ALSFRS-R scores are in the range of 31\\~46 at the time of screening (P-V0).\n\n\\[Exclusion Criteria\\]\n\n1. Subjects who received tracheostomy or use ventilators (including positive pressure ventilators; subjects who use non-invasive ventilation for sleep apnea may be allowed after review) at the time of screening (P-V0).\n2. Subjects who received gastrotomy at the time of screening (P-V0).\n3. Subjects for whom clinical efficacy evaluation is not possible because pulmonary functional tests cannot be conducted at the time of screening (P-V0) or subjects whose forced vital capacity is found to be not greater than 40%of the expected value.\n4. Subjects who fall into above Class II according to the New York Heart Association's functional classification, who have showed myocardial infarction, unstable arrhythmia and/or other significant cardiovascular diseases such as unstable angina in the past 3 months, or who show electrocardiographic signs of myocardial infarction or angina at the time of screening (P-V0) or who received stent insertion or coronary artery bypass grafting.\n5. Subjects who have received other investigational products or edaravone within 3 month or 5 half-lives at the time of screening (P-V0) and lead in period visit L-V1 (evaluated by whichever is longer).\n6. Subjects who have experienced epileptic seizure.\n7. Subjects with severe renal disorder (serum creatinine: not less than 2.0 mg/dL).\n8. Subjects with severe hepatic disorder (ALT, AST, or bilirubin: over 2.0 times of the normal upper limit).\n9. Subjects who show hemorrhagic tendency at the time of screening (PT and aPTT \\> 1.5 x ULN)\n10. Subjects who are found to have active viral infections (HBsAg, HCV Ab, HIV Ab, CMV IgM, EBV IgM, HSV IgM and Treponema pallidum) at the time of screening.\n11. Subjects with hypersensitivity to antibiotics (penicillin or streptomycin).\n12. Subjects who have ever received any cell therapy product for the same disease.\n13. Subjects with any malignant tumor in the past 5 years before screening, except malignant tumors with very low risk of metastasis or death.", "sex": "ALL", "min_age": "25 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Lenzumestrocel", "targeting_mechanism": "Mesenchymal stromal cell therapy that secretes neurotrophic factors and anti-inflammatory cytokines to support degenerating motor neurons.", "targeting_mechanism_pmid": "34890069", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06910384", "title": "A Study of STRO4 in Patients Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "New England Cell Therapeutics, Inc.", "summary": "This study intends to evaluate the safety and efficacy of STR04 administered intravenously in participants with Amyotrophic Lateral Sclerosis.", "interventions": [{"type": "BIOLOGICAL", "name": "STR04"}], "start_date": "2025-08-12", "url": "https://clinicaltrials.gov/study/NCT06910384", "target_entities": [], "locations": [{"facility": "National Neuromuscular Research Institute", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion:\n\n* \"Definite\" or \"Probable\" ALS according to the Revised El Escorial criteria (Airlie House)\n* ALS severity grade 1 or 2 according to the Japan ALS severity classification.\n* ALSFRS-R scores of 2 or more for all items, with scores of 4 for all breathing- related items (i.e., dyspnea, orthopnea, respiratory insufficiency).\n* Within 2 years of ALS onset.\n* %FVC of 80% or more.\n* Aged 18 to \u226475 years at the time of informed consent.\n\nExclusion:\n\n* Current Viral Infection (e.g. HBV, HCV, HIV, HTLV-1, HPVB19, syphilis, COVID- 19).\n* Current low blood cell counts\n* History of cancer, congenital malformations, or chromosomal abnormalities\n* History of allergy to penicillin or streptomycin, or other serious allergies.\n* Current poor medical condition, due to endocrine disease, metabolic disease, immune disease, blood disease, psychiatric disorders, neurological diseases, cardiovascular diseases, respiratory diseases, gastrointestinal diseases, musculoskeletal or connective tissue diseases, renal or urogenital diseases.\n* History of intracranial lesions, or stenosis, dissection or severe atherosclerotic disease of a blood vessel in the head or neck.\n* Current uncontrolled hypertension.\n* Prior treatment with a cell or gene therapy.\n* Currently participating in any other clinical trial.\n* Women who are pregnant, breastfeeding, or plan to become pregnant during study participation\n* Men with plan for their partner to become pregnant during study participation.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "STR04", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06215755", "title": "A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Verge Genomics", "summary": "The primary purpose of this study is to evaluate the safety and tolerability of VRG50635 in participants with ALS.", "interventions": [{"type": "DRUG", "name": "VRG50635"}], "start_date": "2024-01-15", "url": "https://clinicaltrials.gov/study/NCT06215755", "target_entities": [], "locations": [{"facility": "UZ Leuven", "city": "Leuven", "state": "Flemish Brabant", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Stan Cassidy Centre for Rehabilitation (Horizon NB)", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "The Neuro - Montr\u00e9al Neurological Institute-Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "University of Eastern Finland, Brain Research Unit", "city": "Kuopio", "state": "Eastern Finland", "country": "Finland", "status": "", "lat": 62.89238, "lon": 27.67703}, {"facility": "Helsinki University Hospital", "city": "Helsinki", "state": "Uusimaa", "country": "Finland", "status": "", "lat": 60.16952, "lon": 24.93545}, {"facility": "Turku University Hospital", "city": "Turku", "state": "Western Finland", "country": "Finland", "status": "", "lat": 60.45148, "lon": 22.26869}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Provide written informed consent and be willing and able to comply with the requirements of the study protocol.\n2. Must be \u2265 18 and \u2264 75 years of age at the time of signing the informed consent form (ICF).\n3. Have a diagnosis of ALS according to the Gold Coast Diagnostic Criteria.\n4. Have either sporadic amyotrophic lateral sclerosis (sALS) or familial amyotrophic lateral sclerosis (fALS).\n5. Treatment Research Initiative to Cure ALS (TRICALS) risk profile \\> -6.00 and \\< -2.00.\n6. Have slow vital capacity (SVC) \u2265 60% of the predicted value.\n7. Have a score of 3 or 4 on Item #3 (Swallowing) of the Harmonized ALS Functional Rating Scale-Revised (ALS-FRS-R). Participants with a score of 3 can be enrolled with the Sponsor's approval only if they are able to safely swallow capsules.\n8. Have a body weight \u2265 45 kg and body mass index (BMI) \u2265 18 kg/m2.\n9. Participants of childbearing potential are eligible to participate if they are not pregnant or breastfeeding and agree to use one highly effective method of contraception, if sexually active, for the duration of the study through 90 days after the last study drug administration. Participants must not donate eggs for the duration of study through 90 days after the last dose of study drug.\n10. Participants capable of producing sperm and their partners of childbearing potential must agree to use condoms and one highly effective method of contraception, respectively, for the duration of the study through 90 days after the last study drug administration. Participants must not donate sperm for the duration of study through 90 days after the last dose of study drug.\n11. Be able and willing to undergo measurement of at-home mobility using contactless sensors connected to the internet.\n12. Be able and willing to have clinic or at-home visits during the study.\n\nExclusion Criteria:\n\n1. Have active psychiatric disease, substance abuse, neuromuscular weakness other than ALS, or any other medical condition that, in the opinion of the Investigator, might confound the results of the study or interfere with the intake or absorption of the study drug or participation for the full duration of the study.\n2. Have a history of unstable or severe cardiac, pulmonary, neurological, oncological, hepatic, or renal disease or another medically significant illness other than ALS precluding their safe participation in this study.\n3. Have a history of substance use disorder or illicit drug use in the last year (medically prescribed or over-the-counter cannabis use is allowed, if legal in the country).\n4. Have a history of serious infection (e.g., pneumonia, septicemia) \u2264 4 weeks of Screening; infection requiring hospitalization or treatment with intravenous (IV) antibiotics, antivirals, or antifungals within 4 weeks of Screening; or chronic bacterial infection (e.g., tuberculosis) deemed unacceptable as per the Investigator's judgment.\n5. Had major surgery \u2264 4 weeks before Screening.\n6. Be currently taking or planning to take strong cytochrome P450 3A4 (CYP3A4) inhibitors or inducers.\n7. Be currently taking or have discontinued treatment with riluzole \\< 4 weeks before Screening. Participants who have been taking a stable dose of riluzole for \u2265 4 weeks are eligible if they remain on the same dose throughout the duration of the study.\n8. Be taking Radicava (administered orally or IV as approved in the participant's country), Relyvrio, any other approved standard of care treatment, or tauroursodeoxycholic acid (TUDCA) as a dietary supplement administered for \\< 4 weeks prior to Screening or on a schedule of treatment different from the approved standard schedule of treatment. Participants who have completed \u2265 4 weeks of treatment before Screening are eligible if they plan to continue treatment at a stable dose throughout the duration of the study.\n9. Have an active malignancy or history (\u2264 1 years prior to enrollment) of solid, metastatic, or hematologic malignancy. Exception: basal cell carcinoma in situ of the skin that has been adequately treated.\n10. Be diagnosed with long QT syndrome. Any history of clinically significant ventricular arrhythmias (such as ventricular tachycardia, ventricular fibrillation, or Torsades de Pointes) should be discussed with and approved by the study medical monitor prior to enrollment.\n11. Have a prolonged corrected QT interval using Fridericia's formula (QTcF) at the Screening visit ECG \\> 450 ms for male participants and \\> 470 ms for female participants.\n12. Have an active SARS-CoV-2 infection or positive COVID-19 test at Screening.1\n13. Have one or more of the following laboratory test abnormalities at Screening: (a) Positive hepatitis C virus (HCV) antibodies with confirmation by HCV-RNA polymerase chain reaction (PCR) reflex testing; (b) Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb). Note: If a participant is negative for HBsAg but positive for HBcAb, the participant is eligible if the participant tests positive for the antibody to HBsAg reflex testing.\n14. Have uncontrolled seizures.\n15. Have a documented history of attempted suicide within 6 months prior to the Screening visit, or suicidal ideation of category 4 or 5 on the screening Columbia-Suicide Severity Rating Scale (C-SSRS), or be at significant risk for suicide, in the opinion of the Investigator.\n16. For participants of childbearing potential, be pregnant or breastfeeding.\n17. Have received a live vaccine within 14 days before Screening.\n18. Be concurrently participating in any other interventional clinical study or have received treatment with another investigational drug within 4 weeks or 5 half-lives of the investigational agent before the Screening visit, whichever is longer. Participation in observational studies is allowed.\n19. Have received stem cell or gene therapy for ALS at any time in the past.\n20. At the Screening visit, have one or more of the following:\n\n    1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 3.0 \u00d7 upper limit of normal (ULN)\n    2. Bilirubin \\> 1.5 \u00d7 ULN, unless the participant has documented Gilbert syndrome (isolated bilirubin \\> 1.5 \u00d7 ULN is acceptable if bilirubin is fractionated and direct bilirubin is \\< 35%)\n    3. Serum albumin \\< 3 g/dL\n    4. Hemoglobin \\< 9.0 g/dL\n    5. Platelets \\< 30,000/\u03bcL\n    6. Estimated glomerular filtration rate \\< 90 mL/min/1.73 m2 (Modification of Diet in Renal Disease \\[MDRD\\])", "sex": "ALL", "min_age": "18 Years", "max_age": "74 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "VRG50635", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06881979", "title": "High-Tech Rehabilitation Pathway for Chronic Adult Neuromuscular Diseases - Fit4MedRob-Chronic MND Project", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Istituti Clinici Scientifici Maugeri SpA", "summary": "The primary objective is to demonstrate, in a population of chronic neuromuscular disease the non-inferiority of a rehabilitation treatment integrated with robotic and/or technological devices compared to traditional rehabilitation treatment in the level of fatigue.\n\nThe main question it aims to answer is:\n\nAre high-tech rehabilitation interventions, including robotic systems, virtual reality, and stabilometric platforms, not inferior to traditional rehabilitation methods in improving balance, motor function, fatigue levels, sarcopenia, cognitive engagement, and overall quality of life in patients with chronic neuromuscular diseases (NMDs)? Researchers will compare a robotic treatment group, that consists in an high-tech rehabilitation, with a control group, that will receive the traditional rehabilitative treatment.", "interventions": [{"type": "DEVICE", "name": "High-tech rehabilitative treatment"}, {"type": "OTHER", "name": "Rehabilitation"}], "start_date": "2025-04-28", "url": "https://clinicaltrials.gov/study/NCT06881979", "target_entities": [], "locations": [{"facility": "IRCCS Azienda Ospedaliera Universitaria San Martino - Genova", "city": "Genova", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 45.21604, "lon": 11.87211}, {"facility": "Istituti Clinici Scientifici Maugeri IRCCS, Milan Institute", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "Azienda Ospedaliero-Universitaria di Modena", "city": "Modena", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 44.64783, "lon": 10.92539}, {"facility": "Istituti Clinici Scientifici Maugeri", "city": "Montescano", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 45.03196, "lon": 9.28366}, {"facility": "Istituti Clinici Scientifici Maugeri IRCCS", "city": "Pavia", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 45.19205, "lon": 9.15917}, {"facility": "Fondazione Don Carlo Gnocchi Onlus", "city": "Roma", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 44.99364, "lon": 11.10642}, {"facility": "Istituti Clinici Scientifici Maugeri IRCCS", "city": "Telese Terme", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 41.21752, "lon": 14.52681}], "contact_phone": "+39 0250725266", "contact_email": "christian.lunetta@icsmaugeri.it", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with a confirmed diagnosis of chronic neuromuscular diseases (e.g. ALS, CIDP, CMT)\n* Patient able to walk independently or with assistance\n* Patients capable of understanding and adhering to the study protocol.\n* Patients who have provided informed consent to participate in the study\n\nExclusion Criteria:\n\n* Patients with unstable medical conditions (e.g. severe cardiovascular diseases, such as \"New York Heart Association\" - NYHA=4, respiratory distress not compensated by ventilation) that could interfere, in the clinician's judgment, with their ability to safely participate in the study or to perform the assessments related to the protocol.\n* Patients currently participating in other clinical trials that could interfere with this study.\n* Pregnant women.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05498883", "title": "Quality of Life Evaluation with the SRI Questionnaire of ALS Patient with Non-invasive Ventilation", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "This study aims to measure the quality of life of ALS patients by the SRI questionnaire, in two distinct patient groups : Patient requiring initiation of NIV, and patients 24 hours dependent on NIV\n\nThis study also seeks to assess the quality of life of the caregivers with the Zarit Burden interview in those two populations", "interventions": [{"type": "OTHER", "name": "Score completion"}], "start_date": "2022-09-13", "url": "https://clinicaltrials.gov/study/NCT05498883", "target_entities": [], "locations": [{"facility": "Pneumology departement, Piti\u00e9-Salp\u00eatri\u00e8re hospital, GHU APHP-Sorbonne Universit\u00e9", "city": "Paris", "state": "Paris", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nPatients :\n\n* 18 years or more\n* Patient followed for ALS probable or certain with Awaji criteria\n* group 1 : severe chronic respiratory failure, with a decision by the attending physician to start NIV\n* group 2 : complete dependence on NIV\n\nCaregivers :\n\n* 18 years or more\n* Informed and does not object to the study\n* Primary caregiver for more than 3 months of an ALS patient on NIV (either at initiation or with 24 hours NIV dependence)\n* More than 30 hours spent at home per week\n* Met the inclusion criteria for group 1 (NIV initiation) or group 2 (24 hours NIV dependence)\n\nExclusion Criteria:\n\nPatients :\n\n* Patient under legal protection/ guardianship\n* insufficient command of French\n* Severe cognitive impairment, particularly in relation to frontotemporal degeneration\n* No indication criteria for NIV.\n\nCaregivers :\n\n* insufficient command of French\n* Caregiver of a patient with another chronic pathology", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03095989", "title": "An Online Mindfulness Intervention for People With ALS and Their Caregivers", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Harvard University", "summary": "The psychological impact of ALS on both patients and caregivers is high and affects their quality of life (QOL). However, there is minimal research about psychological interventions to improve QOL in the ALS scientific literature.\n\nRecent advances in clinical treatments aimed at improving the health of people with chronic disorders are based on the concept of mindfulness. Mindfulness can be defined as a flexible state of mind resulting from the simple act of actively noticing new things, as opposed to mindlessness, the human tendency to operate on\" autopilot\".\n\nPreliminary data suggests that mindfulness may promote a better QOL for people with ALS and their caregivers. The investigators also found that a mindful attitude was associated with slower disease progression.\n\nThis project's goal is to develop an innovative, web-based online mindfulness training program and intervention, customized for people with ALS and their primary caregivers. It is an active learning intervention, with cognitive exercises and lectures that increase participants' mindfulness. The efficacy of this program for improving QOL, and for reducing anxiety and depression in people with ALS and their caregivers, will be tested with a randomized clinical trial. Assessments immediately post-treatment as well as 3 and 6 months after recruitment will be conducted, comparing subjects undergoing the mindfulness intervention to a control group.", "interventions": [{"type": "BEHAVIORAL", "name": "Mindfulness"}], "start_date": "2015-01", "url": "https://clinicaltrials.gov/study/NCT03095989", "target_entities": [], "locations": [{"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nInclusion Criteria for ALS Subjects:\n\n* Definite, probable, probable laboratory-supported, or possible ALS by revised El-Escorial criteria (Brooks, Miller, Swash, \\& Munsat, 2000).\n* Must have the physical ability, with or without adaptive devices, to use a computer and access the Internet.\n* A forced vital capacity of 50% or greater than the predicted value, measured within 30 days of enrollment. If impaired bulbar function compromises accurate pulmonary function testing as determined by the study neurologist, then a forced vital capacity as low as 40% of predicted is permitted. If taken, stable doses of anti-anxiety or antidepressant medications for 30 days before study entry, and during the study from the T1 to the T2 time point.\n\nInclusion Criteria for Caregivers:\n\n* Must have the physical ability to use a computer and access the Internet.\n\nExclusion Criteria:\n\nExclusion Criteria for ALS Subjects:\n\n* Scores on the Edinburgh Cognitive Assessment (ECAS) within 90 days of study entry, that meet criteria for mild frontotemporal dysfunction (either cognitive or behavioral)\n* Significant cognitive impairment or significant uncontrolled psychiatric disease (for example, schizophrenia, bipolar disorder), in the opinion of the study neurologist.\n* Unsuitable for the study as determined by the study neurologist.\n* Regular meditation or participation in a mindfulness program in past 6 months.\n\nExclusion Criteria for Caregivers:\n\n* Unwillingness to participate in the study\n* Unsuitable for the study as determined by the clinical staff. For example, the clinical staff may consider inappropriate a caregiver who showed no real interest toward the care process or showed signs of severe mental health problems.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06453668", "title": "A Study of TCD601 (Siplizumab) in Newly Diagnosed Adult Amyotrophic Lateral Sclerosis (ALS) Patients", "phase": "PHASE1", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "ITB-Med LLC", "summary": "The purpose of this study is to investigate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of TCD601 (siplizumab) in newly diagnosed adult ALS patients.", "interventions": [{"type": "BIOLOGICAL", "name": "TCD601"}], "start_date": "2024-04-16", "url": "https://clinicaltrials.gov/study/NCT06453668", "target_entities": ["cd4_t_cells"], "locations": [{"facility": "Sk\u00e5ne University Hospital Malm\u00f6", "city": "Malm\u00f6", "state": "", "country": "Sweden", "status": "", "lat": 55.60587, "lon": 13.00073}, {"facility": "Studieenheten Akademiskt Specialistcentrum", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Ume\u00e5 University Hospital", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Male or female patients \u2265 18 to 80 years of age.\n* Diagnosis of ALS by revised El Escorial Criteria, at study entry within 24 months of first symptoms.\n* Patients on existing ALS treatment must have been on a stable dose for 28 days.\n\nKey Exclusion Criteria:\n\n* Patient with severe systemic infections, current or within the two weeks prior to randomization.\n* Subjects who, in the opinion of the investigator, are not capable of giving informed consent for the study or who are unable or unwilling to adhere to the study requirements outlined in the protocol.\n* Use of other investigational products or treatment in another investigational drug study within 30 days of screening\n* Pregnant or nursing (lactating) women.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "TCD601", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04220190", "title": "RAPA-501 Therapy for ALS", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Rapa Therapeutics LLC", "summary": "RAPA-501-ALS is a phase 2/3 expansion cohort study of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).", "interventions": [{"type": "BIOLOGICAL", "name": "RAPA-501 Autologous T stem cells"}], "start_date": "2025-01-02", "url": "https://clinicaltrials.gov/study/NCT04220190", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "(301) 518-3104", "contact_email": "dan@rapatherapeutics.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Male or female patients \u2265 18 years of age.\n2. Patients with sporadic or familial amyotrophic lateral sclerosis (ALS) diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria.\n3. . Less than or equal to 24 months since ALS symptom onset.\n4. Total ALSFRS-R score between 34 and 45.\n5. Must have a source of autologous T cells potentially sufficient to manufacture RAPA-501 cells, as defined by a peripheral CD3+ T cell count \u2265 500 cells per \u03bcl.\n6. Patients may continue riluzole (Rilutek\u00ae), and/or edaravone (Radicava\u00ae), and/or sodium phenylbutyrate/taurusodial (Relyvrio\u2122) if on a stable dose for at least 30 days prior to the screening visit.\n7. Patients must be \u2265 2 two weeks removed from major surgery or investigational therapy.\n8. Patients must have recovered from clinical toxicities (\\[resolution of CTCAE(v5) \\[version 5\\] toxicity to a value of \u2264 2\\].).\n9. Serum creatinine \u2264 less than or equal to 2.0 mg/dL.\n10. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \u2264 3 x upper limit of normal (ULN).\n11. Bilirubin \u2264 1.5 (except if due to Gilbert's disease).\n12. Pulmonary slow vital capacity (SVC) \u2265 70% of predicted normal.\n13. No history of abnormal bleeding tendency.\n14. Voluntary written consent must be given before performance of any study related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient participant at any time without prejudice to future medical care.\n\nExclusion Criteria:\n\n1. Active uncontrolled infection.\n2. Hypertension not adequately controlled by \u2264 3 medications.\n3. History of documented pulmonary embolus within 6 months of enrollment.\n4. Clinically significant cardiac pathology, as defined by: myocardial infarction within 6 months prior to enrollment, Class III or IV heart failure according to NYHA, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.\n5. Patients with history of coronary artery bypass grafting or angioplasty will receive a cardiology evaluation and be considered on a case-by-case basis.\n6. HIV, hepatitis B, or hepatitis C seropositive.\n7. Pregnancy or breastfeeding patients.\n8. Patients of Subjects of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception.\n9. Patients Subjects may be excluded at the Principal Investigator discretion of the PI or if it is deemed that allowing participation would represent an unacceptable medical or psychiatric risk.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RAPA-501", "targeting_mechanism": "RAPA-501 is an autologous hybrid TREG/Th2 cell therapy targeting neuroinflammation in ALS.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00673140", "title": "Far Infrared Irradiation for Control, Management and Treatment of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "GAAD Medical Research Institute Inc.", "summary": "Amyotrophic Lateral Sclerosis (ALS, sometimes called Lou Gehrig's s Disease, or Maladie de Charcot) is a progressive, usually fatal, neurodegenerative disease caused by the degeneration of motor neurons, the nerve cells in the central nervous system that control voluntary muscle movement.\n\nThis study will investigate the use of far infrared radiation for the control, management and treatment of ALS.", "interventions": [{"type": "RADIATION", "name": "Far Infrared Radiation (5\u03bcm to 20\u03bcm wavelength)"}, {"type": "RADIATION", "name": "Far infrared radiation"}], "start_date": "2008-05", "url": "https://clinicaltrials.gov/study/NCT00673140", "target_entities": [], "locations": [{"facility": "The Centre for Incurable Diseases", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with ALS\n\nExclusion Criteria:\n\n* None", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Far Infrared Radiation", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04254913", "title": "Clinical Pharmacology Study of Oral Edaravone in Amyotrophic Lateral Sclerosis Patients With Gastrostomy", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "To evaluate the pharmacokinetics of single doses of edaravone oral suspension in Amyotrophic Lateral Sclerosis Patients with gastrostomy", "interventions": [{"type": "DRUG", "name": "MT-1186"}], "start_date": "2020-01-24", "url": "https://clinicaltrials.gov/study/NCT04254913", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Investigational site", "city": "Chiba", "state": "", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nThe key criteria are listed below.\n\n* Patients aged between 20 and 80 years at the time of informed consent\n* Japanese patients\n* Among patients with ALS, those \"Clinically definite ALS,\" \"Clinically probable ALS\" or \"Clinically probable-laboratory-supported ALS\" according to El Escorial Revised Airlie House criteria\n* ALS Patients with gastrostomy\n* Patients who can consent to contraception\n* Patients who have thoroughly understood the contents of the study and voluntarily provided written informed consent to participate in the study\n\nExclusion Criteria:\n\nThe key criteria are listed below.\n\n* Patients in whom the possibility could not be ruled out that the current symptoms were symptoms of a disease requiring differential diagnosis, such as cervical spondylosis and multifocal motor neuropathy\n* Patients undergoing treatment for malignancy.\n* Patients who have presence of clinically significant liver, heart, or renal disease requiring hospitalization (except ALS) and infections requiring antibiotics. Patients who have a problem in general condition and are judged ineligible by the Investigator\n* Body mass index (BMI) of \\<15.0 or \\>30.0, or a body weight of \\<40 kg\n* Patients judged by the investigator (or subinvestigator) to be unsuitable for the study for any other reason", "sex": "ALL", "min_age": "20 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MT-1186 (edaravone oral suspension)", "targeting_mechanism": "Edaravone reduces oxidative stress by scavenging reactive oxygen species (ROS) and acting as an antioxidant.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07298486", "title": "Impact of Robotic Glove Use on Quality of Life, Grip Strength and Fine Motor Control in ALS", "phase": "NA", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Nova Southeastern University", "summary": "The goal of this clinical trial is to determine the impact of robotic glove use on quality of life, grip strength and fine motor control in participants with Amyotrophic Lateral Sclerosis (ALS). This will provide valuable insights into how an assistive technology intervention can influence functional ability in daily tasks and enhance overall well-being in individuals impacted by ALS.\n\nParticipants will attend 3 in-person clinic visits (BASE, 4 week and 8 week visit) and 2 Telehealth visits (occurring 24 hours after BASE and 4 week visit). The in-person clinic visits will include assessments by either a physical or occupational therapist, followed by assessments and scoring of grip strength, various fine motor strength assessments of affected hand, fine motor coordination assessment of the affected hand and various quality of life scales.\n\nThe participant will be fitted and measured for the appropriately sized robotic glove. Once the fit is confirmed, both the participant and caregiver will receive education on how to don and doff the glove, power it on and off, and follow the study protocol. Once they demonstrate understanding and independence with these steps, the intervention will begin.\n\nThe robotic glove intervention consists of either the participant or caregiver donning the glove and powering it on. A timer will be set for 20 minutes. The participant will sit comfortably at a table with their elbow supported on the table. The PI and/or member of the study team will be present throughout the 20 minute session to monitor for discomfort, fatigue or any additional patient reported symptoms. Once the time is complete, the robotic glove will be powered off and the glove removed. The participant and caregiver will be instructed to perform this one time daily, for 5 days per week for a total of 8 weeks. The PI and study team members will be available for any questions via iPhone or e-mail throughout this time period.\n\nThe Telehealth visits will consist of synchronous video and audio via the Zoom application. The PI and/or study team members will monitor the set up, application and powering on of the Robotic Glove. The treatment of 20 minutes will be monitored and any questions the participant or caregiver have will be answered. This visit's goal is to ensure compliance and proper application of the robotic glove.", "interventions": [{"type": "DEVICE", "name": "Robotic Glove Use"}], "start_date": "2026-02-09", "url": "https://clinicaltrials.gov/study/NCT07298486", "target_entities": [], "locations": [{"facility": "Nova Southeastern University, David and Cathy Husman Neuroscience Institute", "city": "Davie", "state": "Florida", "country": "United States", "status": "", "lat": 26.06287, "lon": -80.2331}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of clinically possible, probable or definite amyotrophic lateral sclerosis by the treating neurologist\n* Have a caregiver that is willing and present to assist with application of robotic glove\n* Reports fine motor deficits or grip strength deficits in at least 1 hand\n* Demonstrates \\> or = 3 lbs. of Grip strength as measured by a dynamometer on affected hand AND/OR Demonstrates Fine motor deficits and has \\> or = 1 lb. of pinch strength (tip to tip, three jaw chuck, or lateral key pinch) as measured by pinch gauge dynamometer\n* Demonstrates a loss of at least 3 lbs. of Grip strength OR 1lb. of pinch strength over a minimum of 1 month period in affected hand\n* Stable dose of efficacious medications for 30 days.\n\nExclusion Criteria:\n\n* History of Rheumatoid Arthritis\n* History of Depuytren's contracture\n* Open wound at affected hand where robotic glove will be placed\n* History of Trigger Finger\n* Complete loss of sensation at the affected hand where robotic glove will be placed\n* History of Carpal Tunnel Syndrome\n* Prior or current use of robotic glove\n* Active physical or occupational therapy treatment interventions on affected hand", "sex": "ALL", "min_age": "18 Years", "max_age": "99 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Robotic glove provides mechanical assistance to restore arm function and improve grip strength through wearable technology.", "targeting_mechanism_pmid": "36724237", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04245709", "title": "Clenbuterol on Motor Function in Individuals With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Dwight Koeberl, M.D., Ph.D.", "summary": "The purpose of this study is to assess the safety and tolerability of clenbuterol (taken by mouth) in subjects with ALS (amyotrophic lateral sclerosis) and to assess the effectiveness of clenbuterol with regard to motor function in subjects with ALS. Subjects will be in this study approximately 24 weeks. The study drug, clenbuterol, is taken twice a day. As part of this study subjects will have the following tests and procedures: medical history, vital signs, physical examination, blood tests, heart and lung function tests, muscle function test, ALSFRS-R (ALS Functional Rating Scale Revised), thyroid function and for women who can become pregnant, pregnancy tests.", "interventions": [{"type": "DRUG", "name": "Clenbuterol"}], "start_date": "2020-02-10", "url": "https://clinicaltrials.gov/study/NCT04245709", "target_entities": ["Beta-2 adrenergic receptor"], "locations": [{"facility": "Duke University Medical center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of possible or more definite ALS according to the El Escorial criteria\n* FVC \\>50% of predicted for age, height and gender.\n* At least four of 12 ALSFRS-R questions scored as 2 or 3 at screening.\n* Diminished but measurable grip strength (1) in at least one hand (females:10-50 pounds; males, 10-70 pounds).\n* Taking riluzole at a stable dose or not taking riluzole at screening.\n* On Radicava at a stable dose for at least 30d or not taking this\n* Life expectancy at least 6 months\n* Able to swallow tablets without crushing.\n* Age: 18+ years at enrollment.\n* Subjects are capable of giving written consent.\n* If sexually active, must agree to use contraceptive or abstinence for duration of treatment\n* Females of child bearing age must have negative pregnancy test at screening\n\nExclusion Criteria:\n\n* Concurrent illness or laboratory abnormalities that could confound the measurement of ALS progression or interfere with the ability to complete the study.\n* Taking any investigational study drug within 30 days of screening or five half-lives of the prior agent.\n* No previous exposure to clenbuterol.\n* Pregnancy\n* Clinically relevant EKG abnormality (arrhythmia, cardiomyopathy)\n* Tachycardia (resting heart rate greater than 100 beats per minute)\n* History of seizure disorder\n* Hyperthyroidism\n* Pheochromocytoma\n* Pregnancy\n* Have any other co-morbid conditions that in the opinion of the study investigator, places the participant at increased risk of complications, interferes with study participation or compliance, or confounds study objectives\n* History of hypersensitivity to 2-agonist drugs such as albuterol, levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline, salmeterol (Serevent).\n* The use of the following concomitant meds is prohibited during the study:\n\ndiuretics (furosemide, Lasix), digoxin (digitalis, Lanoxin);blockers such as atenolol (Tenormin), metoprolol (Lopressor), and propranolol (Inderal); tricyclic antidepressants such as amitriptyline (Elavil, Etrafon), doxepin (Sinequan), imipramine (Janimine, Tofranil), and nortriptyline (Pamelor); monoamine oxidase inhibitors such as isocarboxazid (Marplan), phenelzine (Nardil), rasagiline (Azilect), selegiline (Eldepryl, Emsam), or tranylcypromine (Parnate); or other bronchodilators such as albuterol (Ventolin), levalbuterol (Xopenex), bitolterol (Tornalate), pirbuterol (Maxair), terbutaline (Brethine, Bricanyl), salmeterol (Serevent), isoetherine (Bronkometer), metaproterenol (Alupent, Metaprel), or isoproterenol (Isuprel Mistometer).", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Clenbuterol", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01504009", "title": "Muscle Training of Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Southern Denmark", "summary": "Patients with neuropathic diseases are experiencing increasing muscle weakness, loss of muscle strength and functional abilities during their illness. In healthy people, regular exercise is the best way to maintain or improve muscle strength, endurance and general health status and thereby maintain functioning abilities. Previously, patients with neuromuscular diseases were advised to avoid any kind of physical exercise. However, lately a number of studies have evaluated the effect of training in patients with neuromuscular diseases, and positive effects on the functional abilities have been found. Based on these findings we want to investigate the mechanisms leading to development of muscle atrophy and loss of functional abilities, and to explore the opportunities of reducing muscle wasting and thereby improve the course of the disease development through strength training.\n\nThe main objective is to investigate the effects of strength training on slowing disease progression and reduce the decline in muscle strength and function in patients with amyotrophic lateral sclerosis (ALS). In addition, the aim is to carry out detailed studies of biological processes in muscle tissue in order to unveil mechanisms leading to muscle atrophy, and to examine effects of a strength training program. The goal is to be able to incorporate strength training in the treatment program of these patients in order to maintain muscle strength and function in the individual for as long as possible.\n\nMinimum 10 patients with the disease are included in the study. Through a 12 week period the patients will participate in strength training 2-3 times per week. Muscles biopsies will be taken (i) 12 weeks before commencement of strength training program, (ii) at the beginning of training and (iii) after 12 weeks of strength training. Patients will function as their own controls. Blood samples will be collected simultaneously in order to follow the development of the strength training. Furthermore, participants will be assessed through at number of functional tests and questionnaires evaluating their strength, balance and social/ psychological status.\n\nSubjects are recruited through their association with Odense University Hospital. In the present study, the participants become part of a social network, while participating in organized training sessions, and thus have a possibility to make contact with other ALS patients in the same situation as themselves.", "interventions": [{"type": "OTHER", "name": "Muscle training"}], "start_date": "2011-05", "url": "https://clinicaltrials.gov/study/NCT01504009", "target_entities": [], "locations": [{"facility": "Department of Clinical Research, Odense University Hospital, University of Southern Denmark", "city": "Odense C", "state": "Fyn", "country": "Denmark", "status": "", "lat": 55.40841, "lon": 10.39538}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosed with Amyotrophic lateral sclerosis (ALS)\n\nExclusion Criteria:\n\n* Late stage ALS", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "Swim training modulates skeletal muscle energy metabolism and ameliorates reduction in grip strength in SOD1G93A mice.", "animal_results_pmid": "30634386", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00847847", "title": "Neuromuscular Transmission in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "Consistent data suggest that neuromuscular transmission is impaired in ALS patients. Neuromuscular junctions dysfunction may appear very early in the disease, as shown by data in animal models. The pathogenesis of this neuromuscular transmission impairment is unknown. Nogo A isoform, a possible marker of the disease over-expressed in skeletal muscle of ALS patients, can be involved. We will characterize the pathophysiological mechanisms implicated using a complete study of the structure and function of the NMJ on muscle biopsies, in a group of 20 ALS patients compared to 10 controls.", "interventions": [{"type": "PROCEDURE", "name": "Anconeus Muscle biopsy"}], "start_date": "2009-03", "url": "https://clinicaltrials.gov/study/NCT00847847", "target_entities": ["neuromuscular_transmission", "NogoA"], "locations": [{"facility": "Bruneteau Gaelle", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "1. ALS patients:\n\n   Inclusion criteria :\n   * Aged 18 to 75 (inclusive)\n   * Possible, probable (clinically or laboratory) or definite ALS according to the revised El Escorial criteria\n   * Duration of the disease of less then 12 months\n   * Willing and able to provide a written informed consent\n   * With french social insurance affiliation\n\n   Exclusion criteria :\n   * Cognitive changes or psychiatric condition, inability to give informed consent\n   * patient unable to contact or to be contacted by the investigator in case of emergency\n   * women who are pregnant or nursing\n   * concomitant medication contraindicating muscular biopsy (platelet suppressive agents if treatment can not medically be stopped 2 weeks before surgical procedure, oral anticoagulant therapy)\n   * medical condition contraindicating muscular biopsy (hypo-coagulative disease, allergy to anaesthetic drugs)\n   * medical condition susceptible to influence on EMG examination (concomitant neurological or rheumatological disease)\n2. Controls:\n\n   * adult patients (minimum 18y) without neuromuscular disease\n   * undergoing elbow surgery for local joint or bone disease", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "In SOD1G93A mouse models, neuromuscular junction (NMJ) denervation and distal axonal degeneration occur prior to symptom onset, indicating early NMJ dysfunction in ALS pathogenesis.", "animal_results_pmid": "30320556", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07618585", "title": "Safety and Efficacy of Intrathecal NTF001 Injection in ALS", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ruijin Hospital", "summary": "This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of intrathecal NTF001 injection, an AAV-mediated human neuron-derived neurotrophic factor gene therapy, in patients with amyotrophic lateral sclerosis (ALS).\n\n12 patients with ALS will be enrolled. Each participant will receive a single intrathecal administration of NTF001 and will be followed for 52 weeks after treatment. The primary outcome measures include treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Secondary outcome measures include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, and neurological function.\n\nThis study aims to provide preliminary clinical evidence regarding the safety and potential efficacy of intrathecal NTF001 injection in patients with ALS.", "interventions": [{"type": "GENETIC", "name": "NTF001 Injection"}], "start_date": "2026-04-01", "url": "https://clinicaltrials.gov/study/NCT07618585", "target_entities": ["Neurotrophic factor"], "locations": [{"facility": "Ruijin Hospital, Shanghai Jiao Tong University School of Medicine", "city": "Shanghai", "state": "Shanghai Municipality", "country": "China", "status": "", "lat": 31.22222, "lon": 121.45806}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Voluntarily participate in this study and sign the informed consent form.\n* Agree to comply with study procedures and cooperate with all study-related assessments throughout the study.\n* Male or female patients aged 18 to 65 years.\n* Meet the diagnostic and exclusion criteria for amyotrophic lateral sclerosis according to the Chinese Expert Consensus on the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis 2022 issued by the Neurology Branch of the Chinese Medical Association.\n* Have a history of amyotrophic lateral sclerosis of no more than 5 years.\n\nExclusion Criteria:\n\n* Mini-Mental State Examination (MMSE) score \\< 24.\n* Patient Health Questionnaire-9 (PHQ-9) score \u2265 16.\n* Abnormal liver or renal function, defined as AST or ALT \\> 1.5 \u00d7 upper limit of normal (ULN), or serum creatinine (Cr) \\> 1.5 \u00d7 ULN.\n* Abnormal coagulation function or current use of anticoagulants.\n* Positive infectious disease screening, including positive HBsAg or HBV-DNA, positive HCV-RNA, positive HIV test, or positive syphilis serology.\n* Currently receiving antiviral treatment for hepatitis B or hepatitis C.\n* Unstable or severe systemic diseases, including active tuberculosis, cardiovascular, respiratory, gastrointestinal, urinary, psychiatric or neurological disorders, such as epilepsy, hematological disorders, immune system diseases, or abnormal laboratory findings that, in the opinion of the investigator, make the participant unsuitable for this study.\n* Current or previous history of malignant tumor.\n* History of severe allergic reactions, allergy to contrast agents, or inability to undergo surgical anesthesia.\n* Currently participating in another clinical trial, or participation in another clinical trial within 3 months before screening.\n* Previous receipt of gene therapy before screening.\n* Receipt of stem cell therapy within 6 months before screening.\n* Use of other investigational drugs within 4 weeks before screening or within 5 half-lives of the investigational drug, whichever is longer, or use of any medication that, in the opinion of the investigator, may affect this study.\n* Receipt of a live vaccine within 2 months before screening, or any vaccination within 30 days before screening.\n* History of alcohol dependence or drug addiction, and inability to stop alcohol consumption as instructed during the study.\n* Female participants who are pregnant or breastfeeding.\n* Participants considered unsuitable for enrollment by the investigator.\n* Patients requiring ventilator-assisted ventilation.\n* Patients allergic to the investigational intervention.\n* Patients with obvious signs of dementia.\n* Patients with other psychiatric disorders that may affect disease assessment.\n* Severely obese patients, defined as BMI \\> 35 kg/m\u00b2.", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NTF001 Injection", "targeting_mechanism": "AAV-mediated delivery of human neuron-derived neurotrophic factor gene therapy to provide neuroprotection and motor neuron support.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07095712", "title": "Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "n-Lorem Foundation", "summary": "This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.", "interventions": [{"type": "DRUG", "name": "nL-TARD-001"}], "start_date": "2024-11-25", "url": "https://clinicaltrials.gov/study/NCT07095712", "target_entities": ["TARDBP"], "locations": [{"facility": "Columbia University, Irving Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Houston Methodist", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s)\n* Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records\n* Genetically confirmed neurological disorder\n\nExclusion Criteria:\n\n* Participant has any condition that in the opinion of the Site Investigator would ultimately prevent the completion of study procedures\n* Use of an investigational medication within less than 5 half-lives of the drug at enrollment", "sex": "FEMALE", "min_age": "49 Years", "max_age": "49 Years", "healthy_volunteers": false, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "nL-TARD-001", "targeting_mechanism": "Personalized antisense oligonucleotide designed to target a pathogenic TARDBP variant and reduce TDP-43 protein expression or toxicity.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05882695", "title": "Study of SPG302 in Healthy Volunteers and ALS Participants", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Spinogenix", "summary": "The first-in-human Phase 1 study described herein will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of SPG302 in healthy volunteers and ALS participants", "interventions": [{"type": "DRUG", "name": "SPG302"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2023-07-03", "url": "https://clinicaltrials.gov/study/NCT05882695", "target_entities": ["spg302"], "locations": [{"facility": "Macquarie University", "city": "North Ryde", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.79677, "lon": 151.12436}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Flinders Medical center", "city": "Adelaide", "state": "South Australia", "country": "Australia", "status": "", "lat": -34.92866, "lon": 138.59863}, {"facility": "Nucleus Melbourne (healthy volunteers)", "city": "Melbourne", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.814, "lon": 144.96332}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age 18-55\n* Must be in good health with no significant medical history\n* Clinical laboratory values within normal range or \\< 1.2 times ULN\n* BMI 18-32 (inclusive)\n* Contraceptive use by men or women consistent with local regulations\n* Able and willing to provide written informed consent\n\nExclusion Criteria:\n\n* Any physical or psychological condition that prohibits study completion\n* Known cardiac disease\n* Active or history of malignancy in the past 5 years\n* Serious infection within 1 month of screening\n* Acute illness within 30 days of Day 1\n* Surgery, bone fracture, or major musculoskeletal injury in the past 3 months\n* History of suicidal behavior or suicidal ideation\n* Active cigarette smokers and users of nicotine-containing products\n* HIV, hepatitis B and hepatitis C positive\n* SBP \\>140 or \\<90\n* DBP \\>90 or \\<40\n* HR \\<40 or \\>100\n* QTcF \\>450ms, cardiac arrhythmia, or clinically significant abnormal ECG\n* Prescriptions, over-the-counter, or herbal medication within 7 days\n* Vaccines within 14 days\n* Other investigational products within 30 days\n* Blood donation within 30 days\n* Plasma donation within 7 days\n* Pregnant or breastfeeding\n* Otherwise unfit, on metabolic-altering lifestyle/diet, positive urine drug screen or intake of alcohol or caffeine-containing products\n\nALS Cohort Inclusion Criteria:\n\n* Age 18-80\n* ALS TRICALS risk score\n* Stable dose of standard of care treatment\n* Contraception use by men or women consistent with local regulations\n* Able and willing to provide written informed consent\n\nALS Cohort Exclusion Criteria:\n\n* Underlying physical or psychological condition prohibiting study completion\n* Known cardiac disease\n* Active or history of malignancy in the past 5 years\n* Serious infection within 1 month of screening\n* Acute illness within 30 days of Day 1\n* History of suicidal behavior or suicidal ideation\n* Active cigarette smokers and users of nicotine-containing products\n* Neurodegenerative disease\n* External respiratory support or supplemental oxygen requirement\n* HIV, hepatitis B and hepatitis C positive\n* SBP \\>140 or \\<90\n* DBP \\>90 or \\<40\n* HR \\<40 or \\>100\n* QTcF \\>450ms, cardiac arrhythmia, or clinically significant abnormal ECG\n* Vaccines within 14 days\n* Other investigational products within 30 days\n* Blood donation within 30 days\n* Plasma donation within 7 days\n* Pregnant or breastfeeding\n* Otherwise unfit", "sex": "ALL", "min_age": "18 Years", "max_age": "55 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "SPG302", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05039099", "title": "A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AL-S Pharma", "summary": "The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.", "interventions": [{"type": "DRUG", "name": "AP-101"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-09-02", "url": "https://clinicaltrials.gov/study/NCT05039099", "target_entities": ["ap_101"], "locations": [{"facility": "UC San Diego, ACTRI", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Department of Neurology, University Hospitals", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "ALS clinic at the Kaye Edmonton Clinic, University of Alberta", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "London Health Sciences Centre - Victoria Hospital", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "ALS Research Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute and Hospital / Dr Genge", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Hannover Medical School", "city": "Hanover", "state": "Hanover", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Charit\u00e9", "city": "Berlin", "state": "State of Berlin", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Ulm University Hospital", "city": "Ulm", "state": "Ulm", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Deutsches Zentrum f\u00fcr Neurodegenerative Erkrankungen e.V. (DZNE)", "city": "Bonn", "state": "", "country": "Germany", "status": "", "lat": 50.73438, "lon": 7.09549}, {"facility": "Hanyang University Medical Center", "city": "Seoul", "state": "Seoul", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Studieenheten Akademiskt specialistcentrum, SLSO", "city": "Stockholm", "state": "Stockholm County", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Norrlands universitetssjukhus/ University Hospital of Northern Sweden (NUS)", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* All participants must adhere to contraception restrictions\n* Female participants of childbearing potential must adhere to contraception restrictions\n* Have possible, clinically probable, clinically probable-laboratory supported or definite familial or sporadic ALS in accordance with the El-Escorial criteria or who have a diagnosis of ALS as defined by the Gold Coast Criteria; progressive motor impairment documented by history or repeated clinical examination, preceded by normal motor development, and presence of upper and lower motor neuron dysfunction in at least 1 body region or lower motor neuron dysfunction in at least 2 body regions and investigations excluding other conditions\n* In familial ALS participants, a confirmed pathogenic superoxide dismutase 1 (SOD1) mutation\n* Onset of symptoms (i.e, weakness) within past 24 months prior to screening, at the time of obtaining informed consent\n* Have slow vital capacity (SVC) of greater than or equal to (\\> or =) 50 percentage (%) of predicted values. Participants with SVC of \\<50% of predicted values may be permitted to enter the open-label extension, based on the opinion of the investigator\n* Absence of bilevel positive airway pressure (BiPAP)/proportional assist ventilation (PAV) \\> 4 hours for symptoms attributable to ALS. Use of a CPAP for pre-existing conditions will be allowed\n* If on riluzole, must be on a stable dose.\n* If on edaravone, must have completed 2 cycles and are expected to remain on the same dose throughout the study\n* Able to provide informed consent which includes compliance with the requirements and restrictions\n* Have venous access sufficient to allow for blood sampling\n* Have clinical laboratory test results within the normal reference range for the population or study site, or results with acceptable deviations that are judged to be not clinically significant by the investigator\n\nExclusion Criteria:\n\n* Have participated or currently participating in another clinical trial within 12 weeks of baseline (Day 1)\n* Have undergone a tracheostomy for ALS symptoms\n* Are on nasal intermittent positive pressure ventilation (NIPPV) \\>4 hours per day for the treatment of ALS related symptoms\n* Have other causes of neuromuscular weakness\n* Have cognitive impairment, severe disease in the cardiovascular, hematological, renal system, neurodegenerative disease, pulmonary disorder, or psychiatric illness\n* Pregnant or nursing women\n* Have been exposed to any antisense treatment targeting SOD1 within 6 months of the baseline visit\n* Have undergone stem cell therapy", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AP-101", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05747937", "title": "Longitudinal Assessment of Autonomic and Sensory Nervous System in ALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Istituti Clinici Scientifici Maugeri SpA", "summary": "The goal of this interventional non-pharmacological study is to evaluate, using a multimodal approach, the progression of autonomic and sensory involvement in in amyotrophic lateral sclerosis (ALS) patients enrolled within 18 months from motor onset and its relationship with the progression of overall clinical disability.\n\nThe main questions it aims to answer are:\n\n* Is autonomic dysfunction at diagnosis associated with disease progression and survival in patients with Amyotrophic Lateral Sclerosis ?\n* Can we identify in the skin biomarkers to be used as reliable measures of disease progression and to apply in future clinical trials for patient stratification and to assess response to drug treatment ? Participants at time 0 will receive a full clinical and instrumental examination and a blood sample testing to check inclusion and exclusion criteria, genetic screening for the most common genes associated with ALS (SOD1, FUS, TARDBP and c9orf72), questionnaires about clinical characteristics, quality of life, pain and a multidomain battery of neuropsychological tests, multimodal assessment of the autonomic nervous system including skin biopsy for morphological study. At follow-up we'll perform clinical scales and skin biopsy.\n\nResearchers will compare results from ALS patients with data obtained from a population of age and sex matched healthy subjects.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "Skin biopsy"}, {"type": "DIAGNOSTIC_TEST", "name": "Cardiovascular Reflexes testing"}, {"type": "DIAGNOSTIC_TEST", "name": "Administration of clinical scales evaluating autonomic symptoms, pain small fiber neuropathy symptoms"}, {"type": "DIAGNOSTIC_TEST", "name": "Dinamic Sweat Test"}], "start_date": "2021-05-15", "url": "https://clinicaltrials.gov/study/NCT05747937", "target_entities": [], "locations": [{"facility": "ICS Maugeri - IRCCS of Telese Terme", "city": "Telese Terme", "state": "Benevento", "country": "Italy", "status": "RECRUITING", "lat": 41.21752, "lon": 14.52681}, {"facility": "Department of Neurosciences, Reproductive Sciences and Odontostomatology, University of Naples Federico II", "city": "Naples", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 40.85216, "lon": 14.26811}], "contact_phone": "+390824909257", "contact_email": "maria.nolano@icsmaugeri.it", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS patients will be recruited within 18 months from the motor symptoms onset\n\nExclusion Criteria:\n\n* glucose intolerance or conditions potentially affecting the peripheral nervous system", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01082653", "title": "Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "SUSPENDED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "TCA Cellular Therapy", "summary": "A Phase I, single center, prospective, non-randomized, open label, safety/efficacy study of the infusion of autologous bone marrow-derived stem cells, in 6 patients with Amyotrophic Lateral Sclerosis according to established criteria (1), (2) with a moderate to severe diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.\n\nThe primary purpose of this study is to evaluate safety of the infusion procedure, as assessed by absence of complications at the site of infusion or the appearance of new neurologic deficit not attributed to the natural progression of the disease.\n\nSecondary outcomes will include a)neurological evidence of trends toward a slowing down of the decline of the forced vital capacity (FVC) (3) and of the functional rating scale (ALS-FRS) scores, as assessed at 3-month intervals, b)evidence of a decline of the maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z (4) scores and c)patient evaluation that the treatment was effective and consider the possibility of a new cell product stem cell infusion.\n\nSubjects who fulfill inclusion/exclusion criteria and sign informed consent will undergo an aspiration of bone marrow from the iliac crest for preparation of the cellular product.\n\nThe day of infusion, the investigational product will be injected into the patient's intrathecal space.\n\nAfter cell infusion patients will be followed at WK 2, MN 1, MN 2, MN 6 and a long-term followup at MN 12 in the clinic and/or office. Electromyographic (EMG) studies, Forced vital capacity (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores will have been used to assess the status of the disease before (historical record acceptable if done within three months of Screening Visit) and during the 12-month study period after cell infusion.", "interventions": [{"type": "BIOLOGICAL", "name": "autologous bone marrow-derived stem cells"}], "start_date": "2010-03", "url": "https://clinicaltrials.gov/study/NCT01082653", "target_entities": [], "locations": [{"facility": "TCA Cellular Therapy", "city": "Covington", "state": "Louisiana", "country": "United States", "status": "", "lat": 30.47549, "lon": -90.10042}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Adult male and female subjects \\> 18 years of age.\n2. Good understanding of the protocol and willingness to consent.\n3. Moderate to severe Diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.\n4. Vital capacity at least 50% predicted value for gender, height and age.\n5. More than 6 and less than 36 months of evolution of the disease.\n6. Hematocrit greater than 30 % prior to bone marrow aspiration.\n7. Platelet count greater than 100 Thousand/uL at screening.\n8. INR less than or equal to 1.5.\n\nExclusion Criteria:\n\n1. Any concurrent illness, which affects the bone marrow.\n2. Any concomitant medication that affects the bone marrow.\n3. Previous stem cell therapy.\n4. Any lymphoproliferative disease.\n5. Riluzole with 4 weeks of study entry and at any time during the study.\n6. Hemophiliacs or subjects with bleeding disorders.\n7. Known hypersensitivity to fetal bovine serum\n8. HIV infection.\n9. Serum creatinine \\> 3.0 in subjects not on hemodialysis.\n10. Skin infection at the infusion site or systemic infection\n11. Current smoker.\n12. Active drug or alcohol addiction\n13. Pregnant, planning to become pregnant or not on accepted birth control method if subject is of child bearing potential.\n14. Subjects that are breast feeding.\n15. Any condition that the Principal Investigator considers would render the subject unfit for the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "autologous bone marrow-derived stem cells", "targeting_mechanism": "Stem cells differentiate into support cells such as astrocytes and oligodendrocytes, which benefit degenerating motor neurons by producing growth factors and anti-inflammatory cytokines, providing nutrients and buffering excessive glutamate.", "targeting_mechanism_pmid": "32043626", "animal_results": "In SOD1G93A mice, intravenous injection of hBM-MSC expressing Ngn1 resulted in increased lifespan of 3 days, delayed disease onset of 5 days and reduced motor neuron loss.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02269436", "title": "A follow-on Study to Assess Long-term Safety and Tolerability of i.c.v Administration of sNN0029 in Patients With ALS", "phase": "PHASE1", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Newron Sweden AB", "summary": "This is an open-label, follow-on phase 1 study to assess the long-term safety and tolerability of continuous i.c.v administration of 4 \u03bcg sNN0029/day in patients with ALS who previously participated in study sNN0029-003", "interventions": [{"type": "DRUG", "name": "sNN0029 infusion solution"}], "start_date": "2015-01", "url": "https://clinicaltrials.gov/study/NCT02269436", "target_entities": ["GDNF"], "locations": [{"facility": "Philip Van Damme", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Leonard van den Berg", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Previous participation in sNN0029-003 with completion of 12 weeks study without clinically significant safety concerns\n2. Intact continuity of the Medtronic SynchroMed\u00ae II Infusion System as judged by X-ray of head and abdominal area\n3. Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria\n4. Patient has been given written and verbal information about the continuation study, has had the opportunity to ask questions about the study, and understands the time and procedural commitments\n5. Patient has given oral and / or signed consent (written) to participate in the study. In the event that a patient who gives oral informed consent is not physically able to sign the informed consent form (ICF) due to disease progression, a witness may sign the informed consent form on the patient's behalf\n\nExclusion Criteria:\n\n1. Hypertension defined as blood pressure \\>160 mmHg systolic or \\>90 mmHg diastolic\n2. Ophthalmological examination (fundus photography, visual acuity and perimetry) with any clinically significant findings that imply safety concerns for this study.\n3. Diagnosis of diabetes mellitus\n4. Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that cannot be not managed optimally due to:\n\n   * Anatomical factors at or near the implant site (e.g., vascular abnormalities, neoplasms, or other abnormalities)\n   * Underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver disease, or other medical conditions)\n5. Presence of additional risk factors for thromboembolism such as obesity (Body mass index \\[BMI\\] \\> 35) or use of oestrogens including combined contraceptive pills\n6. Clinically significant abnormalities in haematology or clinical chemistry parameters as assessed by the investigator\n7. Ongoing medical condition that according to the investigator would interfere with the conduct and assessments in the study. Examples are medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of safety and efficacy of investigational product or device performance, or would compromise the ability of the patient to undergo study procedures (e.g., MRI), or to give informed consent\n8. For women only: pregnant, breast feeding and/or for fecund women unwillingness to use adequate contraception during the trial such as:\n\n   * Established use of oral, injected or implanted hormonal methods of contraception that do NOT contain oestrogens\n   * Placement of an intrauterine device\n   * Barrier methods of contraception: Condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "sNN0029", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "In SOD1G37R ALS mice, spinal subpial delivery of AAV9 shRNA-SOD1 silencing vector resulted in potent transduction of neuronal and glial cells throughout the spinal cord and blocked motoneuron degeneration.", "animal_results_pmid": "31873312", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02469675", "title": "Brain and Nerve Stimulation for Hand Muscles in Spinal Cord Injury and ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Bronx VA Medical Center", "summary": "Most neurological injuries such as spinal cord injuries (SCI) and amyotrophic lateral sclerosis (ALS) spare a portion of nerve circuitry. Strengthening spared nerve circuits may be an important method to improve functional recovery.\n\nIn this study, the investigators aim to use non-invasive magnetic and electrical stimulation to strengthen motor circuits between the brain and hands. Magnetic stimulation will be used over the motor cortex (scalp). Two methods of electrical stimulation will be compared: stimulation of the median nerve at the wrist; or direct stimulation of the cervical spinal cord across the skin on the back of the neck. Several different combinations of magnetic and electrical stimulation will be compared to find the conditions that best strengthen nerve circuits between the brain and hands - \"Fire Together, Wire Together\".\n\nPLEASE NOTE, THIS IS A PRELIMINARY STUDY. This study is testing for temporary changes in nerve transmission and hand function. THERE IS NO EXPECTATION OF LONG-TERM BENEFIT FROM THIS STUDY. If we see temporary changes in this study, then future studies would focus on how to prolong that effect.", "interventions": [{"type": "DEVICE", "name": "Transcranial magnetic stimulation"}, {"type": "DEVICE", "name": "Median nerve stimulation"}, {"type": "DEVICE", "name": "Cervical transcutaneous stimulation"}], "start_date": "2015-06", "url": "https://clinicaltrials.gov/study/NCT02469675", "target_entities": ["motor_circuit_strengthening"], "locations": [{"facility": "James J. Peters VA Medical Center, Bronx, NY", "city": "The Bronx", "state": "New York", "country": "United States", "status": "", "lat": 40.84985, "lon": -73.86641}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Males or females age 21-65 years;\n* No history of serious neurological injury or disease; OR\n* Chronic (\\>12 months since injury) incomplete SCI between levels C2-C8 or diagnosis of definite or probable ALS;\n* Incomplete weakness of left or right hand muscles: score of 3 or 4 (out of 5) on manual muscle testing of finger extension, finger flexion, or finger abduction;\n* Detectable F-wave responses of the left or right abductor pollicis brevis muscle to median nerve stimulation;\n* Detectable motor evoked potentials in left or right abductor pollicis brevis muscle to transcranial magnetic stimulation.\n\nExclusion Criteria:\n\n* Multiple spinal cord lesions;\n* History of seizures;\n* Ventilator dependence or patent tracheostomy site;\n* Use of medications that significantly lower seizure threshold, such as tricyclic antidepressants, amphetamines, neuroleptics, dalfampridine, and bupropion;\n* History of stroke, brain tumor, brain abscess, or multiple sclerosis;\n* History of moderate to severe head trauma (loss of consciousness for greater than one hour or evidence of brain contusion or hemorrhage or depressed skull fracture on prior imaging);\n* History of implanted brain/spine/nerve stimulators, aneurysm clips, ferromagnetic metallic implants, or cardiac pacemaker/defibrillator;\n* Significant coronary artery or cardiac conduction disease;\n* Recurrent history over the last 6 months of autonomic dysreflexia;\n* History of bipolar disorder;\n* History of suicide attempt;\n* Active psychosis;\n* Heavy alcohol consumption (greater than equivalent of 5 oz of liquor) within previous 48 hours;\n* Open skin lesions over the face, neck, shoulders, or arms;\n* Pregnancy;\n* Unsuitable for study participation as determined by study physician.", "sex": "ALL", "min_age": "21 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Transcranial magnetic stimulation, Median nerve stimulation, Cervical transcutaneous stimulation", "targeting_mechanism": "Non-invasive magnetic and electrical stimulation to strengthen motor circuits between the brain and hands, targeting spared nerve circuitry to improve functional recovery.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06607900", "title": "hUC-MSC-sEV-001 Nasal Drops for Neurodegenerative Diseases", "phase": "PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Xuanwu Hospital, Beijing", "summary": "To evaluate the safety and preliminary efficacy of human umbilical cord mesenchymal stem cell-derived small extracellular vesicles hUC-MSC-sEV-001 nasal drops in multiple neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, multiple system atrophy, Lewy body dementia, frontotemporal dementia, and amyotrophic lateral sclerosis.", "interventions": [{"type": "DRUG", "name": "hUC-MSC-sEV-001 nasal drops"}], "start_date": "2025-07-01", "url": "https://clinicaltrials.gov/study/NCT06607900", "target_entities": ["neurodegeneration"], "locations": [{"facility": "Xuanwu Hospital, Capital Medical University", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "86-01-83198277", "contact_email": "haojunwei@vip.163.com", "eligibility": {"criteria": "General Criteria:\n\nInclusion Criteria:\n\n1. Age 18-80 years (inclusive), any gender.\n2. Subjects or their legal guardians voluntarily sign a written informed consent form and are able to comply with the study requirements for dosing and follow-up.\n\nExclusion Criteria:\n\n1. Subjects who have received allogeneic mesenchymal progenitor cell therapy or its derived small extracellular vesicles.\n2. Subjects with abnormal nasal anatomy, nasal damage, severe rhinitis, or other nasal conditions that may affect the administration of the investigational product.\n3. Subjects requiring nasogastric tube insertion.\n4. Suffering from other uncontrolled diseases that may interfere with the study results, including but not limited to severe local infection, systemic infection, or immunodeficiency.\n5. Combined with malignant tumors, hematological malignancies, or other serious systemic diseases.\n6. Clinically significant history of allergic reactions, especially drug allergic reactions.\n7. Severe renal insufficiency: creatinine clearance (CrCl) \\< 30 mL/min (calculated by Cockcroft-Gault formula), or other known severe renal diseases.\n8. Peripheral blood hemoglobin (HGB) \\< 100 g/L, absolute neutrophil count (NEUT) \\< 1.5 \u00d7 10\u2079/L, platelet count (PLT) \\< 100 \u00d7 10\u2079/L, white blood cell count (WBC) \\< 4.0 \u00d7 10\u2079/L or \u2265 12 \u00d7 10\u2079/L, serum albumin \\< 30 g/L; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \u2265 3 \u00d7 upper limit of normal (ULN).\n9. HBsAg positive, or HBcAb positive with HBV DNA positive, hepatitis C virus (HCV) antibody positive, peripheral blood HCV RNA positive, HIV antibody positive; CMV DNA positive, syphilis serology positive.\n10. Contraindications to MRI examination (e.g., metal implants) or inability to tolerate MRI (e.g., claustrophobia).\n11. Women of childbearing potential not intending to use effective contraception during the trial or within 90 days after the last dose and with a positive pregnancy test record; pregnant or lactating women; men who are sexually active during the trial or within 90 days after the last dose and not intending to use effective contraception; or men planning to donate sperm during the trial or within 90 days after the last dose.\n12. Vaccination within 1 month prior to the first dose or planned during the period from enrollment until the end of follow-up.\n13. Participation in other clinical drug studies within the past 30 days.\n14. Any condition that, in the investigator's judgment, may compromise the subject's ability to understand and/or comply with the study procedures and/or follow-up.\n\nAlzheimer's Disease (AD) Specific Criteria:\n\nInclusion Criteria:\n\n1. Probable AD as defined by the 2011 National Institute on Aging-Alzheimer's Association (NIA-AA) criteria.\n2. Clinical Dementia Rating (CDR) score \u2264 1.0.\n3. Subjects have an identified, reliable caregiver.\n4. Stable treatment regimen for at least 1 month prior to dosing.\n\nExclusion Criteria:\n\n1. Major history of significant brain injury with persistent neurological impairment (with or without) or known structural brain abnormalities.\n2. Cognitive impairment due to other causes: central nervous system infection, Creutzfeldt-Jakob disease, traumatic dementia, other physical/chemical factors (e.g., drug intoxication, alcoholism, carbon monoxide poisoning), major systemic illnesses (e.g., hepatic encephalopathy, pulmonary encephalopathy), intracranial space-occupying lesions (e.g., subdural hematoma, brain tumor), endocrine disorders (e.g., thyroid disease, parathyroid disease), vitamin B12 or folate deficiency, or any other known causes.\n\nParkinson's Disease (PD) Specific Criteria:\n\nInclusion Criteria:\n\n1. Diagnosis of PD according to the 2015 Movement Disorder Society (MDS) clinical diagnostic criteria for PD.\n2. Hoehn and Yahr stage \u2264 3.\n3. Stable treatment regimen for at least 1 month prior to dosing.\n\nExclusion Criteria:\n\n1\\. Parkinsonism other than PD, including but not limited to progressive supranuclear palsy (PSP), multiple system atrophy (MSA), vascular parkinsonism, drug-induced parkinsonism, essential tremor, primary dystonia.\n\nMultiple System Atrophy (MSA) Specific Criteria:\n\nInclusion Criteria:\n\n1. Diagnosis of possible or probable multiple system atrophy.\n2. Time since diagnosis \\< 3 years from baseline, with an expected survival of at least 3 years.\n3. Stable treatment regimen for at least 1 month prior to dosing.\n\nExclusion Criteria:\n\n1. Modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I score \u2265 14.\n2. Presence of any condition that, in the investigators' judgment, affects the diagnosis or assessment of MSA.\n\nDementia with Lewy Bodies (DLB) Specific Criteria:\n\nInclusion Criteria:\n\n1. Meets the revised consensus criteria for DLB (Fourth Consensus Report of the DLB Consortium, 2017).\n2. Severity of motor symptoms must be \u2264 stage 3 on the Hoehn and Yahr scale.\n3. Clinical Dementia Rating (CDR) score \u2264 1.0.\n4. Stable treatment regimen for at least 1 month prior to dosing.\n\nExclusion Criteria:\n\n1. Patients currently diagnosed with any primary psychiatric disorder (e.g., schizophrenia, bipolar disorder, or major depressive episode) according to DSM-V.\n2. Patients with clinically significant or unstable systemic illness and exposure to toxicants within the past 5 years.\n\nFrontotemporal Dementia (FTD) Specific Criteria:\n\nInclusion Criteria:\n\n1. Meets the 2017 International Research Society (IRS) diagnostic criteria for FTD.\n2. Clinical Dementia Rating (CDR) score \u2264 1.0.\n3. Stable treatment regimen for at least 1 month prior to dosing.\n\nExclusion Criteria:\n\n1\\. Diagnosis of severe central nervous system diseases other than FTD that may be the cause of the patient's FTD symptoms or may affect the study objectives.\n\nAmyotrophic Lateral Sclerosis (ALS) Specific Criteria:\n\nInclusion Criteria:\n\n1. Diagnosis of Amyotrophic Lateral Sclerosis (ALS) meeting the diagnostic criteria (Revised El Escorial Criteria, 2000 or Airlie House Criteria) at the level of definite, probable, or laboratory-supported probable ALS.\n2. Disease duration \u2265 6 months and \u2264 2 years (from the first occurrence of any ALS symptom).\n3. Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score: each individual item score \u2265 2 points (with the three respiratory items - Dyspnea, Orthopnea, and Respiratory Insufficiency - all scoring 4 points).\n4. Stable treatment regimen for at least 1 month prior to dosing.\n\nExclusion Criteria:\n\n1\\. Diagnosis of non-ALS patients based on clinical presentation and available auxiliary clinical examinations (neurophysiology, MRI or other imaging techniques, laboratory tests, etc.).", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "hUC-MSC-sEV-001", "targeting_mechanism": "Human umbilical cord mesenchymal stem cell-derived small extracellular vesicles that may reduce neuroinflammation and oxidative stress in neurodegenerative diseases.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07127172", "title": "GB-PRIME: An Early Feasibility Study of a Precise Robotically Implanted Brain-Computer Interface for the Control of External Devices", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuralink Corp", "summary": "The GB-PRIME Study is an early feasibility study designed to assess the clinical safety and functionality of the Neuralink N1 Implant and R1 Robot. This study involves participants who have tetraparesis, tetraplegia, or a diagnosis that may lead to these conditions.\n\nThe N1 Implant is a wireless, rechargeable device mounted on the skull, connected to electrode threads that are inserted into the brain by the R1 Robot, which is a robotic device specifically designed for this procedure.", "interventions": [{"type": "DEVICE", "name": "N1 Implant"}, {"type": "DEVICE", "name": "R1 Robot"}], "start_date": "2025-07-31", "url": "https://clinicaltrials.gov/study/NCT07127172", "target_entities": [], "locations": [{"facility": "University College London Hospitals NHS Foundation Trust", "city": "London", "state": "Greater London", "country": "United Kingdom", "status": "RECRUITING", "lat": 51.50853, "lon": -0.12574}, {"facility": "The Newcastle upon Tyne Hospitals NHS Foundation Trust", "city": "Newcastle upon Tyne", "state": "Tyne and Wear", "country": "United Kingdom", "status": "RECRUITING", "lat": 54.97328, "lon": -1.61396}], "contact_phone": "(877) 398-4465", "contact_email": "clinical-team-ct@neuralink.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* (a) A diagnosis of a spinal cord injury, brain stem stroke, or other neurological condition causing the participant to be non-ambulant and with bilateral upper limb motor impairment with no expectation of recovery that significantly or completely impairs the participant's ability to manually control a computer, smartphone or tablet with their hands.\n\nOR (b) A diagnosis of Amyotrophic Lateral Sclerosis (ALS) or other progressive neurological condition where the natural history of the disease is well understood and where there is tetraparesis and the expectation in the view of the participants treating neurologist that the disease will progress such that the participant will meet 1a within 1 year of recruitment.\n\n* Life expectancy \u2265 12 months.\n* Ability to communicate in English\n* Presence of a stable caregiver\n\nExclusion Criteria:\n\n* Moderate to high risk for serious perioperative adverse events\n* Active implanted devices\n* Morbid obesity (Body Mass Index \\> 40)\n* History of poorly controlled seizures or epilepsy\n* History of poorly controlled diabetes\n* Requires magnetic resonance imaging (MRI) for any ongoing medical conditions\n* Acquired or hereditary immunosuppression\n* Use of smoking tobacco or other tobacco products\n* Psychiatric or psychological disorder\n* Brain MRI demonstrating hemorrhage, tumor, distorted or adverse anatomy.\n* Any condition which, in the opinion of the Investigator, would compromise your ability to safely participate in the study or undergo the implantation procedure", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "N1 Implant, R1 Robot", "targeting_mechanism": "A wireless, rechargeable brain-computer interface device with electrode threads implanted in the brain to enable control of external devices.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05829330", "title": "Ambulatory Versus Inpatient Initiation of Home Mechanical Ventilation", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Anne Kathrine Staehr-Rye", "summary": "The purpose of this investigation is to see if outpatient initiation of noninvasive home mechanical ventilation combined with closed telemonitoring and follow-up in patients with amyotrophic lateral sclerosis is non-inferior to initiation during admission to the hospital\n\nThe primary hypothesis is that outpatient intiation of noninvasive home mechanical ventilation combined with closed telemonitoring and follow-up is non-inferior to initiation during hospitalization in patients with amyotrophic lateral sclerosis.", "interventions": [{"type": "OTHER", "name": "Outpatient"}, {"type": "OTHER", "name": "Hospitalization"}], "start_date": "2023-09-20", "url": "https://clinicaltrials.gov/study/NCT05829330", "target_entities": [], "locations": [{"facility": "Department of Anaesthesia, Pain and REspiratory Support", "city": "Glostrup Municipality", "state": "", "country": "Denmark", "status": "", "lat": 55.6666, "lon": 12.40377}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age \\> 18 years\n* Diagnosed with amyotrophic lateral sclerosis\n* Indication for start of non-invasive mechanical ventilation\n\nExclusion Criteria:\n\n* No informed consent\n* Does not understand Danish or English\n* Indication for invasive mechanical ventilation\n* No morning baseline PCO2\n* Hospitalization", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04998305", "title": "TJ-68 Clinical Trial in Patients With Amyotrophic Lateral Sclerosis (ALS) and Muscle Cramps", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hiroshi Mitsumoto", "summary": "The primary objective of the study is to demonstrate the safety and potential efficacy of TJ-68 for improving muscle cramps in participants with ALS based on a two-site, randomized, placebo-controlled double-blind multi-period crossover (N-of-1) study design.", "interventions": [{"type": "DRUG", "name": "TJ-68"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2022-09-30", "url": "https://clinicaltrials.gov/study/NCT04998305", "target_entities": ["muscle_cramps"], "locations": [{"facility": "Mayo Clinic", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Columbia University Irving Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosed with ALS, PMA or PLS based on the El Escorial ALS Diagnostic Criteria or based on more recently revised Gold Coast ALS diagnostic criteria\n* Experiences at least one muscle cramp in any muscle per day\n* Age 20 to 84 years old\n* Forced vital capacity is 45% of normal or greater in a seated position\n* Able to swallow liquid via the mouth or be given via a feeding tube\n* Caregiver available to assist with speaking or writing on behalf of the participant if they are not able to speak or write due to the disease\n* Able to comprehend and willing to give (sign) the informed consent\n* Willing to commute to the study site for the frequent visits, including a screening visit (study visits at the end of week 2, 5, 8 and 11)\n* Taking a stable dose of Riluzole (Rilutek), Edaravone (Radicava), and/or sodium phenylbutyrate/taurursodiol (Relyvrio) for at least a month before randomization and not expected to require dose titration or initiation of these medications during the study period\n* Willing to discontinue over-the-counter (OTC) products containing any peony root, Glycyrrhiza, or both\n* Willing to discontinue Mexiletine, Quinine sulfate, or Ranolazine during the study period\n* Willing to avoid food, beverages, and medications that may induce or inhibit metabolism of enzyme of transporters.\n* Willing to refrain from initiation or dose adjustment of baclofen, gabapentin, pregabalin, and/or memantine during the study period (stable dosing of these medications is allowed).\n* Willing to practice contraceptive measures for male and female patients.\n\nExclusion Criteria:\n\n* History of allergic reactions to peony root, Glycyrrhiza, or FD\\&C Yellow No. 5 (tartrazine)\n* Takes any medication known to increase the risk of pseudoaldosteronism or hypokalemia, including corticosteroids and diuretics (except potassium sparing diuretics, such as spironolactone or amiloride)\n* History of pseudoaldosteronism or hypokalemia or current use of potassium supplementation\n* Screening potassium level 3.4 mEq/L or less\n* Screening diastolic blood pressure (DBP) more than 90 mmHg or systolic blood pressure (SBP) more than 150 mmHg after sufficient rest\n* Screening albumin below normal laboratory level either at the Columbia or Mayo Clinic laboratory\n* Screening bicarbonate or carbon dioxide level less than 29 mmol/L, suggesting metabolic alkalosis\n* Screening sodium level greater than 145 mmol/L, suggesting hypernatremia\n* Unstable or active medical or neurological (other than ALS) diseases which require treatment\n* Failure of Capacity Assessment\n* Not able and/or willing to comprehend and sign the informed consent\n* Not able to speak or write English to complete the primary outcome measure, MCS\n* Taking any experimental medication or unapproved medications directed at treating muscle cramps\n* Those who are pregnant or breast feeding\n* Those who have renal or hepatic impairment", "sex": "ALL", "min_age": "20 Years", "max_age": "84 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "TJ-68", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06973629", "title": "Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)", "phase": "PHASE3", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Brainstorm-Cell Therapeutics", "summary": "The goal of this two-part clinical trial is:\n\n1\\) to evaluate the safety and efficacy of Debamestrocel - MSC-NTF (NurOwn) compared to placebo in participants with early symptomatic ALS and moderate disease presentation in ALS; followed by 2) further evaluation by providing NurOwn to all participants in an open label extension period.\n\nResearchers will compare NurOwn to a placebo (a look-alike substance that contains no drug) to evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS.\n\nParticipants will:\n\nReceive NurOwn or a placebo every 8 weeks for 24 weeks. After that, every participant will receive NurOwn every 8 weeks for an additional 24 weeks.\n\nThey will visit the clinic approximately every 8 weeks for checkups and tests.", "interventions": [{"type": "BIOLOGICAL", "name": "Debamestrocel - MSC-NTF (NurOwn)"}, {"type": "BIOLOGICAL", "name": "Placebo"}], "start_date": "2025-06-30", "url": "https://clinicaltrials.gov/study/NCT06973629", "target_entities": ["neurotrophic_factors"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California San Diego Medical Center", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "University of Southern California", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of California, San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Anschutz Medical Campus School of Medicine", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Nova Southeastern University", "city": "Davie", "state": "Florida", "country": "United States", "status": "", "lat": 26.06287, "lon": -80.2331}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Northwestern Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Sean M. Healey & AMG Center For ALS At Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Temple University Of The Commonwealth System of Higher Education", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}], "contact_phone": "201-488-0460", "contact_email": "ClinicalTrial@Brainstorm-cell.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Male and female participants 18 to 75 years old (inclusive) at Screening Visit 1.\n2. ALS diagnosed as laboratory-supported probable, clinically probable, or definite as defined by the revised El Escorial criteria.\n3. Having onset of ALS symptoms, including muscle weakness, within 24 months from Screening Visit 1.\n4. \u22652 points on each item of the ALSFRS-R at the Screening Visit 1.\n5. \u226445 points on ALSFRS-R total score at Screening Visit 1.\n6. Upright slow vital capacity (SVC) measure \u226565% of predicted for gender, height, and age at Screening Visit 1.\n7. Participants must adhere to highly effective methods of contraception as specified in the study protocol.\n\nExclusion Criteria:\n\n1. Prior stem cell therapy of any kind.\n2. Active participation in any other ALS interventional study.\n3. Inability to lie flat for the duration of IT cell treatment and/or bone marrow biopsy, or inability to tolerate study procedures for any other reason.\n4. Any unstable clinically significant medical condition other than ALS\n5. Any history of malignancy, within the previous 5 years, with the exception of localized skin cancers, cervical cancer in-situ or prostate cancer in-situ (with no evidence of metastasis, significant invasion, or reoccurrence within 3 years of baseline).\n6. Primary brain cancer or cancer with CNS involvement is exclusionary.\n7. Other types of motor neuron disease such as primary lateral sclerosis, progressive muscular atrophy, and progressive bulbar palsy.\n8. Usage of a feeding tube at Screening Visit 1 or Screening Visit 2.\n9. Pregnant women or women currently breastfeeding.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Debamestrocel - MSC-NTF (NurOwn)", "targeting_mechanism": "Mesenchymal stromal cells engineered to secrete neurotrophic factors that provide trophic support to degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Bone marrow-derived mesenchymal stem cells showed potential benefit in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1).", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07209943", "title": "Augmented Reality BCI Longitudinal Study for Persons With ALS, Stroke, TBI and SCI Utilizing Cognixion + Apple Vision Pro", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cognixion", "summary": "The goal of this study is refine the usability of a BCI capable communication platform.\n\nThe study will take place in the United States area and will enroll up to 10 participants with late stage ALS, traumatic brain injury (TBI) or spinal cord injury (SCI) that have assistive communication and computer control needs. Each subject will receive an integrated Cognixion + Apple Vision Pro device that includes an augmented reality brain computer interface and associated communication software. The study duration is 3-4 months for each participant.\n\nThe key questions that will be addressed in this study are:\n\n1. Identify the ability of individuals with target indications to use the integrated Cognixion-Apple Vision Pro system to communicate effectively.\n2. Identify the ability of such individuals to learn to use BCI, ET-BCI and other modalities, and to measure their progress over time.\n3. Identify the effectiveness of the different forms of input supported by the combined Cognixion-Apple Vision Pro system (BCI, eye-tracking) in allowing such individuals to communicate and have agency.\n4. Identify how input such as BCI can be optimized to suit the needs of individuals (e.g., specific frequencies that work best for an individual, SNR with different frequencies, number of targets, length of recording for each frequency) and improve overall usability.\n5. Identify the extent to which personalization through a large language model (LLM) affects communication.\n6. Identify the appropriate capabilities to enable through an agentic communication interface.\n\nKey measures include:\n\nITR - information transfer rate SUS - system usability scale", "interventions": [{"type": "DEVICE", "name": "Cognixion + Apple Vision Pro"}], "start_date": "2025-10-16", "url": "https://clinicaltrials.gov/study/NCT07209943", "target_entities": [], "locations": [{"facility": "Cognixion HQ", "city": "Santa Barbara", "state": "California", "country": "United States", "status": "", "lat": 34.42083, "lon": -119.69819}], "contact_phone": "18053200774", "contact_email": "chris@cognixion.com", "eligibility": {"criteria": "Inclusion criteria:\n\n* Must have a designated on-site support individual who can be trained on the Cognixion system\n* Fluent in understanding English\n* 18 years or older\n* Must have one of ALS, spinal cord injury or chronic brain injury and need an assistive communication device\n* Must be able to engage in volitional eye opening and sustain eye opening independently for at least 30 minutes.\n* Must have a way to communicate apart from using the Cognixion device such as vocalizations, head nod, eye blinks, eyebrow raises, etc. At a minimum, reliable, independent way of communicating \"Yes\" and \"No\"\n\nExclusion criteria:\n\n* Disruption in English comprehension, either due to lack of fluent proficiency or due to a developmental/acquired language disorder (e.g. aphasia)\n* Severely hearing impaired or deaf\n* Sensitivity to flashing lights\n* Claustrophobia related to the Apple Vision Pro with comfort adapter\n* History of epilepsy and/or seizures\n* Vision disorders restricting the visual field such as glaucoma, diplopia (double vision), nystagmus (involuntary eye movements)\n* History of vertigo or other vestibular disorders\n* Scalp that is prone to irritation, inflammation, injury, or infectious process", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02000713", "title": "Cervical Spinal Cord Metabolism and Microstructure in Amyotrophic Lateral Sclerosis(ALS)", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "The investigators want to know if magnetic resonance imaging can accurately provide an early diagnosis of amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "OTHER", "name": "MRI"}, {"type": "OTHER", "name": "Clinical Exam"}], "start_date": "2013-10", "url": "https://clinicaltrials.gov/study/NCT02000713", "target_entities": [], "locations": [{"facility": "University of Michigan Hospital", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1\\. Adults age 18 to 80 years of age.\n\nExclusion Criteria:\n\n1. Do not have active substance abuse\n2. Do not have co-morbid psychiatric disease\n3. Do not have opportunistic central nervous system infection\n4. Do not have cerebrovascular disease\n5. Do not have a contraindication for magnetic resonance imaging(MRI)(e.g. cardiac pacemaker, ferromagnetic or metallic implants).\n6. Are not pregnant\n7. Have not had cervical spinal surgery(neck) -", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01758510", "title": "Safety Study of HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hanyang University Seoul Hospital", "summary": "The purpose of this study is to evaluate the safety of HLA-haplo matched Allogenic Bone Marrow Derived stem cells(\"HYNR-CS-Allo inj\"), through intrathecal delivery for the treatment in patients with amyotrophic lateral sclerosis(ALS).\n\nThis study is an open label, dose up and down study using the 3+3 design to assess the safety of HLA-haplo matched Allogenic Bone Marrow Derived stem cells(\"HYNR-CS-Allo inj\")", "interventions": [{"type": "GENETIC", "name": "HYNR-CS-Allo"}], "start_date": "2012-12", "url": "https://clinicaltrials.gov/study/NCT01758510", "target_entities": ["neurodegeneration"], "locations": [{"facility": "Hanyang University Seoul Hospital, Cell Therapy Center for Neurologic Disorders", "city": "Seoul", "state": "Haengdang-dong, Seongdong-gu", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients between 25 and 80 years old\n* Patients who have both signs of lower motor neuron(LMN) and upper motor neuron(UMN) degeneration by clinical, electrophysiological or neuropathologic examination\n* Patients diagnosed as 'Probable' or 'Definite' ALS according to the World Federation of Neurology El Escorial criteria\n* Patients whose duration of disease is within 5 years from the first diagnosis\n* Patients with ALSFRS-R score within 21 to 46 at screening\n* Patients who can visit to a hospital by walk personally or by protector's help\n* Patients who provide the written consent by oneself or his/her legal representative\n* Patients who has HLA-haplo matched Bone marrow donor\n\nExclusion Criteria:\n\n* Patients who doesn't appropriate to the diagnostic criteria of ALS\n* Patients who are diagnosed as primary lateral sclerosis(PLS) or progressive muscular atrophy(PMA)\n* Patients suspected of adverse effect after stem cell injection(patients suspected of malignant tumor, risk group of psychogenic shock, patients with serious hypertension)\n* Patients with ALSFRS-R score below 21 at screening\n* Patients performed ventilator or tracheostomy at screening\n* Patients performed gastrostomy at screening\n* Patients unable to assess the efficacy of this clinical trial due to unattainable Pulmonary Functional Test(PFT) or patients with suspected 40% or less of Forced vital capacity(FVC) at screening\n* Patients with finding of myocardial infarction or angina pectoris according to ECG, patients who have been performed Stenting or Bypass operation at screening\n* Patients who have taken any other drug for clinical trial within the past 3 months at screening entry\n* Patients with epilepsy\n* Patients with severe renal dysfunction(serum creatinine\u22652.0mg/dl)\n* Patients with severe liver dysfunction(ALT, AST, bilirubin\u2265upper limit of normal X 2)\n* Pregnant woman, lactating woman, female patients who has a pregnancy planning or who doesn't agree with adoption of contraception methods proper medically, male patients who doesn't agree with adoption of contraception methods proper to his partner during participating this study\n* Patients with hemorrhagic tendency at screening\n* Patients with virus infection at screening\n* Patients with a known history of hypersensitivity/allergy to penicillin and streptomycin\n* Patients with previous stem cell therapy\n* Patients diagnosed with cancer\n* Patients who have taken any drug that can effect to bone marrow function\n* Patients with any other neurological disease except ALS\n* Patients with psychotic diseases", "sex": "ALL", "min_age": "25 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "HYNR-CS-Allo", "targeting_mechanism": "HLA-haplo matched allogeneic bone marrow-derived stem cells delivered intrathecally to promote neuroprotection and reduce neuroinflammation in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06454682", "title": "An IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With ALS", "phase": "EARLY_PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "RJK Biopharma Ltd", "summary": "The goal of this clinical trial is to evaluate the safety and efficacy of a single intrathecal injection of RJK002 in patients with Amyotrophic Lateral Sclerosis (ALS). The main questions it aims to answer are:\n\n* The safety, tolerability, and preliminary efficacy of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS)\n* The adeno-associated virus (AAV) viral load, changes of biomarkers in serum and cerebrospinal fluid (CSF), and electromyography (EMG) motor unit counts in subjects with ALS treated with a single intrathecal injection of RJK002.\n\nParticipants will receive a single intrathecal administration of investigational product and a systemic immunomodulatory regimen. There will be 3 cohorts: 3E13 vg/person (3 mL), 6E13vg/person (6 mL), and 1.2E14 vg/person (12 mL). 3 subjects will be enrolled in each dose cohort. The dose level will be escalated sequentially from low to high.", "interventions": [{"type": "DRUG", "name": "RJK002 Intrathecal injection"}], "start_date": "2023-09-11", "url": "https://clinicaltrials.gov/study/NCT06454682", "target_entities": ["gene_therapy_aav_vector"], "locations": [{"facility": "The First Affiliated Hospital Fujian Medical University", "city": "Fuzhou", "state": "Fujian", "country": "China", "status": "", "lat": 26.06139, "lon": 119.30611}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Female or male subjects who are \u2265 18 years of age at screening;\n2. Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);\n3. The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be more than 6 months and less than 2 years (inclusive);\n4. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score \u226530 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be full marks;\n5. The forced vital capacity (FVC) of predicted during the screening period is \u226570% at screening;\n6. Body mass index (BMI) greater than 18 kg/m2 at screening;\n7. Subjects who had not taken riluzole, edaravone, or who had been on a stable dose for \u226530 days at screening and continue to take it during the study period (those who have stopped taking riluzole, edaravone for \u2265 30 days at screening may also be included, in the opinion of the Investigator);\n8. Understand and comply with the trial procedure, voluntarily participate in and be willing to undergo genetic testing, and sign the informed consent (the informed consent is voluntarily signed by the subject or the subject's leagally authorized representative in the case that a subject is legally incapable of providing informed consent).\n\nExclusion Criteria:\n\n1. Subjects with other neurological diseases similar to ALS that affect the evaluation of drug efficacy, such as cervical spondylotic myelopathy, syringomyelia, spinal cord and brain stem tumors, hirayama disease, multifocal motor neuropathy, multiple sclerosis, Guillain-Barre syndrome, Parkinson's disease and dementia;\n2. Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;\n3. Subjects with a history of spinal surgery within the last six months after the onset of ALS;\n4. By cytological analysis at screening, the titer of anti-AAV9 neutralizing antibody was positive (\\>1:100);\n5. AST, ALT, bilirubin, glutamyltransferase, or glutamate dehydrogenase \\>1.5 \u00d7 ULN or serum creatinine (Scr) \\> ULN at screening;\n6. Any medical instability deemed by the Investigator to be clinically significant, including but not limited to the presence of cardiovascular and cerebrovascular, hepatic or renal dysfunction, endocrine, psychiatric, or nervous system abnormalities that the investigator believes would interfere with the overall safety or efficacy of the study;\n7. Subjects with history of convulsive seizures or epilepsy (excluding febrile convulsive seizures in childhood);\n8. History of autoimmune diseases (excluding thyroid diseases), myelodysplastic or myeloproliferative diseases, leukemia or lymphoma, or severe scoliosis;\n9. Subjects who are hospitalized for surgery, pulmonary events, or nutritional support within 2 months prior to the screening period or who planned to undergo hospitalization for major surgery during the study period;\n10. Presence of contraindications to lumbar puncture (including, but not limited to, signs or symptoms of skin infection at the administration site and elevated intracranial pressure), receipt of any active intrathecal therapy, presence of implantable shunt tubes for draining cerebrospinal fluid (CSF), presence of implantable central nervous system (CNS) cannula, or any condition that interferes with CSF collection;\n11. Screening for a record of attempted suicide in the six months prior to the screening visit, who indicated four or five categories of suicidal thoughts on the Columbia-Suicide Severity Rating Suicide Scale (C-SSRS) assessment, or who the Investigator judged to be at risk of suicide attempts;\n12. Active infection requiring systemic antiviral or antibacterial therapy at any time between 4 weeks prior to the start of screening and dosing; Active but untreated viral or bacterial infection at any time between 4 weeks prior to the start of screening and dosing; Any febrile illness developed during the 2 weeks prior to the start of screening until treatment was given;\n13. Suspect or have a history of alcohol or drug abuse;\n14. Hepatitis B virus surface antigen, hepatitis C virus antibody, syphilis, or human immunodeficiency virus antibody positive at screening;\n15. Pregnant or lactating female subjects, or subjects of childbearing potential (including heterosexual male subjects and their fertile female partners), who have pregnancy plans during the study period or do not want to use effective contraception during the study period;\n16. Subjects who have been received any stem cell therapy or gene therapy prior to screening;\n17. Subjects vaccinated within 2 weeks prior to screening or expected to be vaccinated during the study period;\n18. Subjects with concomitant or ongoing immunosuppressive therapy within 90 days prior to IP administration;\n19. Subjects who have participated in another clinical trial and received any investigational therapy within 3 months prior to screening;\n20. Subjects who are deemed inappropriate by the investigator to participate in this clinical trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RJK002", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06051123", "title": "Effects of Probiotics in Amyotrophic Lateral Sclerosis-Frontotemporal Dementia Spectrum Disorder (ALS-FTDSD) Patients", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Centre hospitalier de l'Universit\u00e9 de Montr\u00e9al (CHUM)", "summary": "The aim of this study is to assess the impact of a probiotic formulation on participants with ALS-FTDSD. It is hypothesized that participants given the probiotics will have different lipid profiles compared to participants receiving the placebo at different time points.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Probiotic"}, {"type": "DIETARY_SUPPLEMENT", "name": "Placebo"}], "start_date": "2024-01-01", "url": "https://clinicaltrials.gov/study/NCT06051123", "target_entities": ["microbiome"], "locations": [{"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "RECRUITING", "lat": 45.94541, "lon": -66.66558}, {"facility": "Centre Hospitalier de l'Universit\u00e9 de Montr\u00e9al", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}, {"facility": "University of Saskatchewan, Clinical Trials Support Unit", "city": "Saskatoon", "state": "Saskatchewan", "country": "Canada", "status": "RECRUITING", "lat": 52.13238, "lon": -106.66892}], "contact_phone": "514-890-8000", "contact_email": "amelie.bujold.chum@ssss.gouv.qc.ca", "eligibility": {"criteria": "Inclusion criteria:\n\nParticipants must meet all of the following inclusion criteria to be eligible for enrolment into the study:ALS-FTDSD participants\n\n1. Aged 18 years old or greater.\n2. Diagnosis of ALS by El Escorial Criteria revised (possible, probable, probable with lab support and definite).\n3. Onset of weakness or speech impairment no more than 24 months before randomization.\n4. ALSFRS-R equal or superior to 24/48 at screening, with no more than one subscore under 2/4.\n5. SVC greater than or equal to 60% predicted for sex, age and height at screening.\n6. Note on FTD Symptoms: The presence of FTD symptoms is not a requirement for inclusion in this study. Participants with a diagnosis of ALS, whether or not accompanied by FTD symptoms, are eligible for inclusion. No prior or screening diagnosis of FTDSD is required.\n7. Subject has an informant/caregiver who has frequent and sufficient contact to provide accurate information about the patient's cognitive abilities and behaviors to complete the ALS-CBS.\n8. Participants apt to comprehend and sign the ICF.\n9. Participants of child-bearing potential must have a negative serum pregnancy test at screening and agree to use a medically approved method of birth control for the duration of the study. All hormonal birth control must have been in use for a minimum of three months. Acceptable methods of birth control include:\n\n   * Abstinence or agrees to use contraception if planning to become sexually active.\n   * Hormonal contraceptives including oral contraceptives, hormone birth control patch, vaginal contraceptive ring, injectable contraceptives, or hormone implant\n   * Double-barrier method\n   * Intrauterine devices\n   * Non-heterosexual lifestyle or agrees to use contraception if planning on changing to heterosexual partner(s)\n   * Vasectomy of partner at least 6 months prior to screening\n10. Willing to maintain eating habits throughout the study.\n11. Willing to refrain from consuming probiotic supplements and food containing added probiotics and/or prebiotics (e.g., yogurts with live, active cultures or supplements) from the moment of screening until the end of the study.\n\nHealthy controls:\n\n1. Aged 18 years old or greater.\n2. Able to comprehend and willing to sign ICF.\n3. Participants of child-bearing potential must have a negative serum pregnancy test at screening and agree to use a medically approved method of birth control for the duration of the study. All hormonal birth control must have been in use for a minimum of three months. Acceptable methods of birth control include:\n\n   * Abstinence or agrees to use contraception if planning to become sexually active.\n   * Hormonal contraceptives including oral contraceptives, hormone birth control patch, vaginal contraceptive ring, injectable contraceptives, or hormone implant\n   * Double-barrier method\n   * Intrauterine devices\n   * Non-heterosexual lifestyle or agrees to use contraception if planning on changing to heterosexual partner(s)\n   * Vasectomy of partner at least 6 months prior to screening\n4. Willing to maintain eating habits throughout the study.\n5. Willing to refrain from consuming probiotic supplements and food containing added probiotics and/or prebiotics (e.g., yogurts with live, active cultures or supplements) from the moment of screening until the end of the study.\n\nInformant/caregiver\n\n1. Aged 18 years old or greater.\n2. Has frequent and sufficient contact to provide accurate information about the patient's cognitive abilities and behaviors to complete the ALS-CBS.\n3. Able to comprehend and willing to sign ICF\n\nExclusion criteria:\n\nSubjects with any of the following characteristics/conditions will not be included in the study:\n\nALS-FTDSD participants\n\n1. Use of respiratory support (non-invasive ventilation or mechanical respiratory support) at screening.\n2. Significant medical condition or behavioral issues that could interfere with participation in the clinical trial in the principal investigator's opinion.\n3. Use of a feeding tube at randomization.\n4. Use of lipid-lowering drugs for less than 3 months before randomization.\n5. Introduction of lipid-lowering drug unless it is due to the event of acute coronary syndrome or stroke as per Canadian guidelines.\n6. Use of edaravone with stable dosage for less than 2 months before randomization.\n7. Use of riluzole with stable dosage for less than 1 month before randomization.\n8. Introduction of edaravone or riluzole during the clinical trial.\n9. Immunodeficiency (immune-compromised and immune-suppressed participants, e.g., AIDS, lymphoma, participants undergoing long-term corticosteroid treatment, chemotherapy and allograft participants).\n10. Pregnancy (as per serum HCG pregnancy test at screening), planning to be pregnant or currently breastfeeding.\n11. Use of probiotics other than the study medication in the month prior to randomization. Note: participants could be eligible to participate after a 4-week washout period.\n12. Use of any antibiotic drug in the month prior to randomization. Note: participants could be eligible to participate after a 4-week washout period.\n13. Milk and soy allergy, or severe lactose intolerance.\n14. Currently enrolled in another clinical trial.\n\nHealthy controls:\n\n1. Use of lipid-lowering drugs for less than 3 months before randomization.\n2. Introduction of lipid-lowering drug unless it is due to the event of acute coronary syndrome or stroke as per Canadian guidelines.\n3. Pregnancy (as per serum HCG pregnancy test at screening), planning to be pregnant or currently breastfeeding. Note: participants will not take the IP or placebo, but pregnancy is a major factor that could affect lipidomic profiling.\n4. Use of probiotics in the month prior to day 0 of the study (visit 2). Note: participants could be eligible to participate after a 4-week washout period. Although participants will not consume any study product, we aim to maintain environmental factors comparable to the ALS-FTDSD group.\n5. Use of any antibiotic drug in the month prior to day 0 of the study (visit 2). Note: participants could be eligible to participate after a 4-week washout period. Although participants will not consume any study product, we aim to maintain environmental factors comparable to the ALS-FTDSD group.\n6. Milk and soy allergy, or severe lactose intolerance. Note: Although participants will not consume any study product, we aim to maintain environmental factors comparable to the ALS-FTDSD group. We aim to exclude allergies, so participants are maintained on standard diet.\n7. Currently enrolled in another clinical trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Probiotic formulation", "targeting_mechanism": "Modulation of gut microbiota composition to reduce dysbiosis and associated neuroinflammation in ALS.", "targeting_mechanism_pmid": "28129947", "animal_results": "Dysbiosis in ALS is linked to alterations in microbial composition and metabolic dysfunction; targeting the microbiome through probiotics may alleviate disease progression.", "animal_results_pmid": "32501768", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03929068", "title": "Sinemet for Spasticity and Function in Amyotrophic Lateral Sclerosis and Primary Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Washington University School of Medicine", "summary": "Motivated by the success of dopaminergic drugs in treating rigidity associated with Parkinson's disease, some neurologists have used carbidopa-levodopa (Sinemet) to attempt to improve spasticity in ALS and PLS patients. However, data on the efficacy of carbidopa/levodopa is limited. Given the limited data and potential to improve the quality of life of these patients, the effectiveness of carbidopa-levodopa in ALS and PLS patients with severe spasticity should be studied. The investigators hypothesis is that administration of carbidopa-levodopa will improve spasticity in ALS and PLS patients.", "interventions": [{"type": "DRUG", "name": "carbidopa-levodopa"}, {"type": "DRUG", "name": "Placebo Oral Tablet"}], "start_date": "2019-05-13", "url": "https://clinicaltrials.gov/study/NCT03929068", "target_entities": ["dopamine_pathway"], "locations": [{"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of ALS or PLS\n* Age greater than 18 years\n* Clinically significant spasticity.\n\nExclusion Criteria:\n\n* Individuals currently taking carbidopa-levodopa or with known hypersensitivity of any component of carbidopa-levodopa\n* Narrow-angle glaucoma\n* Current use of a non-selective monoamine oxidase inhibitor (MAOI)\n* History of malignant melanoma or suspicious skin lesions\n* History of depression, suicidal ideation, or psychosis\n* History of myocardial infarction, ventricular arrhythmia, or severe cardiopulmonary disease\n* Uncontrolled hypertension\n* Asthma\n* Renal disease\n* Hepatic disease\n* Endocrine disease\n* History of peptic ulcer\n* Pregnant and/or breastfeeding\n* Current participation in another interventional study", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "carbidopa-levodopa", "targeting_mechanism": "Dopaminergic agonist that enhances dopamine neurotransmission to reduce spasticity by targeting dopamine deficiency pathways.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "Parkinson's disease", "repurposed_from_pmid": "31801298"}}
{"nct_id": "NCT06689982", "title": "Tofacitinib in Patients With Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Beijing Tiantan Hospital", "summary": "Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease, of which motor-neuron's degeneration may be associated with neuroinflammation. Tofacitinib is a Janus kinase (JAK) inhibitor that affects cellular hematopoiesis and cellular immune function. At the same time, tofacitinib is suitable for rheumatoid arthritis, psoriatic arthritis, and ankylosing spondylitis. This study is a single center, single arm, proof of concept, clinical trial study, and it is planned to use tofacitinib to carry out a clinical trial to observe the treatment effect of ALS patients.", "interventions": [{"type": "DRUG", "name": "Tofacitinib tablets"}], "start_date": "2024-12-01", "url": "https://clinicaltrials.gov/study/NCT06689982", "target_entities": ["JAK", "neuroinflammation"], "locations": [{"facility": "Beijing Tiantan Hospital", "city": "Beijing", "state": "", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "0086-010-67092222", "contact_email": "yilong528@aliyun.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* 18 years old\u2264 age\u2264 75 years old, males or females;\n* Forced vital capacity \u2265 60% of predicted vital capacity during the screening period;\n* The diagnosis conforms to the diagnostic criteria for amyotrophic lateral sclerosis on the Gold Coast;\n* Based on the analysis of whether the subject significantly deviates from the baseline healthy elderly population, whole exome sequencing (WES) revealed that the embryonic lineage etiology was interpreted as CD8+ T lymphocyte group and Th1 highly activated, and patients with pathways related to neural repair and energy metabolism pathways that were not significantly inhibited;(1.Obtain genomic data of patient embryonic samples (blood or oral swab) through whole exome sequencing (WES) technology;2.Utilizing the Damage Assessment of Genome shotgun (DAGG) system developed by the Turing Darwin Laboratory team, rare coding mutations in patient genomic data are converted into an activity profile of signaling pathways (APSP). If the patient's embryonic genomic interpretation of APSP compared to the baseline healthy population's embryonic APSP shows high activation of CD8+ T lymphocyte group and Th1 activity, and non-significant inhibition of Treg, neural repair, and energy metabolism pathways, the patient may be included in the clinical trial;3.This step is analyzed by the Turing Darwin Laboratory team, and based on the entry criteria of the clinical trial (high activation of CD8+ T lymphocyte group and Th1 activity, and non-significant inhibition of Treg, neural repair, and energy metabolism pathways), a modeling setting for the APSP score threshold for ALS will be established. Patients above the threshold can be included in the trial.\n* Subjects or their legal representatives clearly understand and voluntarily participate in the study and sign the informed consent form;\n* Subjects (including male subjects) are willing to have no birth plan and voluntarily take effective contraceptive measures during the entire study period and within 3 months after the end of the study, and have no plan to donate sperm or eggs.\n\nExclusion Criteria:\n\n* Patients who cannot cooperate with the clinical trial project cycle as determined by professional medical staff;\n* Individual whose use of tofacitinib is forbidden;\n* Absolute lymphocyte counts \\< 500 cells /mm\\^3, absolute neutrophil counts (ANC) \\< 1000 cells /mm\\^3 or hemoglobin level \\< 9 g/dL;\n* Serious infection;\n* Positive HIV test or history of positive test;\n* Positive hepatitis C virus antibody or positive test history;\n* Hepatitis B active infection (hepatitis B surface antigen positive and/or serum HBV DNA positive or serum HBV DNA \\> 2 \u00d7 10\\^8 IU/mL;\n* Positive syphilis test result or positive test history;\n* Lumbar spine diseases or malformation;\n* Have other conditions known to be associated with motor neuron dysfunction that may confuse or obscure an ALS diagnosis;\n* Other psychiatric disorders diagnosed according to DSM-V diagnostic criteria, or significant suicide intent;\n* With severe hepatic insufficiency, renal insufficiency or severe cardiac insufficiency (severe hepatic insufficiency refers to ALT value\u22652.0 times the upper limit of normal value or AST value\u22652.0 times the upper limit of normal value; severe renal insufficiency refers to CRE\u22651.5 times the upper limit of normal value or eGFR\\<40mL/min/1.73m\\^2; severe cardiac insufficiency refers to NYHA class 3-4);\n* Permanently dependent on ventilator-assisted ventilation;\n* Individual who have difficulty communicating verbally to the extent that they are unable to communicate, understand or follow instructions normally, and are unable to cooperate with treatment and evaluation;\n* History of alcohol and drug abuse;\n* Patients who are pregnant, breast-feeding, or who are likely to become pregnant and plan to become pregnant;\n* Patients participating in other clinical trials or using other biological agents, drugs, or devices under investigation;\n* Unable to be cooperative and complete the follow-up due to other reasons.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tofacitinib", "targeting_mechanism": "JAK (Janus kinase) inhibitor that suppresses neuroinflammation by reducing cellular hematopoiesis and immune function, targeting the role of neuroinflammation in motor neuron degeneration.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01849770", "title": "Mexiletine in Sporadic Amyotrophic Lateral Sclerosis (SALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Washington", "summary": "The purpose of this research is to find out if mexiletine is safe and effective in people with Amyotrophic Lateral Sclerosis (ALS). In this trial, participants will be taking either 300 milligrams per day of mexiletine, 900 milligrams per day of mexiletine or placebo (non-active study drug). The safety and efficacy of these doses will be compared to see if one dose is better than the other.", "interventions": [{"type": "DRUG", "name": "Mexiletine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2013-07", "url": "https://clinicaltrials.gov/study/NCT01849770", "target_entities": ["Sodium channel"], "locations": [{"facility": "UCLA, Neuromuscular Research Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of Iowa", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts (Worcester) Memorial Medical Center", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Washington University Medical School", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "SUNY Upstate Medical Center", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "University of Texas Southwestern Medical Center at Dallas", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Sporadic Amyotrophic Lateral Sclerosis (SALS) diagnosed as possible, laboratory-supported probable, probable, or definite ALS as defined by revised El Escorial criteria.\n* Age 18 years or older.\n* Disease duration \u2264 36 months from ALS symptom onset.\n* Capable of providing informed consent and following trial procedures.\n* Subjects must not have taken riluzole for at least 30 days or be on a 50 milligrams twice daily dose of riluzole for at least 60 days prior to randomization (riluzole-na\u00efve subjects are permitted in the study).\n* Subjects must not have taken medication for muscle cramping such as cyclobenzaprine, baclofen, carisoprodol, or methocarbamol, for at least 30 days prior to randomization or be on a stable dose for at least 60 days prior to randomization.\n* Geographic accessibility to the site.\n* Women must not become pregnant for the duration of the study and must be willing to use two contraceptive therapies and have a negative pregnancy test throughout the course of the study.\n* Slow vital capacity (SVC) measure greater than or equal to 50% of predicted for gender, height, and age at the screening visit.\n* Subjects medically able to undergo lumbar puncture (LP) as determined by the investigator (for example, no bleeding disorder, allergy to local anesthetics, a skin infection at or near the LP site, or evidence of high intracranial pressure).\n* Must be able to swallow capsules throughout the course of the study, according to Principal Investigator (PI) judgment.\n* Must have a caregiver assist with dispensing the study drug.\n\nExclusion Criteria:\n\n* Invasive ventilator dependence, such as tracheostomy.\n* Creatinine level greater than 1.5 milligram/deciliter.\n* Serum glutamic oxaloacetic transaminase or (aspartate transaminase) / serum glutamic pyruvic transaminase (alanine aminotransferase) greater than 3 times the upper limit of normal at screening.\n* History of known sensitivity or intolerability to mexiletine or lidocaine.\n* Any history of either substance abuse within the past year, unstable psychiatric disease, cognitive impairment, or dementia.\n* Clinically significant conduction abnormalities on electrocardiogram or a known history of cardiac arrhythmia.\n* Known history of epilepsy.\n* Known history of congestive heart failure (CHF) or history of myocardial infarction within the past 24 months.\n* Use of mexiletine for 60 days prior to Baseline Visit.\n* Exposure to any other experimental agent (off-label use or investigational) including high dose creatine (greater than 10 grams a day) within 30 days prior to Baseline Visit.\n* Use of amiodarone, flecainide, duloxetine, tizanidine, or clozapine.\n* Pregnant women or women currently breastfeeding.\n* Placement of Diaphragm Pacing System (DPS) device less than 60 days prior to Baseline Visit.\n* Planned DPS device implantation after Baseline Visit.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Mexiletine", "targeting_mechanism": "Mexiletine is a local anesthetic with sodium channel blocking properties that may protect motor neurons.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00324454", "title": "Levetiracetam for Cramps, Spasticity and Neuroprotection in Motor Neuron Disease", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Duke University", "summary": "Levetiracetam (Keppra) is used to treat partial onset seizures. Its biological effects suggest it might also be useful in treating 3 aspects of human motor neuron diseases (MNDs) for which no effective therapy exists: cramps, spasticity, and disease progression.", "interventions": [{"type": "BIOLOGICAL", "name": "Levetiracetam"}], "start_date": "2006-05", "url": "https://clinicaltrials.gov/study/NCT00324454", "target_entities": ["gaba_pathway"], "locations": [{"facility": "Duke University ALS Clinic - 932 Morreene Road", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with MNDs (ALS, PLS or PMA)who have cramps with average severity 50/100 points, are able to provide informed consent, have normal renal function and are on a stable riluzole dose.\n\nExclusion Criteria:\n\n* Pregnancy; unstable medical illness, dementia; drug abuse or non-compliance", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Levetiracetam", "targeting_mechanism": "Levetiracetam is an anti-seizure medication whose biological effects suggest potential neuroprotective mechanisms for treating cramps, spasticity, and disease progression in motor neuron disease.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00596115", "title": "Treating Amyotrophic Lateral Sclerosis (ALS) With R(+) Pramipexole Dihydrochloride Monohydrate at 60 mg/Day", "phase": "Expanded Access", "status": "TEMPORARILY_NOT_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "summary": "R(+) pramipexole dihydrochloride monohydrate \\[R(+)PPX\\], an experimental neuroprotective drug, is provided in this open label extension study to ALS patients who have participated in earlier clinical protocols.", "interventions": [{"type": "DRUG", "name": "R(+) pramipexole dihydrochloride monohydrate"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT00596115", "target_entities": ["Dopamine D3 receptor"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Definite diagnosis of ALS\n\nExclusion Criteria:\n\n* No prior participation in R(+)PPX clinical studies", "sex": "ALL", "min_age": "30 Years", "max_age": "80 Years", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "R(+) pramipexole dihydrochloride monohydrate", "targeting_mechanism": "R(+) pramipexole is a dopamine receptor agonist with experimental neuroprotective properties in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01551940", "title": "Toxin Treatment for Amyotrophic Lateral Sclerosis (ALS) Related Sialorrhea", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hospices Civils de Lyon", "summary": "Evaluation of the decrease of the secretion of saliva in patients with amyotrophic lateral sclerosis by a local ultrasound-guided bilateral injection of botulinum toxin type A in parotids and submandibular glands. The investigators want to demonstrate 1 month after the injection, by a multicenter French randomized double blind study, an improvement of at least 25 % of the functional embarrassment due to saliva, estimated with a visual analogue scale, a decrease of the quantity of saliva and a decrease of the embarrassment for the main caregiver.", "interventions": [{"type": "DRUG", "name": "Botox injection"}, {"type": "DRUG", "name": "Placebo injection"}], "start_date": "2012-02", "url": "https://clinicaltrials.gov/study/NCT01551940", "target_entities": ["acetylcholine_signaling"], "locations": [{"facility": "D\u00e9partement de Neurologie, H\u00f4pital de l'H\u00f4tel-Dieu, CHU d'Angers", "city": "Angers", "state": "", "country": "France", "status": "", "lat": 47.47156, "lon": -0.55202}, {"facility": "Centre SLA, Groupement Hospitalier Est, H\u00f4pital Neurologique Pierre Wertheimer, Hospices Civils de Lyon", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "Service de Neurologie Vastel, H\u00f4pital C\u00f4te de Nacre, CHU de Caen", "city": "Caen", "state": "", "country": "France", "status": "", "lat": 49.18585, "lon": -0.35912}, {"facility": "Service de Neurologie, H\u00f4pital Gabriel Montpied, CHU de Clermont-Ferrand", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "", "lat": 45.77969, "lon": 3.08682}, {"facility": "Centre SLA, Service de Neurologie A et pathologies du mouvement, H\u00f4pital Roger Salengro, CHU de Lille", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Service de Neurologie, H\u00f4pital Dupuytren, CHU de Limoges", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Service de Neurologie, H\u00f4pital de la Timone, CHU de Marseille", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Service de Neurologie, H\u00f4pital Central, CHU de Nancy", "city": "Nancy", "state": "", "country": "France", "status": "", "lat": 48.68439, "lon": 6.18496}, {"facility": "Centre de R\u00e9f\u00e9rence maladies Neuromusculaires, Service de M\u00e9decine Physique et R\u00e9adaptation, H\u00f4pital de l'Archet 1, CHU de Nice", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "F\u00e9d\u00e9ration des Maladies du Syst\u00e8me Nerveux, Groupement Hospitalier Piti\u00e9 Salp\u00eatri\u00e8re, AP-HP", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Service de Neurologie, CHU de Saint-Etienne", "city": "Saint-Etienne", "state": "", "country": "France", "status": "", "lat": 45.43389, "lon": 4.39}, {"facility": "D\u00e9partement de Neurologie, H\u00f4pital de Hautepierre, CHRU de Strasbourg", "city": "Strasbourg", "state": "", "country": "France", "status": "", "lat": 48.58392, "lon": 7.74553}, {"facility": "Service d'Explorations Fonctionelles Neuro-Musculaires, H\u00f4pital Rangueil, CHU de Toulouse", "city": "Toulouse", "state": "", "country": "France", "status": "", "lat": 43.60426, "lon": 1.44367}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age \\> 18 years\n* Obtaining of a written consent after information\n* Diagnosis of probable or certain ALS according to the El Escorial criteria of the World Federation and Neurology Committee on Neuromuscular Diseases\n* Patient having a follow-up in an ALS center\n* Sialorrhea with VAS functional embarrassment \\> or equal at 50/100.\n* Patient beneficiary of Social Security regime\n\nExclusion Criteria:\n\n* Evolving disease associated with predictable survival \\< 1 month\n* Patient having previously received an injection of botulinum toxin in the salivary glands\n* Patient taking the other medical treatments for sialorrhea in the 7 days before the inclusion in the study (scopoderm, trihexyph'nidyle, atropine, ipatropium, amitriptyline, clomipramine, oxybutinine, diphenhydramine, beta-blockers)\n* Patient having benefited from radiotherapy or from surgery on the salivary glands\n* Behavioral problems, dementia or other psychiatric problems\n* Myasthenia\n* Known Pregnancy or absence of contraception recognized as effective, breast feeding", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Botulinum toxin type A", "targeting_mechanism": "Botulinum toxin type A blocks acetylcholine release at the neuromuscular junction to reduce excessive salivation in ALS-related sialorrhea.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02936635", "title": "A Study for Patients Who Completed VITALITY-ALS (CY 4031)", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who had completed the double-blind placebo-controlled study of tirasemtiv in ALS (CY 4031).", "interventions": [{"type": "DRUG", "name": "tirasemtiv"}], "start_date": "2016-10-17", "url": "https://clinicaltrials.gov/study/NCT02936635", "target_entities": ["Troponin complex"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "UC Davis Medical Center", "city": "Sacramento", "state": "California", "country": "United States", "status": "", "lat": 38.58157, "lon": -121.4944}, {"facility": "Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Stanford Hospital and Clinics", "city": "Stanford", "state": "California", "country": "United States", "status": "", "lat": 37.42411, "lon": -122.16608}, {"facility": "University of Colorado Hospital Anschutz Outpatient Pavilion", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "George Washington University Medical Center", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami, Miller School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Carol & Frank Morsani Center for Advanced Health Care - University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "The Emory Clinic", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Indiana University", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "University of Michigan Health System", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Health System", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "St. Louis University, Department of Neurology & Psychiatry", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Barnes-Jewish Hospital", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Dartmouth Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Neurological Institute", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Neurosciences Institute, Neurology - Charlotte", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke Neurological Disorders Clinic", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Department of Neurology, Wake Forest School of Medicine", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Ohio State University Wexner Medical Center", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence Brain and Spine Inst. ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Temple University School of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Vanderbilt University Medical Center - Clinical Research Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology, PA", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "UTHSCSA - First Outpatient Research Unit", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "West Virginia University Hospitals", "city": "Morgantown", "state": "West Virginia", "country": "United States", "status": "", "lat": 39.62953, "lon": -79.9559}, {"facility": "Froedtert Memorial Lutheran Hospital", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "UZ Leuven", "city": "Leuven", "state": "Vlaams Brabant", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "University of Calgary", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "QE II Health Sciences, Nova Scotia Health Authority", "city": "Halifax", "state": "Nova Scotia", "country": "Canada", "status": "", "lat": 44.64269, "lon": -63.57688}, {"facility": "McMaster University Medical Centre", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook Health Sciences Center", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Hopital Notre-Dame/CHUM", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "CHU de Quebec - Univerite' Laval", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Hopital Dupuytren, service de neurologie", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Hopital Gui de chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nice - Hopital Pasteur 2", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Hopital Bretonneau", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "University of Ulm, Department of Neurology", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Hannover Medical School, Department of Neurology", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Charite Campus Virchow-Klinikum, Department of Neurology", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Clinical Research Centre Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "IRCCS Istituto Auxologico Italiano - U.O. Neurologia", "city": "Milan", "state": "Lombardy", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda", "city": "Milan", "state": "Lombardy", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Dipartimento di Neuroscienze \"Rita Levi Moltalcini\" A.O.U. Citta della Salute e della Scienza di Torino P.O. \"Molinette\"", "city": "Turin", "state": "Piedmont", "country": "Italy", "status": "", "lat": 45.07049, "lon": 7.68682}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Santa Maria - Centro Hospitalar Lisboa Norte", "city": "Lisbon", "state": "", "country": "Portugal", "status": "", "lat": 38.72509, "lon": -9.1498}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "King's College Hospital NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal consent is given, a Legal Designee of the patient must sign the ICF form\n* Completed participation on study drug and the Follow-Up Visit in the CY 4031 study\n* Male patients, who have not had a vasectomy AND confirmed zero sperm count, must agree for the duration of their participation in the study to either:\n\n  * Use a condom during sexual intercourse with female partners who are of childbearing potential AND to have female partners use a highly effective means of contraception OR\n  * Abstain from sexual intercourse during participation in the study\n* Female patients who are not post-menopausal (\u2265 1 year) or sterilized, must:\n\n  * Not be breastfeeding\n  * Have a negative pregnancy test\n  * Have no intention to become pregnant during participation in the study AND\n  * Practice sexual abstinence, defined as refraining from intercourse during the duration of the study OR if male partners are not vasectomized with a confirmed zero sperm count, require use of a condom AND use of a highly effective contraceptive measure\n\nExclusion Criteria:\n\n* Has a diaphragm pacing system (DPS) at study entry or anticipate DPS placement during the course of the study\n* Has taken an investigational study drug (other than tirasemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater\n* Use of tizanidine and theophylline-containing medications during study participation\n* Participation or planning to participate in any form of stem cell therapy for the treatment of ALS or another investigational drug", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tirasemtiv", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03049046", "title": "CC100: Phase 1 Multiple-Dose Safety and Tolerability in Subjects With ALS", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Chemigen, LLC", "summary": "Approximately 21 subjects with amyotrophic lateral sclerosis (ALS) will be randomized (6 to 1) to receive by mouth seven morning doses of CC100 or placebo for 7 days. Subjects are required to stay in the Clinic for approximately 9 hours following the first and last dose. Subjects will also have a mid-week clinic visit and will be contacted by phone within 3 to 5 days after the last dose.\n\nFunding Source - FDA OOPD", "interventions": [{"type": "DRUG", "name": "CC100"}, {"type": "DRUG", "name": "Placebos"}], "start_date": "2017-04-07", "url": "https://clinicaltrials.gov/study/NCT03049046", "target_entities": ["cc100"], "locations": [{"facility": "Indiana University, IU Health Physicians Neurology", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "RECRUITING", "lat": 39.76838, "lon": -86.15804}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Have definite or probable ALS with a forced vital capacity of \\>60% predicted.\n* Men must practice a reliable method of birth control during study and for 2 weeks following study. Women must be non-fertile or post-menopausal.\n* Riluzole is allowed if dose has been stable for at least 30 days. Other allowed medications: lipid-lowering drugs, anti-hypertensives, anti-depressants, oral medications for type II diabetes, estrogen replacement therapy, thyroid replacement therapy, antihistamines, antacids, nonsteroidal anti-inflammatory drugs (except indomethacin), histamine H2-receptor antagonists, proton-pump inhibitors, calcium supplements, topical eye medications, and topical antibiotics.\n\nExclusion Criteria:\n\n* Greater than 250 pounds\n* Have serious or unstable illnesses as determine by the investigator.\n* Have current or a history of asthma or severe drug allergies or pollen allergy.\n* Have had serious infectious disease affecting the brain within the preceding 5 years; or have existing evidence of serious infection.\n* Have laboratory test values that are considered clinically significant as determined by the investigators.\n* Have ECG abnormalities that are clinically significant.\n* Have donated blood (a pint or more) or received an experimental drug within 30 days prior to dosing.\n* Have a history of chronic alcohol or drug abuse within the past 2 years.", "sex": "ALL", "min_age": "18 Years", "max_age": "64 Years", "healthy_volunteers": false, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "CC100", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01879241", "title": "Study of Rasagiline in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Ulm", "summary": "The primary objective of the trial is to investigate the survival time (the time from randomization until death or end of the trial) compared between control group and experimental group.\n\nThis is a prospective, multicenter, randomized, stratified, parallel-group, double-blind trial comparing placebo with 1 mg/d rasagiline as add-on therapy to 100 mg riluzole in amyotrophic lateral sclerosis (ALS) in 250 enrolled patients. For entry, the El Escorial Criteria for the diagnosis of ALS will be used. The patients have to be stable on riluzole at least 4 weeks prior to randomization.", "interventions": [{"type": "DRUG", "name": "Rasagiline"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2013-06", "url": "https://clinicaltrials.gov/study/NCT01879241", "target_entities": ["MAOA"], "locations": [{"facility": "Department of Neurology, University of Ulm", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Department of Neurology, Technische Universit\u00e4t M\u00fcnchen", "city": "Munich", "state": "Bavaria", "country": "Germany", "status": "", "lat": 48.13743, "lon": 11.57549}, {"facility": "Department of Neurology, Universty of Regensburg", "city": "Regensburg", "state": "Bavaria", "country": "Germany", "status": "", "lat": 49.01513, "lon": 12.10161}, {"facility": "Department of Neurology, University of Wuerzburg", "city": "W\u00fcrzburg", "state": "Bavaria", "country": "Germany", "status": "", "lat": 49.79391, "lon": 9.95121}, {"facility": "Department of Neurology, Deutsche Klinik f\u00fcr Diagnostik", "city": "Wiesbaden", "state": "Hesse", "country": "Germany", "status": "", "lat": 50.08601, "lon": 8.24435}, {"facility": "Department of Neurology, University of Goettingen", "city": "G\u00f6ttingen", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 51.53443, "lon": 9.93228}, {"facility": "Department of Neurology, Medical School Hannover", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Department of Neurology, University of Rostock", "city": "Rostock", "state": "Mecklenburg-Vorpommern", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Department of Neurology, Universty of Bonn", "city": "Bonn", "state": "Nordrhrein-Westfalen", "country": "Germany", "status": "", "lat": 50.73438, "lon": 7.09549}, {"facility": "Neurologische Universit\u00e4tsklinik Bergmannsheil", "city": "Bochum", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Department of Neurology, Universty of Muenster", "city": "M\u00fcnster", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.96236, "lon": 7.62571}, {"facility": "Department of Neurology, TU Dresden", "city": "Dresden", "state": "Saxony", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Department of Neurology, University of Halle-Wittenberg", "city": "Halle", "state": "Saxony-Anhalt", "country": "Germany", "status": "", "lat": 51.48158, "lon": 11.97947}, {"facility": "Department of Neurology, University of Jena", "city": "Jena", "state": "Thuringia", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Department of Neurology, Humboldt University", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Possible, probable (clinically or laboratory) or definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria\n* Disease duration more than 6 months and less than 3 years (inclusive). Disease onset defined as date of first muscle weakness, excluding fasciculations and cramps\n* Vital capacity more than 50% of normal (slow vital capacity; best of three measurements)\n* Age: \u2265 18 years\n* Continuously treated with 100 mg riluzole for at least four weeks\n* Capable of thoroughly understanding all information given and giving full informed consent according to GCP\n* Women of childbearing age must be non-lactating and surgically sterile or using a highly effective method of birth control and have a negative pregnancy test. Acceptable methods of birth control with a low failure rate i.e. less than 1% per year) when used consistently and correct are such as implants, injectables, combined oral contraceptives, hormonal intrauterine devices (IUDs), or double-barrier methods (condom or diaphragm with spermicidal agent or IUD), sexual abstinence or vasectomized partner\n\nExclusion Criteria:\n\n* Previous participation in another clinical study within the preceding 12 weeks\n* Tracheostomy or assisted ventilation of any type during the preceding three months\n* Gastrostomy\n* Any medical condition known to have an association with motor neuron dysfunction which might confound or obscure the diagnosis of ALS\n* Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment\n* Patients on sympathomimetic agents. This includes pseudoephedrine, phenylephrine, phenylpropanolamine, and ephedrine.\n* Patients on analgesics with serotoninergic properties such as meperidine, tramadol, methadone and propoxyphene.\n* Patients on serotonin reuptake inhibitors (SSRIs). This includes fluoxetine or fluvoxamine.\n* Patients on dextromethorphan, St. John's wort, cyclobenzaprine or other MAO inhibitors (selective or non-selective)\n* Patients taking Antidepressants\n* Confirmed hepatic insufficiency or abnormal liver function (ASAT and/or ALAT greater than 3 times the upper limit of the normal range)\n* Renal insufficiency (serum creatinine more than 2.26 mg/dL)\n* Evidence of major psychiatric disorder or clinically evident dementia precluding evaluation of symptoms\n* Known hypersensitivity to any component of the study drug\n* Liable to be not cooperative or comply with the trial requirements (as assessed by the investigator), or unable to be reached in the case of emergency\n* Female with childbearing potential, if no adequate contraceptive measures are used\n* Pregnancy or breast-feeding females", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Rasagiline", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01999803", "title": "A Safety Study of sNN0029 Administration Via Intracerebroventricular Route to Patients With ALS", "phase": "PHASE1", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Newron Sweden AB", "summary": "This is a phase I, multicentre randomised, double-blind, placebo-controlled trial to assess the safety and tolerability of continuous i.c.v. administration of sNN0029 infusion solution at a dose of 4\u00b5g/day in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "sNN0029"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2014-09", "url": "https://clinicaltrials.gov/study/NCT01999803", "target_entities": ["snn0029"], "locations": [{"facility": "Philip Van Damme", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Leonard van den Berg", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised World Federation of Neurology (WFN) El Escorial criteria.\n* If patients are being treated with riluzole, they must have been on a stable dose for at least the past 30 days prior to screening.\n* The patient is, in the opinion of the investigator, medically fit to undergo the surgery required for stereotactic implantation of the catheter and infusion pump.\n\nExclusion Criteria:\n\n1. Impaired respiratory function judged to pose a risk to the patient during anaesthesia for the device implantation.\n2. Hypertension defined as blood pressure \\>160 mmHg systolic or \\>90 mmHg diastolic.\n3. Values for coagulation parameters including platelet count, normalised prothrombin complex (PK-INR), activated partial thromboplastin time (APTT) outside normal ranges.\n4. Ophthalmological examination (fundus photography, visual acuity and perimetry) with any clinically significant findings that imply safety concerns for this study.\n5. Diagnosis of diabetes mellitus.\n6. History of structural brain disease other than ALS, including tumours and hyperplasia.\n7. An MRI of the brain and cervical spine, and an Magnetic Resonance Angiography (MRA) of the brain with findings of tumours or potential sources of pathological bleedings, or abnormality that may interfere with the assessments of safety or efficacy or that would, in the judgment of the investigator, represent a surgical risk to the patient. If an MRI and/or MRA has been performed within 1 month prior to screening, the results from that examination can be used.\n8. Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection), alters wound healing (e.g., including bleeding disorders), or renders chronic i.c.v. delivery or device implants medically unsuitable.\n9. Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that cannot be not managed optimally due to:\n\n   i. anatomical factors at or near the implant site (e.g., vascular abnormalities, neoplasms, or other abnormalities), ii. underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., haemophilia, Von Willebrand's disease, liver disease, or other medical conditions) iii. administration of any antiplatelet or anticoagulant medication in the preoperative period\n10. A personal history of thromboembolic disease. A family history of thromboembolic disease will prompt a laboratory assessment to exclude hereditary liability before the patient is declared eligible.\n11. Presence of additional risk factors for thromboembolism such as obesity (BMI \\> 35) or use of oestrogens including combined contraceptive pills.\n12. Presence of an implanted shunt for the drainage of CSF or an implanted Central Nervous System (CNS) catheter.\n13. Clinically significant abnormalities in haematology or clinical chemistry parameters as assessed by the investigator.\n14. Serological evidence of Hepatitis B virus (HBV), Hepatitis C virus (HCV) or Human immunodeficiency virus (HIV)\n15. Ongoing medical condition that according to the investigator would interfere with the conduct and assessments in the study. Examples are medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of safety and efficacy of investigational product or device performance, or would compromise the ability of the patient to undergo study procedures (e.g., MRI), or to give informed consent.\n16. Participation in another clinical trial with an investigational drug or device within 3 months prior to screening visit.\n17. For women only: pregnant, breast feeding and/or for fecund women unwillingness to use adequate contraception during the trial such as:\n\n    * Established use of oral, injected or implanted hormonal methods of contraception that do NOT contain oestrogens.\n    * Placement of an intrauterine device.\n    * Barrier methods of contraception: Condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "sNN0029", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02759003", "title": "Nightime NIV Initiation in Amyotrophic Lateral Sclerosis in an Outpatient Setting", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fondazione Salvatore Maugeri", "summary": "In patients with amyotrophic lateral sclerosis (ALS), non-invasive mechanical ventilation (NIV) is usually initiated in an in-hospital regime. The investigators evaluated if NIV initiated in an outpatient setting can be as effective as regards patients' adherence. The investigators also evaluated factors predicting NIV adherence and disease progression.", "interventions": [{"type": "PROCEDURE", "name": "Nightime NIV initiation"}], "start_date": "2011-03", "url": "https://clinicaltrials.gov/study/NCT02759003", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* consecutive patients\n* definite ALS diagnosis\n* aged \\> 18 years\n* in clinically stable condition\n* referred to the ALS outpatient clinics of the Fondazione Salvatore Maugeri Institute of Lumezzane (Brescia) and the Istituto Don Gnocchi Onlus (Milano), Italy for respiratory functional assessment for the purpose of early initiation of NIV\n* no chest infections during the previous 3 months.\n\nExclusion Criteria:\n\n* cognitive impairment\n* refusal to participate\n* severe comorbidities and contraindications to NIV (arrhythmias, cardiac failure, history of pneumothorax)\n* distance from hospital \\> 40 km, travel problems to attend the outpatient clinic", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07093268", "title": "Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Brain Trust Bio", "summary": "The purpose of this study is to investigate the safety and tolerability of intrathecal riluzole in adults with amyotrophic lateral sclerosis.", "interventions": [{"type": "DRUG", "name": "Intrathecal Riluzole"}], "start_date": "2025-08-15", "url": "https://clinicaltrials.gov/study/NCT07093268", "target_entities": ["riluzole"], "locations": [{"facility": "Sunshine Coast University Hospital", "city": "Birtinya", "state": "Queensland", "country": "Australia", "status": "", "lat": -26.74322, "lon": 153.11913}, {"facility": "Flinders Medical Centre", "city": "Bedford Park", "state": "South Australia", "country": "Australia", "status": "", "lat": -35.02204, "lon": 138.56815}], "contact_phone": "+1 857 285 8300", "contact_email": "chen.benkler@gmail.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Men and women aged 18 years or older.\n* Participants are ambulatory with or without an assistive device.\n* Sporadic or familial ALS diagnosis with possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.\n* Slow vital capacity (SVC) measure \u226570% of predicted for gender, height, and age.\n* Medically able to undergo implantation of the SynchroMed II Infusion Pump according to the judgment of the investigator, or the presence of a previously implanted IT pump (not to be used for concurrent IT infusion of another IT agent).\n* Capable of reading and providing informed consent and following study procedures.\n* Geographic accessibility to the study center and the ability to travel to the clinic for study visits by ground transportation.\n* Women must not be able to become pregnant (eg, post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and 3 months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device in place for \u22653 months, barrier method in conjunction with spermicide, or another adequate method.\n* Taking and tolerating oral riluzole 50 mg twice a day for at least 30 days prior screening and willingness to continue oral riluzole throughout duration of the study.\n* Patients may take other drugs approved for treatment of ALS at the dose prescribed by their neurologist.\n\nExclusion Criteria:\n\n* Participants with bulbar-onset ALS\n* Participants at risk of increased bleeding or uncontrolled bleeding during the SynchroMed II Infusion Pump implantation or following explant. This includes but is not limited to:\n\n  1. Anatomical factors at or near the site of implantation;\n  2. Underlying disorders of the coagulation cascade or platelet function (eg, hemophilia, Von Willebrand's disease, liver disease);\n  3. Administration of antiplatelet or anticoagulant medication within 7 days before or after pump implantation (eg, aspirin, clopidogrel bisulfate, rivaroxaban, nonsteroidal anti-inflammatory agents \\[NSAIDs\\]), or\n  4. Use of nutritional supplements (eg, St John's Wort) within 7 days before or after pump implantation.\n* Presence of infection including but not limited to: meningitis, ventriculitis, skin infection, bacteremia, or septicemia.\n* Testing positive for HIV (anti-HIV antibody), HBV (HBV surface antigen) or HCV (anti-HCV antibody; HCV RNA if anti-HCV antibody is positive) at screening.\n* Inability to have the infusion pump implanted \u2264 2.5 cm below the skin surface.\n* Body weight and size unable to accept the infusion pump bulk and weight.\n* Spinal anomalies which would complicate the implantation and fixation of the catheter for IP delivery.\n* Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) value \\> 2.0 times the upper normal.\n* A life expectancy of less than 6 months, based on the judgment of the investigator.\n* Presence of tracheostomy.\n* The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair the ability of the participant to provide informed consent, per investigator judgment.\n* History of active substance abuse within the prior year.\n* At risk for committing suicide per investigator judgment.\n* Clinically significant history of unstable or severe cardiac, oncologic, hepatic, or renal disease, or other medically significant illness.\n* Pregnant women or women currently breastfeeding.\n* Exposure to any investigational drug, device, or biologic within 30 days of screening.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Riluzole", "targeting_mechanism": "Riluzole blocks glutamatergic neurotransmission in the CNS to exert neuroprotective effects.", "targeting_mechanism_pmid": "32847483", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00230074", "title": "A Long-term Extension Study of TCH346 and Placebo Administered Once Daily in Patients With Amyotrophic Lateral Sclerosis(ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Novartis", "summary": "This is a study to evaluate the safety and clinical effects of 4 oral doses of TCH346 compared to placebo in patients with mild or mild to moderate stages of ALS.", "interventions": [{"type": "DRUG", "name": "TCH346"}], "start_date": "2004-11", "url": "https://clinicaltrials.gov/study/NCT00230074", "target_entities": ["tch346"], "locations": [{"facility": "Novartis", "city": "East Hanover", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.8201, "lon": -74.36487}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Completed original protocol, Study No. CTCH346A2211\n* Be capable of satisfying the requirements of the extension protocol and must sign informed consent after the nature of the extension protocol has been fully explained\n\nExclusion Criteria:\n\n* Exclusion criteria as described in the original protocol will remain applicable into the extension protocol\n\nOther protocol-defined exclusion criteria may apply.", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "TCH346", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02868580", "title": "Safety and Tolerability of Antiretroviral (Triumeq) in Patients With Amyotrophic Lateral Sclerosis (ALS).", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuroscience Trials Australia", "summary": "This is a phase 2a open label, multicentre design study to investigate the safety of Triumeq in patients with ALS at 24 weeks post treatment. In this phase 2a study the investigators aim to determine whether a combination of anti-retroviral therapy, Triumeq (dolutegravir 50mg, abacavir 600mg, lamivudine 300mg) is tolerated and safe in patients with ALS. As secondary outcomes, ALSFRS-R, ALSQOL, physical examination, neurophysical parameters and respiratory and muscle function will be evaluated. Blood and urine samples will be stored for possible future analysis for viral activity. Subjects will be screened for the study after signing an approved Informed consent document.", "interventions": [{"type": "DRUG", "name": "Triumeq"}], "start_date": "2016-10", "url": "https://clinicaltrials.gov/study/NCT02868580", "target_entities": ["triumeq"], "locations": [{"facility": "Macquarie Neurology", "city": "North Ryde", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.79677, "lon": 151.12436}, {"facility": "Westmead Hospital", "city": "Parramatta", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.8178, "lon": 151.00348}, {"facility": "Brain and Mind Centre", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}, {"facility": "Calvary Health Care Bethlehem", "city": "Caulfield South", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.89562, "lon": 145.02597}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nSubjects must meet all of the following inclusion criteria to be eligible to participate in this study:\n\n* Age 18-75 years at the time of the screening visit\n* Able to provide informed consent and comply with study procedures\n* Sporadic ALS diagnosed as probable, laboratory-supported probable or definite according to the World Federation of Neurology El Escorial revised criteria as determined by a neurologist with neuromuscular sub-specialty training\n* Diagnosis \\<24 months from date of enrolment\n* (Forced) Vital capacity at least 60% of predicted value for gender, height and age at the screening visit\n* Must be on a stable dose of riluzole for at least 30 days prior to the screening visit.\n* Subject has established care with a neurologist at one of the four specialized ALS clinics involved in the study and will maintain this clinical care throughout the study.\n* Subjects can participate in clinical registries, but will be excluded to this protocol if they are participating in a clinical trial involving additional or investigative treatment exposure.\n\nExclusion Criteria:\n\nA participant will be excluded if he or she has any of the following:\n\n* Dependence on mechanical ventilation at the time of screening\n* Gastrostomy at the time of screening\n* Absence of Upper Motor Neuron Signs\n* Participation in any other investigational drug trial or using investigational drug (within 12 weeks prior to screening)\n* Known hypersensitivity to dolutegravir, abacavir or lamivudine, or to any of the excipients\n* Presence of the HLA-B\\*5701 allele at screening\n* Presence of a monogenic cause of ALS (e.g. known mutation in SOD1, expansion in c9orf72 etc.)\n* History of positive test or positive result at screening for HIV\n* Subjects positive for Hepatitis B at screening (+HBsAg), or anticipated need for Hepatitis C virus (HCV) therapy during the study\\*;\n* Women must not be able to become pregnant (post menopausal for \\>1 year, surgically sterile, adequate contraception) or breastfeed for the duration of the study. Women of childbearing potential must have a negative pregnancy test at screening and be non-lactating\n* Other interventional clinical trial\n* Subject is taking medication contraindicated with Triumeq. Dofetilide (or pilsicainide \\[available in Japan\\]) is prohibited as DTG may inhibit its renal tubular secretion resulting in increased dofetilide concentrations and potential for toxicity.\n* Presence of any of the following clinical conditions at the time of screening:\n\nDrug or alcohol abuse Unstable medical disease (such as unstable angina or chronic obstructive pulmonary disease), or active infectious disease (such as Hepatitis B or C or tuberculosis), or current malignancy Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days of the screening visit. This exclusion criteria is based on a prior psychiatric diagnosis that is unstable as determined by the subject's treating Psychiatrist Dementia as previously diagnosed by a medical practitioner\n\n\u2022 Safety Laboratory Criteria at the screening visit: Alanine aminotransferase (ALT) \\>5 times the upper limit of normal (ULN), OR ALT \\>3xULN Total bilirubin, lactate, triglycerides, amylase, or lipase greater than 2.0 times the upper limit of normal Subject has creatinine clearance of \\<50 mL/min via Cockroft-Gault method Subjects with moderate to severe hepatic impairment (Class B or greater) as determined by Child-Pugh classification; Absolute neutrophil count of \\< 1 x 109/L Platelet concentration of \\< 100 x 109/L Haemoglobin \\< 100g/L", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Triumeq", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05740813", "title": "HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.\n\nRegimen F will evaluate the safety and efficacy of a single study drug, ABBV-CLS-7262, in participants with ALS.", "interventions": [{"type": "DRUG", "name": "ABBV-CLS-7262 Dose 1"}, {"type": "DRUG", "name": "ABBV-CLS-7262 Dose 2"}, {"type": "DRUG", "name": "Matching Placebo"}], "start_date": "2023-03-23", "url": "https://clinicaltrials.gov/study/NCT05740813", "target_entities": ["abbv_cls_7262"], "locations": [{"facility": "Healey Center for ALS at Mass General", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\nExclusion Criteria:\n\n* The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\n  1. Based on the metabolism of the compound, the concomitant use of certain inhibitors and inducers of cytochrome P450 enzymes.\n  2. Any clinically significant ECG abnormalities.\n  3. Clinically significant clinical laboratory abnormalities.", "sex": "ALL", "min_age": "18 Years", "max_age": "100 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ABBV-CLS-7262", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05041114", "title": "SWITCH II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Synchron Medical, Inc.", "summary": "The Synchron Motor Neuroprosthesis (MNP) is intended to be used in subjects with severe motor impairment, unresponsive to medical or rehabilitative therapy and a persistent functioning motor cortex. The purpose of this research is to evaluate safety and feasibility.\n\nThe MNP is a type of implantable brain computer interface which bypasses dysfunctional motor neurons. The device is designed to restore the transmission of neural signal from the cerebral cortex utilized for neuromuscular control of digital devices, resulting in a successful execution of non-mechanical digital commands.", "interventions": [{"type": "DEVICE", "name": "Motor Neuroprosthesis"}], "start_date": "2022-04-21", "url": "https://clinicaltrials.gov/study/NCT05041114", "target_entities": [], "locations": [{"facility": "Sydney Local Health District", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}, {"facility": "Metro North Health", "city": "Brisbane", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.46794, "lon": 153.02809}, {"facility": "Melbourne Health", "city": "Melbourne", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.814, "lon": 144.96332}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Severe motor impairment\n2. Able to give consent\n3. Appropriate candidate for neurointerventional procedure\n4. Able and willing to access all clinical testing and not impeded by geographical location\n5. Proficient in English\n6. Have a study partner\n\nExclusion Criteria:\n\n1. Active condition resulting in immunosuppression\n2. Unsuitable for general anaesthetic\n3. Anaphylactic allergy to contrast media\n4. Allergy to nickel\n5. History of pulmonary embolism\n6. History of recent deep vein thrombosis\n7. Psychiatric or psychological disorder\n8. No study partner or caregiver\n9. Unable to provide evidence of COVID vaccination", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Motor Neuroprosthesis", "targeting_mechanism": "An implantable brain-computer interface that bypasses dysfunctional motor neurons to restore transmission of neural signals from the cerebral cortex for neuromuscular control.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00965497", "title": "Escitalopram (Lexapro) for Depression MS or ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of South Carolina", "summary": "The purpose of this study is to see if escitalopram (Lexapro) improves symptoms of major depressive disorder in patients who have ALS or MS.", "interventions": [{"type": "DRUG", "name": "escitalopram"}], "start_date": "2009-07", "url": "https://clinicaltrials.gov/study/NCT00965497", "target_entities": ["SLC6A4"], "locations": [{"facility": "University of South Carolina School of Medicine", "city": "Columbia", "state": "South Carolina", "country": "United States", "status": "", "lat": 34.00071, "lon": -81.03481}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients between 18 and 70 years of age with documented ALS or MS,\n* DSM-IV episode of non-psychotic Major Depression,\n* \u226514 score on the 17-item HAM-D,\n* Ability to give informed consent.\n\nExclusion Criteria:\n\n* History of psychotic disorders,\n* Psychotic depression,\n* Bipolar depression,\n* Suicide risk,\n* History of substance abuse in the previous 6 months,\n* History of unstable medical disorders,\n* Pregnancy or planning for pregnancy,\n* Severity of ALS or MS that limits participating in the study protocol.", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "escitalopram", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03835507", "title": "Randomized, Double-blind, Safety and Efficacy of Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hanyang University Seoul Hospital", "summary": "Erythropoietin is neuroprotective in animal models of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS). The aim of this study was to determine the safety and feasibility of repetitive high-dose recombinant human erythropoietin (rhEPO) therapy in ALS patients.", "interventions": [{"type": "DRUG", "name": "recombinant human erythropoietin(rhEPO)"}], "start_date": "2016-06-20", "url": "https://clinicaltrials.gov/study/NCT03835507", "target_entities": ["EPO"], "locations": [{"facility": "Hanyang Medical Center", "city": "Seoul", "state": "", "country": "South Korea", "status": "RECRUITING", "lat": 37.566, "lon": 126.9784}], "contact_phone": "+82-2-2290-8367", "contact_email": "jinseok.park0@gmail.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age between 25 to 80\n* upper motor neuron signs and lower motor neuron signs were identified in neurological examination.\n* Meet the revised El Escorial Criteria for clinically possible, probable-laboratory -supported, probable, definite ALS.\n* Disease duration \\< 3 years (Within 3 years from symptom onset)\n* ALSFRS-R score between 21 to 46\n* Patient who can visit an outpatient under the aid of his or her own walking or caregivers.\n* The person who have agreed in writing to participate in this clinical trial by themselves and the legal representative\n* FVC over 50% at screening\n\nExclusion Criteria:\n\n* Person who were not compatible with ALS\n* Patient with PLS or PMA\n* A group of patients who are concerned about the adverse effects of the drug administration (e.g. malignant hypertension,...)\n* ALSFRS-R score below 20 at screening\n* Ventilator user or Tracheostomy state patients at screening\n* Gastrostomy state at screening\n* FVC below 50% at screening or patient who cannot perform FVC test.\n* EKG abnormality, history of coronary stent , CABG at screening\n* Person who was given another clinical trial drug three months prior to screening.\n* History of seizure/ epilepsy\n* Abnormal renal function (serem creatinine \\> 2.0mg/dl)\n* Abnormal liver function(AST/ALT/bilirubin over 2 times the upper normal limit\n* Pregnant\n* Bleeding tendency at screening\n* Infectious disease at screening\n* Drug sensitivity\n* Person who injected erythropoietin 6 months prior to screening\n* Malignant tumor\n* Other neurological disease (stroke, parkinson's disease, dementia...)\n* Psychological disease\n* Hb more than 16g/dL", "sex": "ALL", "min_age": "25 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "recombinant human erythropoietin (rhEPO)", "targeting_mechanism": "Neuroprotective agent that reduces neurodegeneration in animal models of neurodegenerative diseases including ALS", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04082832", "title": "CuATSM Compared With Placebo for Treatment of ALS/MND", "phase": "PHASE2, PHASE3", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Collaborative Medicinal Development Pty Limited", "summary": "Multicenter, randomized, double-blind, placebo controlled study to assess the tolerabilty and efficacy of CuATSM in patients with ALS/MND. Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment.", "interventions": [{"type": "DRUG", "name": "Cu(II)ATSM"}, {"type": "DRUG", "name": "Placebos"}], "start_date": "2019-09-30", "url": "https://clinicaltrials.gov/study/NCT04082832", "target_entities": ["SOD1"], "locations": [{"facility": "Macquarie University", "city": "Macquarie", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": null, "lon": null}], "contact_phone": "(415) 444 9600", "contact_email": "Kay.Noel@colMedDev.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* signed informed consent\n* familial or sporadic ALS/MNS by Awaji-shima Consensus Recommendations\n* not taking riluzole or on stable dose of riluzole for 4 weeks prior to screening visit\n* no prior exposure to agents other than riluzole for treatment of ALS\n* adequate bone marrow reserve, renal and liver function\n* women of childbearing potential must have a negative pregnancy test and be non-lactating\n* women and men with partners of childbearing potential must take effective contraception while on treatment\n\nExclusion Criteria:\n\n* presence of a gastrointestinal disorder (eg, malabsorption) that might jeopardize intestinal absorption of study drug\n* inability to perform seated SVC\n* known immune compromising illness or treatment\n* drug abuse or alcoholism\n* clinically significant or active cardiovascular disease\n* acute or chronic infection\n* diagnosis of malignancy within 2 years prior to screening\n* dementia that may affect patient understanding and/or compliance with study requirements and procedures\n* current use of strong inducers or inhibitors of CYPs 2C19 and 2D6\n* current us of medications (other than riluzole) that are metabolized predominantly by CYP 1A2", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Cu(II)ATSM", "targeting_mechanism": "Copper delivery to the central nervous system that restores SOD1 copper cofactor levels and reduces oxidative stress", "targeting_mechanism_pmid": "26826269", "animal_results": "In SOD(G93A) mice co-expressing the Copper-Chaperone-for-SOD, CuATSM treatment rescued early death (from 8-13 days to survival beyond that period) and effectively treated motor neuron disease", "animal_results_pmid": "26826269", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00753571", "title": "Cistanche Total Glycosides for Amyotrophic Lateral Sclerosis: A Randomized Control Trial (RCT) Study Assessing Clinical Response", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University", "summary": "This study will examine the effectiveness of Cistanche Total Glycosides(CTG) in treating patients with amyotrophic lateral sclerosis (ALS) - a fatal neurological degenerative disease that causes adult-onset, progressive motor neurons loss in the spinal cord, brain stem and motor cortex. Patients develop progressive wasting and weakness of both upper and lower limbs, bulbar and respiratory muscles. Usually death from respiratory failure typically is within 3-5 years of diagnosis. Although there are various treatments for ALS, riluzole is the only approved treatment to delay the disease progression. Cistanche Total Glycosides is an approved drug that has protective effects. It acts anti-apoptosis by activating several protective pathways, stimulates neuronal differentiation of adult neural stem cells in the brain, and improves long-term recovery. CTG is a highly attractive candidate for the treatment of neurodegenerative conditions such as ALS.\n\nPatients 18 to 65 years of age who have had mild to moderately severe ALS for 0.5 to 2 years of duration may be eligible for this study. Candidates will be screened with a medical history and possible review of medical records, physical examination, blood test, urine and stool analyses, electrocardiogram, electrophysiological examination, neurological imaging and, for women, a pregnancy test.\n\nParticipants will have drug therapy according to randomized number. One group receives CTG while other group receives placebo. For the procedure, patients are given a medication to lessen anxiety and any discomfort. Patients receive drugs for 9 months. The CTG dosage is 1.8g/day. Physical examination and interview, Appel ALS scale and ALS-Functional Rating Scale will be done in 28 days and 3, 6, 9months. Electrophysiological examination will be tested per 3 months. Blood samples will be collected on treat28 days and 3, 6, 9months.", "interventions": [{"type": "DRUG", "name": "Cistanche Total Glycosides"}], "start_date": "2008-01", "url": "https://clinicaltrials.gov/study/NCT00753571", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Dongsheng Fan, MD; Liping Wang,MD", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "RECRUITING", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "+861082265250", "contact_email": "dsfan@sina.com ; chinaals@gmail.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* All subjects must have a verifiable diagnosis of ALS of 0.5 to 2 years' duration.\n* The diagnosis must be supported by the Revised Criteria of the World Federation of Neurology.\n* The grades of diagnosis must be clinically definite ALS or clinically probable ALS.\n* All subjects must be over age 18 and below 65.\n* The ALS is mildly to moderate based on ALS Health State Scale.\n* Electrophysiological features show CMAP amplitude of motor nerve normal or mild declining.\n* Serum creatine kinase is normal or mild upper, less than 500U/L.\n\nExclusion Criteria:\n\n* If anyone of the above eligibility requirements is not met\n* Use of any other investigational agent within 30 days beginning the treatment phase of this study\n* Severe cardiac, pulmonary, hepatic or/and hematic disease\n* HIV positivity or signs and symptoms consistent with HIV infection\n* Pregnant or nursing women\n* History of cancer with less than 5 years documentation of a disease-free state\n* History of anaphylactic reaction or hypersensitivity to G-CSF or proteins derived from E.coli\n* Alcohol or drug abuse in recent 1 year\n* Can't understand or obey the rules of treatment\n* Blood donor in recent 30 days", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Cistanche Total Glycosides", "targeting_mechanism": "Antioxidant and anti-inflammatory agent targeting oxidative stress and inflammatory pathways in neurodegeneration", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00243932", "title": "Clinical Trial of High Dose CoQ10 in ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Columbia University", "summary": "The purpose of this study is to determine the efficacy and preferred dose of CoQ10 in individuals with ALS for a possible future phase III study.", "interventions": [{"type": "DRUG", "name": "coenzyme Q10"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2005-04", "url": "https://clinicaltrials.gov/study/NCT00243932", "target_entities": ["mitochondrial_dysfunction"], "locations": [{"facility": "University of Arkansas for Medical Sciences, Department of Neurology", "city": "Little Rock", "state": "Arkansas", "country": "United States", "status": "", "lat": 34.74648, "lon": -92.28959}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of California at San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Health Sciences, Dept of Neurology", "city": "Denver", "state": "Colorado", "country": "United States", "status": "", "lat": 39.73915, "lon": -104.9847}, {"facility": "Yale University School of Medicine, Department of Neurology", "city": "New Haven", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.30815, "lon": -72.92816}, {"facility": "Northwestern University, Department of Neurology,", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Chicago, Department of Neurology", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Kentucky, Dept of Neurology, College of Medicine", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Brigham and Women's Hospital , Department of Neurology", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Baystate Medical Center, Division of Critical Care Research", "city": "Springfield", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.10148, "lon": -72.58981}, {"facility": "Minneapolis Medical Research Foundation, ,", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Washington University in St. Louis School of Medicine, Department of Neurology", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Columbia Presbyterian Medical Center, The Neurological Institute", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "State University of New York Upstate Medical, Neurology Department", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Drexel University, Dept of Neurology", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Texas, Health Science Center at San Antonio, Division of Neurology", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Vermont, Neurology Department", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinical diagnosis of definite, probable, or laboratory-supported probable ALS\n* Negative pregnancy test for women of childbearing age and adequate birth control measures\n* Subjects must be able and willing to give informed consent and must be capable of complying with the trial procedures\n* Forced Vital Capacity (FVC) \\>/= 60% of predicted\n* Age 21 to 85 years, inclusive\n* Disease duration of less than 5 years\n* Subjects may take riluzole (without change in dose for more than 30 days before enrollment)\n* Patients who have taken CoQ10 in the past will be eligible if they stop at least 30 days before enrollment\n* Patients who have taken vitamin E in the past will be eligible if they stop at least 14 days before enrollment\n\nExclusion Criteria:\n\n* Dependency on mechanical ventilation (non-invasive ventilation \\> 23 hours)\n* Severe and unstable concomitant medical or psychiatric illness\n* Insufficiently controlled diabetes mellitus\n* Concomitant warfarin therapy\n* Women who are breast feeding or have a high likelihood of pregnancy\n* Significant hepatic dysfunction\n* Forced Vital Capacity (FVC) less than 60%\n* Exposure to CoQ10 within 30 days of enrollment\n* Exposure to other experimental medications within 30 days of enrollment\n* Exposure to vitamin E within 14 days of enrollment\n* Sensitivity to color additive FD\\&C Yellow No. 5\n* Sensitivity to aspirin", "sex": "ALL", "min_age": "21 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "coenzyme Q10 (CoQ10)", "targeting_mechanism": "Mitochondrial electron transport chain cofactor that enhances ATP production and reduces oxidative stress in mitochondria", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05189106", "title": "Neurodegenerative Alzheimer's Disease and Amyotrophic Lateral Sclerosis (NADALS) Basket Trial", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "This is an open-label, biomarker-driven basket trial of baricitinib in people with subjective cognitive disorder, mild cognitive impairment, Alzheimer's disease (AD), Amyotrophic lateral sclerosis (ALS), or asymptomatic carriers of an ALS-related gene, such as a hexanucleotide expansion in the C9ORF72 gene, with evidence of abnormal inflammatory signaling in cerebrospinal fluid (CSF) at baseline. Each participant will be treated with baricitinib for 24 weeks; no placebo will be given. Participants will receive baricitinib 2 mg per day by mouth for the first 8 weeks and baricitinib 4 mg per day by mouth for the remaining 16 weeks. This proof of concept trial will ascertain whether baricitinib at 2 mg per day, 4 mg per day, or both reaches therapeutic levels in the CSF and suppresses inflammatory biomarkers associated with type I interferon signaling among the study participants.", "interventions": [{"type": "DRUG", "name": "Baricitinib"}], "start_date": "2022-12-05", "url": "https://clinicaltrials.gov/study/NCT05189106", "target_entities": ["JAK", "C9orf72", "neuroinflammation"], "locations": [{"facility": "Massachusetts General Hospital - ALS Site", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Massachusetts General Hospital - AD Site", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria\n\nStudy participants meeting all of the following criteria will be allowed to enroll in the study:\n\n1. Must be 55-90 years old, inclusive and have one of the following:\n\n   * Subjective cognitive decline(SCD)\n   * Minor neurocognitive disorder(mild cognitive impairment(MCI))\n   * Major neurocognitive disorder(possible or probable AD) OR\n\n   Must be 18-80 years old, inclusive and have one of the following:\n   * Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by the revised El Escorial criteria\n   * Asymptomatic carrier of an ALS-causative mutation per CLIA-certified genetic testing results (MGH site only)\n2. Screening CSF level of CCL2 level \u2265 250 pg/mL\n3. Up-to-date immunization records per CDC guidelines\n\n   * Routine vaccinations should be administered at a minimum of 14 days prior to any study visit with an LP\n4. Must have received the Recombinant Zoster Vaccine (RZV, also known as Shingrix) within 4 years prior to enrollment. Note: Only one dose of RZV is needed prior to the Baseline Visit.\n5. Must be fully vaccinated for COVID-19 per CDC guidelines\n\n   * If a participant is planning to receive a COVID-19 booster shot, should be administered a minimum of 14 days prior to the Screening LP.\n6. For participants with ALS:\n\n   * Must either not be taking or be on a stable dose of any FDA approved treatment for ALS for at least 30 days or at least 1 cycle prior to screening\n   * ALSFRS-R score \u2265 27\n   * Must be ambulatory, defined as able to walk at least within the home every day. Use of gait assistive devices is allowed. Some use of a wheelchair is also allowed.\n   * Greater than 12-month life expectancy in the opinion of the investigator\n\n   For participants with AD:\n   * MoCA score \u2265 8\n   * The participant must have a study partner that can accompany them to every visit and co-sign any informed consent document.\n   * Must either not be taking or be on a stable dose of any FDA approved treatment for AD for at least 30 days prior to screening. Participants cannot be taking Aducanumab(see exclusion criterion #15).\n7. Ability to medically undergo LP in the opinion of the investigator (e.g., no bleeding disorder, allergy to local anesthetics, prior lumbar surgery which might make LP difficult, a skin infection at or near the LP site, evidence of high intracranial pressure, or anticipated difficulty getting into position for LP).\n8. Capable of providing informed consent and following study procedures.\n\n   * In the case that a participant lacks the ability to provide informed consent, informed consent will be obtained from the participant's surrogate representative and assent obtained from the participant.\n\nExclusion Criteria\n\nStudy participants meeting any of the following criteria during screening evaluations will be excluded from entry into the study:\n\n1. Women who are pregnant, breastfeeding, or planning to become pregnant during the trial\n2. Any unstable clinically significant medical condition other than ALS or AD (e.g., within six months of baseline, including but not limited to myocardial infarction, angina pectoris, congestive heart failure, or neoplasm undergoing active treatment).\n3. Active cancer or history of cancer, except for the following: basal cell carcinoma, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 5 years. Active cancer includes cancers with current disease manifestations or therapy that could adversely affect participant safety and longevity, create the potential for drug-drug interactions, or compromise the interpretation of study results.\n4. History of diverticulitis or bowel perforation.\n5. Active ulcerative colitis, Crohn's disease, and history of peptic ulcer disease within the past 5 years or after the age of 65.\n6. Active, serious infection, including localized infection in the opinion of the investigator.\n7. Positive for latent or active tuberculosis (TB). Note: Patients with a history of latent or active TB must have had an adequate course of treatment documented prior to study participation.\n8. Evidence of active hepatitis B or C infection.\n9. History of severe hepatic or renal impairment.\n10. eGFR \\< 60 mL/min/1.73 m2\n11. Have any of the following specific abnormalities on screening laboratory tests:\n\n    * ALT or AST \\>2.5x upper limits of normal (ULN)\n    * Alkaline phosphatase (ALP) \u22652x ULN\n    * Total bilirubin \u22651.5x ULN, Note: patients with elevated bilirubin secondary to Gilbert's disease are eligible to participate in the study.\n    * Hemoglobin \\<10 g/dL (100.0 g/L)\n    * Total white blood cell count \\<3000 cells/\u03bcL (\\<3.00 x 103/\u03bcL or \\<3.00 billion/L)\n    * Neutropenia (absolute neutrophil count \\[ANC\\] \\<1500 cells/\u03bcL) (\\<1.50 x 103/\u03bcL or \\<1.50 billion/L)\n    * Lymphopenia (lymphocyte count \\<1000 cells/\u03bcL) (\\<1.00 x 103/\u03bcL or \\<1.00 billion/L)\n    * Thrombocytopenia (platelets \\<100,000 cells/\u03bcL) (\\<100 x 103/\u03bcL or \\<100 billion/L)\n    * Laboratory abnormalities in vitamin B12, thyroid stimulating hormone (TSH), or other common laboratory parameters that might contribute to cognitive dysfunction\n12. Personal history of pulmonary embolus (provoked or unprovoked) or deep vein thrombosis, or a history of unprovoked pulmonary embolus in a first-degree family member.\n13. Treatment with anticoagulants that, in the opinion of the investigator, would compromise the safety of the participant.\n14. Previous therapy with baricitinib.\n15. Current use of strong Organic Anion Transporter 3(OAT3) inhibitors (e.g., probenecid) or other prohibited medication (refer to Section 6.7.1) within 5.5 half-lives or 30 days of screening, whichever is longer.\n\n    * For participants with AD: Current use of Aducanumab or within 30 days of screening.\n16. Receiving other experimental interventions for AD or ALS within 5.5 half-lives or 30 days of screening, whichever is longer.\n17. Use of permanent assisted ventilation (invasive ventilation via tracheostomy, or \\>22 hours of non-invasive ventilation per day, e.g., via BiPAP).\n18. Have had any major surgery within 8 weeks prior to screening or will require major surgery during the study that, in the opinion of the investigator, would pose an unacceptable risk to the patient. Note: Placement of a gastrostomy tube and an intravenous port are not considered major surgery.", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Baricitinib", "targeting_mechanism": "JAK1/JAK2 inhibitor that reduces neuroinflammation and abnormal inflammatory signaling in the central nervous system", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02750982", "title": "Laughter Therapy Effects on Mood, Stress and Self-efficacy in People With Neurological Diseases.", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Brown, Theodore R., M.D., MPH", "summary": "This is a prospective investigation of the effects of Laughter therapy (LT) on perceived stress, self-efficacy, mood and other wellness measures in people with the following neurological conditions: Alzheimer's disease, amyotrophic lateral sclerosis, brain injury, Huntington's Disease, multiple sclerosis, Parkinson's Disease, post-stroke, spinal cord injury.", "interventions": [{"type": "OTHER", "name": "Laughter Therapy"}], "start_date": "2016-07", "url": "https://clinicaltrials.gov/study/NCT02750982", "target_entities": [], "locations": [{"facility": "Evergreen Healthcare", "city": "Kirkland", "state": "Washington", "country": "United States", "status": "", "lat": 47.68149, "lon": -122.20874}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis based on medical record review of one of the following neurological diseases: Alzheimer's disease, amyotrophic lateral sclerosis, brain injury, Huntington's Disease, Multiple Sclerosis, Parkinson's Disease, Post-Stroke, Spinal Cord Injury.\n* Medically stable for at least 2 months.\n* Not participating in Laughter therapy for 30 days prior to screening.\n\nExclusion Criteria:\n\n* Females who are pregnant\n* Any unstable medical condition\n* Severe cognitive deficits that would interfere with participation (e.g. unable to follow commands).\n* Severe abdominal pain, chest pain or back pain.\n* Abdominal, chest or back surgery within 90 days.\n* Psychosis or severe mental illness.\n* Untreated hernia.\n* Persistent cough.\n* Advanced hemorrhoids.\n* Epilepsy.\n* Uncontrolled Hypertension - SBP \\>170 or DBP \\>105.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Laughter Therapy", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05562960", "title": "Plasmapheresis in Amyotrophic Lateral Sclerosis With Autoantibody Against NRIP", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Taiwan University Hospital", "summary": "Patient with amyotrophic lateral sclerosis (ALS) having anti-NRIP autoantibody showed titer-dependent detrimental Effects. Plasmapheresis might benefit this subgroup of patients via removal of anti-NRIP autoantibody", "interventions": [{"type": "PROCEDURE", "name": "Plasmapheresis"}], "start_date": "2023-05-01", "url": "https://clinicaltrials.gov/study/NCT05562960", "target_entities": ["NRIP"], "locations": [{"facility": "National Taiwan University Hospital", "city": "Taipei", "state": "", "country": "Taiwan", "status": "", "lat": 25.05306, "lon": 121.52639}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patients with amyotrophic lateral sclerosis (ALS) at the age more than 20 years and having plasma anti-NRIP autoantibody.\n2. Agree to receive plasmapheresis intervention.\n3. Agree to participate in the trial and receive serial examinations and follow up.\n\nExclusion Criteria:\n\n1. Patients without plasma anti-NRIP autoantibody.\n2. Patients requiring permanent ventilator support for ALS progression.\n3. Not able to receive plasmapheresis or trial-related examinations.\n4. Under pregnancy.\n5. Blood fibrinogen level less than 50 mg/dl.\n6. Specific ALS subtypes, including primary lateral sclerosis, progressive muscular atrophy, flail arm syndrome, or flail leg syndrome.", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Plasmapheresis", "targeting_mechanism": "Removal of anti-NRIP autoantibodies to reduce titer-dependent detrimental effects in ALS patients.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05903690", "title": "Safety and Tolerance of RAG-17 in Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation", "phase": "EARLY_PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Beijing Tiantan Hospital", "summary": "The goal of this clinical trial is to evaluate the safety, tolerability and pharmacokinetics of RAG-17 in adult amyotrophic lateral sclerosis (ALS) patients with SOD1 mutation. Patients will receive drug treamtent via dose escalation which ranging from minimum of 60 mg to the maximum tolerated dose (MTD), after reaching the tolerated dose, a fixed dose of the drug is given once every two months for continuous treatment, and the total treatment cycle is 8 months. The duration of this study is two years.", "interventions": [{"type": "DRUG", "name": "RAG-17"}], "start_date": "2023-05-24", "url": "https://clinicaltrials.gov/study/NCT05903690", "target_entities": ["SOD1"], "locations": [{"facility": "Beijing Tiantan Hospital", "city": "Beijing", "state": "", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients who are judged by professional medical staff to still be able to carry out the clinical trial project cycle;\n* 18 years old \u2264 age \u2264 75 years old, males or females;\n* ALS patients with confirmed SOD1 gene mutations document (known SOD1 mutation sites and related disease progression have been reported);\n* Forced vital capacity \u2265 50% of predicted vital capacity during the screening period;\n* Diagnosis of confirmed or probable familial or sporadic ALS in accordance with the revised EI Escorial diagnostic criteria for amyotrophic lateral sclerosis of the World Federation of Neurology;\n* The patient or patient's legal representative clearly understands and voluntarily participates in the study and signs the informed consent form;\n* Subjects (including male subjects) are willing to have no birth plan and voluntarily take effective contraceptive measures during the entire study period and within 3 months after the end of the study, and have no plan to donate sperm or eggs.\n\nExclusion Criteria:\n\n* Patients with SOD1 mutations occurring at nucleotides 44 to 66 (calculated from the start of SOD1 protein translation), patients with P.F21C mutation;\n* Patients who have previously received or are currently receiving Tofersen treatment;\n* HIV test positive or history of positive tests;\n* Positive hepatitis C virus antibody or history of positive tests;\n* Active hepatitis B infection (positive hepatitis B surface antigen and/or positive hepatitis B core antibody);\n* Have used other investigational drugs within 1 month or within 5 drug half-lives;\n* Diseases and deformities of the lumbar spine;\n* Have other conditions known to be associated with motor neuron dysfunction that may confuse or obscure an ALS diagnosis;\n* Other psychiatric disorders diagnosed according to DSM-V diagnostic criteria, or significant suicide intent;\n* With severe hepatic insufficiency, renal insufficiency or severe cardiac insufficiency (severe hepatic insufficiency refers to ALT value\u22652.0 times the upper limit of normal value or AST value\u22652.0 times the upper limit of normal value; severe renal insufficiency refers to CRE\u22651.5 times the upper limit of normal value or eGFR\\<40mL/min/1.73m2; severe cardiac insufficiency refers to NYHA class 3-4);\n* Permanently dependent on ventilator-assisted ventilation;\n* History of alcohol and drug abuse;\n* Patients who are pregnant, breast-feeding, or who are likely to become pregnant and plan to become pregnant;\n* Patients participating in other clinical trials or using other biological agents, drugs or devices under investigation;\n* Patients who have received any vaccinations within 28 days;\n* Contraindications to MRI (eg, claustrophobia);\n* Unable to be cooperative and complete the follow-up due to other reasons.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RAG-17", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02437110", "title": "HERV-K Suppression Using Antiretroviral Therapy in Volunteers With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)", "summary": "Background:\n\nSome people with Amyotrophic Lateral Sclerosis (ALS) have a high level of the virus HERV-K in their blood. Researchers do not think this virus causes ALS. But they don t know why some people with ALS have a high level of it. They want to know if HERV-K can be suppressed by drugs that are used to treat HIV infection.\n\nObjectives:\n\nTo learn how drugs usually taken for HIV infection affect people with Amyotrophic Lateral Sclerosis (ALS).\n\nEligibility:\n\nAdults at least 18 years old with ALS and high levels of HERV-K but no HIV.\n\nDesign:\n\nInterested participants can contact the study team and, if eligible, the study team will arrange for a screening blood draw to determine the HERV-K level.\n\nParticipants with a high HERV-K level will be screened with medical history, physical exam, questionnaires, nerve conduction test, lumbar puncture, and blood and breathing tests.\n\nAfter screening, participants will start taking the 4 study drugs.\n\nParticipants will have up to 12 study visits over a period of 72 weeks. After starting study drugs, they will have study visits at Weeks 1 and 4 and then every 4 weeks until Week 28. They will be asked how they are feeling and have an exam and blood drawn. At 3 visits, they will have tests of nerve conduction, breathing, and their ALS symptoms.\n\nAt Week 24, they will stop taking the study drugs and may have a repeat lumbar puncture.\n\nAfter the Week 48 visit, their participation is finished.", "interventions": [{"type": "DRUG", "name": "Darunavir"}, {"type": "DRUG", "name": "Ritonavir"}, {"type": "DRUG", "name": "Dolutegravir"}, {"type": "DRUG", "name": "Tenofovir alafenamide (TAF)"}], "start_date": "2019-04-01", "url": "https://clinicaltrials.gov/study/NCT02437110", "target_entities": ["herv_k"], "locations": [{"facility": "National Institutes of Health Clinical Center", "city": "Bethesda", "state": "Maryland", "country": "United States", "status": "", "lat": 38.98067, "lon": -77.10026}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "INCLUSION CRITERIA:\n\nSubjects must meet all of the following inclusion criteria to be eligible to participate in this study:\n\nAge 18 years or older at the time of the screening visit.\n\nAble to provide informed consent and comply with study procedures.\n\nALS diagnosed as probable, laboratory-supported probable or definite according to the World Federation of Neurology El Escorial revised criteria as determined by a neurologist with neuromuscular subspecialty training.\n\nA ratio of HERV-K:RPP greater than or equal to 13 measured by quantitative PCR at the screening visit.\n\nDuration of disease less than 2 years, or if greater than 2 years, disease progression at a rate that in the judgement of the investigator would allow for completion of the study.\n\nIf taking riluzole or edaravone, must be on a stable dose for at least 30 days prior to the screening visit, or stopped taking riluzole or edaravone at least 30 days prior to the screening visit.\n\nSubject has a competent caregiver who can and will be responsible for administering study drug. If there is no caregiver, another qualified individual must be available to do this.\n\nSubject has established care with a neurologist and will maintain this clinical care throughout the study.\n\nSubject has had neuroimaging within the last 24 months for participants enrolling at the NIH Clinical Center.\n\nEXCLUSION CRITERIA:\n\nA participant will be excluded if he or she has any of the following:\n\nDependence on daytime mechanical ventilation (invasive or non-invasive, including Continuous Positive Airway Pressure (CPAP) or Bilevel Positive Airway Pressure (BiPap) at the time of the screening visit.\n\nParticipation in any other investigational drug trial or using investigational drug (within 4 weeks prior to the Day 0 visit and thereafter).\n\nHistory of severe sulfonamide allergy (i.e. anaphylaxis).\n\nHistory of positive test or positive result at screening for HIV or HTLV-1.\n\nParticipants must not be able to become pregnant (e.g., post-menopausal for at least one year, surgically sterile, or using adequate methods of contraception) or breastfeed for the duration of the study. Adequate methods of contraception include: implanted contraception, intrauterine device in place for at least 3 months, or barrier method in conjunction with spermicide. Participants of childbearing potential must have a negative pregnancy test at screening and be non-lactating.\n\nPresence of any of the following clinical conditions at the time of screening:\n\nDrug abuse or alcoholism\n\nUnstable medical disease (such as unstable angina or chronic obstructive pulmonary disease), or active infectious disease (such as Hepatitis C or tuberculosis), or current malignancy\n\nUnstable psychiatric illness defined as psychosis or untreated major depression within 90 days of the screening visit\n\nDementia\n\nDiabetes mellitus\n\nHemophilia\n\nUse of contraindicated medications: amiodarone, dronedarone, lovastatin, simvastatin, rifampin, rifapentine, rifabutin, cisapride, pimozide, midazolam, triazolam, dihydroergotamine, ergonovine, ergotamine, methylergonovine, St. John s wort, alfuzosin, salmeterol, sildenafil for pulmonary arterial hypertension, oxcarbazepine, phenobarbital, phenytoin or dofetilide.\n\nSafety Laboratory Criteria at the screening visit:\n\nAlanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3.0 times the upper limit of normal\n\nSerum creatinine, serum phosphorous, total bilirubin, triglycerides, amylase, or lipase greater than 2.0 times the upper limit of normal\n\nEstimated glomerular filtration rate \\<60mg/dl.\n\nPlatelet concentration of \\<100,000/ (micro)l.\n\nPT and PTT \\>1.2 times the upper limit of normal for participants enrolling at the NIH Clinical Center.\n\nHemoglobin \\<10mg/dL.\n\nPositive Hepatitis B Surface Antigen and Hepatitis C Virus Antigen", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Darunavir, Ritonavir, Dolutegravir, Tenofovir alafenamide (TAF)", "targeting_mechanism": "Suppression of human endogenous retrovirus K (HERV-K) using antiretroviral therapy.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03455309", "title": "Evaluation of NDV-3A Vaccine in Preventing S. Aureus Colonization", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "NovaDigm Therapeutics, Inc.", "summary": "The proposed study aims to further evaluate the safety and immunogenicity of a candidate S. aureus vaccine NDV-3A, as well as its efficacy against acquisition of S. aureus", "interventions": [{"type": "BIOLOGICAL", "name": "NDV-3A"}, {"type": "BIOLOGICAL", "name": "Placebo"}], "start_date": "2018-01-30", "url": "https://clinicaltrials.gov/study/NCT03455309", "target_entities": [], "locations": [{"facility": "Fort Benning", "city": "Fort Benning", "state": "Georgia", "country": "United States", "status": "", "lat": 32.35237, "lon": -84.96882}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Active duty, male subject, 17-35 years of age, inclusive, at the time of screening.\n* Assigned to one of the selected companies/battalions\n* Informed of the nature of the study and has agreed to and is able to read, review, and sign the informed consent document prior to screening.\n* Free of known significant health problems as established by the requirements to be enrolled in a military training program before entering into the study.\n* Agrees to be reachable by phone, email or letter at 6 months post-vaccination.\n\nExclusion Criteria:\n\n* Reports receiving any investigational drug, investigational vaccine, or investigational device within 30 days prior to dosing; subjects will be allowed to receive routine vaccinations associated with training and any other prescribed medications not in the exclusion criteria.\n* Presence of clinically significant SSTI (e.g., cellulitis, abscess) at screening or other skin or skin structure infections that would confound the interpretation of clinical response.\n* Reports a history of allergic response(s), anaphylaxis, or other serious reactions to previous vaccinations.\n* Reports a history of allergies to yeast\n* Reports a history of anaphylaxis or other serious reactions to aluminum.\n* Reports a history of autoimmune disease (psoriasis, etc.)\n* Seropositive for HIV antibody.\n* Reports the use of any immunosuppressive drugs, including systemic corticosteroids (more than 14 days at a dose of \\>20 mg/day prednisone or equivalent), within 4 weeks prior to dosing.\n* Reports receiving any blood products within 3 months prior to dosing.\n* Reports donating blood/plasma within 28 days prior to dosing.\n* Illness causing temperature \u2265 100.4\u00b0F\n* Evidence of abnormal, unresolved laboratory results in the subject's medical record for the following tests: hemoglobin, white blood cell count, platelet count, creatinine, and alanine aminotransferase\n* Any other medical and/or social reason which, in the opinion of the investigator(s), would increase the subject's risk of having an adverse reaction as a result of participation in the study.", "sex": "MALE", "min_age": "17 Years", "max_age": "35 Years", "healthy_volunteers": true, "std_ages": ["CHILD", "ADULT"]}, "mechanism_summary": {"compound": "NDV-3A", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04577404", "title": "Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "This is a Phase 3, international, multicenter, open-label, long-term extension study. The primary objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) for up to 96 weeks.", "interventions": [{"type": "DRUG", "name": "MT-1186"}], "start_date": "2020-10-29", "url": "https://clinicaltrials.gov/study/NCT04577404", "target_entities": ["oxidative_stress"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Neuromuscular Research Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Woodland Research Northwest", "city": "Rogers", "state": "Arkansas", "country": "United States", "status": "", "lat": 36.33202, "lon": -94.11854}, {"facility": "University of Colorado Anschutz Medical Campus", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "UF Health Cancer Center", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Emory University - School of Medicine", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Neurology Associates, P.C - Lincoln", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Wake Forest University Baptist Medical Center (WFUBMC) - The J. Paul Sticht Center on Aging and Rehabilitation", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Penn State Hershey Children's Hospital", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Alleghany General Hospital", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Texas Neurology, PA", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Medical College of Wisconsin", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "Heritage Medical Research Clinic - University Of Calgary", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "CHU-Nice - Hopital Pasteur 2", "city": "Nice", "state": "Cedex 1", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Centre Hospitalier Esquirol", "city": "Limoges", "state": "Marcland", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Centre Hospitalier Universitaire (CHU) de Bordeaux", "city": "Bordeaux", "state": "", "country": "France", "status": "", "lat": 44.84124, "lon": -0.58046}, {"facility": "Hopital Pierre Wertheimer - Hopital Neurologique", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Deutsche Klinik fuer Diagnostik", "city": "Wiesbaden", "state": "Hesse", "country": "Germany", "status": "", "lat": 50.08601, "lon": 8.24435}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)", "city": "Milan", "state": "Lombardy", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Universita degli Studi di Torino - Centro Regionale Esperto Per La Sclerosi Laterale Amiotrofica (CRESLA)", "city": "Turin", "state": "Piedmont", "country": "Italy", "status": "", "lat": 45.07049, "lon": 7.68682}, {"facility": "Fondazione Serena Onlus - Azienda Ospedaliera Niguarda Ca Granda - Centro Clinico Nemo (Neuro Muscular Omnicentre)", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Aichi Medical University Hospital", "city": "Nagakute-shi", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "National Hospital Organization Chibahigashi National Hospital", "city": "Chiba", "state": "Chiba", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "Murakami Karindoh Hospital", "city": "Fukuoka", "state": "Fukuoka", "country": "Japan", "status": "", "lat": 33.6, "lon": 130.41667}, {"facility": "Fukushima Medical University Hospital", "city": "Fukushima", "state": "Fukushima", "country": "Japan", "status": "", "lat": 37.75, "lon": 140.46667}, {"facility": "National Hospital Organization Hokkaido Medical Center", "city": "Sapporo", "state": "Hokkaido", "country": "Japan", "status": "", "lat": 43.06667, "lon": 141.35}, {"facility": "National Hospital Organization Iou National Hospital", "city": "Kanazawa", "state": "Ishikawa-ken", "country": "Japan", "status": "", "lat": 36.6, "lon": 136.61667}, {"facility": "Kagawa University Hospital", "city": "Kita-gun", "state": "Kagawa-ken", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Kitasato University Hospital", "city": "Sagamihara", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.56707, "lon": 139.24167}, {"facility": "Yokohama City University Hospital", "city": "Yokohama", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.43333, "lon": 139.65}, {"facility": "National Hospital Organization Kumamoto Saishun Medical Center", "city": "Koshi-shi", "state": "Kumamoto", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Tohoku University Hospital", "city": "Sendai", "state": "Miyagi", "country": "Japan", "status": "", "lat": 38.26667, "lon": 140.86667}, {"facility": "Niigata University Medical And Dental Hospital", "city": "Niigata", "state": "Niigata", "country": "Japan", "status": "", "lat": 37.92259, "lon": 139.04125}, {"facility": "Kansai Electric Power Hospital Recruiting", "city": "Fukushima-ku, Osaka-shi", "state": "Osaka", "country": "Japan", "status": "", "lat": 34.69379, "lon": 135.50107}, {"facility": "National Hospital Organization Toneyama Medical Center", "city": "Toyonaka-shi", "state": "Osaka", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Shiga University of Medical Science Hospital", "city": "\u014ctsu", "state": "Shiga", "country": "Japan", "status": "", "lat": 35.0, "lon": 135.86667}, {"facility": "National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders", "city": "Shizuoka", "state": "Shizuoka", "country": "Japan", "status": "", "lat": 34.98333, "lon": 138.38333}, {"facility": "Juntendo University Hospital", "city": "Bunkyo-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Tokyo Metropolitan Neurological Hospital", "city": "Fuch\u016b", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.67452, "lon": 139.48216}, {"facility": "Toho University Omori Medical Center", "city": "\u014cta-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.56126, "lon": 139.71605}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Subjects must provide signed and dated informed consent form (ICF) to participate in the study. Subjects must be able (in the judgment of the Investigator) to understand the nature of the study and all risks involved with participation in the study.\n2. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.\n3. Subjects who successfully completed Study MT-1186-A01.\n\nExclusion Criteria:\n\n1. Subjects of childbearing potential unwilling to use a highly effective method of contraception from Visit 1 until 3 months after the last dose of study medication.\n2. Subjects who have a significant risk of suicide. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a specific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the C-SSRS at Visit\n3. Subjects who are not eligible to continue in the study, as judged by the Investigator.\n4. Subjects who are unable to take their medications orally or through a PEG/RIG tube.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MT-1186", "targeting_mechanism": "Oral edaravone acts as a free radical scavenger to reduce oxidative stress in motor neurons.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02870634", "title": "Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Collaborative Medicinal Development Pty Limited", "summary": "Multicenter, open-label , single and multiple dose-escalation and pharmacokinetic study", "interventions": [{"type": "DRUG", "name": "Cu(II)ATSM"}], "start_date": "2016-11-16", "url": "https://clinicaltrials.gov/study/NCT02870634", "target_entities": ["SOD1"], "locations": [{"facility": "Macquarie University", "city": "Sydenham", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.91669, "lon": 151.16798}, {"facility": "Calvary Health Care Bethlehem", "city": "Caulfield", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.88251, "lon": 145.02288}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Signed informed consent prior to initiation of any study-specific procedures;\n* Familial or sporadic ALS/MND defined as clinically possible, probable, or definite by Awaji-shima Consensus Recommendations;\n* First ALS/MND symptoms occurred no more than 2 years prior to screening visit;\n* Seated FVC \u2265 70% and SNP \u2265 50% of predicted value;\n* Not taking riluzole or on a stable dose of riluzole for at least 4 weeks prior to screening visit (participants are not allowed to start taking riluzole during the study);\n* Age between 18 and 75 years at time of informed consent;\n* Patient has a competent caregiver who can and will be responsible for administration of study drug;\n* Adequate bone marrow reserve, renal and liver function:\n\n  * absolute neutrophil count \u2265 1500/\u00b5L\n  * lymphocyte count \\< 48%\n  * platelet count \u2265 150,000/\u00b5L\n  * hemoglobin \u2265 11 g/dL\n  * creatinine clearance \u2265 60 mL/min (Cockroft \\& Gault formula)\n  * ALT and/or AST \u2264 2 x ULN\n  * total bilirubin \u2264 1.5 x ULN\n  * serum albumin \u2265 2.8 g/dL\n* Women and men with partners of childbearing potential must take effective contraception while on study and women of childbearing potential must have a negative pregnancy test and be non-lactating at screening\n\nExclusion Criteria:\n\n* Inability to swallow oral medications or presence of GI disorder deemed to jeopardize intestinal absorption of Cu(II)ATSM\n* Dependence of mechanical ventilation (non-invasive or invasive) for any part of day or night\n* Exposure to any other investigational agent within 3 months or two investigational agents within 6 months prior to screening visit\n* Active GI disease (except gastrointestingal reflux disease) within 30 days of screening visit\n* Known immune compromising illness or treatment\n* Presence of any of the following clinical conditions\n\n  * drug abuse or alcoholism\n  * unstable cardiac, pulmonary, renal, hepatic, endocrine or hematologic disorder\n  * active infectious disease\n  * AIDS or AIDS-related complex\n  * current malignancy\n  * unstable psychiatric illness, defined as psychosis or untreated major depression within 90 days of screening visit\n  * neuromuscular disease other than ALS/MND\n* Dementia that may affect either outcome measures or patient understanding and/or compliance with study requirements and procedures\n* Use of anticoagulants at therapeutic doses within 7 days prior to screening visit\n* Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Cu(II)ATSM", "targeting_mechanism": "Cu(II)ATSM delivers copper to the central nervous system to restore copper-dependent SOD1 function and antioxidant defense in motor neurons.", "targeting_mechanism_pmid": "26826269", "animal_results": "CuATSM treatment rescued early death in SOD(G93A) mice co-expressing the copper-chaperone-for-SOD, with treated pups surviving beyond the 8\u201313 day mortality window seen in untreated animals.", "animal_results_pmid": "26826269", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06849609", "title": "A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS", "phase": "EARLY_PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hong Chen", "summary": "The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of VGN-R13 as a treatment of Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "BIOLOGICAL", "name": "VGN-R13"}], "start_date": "2025-01-16", "url": "https://clinicaltrials.gov/study/NCT06849609", "target_entities": ["motor_neuron_degeneration"], "locations": [{"facility": "TongJi Hospital", "city": "Wuhan", "state": "Hubei", "country": "China", "status": "RECRUITING", "lat": 30.58333, "lon": 114.26667}], "contact_phone": "13296508243", "contact_email": "chenhong1129@hotmail.com", "eligibility": {"criteria": "key Inclusion Criteria:\n\n1. Fully understand the purpose and risks of the study and voluntarily provide a signed and dated informed consent form.\n2. Aged \u226518 years, male or female;\n3. A diagnosis of ALS according to the World Federation of Neurology El Escorial criteria (revised according to the Airlie House Conference 1998 \\[Brooks 2000\\]), and confirmed through genetic diagnosis to exclude pathogenic mutations in the superoxide dismutase 1 (SOD1) and/or FUS genes;\n4. The duration of the disease from the first symptom (any ALS symptom) prior to the screening must be less than 2 years (inclusive);\n5. Forced Vital Capacity (FVC) adjusted for gender, age, and height (sitting position) \u226550% of the predicted value.\n6. Discontinued riluzole for more than five half-lives prior to screening (and is not expected to resume during the study) or has been on a stable dose of riluzole for \u226530 days and continue to maintain this dose during the study period.\n7. Discontinued edaravone for more than five half-lives prior to screening (and is not expected to resume edaravone during the study) or is receiving the standard edaravone treatment regimen at screening and has maintained a stable dose for \u226560 days (two treatment cycles) prior to administration, and continue to maintain this dose during the study period.\n\nKey Exclusion Criteria:\n\n1. There are other diseases related to motor neuron dysfunction (progressive bulbar palsy, primary lateral sclerosis, cervical spondylosis, lumbar spondylosis, etc., idiopathic inflammatory myopathy), which may confuse or cover up the diagnosis of ALS.\n2. Presence of risk for increased or uncontrolled bleeding and/or risk of bleeding that is not managed optimally and could place a participant at an increased risk for intraoperative or postoperative bleeding. These could include, but are not limited to, anatomical factors at or near the LP site (e.g., vascular abnormalities, neoplasms, or other abnormalities) and underlying disorders of the coagulation cascade, platelet function, or platelet count (e.g., hemophilia, Von Willebrand's disease, liver disease).\n3. Current severe liver or kidney or cardiovascular disease or coagulation dysfunction, autoimmune deficiency, or uncontrolled autoimmune disease or need immunosuppressive long-term treatment, poorly controlled diabetes (HBA1C \u22657% at screening) or high blood pressure, presence of sever gastrointestinal ulcers or history of gastrointestinal bleeding;\n4. Presence of active infection that needs system treatment;\n5. Presence or history of malignant tumors within 5 years prior to screening;\n6. Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression \u2264 90 days, as determined by the Investigator.\n7. Current using medications including, drugs, herbal or OTC medications that strongly inhibit or induce CYP3A4 or P-glycoprotein (P-gp), e.g., metoclopramide, grapefruit juice, ketoconazole, erythromycin;\n8. Received another drug for the treatment of ALS disease (including but not limited to sodium phenylbutyrate (PB), taurine diol (TURSO), tauroursodeoxycholic acid (TUDCA) within 1 month before the first dose) or ursodeoxycholic acid (UDCA), biologics or Other investigational drugs with a discontinuation period shorter than five half-lives;\n9. Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication (e.g., clopidogrel) for 7 days before or 48 hours after an LP\uff1b\n10. Received systemic immunosuppressive therapy within 3 months prior to screening;\n11. Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter\uff1b\n12. Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter\uff1b\n13. Participation in gene therapy or stem cell transduction therapy at any time prior to screening\uff1b\n14. Positive test result for HIV, positive treponemal antibody for syphilis, Active hepatitis B or hepatitis C infection, Active tuberculosis infection;\n15. Clinically significant abnormalities in hematology or clinical chemistry parameters, as determined by the Investigator, which would render the participant unsuitable for enrollment;\n16. Clinically significant, as determined by the Investigator, 12-lead ECG abnormalities, including corrected QT interval using Fridericia's correction method of \\> 450 ms for males and \\> 470 ms for females;\n17. History of systemic hypersensitivity reaction to investigational product, the excipients contained in the formulation, or prophylactic immunosuppressant;\n18. Contraindicated use of corticosteroids and sirolimus;\n19. History of drug abuse or alcoholism within \u2264 6 months of study enrollment that would limit participation in the study, as determined by the Investigator;\n20. Alcohol abuse (drinking more than 14 units of alcohol per week) or smoking more than 5 cigarettes per day on average in the 6 months prior to screening;\n21. Pregnant or breastfeeding women;\n22. Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make the participant unsuitable for enrollment;", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "VGN-R13", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02193893", "title": "Biological Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Pomeranian Medical University Szczecin", "summary": "The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic lateral sclerosis.", "interventions": [{"type": "OTHER", "name": "Biological: Cell-based therapeutics"}, {"type": "OTHER", "name": "Symptomatic treatment of ALS"}], "start_date": "2010-01", "url": "https://clinicaltrials.gov/study/NCT02193893", "target_entities": ["neuroprotection"], "locations": [{"facility": "Department of Neurology of Pomeranian Medical University in Szczecin", "city": "Szczecin", "state": "Poland", "country": "Poland", "status": "", "lat": 53.42894, "lon": 14.55302}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)\n* good understanding of the protocol and willingness to consent\n* patient is mentally intact and psychologically stable\n* signed informed consent\n\nExclusion Criteria:\n\nConcomitant of other systemic disease or diseases:\n\n* inflammation (high protein or lymphocytosis in the CSF), active infections.\n* diabetes,\n* cardio-vascular disorders,\n* cancer,\n* autoimmune diseases\n* renal failure,\n* impaired hepatic function.\n* subject is a respiratory dependent.\n* subject unwilling or unable to comply with the requirements of the protocol.\n* patient has been treated previously with any cellular therapy.", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous bone marrow-derived stem/progenitor cells", "targeting_mechanism": "Stem cells can differentiate into support cells such as astrocytes, oligodendrocytes or microglia, which may benefit degenerating motor neurons by producing growth factors and anti-inflammatory cytokines, providing nutrients and buffering excessive glutamate.", "targeting_mechanism_pmid": "32043626", "animal_results": "Transplantation of clinical-grade human neural stem cells in SOD1 rats reduced neuroinflammation, prolonged survival and delayed disease progression.", "animal_results_pmid": "31024007", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00072709", "title": "Study Evaluating TCH346 and Placebo Administered Once Daily in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Novartis Pharmaceuticals", "summary": "This is a global multicenter study designed to evaluate the safety and clinical effects of 4 oral doses of TCH346 (1.0, 2.5, 7.5, and 15 mg) compared to placebo in patients with mild or mild to moderate stages of ALS. The study consists of 3 phases: screening (up to 2 weeks), run-in (16 weeks), and a double-blind treatment phase of variable duration (at least 24 weeks).", "interventions": [{"type": "DRUG", "name": "TCH346"}], "start_date": "2003-09", "url": "https://clinicaltrials.gov/study/NCT00072709", "target_entities": ["mitochondrial_dysfunction"], "locations": [{"facility": "Novartis USA", "city": "East Hanover", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.8201, "lon": -74.36487}, {"facility": "Novartis Belgium", "city": "Vilvoorde", "state": "", "country": "Belgium", "status": "", "lat": 50.92814, "lon": 4.42938}, {"facility": "Novartis CANADA", "city": "Dorval", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.4473, "lon": -73.75335}, {"facility": "Novartis France", "city": "Rueil-Malmaison", "state": "", "country": "France", "status": "", "lat": 48.8765, "lon": 2.18967}, {"facility": "Novartis Germany", "city": "Nuremberg", "state": "", "country": "Germany", "status": "", "lat": 49.45421, "lon": 11.07752}, {"facility": "Novartis Italy", "city": "Saronno", "state": "", "country": "Italy", "status": "", "lat": 45.62513, "lon": 9.03517}, {"facility": "Novartis Netherlands", "city": "Arnhem", "state": "", "country": "Netherlands", "status": "", "lat": 51.98, "lon": 5.91111}, {"facility": "Novartis Switzerland", "city": "Bern", "state": "", "country": "Switzerland", "status": "", "lat": 46.94809, "lon": 7.44744}, {"facility": "Novartis UK", "city": "Frimley", "state": "", "country": "United Kingdom", "status": "", "lat": 51.31667, "lon": -0.74544}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion criteria:\n\n* clinical diagnosis of laboratory-supported probable, probable, or definite ALS;\n* sporadic or familial ALS;\n* ALS symptom onset for no more than 3 yrs at study entry;\n* FVC equal to or more than 70%;\n* patients who are either riluzole naive or patients who are receiving concomitant treatment with riluzole at a stable dose of riluzole (50 mg bid) at study start.\n\nExclusion criteria:\n\n* Known or suspected chronic infectious disease including HIV, hepatitis B, or hepatitis C.\n* Clinically significant ECG abnormalities.\n* Known hypersensitivity to study drug or related drugs (e.g. selegiline, MAO-A and B inhibitors, or tricyclic antidepressants).\n* Patients treated with potent inhibitors of CYP1A2 or CYP3A4 (a list of such inhibitors will be provided to the investigator).\n* Treatment with MAO-A and B inhibitors (including selegiline) within the past 30 days.", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "TCH346", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03324399", "title": "A Study of Protein Metabolism, Microbiome and Investigational Probiotic Use in Patients With ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Avera McKennan Hospital & University Health Center", "summary": "ALS, also known as \"Lou Gehrig's\" disease, is a neurodegenerative disease which is fatal. Treatment for ALS is limited and currently consists of primary symptom relief or support. In addition, time from diagnosis to death averages 3-5 years. New Biotic, LLC has submitted an Orphan Drug Designation Application for an investigational probiotic and have indicated the need for more study of this orphaned drug in ALS patients.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "probiotic"}], "start_date": "2017-06-01", "url": "https://clinicaltrials.gov/study/NCT03324399", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Avera Medical Group Palliative Medicine Sioux Falls", "city": "Sioux Falls", "state": "South Dakota", "country": "United States", "status": "", "lat": 43.54369, "lon": -96.72796}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of sporadic ALS, definite or probable disease,-revised El Escorial criteria\n* Patient must be able to understand the purpose and procedures of the study, sign informed consent and comply with requirements of the protocol.\n* Age 18 and older.\n* Normal serum Magnesium (1.7 - 2.3 mg/dL) and Manganese (4.7 - 18.3 ng/mL) levels or adequate supplement to obtain normal serum Mg (if Manganese levels are low (\\<1.7 mg/dL), Hair Manganese will be evaluated before starting probiotic, and inclusion to the protocol will be at the principal investigators discretion).\n\nExclusion Criteria:\n\n* Need for consumption of frequent antibiotics, gut pH increasing medications, and/or alkaline water.\n* Patient unable to maintain regular follow up or submit to informed consent\n* Stool pH \\>7.5 - The ideal stool pH for growth and function of the investigational probiotic is 6-6.5.\n* Patients who are judged to be ineligible for study entry by investigator or sub-investigator.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "probiotic", "targeting_mechanism": "Modulation of gut microbiota composition to reduce neuroinflammation and support motor neuron function through alterations in microbial dysbiosis associated with ALS pathogenesis.", "targeting_mechanism_pmid": "28129947", "animal_results": "Unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05880563", "title": "Investigation of Cannabinoid 2-receptor Expression in the Brain and Spine of ALS-patients Compared to Healthy Controls With PET (18F-RoSMALS)", "phase": "PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Markus Weber", "summary": "This clinical trial is a phase 1 study in which investigations with the weakly radioactive substance \\[18F\\]-RoSMA-18-d6 are being carried out for the first time.\n\nThis radiolabeled substance will be used to study a specific protein in the brain and spinal cord of patients with ALS and healthy individuals. This particular protein, the cannabinoid type 2 receptor, is thought to play a role in the disease process of ALS. Furthermore, it is assumed that this protein is found more frequently in the brain and spinal cord of patients with ALS compared to healthy individuals.\n\nThe following questions will be answered by this clinical trial.\n\n1. Is this protein found, as suspected, increased in the brain and spinal cord of ALS patients compared to healthy individuals ?\n2. Does the amount of this protein change during the course of the disease?\n3. Are there any correlations between the observed changes in the amount of protein and the assessment of the course of the disease?", "interventions": [{"type": "DRUG", "name": "[18F]-RoSMA-18-d6"}], "start_date": "2023-08", "url": "https://clinicaltrials.gov/study/NCT05880563", "target_entities": ["CNR2"], "locations": [{"facility": "Muskelzentrum/ALS-Clinic, Kantonsspital St. Gallen", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria \\_ALS-patients:\n\n* Age \u226518 years\n* Able to provide written informed consent prior to study participation\n* Body weight of \u2265 50 kg and a body mass index (BMI) between 19-30 kg/m2\n* Vital signs measured after three minutes resting in the supine position must be within the following ranges:\n\n  * oral body temperature: 35.0-37.5 \u00b0C\n  * systolic blood pressure: 90-140 mm Hg\n  * diastolic blood pressure: 50-90 mm Hg\n  * pulse rate: 40-90 bpm\n* Clinically probable, probable laboratory supported, or definite ALS-diagnosis according to the revised version of the El Escorial World Federation of Neurology criteria (EEC) (46)\n* Disease duration \u2264 18 months since date of diagnosis\n* Slow vital capacity (sVC) \u2265 80 % of normal (best of three measurements)\n* Pre-study ALSFRS-R progression between disease onset and screening of -0.4 points/month or worse (calculated by ALSFRS-R score decline from 48 divided by months since symptom onset until screening)\n* Patient has to be on a stable dose of disease modifying treatments (Edaravone 60 mg i.v.\n\non ten days/month, Riluzole 100 mg/day)\n\nnclusion Criteria \\_Healthy controls\n\n* Age \u2265 18 years\n* Able to provide written informed consent prior to study participation\n* Body weight of \u2265 50 kg and a body mass index (BMI) between 19-30 kg/m2\n* Vital signs measured after 3 minutes resting in the supine position must be within the following ranges:\n\n  * oral body temperature: 35.0-37.5 \u00b0C\n  * systolic blood pressure: 90-140 mm Hg\n  * diastolic blood pressure: 50-90 mm Hg\n  * pulse rate: 40-90 bpm\n\nExclusion Criteria\\_ALS-patients\n\n* Previous participation in another clinical study involving trial medication within the preceding 12 weeks prior to \\[18F\\]RoSMA-18-d6 administration\n* Tracheostomy or continuous assisted ventilation of any type, or any other significant pulmonary disorder not attributed to ALS\n* Structural brain or spinal cord abnormalities on MRI (e.g. evidence of stroke, infarct, or other space-occupying lesion or structural abnormality in brain and/or spinal cord.)\n* Significant illness within two weeks prior to dosing\n* History of clinically significant drug allergy; history of atopic allergy (asthma, urticaria, eczematous dermatitis). A known hypersensitivity to the study drug or drugs similar to the study drug\n* History of allergic reaction to drugs or anaphylactic shock.\n* History of myocardial infarction or history of treated cancer\\[18F\\]RoSMA-18-d6 in ALS brain and spinal cord\\_ Version 1.1\\_29.11.2022 Page 45 of 98\n* Current clinically significant systemic illness or symptoms (e.g., respiratory or cardiovascular disease) that may deteriorate or affect the patient's safety or ability to cooperate during the study.\n* Gastrostomy\n* Any medical condition known to have an association with motor neuron dysfunction or involving neuromuscular weakness or another neurodegenerative disease, e.g. Parkinson's Disease (PD) or Alzheimer's disease (AD), which might confound or obscure the diagnosis of ALS\n* Major internal disorders (e.g. arterial hypertension, diabetes, evidence suggestive of liver or renal disease (bilirubin \\>1.6 mg/dL, creatinine \\> 150 \u03bcM).\n* Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment\n* Donation or loss of 400 mL or more of blood within eight weeks prior to dosing.\n* Clinically relevant abnormalities on blood screening (see 4.3.1)\n* Use of any prescription drug with central action or over-the-counter (OTC) medication within two weeks prior to dosing, except ALS-medication and Paracetamol which are acceptable.\n* Use of tobacco products in the previous three months\n* History of drug (e.g. Cannabis) or alcohol abuse within 12 months prior to dosing\n* Pregnancy or breast feeding\n\nExclusion criteria\\_healthy volunteers:\n\n* Significant neurological or psychiatric disease\n* Significant illness within two weeks prior to dosing\n* History of clinically significant drug allergy; history of atopic allergy (asthma, urticaria, eczematous dermatitis). A known hypersensitivity to the study drug or drugs similar to the study drug.\n* History of myocardial infarction or history of treated cancer\n* Current clinically significant systemic illness or symptoms (e.g., respiratory or cardiovascular disease) that may deteriorate or affect the patient's safety or ability to cooperate during the study.\n* Major internal disorders (e.g. arterial hypertension, diabetes, evidence of suggestive liver or renal disease (bilirubin \\>1.6 mg/dL, creatinine \\> 150 \u03bcM).\n* Other clinically significant abnormality on physical, neurological, or laboratory examination that, in the opinion of the investigator precludes the patient from the study.\n* Clinically significant abnormality on electrocardiogram (ECG) that, in the opinion of the investigator, precludes the patient from the study.\n* A past medical history of clinically significant ECG abnormalities or a family history of a prolonged QT-interval syndrome.\n* Structural brain or spinal cord abnormalities on MRI (e.g., evidence of stroke, infarct, or other space-occupying lesion or structural abnormality in brain and/or spinal cord). History of allergic disease or anaphylactic shock.\n* Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment\n* Use of any prescription drug or over-the-counter (OTC) medication within two weeks prior to dosing. Paracetamol is acceptable.\n* Use of tobacco products in the previous three months.\n* History of drug (e.g. Cannabis) or alcohol abuse within 12 months prior to dosing.\n* Participation in any clinical investigation within four weeks prior to dosing or any ever participation in a research study with an amyloid lowering objective.\n* Donation or loss of 400 mL or more of blood within eight weeks prior to dosing.\n* Clinically relevant abnormalities on blood screening (see 4.3.1)\n* Significant radiation exposure, especially in the last quarter (either X-ray or nuclear medicine studies). Any earlier nuclear medicine studies.\n* Pregnancy or breast feeding", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "[18F]-RoSMA-18-d6", "targeting_mechanism": "A radiolabeled PET tracer that binds to cannabinoid type 2 receptors in the brain and spinal cord to assess their expression as a biomarker in ALS pathophysiology.", "targeting_mechanism_pmid": "", "animal_results": "Unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04332198", "title": "Neurobiological and Immunological Mechanisms of Dyspnea in ALS (BIOPNEA)", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Association pour le D\u00e9veloppement et l'Organisation de la Recherche en Pneumologie et sur le Sommeil", "summary": "Dyspnea; subjective experience of respiratory discomfort; which produces negative emotional experience, is the most common symptom of patients afflicted with chronic respiratory failure and its treatments are limited. Amyotrophic Lateral Sclerosis (ALS) related - dyspnea, due to diaphragmatic dysfunction, is similar to dyspnea during mechanical inspiratory load (activation of the supplementary motor area, SMA). The perception of pain and dyspnea is processed in similar brain areas (insula, dorsal anterior cingulate cortex, amygdala and medial thalamus) and in ALS; relieving dyspnea by noninvasive ventilation (NIV) is associated with decreased pain thresholds. Otherwise, it is reported systemic elevations of pro-inflammatory cytokines in chronic pain patients, correlating with intensity of pain, and during respiratory load in healthy volunteers.\n\nThe objectives are to evaluate the cytokines and endorphins rates variations after initiation of NIV in ALS patients, and to correlate cytokines and endorphins rates with the intensity of the affective component and the intensity of the sensory component of dyspnea.\n\nThe investigators will perform a prospective, experimental study, including 30 ALS patients. Dyspnea, ventilatory and cardiac settings, electromyographic recording of the scalene muscle and biological assays (ACTH, endorphin, Neuropeptide P, BDNF, IL1, IL6, IL8, IL10, TNF), will be measured during spontaneous breathing and during NIV at different times after initiation.\n\nThe investigators expect a reduction of immunological and neurobiological markers after relieving dyspnea by NIV. This work could lead to the development of new treatments for dyspnea.", "interventions": [{"type": "OTHER", "name": "biological test"}], "start_date": "2023-07", "url": "https://clinicaltrials.gov/study/NCT04332198", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n\\- Patients with chronic respiratory failure with diaphragmatic dysfunction linked to amyotrophic lateral sclerosis and requiring long-term NIV\n\nExclusion Criteria:\n\n* Other respiratory disease (COPD, asthma, obstructive sleep apnea)\n* Alcohol or psychotropic drug the lasts 24 hours\n* Cognitive impairment\n* Smoking \\> 10 PA\n* Exacerbation or infection 6 weeks earlier\n* Endstage of illness\n* Chronic or acute pain (VAS \\> 3)", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "biological test", "targeting_mechanism": "Unknown", "targeting_mechanism_pmid": "", "animal_results": "Unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03505021", "title": "Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Orion Corporation, Orion Pharma", "summary": "This study will evaluate whether prolonged oral levosimendan can preserve respiratory function more effectively than placebo, resulting in better patient functionality as measured by the ALSFRS-R scale. In this randomized, double-blind, placebo-controlled, parallel-group, multicenter study, subjects are allocated in a 2:1 ratio to receive either levosimendan (1 -2 mg daily) or placebo for 48 weeks. The primary endpoint is slow vital capacity (SVC) at 12 weeks, with the impact on patient function assessed through 48 weeks, adjusted for patient outcome, using ALSFRS-R (combined assessment of function and survival, CAFS). Other important efficacy measures include time to respiratory events, clinical global impression (CGI), assessment of dyspnea using the Borg scale and sleep scales (Pittsburgh sleep quality index and Epworth sleepiness scale). Patient safety is monitored using conventional methods including adverse events, safety laboratory tests, vital signs and 12-lead EKG. Following screening and baseline visits, patients attend the clinic at 2, 4, 8, 12, 24, 36 and 48 weeks, with telephone assessments conducted at weeks 18, 30 and 42. An end of study visit is performed 14-25 days after the last study treatment administration. The study will be monitored by an independent data and safety monitoring board. A long-term extension study will be available for patients completing the study.", "interventions": [{"type": "DRUG", "name": "Levosimendan"}, {"type": "DRUG", "name": "Placebo for levosimendan"}], "start_date": "2018-06-21", "url": "https://clinicaltrials.gov/study/NCT03505021", "target_entities": ["cardiac_muscle_function"], "locations": [{"facility": "Phoenix Neurological Associates", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Neuromuscular Research Center and Neuromuscular Clinic of Arizona", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Colorado Springs Neurological Associates", "city": "Colorado Springs", "state": "Colorado", "country": "United States", "status": "", "lat": 38.83388, "lon": -104.82136}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "Georgetown University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "The George Washington Medical Faculty Associates - Foggy Bottom North Pavilion", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Providence Holy Cross Medical Center", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "University of Florida Health - Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Mayo Clinic - Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University School of Medicine", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Augusta University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Northwestern University Feinberg School of Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Chicago", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kentucky Chandler Medical Center", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Kentucky Neuroscience Research", "city": "Louisville", "state": "Kentucky", "country": "United States", "status": "", "lat": 38.25424, "lon": -85.75941}, {"facility": "The Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Mayo Clinic - Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "HealthPartners Specialty Center", "city": "Saint Paul", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.94441, "lon": -93.09327}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Mount Sinai Beth Israel", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia Presbyterian Hospital", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Neurosciences Institute - 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Imaging & Neurosciences Center", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Vermont Medical Center", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "Swedish Neuroscience Institute", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Froedtert Hospital", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "Brain and Mind Centre", "city": "Camperdown", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.88965, "lon": 151.17642}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Brisbane", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.46794, "lon": 153.02809}, {"facility": "Flinders Medical Centre", "city": "Bedford Park", "state": "South Australia", "country": "Australia", "status": "", "lat": -35.02204, "lon": 138.56815}, {"facility": "Calvary Health Care Bethlehem", "city": "Caulfield South", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.89562, "lon": 145.02597}, {"facility": "Perron Institute for Neurological and Translational Science", "city": "Murdoch", "state": "Western Australia", "country": "Australia", "status": "", "lat": -32.06987, "lon": 115.83757}, {"facility": "Universit\u00e4t Innsbruck", "city": "Innsbruck", "state": "Tyrol", "country": "Austria", "status": "", "lat": 47.26266, "lon": 11.39454}, {"facility": "Salzkammergut-Klinikum V\u00f6cklabruck", "city": "V\u00f6cklabruck", "state": "Upper Austria", "country": "Austria", "status": "", "lat": 48.00279, "lon": 13.65652}, {"facility": "Medizinische Universit\u00e4t Wien", "city": "Vienna", "state": "", "country": "Austria", "status": "", "lat": 48.20849, "lon": 16.37208}, {"facility": "Universitaire Ziekenhuis Leuven", "city": "Leuven", "state": "Flemish Brabant", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Centre Hospitalier R\u00e9gional de la Citadelle", "city": "Li\u00e8ge", "state": "Liege", "country": "Belgium", "status": "", "lat": 50.63373, "lon": 5.56749}, {"facility": "Algemeen Ziekenhuis St. Lucas Gent", "city": "Ghent", "state": "Oost-Vlaanderen", "country": "Belgium", "status": "", "lat": 51.05, "lon": 3.71667}, {"facility": "Alberta Health Services - Neuromuscular Clinic", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "Moncton Hospital, Southeast Regional Health Authority", "city": "Moncton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 46.09454, "lon": -64.7965}, {"facility": "McMaster University Medical Centre", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Centre De Recherche Du Centre Hospitalier de l'Universite de Montreal - Hopital Notre-Dame", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Centre Hospitalier Affilie Universitaire de Quebec", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Neurologian Poliklinikka - Meilahden Tornisairaala 3", "city": "Helsinki", "state": "", "country": "Finland", "status": "", "lat": 60.16952, "lon": 24.93545}, {"facility": "Etel\u00e4-Karjalan keskussairaala", "city": "Lappeenranta", "state": "", "country": "Finland", "status": "", "lat": 61.05871, "lon": 28.18871}, {"facility": "Turku University Hospital", "city": "Turku", "state": "", "country": "Finland", "status": "", "lat": 60.45148, "lon": 22.26869}, {"facility": "Centre Hospitalier Universitaire de Limoges", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "H\u00f4pital Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "H\u00f4pital Pasteur", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Centre Hospitalier R\u00e9gional et Universitaire de Tours H\u00f4pital Bretonneau", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Universit\u00e4tsklinikum Ulm", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Deutsche Klinik f\u00fcr Diagnostik", "city": "Wiesbaden", "state": "Hesse", "country": "Germany", "status": "", "lat": 50.08601, "lon": 8.24435}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4tsmedizin Rostock", "city": "Rostock", "state": "Mecklenburg-western-pommerania", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Alfried Krupp Krankenhaus R\u00fcttenscheid", "city": "Essen", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.45657, "lon": 7.01228}, {"facility": "Universit\u00e4tsklinikum M\u00fcnster", "city": "M\u00fcnster", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.96236, "lon": 7.62571}, {"facility": "Universit\u00e4tsklinikum Carl Gustav Carus", "city": "Dresden", "state": "Saxony", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Universit\u00e4tsklinikum Jena", "city": "Jena", "state": "Thuringia", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Charit\u00e9 Universit\u00e4tsmedizin Berlin - Campus Virchow-Klinikum", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Beaumont Hospital - Ireland", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Azienda Ospedaliera Universitaria San Martino", "city": "Genova", "state": "", "country": "Italy", "status": "", "lat": 45.21604, "lon": 11.87211}, {"facility": "Azienda Ospedaliera Universitaria-Maggiore della Carit\u00e0 di Novara", "city": "Novara", "state": "", "country": "Italy", "status": "", "lat": 45.44694, "lon": 8.62118}, {"facility": "ICS Maugeri Spa SB", "city": "Pavia", "state": "", "country": "Italy", "status": "", "lat": 45.19205, "lon": 9.15917}, {"facility": "Azienda Ospedaliero-Universitaria Pisana Ospedale Santa Chiara", "city": "Pisa", "state": "", "country": "Italy", "status": "", "lat": 43.70853, "lon": 10.4036}, {"facility": "Policlinico Umberto I di Roma", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}, {"facility": "Azienda Ospedaliera - Universitaria Citt\u00e0 della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Academisch Medisch Centrum", "city": "Amsterdam", "state": "North Holland", "country": "Netherlands", "status": "", "lat": 52.37403, "lon": 4.88969}, {"facility": "Universitair Medisch Centrum Utrecht - Rudolf Magnus Instituut voor Neurowetenschappen", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital de Basurto", "city": "Bilbao", "state": "Vizcaya", "country": "Spain", "status": "", "lat": 43.26271, "lon": -2.92528}, {"facility": "Hospital Universitari de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario Reina Sofia", "city": "C\u00f3rdoba", "state": "", "country": "Spain", "status": "", "lat": 37.89155, "lon": -4.77275}, {"facility": "Hospital San Rafael - Madrid", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Universitario y Polit\u00e9cnico de La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Norrlands Universitetssjukhus", "city": "Ume\u00e5", "state": "V\u00e4sterbotten County", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "Centralsjukhuset Karlstad", "city": "Karlstad", "state": "", "country": "Sweden", "status": "", "lat": 59.3793, "lon": 13.50357}, {"facility": "Karolinska Universitetssjukhuset", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Barts Health NHS Trust", "city": "London", "state": "England", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "King's College Hospital NHS Foundation Trust", "city": "London", "state": "England", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "The Walton Centre NHS Foundation Trust", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Written or verbal informed consent (IC) for participation in the study\n* Male or female subjects with diagnosis of laboratory supported probable, probable or definite ALS according to El Escorial revised criteria. Full electromyogram (EMG) report available consistent with ALS (but not necessarily fulfilling the electrodiagnostic criteria for ALS) from an experienced neurophysiologist\n* Able to swallow study treatment capsules, and in the opinion of the investigator, is expected to continue to do so during the study\n* Sitting SVC between 60-90% of the predicted value for age, height and sex at screening visit\n* Disease duration from symptom onset (defined by first muscle weakness or dysarthria) 12-48 months at the time of visit 1 (baseline)\n* Able to perform supine SVC in an adequate and reliable way at screening and baseline visits as judged by the investigator\n* Subjects with or without riluzole and/or edaravone. If using riluzole (any daily dose up to 100 mg), the dose must have been stable for at least 4 weeks before the screening visit and should not be changed during the study. If using edaravone, the treatment should have been started at least 4 weeks before the screening visit (at least one 28-day treatment cycle as indicated) and should not be changed during the study. If not on riluzole and/or edaravone, the respective treatments should not be started during the study\n\nExclusion Criteria:\n\n* Subject in whom other causes of neuromuscular weakness have not been excluded\n* Subject with a diagnosis of another neurodegenerative disease (e.g. Parkinson's or Alzheimer's disease)\n* Assisted ventilation of any type within 3 months before the screening visit or at screening\n* Any use of a diaphragm pacing system (DPS) within 3 months before the screening visit\n* Any form of stem cell or gene therapy for the treatment of ALS\n* Known hypersensitivity to levosimendan\n* Administration of levosimendan within 3 months before the screening visit or previous participation in the present phase III study or earlier study with oral levosimendan in ALS patients (LEVALS)\n* Any use of tirasemtiv or reldesemtiv within 1 month before the screening visit.\n* Participation in a clinical trial with any experimental treatment within 30 days or within 5 half-lives of that treatment (whichever is longer) before the screening visit\n* Any botulinum toxin use within 3 months before the screening visit\n* Recorded diagnosis or evidence of major psychiatric diagnosis, significant cognitive impairment or clinically evident dementia that may interfere with the patient's ability to comply with study procedures\n* Pulmonary illness (e.g. asthma or COPD) requiring regular treatment\n* Haemodynamically significant uncorrected valve disease or hypertrophic cardiomyopathy or restrictive cardiomyopathy\n* Any cardiovascular event (e.g. myocardial infarction, HF, arrhythmia or stroke) requiring hospitalisation within 3 months before the screening visit\n* History of Torsades de Pointes (TdP) or diagnosed long QT-syndrome\n* History of life-threatening ventricular arrhythmia, unless treated with reliable measures to prevent recurrence (e.g. with placement of implantable cardioverter defibrillator \\[ICD\\] or catheter ablation)\n* History of second or third degree atrioventricular (AV) block or sinus node disease at screening, if not treated with pacemaker\n* HR repeatedly \\> 100 bpm in the 12-lead ECG after a 5-minute rest at screening. If the HR is \\> 100 bpm in the first recording, then the second recording must be done after another 5 min rest to confirm HR \\> 100 bpm\n* Systolic blood pressure (SBP) \\< 90 mmHg at screening\n* Potassium \\< 3.7 mmol/l or \\> 5.5 mmol/l at screening\n* Severe renal impairment (creatinine clearance \\< 30 ml/min at screening), creatinine \\> 170 \u03bcmol/l at screening or on dialysis\n* Blood haemoglobin \\< 10 g/dl at screening or blood donation or loss of significant amount of blood within 60 days before the screening visit\n* Clinically significant hepatic impairment at the discretion of the investigator\n* Body mass index (BMI) \u2264 18.5kg/m2 (BMI = weight/height2)\n* Women who are lactating or of reproductive age without a negative pregnancy test and without a commitment to using a highly effective method of contraception (e.g. oral hormonal contraceptives associated with inhibition of ovulation, intrauterine devices and long acting progestin agents), if sexually active during the study, and for 1 month after the last dose of the study treatment. Women who are postmenopausal (1 year since last menstrual cycle), surgically sterilised or who have undergone a hysterectomy are considered not to be reproductive and can be included\n* Patient judged to be actively suicidal by the investigator during 3 months before the screening visit\n* Patients with known history of human immunodeficiency virus (HIV) infection\n* Any other clinically significant cardiovascular, gastrointestinal, hepatic, renal, neurological or psychiatric disorder or any other major concurrent illness that in the opinion of the investigator could interfere with the interpretation of the study results or constitute a health risk for the subject if he/she took part in the study", "sex": "ALL", "min_age": "18 Years", "max_age": "120 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Levosimendag", "targeting_mechanism": "Unknown", "targeting_mechanism_pmid": "", "animal_results": "Unknown", "animal_results_pmid": "", "repurposed_from": "Unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06199284", "title": "Atalante Exoskeleton in the Rehabilitation of Patients With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "SUSPENDED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Institut de Recherche sur la Moelle \u00e9pini\u00e8re et l'Enc\u00e9phale", "summary": "Using a MRI gait motor imagery paradigm in ALS patients in order to study how ALS affects the function of the central neural networks involved in gait function, we showed a reorganization of the motor networks that represents a compensatory response to the dysfunction of the networks involved in gait function. Our main hypothesis is that by providing coherent proprioceptive input to the sensorimotor integration areas, gait training with an exoskeleton may boost compensatory network reorganization and help to maintain function. We hypothesize that this can be achieved through a locomotion training strategy that reproduces normal gait motor patterns and appropriate sensory feedback. Gait training with an exoskeleton can meet these needs. The Atalante exoskeleton offers unique potential thanks to its cutting-edge technological features, hands-free functions and availability in numerous centers across Europe. Evaluation of its safety and efficacy in ALS is of the utmost interest in order to generalize this new approach in ALS.", "interventions": [{"type": "DEVICE", "name": "Atalante exoskeleton"}], "start_date": "2024-01-31", "url": "https://clinicaltrials.gov/study/NCT06199284", "target_entities": [], "locations": [{"facility": "Station Debout", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "H\u00f4pital Piti\u00e9-Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Institut de myologie", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with amyotrophic lateral sclerosis (defined according to the El Escorial criteria: possible, probable or definite), at the time of diagnosis\n* Male or female, between 18 and 70 years of age\n* Slowly and moderately evolving patients \u2206 ALS Progression rate (FS) \u2264 1.11 (The ALS progression rate was calculated as follows: (48 - ALSFRS-R at screening visit) / (duration from onset to this visit (month)) (Kimura et al., 2006). ALSFRS-R progression will be classified as slow (\u2206FS \u2264 0.47), intermediate (0.47 \u2264 \u2206PR \u22641.11) or fast (\u2206PR \\> 1.11) (Labra et al., 2016).\n* Manual muscle testing \u2265 3 for deltoid muscle and \u2265 4 for neck flexors and extensors muscles\n* Ambulatory status: a stable gait deficit with a total score between 3 and 6 on the ALSFRS-R sub-items \"walking\" and \"climbing stairs\"\n* French speaking patient\n* Patient affiliated with the French social security system\n* Signed informed consent\n* Measurements related to the use of the Atalante exoskeleton:\n* Height between 155 and 190 cm\n* Weight \\< 90 kg\n* Pelvis width \\< 46 cm in seated position\n* Thigh length between 56.8 and 64.8 cm\n* Leg length \\> 45.7cm or less than:\n* 60.7cm if ankle dorsi flexion is \u226516\u00b0\n* 57.7cm if ankle dorsi flexion is \u226513\u00b0and \\<16\u00b0\n* 56.7cm if ankle dorsi flexion is\\>10\u00b0and \u226413\u00b0\n* 55.7cm if ankle dorsi flexion is \u2265 0\u00b0and \u226410\u00b0\n* Joint amplitudes of lower limbs :\n* Hip: flexion 90\u00b0, extension 5\u00b0, medial rotation 10\u00b0, lateral rotation 20\u00b0, abduction 17\u00b0, adduction 10\u00b0\n* Knee: flexion 5-110\u00b0\n* Ankle: dorsiflexion (knee straight) 0\u00b0, plantarflexion 9\u00b0, eversion18\u00b0, inversion 18\u00b0.\n\nExclusion Criteria:\n\n* Osteoporosis at the femoral and/or lumbar level (T score \u2264 -2.5), confirmed by bone densitometry at both the femoral and lumbar sites.\n* Pressure ulcers in areas of contact with the exoskeleton\n* Severe spasticity (greater than Ashworth 3) for adductor, hamstring, quadriceps and sural triceps muscles or uncontrolled clonus\n* Cardiac or respiratory contraindications to physical effort\n* Cognitive impairment that can affect comprehension\n* Pregnancy or attempted pregnancy", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Atalante exoskeleton", "targeting_mechanism": "A robotic exoskeleton that provides proprioceptive input to engage motor cortex compensatory network reorganization and improve gait function through motor learning.", "targeting_mechanism_pmid": "", "animal_results": "Unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07543367", "title": "INdependence Through Endovascular Neuroprosthetic Technology (INTENT): an Early Feasibility Study", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Synchron, Inc.", "summary": "INdependence Through Endovascular Neuroprosthetic Technology (INTENT): an Early Feasibility Study", "interventions": [{"type": "DEVICE", "name": "Stentrode"}], "start_date": "2026-04", "url": "https://clinicaltrials.gov/study/NCT07543367", "target_entities": [], "locations": [{"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Buffalo Neurosurgery", "city": "Buffalo", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 42.88645, "lon": -78.87837}, {"facility": "Mount Sinai Hospital", "city": "New York", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Pittsburgh Medical Center (UPMC)", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "NOT_YET_RECRUITING", "lat": 40.44062, "lon": -79.99589}, {"facility": "University of Texas Southwestern Medical Center (UTSW)", "city": "Dallas", "state": "Texas", "country": "United States", "status": "NOT_YET_RECRUITING", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Able to provide informed consent to participate in the study.\n* Diagnosis of ALS, with bilateral upper-limb paresis.\n* ALS must be refractory to treatment and have been present for a minimum of six months.\n* Aged 22 years or older.\n* Life expectancy greater than 12 months post-implantation.\n* Preserved precentral gyrus assessed using CT.\n* Suitable vascular anatomy assessed using CT venography.\n* Suitable anatomy for subcutaneous pocket creation.\n* Able to undergo anesthesia.\n* Willing and able to comply with all investigational requirements, including clinical testing visits and training visits in the home.\n* Caregiver(s) willing and able to facilitate study visits, including visits to the study site and in the home, and BCI use outside of study visits (e.g., device charging).\n* Patient and caregiver fluent in English.\n* Suitable home environment for BCI training.\n\nExclusion Criteria:\n\n* Active infection or unexplained fever in the 48 hours prior to informed consent.\n* Major psychiatric disorder that may adversely impact the participant's safety or study compliance, including severe depression, psychotic features, personality disorder, severe emotional lability, or substance abuse.\n* Diagnosis of ALS-FTD or another dementia.\n* Active implanted device (e.g., deep brain stimulator, cardiac defibrillator, pacemaker, vagal nerve stimulator, spinal cord stimulator, diaphragmatic pacer, etc.).\n* Known allergy to patient-contacting materials included in the implanted device.\n* Contraindication to angiographic imaging or iodine contrast media.\n* History of central venous sinus thrombosis.\n* Recent history of new venous thromboembolic event (in the 6 months prior to implant), recurrent history of venous thromboembolic disease, or hypercoagulable state.\n* Contraindication to antithrombotic therapy.\n* Participant is at substantially increased risk of infection, including immunocompromised status, recurrent or chronic infection, or poorly controlled diabetes mellitus.\n* Significant risk of non-healing of the subcutaneous pocket incision, including history of chronic non-healing surgical wounds or poorly controlled diabetes mellitus.\n* Pregnant or breast feeding.\n* Patients who are currently enrolled in any other clinical trial that would confound interpretation of safety or effectiveness data or may interfere with the ability to meet study requirements.\n* Any other disease or disorder that could significantly affect participation in the study. Examples may include corrected vision insufficient for viewing computer screens or hearing insufficient for following verbal instructions, which might impact the participant's ability to participate in BCI training and testing.", "sex": "ALL", "min_age": "22 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Stentrode", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04066244", "title": "Study of Safety and Proof of the Mechanism of BLZ945 in ALS Patients", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Novartis Pharmaceuticals", "summary": "It was an open label study to evaluate safety, tolerability and brain microglia response in participants with Amyotrophic Lateral Sclerosis (ALS) following multiple doses of BLZ945.", "interventions": [{"type": "DRUG", "name": "BLZ945"}], "start_date": "2019-12-30", "url": "https://clinicaltrials.gov/study/NCT04066244", "target_entities": ["CSF1R"], "locations": [{"facility": "Yale University School of Medicine", "city": "New Haven", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.30815, "lon": -72.92816}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Novartis Investigative Site", "city": "Turku", "state": "", "country": "Finland", "status": "", "lat": 60.45148, "lon": 22.26869}, {"facility": "Novartis Investigative Site", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Able to communicate well with the investigator, to understand and comply with the study visits and procedures of the study\n* Written informed consent must be obtained before any assessment is performed.\n* Male and female participants who are 18 years old or older at screening, and who are diagnosed with familial or sporadic ALS according to the World Federation of Neurology Revised El Escorial criteria of either bulbar or limb onset.\n* Disease duration from symptoms onset no longer than 48 months at the screening visit.\n* Females of childbearing potential must have a negative pregnancy test at screening and/or baseline.\n* Treatment with approved ALS therapies is allowed, but participants need to be on a stable dose and regimen for at least 30 days prior to baseline.\n* Having completed the Core Treatment Period to be able to continue in the Extended Treatment Period.\n\nExclusion Criteria:\n\n* A history of clinically significant ECG abnormalities\n* Active medical or neurologic diseases other than ALS, that in the opinion of the investigator would limit their participation in the current study.\n* Use of other investigational drugs within 5 half-lives of screening, or until the expected PD effect has returned to baseline, whichever is longer; or longer if required by local regulations.\n* History of hypersensitivity to any of the study treatments or excipients or to drugs of similar chemical classes.\n* Presence of human immunodeficiency virus (HIV) infection based on screening lab results.\n* Evidence of active or latent tuberculosis as assessed by Quantiferon testing at the screening visit.\n* Positive serology for hepatitis B surface antigen, or hepatitis C antibodies confirmed by an appropriate licensed test at screening.\n* Signs or symptoms, in the judgement of the investigator, of a clinically significant systemic viral, bacterial or fungal infection within 30 days prior to the screening visit.\n* Cardiac disorders, such as recent cardiac history (within 6 months of screening) of acute coronary syndrome, acute heart failure, or significant ventricular arrhythmia at the screening visit or participants with a history of severe pulmonary hypertension. Participants with cardiac failure class 3 or 4 of the NYHA classification, or history of reduced LVEF or individuals with implanted cardiac pacemaker, or defibrillator.\n* Significant hematological laboratory abnormalities.\n* Clinical evidence of liver disease or liver injury or any of the following hepatic conditions at the screening visit:\n* Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 14 days after last dose of BLZ945.\n* Pregnant or nursing female participants\n* Sexually active males unless they use a condom during intercourse while taking the drug during treatment, for 14 days after stopping BLZ945 and should not father a child in this period.\n* History or presence of impaired renal function at the screening visit.\n* Active suicidal ideation.\n* History of drug abuse or harmful alcohol use within the 12 months prior to dosing within the judgement of the investigator, or evidence of such abuse as indicated by the laboratory assays conducted during screening.\n* Active GI conditions such as Barrett's esophagus, achalasia, esophageal varices and active or history of esophageal cancer, pre-existing pancreatic disease at screening visit.\n* History of active vasculitis or history of autoimmune disease autoimmune disease associated with vasculitis (eg., RA, SLE, Sj\u00f6grens disease, scleroderma).\n* History or active cardiac valve disorder, congenital valve disease, or other clinical condition that might affect cardiac valve function\n* Use of systemic anticoagulation that cannot be temporarily paused before study procedures\n* Abnormal values on CT scan or echocardiography, signs of vasculitis, or evidence of a significant medical condition meeting treatment discontinuation criteria will be exclusionary for continuation in the extended treatment period.\n* Participants who are planning to initiate treatment with an additional approved ALS therapy in the next 24 weeks are not allowed to continue in the extended treatment period.", "sex": "ALL", "min_age": "18 Years", "max_age": "100 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "BLZ945", "targeting_mechanism": "A CSF1R inhibitor that targets microglial activation in the central nervous system.", "targeting_mechanism_pmid": "", "animal_results": "In SOD1G93A mice, the temporal evolution of the immune response and microglial activation is associated with disease progression, demonstrating microglial involvement in ALS neurodegeneration.", "animal_results_pmid": "31597644", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03338114", "title": "Study to Evaluate the Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Upper or Lower Extremity Muscles Most Affected in Subjects With ALS", "phase": "PHASE1, PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Flex Pharma, Inc.", "summary": "The FLX-787-106 study will determine how well FLX-787-ODT works to reduce fasciculations in patients with Amyotrophic Lateral Sclerosis (ALS). The study will measure how often fasciculations occur, and monitor any side effects that might develop while taking the investigational product. Participants will be assessed before and after taking a single dose of FLX-787-ODT. Approximately 15 people will take part in this study at one center in the United States. Participants will be in the study for a single clinic visit and receive a telephone call 7 days later to monitor for side effects.", "interventions": [{"type": "DRUG", "name": "FLX-787-ODT (orally disintigrating tablet)"}], "start_date": "2017-11", "url": "https://clinicaltrials.gov/study/NCT03338114", "target_entities": ["fasciculation_neuromuscular_function"], "locations": [{"facility": "Wake Forest University Health Sciences", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Documented diagnosis of ALS diagnosis of less than 3 years.\n* Expected survival \\> 6 months\n* Frequent fasciculations noted during clinical examination of any single muscle (\\>6 visible fasciculations per minute) observed in Part I\n* Normal oral cavity exam at screening\n* Proficient in English\n* Male subjects will either be surgically sterile or agree to use a medically acceptable method of contraception during sexual contact with females of childbearing potential, and will refrain from sperm donation for 45 days following the study\n* Male subjects will either be surgically sterile or agree to use a medically acceptable method of contraception during sexual contact with females of childbearing potential, and will refrain from sperm donation for 45 days following the study\n\nExclusion Criteria:\n\n* Presence of clinically significant or unstable condition that would result in an increased risk of study participation or difficulty in interpretation of the study results\n* Tremor or other movement disorder that would interfere with recording\n* Inability to lie flat\n* Presence of major gastrointestinal disorders, such as inflammatory bowel disease, diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or esophageal lesions/ulcers\n* Presence of laryngospasm or significant swallowing problems\n* Presence of percutaneous endoscopic gastrostomy, esophagogastroduodenoscopy, or G-tube\n* Inability to tolerate a spicy sensation in the mouth or stomach\n* Actively using illicit drugs or history of chronic substance abuse within the past year prior to screening, including abuse of alcohol\n* Participated in a clinical study (except natural history studies without administration of an investigational product) within 30 days prior to screening\n* Pregnant, breastfeeding, or planning to become pregnant\n* Blood pressure of \u2265160 mmHg systolic and/or \u2265100 mmHg diastolic\n* Clinically significant abnormalities in laboratory findings (including screening complete electrolyte panel, complete blood count, liver function tests).", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "FLX-787-ODT", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01730716", "title": "Dose Escalation and Safety Study of Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuralstem Inc.", "summary": "The study is to determine the feasibility, safety, toxicity, and maximum tolerated (safe) dose of human spinal derived neural stem cell transplantation for the treatment of Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DEVICE", "name": "Human spinal cord stem cell implantation"}], "start_date": "2013-05", "url": "https://clinicaltrials.gov/study/NCT01730716", "target_entities": [], "locations": [{"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Have the ability to understand the requirements of the study, provide written informed consent, understand and provide written authorization for the use and disclosure of Protected Health Information (PHI) \\[per Health Insurance Portability and Accountability Act (HIPAA) Privacy Ruling\\] and comply with the study procedures.\n2. Subjects with sporadic or familial ALS, meeting the definition of laboratory-supported probable, probable or definite ALS according to the World Federation of Neurology El Escorial Criteria (Appendix A). At the time of enrollment subjects should be within 24 months of symptom onset.\n3. Age 18 years or older.\n4. Females must have a negative serum pregnancy test and practice an acceptable method of contraception or be of non-childbearing potential (post-menopausal for at least 2 years or surgically sterile \\[hysterectomy, oophorectomy or surgical sterilization\\]).\n5. Geographic accessibility to the study center and the ability to travel to the clinic for study visits.\n6. Presence of a willing and able caregiver.\n7. Medically able to undergo lumbar and/or cervical laminectomy or laminoplasty as determined by the site Principal Investigator and neurosurgeon.\n8. Medically able to tolerate the immunosuppression regimen consisting of basiliximab, tacrolimus, mycophenolate mofetil, prednisone and methylprednisolone as determined by the site PI.\n9. Agrees to the visit schedule as outlined in the informed consent.\n10. Not taking riluzole (Rilutek\u00ae) or on a stable dose for \u2265 30 days.\n11. Vital capacity \u2265 60% of predicted normal for age, height and gender measured in the seated position and \u226550% in supine position during the 7 days prior to surgery.\n12. Ambulatory subjects with extremity weakness and/or spasticity due to ALS. Patients undergoing lumbar surgery must have demonstrable weakness or spasticity in one or both lower extremities. Patients undergoing cervical surgery must have demonstrable weakness or spasticity in one or both upper extremities, with at least antigravity strength. Subjects must have normal neck extensor and flexor strength.\n\nExclusion Criteria:\n\n1. Etiology of paraplegia or weakness is due to causes other than ALS.\n2. A positive result on the Panel Reactive Antibody (PRA) test, with the presence of specific HLA antibodies matching the HLA DNA profile of the donor cells.\n3. Any known immunodeficiency syndrome.\n4. Receipt of any investigational drug, device or biologic within 30 days of surgery.\n5. Any concomitant medical disease or condition limiting the safety to participate:\n\n   1. Coagulopathy\n   2. Active uncontrolled infection\n   3. Hypotension requiring vasopressor therapy\n   4. Previous spinal surgery that the neurosurgeon deems to be an obstacle to the planned transplantation\n   5. Skin breakdown over the site of surgery\n   6. Malignancy (except for non-melanoma skin cancer)\n   7. Spinal stenosis severe enough to preclude surgery (as determined by the neurosurgeon)\n   8. Pre-existing kyphosis by preoperative MRI or X-ray\n   9. Less than 5/5 grade in neck extension and strength test at the time of surgery.\n6. Creatinine \\>1.5, liver function tests (SGOT/SGPT, Bilirubin, Alk Phos) \\> 2x upper limit of normal, hematocrit/hemoglobin \\< 30/10, total WBC \\< 4000, uncontrolled hypertension (systolic \\> 180 or diastolic \\> 100) or uncontrolled diabetes (defined as hemoglobin A1C \\>8), evidence of GI bleeding by hemoccult test, tuberculosis (TB test: PPD), serologic evidence of current infection with a hepatitis virus or human immunodeficiency virus (HIV).\n7. Presence of any of the following conditions:\n\n   1. Current drug abuse or alcoholism\n   2. Unstable medical conditions\n   3. Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening\n8. Any condition or ALS disease phenotype that the site PI feels may interfere with participation in the study or in the interpretation of study endpoints.\n9. Any condition that the neurosurgeon feels may pose complications for the surgery.\n10. Known hypersensitivity to basiliximab, tacrolimus, mycophenolate mofetil, prednisone or methylprednisolone.\n11. Inability to provide informed consent as determined by the site PI.\n12. Inadequate family or caregiver support as determined by the site PI.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Human spinal cord stem cell implantation", "targeting_mechanism": "Human spinal cord-derived neural stem cells that can differentiate into motor neurons and support cells (astrocytes, oligodendrocytes) to provide trophic support through growth factors and anti-inflammatory cytokines.", "targeting_mechanism_pmid": "", "animal_results": "Preclinical stem cell studies in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1) demonstrated potential benefit of stem cell therapy, serving as stimulus for human trials.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02164253", "title": "Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Lille", "summary": "The FAIR-ALS study is to investigate the safety and efficacy of a scavenger treatment of iron deferiprone, which would reduce the brain iron to limit the development of amyotrophic lateral sclerosis.\n\nIt has been shown an excess of iron in the central nervous system carrying a sporadic ALS patients. Iron overload associated with a loss of motor neurons may explain the signs of the disease (atrophy).\n\nThe investigators discuss the hypothesis that reducing excess iron, the investigators can reduce the loss of neurons and thus the progression of signs of the disease.", "interventions": [{"type": "DRUG", "name": "Deferiprone"}], "start_date": "2013-09", "url": "https://clinicaltrials.gov/study/NCT02164253", "target_entities": ["iron_homeostasis"], "locations": [{"facility": "H\u00f4pital Roger Salengro, CHRU de Lille", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Amyotrophic lateral sclerosis defined in accordance to the El Escorial criteria (possible, probable or defined)\n* 18 to 85 years old patient, male or female\n* Patient with social security cover\n\nExclusion Criteria:\n\n* Achieved respiratory defined by a FVC \\<70%\n* Evolution of more than 24 months\n* Demented subject\n* Severe malnutrition\n* Patients with treatment potentially at risk of agranulocytosis and neutropenia\n* Patients with a history of agranulocytosis or iatrogenic under haematological disease\n* Incapable of giving consent\n* Indication against MRI\n* Indication against lumbar puncture\n* Patient refused lumbar puncture\n* Hypersensitivity to iron chelators\n* Concomitant treatment with antacids containing aluminum\n* Presence of another serious illness to life-threatening or disabling cons to the use of the treatment mixture of oxygen and nitrous oxide equally", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Deferiprone", "targeting_mechanism": "An iron chelator that scavenges excess iron in the central nervous system to reduce iron-associated oxidative stress and motor neuron degeneration.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04575727", "title": "Exploratory Evaluation of [11C]MPC6827", "phase": "EARLY_PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Columbia University", "summary": "This is a phase 0 study that will enable an assessment of biodistribution and estimation of absorbed dose in humans based on data collected from five healthy volunteers, which is typically the minimum number required by the FDA for first-in-human studies to assess dosimetry of a new tracer. The evaluation of the brain imaging of thirty additional subjects in the 2nd part of the study will lead to a descriptive assessment of the targeting and pharmacokinetics of MPC6827 in the brain and between normal and diseased brain.", "interventions": [{"type": "DRUG", "name": "[11C]MPC6827"}], "start_date": "2021-01-08", "url": "https://clinicaltrials.gov/study/NCT04575727", "target_entities": [], "locations": [{"facility": "Cuimc / Nyp", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria (Healthy Volunteers)\n\n1. All volunteers must be 18 years of age or older, able to read, understand and voluntarily sign and informed consent document.\n2. Volunteers must have no current medical history of brain disease\n3. Negative pregnancy test if female of childbearing potential.\n4. Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry for the duration of study participation.\n\nInclusion Criteria (Alzheimer's Disease or ALS)\n\n1. All volunteers must be 18 years of age or older, able to read, understand and voluntarily sign and informed consent document.\n2. Subjects must have a diagnosis of Alzheimer's Disease or ALS for which they are under a physician's care.\n3. Subjects must have a negative pregnancy test if female of childbearing potential\n4. Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry for the duration of the study participation.\n\nExclusion Criteria:\n\n1. Participants with evidence of brain disease other than ALS or Alzheimer's Disease at the time of enrolment up to agent administration are to be excluded from the study.\n2. Concomitant medication use that, in the judgement of the investigator, would make the participant inappropriate for enrolment.\n3. Severe concurrent disease, infection, or medical co-morbidity that, in the judgement of the investigator, would make the participant inappropriate for enrolment.\n4. Participants who are receiving other investigational radiation drugs.\n5. Women who are pregnant or breast feeding.\n6. Subjects who are unable to tolerate PET/CT imaging", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "[11C]MPC6827", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03039673", "title": "MIROCALS: Modifying Immune Response and OutComes in ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Centre Hospitalier Universitaire de N\u012bmes", "summary": "MIROCALS is a phase II study of ld-IL-2 as a therapeutic agent for ALS. A randomized (1:1), placebo-controlled, double-blind, parallel group trial will be carried out to assess ld-IL-2 safety and clinical efficacy on survival and functional decline in newly diagnosed ALS patients treated for 18 months. Randomization will be stratified according to (i) country (n = 2 levels: UK, France) and (ii) site of onset (n= 2 levels: bulbar vs limb onset).\n\nThe primary objective to evaluate the clinical efficacy and safety of the experimental drug (ld IL-2) over an 18 months period in order to establish the proof of concept (PoC) that modifying immune responses through the enhancement of regulatory T cells modifies the rate of ALS disease progression.", "interventions": [{"type": "DRUG", "name": "Riluzole"}, {"type": "DRUG", "name": "IL-2"}, {"type": "DRUG", "name": "5% glucose water solution"}], "start_date": "2017-06-19", "url": "https://clinicaltrials.gov/study/NCT03039673", "target_entities": ["IL-2"], "locations": [{"facility": "CHRU de Lille - H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges - H\u00f4pital Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "HCL - H\u00f4pital Neurologique P. Wertheimer", "city": "Lyon", "state": "", "country": "France", "status": "", "lat": 45.74906, "lon": 4.84789}, {"facility": "APHM - H\u00f4pital de la Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHRU de Montpellier - H\u00f4pital Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nice - H\u00f4pital Pasteur", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "APHP - Groupe Hospitalier Piti\u00e9-Salpetri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "CENTRE HOSPITALIER DE SAINT BRIEUC - H\u00f4pital Yves Le Foll", "city": "Saint-Brieuc", "state": "", "country": "France", "status": "", "lat": 48.51513, "lon": -2.76838}, {"facility": "CHU de Strasbourg - H\u00f4pital de Hautepierre", "city": "Strasbourg", "state": "", "country": "France", "status": "", "lat": 48.58392, "lon": 7.74553}, {"facility": "CHRU de Tours - H\u00f4pital Bretonneau", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Trafford Centre for Biomedical Research", "city": "Brighton", "state": "", "country": "United Kingdom", "status": "", "lat": 50.82838, "lon": -0.13947}, {"facility": "Institute of Neurological Sciences, Queen Elizabeth University Hospital", "city": "Glasgow", "state": "", "country": "United Kingdom", "status": "", "lat": 55.86515, "lon": -4.25763}, {"facility": "North-East London and Essex MND Regional Care Centre", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "King's MND Care and Research Centre", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Centre for Neuromuscular Diseases - National Hospital of Neurology", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Salford Royal NHS Foundation Trust, Neurology Dept", "city": "Manchester", "state": "", "country": "United Kingdom", "status": "", "lat": 53.48095, "lon": -2.23743}, {"facility": "Sheffield Care and Research Centre", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Main Inclusion criteria\n\n* Patient is 18 years old and less than 76 years old\n* Possible, Probable, Probable laboratory-supported or Definite ALS as defined by El Escorial Revised ALS diagnostic criteria\n* Disease duration \\<= 24 months\n* Slow Vital capacity \\>= 70% of normal\n* No prior or present riluzole treatment\n* Lumbar punctures accepted by patient and done\n\nMain Exclusion criteria\n\n* Other neurodegenerative disease that could explain signs or symptoms\n* Contra indication for lumbar puncture (history of allergy to xylocaine, presence of contra-indicated treatment, or coagulation test abnormality, clinically significant coagulopathy or thrombocytopenia)\n* Non authorized treatment\n* Other disease or disorders that could preclude functional assessment, or life-threatening disorders\n* Any documented, active, past or present, auto-immune disorders except asymptomatic Hashimoto thyroiditis\n* Using assisted ventilation\n* Feeding through gastrostomy or nasogastric tube\n* Women of child-bearing potential or sexually active man without contraception\n* Pregnant or breast feeding woman\n* Any clinically significant laboratory abnormality (excepting cholesterol, triglyceride, glucose, CK, ferritin)\n* History of documented symptomatic and treated asthma within the past 5 years", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ld-IL-2", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03876002", "title": "Evaluation of Microglial Activation in ALS With [18F]PBR06 (Peripheral Benzodiazepine Receptor-06) PET", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Molecular NeuroImaging", "summary": "The overall goal of this protocol is to evaluate microglial activation in the brain using \\[18F\\]PBR06 in patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "[18F]PBR06"}], "start_date": "2016-06-28", "url": "https://clinicaltrials.gov/study/NCT03876002", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Molecular NeuroImaging, LLC", "city": "New Haven", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.30815, "lon": -72.92816}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria (for all subjects):\n\n* Written informed consent must be obtained before any assessment is performed.\n* Provide signed and dated written informed consent.\n* Female subjects must be documented by medical records or physician's note to be either surgically sterile (by means of hysterectomy, bilateral oophorectomy, or tubal ligation) or post-menopausal for at least 1 year or, if they are of child-bearing potential, must commit to the use of a barrier method of contraception for the study duration,\n* Male subjects and their partners of childbearing potential must commit to the use of a barrier method of contraception for the study duration.\n* Male subjects must not donate sperm for the study duration.\n* Able to lie supine on camera bed for a reasonable period of time.\n* Willing and able to cooperate with study procedures.\n* Women of child bearing potential must have a negative pregnancy test at screening.\n\nInclusion criteria specific to healthy volunteer subjects:\n\n* Males and females aged 45-80 years, healthy with no clinically relevant finding on physical examination at screening and upon reporting for the \\[18F\\]PBR06 imaging sessions.\n* No family history of ALS or frontotemporal dementia.\n* C-reactive protein level \u226410 mg/dL Inclusion criteria specific to ALS subjects\n* Males and females aged 18 to 80 years.\n* Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by the modified El Escorial criteria;\n* Subjects with a diagnosis of possible ALS must have upper motor neuron (UMN) signs to be eligible\n* ALS cognitive behavioral screen score \\>10 on the cognitive scale and/or \\>32 on the behavioral scale;\n* Forced vital capacity of \u226540%; or if in the opinion of the investigator can lay flat for up to 90 minutes. If an forced vital capacity (FVC) has been performed within the past 6 months; this data may be used at the discretion of the investigator.\n* Medications for ALS subjects should be stable for 30 days prior to the Screening Visit and remain unchanged during the subject's participation in the study.\n\nExclusion Criteria (for all subjects):\n\n* Low affinity translocator protein (TSPO) binders determined by having a Thr/Thr polymorphism in the TSPO gene at screening.\n* Treatment with immunosuppressive agents (eg. prednisone, solumedrol) within 30 days of baseline and follow-up imaging.Treatment with anti-inflammatory agents including any medications in the following classes of NSAIDS: carboxylic acids, enolic acids, cyclooxygenase (COX) II Inhibitors within 14 days of baseline and follow-up imaging. Treatment with benzodiazepines within 5 half-lives prior to any \\[18F\\]PBR06 imaging visit.\n* For subjects undergoing arterial line placement, treatment with any antihemostasis medication (e.g., warfarin, heparin, thrombin inhibitors, Factor Xa inhibitors, streptokinase, urokinase, tissue plasminogen activators) within 2 weeks of the planned arterial line placement of either the baseline or follow-up imaging.\n* For subjects undergoing arterial line placement, a Modified Allen's test indicating insufficient ulnar artery blood supply to the hand.\n* Current or prior history of alcohol or drug abuse.\n* Current tobacco use including cigarettes, cigars, and chewing tobacco, or nicotine-containing products such as gum, patch, or \"electronic cigarettes\".\n* Positive urine drug screen or urine cotinine screen, with the exception of tetrahydrocannabinol (THC) in ALS subjects for whom medical marijuana is prescribed.\n* Laboratory tests with clinically significant abnormalities and/or clinically significant unstable medical illness.\n* Subject has received an investigational therapeutic within 30 days prior to screening.\n* Radiation exposure over the past year (including prior participation in other research protocols, clinical care or participation in this study) exceeding the effective dose of 50 millisievert (mSv), which would be above the acceptable annual limit established by the US Federal Guidelines.\n* Pregnancy, lactating or breastfeeding.\n* Evidence of clinically significant gastrointestinal, cardiovascular, hepatic, renal, hematological, neoplastic, endocrine, alternative neurological, immunodeficiency, pulmonary, or other disorder or disease.\n* Unsuitable veins for repeated venipuncture.\n* MRI exclusion criteria include: Findings that may interfere with interpretation of the \\[18F\\]PBR06 PET imaging, including but not limited to: significant cortical cerebrovascular disease, infectious disease, space-occupying lesions, hydrocephalus or other abnormalities associated with central nervous system (CNS) disease.\n* Contraindications to MRI imaging, including but not limited to: Implants such as implanted cardiac pacemakers or defibrillators, insulin pumps, cochlear implants, metallic ocular foreign body, implanted neural stimulators, CNS aneurysm clips and other medical implants that have not been certified for MRI, or history of claustrophobia, which in the opinion of the investigator is severe and prohibits imaging.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "[18F]PBR06", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00600873", "title": "R(+)PPX High Dose Treatment of ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "summary": "R(+)pramipexole is administered in escalating doses to patients with early ALS. Plasma and spinal fluid levels of R(+)PPX are monitored, in addition to biochemical markers of oxidative stress.", "interventions": [{"type": "DRUG", "name": "R(+) pramipexole dihydrochloride monohydrate"}], "start_date": "2007-08", "url": "https://clinicaltrials.gov/study/NCT00600873", "target_entities": ["oxidative_stress"], "locations": [{"facility": "University of Virginia", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* definite ALS no prior exposure to R(+)PPX\n\nExclusion Criteria:\n\n* ALSFRS at baseline \\<40 FVC at baseline \\<70%", "sex": "ALL", "min_age": "30 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "R(+) pramipexole dihydrochloride monohydrate", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03618966", "title": "Neuromuscular Magnetic Stimulation in ALS Patients", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Roma La Sapienza", "summary": "Aim of the study is to verify whether neuromuscular magnetic stimulation can improve muscle function in spinal-onset Amyotrophic Lateral Sclerosis (ALS) patients.", "interventions": [{"type": "DEVICE", "name": "Neuromuscular magnetic stimulation (NMMS)"}], "start_date": "2014-11-01", "url": "https://clinicaltrials.gov/study/NCT03618966", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of probable or definite ALS with spinal-onset\n* right-handed patients\n* a bilateral symmetric muscular deficit in flexor carpi radialis muscle or flexor digitorum profundus muscle (defined by a MRC Muscle Scale score of 3-4/5)\n\nExclusion Criteria:\n\n* history of epilepsy or severe headaches,\n* pregnancy or breast-feeding\n* patients with implanted cardiac pacemaker, neurostimulators, surgical clips or medical pumps\n* presenting any other comorbid condition affecting the possibility of completing the study", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07703462", "title": "Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS", "phase": "PHASE1, PHASE2", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "n-Lorem Foundation", "summary": "This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.", "interventions": [{"type": "DRUG", "name": "nL-TARDB-002"}], "start_date": "2026-12", "url": "https://clinicaltrials.gov/study/NCT07703462", "target_entities": ["TARDBP"], "locations": [{"facility": "Jefferson Health", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Informed consent/assent provided by the participant (when appropriate), and/or participants parent(s) or legally authorized representative(s)\n* Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records\n* Genetically confirmed neurological disorder\n\nExclusion Criteria:\n\n* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures\n* Use of an investigational medication within less than 5 half-lives of the drug at enrollment", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "nL-TARDB-002", "targeting_mechanism": "Antisense oligonucleotide (ASO) designed to target pathogenic variants in TARDBP to suppress mutant TDP-43 expression in motor neurons.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01494480", "title": "The Clinical Trial on the Use of Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "General Hospital of Chinese Armed Police Forces", "summary": "Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intrathecal injection of umbilical cord mesenchymal stem cells can secret trophic factors that keep the motorneurons functional. The investigators have designed a phase I/II clinical trial to check the feasibility of this approach in humans.", "interventions": [{"type": "PROCEDURE", "name": "stem cell transplantation"}], "start_date": "2012-03", "url": "https://clinicaltrials.gov/study/NCT01494480", "target_entities": ["neuroprotection_motor_neuron_survival"], "locations": [{"facility": "Yihua An", "city": "Beijing", "state": "", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnose established following the World Federation of Neurology criteria\n* More than 6 and less than 36 months of evolution of the disease\n* Medullar onset of the disease\n* More than 20 and less than 65 years old\n* Forced Vital Capacity equal or superior to 50%\n* Total time of oxygen saturation \\<90% inferior to 2% of the sleeping time\n* Signed informed consent\n\nExclusion Criteria:\n\n* Neurological or psychiatric concomitant disease\n* Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube\n* Concomitant systemic disease\n* Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 12 months\n* Inclusion in other clinical trials\n* Unability to understand the informed consent", "sex": "ALL", "min_age": "20 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "umbilical cord mesenchymal stem cells", "targeting_mechanism": "Stem cell transplantation to secrete trophic factors that preserve motor neuron function and provide neuroprotection.", "targeting_mechanism_pmid": "", "animal_results": "Neural progenitor cells transduced with GDNF (glial cell line-derived neurotrophic factor) protected spinal motor neurons in animal models.", "animal_results_pmid": "36064599", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04686110", "title": "Analysis of Capillary Retinal and Papillary Vascularization in Patients With Amyotrophic Lateral Sclerosis - CAPISLA", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Angers", "summary": "Amyotrophic Lateral Sclerosis, or Charcot's disease, is a neurodegenerative disease affecting motor neurons. The disease affects between 5 and 10 people per 100,000 in the world, nearly 7,000 patients are affected in France. The only therapeutic treatment available to date in France is riluzole, which slows the progression of the disease. Amyotrophic Lateral Sclerosis is the first degenerative disease affecting motor neurons. However, recent evidence suggests that the impairment extends beyond motor neurons alone.\n\nOptical Coherence Tomography analyzes made it possible to highlight ophthalmologic damage in patients with Amyotrophic Lateral Sclerosis, in particular at the macula and papilla, although some results are contradictory.\n\nNo angiographic Optical Coherence Tomography analysis has been performed to date in patients with Amyotrophic Lateral Sclerosis. However, in the hypothesis of microvascular involvement participating in the pathophysiology of neurodegeneration in Amyotrophic Lateral Sclerosis, these examinations could provide relevant clinical and pathophysiological data by studying the retinal microvascularization of patients with the disease.", "interventions": [{"type": "OTHER", "name": "angiographic optical coherence tomography"}], "start_date": "2021-02-12", "url": "https://clinicaltrials.gov/study/NCT04686110", "target_entities": [], "locations": [{"facility": "CHU Angers", "city": "Angers", "state": "", "country": "France", "status": "RECRUITING", "lat": 47.47156, "lon": -0.55202}], "contact_phone": "0241353637", "contact_email": "jeanne.muller@chu-angers.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\nPatient Amyotrophic Lateral Sclerosis :\n\n* Patient diagnosed with bulbar or spinal ALS defined according to El Escorial criteria (probable or certain)\n* Hospitalized in a day hospital at the Angers University Hospital as part of his usual follow-up\n\nControl subject :\n\n* Subject not affected by the disease studied and without a history of neurological disease.\n* Subject matched in age and sex to a case (patient)\n\nFor all participants:\n\n* Major upon inclusion\n* Signature of informed consent to participate in the protocol\n\nExclusion Criteria:\n\nPatient Amyotrophic Lateral Sclerosis and control subject :\n\n* Simultaneous participation in another intervention protocol with an experimental treatment\n* Subject unable to express consent\n* Known ophthalmologic pathology (maculopathy, glaucoma, optic neuropathy, retinopathy whatever the etiology)\n* Diabetic subject\n* Cardiovascular history\n* Inability to perform the ophthalmological examinations of the study\n* Pregnant, lactating or parturient woman\n* Subject under duress psychiatric care\n* Subject to legal protection\n* Subject not affiliated or not beneficiary of a social security scheme", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "This is a diagnostic imaging study using angiographic optical coherence tomography to analyze retinal and papillary vascularization as a biomarker for ALS; it is not a therapeutic intervention trial.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02236897", "title": "PET Imaging in ALS Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "This is a pilot study to evaluate a potential imaging biomarker for aiding diagnosis and monitoring progression of ALS, based on a well established basic science pathway, published human autopsy data, preliminary data in ALS mutant mice, and our recently published data using brain PET scans to image the metabotropic glutamate receptor type 5 (mGluR5) in healthy human volunteers.", "interventions": [{"type": "OTHER", "name": "PET Scanning"}], "start_date": "2013-08", "url": "https://clinicaltrials.gov/study/NCT02236897", "target_entities": ["MGluR5"], "locations": [{"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female ALS patients, ranging in age from 18-80.\n* Must meet El Escorial Criteria for Probable or Definite ALS.\n* Disease duration \\>1 year, but \\<3 years.\n* Weakness in at least two extremities.\n* Forced vital capacity less than 80% and greater than 50%.\n\nExclusion Criteria:\n\n* Documented orthopnea or otherwise unable to lie flat in a PET scanner for 90 minutes.\n* Presence of pacemakers, aneurysm clips, shrapnel, or other implanted metallic devices that would preclude an MRI scan.\n* Absence of sufficient collateral arterial circulation for radial arterial line placement in both wrists.\n* Significant abnormalities of hepatic or renal function, or illicit substance use.\n* Positive drug screen. (Subjects currently taking prescribed narcotic medication who have a positive drug screen for this medication will not be excluded. Medication history will be obtained during screening).\n* Weighs \\> 350 lbs.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "This is a diagnostic imaging study using PET scanning to image metabotropic glutamate receptor type 5 (mGluR5) as a potential biomarker for ALS diagnosis and disease progression monitoring; it is not a therapeutic intervention trial.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01786174", "title": "Gilenya in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "The purpose of this study is to determine whether Gilenya, also known as fingolimod, is safe and tolerable in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Gilenya"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2013-08", "url": "https://clinicaltrials.gov/study/NCT01786174", "target_entities": ["S1P1"], "locations": [{"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Georgia Regents University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 18 years or older.\n2. Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria (Appendix 1).\n3. Onset of weakness or spasticity due to ALS \u2264 2 years (24 months) prior to Baseline Visit.\n4. Slow vital capacity (SVC) measure \u226565% of predicted for gender, height, and age at the screening visit.\n5. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to randomization (riluzole-na\u00efve subjects are permitted in the study).\n6. Subjects must be able to swallow oral medication at the Screening Visit and expected to be able to swallow the capsule throughout the course of the study.\n7. Capable of providing informed consent and following trial procedures.\n8. Geographically accessible to the site.\n9. Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and three months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for \u2265 3 months, barrier method in conjunction with spermicide, or another adequate method.\n10. Subjects must agree not to take live attenuated vaccines (including seasonal flu vaccine) 30 days before randomization, throughout the duration of the trial and for 60 days following the trial.\n\nExclusion Criteria:\n\n1. Prior use of fingolimod (Gilenya\u00ae).\n2. History or presence of cardiac conditions including:\n\n   1. Cardiovascular or cerebrovascular disease in the previous 6 months (eg. myocardial infarction, unstable angina, or stroke)\n   2. Congestive heart failure\n   3. First, second- or third-degree atrioventricular block, sick sinus syndrome, or other serious cardiac rhythm disturbances\n   4. Any history of Torsades de Pointes\n3. Treatment with a prohibited medication within 30 days of the Baseline Visit:\n\n   a. Class Ia or III antiarrhythmic medications: i.e., Quinidine, Sotalol Includes Nuedexta b. QT interval prolonging medications c. Ketoconazole d. Beta-blockers e. Calcium channel blockers f. Immunosuppressant medication g. Chemotherapeutic (anti-neoplastic) medications\n4. Evidence on examination or ECG of bradycardia (\\<55 bpm), QTc \\>450ms for women or \\>430 msec for men, or 1st degree or higher conduction block.\n5. History of unexplained syncope or cardiac syncope.\n6. Serum AST and ALT value \\>2.0 times the upper normal limit.\n7. Active infection (acute or chronic).\n8. History of diabetes.\n9. History of macular edema or uveitis.\n10. History of lymphopenia.\n11. History of acquired or inherited immune deficiency syndrome, including leukopenia.\n12. History of severe untreated chronic obstructive sleep apnea.\n13. Exposure to any other agent currently under investigation for the treatment of patients with ALS (off-label use or investigational) within 30 days of the Baseline Visit.\n14. Presence of tracheostomy.\n15. Use of non-invasive ventilation for hypoventilation due to ALS (such as BiPAP).\n16. Presence of feeding tube.\n17. Presence of diaphragmatic pacing system.\n18. The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to PI judgment, or a history of active substance abuse within the prior year.\n19. Clinically significant history of unstable or severe cardiac, oncologic, hepatic, or renal disease, or other medically significant illness.\n20. Pregnant women or women currently breastfeeding.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Gilenya (fingolimod)", "targeting_mechanism": "Sphingosine 1-phosphate receptor modulator that crosses the blood-brain barrier and modulates immune cell trafficking to reduce neuroinflammation in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04840823", "title": "Enoxacin for Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "McGill University", "summary": "The study will assess the safety of the drug enoxacin at specific dose levels in adults with ALS.", "interventions": [{"type": "DRUG", "name": "Enoxacin"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-03-26", "url": "https://clinicaltrials.gov/study/NCT04840823", "target_entities": ["PINK1"], "locations": [{"facility": "Montreal Neurological Institute-Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of familial or sporadic ALS\n* FVC of \u2265 50 percent predicted\n* If female, is not breastfeeding and is not pregnant\n* Has been on a stable dose of riluzole, or has not taken riluzole, for at least 30 days prior to screening\n* If taking concomitant edaravone at study entry, must have completed at least one cycle of edaravone therapy prior to screening\n* Not currently taking and has not taken for at least 30 days prior to screening any Theophylline containing medications, clozapine, or duloxetine\n* No active infection in the 30 days prior to randomization\n* Has not taken any fluoroquinolone antibiotics for at least 30 days prior to screening\n\nExclusion Criteria:\n\n* Hypersensitivity/allergy to fluoroquinolones\n* Diagnosed with another neurodegenerative disease\n* Significant pulmonary disorder not attributed to ALS, central nervous system disorder associated with seizures, myasthenia gravis, active rheumatologic disease, tendinopathy, or any severe uncontrolled medical condition (other than ALS)\n* Severe renal impairment or impaired liver function\n* Baseline prolongation of QT interval/corrected QT interval (QTc) at screening, treatment with any agent that may prolong Qt/QTc interval, or history of any other at-risk other cardiac condition\n* Currently enrolled in another clinical trial involving an experimental drug or device", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Enoxacin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05021536", "title": "Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment", "phase": "PHASE3", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Amylyx Pharmaceuticals Inc.", "summary": "The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS", "interventions": [{"type": "OTHER", "name": "Placebo"}, {"type": "DRUG", "name": "AMX0035"}], "start_date": "2021-10-28", "url": "https://clinicaltrials.gov/study/NCT05021536", "target_entities": ["TARDBP", "neuroinflammation"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of Southern California", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center Research Institute", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Augusta University Neuroscience Center", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins University School of Medicine Outpatient Center", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Healey & AMG Center for ALS Research at Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Hennepin Healthcare Research Institute", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Somnos Clinical Research", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Rutgers University", "city": "New Brunswick", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.48622, "lon": -74.45182}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of North Carolina at Chapel Hill", "city": "Chapel Hill", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.9132, "lon": -79.05584}, {"facility": "Wake Forest University Health Sciences", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Lewis Katz School of Medicine at Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Virginia Commonwealth University", "city": "Henrico", "state": "Virginia", "country": "United States", "status": "", "lat": 36.59264, "lon": -78.61611}, {"facility": "Swedish Neuroscience Institute", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "University Hospitals Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Hospices Civils de Lyon H\u00f4pital Neurologique Pierre Wertheimer Cellule Mutualis\u00e9e de Recherche Clinique (CMRC)", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "Hopital Gabriel Montpied Service de Neurologie", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "", "lat": 45.77969, "lon": 3.08682}, {"facility": "CHRU de Lille - H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges - H\u00f4pital Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "H\u00f4pitaux Universitaires de Marseille Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHU de Montpellier", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU Nice", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "H\u00f4pital de la Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Le Centre Hospitalier R\u00e9gional Universitaire de Tours", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Charit\u00e9 - Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Uniklinikum Dresden", "city": "Dresden", "state": "", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Hannover Medical School", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Jena University Hospital", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Medizinische Fakult\u00e4t Mannheim der Universit\u00e4t Heidelberg", "city": "Mannheim", "state": "", "country": "Germany", "status": "", "lat": 49.4891, "lon": 8.46694}, {"facility": "University Medical Center Rostock", "city": "Rostock", "state": "", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Ulm University Medical Centre", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Trinity College Dublin/Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Universit\u00e0 degli Studi di Bari Aldo Moro", "city": "Bari", "state": "", "country": "Italy", "status": "", "lat": 41.12066, "lon": 16.86982}, {"facility": "Centro Clinico NEMO", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "University of Milan Medical School", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Azienda Ospedaliero Universitaria Di Modena", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Universit\u00e0 degli Studi della Campania Luigi Vanvitelli", "city": "Naples", "state": "", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}, {"facility": "University of Padua", "city": "Padova", "state": "", "country": "Italy", "status": "", "lat": 44.38225, "lon": 11.14261}, {"facility": "University of Torino", "city": "Turin", "state": "", "country": "Italy", "status": "", "lat": 45.07049, "lon": 7.68682}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Centrum Medyczne Linden", "city": "Krakow", "state": "", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}, {"facility": "City Clinic Warsaw", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Centro Hospitalar Universit\u00e1rio Lisboa-Norte", "city": "Lisbon", "state": "", "country": "Portugal", "status": "", "lat": 38.72509, "lon": -9.1498}, {"facility": "Hospital del Mar", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitari de Bellvitge-IDIBELL", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Biodonostia Health Research Institute; Hospital Universitario Donostia", "city": "San Sebasti\u00e1n", "state": "", "country": "Spain", "status": "", "lat": 43.56667, "lon": -5.9}, {"facility": "Hospital Universitario y Polit\u00e9cnico La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Karolinska Institutet", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Ume\u00e5 University Hospital", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "King's College London", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "UCL Queen Square Institute of Neurology", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "University of Plymouth", "city": "Plymouth", "state": "", "country": "United Kingdom", "status": "", "lat": 50.37153, "lon": -4.14305}, {"facility": "Salford Royal Hospital Barnes", "city": "Salford", "state": "", "country": "United Kingdom", "status": "", "lat": 53.48771, "lon": -2.29042}, {"facility": "Sheffield Institute for Translational Neuroscience (SITraN)", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female, at least 18 years of age\n* Diagnosis of ALS (definite or clinically probable)\n* Time since onset of first symptom of ALS should be \\<24 months prior to randomization;\n* If the participant is to be treated with riluzole and/or edaravone during the course of the trial, then treatment with riluzole and/or edaravone was, at the time of the screening visit, started and maintained at a stable regimen for at least 14 days for riluzole and/or for a full treatment cycle for edaravone;\n* Capable of providing informed consent\n* Capable and willing to follow trial procedures including visits to the trial clinic and visit requirements;\n* Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug. Women must not be planning to become pregnant for the duration of the study and 3 months after last dose of study drug\n* Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide for sperm donation for the duration of the study and 3 months after last dose of study drug\n\nExclusion Criteria:\n\n* Presence of tracheostomy or permanent assisted ventilation(PAV)\n* Slow Vital Capacity (SVC) less than 55%\n* History of known allergy to phenyl butyrate or bile salts\n* Abnormal liver function defined as bilirubin levels and/or aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \\> 5 times the upper limit of the normal (obtained within 12 weeks from first dose)\n* Renal insufficiency as defined by eGFR \\<60 mL/min/1.73m\\^2 (obtained within 12 weeks from first dose)\n* Pregnant women (confirmed by a pregnancy test within 7 days of first dose) or women currently breastfeeding\n* Current severe biliary disease which may result in the Investigator medical judgement in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gangrene of the gallbladder, abscess of the gallbladder\n* History of Class III/IV heart failure (per New York Heart Association - NYHA)\n* Participant under severe salt restriction where the added salt intake due to treatment would put the participant at risk, in the Investigator clinical judgment\n* Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, according to Investigator judgment\n* Clinically significant unstable medical condition (other than ALS) (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, severe laboratory test anomaly or clinically significant electrocardiogram \\[ECG\\] changes) that would pose a risk to the participant if he/she were to participate in the trial, according to Investigator judgment\n* Previous treatment for ALS with cellular therapies or gene therapies\n* Currently enrolled in another trial involving use of an investigational therapy\n* Previous treatment with PB or taurursodiol within 30 days from Screening\n* Implantation of Diaphragm Pacing System (DPS)\n* Currently or previously treated within the last 30 days or planned exposure to any prohibited medications listed in Section 6.8 of the protocol", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AMX0035", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04057898", "title": "Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "MediciNova", "summary": "A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.", "interventions": [{"type": "DRUG", "name": "MN-166"}, {"type": "DRUG", "name": "placebo"}], "start_date": "2020-05-28", "url": "https://clinicaltrials.gov/study/NCT04057898", "target_entities": ["neuroinflammation"], "locations": [{"facility": "University of California", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Augusta University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Indiana University IU Health Neuroscience Center", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Hennepin Healthcare Research Institute", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Lehigh Valley Health Network", "city": "Allentown", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.60843, "lon": -75.49018}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Alberta Hospital", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "McMaster University Medical Center", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "Sunnybrook Research Institute", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "University of Saskatchewan - Sastakoon Hospital", "city": "Saskatoon", "state": "Saskatchwean", "country": "Canada", "status": "", "lat": 52.13238, "lon": -106.66892}, {"facility": "Hopital de L'Enfant-Jesus, CHU de Quebec-Universite Laval", "city": "Qu\u00e9bec", "state": "", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Major Inclusion Criteria:\n\n* Male or female subjects age 18 - 80 years, inclusive;\n* Diagnosis of familial or sporadic ALS as defined by the El Escorial-Revised (2000) research diagnostic criteria for ALS \\[clinically definite, clinically probable, probable-laboratory-supported\\];\n* ALS onset of \u226418 months from first clinical signs of weakness prior to screening;\n* If currently using riluzole, subject must be on a stable dose for at least 30 days prior to initiation of study drug;\n* If currently using edaravone, subject should have completed at least 14 days of their initial treatment cycle prior to initiation of study drug;\n* Last documented pulmonary function test result (i.e., slow vital capacity or forced vital capacity) must be greater than or equal to 70% predicted;\n* Able to swallow study medication capsules;\n* No known allergies to the study drug or its excipients;\n* Received pneumococcal vaccine within 6 years prior to starting clinical trial.\n\nMajor Exclusion Criteria:\n\n* Confirmed hepatic insufficiency or abnormal liver function (AST and/or ALT \\>3 times upper limit of normal);\n* Currently diagnosed with a clinically significant psychiatric disorder or dementia that would preclude evaluation of symptoms;\n* Currently use or treated with parenteral (intramuscular or intravenous) high dose (\\>25 mg/week) Vitamin B12 within 30 days prior to study drug administration;\n* Poor peripheral venous access that will limit the ability to draw blood as judged by the Investigator;\n* Currently participating, or has participated in a study with an investigational or marketed compound or device within 30 days or 5 half-lives, whichever is shorter, prior to signing the informed consent;\n* Use of tracheostomy or \\>22/24-hour ventilatory support.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MN-166 (Ibudilast)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00855400", "title": "Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia", "summary": "The purpose of this clinical trial is to asses the feasibility and the security of the intraspinal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients.", "interventions": [{"type": "PROCEDURE", "name": "Laminectomy and bone marrow stem cells transplantation"}, {"type": "PROCEDURE", "name": "Autologous bone marrow cells collection"}], "start_date": "2007-02", "url": "https://clinicaltrials.gov/study/NCT00855400", "target_entities": ["neuroprotection_motor_neuron_survival"], "locations": [{"facility": "Hospital Universitario Virgen de la Arrixaca", "city": "El Palmar", "state": "Murcia", "country": "Spain", "status": "", "lat": 37.93939, "lon": -1.16095}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnose established following the World Federation of Neurology criteria\n* More than 6 and less than 36 months of evolution of the disease\n* Medullar onset of the disease\n* More than 20 and less than 65 years old\n* Forced Vital Capacity equal or superior to 50%\n* Total time of oxygen saturation \\<90% inferior to 2% of the sleeping time\n* Signed informed consent\n\nExclusion Criteria:\n\n* Neurological or psychiatric concomitant disease\n* Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube\n* Concomitant systemic disease\n* Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 12 months\n* Inclusion in other clinical trials\n* Unability to understand the informed consent", "sex": "ALL", "min_age": "20 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous bone marrow stem cells", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03204500", "title": "Dual Treatment With Lithium and Valproate in ALS.", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "El Instituto Nacional de Neurologia y Neurocirugia Manuel Velasco Suarez", "summary": "This is a pilot study in 40 subjects with definite ALS to evaluate the efficacy of valproate and lithium carbonate. After a random assignation of the dual treatment vs. placebo, a follow-up of 20 months will allow to know the clinical and functional evolution so as the status of biomarkers under each treatment.", "interventions": [{"type": "COMBINATION_PRODUCT", "name": "Active treatment with dual therapy"}, {"type": "DRUG", "name": "Placebos"}], "start_date": "2016-05", "url": "https://clinicaltrials.gov/study/NCT03204500", "target_entities": ["histone_deacetylase_inhibition_neuroprotection", "wnt_signaling_pathway"], "locations": [{"facility": "Instituto Nacional de Neurologia Y Neurocirugia Mvs", "city": "Mexico City", "state": "Mexico City", "country": "Mexico", "status": "", "lat": 19.42847, "lon": -99.12766}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* patients aged 40 to 70 years\n* of both genders\n* female patients who are either postmenopausal for at least 24 months or who are able to practice 2 methods of contraception.\n* Clinical diagnosis of definite ALS supported by neurophysiological studies, according to El Escorial reviewed criteria and Awaji criteria.\n* Sporadic ALS, a priori.\n* Onset of weakness for 1 year \u00b1 6 months\n* Vital capacity of at least 60 % of the predicted value\n* Other treatment (with riluzole or not) at fixed dosis 2 months before and during all the clinical trial.\n* Patients who are willing to give informed consent\n* Without gastrostomy\n* Without jejunostomy\n* Without traqueostomy\n\nExclusion Criteria:\n\n* Age less than 25 years\\*\\*\n* Patients with uncontrolled diabetes\n* Patient with heart failure\n* Patient with respiratory vital capacity \\< 60%\n* Hepatic failure\n* Dysthyroidism\n* Do not give or sign informed consent\n* Women in lactation, pregnancy or possibility of pregnancy\n* Patients with significant sensory abnormalities and uncompensated medical illnesses\n* Laboratory abnormalities consistent with clinically significant cardiovascular, respiratory, haematological, metabolic, hepatic and renal disease.\n* Patients with gastrostomy\n* With jejunostomy\n* With nasogastric tube\n* Tracheotomy and invasive ventilation\n* Treatment with investigational drug within 3 months prior to screening\n\n  * Patients aged 26 to 39 years can be included at the discretion of medical researchers.", "sex": "ALL", "min_age": "40 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Valproate and lithium carbonate", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03136809", "title": "ALS Treatment Extension Study", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Collaborative Medicinal Development Pty Limited", "summary": "Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment.", "interventions": [{"type": "DRUG", "name": "Cu(II)ATSM"}], "start_date": "2018-01-18", "url": "https://clinicaltrials.gov/study/NCT03136809", "target_entities": ["copper_ii_complex"], "locations": [{"facility": "Macquarie University", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Signed informed consent prior to initiation of any study-specific procedures and treatment\n* Documented completion of protocol-specific assessments following completion of six 28-day treatment cycles in study CMD-2016-001\n* Principal Investigator considers the patient would benefit from continued treatment with Cu(II)ATSM\n* Not taking riluzole or on the same (or lower) dose used during the CMD-2016-001 study\n* Adequate bone marrow reserve, renal and liver function\n* Women and men with partners of childbearing potential must take effective contraception while on study treatment\n\nExclusion Criteria:\n\n* Inability to swallow oral medications or presence of a gastrointestinal disorder (e.g.,malabsorption) deemed to jeopardize intestinal absorption of study drug\n* Dependence on mechanical ventilation (invasive or non-invasive) for any part of day or night, where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use\n* Dementia that may affect either outcome measures or patient understanding and/or compliance with study requirements and procedures\n* Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Cu(II)ATSM", "targeting_mechanism": "Delivers copper to the central nervous system to restore copper homeostasis and prevent motor neuron degeneration.", "targeting_mechanism_pmid": "26826269", "animal_results": "CuATSM treatment extended lifespan in SOD1(G93A) mice and rescued early mortality in SOD(G93A) mice co-expressing the Copper-Chaperone-for-SOD when applied dermally.", "animal_results_pmid": "26826269", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04226144", "title": "Breath Stacking Technique Associated With Expiratory Muscle Training in Amyotrophic Lateral Sclerosis Patients", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Escola Superior de Ciencias da Saude", "summary": "it will be conducted a randomized parallel controlled trial with patients diagnosed with Amyotrophic Lateral Sclerosis (ALS) to compare two techniques to lung recruitment and cough augmentation, to assess their effects on pulmonary function, global functionally, swallowing and ability to speech in these population.", "interventions": [{"type": "DEVICE", "name": "Breath Stacking Group"}, {"type": "DEVICE", "name": "Breath stacking and EMT"}], "start_date": "2020-01-06", "url": "https://clinicaltrials.gov/study/NCT04226144", "target_entities": ["respiratory_function"], "locations": [{"facility": "Hospital de Apoio de Brasilia", "city": "Bras\u00edlia", "state": "Federal District", "country": "Brazil", "status": "", "lat": -15.77972, "lon": -47.92972}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of neuromuscular disease confirmed by neurologists at the referral center for neuromuscular diseases at Bras\u00edlia prior to screening for recruitment\n* age over 18 years\n* preserved cognition, evidenced by a score greater than or equal to 24 points in the Mini-Mental Status Exam;\n* no barium allergies\n* without tracheostomy or invasive mechanical ventilation\n* no diaphragmatic pacemaker\n* without associated respiratory disease\n\nExclusion Criteria:\n\n* pregnancy\n* previous kidney disease or other concomitant diseases .respiratory diseases and hospitalization in intensive care units (ICUs) during the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00424463", "title": "Expanded Controlled Study of Safety and Efficacy of MCI-186 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "This is a long-term, double-blind, placebo-controlled study of MCI-186 to treat ALS. This study is the long-term extension of Study NCT00330681; Study NCT00330681 is a Phase 3, randomized, double-blind, placebo control, parallel assignment, 24-week study in the treatment of ALS. The objectives of this study are to assess the efficacy and safety of long-term intermittent therapy with 60 mg MCI-186 to ALS patients.", "interventions": [{"type": "DRUG", "name": "MCI-186"}, {"type": "DRUG", "name": "Placebo of MCI-186"}], "start_date": "2007-01-31", "url": "https://clinicaltrials.gov/study/NCT00424463", "target_entities": ["oxidative_stress"], "locations": [{"facility": "National Hospital Organization Miyagi National Hospital", "city": "Watari-gun", "state": "Miyagi", "country": "Japan", "status": "", "lat": null, "lon": null}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients who ware completed drug administration without discontinuation in the preceding confirmatory study NCT00330681.\n\nExclusion Criteria:\n\n* Patients with such complications as Parkinson's disease, schizophrenia, dementia, renal failure, or other severe complication, and patients who have the anamnesis of hypersensitivity to edaravone.\n* Patients whose creatinine clearance is 50mL/min or less at the time of completion of drug administration in the study NCT00330681.\n* Pregnant, lactating, and probably pregnant patients, and patients who want to become pregnant, and patients who can not agree to contraception.\n* Patients who are participating in other clinical trials except the study NCT00330681.\n* In addition to the above exclusion criteria, patients judged to be inadequate to participate in this study by their physician.", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MCI-186", "targeting_mechanism": "Reduces oxidative stress through antioxidant mechanisms.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05176093", "title": "A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Helixmith Co., Ltd.", "summary": "The purpose of this study is to evaluate the long-term safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis who were previously randomized, received treatment, and completed the Day 180 Visit of Study VMALS-002-2. Safety will be assessed by incidences of treatment-emergent adverse events, treatment emergent serious adverse events, adverse events of special interest, and the clinically significant laboratory values.", "interventions": [{"type": "BIOLOGICAL", "name": "Engensis"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2021-11-14", "url": "https://clinicaltrials.gov/study/NCT05176093", "target_entities": ["motor_neuron_degeneration"], "locations": [{"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Hanyang University Medical Center", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Participants who complete the Day 180 Visit in VMALS-002-2 are eligible to enroll in this extension study, VMALS-002-2b.\n\nExclusion Criteria:\n\n\\- None", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Engensis", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06719947", "title": "HD-tDCS in Amyotrophic Lateral Sclerosis: A Multicenter Randomized Controlled Trial", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Universidade Federal do Rio Grande do Norte", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a nervous system disease that causes muscle weakness and rapidly progresses to the loss of mobility and functionality. Studies suggest that High-Definition Transcranial Direct Current Stimulation (HD-tDCS) is a technique for modulating motor cortical hyperexcitability. However, evidence on the use of HD-tDCS as a neuromodulator of the diaphragmatic motor cortex in people with ALS is inconclusive.", "interventions": [{"type": "DEVICE", "name": "Active HD-tDCS"}, {"type": "DEVICE", "name": "Simulated HD-tDCS"}], "start_date": "2025-11-28", "url": "https://clinicaltrials.gov/study/NCT06719947", "target_entities": ["cortical_hyperexcitability"], "locations": [{"facility": "Universidade de Bras\u00edlia - Campus Ceil\u00e2ndia", "city": "Bras\u00edlia", "state": "Federal District", "country": "Brazil", "status": "RECRUITING", "lat": -15.77972, "lon": -47.92972}, {"facility": "PneumoCardioVascular Lab - HUOL/UFRN", "city": "Natal", "state": "Rio Grande do Norte", "country": "Brazil", "status": "RECRUITING", "lat": -5.795, "lon": -35.20944}, {"facility": "Universidad Aut\u00f3noma de Chile", "city": "Santiago", "state": "Santiago Metropolitan", "country": "Chile", "status": "NOT_YET_RECRUITING", "lat": -33.45694, "lon": -70.64827}, {"facility": "Universidad do Chile", "city": "Santiago", "state": "Santiago Metropolitan", "country": "Chile", "status": "NOT_YET_RECRUITING", "lat": -33.45694, "lon": -70.64827}], "contact_phone": "+55(84)99426-7896", "contact_email": "guilherme.fregonezi@ufrn.br", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Both sexes; diagnosis of ALS according to the revised El Escorial criteria;\n* Age between 18 and 80 years;\n* Forced Vital Capacity greater than 50% of predicted;\n* Sniff nasal inspiratory pressure greater than 40 cmH2O;\n* A telephone number to contact the care team and who signed the study consent form.\n\nExclusion Criteria:\n\n* Subjects who are unable to understand or perform any of the study procedures;\n* Subjects who do not agree to participate or voluntarily request withdrawal from the study at any time;\n* Subjects with cardiac, respiratory, or musculoskeletal comorbidities;\n* Subjects using invasive mechanical ventilation;\n* Subjects with a tracheostomy;\n* Subjects with a pacemaker;\n* Subjects with metallic brain implants or other electronic implants;\n* Subjects with a cochlear implant;\n* Subjects with epileptic activity or a history of epilepsy, or a family history of epilepsy;\n* Subjects with a history of stroke or tumor;\n* Subjects prone to severe hemodynamic fluctuations, acute infectious processes, and/or inflammatory conditions;\n* Pregnant women at the time of recruitment;\n* Subjects who are unable to complete the intervention protocol.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "High-Definition Transcranial Direct Current Stimulation modulates motor cortical hyperexcitability, potentially targeting the diaphragmatic motor cortex.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05568615", "title": "Extension Study Following the Studies MT-1186-A03 or A04 to Evaluate the Safety of Oral Edaravone in Subjects With ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "The purpose of this study is to evaluate the safety of oral edaravone at a dose of 105 mg administered once daily for 10 days out of a 14-day period, followed by a 14-day drug-free period. This study will be continued until the earlier date when oral edaravone is commercially available at each site in Japan or August 2023.", "interventions": [{"type": "DRUG", "name": "MT-1186"}], "start_date": "2022-10-26", "url": "https://clinicaltrials.gov/study/NCT05568615", "target_entities": ["oxidative_stress"], "locations": [{"facility": "National Hospital Organization Higashinagoya National Hospital", "city": "Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "National Hospital Organization Chibahigashi National Hospital", "city": "Chiba", "state": "Chiba", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "Fukushima Medical University Hospital", "city": "Fukushima", "state": "Fukushima", "country": "Japan", "status": "", "lat": 37.75, "lon": 140.46667}, {"facility": "Kagawa University Hospital", "city": "Kita-gun", "state": "Kagawa-ken", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Kitasato University Hospital", "city": "Sagamihara-shi", "state": "Kanagawa", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Yokohama City University Hospital", "city": "Yokohama", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.43333, "lon": 139.65}, {"facility": "National Hospital Organization Kumamoto Saishun Medical Center", "city": "K\u014dshi", "state": "Kumamoto", "country": "Japan", "status": "", "lat": 32.89271, "lon": 130.77567}, {"facility": "National Hospital Organization Osaka Toneyama Medical Center", "city": "Toyonaka-shi", "state": "Osaka", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Shiga University of Medical Science Hospital", "city": "\u014ctsu", "state": "Shiga", "country": "Japan", "status": "", "lat": 35.0, "lon": 135.86667}, {"facility": "Tokyo Metropolitan Neurological Hospital", "city": "Fuchu-shi", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "National Epilepsy Center NHO Shizuoka Institute of Epilepsy and Neurological Disorders", "city": "Shizuoka", "state": "", "country": "Japan", "status": "", "lat": 34.98333, "lon": 138.38333}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subjects must provide a signed and dated informed consent form (ICF) to participate in the study.\n* Subjects must be able (in the judgment of the Investigator) to understand the nature of the study and all risks involved with participation in the study\n* Subjects must be willing to cooperate and comply with all protocol restrictions and requirements.\n* Subjects who successfully complete Week 96 of Study MT-1186-A03 or Week 48 of Study MT-1186-A04 and have been compliant with study drug (80-120%).\n\nExclusion Criteria:\n\n* Subjects of childbearing potential unwilling to use a highly effective method of contraception from the Visit #1 until 3 months after the last dose of study medication.\n* Subjects who have a significant risk of suicide. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a specific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the C-SSRS at Week 96 of the A03 study or at Week 48 of the A04 study.\n* Subjects who are not eligible to continue in the study, as judged by the Investigator.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MT-1186", "targeting_mechanism": "Free radical scavenger and antioxidant that reduces oxidative stress", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02288091", "title": "A Pilot Study of Inosine in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "This is a single center, open label, 12-week study of inosine treatment. Inosine treatment leads to an increase in the levels of urate (uric acid) in the blood.\n\nThe primary objective of the study is to determine the tolerability of oral administration of inosine.\n\nSecondary study objectives include the measurement of biomarkers of oxidative stress and damage in response to inosine treatment.", "interventions": [{"type": "DRUG", "name": "Inosine"}], "start_date": "2015-01", "url": "https://clinicaltrials.gov/study/NCT02288091", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 18 years or older.\n2. Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria (Appendix 1).\n3. Capable of providing informed consent and following trial procedures.\n4. Serum urate \\< 5.5 mg/dl at screening (i.e. below the population median serum urate levels).\n5. Willingness to undergo magnetic resonance spectroscopy (MRS) at Baseline and at Week 12 of the study.\n6. Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and 3 months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for \u2265 3 months, barrier method in conjunction with spermicide, or another adequate method.\n\nExclusion Criteria:\n\n1. History of urolithiasis.\n2. Urine pH \\< 5.5 at screening (as acidic urine is a major determinant of uric acid urolithiasis).\n3. Urate crystalluria at Screening.\n4. History of gout.\n5. History of stroke or myocardial infarction.\n6. History of symptomatic coronary artery disease (e.g. angina pectoris) or symptomatic peripheral arterial disease within 1 year prior to Screening.\n7. Symptomatic congestive heart failure with a documented ejection fraction below 45%.\n8. Poorly controlled arterial hypertension (SBP\\>160mmHg or DBP\\>100mmHg at Screening).\n9. Contraindications to undergo magnetic resonance spectroscopy (MRS) at Baseline and at Week 12 of the study such as history of claustrophobia, inability to lie flat for approximately one hour, or metal implants (metal pins or plates, extensive non-removable dental work, cerebral aneurysm clips, pacemaker).\n10. Women who are pregnant or lactating.\n11. The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to PI judgment, or a history of active substance abuse within the prior year.\n12. Anything that, in the opinion of the investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.\n13. Use of the following within 30 days prior to Screening: inosine, allopurinol, probenecid, more than 300mg vitamin C daily (note that a subject may take a standard multivitamin up to one tablet or capsule daily). Use of thiazides is permissible as long as the subject is on a stable dose from 1 week prior to Screening.\n14. Known hypersensitivity or intolerability to inosine.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Inosine", "targeting_mechanism": "Increases urate levels to provide antioxidant protection against oxidative stress", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06315608", "title": "MRG-001 in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "MedRegen LLC", "summary": "The proposed study is an Open-Label, Single-Dose Study to Assess the Safety, and Pharmacodynamics (PD) signals of MRG-001 in Patients with Amyotrophic Lateral Sclerosis (ALS). MRG-001 will be administered subcutaneously 3 times per week for 2 weeks. This cycle will be repeated for 3 months. In total, patients are expected to receive 18 injections over the span of 3 months.", "interventions": [{"type": "DRUG", "name": "MRG-001"}], "start_date": "2025-07-01", "url": "https://clinicaltrials.gov/study/NCT06315608", "target_entities": ["neuroinflammation"], "locations": [], "contact_phone": "443-759-8563", "contact_email": "info@medregenco.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Able to provide written informed consent (either from patient or patient's legally acceptable representative and complying with study procedures, in the PI's opinion.\n* Male or female patients between 18-75 years.\n* Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS defined by revised El Escorial criteria.\n* Time since onset of weakness due to ALS \u2264 48 months at the time of the Screening Visit\n* Vital Capacity \u2265 50% of predicted capacity for age, height, and sex at the time of the Screening Visit measured by Slow Vital Capacity (SVC), or Forced Vital Capacity (FVC).\n* Patients must either not take Riluzole or be on a stable dose of Riluzole for \u2265 30 days prior to the Master Protocol Screening Visit. Riluzole-na\u00efve participants are permitted in the study.\n* Participants must either not take Edaravone or have completed at least one cycle of edaravone prior to the Master Protocol Screening Visit. Edaravone-na\u00efve participants are permitted in the study.\n* Participants must either not take Relyvrio (AMX0035) or be on a stable dose of Relyvrio for \u2265 30 days prior to the Master Protocol Screening Visit. Relyvrio-na\u00efve participants are permitted in the study.\n* Women of child-bearing potential (defined as females who are not surgically sterile or who are not over the age of 52 and amenorrhoeic for at least 12 months) must utilize appropriate birth control throughout the study duration.\n* Male patients must agree to use a medically acceptable method of contraception /birth control throughout the study duration.\n\nExclusion Criteria:\n\n* Subjects who meet one or more of the following criteria will not be considered eligible to participate in the clinical study:\n* Participation in another interventional clinical trial (drug or device) within 30 days of Screening and at any time during the study.\n* Significant pre-existing organ dysfunction prior to randomization:\n* Lung: Receiving supplemental home oxygen therapy at baseline for pre-existing medical condition (other than COVID-19), as documented in medical record.\n* Heart: Pre-existing congestive heart failure defined as an ejection fraction \\<20% as documented in the medical record. Clinically significant ventricular arrhythmias (ventricular tachycardia, ventricular fibrillation), unstable angina, myocardial infarction (past 3 months), heart and coronary vessel surgery (past 3 months), significant valvular heart disease, uncontrolled arterial hypertension with systolic blood pressure \\>180 mm Hg and diastolic blood pressure \\>110 mm Hg.\n* Renal: End-stage renal disease requiring renal replacement therapy or creatinine clearance \\<50 mL/min.\n* Hematologic: Baseline platelet count \\<30,000/mm3 or hemoglobin levels \\<6.0 g/dL.\n* Neurological: Stage \u22653 hepatic encephalopathy by West Haven criteria.\n* History of splenectomy or splenomegaly (spleen weighing \\> 750 g).\n* Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.\n* Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.\n* Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational) including tofersen (Qalsody).\n* History of splenectomy or splenomegaly (spleen weighing \\>750 g).\n* Co-infection with human immunodeficiency virus (HIV).\n* History of organ or bone marrow transplantation, other than a corneal transplant.\n\nor recent (within 3 months) chronic use of immunosuppressive drugs (tacrolimus, mycofenolate mofetil, cyclosporine, rapamycine, hydrochloroquine, azathiopurine, methotrexate), e.g., biologicals, JAK1/2 inhibitors, interferons, interleukins or (prednisone or related corticosteroids are allowed).\n\n* Hypersensitivity to either of the components of MRG-001.\n* If female, known pregnancy, or has a positive serum pregnancy test, or lactating/breastfeeding.\n* Underlying diseases that, in the opinion of the site investigator, might be complicated or exacerbated by proposed treatments or might confound assessment of study drug.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MRG-001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04322149", "title": "Multiple Doses of AT-1501-A201 in Adults With ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Anelixis Therapeutics, LLC", "summary": "This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40 ligand (CD40L). Approximately 54 adults with Amyotrophic Lateral Sclerosis (ALS) will be enrolled into the study in the United States and Canada at approximately 13 ALS treatment sites.\n\nParticipants will be enrolled into one of four ascending doses.", "interventions": [{"type": "DRUG", "name": "AT-1501"}], "start_date": "2020-10-16", "url": "https://clinicaltrials.gov/study/NCT04322149", "target_entities": ["CD40L"], "locations": [{"facility": "Barrows Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Augusta University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "University of Indiana", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "The University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University Medical Center", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Hospital for Special Surgery (HSS)", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Providence Brain & Spine Institute", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Texas Neurology, P.A.", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Houston Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. ALS diagnosed as possible, laboratory supported probable, probable, or definite as defined by revised El Escorial criteria\n2. ALS Functional Rating Scale - Revised (ALSFRS-R) Aggregate score of 37 or greater\n3. No more than 24 months from diagnosis\n\nExclusion Criteria:\n\n1. Any other central or peripheral nervous system disease that may interfere with the evaluation of ALS or its progression\n2. Presence of a tracheostomy, or use of permanent assistive ventilation (ventilatory support for 23 hours per day or more)\n3. History of malignancy within the previous 5 years, except for localized non-melanoma skin cancers\n4. Abnormal function of the immune system resulting from:\n\n   * Clinical conditions affecting the immune system (e.g. HIV infection, agammaglobulinemia),\n   * Systemic administration of corticosteroids (PO/IV/IM) at a dose equivalent to 20 mg/day of prednisone for more than 14 consecutive days within 90 days prior to screening,\n   * Administration of anti-neoplastic and/or immunomodulating agents (e.g. Tumor necrosis factor alpha (TNF \u03b1) antagonists or anti-B cell antibodies) or radiotherapy within 1 year prior to screening.\n5. Recipient of Stem Cell or Gene Therapy\n6. Positive test for Hepatitis B surface antigen, Hepatitis C antibody, or HIV.\n7. History of deep venous thrombosis or pulmonary embolism\n8. History of active substance abuse within the past 2 years\n9. History of stroke, poorly controlled or significant cardiovascular disease, diabetes", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AT-1501", "targeting_mechanism": "Humanized monoclonal antibody antagonist to CD40 ligand (CD40L) that modulates immune response", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07091799", "title": "Interest of Measuring P2X4 Receptors on Blood Monocytes as a Diagnostic Marker in Amyotrophic Lateral Sclerosis: P2X4 as a Diagnostic Biomarker for ALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Bordeaux", "summary": "Amyotrophic lateral sclerosis (ALS) is the most common form of motor neuron disease and is characterized by the degeneration of motor neurons leading to progressive paralysis and death within 3 to 5 years after diagnosis. To date, no key mechanism had been identified. Our associated laboratory has identified the P2X4 purinergic pathway that appears to be involved in the pathogenesis of ALS. Our goal is to verify these results at the human level in order to have a proof of concept of P2X4's role as a biomarker of the disease.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "P2X4 receptors in blood samples"}], "start_date": "2026-01-15", "url": "https://clinicaltrials.gov/study/NCT07091799", "target_entities": ["P2X4"], "locations": [{"facility": "H\u00f4pital Pellegrin", "city": "Bordeaux", "state": "", "country": "France", "status": "RECRUITING", "lat": 44.84124, "lon": -0.58046}], "contact_phone": "05 57 82 13 70", "contact_email": "gwendal.le-masson@chu-bordeaux.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* For ALS group: Person presenting a probable or confirmed diagnosis of ALS according to the criteria of EI Escorial.\n* Adult.\n* Person affiliated or beneficiary of a social security scheme.\n* Free, informed and written consent signed by the participant or by a third person (in case of physical incapacity of the participant), after information on the study.\n\nExclusion Criteria:\n\n* People undergoing immunosuppressive or corticosteroid treatments.\n* Participation in a research protocol with an experimental treatment.\n* People placed under guardianship, curatorship or legal protection.\n* For healthy volunteer, people directly related to the patient (siblings, descendants and ancestry).", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "P2X4 receptors in blood samples", "targeting_mechanism": "P2X4 purinergic pathway involved in ALS pathogenesis via intracellular calcium dysregulation", "targeting_mechanism_pmid": "27453058", "animal_results": "Several ALS-related misfolded proteins including mutants of SOD1 or TDP-43 lead to a significant increase in surface P2X4 receptor density and function in SOD1-G93A mice", "animal_results_pmid": "35852606", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06021938", "title": "Alleviating Persistent Dyspnea in Amyotrophic Lateral Sclerosis Patients Treated With Non-Invasive Ventilation Through Immersive Virtual Reality", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "The evolution of amyotrophic lateral sclerosis (ALS) is marked by dyspnea, anxiety and pain, major determinants of suffering induced by this disease. The only palliative treatment for respiratory failure is non-invasive ventilation (NIV), which compensates failing respiratory muscles and relieves dyspnea, improves quality of life and increases life expectancy. In ALS patients, the persistence of dyspnea outside of NIV sessions has highlighted the need for therapeutic alternatives in the treatment of persistent dyspnea, including immersive virtual reality (IVR) and auditory distraction through music (music therapy). This study evaluates the effect of IVR on respiratory discomfort in ALS patients with persistent dyspnea treated with NIV.", "interventions": [{"type": "DEVICE", "name": "Immersive virtual reality (IVR) & Music therapy"}], "start_date": "2024-04-04", "url": "https://clinicaltrials.gov/study/NCT06021938", "target_entities": [], "locations": [{"facility": "Service de Pneumologie", "city": "Paris", "state": "France", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "01 42 16 77 71", "contact_email": "capucine.morelot@aphp.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* \u2265 18 years old\n* Diagnosis of ALS confirmed according to the revised criteria of El Escorial\n* Respiratory failure due to diaphragmatic dysfunction treated by non-invasive ventilation for more than a month\n* Care provided in an ambulatory setting (day care hospital)\n* Persistent dyspnea at rest \u2265 3 across a numerical scale (0 to 10) in a semi-sitting position\n* Stable clinical condition, i.e., no episode of acute cardiac, respiratory and/or neurological failure leading to hospitalization in the previous 4 weeks\n* Free, prior and informed written consent about the study has been obtained\n* Benefiting a social security (French health insurance system)\n\nExclusion Criteria:\n\n* Neurological disorders according to a neurological evaluation dating from less than one year, in particular diagnosed dementia (frontotemporal dementia, Alzheimer's disease, etc.), brain pathology (tumor, stroke, Parkinson's disease, etc.)\n* Diagnosed psychiatric illness (severe depression, psychosis) or receiving antipsychotic treatment\n* Acrophobia\n* Claustrophobia\n* Photophobia\n* Hearing loss\n* Visual impairment\n* Subject under guardianship or curatorship", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06649955", "title": "Controlling Amyotrophic Lateral Sclerosis Motor Neuron Excitability Study", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "PathMaker Neurosystems Inc.", "summary": "Following completion of the ALS Early Feasibility Study of the MyoRegulator\u00ae device for treatment of ALS (NCT06165172), the CALM study will further assess the feasibility of the MyoRegulator\u00ae device to treat ALS in an expanded number of individuals with ALS. CALM will gather additional preliminary evidence of clinical safety and potential effectiveness in this patient population with a longer follow-up period and additional secondary endpoints in a single-arm study prior to commencing a larger sham-controlled pivotal trial.", "interventions": [{"type": "DEVICE", "name": "Multi-site direct current stimulation (DCS)"}], "start_date": "2025-02-10", "url": "https://clinicaltrials.gov/study/NCT06649955", "target_entities": [], "locations": [{"facility": "Beth Israel Deaconess Medical Center", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "617-667-3083", "contact_email": "tokanlom@bidmc.harvard.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. 18-80 years of age inclusive\n2. Sporadic or familial ALS diagnosed as clinically possible, probable, lab-supported probable, or definite ALS as defined by revised El Escorial criteria\n3. Less than or equal to 3 years since ALS symptom onset\n4. Slow Vital Capacity \u2265 50% of predicted capacity at the time of Screening as determined using a portable spirometer\n5. For TTNCS: Median CMAP \u2265 1.5 mV\n6. Willing to forgo botulinum toxin, phenol or alcohol injections, intrathecal baclofen, digitalis, and morphine for the study duration\n7. Willing to refrain from participation in any other therapeutic clinical trial or investigational product for ALS for the duration of this study\n8. Women must not be able to become pregnant (e.g., post-menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and 3 months after study completion.\n9. Stable dose of rilutek (Riluzole), edaravone (Radicava), or tofersen (Qualsody) and oral medications for muscle spasms/cramps (e.g. mexiletine, quinine, quinidine, magnesium, gabapentin, oxcarbazepine, baclofen) for at least 30 days prior to the onset of participation in the study\n10. ALS Functional Rating Score (ALSFRS-R) of greater than or equal to 35\n11. Willing and able to give informed consent\n\nExclusion Criteria:\n\n1. Study participants who are on permanent assisted ventilation (PAV) defined as \\>22h of noninvasive or invasive ventilation a day for \\> 7 consecutive days.\n2. Study participants who have been diagnosed with ALS having only clinical bulbar involvement\n3. Implanted intrathecal pump\n4. Prior botulinum toxin injection(s) at any site within 12 weeks of study enrollment\n5. Prior phenol or alcohol injections for spasticity within 6 months of study enrollment\n6. Presence of potential tsDCS and/or TMS risk factors:\n\n   1. Damaged skin at the stimulation sites (i.e., skin with ingrown hairs, acne, razor nicks, wounds that have not healed, recent scar tissue, broken skin, etc.)\n   2. Presence of an electrically, magnetically or mechanically activated implant (including cardiac pacemaker) or any other electrically sensitive support system with the exception of loop recorders\n   3. Ferromagnetic metal in the head, neck or any site of stimulation including, but not limited to, aneurysm clips, implanted medication pumps, implanted brain stimulators, pacemakers, cochlear implants, implanted metal prostheses or metal due to any injury; dental fillings are permitted. Jewelry must be removed during stimulation\n   4. Seizures or unexplained spells of loss of consciousness during the previous 12 months\n   5. Any cardiac abnormality that may be exacerbated by transthoracic electrical stimulation\n   6. History of cord lesions or previous spinal surgery that may interfere with procedure as determined by the study MD\n   7. History of intracranial brain lesions, cortical stroke or previous neurosurgery that may interfere with TMS (e.g., in regions to be stimulated for TMS evaluations) as reviewed and approved by the study MD\n7. Any medical condition that would prevent the participant from being able to participate in the clinical outcome measures\n8. Pregnant females, as determined by a pregnancy test at V1 (in females of child-bearing potential)", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Direct current stimulation to modulate motor neuron excitability.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04490148", "title": "Remote Pulmonary Function Testing and Nurse Coaching in ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Milton S. Hershey Medical Center", "summary": "Comparison of respiratory outcomes in patients receiving telemedicine-guided remote pulmonary function testing (rPFT) with or without the additional support of nurse coaching. This is a randomized controlled study which assesses the effects rPFT and coaching on respiratory outcomes and quality of life.", "interventions": [{"type": "DEVICE", "name": "remote pulmonary function testing"}, {"type": "DEVICE", "name": "standard pulmonary function testing"}, {"type": "BEHAVIORAL", "name": "Nurse Respiratory Health Coaching (NRHC)"}], "start_date": "2020-07-01", "url": "https://clinicaltrials.gov/study/NCT04490148", "target_entities": [], "locations": [{"facility": "Hershey Medical Center ALS Clinic", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nPatients:\n\n1. Possess a diagnosis of definite, probable, probable laboratory-supported, or possible ALS by revised El Escorial research criteria \\[Brooks2000\\].\n2. Be 18 years of age or older.\n3. Have a caregiver available to participate in the study\n4. Symptom onset within the last three years.\n5. Have a computer and home internet service sufficient for engaging in telemedicine sessions.\n6. Have a second device capable of downloading the spirometer application from an app store (Android- or iOS-based smartphone or tablet).\n\nCaregivers:\n\n1. Be 18 years of age or older, of either gender.\n2. Be able and willing to provide informed consent.\n\nExclusion Criteria:\n\nPatients:\n\n1. Use of NIV or diaphragm pacer at time of obtaining informed consent.\n2. FVC \u226450% predicted or MIP \\> -60 cm of water.\n3. ALS Functional Rating Scale (ALSFRS-R) \\[Cedarbaum1999\\] score on day of screening of \u22652 on items for speech, swallowing, and salivation. These items are indicators of bulbar dysfunction, which limits the reliability of PFT administration.\n4. Cognitive impairment, as judged by the ALS clinic neurologist, that prevents participation in the study.\n\nCaregivers: None", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06819358", "title": "Individualized Functional Imaging-Guided Repetitive Transcranial Magnetic Stimulation (rTMS) for Treating Postural Gait Disorders in Patients with Amyotrophic Lateral Sclerosis (ALS): a Randomized, Crossover, Controlled, Double-Blind Clinical Study", "phase": "EARLY_PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University Third Hospital", "summary": "This study is a randomized, crossover, controlled, double-blind clinical trial. Patients (n=45) were randomly divided into Group A and Group B. Patients in Group A will receive 2 weeks (10800 Hz daily, 5 days\u00d72) of Transcranial magnetic stimulation\uff08TMS\uff09 treatment, while patients in Group B will receive sham stimulation with the same frequency. After a 4-week washout period, the two groups cross over. Patients in Group A will receive sham stimulation, and patients in Group B will receive TMS treatment for 2 weeks (10800 Hz daily, 5 days\u00d72). Clinical functional scales, imaging evaluations, and gait analysis will be conducted at baseline, after 2 weeks of treatment, before crossover treatment, and after 2 weeks of crossover treatment. The therapist, patients, and assessors will be all blinded throughout the study.", "interventions": [{"type": "DEVICE", "name": "Transcranial Magnetic Stimulation"}], "start_date": "2025-02-14", "url": "https://clinicaltrials.gov/study/NCT06819358", "target_entities": [], "locations": [], "contact_phone": "+86 15801224009", "contact_email": "15801224009@163.COM", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Aged 18-80 years;\n2. Diagnosis of motor neuron disease at probable level or above based on Estorial criteria;12\n3. Meet UMND ALS diagnosis criteria: at least three segments of upper motor neuron damage localized to 1-2 muscles, or EMG indicating loss of innervation in 1-2 muscles;13\n4. Presence of lower limb dysfunction: Berg balance scale score below 40;\n5. Capable of standing independently for more than 30 seconds and able to walk or walk with assistance;\n6. Stable medication dosage for at least one month;\n7. FVC \\> 60%;\n8. Signed informed consent.\n\nExclusion Criteria:\n\n1. History of substance abuse within the past 6 months;\n2. History of epilepsy or first-degree relative with epilepsy;\n3. Patients with severe systemic diseases in the heart, lungs, liver, or kidneys that cannot be controlled with routine medications, based on laboratory results;\n4. Patients with severe depression or anxiety (HAMD-17 score \u226518; HAMA score \u226521) or diagnosed with other mental illnesses;\n5. Patients with a life expectancy of less than one year due to reasons other than neurodegenerative diseases;\n6. Pregnant women or those planning to become pregnant;\n7. Individuals with a pacemaker, cochlear implant, or other metallic foreign bodies and any implanted electronic equipment, or those with contraindications for MRI scanning or TMS treatment, such as claustrophobia;\n8. Patients who have received TMS, transcranial electrical stimulation, transcranial focused ultrasound, or other neuromodulation treatments within three months prior to enrollment;\n9. Other abnormal examination results deemed unsuitable for participation by the research investigator;\n10. Inability to cooperate for follow-ups due to geographical or other reasons;\n11. Participation in other clinical research trials.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Repetitive transcranial magnetic stimulation to modulate motor cortex function and motor neuron activity.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01232738", "title": "Trial of Safety and Efficacy of Rasagiline in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Yunxia Wang, MD", "summary": "ALS is a disorder that weakens motor strength and lung function. Rapid loss of motor neurons in the brain and spinal cord of ALS patients causes the symptoms of increasing weakness and loss of muscle function. While there are drugs to help relieve symptoms of ALS, there is no cure for ALS.\n\nRasagiline is a drug with possible neuroprotective characteristics. Neuroprotective means that the nervous system may be protected against weakening. It is known that rasagiline has possible neuroprotective characteristics and it is approved for use for patients with another disorder, the effectiveness of rasagiline for patients with ALS has not been tested.", "interventions": [{"type": "DRUG", "name": "rasagiline"}], "start_date": "2011-12", "url": "https://clinicaltrials.gov/study/NCT01232738", "target_entities": ["Monoamine oxidase B"], "locations": [{"facility": "Phoenix Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Iowa", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University Of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "University of Nebraska Medical Center", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "", "lat": 41.25626, "lon": -95.94043}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Tennessee", "city": "Memphis", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.14953, "lon": -90.04898}, {"facility": "The Methodist Hospital System", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "McGill University", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. A clinical diagnosis of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criteria, by the study investigator (Appendix IV).\n2. 21 to 80 years of age inclusive.\n3. VC greater or equal to 75% of predicted at screening and baseline.\n4. Onset of weakness within 3 years prior to enrollment.\n5. If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit.\n6. Women of childbearing age must be non-lactating and surgically sterile or using an effective method of birth control and have a negative pregnancy test.\n7. Willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).\n\nExclusion criteria\n\n1. Requirement for tracheotomy ventilation or non-invasive ventilation for \\> 23 hours per day.\n2. Patients on sympathomimetic agents. This includes pseudoephedrine, phenylephrine, phenylpropanolamine, and ephedrine.\n3. Patients on analgesics with serotoninergic properties such as meperidine, tramadol, methadone and propoxyphen, flexeril.\n4. Patients on fluoxetine or fluvoxamine.\n5. Patients taking amitriptyline \\> 50 mg/d, trazodone and sertraline \\> 100 mg/d, citalogram \\> 20 mg/d or paroxetine \\> 30 mg/d.\n6. Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc).\n7. Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc) over the last 30 days.\n8. History of renal disease.\n9. History of liver disease.\n10. Current pregnancy or lactation.\n11. Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.\n12. History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols.\n13. VC \\< 75% of predicted.\n14. Receipt of any investigational drug within the past 30 days.\n15. Women with the potential to become pregnant who are not practicing effective birth control.", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "rasagiline", "targeting_mechanism": "Monoamine oxidase B (MAO-B) inhibition with neuroprotective properties.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05470478", "title": "iBCI Optimization for Veterans With Paralysis", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "VA Office of Research and Development", "summary": "VA research has been advancing a high-performance brain-computer interface (BCI) to improve independence for Veterans and others living with tetraplegia or the inability to speak resulting from amyotrophic lateral sclerosis, spinal cord injury or stoke. In this project, the investigators enhance deep learning neural network decoders and multi-state gesture decoding for increased accuracy and reliability and deploy them on a battery-powered mobile BCI device for independent use of computers and touch-enabled mobile devices at home. The accuracy and usability of the mobile iBCI will be evaluated with participants already enrolled separately in the investigational clinical trial of the BrainGate neural interface.", "interventions": [{"type": "DEVICE", "name": "Mobile neural decoding platform (mobile iBCI)"}], "start_date": "2026-10-02", "url": "https://clinicaltrials.gov/study/NCT05470478", "target_entities": [], "locations": [{"facility": "Providence VA Medical Center, Providence, RI", "city": "Providence", "state": "Rhode Island", "country": "United States", "status": "", "lat": 41.82399, "lon": -71.41283}], "contact_phone": "(401) 273-7100", "contact_email": "Kate.Barnabe@va.gov", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Inclusion criteria are extensive and are determined by the associated BrainGate IDE(clinicaltrials.gov # NCT00912041)\n* Informally, participants will be tetraplegic or anarthric with little or no functional use of the arms and legs\n\nExclusion Criteria:\n\n* Exclusion criteria are extensive and are determined by the associated BrainGate IDE(clinicaltrials.gov # NCT00912041).", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00542412", "title": "CARE Canadian ALS Riluzole Evaluation", "phase": "PHASE4", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sanofi", "summary": "Evaluate the efficacy of riluzole 50-mg bid defined by comparing the percentage of riluzole-treated subjects who experienced death, permanently assisted ventilation (PAV) or tracheostomy, to a group of recent historical controls for the treatment of amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Riluzole"}], "start_date": "2001-01", "url": "https://clinicaltrials.gov/study/NCT00542412", "target_entities": ["glutamate"], "locations": [{"facility": "Sanofi-Aventis", "city": "Laval", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.56995, "lon": -73.692}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of ALS confirmed by the following definition:\n\n  (a)\"Probable\" or \"Definite\" Amyotrophic Lateral Sclerosis (ALS) according to the El Escorial criteria(b)\"Peripheral\" onset form (limb involvement) or a \"Bulbar\" form of ALS with a duration of five years, based on inquiry for the earliest symptoms of the disease\n* A subject who simultaneously presents with bulbar and peripheral signs at onset of ALS disease should be stratified to the bulbar onset group. The neurologic progression of such subjects matches that of the bulbar onset ALS subjects.\n* Pulmonary Function: forced vital capacity (FVC) must be 3 60% at study entry.\n* Females of childbearing potential must be documented to be using acceptable birth control methods such as an IUD or oral contraceptives.\n\nExclusion Criteria:\n\n* Previous treatment with riluzole\n* Tracheostomy, or expected to undergo a tracheostomy within two months after study inclusion\n* Signs of clinical dementia and/or major psychiatric disorders\n* Serious concomitant disease or handicap likely to interfere with the subject's assessments or impact on the subject's survival\n* A multiple conduction block has been shown on nerve conduction studies by electromyogram\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Riluzole", "targeting_mechanism": "Riluzole is an anti-glutamatergic agent that blocks glutamatergic neurotransmission in the CNS to exert neuroprotective effects by targeting excitotoxicity.", "targeting_mechanism_pmid": "31141951", "animal_results": "Riluzole does not improve lifespan or motor function in three ALS mouse models.", "animal_results_pmid": "29221425", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03537807", "title": "Expanded Access Protocol of BHV-0223 for Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Biohaven Pharmaceuticals, Inc.", "summary": "This is an open label expanded access protocol for the treatment of up to approximately 250 adult patients with amyotrophic lateral sclerosis (ALS) who have difficulty swallowing oral riluzole tablets and may be able to derive benefit from treatment with an alternative oral formulation of riluzole.", "interventions": [{"type": "DRUG", "name": "Riluzole"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT03537807", "target_entities": ["glutamate"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with diagnosed ALS of any type or duration\n* Current or previous treatment with oral riluzole tablets, or patients who have never taken riluzole oral tablets, or patients who have successfully completed a clinical trial with BHV-0223 and were not withdrawn prematurely due to adverse events\n* Swallowing difficulties, or patient or caregiver report choking one or more times per week, or investigator deems appropriate to treat with sublingual BHV-0223 because (s)he deems the patient cannot be satisfactorily treated with Rilutek\u00ae\n* Adequate hepatic function\n\nExclusion Criteria:\n\n* Patient with history of severe hypersensitivity reaction to riluzole oral tablets or BHV-0223\n* Patient is known to have any other acute or chronic liver disease", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Riluzole", "targeting_mechanism": "Riluzole is a benzothiazole derivative that blocks glutamatergic neurotransmission in the CNS to exert neuroprotective effects.", "targeting_mechanism_pmid": "32847483", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04140136", "title": "The Efficacy and Safety of Vitamin E Mixed Tocotrienols In Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Malaya", "summary": "There is currently no effective treatment in ALS. Oxidative stress, probably interacting with other neurodegenerative processes, is hypothesized to play a leading role in pathogenesis. These include mechanisms that promote glutamate excitotoxicity, mitochondrial dysfunction and axonal dysfunction.\n\nIn a transgenic mouse model of fALS that develops a disease with a clinical phenotype similar to ALS, dietary vitamin E supplementation delayed disease onset and slowed progression, although it did not prolong survival. When used as an experimental therapy in human trials, vitamin E did not affect survival significantly, but possibly slowed ALS progression. Two large, prospective epidemiologic studies suggest that longterm use of vitamin E supplements could be inversely associated with risk of ALS or ALS death. In another study, higher baseline serum \u03b1-tocopherol was associated with lower subsequent risk of ALS. A modest, non-significant protective effect from supplementation was seen in subjects with baseline serum \u03b1-tocopherol levels below median levels. In the current study, we aim to investigate the effects of tocotrienols in patients with ALS, particularly in delaying disease progression as well as assessing its safety profile in this group of patients.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Tocotrienols"}, {"type": "DIETARY_SUPPLEMENT", "name": "Placebo"}], "start_date": "2019-06-17", "url": "https://clinicaltrials.gov/study/NCT04140136", "target_entities": ["oxidative_stress", "glutamate_excitotoxicity", "mitochondrial_dysfunction"], "locations": [{"facility": "Clinical Investigation Centre (CIC)", "city": "Kuala Lumpur", "state": "", "country": "Malaysia", "status": "RECRUITING", "lat": 3.1412, "lon": 101.68653}], "contact_phone": "+603-79492622", "contact_email": "wangpl@ummc.edu.my", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients who have a decrease of 1 to 4 points on the ALSFRS-R score during the 12-weeks observation period prior to screening and enrollment\n* Patients of less than 2 years after the diagnosis of ALS.\n* Patients without respiratory symptoms (orthopnea, dyspnea)\n* Capable of giving signed informed consent\n\nExclusion Criteria:\n\n* Patients who have developed respiratory failure necessitating ventilation\n* Patients who have developed unsafe swallowing necessitating enteral feeding tube insertion\n* Patients with other neurodegenerative disease such as Parkinson's disease and significant mental health illness\n* Patients with certain concomitant diseases which may affect the assessment of safety/efficacy i.e. malignancy within the last 5 years, congestive heart disease, liver disease, kidney failure, bleeding disorders and other autoimmune diseases etc.\n* Pregnant, lactating, and probably pregnant patients.\n* Patients taking vitamin E tocopherol or tocotrienols supplements within 1 month from screening and randomisation.\n* Patients who have participated in other trials within 12 weeks before consent, or who are participating in other clinical trials at present.\n* Women of child bearing potential or nursing mother, unless they are willing to practice effective contraceptive measures.", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tocotrienols", "targeting_mechanism": "Tocotrienols are a form of vitamin E that act as antioxidants to reduce oxidative stress and its contribution to neurodegenerative pathogenesis.", "targeting_mechanism_pmid": "33274002", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00879593", "title": "Nocturnal PtcCO2 Monitoring in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Association Nationale pour les Traitements A Domicile, les Innovations et la Recherche", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting motoneurons, with a prevalence around 5/100.000. Respiratory muscle involvement is a major feature in ALS and remains the main prognostic factor. Timing and rate of progression of this respiratory muscle involvement is also highly variable among individuals.\n\nRespiratory manifestations justify a careful follow up including clinical evaluation, pulmonary function tests and blood gases. Prognostic value of respiratory muscle assessment has been clearly demonstrated in ALS, although several cut off values have been published. The clinical benefit of non invasive ventilation (NIV) is well established in ALS, but the optimal criteria for its initiation remain debated .\n\nThe 1999 consensus for NIV selected classical criteria to consider NIV in patients with respiratory symptoms suggesting hypoventilation: daytime hypercapnia (PaCO2 \\> 45 mmHg), nocturnal SaO2 \\< 89 % more than 5 consecutive minutes and for progressive neuromuscular disorders (NMD) (mainly ALS), a vital capacity (VC) \\< 50 % pred or a PImax \\< 60 cmH2O.\n\nBesides daytime clinical and PFT assessment, nocturnal evaluation is essential in ALS. The prevalence of sleep apnea ranges from 16 % to 76 %.\n\nTranscutaneous PCO2 (tcPCO2) is an attractive technique to evaluate non invasively nocturnal hypoventilation. The technique is well validated in different settings. Its use in neuromuscular disorders (NMD) is recent. In particular one study has demonstrated a high predictive value of tcPCO2 for the development of daytime hypoventilation within 1 year. To our knowledge, this technique has not been specifically assessed in ALS. There is a potential role for nocturnal PtcCO2 monitoring in the close follow up of ALS patients. Indeed, a close respiratory follow up of ALS patients is essential to determine the optimal timing of NIV, avoiding the occurence of unexpected acute respiratory failure.", "interventions": [{"type": "DEVICE", "name": "PtcCO2"}], "start_date": "2009-04", "url": "https://clinicaltrials.gov/study/NCT00879593", "target_entities": [], "locations": [{"facility": "PEREZ", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "P\u00f4le des maladies respiratoires et service EFR- Centre hospitalier Regional Universitaire", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Amyotrophic lateral sclerosis :definite, probable or probable with EMG (Airlie House Criteria, 1998).\n* Forced vital capacity \\>70% pred.\n* Daytime PaCO2 \\<43 mmHg.\n* Venous HCO3- \\<28 mmol/L\n\nExclusion Criteria:\n\n* Patients unable to perform pulmonary function tests or nocturnal recordings.\n* Coexisting significant lung disease: moderate to severe asthma or COPD\n* Current NIV, CPAP or oxygen therapy.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02655614", "title": "A Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Participants With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Genentech, Inc.", "summary": "This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134. It will include three components: a Single-Ascending-Dose (SAD) stage, a Multiple-Ascending-Dose (MAD) stage, and an Open-Label Safety Expansion (OSE) stage.", "interventions": [{"type": "DRUG", "name": "GDC-0134"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Rabeprazole"}, {"type": "DRUG", "name": "Midazolam"}, {"type": "DRUG", "name": "Caffeine"}], "start_date": "2016-05-31", "url": "https://clinicaltrials.gov/study/NCT02655614", "target_entities": ["TARDBP"], "locations": [{"facility": "Forbes Norris Mda/als Ctr; Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic Hospital - Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami Miller School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "The Emory ALS Clinic", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Johns Hopkins University School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Wake Research Associates", "city": "Raleigh", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.7721, "lon": -78.63861}, {"facility": "New Orleans Center for Clinical Research", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}, {"facility": "MUCH - Montreal Neurological Institute & Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female participants with a diagnosis of possible, laboratory-supported probable, probable, or definite ALS according to modified El Escorial criteria\n* Upright forced vital capacity of at least 50 percent (%)\n* Ability to fast from food for 8 hours prior to dosing and 2 hours after dosing\n\nExclusion Criteria:\n\n* Currently taking riluzole unless on a stable dose for the 3 months prior to Day -1 and without current liver enzyme or liver function abnormalities\n* Currently taking edaravone unless after completion of at least the second 14-day drug-treatment period, as long as Day 1 occurs during a drug-free period at least 24 hours after the last edaravone dose and at least 5 days prior to the first dose of the next cycle\n* Positive for hepatitis C antibody, hepatitis B surface antigen, or human immunodeficiency virus (HIV) antibody\n* Clinically significant thrombocytopenia\n* Currently taking nutritional/herbal supplements, except for over-the-counter vitamins that are within Recommended Dietary Allowance (RDA), unless discontinued at least 7 days prior to Day -1, except upon approval of both the investigator and Sponsor\n* For participants participating in a designated drug-drug interaction (DDI) cohort in the MAD stage of the study, who require midazolam/caffeine administration: known allergy, religious prohibition, or other condition limiting midazolam or caffeine administration", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "GDC-0134", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03474263", "title": "IC14 for Rapidly Progressive Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Implicit Bioscience", "summary": "Patients with rapidly progressive ALS will be assigned to IC14 intravenously on Day 1-4. This 4-day course will be repeated on Days 8-11. Patients will all undergo MR-PET scans at two time points: before treatment onset and after the last treatment cycle. This scan will measure areas of ALS disease activity and assess response to IC14 treatment. MR-PET scans will be compared to historical controls.", "interventions": [{"type": "BIOLOGICAL", "name": "Biologic: IC14 (monoclonal antibody against human CD14)"}], "start_date": "2019-09-01", "url": "https://clinicaltrials.gov/study/NCT03474263", "target_entities": ["CD14", "neuroinflammation"], "locations": [{"facility": "Royal Brisbane & Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Capable of providing informed consent and informed consent form signed prior to initiation of any study-specific procedures.\n2. Familial or sporadic ALS defined as clinically possible, probable, or definite by El Escorial Criteria.\n3. Rapidly progressive ALS defined by the Revised ALS Functional Rating Scale (ALSFRS-R) slope \u22651 (48 minus ALSFRS-R score at screening / disease duration in months \u2265 1).\n4. Upper Motor Neuron Burden Score of \u2265 25 (out of 45) at screening\n5. First symptoms of ALS within 3 years of the screening visit\n6. Age between 18 and 80 years at the time of the screening visit.\n7. Not taking riluzole or edaravone or on a stable dose of riluzole or edaravone for at least 3 months prior to screening visit.\n8. Adequate bone marrow reserve, renal and liver function:\n\n   1. absolute neutrophil count \u2265 1500/\u00b5L\n   2. lymphocyte count \\< 6000/\u00b5L\n   3. platelet count \u2265 150,000/\u00b5L\n   4. hemoglobin \u2265 11 g/dL\n   5. creatinine clearance \u2265 60 mL/min\n   6. alanine transaminase (ALT) and/or aspartate transaminase (AST) \u2264 3x upper limits of normal (ULN)\n   7. total bilirubin \u2264 1.5x ULN\n   8. serum albumin \u2265 2.8 g/dL\n9. Females of childbearing potential should be using and committed to continue using one of the following acceptable birth control methods:\n\n   1. Sexual abstinence (inactivity) for 1 month prior to screening through study completion; or\n   2. Intrauterine device (IUD) in place for at least 3 months prior to study through study completion; or\n   3. Stable hormonal contraception for at least 3 months prior to study through study completion; or\n   4. Surgical sterilization (vasectomy) of male partner at least 6 months prior to study.\n10. To be considered of non-childbearing potential, females should be surgically sterilized (bilateral tubal ligation, hysterectomy, or bilateral oophorectomy at least 2 months prior to study) or be post-menopausal and at least 3 years since last menses.\n11. Males with female partners of childbearing potential must use contraception through study completion.\n12. Ability to safely lie flat for 90 min for magnetic resonance-positron emission tomography (MR-PET) procedures in the opinion of the Investigator.\n13. Patients must also have a genotype associated with a high or mixed affinity translocator protein (TSPO) (Ala/Ala or Ala/Thr) and ability to safely undergo MR-PET scans based on the opinion of the Investigator.\n\nExclusion Criteria:\n\n1. Dependence on invasive or non-invasive ventilation, defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use; presence of tracheostomy at screening; or presence of diaphragm pacing system at screening.\n2. Exposure to any experimental treatment for ALS within the last 30 days or five half-lives, whichever is longer.\n3. Treatment within 12 months with immunomodulator or immunosuppressant agent (including but not limited to cyclophosphamide, cyclosporine, interferon-\u03b1, interferon-\u03b2-1a, rituximab, alemtuzumab, azathioprine, etanercept, infliximab, adalimumab, certolizumab, golimumab, anakinra, rilonacept, secukinumab, tocilizumab, mycophenolate mofetil, methotrexate, cell-depleting agents, total lymphoid irradiation, dimethyl fumarate). Treatment with intravenous immunoglobulin (IVIG) within 2 months. Non-steroidal anti-inflammatory drugs (NSAIDs) are acceptable.\n4. Exposure at any time to any cell or gene therapies under investigation for the treatment of ALS.\n5. Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other opportunistic infections; or major episode of infection requiring hospitalization or treatment with intravenous (IV) antibiotics within 4 weeks.\n6. Live-attenuated vaccines within 30 days before dosing. Subjects must agree to forego live-attenuated vaccines throughout the study, including 60 days after the last dose of study drug.\n7. History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies.\n8. History of one or more of the following: cardiac insufficiency (New York Heart Association \\[NYHA\\] III/IV), uncontrolled cardiac arrhythmias, unstable ischemic heart disease, or uncontrolled hypertension (systolic blood pressure \\> 170 mmHg or diastolic blood pressure \\> 110 mmHg).\n9. History of myocardial infarction, or cerebrovascular accident.\n10. Unstable pulmonary, renal, hepatic, endocrine or hematologic disease.\n11. Autoimmune disease, mixed connective tissue disease, scleroderma, polymyositis, or significant systemic involvement secondary to rheumatoid arthritis.\n12. Evidence of active malignant disease, malignancies diagnosed within the previous 5 years, or breast cancer diagnosed within the previous 5 years (except skin cancers other than melanoma).\n13. History of human immunodeficiency virus infection or other immunodeficiency illness.\n14. Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days.\n15. History of drug abuse (not including marijuana use) or alcoholism within the past 12 months.\n16. Significant neuromuscular disease other than ALS.\n17. Other ongoing disease that may cause neuropathy, such as toxin exposure, dietary deficiency, uncontrolled diabetes, hyperthyroidism, cancer, systemic lupus erythematosus or other connective diseases, infection with HIV, hepatitis B virus (HBV), or hepatitis C (HCV), Lyme disease, multiple myeloma, Waldenstr\u00f6m's macroglobulinemia, amyloid, and hereditary neuropathy.\n18. Pregnancy or breastfeeding.\n19. Deprivation of freedom by administrative or court order.\n20. Any contraindication to undergo magnetic resonance imaging (MRI) studies such as history of a cardiac pacemaker or pacemaker wires; metallic particles in the body; vascular clips in the head; prosthetic heart valves; or severe claustrophobia.\n21. Unwilling or unable to discontinue benzodiazepine usage \\[other than lorazepam (Ativan\u00ae), clonazepam (Klonopin\u00ae), or zolpidem (Ambien\u00ae)\\] for one day prior to and during scanning.\n22. Research imaging-related radiation exposure exceeds current institutional Radiology Department guidelines", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "IC14", "targeting_mechanism": "Monoclonal antibody against human CD14 to modulate innate immune activation and neuroinflammation in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02714036", "title": "A Biomarker Study to Evaluate MN-166 in Subjects With Amyotrophic Literal Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "MediciNova", "summary": "This is a multi-center, open-label study of MN-166 (ibudilast) in subjects with ALS. To be eligible subjects must meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS. Safety, tolerability, blood, neuro-imaging biomarkers, and clinical outcomes will be collected on all subjects. Subjects will receive study drug for 36 weeks.\n\nThe study will consist of a Screening Phase (up to 6 weeks), an Open-Label Treatment Phase (36 weeks) and an Off-Treatment Follow-up Phase (4 Weeks).\n\nNumber of Subjects (Planned):\n\nApproximately 45 subjects are planned to be screened with the goal of enrolling 35 subjects.", "interventions": [{"type": "DRUG", "name": "ibudilast"}, {"type": "DRUG", "name": "Ibudilast"}], "start_date": "2016-05-06", "url": "https://clinicaltrials.gov/study/NCT02714036", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "South Shore Neurologic Associates, P.C.", "city": "Patchogue", "state": "New York", "country": "United States", "status": "", "lat": 40.76565, "lon": -73.01511}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Subjects must be diagnosed as having possible, probable, probable-laboratory supported, or definite ALS, either sporadic or familial according to modified El Escorial criteria.\n2. Age 18 or above, able to provide informed consent, and safely comply with study procedures.\n3. Vital capacity (VC) of at least 50% predicted value for gender, height and age at screening visit, or in the opinion of the study physician, able to safely tolerate study procedures. (Not applicable to flexible arm)\n4. Subject must be able to swallow oral medication at the Baseline Visit and expected to be able to swallow the capsules throughout the course of the study.\n5. Subject must not have taken riluzole for at least 30 days or be on a stable dose of riluzole for at least 30 days, prior to screening (riluzole-na\u00efve participants are permitted in the study). (Not applicable to flexible arm)\n6. Women must not be able to become pregnant (e.g. post-menopausal, surgically sterile, or using adequate birth control) for the duration of the study and 3 months after study completion.\n7. Males should practice contraception for the duration of the study and 3 months after completion.\n8. Ability to safely lie flat for 90 min for PET procedures in the opinion of the study physician. (Not applicable to flexible arm)\n9. High or mixed affinity to bind TSPO protein (Ala/Ala or Ala/Thr) (not applicable to flexible arm).\n10. Upper motor Neuron Burden (UMNB) Score \u226525 (out of 45) at screening visit. (Not applicable to flexible arm)\n\nExclusion Criteria:\n\n1. Abnormal liver function defined as AST and/or ALT \\> 3 times the upper limit of the normal.\n2. Renal insufficiency as defined by a serum creatinine \\> 1.5 times the upper limit of normal.\n3. The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent, according to PI judgment.\n4. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if they were to participate in the study.\n5. History of HIV, clinically significant chronic hepatitis, or other active infection.\n6. Active inflammatory condition of autoimmune disorder (Not applicable to flexible arm)\n7. Females must not be lactating or pregnant.\n8. Active participation in another ALS clinical trial or exposure to an off-label ALS experimental treatment within 30 days of the Baseline Visit (Not applicable to flexible arm)\n9. Exposure to immunomodulatory medications within 30 days of the Baseline Visit. (Not applicable to flexible arm)\n10. Any contraindication to undergo MRI studies such as\n\n    * History of a cardiac pacemaker or pacemaker wires\n    * Metallic particles in the body\n    * Vascular clips in the head\n    * Prosthetic heart valves\n    * Claustrophobia (Not applicable to flexible arm)\n11. Radiation exposure that exceeds the site's current guidelines (Not applicable to flexible arm)\n12. EKG finding of QTc prolongation \\> 450 msec for males and \\> 470 msec for females at screening or baseline.\n13. Not on any prohibitive medication or known QT prolonging medication:", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ibudilast", "targeting_mechanism": "Inhibitor of neuroinflammation and glial cell activation to reduce CNS inflammation in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06704347", "title": "Safety Study of XT-150 in Participants With ALS", "phase": "PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Xalud Therapeutics, Inc.", "summary": "This is a Phase 1, open-label, multi-center safety study of XT-150 in adult participants with Amyotrophic Lateral Sclerosis (ALS).\n\nParticipants providing informed consent and meeting all study eligibility criteria will be enrolled in the study and will receive a single injection of XT-150 at the Baseline visit. Follow-up visits will occur over 180 days (6 months) after the injection.\n\n8 participants (4 participants per dose level) will be enrolled sequentially in up to 2 ascending, single dose cohorts: Cohort 1: 1.5 mg XT-150 Cohort 2: 4.5 mg XT-150", "interventions": [{"type": "BIOLOGICAL", "name": "XT-150"}], "start_date": "2027-03", "url": "https://clinicaltrials.gov/study/NCT06704347", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Barrow Neurological Institute (St. Joseph's)", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Henry Ford Health", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}], "contact_phone": "212-301-6673", "contact_email": "medical.information@xaludthera.com", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Adults between 18 and 80 years of age\n* Male or female, if of childbearing potential or sexually active, strict contraception required\n* Have ALS diagnosed by a doctor (specifically, sporadic or familial ALS diagnosed as clinically probable, lab-supported probable or definite ALS defined by the El Escorial criteria)\n* Have had symptoms of ALS (muscle weakness) within 36 months of starting this study\n* Have the ability to slowly exhale a volume of air at least 60% of what is expected for the participant's sex, height and age\n* Have not received treatment for ALS or are currently on a stable dose of an approved treatment for ALS. Patients currently receiving Tofersen are not eligible.\n* Able to receive the study injection intrathecally, determined by the study doctor\n* Able to undergo the study procedures and adhere to the study visit schedule at the time of study entry, with an estimated life expectancy of 6 months or greater\n\nKey Exclusion Criteria:\n\n* Have an implanted shunt to drain cerebrospinal fluid (CSF) or an implanted CNS catheter\n* Have an implanted of diaphragm pacing system\n* Tracheostomy\n* History or current diagnosis of cardiac conditions or ECG abnormalities indicating significant risk of safety for participants in the study\n* History or current diagnosis of respiratory conditions such as COPD\n* History or current diagnosis of cancer, chemical meningitis, HIV, Hep B, Hep C, uncontrolled diabetes\n* Presence of an autoimmune condition (for example, rheumatoid arthritis or lupus) requiring treatment or immunodeficiency\n* Clinical or laboratory evidence of hepatic or renal disease/injury.\n* Taking any prohibited medications\n* Women who are pregnant or nursing\n* Use of any investigational drugs or devices within 30 days or 5 half-lives of the study agent (whichever is longer). Exception: Observational, non-interventional clinical studies are allowed in the opinion of the study doctor.\n* Any other condition that the study doctor feels could compromise the participant's safety, ability to communicate with the study staff, or the quality of the data", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "XT-150", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02623699", "title": "An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogen", "summary": "The primary objectives of Parts A and B of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending doses of tofersen in adults with ALS and a documented superoxide dismutase 1 (SOD1) mutation. The primary objective of Part C of this study is to evaluate the clinical efficacy of tofersen administered to adults with ALS and a confirmed SOD1 mutation.\n\nThe secondary objective of Parts A and B of this study is to evaluate the effects of tofersen on levels of total SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, pharmacodynamic (PD), and biomarker effects of tofersen.", "interventions": [{"type": "DRUG", "name": "Tofersen"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2016-01-20", "url": "https://clinicaltrials.gov/study/NCT02623699", "target_entities": ["SOD1"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California San Diego Medical Center", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic in Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Bioclinica Research", "city": "Orlando", "state": "Florida", "country": "United States", "status": "", "lat": 28.53834, "lon": -81.37924}, {"facility": "Emory University Hospital", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern University Feinberg School of Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Henry Ford Hospital", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Mayo Clinic - Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates, P.C.", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "The Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "New Orleans Center for Clinical Research/Volunteer Research Group, an AMR Company", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Westmead Hospital", "city": "Westmead", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.80383, "lon": 150.98768}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "University of Calgary - Health Sciences Centre", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Research Site", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Bispebjerg Hospital", "city": "Copenhagen", "state": "", "country": "Denmark", "status": "", "lat": 55.67594, "lon": 12.56553}, {"facility": "Hopital Pitie Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "University of Ulm", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "ALS Center - Dept. of Neuroscience \"Rita Levi Montalcini\", University of Turin", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "The University of Tokyo Hospital", "city": "Bunky\u014d City", "state": "", "country": "Japan", "status": "", "lat": 35.5331, "lon": 139.4217}, {"facility": "Research Site", "city": "Fukuoka", "state": "", "country": "Japan", "status": "", "lat": 33.6, "lon": 130.41667}, {"facility": "Research Site", "city": "Kagoshima", "state": "", "country": "Japan", "status": "", "lat": 31.56667, "lon": 130.55}, {"facility": "Research Site", "city": "Shinjuku-ku", "state": "", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Research Site", "city": "Suita-Shi", "state": "", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Research Site", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Research Site", "city": "Yangsan", "state": "Gyeongsangnam-do", "country": "South Korea", "status": "", "lat": 35.34199, "lon": 129.03358}, {"facility": "Research Site", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Research Site", "city": "London", "state": "Greater London", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Research Site", "city": "Sheffield", "state": "South Yorkshire", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria: Part A and B\n\n* Weakness attributable to ALS and documented SOD1 mutation at Screening Visit 2.\n* A forced vital capacity (FVC) \u226550% of predicted value as adjusted for sex, age, and height (from the sitting position). Participants with stable FVC \\<50% but \u226545%, whose FVC has not declined by more than 5% in the last 6 months may be considered for inclusion, at the discretion of the Investigator.\n* If taking riluzole, participant must be on a stable dose for \u226530 days prior to Day 1 and expected to remain at that dose until the final study visit.\n* Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.\n\nKey Exclusion Criteria: Part A and B\n\n* History of or positive test result for human immunodeficiency virus.\n* History of, or positive test result at Screening, for hepatitis C virus antibody.\n* Current hepatitis B infection (defined as positive for hepatitis B surface antigen \\[HBsAg\\] and/or hepatitis B core antibody \\[HBcAb\\]). Participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive hepatitis B surface antibody immunoglobulin G, and positive HBcAb) or vaccination (defined as positive anti-HBs) are eligible to participate in the study.\n* Treatment with another investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering ribonucleic acid, stem cell therapy, or gene therapy is allowed.\n* Current enrollment in any other interventional study.\n* Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis (N4-methylthiosemicarbazone)) or pyrimethamine.\n* Current or anticipated need, in the opinion of the Investigator, of a diaphragm pacing system (DPS) during the study period.\n\nKey Inclusion Criteria: Part C\n\n* Weakness attributable to ALS and confirmed SOD1 mutation at Screening Visit.\n* If taking riluzole, participant must be on a stable dose for \u226530 days prior to Day 1 and expected to remain at that dose until the final study visit.\n* If taking edaravone, participant must have initiated edaravone \u226560 days (2 treatment cycles) prior to Day 1 and expected to remain at that dose until the final study visit, unless the Investigator determines that edaravone should be discontinued for medical reasons, in which case it may not be restarted during the study. Edaravone may not be administered on dosing days of this study.\n* Medically able to undergo the study procedures and to adhere to the visit schedule at the time of study entry, as determined by the Investigator.\n\nKey Exclusion Criteria: Part C\n\n* History of or positive test result for human immunodeficiency virus.\n* Current hepatitis C infection (defined as positive hepatitis C virus \\[HCV\\] antibody and detectable HCV ribonucleic acid \\[RNA\\]). Participants with positive HCV antibody and undetectable HCV RNA are eligible to participate in the study (United States Centers for Disease Control and Prevention).\n* Current hepatitis B infection (defined as positive for HBsAg and/or anti-HBc). participants with immunity to hepatitis B from previous natural infection (defined as negative HBsAg, positive anti-HBc, and positive anti-HBs) or vaccination (defined as negative HBsAg, negative anti-HBc, and positive anti-HBs) are eligible to participate in the study.\n* Treatment with another investigational drug (including investigational drugs for ALS through compassionate use programs), biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Specifically, no prior treatment with small interfering RNA, stem cell therapy, or gene therapy is allowed.\n* Current enrollment in any other interventional study.\n* Current or recent (within 1 month) use, or anticipated need, in the opinion of the Investigator, of copper (II) (diacetyl-bis(N4-methylthiosemicarbazone)) or pyrimethamine.\n* Current or anticipated need, in the opinion of the Investigator, of a DPS during the study period.\n\nNOTE: Other protocol defined Inclusion/Exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tofersen", "targeting_mechanism": "Antisense oligonucleotide that targets and reduces SOD1 mRNA expression in patients with SOD1 mutations.", "targeting_mechanism_pmid": "37975798", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05328492", "title": "Volume Mode Non-invasive Ventilation in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospitals Coventry and Warwickshire NHS Trust", "summary": "The purpose of this study is to assess the efficacy of using intelligent volume assured pressure support (iVAPS-AE) versus spontaneous timed (ST) modes of non-invasive ventilation (NIV) in patients diagnosed with amyotrophic lateral sclerosis (ALS).\n\nThe investigators believe that the use of iVAPS-AE mode NIV over a 90 day period will produce NIV compliance data and health-related quality of life (HRQOL) scores that are equivalent or no worse compared to ST mode NIV.", "interventions": [{"type": "DEVICE", "name": "iVAPS-AE"}, {"type": "DEVICE", "name": "ST-mode"}], "start_date": "2022-03-15", "url": "https://clinicaltrials.gov/study/NCT05328492", "target_entities": [], "locations": [{"facility": "University Hospital Coventry and Warwickshire NHS Trust", "city": "Coventry", "state": "West Midlands", "country": "United Kingdom", "status": "RECRUITING", "lat": 52.40656, "lon": -1.51217}], "contact_phone": "02476966734", "contact_email": "edward.parkes@uhcw.nhs.uk", "eligibility": {"criteria": "Inclusion Criteria:\n\nPatients with respiratory failure secondary to ALS (diagnosed either at an MND MDT or specialist neurology clinic) according to criteria set out in the NICE guideline (NG42) (2016); Motor neurone disease: assessment and management.\n\n* Patients able to provide informed consent to take part in the research study.\n* Patients not contraindicated to commence NIV in accordance with local protocol.\n* Patients not currently enrolled in another research study that could alter disease progression.\n\nExclusion Criteria:\n\n* Acutely unwell or medically complicated patients as assessed by lead investigator. These patients will be urgently reviewed by a dedicated Consultant Physician. The Principal Investigator will be immediately informed.\n* An inability to provide informed consent.\n* An inability to use NIV.\n* Patients whom are contraindicated to commence NIV in accordance with local protocol.", "sex": "ALL", "min_age": "18 Years", "max_age": "100 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07082192", "title": "A Study to Evaluate the Efficacy and Safety of Different Doses of CB03-154 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shanghai Zhimeng Biopharma, Inc.", "summary": "The goal of this clinical trial is to learn if drug CB03-154 works to treat ALS in adults. It will also learn about the safety of drug CB03-154.\n\nThe main questions it aims to answer are:\n\n* Does drug CB03-154 have an effect on delaying disease progression, improving function, and prolonging survival in adult ALS patients?\n* What medical problems do patients have when taking drug CB03-154? Researchers will compare drug CB03-154 to a placebo (a look-alike substance that contains no drug) to see if drug CB03-154 works to treat ALS.\n\nParticipants (adult ALS patients) will:\n\n* Take drug CB03-154 or a placebo every day for 39 weeks (an additional 39 weeks would be required if entering the open-label extension phase).\n* Visit the clinic approximately every 2-3 months for checkups and tests, and there is also telephone follow-up in between.\n* Keep a diary of daily medication (CB03-154 or other concomitant medications), and if there are any unplanned medications, the reason (disease or symptoms) also need be recorded.", "interventions": [{"type": "DRUG", "name": "Test drug CB03-154 5mg group"}, {"type": "DRUG", "name": "Test drug CB03-154 10mg group"}, {"type": "DRUG", "name": "Test drug CB03-154 15mg group"}, {"type": "DRUG", "name": "Placebo Group"}], "start_date": "2025-09-29", "url": "https://clinicaltrials.gov/study/NCT07082192", "target_entities": ["Tau"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Agree to follow the treatment plan and trial procedures of this study, and sign the written informed consent form.\n2. Male or female, aged 18 to 65 years, inclusive.\n3. The weight of subjects during the screening period must not be less than 45 kg, and the BMI must not be less than 18 kg/m2.\n4. Diagnosed according to the Revised EI Escorial diagnostic criteria set by the World Federation of Neurology: definite ALS, probable ALS, lab supported probable ALS, or possible ALS.\n5. Less than or equal to 24 months since ALS symptom onset at Screening, and estimated survival time of \u22651 year as per the Investigator's judgement.\n6. Forced vital capacity (FVC) \\>80% of predicted value for gender, height, and age at Screening.\n7. Able to swallow oral medication (tablets) at Screening as judged by the investigator.\n8. For participants taking riluzole: Dose must be stable for at least 4 weeks prior to Screening and participant must be expected to remain on treatment for the duration of the trial. Participants receiving riluzole should maintain the same dose throughout the study.\n9. Currently not receiving edaravone treatment or is in the schedule of edaravone treatment cycle. Participants receiving edaravone treatment must complete at least one cycle of treatment before the screening visit and continue stable dose edaravone treatment throughout the study.\n10. Women of childbearing potential (WOCBP) must use an approved highly effective contraception for at least one menstrual cycle before the first dose of the investigational product and for at least 3 months after the last dose of the investigational product. Similarly, men must start using effective contraception before the first dose of the investigational product and continue for at least 3 months after the last dose of the investigational product, with no plans for procreation. Male participants cannot donate sperm for at least 3 months during the trial and after the last dose of the investigational product and female participants cannot donate or freeze eggs during the trial and for at least 3 months after the last dose of the investigational product.\n\nExclusion Criteria:\n\n1. Significant cognitive impairment, mental disorders (such as schizophrenia, bipolar disorder), other neurodegenerative diseases (such as Parkinson's disease, Alzheimer's disease, frontotemporal dementia, etc.), substance abuse or other causes leading to neuromuscular weakness (such as myasthenia gravis), or other conditions that may interfere with the participants' participation in clinical study or, in the investigator's judgment, may interfere with outcome assessment or affect the completion of the trial.\n2. Serum aspartate aminotransferase (AST), alanine aminotransferase (ALT), or bilirubin\\>2.0\u00d7 upper limit of normal at screening.\n3. Estimated glomerular filtration rate \\<59 mL/min/1.73m2 at Screening (using the Cockcroft-Gault formula to calculate eGFR: eGFR (mL/min/1.73m2) = Ccr \u00d7 0.84 \u00d7 1.73 / BSA; Ccr (mL/min) = \\[(140 - age) \u00d7 weight (kg)\\] / \\[72 \u00d7 Scr (mg/dL)\\], females multiply the result by 0.85, and Scr is the serum creatinine; BSA (m2) = 0.007184 \u00d7 weight (kg)\\^0.425 \u00d7 height (cm)\\^0.725).\n4. D-dimer\\>2.0\u00d7 upper limit of normal or venous ultrasound of the lower limbs shows deep vein thrombosis at screening or a history of venous thrombosis.\n5. Assistance with ventilation support or tracheostomy or tube feeding status or having a central venous catheter is required at screening.\n6. A history of unexplained syncope, family history of syncope, or a history of convulsions or epilepsy (excluding the history of febrile seizures in childhood), or unstable medical condition, serious heart issues (e.g., corrected QTcF interval: males \\>450ms, females \\>470ms, torsades de pointes, NYHA class 3 or higher heart failure, myocardial infarction or unstable angina within 6 months prior to screening), lung, liver, kidney diseases, or tumors, or other clinically significant diseases or medical history (excluding ALS), participation in this study could threaten the safety of the participants.\n7. Current clinically significant urinary retention, or current use of medications for urinary retention, or clinically significant abnormalities in residual urinary bladder ultrasound at screening.\n8. Clinically significant ophthalmological abnormalities found in visual acuity examination (best corrected visual acuity), fundus photography, OCT examination, etc. during screening period, or clinically significant fundus lesions or retinopathy known or recorded in medical history.\n9. Hepatitis B surface antigen (HBsAg) positive, or hepatitis C antibody (HCVAb) positive and hepatitis C virus ribonucleic acid (HCV-RNA) test higher than the lower limit of detection, or human immunodeficiency virus antibody (HIVAb) positive, or Treponema pallidum (TP) antibody positive (also judged as active infection by the investigator) at screening.\n10. Severe infections (e.g., infectious pneumonia, sepsis) within 4 weeks prior to screening, or infections requiring hospitalization or intravenous administration of antibiotics, antiviral drugs, or antifungal medications, or chronic active bacterial infections (e.g., tuberculosis) deemed by the investigator to be unsuitable for participation in this trial.\n11. Received Tofersen treatment before screening.\n12. Significant risk of suicidality based on the Investigator's opinion or with an answer of \"yes\" on either item 4 or item 5 of the Suicidal Ideation Section of the Columbia Suicide Severity Rating Scale (C-SSRS) or any answer of \"yes\" within the Suicidal Behavior Section of the C SSRS within the 6 months before Screening.\n13. Exposure to any other investigational medicinal product or product within 4 weeks or 5 half-lives of the investigational product (whichever is longer) prior to Screening; or exposure to monoclonal antibody drug within 6 months prior to Screening (or if the washout period at the time of screening has not reached more than 3 months), or had received cell therapy or gene therapy at any time in the past.\n14. Treatment with CYP3A4 strong inducers or strong inhibitors prior to screening, and washout time of more than 5 half-lives of the drug was not reached before enrollment\u3002\n15. Pregnant, currently breastfeeding women or women with a positive pregnancy test at screening.\n16. Known history of allergy to any component of the investigational medicinal product.\n17. History of drug abuse within 12 months of Screening.\n18. Anything else that, in the opinion of the Investigator, would place the participant at increased risk or preclude the participant's full compliance with or completion of the trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CB03-154", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00409721", "title": "The Effect of Memantine on Functional Outcomes and Motor Neuron Degeneration in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Alberta", "summary": "The purpose of the study is to investigate the effects of memantine in ALS patients using functional outcome measures.", "interventions": [{"type": "DRUG", "name": "Memantine"}], "start_date": "2007-03", "url": "https://clinicaltrials.gov/study/NCT00409721", "target_entities": ["glutamate_excitotoxicity"], "locations": [{"facility": "Calgary ALS Neuromuscular Clinic", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta ALS Clinic", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* El Escorial Classification of laboratory supported probable, probable,or definite ALS\n* Age 18 - 80 years,\n* ALS symptoms for no more than 3 years,\n* FVC greater than or equal to 60% predicted,\n* Riluzole na\u00efve or have been on a stable dose of Riluzole for at least 2 months,\n* Patients must have the ability to attend monthly study visits in Edmonton or Calgary, Alberta\n\nExclusion Criteria:\n\n* Presence of significant sensory abnormalities, dementia, other neurologic diseases, uncompensated medical illness and psychiatric illness\n* Female patients who are breastfeeding\n* Use of concurrent investigational drugs,\n* Patient unlikely to comply with study requirements\n* Poor adherence to study protocol during run-in phase", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Memantine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07161999", "title": "Study of COYA 302 for the Treatment of ALS", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Coya Therapeutics", "summary": "The ALSTARS trial will be conducted across 20-25 sites in the US and Canada, and will evaluate the safety and efficacy of an investigational treatment called COYA 302 for adults with Amyotrophic Lateral Sclerosis (ALS).\n\nCOYA 302 is an investigational and proprietary biologic combination therapy with a dual immunomodulatory mechanism of action intended to enhance the anti-inflammatory function of regulatory T cells (Tregs) and suppress the inflammation produced by activated monocytes and macrophages. It is comprised of low dose interleukin-2 (LD IL-2) and DRL\\_AB (a biosimilar candidate for abatacept). Participants will be randomly assigned to receive one of 2 regimens of COYA 302 or placebo (an inactive substance) in a 1:1:1 ratio for 24 weeks in the double-blind (DB) period. Those who complete this part of the study will be eligible to receive one of the two regimens of COYA 302 for an additional 24 weeks in a blinded active extension phase (EXT).\n\nThe study will assess changes in disease progression using established ALS clinical outcome measures, including the ALS Functional Rating Scale-Revised (ALSFRS-R), neurofilament (NfL), maximal inspiratory pressure (MIP), slow vital capacity (SVC), and neurological assessments. Additional objectives include evaluation of biomarkers and safety through routine clinical assessments and adverse event monitoring.", "interventions": [{"type": "DRUG", "name": "COYA 302"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2025-10-01", "url": "https://clinicaltrials.gov/study/NCT07161999", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "RECRUITING", "lat": 33.44838, "lon": -112.07404}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.77493, "lon": -122.41942}, {"facility": "Nova Southeastern University", "city": "Davie", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.06287, "lon": -80.2331}, {"facility": "University of Florida Clinical and Translational Research Center", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 29.65163, "lon": -82.32483}, {"facility": "University Of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "RECRUITING", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "RECRUITING", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "RECRUITING", "lat": 42.27756, "lon": -83.74088}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "RECRUITING", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates, P.C. Somnos Clinical Research", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "RECRUITING", "lat": 40.8, "lon": -96.66696}, {"facility": "Columbia University Medical Center ALS Center", "city": "New York", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 40.71427, "lon": -74.00597}, {"facility": "Thomas Jefferson University-Weinberg ALS Center", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 39.95238, "lon": -75.16362}, {"facility": "Temple Neurology", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 39.95238, "lon": -75.16362}, {"facility": "Austin Neuromuscular Center; National Neuromuscular Research Institute, PLLC", "city": "Austin", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 30.26715, "lon": -97.74306}, {"facility": "Texas Neurology, PA", "city": "Dallas", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 32.78306, "lon": -96.80667}, {"facility": "Houston Methodist Stanley H. Appel Department of Neurology", "city": "Houston", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 29.76328, "lon": -95.36327}, {"facility": "The University of Texas Health Science Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of British Columbia", "city": "Vancouver", "state": "British Columbia", "country": "Canada", "status": "RECRUITING", "lat": 49.24966, "lon": -123.11934}, {"facility": "London Health Sciences Center", "city": "London", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 42.98339, "lon": -81.23304}, {"facility": "University of Toronto/Sunnybrook Health Sciences Center", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}, {"facility": "Hopital Neurologique de Montreal", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "800-587-8170", "contact_email": "clinicaltrials@coyatherapeutics.com", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n1. Sporadic or familial ALS, diagnosed as clinically probable, lab-supported probable, or definite ALS according to the revised El Escorial criteria\n2. Male or female participants aged 18 to 80\n3. Time since onset of ALS symptoms \u226428 months from Screening.\n4. ALSFRS-R total score \u226535 at Screening\n5. Rate of progression at baseline between -0.5 and -1.5 points per month on ALSFRS-R total score.\n6. SVC \u226560% of predicted capacity.\n7. Participants receiving riluzole must be on a stable dose for at least 30 days prior to Screening, with intent to stay on stable dosage throughout the study. If not on a stable dose of riluzole for at least 30 days prior to Screening, willing to refrain from initiation of the agent for the duration of the trial.\n8. Participants receiving edaravone (intravenous \\[IV\\] or oral, RADICAVA\u00ae) must have completed at least one treatment cycle prior to Screening, with intent to remain on stable dosage throughout the study. If participant has not completed at least one treatment cycle of edaravone at the time of Screening, willing to refrain from initiation of the agent for the duration of the trial.\n9. Participants receiving tofersen (QALSODY\u00ae) must have completed 90 days of treatment prior to Screening, with intent to remain on stable dosage throughout the study. If participant has not completed at least 90 days of tofersen at the time of Screening, willing to refrain from initiation of the agent for the duration of the trial.\n\nKey Exclusion Criteria:\n\n1. Any clinically significant and/or unstable medical (including active systemic infections requiring treatment), surgical, or psychiatric condition or laboratory abnormality other than ALS, in the judgement of the Investigator.\n2. Active suicidality (e.g., any suicide attempts within the past 12 months or any current suicidal intent, including a plan, as assessed by the C-SSRS, score of \"YES\" on questions 4 or 5; and/or based on clinical evaluation by the Investigator).\n3. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels greater than 3 times the upper limit of normal (ULN).\n4. Significant renal impairment as determined by estimated glomerular filtration rate (eGFR) of \\<60 mL/min.\n5. Pre-existing chronic obstructive pulmonary disease or significant pulmonary impairment including those with an FEV1 \u2264 2 liters or \\< 75% predicted for height and age, in the judgement of the Investigator.\n6. Clinically significant history of cardiac function impairment including cardiac ejection fraction below 40%, ventricular wall motion abnormalities, or coronary artery disease.\n7. Any organ allografts.\n8. A positive tuberculosis (TB) test indicating a latent TB infection or a positive test for viral hepatitis.\n9. Currently receiving or have received abatacept treatment within 75 days prior to Screening.\n10. Currently receiving or have received interleukin-2 (IL-2) treatment within 30 days prior to Screening.\n11. Currently receiving or expected to receive immunosuppressant therapy (e.g., cyclosporine, sirolimus, tacrolimus, mycophenolate mofetil, systemic steroids) over the course of the study.\n12. Planning to receive a live vaccine during the study or within 3 months of discontinuation.\n13. Current participation in another interventional clinical trial and/or participation in any investigational medication or device clinical trial within 30 days prior to Screening or 5 half-lives of elimination of the investigational medication, whichever is longer.\n14. Previous participation in any COYA 302 (LD rhIL-2 and DRL\\_AB) study.\n15. Uncontrolled autoimmune condition.\n16. Presence of an indwelling central catheter.\n\nNOTE: Other protocol defined Inclusion/Exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "COYA 302", "targeting_mechanism": "Dual immunomodulatory mechanism that enhances anti-inflammatory function of regulatory T cells (Tregs) and suppresses inflammation produced by activated monocytes and macrophages.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01935518", "title": "A Clinical Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University Third Hospital", "summary": "This study will examine whether fasudil is effective and safe in treating patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Fasudil"}], "start_date": "2013-09", "url": "https://clinicaltrials.gov/study/NCT01935518", "target_entities": ["Rho kinase"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "", "country": "China", "status": "RECRUITING", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "0086-15611908107", "contact_email": "dsfan@sina.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinical diagnosis of laboratory-supported probable, probable, or definite ALS\n* Age: 18-70 years\n* Disease duration: 3-36 months\n* Forced vital capacity: at least 60% of predicted\n* ALSFRS-R: at least 30, respiratory items: at least 10\n* Decline of ALSFRS-R in the last 3 months before enrollment: 1-8\n* Must take riluzole, on a stable dose for at least 30 days prior to baseline visit with no serious side effects. They must continue the riluzole treatment for at least 6 months after enrollment.\n* Patients of childbearing potential must be using an effective method of birth control\n* Willing and able to give informed consent\n\nExclusion Criteria:\n\n* Familial ALS\n* Pregnant or nursing women\n* Patients after tracheotomy or continuous ventilator-dependent (time with non-invasive ventilator more than 22 hours per day for 7 consecutive days.)\n* After percutaneous endoscopic gastrostomy\n* Alanine Transaminase (ALT) or Aspartate Transaminase (AST): at least 3 times the upper limit of normal\n* Abnormal creatinine or urea nitrogen\n* Severe cardiac disease, pulmonary disease, hematic disease, autoimmune disease, mental disease, dementia and substance abuse\n* History of malignancy\n* History of intracranial hemorrhage\n* History of severe bleeding of digestive tract, lungs, nose and skin\n* Allergic to fasudil\n* Participating in other clinical studies or using other investigational drugs at present", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Fasudil", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00690118", "title": "Study of Pioglitazone in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Ulm", "summary": "Primary objective:\n\nEfficacy of pioglitazone (45 mg/day) as add-on therapy to standard therapy with riluzole in patients with ALS compared to placebo in terms of survival (mortality defined exclusively as death).\n\nThis is a prospective, multicentre, randomised, stratified, parallel-group, double-blind trial comparing placebo with 45 mg pioglitazone as add-on therapy to 100 mg riluzole in ALS in 220 enrolled patients. For entry, the El Escorial Criteria for diagnosis will be used. The duration of treatment will be 18 months. The primary endpoint will be subjected to a confirmatory analyses. Secondary variables will be incidence of tracheotomy or non-invasive ventilation, ALS Functional Rating Scale, Quality of life and safety variables.", "interventions": [{"type": "DRUG", "name": "pioglitazone"}, {"type": "DRUG", "name": "placebo"}], "start_date": "2008-05", "url": "https://clinicaltrials.gov/study/NCT00690118", "target_entities": ["insulin_sensitivity_metabolic_dysfunction"], "locations": [{"facility": "Department of Neurology, University of Ulm", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Department of Neurology and Center for Palliative Medicine, University of Munich", "city": "Munich", "state": "Bavaria", "country": "Germany", "status": "", "lat": 48.13743, "lon": 11.57549}, {"facility": "Department of Neurology, Universty of Regensburg", "city": "Regensburg", "state": "Bavaria", "country": "Germany", "status": "", "lat": 49.01513, "lon": 12.10161}, {"facility": "Department of Neurology, University of Wuerzburg", "city": "W\u00fcrzburg", "state": "Bavaria", "country": "Germany", "status": "", "lat": 49.79391, "lon": 9.95121}, {"facility": "Department of Neurology, Deutsche Klinik f\u00fcr Diagnostik", "city": "Wiesbaden", "state": "Hesse", "country": "Germany", "status": "", "lat": 50.08601, "lon": 8.24435}, {"facility": "Department of Neurology, University of Goettingen", "city": "G\u00f6ttingen", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 51.53443, "lon": 9.93228}, {"facility": "Department of Neurology, Medical School Hannover", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Department of Neurology, University of Rostock", "city": "Rostock", "state": "Mecklenburg-Vorpommern", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Department of Neurology, Universty of Bonn", "city": "Bonn", "state": "Nordrhrein-Westfalen", "country": "Germany", "status": "", "lat": 50.73438, "lon": 7.09549}, {"facility": "Neurologische Universit\u00e4tsklinik Bergmannsheil", "city": "Bochum", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Department of Neurology, Universty of Muenster", "city": "M\u00fcnster", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.96236, "lon": 7.62571}, {"facility": "Department of Neurology, TU Dresden", "city": "Dresden", "state": "Saxony", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Department of Neurology, University of Halle-Wittenberg", "city": "Halle", "state": "Saxony-Anhalt", "country": "Germany", "status": "", "lat": 51.48158, "lon": 11.97947}, {"facility": "Department of Neurology, University of Jena", "city": "Jena", "state": "Thuringia", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Department of Neurology, Humboldt University", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* possible, probable (clinically or laboratory) or definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria\n* disease duration more than 6 months and less than 3 years\n* best-sitting FVC between 50% and 95% of predicted normal\n* continuously treated with 100 mg riluzole daily, for at least one month\n* onset of progression weakness within 36 months prior to study\n* women of childbearing age be non-lactating and surgically sterile or using a highly effective method of birth control and have a negative pregnancy test\n* capable of thoroughly understanding all information given and giving full informed consent according to GCP\n\nExclusion Criteria:\n\n* previous participation in another clinical study within the preceding three months\n* tracheotomy or assisted ventilation of any type during the preceding three months\n* gastrostomy\n* any medical condition known to have an association with motor neuron dysfunction which might confound or obscure the diagnosis of ALS\n* presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment\n* confirmed hepatic insufficiency or abnormal liver function (ASAT and/or ALAT more than 1.5 upper limit of normal)\n* renal insufficiency (serum creatinine more than 2.26 mg/dl)\n* evidence of major psychiatric disorder or clinically evident dementia precluding evaluation of symptoms\n* known hypersensitivity to any component of the study drugs\n* likely to be not cooperative or comply with the trial requirements (as assessed by the investigator), or unable to be reached in the case of an emergency\n* other antidiabetics\n* heart failure or heart failure in the patients history (NYHA I to IV)\n* history of macular oedema\n* treatment with thiazolidinediones within 3 months prior to screening\n* known or suspected history of alcohol and/or drug abuse\n* treatment with gemfibrozil within 3 months prior to screening", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "pioglitazone", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06968468", "title": "Resiliency Intervention for Patients With ALS and Their Care-Partners", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "The purpose of this study is to pilot a resiliency and coping intervention for persons recently diagnosed with Amyotrophic Lateral Sclerosis (ALS) and their primary informal caregivers. The data investigators gather in this study will be used to further refine our intervention.", "interventions": [{"type": "BEHAVIORAL", "name": "Resilient Together ALS"}], "start_date": "2026-05-13", "url": "https://clinicaltrials.gov/study/NCT06968468", "target_entities": [], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "617-643-7641", "contact_email": "crush2@mgh.harvard.edu", "eligibility": {"criteria": "Inclusion Criteria for patient with ALS:\n\n* recent (within \\~2 months) of first appointment at Healey Center for diagnosis of ALS documented in Epic (based on recommendations from neurologists)\n* ability to understand the study and research protocol as determined by a standardized teach-back method of assessment\n* ability to communicate by writing, speaking, or assistive communication device\n\nInclusion Criteria for dyad:\n\n* English speaking adults\n* dyad lives together\n* at least one partner endorses clinically significant emotional distress during screening (\\>7 on either subscale of the HADS)\n\nExclusion Criteria:\n\n* patient with ALS with comorbid terminal diagnosis or severe mental health disorder that limits ability to participate (by self-report, as determined by our clinical team);\n* cognitive disorder that limits ability to participate (per neurologist)\n* inability or unwillingness to use live video technology (will teach any dyads who have low technology literacy how to use live video technology)", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03472950", "title": "Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Kansas Medical Center", "summary": "The purpose of this research study is to evaluate the safety and effectiveness of Ranolazine, and how well it is tolerated in patients with Amyotrophic Lateral Sclerosis (ALS). Ranolazine is an FDA approved drug that is used for decreasing chest pain.", "interventions": [{"type": "DRUG", "name": "Ranolazine 500 MG"}, {"type": "DRUG", "name": "Ranolazine 1000 MG"}], "start_date": "2018-06-11", "url": "https://clinicaltrials.gov/study/NCT03472950", "target_entities": ["late_sodium_current_cardiac_myocyte_function"], "locations": [{"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with clinically definite, probable, laboratory supported probable, or possible ALS per revised El Escorial criteria\n* Cramp frequency greater than 4 cramps per week during 2 week run in\n* ALS functional rating scale-revised (ALSFRS-R) score of greater than 24\n* Able to lie on back for study procedures\n\nExclusion Criteria:\n\n* Tracheostomy invasive ventilation, or use of non-invasive ventilation greater than 12 hours per day\n* Pregnant or lactating\n* Participation in a prior experimental drug trial less than 30 days prior to screening\n* Patients taking ranolazine\n* Patients taking medications which are contraindicated for use with ranolazine such as strong CYP3 inhibitors (ketoconazole, clarithromycin, nelfinavir), and CYP3 inducers (rifampin, phenobarbital)\n* Patients with clinically significant medical comorbidities (hepatic, renal, cardiac, etc)\n* Patients with baseline QT interval prolongation on Electrocardiography (ECG)\n* Patients pre-disposed to secondary QT prolongation for other health conditions like family history of congenital long QT syndrome, heart failure, bradycardia, or cardiomyopathies", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Ranolazine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07118319", "title": "The Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.", "summary": "A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects with Amyotrophic Lateral Sclerosis", "interventions": [{"type": "DRUG", "name": "Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)"}, {"type": "DRUG", "name": "Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)"}], "start_date": "2025-09-15", "url": "https://clinicaltrials.gov/study/NCT07118319", "target_entities": ["motor_neuron_replacement_regeneration"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "RECRUITING", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "+86 21 64027719", "contact_email": "CEO@xellsmart.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. 18-75 years of age (inclusive of 18 and 75 years), regardless of gender;\n2. Diagnosed of definite or probable ALS according to the revised EI Escorial criteria;\n3. Respiratory function FVC at baseline was \u226570% of the predicted value (FVC%);\n4. Patients with birth-potential (both male and female) must agree to use effective non-drug contraceptive measures from the time of signing the informed consent until 6 months after the conclusion of the trial;\n5. Volunteer to participate in the clinical study, understand and sign the informed consent form.\n\nExclusion Criteria:\n\n1. Subject has signs and symptoms of neuromuscular weakness, and other causes of muscle weakness cannot be ruled out;\n2. Baseline body mass index (BMI) \\< 18.5 kg/m\u00b2;\n3. Primary lateral sclerosis presenting only with upper motor neuron symptoms;\n4. Significant psychiatric disorders that the investigator assesses may affect evaluation;\n5. Diseases causing neurological or muscular dysfunction, such as metabolic muscle diseases or myasthenia gravis;\n6. Diagnosed autoimmune diseases with uncontrolled severe arthritis or other conditions (e.g., lameness) that the investigator assesses may affect evaluation;\n7. Acute active infections requiring antibiotics, antivirals, or antifungals that occurred within the 2 weeks prior to screening and are not controlled;\n8. Subject diagnosed with active pulmonary tuberculosis or treated for suspected tuberculosis;\n9. Diagnosed severe pulmonary diseases that the investigator assesses may affect evaluation;\n10. Poorly controlled hypertension;\n11. Previous or detected cardiac abnormalities;\n12. A history of cirrhosis, chronic hepatitis, or liver function at screening;\n13. A history of chronic kidney disease;\n14. Previous history of bleeding, abnormal clotting, or being treated with anticoagulation;\n15. Active hepatitis B, active hepatitis C, human immunodeficiency virus (HIV) antibody or treponema pallidum antibody positive at screening;\n16. History of severe trauma or surgery and may affect the assessment judged by investigator;\n17. Subjects with contraindications to lumbar puncture, including menifestations of injection site infection or high intracranial pressure.\n18. Those who have had a malignant tumor within 5 years prior to screening or are undergoing antitumor therapy;\n19. Have participated in other clinical trials within 3 months prior to screening;\n20. Pregnant or breastfeeding women;\n21. Subject who is judged by the investigator to be unsuitable for the clinical trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Human Allogeneic Induced Pluripotent Stem Cells (iPSCs)-Derived Motor Neuron Progenitor Cells (XS228CN)", "targeting_mechanism": "Motor neuron progenitor cells to replace or support degenerating motor neurons", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02988297", "title": "Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Revalesio Corporation", "summary": "The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "RNS60"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2026-10", "url": "https://clinicaltrials.gov/study/NCT02988297", "target_entities": ["oxidative_stress_cellular_redox_balance"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Probable, probable-laboratory supported, or definite ALS, either sporadic or familial according to modified El Escorial criteria\n* Disease duration \\< 3 years\n* Age 18 to 80\n* Able to provide informed consent and to comply with study procedures\n* Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to screening (riluzole-na\u00efve participants are permitted in the study)\n* Women must not be lactating or able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control) for the duration of the study, and for 3 months after study completion\n* Men should practice contraception for the duration of the study and for 3 months after completion\n\nExclusion Criteria:\n\n* Diagnosis of a motor neuron disease other than ALS (e.g., primary lateral sclerosis, progressive muscular atrophy, progressive bulbar palsy)\n* Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if they were to participate in the study or that would impact survival or functional disability in the next 12 months\n* Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \\> 3 times the upper limit of the normal\n* Renal insufficiency (Glomerular Filtration Rate \\< 60)\n* Active pulmonary disease\n* Prior poor compliance with an inhalation device\n* The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent, according to judgment by the principal investigator.\n* History of human immunodeficiency virus (HIV) infection, clinically significant chronic hepatitis, or other active infection.\n* Active participation in another ALS clinical trial within 30 days of the Screening Visit\n* Other experimental treatments or anti-inflammatory/immunomodulatory medications used in the preceding 3 months", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RNS60", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04557410", "title": "Open Label Study: Treatment of ALS Fatigue With PolyMVA", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Missouri-Columbia", "summary": "Amyotrophic lateral sclerosis (ALS) is a disease that causes the death of upper and lower motor neurons. ALS symptoms are characterized by stiffness, muscle twitching, and worsening weakness due to muscle breakdown. Onset of symptoms are typically arm or leg weakness or difficulty speaking or swallowing and gradual development of overall body weakness. The cause is unknown and there is no cure for ALS.\n\nPoly MVA was found to substantially lower fatigue and improve quality of life in a pilot study of patients with varied medical disorders. The reduction in fatigue was also observed in a small series of patients enrolled in an open label study for patients with gliomas.\n\nIn this study, we want to find out more about a dietary supplement, called Poly MVA (also called the study drug in this form), for people with ALS. We want to find out if Poly MVA reduces the symptoms of fatigue and depression when taken daily. The supplement contains vitamins, minerals and amino acids (proteins) and has been used by patients with other medical conditions to help with their fatigue and quality of life.", "interventions": [{"type": "DRUG", "name": "PolyMVA"}], "start_date": "2020-09-23", "url": "https://clinicaltrials.gov/study/NCT04557410", "target_entities": ["mitochondrial_function_oxidative_stress"], "locations": [{"facility": "University of Missouri-Columbia School of Medicine", "city": "Columbia", "state": "Missouri", "country": "United States", "status": "", "lat": 38.95171, "lon": -92.33407}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Definite ALS\n* Severe fatigue (defined by FSS \\> 4.0)\n* Expanded Disability Status Scale (EDSS) (measure of neurological impairment) 0 - 7.5 Able to comply with study procedures\n* Stable medication for the past month prior to enrollment.\n\nExclusion Criteria:\n\n* na", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "PolyMVA", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04436497", "title": "HEALEY ALS Platform Trial - Regimen A Zilucoplan", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.\n\nRegimen A will evaluate the safety and efficacy of a single study drug, zilucoplan, in participants with ALS.", "interventions": [{"type": "DRUG", "name": "Zilucoplan"}, {"type": "DRUG", "name": "Matching Placebo"}], "start_date": "2020-07-29", "url": "https://clinicaltrials.gov/study/NCT04436497", "target_entities": ["Complement component C5"], "locations": [{"facility": "Healey Center for ALS at Mass General", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* The following inclusion criterion is in addition to the inclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\n  1. Vaccination with a quadrivalent meningococcal vaccine and meningococcal serotype B vaccine at least 14 days prior to the first dose of study drug at the Baseline (Day 0) visit. Meningococcal vaccines (including boosters) should be administered in accordance with the study's vaccination worksheet.\n\nExclusion Criteria:\n\n* The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\n  1. History of meningococcal disease.\n  2. Prior treatment with a complement inhibitor.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Zilucoplan", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01823380", "title": "Influence of the Vitamin D Blood Levels on the Amyotrophic Lateral Sclerosis Phenotype", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Montpellier", "summary": "The main objective of this study is to investigate the correlation between the rate of motor decline and blood levels of Vitamin D total. Secondary objectives are to investigate the relationship between blood levels of vitamin D and total disease duration of ALS, forced vital capacity, weight loss, age of onset and the start site of ALS.", "interventions": [{"type": "PROCEDURE", "name": "Blood test"}], "start_date": "2012-09", "url": "https://clinicaltrials.gov/study/NCT01823380", "target_entities": [], "locations": [{"facility": "UH Montpellier", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subject with possible, probable or definite Amyotrophic Lateral Sclerosis (ALS).\n* ALS operating for less than three years at dosage of vitamin D time.\n* Subject monitored in the center ALS of Montpellier for 6 months.\n* Subject agreeing to give his consent in writing or orally if the patient SLA is unable to write\n\nExclusion Criteria:\n\n* Subject has received a Vitamine D treatment in the six months preceding the inclusion\n* Subject with a clinical condition on the inclusion day that makes it highly probable death in the year (quadriplegic patient, subject ventilated for respiratory failure in ALS, major malnutrition) or with ALSFRS-R score \\<20.\n* Pregnant or breastfeeding women\n* Subject not covered by a social security scheme.\n* Subject under guardianship\n* Adult protected by the law", "sex": "ALL", "min_age": "18 Years", "max_age": "95 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02795052", "title": "Neurologic Stem Cell Treatment Study", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "MD Stem Cells", "summary": "This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will provide improvement in neurologic function for patients with certain neurologic conditions. http://mdstemcells.com/nest/", "interventions": [{"type": "PROCEDURE", "name": "Intravenous and Intranasal BMSC"}], "start_date": "2016-06", "url": "https://clinicaltrials.gov/study/NCT02795052", "target_entities": ["neural_regeneration_neuroprotection"], "locations": [{"facility": "MD Stem Cells", "city": "Westport", "state": "Connecticut", "country": "United States", "status": "RECRUITING", "lat": 41.14149, "lon": -73.3579}, {"facility": "MD Stem Cells", "city": "Coral Springs", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.27119, "lon": -80.2706}, {"facility": "The Saudi-German Hospital", "city": "Dubai", "state": "United Arab Emirates", "country": "United Arab Emirates", "status": "RECRUITING", "lat": 25.07725, "lon": 55.30927}], "contact_phone": "203-423-9494", "contact_email": "stevenlevy@mdstemcells.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Have documented functional damage to the central or peripheral nervous system unlikely to improve with present standard of care.\n2. Be at least 6 months post-onset of the disease.\n3. If under current medical therapy (pharmacologic or surgical treatment) for the condition be considered stable on that treatment and unlikely to have reversal of the associated neurologic functional damage as a result of the ongoing pharmacologic or surgical treatment.\n4. In the estimation of Dr. Weiss and the neurologists have the potential for improvement with BMSC treatment and be at minimal risk of any potential harm from the procedure.\n5. Be over the age of 18 and capable of providing informed consent.\n6. Be medically stable and able to be medically cleared by their primary care physician or a licensed primary care practitioner for the procedure. Medical clearance means that in the estimation of the primary care practitioner, the patient can reasonably be expected to undergo the procedure without significant medical risk to health.\n\nExclusion Criteria:\n\n1. All patients must be capable of an adequate neurologic examination and evaluation to document the pathology. This will include the ability to cooperate with the exam.\n2. Patients must be capable and willing to undergo follow up neurologic exams with the sub-investigators or their own neurologists as outlined in the protocol.\n3. Patients must be capable of providing informed consent.\n4. In the estimation of Dr. Weiss the BMSC collection and treatment will not present a significant risk of harm to the patient's general health or to their neurologic function. .\n5. Patients who are not medically stable or who may be at significant risk to their health undergoing the procedure will not be eligible.\n6. Women of childbearing age must not be pregnant at the time of treatment and should refrain from becoming pregnant for 3 months post treatment.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous bone marrow derived stem cells (BMSC)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "Bone marrow-derived stem cells demonstrate potential for motor neuron protection in SOD1G93A mouse models of ALS.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03900247", "title": "Restore Motor Function Through Robotic Arm Exoskeleton and Brain Computer Interface", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Aarhus", "summary": "The current project will aid patients with motor impairment to reduce the need for homecare. Specifically the aim is to develop and implement a robotic exoskeleton and brain computer interface to assist and eventually perform arm and hand movement in patients with the progressive neurodegenerative disease ALS. This proposal brings together state-of-the-art robotic technology, EEG-based brain computer interface (BCI) know-how, clinical expertise, patient perspective and industrial partners to develop and implement a robotic arm/hand device that will adapt, with increasing brain-computer control, based on the need of the patient. In short the BCI will measure electroencephalography (EEG) from the surface of the scalp and recognize signature EEG as the patient intents to move. As the patient loses muscle power the BCI robotic-device will gradually take over and support motor activity, even when the patient is totally paralyzed. As the device supports hand/arm function only, the investigators aim to address ADLs associated to hand function, specifically eating activities.", "interventions": [{"type": "DEVICE", "name": "REMAP EEG based BCI and SEM-Glove BioServo"}], "start_date": "2019-03-18", "url": "https://clinicaltrials.gov/study/NCT03900247", "target_entities": [], "locations": [{"facility": "Center of Functionally Integrative Neuroscience", "city": "Aarhus", "state": "", "country": "Denmark", "status": "", "lat": 56.15674, "lon": 10.21076}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS according to the latest revision of El Escorial Criteria (Ludorph et al. 2015)\n* Loss of muscle force or fine motor skills in a hand\n\nExclusion Criteria:\n\n* Other severe Neurological or Psychiatric disease\n* Drug or Alcohol dependency\n* Pregnancy\n* Severe cognitive disturbances found to impede with study completion", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "REMAP EEG based BCI and SEM-Glove BioServo", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01639391", "title": "Creation of a Bank of Fibroblast From Patients With Amyotrophic Lateral Sclerosis: Pilot Study", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Institut Pasteur", "summary": "Patients with ALS will be included in the reference center for ALS in hospital La Piti\u00e9 Salp\u00eatri\u00e8re, Paris.\n\nThe study proposes to investigate the pathophysiology of ALS by setting up a fibroblast bank for studying molecular, cellular and genetic parameters of the pathology.\n\niPS (induced pluripotent stem cells) and then differentiated cells will be generated.\n\nThe pathophysiology of ALS will be studied on the 3 types of cells (fibroblasts, iPS, differentiated cells).", "interventions": [{"type": "PROCEDURE", "name": "skin biopsy"}], "start_date": "2012-11-29", "url": "https://clinicaltrials.gov/study/NCT01639391", "target_entities": [], "locations": [{"facility": "Centre r\u00e9f\u00e9rent maladies rares SLA", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* patients over 18 years old\n* ALS sporadic or familial or unknown\n\nExclusion Criteria:\n\n* known cutaneous disease not allowing the biopsy\n* platelets less than 10 000/m3\n* suspected or known xylocain allergy", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04257760", "title": "Evaluation of Palliative Care for Patients With ALS and Their Caregivers", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ottawa Hospital Research Institute", "summary": "Rationale: Amyotrophic lateral sclerosis (ALS) is a degenerative illness which currently has no medical cure. It is routinely accompanied by a significant symptom burden including high levels of distress in patients and their caregivers. As a result, an early palliative care approach is recommended in the ALS population. Palliative care has been shown to have positive effects on the quality of life in patients and caregivers in other life limiting illness such as cancer and multiple sclerosis. Unfortunately, our understanding of the palliative care needs in ALS is limited and the efficacy of palliative care involvement is poorly understood. Furthermore, ALS patients are largely underserved by palliative care in Ontario, with \\<50% of ALS patients receiving palliative care even in the last year of life. Hypothesis: The investigators hypothesize that ALS patients will be agreeable to palliative care consultations and that this will improve the quality of life of patients and their caregivers. Specific Aims: This project seeks to initiate routine palliative care consultation in an interdisciplinary ALS clinic to: 1) improve patient and caregiver quality of life, 2) further understand the palliative care needs of the ALS population and 3) identify which patients and caregivers are most likely to benefit from palliative care consultation, thus guiding clinicians on when to refer in the future. Significance: This study is the first investigate the feasibility and efficacy of palliative care consultation in the ALS population, and its effects on quality of life. It has the potential to provide increased support to patients as well as caregivers. Finally, this study will aid in our understanding of the optimal time to involve palliative care in the ALS population and will act as a foundation on which larger, controlled studies can be built.", "interventions": [{"type": "BEHAVIORAL", "name": "Palliative Care consultation outside standard of care"}], "start_date": "2020-10-08", "url": "https://clinicaltrials.gov/study/NCT04257760", "target_entities": [], "locations": [{"facility": "The Ottawa Hospital", "city": "Ottawa", "state": "Ontario", "country": "Canada", "status": "", "lat": 45.41117, "lon": -75.69812}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nPatient with ALS at the Ottawa Hospital ALS clinic or caregiver of participating patient.\n\nExclusion Criteria:\n\nSignificant cognitive impairment Prior palliative care consultation", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06249412", "title": "The Importance of Positive Expiratory Pressure Associated With the In-exsufflator in ALS Patients", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Groupe Hospitalier du Havre", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder that impairs motor neurons, with a life expectancy of 2 to 7 years after diagnosis. ALS manifests as 'spinal' when it primarily affects limbs, or 'bulbar' when it impairs speech and swallowing. The disease progressively weakens all skeletal muscles, causing respiratory issues and increased risk of lung infections due to ineffective coughing. Mechanical cough assistance via In-exsufflation therapy/ mechanical in-exsufflator devie (INEX/MI-E) applies positive and negative airway pressures non-invasively to improve coughing. However, MI-E may fail in some ALS patients due to airway collapse, often related to brainstem muscle dysfunction.Research by Andersen et al. in 2017 highlighted that during MI-E, ALS patients often experience adverse laryngeal movements, which can obstruct airways and reduce the therapy's effectiveness. To combat this, they suggested individualized MI-E settings to minimize airway collapse. Modern MI-E devices, such as the EOVE-70, offer adjustable positive expiratory pressure (PEP) between cycles to potentially enhance airway stability and coughing efficiency. The current study focuses on the impact of PEP during therapy pauses on the peak expiratory flow rate in ALS patients, which could lead to improved therapeutic outcomes.", "interventions": [{"type": "DEVICE", "name": "MI-E with PEP function activated during the pause"}, {"type": "DEVICE", "name": "MI-E without PEP function activated during the pause"}], "start_date": "2024-12-19", "url": "https://clinicaltrials.gov/study/NCT06249412", "target_entities": [], "locations": [{"facility": "GH Havre", "city": "Le Havre", "state": "Normandy", "country": "France", "status": "RECRUITING", "lat": 49.49346, "lon": 0.10785}], "contact_phone": "+332 32 73 32 32", "contact_email": "yann.combret@ch-havre.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female patient aged over 18 years\n* Patient with Amyotrophic Lateral Sclerosis (ALS)\n* Patients with established or beginning bulbar disorders, identified by the healthcare team (speech impairment, hypersalivation, or swallowing difficulties)\n* Patient naive to INEX therapy but prescribed for its installation or patient already treated by an INEX device\n* Patient followed by the ALS mobile team of the Groupe Hospitalier du Havre or the CHU of Dijon\n* Patient whose disease progression kinetics is medically deemed compatible with inclusion in the study\n* Patient willing to participate in the research after receiving adequate information and the information letter.\n* Patient affiliated with social security or a beneficiary of such a scheme.\n\nExclusion Criteria:\n\n\"\u25cf Patient not presenting an episode of infection or a past episode of respiratory infection less than one month old\n\n* Mental illness interfering with the proper use of the device\n* History of laryngospasm\n* Inability to come for consultation with the ALS team of the Groupe Hospitalier du Havre or the CHU of Dijon\n* Pregnancy\n* Person deprived of liberty by judicial or administrative decision, person subject to a legal protection measure (patient under guardianship or curatorship) Article L1121-8.\n* Appearance of a non-inclusion criterion\n* Refusal to participate after inclusion\n* Death from any cause\"", "sex": "ALL", "min_age": "18 Years", "max_age": "100 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04309604", "title": "IC14 for ALS Patients Expanded Access", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Implicit Bioscience", "summary": "The primary objective is to provide the investigational product, IC14, at the dose of 8 mg/kg intravenously every 2 weeks for 12 weeks to 6 participants with amyotrophic lateral sclerosis (ALS). No clinical hypotheses are being tested. An extension for 6 additional doses every 2 weeks will be allowed if the drug is safe and well tolerated.A second extension for 14 doses every 2 weeks will be allowed if the drug is safe and well tolerated.", "interventions": [{"type": "BIOLOGICAL", "name": "IC14"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT04309604", "target_entities": ["C5 complement component"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Signed informed consent prior to initiation of any program-specific procedures.\n2. Familial or sporadic ALS defined as clinically possible, probable, or definite by Awaji-Shima Consensus Recommendations or other standard guidance.\n3. Adequate hematologic, renal, and liver function as defined by the patient's physician.\n4. Females of childbearing potential should be using and committed to continue using one of the following acceptable birth control methods:\n\n   1. Sexual abstinence (inactivity) for 1 month prior to screening through study completion; or\n   2. Intrauterine device (IUD) in place for at least 3 months prior to study through study completion; or\n   3. Stable hormonal contraception for at least 3 months prior to study through study completion; or\n   4. Surgical sterilization (vasectomy) of male partner at least 6 months prior to study.\n5. To be considered of non-childbearing potential, females should be surgically sterilized (bilateral tubal ligation, hysterectomy, or bilateral oophorectomy at least 2 months prior to study) or be post-menopausal and at least 3 years since last menses.\n6. Males with female partners of childbearing potential must use contraception through study completion.\n\nExclusion Criteria:\n\n1. Dependence on invasive mechanical ventilation, defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use; presence of tracheostomy at screening;\n2. Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other opportunistic infections; or major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks of baseline.\n3. Immunized with live-attenuated vaccines within 30 days before dosing. Participants must agree to forego live-attenuated vaccines throughout the program, including 12 weeks after the last dose of IC14.\n4. History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies.\n5. Pregnancy or breastfeeding.\n6. Presence of any of the following clinical conditions:\n\n   * History of one or more of the following: cardiac insufficiency (New York Heart Association III/IV), uncontrolled cardiac arrhythmias, unstable ischemic heart disease, or uncontrolled hypertension (systolic blood pressure \\> 170 mmHg or diastolic blood pressure \\> 110 mmHg).\n   * History of venous thromboembolic disease within 6 months, myocardial infarction, or cerebrovascular accident.\n   * Unstable pulmonary, renal, hepatic, endocrine or hematologic disease.\n   * Autoimmune disease, mixed connective tissue disease, scleroderma, polymyositis, or significant systemic involvement secondary to rheumatoid arthritis.\n   * Evidence of active malignant disease, malignancies diagnosed within the previous 5 years, or breast cancer diagnosed within the previous 5 years (except skin cancers other than melanoma).\n   * History of human immunodeficiency virus infection or other immunodeficiency illness.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "IC14", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07369076", "title": "A Clinical Trial to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Nura Bio", "summary": "The purpose of this trial is to learn about the effects of NB-4746 compared with placebo in people with amyotrophic lateral sclerosis.\n\nThe questions this trial aims to answer in comparing NB-4746 to placebo are:\n\n* What adverse events associated with taking NB-4746 are reported during this trial? (An adverse event is any sign or symptom that participants have during a trial. Adverse events may or may not be caused by treatments in the trial.)\n* How does NB-4746 move into, through, and out of the body of the participants?\n* What is the change in the level of neurofilament light (NfL) in the participants' blood? (NfL is a marker used to measure the extent of damage to the nerve cells.)\n\nThis trial has 2 parts. The trial doctors will start Part A before starting Part B of the trial. Participants have an option to enter the open label extension after completing Part A or Part B.\n\nPart A: Participants will be randomly placed into 1 of the 3 groups. There are equal chances to be assigned to either group. Group 1: Participants will receive NB-4746 capsules at a low dose to take by mouth twice daily for 1 month. Group 2: Participants will receive NB-4746 capsules at a high dose to take by mouth twice daily for 1 month. Group 3: Participants will receive placebo capsules to take twice daily for approximately 1 month.\n\nPart B: Participants will be randomly placed into 1 of the 2 groups. There are equal chances to be assigned to either group. Group 1: Participants will receive NB-4746 capsules at a dose determined by Part A to take by mouth twice daily for 12 weeks. Group 2: Participants will receive placebo capsules to take twice daily for approximately 12 weeks.\n\nNone of the participants, trial doctors, or trial staff will know which treatment the participants will receive during Part A or B. Some trials are done this way because knowing what treatment the participants receive can affect the results of the trial. Doing a trial this way helps to make sure that the results are looked at with fairness across all treatments.\n\nOpen-Label Extension: Upon the completion of Part A or Part B, the doctor will verify the participant's willingness to continue receiving study treatment. This open label extension continues until each participant completes up to 1 year of treatment. The trial doctors will check participants' ALS and general health throughout the trial.", "interventions": [{"type": "DRUG", "name": "NB-4746 High dose"}, {"type": "DRUG", "name": "NB-4746 Low dose"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "NB-4746 (Dose TBD)"}], "start_date": "2026-03-04", "url": "https://clinicaltrials.gov/study/NCT07369076", "target_entities": ["astrocyte_activation_neuroinflammation"], "locations": [{"facility": "Concord Repatriation General Hospital", "city": "Concord", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": -33.84722, "lon": 151.10381}, {"facility": "Neuroscience Research Australia", "city": "Randwick", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": -33.91439, "lon": 151.24895}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Brisbane", "state": "Queensland", "country": "Australia", "status": "RECRUITING", "lat": -27.46794, "lon": 153.02809}, {"facility": "Flinders Medical Centre", "city": "Bedford Park", "state": "South Australia", "country": "Australia", "status": "RECRUITING", "lat": -35.02204, "lon": 138.56815}, {"facility": "Calvery Health care Bethlehem", "city": "Caulfield South", "state": "Victoria", "country": "Australia", "status": "RECRUITING", "lat": -37.89562, "lon": 145.02597}, {"facility": "Perron Institute", "city": "Nedlands", "state": "Western Australia", "country": "Australia", "status": "RECRUITING", "lat": -31.98184, "lon": 115.8073}, {"facility": "Stan Cassidy Center for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "RECRUITING", "lat": 45.94541, "lon": -66.66558}, {"facility": "Genge Partners", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "801-918-7637", "contact_email": "lmehta@nurabio.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n\\- ALS Criteria for Phase 1b:\n\n1. Participants in Phase 1b must have:\n\n   1. Diagnosis of ALS per Gold Coast Criteria; and\n   2. Symptom onset \u226448 months prior to randomization on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in the limbs, bulbar region, or trunk. Weakness in the bulbar region includes dysarthria and dysphagia.\n\n      * ALS Criteria for Phase 2:\n2. Participants in Phase 2 must have:\n\n   1. Diagnosis of ALS per Gold Coast Criteria; and\n   2. Symptom onset \u226424 months prior to randomization on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in the limbs, bulbar region, or trunk. Weakness in the bulbar region includes dysarthria and dysphagia.\n\n      * Additional Inclusion Criteria (All Participants):\n3. Male or female participants aged \u226518 years and \u226480 years at the time of signing informed consent.\n4. Participants must demonstrate functional oral intake and be independent of enteral nutritional support (e.g., PEG, G-tube) at the time of screening per Principal Investigator's judgement.\n5. Slow vital capacity (SVC) \u226560% of predicted at Screening.\n6. If taking riluzole, participant must be on a stable dose for \u226560 days prior to Screening lab blood draw.\n7. If taking edaravone, participant must have completed at least 1 cycle of edaravone prior to screening labs.\n8. Willing to adhere to contraceptive requirements during the study period as described in Appendix 1.\n9. Capable of giving signed informed consent, as described in Section 13.2.2, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n\nExclusion Criteria:\n\n1. Presence of tracheostomy or permanent assisted ventilation; defined as \\> 22 hours daily of mechanical ventilation for more than 1 week (7 days).\n2. History or presence of clinically significant uncontrolled medical conditions (other than ALS) that include cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study drugs; or interfering with the interpretation of data as determined by the Investigator.\n3. Lifetime history of cancer except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years. Fully resected basal cell carcinoma and squamous cell carcinoma with no evidence of recurrence for 1 year are permitted.\n4. Female participants who have a positive serum pregnancy test at Screening, positive urine pregnancy test on Day 1, or who are breastfeeding on Day 1.\n5. Has a spinal deformity or other condition that may prevent the performance of a lumbar puncture.\n6. International Normalized Ratio (INR) \\>1.4, platelet count \\<50,000/\u03bcL, or use of warfarin, heparin, or direct oral anticoagulants.\n7. History of Class III/IV heart failure (per New York Heart Association \\[NYHA\\]).\n8. Inability to swallow or tolerate oral medications at Screening.\n9. Current participation in any other investigational drug study or receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) before Screening.\n10. Known sensitivity to the NB-4746 study drug or any of the formulation components.\n11. Has received NB-4746 at any time prior to initial Screening.\n12. Any other reason that, in the opinion of the Investigator, would confound the conduct of this study or the interpretation of the results or that could compromise the participant's safety.\n13. Currently taking or planning to receive tofersen for the treatment of ALS.\n14. If a participant does not transition to the OLE in \u226430 days following completion of the main study, the participant will need to be rescreened for laboratory criteria and contraception/pregnancy criteria.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NB-4746", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04800926", "title": "Xavier Electromyographic Wheelchair Control for Limited Mobility Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mayo Clinic", "summary": "The purpose of this study is to assess the functional mobility and self-reported satisfaction with the Xavier electromyography hands-free wheelchair control system in comparison with a standard joystick.", "interventions": [{"type": "DEVICE", "name": "Xavier wheelchair controller"}], "start_date": "2019-01-11", "url": "https://clinicaltrials.gov/study/NCT04800926", "target_entities": [], "locations": [{"facility": "Mayo Clinic in Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS diagnosis by revised el Escorial criteria (definite, probable or probable laboratory supported)\n* Age 18-89\n* Limited mobility with use of motorized wheelchair at screening-time\n* Impairment of hand function limiting the use of a standard joystick control\n* Caregiver willing to assist with transfers into wheelchair and application of controllers\n* Ability to attend study visits with a motorized wheelchair\n* Ability to communicate and answer patient reported outcome measure questions\n\nExclusion Criteria:\n\n* Cognitive impairment prohibiting safe independent mobility as defined by an ALS-Cognitive Behavioral Screen (ALS-CBS) score of \\<10 or the opinion of the investigator\n* A sensory impairment prohibiting safe independent mobility in the opinion of the investigator\n* Allergy to adhesives or electrode gels (required for EMG electrodes)\n* Skin breakdown over the temporalis muscle that would predispose to further breakdown and/or infection with electrodes\n* Severe loss of facial muscle functionality or control that would preclude EMG electrode efficacy\n* Subjects who do not have the capacity to consent", "sex": "ALL", "min_age": "18 Years", "max_age": "89 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01709149", "title": "Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "The purpose of this research study is to evaluate the safety and effectiveness of CK-2017357 when taken with or without riluzole (also called Rilutek\u00ae) in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "CK-2017357"}, {"type": "OTHER", "name": "Placebo tablets"}, {"type": "DRUG", "name": "Riluzole"}], "start_date": "2012-10", "url": "https://clinicaltrials.gov/study/NCT01709149", "target_entities": ["Skeletal muscle fiber"], "locations": [{"facility": "Barrow Neurology", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California, San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "UC Irvine ALS & Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Coordinated Clinical Research", "city": "San Diego", "state": "California", "country": "United States", "status": "", "lat": 32.71571, "lon": -117.16472}, {"facility": "California Pacific Medical Center Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "The George Washington University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Mayo Clinic Florida Department of Neurology", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Emory University, School of Medicine", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Georgia Health Sciences University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Indiana University Department of Neurology", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Hospital", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "St Mary's Healthcare", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "Hennepin County Medical Center - Berman Center for Research", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Saint Louis University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Dartmouth Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Rochester", "city": "Rochester", "state": "New York", "country": "United States", "status": "", "lat": 43.15478, "lon": -77.61556}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Medical Center Department of Neurology", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest University, School of Medicine", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Ohio State University Department of Neurology", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Oregon Health & Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Hershey Neuroscience Clinics", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel Neurology", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburgh", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Vanderbilt University Medical Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Baylor College of Medicine", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "UTHSCSA Department of Neurology", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Virginia", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "West Virginia University Department of Neurology", "city": "Morgantown", "state": "West Virginia", "country": "United States", "status": "", "lat": 39.62953, "lon": -79.9559}, {"facility": "Medical College of Wisconsin", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "Heritage Medical Research", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta Hospital", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "University of British Columbia", "city": "Vancouver", "state": "British Columbia", "country": "Canada", "status": "", "lat": 49.24966, "lon": -123.11934}, {"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "QE II Health Sciences Centre", "city": "Halifax", "state": "Nova Scotia", "country": "Canada", "status": "", "lat": 44.64269, "lon": -63.57688}, {"facility": "McMaster University Medical Centre", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "Queen's University : Kingston General", "city": "Kingston", "state": "Ontario", "country": "Canada", "status": "", "lat": 44.22976, "lon": -76.48098}, {"facility": "London Health Sciences", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Univ. of Toronto - Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "H\u00f4pital Notre Dame (CHUM) Centre Hospitalier de l'Universite de Montreal", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "CHU de Quebec: Hopital l'Enfant-Jesus", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "CHRU de Lille - H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges - H\u00f4pital Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "H\u00f4pital La Timone Adulte", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHU Montepellier", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "H\u00f4pital Archet 1", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "H\u00f4pital de la Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "H\u00f4pital Bretonneau", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Charite Universit\u00e4tsmedizin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Hannover Medical School", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "University of Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Trinity College, Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Carlos III", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Barts and the London MND & the Centre Royal London Hospital", "city": "Whitechapel", "state": "London", "country": "United Kingdom", "status": "", "lat": 51.51382, "lon": -0.06583}, {"facility": "Walton Centre for Neurology and Neurosurgery", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "Kings College Hospital NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "John Radcliffe Hospital", "city": "Oxford", "state": "", "country": "United Kingdom", "status": "", "lat": 51.75222, "lon": -1.25596}, {"facility": "Plymouth Hospitals NHS Trust", "city": "Plymouth", "state": "", "country": "United Kingdom", "status": "", "lat": 50.37153, "lon": -4.14305}, {"facility": "Sheffield Institute for Translational Neuroscience", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to comprehend and willing to sign an Informed Consent Form (ICF)\n2. Male or female 18 years of age or older\n3. A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria)\n4. Upright Slow Vital Capacity (SVC) \\>50 % of predicted for age, height and sex\n5. At least 4 of the 12 ALSFRS-R questions must be scored 2 or 3\n6. Diminished but measurable maximum voluntary grip strength in at least one hand; i.e., between 10 and 50 pounds (females) and 10 and 70 pounds (males)\n7. Able to swallow tablets without crushing\n8. A caregiver (if one is needed) who can and will observe and report the patient's status\n9. Pre-study clinical laboratory findings within normal range or, if outside of the normal range, deemed not clinically significant by the Investigator\n10. Male patients must agree for the duration of the study and 10 weeks after the end of the study to use a condom during sexual intercourse with female partners who are of reproductive potential and to have female partners use an additional effective means of contraception (e.g., diaphragm plus spermicide, or oral contraceptives) or the male patient must agree to abstain from sexual intercourse during and for 10 weeks after the end of the study\n11. Female patients must be post-menopausal (\u2265 1 year) or sterilized, or, if of childbearing potential, not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the course of the study, and use contraceptive drugs or devices as detailed in item 10 for the duration of the study and for 10 weeks after the end of the study\n12. Patients must be either on a stable dose of riluzole 50 mg twice daily for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and are willing not to begin riluzole use during the conduct of this study.\n\nExclusion Criteria:\n\n1. Any use of non-invasive positive pressure ventilation (NIPPV, e.g. continuous positive airway pressure \\[CPAP\\] or bilevel positive airway pressure \\[BiPAP\\]) for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation\n2. Patients with a diaphragm pacing system (DPS) at study entry or who anticipate DPS placement during the course of the study\n3. Body Mass Index (BMI) of 19.0 kg/m2 or lower\n4. Unwilling to discontinue tizanidine and theophylline-containing medications during study participation\n5. Serum chloride \\< 100 mmol/L\n6. Neurological impairment due to a condition other than ALS, including history of transient ischemic attack (TIA) within the past year\n7. Presence at screening of any medically significant cardiac, pulmonary, gastrointestinal (GI), musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data\n8. Has taken any investigational study drug within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing\n9. Previously received CK-2017357 in any previous clinical trial", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CK-2017357", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06111014", "title": "Continuation Study for Latozinemab", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Alector Inc.", "summary": "Continuation study to provide continued access to latozinemab for participants who have previously participated in a latozinemab study", "interventions": [{"type": "DRUG", "name": "Latozinemab"}], "start_date": "2023-12-08", "url": "https://clinicaltrials.gov/study/NCT06111014", "target_entities": ["Amyloid beta"], "locations": [{"facility": "Dignity Health - Arizona", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Mayo Comprehensive Cancer Center - PPDS", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Irving Institute for Clinical and Translational Research", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Cincinnati Gardner Neuroscience Institute", "city": "Cincinnati", "state": "Ohio", "country": "United States", "status": "", "lat": 39.12711, "lon": -84.51439}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Parkwood Institute", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Groupe Hospitalier Piti\u00e9 Salp\u00e9tri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Universit\u00e4tsklinikum Ulm - Leimgrubenweg 12-14", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "IRCCS - Centro S. Giovanni di Dio Fatebenefratelli", "city": "Brescia", "state": "", "country": "Italy", "status": "", "lat": 45.53558, "lon": 10.21472}, {"facility": "Fondazione IRCCS C\u00e0 Granda Ospedale Maggiore Policlinico -Via Fracesco Sforza 35", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Azienda Ospedaliero Universitaria Di Modena Policlinico", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Erasmus MC-Dr. Molewaterplein 40", "city": "Rotterdam", "state": "", "country": "Netherlands", "status": "", "lat": 51.9225, "lon": 4.47917}, {"facility": "Centro Hospitalar E Universitario de Coimbra EPE", "city": "Coimbra", "state": "", "country": "Portugal", "status": "", "lat": 40.20686, "lon": -8.41996}, {"facility": "Centro Hospitalar Universit\u00e1rio Lisboa Norte, EPE - Hospital de Santa Maria", "city": "Lisbon", "state": "", "country": "Portugal", "status": "", "lat": 38.72509, "lon": -9.1498}, {"facility": "Karolinska Universitetssjukhuset Huddinge", "city": "Huddinge", "state": "", "country": "Sweden", "status": "", "lat": 59.23705, "lon": 17.98192}, {"facility": "University College London", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Written informed consent must be obtained and documented (from the participant or, where jurisdictions allow it, from their legal decision maker).\n* Has successfully completed participation in their parent latozinemab study.\n* Female participants must be nonpregnant and nonlactating.\n* Male participants must agree to acceptable contraception use.\n\nExclusion Criteria:\n\n* Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.\n* Clinically significant heart disease, liver disease or kidney disease. History or evidence of clinically significant brain disease other than FTD.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Latozinemab", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00537446", "title": "Effect of Noninvasive Ventilation on Lung Function in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Columbia University", "summary": "Amyotrophic Lateral Sclerosis (ALS), or \"Lou Gehrig's Disease\", is a fatal disorder that causes progressive degeneration and weakening of the muscles of breathing, leading to breathing insufficiency and eventually breathing failure. This breathing insufficiency is commonly treated with a breathing assistance device, known as noninvasive positive pressure ventilation (NIPPV). While generally well tolerated and accepted, it is not clear whether or to what extent NIPPV in fact helps breathing function: some data suggest that NIPPV preserves breathing function over time, whereas other data suggest that it actually causes breathing function to decline more quickly. No studies have shown what the acute effect of NIPPV is on breathing muscle function in ALS patients.\n\nThis study will test the hypothesis that the acute use of NIPPV, at pressure levels that are in common clinical use, will cause measurable changes in tests of breathing function, compared to baseline and to lower levels of NIPPV. We expect that the results of this study will help to clarify whether and to what extent NIPPV assists respiratory muscle function in patients with ALS.", "interventions": [{"type": "DEVICE", "name": "noninvasive positive pressure ventilation"}, {"type": "DEVICE", "name": "noninvasive positive pressure ventilation"}], "start_date": "2007-09", "url": "https://clinicaltrials.gov/study/NCT00537446", "target_entities": [], "locations": [{"facility": "Eleanor and Lou Gehrig ALS/MDA Center at Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* clinical diagnosis of amyotrophic lateral sclerosis\n* clinical indication to start noninvasive ventilation (forced vital capacity \\< 50% predicted or signs/symptoms of respiratory insufficiency)\n* age 18 to 80 years old\n\nExclusion Criteria:\n\n* prior institution of NIPPV\n* inability to safely use NIPPV\n* indications for tracheostomy assisted ventilation because of inability to clear secretions from the airway\n* inability or unwillingness to perform pulmonary function testing\n* presence of advanced dementia.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05395624", "title": "Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ashvattha Therapeutics, Inc.", "summary": "This is a Phase 1/2 study to evaluate the safety and tolerability of 18F-OP-801 in subjects with ALS, AD, MS, PD and age-matched HVs. 18F-OP-801 is intended as a biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation.", "interventions": [{"type": "DRUG", "name": "18F-OP-801"}], "start_date": "2023-02-02", "url": "https://clinicaltrials.gov/study/NCT05395624", "target_entities": ["neuroinflammation"], "locations": [{"facility": "UCSF", "city": "San Francisco", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.77493, "lon": -122.41942}, {"facility": "Stanford University", "city": "Stanford", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.42411, "lon": -122.16608}, {"facility": "Mayo Clinic Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 30.33218, "lon": -81.65565}], "contact_phone": "408-476-4172", "contact_email": "jcheung@avttx.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Has the ability to understand and sign the written ICF and local medical privacy authorization forms, which must be obtained prior to the conduct of any study related procedures.\n2. Female subjects of non-childbearing potential must be either surgically sterile (hysterectomy, bilateral tubal ligation, salpingectomy, and/or bilateral oophorectomy at least 26 weeks before the Screening Visit) or postmenopausal, defined as spontaneous amenorrhea for at least 2 years, with follicle-stimulating hormone (FSH) in the postmenopausal range at screening, based on the local laboratory's defined ranges.\n3. Female subjects of childbearing potential (i.e., ovulating, premenopausal, and not surgically sterile) and all male subjects must agree to practice abstinence from sexual intercourse or use a medically accepted contraceptive regimen (including hormonal contraceptives) during their participation in the study and for 90 days (males) or 6 months (females) after Day 1. Medically accepted contraceptive methods are defined as those with 90% or greater efficacy and are as follows:\n\n   1. Male subjects: condoms or surgical sterilization of subject at least 26 weeks before the Screening Visit (i.e., vasectomy).\n   2. Female subjects:\n\n      1. Surgical sterilization at least 26 weeks before the Screening Visit (includes hysterectomy or bilateral tubal ligation, bilateral oophorectomy, or salpingectomy);\n      2. Intrauterine device or diaphragm with spermicide for at least 12 weeks before the Screening Visit; or\n      3. Hormonal contraception (oral, implant, injection, ring, or patch) for at least 12 weeks before the Screening Visit.\n4. If male, subjects must agree to abstain from sperm donation through 90 days after the Day 1 Visit.\n5. Female subjects may not be pregnant, lactating, or breastfeeding.\n6. Female subjects of childbearing potential must have negative result for pregnancy test at Screening and Check-in.\n7. Subjects must have an estimated glomerular filtration rate (eGFR) of \\>45 mL/min/1.73m2 at Screening.\n8. C-reactive protein level \u226410 mg/dL.\n9. Subjects must be willing and able to abide by all study requirements and restrictions.\n\n   Inclusion Criteria Specific to ALS Subjects:\n10. Adult (Age 18 to 80, inclusive) at the Screening Visit\n11. Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by the modified El Escorial criteria.\n12. Forced vital capacity (FVC) of \u226550%; or if in the opinion of the investigator can lay flat for up to 90 minutes. If FVC has been performed within the past 6 months, this data may be used at the discretion of the investigator.\n13. For ALS subjects, medication changes within 30 days prior to the Screening Visit should be discussed with the Medical Monitor.\n\n    Inclusion Criteria Specific to AD Subjects\n14. Adult (Age 40 to 80, inclusive) at the Screening Visit\n15. Clinical diagnosis of early stage dementia, Alzheimer type, plus positive A\u03b2 and tau PET imaging, cerebrospinal fluid (CSF) and/or plasma biomarkers consistent with 2018 NIA-AA criteria\n16. MMSE score \\>20 at Screening\n17. AD medication changes within 30 days prior to the Screening Visit should be discussed with the Medical Monitor.\n\n    Inclusion Criteria Specific to MS Subjects:\n18. Adult (Age 18 to 70, inclusive) at the Screening Visit\n19. MS medication changes within 30 days prior to the Screening Visit should be discussed with the Medical Monitor.\n\n    Inclusion criteria specific to subjects with RRMS:\n20. Diagnosis of RRMS based on 2017 McDonald criteria\n21. If not newly diagnosed, subjects should have at least 1 documented relapse in the last 24 months.\n22. \"Active disease\" subjects should have at least 1 Gadolinium-enhancing (Gd+) T1-weighted brain or spinal cord lesion at Screening MRI.\n23. \"Disease in remission\" subjects should have no Gd+ T1-weighted brain or spinal cord lesions at Screening MRI and stable clinical symptoms for at least 3 months prior to Day 1.\n24. EDSS score between 2.0 and 5.5 inclusive at Screening\n\n    Inclusion criteria specific to subjects with progressive MS:\n25. Diagnosis of PPMS or SPMS based on 2017 McDonald criteria\n26. EDSS score between 3.0 and 6.5 inclusive at Screening\n27. No evidence of relapse in the prior 6 months\n28. Neurological exam and symptom stability for \u226530 days prior to Day 1\n29. Documented evidence of disability progression in the past 24 months not temporally related to a relapse\n\n    Inclusion Criteria Specific to PD Subjects:\n30. Adult (Age 55 to 80, inclusive) at the Screening Visit\n31. Diagnosis of definite, idiopathic Parkinson's disease according to UK Parkinson's Society Brain Bank diagnostic criteria\n32. PD medication changes within 30 days prior to the Screening Visit should be discussed with the Medical Monitor.\n\nOverall Exclusion Criteria - For All Subjects:\n\nSubjects meeting any of the following criteria will be excluded from this study:\n\n1. Body weight \\>120 kg\n2. Evidence of clinically significant or past medical history of hematologic, renal, endocrine, pulmonary, cardiac, gastrointestinal, hepatic, psychiatric, neurologic, immunologic, allergic disease (including multiple or clinically significant drug allergies) or any other condition that, in the opinion of the Investigator, might significantly interfere with the absorption, distribution, metabolism or excretion of study drug or place the subject at an unacceptable risk as a participant in this study\n3. History of recurrent kidney or liver malignancy\n4. Pacemaker or defibrillator or any non-removable metallic foreign objects in the body not compatible with MRI\n5. Inability to lie in a PET/CT or PET/MRI scanner for up to 90 minutes at a time\n6. Laboratory results (serum chemistry, hematology, coagulation and urinalysis) outside the normal range at Screening and Check-In and considered clinically significant in the opinion of the Investigator. Any elevation of aspartate transaminase (AST) and alanine transaminase (ALT) more than 3 times above the upper limit of normal at screening and/or check-in is exclusionary. One retest of an exclusionary laboratory result is allowed at the discretion of the Investigator and with approval from the Medical Monitor.\n7. Resolved acute illness considered clinically significant by the Investigator within 10 days prior to Screening\n8. History of alcoholism or drug abuse within 2 years prior to Screening. No cannabinoid drug use for at least 10 days prior to Day 1.\n9. Positive urine drug test, marijuana test or cotinine test at Screening or Check-In\n10. Any immunizations within the 28 days prior to screening\n11. Received any other investigational medicinal product within 30 days or 5 half-lives (whichever is longer) prior to Day 1\n12. Corticosteroid treatment (e.g., prednisone, solumedrol) within 30 days of Baseline\n13. Treatment with any of the following classes of nonsteroidal anti-inflammatory drugs (NSAIDS): carboxylic acids, enolic acids, cyclooxygenase (COX) II inhibitors within 14 days of Day 1\n14. Lost or donated \\>450 mL of whole blood or blood products within 30 days prior to Screening\n15. MRI exclusion criteria: findings that may interfere with interpretation of the PET imaging, including but not limited to significant cortical/subcortical cerebrovascular disease, infectious disease, space-occupying lesions, hydrocephalus or other abnormalities associated with CNS disease not related to ALS, AD, MS or PD\n16. CT exclusion criteria include any medical device or metallic implant that may interfere with image acquisition or affect image reconstruction (e.g., CT attenuation correction).\n17. Investigator has reason to believe that the subject may be unable to fulfill the protocol visit schedule or requirements\n18. Has any finding that, in the view of the Investigator and Medical Monitor, would compromise the subject's safety in the trial\n\n    Exclusion Criteria Specific to MS Subjects:\n19. Clinical signs or laboratory findings suggestive of neuromyelitis optica (NMO) spectrum disorders or myelin oligodendrocyte glycoprotein (MOG)-associated encephalomyelitis (i.e., presence of anti-NMO \\[aquaporin-4\\] antibodies or anti-MOG antibodies)\n20. Diagnosis of progressive multifocal leukoencephalopathy (PML)\n\n    Exclusion Criteria Specific to PD Subjects:\n21. Secondary, atypical, or genetic parkinsonism\n\n    Exclusion Criteria Specific to HV Subjects:\n22. Clinically relevant finding on physical examination at Screening\n23. Family history of neurological disease that may confound interpretation of imaging results\n24. History of any central nervous system disorder or brain trauma that could cause imaging abnormalities in the opinion of the Principal Investigator and Medical Monitor", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "18F-OP-801", "targeting_mechanism": "Biomarker for PET imaging of activated microglia and macrophages in regions of neuroinflammation", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02460679", "title": "Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "PTC Therapeutics", "summary": "This is an open label study with 30-day run in phase to establish baseline parameters, 90-day treatment phase, and a 90-day withdrawal phase to determine long-term effects, duration of treatment response, and potential effects of EPI-589 therapy on known trajectory.", "interventions": [{"type": "DRUG", "name": "EPI-589"}], "start_date": "2016-01-14", "url": "https://clinicaltrials.gov/study/NCT02460679", "target_entities": ["TARDBP"], "locations": [{"facility": "Cedar's Sinai", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Providence Brain and Spine Institute ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of possible, probable, laboratory supported probable, or definite ALS by E1 Escorial Criteria\n* Forced vital capacity (FVC) \u2265 70% of predicted\n* Weakness onset within 3 years\n* Agreement to use contraception if within reproductive years\n* Willingness and ability to comply with study procedures\n* Stable regimen of dietary supplements and /or riluzole for at least 30 days prior to enrollment\n* Abstention from use of other investigative or non-approved drugs\n* Participants must be able to swallow 0.375 \\* 0.700 inch tablets\n\nExclusion Criteria:\n\n* Allergy to EPI-589\n* Use of ventilation\n* Participation in other intervention studies\n* Diagnosis of any other neurologic disease\n* Malignancy within the past 2 years\n* History of stroke\n* History of brain surgery\n* Hepatic insufficiency with liver function tests (LFTs) greater than 3 times upper limit of normal (ULN)\n* Renal insufficiency requiring dialysis\n* End stage cardiac failure\n* Participation in a trial of a device, drug, or other therapy for ALS within 3 months of screening or during the trial", "sex": "ALL", "min_age": "21 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "EPI-589", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03945279", "title": "A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BIIB100 Administered Orally to Adults With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogen", "summary": "The primary objective of this study is to evaluate the safety, tolerability of single-ascending doses of BIIB100 in adults with amyotrophic lateral sclerosis (ALS). The secondary objective of the study is to characterize the pharmacokinetic profile of BIIB100.", "interventions": [{"type": "DRUG", "name": "BIIB100"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2019-05-30", "url": "https://clinicaltrials.gov/study/NCT03945279", "target_entities": ["TBK1"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California San Diego Medical Center", "city": "San Diego", "state": "California", "country": "United States", "status": "", "lat": 32.71571, "lon": -117.16472}, {"facility": "Mayo Clinic Hospital", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Johns Hopkins University, Dept of Neurology", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Research Site", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Research Site", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Research Site", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Alliance for Multispecialty Research NOCCR/VRG", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria.\n* Participants taking concomitant riluzole at study entry must be on a stable dose for greater than or equals to (\\>=) 30 days prior to the first dose of study treatment (Day 1). Participants taking concomitant riluzole must be willing to continue with the same dose regimen throughout the study, unless the Investigator determines that riluzole should be discontinued for medical reasons, in which case it may not be restarted during the study.\n* Participants taking concomitant edaravone at study entry must be on a stable dose for \\>= 60 days prior to the first dose of study treatment (Day 1).\n* Adequate respiratory function as indicated by slow vital capacity (SVC) \\>= 65% of predicted value as adjusted for sex, age, and height (from the sitting position).\n\nKey Exclusion Criteria:\n\n* Ongoing medical condition (e.g., wasting or cachexia, severe anemia) that would, in the opinion of the Investigator, interfere with the conduct or assessments of the study.\n* Significant cognitive impairment or unstable psychiatric illness, including psychosis, suicidal ideation, suicide attempt, or untreated major depression less than or equals to (\\<=) 90 days of Screening, which in the opinion of the Investigator would interfere with the study procedures.\n* Treatment with drugs that are transported by Breast Cancer Resistance Protein (BCRP) and P-glycoprotein (P-gp) including, but not limited to, rosuvastatin, sulfasalazine, dabigatran, digoxin and fexofenadine.\n* Current enrollment or plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 30 days or 5 half-lives of the agent, whichever is longer, prior to the Baseline Visit (pre-dose on Day 1). Participation in a noninterventional study focused on ALS natural history may be allowed at the discretion of the Investigator and after consultation with the Sponsor.\n\nNote: Other protocol defined Inclusion/Exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "BIIB100", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04944784", "title": "A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "The purpose of this study is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.", "interventions": [{"type": "DRUG", "name": "Reldesemtiv"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-08-16", "url": "https://clinicaltrials.gov/study/NCT04944784", "target_entities": ["neuromuscular_junction_function"], "locations": [{"facility": "St. Joseph's Hospital & Medical Center - Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Irvine - ALS & Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center - Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Stanford Hospital and Clinics", "city": "Stanford", "state": "California", "country": "United States", "status": "", "lat": 37.42411, "lon": -122.16608}, {"facility": "University of Colorado Hospital Anschutz Outpatient Pavilion", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "GW Medical Faculty Associates", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "University of Florida Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida - Carol and Frank Morsani Center for Advanced Health Care", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Duchossois Center for Advanced Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Indiana University", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "The University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins Outpatient Center", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital - Neurological Clinical Research Institute", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Memorial Medical Center/Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Michigan Medicine", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Health System", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Washington University School of Medicine - Center for Advance Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates, PC", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Rochester Medical Center", "city": "Rochester", "state": "New York", "country": "United States", "status": "", "lat": 43.15478, "lon": -77.61556}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Atrium Health Neuroscience Institute - Charlotte", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Oregon Health and Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Health Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Lewis Katz School of Medicine at Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Vanderbilt University Medical Center - Clinical Research Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology, P.A.", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Vermont Medical Center", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "VCU Neuroscience Orthopaedic and Wellness Center (NOW)", "city": "Henrico", "state": "Virginia", "country": "United States", "status": "", "lat": 36.59264, "lon": -78.61611}, {"facility": "Froedtert Hospital", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "Brain and Mind Centre", "city": "Camperdown", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.88965, "lon": 151.17642}, {"facility": "Concord Repatriation General Hospital", "city": "Concord", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.84722, "lon": 151.10381}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Flinders Medical Centre", "city": "Bedford Park", "state": "South Australia", "country": "Australia", "status": "", "lat": -35.02204, "lon": 138.56815}, {"facility": "The Perron Institute", "city": "Nedlands", "state": "Western Australia", "country": "Australia", "status": "", "lat": -31.98184, "lon": 115.8073}, {"facility": "UZ Leuven Gasthuisberg, Department of Neurology", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "University of Calgary - Heritage Medical Research Clinic", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "McMaster University Medical Centre", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Ottawa Hospital Research Institute - Civic Campus", "city": "Ottawa", "state": "Ontario", "country": "Canada", "status": "", "lat": 45.41117, "lon": -75.69812}, {"facility": "Sunnybrook Research Institute", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Centre de recherche du CHUM", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "McGill University, Montreal Neurological Institute & Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "CHU de Quebec-Universit\u00e9 Laval", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Saskatoon City Hospital", "city": "Saskatoon", "state": "Saskatchewan", "country": "Canada", "status": "", "lat": 52.13238, "lon": -106.66892}, {"facility": "Deparment of Neurology Bispebjerg University Hospital", "city": "Copenhagen", "state": "", "country": "Denmark", "status": "", "lat": 55.67594, "lon": 12.56553}, {"facility": "CRC SLA de Lyon", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "CHRU de Lille Hopital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges - Hopital Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "CHU de la Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHU de Nice - H\u00f4pital Pasteur 2", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Hopital La Pitie Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "CHRU de Tours, Hopital Bretonneau, Clinical Research Center", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Charit\u00e9 - Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Universitatsklinikum Bonn", "city": "Bonn", "state": "", "country": "Germany", "status": "", "lat": 50.73438, "lon": 7.09549}, {"facility": "Medical School Hannover - Department of Neurology", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universitatsklinikum Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Universit\u00e4tsklinikum Schleswig Holstein", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "", "lat": 53.86893, "lon": 10.68729}, {"facility": "Universitatsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "RSCI Education and Research Centre, Beaumont Hospital", "city": "Beaumont", "state": "Dublin", "country": "Ireland", "status": "", "lat": 53.38721, "lon": -6.22713}, {"facility": "Ospedale San Luca", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Centro Clinical Nemo - Fondazione Serena Onlus", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Instituti Clinici Scientifici Maugeri", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "AOU Citt\u00e0 della Salute e Scienza (Molinette),", "city": "Turin", "state": "", "country": "Italy", "status": "", "lat": 45.07049, "lon": 7.68682}, {"facility": "UMC Utrecht, Department of Neurology, ALS Center", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "City Clinic Research", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Centro Hospitalar Universitario Lisboa Norte, Department of Neurology", "city": "Lisbon", "state": "", "country": "Portugal", "status": "", "lat": 38.72509, "lon": -9.1498}, {"facility": "Hospital Universitari de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario Basurto", "city": "Bilbao", "state": "", "country": "Spain", "status": "", "lat": 43.26271, "lon": -2.92528}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Universitari i Politecnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Neurologimottagningen Skane University Hospital", "city": "Malm\u00f6", "state": "", "country": "Sweden", "status": "", "lat": 55.60587, "lon": 13.00073}, {"facility": "Studieenheten Akademiskt Specialistcentrum, Sabbatsberg Hospital", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Muskelzentrum/ALS Clinic", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "The Walton Centre NHS Foundation Trust", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "Maurice Wohl Clinical Neuroscience Institute", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Males or Females between the ages of 18 and 80 years of age, inclusive\n* Diagnosis of familial or sporadic ALS (defined as meeting the laboratory-supported probable, probable, or definite criteria for ALS according to the World Federation of Neurology El Escorial criteria). Patients who meet the possible criteria are eligible if they have lower motor neuron findings; those who have purely upper motor neuron findings are ineligible.\n* First symptom of ALS \u2264 24 months prior to screening. The qualifying first symptoms of ALS are limited to manifestations of weakness in extremity, bulbar, or respiratory muscles.\n* ALSFRS-R total score \u2264 44 at screening. Patients with a total score of 45 or higher may be rescreened 60\u00b17 days following the original screening date.\n* Upright FVC \u2265 65.0% of predicted for age, height, sex and ethnicity at screening according to Global Lung Initiative equation\n* Must be either on riluzole for \u2265 30 days prior to screening or have not taken it for at least 30 days prior to screening\n* Must have completed at least 2 cycles of edaravone at the time of screening or have not received it for at least 30 days prior to screening\n* Able to swallow whole tablets\n\nExclusion Criteria:\n\n* eGFRCysC \\< 45.0 mL/min/1.73 m2 at screening\n* Urine protein/creatinine ratio \\> 1 mg/mg (113 mg/mmol) at screening\n* Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \u2265 3-times the upper limit of normal (ULN)\n* Total bilirubin (TBL), direct or indirect bilirubin above the ULN.\n* Cognitive impairment, related to ALS or otherwise that impairs the patient's ability to understand and/or comply with study procedures and provide informed consent\n* Other medically significant neurological conditions that could interfere with the assessment of ALS symptoms, signs or progression.\n* Has a tracheostomy", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Reldesemtiv", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03651349", "title": "To Determine the Maximum Tolerated Dose (MTD) of HK-001 in Healthy Volunteers", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Everfront Biotech Co., Ltd.", "summary": "Eligible subjects will receive either different dosages of HK-001 or placebo in a 3:1 ratio in 1 of the 7 dose cohorts. After single dose administration, followed by an independent Data and Safety Monitoring Board (DSMB) meeting for safety assessments (including the available plasma pharmacokinetic profile), the subjects will be allowed to receive (Z)-BP or placebo twice a day orally at the study site for 14 consecutive days and follow up on the 28th day after the last dose administration by a site visit. The study drugs (including placebo) will be administered at the study site by following the investigator's instructions to either perform blood sampling for pharmacokinetic evaluation or maximize the treatment compliance.\n\nThere will be 7 cohorts and subjects will be randomized into cohorts consisting of 8 subjects each (6 active and 2 placebo controls per cohort). Dose cohorts will be escalated sequentially from low to high dose (50 mg, BID; 100 mg, BID; 150 mg, BID; 225 mg, BID; 300 mg, BID; 400 mg, BID; 525 mg, BID) by following a modified Fibonacci sequence, and based on the decision of an independent DSMB at a set time point. Following all subjects of a cohort complete the safety and PK evaluation after receiving the last dose administration, a cohort at the next dose level will be launched if the DSMB does not identify significant safety concerns after reviewing safety data and PK profiles.", "interventions": [{"type": "DRUG", "name": "HK-001"}, {"type": "DRUG", "name": "Placebo control"}], "start_date": "2021-02-01", "url": "https://clinicaltrials.gov/study/NCT03651349", "target_entities": [], "locations": [{"facility": "Hualien Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation", "city": "Hualien City", "state": "", "country": "Taiwan", "status": "", "lat": 23.97694, "lon": 121.60444}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Subject's age is no less than 20 years old.\n2. Subjects whose body mass index (BMI) at screening is within a range of \u226518.5 kg/m2 and \\<25.0 kg/m2.\n\n   BMI = Body Weight (kg) / \\[Height (m)\\]2 And body weight is not less than 50 kg and 45 kg for males and females, respectively.\n3. Subject's medical history shows no contraindication to the test medications \\[hypersensitivity to (Z)-BP or any component of test and reference products\\].\n4. Subjects who are judged to be in good health by the investigator based upon the results of physical examinations (PEs) and chest X-ray (within 60 days prior to the first study dose), and all items of routine laboratory tests, including serum biochemistry, hematology and urinalysis, are within normal range as judged by the site. Assessment items of blood biochemistry include electrolytes (sodium, potassium, chloride, calcium, phosphorus), albumin, total cholesterol, total bilirubin, ALP, SGOT, SGPT, GGT, BUN, PT, APPT, serum creatinine, triglyceride, glucose, amylase, lipase, ACTH, aldosterone, cortisol, T3, free T4, TSH, and uric acid. Assessment items of hematology tests include red blood cell (RBC), white blood cell (WBC) and platelet count; differential WBC count including neutrophils, lymphocytes, monocytes, eosinophils, and basophils; hemoglobin, and hematocrit. Assessment items of urinalysis include appearance, gravity, pH, erythrocyte, leukocyte, epithelial cells, glucose, protein, ketones, and nitrite\n5. Female subjects show negative pregnancy test results within 30 days prior to the first study dose.\n6. Subjects did not take any of the following medications in the specified durations:\n\n   * Any medication (except contraceptives) within 14 days prior to the first dose of the study\n   * Any enzyme inducer or inhibitor within 30 days prior to the first dose of the study\n7. Subjects understood and have signed the written informed consent form.\n\nExclusion Criteria:\n\n1. Subjects with any properly diagnosed disease within 30 days prior to the first dose of the study\n2. Subjects with a clinically significant hematological, endocrinal, cardiovascular, hepatic, renal, gastrointestinal, and/or pulmonary disorders; subjects with any predisposing condition that might interfere with the absorption, distribution, metabolism and excretion of drugs; subjects who have had any previous gastrointestinal surgery, except appendectomy if performed \\>90 days prior to the first dose of the study\n3. Subjects who have received any known hepatic or renal clearance-altering agents (e.g., erythromycin, cimetidine, barbiturates, phenothiazine, clarithromycin, trolearndomycin, ketoconazole, miconazolem fluconazole, itraconazole) for a period of up to 30 days prior to the first dose of the study\n4. Subjects had participated in investigational drug trials and took any investigational drugs within 60 days prior to the first study dose.\n5. Subjects had blood donation for more than 250 mL within 60 days prior to the first dose of the study.\n6. Subjects had a history of drug abuse or alcohol abuse according to DSM IV criteria.\n7. Subjects who are smokers or have smoking history\n8. Subjects who cannot stop caffeine-intakes for 48 hours prior to the first study dose and during the entire study period.\n9. Subjects who are pregnant or lactating\n10. For enrollment of female subjects with child-bearing potential, the subject must be practicing sexual abstinence or be using and willing to continue to use a medically acceptable form of birth control for at least 1 month prior to screening (that period will extend to 3 months for oral contraceptive use) and for at least 30 days after the last dose of study drug. For a subject to be considered not to be of child-bearing potential, she must have been amenorrheic for at least 2 years, or must have had a hysterectomy, a bilateral tubal ligation, and/or a bilateral oophorectomy (as determined by the medical history). The male partner of a female study subject with childbearing potential must use a condom and ensure that his partner uses a suitable method of contraception as outlined above.\n11. Subjects who are inappropriate to participate in this study, as judged by the clinical investigator\n12. Subjects who have been tested positive for the following tests:\n\n    * Human immunodeficiency virus (HIV)\n    * Hepatitis B virus (HBV)\n    * Hepatitis C virus (HCV)", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "HK-001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05105958", "title": "Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Zurich", "summary": "Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative condition, mainly characterized by progressive weakness and wasting of the limbs, the respiratory and bulbar muscles. Respiratory insufficiency leads to a fatal outcome after a mean diseases duration of only three to five years. The disease is characterized by pathological accumulations of a protein called TDP-43, which can be found large cortical and sub-cortical areas of post-mortem ALS brains.\n\nNo causal treatment for this condition is known to date, and there is a large unmet need to develop new strategies in order to halt or slow down its progression.\n\nThe aim of this study is to test the safety and tolerability of Tideglusib, a treatment that is already in clinical trials for other neuromuscular conditions, in patients with ALS. It is assumed that this drug may have a significant therapeutic benefit in this population due to his mode of action: In the ALS mouse model, Tideglusib decreases significantly the amount of accumulated TDP-43 proteins within the cells.", "interventions": [{"type": "DRUG", "name": "Tideglusib"}], "start_date": "2025-12-01", "url": "https://clinicaltrials.gov/study/NCT05105958", "target_entities": ["TARDBP", "GSK3"], "locations": [{"facility": "University Hospital Bern", "city": "Bern", "state": "", "country": "Switzerland", "status": "", "lat": 46.94809, "lon": 7.44744}, {"facility": "University Hospital Geneva", "city": "Geneva", "state": "", "country": "Switzerland", "status": "", "lat": 46.20222, "lon": 6.14569}, {"facility": "University Hospital Lausanne", "city": "Lausanne", "state": "", "country": "Switzerland", "status": "", "lat": 46.516, "lon": 6.63282}, {"facility": "Kantonsspital St. Gallen", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "University Hospital Zurich", "city": "Zurich", "state": "", "country": "Switzerland", "status": "", "lat": 47.36667, "lon": 8.55}], "contact_phone": "0795531171", "contact_email": "annemarie.hubers@hcuge.ch", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Possible, probable (clinically or laboratory supported) or definite ALS according to the revised version of the El Escorial criteria\n* Disease duration \\< 18 months\n* Vital capacity of more than 60% of normal (defined as slow vital capacity, best of three measurements)\n* Age more than 18 years\n* On a stable dose of riluzole for at least four weeks or not taking riluzole\n* On a stable dose of edaravone for at least four weeks or not taking edaravone\n* Capable of thoroughly understanding all information given and giving full informed consent according to GCP\n\nExclusion Criteria:\n\n* Previous participation in another clinical study within the preceding 12 weeks\n* Proven SOD1- or FUS - mutation\n* Tracheostomy or assisted ventilation of any type during the preceding three months\n* Pregnancy or breast-feeding females\n* Any medical condition known to have an association with motor neuron dysfunction which might confound or obscure the diagnosis of ALS\n* Presence of any concomitant life-threatening disease or impairment likely to interfere with functional assessment\n* Evidence of a major psychiatric disorder or clinically evident dementia precluding evaluation of symptoms\n* Alcoholism\n* Cardiovascular disorder/arrhythmia\n* Impaired kidney function, defined as creatinine levels of 2.5 x upper limit of normal (ULN)\n* Impaired liver function, defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) of 3 x ULN\n* Liable to be not cooperative or comply with trial requirements as assessed by the investigator, or unable to be reached in the case of emergency", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tideglusib", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04651855", "title": "The Evaluation of the Effect of Mesenchymal Stem Cells on the Immune System of Patients With ALS", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Polski Bank Komorek Macierzystych JSC (PBKM)", "summary": "The objective of this study is to evaluate the safety of intrathecal administration of Wharton's Jelly Mesenchymal Stem Cells (WJMSC) and the impact on the immune system of patients with Amyotrophic Lateral Sclerosis.", "interventions": [{"type": "DRUG", "name": "Mesenchymal stem cells isolated from Wharton's jelly"}], "start_date": "2020-12-02", "url": "https://clinicaltrials.gov/study/NCT04651855", "target_entities": ["neuroinflammation"], "locations": [{"facility": "JST sp. z o.o.", "city": "Cz\u0119stochowa", "state": "", "country": "Poland", "status": "", "lat": 50.79646, "lon": 19.12409}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Adult patients (at least 18 years old)\n2. The minimum patient's weight is not less than 40 kg\n3. Diagnosis of sporadic ALS, definite or probable, as defined by El Escorial World Federation of Neurology criteria\n4. History of ALS symptoms less than 2 years duration from the first symptoms of the disease\n5. More than 6 months from diagnosis of the disease\n6. Disease progression at 6 past months at least 3 points during this period of time assessed in ALSFRS-R scale\n7. ALSFRS-R scale of at least 30 at screening appointment\n8. Forced vital capacity \\>70% of predicted value for age, gender and height\n9. Treatment with stable dose of riluzole(2x 50mg per 24h) before baseline visit (for at least 1 month)\n10. Capable of providing written informed consent\n11. Able to comply with study requirements and willing to follow all study procedures and follow-up visits\n12. Women of child-bearing age and men with partners of child-bearing potential must agree to use two forms of contraceptive therapy throughout the course of the trial\n13. Women of child-bearing age must undergo pregnancy test\n14. Polish-language native speakers or patients who are proficient in the Polish language\n\nExclusion Criteria:\n\n1. Pregnancy or breastfeeding\n2. Tracheostomy\n3. Ventilator dependence\n4. Renal disease with creatinine \\>2mg/dl\n5. Liver disease with ALT, AST or GGTP 2-fold higher than upper normal limit\n6. Positive test for HBV, HCV, HIV with NAT method\n7. Positive tests for syphilis\n8. Any other clinically significant abnormalities on laboratory evaluation\n9. Any condition that would compromise ability of undergoing lumbar puncture\n10. Active systemic disease\n11. Autoimmune disease (Hashimoto disease under control is allowed)\n12. Uncontrolled diabetes (HbA1c \\> 8%)\n13. Pulmonary disease that could affect interpretation of spirometry\n14. Neurological concomitant disease\n15. Unstable psychiatric concomitant disease\n16. High risk of suicide\n17. History of substance abuse within past year\n18. History of malignancy, within the previous 5 years, including melanoma with exception of localized skin cancers\n19. Any other clinically significant medical condition that can compromise patient's safety in the opinion of the investigator\n20. Treatment with immunomodulatory drugs (for example immunoglobulins, corticosteroids or other immunosuppressant) in last 6 months\n21. Participation in another clinical trial in last 6 months\n22. Previous cellular therapy of any kind\n23. Hypersensitivity to any component used in the cell culture\n24. Nuchal rigidity and other signs of meningitis\n25. Patients on chronic anticoagulation treatment (heparin/ warfarin/acenocoumarol/(N)OAC)", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Mesenchymal stem cells isolated from Wharton's jelly", "targeting_mechanism": "Intrathecal-delivered stem cells differentiate into support cells and produce growth factors and anti-inflammatory cytokines to modulate neuroinflammation and benefit degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07221240", "title": "Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Amisodin in Healthy Adult Subjects With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "PRG Science & Technology Co., Ltd.", "summary": "Researchers will evaluate the safety, tolerability, and pharmacokinetics (PK) of orally administered Amisodin in healthy adult subjects through a randomized, double-blind, placebo-controlled Phase 1 study consisting of two parts: single ascending dose (SAD) and multiple ascending dose (MAD). The food effect will be assessed in one cohort in Part1. Approximately 48 healthy, adult subjects are planned to be enrolled in total. Subjects will participate in only one part and one cohort.", "interventions": [{"type": "DRUG", "name": "Amisodin"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2025-10-01", "url": "https://clinicaltrials.gov/study/NCT07221240", "target_entities": ["NEK1"], "locations": [{"facility": "Pharmaron, Inc.", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "+82 051-583-8703", "contact_email": "claudiajang@kcrnresearch.com", "eligibility": {"criteria": "Inclusion Criteria:\n\nSubjects must fulfill all of the following inclusion criteria to be eligible for participation in the study:\n\n* 1\\. Healthy, adult, male or female (of non-childbearing potential only)\\*, 18 55 years of age, inclusive, at the screening visit.\n\n  * Females of non-childbearing potential are defined as follows:\n\n    * Females who have undergone one of the following sterilization procedures at least 6 months prior to the first dosing:\n\n      o Hysteroscopic sterilization\n\n      o Bilateral tubal ligation or bilateral salpingectomy\n\n      o Hysterectomy\n      * Bilateral oophorectomy or\n    * Females who are postmenopausal with amenorrhea for at least 1 year prior to the first dosing and have follicle-stimulating hormone (FSH) serum levels consistent with postmenopausal status. 2. Male subjects must follow protocol specified contraception guidance as described in Section 7.4.5 Contraception Requirements and agree to refrain from sperm donation until 90 days after the last dosing. 3. Continuous non-smoker who has not used nicotine-containing products for at least 3 months prior to the first dosing based on subject self-reporting.\n\n      4\\. Body mass index (BMI) \u2265 18.0 and \\< 32.0 kg/m2 at the screening visit. 5. Medically healthy with no clinically significant medical history, physical examination, laboratory profiles, or vital signs, as deemed by the PI or designee at the screening visit, including the following:\n    * Seated blood pressure is \u2265 90/50 mmHg and \u2264 140/90 mmHg\n    * Liver function tests at or below limit or normal range\n    * Estimated glomerular filtration rate (estimated by Modification of Diet in Renal Disease Study equation \\[MDRD\\] method) \u2265 90 mL/min/1.73 m\u00b2 6. No ECG findings of clinical significance as judged by the PI or qualified designee at the screening visit and at first check in, including each criterion as listed below:\n    * Normal sinus rhythm (heart rate between 40 and 100 bpm, inclusive)\n    * QTcF interval \u2264 450 msec (males) or \u2264 460 msec (females)\n    * QRS interval \\< 110 msec; if \\> 110 msec, result will be confirmed by a manual over read\n    * PR interval \u2264 210 msec 7. Understands the study procedures in the informed consent form (ICF) and be willing and able to comply with the protocol.\n\nExclusion Criteria:\n\nSubjects must not be enrolled in the study if they meet any of the following criteria:\n\n* 1\\. Is mentally or legally incapacitated or has significant emotional problems at the time of the screening visit or expected during the conduct of the study.\n\n  2\\. History or presence of clinically significant medical or psychiatric condition or disease in the opinion of the PI or designee. 3. History of any illness that, in the opinion of the PI or designee, might confound the results of the study or poses an additional risk to the subject by their participation in the study. 4. History or presence of alcohol or drug abuse within the past 2 years prior to the first dosing. 5. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the study drug, study drug excipients, or antihistamines.\n\n  6\\. Allergy to band aids, adhesive dressing, or medical tape. 7. Female subjects of childbearing potential. 8. Female subject with a positive pregnancy test at the screening visit or at first check-in or who is lactating. 9. Positive urine drug or serum alcohol results at the screening visit or first check-in. 10. Positive results at the screening visit for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV). 11. Unable to refrain from or anticipates the use of:\n\n  \u2022 Any drugs, including prescription and non-prescription medications, herbal remedies, or vitamin supplements beginning 14 days prior to the first dosing.\n\n  \u2022 Any drugs known to be significant inducers of cytochrome P450s (CYP) and/or P-glycoprotein (P-gp), including St. John's Wort, for 28 days prior to the first dosing.\n\n  \u2022 Any drugs that prolong the QT/QTc interval within 14 days (or 5 half lives, whichever is longer) prior to the first dosing.\n\n  12\\. Has been on a diet incompatible with the on-study diet, in the opinion of the PI or designee, within the 30 days prior to the first dosing. 13. Donation of blood or significant blood loss within 56 days prior to the first dosing. 14. Plasma donation within 7 days prior to the first dosing 15. Participation in another clinical study within 90 days prior to the first dosing.\n\nThe 90-day window will be derived from the date of the last blood collection or dosing, whichever is later, in the previous study to Day 1 of the current study.\n\n16\\. Any other reason determined by the PI or designee, in their opinion, that would prevent the subject's participation in the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "55 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "Amisodin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05136222", "title": "Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Melbourne", "summary": "A two-arm, individual participant randomised controlled, assessor-blinded trial in 7 MND care centres across Australia will be undertaken.", "interventions": [{"type": "OTHER", "name": "Intervention polysomnography"}, {"type": "OTHER", "name": "Sham polysomnography"}], "start_date": "2021-12-15", "url": "https://clinicaltrials.gov/study/NCT05136222", "target_entities": ["respiratory_function"], "locations": [{"facility": "Flinders Medical Centre", "city": "Adelaide", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -34.92866, "lon": 138.59863}, {"facility": "The Prince Charles Hospital", "city": "Brisbane", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -27.46794, "lon": 153.02809}, {"facility": "Motor Neurone Disease Australia", "city": "Canberra", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -35.28346, "lon": 149.12807}, {"facility": "Austin Health", "city": "Melbourne", "state": "", "country": "Australia", "status": "RECRUITING", "lat": -37.814, "lon": 144.96332}, {"facility": "Australian MND Registry", "city": "Melbourne", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -37.814, "lon": 144.96332}, {"facility": "FightMND", "city": "Melbourne", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -37.814, "lon": 144.96332}, {"facility": "Institute for Breathing and Sleep", "city": "Melbourne", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -37.814, "lon": 144.96332}, {"facility": "Monash University", "city": "Melbourne", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -37.814, "lon": 144.96332}, {"facility": "University of Melbourne", "city": "Melbourne", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -37.814, "lon": 144.96332}, {"facility": "Sir Charles Gairdner Hospital", "city": "Perth", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -31.95224, "lon": 115.8614}, {"facility": "Macquarie University", "city": "Sydney", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -33.86785, "lon": 151.20732}, {"facility": "Royal Prince Alfred Hospital", "city": "Sydney", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -33.86785, "lon": 151.20732}, {"facility": "Westmead Hospital", "city": "Sydney", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -33.86785, "lon": 151.20732}], "contact_phone": "+613 9496 3871", "contact_email": "david.berlowitz@austin.org.au", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age \\>18 years\n* Clinical indication to commence long term NIV\n* Confirmed clinical diagnosis of underlying condition\n\nExclusion Criteria:\n\n* Medically unstable\n* Hypoventilation attributable to medications with sedative/respiratory depressant side- effects\n* Use of NIV for more than 1 month in the previous 3 months\n* Inability to provide informed consent\n* Previous intolerance of NIV", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03326622", "title": "Exercise and Disease Progression in Amyotrophic Lateral Sclerosis Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Lisbon", "summary": "This study evaluated the influence of a tailored aerobic exercise protocol on the functional outcome in ALS patients. In addition, the investigators compare some CPET variables collected during exercise testing in both groups.", "interventions": [{"type": "OTHER", "name": "standard care"}, {"type": "OTHER", "name": "moderate exercise"}], "start_date": "2013-07-01", "url": "https://clinicaltrials.gov/study/NCT03326622", "target_entities": ["mitochondrial_function"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Consecutive patients diagnosed with definite, probable, or probable laboratory supported ALS\n* Disease duration from first symptoms between 6-24 months to exclude slow and fast progression\n* ALSFRS-R \u2265 30\n* FVC (%predicted) \u2265 70%\n\nExclusion Criteria:\n\n* Other medical conditions, like cardiac insufficiency and lung disorders or others conditions limiting exercise training;\n* Heavy smoking habits with laboratorial evidence of significant bronchial constriction;\n* Signs of associated dementia or psychiatric disorders.\n\nNote: None of the patients were on tube feeding, invasive or non-invasive mechanical ventilation at admission of study protocol (T1).", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01609283", "title": "A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mayo Clinic", "summary": "The purpose of this study is to determine determine the safety of intraspinal delivery of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis (ALS) using a dose-escalation study.", "interventions": [{"type": "BIOLOGICAL", "name": "autologous mesenchymal stem cells"}], "start_date": "2012-05", "url": "https://clinicaltrials.gov/study/NCT01609283", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* All patients must have clinically-defined ALS as defined by the World Federation of Neurology criteria\n* Age greater than 18 years\n* If female, must be post-menopausal or had a hysterectomy\n* Permanent resident or citizen of the United States\n* History of a chronic onset of a progressive motor weakness of greater than one year, but less than two years duration\n* Must have vital capacity greater than 65% of predicated for age, gender, and body type\n* Able to comply with protocol requirements, including MRI testing\n* Can provide written informed consent\n\nExclusion Criteria:\n\n* Any clinically significant medical condition (e.g., within six months of baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure) that, in the opinion of the investigator, would compromise the safety of patient.\n* Autoimmunity, including Crohn's disease, rheumatoid arthritis, psoriasis\n* Malignancy including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline). Any other malignancy will not be allowed.\n* Active systemic or local infection near the lumbar puncture site\n* Other active systemic disease as defined by laboratory abnormalities\n* Use of herbal medications or other unapproved drugs\n* Enrolled in an investigational drug trial within 30 days of baseline visit\n* Kokmen Short Test of Mental Status score \\<32\n* Beck's Depression Inventory score \\>18\n* Presence of a tracheostomy\n* Ventilator dependent", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "autologous mesenchymal stem cells", "targeting_mechanism": "Intraspinal-delivered mesenchymal stem cells differentiate into support cells (astrocytes, oligodendrocytes, microglia) and produce growth factors and anti-inflammatory cytokines to protect degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "ASC-Exosomes derived from adipose-derived mesenchymal stem cells ameliorated disease progression in the SOD1(G93A) murine ALS model.", "animal_results_pmid": "32455791", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04868994", "title": "Magnetic Imaging for Diagnostic of Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Toulouse", "summary": "Nearly 60% of Amyotrophic Lateral Sclerosis (ALS) patients have a low level of diagnostic certainty (possible, probable) at the time of diagnosis. In the absence of biomarkers, this diagnosis is based, among other things, on the demonstration of the diffusion of signs of denervation by electroneuromyography (ENMG). The objective of this study is to improve the earliness and the level of diagnostic certainty by better demonstrating the diffusion of the denervation process by whole body muscular MRI.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "Whole Body MRI and ENMG"}], "start_date": "2021-06-01", "url": "https://clinicaltrials.gov/study/NCT04868994", "target_entities": [], "locations": [{"facility": "Pascal CINTAS", "city": "Toulouse", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.60426, "lon": 1.44367}], "contact_phone": "05 61 77 94 40", "contact_email": "cintas.p@chu-toulouse.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Consent form signed by the patient\n* Patients suspected ALS defined according Awaji Shima criteria (possible, probable, defined)\n* Clinical assessment of upper motor neuron involvement\n* Electrophysiologic assessment of lower motor neuron involvement\n\nExclusion Criteria:\n\n* inability to give informed consent\n* a contraindication to MRI\n* respiratory failure impairing ability to lie flat in the scanner.\n* Patient placed under judicial protection or under another protective regime,\n* Females who are pregnant", "sex": "ALL", "min_age": "18 Years", "max_age": "99 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03757351", "title": "Study to Evaluate DNL747 in Subjects With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sanofi", "summary": "The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple oral doses of DNL747 in subjects with Amyotrophic Lateral Sclerosis in a cross-over design", "interventions": [{"type": "DRUG", "name": "DNL747"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2018-12-14", "url": "https://clinicaltrials.gov/study/NCT03757351", "target_entities": ["dnl747"], "locations": [{"facility": "Bioclinica", "city": "Orlando", "state": "Florida", "country": "United States", "status": "", "lat": 28.53834, "lon": -81.37924}, {"facility": "PRA Health Sciences", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "CHDR", "city": "Leiden", "state": "South Holland", "country": "Netherlands", "status": "", "lat": 52.15833, "lon": 4.49306}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria (Double-Blind Part):\n\n* Women of non-childbearing potential and men, aged 21-80 years\n* Willingness and ability to complete all aspects of the study; participant should be capable of completing assessments either alone or with help of a caregiver\n* Diagnosis of laboratory-supported probable, probable, or definite (sporadic or familial) ALS according to the El Escorial World Federation of Neurology revised research diagnostic criteria\n* Less than 3 years since symptom onset\n* Forced vital capacity (FVC) \\>50% predicted measured within 30 days of screening\n* If subject is taking approved ALS treatments (riluzole and/or edaravone), doses must be stable for \u22652 months prior to screening and subject is expected to stay on a stable regimen throughout the study\n\nKey Exclusion Criteria (Double-Blind Part):\n\n* History of a clinically significant non-ALS neurologic disorder (other than frontal temporal lobe dementia), including, but not limited to, muscular dystrophy, spinal stenosis, peripheral neuropathy, inherited neuropathies, AD, Parkinson's disease, Lewy body dementia, vascular dementia, Huntington's disease, epilepsy, stroke, multiple sclerosis, brain tumor, or brain infection or abscess\n* Unstable or poorly controlled comorbid disease process of any organ system currently requiring active treatment or likely to require treatment adjustment during the study\n\nKey Inclusion Criteria (Open-Label Extension):\n\n* Successful completion of both periods of the the double-blind, crossover part of the study\n* Continued diagnosis of laboratory-supported probable, probable, or definite (sporadic or familial) ALS according to the El Escorial World Federation of Neurology revised research diagnostic criteria\n\nKey Exclusion Criteria (Open-Label Extension):\n\n* Presence of laboratory abnormalities, physical examination findings, or AEs determined to be clinically significant by the investigator from the double-blind part of the study that have not resolved by the final follow-up visit as part of the double-blind study period\n* New diagnosis of clinically significant neurological disorder (other than frontal temporal lobe dementia)", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "DNL747", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01119001", "title": "A P300 Brain Computer Interface Keyboard to Control Assistive Technology For Use by People With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "People with Amyotrophic Lateral Sclerosis (ALS) will use a P300 based brain computer interface (BCI) keyboard to type in assistive technology devices. The results of this study will be compared with a previous study of a P300 BCI keyboard used by healthy volunteers.", "interventions": [{"type": "DEVICE", "name": "P300 Brain Computer Interface Keyboard"}], "start_date": "2010-02", "url": "https://clinicaltrials.gov/study/NCT01119001", "target_entities": [], "locations": [{"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age 18 or older.\n* Diagnosed with cerebral palsy, spinal cord injury, neuromuscular disease, or ALS that results in impaired hand and arm function making it difficult to manipulate objects (if at all) and requires help to prepare or modify activities\n* Able to see the BCI display\n* Able to give informed consent\n* Able to understand and remember instructions concerning participation\n* Able to communicate effectively at least with familiar conversation partners\n\nExclusion Criteria:\n\n* Are unable to give informed consent.\n* Are currently experiencing open head sores\n* Have a history of photo-sensitive epilepsy\n* Have a history of cognitive deficits requiring accommodation or special education services\n* Are unable to sit without moving the head and neck for at least 15 minutes", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04468919", "title": "Optimizing BCI-FIT: Brain Computer Interface - Functional Implementation Toolkit", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Oregon Health and Science University", "summary": "This project adds to non-invasive BCIs for communication for adults with severe speech and physical impairments due to neurodegenerative diseases. Researchers will optimize \\& adapt BCI signal acquisition, signal processing, natural language processing, \\& clinical implementation. BCI-FIT relies on active inference and transfer learning to customize a completely adaptive intent estimation classifier to each user's multi-modality signals simultaneously. 3 specific aims are: 1. develop \\& evaluate methods for on-line \\& robust adaptation of multi-modal signal models to infer user intent; 2. develop \\& evaluate methods for efficient user intent inference through active querying, and 3. integrate partner \\& environment-supported language interaction \\& letter/word supplementation as input modality. The same 4 dependent variables are measured in each SA: typing speed, typing accuracy, information transfer rate (ITR), \\& user experience (UX) feedback. Four alternating-treatments single case experimental research designs will test hypotheses about optimizing user performance and technology performance for each aim.Tasks include copy-spelling with BCI-FIT to explore the effects of multi-modal access method configurations (SA1.3a), adaptive signal modeling (SA1.3b), \\& active querying (SA2.2), and story retell to examine the effects of language model enhancements. Five people with SSPI will be recruited for each study. Control participants will be recruited for experiments in SA2.2 and SA3.4. Study hypotheses are: (SA1.3a) A customized BCI-FIT configuration based on multi-modal input will improve typing accuracy on a copy-spelling task compared to the standard P300 matrix speller. (SA1.3b) Adaptive signal modeling will allow people with SSPI to typing accurately during a copy-spelling task with BCI-FIT without training a new model before each use. (SA2.2) Either of two methods of adaptive querying will improve BCI-FIT typing accuracy for users with mediocre AUC scores. (SA3.4) Language model enhancements, including a combination of partner and environmental input and word completion during typing, will improve typing performance with BCI-FIT, as measured by ITR during a story-retell task. Optimized recommendations for a multi-modal BCI for each end user will be established, based on an innovative combination of clinical expertise, user feedback, customized multi-modal sensor fusion, and reinforcement learning.", "interventions": [{"type": "BEHAVIORAL", "name": "BCI-FIT multi-modal access"}, {"type": "BEHAVIORAL", "name": "BCI-FIT adaptive signal modeling"}, {"type": "BEHAVIORAL", "name": "BCI-FIT active querying"}, {"type": "BEHAVIORAL", "name": "BCI-FIT language modeling"}], "start_date": "2022-07-15", "url": "https://clinicaltrials.gov/study/NCT04468919", "target_entities": [], "locations": [{"facility": "Oregon Health & Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nControls\n\n* Able to read and communicate in English\n* Capable of participating in study visits lasting 1-3 hours\n* Adequate visuospatial skills to select letters, words, or icons to copy or generate messages\n* Live within a 2-hour drive of OHSU or is willing to travel to OHSU\n\nParticipants with severe speech and physical impairment:\n\n* Adults between 18-89 years of age\n* SSPI that may result from a variety of degenerative or neurodevelopmental conditions, including but not limited to: Duchenne muscular dystrophy, Rett Syndrome, ALS, brainstem CVA, SCI, and Parkinson-plus disorders (MSA, PSP)\n\n  * Able to read and communicate in English with speech or AAC device\n  * Capable of participating in study visits lasting 1-3 hours\n* Adequate visuospatial skills to select letters, words or icons to copy or generate basic messages\n* Life expectancy greater than 6 months\n* Able to give informed consent or assent according to IRB approved policy\n\nExclusion Criteria:\n\n* Participants with severe speech and physical impairment:\n\n  * Unstable medical conditions (fluctuating health status resulting in multiple hospitalizations within a 6 week interval)\n\n    * Unable to tolerate weekly data collection visits\n    * Photosensitive seizure disorder\n    * Presence of implanted hydrocephalus shunt, cochlear implant or deep brain stimulator\n    * High risk of skin breakdown from contact with data acquisition hardware.", "sex": "ALL", "min_age": "18 Years", "max_age": "89 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07396818", "title": "Kamlanoflast In Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Inflammasome Therapeutics", "summary": "This is a study of Kamlanoflast in patients with ALS. Kamlanoflast is orally administered over 24 weeks. Its effects on inflammatory and functional parameters will be studied. Information on safety and tolerability will be collected.", "interventions": [{"type": "DRUG", "name": "Kamlanoflast"}, {"type": "DRUG", "name": "Kamlanoflast"}], "start_date": "2026-02", "url": "https://clinicaltrials.gov/study/NCT07396818", "target_entities": ["kamlanoflast"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Diagnosis of definite, probable, laboratory-supported probable, or possible ALS by revised El Escorial research criteria.\n2. Ages 18 to 75 years.\n3. Onset of weakness within three years of study enrollment.\n4. ALS with progression, characterized either by:\n\ni. a reduction of 0.5 points per month or greater on the ALS Functional Rating Scale-Revised (ALSFRS-R), which will be calculated based on (most recent ALSFRS-R at least 12 weeks from screening - ALSFRS-R at screening)/time interval; or ii. a calculated progression rate: (48 - ALSFRS-R at \"time of diagnosis\") / duration from onset to diagnosis (month) that is 0.5 points per month or greater.\n\ne) Plasma NfL levels \u2265 2 times the upper limit of the age-specific reference values for normal at the measuring laboratory at screening.\n\nf) Capable of providing informed consent. g) Capable and willing to follow study protocol. h) Ability to swallow pills and liquids at the time of the screening visit and, in the investigator's opinion have the ability to swallow for the duration of the study OR can be fed via a Gastrostomy (G) tube or Percutaneous Endoscopic capacity (PEG) tube.\n\ni) Slow vital capacity (SVC) \\> 65% of predicted value for gender, height, and age (participants perform SVC for three trials and the best SVC will be used).\n\nj) Females of childbearing potential must agree to abstain from sex or use adequate method of contraception for the duration of the study period and for 28 days after the last dose of study drug.\n\nk) Males must agree to abstain from sex or use adequate method of contraception for the duration of the study period and for 28 days after the last dose of study drug.\n\nl) If an approved therapy for ALS is used during the study, a steady dose must be used as follows: i. Participants who do not currently receive riluzole and do not plan to receive riluzole during the study period. Participants receiving riluzole are on a stable dose for at least 4 weeks before enrollment. Participants receiving riluzole are expected to remain on the same dose throughout the duration of the study.\n\nii. Participants who do not currently receive edaravone and do not plan to receive edaravone during the study period. Participants receiving edaravone must have completed at least 1 cycle of treatment before enrollment and are expected to continue edaravone treatment throughout the duration of the study.\n\nExclusion Criteria:\n\n1. Inability to follow the study protocol, based on the investigator's assessment.\n2. Pregnant or nursing women.\n3. Recently\uff08within 28 days\uff09received other experimental treatments.\n4. Presence of any active infections or inflammatory diseases at the time of enrollment that may confound the assessment of levels of inflammatory markers.\n5. Taking any medication or supplements with anti-inflammatory effects, including but not limited to prednisone, colchicine, or curcumin.\n6. Taking Qalsody (tofersen).\n7. Taking any medications containing nucleotide reverse transcriptase inhibitors (NRTIs), including but not limited to Abacavir, Emtricitabine, Lamivudine, or Zidovudine; trade names Atripla, Biktarvy, Cimduo, Combivir, Complera, Delstrigo, Descovy, Dovato, Emtriva, Epivir, Epzicom, Genvoya, Odefsey, Retrovir, Stribild, Symfi, Symtuza, Triumeq, Trizivir, Truvada, Ziagen.\n8. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to investigator's judgment (e.g., cardiovascular instability, systemic infection), or clinically significant laboratory abnormality.\n9. Clinically significant abnormal liver or kidney function at baseline (pre-dose). The following values \\[alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 3 times the upper limit of normal (ULN) or estimated Glomerular Filtration Rate (eGFR) \\< 30 mL/min/1.73m2\\] are exclusionary regardless of clinical symptoms.\n10. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, in the investigator's opinion.\n11. Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.\n12. Non-invasive ventilation, tracheostomy, oxygen supplementation for primary pulmonary pathology.\n13. Current / anticipated need of diaphragm pacing system (DPS).\n14. History of prior AAV gene therapy for any indication;\n15. Presence of any clinically relevant diseases that, in the research team's opinion, would prevent the subject from completing the study, including but not limited to severe cognitive dysfunction or medical conditions other than ALS that affect physical function or life expectancy.\n16. Plan to move away from the study site within the next 6 months.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Kamlanoflast", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01384162", "title": "An Open Label, Safety and Tolerability Continuation Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Newron Sweden AB", "summary": "This is an open label safety and tolerability continuation study of intracerebroventricular administration of sNN0029, containing the growth factor VEGF165, in patients with amyotrophic lateral sclerosis that have previously participated in study sNN0029-001. The intention of the study is to investigate safety and tolerability of intracerebroventricular administration of sNN0029 and whether it can improve motor function and prolong survival in patients with ALS.", "interventions": [{"type": "DRUG", "name": "sNN0029"}], "start_date": "2009-06", "url": "https://clinicaltrials.gov/study/NCT01384162", "target_entities": ["VEGF"], "locations": [{"facility": "University Hospital Leuven, Department of Neurology", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Clinical diagnosis of ALS classified as definite, or probable with or without additional laboratory evidence, according to the revised WFN El Escorial criteria (Appendix B).\n2. Previous participation in study sNN0029-001 with completion of 12 weeks study drug administration.\n3. To have completed the investigations associated with safety in study sNN0029-001 without development of clinically significant safety concerns.\n4. Patient has been given written and verbal information about the continuation study, has had the opportunity to ask questions about the study, and understands the time and procedural commitments.\n5. Patient has given oral and / or signed consent (written) to participate in the study. In the event that a patient who gives oral informed consent is not physically able to sign the informed consent form (ICF) due to disease progression, a witness may sign the ICF on the patient's behalf.\n\nExclusion Criteria:\n\n1. Hypertension defined as blood pressure \\>160 mmHg systolic or \\>90 mmHg diastolic.\n2. Proliferative retinopathy.\n3. Non-proliferative retinopathy of moderate severity or higher.\n4. Concurrent clinically significant dementia as determined by the investigator.\n5. Concurrent clinically significant depression as determined by the investigator.\n6. Need for administration of any antiplatelet or anticoagulant medication (e.g. aspirin, Plavix, non-steroidal anti-inflammatory drugs \\[NSAIDs\\]). Low dose aspirin or occasional use of NSAIDs is allowed (See Appendix E).\n7. Clinically significant abnormalities in haematology or clinical chemistry parameters as assessed by the investigator.\n8. For female patients, ongoing pregnancy or planned pregnancy\n9. Breast feeding", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "sNN0029", "targeting_mechanism": "Intracerebroventricular delivery of VEGF165 growth factor to promote motor neuron survival and function.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03944447", "title": "Outcomes Mandate National Integration With Cannabis as Medicine", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "OMNI Medical Services, LLC", "summary": "This will be a multistate, multicenter clinical study to determine the efficacy and safety of medical cannabis for a wide variety of chronic medical conditions.", "interventions": [{"type": "DRUG", "name": "Cannabis, Medical"}, {"type": "DEVICE", "name": "RYAH-Medtech Inhaler"}], "start_date": "2018-12-01", "url": "https://clinicaltrials.gov/study/NCT03944447", "target_entities": [], "locations": [{"facility": "OMNI Medical Services", "city": "Boca Raton", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.35869, "lon": -80.0831}, {"facility": "OMNI Medical Services", "city": "Bradenton", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 27.49893, "lon": -82.57482}, {"facility": "OMNI Medical Services", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.12231, "lon": -80.14338}, {"facility": "OMNI Medical Services", "city": "Fort Myers", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.62168, "lon": -81.84059}, {"facility": "OMNI Medical Services", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 29.65163, "lon": -82.32483}, {"facility": "OMNI Medical Services", "city": "Merritt Island", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 28.359, "lon": -80.69}, {"facility": "OMNI Medical Services", "city": "Miami", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 25.77427, "lon": -80.19366}, {"facility": "OMNI Medical Services", "city": "Ocoee", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 28.56917, "lon": -81.54396}, {"facility": "OMNI Medical Services", "city": "Pensacola", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 30.42131, "lon": -87.21691}, {"facility": "OMNI Medical Services", "city": "Pompano Beach", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.23786, "lon": -80.12477}, {"facility": "OMNI Medical Services", "city": "Tampa", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 27.94752, "lon": -82.45843}, {"facility": "OMNI Medical Services", "city": "Wesley Chapel", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 28.23973, "lon": -82.32787}, {"facility": "OMNI Medical Services", "city": "Beechwood", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 39.20839, "lon": -84.39911}, {"facility": "OMNI Medical Services", "city": "Bowling Green", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 41.37477, "lon": -83.65132}, {"facility": "OMNI Medical Services", "city": "Sandusky", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 41.44894, "lon": -82.70796}, {"facility": "OMNI Medical Services", "city": "Toledo", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 41.66394, "lon": -83.55521}, {"facility": "OMNI Medical Services", "city": "Toledo", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 41.66394, "lon": -83.55521}], "contact_phone": "419-214-3220", "contact_email": "ryan.lakin.md@gmail.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinical diagnosis of a Qualifying Condition for Medical Marijuana\n* Must be 18 years or older unless they have consent from their parent or legal guardian as defined under state law parameters\n* Must be willing to complete online surveys at baseline and the follow up points in this study\n\nExclusion Criteria:\n\n* Pregnancy\n* Breastfeeding\n* Inability to provide informed consent\n* Inability to complete study visits or questionnaires\n* Active suicidality or psychosis, that could be exacerbated by the administration of cannabis", "sex": "ALL", "min_age": "7 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Medical Cannabis (THC:CBD)", "targeting_mechanism": "Targeting CB2 receptors on astrocytes and other endocannabinoid system elements to exert anti-excitotoxicity, anti-oxidant and anti-inflammatory effects in ALS.", "targeting_mechanism_pmid": "33486755", "animal_results": "In SOD1(G93A) transgenic mice, the cannabigerol quinone derivative VCE-003.2 showed neuroprotective effects, suggesting cannabinoids can provide benefit in ALS models.", "animal_results_pmid": "30076846", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03114215", "title": "Effect of MD1003 in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "MedDay Pharmaceuticals SA", "summary": "This is a 6-month double blind randomized 2:1 placebo-controlled study with two arms (placebo, biotin 300 mg/day). The study will be followed by a 6-month extension phase during which all patients will receive biotin 300 mg/day.", "interventions": [{"type": "DRUG", "name": "MD1003"}, {"type": "DRUG", "name": "Placebo oral capsule"}], "start_date": "2016-06-29", "url": "https://clinicaltrials.gov/study/NCT03114215", "target_entities": ["biotin"], "locations": [{"facility": "Hopital Gui De Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age: 25 to 80 years, inclusive\n* Male or female subjects with probable or confirmed ALS (revised international El Escorial criteria, Forbes et al., 2001).\n* Patients presenting first motor deficits due to ALS for a maximum of three years at the first consultation in an ALS centre.\n* Patients monitored for at least 6 months in an ALS centre or for whom the previous monitoring parameters are available (excepted for MIP and SNIP).\n* Patients who have lost at least 5 points on the ALSFRS-R (ALS functional rating scale) during the last 12 months or at least 2 points during the preceding 6 months\n* Patients who have been treated with riluzole for at least 3 months at a stable dose. In case of intolerance to this product or refusal for this treatment, patients who have not been treated with riluzole for at least 1 month before inclusion\n* For patients with spinal form (onset of the disease affecting limbs) or respiratory form, slow vital capacity \\> 60% of predicted value.\n* For patients with a bulbar form, slow vital capacity \\> 60% of theoretical value or, if spirometry not assessable (severe bulbar disability), patient should not have significant abnormality in both nocturnal capnography and nocturnal oximetry (median pCO2 (carbon dioxide partial pressure ) \\< 52 mmHg, SaO2 (arterial oxygen saturation ) \\< 90% less than 5% of the time during night) less than 3 months prior inclusion.\n* Patients who are willing to give written consent (or oral consent in the presence of a trusted person if the patient is no longer able to write)\n* Patients likely to be able to participate in all scheduled evaluation and complete all required study procedures (except for spirometry in bulbar patients with severe disability).\n\nExclusion Criteria:\n\n* Patients on non-invasive ventilation for respiratory insufficiency due to ALS for more than 10 hours a day\n* Patients with an ALSFRS-R score at inclusion of \\< 20 (maximum score without disability = 48)\n* Patients who have lost less than 5 points on the ALSFRS-R during the last year or less than 2 points during the preceding 6 months\n* Patients with a gastrostomy\n* Patients who have lost more than 15% of their reference weight (defined as weight before disease onset)\n* Patient with dyspnoea at rest or with the least effort (score \\< 3 on the dyspnoea item of the ALSFRS-R)\n* Patients with dementia\n* Patient with severe or rapidly progressive form of ALS for whom the investigator estimates the life expectancy less than 3 months\n* Patients with another progressive disease that has not been stabilized at the time of inclusion\n* Patients with cancer, except basal cell carcinoma, for less than 5 years, or who require continuous treatment for cancer even if it is older\n* Pregnant women.\n* Subject who are not covered by a social security scheme.\n* Subject under temporary or permanent Judicial Protection.\n* Contraception: Both male subjects, and female subjects who are not either surgically sterile (tubal ligation/obstruction or removal of ovaries or uterus) or post-menopausal (no spontaneous menstrual periods for at least one year confirmed by a negative hormone panel), must commit to using two highly effective method of birth control for the duration of the study and for two months after the treatment termination.", "sex": "ALL", "min_age": "25 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MD1003", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00696332", "title": "Talampanel for Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.", "summary": "The purpose of this study is to assess the efficacy, tolerability and safety of oral administration of talampanel compared to a placebo in subjects with ALS.", "interventions": [{"type": "DRUG", "name": "Talampanel"}, {"type": "DRUG", "name": "Talampanel"}, {"type": "OTHER", "name": "placebo"}], "start_date": "2008-09", "url": "https://clinicaltrials.gov/study/NCT00696332", "target_entities": ["glutamate_excitotoxicity"], "locations": [{"facility": "CA Medical Center for Movement Disorders-Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Kansas Medical Center - Dept of Neurology", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins OPC - Meyer Bldg", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital-Neurology Clinical Trials Unit", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Columbia University - Neurology Institute", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Academic Hospital University of Leuven - ALS dept", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "ALS Centre", "city": "Vancouver", "state": "British Columbia", "country": "Canada", "status": "", "lat": 49.24966, "lon": -123.11934}, {"facility": "London Health Sciences Centre Motor Neuro Diseases Clinic", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Montreal Neurological Institute", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "C.H.U. La Timone - Service de Neurologie", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "C.H.U. de Montpellier - Hopital Gui de Chauliac - Service des Explorations Neurologiques", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Hopital La Pitie Salpetriere - Federation de Neurologie", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Universitaetsklinik Berlin-Charite, Campus Virchow Klinikum, Neurologische Klinik", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Berufsgenossenschaftliche Klinik Bergmannsheil, Neurologische Klinik", "city": "Bochum", "state": "", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Universitaet Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Semmelweis University, Department of Neurology", "city": "Budapest", "state": "", "country": "Hungary", "status": "", "lat": 47.49835, "lon": 19.04045}, {"facility": "Sourasky MC -EMG Unit", "city": "Tel Aviv", "state": "", "country": "Israel", "status": "", "lat": 32.08088, "lon": 34.78057}, {"facility": "Fondazione \"S.Maugeri\" Clinica della Riabilitazione IRCCS-Istituto Scientifico di Lissone", "city": "Lissone (MI)", "state": "", "country": "Italy", "status": "", "lat": 45.61236, "lon": 9.23985}, {"facility": "Centro Clinico NEMO", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Azienda Ospedaliero Universitaria San Giovanni Battista di Torino - Dipartimento di Neuroscienze", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Carlos III", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Subjects with sporadic or familial ALS classified as definite, probable, or laboratory-supported probable ALS according to the revised El Escorial criteria.\n\nInclusion Criteria:\n\n1. Diagnosis of definite or probable ALS in accordance with the El-Escorial criteria.\n2. Subject has experienced his/her first ALS symptoms within 3 years prior to the screening visit.\n3. Slow VC test equal to or greater than 70% of the predicted value.\n4. The sum of the 3 respiratory items on the ALSFRS-R must total at least 10 points.\n5. Subjects taking riluzole must be on a stable dose for at least 8 weeks prior to screening visit.\n6. Ages 18-80 (inclusive)\n\nExclusion Criteria:\n\n1. The use of invasive or non-invasive ventilation.\n2. Subject having undergone gastrostomy.\n3. Subject with any clinically significant or unstable medical condition.\n4. Subject participating in any other investigational drug trial or using investigational drug (within 12 weeks prior to screening and thereafter).\n5. Females who are pregnant or nursing.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Talampanel", "targeting_mechanism": "AMPA receptor antagonist that blocks glutamatergic excitotoxicity to protect motor neurons from glutamate-mediated cell death.", "targeting_mechanism_pmid": "27350567", "animal_results": "The AMPA receptor antagonist perampanel robustly rescued ALS pathology in sporadic ALS model mice, with oral administration reducing disease progression markers.", "animal_results_pmid": "27350567", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07414212", "title": "Evaluation of the Combined Therapy of EH-301 and N-acetylcysteine Together With Riluzole in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogipuzkoa Health Research Institute", "summary": "This study is designed to evaluate whether a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis (ALS). Researchers will assess changes in disease progression using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard tool for measuring daily functioning in people with ALS.\n\nThe main question is whether taking NAC together with EH-301 can prevent symptom worsening and possibly improve existing ALS symptoms.\n\nParticipants will be randomly assigned to receive either the active combination (NAC + EH-301) or matching placebos for 6 months. During this period, they will attend regular clinic visits for evaluations, tests, and safety monitoring.\n\nAfter completing the initial 6-month phase, all participants may choose to join a 6-month open-label extension, where everyone receives the active treatment regardless of their original group.", "interventions": [{"type": "DRUG", "name": "Acetylcysteine"}, {"type": "DIETARY_SUPPLEMENT", "name": "EH301"}, {"type": "DRUG", "name": "Riluzole"}], "start_date": "2025-11-25", "url": "https://clinicaltrials.gov/study/NCT07414212", "target_entities": ["oxidative_stress", "riluzole"], "locations": [{"facility": "Hospital Universitario Donostia", "city": "San Sebasti\u00e1n", "state": "Guipuzcoa", "country": "Spain", "status": "RECRUITING", "lat": 43.56667, "lon": -5.9}], "contact_phone": "+34 943006140", "contact_email": "lara.alamedacalvo@bio-gipuzkoa.eus", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patients diagnosed with ALS according to the Gold Coast criteria;\n2. Disease duration \u2264 18 months;\n3. Men and women aged 18 to 75 years;\n4. Total ALSFRS-R score \u2265 30 for all 12 categories;\n5. Forced vital capacity (FVC) \u226570%;\n6. The participant must receive treatment with Riluzole (50 mg twice daily) for \u226530 days prior to Day 1 (Visit 2) and is expected to remain on that dose until the final study visit.\n7. Willingness and ability of the patient to comply with the requirements of the protocol during the study;\n8. Sign written informed consent prior to any study-related procedure;\n9. Acceptance by women to use at least one highly effective method of contraception during the study 30 days prior to taking the nutraceutical and investigational drug and throughout the study. The following are considered highly effective contraceptive methods:\n\n   * Combined hormonal methods (oral, patches, injectables, or implants).\n   * Hormonal or copper intrauterine devices (IUDs).\n   * Previous surgical sterilization (bilateral tubal ligation).\n   * Total sexual abstinence when consistent with the patient's usual lifestyle.\n10. Acceptance by men included in the study of the use of condoms in combination with an effective contraceptive method used by their female partner of childbearing age during treatment.\n\nExclusion Criteria:\n\n1. Presence of other neurodegenerative diseases;\n2. Significant cognitive impairment and/or dementia;\n3. Any psychiatric illness that could interfere with the study;\n4. Use of dietary supplements with high doses of vitamin B3 in the 30 days prior to inclusion in the study;\n5. Severe heart disease;\n6. Moderate to severe lung disease, such as emphysema, stage III-IV COPD;\n7. Uncontrolled chronic asthma;\n8. Active cancer;\n9. Any metabolic, neoplastic, physically or mentally debilitating disease that could put the subject at risk or interfere with the study results;\n10. Genetically confirmed mitochondrial disease;\n11. Tracheostomized and/or gastrostomized patients;\n12. Participation in any clinical trial with an investigational product within 30 days or five half-lives of the previous agent, whichever is longer, prior to dosing;\n13. Any clinically significant laboratory abnormality that could directly affect compliance or safety;\n14. Allergy to NAC or any excipient, either in the investigational drug or in the EH301 nutraceutical;\n15. Patients with a short life expectancy in the investigator's judgment.\n16. \\[Women only\\] Pregnancy or breastfeeding for women of childbearing potential (i.e., \\<2 years postmenopausal or not surgically sterile);\n17. The participant is unwilling to use highly effective contraception during the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "EH-301 and N-acetylcysteine (NAC) combined with Riluzole", "targeting_mechanism": "N-acetylcysteine acts as an antioxidant to counteract oxidative stress, while riluzole blocks glutamatergic neurotransmission to reduce excitotoxicity; combined approach targets both oxidative stress and excitatory toxicity in ALS.", "targeting_mechanism_pmid": "32847483", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "Riluzole is approved for ALS treatment; NAC is a known antioxidant used in various clinical contexts.", "repurposed_from_pmid": "32847483"}}
{"nct_id": "NCT03489200", "title": "EH301 for the Treatment of ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Elysium Health", "summary": "The objective of this trial is to evaluate the efficacy and tolerability of EH301 in patients with amyotrophic lateral sclerosis. Patients with ALS are randomized to receive either EH301 or placebo daily and undergo active evaluation for 6 months.", "interventions": [{"type": "OTHER", "name": "EH301"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2017-01", "url": "https://clinicaltrials.gov/study/NCT03489200", "target_entities": ["eh301"], "locations": [{"facility": "Universidad Cat\u00f3lica de Valencia San Vicente M\u00e0rtir", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* 18 years or older\n* Diagnosis of probable or definite (sporadic or familial) ALS by El Escorial criteria\n* Onset of symptomatology for more than 6 months\n* If female: not lactating; negative pregnancy test; agree to use an effective method of birth control throughout study\n\nExclusion Criteria:\n\n* Tracheostomy, invasive ventilation, or non-invasive positive pressure ventilation\n* Gastrostomy\n* Evidence of major psychiatric disorder or clinically evident dementia\n* Diagnosis of a neurodegenerative disease in addition to ALS\n* Current medication apart from riluzole that in the opinion of the investigator would make the patient unsuitable for study participation\n* Recent history (within the previous 6 months) or current evidence of alcohol or drug abuse\n* Concurrent unstable disease involving any system (e.g. carcinoma other than basal cell carcinoma), any cardiac dysrhythmia, myocardial infarction, clinical or ECG signs of myocardial ischemia, cardiac insufficiency, angina symptoms, current symptoms of coronary artery disease, or any other condition that in the opinion of the investigator would make the patient unsuitable for study participation\n* Baseline QTc (Bazett) \\> 450 msec for males and \\> 470 msec for females\n* Known hepatitis B/C or HIV positive serology\n* Renal impairment defined as blood creatinine \\> 2x ULN\n* Hepatic impairment and/or liver enzymes (ALT or AST) \\> 3x ULN\n* Hemostasis disorders or current treatment with oral anticoagulants\n* Participated in any other investigational drug or therapy study with a non-approved medication, within the previous 3 months\n* No medical insurance", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "EH301", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00748501", "title": "Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sangamo Therapeutics", "summary": "The purpose of the study is to evaluate the effects of the investigational drug, SB-509 on progression of the disease in subjects with ALS", "interventions": [{"type": "DRUG", "name": "SB-509"}], "start_date": "2008-09", "url": "https://clinicaltrials.gov/study/NCT00748501", "target_entities": ["GDNF"], "locations": [{"facility": "Coordinated Clinical Research", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "University of California, Irvine; MDA ALS and Neuromuscular Center,", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center (CPMC), The Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "The University of Kansas Medical Center (KU)", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Nerve and Muscle Center of Texas", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female between the ages of 18 and 85 with clinical diagnosis of ALS\n* Forced Vital Capacity (FVC) \\> 60% of predicted\n* Less than 3 years of ALS since the onset of the first symptom with clinical evidence of limb muscle atrophy and weakness.\n* Subjects taking Riluzole must have been at a stable dose for at least 30 days with no evidence of toxicity\n* Female of childbearing potential and male of child-creating potential must agree to use a medically acceptable physical barrier (condom, diaphragm, and cervical cap) through the treatment phase and for at least 30 days after the last study treatment.\n\nExclusion Criteria:\n\n* Women who are pregnant or currently breast-feeding\n* Dependent upon invasive or non-invasive artificial ventilation\n* Patients with bulbar onset ALS or with other active neuromuscular/ neurodegenerative diseases.\n* Type 1 or Type 2 diabetes.\n* Evidence of chronic or active heart, liver, kidney, or lung diseases, or Age-related macular degeneration.\n* Current or history of known immune or immunodeficiency disorders\n* Patients with cognitive impairment with significant decision making incapacity, or major depression, or schizophrenia, or dementia (e.g. Alzheimer's disease).\n* Malignancy or history of malignancy, except it has been in complete remission for at least 5 years\n* Pre-cancerous conditions (e.g. Barrett's Esophagus, dysplasias) or benign tumors which have the potential for significant growth due to VEGF stimulation.", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "SB-509", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01933321", "title": "Effect of Intrathecal Administration of Hematopoietic Stem Cells in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hospital Universitario Dr. Jose E. Gonzalez", "summary": "Autologous cell therapy in patients with ALS can stimulate neuroplasticity, modifying the neurodegenerative process and stops the clinical progression of disease.", "interventions": [{"type": "BIOLOGICAL", "name": "Intrathecal autologous stem cell"}], "start_date": "2012-09", "url": "https://clinicaltrials.gov/study/NCT01933321", "target_entities": ["neuroplasticity"], "locations": [{"facility": "Servicio Hematolog\u00eda Hospital Universitario", "city": "Monterrey", "state": "Nuevo Le\u00f3n", "country": "Mexico", "status": "", "lat": 25.68435, "lon": -100.31721}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* patients with a confirmed diagnosis of ALS according to El Escorial criteria. Diagnosis-time less than four years.\n* Over 18 years old. Forced vital capacity \u2265 40%.\n* One year of evolution.\n* Adequate nutritional state\n\nExclusion Criteria:\n\n* Severe bulbar ALS involucre.\n* Inadequate nutritional status.\n* Spondylotic myelopathy, or abnormalities in imaging study.\n* Having concomitant neurological or psychiatric disease.\n* Systemic disease with poor-control.\n* History of treatment with steroids or immunoglobulins in the last year.\n* Participate in the past three months in a Clinical Trial.\n* History of malignancy or cancer today.\n* Intracranial hypertension.\n* Clinical suggestive data of infection in the site of lumbar puncture.\n* Tracheostomy.\n* Use of mechanical ventilation. Forced vital capacity less than 40%. HIV-seropositive or presence of antibodies against hepatitis B or C in the serological studies.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Intrathecal autologous stem cell", "targeting_mechanism": "Autologous hematopoietic stem cells to stimulate neuroplasticity and support degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Preclinical stem cell studies in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1) demonstrated potential benefit of stem cell therapy for ALS.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04454840", "title": "Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University Third Hospital", "summary": "To evaluate the safety and effectiveness of intravenous infusion of plasma from healthy young people for the treatment of amyotrophic lateral sclerosis.", "interventions": [{"type": "BIOLOGICAL", "name": "Plasma from healthy young people treatment + Riluzole"}, {"type": "DRUG", "name": "Riluzole"}], "start_date": "2016-05-01", "url": "https://clinicaltrials.gov/study/NCT04454840", "target_entities": ["plasma_factors_rejuvenation_factors"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Meet the following diagnosis standard: confirmed, proposed and laboratory supported diagnosis;\n* Age 50-70 years old ;\n* 3-18 months course of disease;\n* Forced vital capacity (FVC) \u226570% predicted value;\n* Total amyotrophic lateral sclerosis Functional Rating Scale score \u226536, scores of respiratory related items \u226510;\n* Take Riluzole regularly before participate in this trial (25\\~50mg twice a day for at least 30 days continuously) without obvious side effects and can continue to take for 22 months;\n* Participants of childbearing age take reasonable and effective contraceptive measures from the time of enrollment to the end of follow-up;\n* Signed informed consent.\n\nExclusion Criteria:\n\n* Familial amyotrophic lateral sclerosis;\n* Female during pregnancy and lactation;\n* Positive hepatitis B, hepatitis C or HIV in screening\n* History of cytomegalovirus and malaria infection;\n* After tracheotomy and ventilator-dependent state (daily use of non-invasive ventilator \u2265 22 hours for 7 consecutive days);\n* After percutaneous gastrostomy (PEG) operation;\n* Has had allergic reactions and other adverse reactions during blood transfusion;\n* Have diseases of the blood system (including Immunoglobulin A deficiency);\n* alanine transaminase, Aspartate transaminase\u2265 3 times the upper limit of normal;\n* Abnormal renal function (Cr, BUN);\n* History of malignant tumors;\n* Combining severe cardiopulmonary diseases, autoimmune diseases, mental diseases, substance abuse history, etc;\n* Currently participating in other clinical studies or using other drugs in researching.", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Plasma from healthy young people", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04204889", "title": "Trial of Oxaloacetate in ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Omar Jawdat", "summary": "The purpose of this study is to determine the safety and the maximal tolerated dose of Oxaloacetate (OAA) in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Oxaloacetic Acid"}], "start_date": "2020-03-17", "url": "https://clinicaltrials.gov/study/NCT04204889", "target_entities": ["mitochondrial_metabolism"], "locations": [{"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* A clinical diagnosis by a study investigator of laboratory-supported probable, probable, or definite ALS, according to the modified El Escorial criteria\\[1\\]\n* Vital capacity (VC) greater or equal to 50% of predicted\n* Diagnosis with ALS within 3 years prior to enrollment\n* If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit\n* Women of childbearing age must use protection against pregnancy.\n\nExclusion Criteria:\n\n* Requirement for tracheotomy ventilation or non-invasive ventilation for \\> 23 hours per day\n* Diagnosis of other neurodegenerative diseases (e.g., Parkinson disease, Alzheimer disease)\n* Clinically significant history of unstable medical illness (e.g., unstable angina, advanced cancer) over the last 30 days\n* Current pregnancy or lactation\n* Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures\n* Receipt of any investigational drug within the past 30 days from enrollment", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Oxaloacetic Acid", "targeting_mechanism": "Oxaloacetate restores mitochondrial bioenergetic function to address mitochondrial dysfunction in ALS motor neurons.", "targeting_mechanism_pmid": "33398403", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00214110", "title": "Tamoxifen Therapy in Amyotrophic Lateral Sclerosis [ALS]", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Wisconsin, Madison", "summary": "This is a single-center, phase 2 randomized clinical trial of tamoxifen on mean percent predicted isometric muscle strength in patients with amyotrophic lateral sclerosis (ALS). The purpose is to determine whether the triphenylethylenetamoxifen, used as adjuvant therapy in the treatment of breast cancer, can delay the loss of isometric muscle strength in ALS patients.", "interventions": [{"type": "DRUG", "name": "Tamoxifen"}], "start_date": "2001-01", "url": "https://clinicaltrials.gov/study/NCT00214110", "target_entities": ["Estrogen receptor"], "locations": [{"facility": "University of Wisconsin", "city": "Madison", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.07305, "lon": -89.40123}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* clinically probable-laboratory supported, clinically probable, or clinically definite amyotrophic lateral sclerosis\n\nExclusion Criteria:\n\n* Allergic or idiosyncratic response to tamoxifen.\n* Other active neurologic diseases that may produce weakness, sensory loss, or autonomic symptoms.\n* Psychiatric, psychological, or behavioral symptoms that would interfere with the subject's ability to participate in the trial.\n* Clinically significant cardiac, pulmonary, gastrointestinal, hematologic, or endocrine (poorly controlled insulin-dependent diabetes mellitus or hyperthyroidism) disease that may confound interpretation of the study results.\n* Previous kidney or pancreas transplants.\n* Significant hepatic or renal disease (AST \\> 5 times normal, serum creatinine \\> 2.0 mg/dL for males or \\> 1.8 mg/dL for females).", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tamoxifen", "targeting_mechanism": "Stabilization of neuromuscular junctions through modulation of estrogen signaling.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "breast cancer", "repurposed_from_pmid": "32481440"}}
{"nct_id": "NCT04632225", "title": "Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Helixmith Co., Ltd.", "summary": "The purpose of this study was to evaluate the safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis as compared to Placebo. Safety will be assessed by incidences of treatment-emergent adverse events, treatment-emergent serious adverse events, injection site reactions and other adverse events of special interest, and the clinically significant laboratory values after injections of Engensis compared to Placebo. Exploratory endpoints include assessment of muscle function using the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale subscores for Fine and Gross Motor Function; muscle strength by quantitative testing using handheld dynamometry and the Accurate Test of Limb Isometric Strength where available; quality of life using the Amyotrophic Lateral Sclerosis Assessment Questionnaire-40; patient global impression of change, clinical global impression of change, and clinical global impression of severity; and evaluation of lung function using Slow Vital Capacity. Muscle biopsies will be performed during the study for future biomarker analyses.", "interventions": [{"type": "BIOLOGICAL", "name": "Engensis"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2021-03-09", "url": "https://clinicaltrials.gov/study/NCT04632225", "target_entities": ["engensis"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center, Barrows Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins University Department of Neurology", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Hanyang University Medical Center", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Clinically definite or probable Amyotrophic Lateral Sclerosis or laboratory-supported probable Amyotrophic Lateral Sclerosis as defined in the revised El Escorial/Airlie House diagnostic criteria\n2. The site of onset of Amyotrophic Lateral Sclerosis symptoms is a limb and experiencing symptoms of lower motor dysfunction (e.g., weakness, atrophy, cramps, poor circulation, etc.) with upper motor neuron symptoms (e.g., weakness, brisk reflexes, spasticity)\n3. Onset of Amyotrophic Lateral Sclerosis symptoms \u2264 4 years\n4. Slow Vital Capacity \u2265 50% of predicted value at Screening\n5. Not taking riluzole, or on a stable dose (defined as no noted toxicities) for at least 30 days prior to Screening and throughout the study\n6. Not taking edaravone or on a maintenance cycle for at least 30 days prior to Screening and throughout the study\n7. For females of childbearing potential, a negative urine pregnancy test at Screening and on Day 0\n8. Male Participants and their female partners must agree to use double-barrier contraception during the study or provide proof of postmenopausal state (minimum 1 year) or surgical sterility\n9. Male Participants must not donate sperm during the study\n10. Female Participants must be nonpregnant, nonlactating, and either postmenopausal for at least 1 year, or surgically sterile for at least 3 months, or agree to use double-barrier contraception from 28 days prior to randomization (Day 0) and/or their last confirmed menstrual period prior to study randomization (whichever is longer) until the end of the study\n11. Capable of complying and willing to comply with the requirements and restrictions in the informed consent form and this protocol\n12. Willing to forgo new experimental Amyotrophic Lateral Sclerosis treatments for at least 6 months following randomization\n\nExclusion Criteria:\n\n1. Progressive or degenerative neurological disorder such as Alzheimer's disease, Parkinson's disease, vascular dementia, multiple sclerosis, and other neurological or vascular disorders felt by the Investigator to preclude participation\n2. Requires tracheotomy ventilation or noninvasive ventilation related to bulbar function\n3. Evidence by physical examination, history, or laboratory evaluation of significant concomitant disease with a life expectancy of \\< 6 months at Screening\n4. International Normalized Ratio values \\>2.0\n5. Platelet count \\<100,000/\u00b5L\n6. Inflammatory disorder of the blood vessels (inflammatory angiopathy or vasculitis, such as Buerger's disease)\n7. Active infection (chronic infection or severe active infection that may compromise the Participant's wellbeing or participation in the study in the Investigator's judgment)\n8. Chronic inflammatory disease (e.g., Crohn's disease, rheumatoid arthritis)\n9. Positive human immunodeficiency virus or human T-cell lymphotrophic virus I/II test at Screening\n10. Active acute or chronic hepatitis B\n11. Active hepatitis C\n12. Immunosuppression due to underlying disease (e.g., rheumatoid arthritis, systemic lupus erythematosus) or to currently receiving immunosuppressive drugs, (e.g., chemotherapy, corticosteroids) or to radiation therapy\n13. Stroke or myocardial infarction within 3 months prior to Screening\n14. Active deep vein thrombosis\n15. Recent history (\\< 3 years) or presence of cancer except basal cell carcinoma or squamous cell carcinoma of the skin that was excised and has shown no evidence of recurrence for at least 1 year\n16. Major psychiatric disorder diagnosed in the past 6 months that has not been stabilized or in the Investigator's opinion would not allow the patient to participate in the scheduled procedures\n17. Use of an investigational drug for the treatment of Amyotrophic Lateral Sclerosis in the past 30 days or 5 half-lives (if available), whichever is longer, or previous participation in a clinical study with Engensis\n18. Stem cell administration for investigational treatment of Amyotrophic Lateral Sclerosis or other conditions in the 6 months prior to Screening", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Engensis", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04240925", "title": "Tolerability, Safety and Efficacy of Sigh Breaths During NIMV in Motor Neuron Disease", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ospedale San Raffaele", "summary": "Non-invasive mechanical ventilation (NIMV) is the recommended standard of care as initial therapy for patients with motor neuron disease (MND) with deterioration of the respiratory function.\n\nSIGH\\_01 study is aimed at investigating the tolerability, safety profile and efficacy of sigh breaths during non-invasive mechanical ventilation in patients with MND in comparison to the standard ventilation support protocol.", "interventions": [{"type": "DEVICE", "name": "NIMV with sigh breaths"}, {"type": "DEVICE", "name": "Standard NIMV"}], "start_date": "2018-05-25", "url": "https://clinicaltrials.gov/study/NCT04240925", "target_entities": [], "locations": [{"facility": "Ospedale San Raffaele", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Willing and able to give informed consent\n* MND diagnosis according to El-Escorial criteria\n* Non-invasive ventilation indications in accordance with the international guidelines\n\nExclusion Criteria:\n\n* Inability to adhere to study visit schedule or lack of reliable caretaker\n* Presence of dementia\n* History of arrhythmia, heart failure or pneumothorax", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Non-invasive mechanical ventilation with sigh breaths to support respiratory function in motor neuron disease.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03334786", "title": "Study to Evaluate Safety & Efficacy of FLX-787-ODT to Treat Fasciculations in Tongue and Appendicular Muscle in Adult Subjects With ALS", "phase": "PHASE1, PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Flex Pharma, Inc.", "summary": "The FLX-787-107 study will determine how well FLX-787-ODT works to reduce fasciculations in patients with Amyotrophic Lateral Sclerosis (ALS). The study will measure how often fasciculations occur, if tongue and muscle strength, speech, and swallowing are affected, and monitor any side effects that might develop while taking the investigational product. Participants will be assessed before and after taking a single dose of FLX-787-ODT. Approximately 15 people will take part in this study at one center in the United States. Participants will be in the study for a single clinic visit and receive a telephone call 7 days later to monitor for side effects.", "interventions": [{"type": "DRUG", "name": "FLX-787-ODT"}], "start_date": "2018-04-05", "url": "https://clinicaltrials.gov/study/NCT03334786", "target_entities": ["Sodium channel"], "locations": [{"facility": "Beth Israel Deaconess Medical Center", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Documented diagnosis of ALS diagnosis of less than 5 years.\n* Greater than 6 fasciculations per minute noted at least in the tongue by clinical, ultrasound, or EMG evaluation.\n* Normal oral cavity exam at screening.\n\nExclusion Criteria:\n\n* Presence of clinically significant or unstable condition that would result in an increased risk of study participation or difficulty in interpretation of the study results.\n* Tremor or other movement disorder that would interfere with recording.\n* Presence of major gastrointestinal disorders, such as inflammatory bowel disease, diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or esophageal lesions/ulcers.\n* Presence of laryngospasm or significant swallowing problems.\n* Inability to tolerate a spicy sensation in the mouth or stomach.\n* Actively using illicit drugs or history of chronic substance abuse within the past year prior to screening, including abuse of alcohol.\n* Participated in a clinical study (except natural history studies without administration of an investigational product) within 30 days prior to screening.\n* Pregnant, breastfeeding, or planning to become pregnant.\n* Blood pressure of \u2265160 mmHg systolic and/or \u2265100 mmHg diastolic.\n* Clinically significant abnormalities in laboratory findings (including screening complete electrolyte panel, complete blood count, liver function tests).", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "FLX-787-ODT", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05163886", "title": "Study of Safety, Tolerability, and Biological Activity of LAM-002A in C9ORF72-Associated Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "OrphAI Therapeutics", "summary": "This is a clinical trial to evaluate the safety, tolerability, and biological effect of LAM-002A in adults with C9ORF72-associated ALS (C9ALS).", "interventions": [{"type": "DRUG", "name": "LAM-002A"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2021-12-23", "url": "https://clinicaltrials.gov/study/NCT05163886", "target_entities": ["C9orf72"], "locations": [{"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "Johns Hopkins University School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Diagnosis of C9ORF72-associated ALS with BOTH of the following:\n\n   1. Documentation of a clinical genetic test demonstrating the presence of a pathogenic repeat expansion in C9ORF72. If there is a strong clinical suspicion for C9ALS based on C9-positive family history and El Escorial Criteria consistent with a diagnosis of ALS, clinical testing for the C9ORF72 repeat expansion may be performed with study screening labs at the discretion of the Site Investigator (SI), medical monitor, and study sponsor.\n\n      AND\n   2. Must meet possible, laboratory-supported probable, probable, or definite criteria for diagnosing ALS by revised El Escorial criteria (Brooks 2000).\n2. Age 18 or older\n3. Capable of providing informed consent at the Screening Visit and complying with study procedures throughout the study, in the SI's opinion, and at the discretion of the medical monitor and study sponsor.\n4. In the case that a participant lacks the ability to provide informed consent. Informed consent will be sought from the participant's surrogate representative.\n5. Able to safely swallow study drug capsule at screening and throughout study. May use thickened substances to assist in swallowing drug.\n6. Vital Capacity greater than and equal to 50% of predicted at the time of the Screening Visit measured by Slow Vital Capacity (SVC), or, if required due to COVID-19 pandemic-related restrictions and with Sponsor approval, Forced Vital Capacity (FVC) measured in-person or via telemedicine.\n7. Participants must either not take or be on a stable dose of riluzole (as either a tablet or oral suspension) for greater than 30 days prior to the Screening Visit. Riluzole-na\u00efve participants are permitted in the study.\n8. Participants must either not take edaravone or have completed at least one 14-day cycle with plan for continuation of edaravone prior to the Screening Visit. Participants must be off cycle and at least 2 days after the last dose administration of edaravone at the time of study visit. Edaravone-na\u00efve participants are permitted in the study.\n9. Participants must be able to complete all study procedures, including the lumbar punctures (LP) at the time of the Screening Visit, in the SI's opinion.\n10. Geographically accessible to the site.\n\nExclusion Criteria:\n\n1. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to the SI's judgment \\[e.g., cardiovascular instability, systemic infection, or clinically significant laboratory abnormality or electrocardiogram (ECG) changes\\].\n\n   1. Gastrointestinal disease (e.g., gastric, or intestinal bypass surgery, jejunostomy tube, pancreatic enzyme insufficiency, malabsorption syndrome, symptomatic inflammatory bowel disease, chronic diarrheal illness, bowel obstruction) that might interfere with drug absorption or with interpretation of gastrointestinal AEs. Gastrostomy tube placement is allowed prophylactically or to supplement nutrition/hydration but may not be used for study drug administration.\n   2. Hepatic profile showing any of the following:\n\n   i. Serum alanine aminotransferase (ALT) greater than 5 \u00d7 upper limit of normal (ULN).\n\n   ii. Serum aspartate aminotransferase (AST) greater than 5 \u00d7 ULN.\n\n   iii. Serum bilirubin greater than 1.5 \u00d7 ULN.\n\n   c. Renal profile showing an estimated creatinine clearance (eClCR) less than 30 mL/minute (with eClCR to be calculated by the method at the laboratory performing the serum creatinine test).\n2. Presence of a neurodegenerative cognitive or motor syndrome (e.g., Alzheimer's disease, Parkinson's disease) not related to the C9ORF72 repeat expansion.\n3. Presence of unstable psychiatric disease or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.\n4. Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years. Active cancer includes cancers with current disease manifestations or therapy that could adversely affect subject safety and longevity, create the potential for drug-drug interactions, or compromise the interpretation of study results.\n5. Prior solid organ transplantation.\n6. Ongoing immunosuppressive therapy including systemic or enteric corticosteroids at screening or for the duration of the trial, at the discretion of the site investigator and medical monitor.\n7. Use within 5 days prior to randomization or for the duration of the trial of a strong inhibitor or inducer of cytochrome P450 (CYP) 3A4 or expected requirement for chronic use of a strong inhibitor or inducer of CYP3A4 during study therapy.\n8. Use within 5 days prior to randomization or for the duration of the trial of drug that is a moderate-to-strong substrate of CYP2C9 (including warfarin, tolbutamide, phenytoin, glimepiride) or expected requirement for chronic use of such drugs during study therapy, at the discretion of the site investigator and medical monitor.\n9. Use of investigational treatments for ALS (off-label use or active participation in a clinical trial) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Screening Visit.\n10. Exposure at any time to any gene therapies under investigation for the treatment of ALS (off-label use or investigational).\n11. If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception for the duration of the trial and for 3 months after discontinuing treatment.\n12. If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 3 months after discontinuing study treatment.\n13. Anything that would place the participant at increased risk or preclude the participant's full compliance with or completion of the study, in the SI's opinion.\n14. If a participant is being re-screened, the disqualifying condition has not been resolved, or the mandatory wash-out duration has not occurred.\n15. Contraindication to undergoing a lumbar puncture (LP) in the SI's opinion. Participants undergoing the LP must not be currently taking anticoagulation and antiplatelet medications such as warfarin and clopidogrel bisulfate (Plavix\u2122), that would be a contraindication to LP; aspirin and non-steroidal anti-inflammatories are allowed.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "LAM-002A", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05619783", "title": "Extension Study Evaluating The Safety And Tolerability of AMX0035", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Amylyx Pharmaceuticals Inc.", "summary": "The primary objective is to evaluate the safety and tolerability of AMX0035 over 108 weeks of open label treatment for participants previously enrolled in Study A35-004 (PHOENIX).", "interventions": [{"type": "DRUG", "name": "AMX0035"}], "start_date": "2022-12-29", "url": "https://clinicaltrials.gov/study/NCT05619783", "target_entities": ["TARDBP"], "locations": [{"facility": "University Hospitals Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Hospices Civils de Lyon H\u00f4pital Neurologique Pierre Wertheimer Cellule Mutualis\u00e9e de Recherche Clinique (CMRC)", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "Hopital Gabriel Montpied Service de Neurologie", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "", "lat": 45.77969, "lon": 3.08682}, {"facility": "CHRU de Lille - H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges - H\u00f4pital Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "H\u00f4pitaux Universitaires de Marseille Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHU de Montpellier", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU Nice", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "H\u00f4pital de la Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Le Centre Hospitalier R\u00e9gional Universitaire de Tours", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Uniklinikum Dresden", "city": "Dresden", "state": "", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Hannover Medical School", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Jena University Hospital", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Medizinische Fakult\u00e4t Mannheim der Universit\u00e4t Heidelberg", "city": "Mannheim", "state": "", "country": "Germany", "status": "", "lat": 49.4891, "lon": 8.46694}, {"facility": "University Medical Center Rostock", "city": "Rostock", "state": "", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Ulm University Medical Centre", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Trinity College Dublin/Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Universit\u00e0 degli Studi di Bari Aldo Moro", "city": "Bari", "state": "", "country": "Italy", "status": "", "lat": 41.12066, "lon": 16.86982}, {"facility": "Centro Clinico NEMO", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "IRCCS - Ospedale San Raffaele", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "University of Milan Medical School", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "IRCCS - Istituto Auxologico italiano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Azienda Ospedaliero Universitaria Di Modena", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Universit\u00e0 degli Studi della Campania Luigi Vanvitelli", "city": "Naples", "state": "", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}, {"facility": "University of Padua", "city": "Padova", "state": "", "country": "Italy", "status": "", "lat": 44.38225, "lon": 11.14261}, {"facility": "Universit\u00e0 degli Studi di Bari Aldo Moro", "city": "Tricase", "state": "", "country": "Italy", "status": "", "lat": 39.93018, "lon": 18.35421}, {"facility": "University of Torino", "city": "Turin", "state": "", "country": "Italy", "status": "", "lat": 45.07049, "lon": 7.68682}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Centrum Medyczne Linden", "city": "Krakow", "state": "", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}, {"facility": "City Clinic Warsaw", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Centro Hospitalar Universit\u00e1rio Lisboa-Norte", "city": "Lisbon", "state": "", "country": "Portugal", "status": "", "lat": 38.72509, "lon": -9.1498}, {"facility": "Hospital del Mar", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitari de Bellvitge-IDIBELL", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario de Basurto", "city": "Bilbao", "state": "", "country": "Spain", "status": "", "lat": 43.26271, "lon": -2.92528}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Biodonostia Health Research Institute; Hospital Universitario Donostia", "city": "San Sebasti\u00e1n", "state": "", "country": "Spain", "status": "", "lat": 43.56667, "lon": -5.9}, {"facility": "Hospital Universitario y Polit\u00e9cnico La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Karolinska Institutet", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Ume\u00e5 University Hospital", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "The Walton Centre NHS Trust", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "King's College London", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "UCL Queen Square Institute of Neurology", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "University of Plymouth", "city": "Plymouth", "state": "", "country": "United Kingdom", "status": "", "lat": 50.37153, "lon": -4.14305}, {"facility": "Salford Royal Hospital Barnes Clinical Research Team", "city": "Salford", "state": "", "country": "United Kingdom", "status": "", "lat": 53.48771, "lon": -2.29042}, {"facility": "Sheffield Institute for Translational Neuroscience (SITraN)", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Previous participation in Study A35-004 (PHOENIX), including completion of the randomized controlled phase through Week 48 (this timepoint may be upcoming at the time of screening). Participants who do not complete randomized-controlled phase through Week 48 for medical reasons may be included on a case-by-case basis, in consultation with the Sponsor;\n2. Capable of providing informed consent;\n3. Capable and willing to follow trial procedures including visits to the trial clinic, remote visits, and survival status reporting requirements;\n4. Women of childbearing potential (WOCBP; e.g., not post-menopausal for at least one year or surgically sterile must agree to use adequate birth control for the duration of the trial and 3 months after the last dose of AMX0035;\n\n   1. 12 months of spontaneous amenorrhea or 6 months of spontaneous amenorrhea with serum Follicle-stimulating hormone (FSH) levels \\> 40 mIU/ml (milli-international units per milliliter) or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy.\n   2. Acceptable contraception methods for use in this trial are:\n\n      * Hormonal methods, such as birth control pills, patches, injections, vaginal ring, or implants;\n      * Barrier methods (such as a condom or diaphragm) used with a spermicide (a foam, cream, or gel that kills sperm);\n      * Intrauterine device (IUD);\n      * Abstinence (no heterosexual sex);\n      * Unique partner who is surgically sterile (men) or not of childbearing potential (female).\n5. Women must not be pregnant or planning to become pregnant for the duration of the trial and 3 months after last dose of AMX0035;\n6. Men must agree to practice contraception for the duration of the trial and for at least 3 months after last dose of AMX0035;\n7. Men must not plan to father a child or to provide sperm for donation for the duration of the trial and 3 months after the last dose of AMX0035\n\nExclusion Criteria:\n\n1. History of known allergy to phenyl butyrate or bile salts;\n2. Abnormal liver function defined as bilirubin levels and/or aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \\> 5 times the upper limit of the normal (obtained within 12 weeks from first dose);\n3. Renal insufficiency as defined by estimated glomerular filtration rate (eGFR) \\<60 mL/min/1.73m2 normal (obtained within 12 weeks from first dose);\n4. Pregnant women or women currently breastfeeding;\n5. Current severe biliary disease which may result in the Investigator medical judgement in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gangrene of the gallbladder, abscess of the gallbladder;\n6. History of Class III/IV heart failure (per New York Heart Association - NYHA);\n7. Participant under severe salt restriction where the added salt intake due to treatment would put the participant at risk, in the Investigator clinical judgment;\n8. Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, according to Investigator judgment;\n9. Clinically significant unstable medical condition (other than ALS) (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, severe laboratory test anomaly or clinically significant electrocardiogram \\[ECG\\] changes) that would pose a risk to the participant if he/she were to participate in the trial, according to Investigator judgment;\n10. Currently enrolled in another trial (excluding Study A35-004 (PHOENIX)) involving use of an investigational therapy (or within 5 plasma half-lives) prior to first dose at Baseline Visit;\n11. Implantation of Diaphragm Pacing System (DPS);\n12. Currently or previously treated within the last 30 days (or 5 half-lives, whichever is longer) from first dose at the Baseline Visit or planned exposure during the treatment period to any prohibited medications listed in Section 6.7 of the protocol.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AMX0035", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02418546", "title": "Electronic-health Application To Measure Outcomes REmotely Clinical Trial", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "This is a phase II feasibility, safety, tolerability and preliminary efficacy study of an e-Health application versus in-person nutritional counseling to maintain or increase weight in patients with neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), Parkinson's Disease (PD) and Huntington's disease (HD). Primary Objectives include the feasibility, safety, tolerability and efficacy of an e-Health application to maintain or increase body weight compared to in-person nutritional counseling. Secondary Objectives are to measure the number of calories required to maintain or increase body weight in neurodegenerative diseases at all stages of the disease. Tertiary Objectives are to test the effects of an e-Health application compared to in-person nutritional counseling on disease progression using the ALSFRS-R, UHDRS or UDysRS, on survival, and on quality of life using the PROMIS SF v1.1 scale.", "interventions": [{"type": "BEHAVIORAL", "name": "In-Person Nutritional Counseling by a Registered Dietitian"}, {"type": "BEHAVIORAL", "name": "Nutritional counseling using an e-Health Application"}], "start_date": "2015-04", "url": "https://clinicaltrials.gov/study/NCT02418546", "target_entities": ["nutritional_status"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Adults with neurodegenerative diseases such as ALS, PD or HD with or without a history of unintentional weight loss.\n2. Male or female subjects aged 18 years or older.\n3. Participants must be capable of providing informed consent and complying with trial procedures.\n4. Participants must have an MGH swallowing screening tool score\\>5 at the time of the screening visit\n5. Participants or a designated caregiver must be able to obtain home weights and communicate to their RD\n\nExclusion Criteria:\n\n1. Clinical evidence of unstable medical or psychiatric illness, in the investigator's judgment, which would prevent the participant from completing their assessments.\n2. BMI \\> 35 combined with a history of cardiovascular disease; or a history of diabetes regardless of BMI.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06493279", "title": "Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "RJK Biopharma Ltd", "summary": "The goal of the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D).", "interventions": [{"type": "DRUG", "name": "RJK002 Intrathecal injection"}], "start_date": "2024-09-24", "url": "https://clinicaltrials.gov/study/NCT06493279", "target_entities": ["ATXN2"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Main Inclusion Criteria:\n\n1. Female or male subjects who are \u2265 18 years of age at screening;\n2. Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);\n3. The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be less than 2 years (inclusive);\n4. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score \u226530 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be \u22653;\n5. The forced vital capacity (FVC) of predicted during the screening period is \u226570% at screening;\n6. Body mass index (BMI) greater than 18 kg/m2 at screening;\n\nMain Exclusion Criteria:\n\n1. Subjects with other neurological diseases similar to ALS that affect the evaluation of drug efficacy, such as cervical spondylotic myelopathy, syringomyelia, spinal cord and brain stem tumors, hirayama disease, multifocal motor neuropathy, multiple sclerosis, Guillain-Barre syndrome, Parkinson's disease and dementia;\n2. Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);\n3. Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RJK002", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07407725", "title": "Clinical Outcome Assessment for AT & BCI", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shirley Ryan AbilityLab", "summary": "Many individuals with severe motor impairments rely on Assistive Technologies (ATs) or Brain-Computer Interfaces (BCIs) to interact with digital devices such as their computers. Clinicians and researchers currently lack a common framework to objectively quantify how much a given AT or BCI improves real-world function or to compare across tools. This project seeks to address this gap by developing a standardized method to objectively assess or compare the functional benefit of these tools on digital independence, i.e., the ability to independently operate computers, phones, and other digital systems, by creating a unique Digital Assessment Interface (DAI).\n\nThis assessment will be a simulation of online and digital activities that prior work has determined is important to functional daily living in the digital domain. Participants will complete this assessment with various ATs and BCIs, and these scores will be used to create an index, which will be comprised of performance outcomes, clinician-reported outcomes, and patient-reported outcomes.\n\nThe tool aims to quantify and compare digital task performance across devices and user populations. The primary objective of this study is to develop an index. The index will quantify functional performance of individuals using various ATs and BCIs. The secondary objectives are to extensively evaluate the psychometric properties of the index, such as the validity, responsiveness, reliability, and floor/ceiling effects both globally and across different devices and impairment levels, ensuring that it can reliably measure the impact of an AT or BCI on a user's ability to independently operate digital systems; and to characterize the familiarization and use of specific BCI and AT systems with reference to a normative healthy control population.", "interventions": [{"type": "DEVICE", "name": "Eye Tracker"}, {"type": "DEVICE", "name": "Mouth Operated Joystick"}, {"type": "DEVICE", "name": "Non-invasive electroencephalogram (EEG) Headset"}, {"type": "DEVICE", "name": "Implantable Brain-Computer-Interface"}, {"type": "DEVICE", "name": "Personal Assistive Technology"}, {"type": "DEVICE", "name": "Voice Control"}], "start_date": "2026-01-08", "url": "https://clinicaltrials.gov/study/NCT07407725", "target_entities": [], "locations": [{"facility": "Shirley Ryan AbilityLab", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "RECRUITING", "lat": 41.85003, "lon": -87.65005}], "contact_phone": "312-238-6875", "contact_email": "ajayaraman@sralab.org", "eligibility": {"criteria": "Enrollment will involve three different cohorts, namely a cohort of healthy participant, a cohort of ALS/SCI participant who underwent the implant of an invasive BCI device, and a cohort of ALS/SCI participants without any implanted BCI device.\n\nBelow are listed the inclusion and exclusion criteria for each diagnostic group.\n\nSpinal Cord Injury (SCI):\n\nInclusion Criteria:\n\n* Age at or above 18 years old;\n* Diagnosis of spinal cord injury, at the level of T1 or above levels (between C1 and T1);\n* Ability to communicate independently or with a support device, or with a legal representative;\n* Ability to participate in a study session for about 3 hours (e.g., endurance, fatigue), which may include breaks as needed.\n\nExclusion Criteria:\n\n* Participation in another trial that would conflict with the current study or clinical endpoint interference may occur.\n\nAmyotrophic Lateral Sclerosis (ALS):\n\nInclusion criteria:\n\n* Age at or above 18 years old;\n* Diagnosis of amyotrophic lateral sclerosis;\n* Ability to communicate independently, with a support device, or with a legal representative;\n* Ability to participate in a study session for about 3 hours (e.g., endurance, fatigue), which may include breaks as needed.\n\nExclusion criteria:\n\n\u25cf Participation in another trial that would conflict with the current study or clinical endpoint interference may occur.\n\nHealthy Controls:\n\nInclusion criteria:\n\n* Age at or above 18 years old;\n* No history of neurological or psychiatric disorders;\n* Ability to provide written informed consent;\n* Ability to participate in a study session for about 3 hours (e.g., endurance, fatigue), which may include breaks as needed.\n\nExclusion criteria:\n\n* Participation in another trial that would conflict with the current study or clinical endpoint interference may occur;\n* Cognitive, visual, or auditory deficits that would interfere with study participation;\n* Current or prior diagnosis or condition that could confound study assessments.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02789059", "title": "Muscle Oxygenation in Effort in Neuromuscular Diseases", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Lille", "summary": "Previous studies showed modifications of muscle oxygenation parameters in muscular dystrophies du to an impairment or an absence of dystrophin.\n\nOur study aim at assessing muscle oxygenation during effort in different neuromuscular diseases (muscular dystrophies related and not related to dystrophin, non dystrophic myopathies and motor neuron diseases) compared to a group of healthy controls. Patients and controls are invited to perform an inframaximal , standardized effort of the knee extensors by the mean of an isokinetic dynamometer. Muscle oxygenation parameters are assessed through a Near Infrared Spectroscopy (NIRS) Device.\n\nIn patients affected by dystrophin related myopathies, a muscle biopsy will be performed in order to analyse mitochondrial oxygenation parameters and mitochondrial phenotype.\n\nOur Hypothesis is that muscle oxygenation is impaired in dystrophin related muscular dystrophies compared to other neuromuscular diseases and healthy controls because of lack of muscle capillary vessels dilatation during effort and impairment of mitochondrial function.", "interventions": [{"type": "OTHER", "name": "muscle oxygenation"}], "start_date": "2015-07-02", "url": "https://clinicaltrials.gov/study/NCT02789059", "target_entities": ["muscle_oxygenation"], "locations": [{"facility": "H\u00f4pital Amiens Nord, Service de Neurologie", "city": "Amiens", "state": "", "country": "France", "status": "", "lat": 49.9, "lon": 2.3}, {"facility": "CHRU de Lille, H\u00f4pital Swyngedhauw", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "H\u00f4pital S\u00e9bastopol, CHU de Reims", "city": "Reims", "state": "", "country": "France", "status": "", "lat": 49.26526, "lon": 4.02853}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* healthy subjects and\n* subjects affected by one of the fallowing neuromuscular diseases: Becker Muscular dystrophy Facioscapulohumeral dystrophy, Limb Girdle Muscular Dystrophy , Congenital Myopathy , Spinal Muscular Atrophy Charcot Marie Tooth Disease and Amyotrophic Lateral Sclerosis ,\n* able to walk\n* presenting a manual muscle testing of at Least 4/5 on the quadriceps according to the Medical research Council\n\nExclusion Criteria:\n\n* musculoskeletal pain of the quadriceps\n* other neurological disorders\n* Heart failure arrhythmia, uncontrolled hypertension, angina pectoris\n* dyspnoea \\>2 according to the NYHA\n* Peripheral artery disease\n* BMI \\>30kg.m-2.", "sex": "MALE", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04176224", "title": "Clinical Pharmacology Study of Oral Edaravone in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "To evaluate the pharmacokinetics of single doses of edaravone oral suspension in Patients with Amyotrophic Lateral Sclerosis", "interventions": [{"type": "DRUG", "name": "MT-1186"}], "start_date": "2019-04-17", "url": "https://clinicaltrials.gov/study/NCT04176224", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Investigational site", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nThe key criteria are listed below.\n\n* Patients aged between 20 and 75 years at the time of informed consent\n* Japanese patients\n* Among patients with ALS, those \"Clinically definite ALS,\" \"Clinically probable ALS\" or \"Clinically probable-laboratory-supported ALS\" according to El Escorial Revised Airlie House criteria\n* Patients who can consent to contraception\n* Patients who have thoroughly understood the contents of the study and voluntarily provided written informed consent to participate in the study\n\nExclusion Criteria:\n\nThe key criteria are listed below.\n\n* Patients in whom the possibility could not be ruled out that the current symptoms were symptoms of a disease requiring differential diagnosis, such as cervical spondylosis and multifocal motor neuropathy\n* Patients undergoing treatment for malignancy\n* Patients who have presence of clinically significant liver, heart, or renal disease requiring hospitalization (except ALS) and infections requiring antibiotics. Patients who have a problem in general condition and are judged ineligible by the Investigator\n* Body mass index (BMI) of \\<18.0 or \\>30.0, or a body weight of \\<50 kg\n* Patients judged by the investigator (or subinvestigator) to be unsuitable for the study for any other reason", "sex": "ALL", "min_age": "20 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MT-1186 (edaravone)", "targeting_mechanism": "Edaravone is a free radical scavenger that reduces oxidative stress by neutralizing reactive oxygen species.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06215391", "title": "Customized Masks in Non-Invasive Mechanical Ventilation", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hospital Universitario 12 de Octubre", "summary": "In non-invasive mechanical ventilation (NIMV), the interface is the primary determinant of success, as adherence and quality of therapy mainly depend on it. The aim of this study is to investigate the usefulness of a customised mask approach to minimise leakage and upper airway obstruction. It will focus on ventilator registries and changes in the way they can be corrected with these customised masks.\n\nThe process involves 3D face scanning and dedicated computer-aided design. The processing and manufacturing of the masks is based on additive manufacturing through 3D printing.", "interventions": [{"type": "DEVICE", "name": "3d printed mask"}], "start_date": "2023-12-10", "url": "https://clinicaltrials.gov/study/NCT06215391", "target_entities": [], "locations": [{"facility": "Hospital Universitarios 12 de Octubre", "city": "Madrid", "state": "Madrid", "country": "Spain", "status": "RECRUITING", "lat": 40.4165, "lon": -3.70256}], "contact_phone": "913908492", "contact_email": "lauragramos@hotmail.es", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patient enrolled in the Home Mechanical Ventilation (HMV) program.\n* Adherence of \\>4 hours per day.\n* In the case of COPD, inspiratory pressures \\>18 cmH2O.\n* Users of a Resmed HMV device to standardize and facilitate the analysis of the respirator log.\n* Commercial masks (MC) in optimal condition, as assessed by the mechanical ventilation unit responsible during outpatient ventilation consultations.\n* Presence of residual leaks (\\>5 LPM on average according to the respirator log - ResScan, unintentional leaks, with intentional leaks excluded by software).\n\nExclusion Criteria:\n\n* Patients with tracheostomy or scheduled for tracheostomy.\n* Patients on a waiting list for lung transplantation.\n* Patients using HMV devices from manufacturers other than ResMed.\n* Users who alternate between various MC models, where homogeneity in interface use cannot be assured.\n* Refusal to provide consent.\n* Patients with documented allergies to components of medical-grade silicone used in the production of M3D.\n* Exacerbations requiring hospitalization or changes in medication or the respirator in the last 3 months.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "3D printed mask", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04667013", "title": "A Multiple Dose Study to Investigate Safety, Tolerability and Pharmacokinetics of TBN", "phase": "PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Guangzhou Magpie Pharmaceuticals Co., Ltd.", "summary": "The purpose of this study is to assess the safety, tolerability and pharmacokinetics (i.e. how study drug is taken up by the body) of TBN in healthy participants.", "interventions": [{"type": "DRUG", "name": "Tetramethylpyrazine nitrone (TBN) tablet / Placebo"}], "start_date": "2028-09-01", "url": "https://clinicaltrials.gov/study/NCT04667013", "target_entities": ["oxidative_stress"], "locations": [], "contact_phone": "305-547-5857", "contact_email": "david.wyatt@syneoshealth.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Male or non-childbearing potential female, non-smoker (no use of tobacco or nicotine products within 3 months prior to screening), \u2265 18 and \u2264 50 years of age, with BMI \\> 18.0 and \\< 30.0 kg/m2 and body weight \u2265 50.0 kg for males and \u2265 45.0 kg for females.\n2. Healthy as defined by:\n\n   1. The absence of clinically significant illness and surgery within 4 weeks prior to the first dosing. Subjects vomiting within 24 hours pre-dose will be carefully evaluated for upcoming illness/disease. Inclusion pre-dosing is at the discretion of the Investigator.\n   2. The absence of clinically significant history of neurological, endocrine, cardiovascular, respiratory, hematological, immunological, psychiatric, gastrointestinal, renal, hepatic, and metabolic disease.\n3. Non-childbearing potential female is defined as:\n\n   1. Post-menopausal female (absence of menses for 12 months prior to the first study drug administration, bilateral oophorectomy or hysterectomy with bilateral oophorectomy at least 6 months prior to the first study drug administration);\n\n      or\n   2. Surgically sterile female (hysterectomy or tubal ligation at least 6 months prior to drug administration).\n4. Male subjects who have not been vasectomized for at least 6 months prior, and who are sexually active with a female partner of childbearing potential (childbearing potential females are defined as women that are neither post-menopausal nor surgically sterile) must be willing to use one of the following acceptable contraceptive methods from the first study drug administration until at least 90 days after the last study drug administration:\n\n   1. Simultaneous use of a male condom and, for the female partner, hormonal contraceptives used since at least 4 weeks prior or intra-uterine contraceptive device placed since at least 4 weeks prior;\n   2. Simultaneous use of a male condom and, for the female partner, a diaphragm or cervical cap with intravaginally applied spermicide.\n5. Male subjects (including men who have had a vasectomy) with a pregnant partner must agree to use a condom from the first study drug administration until at least 90 days after the last study drug administration.\n6. Male subjects must be willing not to donate sperm until 90 days following the last study drug administration.\n7. Capable of consent.\n\nExclusion Criteria:\n\n1. Any clinically significant abnormality at physical examination, clinically significant abnormal laboratory test results or positive test for hepatitis B, hepatitis C, or HIV found during medical screening.\n2. Positive urine drug screen, alcohol breath test, or urine cotinine test at screening.\n3. History of allergic reactions to TBN or other related drugs, or to any excipient in the formulation.\n4. Positive pregnancy test at screening.\n5. Clinically significant ECG abnormalities or vital sign abnormalities, systolic BP lower than 90 or over 140 mmHg, diastolic BP lower than 50 or over 90 mmHg, or heart rate (HR) less than 50 or over 100 bpm; orthostatic BP: decrease in systolic BP of 20 mmHg or higher, decrease in diastolic BP of 10 mmHg or higher, or increase in HR of 30 bpm or higher within 2 to 3 minutes after passing from a supine to a standing position at screening.\n6. History of significant alcohol abuse within 1 year prior to screening or regular use of alcohol within 6 months prior to the screening visit (more than 14 units of alcohol per week \\[1 unit = 150 mL of wine, 360 mL of beer, or 45 mL of 40% alcohol\\]).\n7. History of significant drug abuse within 1 year prior to screening or use of soft drugs (such as marijuana) within 3 months prior to the screening visit or hard drugs (such as cocaine, phencyclidine (PCP), crack, opioid derivatives including heroin, and amphetamine derivatives) within 1 year prior to screening.\n8. Participation in a clinical research study involving the administration of an investigational or marketed drug or device within 30 days prior to the first dosing, administration of a biological product in the context of a clinical research study within 90 days prior to the first dosing, or concomitant participation in an investigational study involving no drug or device administration.\n9. Use of medications for the timeframes specified below, with the exception of medications exempted by the Investigator on a case-by-case basis because they are judged unlikely to affect the pharmacokinetics (PK) profile of the study drug or subject safety (e.g., topical drug products without significant systemic absorption):\n\n   1. Prescription medications within 14 days prior to the first dosing;\n   2. Over-the-counter products and natural health products (including herbal remedies, homeopathic and traditional medicines, probiotics, food supplements such as vitamins, minerals, amino acids, essential fatty acids, and protein supplements used in sports) within 7 days prior to the first dosing, with the exception of the occasional use of acetaminophen (up to 2 g daily);\n   3. Depot injection or implant of any drug within 3 months prior to the first dosing;\n   4. Any drugs known to induce or inhibit hepatic drug metabolism (including St. John's wort) within 30 days prior to the first dosing.\n10. Donation of plasma within 7 days prior to dosing. Donation or loss of blood (excluding volume drawn at screening) of 50 mL to 499 mL of blood within 30 days, or more than 499 mL within 56 days prior to the first dosing.\n11. Any reason which, in the opinion of the Investigator, would prevent the subject from participating in the study.\n12. Breast-feeding subject.\n13. Any history of thyroid/gland abnormalities.\n14. Any history of suicidal ideation or suicidal behavior (within 2 years prior to screening), as assessed by the Columbia-Suicide Severity Rating Scale (C-SSRS) (baseline version).", "sex": "ALL", "min_age": "18 Years", "max_age": "50 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "Tetramethylpyrazine nitrone (TBN)", "targeting_mechanism": "Tetramethylpyrazine nitrone activates the PGC-1alpha/Nrf2/HO-1 pathway to reduce oxidative stress and improve mitochondrial function in ALS.", "targeting_mechanism_pmid": "33152451", "animal_results": "TBN improves motor dysfunction and reduces pathological manifestations in ALS mice via activation of the PGC-1alpha/Nrf2/HO-1 pathway.", "animal_results_pmid": "33152451", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05299658", "title": "An Open-Label Extension for the Phase 2 Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Clene Nanomedicine", "summary": "This is an optional open-label extension to participants that have completed the clinical trial CNMAu8.205.", "interventions": [{"type": "DRUG", "name": "CNMAu8"}], "start_date": "2021-11-13", "url": "https://clinicaltrials.gov/study/NCT05299658", "target_entities": ["bioenergetics"], "locations": [{"facility": "Concord Hospital", "city": "Concord", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.84722, "lon": 151.10381}, {"facility": "Neuroscience Research Australia", "city": "Randwick", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.91439, "lon": 151.24895}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Participants must have completed the randomized placebo-controlled Treatment Period without compliance issues.\n2. Able to understand and give written informed consent to participate in the open-label extension.\n3. If referred from a third party (neurologist or a State based ALS organisation), participant agrees to maintain transfer of care to a neurologist participating in the study.\n\nExclusion Criteria:\n\n1. Lack of treatment compliance during the randomized placebo controlled Treatment Period.\n2. Positive pregnancy test at the Week 36 visit, or, females who plan to get pregnant during the course of this extension or within 6 months of the end of this extension.\n3. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.\n4. Patient with clinically significant abnormalities in haematology, blood chemistry, ECG, or physical examination identified during the W36 visit which according to Investigator may interfere with continued participation.\n5. Patients with clinically significant hepatic or renal dysfunction or clinical laboratory findings that would limit the interpretability of change in liver or kidney function, or those with low platelet counts (\\< 150 x 10\\^9 per liter) or eosinophilia (absolute eosinophil count of \u2265 500 eosinophils per microliter) at the Week 36 visit.\n6. Patient is considered a suicide risk in the opinion of the Investigator, has previously made a suicide attempt, or is currently demonstrating active suicidal ideation. Subjects with intermittent passive suicidal ideation are not necessarily excluded based on the assessment of the Investigator.", "sex": "ALL", "min_age": "30 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CNM-Au8", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02670226", "title": "Muscular Biomarkers in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Tours", "summary": "The first objective is to find some biomarkers, or a profile of biomarkers of ALS to help to diagnosis. The second objective is to better understand the pathogenesis of this disease by the exploration of muscle, blood and satellite cells metabolomes and transcriptomes.", "interventions": [{"type": "OTHER", "name": "Samples"}], "start_date": "2016-03-29", "url": "https://clinicaltrials.gov/study/NCT02670226", "target_entities": [], "locations": [{"facility": "Service de chirurgie orthop\u00e9dique et traumatologique, CHRU de TOURS", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Service de Neurologie, CHRU de TOURS", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Case group selection criteria:\n\nInclusion Criteria:\n\n* Age \u2265 18 years and \u2265 75 years\n* ALS according to the El Escorial criteria\n* Patients affiliated to social security scheme\n* Informed consent signed by the patient\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding women\n* Contraindication to biopsy\n* Contraindication to local anesthesia\n* Treatment with oral or injectable anticoagulants, antiplatelet (except aspirin)\n* Unbalanced Diabetes\n* Systemic corticosteroid treatment\n* Treatment against cramps or twitching may affect muscle metabolism\n\nControl group selection criteria:\n\nInclusion Criteria:\n\n* Age \u2265 18 years and \u2265 75 years\n* No neuronal disease\n* Patients affiliated to social security scheme\n* Informed consent signed by the patient\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding women\n* Contraindication to biopsy\n* Treatment with oral or injectable anticoagulants, antiplatelet (except aspirin)\n* Unbalanced Diabetes\n* Systemic corticosteroid treatment\n* Treatment against cramps or twitching may affect muscle metabolism", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02537132", "title": "\"New Perspectives of Adaptation to NIV in ALS\"", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fondazione Don Carlo Gnocchi ETS", "summary": "The purpose of this study is to demonstrate that the adaptation to the Non Invasive Ventilation (NIV) at home is not worse when compared with an adaptation performed in inpatient settings.", "interventions": [{"type": "OTHER", "name": "Adaptation and training to Non Invasive Ventilation (NIV)"}], "start_date": "2015-01", "url": "https://clinicaltrials.gov/study/NCT02537132", "target_entities": [], "locations": [{"facility": "Michele Vitacca", "city": "Lumezzane", "state": "Brescia", "country": "Italy", "status": "", "lat": 45.64789, "lon": 10.26487}, {"facility": "Paolo Banfi", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS diagnosis according to the criteria of El Escorial;\n* Clinical indication to NIV (nocturnal hypoventilation and respiratory muscle weakness confirmed according to the criteria normally accepted);\n* No respiratory infection within 3 months\n* Severe disability (ALS-FRS \\<31);\n* Age \\> 18 years;\n* The voluntary participation to the Study.\n\nExclusion Criteria:\n\n\\-- Previous episodes of pneumothorax;\n\n* Comorbid cardiac and / or renal important\n* Severe cognitive impairment;\n* Refusal of the patient at the time of Informed Consent.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04993755", "title": "A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Transposon Therapeutics, Inc.", "summary": "This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).", "interventions": [{"type": "DRUG", "name": "TPN-101, 400 mg/day"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-10-01", "url": "https://clinicaltrials.gov/study/NCT04993755", "target_entities": ["C9orf72"], "locations": [{"facility": "University of California San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "University of California Irvine - ALS & Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "UCSF Neurosciences Clinical Research Unit (NCRU)", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "John Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Johns Hopkins Outpatient Center", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Mayo Family Clinic Northwest", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University Medical Center - The Neurological Institute of New York", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "The University of North Carolina at Chapel Hill, Department of Neurology", "city": "Chapel Hill", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.9132, "lon": -79.05584}, {"facility": "VIB-KU Leuven Center for Brain & Disease Research", "city": "Leuven", "state": "Flemish Brabankt", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "CHU Lille - CMRR H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU Dupuytren, Limoges", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Universitaetsklinikum Ulm - Klinik fuer Neurologie", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Complejo Hospitalario Universitario de Santiago (CHUS)", "city": "Santiago de Compostela", "state": "A Coru\u00f1a", "country": "Spain", "status": "", "lat": 42.88052, "lon": -8.54569}, {"facility": "Hospital de la Santa Creu i Sant Pau", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario Vall d'Hebron", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitari I Polit\u00e8cnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Documentation of a clinical genetic test demonstrating a hexanucleotide repeat expansion (HRE) in the C9orf72 gene\n* Has a reliable caregiver/informant to accompany the patient to all study visits\n\nFor patients with ALS (with or without FTD):\n\n* Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria\n* Onset of weakness within 3 years prior to Screening\n* Slow vital capacity (SVC) \u2265 60% of predicted normal adjusted for sex, age, and height (from the sitting position)\n* Able to perform reproducible pulmonary function tests.\n* ALS Functional Rating Scale-Revised (ALSFRS-R) \u2265 30 and score of 3 or 4 on Item #3 (swallowing) at Screening\n\nFor patients with FTD:\n\n* A gradual, progressive decline in behavior, language, or motor function consistent with mild cognitive impairment, mild behavioral impairment, mild cognitive/behavioral impairment, behavioral variant FTD, primary progressive aphasia, or amnestic syndrome\n* CDR Dementia Staging Instrument plus National Alzheimer's Coordinating Center Behavior and Language Domains (CDR plus NACC FTLD) global score of 0.5-2.0 at Screening\n\nExclusion Criteria:\n\n* Presence of other significant neurological or psychiatric disorders\n* History of clinically significant brain abnormality\n* Clinically significant medical illness\n* Tracheostomy or diaphragmatic pacing\n* Autoimmune disease requiring treatment or management (quiescent rheumatoid arthritis, psoriasis, or controlled Type 1 diabetes are acceptable)\n* History of human immunodeficiency virus (HIV) or hepatitis B infection, or any active infection during Screening, unless the patient will have been symptom-free for at least 30 days prior to randomization", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "TPN-101", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06710626", "title": "Control of Assistive Devices Via Brain-Computer Interface Technology", "phase": "NA", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuralink Corp", "summary": "The CONVOY Study is a clinical trial designed to explore the feasibility of participants from the PRIME Study (NCT06429735) using the N1 Implant to control various assistive devices. The main goal is to determine whether participants can successfully modulate their brain activity to control devices, such as an Assistive Robotic Arm (ARA). This study will assess the effectiveness, consistency, and safety of neural control using the ARA and other assistive devices.", "interventions": [{"type": "DEVICE", "name": "Assistive Robotic Arm"}], "start_date": "2024-11-25", "url": "https://clinicaltrials.gov/study/NCT06710626", "target_entities": [], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Continued enrollment in the PRIME Study.\n* Implanted with the N1 Implant.\n\nExclusion Criteria:\n\n* Explantation or deactivation of the N1 Implant.\n* Insufficient N1 Implant BCI performance demonstrated.\n* Lack of a suitable physical space to perform research sessions.\n* Any condition which, in the opinion of the Investigator, would compromise the candidate's ability to safely participate in the study.", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "N1 Implant and Assistive Robotic Arm", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04302870", "title": "Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Edinburgh", "summary": "MND-SMART is investigating whether selected drugs can slow down the progression of motor neuron disease (MND) and improve survival.\n\nThe study is 'multi-arm' meaning more than one treatment will be tested at the same time. The trial started with 3 arms; drug 1 (memantine), drug 2 (trazodone) and placebo (dummy drug). A third drug, amantadine, was added in April 2023. A fourth drug, tacrolimus, was added in March 2025 in Edinburgh and across all sites in April 2025. The first two drugs, memantine and trazodone, were removed from the trial in September 2023 due to lack of benefit. The trial currently has 4 recruiting arms; amantadine, liquid placebo (matched to amantadine), tacrolimus, and tablet placebo (matched to tacrolimus). This allows the evaluation of each drug versus placebo. Participants will be randomly allocated between the treatment arms they are eligible for. Medicines being tested are already approved for use in other conditions.\n\nMND-SMART has an 'adaptive' design. This means medicines being studied can change according to emerging results. Treatments shown to be ineffective can be dropped and new drugs can be added over the duration of the study. This will allow many treatments, over time, to be efficiently and definitively evaluated.\n\nThe medicines being tested have been selected following a rigorous process involving a systematic, unbiased, and comprehensive review of past clinical trials data, as well as information from pre-clinical research (studies in laboratories), for MND and other related neurodegenerative disorders. Drugs have been ranked for inclusion in MND-SMART by a group of independent MND experts according to set criteria. These include consideration of how the drugs work, their safety profiles, and the quality of previous studies.\n\nNew drugs will be selected for investigation in MND-SMART based on continuous review of constantly updated scientific evidence as well as findings from state-of-the-art human stem cell based drug discovery platforms. These can be added by substantial amendment to the protocol.", "interventions": [{"type": "DRUG", "name": "Memantine Hydrochloride Oral Solution"}, {"type": "DRUG", "name": "Trazodone Hydrochloride oral solution"}, {"type": "DRUG", "name": "Placebo oral solution"}, {"type": "DRUG", "name": "Amantadine Hydrochloride Oral Solution"}, {"type": "DRUG", "name": "Tacrolimus 1Mg Cap"}, {"type": "DRUG", "name": "Placebo capsule"}], "start_date": "2020-02-27", "url": "https://clinicaltrials.gov/study/NCT04302870", "target_entities": ["nmda_receptor_antagonism", "serotonin_modulation", "calcineurin_inhibition"], "locations": [{"facility": "Southern Health and Social Care Trust, Craigavon Area Hospital", "city": "Portadown", "state": "County Armagh", "country": "United Kingdom", "status": "RECRUITING", "lat": 54.42302, "lon": -6.44434}, {"facility": "Aberdeen Royal Infirmary", "city": "Aberdeen", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 57.14369, "lon": -2.09814}, {"facility": "University Hospitals of Birmingham NHS Foundation Trust", "city": "Birmingham", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 52.48142, "lon": -1.89983}, {"facility": "University Hospitals Sussex NHS Foundation Trust", "city": "Brighton", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 50.82838, "lon": -0.13947}, {"facility": "West Suffolk NHS Foundation Trust", "city": "Bury St Edmunds", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 52.2463, "lon": 0.71111}, {"facility": "Cambridge University Hospitals NHS Foundation Trust", "city": "Cambridge", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 52.2, "lon": 0.11667}, {"facility": "Cardiff and Vale University Local Health Board", "city": "Cardiff", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 51.48, "lon": -3.18}, {"facility": "Clinical Research Centre , Ninewells Hospital", "city": "Dundee", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 56.46913, "lon": -2.97489}, {"facility": "Anne Rowling Regenerative Neurology Clinic", "city": "Edinburgh", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 55.95206, "lon": -3.19648}, {"facility": "Royal Devon and Exeter Hospital", "city": "Exeter", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 50.7236, "lon": -3.52751}, {"facility": "Queen Elizabeth University Hospital Clinical Research Facility", "city": "Glasgow", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 55.86515, "lon": -4.25763}, {"facility": "NHS Highland Clinical Research Facility, Raigmore Hospital", "city": "Inverness", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 57.47908, "lon": -4.22398}, {"facility": "East Suffolk and North Essex NHS Foundation Trust", "city": "Ipswich", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 52.05917, "lon": 1.15545}, {"facility": "Royal London Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 51.50853, "lon": -0.12574}, {"facility": "St George's University Hospitals NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 51.50853, "lon": -0.12574}, {"facility": "King's College Hospital NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 51.50853, "lon": -0.12574}, {"facility": "Newcastle upon Tyne Hospitals NHS Foundation Trust", "city": "Newcastle upon Tyne", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 54.97328, "lon": -1.61396}, {"facility": "Norfolk and Norwich University Hospitals NHS Foundation Trust", "city": "Norwich", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 52.62783, "lon": 1.29834}, {"facility": "University Hospitals of Dorset NHS Trust", "city": "Poole", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 50.71429, "lon": -1.98458}, {"facility": "Clinical Research Facility Salford Royal NHS Foundation Trust", "city": "Salford", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 53.48771, "lon": -2.29042}, {"facility": "Sheffield Teaching Hospitals NHS Foundation Trust", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 53.38297, "lon": -1.4659}, {"facility": "Clinical Research Facility University Hospital Southampton", "city": "Southampton", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 50.90395, "lon": -1.40428}], "contact_phone": "0131 465 9612", "contact_email": "siddharthan.chandran@ed.ac.uk", "eligibility": {"criteria": "Participants will be considered eligible for randomisation if they fulfil all the core inclusion criteria and none of the exclusion criteria as defined below. In addition, investigators must simultaneously check and ensure participants do not meet any of the drug specific exclusion criteria. If exclusion criteria are met for an arm, participants can still be considered for other arms and randomised accordingly to eligible arms.\n\nCore inclusion criteria:\n\n* Confirmed diagnosis of MND. This includes the following subtypes: ALS by El Escorial Criteria (possible, probable, and definite) or Gold Coast Criteria, Primary Lateral Sclerosis, and Progressive Muscular Atrophy\n* Over 18\n* Women of childbearing potential according to CTFG guidelines must have a negative pregnancy test within 7 days prior to, or at, the baseline visit\n* Women of childbearing potential and fertile men must be using an appropriate method of contraception to avoid any unlikely teratogenic effects of the selected drugs from time of consent, to 4 weeks after treatment inclusive\n* Willing and able to comply with the trial protocol and ability to understand and complete questionnaires\n* Written informed consent (in the case of limb dysfunction verbal consent can be given in the presence of a witness who can sign)\n\nCore Exclusion Criteria:\n\n* Patients diagnosed with Frontotemporal Dementia (FTD-MND) or any other significant psychiatric disorder that prevents informed consent being given.\n* Alcoholism (current self-reported - at the investigator's discretion)\n* Active suicide ideation assessed using the Columbia-Suicide Severity Rating Scale\n* On concurrent investigational devices and medication (including biological therapy)\n* Pregnancy or breast-feeding females\n* If ALT, ALP, bilirubin or GGT \\>3 times the upper limit of normal.\n* If creatinine clearance (creatinine clearance or eGFR) \\<35 ml/min.\n* If TSH \\<0.2mU/l (if possible to test free T4, then Serum free T4 \\>25pmol/l)\n* If corrected QT interval on 12 lead ECG \\>500 ms\n* Patient's diagnosed with ventricular arrhythmias, significant heart block (at the investigator's discretion)) or in the immediate recovery period after myocardial infarction (\\< 6 weeks).\n* Patients who the PI considers will not be able to comply with the study protocol.\n\nAmantadine Exclusion Criteria:\n\n* Patients in the manic phase of bipolar disorder.\n* Patients with history of proven peptic ulcer confirmed on endoscopy\n* Patients with active epilepsy\n* Already taking the IMP in this comparison\n* Known hypersensitivity, including hereditary fructose intolerance, or adverse reaction to the active substances and their excipients (as per SPCs for this comparison) or any past medical history contraindicating use of the IMP in this comparison\n\nTacrolimus Exclusion Criteria:\n\n* Poorly controlled hypertension (Systolic BP\\>180 mmHg or Diastolic BP\\>100mmHg)\n* Poorly controlled diabetes (HbA1c\\>6.4% or 48mmol/mol)\n* Hypertrophic cardiomyopathy or history of QT prolongation (including family history), congestive heart failure, bradyarrhythmias, and electrolyte abnormalities\n* History of bleeding disorders or significant haematological or immune diseases including, congenital or acquired immune deficiency, anaemia (Hb\\<130g/L for males and Hb\\<120 g/L in females) and thrombocytopenia (platelet count \\<150 \u00d7 109/L), use of other biological agents and immunosuppressant medications including oral/IV steroids\n* Active or chronic infection (at PI discretion)\n* History of Hepatitis B or C\n* History of lymphoma and active malignancy\n* Risk of dehydration due to reduced oral intake and lack of parenteral route\n* Patient's contraindicated to tacrolimus according to SPC section 4.3\n* Use of concomitant medications that interacts with tacrolimus according to the SPC, including but not limited to strong CYP3A4 inhibitors (i.e. azoles, protease inhibitors) or CYP3A4 inducers (rifampicin, phenytoin, carbamazepine), barbiturates, macrolides, digoxin, statins, PPI inhibitors, ergotamine, tricyclic antidepressants, herbal supplements (St. John's wort, extracts of Schisandra sphenanthera)\n* Inability to swallow capsules\n* Already taking the IMP in this comparison\n* Known hypersensitivity, including lactose and gelatin intolerance, or adverse reaction to the active substances and their excipients (as per SPCs for this comparison) or any past medical history contraindicating use of the IMP in this comparison\n* Receipt of a live attenuated vaccine within four weeks prior to receipt of tacrolimus. These include, but are not limited to live influenza vaccine (Fluenz Tetra), Shingles (varicella zoster virus) Zostavax, Varicella (Varilrix, Varilvax), Oral typhoid (Ty21a), and yellow fever vaccines.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Memantine Hydrochloride, Trazodone Hydrochloride, Amantadine Hydrochloride, Tacrolimus", "targeting_mechanism": "Amantadine is a weak NMDA receptor antagonist that increases dopamine release and blocks dopamine reuptake", "targeting_mechanism_pmid": "37166702", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07017946", "title": "Intestinal Microbiome Transplant in ALS", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Duke University", "summary": "This is a 24-week study of intestinal microbiome transplant in people with ALS. All participants will be evaluated clinically in person for 4 visits. Blood and mailed/ fresh stool samples will also be collected. Blood samples will be used to determine changes in neurofilament light chain over time. Stool samples will be processed for microbiome analysis. Participants will have phone visits to further evaluate safety and tolerability. They will then undergo antibiotic conditioning and a standard bowel preparation before being assigned to the investigational product, MTP-101C .", "interventions": [{"type": "DRUG", "name": "MTP-101C"}], "start_date": "2026-01-27", "url": "https://clinicaltrials.gov/study/NCT07017946", "target_entities": ["microbiome"], "locations": [{"facility": "Duke University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "RECRUITING", "lat": 35.99403, "lon": -78.89862}], "contact_phone": "919-613-2681", "contact_email": "ALSresearch@duke.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of ALS according to Gold Coast Criteria\n* Age: 18+ years at enrollment\n* Fast-progressing (ALSFRS-R change of at least 1.5 points per month between last 2 measurements at screening)\n* Modestly but not severely affected (ALSFRS-R score at or above 24 at screening)\n* Able to swallow capsules and expected to be able to for the duration of the trial (ALSFRS-R \"swallowing\" score of 3 or 4 at screening)\n* Expected to survive for the duration of the trial\n* Taking any combination of riluzole, edaravone, and/or tofersen at a stable dose for 30 days prior to screening, or not taking any of these and not expected to during the study.\n* Capable of giving written consent.\n* If sexually active, must agree to use contraceptive or abstinence for duration of treatment.\n* Females of child-bearing age must have negative pregnancy test at screening.\n\nExclusion Criteria:\n\n* Concurrent illness or laboratory abnormalities that could confound the measurement of ALS progression or the microbiome or interfere with the ability to complete the study.\n* Taking probiotics, nutraceuticals, or herbal remedies within 2 weeks of screening\n* Taking antibiotics within 3 months of screening.\n* Taking any investigational study drug within 30 days of screening or five half-lives of the prior agent.\n* Previous exposure to MTT.\n* Pregnancy.\n* Known specific food allergy with anaphylaxis\n* Other co-morbid conditions that, in the opinion of the study investigator, place the participant at increased risk of complications, interfere with study participation or compliance, or confound study objectives.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MTP-101C", "targeting_mechanism": "Microbiome-based therapeutic targeting gut dysbiosis to restore healthy gut microbiota composition and alleviate neurodegenerative disease progression through neuroimmune mechanisms", "targeting_mechanism_pmid": "32503641", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07595666", "title": "Comparison of NeuroEXPLORER PET/CT Versus Standard-of-care PET/CT of the Head and Neck Region.", "phase": "NA", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "prof. dr. Koen Van Laere", "summary": "The goal of this clinical study is to compare the current standard-of-care PET/CT scanner with the new NeuroEXPLORER PET/CT, with the aim of demonstrating the added diagnostic value of the new scanner. As this is the first device of its kind in Europe, the study will also evaluate the safety and general feasibility of the NeuroEXPLORER system for head and neck PET studies.\n\nDue to to its high resolution, the NeuroEXPLORER enables an accurate investigation of areas up to 20 times smaller than what can be visualized with standard clinical PET scanners, providing images with a very high level of detail. This scanner is specifically designed for imaging the brain, spinal cord, and neck region, to support the diagnosis and monitoring of neurological, psychiatric, and other disorders in the head and neck.\n\nThe main questions this study aims to answer are:\n\n* Does the NeuroEXPLORER provide more detailed information with better diagnostic quality compared to standard clinical PET/CT scans for head and neck disorders?\n* Is the NeuroEXPLORER system safe and effective in performing head and neck studies?\n\nParticipants will:\n\n* receive clear information about the study, including its organization, possible risks, and potential benefits. Based on this, they will be able to give their informed consent by signing an informed consent form before the study begins.\n* visit the clinic (UZ Leuven) once for two consecutive PET/CT scans: first the prescribed clinical scan, followed by a shorter scan with the NeuroEXPLORER.\n* before the start of the regular PET/CT scan, have a small catheter placed in a blood vessel in the forearm. Through this, a tracer will be administered. A tracer is a small amount of slightly radioactive material that helps to produce images of processes inside the body. Depending on the tracer used, the PET/CT scan may start immediately or after a short waiting period.\n* lie on their back on the scanner bed for 10 to 30 minutes during the clinical scan.\n* after the clinical scan, undergo a 15-20 minute scan on the NeuroEXPLORER.\n* leave the hospital after completing both scans.\n* afterwards, be asked whether they experienced any discomfort during the study. In addition, they will receive a one-time follow-up phone call within a week after the scan to check if they experienced any discomfort in the days following the study.", "interventions": [{"type": "DEVICE", "name": "Standard-of-care PET/CT"}, {"type": "DEVICE", "name": "NeuroEXPLORER"}], "start_date": "2025-09-30", "url": "https://clinicaltrials.gov/study/NCT07595666", "target_entities": [], "locations": [{"facility": "UZ Leuven", "city": "Leuven", "state": "Vlaams-Brabant", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* All subjects referred for standard-of-care PET in the indications for the substudies are eligible.\n\nExclusion Criteria:\n\n* Subject is unwilling to avoid unusual, unaccustomed, or strenuous physical activity beginning 4 days prior to tracer injection up to 1 day after tracer injection.\n\n  * Subject does not understand the study procedures.\n  * Subject is unwilling or unable to perform all of the study procedures or is considered unsuitable in any way by the principal investigator.\n  * Subject has had exposure to ionizing radiation (\\> 1 mSv) in other research studies within the last 12 months.\n  * Subject does not agree that incidental findings are communicated to the general practitioner and to the participant him/herself.\n  * Incapacitated patients that cannot give consent by themselves (in particular for substudies 1-3 in neurodegeneration).", "sex": "ALL", "min_age": "28 Days", "max_age": "", "healthy_volunteers": true, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06903286", "title": "Extension Study of Participants From SPG302-ALS-001", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Spinogenix", "summary": "This study will evaluate the long-term safety and efficacy of participants enrolled in SPG302-ALS-101 with Amyotrophic Lateral Sclerosis (ALS)", "interventions": [{"type": "DRUG", "name": "SPG302"}], "start_date": "2025-05-29", "url": "https://clinicaltrials.gov/study/NCT06903286", "target_entities": ["SMN1"], "locations": [{"facility": "Macquarie University", "city": "North Ryde", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.79677, "lon": 151.12436}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Flinders Medical Center", "city": "Adelaide", "state": "South Australia", "country": "Australia", "status": "", "lat": -34.92866, "lon": 138.59863}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n\u2022 Must have participated in all study activities of SPG302-ALS-001, the parent study\n\nExclusion Criteria:\n\n* Unable to reliably and regularly swallow whole oral medications on a daily basis.\n* Medical conditions that investigator or sponsor determine would interfere with participation in clinical trial", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "SPG302", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02665663", "title": "Tongue Strength in Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique Hopitaux De Marseille", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease with a poor prognosis that occurs in adults 64 years on average. Its prevalence is 4 to 6/100 000 inhabitants.\n\nSwallowing disorders occur during evolution and involve the prognosis of patients in the short term by the association of dysphagia with severe malnutrition, and aspiration. The issue of phoniatric monitoring is to detect early onset of the swallowing disorders to develop strategies for respiratory protection, food adapted to disturbances, and speech therapy.\n\nThe objective of this study is to compare the tongue force in patients with amyotrophic lateral sclerosis at the time of diagnosis and at the onset of swallowing disorders compared to healthy subjects, with the dynamic palatography device developed in the Laboratoire Parole et Langage (UMR 7309, CNRS-Universit\u00e9 Aix-Marseille, Aix-en-Provence), which allows the measurement of the strength and duration of the pression of the tongue on the palate.", "interventions": [{"type": "DEVICE", "name": "medical device"}], "start_date": "2016-02", "url": "https://clinicaltrials.gov/study/NCT02665663", "target_entities": [], "locations": [{"facility": "Service ORL Assistance Publique H\u00f4pitaux de Marseille", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}], "contact_phone": "04 91 38 60 71", "contact_email": "aude.lagier@ap-hm.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Adults and legally responsible/ valid medical insurance\n* patients : ALS diagnostic already announced\n* healthy subjects : EAT-10 score\\<2\n\nExclusion Criteria:\n\n* Non pregnant and non baby feeding\n* Presence of risk factor or suspicion of Creutzfeld Jacob Disease\n* Allergy/intolerance to the glueing paste\n* Antecedent of pathology of the aerodigestive tract\n* Other neurologic disease\n* Morphologic anomaly of the aerodigestive tract\n* Excessive gag reflex", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06008249", "title": "Platform Trial to Assess the Efficacy of Multiple Drugs in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Stichting TRICALS Foundation", "summary": "The objective of this phase III, placebo-controlled platform study is to investigate the efficacy of drugs for patients with ALS (Amyotrophic lateral sclerosis).", "interventions": [{"type": "DRUG", "name": "Lithium Carbonate 400 MG"}], "start_date": "2021-08-09", "url": "https://clinicaltrials.gov/study/NCT06008249", "target_entities": ["Glycogen synthase kinase-3"], "locations": [{"facility": "Flinders Medical Centre", "city": "Adelaide", "state": "", "country": "Australia", "status": "", "lat": -34.92866, "lon": 138.59863}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Brisbane", "state": "", "country": "Australia", "status": "", "lat": -27.46794, "lon": 153.02809}, {"facility": "Calvary Health Care Bethlehem", "city": "Parkdale", "state": "", "country": "Australia", "status": "", "lat": -37.99187, "lon": 145.08128}, {"facility": "Perron Institute", "city": "Perth", "state": "", "country": "Australia", "status": "", "lat": -31.95224, "lon": 115.8614}, {"facility": "The University of Sydney (Royal prince Alfred hospital)", "city": "Sydney", "state": "", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}, {"facility": "Concord hospital Sydney", "city": "Sydney", "state": "", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}, {"facility": "University Hospital Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "Utrecht", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Bellvitge University Hospital", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Karolinska University Hospital", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "King's College Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "University College London Hospital NHS", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "University Hospitals of North Midlands NHS Trust", "city": "Stoke-on-Trent", "state": "", "country": "United Kingdom", "status": "", "lat": 53.00415, "lon": -2.18538}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. \u2265 18 years at the time of screening.\n2. Diagnosis of ALS according to the revised El Escorial criteria (possible, probable-laboratory supported, probable or definite).\n3. Capable of providing informed consent and complying with trial procedures, including randomization to sub-studies.\n4. TRICALS risk profile \\> -6.0 and \\< -2.0 \\*\\*\n5. The use of riluzole will be permitted during the study. Subjects taking riluzole must be on a stable dose for at least 30 days prior to the baseline visit, or stopped taking riluzole at least 30 days prior to the baseline visit.\n6. Women of childbearing potential\\* must have a negative pregnancy test at baseline and be non-lactating.\n7. Men must agree to practice contraception for the duration of the trial and for at least 3 months after last dose of study drug.\n8. Men must not plan to father a child or to provide sperm for donation for the duration of the trial and 3 months after the last dose of study drug.\n9. Women must not be able to become pregnant (e.g. post-menopausal\\*\\*\\*, surgically sterile or using effective birth control methods) for the duration of the study. Effective contraceptives are defined as having a failure rate of less than 1% per year when used consistently and correctly and, when applicable, in accordance with the product label, including: abstinence, hormonal contraception, intrauterine device in place for \u2265 3 months Appendix 1). Women of childbearing potential must have a negative pregnancy test at baseline, and be non-lactating. Women who are pregnant or are actively seeking to become pregnant, and women of reproductive potential who are not using effective contraceptives are excluded.\n\nExclusion Criteria:\n\n1. Laboratory Criteria at baseline:\n\n   * ALT (alanine transaminase) \u2265 5 times upper limit of normal (ULN)\n   * AST (aspartate aminotransferase) \u2265 3 times ULN\n   * Bilirubin \u2265 1.5 times ULN\n   * Estimated glomerular filtration rate (eGFR) \\< 50 mL / min / 1.73 m2 based on Cystatin C, if not available eGFR can also be calculated based on creatinine clearance.\n   * Platelet concentration of \\< 100 x109 per L\n   * Absolute neutrophil count of \\< 1x109 per L\n   * Haemoglobin \\< 100 g/L (\\<6.2 mmol/L)\n   * Amylase \\& lipase \u2265 2 times ULN (suspected pancreatitis)\n   * Lactate \u2265 2 times ULN (suspected lactate acidosis)\n2. Moderate to severe hepatic impairment according to Child-Pugh classification (Class B or higher; score \u2265 7). Child-Pugh classification is based on bilirubin, albumin, International Normalized Ratio (INR) and presence of encephalopathy or ascites.\n3. Participation in any other investigational drug trial or using investigational drug (within 30 days prior to screening).\n4. Hypothyroidism unresponsive to thyroid hormone supplementation.\n5. Subjects using non-invasive ventilation (NIV, \u226522 h per day) or having a tracheostomy.\n6. Subjects taking edaravone within 30 days prior to screening. Edaravone is approved by the FDA, but remains an investigational product in Europe and Australia.\n7. Clinically significant history of unstable or severe cardiac (e.g. congestive heart failure, coronary insufficiency and arrhythmias), oncological, hepatic or renal disease, neuromuscular diseases, significant pulmonary disorder or other medically significant illness.\n8. Drug or alcohol abuse.\n9. Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days of the screening visit. This exclusion criterion is based on a prior psychiatric diagnosis that is unstable as determined by the subject's treating Psychiatrist.\n10. Presence of frontotemporal dementia which prevents informed consent.\n\nLithium carbonate study-specific exclusion criteria:\n\n1. Patients heterozygous or homozygous for the A-allele of rs12608932 (UNC13A)\n2. Known allergy or hypersensitivity to lithium, or its excipients, or to the components of the placebo.\n3. Brain injury with posttraumatic epilepsy or neurologic deficit, excluding a concussion in the medical history. Brain infarction is an exclusion criterion, a transient ischemic attack is not.\n4. Addison disease.\n5. Patients with the following co-medication: antipsychotics, digoxin and calcium antagonists, carbamazepine, methyldopa, verapamil and diltiazem.\n6. Brugada Syndrome or family history of Brugada Syndrome.\n7. Plasma sodium \\<120 mmol/L", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Lithium Carbonate", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04950933", "title": "The Treatment of Amyotrophic Lateral Sclerosis With Huollingshengji Granules", "phase": "PHASE2, PHASE3", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University Third Hospital", "summary": "This study intends to evaluate the efficacy and safety of Dong Lingsheng Ji Granule in the treatment of amyotrophic lateral sclerosis (spleen deficiency, kidney-yang deficiency syndrome) in comparison with riluzole, so as to provide data support for marketing application or subsequent clinical research design.", "interventions": [{"type": "DRUG", "name": "Huolingshengji Granules"}, {"type": "DRUG", "name": "Riluzole tablet"}], "start_date": "2020-06-01", "url": "https://clinicaltrials.gov/study/NCT04950933", "target_entities": ["riluzole"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijin", "state": "", "country": "China", "status": "RECRUITING", "lat": 35.93305, "lon": 111.40836}], "contact_phone": "+86 13701023871", "contact_email": "dsfan@sina.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Meet the Western diagnostic criteria for amyotrophic lateral sclerosis (ALS) (clinically confirmed ALS, clinically likely ALS or clinically likely ALS- laboratory support);\n2. The score of the modified amyotrophic lateral sclerosis function scale (ALSFRS-R) was \u22652 points for each item (among which dyspnea, sitting breathing and respiratory insufficiency were all 4 points);\n3. The percentage of forced vital capacity in the predicted value (FVC%) \u226570%;\n4. the duration of the disease is 3 years or less (from the first onset of any symptoms of ALS);\n5. TCM syndrome differentiation for deficiency of temper, kidney Yang deficiency syndrome;\n6. Age 45-70 (including 45 and 70), gender unlimited;\n7. Voluntarily participate in the clinical trial, give informed consent and sign informed consent.\n\nExclusion Criteria:(1) Patients diagnosed with familial ALS; (2) Those who have undergone gastrostomy; (3) patients with other neurological diseases similar to ALS, such as cervical spondylotic myelopathy, lumbar spondylopathy, dementia, etc., which may affect the evaluation of drug effectiveness; (4) electromyogram detection found motor nerve conduction block, sensory nerve conduction abnormality, imaging examination (CT or MRI) found that can explain the clinical manifestations of substantial lesions; (5) Patients who had been treated with riluzole or edaravone within 3 months before enrollment; (6) patients with a history of spinal surgery after the onset of ALS; (7) aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \\&gt; 1.5 times the upper limit of the normal reference value, or blood muscle (SCR) \\>; Upper limit of normal reference value; (8) Patients with other serious primary diseases of the nervous system, heart, lung, hematopoietic system or endocrine system and psychosis; (9) Suspected or have a history of alcohol and drug abuse; (10) Pregnant women or lactating women, subjects of reproductive age (including male subjects with heterosexual behavior and their female partners with fertility potential) have pregnancy plans or are unwilling to take effective contraceptive measures within 3 months from the beginning of screening to the end of drug withdrawal; (11) People who are known or suspected to have a history of allergy to the test drug and its excipients; (12) Screening participants who had participated in other clinical trials within the previous 3 months; (13) Those considered by the researcher to be unsuitable to participate in this clinical trial.", "sex": "ALL", "min_age": "45 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Huolingshengji Granules", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07341984", "title": "A Physiotherapy Intervention Study in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Charlotte Vogt", "summary": "This study investigates whether an individualized physiotherapy program, tailored to each patient's specific motor deficits, can better support physical function compared with usual care physiotherapy in people with ALS.\n\nThe individualized program is guided by diagnostic assessments using a robotic leg press system, which helps identify strengths and weaknesses in muscle function and movement control.\n\nParticipants will receive either individualized physiotherapy or standard physiotherapy and will be followed for 12 months. The aim of the study is to improve physiotherapy strategies for people with ALS in a safe and patient-centered manner.", "interventions": [{"type": "OTHER", "name": "Individualized Physiotherapy"}], "start_date": "2025-12-16", "url": "https://clinicaltrials.gov/study/NCT07341984", "target_entities": [], "locations": [{"facility": "ALS Clinic", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "RECRUITING", "lat": 47.42391, "lon": 9.37477}], "contact_phone": "0041714943581", "contact_email": "charlotte.vogt@h-och.ch", "eligibility": {"criteria": "Inclusion Criteria:\n\n* diagnosis of clinically probable, probable laboratory-supported, or definite ALS (revised El Escorial criteria) or upper motor neuron only (OPM classification \\[onset, propagation, motoneuron involvement\\])\n* age \u226518 years; ability to understand study information and provide written informed consent\n* Willingness and ability to perform individualized exercise according to the study protocol (approximately three to five sessions of 30 minutes per week) for the duration of the intervention period\n* Individuals of all sexes and gender identities are eligible for inclusion\n\nExclusion Criteria:\n\n* pregnancy, tracheostomy, continuous assisted ventilation, or other significant non-ALS pulmonary disease\n* other neurodegenerative or neuromuscular conditions that may confound assessments\n* concomitant life-threatening disease or impairment interfering with functional assessment", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00839033", "title": "Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "The hypothesis is that a mechanical insufflation-exsufflation (MI-E) is associated with a decrease in the number of intubations and more rapid clinical improvement in children and adults with neuromuscular disease who are admitted for an acute respiratory exacerbation.In this prospective, randomised, multicenter study, 55 patients will be treated with standard treatment and a MI-E, and 55 patients with standard treatment and standard respiratory physiotherapy. The primary objective is the reduction of the number of patients requiring invasive ventilatory support (endotracheal intubation or tracheotomy) in the group treated with MI-E (MI-E group). The main secondary objectives are a reduction in hospital stay and an improvement in clinical condition, dyspnea and respiratory muscle function.", "interventions": [{"type": "DEVICE", "name": "mechanical insufflation - exsufflation"}, {"type": "DEVICE", "name": "Standard respiratory physiotherapy"}], "start_date": "2009-06", "url": "https://clinicaltrials.gov/study/NCT00839033", "target_entities": [], "locations": [{"facility": "Hospital Armand Trousseau, Pediatric Pulmonology Department and INSERM UMR S-893", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nPediatric or adult patients with chronic neuromuscular disorders, such as spinal muscular atrophy, Duchenne muscular dystrophy, other congenital myopathy, or amyotrophic lateral sclerosis (ALS), hospitalized for acute respiratory failure, as defined by:\n\n* Persistent bronchial encumbrance (\\> 2 days) despite regular treatment in the homecare setting, associated with-Oxygen desaturation on room air, defined by a pulse oximetry (SaO2) \\<95%) or\n* In patients not receiving long-term NPPV: the need to institute NPPV-In patients receiving long-term NPPV: the need to increase the daily length of NPPV by at least 25%.\n\nExclusion Criteria:\n\n* Need for immediate intubation (alteration in consciousness, coma, hemodynamic disorders)\n* Multiple organ failure (e.g., associated cardiac failure)\n* In adults: respiratory rate \\>30/min, pH \\< 7.35, PaCO2 \\> 50 mm Hg\n* Facial deformity or anomaly which prevents the use of a mouthpiece or mask\n* Patients who signed a refusal to be intubated regardless of the progression of their disease\n* Patients on long-term oxygen therapy\n* Tracheotomized patients\n* Patients requiring the use of an intrapulmonary percussive ventilation device during hospitalization\n* Acute neuromuscular disorder of known or unknown etiology\n* Associated lung disease such as chronic obstructive pulmonary disease (COPD)\n* Refusal of patient consent and/or parental consent in the case of a minor\n* Uncooperative patients\n* Patients \\< 4 years old", "sex": "ALL", "min_age": "4 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00047723", "title": "Minocycline to Treat Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Institute of Neurological Disorders and Stroke (NINDS)", "summary": "The purpose of this trial is to test the safety, tolerability, and effectiveness of minocycline compared to placebo in patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "minocycline"}], "start_date": "2003-01", "url": "https://clinicaltrials.gov/study/NCT00047723", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Mayo Clinic", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "University of California, Irvine", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "University of California Department of Neurology", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Univ. of Colorado Health Sciences Center", "city": "Denver", "state": "Colorado", "country": "United States", "status": "", "lat": 39.73915, "lon": -104.9847}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Illinois", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Indiana University School of Medicine", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Kentucky", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Hennepin County Med Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "UMDNJ/Robert Wood Johnson Medical Center", "city": "New Brunswick", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.48622, "lon": -74.45182}, {"facility": "University of New Mexico", "city": "Albuquerque", "state": "New Mexico", "country": "United States", "status": "", "lat": 35.08449, "lon": -106.65114}, {"facility": "Columbia Unversity, Eleanor and Lou Gehrig MDA/ALS Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest University", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Metro Health Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Oregon Health & Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Drexel University College of Medicine, Hahnemann Campus", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Texas Southwestern", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Methodist Hospital", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Texas Health Sciences Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Vermont", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "Virginia Mason Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "To be eligible for enrollment in this study, subjects must meet the following eligibility criteria within fourteen days prior to randomization:\n\nInclusion criteria:\n\n* A clinical diagnosis of laboratory-supported probable, probable or definite ALS, according to modified EL Escorial criteria.\n* FVC greater or equal to 75% of predicted.\n* Onset of weakness within 3 years prior to enrollment.\n* If patients are receiving riluzole they must be on a stable dose for at least the past thirty days.\n* Women of childbearing age must be non-lactating and surgically sterile or using an effective method of birth control and have a negative pregnancy test (adequate birth control includes use of intra-uterine device or oral contraceptives plus a barrier method, e.g. condom, diaphragm).\n* Willing and able to give signed informed consent that has been approved by your Institutional Review Board (IRB).\n\nExclusion criteria:\n\n* Requirement for tracheotomy ventilation (or non-invasive ventilation \\> 23 hours/day).\n* Diagnosis of other neurodegenerative diseases (Parkinson's disease, Alzheimer's disease, etc).\n* FVC \\< 75% of predicted.\n* A clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc) over the last 30 days.\n* History of renal disease (screening creatinine greater than 1.5).\n* History of liver disease (screening alanine aminotransferase greater than 3 times the upper limit of normal).\n* History of hematologic disease (screening white blood cell count less than 3,800/mm3).\n* History of system lupus erythematosis (or screening ANA of 1:160 or greater).\n* Treatment with any medications that may cause lupus-like symptoms within 4 weeks of baseline visit (e.g. procainamide, hydralazine).\n* History of vestibular disease (excluding benign position vertigo).\n* Pregnancy or lactation.\n* Allergy to tetracycline antibiotics.\n* Use of minocycline within thirty days of enrollment (baseline visit).\n* Use of anti-epileptic medications other than gabapentin.\n* Limited mental capacity rendering the subject unable to provide written informed consent or comply with evaluation procedures.\n* History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols.\n* Use of any investigational drug within the past 30 days (Creatine, Vioxx, Celebrex, Topiramate).\n* Women with the potential to become pregnant who are not practicing effective birth control.", "sex": "ALL", "min_age": "21 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "minocycline", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03100110", "title": "NeuroCognitive Communicator: Safety Study", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ottawa Hospital Research Institute", "summary": "Individuals suffering from tetraplegia as a result of cervical spinal cord injury, brainstem stroke, or amyotrophic lateral sclerosis (ALS) cannot independently perform tasks of daily living. In many cases, these conditions do not have effective therapies and the only intervention is the provision of assistive devices to increase independence and quality of life. However, currently available devices suffer from usability issues and are limiting for both the patient and caregiver. One of the most progressive alternative strategies for assistive devices is the use of brain-computer interface (BCI) technology to translate intention signals directly from sensors in the brain into computer or device action. Preclinical primate research and recent human clinical pilot studies have demonstrated success in restoring function to disabled individuals using sensors implanted directly in motor regions of the brain. Other preclinical primate research has demonstrated effective intention translation from sensors implemented in cognitive regions of the brain and that this information complements information from the motor regions. The current proposal seeks to build on these studies and to test the safety aspects related to implanting two sensors, each a microelectrode array, into both the motor and cognitive regions of the brain in motor impaired humans. Secondary objectives include feasibility evaluation of the complementary sensors in their ability to support effective assistive communication.", "interventions": [{"type": "DEVICE", "name": "NeuroCognitive Communicator"}], "start_date": "2019-05-13", "url": "https://clinicaltrials.gov/study/NCT03100110", "target_entities": [], "locations": [{"facility": "The Ottawa Hospital", "city": "Ottawa", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 45.41117, "lon": -75.69812}], "contact_phone": "647-563-3141", "contact_email": "rdoole@ohri.ca", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Documented diagnosis of a complete or incomplete cervical spinal cord injury, with stable neurological deficits greater than 1 year, or ALS with equivalent degree of deficit.\n* Maintain some level of communication, enough to independently provide informed consent for the study.\n* Deemed healthy for surgery.\n* Good psychological and social stability.\n* Prospective participants with ALS must already have an advanced directive with regard to ventilation.\n* Live within a one-hour travel duration of the site.\n\nExclusion Criteria:\n\n* Presence of previous certain implanted devices.\n* In the opinion of the investigator, the presence of other serious disease or disorder that could affect ability to participate in this study.\n* Ongoing participation in another clinical trial.\n* Individuals who are immunosuppressed or who have conditions that typically result in immunocompromise (eg. chronic corticosteroid use, immunomodulators, chemotherapy).\n* Presence of clinical depression that is not medically optimized, as screened by a neuropsychologist on our team.\n* Presence of cognitive deficits, as assessed by a neuropsychologist on our team, that would preclude completion of some cognitively challenging tasks.\n* The participant has plans to move outside the study radius within the study period.", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NeuroCognitive Communicator", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02645461", "title": "Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Roma La Sapienza", "summary": "Amyotrophic lateral sclerosis (ALS) is a fatal disease leading to motor neuron degeneration and progressive paralysis. Other studies have revealed defects in skeletal muscle even in absence of motor neuron anomalies, focusing on acetylcholine receptors (AChRs) and supporting the so-called \"dying-back\" hypothesis. Outcome of this study will be to understand if the endocannabinoid palmitoylethanolamide (PEA) can reduce the rundown of AChRs currents in ALS muscle, and if it can modify ALS patients' clinical and electrophysiological parameters.", "interventions": [{"type": "DRUG", "name": "endocannabinoid palmitoylethanolamide (PEA)"}, {"type": "DRUG", "name": "Riluzole"}], "start_date": "2014-01", "url": "https://clinicaltrials.gov/study/NCT02645461", "target_entities": ["Acetylcholine receptors", "endocannabinoid_signaling"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of ALS according to the El-Escorial criteria;\n* Age\\> 18 years;\n* ALS Functional Rating Scale-Revised (ALSFRS- r) score\\> 20;\n* Forced Vital Capacity (FVC)\\> 30%;\n* Treatment with Riluzole.\n\nExclusion Criteria:\n\n* Other diseases motor neurons;\n* Experimental treatments in the previous three months;\n* Pregnant or breast-feeding;\n* Contraindications to the use of riluzole;\n* Patients undergoing tracheostomy, enteral or parenteral supply;\n* Severe psychiatric disorders.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "palmitoylethanolamide (PEA)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01089010", "title": "A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "The primary objective of this study is to demonstrate a pharmacodynamic effect of CK 2017357 on measures of skeletal muscle function or fatigability in patients with ALS.", "interventions": [{"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "250 mg CK-2017357"}, {"type": "DRUG", "name": "500 mg CK-2017357"}], "start_date": "2010-03", "url": "https://clinicaltrials.gov/study/NCT01089010", "target_entities": ["skeletal_muscle_function"], "locations": [{"facility": "Phoenix Neurological Associates, Ltd.", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University Neurology Associates", "city": "Fresno", "state": "California", "country": "United States", "status": "", "lat": 36.74773, "lon": -119.77237}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Kentucky", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "SUNY Upstate Medical Center", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Drexel University College of Medicine, Dept of Neurology", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Penn State", "city": "University Park", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.80201, "lon": -77.85639}, {"facility": "The University of Texas Health Science Center at San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Vermont", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria\n\nFor enrollment, patients were required to satisfy all of the following criteria at baseline:\n\n1\\. Able to comprehend and willing to sign an Informed Consent Form (ICF)\n\n1. A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria) (Brooks, Miller et al. 2000)\n2. Males or females 18 years of age or older\n3. Body Mass Index (BMI) of 18.0 to 30.0 kg/m2, inclusive\n4. Maximum voluntary grip strength in at least one hand between 10 and 40 pounds (females) or 10 and 60 pounds (males)\n5. Able to swallow capsules with water\n6. Upright Slow Vital Capacity (SVC) \\> 40% of predicted for age, height, and sex \\[See Appendix 16.6.1\\]\n7. Able to perform pulmonary function tests\n8. Pre-study clinical laboratory findings (including troponin I \\[TnI\\] and creatine phosphokinase \\[CPK\\]) within normal range, or, if outside of the normal range, deemed not clinically significant by the Investigator\n9. For female patients only: The patient is post-menopausal (\u2265 1 year) or sterilized, or if she is of childbearing potential, she is not breastfeeding, her pregnancy test is negative, she has no intention to become pregnant during the course of the study, and she is using contraceptive drugs or devices for the duration of the study and for 10 weeks after the end of the study.\n\nFor male patients only: Male patients agree for the duration of the study and 10 weeks after the end of the study to use a condom during sexual intercourse with female partners who are of reproductive potential and to have female partners use an additional effective means of contraception (e.g., diaphragm plus spermicide or oral contraceptives) or the male patient must agree to abstain from sexual intercourse for 10 weeks after the end of the study.\n\nExclusion Criteria\n\nPatients satisfying any of the following criteria at baseline were excluded from enrollment:\n\n1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 3 times the upper limit of normal (ULN)\n2. Life expectancy \\< 3 months\n3. Participation in any trial in which receipt of investigational study drug occurred within 30 days prior to dosing\n4. Any prior treatment with CK-2017357\n5. In the opinion of the Investigator, the patient is not suitable to participate in the study", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CK-2017357", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03800524", "title": "Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS", "phase": "PHASE3", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Humanitas Mirasole SpA", "summary": "This is a Phase III, multi-centre, randomized, double-blind, placebo-controlled, parallel-group study to evaluate Safety and Efficacy of Tauroursodeoxycholic (TUDCA) as add-on Treatment in Patients Affected by Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Tauroursodeoxycholic Acid"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2019-02-22", "url": "https://clinicaltrials.gov/study/NCT03800524", "target_entities": ["apoptosis"], "locations": [{"facility": "Katholieke Universiteit Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Centre Hospitalier Universitaire de Bordeaux", "city": "Bordeaux", "state": "", "country": "France", "status": "", "lat": 44.84124, "lon": -0.58046}, {"facility": "Centre Hospitalier Universitaire Limoges", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Centre Hospitalier Universitaire de Montpellier", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Centre Hospitalier Regional Universitaire de Tours", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Charit\u00e9 - Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Universit\u00e4tsklinikum Carl Gustav Carus Dresden", "city": "Dresden", "state": "", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Alfried Krupp Krankenhaus R\u00fcttenscheid", "city": "Essen", "state": "", "country": "Germany", "status": "", "lat": 51.45657, "lon": 7.01228}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4tsklinikum Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Universit\u00e4t Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Trinity College Dublin", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "IRCCS Istituto Auxologico Italiano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "NEuroMuscular Omnicentre. Fondazione Serena Onlus", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "AOU Universit\u00e0 degli Studi della Campania \"Luigi Vanvitelli\"", "city": "Naples", "state": "", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}, {"facility": "IRCCS Istituto Clinico Humanitas", "city": "Rozzano", "state": "", "country": "Italy", "status": "", "lat": 45.38193, "lon": 9.1559}, {"facility": "Azienda Ospedaliera Santa Maria di Terni", "city": "Terni", "state": "", "country": "Italy", "status": "", "lat": 42.56335, "lon": 12.64329}, {"facility": "AOU Citt\u00e0 della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "The Walton Centre NHS Foundation Trust", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "Plymouth Hospitals NHS Trust", "city": "Plymouth", "state": "", "country": "United Kingdom", "status": "", "lat": 50.37153, "lon": -4.14305}, {"facility": "Lancashire Teaching Hospitals NHS Foundation Trust", "city": "Preston", "state": "", "country": "United Kingdom", "status": "", "lat": 53.76282, "lon": -2.70452}, {"facility": "Salford Royal NHS Foundation Trust", "city": "Salford", "state": "", "country": "United Kingdom", "status": "", "lat": 53.48771, "lon": -2.29042}, {"facility": "University of Sheffield", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}, {"facility": "Royal Stoke University Hospital", "city": "Stoke", "state": "", "country": "United Kingdom", "status": "", "lat": 53.25, "lon": -2.86667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Probable laboratory-supported, probable, or definite ALS, as defined by El Escorial Revised ALS diagnostic criteria at screening visit (month -3)\n* Disease duration \u2264 18 months at screening visit (month -3)\n* Able to perform reproducible pulmonary function tests at screening visit (month -3)\n* Forced vital capacity or slow vital capacity \u226570% of normal at screening visit (month -3)\n* Stable on riluzole treatment for 3 months in the lead-in period\n* Signed informed consent at screening visit (month -3)\n\nExclusion Criteria:\n\n* Treatment with edaravone\n* Other causes of neuromuscular weakness\n* Presence of other neurodegenerative diseases\n* Significant cognitive impairment, clinical dementia or psychiatric illness\n* Severe cardiac or pulmonary disease\n* Other diseases precluding functional assessments\n* Other life-threatening diseases\n* Any use of non-invasive ventilation (e.g. continuous positive airway pressure, non-invasive bi-level positive airway pressure or non-invasive volume ventilation) for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation\n* Gastrointestinal disorder that is likely to impair absorption of study drug from the gastrointestinal tract\n* Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is longer, prior to dosing\n* Any clinically significant laboratory abnormality\n* Other concurrent investigational medications\n* Active peptic ulcer\n* Previous surgery or infections of small intestine\n* Patients unable to easily swallow the treatment pills\n* Acute inflammation of the gallbladder or bile ducts\n* Occurrence of frequent biliary colic, biliary infections, severe pancreatic abnormalities\n* Bile duct obstruction, calcified X-ray opaque gallstones and reduced mobility of the gallbladder\n* Subjects who weigh 88 lbs (40 kg) or less\n* Aspartate aminotransferase or alanine aminotransferase concentrations more than 3 times the upper limit of normal\n* Creatinine clearance 50 ml/min or less\n* Any clinically significant neurological, haematological, autoimmune, endocrine, cardiovascular, neoplastic, renal, gastrointestinal, or other disorder that, in the Investigator's opinion, could interfere with the subject's participation in the study, place the subject at increased risk, or confound interpretation of study results\n* Consideration by the investigator, for any reason, that the subject is an unsuitable candidate to receive TUDCA or that the subject is unable or unlikely to comply with the dosing schedule or study evaluations\n* The patient of reproductive potential is sexually active and is not willing to use highly effective contraception during the study and up to 90 days after the day of last dose\n* The patient is pregnant or breast feeding", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tauroursodeoxycholic Acid (TUDCA)", "targeting_mechanism": "Tauroursodeoxycholic acid exhibits anti-apoptotic and neuroprotective activities against neurodegenerative diseases mediated by protein homeostasis and apoptotic pathways.", "targeting_mechanism_pmid": "35659112", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03705390", "title": "A Safety and Tolerability Study of ILB\u00ae in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Birmingham", "summary": "This is a phase II study to determine the safety and tolerability of ILB\u00ae, a type of low molecular weight dextran sulfate, in patients with Motor Neurone Disease (MND)/ Amyotrophic Lateral Sclerosis (ALS)", "interventions": [{"type": "DRUG", "name": "ILB\u00ae"}], "start_date": "2019-03-29", "url": "https://clinicaltrials.gov/study/NCT03705390", "target_entities": ["neuroinflammation"], "locations": [{"facility": "University Hospitals Birmingham NHS Foundation Trust", "city": "Birmingham", "state": "West Midlands", "country": "United Kingdom", "status": "", "lat": 52.48142, "lon": -1.89983}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Patients \u226518 years and who have provided written informed consent to participate in the study\n2. Prior to trial entry patients will have a definite diagnosis of ALS according to El Escorial Criteria. All patients will demonstrate either:\n\n   * presence of Upper Motor Neuron (UMN) (increased tone, brisk reflexes) as well as Lower Motor Neuron (LMN) (weakness,\n   * wasting and fasciculation) signs in the bulbar region and at least two of the other spinal regions (cervical, thoracic or lumbosacral) or\n   * presence of UMN and LMN signs in all three spinal regions (cervical, thoracic or lumbosacral)\n3. Electrophysiological tests (Electromyography (EMG) / Nerve Conduction Study (NCS)) that supports the diagnosis of Motor Neurone Disease (MND) and to exclude mimic disorders\n4. Forced Vital Capacity (FVC) \u226550% of predicted value for gender, height and age at screening and a mean Sniff Nasal Inspiratory Pressure (SNIP) \u226550% of predicted value for age\n5. Adequate haematological function (Hb\u226510g/dl absolute neutrophil count \u22651.5x109/L and a platelet count \u226560 x109/L\n6. International Normalised Ratio (INR) \u2264 1.5, Activated Partial Thromboplastin Time (aPTT) 30 - 40 seconds, Prothrombin Time (PT) 11-13.5 seconds\n7. Patient willing and able to comply with schedule visits, treatment plan and other study procedures.\n8. Patients taking Riluzole must have discontinued treatment \u226528 days prior to study entry (and following consent to take part in the study)\n9. Women Of Child Bearing Potential (WOCBP) who agree to use highly effective means of contraception (as defined in the Heads of Medicines Agencies\\_Clinical Trials Facilitation Group (HMA\\_CTFG) guideline (see Appendix 8) and in combination with a barrier contraception method (condom, diaphragm or cap) for the entirety of the study\n\nExclusion Criteria:\n\n1. Patients classified as either probable or possible ALS according to El Escorial Criteria.\n2. Subjects in whom other causes of neuromuscular weakness have not been excluded\n3. Assisted ventilation of any type within 3 months before the screening visit or at screening\n4. Patients requiring Radiologically Inserted Gastrostomy (RIG) or Percutaneous Endoscopic Gastroscopy (PEG) feeding\n5. Involvement in any other interventional study involving use of another Investigational Medicinal Product (IMP) or biological product, within 3 months of screening\n6. Any use of antioxidants, edaravone, tirasemtiv or CK-2127107 within 1 month before the screening visit\n7. Any botulinum toxin use within 3 months before the screening visit.\n8. Any form of stem cell or gene therapy for the treatment of amyotrophic lateral sclerosis (ALS)\n9. Neuroimaging of brain and cervical spine with Magnetic Resonance imaging (MRI) indicating compressive myelopathy as an alternate diagnosis\n10. Laboratory examinations including Acetylcholine receptor (AChR) antibodies and Muscle Specific Kinase (MuSK) antibodies to exclude Bulbar onset Myasthenia gravis from Bulbar onset Motor neuron disease as an alternate diagnosis and Antinuclear Antibodies (ANA), Anti-neutrophil cytoplasmic antibodies (ANCA), Extractable Nuclear Antigen (ENA) antibodies, Creatine Kinase (CK), electrophoresis and immunoglobulin indicating an alternate diagnosis for muscle disease like Myositis\n11. Abnormal liver function defined as Aspartate Transaminase (AST) and/or Alanine Transaminase (ALT) \\>3 times upper limit of normal\n12. Any head trauma, intracranial or spinal surgery within 3 months of trial entry\n13. Patients who have had recurrent falls will be excluded to reduce the risk of intracerebral haemorrhage with this IMP\n14. Current use of an anticoagulant e.g Warfarin, Aspirin, Clopidogrel, any novel anticoagulants (NOAC)s or low molecular weight subcutaneous heparin\n15. Uncontrolled severe hypertension defined as systolic blood pressure (SBP) \u2265 220 mmHg or diastolic blood pressure (DBP) \u2265120 mmHg\n16. Current or previous history of heparin-induced thrombocytopenia\n17. Active peptic ulcer disease\n18. Known hypersensitivity to sulphur\n19. Severe liver insufficiency\n20. Patients with evidence of major psychiatric illness, significant cognitive impairment or clinically evident dementia that may interfere with the patients' ability to comply with study procedures\n21. Pulmonary illness (e.g asthma or Chronic Obstructive Pulmonary Disease (COPD)) requiring regular treatment\n22. Patient judged to be actively suicidal by the investigator during 3 months before the screening visit\n23. Subjects with a diagnosis of another neurodegenerative disease (e.g. Parkinson's disease, Alzheimer's disease and Frontotemporal dementia)\n24. Pregnant and/or breastfeeding.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ILB\u00ae (low molecular weight dextran sulfate)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05286372", "title": "An Intermediate Size Expanded Access Protocol of AMX0035 for ALS", "phase": "Expanded Access", "status": "APPROVED_FOR_MARKETING", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Amylyx Pharmaceuticals Inc.", "summary": "The Expanded Access Program will provide access and assess the safety of AMX0035 for the treatment of people living with ALS.", "interventions": [{"type": "DRUG", "name": "AMX0035"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT05286372", "target_entities": ["TARDBP"], "locations": [{"facility": "University of Southern California", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "The Kaiser Permanente Medical Group", "city": "Oakland", "state": "California", "country": "United States", "status": "", "lat": 37.80437, "lon": -122.2708}, {"facility": "Nova Southeastern University", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "Holy Cross Health", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "The Sean M. Healey & AMG Center for ALS Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Neurology Associates, P.C. / Somnos Clinical Research", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Duke University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Thomas Jefferson University Weinberg ALS Center", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Texas Tech University Health Sciences Center El Paso", "city": "El Paso", "state": "Texas", "country": "United States", "status": "", "lat": 31.75872, "lon": -106.48693}, {"facility": "Virginia Commonwealth University", "city": "Richmond", "state": "Virginia", "country": "United States", "status": "", "lat": 37.55376, "lon": -77.46026}, {"facility": "Swedish Neuroscience Institute", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "University of Washington School of Medicine", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Providence St. Luke's Rehabilitation Medical Center", "city": "Spokane", "state": "Washington", "country": "United States", "status": "", "lat": 47.65966, "lon": -117.42908}, {"facility": "University of Wisconsin", "city": "Madison", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.07305, "lon": -89.40123}, {"facility": "Hispanic Alliance for Research & Translational Research", "city": "San Juan", "state": "", "country": "Puerto Rico", "status": "", "lat": 18.46633, "lon": -66.10572}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female, at least 18 years of age (inclusive);\n* Diagnosis of ALS made by a physician experienced with the management of ALS;\n* \\>36 months from symptom onset defined as first weakness\n* Capable of providing informed consent;\n* Capable of and willing to follow program procedures.\n* Participants who have established care with a physician experienced in treating patients with ALS involved in the program and will maintain this clinical care throughout the duration of their time in the program.\n* Women of childbearing potential (e.g., not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the program and 3 months after last dose of AMX0035;\n\n  * Women must not be planning to become pregnant for the duration of the program and 3 months after last dose of study drug\n* Men must agree to practice contraception for the duration of the program and for at least 3 months after last dose of program drug;\n\n  * Men must not plan to father a child or provide sperm\n\nExclusion Criteria\n\n* Currently enrolled in a therapeutic study involving the use of an investigational therapy;\n* Dependence on invasive mechanical ventilation, defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use; presence of tracheostomy at Screening;\n\n  * No current need for tracheostomy or PAV (defined as more than 22 hours daily of mechanical ventilation for more than one week (7 days) or based on the site investigator's judgment; no need anticipated for the next 12 weeks\n* In the judgment of the Investigator, the participant's expected survival is less than 6 months\n* History of known allergy to the following: PB, bile salts, excipient/constituents of the formulation;\n* Abnormal liver function defined as AST and/or ALT \\>3 times the upper limit of the normal (obtained within 12 weeks from first dose);\n* Renal insufficiency as defined by eGFR \\<60 mL/min/1.73m2 (obtained within 12 weeks from first dose);\n* Pregnant women or women currently breastfeeding;\n* Current severe biliary disease which may result in the investigator medical judgement in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder;\n* History of Class III/IV heart failure (per New York Heart Association - NYHA);\n* Participant under severe salt restriction where the added salt intake due to treatment would put the participant at risk, in the Site Investigator clinical judgment;\n* Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed c consent, according to Site Investigator judgment;\n* Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant if they were to participate in the program, according to Site Investigator judgment;\n* Treatment, current or within 90 days from screening with any cell therapies or gene therapies;\n* Implantation of Diaphragm Pacing System (DPS);\n* Anything that, in the opinion of the Site Investigator, would place the participant at increased risk or preclude the participant's full compliance with or completion of the program;\n* Current or planned exposure to any prohibited medications listed in Section 6.8.1 of the protocol", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AMX0035", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04950647", "title": "Efficacy and Safety of Nitrazine in the Treatment of ALS", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University Third Hospital", "summary": "To evaluate the trend of safety and effectiveness of Nitroketazine tablets for ALS patients, and to explore the best effective dose.", "interventions": [{"type": "DRUG", "name": "Nitrofurazone Group 1"}, {"type": "DRUG", "name": "Nitroketazine Group 2"}, {"type": "DRUG", "name": "placebo group"}], "start_date": "2020-07-01", "url": "https://clinicaltrials.gov/study/NCT04950647", "target_entities": [], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "RECRUITING", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "+86 13701023871", "contact_email": "dsfan@sina.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 45-70, gender unlimited (including 45 and 70);\n2. diagnoses in accordance with the confirmed and proposed ALS diagnostic standards ofthe Revised World Federation of Neurology (1998);\n3. The duration of disease from onset to randomization of subjects is less than 2 years;\n4. Before randomization, ALSFRS-R scores were \u22652 points, and respiratory function items were 4 points;\n5. ALSFRS-R decreased by 1-4 points in screening period 3A (\u22651 and lt; 4);\n6. Random pre-respiratory function Forced Vital Capacity (%FVC) \u226580%;\n7. Understand and abide by the test procedures, participate voluntarily, and sign the informed consent (the informed consent should be signed by the person or the guardian voluntarily).\n\nExclusion Criteria:\n\n1. Familial ALS (judged by family history);\n2. Patients with significant cognitive impairment (MMSE: illiteracy group \\&lt; 19 points, elementary school. 22 points, S26 points in the junior high school and above group (more than 8 years of education);\n3. obvious dysphagia;\n4. Severe renal insufficiency: creatinine clearance. 30 mL/min (Cockcroft-Gault formula), or other known severe renal insufficiency;\n5. Severe liver function impairment: ALT, AST\\&GT; 3 times the upper limit of normal value, or other known liver diseases such as acute or chronic active hepatitis, cirrhosis, etc.;\n6. In the screening stage, patients with heart failure who developed acute myocardial infarction or underwent interventional therapy within the last 6 months (grade II1-IV according to NYHA);\n7. Complicated with malignant tumors, serious diseases of blood, digestion or other systems.\n8. Allergic to experimental drugs or ligustrazine;\n9. Pregnancy and lactation;\n10. Participated in, or is participating in, other clinical trials within 30 days prior to screening;\n11. The investigator did not consider it appropriate to participate in this study.", "sex": "ALL", "min_age": "45 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Nitroketazine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00573443", "title": "Safety and Efficacy of AVP-923 in PBA Patients With ALS or MS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Avanir Pharmaceuticals", "summary": "Objectives of the study are to evaluate the safety, tolerability, and efficacy of two different doses of AVP-923 (capsules containing either 30 mg of dextromethorphan hydrobromide and 10 mg of quinidine sulfate \\[AVP-923-30\\] or 20 mg of dextromethorphan hydrobromide and 10 mg of quinidine sulfate \\[AVP-923-20\\]) when compared to placebo, for the treatment of PBA in a population of patients with amyotrophic lateral sclerosis (ALS) or multiple sclerosis (MS) over a 12-week period. An additional objective is to determine the pharmacokinetic parameters of the two different doses of AVP-923 in a subset of the study population.\n\nPseudobulbar Affect (PBA) is a condition characterized by involuntary, sudden and frequent episodes of laughing and/or crying out of proportion or incongruous to the underlying emotion of happiness or sadness Other terms used to describe this condition include emotional lability, emotionalism, emotional incontinence, emotional discontrol, excessive emotionalism, and pathological laughing and crying. The outbursts can occur spontaneously or in response to provocative stimuli such as questions or events.\n\nA body of evidence suggests that PBA can be modulated through pharmacologic intervention.\n\nDextromethorphan (DM) is a low-affinity uncompetitive antagonist of the N-Methyl-D-aspartate (NMDA) receptor, reducing the level of excitatory activity. DM also acts at the phencyclidine-binding site, which is part of the NMDA receptor complex. DM is a sigma receptor agonist, suppressing the release of excitatory neurotransmitters.\n\nQuinidine (Q) is a known potent inhibitor of cytochrome P450 2D6 (CYP2D6), that decreases the metabolism of dextromethorphan and helps to achieve sustained and therapeutic levels of this drug.", "interventions": [{"type": "DRUG", "name": "dextromethorphan hydrobromide 20 mg and quinidine sulfate 10 mg"}, {"type": "DRUG", "name": "dextromethorphan hydrobromide 30 mg and quinidine sulfate 10 mg"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2007-12", "url": "https://clinicaltrials.gov/study/NCT00573443", "target_entities": ["pseudobulbar_affect"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Neuromuscular Research Center", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "South Coast Clinical Trials", "city": "Anaheim", "state": "California", "country": "United States", "status": "", "lat": 33.83529, "lon": -117.9145}, {"facility": "UCI Medical Center", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "Center for Neurologic Study", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "UCLA School of Medicine", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "The Forbes Norris MDA/ALS Research Center - California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "The ALS Center at UCSF", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado at Denver & Health Science Center", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Neuroscience Center", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Suncoast Neuroscience Associates", "city": "St. Petersburg", "state": "Florida", "country": "United States", "status": "", "lat": 27.77086, "lon": -82.67927}, {"facility": "The ALS Center at Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Neurology Specialists of Decatur of Decatur", "city": "Decatur", "state": "Georgia", "country": "United States", "status": "", "lat": 33.77483, "lon": -84.29631}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Consultants in Neurology", "city": "Northbrook", "state": "Illinois", "country": "United States", "status": "", "lat": 42.12753, "lon": -87.82895}, {"facility": "University of Kentucky Health Care - Dept. of Neurology", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "The John Hopkins Universitiy", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusets General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Baystate Medical Center", "city": "Springfield", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.10148, "lon": -72.58981}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Hospital", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "St.Louis University - Neuromuscular Clinic", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Advanced Neurology Specialists", "city": "Great Falls", "state": "Montana", "country": "United States", "status": "", "lat": 47.50024, "lon": -111.30081}, {"facility": "Neurology Associates", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Universitiy of Nevada", "city": "Las Vegas", "state": "Nevada", "country": "United States", "status": "", "lat": 36.17497, "lon": -115.13722}, {"facility": "Upstate Clinical Research", "city": "Albany", "state": "New York", "country": "United States", "status": "", "lat": 42.65258, "lon": -73.75623}, {"facility": "Jacobs Neurological Institute", "city": "Buffalo", "state": "New York", "country": "United States", "status": "", "lat": 42.88645, "lon": -78.87837}, {"facility": "Mount Sinai Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Neurological Institute - Columbia Presbyterian Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke Universitiy Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Department of Neurology - The Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Ohio State Universitiy", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Oregon Health Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Drexel University - Department of Neurology", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "The ALS Center - Penn Neurological Institute - The University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Vanderbilt University", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "The Methodist Hospital - Baylor College of Medicine", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Department of Neuropsychiatry - Texas Tech University", "city": "Lubbock", "state": "Texas", "country": "United States", "status": "", "lat": 33.57786, "lon": -101.85517}, {"facility": "University of Texas Health Science Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "Universitiy of Vermont", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "West Virginia University", "city": "Morgantown", "state": "West Virginia", "country": "United States", "status": "", "lat": 39.62953, "lon": -79.9559}, {"facility": "Dean Foundation", "city": "Madison", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.07305, "lon": -89.40123}, {"facility": "FACENE", "city": "Buenos Aires", "state": "Buenos Aires F.D.", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "IADIN", "city": "Buenos Aires", "state": "Buenos Aires F.D.", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "Hospital Italiano", "city": "Buenos Aires", "state": "Buenos Aires F.D.", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "INEBA", "city": "Buenos Aires", "state": "Buenos Aires F.D.", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "Hospital Ramos Mejia", "city": "Buenos Aires", "state": "Buenos Aires F.D.", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "Hospital Britanico", "city": "Buenos Aires", "state": "Buenos Aires F.D.", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "Policlinico Bancario", "city": "Buenos Aires", "state": "Buenos Aires F.D.", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "FLENI", "city": "Buenos Aires", "state": "Buenos Aires F.D.", "country": "Argentina", "status": "", "lat": -34.61315, "lon": -58.37723}, {"facility": "Hospital Militar Regional de Cordoba", "city": "C\u00f3rdoba", "state": "C\u00f3rdoba Province", "country": "Argentina", "status": "", "lat": -31.40648, "lon": -64.18853}, {"facility": "Instituto Medico Rodriguez Alfici", "city": "Godoy Cruz", "state": "Mendoza Province", "country": "Argentina", "status": "", "lat": -32.92533, "lon": -68.84428}, {"facility": "Instituto de Neurociencias Rosario", "city": "Rosario", "state": "Santa Fe Province", "country": "Argentina", "status": "", "lat": -32.94682, "lon": -60.63932}, {"facility": "Santa Casa de Misericordia de Belo Horizonte", "city": "Belo Horizonte", "state": "M G", "country": "Brazil", "status": "", "lat": -19.92083, "lon": -43.93778}, {"facility": "Hospital de Cl\u00ednicas-UFPR", "city": "Curitiba", "state": "Paran\u00e1", "country": "Brazil", "status": "", "lat": -25.42778, "lon": -49.27306}, {"facility": "Hospital da Restaura\u00e7\u00e3o", "city": "Recife", "state": "Pernambuco", "country": "Brazil", "status": "", "lat": -8.05389, "lon": -34.88111}, {"facility": "Hospital Universit\u00e1rio Clementino Fraga Filho", "city": "Rio de Janeiro", "state": "Rio de Janeiro", "country": "Brazil", "status": "", "lat": -22.90642, "lon": -43.18223}, {"facility": "Hospital Moinhos de Vento", "city": "Porto Alegre", "state": "Rio Grande do Sul", "country": "Brazil", "status": "", "lat": -30.03283, "lon": -51.23019}, {"facility": "Hospital das Cl\u00ednicas da Faculdade de Medicina da Universidade S\u00e3o Paulo", "city": "S\u00e3o Paulo", "state": "S\u00e3o Paulo", "country": "Brazil", "status": "", "lat": -23.5475, "lon": -46.63611}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Main Inclusion Criteria:\n\n* The patient has a diagnosis of Amyotrophic Lateral Sclerosis (according to El Escorial Criteria, WFN, 1998) and the time from diagnosis of ALS is not be longer than 30 months, or the patient has a diagnosis of multiple sclerosis or probable multiple sclerosis (according to McDonald criteria, 2001)\n* The patient has a clinical history and clinical relevant symptoms of Pseudobulbar Affect (PBA)\n* CNS-LS score at baseline is 13 or greater\n\nMain Exclusion Criteria:\n\n* Patients with myasthenia gravis\n* Any personal history of complete heart block, QTc prolongation, or torsades de pointes\n* Any family history of congenital QT interval prolongation syndrome\n* Patients with known sensitivity to quinidine, dextromethorphan or opiate drugs (codeine, etc.)", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "dextromethorphan hydrobromide and quinidine sulfate (AVP-923)", "targeting_mechanism": "Dextromethorphan is an NMDA receptor antagonist that suppresses uncontrollable emotional expression (pseudobulbar affect)", "targeting_mechanism_pmid": "28070747", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "labile emotionality (pseudobulbar affect)", "repurposed_from_pmid": "28070747"}}
{"nct_id": "NCT02405403", "title": "Microglial Activation Role In ALS (MARIA)", "phase": "EARLY_PHASE1", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Tours", "summary": "Neuroinflammation, characterized in particular by microglia activation, is an essential component of Amyotrophic Lateral Sclerosis (ALS) pathogenesis. Translocator Protein (TSPO) is recognized as a specific and sensitive biomarker of neuroinflammation, reflecting disease activity. An experimental radiopharmaceutical specific of TSPO expression, namely \\[18F\\]DPA714, allow to quantify this microglial activation using Positon Emission Tomography (PET) imaging.\n\nThe purpose of this study is to longitudinally correlate the spatial distribution of neuroinflammation with the pro- or anti-inflammatory state of activated microglia cells in ALS, in order to evaluate neurotoxic or neuroprotective microglia activity, by complementary approaches in 20 ALS patients:\n\n* in vitro: measuring concentrations of several pro- and anti-inflammatory cytokines secreted by microglial cells in the cerebrospinal fluid (CSF).\n* in vivo: \\[18F\\]DPA714 PET imaging. These assays will be performed in the framework of the clinical follow-up of ALS patients, at the diagnosis of ALS disease and 6 months latter.", "interventions": [{"type": "DRUG", "name": "[18F]DPA-714 PET"}], "start_date": "2015-03", "url": "https://clinicaltrials.gov/study/NCT02405403", "target_entities": ["neuroinflammation", "TSPO"], "locations": [{"facility": "University Hospital", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Signed informed consent\n* Age \u2265 18 years old\n* Patient with probable or definite sporadic Amyotrophic Lateral Sclerosis (ALS) form according to the modified criteria of El Escorial\n* Treated with riluzole 2 weeks\n* Evolution less than 18 months\n* Mini-Mental State Examination (MMS) score \u2265 26 and Frontal Assessment Battery (FAB) (normal)\n* Affiliated to a social security system\n\nExclusion Criteria:\n\n* Another unbalanced progressive pathology\n* Vascular diseases (hypertension, diabetes, smoking, dyslipidemia) unbalanced\n* Forced vital capacity \\<75%\n* Weight loss\\> 10% of the weight before disease\n* Status \"low affinity binder\" or \"mixed affinity binder\", the TSPO respect to the \\[18 F\\] DPA-714, which can interfere with the process of neuroinflammation: drugs with anti-inflammatory drugs (NSAIDs, corticosteroids, azathioprine, anti-tumor necrosis factor (TNF), antibiotics)\n* Benzodiazepine in the week before the PET scan \\[18F\\] DPA-714 given the potential consequences for TSPO receivers\n* Contraindications to MRI in patients with:\n\n  1. Metallic foreign body eye.\n  2. Any implanted electronic medical irremovably (pacemaker, neurostimulator, cochlear implants ...)\n  3. Metal heart valve,\n  4. Vascular clips formerly located on cranial aneurysm.\n* Treatment in the month before the PET scan \\[18F\\] DPA-714 antagonist N-methyl-D-aspartate (NMDA) (memantine)\n* Pregnant women, lactating women, and women in age for procreation and without reliable contraception or without history of hysterectomy\n* \u25e6Person under guardianship", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "[18F]DPA-714", "targeting_mechanism": "Translocator Protein (TSPO) ligand used as a PET imaging biomarker to quantify microglial activation and neuroinflammation", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07095686", "title": "Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS", "phase": "PHASE1, PHASE2", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "n-Lorem Foundation", "summary": "This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for individual participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10", "interventions": [{"type": "DRUG", "name": "nL-CHCHD-001"}], "start_date": "2024-10-02", "url": "https://clinicaltrials.gov/study/NCT07095686", "target_entities": ["CHCHD10"], "locations": [{"facility": "Columbia University, Irving Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representatives(s)\n* Ability to travel to the study site and adhere to study related follow-up examinations and/or procedures and provide access to participant's medical records\n* Genetically confirmed neurological disorder\n\nExclusion Criteria:\n\n* Participant has any condition that in the opinion of the Site Investigator would ultimately prevent the completion of study procedures\n* Use of an investigational medication within less than 5 half-lives of the drug at enrollment", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "nL-CHCHD-001", "targeting_mechanism": "Personalized antisense oligonucleotide designed to target pathogenic variants in CHCHD10 gene", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07224256", "title": "VOICE: An Early Feasibility Study of a Precise Robotically Implanted Brain-Computer Interface for Communication Restoration", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuralink Corp", "summary": "The VOICE Study is an early feasibility study to evaluate the initial clinical safety and efficacy of the N1 and R1 Systems device design concept in providing an ability to communicate. The Neuralink N1 Implant is intended to provide the ability to communicate to individuals with severe and irreversible speech production impairment. It is indicated for adults with neurological conditions of the central speech pathways who have impaired upper limb function. The N1 Implant is a skull-mounted, wireless, rechargeable implant connected to electrode threads that are implanted in the brain by the R1 Robot, a robotic electrode thread inserter.", "interventions": [{"type": "DEVICE", "name": "N1 Implant"}, {"type": "DEVICE", "name": "R1 Robot"}], "start_date": "2025-10-03", "url": "https://clinicaltrials.gov/study/NCT07224256", "target_entities": [], "locations": [{"facility": "The University of Texas Southwestern Medical Center", "city": "Dallas", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "+1 (650) 250-0520", "contact_email": "voice-study@neuralink.com", "eligibility": {"criteria": "Inclusion Criteria:\n\nAdults with diagnosis of ALS, PLS, stroke, or Spinal Cord Injury who have severe speech impairment and impaired upper limb function.\n\n* Life expectancy \u2265 12 months.\n* Ability to communicate in English\n* Presence of a stable caregiver\n\nExclusion Criteria\n\n* Moderate to high risk for serious perioperative adverse events\n* Morbid obesity (Body Mass Index \\> 40)\n* History of poorly controlled seizures or epilepsy\n* History of poorly controlled diabetes\n* Requires magnetic resonance imaging (MRI) for any ongoing medical conditions\n* Acquired or hereditary immunosuppression\n* Psychiatric or psychological disorder\n* Brain MRI demonstrating hemorrhage, tumor, distorted or adverse anatomy.\n* Any condition which, in the opinion of the Investigator, would compromise your ability to safely participate in the study or undergo the implantation procedure", "sex": "ALL", "min_age": "22 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Brain-computer interface device intended to restore communication in individuals with severe speech impairment due to motor neuron disease", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04313166", "title": "Treatment Continuation Study for Patients With ALS/MND Who Completed Study CMD-2019-001", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Collaborative Medicinal Development Pty Limited", "summary": "Provides up to six months treatment with CuATSM for subjects who have successfully completed study CMD-2019-001", "interventions": [{"type": "DRUG", "name": "Cu(II)ATSM"}], "start_date": "2020-03-19", "url": "https://clinicaltrials.gov/study/NCT04313166", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Macquarie University", "city": "Macquarie Park", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.78105, "lon": 151.12757}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* signed informed consent prior to initiation of any study-specific procedures and treatment\n* documented completion of protocol-specified assessments following completion of 24 weeks treatment on study CMD-2019-002\n* Investigator considers patient has been tolerating treatment on study CMD-2019-001 and may benefit from continued treatment with CuATSM\n\nExclusion Criteria:\n\n* not dependent on mechanical ventilation", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Cu(II)ATSM", "targeting_mechanism": "Copper(II) complex that delivers bioavailable copper to the CNS to restore SOD1 activity and counteract oxidative stress", "targeting_mechanism_pmid": "26826269", "animal_results": "CuATSM treatment prevented early mortality and motor neuron degeneration in SOD1(G93A) mice co-expressing copper-chaperone-for-SOD, rescuing runted pups that normally died in 8-13 days without treatment", "animal_results_pmid": "26826269", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07509125", "title": "Ultra-High Resolution PET in Aging, Neurodegeneration and Psychotic Disorders", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Universitaire Ziekenhuizen KU Leuven", "summary": "The goal of this study is to use ultra-high-resolution (UHR) PET imaging to better understand how the brain and spinal cord change in healthy aging and in neurological and psychiatric disorders such as Alzheimer's disease (AD), Parkinson's disease and related movement disorders, amyotrophic lateral sclerosis (ALS), and psychotic disorders. Researchers will use the NeuroExplorer PET/CT system, a new scanner that can show very small structures in the brain and spinal cord in much more detail than regular PET.\n\nThe main questions this study aims to answer are:\n\n* How do small but important brain regions (like the locus coeruleus, substantia nigra, and thalamic nuclei) change in healthy aging?\n* What early brain changes occur in neurodegenerative and psychotic disorders, and can they help improve early diagnosis?\n\nParticipants will:\n\n* Undergo PET and MRI brain scans using different tracers that measure brain metabolism (18F-FDG), synaptic density (\u00b9\u2078F-SynVesT-1), dopamine transporters (\u00b9\u2078F-PE2I), and tau protein buildup (\u00b9\u2078F-MK6240).\n* Complete cognitive and clinical assessments related to memory, mood, and motor or psychiatric symptoms, depending on their group.\n\nThis study will include healthy volunteers and patients with mild cognitive impairment due to Alzheimer\u00b4s disease, ALS, Parkinson's disease and related disorders, or psychotic disorders.\n\nThe results will help create detailed brain imaging maps for healthy aging and identify early biomarkers for different diseases to support better diagnosis and treatment in the future.", "interventions": [{"type": "OTHER", "name": "UHR PET/CT scan of the brain with \u00b9\u2078F-FDG"}, {"type": "OTHER", "name": "UHR PET/CT scan of the brain with \u00b9\u2078F-PE2I"}, {"type": "OTHER", "name": "UHR PET/CT scan of the brain with \u00b9\u2078F-SynVesT-1"}, {"type": "OTHER", "name": "UHR PET/CT scan of the brain with \u00b9\u2078F-MK6240"}, {"type": "OTHER", "name": "3T MRI imaging of the brain"}], "start_date": "2026-02-13", "url": "https://clinicaltrials.gov/study/NCT07509125", "target_entities": [], "locations": [{"facility": "UZ Leuven", "city": "Leuven", "state": "Vlaams-Brabant", "country": "Belgium", "status": "RECRUITING", "lat": 50.87959, "lon": 4.70093}], "contact_phone": "+32 16 34 37 11", "contact_email": "koen.vanlaere@uzleuven.be", "eligibility": {"criteria": "Inclusion Criteria:\n\n* WP1: Healthy controls\n* Age between 18 and 90 years old (15 aged 18-50 years and 25 aged 50 90 years);\n* Subject is judged to be in good health by the investigator on the basis of medical history, physical examination including vital signs and clinical laboratory tests;\n* No history or evidence of current major neurological, internal or psychiatric disorder, based on the medical assessment as described hereabove and neuropsychological assessment;\n* No evidence of cognitive impairment as assessed by a Montreal Cognitive Assessment (MoCA) score of 26 or higher at baseline;\n* In subjects \\< 60 years of age, a normal structural MRI scan as assessed by expert radiologist.\n* In subjects \\>= 60 years of age white matter hyperintensities corresponding to a WML (white matter lesion) score \\<= 2 (of 3) on the Age-Related White Matter changes scale are acceptable;\n* When older than 50 years of age, the volunteer is willing to undergo a p- tau217 blood sample.\n* WP2: Dementia\n* Patient has a clinical diagnosis of biomarker-proven prodromal AD\n* WP3: ALS spectrum\n* Subject must meet El Escorial Criteria (30) and Awaji-Shima criteria (31) for at least possible ALS;\n* WP4: Movement disorders\n* (all): Patient (or legal representative, when applicable) is able to understand the patient information form and give written informed consent.\n* Parkinson\u00b4s disease (PD):\n* Patient has clinically established PD based on the Movement Disorder Society (MDS) diagnostic criteria (32);\n* Patient has an abnormal 18F-PE2I PET;\n* No evidence of cognitive impairment as assessed by a Montreal Cognitive Assessment (MoCA) score of 26 or higher at baseline.\n* Multiple system atrophy (MSA)\n* Patient has clinically established or clinically probable MSA-P based on the\n* Movement Disorder Society (MDS) diagnostic criteria (33);\n* Patient has an abnormal 18F-PE2I PET.\n* Progressive supranuclear palsy (PSP)\n* Patient has an abnormal 18F-PE2I PET;\n* Patient has clinically established probable PSP according to the latest MDS criteria\n* Dementia with Lewy bodies (DLB)\n* Patient has probable DLB by consensus criteria (cognitive impairment MoCA \\< 26 + visual hallucinations and/or fluctuating alertness);\n* Patient has an abnormal 18F-PE2I PET.\n* Idiopathic REM sleep behavior disorder (iRBD)\n* Patient has Polysomnography-confirmed iRBD;\n* No evidence of cognitive impairment as assessed by a Montreal Cognitive Assessment (MoCA) score of 26 or higher at baseline;\n* No clinical evidence of parkinsonism at baseline.\n* WP5: Psychosis\n* DSM 5 criteria for a non-affective schizophrenia spectrum psychotic disorder;\n* Age between 18 and 55 years old for adult-onset psychosis, onset of psychosis (and age) above 60 years old for very late onset psychosis.\n\nExclusion Criteria:\n\n* Subject has a history of any major (other) internal, psychiatric or neurological disease that may interfere with the investigations (especially liver and kidney disease, uncontrolled diabetes, cancer, severe depression, stroke, severe TBI);\n* Subject is currently a user (including recreational use) of any illicit drugs, including cannabis, or has a history of drug or alcohol abuse;\n* Subject chronically uses medication that has central nervous system effects (e.g. strong painkillers such as opioids, neuroleptics,..; ) (other than prescribed for the illness in case of patients);\n* Subject has had exposure to ionizing radiation (\\> 1 mSv) in other research studies within the last 12 months;\n* Subject has a contra-indication for MRI scanning;\n* Subject suffers from claustrophobia or cannot tolerate confinement during PET-MRI scanning procedures; subject cannot lie still for (at least) 60 minutes inside the scanner;\n* (For subjects with arterial sampling): The subject is hypersensitive to lidocaine (used for local anaesthesia during the placement of the arterial catheter), has an abnormal Allen test (a test to check blood flow in the arteries of the forearm) or is on anti-coagulant therapy;\n* Subject (or his/her legal representative) does not understand the study procedures;\n* Subject is unwilling or unable to perform all of the study procedures, or is considered unsuitable in any way by the principal investigator;\n* Subject is potentially pregnant (hCG test can be done if doubt exists).", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01759797", "title": "Intravenous Transplantation of Mesenchymal Stem Cell in Patients With ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Royan Institute", "summary": "ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). ALS is the most common of the five motor neuron diseases.Riluzole (Rilutek) is the only treatment that has been found to improve survival but only to a modest extent. It lengthens survival by several months, and may have a greater survival benefit for those with a bulbar onset. It also extends the time before a person needs ventilation support.Stem cell transplantation is a new hopeful way to improve the patients conditions and reduce the period of disabilities.", "interventions": [{"type": "BIOLOGICAL", "name": "intra venous injection of stem cell"}], "start_date": "2013-01", "url": "https://clinicaltrials.gov/study/NCT01759797", "target_entities": [], "locations": [{"facility": "Royan Institute", "city": "Tehran", "state": "", "country": "Iran", "status": "", "lat": 35.69439, "lon": 51.42151}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* \\- Age:18-65\n* both gender\n* duration of disease\\<2 years\n* FVC\\>40% ALS-FRS\\>26\n\nExclusion Criteria:\n\n* \\- neurological and psychiatric concomitant disease\n* concomitant systemic disease\n* treatment with corticosteroid,Ig,immunosuppressive during 12 months.", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "intravenous mesenchymal stem cells", "targeting_mechanism": "Stem cells differentiate into support cells (astrocytes, oligodendrocytes, microglia) and produce growth factors and anti-inflammatory cytokines to promote motor neuron survival and provide blood-spinal cord barrier repair.", "targeting_mechanism_pmid": "32043626", "animal_results": "In SOD1G93A mice, intravenous bone marrow-derived mesenchymal stem cells expressing Ngn1 increased lifespan by 3 days, delayed disease onset by 5 days, and reduced motor neuron loss, though cells were mostly found in the kidney with very few in the brainstem and spinal cord.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03975608", "title": "Psychological Therapy for Patients With ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Leipzig", "summary": "Amyotrophic lateral sclerosis (ALS) is a disease that is inevitably fatal. To be diagnosed with a terminal illness such as ALS deeply affects one's personal existence and goes along with significant changes regarding the physical, emotional, and social domains of the patients' life. This pilot study will test a manualized, individual psychotherapeutic intervention to relieve distress and promote psychological well-being in ALS patients. A total of 5 patients will receive the intervention. The investigators will gather important information regarding the feasibility of the intervention (i.e., response rate, patient and therapist adherence, and patient satisfaction), which may be used for conducting a future randomized controlled trial. Various domains of quality of life will be assessed before the intervention (T0), after the intervention (T1) and at 3-months-follow-up (T2) in order to test for preliminary efficacy of the intervention.", "interventions": [{"type": "BEHAVIORAL", "name": "Adaptation of \"CALM\""}], "start_date": "2019-02-11", "url": "https://clinicaltrials.gov/study/NCT03975608", "target_entities": [], "locations": [{"facility": "University Medical Center Leipzig", "city": "Leipzig", "state": "Saxony", "country": "Germany", "status": "", "lat": 51.33962, "lon": 12.37129}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* confirmed diagnosis of Amyotrophic Lateral Sclerosis\n* minimum age of 18 years\n* fluent in German language\n* ability to visit the institution providing the intervention at the start of therapy (in the course of treatment, telephone-sessions may be offered)\n* ability to report on thoughts and feelings (by speaking, writing or via communication devices)\n* cognitive ability to give written informed consent\n* expected remaining lifetime of at least 9 months\n\nExclusion Criteria:\n\n* inability for communicate (neither via speaking, writing or communication devices)\n* currently in psychotherapeutic treatment", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05404867", "title": "Study of Structural and Functional Brain Connectivity Changes in ALS (CoALS-II)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Dartmouth-Hitchcock Medical Center", "summary": "This study will try to understand the difference in brain structure between ALS patients and healthy people of similar age. ALS is a condition affecting the nervous system with disruption of the brain networks. This study aims to understand these disruptions and determine their significance in ALS.", "interventions": [{"type": "OTHER", "name": "fMRI"}], "start_date": "2022-06-08", "url": "https://clinicaltrials.gov/study/NCT05404867", "target_entities": [], "locations": [{"facility": "Dartmouth-Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Based on El Escorial Criteria we will select Probable or Definite ALS.\n* Healthy controls age matched to ALS patients\n* Subjects should be able to lie down in a scanner and undergo an hour-long MRI study\n* Subjects should be able to understand instructions, provide consent and perform a hand task while inside the scanner\n\nExclusion Criteria:\n\n* Unable to undergo MRI due to any reason\n* Age \\<18\n* ALS patients having high aspiration risk\n* Patients having cognitive limitations which prevents them from understanding the study requirements, providing consent or perform tasks inside the scanner.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05695521", "title": "Regulatory T Cells for Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cellenkos, Inc.", "summary": "Phase 1 Safety Run-in Study of 6 patients followed by Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803, neurotropic, allogeneic, umbilical cord blood derived T regulatory (Treg) cells in additional 60 patients with Amyotrophic Lateral Sclerosis.", "interventions": [{"type": "BIOLOGICAL", "name": "CK0803"}, {"type": "OTHER", "name": "Excipient"}], "start_date": "2023-04-03", "url": "https://clinicaltrials.gov/study/NCT05695521", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Columbia University Irving Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Baylor College of Medicine", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Michael E. DeBakey Veterans Affairs Medical Center", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Ability of the subject or his/her legally authorized representative to provide informed consent.\n* Adult ALS subjects (\u226518 years of age)\n* Diagnosis of ALS, according to the Revised El Escorial Criteria for ALS\n* Subjects with disease onset \u2264 5 years\n* Upright (sitting position) Slow Vital Capacity (SVC) as adjusted for sex, age and height \u2265 50% predicted\n* Subjects must have documented ALSFRSR score of 36-45 at baseline.\n* Subjects taking concomitant Riluzole or Edaravone or Albrioza at study entry must be on a stable dose for \u2265 30 days prior to the first dose of study treatment (Day 1).\n* Screening values of coagulation parameters including platelet count, international normalized ratio (INR), prothrombin time (PT), and activated partial thromboplastin time (APTT) should be within normal ranges.\n* Agree to practice highly effective contraception during the study and continue contraception for 90 days after their last dose of study treatment.\n\nExclusion Criteria:\n\n* Uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol medical monitor is the final arbiter of eligibility.\n* Antiplatelet or anticoagulant therapy within the 14 days prior to Day 1 or anticipated use during the study, including but not limited to daily aspirin including low dose aspirin (defined as \u2264 150 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban and apixaban\n* Clinically significant low platelet count (defined as \\< 100,000/mm3), coagulation tests, or laboratory abnormalities that would render a subject unsuitable for inclusion\n* Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator\n* Have any other conditions, which, in the opinion of the Investigator would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the study\n* Concurrent participation in any other interventional clinical study\n* Treatment with another investigational drug, biological agent, or device, including, but not limited to sodium phenylbutyrate, within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer\n* Treatment of cancer in the last 5 years (except in situ carcinoma of the cervix or basal cell carcinoma)\n* Female subjects who are pregnant or currently breastfeeding\n* Other unspecified reasons that, in the opinion of the Investigator or Sponsor, make the subject unsuitable for enrollment.", "sex": "ALL", "min_age": "18 Years", "max_age": "95 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CK0803", "targeting_mechanism": "Allogeneic umbilical cord blood-derived T regulatory (Treg) cells that modulate neuroinflammatory responses to suppress excessive inflammatory activation in the CNS.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05237284", "title": "Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sanofi", "summary": "This was a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820 compared with placebo in male and female participants, 18 to 80 years of age with ALS followed by an open-label, long-term extension period.\n\nStudy ACT16970 consisted of 2 parts (A and B) as follows:\n\nPart A was a 24-week, double blind, placebo-controlled part, preceded by a screening period of up to 4 weeks before Day 1.\n\nOn Day 1 of Part A, participants were randomized in a 2:1 ratio to the SAR443820 treatment arm or matching placebo arm as listed below:\n\n* Treatment arm: SAR443820, BID\n* Placebo arm: Placebo, BID\n\nRandomization was stratified by the geographic region of the study site, region of ALS onset (bulbar vs other areas), use of riluzole (yes vs no), use of edaravone (yes vs no) and use of the combination of sodium phenylbutyrate and taurursodiol (named Relyvrio in the United States of America \\[USA\\] and Albrioza in Canada) (yes vs no). Participants attended in-clinic study assessments at baseline (Day 1), Week 2, Week 4, Week 6, Week 8, Week 10, Week 12, Week 16, Week 20, Week 21, Week 22, Week 23, and Week 24. All ongoing participants at Week 24 rolled to open-label extension Part B. The Week 24 Visit was the end of Part A and the beginning of Part B.\n\nPart B was an open-label, long-term extension period that starts from Week 24 and continues up to Week 106. The objectives of Part B were to provide extended access to SAR443820 participants in Part A and to further evaluate the safety and efficacy of long-term SAR443820 treatment. The treatment assignment of participants at randomization in Part A remained blinded to Investigators, participants, and site personnel until the end of Part B. Every participant, except those who discontinued Investigational Medicinal Product (IMP) treatment permanently in Part A, received BID oral tablets of SAR443820 in Part B.", "interventions": [{"type": "DRUG", "name": "SAR443820"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2022-04-13", "url": "https://clinicaltrials.gov/study/NCT05237284", "target_entities": ["sar443820"], "locations": [{"facility": "UC San Diego Health Site Number : 8400022", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "USC Site Number : 8400008", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Irvine Site Number : 8400012", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center Site Number : 8400015", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Site Number : 8400025", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Georgetown University Medical Center Site Number : 8400020", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Mayo Clinic Site Number : 8400029", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "AdventHealth Medical Group - Neurology at Winter Park Site Number : 8400006", "city": "Winter Park", "state": "Florida", "country": "United States", "status": "", "lat": 28.6, "lon": -81.33924}, {"facility": "Northwestern Medical Group, Department of Neurology Site Number : 8400003", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins University Site Number : 8400028", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital Site Number : 8400001", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Mount Sinai - Union Square Site Number : 8400002", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Penn State Milton S. Hershey Medical Center Site Number : 8400004", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "University of Pennsylvania Site Number : 8400021", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Thomas Jefferson University Hospital Site Number : 8400014", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Utah Site Number : 8400009", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "Froedtert Hospital & Medical College of Wisconsin Site Number : 8400010", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "Investigational Site Number : 0560001", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Investigational Site Number : 1240004", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Investigational Site Number : 1240007", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "Investigational Site Number : 1240006", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Investigational Site Number : 1240008", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Investigational Site Number : 1240002", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Investigational Site Number : 1240001", "city": "Qu\u00e9bec", "state": "", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Investigational Site Number : 1560001", "city": "Beijing", "state": "", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}, {"facility": "Investigational Site Number : 1560003", "city": "Chengdu", "state": "", "country": "China", "status": "", "lat": 30.66667, "lon": 104.06667}, {"facility": "Investigational Site Number : 1560005", "city": "Guangzhou", "state": "", "country": "China", "status": "", "lat": 23.11667, "lon": 113.25}, {"facility": "Investigational Site Number : 1560002", "city": "Hangzhou", "state": "", "country": "China", "status": "", "lat": 30.29365, "lon": 120.16142}, {"facility": "Investigational Site Number : 1560004", "city": "Wuhan", "state": "", "country": "China", "status": "", "lat": 30.58333, "lon": 114.26667}, {"facility": "Investigational Site Number : 1560006", "city": "Xi'an", "state": "", "country": "China", "status": "", "lat": 34.25833, "lon": 108.92861}, {"facility": "Investigational Site Number : 2500007", "city": "Caen", "state": "", "country": "France", "status": "", "lat": 49.18585, "lon": -0.35912}, {"facility": "Investigational Site Number : 2500006", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Investigational Site Number : 2500002", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Investigational Site Number : 2500003", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Investigational Site Number : 2500004", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Investigational Site Number : 2500005", "city": "Vand\u0153uvre-l\u00e8s-Nancy", "state": "", "country": "France", "status": "", "lat": 48.66115, "lon": 6.17114}, {"facility": "Investigational Site Number : 2760004", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Investigational Site Number : 2760003", "city": "Dresden", "state": "", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Investigational Site Number : 2760008", "city": "Haag in OB", "state": "", "country": "Germany", "status": "", "lat": null, "lon": null}, {"facility": "Investigational Site Number : 2760005", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Investigational Site Number : 2760002", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "", "lat": 53.86893, "lon": 10.68729}, {"facility": "Investigational Site Number : 2760001", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Investigational Site Number : 2760009", "city": "W\u00fcrzburg", "state": "", "country": "Germany", "status": "", "lat": 49.79391, "lon": 9.95121}, {"facility": "Investigational Site Number : 3800001", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Investigational Site Number : 3800004", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Investigational Site Number : 3800002", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Investigational Site Number : 3920003", "city": "Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "Investigational Site Number : 3920004", "city": "Ichikawa-shi", "state": "Chiba", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Investigational Site Number : 3920006", "city": "Tokushima", "state": "Tokushima", "country": "Japan", "status": "", "lat": 34.06667, "lon": 134.56667}, {"facility": "Investigational Site Number : 3920005", "city": "Fuchu-shi", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Investigational Site Number : 3920001", "city": "\u014cta-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.56126, "lon": 139.71605}, {"facility": "Investigational Site Number : 3920002", "city": "Koshi-shi", "state": "", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Investigational Site Number : 5280001", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Investigational Site Number : 6160001", "city": "Krakow", "state": "", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}, {"facility": "Investigational Site Number : 6160002", "city": "Ksawer\u00f3w", "state": "", "country": "Poland", "status": "", "lat": 51.68288, "lon": 19.4028}, {"facility": "Investigational Site Number : 7240005", "city": "Barcelona", "state": "Barcelona [Barcelona]", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Investigational Site Number : 7240002", "city": "L'Hospitalet de Llobregat", "state": "Barcelona [Barcelona]", "country": "Spain", "status": "", "lat": 41.35967, "lon": 2.10028}, {"facility": "Investigational Site Number : 7240003", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Investigational Site Number : 7240001", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Investigational Site Number : 7520002", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Investigational Site Number : 7520001", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "Investigational Site Number : 8260002", "city": "Plymouth", "state": "Devon", "country": "United Kingdom", "status": "", "lat": 50.37153, "lon": -4.14305}, {"facility": "Investigational Site Number : 8260003", "city": "Stoke-on-Trent", "state": "Staffordshire", "country": "United Kingdom", "status": "", "lat": 53.00415, "lon": -2.18538}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of possible, clinically probable ALS, clinically probable laboratory supported ALS, or clinically definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria\n* Time since onset of first symptom of ALS \u22642 years.\n* Slow Vital Capacity (SVC) \u226560% of the predicted value.\n* Had to be able to swallow the study tablets at the screening visit.\n* Either not currently receiving riluzole or on a stable dose of riluzole for at least 4 weeks before the screening visit. Participants receiving riluzole were expected to remain on the same dose throughout the duration of the study.\n* Either not currently receiving edaravone or on the approved standard schedule of edaravone treatment. Participants receiving edaravone had to have completed at least 1 cycle of treatment before the screening visit and were expected to continue edaravone treatment throughout the duration of the study.\n* Either not currently receiving the combination of sodium phenylbutyrate and taurursodiol or on the approved standard schedule of the combination of sodium phenylbutyrate and taurursodiol treatment for at least 4 weeks before the screening visit. Participants receiving the combination of sodium phenylbutyrate and taurursodiol were expected to remain on the approved standard schedule throughout the duration of the study.\n* Participants with a body weight no less than 45 kg and body mass index no less than 18 kg/m2 at the screening visit\n* Female participants with childbearing potential were eligible to participate if they were not pregnant or breastfeeding and agreed to use adequate contraceptive method during study intervention period and for at least 32 days after the last dose of study drug.\n* Male participants had to agree to use highly effective contraceptive method during the study period and for at least 92 days following their last dose of the study drug. Male participants were not donate sperms for the duration of study and 92 days after last dose of study drug.\n\nExclusion Criteria:\n\n* A history of seizure (History of febrile seizure during childhood was allowed).\n* Having central IV lines, such as a peripherally inserted central catheter (PICC XE ' PICC ' \\\\f Abbreviation \\\\t 'peripherally inserted central catheter' ) or midline or portacath lines.\n* With significant cognitive impairment, psychiatric disease, other neurodegenerative disorder (eg, Parkinson disease or AD), substance abuse other causes of neuromuscular weakness, or any other condition that would make the participants unsuitable for participating in the study or could interfere with assessment or completing the study in the opinion of the Investigator.\n* History of recent serious infection (eg, pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with IV antibiotics, antivirals, or antifungals within 4 weeks of screening; or chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the Investigator's judgment.\n* With active herpes zoster infection within 2 months prior to the screening visit.\n* A documented history of attempted suicide within 6 months prior to the screening visit, present with suicidal ideation of category 4 or 5 on the Columbia Suicide Severity Rating Scale (CSSRS) , or in the Investigator's judgment are at risk for a suicide attempt.\n* History of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or another medically significant illness other than ALS precluding their safe participation in this study.\n* Participants who were pregnant or were currently breastfeeding.\n* A known history of allergy to any ingredients of SAR443820.\n* Currently or previously treated with any strong or moderate CYP3A4 inhibitors or strong CYP3A4 inducers within the specified washout period before the screening visit.\n* Received a live vaccine within 14 days before the screening visit.\n* Participants with concurrent participation in any other interventional clinical study or who had received treatment with another investigational drug (eg sodium phenylbutyrate or taurursodiol ) within 4 weeks or 5 halflives of the investigational agent before the screening visit, whichever is longer.\n* Participants who had received stem cell or gene therapy for ALS at any time in the past.\n* Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\>3.0 \u00d7 upper limit of normal (ULN)\n* Bilirubin \\>1.5 \u00d7 ULN unless the participant had documented Gilbert syndrome (isolated bilirubin \\>1.5 \u00d7 ULN was acceptable if bilirubin was fractionated and direct bilirubin is \\<35%)\n* Serum albumin \\<3.5 g/dL\n* Estimated glomerular filtration rate \\<60 mL/min/1.73 m2 (Modification of Diet in Renal Disease \\[MDRD\\])\n\nThe above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "SAR443820", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03986671", "title": "Transmembrane Electromyography (TM-EMG) for the Assessment of Neuromuscular Function in the Oropharynx", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Powell Mansfield Inc.", "summary": "This is a pilot study to examine the diagnostic utility of a novel transmembrane surface sensor, and compare signals obtained with the transmembrane sensor to conventional needle EMG signals from healthy volunteers to those with documented neurologic pharyngeal muscle dysfunction (ALS and muscular dystrophy) and to those with severe OSA.", "interventions": [{"type": "DEVICE", "name": "Transmembrane EMG Oropharynx Probe"}], "start_date": "2019-04-30", "url": "https://clinicaltrials.gov/study/NCT03986671", "target_entities": [], "locations": [{"facility": "SENTA Clinic", "city": "San Diego", "state": "California", "country": "United States", "status": "", "lat": 32.71571, "lon": -117.16472}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age: 18-70\n* Must be able to pause use of anticoagulation, NSAID and multi-vitamins for appropriate period prior to study test.\n* Must be willing to stop any type of smoking or vaping 10 days prior to testing\n\nA cohort of participants with documented neurological disorders involving upper airway striated muscles including ALS and muscular dystrophy with the presence of bulbar symptoms.\n\nA cohort of participants diagnosed with moderate to severe OSA proven by an in-lab PSG, including the following criteria:\n\n* AHI \\> 25\n* Nadir SaO2 \\< 85%\n* not currently using CPAP\n\nA cohort of healthy participants that meet the following criteria:\n\n* Normal craniofacial anatomy\n* BMI \\< 30\n\nExclusion Criteria:\n\n* Allergy to topical anesthetic\n* 4 or more alcoholic drinks on the same occasion on 5 or more days in the past month\n* Prior cancer, or radiation to the head or neck\n* Craniofacial anatomical disorders\n* Presence of any underlying medical, surgical or psychiatric disorder that would preclude participation as determined by the principal investigator", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05842941", "title": "HEALEY ALS Platform Trial - Regimen G DNL343", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.\n\nRegimen G will evaluate the safety and efficacy of a single study drug, DNL343, in participants with ALS.", "interventions": [{"type": "DRUG", "name": "DNL343"}, {"type": "DRUG", "name": "Matching Placebo"}], "start_date": "2023-05-24", "url": "https://clinicaltrials.gov/study/NCT05842941", "target_entities": ["dnl343"], "locations": [{"facility": "Healey Center for ALS at Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\nExclusion Criteria:\n\n* The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\n  1. Diagnosis of epilepsy or seizure within 6 months of randomization\n  2. Hypersensitivity to DNL343 or any of the excipients contained within the DNL343 drug product\n  3. The concomitant use of prescription or over-the-counter (OTC) medications that are inducers of certain cytochrome P450 enzymes, substrates of certain cytochrome P450 enzymes, or substrates of certain drug transporters.", "sex": "ALL", "min_age": "18 Years", "max_age": "100 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "DNL343", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02059759", "title": "Immuno-modulation in Amyotrophic Lateral Sclerosis- a Phase II Study of Safety and Activity of Low Dose Interleukin-2", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Centre Hospitalier Universitaire de N\u012bmes", "summary": "The primary objective is to evaluate in ALS patients the regulatory T cell early response to two low-doses of IL-2 at 1 and 2 MIU per day after one course of 5 consecutive days comparatively to placebo.", "interventions": [{"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "1.0 MIU IL-2 per day"}, {"type": "DRUG", "name": "2.0 MIU IL-2 per day"}], "start_date": "2015-09", "url": "https://clinicaltrials.gov/study/NCT02059759", "target_entities": ["IL-2"], "locations": [{"facility": "CHRU de Montpellier - H\u00f4pital Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* The patient has been correctly informed\n* The patient must have given his/her informed and signed consent.\n* The patient must be insured or beneficiary of a health insurance plan.\n* The patient is at least 18 years old and less than 75 years old\n* Probable, or laboratory-supported probable or definite ALS as defined by El Escorial Revised ALS diagnostic criteria (according to Airlie House Conference 1988)\n* Stable on riluzole treatment for more than 3 months with liver function test results \\< 2ULN\n* Disease duration \u2264 5 years\n* Vital capacity \u2265 70% of normal\n* Ability to swallow without the requirement for nasogastric or PEG feeding\n* Agreement for patient to use an adequate method of contraception throughout the study and for 2 weeks after post study visit\n* The patient is available and willing to participate in seven study visits occurring at the CHU within the next six months\n\nExclusion Criteria:\n\n* The patient is participating in another interventional study\n* Within the past three months, the patient has participated in another interventional\n* The patient is in an exclusion period determined by a previous study\n* The patient is under judicial protection\n* The patient is an adult under guardianship\n* The patient refuses to sign the consent\n* It is impossible to correctly inform the patient\n* Other life threatening disease\n* Presence of contra-indicated concomitant treatments or with potential neuroprotective benefit (see section 11.2 of the protocol)\n* Presence of tracheostomy or non-invasive ventilation\n* Use of Percutaneous endoscopic gastrostomy (PEG) or nasogastric tube\n* Presence of clinical infection (treated or untreated)\n* Positive serology for CMV, EBV (confirmed by viral load), or HIV\n* Vaccination within 8 weeks prior to first experimental dosing\n* Other disease precluding functional assessments\n* Cancer within the past 5 years (except stable non-metastatic basal cell skin carcinoma or in situ carcinoma of the cervix)\n* Severe cardiac or pulmonary disease\n* Documented auto-immune disorders except asymptomatic Hashimoto thyroiditis\n* Women of child bearing age without contraception or pregnant or breast feeding\n* Any clinically significant laboratory abnormality (excepting cholesterol, triglyceride and glucose)", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Interleukin-2 (IL-2)", "targeting_mechanism": "Stimulates expansion and differentiation of regulatory T cells to modulate immune response in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02969759", "title": "Bioenergetics and Protein Metabolism in Sporadic Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Angers", "summary": "INTRODUCTION: Amyotrophic Lateral Sclerosis (ALS) is a degenerative disease of the motor neurones of the brain and the spinal cord. The pathophysiological mechanisms of the disease remain unknown. The average age of onset of ALS is about 60 years old, and the mean survival of patients is about 2 years. The disease is responsible for a progressive paralysis leading to death from respiratory failure. The only treatment available is the Riluzole, with a very modest efficiency on the progression of the disease. ALS is the third neurodegenerative disease, affecting 6000 persons in France, 150 000 in the world. Among the protagonists involved in the occurrence of the disease, mitochondrial perturbations and protein accumulations seem to be central elements.\n\nOBJECTIVES: To precise the implication of energy and protein metabolism in the sporadic forms of ALS, to identify potential biomarkers of the disease and to test new therapies.\n\nMETHODS: The investigators will study cell growth, bioenergetics, mitochondrial dynamics, free-radicals production, presence of cytoplasmic inclusions, cytoskeleton structure and stress response in primary skin fibroblasts obtained from sporadic ALS patients. The study will be conducted over a period of three years in 3 centres specialized respectively in motor neuron diseases, mitochondrial metabolism and neuronal cytoskeleton.\n\nPROSPECTS: If the investigators achieve to identify differences between ALS fibroblasts and controls, the results will be key elements to reinforce the hypothesis of a systemic disease with an important metabolic participation, to better define ALS pathophysiological mechanisms, to find potential biomarkers and to test new therapies.", "interventions": [{"type": "OTHER", "name": "Skin Biopsy"}], "start_date": "2016-11", "url": "https://clinicaltrials.gov/study/NCT02969759", "target_entities": ["bioenergetics", "protein_metabolism"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion criteria :\n\n* a free signed an written consent\n* aged between 18 and 80 years old\n* ALS defined by El Escorial Criteria\n* normal neurological examination\n\nExclusion criteria:\n\n* comorbidity or treatment susceptible to impact on metabolism\n* differential diagnosis suspected, early and late forms (\\<6 month \\> 3 years)\n* ALS not defined by El Escorial Criteria\n* withdrawal of consent to participate", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02447952", "title": "Exploratory Study of Biotelemetry in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "GlaxoSmithKline", "summary": "Study 201283 is an exploratory, non-controlled, non-drug study in Amyotrophic Lateral Sclerosis (ALS) subjects. This study is being conducted as the first step for developing new meaningful measure(s) which might prove to be more effective than existing measures for monitoring clinical function and disease course in ALS. The objective of this study is to test novel measures of movement/physical activity, heart rate and speech and explore how they measure disease progression by evaluating their relationship to gold standard measures of function. This study will be conducted in two phases. A variable length Pilot Phase to test biotelemetry instruments and algorithms reliability and ease of use/acceptance. Approximately 5 subjects will have at least 1 clinic visit to perform a series of set reference tasks while wearing the accelerometer and electrode. Subjects will also continuously wear the accelerometer and electrode in their routine home-life setting for approximately 3 days after the clinic visit (i.e., home monitoring). Subjects in the Pilot Phase will continue in the study and participate in the Core Study Phase. A 48 week Core Study Phase will be conducted to evaluate how measures of movement/physical activity, speech and Heart Rate Variability (HRV) relate to ALS disease progression. During this phase, a maximum of 25 subjects will be enrolled. Subjects will attend 5 clinic visits to perform gold standard measures of function and perform a series of set reference tasks while wearing the accelerometer and electrode. In between clinic visits, every month subjects will attach the accelerometer and electrode and wear it for approximately 3 days in their home. A telephone contact with the subject will be made by the site at the end of each 3-day home monitoring period.\n\nAll third party trademark rights are the rights of their respective owners.", "interventions": [{"type": "DEVICE", "name": "Faros Sensor (FS) and LifeInsight Hub"}, {"type": "DEVICE", "name": "Fast Fix electrode patch"}, {"type": "PROCEDURE", "name": "Quantitative Measure of Speech (Core Phase Only)"}], "start_date": "2015-06-30", "url": "https://clinicaltrials.gov/study/NCT02447952", "target_entities": [], "locations": [{"facility": "GSK Investigational Site", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "GSK Investigational Site", "city": "Oxford", "state": "", "country": "United Kingdom", "status": "", "lat": 51.75222, "lon": -1.25596}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Between 18 and 80 years of age, inclusive, at the time of signing the informed consent.\n* Diagnosed with ALS by a neurologist with expertise in ALS. For subjects with bulbar onset there must be objective limb involvement of at least one limb.\n* Diagnosed with ALS within 18 months of symptom onset.\n* Subjects must be ambulatory (i.e., must not be confined to a wheelchair).\n* Male and female subjects.\n* Capable of giving signed (or verbal consent or assent where applicable) informed consent as described in Protocol which includes compliance with the requirements and restrictions listed in the consent form and in protocol.\n* Capable and willing to comply with the requirements of the protocol (either by themselves or with assistance).\n\nExclusion Criteria:\n\n* Neurological (other than the subject's ALS) or non-neurological co-morbidities (e.g. joint disease, respiratory disease) which limit mobility.\n* Clinically significant cognitive impairment in the opinion of the investigator.\n* Regionally restricted forms of ALS, or other atypical variants: Isolated corticobulbar pattern of ALS with normal ambulation; Flail arm syndrome; Primary lateral sclerosis; Signs of chronic partial denervation restricted to a single limb; ALS parkinsonism dementia complex\n* Subjects requiring mechanical ventilation (non-invasive ventilation for sleep apnoea is allowed).\n* Historical or current evidence of clinically significant uncontrolled disease which, in the opinion of the investigator, would put the safety of the subject at risk through participation or impact the study assessments or endpoints.\n* Presence of an active implantable cardiac medical device (e.g., pacemaker or implantable cardioverter-defibrillator) or at a high risk for needing external defibrillation.\n* History of skin hypersensitivity to adhesives.\n* Current participation in a clinical trial which in the opinion of the investigator and GSK medical monitor might impact the objectives of this study.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01123148", "title": "A P300 Brain Computer Interface to Operate Power Wheelchair Tilt", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "The investigators want to develop a brain-computer interface (BCI) that will eventually allow people who are completely paralyzed to independently control the tilt feature on their power wheelchairs. This study will allow healthy volunteers to test the feasibility and accuracy of controlling a BCI using only their brain signals while seated in a tilting wheelchair.", "interventions": [{"type": "DEVICE", "name": "Using a BCI to control wheelchair tilt"}], "start_date": "2010-01", "url": "https://clinicaltrials.gov/study/NCT01123148", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age 18 or older.\n* Able to read text on a computer screen\n* Able to understand and remember instructions concerning participation.\n\nExclusion Criteria:\n\n* Unable give informed consent.\n* Unable to understand and follow instructions.\n* Have abnormal tone or uncontrolled movements in the head-and-neck that would interfere with EEG recordings.\n* Known to have photosensitive epilepsy.\n* Open head lesions or sores.", "sex": "ALL", "min_age": "18 Years", "max_age": "99 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04562831", "title": "The NO-ALS Study: A Trial of Nicotinamide/Pterostilbene Supplement in ALS.", "phase": "NA", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Haukeland University Hospital", "summary": "Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is 3 years. Apart from Riluzole, there is no effective treatment. Care of advanced ALS will have a cost of 4-8 million NOK per year\n\nResearch i.a. from the investigators department has shown that increased activity in histone deacetylation enzymes (sirtuins) together with increased access to NAD can delay disease progression. Nicotinamide riboside (NR) can increase cells' access to NAD and Pterostilben will stimulate sirtuins.\n\nThe investigators want to study whether combination therapy with NR and Pterostilben can inhibit neurodegeneration in ALS and thereby delay disease development, increase survival and improve quality of life in ALS.\n\nIn the study, the investigators will use 2 different dosages on the active treatment and strength calculations show that 180 patients are needed to show a rather weak effect. Patients will be recruited in collaboration with hospitals in Helse Vest, AHUS, Drammen, OUS and St. Olavs hospital.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "EH301 (Nicotinamide Riboside/Pterostilbene)"}], "start_date": "2020-10-07", "url": "https://clinicaltrials.gov/study/NCT04562831", "target_entities": ["Histone deacetylases"], "locations": [{"facility": "Haukeland University Hospital", "city": "Bergen", "state": "", "country": "Norway", "status": "", "lat": 60.39299, "lon": 5.32415}, {"facility": "Vestre Viken HF", "city": "Drammen", "state": "", "country": "Norway", "status": "", "lat": 59.74389, "lon": 10.20449}, {"facility": "Helse F\u00f8rde HF", "city": "F\u00f8rde", "state": "", "country": "Norway", "status": "", "lat": 61.45217, "lon": 5.85717}, {"facility": "Helse Fonna HF", "city": "Haugesund", "state": "", "country": "Norway", "status": "", "lat": 59.41378, "lon": 5.268}, {"facility": "Akershus University Hospital", "city": "L\u00f8renskog", "state": "", "country": "Norway", "status": "", "lat": null, "lon": null}, {"facility": "Oslo University Hospital", "city": "Oslo", "state": "", "country": "Norway", "status": "", "lat": 59.91273, "lon": 10.74609}, {"facility": "Stavanger University Hospital", "city": "Stavanger", "state": "", "country": "Norway", "status": "", "lat": 58.97005, "lon": 5.73332}, {"facility": "Universitetssykehuset Nord-Norge", "city": "Troms\u00f8", "state": "", "country": "Norway", "status": "", "lat": 69.6489, "lon": 18.95508}, {"facility": "St.Olavs Hospital HF", "city": "Trondheim", "state": "", "country": "Norway", "status": "", "lat": 63.43049, "lon": 10.39506}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nArm 1 (newly diagnosed ALS patients)\n\n* Have a clinical diagnosis of probable ALS according to the revised El Escorial criteria.\n* MR of the brain and cervical spine cannot explain symptoms.\n* Diagnosed with likely ALS within 6 months from enrolment and treated with Riluzole 50mg x 2\n* Symptom onset no longer than 2 year prior to inclusion.\n* ALS-FRC-R of 36 or more (not any item below 2).\n* Age equal to or greater than 35 years at time of enrollment\n\nArm 2 (earlier diagnosed ALS patients)\n\n* Have a clinical diagnosis of probable ALS according to the revised El Escorial criteria.\n* MR of the brain and cervical spine cannot explain symptoms.\n* Treated with Riluzole 50mg x 2.\n\nExclusion Criteria:\n\n* Dementia, FTD or other neurodegenerative disorder at baseline visit\n* Any psychiatric disorder that would interfere with compliance in the study.\n* Use of high dose vitamin B3 supplementation within 30 days of enrollment\n* Metabolic, neoplastic, or other physically or mentally debilitating disorder at baseline visit.\n* Genetically confirmed mitochondrial disease\n* Patients who become tracheostomized as part of the treatment of ALS\n* Patients with short expected survival at the discretion of the investigator. Such cases cannot be expected to follow protocol procedures.", "sex": "ALL", "min_age": "35 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "EH301 (Nicotinamide Riboside/Pterostilbene)", "targeting_mechanism": "Increases NAD availability and sirtuin activity to enhance histone deacetylation and delay disease progression.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00886977", "title": "Efficacy and Safety of YAM80 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Yoshino Neurology Clinic", "summary": "The efficacy and safety are evaluated when YAM80 is administered orally to the patients of Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "YAM80"}], "start_date": "2009-04", "url": "https://clinicaltrials.gov/study/NCT00886977", "target_entities": ["yam80"], "locations": [{"facility": "Yoshino Neurology Clinic", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients aged between 25 and 65 years\n* ALS patients who can visit the clinic for six months\n* Forced Vital Capacity (FVC) \\> 70%\n* Patients who can walk by themselves\n* Change in ALSFRS-R score from -1 to -4 during 12 weeks before the initial administration\n* Patients who are willing to give informed consent\n\nExclusion Criteria:\n\n* Tracheotomy and invasive ventilation\n* Pregnant or possibly pregnant female patients\n* Female patients of childbearing potential who cannot practice contraception during and two years after the administration, and male patients who cannot practice contraception during and six months after the administration\n* Patients with clinically significant conditions such as cardiovascular, respiratory, haematological, and renal diseases.\n* Patients who are being treated with investigational drugs\n* Patients who are treated with other ALS drugs within 2 weeks prior to the first administration", "sex": "ALL", "min_age": "25 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "YAM80", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06726577", "title": "TP04HN106 in the Treatment of Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Talengen Institute of Life Sciences, Shenzhen, P.R. China.", "summary": "This trial adopts a multicenter, randomized, double-blind, placebo-controlled parallel design.\n\nThis experiment is divided into two groups: the experimental drug group and the placebo group. Successful participants will be randomly assigned to the two groups, with an expected enrollment of 60 participants. There will be 30 participants in the experimental drug group and 30 participants in the placebo group. During the treatment period, the experimental drug group received intravenous injections of 0.5mL/kg TP04HN106 each time; The placebo group received intravenous injections of 0.5mL/kg of saline each time. During the extension period, all subjects received intravenous injection of 0.5mL/kg TP04HN106. In the experiment, all subjects received Liraglutide tablets as the standard baseline treatment.\n\nThe subjects who were successfully screened in the experiment were enrolled in sequence, and the safety, tolerability, efficacy, and pharmacokinetic characteristics of the experimental drug were evaluated after administration. The entire trial includes a screening period of 1 week, a treatment period of 12 weeks (including 3 treatment cycles, each treatment cycle of 4 weeks), an extension period of 12 weeks (including 3 treatment cycles, each treatment cycle of 4 weeks), and a follow-up period of 4 weeks. In addition, some subjects underwent a 1-week single dose PK study before the start of the treatment period; In addition, during the first treatment cycle of the treatment period, some subjects were selected for multiple dosing PK studies.\n\nWe plan to conduct a single dose PK study among 12 subjects, with 6 subjects in the experimental group and 6 subjects in the control group; Multiple dose PK studies were conducted among 12 subjects, with 6 subjects in the experimental group and 6 sujects in the control group. It is not allowed for the same subject to participate in both single dose and multiple dose PK studies simultaneously.\n\nThe 1st to 12th subjects planned to be enrolled in the trial will undergo a single dose PK study. After the first dose, venous blood will be collected from the 12 subjects according to the blood sample collection requirements, and their PK characteristics will be evaluated. The observation period for single dose administration is one week. After completing the final blood sample collection and safety assessment, the subjects enter the treatment period, extension period, and follow-up period.\n\nThe 13th to 24th subjects planned to be enrolled in the trial will undergo multiple dose PK studies. These 12 subjects will have their venous blood collected according to the blood sample collection requirements during the first treatment cycle of the treatment period, and their PK characteristics will be evaluated. After completing the treatment period, the subjects will enter the extension period and follow-up period.", "interventions": [{"type": "DRUG", "name": "TP04HN106"}, {"type": "DRUG", "name": "Saline"}], "start_date": "2026-05-10", "url": "https://clinicaltrials.gov/study/NCT06726577", "target_entities": ["tp04hn106"], "locations": [{"facility": "The First Affiliated Hospital of Soochow University", "city": "Suzhou", "state": "Jiangsu", "country": "China", "status": "RECRUITING", "lat": 31.30408, "lon": 120.59538}], "contact_phone": "0086-89290018", "contact_email": "jnl@talengen-pharma.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Meet the diagnostic criteria for amyotrophic lateral sclerosis (ALS) (Gold Coast Criteria 2020\uff09;\n2. Age \u226518 years old, male or female;\n3. The amyotrophic lateral sclerosis Function Rating Scale (ALSFRS-R\uff09of pre-visit subjects should be \u22651 score for dyspnea, upright breathing and respiratory dysfunction;\n4. Pre-randomized subjects received stable dose of riluzole tablets for \u22657 days, and should maintain the treatment until the last study visit;\n5. Voluntarily participate in clinical trials, sign informed consent, and understand and comply with study procedures.\n\nExclusion Criteria:\n\n1. The subject is known to be allergic to the investigational drug or its excipients;\n2. The subject has a disease or injury that interferes with functional assessment or threatens life, or is accompanied by a serious irreversible disease of the heart, lung, liver, or brain, or is accompanied by a failure of different organs (for patients with respiratory failure, only patients diagnosed as type I or type II respiratory failure are excluded);\n3. The subject has a major mental illness or cognitive dysfunction;\n4. Patients with a history of secondary or above surgery within one month before the screening period;\n5. The subjects participated in other clinical studies within 1 month;\n6. The subjects are pregnant or lactating women;\n7. Poor compliance or other researchers believe that there are any circumstances that are not suitable for inclusion.\n\n   \\-", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "TP04HN106", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05031351", "title": "NF-\u03baB Inhibition in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sunnybrook Health Sciences Centre", "summary": "This is a Phase II, single centre, randomized, parallel, double blind, placebo-controlled clinical trial to determine the safety of Withania somnifera in participants with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Withania somnifera"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-10-19", "url": "https://clinicaltrials.gov/study/NCT05031351", "target_entities": ["nf_b", "neuroinflammation"], "locations": [{"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}], "contact_phone": "416-480-6100", "contact_email": "jake.wimmer@sri.utoronto.ca", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosed with laboratory supported probable, clinically possible, probable or definite ALS according to the World Federation of Neurology Revised El Escorial criteria (83) (Appendix A)\n* Disease duration from symptom onset no greater than 36 months at the Screening Visit\n* Aged 18 years or older\n* Capable of providing informed consent and complying with study procedures\n* If taking riluzole, on a stable dose for at least 30 days prior to Screening Visit\n* If taking edaravone, on a stable dose for at least one cycle prior to Screening Visit\n* If on BiPAP, average usage of no more than 12 hours per day at time of Screening Visit\n* Able to swallow a capsule at Baseline Visit\n* Fluency in English or French\n\nExclusion Criteria:\n\n* Exposure to any investigational agent or Withania somnifera (Ashwagandha) within 30 days prior to the Screening Visit; simultaneous participation in other observational studies is allowed upon Site Investigator approval\n* Presence of any of the following clinical conditions:\n\n  1. Substance abuse within the past year\n  2. Unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active malignancy or infectious disease\n  3. Acquired Immunodeficiency Syndrome (AIDS) or AIDS-related complex\n  4. Unstable psychiatric illness defined as psychosis (hallucinations or delusions) or untreated major depression within 90 days prior to the Screening Visit\n* Hypersensitivity or allergy to Withania somnifera\n* Uncontrolled diabetes with severe associated complications (such as neuropathy)\n* Untreated hypertension, active stomach ulcers, or untreated thyroid disorder\n* Previously diagnosed auto-immune condition with or without neurological manifestations (e.g. multiple sclerosis (MS), systemic lupus erythematosus (SLE), rheumatoid arthritis, etc.)\n* Current or planned use of oral, intramuscular or intravenous steroid drugs (such as prednisone, prednisolone, dexamethasone, triamcinolone, methylprednisolone, oxandrolone, and others) or immunosuppressant drugs (azathioprine, mycophenolate, tacrolimus, sirolimus, cyclophosphamide, and others) for more than 7 days\n* Planned consumption of alcohol, other drugs or natural health products with sedative and anxiolytics properties while taking study drugs (8 week duration)\n* Current or planned use of continuous subcutaneous, intravenous or oral anticoagulant drugs\n* Scheduled for surgery under general anesthetic within 14 days of Screening Visit\n* Pregnancy or planned pregnancy. Women of childbearing potential must have a negative pregnancy test and be non-lactating at the Screening Visit\n* Insertion of a diaphragm pacing system", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Withania somnifera", "targeting_mechanism": "NF-\u03baB inhibition to reduce neuroinflammation in ALS", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03456882", "title": "The Effect of RNS60 on ALS Biomarkers", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mario Negri Institute for Pharmacological Research", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a rare lethal neurodegenerative disease involving inflammation. Riluzole, the only drug for ALS, improves median survival by 3 months. This prompts new treatments of ALS. RNS60 is an experimental drug with favorable effects in preclinical studies of neuroinflammation and neurodegeneration. Based on significant efficacy demonstrated in preclinical studies and its excellent clinical safety profile, RNS60 is a promising candidate for a drug to treat ALS. Developing a pharmacodynamic marker will be a first and important step for dose finding and exploration of the mechanism of action in human, and pave the way to trials measuring drug efficacy.\n\nThe Investigator propose a multicenter, randomized, double-blind, placebo-controlled, parallel group, Phase II trial. The study centers will be located in Italy and at Massachusetts General Hospital (MGH) in Boston. A total of 142 ALS patients will be randomly assigned to RNS60 or placebo (administered by intravenous infusion once/week and inhaled via nebulization every morning for 24 weeks). All participants will also take riluzole (50-mg tablet twice/day). Blood samples for biomarker analysis (protein, RNA) will be collected in the screening period, on day 1, week 4,12 and 24. Both safety and potential therapeutic effects of RNS60 will be also assessed.", "interventions": [{"type": "DRUG", "name": "RNS60"}], "start_date": "2017-05-30", "url": "https://clinicaltrials.gov/study/NCT03456882", "target_entities": ["neuroinflammation", "neurodegeneration"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Azienda Opsedaliera Universitaria Consorziale Policlinico- Universit\u00e0 degli studi di Bari", "city": "Bari", "state": "", "country": "Italy", "status": "", "lat": 41.12066, "lon": 16.86982}, {"facility": "Spedali civili di Brescia", "city": "Brescia", "state": "", "country": "Italy", "status": "", "lat": 45.53558, "lon": 10.21472}, {"facility": "IRCCS Azienda Ospedaliera Universitaria San Martino IST", "city": "Genova", "state": "", "country": "Italy", "status": "", "lat": 45.21604, "lon": 11.87211}, {"facility": "Azienda Ospedaliera Universitaria POLICLINICO \"G. MARTINO\"", "city": "Messina", "state": "", "country": "Italy", "status": "", "lat": 38.19394, "lon": 15.55256}, {"facility": "Ospedale San Raffaele", "city": "Miano", "state": "", "country": "Italy", "status": "", "lat": 40.88816, "lon": 14.25339}, {"facility": "Centro Clinico NEMO - Fondazione Serena Onlus", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Presidio Ospedaliero Provinciale - Nuovo Ospedale Civile \"S. Agostino Estense\"", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Azienda Ospedaliera Universitaria della Seconda Univ. Degli Studi di Napoli (AOU-SUN)", "city": "Naples", "state": "", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}, {"facility": "Azienda Ospedaliero Universitaria Maggiore della Carit\u00e0", "city": "Novara", "state": "", "country": "Italy", "status": "", "lat": 45.44694, "lon": 8.62118}, {"facility": "Ospedale San Francesco ASSL Nuoro", "city": "Nuoro", "state": "", "country": "Italy", "status": "", "lat": 40.31991, "lon": 9.32568}, {"facility": "Azienda Ospedaliera di Padova-Universit\u00e0 degli studi di Padova", "city": "Padova", "state": "", "country": "Italy", "status": "", "lat": 44.38225, "lon": 11.14261}, {"facility": "Azienda Ospedaliera Universitaria Policlinico \"P Giaccone\"", "city": "Palermo", "state": "", "country": "Italy", "status": "", "lat": 38.1166, "lon": 13.3636}, {"facility": "Istituto Neurologico Nazionale \"C. Mondino\"", "city": "Pavia", "state": "", "country": "Italy", "status": "", "lat": 45.19205, "lon": 9.15917}, {"facility": "Azienda Ospedaliero-Universitaria Pisana,", "city": "Pisa", "state": "", "country": "Italy", "status": "", "lat": 43.70853, "lon": 10.4036}, {"facility": "Azienda Ospedaliero-Universitaria Pisana", "city": "Pisa", "state": "", "country": "Italy", "status": "", "lat": 43.70853, "lon": 10.4036}, {"facility": "Centro Clinico Nemo- Policlinico Gemelli", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}, {"facility": "POLICLINICO UMBERTO I - Universit\u00e0 di Roma \"La Sapienza\"", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}, {"facility": "IRCCS Casa sollievo della Sofferenza", "city": "San Giovanni Rotondo", "state": "", "country": "Italy", "status": "", "lat": 41.70643, "lon": 15.7277}, {"facility": "Azienda Ospedaliera Universitaria Senese (AOUS)", "city": "Siena", "state": "", "country": "Italy", "status": "", "lat": 43.31822, "lon": 11.33064}, {"facility": "Azienda Ospedaliera \"Santa Maria\" di Terni", "city": "Terni", "state": "", "country": "Italy", "status": "", "lat": 42.56335, "lon": 12.64329}, {"facility": "Azienda Ospedaliero-Universitaria Citt\u00e0 della Salute e della Scienza di Torino.", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Azienda Ospedaliera \"Card. G. Panico\"", "city": "Tricase", "state": "", "country": "Italy", "status": "", "lat": 39.93018, "lon": 18.35421}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 18 through 80 years inclusive;\n2. Geographically accessible to the site and able to come to the site once a week for 24 weeks;\n3. Definite, probable, probable laboratory supported ALS diagnosis according to the revised El Escorial criteria; 4) Disease duration 6 to 24 months from symptom onset;\n\n5\\) Self sufficiency: Satisfactory bulbar and spinal function (score 3+ on the ALSFRS-R for swallowing, cutting food and handling utensils, and walking); 6) Satisfactory respiratory function (FVC \u226580% of predicted); 7) Documented progression of symptoms in the last three months, as measured by the ALSFRS-R scale; 8) Ability to understand and comply with the study requirements and to give written informed consent personally or via a legally authorized representative; 9) Treatment with riluzole 50 mg twice/day for at least 1 month prior to screening visit.\n\nSelf sufficiency: this term reflect independence in daily living activities. It is an intuitive parameter to indicate preservation of key functional activities, and - not least - it has shown to be a valid and reliable measure\n\nExclusion Criteria:\n\n1. History of HIV, clinically significant chronic hepatitis, antecedent polio infection, or other active infection;\n2. Motor neuron disease (MND) other than ALS;\n3. Involvement of systems other than motor possibly determining a functional impairment (as measured by the end-points) for the entire duration of the study;\n4. Other severe clinical conditions (e.g., cardiovascular disorders, neoplasms) with impact on survival or functional disability in the next 12 months;\n5. Renal insufficiency as defined by a serum creatinine \\> 1.5 times the upper limit of normal;\n6. Poor compliance with previous treatments;\n7. Other experimental treatments in the preceding 3 months;\n8. Women who are lactating or able to become pregnant (e.g. who are not post menopausal, surgically sterile, or using inadequate birth control) and men unable to practice contraception for the duration of the treatment and 3 months after its completion;\n9. Unwillingness or inability to take riluzole; 10) Poor capability to use an inhalation device;\n10. Abnormal liver function defined as AST and/or ALT \\> 3 times the upper limit of the normal.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RNS60", "targeting_mechanism": "Reduces neuroinflammation and neurodegeneration", "targeting_mechanism_pmid": "37433768", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00919555", "title": "Combination Therapy in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Phoenix Neurological Associates, LTD", "summary": "The purpose of the study is to determine the safety and the efficacy of Tretinoin and Pioglitazone HCL in patients with ALS who are currently on Riluzole.", "interventions": [{"type": "DRUG", "name": "Pioglitazone and Tretinoin"}, {"type": "DRUG", "name": "Tretinoin and Pioglitazone HCL"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2008-06", "url": "https://clinicaltrials.gov/study/NCT00919555", "target_entities": ["PPAR\u03b3", "Retinoic acid receptors"], "locations": [{"facility": "Phoenix Neurological Associates, LTD", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* El Escorial Classification of laboratory supported probable, probable, or definite ALS\n* Age 18 - 85 years\n* Male or female\n* FVC greater than or equal to 70% predicted\n\nExclusion Criteria:\n\n* Patients with FVC below 1.5 L or below 70% predicted\n* History of liver disease\n* Severe renal failure (CrCl\\<30)\n* History of coronary artery disease requiring placement of stents, bypass surgery or previous myocardial infarction\n* EKG at baseline with evidence for previous myocardial infarction, cardiomyopathy, or arrhythmia\n* History of intolerance to Riluzole, Tretinoin, or Pioglitazone HCL\n* History of diabetes\n* Any other comorbid condition which would make completion of trial unlikely", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Pioglitazone and Tretinoin", "targeting_mechanism": "Pioglitazone acts through peroxisome proliferator-activated receptors (PPAR-\u03b3) and modulates the hypothalamic melanocortin pathway; Tretinoin acts as a retinoid receptor agonist", "targeting_mechanism_pmid": "26984187", "animal_results": "Pioglitazone modulates alterations in the hypothalamic melanocortin pathway in ALS mouse models", "animal_results_pmid": "26984187", "repurposed_from": "type 2 diabetes (Pioglitazone); acute promyelocytic leukemia (Tretinoin)", "repurposed_from_pmid": "26984187"}}
{"nct_id": "NCT07312240", "title": "LONgitudinal and Integrated Evaluation of Biomarkers in reLation to phenotYpe in ALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Istituto Auxologico Italiano", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by progressive degeneration of upper and lower motor neurons, leading to paralysis and death. Despite its uniformly fatal outcome, ALS shows marked clinical heterogeneity with respect to phenotype, progression rate, cognitive involvement, and survival. This heterogeneity limits prognostic accuracy and complicates patient stratification in both clinical practice and research settings.\n\nNeurochemical biomarkers have emerged as promising tools to improve diagnosis, prognostication, and understanding of ALS pathophysiology. Among them, neurofilament light chain (NfL) represents the most established biomarker, reflecting axonal degeneration. Additional biomarkers, including glial fibrillary acidic protein (GFAP), phosphorylated tau (p-tau181), and Alzheimer's disease-related markers (A\u03b242 and A\u03b240), may provide complementary information regarding astroglial activation, motor neuron subtype involvement, and cognitive-behavioral features. However, the phenotypic correlates, longitudinal trajectories, and biological determinants of these biomarkers in ALS are not yet fully understood.\n\nThe LONELYALS study is an ongoing, monocentric, observational cohort study with a case-control component, designed to investigate the relationships between ALS phenotype and a comprehensive panel of cerebrospinal fluid (CSF) and blood biomarkers. The study will enroll 140 adult patients with ALS and collect longitudinal clinical, neuropsychological, biological, and laboratory data over a follow-up period of up to 36 months. By integrating biomarker measurements with detailed phenotypic characterization, the study aims to clarify biomarker origins, determinants, and prognostic value, and to identify novel CSF biomarkers relevant to ALS.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "Lumbar Puncture for analysis of Cerebrospinal Fluid"}, {"type": "DIAGNOSTIC_TEST", "name": "Deep Phenotyping"}, {"type": "DIAGNOSTIC_TEST", "name": "Routine blood chemistry analysis and genetic analysis"}], "start_date": "2025-04-17", "url": "https://clinicaltrials.gov/study/NCT07312240", "target_entities": [], "locations": [{"facility": "Istituto Auxologico Italiano IRCCS", "city": "Milan", "state": "Lombardy", "country": "Italy", "status": "RECRUITING", "lat": 45.46427, "lon": 9.18951}], "contact_phone": "+3902619111", "contact_email": "f.verde@auxologico.it", "eligibility": {"criteria": "Inclusion Criteria ALS patients:\n\n* diagnosis of Amyotrophic Lateral Sclerosis (ALS);\n* age \u226518 y;\n* feasibility of lumbar puncture (LP);\n* informed consent.\n\nExclusion Criteria ALS patients:\n\n* severe medical comorbidities;\n* recent traumatic, inflammatory, vascular, or neoplastic Central Nervous System disease; contraindications to LP.\n\nInclusion Criteria Controls:\n\n* age \u226518 y;\n* individuals undergoing LP for neurological symptoms;\n* no evidence of nervous system pathology;\n* informed consent.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03652805", "title": "A Study of IPL344 in the Treatment of ALS Patients", "phase": "PHASE1, PHASE2", "status": "SUSPENDED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Immunity Pharma Ltd.", "summary": "This is a prospective, open-label, phase 1/2a study, dose escalation, to evaluate tolerability, safety, and PK of I.V. administered IPL344 in participants with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "IPL344"}], "start_date": "2018-08-01", "url": "https://clinicaltrials.gov/study/NCT03652805", "target_entities": [], "locations": [{"facility": "Hadassah Medical Center -Motor Neuron Disease Clinic", "city": "Jerusalem", "state": "", "country": "Israel", "status": "", "lat": 31.76904, "lon": 35.21633}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Male or female participants ages \u226518 to 80 years\n2. Consenting participants fulfilling the El Escorial criteria for probable and definite ALS (sporadic and familial)\n3. Participant has ALSFRS-R score \\>20, the latest ALSFRS-R test should be no more than 6 weeks before screening visit, AND:\n\n   1. a disease progression rate greater than 0.55 ALSFRS-R point per month on average, over at least 4 months, prior to the latest ALSFRS-R test OR\n   2. a decline of at least 3 points in ALSFRS-R score within the last 4 months prior to the latest ALSFRS-R test\n4. Previous data of Force Vital Capacity (FVC) of \u226560% at least 3 months before screening and not more than 12 months.\n5. Written informed consent consistent with ICH-GCP and local laws, signed prior to any study procedures being performed.\n6. BMI 18.5 to 30 kg/m2 inclusive and weigh at least 50 kg and no more than 100 kg.\n7. If taking riluzole or edaravone, the participant must be on a stable dose for \u226530 days prior to Day 1 and expected to remain at that dose until the final study visit.\n8. Medically able to undergo the study procedures, and to adhere to the visit schedule at the time of study entry.\n9. Medically is able and willing to undergo placement and maintain a central venous catheter as determined by the investigator.\n10. Participant has a competent caregiver or qualified individual who can and will be responsible for the administration of study drug and reporting home activities.\n11. Geographic accessibility to the study site\n12. Females must not be lactating or pregnant at Screening, as documented by a negative beta-human chorionic gonadotropin \\[\u00df-hCG\\] (or human chorionic gonadotropin \\[hCG\\].\n13. Women of child-bearing potential or males whose partners are women of child-bearing potential use an effective method of contraception throughout the trial.\n\nExclusion Criteria:\n\n1. Concurrent therapy that, in the PI's opinion, would interfere with the evaluation of the safety or efficacy of the study medication.\n2. Co-existing psychiatric disorder excluding a depression disorder occurred after ALS diagnosis.\n3. Participant is a respiratory dependent.\n4. Subjects with a significant pulmonary disorder not attributed to ALS.\n5. Slow Vital Capacity (SVC) \\<60.\n6. Presence of any other condition or circumstance that, in the judgment of the Investigator, might contraindicate or increase the risk to the participant or decrease the chance of obtaining satisfactory data to achieve the objectives of the study.\n7. History of HIV, positive HBV or HCV serology.\n8. Participants suffering from significant cardiac, or any other disease that may endanger the participant or interfere with the ability to interpret the results.\n9. A participant with active infections.\n10. Documented active cancer.\n11. Treatment with another investigational drug, biological agent, or device within 2 months of the first dose, or investigational cell therapy within 6 months of the first dose.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "IPL344", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04972487", "title": "Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis", "phase": "Expanded Access", "status": "APPROVED_FOR_MARKETING", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Biogen", "summary": "The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism in order to address a high unmet medical need in this population.", "interventions": [{"type": "DRUG", "name": "Tofersen"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT04972487", "target_entities": ["SOD1"], "locations": [{"facility": "Research Site", "city": "Anchorage", "state": "Alaska", "country": "United States", "status": "", "lat": 61.21806, "lon": -149.90028}, {"facility": "Research Site", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "Research Site", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Research Site", "city": "San Diego", "state": "California", "country": "United States", "status": "", "lat": 32.71571, "lon": -117.16472}, {"facility": "Research Site", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Research Site", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Research Site", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Research Site", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Research Site", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Research Site", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Research Site", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Research Site", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Research Site", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Research Site", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}, {"facility": "Research Site", "city": "Amherst", "state": "New York", "country": "United States", "status": "", "lat": 42.97839, "lon": -78.79976}, {"facility": "Research Site", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Research Site", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Research Site", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Research Site", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Key Inclusion Criteria:\n\n* Medically able to undergo the program procedures, as determined by the treating healthcare professional (HCP).\n* Weakness attributable to ALS and associated with a mutation in the SOD1 gene (SOD1-ALS).\n\nKey Exclusion Criteria:\n\n* Previous or current participation in a clinical trial of tofersen.\n* Use of an investigational medicinal product (IMP) for amyotrophic lateral sclerosis (ALS) within 5 half-lives of the IMP before the first dose of tofersen.\n* Participant's primary place of residence is outside of the country of treatment.\n\nNOTE: Other protocol defined Inclusion/Exclusion criteria may apply", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Tofersen", "targeting_mechanism": "Antisense oligonucleotide targeting SOD1 mRNA to reduce mutant SOD1 protein expression.", "targeting_mechanism_pmid": "37975798", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00204464", "title": "Study of the Effects Strengthening Exercises in Individuals With ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Saskatchewan", "summary": "Amyotrophic Lateral Sclerosis (ALS) is the most common motor neuron disease MND) among adults. Motor neurons in the spinal cord, brain stem, and cerebral motor cortex degenerate and create a variety of upper (UMN) and lower motor neuron (LMN) clinical signs and symptoms, with the most frequently presenting symptom being focal weakness beginning in the leg, arm, or bulbar muscles, occurring in more than 70% of patients. Despite the high incidence of muscle weakness in patients with ALS, only two case studies evaluating the effects of specific muscle strengthening and endurance exercise programs in this patient population have been published, and the effects of resistive exercise programs in patients with ALS have not been well studied. Some have discouraged exercise programs in patients with ALS because of fear of overuse weakness. Yet, in patients with other neuromuscular diseases, resistive exercise programs have been shown to be beneficial and have not produced overuse weakness. The purpose of this study is to determine the effects of six months strengthening program on strength, function, fatigue and quality of life in individuals with ALS.", "interventions": [{"type": "PROCEDURE", "name": "Exercise"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT00204464", "target_entities": [], "locations": [{"facility": "Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* definite and/or probable diagnosis of ALS\n* early stages of the disease\n\nExclusion Criteria:\n\n* history of neuromuscular dysfunction not related to ALS\n* active, confounding medical conditions\n* unwillingness or inability to comply with the protocol\n* FVC of less than 90% predicted\n* ALSFRS score of less than 30", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06256107", "title": "Virtual Reality in Motor Neurone Disease", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Lancashire Teaching Hospitals NHS Foundation Trust", "summary": "Motor Neurone Disease (MND) is a chronic progressive neurological condition where people experience weakness of muscles leading to pain and restriction of movement as well as problems with swallowing, breathing and communication. The purpose of this study is to establish if Virtual Reality is useful for people with MND and if it helps improve their well being.", "interventions": [{"type": "DEVICE", "name": "Virtual Reality Headset"}], "start_date": "2021-01-13", "url": "https://clinicaltrials.gov/study/NCT06256107", "target_entities": [], "locations": [{"facility": "Lancashire Teaching Hospitals NHS Foundation Trust", "city": "Preston", "state": "", "country": "United Kingdom", "status": "", "lat": 53.76282, "lon": -2.70452}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Diagnosis of MND\n2. Sufficient cognitive ability to understand instructions with regards using the VR kit (ECAS score - minimum 100/136)\n3. Has sufficient motor ability/dexterity to use the kit or a carer who will be able to assist with the use of the kit.\n4. Can tolerate light and have sufficient head control to wear the head set.\n5. English speaking\n\nExclusion Criteria:\n\n1. Poor cognition - inability to understand instructions regarding the use of VR (Total ECAS Score \\<100/136)\n2. Unable to tolerate light/unable to wear the head set\n3. Light sensitive epilepsy, severe vertigo or dizziness.\n4. Non English speaking", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04948645", "title": "A Phase 1 Study to Investigate the Safety and Pharmacokinetics of Fosigotifator in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AbbVie", "summary": "Fosigotifator is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is an up to 156-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be up to a 152-week active treatment extension (ATE) during which all subjects will receive Fosigotifator.", "interventions": [{"type": "DRUG", "name": "Fosigotifator"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-09-22", "url": "https://clinicaltrials.gov/study/NCT04948645", "target_entities": [], "locations": [{"facility": "UC Irvine Health ALS and Neuromuscular Center", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "Forbes Norris MDA/ALS Research and Treatment Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Johns Hopkins ALS Clinical Trials Unit", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Healey & AMG Center for ALS Research", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "University of Calgary - Heritage Medical Research Clinic", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook Research Institute", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Centre Hospitalier de l'Universite de Montreal (CHUM)", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Must have an identified, reliable caregiver.\n* Confirmed diagnosis of Familial Amyotrophic Lateral Sclerosis (ALS) or Sporadic ALS.\n* First ALS symptoms occurred \\<= 36 months before screening.\n* Able to swallow solids.\n* No known active COVID-19 infection at screening.\n* Slow vital capacity (SVC) \\>= 50% predicted value (for sex, age, ethnic origin, and height) at screening.\n* If taking concomitant standard-of-care medications approved for the treatment of ALS (or their components), subjects must be on a stable dose of the medication(s) for \\>30 days prior to Baseline in order to enter the study. For edaravone, a stable dose is defined by having completed 2 treatment cycles prior to Baseline.\n\nExclusion Criteria:\n\n* History of dementia/severe cognitive problems at screening.\n* History of clinically significant medical conditions (other than ALS) or any other reason, including any physical, psychological, or psychiatric condition that, in the opinion of the Investigator, would compromise the safety or interfere with the subject's participation in the study, or would make the subject an unsuitable candidate to receive study drug, or would put the subject at risk by participating in the study.\n* History of abnormal screening laboratory or imaging results that, in the opinion of the Investigator, are indicative of any significant cardiac, endocrinologic, hematologic, hepatic, immunologic, infectious, metabolic, urologic, pulmonary, gastrointestinal, dermatologic, psychiatric, renal, neurologic, and/or other major disease that would preclude administration of Fosigotifator.\n* Documented active or suspected malignancy or history of any malignancy within the last 5 years except for successfully treated non-melanoma skin cancer or localized carcinoma in situ of the cervix.\n* If female, is known to be pregnant, breastfeeding, considering becoming pregnant, or donating/banking eggs during the study or within 30 days or \\>5 half-lives (whichever is longer) after the last dose of study drug.\n* If male, plans to donate sperm or father a child during the study or within 30 days after the last dose of study drug.\n* Known to have received any investigational product within 30 days or 5 half-lives of the drug (whichever is longer) prior to the first dose of study drug or is currently enrolled in another clinical study.\n* History of Fosigotifator use prior to participation in this study.\n* Recent (within 6 months prior to Screening) history of drug or alcohol abuse.\n* Previous participation in a stem cell clinical study for treatment of ALS.\n* Current or anticipated use of diaphragmatic pacing during the study period.\n* Tracheostomy or use of non-invasive ventilatory support \\>= 22 hours a day.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Fosigotifator", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07169175", "title": "A Phase \u2160/\u2161a Study of SNUG01 in Adult Subjects With ALS", "phase": "PHASE1, PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "SineuGene Therapeutics Co., Ltd.", "summary": "The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "SNUG01"}], "start_date": "2025-12-01", "url": "https://clinicaltrials.gov/study/NCT07169175", "target_entities": [], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Peking University Third Hospital", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}, {"facility": "Fujian Medical University Union Hospital", "city": "Fuzhou", "state": "Fujian", "country": "China", "status": "", "lat": 26.06139, "lon": 119.30611}, {"facility": "Second Affiliated Hospital Zhejiang University School of Medicine", "city": "Hangzhou", "state": "Zhejiang", "country": "China", "status": "", "lat": 30.29365, "lon": 120.16142}], "contact_phone": "+8617274855306", "contact_email": "patient@sineugene.com", "eligibility": {"criteria": "* Key Inclusion Criteria\\*\\*:\n\n  * Subjects who are able to provide written informed consent form (ICF).\n  * Subjects who are males or females must be \u2265 18 years and \u2264 80 years of age at the screening visit.\n  * Subjects who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised version of the El Escorial World Federation of Neurology criteria.\n  * Subjects must have an ALS disease duration (from first symptom onset to the screening visit) \u2264 2 years.\n  * Subjects with a body mass index (BMI) \u2265 19 kg/m2 at the screening visit.\n  * Subjects whose percent-predicted Forced Vital Capacity (%FVC) is \u2265 70% or percent-predicted Slow Vital Capacity (%SVC) is \u2265 60%, adjusted for sex, age, and height at the screening visit.\n  * The ALSFRS-R score \u2265 30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be full marks.\n* Key Exclusion Criteria\\*\\*:\n\n  * Serum Anti-AAV9 neutralizing antibody titer \u2265 1:100.\n  * Current or previous exposure to gene therapy, stem cell products, and solid organ transplantation.\n  * Subjects who have implanted or are estimated to require a diaphragmatic pacing system during the study period.\n  * Any thromboembolic event, such as deep vein thrombosis, pulmonary arteriovenous embolism, and jugular vein embolism, has occurred within 6 months before the administration.\n  * Suffering from autoimmune diseases or ongoing immune-related therapy, except intranasal, inhalation, ocular, topical, intra-articular corticosteroid therapy or corticosteroid physiological replacement therapy.\n  * Active or chronic uncontrolled infection within 4 weeks before the administration, deemed unacceptable in the discretion of the investigator.\n  * Evidence of human immunodeficiency virus (HIV) and treponema pallidum (TP) infection, as documented by the treatment for HIV or TP, or by HIV or TP antibodies positivity at the screening visit.\n  * Has a positive serum pregnancy test at screening (females of childbearing potential only), a positive urine or serum pregnancy test at baseline (Day -1. females of childbearing potential only), or is nursing.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "SNUG01", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04428775", "title": "A Safety and Biomarker Study of ALZT-OP1a in Subjects With Mild-Moderate ALS Disease", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AZTherapies, Inc.", "summary": "This is a Phase IIa, randomized, open-label, multi-center, multi-dose study for subjects with mild to moderate ALS. The protocol is designed to determine whether ALZT-OP1a treatment will positively impact neuro-inflammatory biomarkers and slow down or arrest functional decline in subjects with mild to moderate ALS.", "interventions": [{"type": "DRUG", "name": "ALZT-OP1a (cromolyn)"}], "start_date": "2020-09-08", "url": "https://clinicaltrials.gov/study/NCT04428775", "target_entities": ["neuroinflammation"], "locations": [{"facility": "UCSD Altman Clinical and Translational Research Institute", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Wake Forest School of Medicine", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Oregon Health & Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female subjects aged 18-75 years, both inclusive;\n* Must provide written informed consent before any study related procedures;\n* Should be capable to complete all trial related procedures, assessments and visits in the judgement of Investigator;\n* Familial or sporadic ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria;\n* Disease duration from ALS diagnosis \u226424 months;\n* ALSFRS-R total score \u2265 36 at screening visit;\n* ALSFRS-R Breathing sub-score should be \u22659 at the time of screening;\n* ALSFRS-R Bulbar sub-score should be \u22659 at the time of screening;\n* Peak inspiratory flow rate (PIFR) \u2265 100 L/minute;\n* Forced vital capacity (FVC) \\>70% of predicted value;\n* Participant must be receiving treatment with stable dose of standard of care treatment for \u226530 days prior to signing informed consent.\n\nExclusion Criteria:\n\n* Subjects with bulbar-onset ALS;\n* Any use of non-invasive ventilation (e.g., continuous positive airway pressure, non-invasive bi-level positive airway pressure or non-invasive volume ventilation) for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation;\n* Any other significant neurological disorder which can interfere with study assessments, e.g., significant cognitive impairment and/or clinical dementia;\n* Significant psychiatric illness like schizophrenia, bipolar disorder etc. Subjects with depression can be included, only if the depression has been stable and no episode of major depression has occurred in past one year;\n* Severe cardiac disease (e.g.,corrected QT interval \\> 500ms), Torsade de Pointes, evidence of significant heart failure (New York Heart Association \\[NYHA\\] Class 3 or greater, myocardial infarction or unstable angina in the 6 months prior to screening);\n* Any moderate-to-severe pulmonary disease or difficulty taking inhaled drugs;\n* Inability to tolerate the administration of an oral inhaled powder via dry powder inhaler (DPI);\n* Has taken any investigational study drug within 30 days or five half-lives of the drug, whichever is longer, prior to dosing;\n* Currently taking cromolyn, or has taken cromolyn, within the past 12 months;\n* Allergy to cromolyn or cromolyn products, such as Intal\u00ae, Nasalcrom\u00ae, Opticrom\u00ae, Gastrocrom\u00ae, etc.;\n* Taking inhaled protein products on a chronic basis (such as insulin, parathyroid hormone \\[PTH\\], etc.);\n* Subjects who weigh 88 lb (40 kg) or less, or, body mass index (BMI) of \\<17.5 or \\>35.0 at screening;\n* Moderate-to-severe liver disease: aspartate aminotransferase (AST), alanine aminotransferase (ALT), or bilirubin concentrations \\>3 times the upper limit of normal; patients with hepatic diseases such as hepatic cirrhosis, hepatic cancer and active hepatitis\n* Moderate-to-severe renal disease: creatinine clearance \\<45 mL/min/1.73 m2 (by Cockcroft-Gault calculation);\n* Any clinically significant disorder or laboratory abnormality that, in the investigator's opinion, could interfere with the subject's participation in the study, place the subject at increased risk, or confound interpretation of the study results;\n* Pregnant or breast-feeding females or sexually active females with childbearing potential, if no adequate contraceptive measures are used.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "cromolyn (ALZT-OP1a)", "targeting_mechanism": "Reduces neuroinflammation to slow or arrest functional decline in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07567664", "title": "Tracking and Predicting How Brain Damage Spreads in Neurodegenerative Diseases", "phase": "NA", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "IRCCS San Raffaele", "summary": "Neurodegenerative diseases, including frontotemporal lobar degeneration (FTLD) spectrum syndromes, are characterized by the accumulation of insoluble protein aggregates in the central nervous system. A common feature of these diseases is that pathological changes accumulate over time following a stereotyped spatial pattern, which contributes to the onset and progression of clinical symptoms. Until recently, the causes of such progression were still unknown. Recent pathological and neuroimaging studies have, however, suggested that insoluble and pathological protein aggregates are able to alter the conformation of neighboring proteins and spread through cell-to-cell transmission. According to this theory, called the 'brain connectome,' the brain network is established as a set of nodes, which correspond to different anatomical regions.\n\nThese brain networks are highly connected to each other and their internal organization is fundamental for an efficient integration of information coming from different regions and to guarantee adequate levels of motor/cognitive performance. Thanks to magnetic resonance studies and research in the field of brain networks, it is possible to understand the pathophysiology of neurodegenerative diseases and reveal the connectivity profiles associated with different clinical outcomes.\n\nThe main objective of this project is to explore the mechanisms of neurodegeneration associated with the different FTLD spectrum syndromes, and in particular the hypothesis that the neurodegenerative process is driven by the structural architecture of the brain 'connectome'. The ultimate goal is to apply mathematical models to structural and functional connectivity data to predict the evolution of the neurodegenerative process in sporadic and genetic forms of Frontotemporal Lobar Degeneration Disease.\n\nThis study aims to investigate the spatiotemporal progression of neurodegeneration in frontotemporal lobar degeneration (FTLD) using advanced neuroimaging and connectomics. 360 patients with sporadic FTLD (including bvFTD, semantic and nonfluent PPA, PSPs, CBS, and ALS) and 65 patients with genetic FTLD (MAPT, GRN, and C9orf72 mutati will be enrolled. The study also plans to enroll 120 subjects who are members of families carrying FLTD-associated mutations (including 60 mutation carriers). Finally, 100 healthy controls will also be enrolled, including 50 young healthy controls and 50 healthy controls comparable with patients by sex and age. Participants will undergo clinical, neuropsychological, and behavioral assessments, blood and Cerebrospinal fluid (CSF) collection, and multimodal 3Tesla Magnetic Resonance Imaging MRI at baseline and every 6 months for up to 2 years. Primary objectives include mapping longitudinal changes in structural and functional brain networks, developing predictive models of network degeneration and clinical decline, and characterizing protein-specific patterns of network degeneration. Secondary aims include identifying early network biomarkers in presymptomatic carriers and correlating network changes with biological markers.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "3 Tesla MRI without contrast medium"}, {"type": "GENETIC", "name": "Blood sample for genetic analysis"}, {"type": "DIAGNOSTIC_TEST", "name": "Cerebrospinal fluid sampling (CSF)"}, {"type": "DIAGNOSTIC_TEST", "name": "Neurological evaluation"}, {"type": "DIAGNOSTIC_TEST", "name": "Neuropsychological evaluation"}], "start_date": "2017-06-01", "url": "https://clinicaltrials.gov/study/NCT07567664", "target_entities": ["protein_aggregation"], "locations": [{"facility": "IRCCS San Raffaele", "city": "Milan", "state": "Lombardy", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nAdult participants, under 85 years of age, diagnosed with bvFTD, semantic variant PPA, non-fluent variant PPA, PSP, CBS, and early-stage ALS, according to the criteria of Rascovsky (2011), Gorno-Tempini (2011), Litvan (1996), Armstrong (2013), and Brooks (2000), respectively;\n\nParticipants with genetic forms of FTLD associated with mutations in the c9orf72, GRN, MAPT genes, and asymptomatic family members related to FTLD patients carrying such mutations\n\nHealthy participants (age between 20 and 30 years old); Healthy participants matched to patients for age and sex\n\nExclusion Criteria:\n\n* Participants with a history of other neurological and/or psychiatric disorders, head trauma, alcohol or psychoactive substance use, or a family history of other neurodegenerative diseases.", "sex": "ALL", "min_age": "20 Years", "max_age": "85 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04797845", "title": "Patient's TeleMonitoring With Amyotrophic Lateral Sclerosis Treated by Non Invasive Ventilation at Home.", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Toulouse", "summary": "Single-center, prospective pilot study on patients with amyotrophic lateral sclerosis fitted with noninvasive ventilation. The objective is to assess the satisfaction of remote monitoring of patients on non-invasive ventilation after 12 months.", "interventions": [{"type": "OTHER", "name": "teleconsultation"}], "start_date": "2021-04-12", "url": "https://clinicaltrials.gov/study/NCT04797845", "target_entities": [], "locations": [{"facility": "Toulouse University Hospital", "city": "Toulouse", "state": "", "country": "France", "status": "", "lat": 43.60426, "lon": 1.44367}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "o inclusion criteria:\n\n* Patient over 18 years,\n* Patient with Amyotrophic Lateral Sclerosis (ALS),\n* Already fitted by Non Invasive Ventilation (NIV),\n* Patient having signed the informed consent\n* Patient affiliated to a social security scheme,\n* Patient with a correct understanding of the French language,\n* Patient with access to an internet connection at home.\n\nFor the caregiver:\n\n* Adult person\n* Be the patient's primary caregiver\n* Have signed the informed consent intended for the caregiver\n\n  o exclusion criteria:\n* Patient with another cause of chronic respiratory failure: other neuromuscular diseases, Chronic obstructive pulmonary disease (COPD), diffuse interstitial lung disease,\n* Patient under guardianship or under judicial protection,\n* Patient dependent at least 20 hours out of 24 of the non invasive Ventilation (NIV).", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06429735", "title": "Precise Robotically IMplanted Brain-Computer InterfacE", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuralink Corp", "summary": "The PRIME Study is a first-in-human early feasibility study to evaluate the initial clinical safety and device functionality of the Neuralink N1 Implant and R1 Robot device designs in participants with tetraparesis or tetraplegia. The N1 Implant is a skull-mounted, wireless, rechargeable implant connected to electrode threads that are implanted in the brain by the R1 Robot, a robotic electrode thread inserter.", "interventions": [{"type": "DEVICE", "name": "N1 Implant"}, {"type": "DEVICE", "name": "R1 Robot"}], "start_date": "2024-01-09", "url": "https://clinicaltrials.gov/study/NCT06429735", "target_entities": [], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "RECRUITING", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 25.77427, "lon": -80.19366}], "contact_phone": "(877) 398-4465", "contact_email": "clinical-team-ct@neuralink.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Severe quadriplegia (tetraplegia) due to spinal cord injury or amyotrophic lateral sclerosis (ALS) for at least 1 year without improvement, where quadriplegia is defined as having very limited or no hand, wrist, and arm movement and all levels below\n* Life expectancy \u2265 12 months.\n* Ability to communicate in English\n* Presence of a stable caregiver\n\nExclusion Criteria\n\n* Moderate to high risk for serious perioperative adverse events\n* Active implanted devices\n* Morbid obesity (Body Mass Index \\> 40)\n* History of poorly controlled seizures or epilepsy\n* History of poorly controlled diabetes\n* Requires magnetic resonance imaging (MRI) for any ongoing medical conditions\n* Acquired or hereditary immunosuppression\n* Use of smoking tobacco or other tobacco products\n* Psychiatric or psychological disorder\n* Brain MRI demonstrating hemorrhage, tumor, distorted or adverse anatomy.\n* Any condition which, in the opinion of the Investigator, would compromise your ability to safely participate in the study or undergo the implantation procedure", "sex": "ALL", "min_age": "22 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00635960", "title": "Growth Hormone in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Federico II University", "summary": "Several drugs have been proposed for ALS. These drugs included: Topiramate, Lamotrigine, creatine, Vit. E, Pentoxifylline, etc. Although most of the trials showed a positive trend, none of them reached a statistically significant result. The only exception is the Riluzole trial, that demonstrated a small but significant reduction in mortality between treated and untreated patients. Aim of our study is to determine if the add-on of GH to treatment with Riluzole is able to reduce neuronal loss in the motor cortex of ALS patients.", "interventions": [{"type": "DRUG", "name": "Growth Hormone (Somatropin)"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2007-03", "url": "https://clinicaltrials.gov/study/NCT00635960", "target_entities": ["growth_hormone_signaling"], "locations": [{"facility": "Diparimento di Scienze Neurologiche", "city": "Naples", "state": "", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}, {"facility": "Istituto Biostrutture e Bioimmagini, Consiglio Nazionale delle Ricerche", "city": "Naples", "state": "", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Definite/probable ALS according to El Escorial criteria\n* Aged \\> 40, \\< 85 years\n* Progression from onset\n* Disease duration \u22643 years\n* Treatment with Riluzole\n\nExclusion Criteria:\n\n* Rapid disease progression in the first 6 months after diagnosis\n* Patients with tracheostomy and/or Gastrostomy\n* Disease duration \\> 3 years\n* Patient with exclusive bulbar or 2\u00b0 motorneuron involvement\n* Hepatic/renal failure\n* Pregnant or breastfeeding\n* Signs of active neoplasia\n* Complicated Diabetes\n* Severe hypertension\n* Unable to undergo MRI exams", "sex": "ALL", "min_age": "40 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Somatropin (Growth Hormone)", "targeting_mechanism": "Growth hormone (GH)/IGF-1 axis promotes neurotrophic and neuroregenerative effects in the CNS.", "targeting_mechanism_pmid": "29149058", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03887338", "title": "Systematic Laryngoscopic Evaluation of Upper Airways in Ventilated ALS Patients in Portugal and in Norway", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Tiina Maarit Andersen", "summary": "The study examines if titration of Non-Invasive mechanical ventilation (NIV) settings during ongoing laryngoscopic visualization can improve the compliance of NIV in subjects with Amyotrophic Lateral Sclerosis (ALS). The study is a multicentre study between Norwegian National Advisory Unit on Long-term Mechanical Ventilation at the Thoracic Department, Haukeland University Hospital, Bergen, Norway and Centro Hospital Tras-os-Montes e Alto Douro, Vila Real, Portugal.", "interventions": [{"type": "OTHER", "name": "Transnasal Fiberoptic Laryngoscopy"}], "start_date": "2019-03-21", "url": "https://clinicaltrials.gov/study/NCT03887338", "target_entities": [], "locations": [{"facility": "Norwegian National Advisory Unit on Long-term Mechanical Ventilation at Thoracic Department, Haukeland University Hospital", "city": "Bergen", "state": "Hordaland", "country": "Norway", "status": "", "lat": 60.39299, "lon": 5.32415}, {"facility": "Centro Hospitalar Tras-os-Montes e Alto Douro", "city": "Vila Real", "state": "", "country": "Portugal", "status": "", "lat": 41.3001, "lon": -7.7432}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients diagnosed Amyotrophic Lateral Sclerosis (ALS) due to El Escorial criteria\n* Must have already established Non-invasive ventilation (NIV)\n\nExclusion Criteria:\n\n* Ages under 18 years\n* Unstable ischemic heart disease\n* Oncological disease\n* Ventilatory support dependency (more than 16h per day)", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02590276", "title": "Predict to Prevent Frontotemporal Lobar Degeneration (FDT) and Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "The project focuses on C9orf72, the most frequent genetic form of frontotemporal dementias (FTD, or frontotemporal lobar degeneration, FTLD) and amyotrophic lateral sclerosis (ALS). FTD is the second commonest cause of degenerative dementia in presenium after Alzheimer's disease. Behavioural and cognitive impairments progressively lead to dementia. ALS produces progressive muscle weakness leading to the death in 2 to 4 years. Since 2006, major discoveries have linked FTLD and ALS:\n\n1. TDP-43 aggregates in neurons and\n2. C9orf72 mutations is a major genetic cause in both disorders.\n\nTwo major pathological subtypes are now defined in FTD, FTD-TDP and FTD-TAU. C9orf72 mutations (associated to FTD-TDP) are the most frequent genetic causes of FTD (15%), FTD-ALS (65%) and ALS (40%).\n\nFTD is difficult at an early stage; and no clinical, biological or imaging features can predict the underlying pathology in living patients. Therapeutic perspectives emerged against tau aggregation, progranulin deficit and C9orf72 expansion (antisense). Presymptomatic carriers of genetic FTD would benefit, before onset of symptoms, from these therapeutic that would delay or prevent the disease. At this step, it becomes crucial to develop markers to know how many years before symptoms, does the pathological progress begin, to treat the patients at the most early stage of the disease. Markers are also needed to predict the pathology (FTD-TDP/FTD-tau) in patients that will be eligible for trials targeting specific pathological lesion.", "interventions": [{"type": "BEHAVIORAL", "name": "characterization"}], "start_date": "2015-10-08", "url": "https://clinicaltrials.gov/study/NCT02590276", "target_entities": ["C9orf72"], "locations": [{"facility": "Groupe Hospitalier Piti\u00e9-Salp\u00eatri\u00e8re - Charles Foix", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age \u2265 18\n* Signed informed consent for genetic and clinical study\n* To be carrier of a C9ORF72 mutation - Diagnosis criteria of FTD or ALS\n* To be French-speaking\n* To be affiliated to the social security scheme\n* Absence of another intercurrent neurological pathology (vascular cerebral accident, tumor, etc.....)\n\nInclusion criteria for asymptomatic relatives\n\n* Age \u2265 18\n* To be first degree relative of a person carrying a C9ORF72 mutation OR first degree relative of FTD or ALS deceased patient whose C9ORF72 mutation as been identified in the family\n* Signed informed consent for genetic and clinical study\n* To be French-speaking\n* To be affiliated to the social security scheme\n* Absence of proven neurologic disorders or an intercurrent neurological pathology (vascular cerebral accident, tumor, etc.....)\n\nExclusion Criteria:\n\n* Contra-indication to perform a brain MRI (wearing of pacemaker, cardiac valve or incompatible vascular MRI surgical equipment , neurosurgery or surgery vascular equipment, surgical equipment likely to concentrate the radio frequency field, intra ocular or intra cerebral metal foreign body, claustrophobia, wearing a non-compliant radio intrauterine device ),\n* Inability to lie one hour without moving\n* PET-FDG contra-indication\n* Breastfeeding and pregnant women\n* Human chorionic gonadotrophin (B\u00e9t\u00e2-HCG) positive determination or Positive urine pregnancy test for women of childbearing age\n\nExclusion criteria for related asymptomatic :\n\n* Clinical proven signs of FTD, language disorder, praxis disorder, mnemic, of parkinson's syndrome or amyotrophic lateral sclerosis.\n* Counter-indication to perform a brain MRI (wearing of pacemaker, cardiac valve or incompatible vascular MRI surgical equipment , neurosurgery or surgery vascular equipment, surgical equipment likely to concentrate the radio frequency field, intra ocular or intra cerebral metal foreign body, claustrophobia, wearing a non-compliant radio intrauterine device )\n* Severe chronic alcoholism\n* PET-FDG contre-indication\n* Inability to lie one hour without moving\n* B\u00e9t\u00e2-HCG positive determination or Positive urine pregnancy test for women of childbearing age", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00706147", "title": "Phase II/III Randomized, Placebo-controlled Trial of Arimoclomol in SOD1 Positive Familial Amyotrophic Lateral Sclerosis", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Miami", "summary": "The purpose of this study will be to demonstrate the safety, tolerability, and efficacy of arimoclomol in subjects with SOD1 positive familial Amyotrophic Lateral Sclerosis (ALS). This type of ALS is HEREDITARY (runs in families), and at least one other person in the family must have had ALS.\n\nStudy hypotheses: Arimoclomol, taken at a dose of 200 mg three times daily will improve survival as defined by time to death, tracheostomy or permanent assisted ventilation. In addition, it will be safe and well tolerated in subjects with SOD1 positive familial ALS.\n\nFunding Source - FDA-OOPD", "interventions": [{"type": "DRUG", "name": "Arimoclomol"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2009-01", "url": "https://clinicaltrials.gov/study/NCT00706147", "target_entities": ["SOD1", "heat_shock_response"], "locations": [{"facility": "University of Miami Miller School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Type of ALS that is hereditary (runs in families) only.\n* El Escorial criteria for familial ALS and a family history of a pathogenic mutation in a gene known to be associated with ALS, such as the SOD1 gene.\n* Willingness to undergo genetic testing and to learn the results.\n* Demonstrable mutation in the SOD1 gene that is reported to be associated with a rapid rate of disease progression (i.e. A4V, A4T, C6F, C6G, V7E, L8Q, G10V, G41S, H43R, H48Q, D90V, G93A, D101H, D101Y, L106V, I112M, I112T, R115G, L126X, G127X, A145T, V148G, V148I) or possibly associated with rapidly progressive disease (E21G, G37R, L38V, D76Y, L84F, L84V, N86S, D90A het, G93R, I104F, I113T, L144F, L144S).\n* Age 18 years or older; male or female.\n* Capable of providing informed consent and complying with trial procedures.\n* Diagnosis within less than 9 months of the anticipated date of the baseline visit AND study participants' subjective evaluation that they expect their physical condition to permit travel to the study site for both the baseline and 2-month study visits.\n* Women must not be able to become pregnant (e.g. post menopausal for at least one year, surgically sterile, or practicing adequate birth control methods) for the duration of the study. Adequate contraception includes: oral contraception, implanted contraception, intrauterine device in place for at least 3 months, or barrier method in conjunction with spermicide.\n* Women of childbearing potential must have a negative pregnancy test at screening visit and be non-lactating.\n* Willing to remain on a stable dose of Riluzole or to remain off Riluzole for the duration of the trial.\n* Identifiable local medical doctor to assist with urgent care of any medical complications that may arise.\n* Absence of any of the exclusion criteria.\n\nExclusion Criteria:\n\n* History of known sensitivity or intolerability to Arimoclomol or to any other related compound.\n* Exposure to any investigational drug within 30 days of the screening visit.\n* Presence of any of the following clinical conditions:\n\n  * Substance abuse within the past year.\n  * Unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active infectious disease.\n  * AIDS or AIDS-related complex.\n  * Unstable psychiatric illness defined as psychosis (hallucinations or delusions), untreated major depression within 90 days of the screening visit.\n  * Positive pregnancy test at screening visit.\n* Screening laboratory values:\n\n  * Creatinine greater than 1.5.\n  * Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST). greater than 3.0 times the upper limit of normal.\n  * Total bilirubin greater than 2.0 times the upper limit of normal.\n  * White blood cell (WBC) count less than 3,500/mm3.\n  * Platelet concentration less than 100,000/ul.\n  * Hematocrit level less than 33 for female or less than 35 for male.\n* Female patients who are breast-feeding.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Arimoclomol", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04380649", "title": "Development and Test of a Headset for BCI Until Obtaining an Efficient and Comfortable System That Can be Used in Daily Practice by ALS People", "phase": "NA", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Centre Hospitalier Universitaire de Nice", "summary": "\"Brain-computer interfaces (BCIs) are computer-based systems that acquire brain signals, analyze them, and translate them into commands that are relayed to an output device to carry out a desired action. BCIs represent a very active and promising field of research among devices for people with severe motor disabilities. As the currently available systems correspond to research prototypes, they are not adapted to daily live situations. On the other hand, some systems have recently been commercialized, principally for video games but they are not satisfactory for use as a substitute technology in disability.\n\nA BCI's prototype for alternative communication using a virtual keyboard, the P300 Speller, has been developed by the National Institute for Research in Digital Science and Technology (Athena team - Nice University). This prototype includes an EEG-cap with gel based active electrodes. A recent study conducted on 20 patients with ALS (University Hospital, Nice) demonstrated the usability of the system and the patient satisfaction concerning the ease of use and utility. To achieve a system that can be used in daily live in severely disabled patients, technical developments are necessary.\n\nThe investigators have conceptualized and developed an ergonomic, comfortable, headset, including dry electrodes to allow a prolonged use of the system.\n\nThe purpose of the study conducted all along the development of the headset is to improve the developed system until a successful system is achieved. This study is a monocentric usability study conducted on ALS people.", "interventions": [{"type": "DEVICE", "name": "New headset prototype"}], "start_date": "2023-02-13", "url": "https://clinicaltrials.gov/study/NCT04380649", "target_entities": [], "locations": [{"facility": "CHU de Nice", "city": "Nice", "state": "Provence-Alpes-C\u00f4te d'Azur Region", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion criteria\n\n1. Age \u2265 18 years old\n2. For women of childbearing potential, use of contraception for the duration of the study\n3. Diagnosis of suspected ALS, possible, probable with ENMG, probable, defined according to the criteria of the World Federation of Neurology (revised El Escorial criteria, Airlie House Conference 1998)\n4. Understanding of the objective of the study after describing the principle of the P300 Speller and the course of the study\n5. Ability to follow the study procedure and to comply with the schedule of visits when entering the study\n6. Expression of a P300 wave under the conditions of the study\n\nExclusion criteria\n\n1. Psychiatric illness or dementia that may interfere with the patient's ability to follow study procedures\n2. History of photosensitive epilepsy\n3. Patient subject to protective measures\n4. Non-correctable visual disturbances\n5. Limited ability to concentrate.\n6. Women who are pregnant or breastfeeding or who have planned a pregnancy during the course of the study.\n7. Persons deprived of their liberty by a judicial or administrative decision", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01622088", "title": "Phase 3 Extension Study of Dexpramipexole in ALS", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.", "interventions": [{"type": "DRUG", "name": "Dexpramipexole"}], "start_date": "2012-06", "url": "https://clinicaltrials.gov/study/NCT01622088", "target_entities": ["mitochondrial_function"], "locations": [{"facility": "Barrow Neurological Institute - St. Joseph's Hospital", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California at San Francisco - Fresno", "city": "Fresno", "state": "California", "country": "United States", "status": "", "lat": 36.74773, "lon": -119.77237}, {"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "University of California, Davis", "city": "Sacramento", "state": "California", "country": "United States", "status": "", "lat": 38.58157, "lon": -121.4944}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "Mayo Clinic - Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami Miller School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of South Florida Medical Center", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Indiana University", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "St. Mary's Health Care", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Mayo Clinic - Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates, P.C.", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "University of Nevada School of Medicine", "city": "Las Vegas", "state": "Nevada", "country": "United States", "status": "", "lat": 36.17497, "lon": -115.13722}, {"facility": "Dartmouth-Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Research Foundation of the State University of New York", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest University", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University College of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "ALS Center at Penn", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburgh Medical Center", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Vanderbilt University Medical Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Texas Health Sciences Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Prince of Wales Hospital", "city": "Randwick", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.91439, "lon": 151.24895}, {"facility": "Westmead Hospital", "city": "Westmead", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.80383, "lon": 150.98768}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Calvary Health Care Bethlehem", "city": "Melbourne", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.814, "lon": 144.96332}, {"facility": "AZ St-Lucas", "city": "Ghent", "state": "", "country": "Belgium", "status": "", "lat": 51.05, "lon": 3.71667}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Univ of Calgary / Foothills MC", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "CHUM - Hopital Notre Dame", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Mcgill University", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "London Health Sciences Centre", "city": "London", "state": "", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook and Women's College and Health Sciences Centre", "city": "Toronto", "state": "", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "University of British Columbia", "city": "Vancouver", "state": "", "country": "Canada", "status": "", "lat": 49.24966, "lon": -123.11934}, {"facility": "CHRU de Lille - H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Centre Hospitalier La Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHU Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nice - H\u00f4pital de l'Archet 1", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "H\u00f4pital La Piti\u00e9 Salp\u00e9tri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Charit\u00e9 - Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Bergmannsheil Gmbh", "city": "Bochum", "state": "", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Medizinische Hochschule Hannover (MHH)", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4tsklinikum Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "University of Ulm, RKU", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Academisch Medisch Centrum", "city": "Amsterdam", "state": "", "country": "Netherlands", "status": "", "lat": 52.37403, "lon": 4.88969}, {"facility": "UMC St. Radboud", "city": "Nijmegen", "state": "", "country": "Netherlands", "status": "", "lat": 51.8425, "lon": 5.85278}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Universitario de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Vall d'Hebron", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital La Paz", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Carlos III", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Sahlgrenska Universitetssjukhuset", "city": "Gothenburg", "state": "", "country": "Sweden", "status": "", "lat": 57.70716, "lon": 11.96679}, {"facility": "Karolinska Universitetssjukhuset, Solna", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Queen Elizabeth Hospital", "city": "Birmingham", "state": "", "country": "United Kingdom", "status": "", "lat": 52.48142, "lon": -1.89983}, {"facility": "Walton Centre for Neurology & Neurosurgery", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "Kings College Hospital NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Newcastle University Hospital - Clinical Ageing Research Unit", "city": "Newcastle", "state": "", "country": "United Kingdom", "status": "", "lat": 54.21804, "lon": -5.88979}, {"facility": "John Radcliffe Hospital", "city": "Oxford", "state": "", "country": "United Kingdom", "status": "", "lat": 51.75222, "lon": -1.25596}, {"facility": "Sheffield Institute for Transnational Neuroscience", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subject has the ability to understand the purpose and risks of the study and provide signed and dated informed consent (or have the consent confirmed by a witness if unable to write) and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.\n* Subject was enrolled in either CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).\n* Subject has completed their last visit in Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).\n* Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment.\n\nExclusion Criteria:\n\n* Subject withdrew prematurely from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).\n* Subject permanently discontinued study treatment in Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) for any reason other than enrollment into this study.\n* Subject from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) has a significant change in medical history (including laboratory tests or a clinically significant condition) that in the opinion of the Investigator would impair the subject's medical fitness for participation and preclude treatment.\n* Female subject who is pregnant or breastfeeding.\n* Subject is currently enrolled in any investigational drug study other than Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).\n* Subject is taking pramipexole, other dopamine agonists, any other agent with dopaminergic activity, or any other disallowed concomitant medication.\n* Subject is unwilling or unable to comply with the requirements of the protocol including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to comply with the protocol. At a minimum, subjects who are not able to travel to the study site must be willing to agree to remote blood draws for clinical laboratory evaluations and telephone visits to report Adverse Events, concomitant medications, and Amyotrophic Lateral Sclerosis Functional Rating Scale (revised) (ALSFRS-R) scores.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Dexpramipexole", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04518540", "title": "Explore Neuroprotective Effect of Lipoic Acid in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Second Affiliated Hospital, Zhejiang University, School of Medicine", "summary": "In this proposed study, the investigators will evaluate the safety and efficacy of lipoic acid in treatment of Amyotrophic lateral sclerosis (ALS). The study will recruit 150 AD patients, and then these patients will be randomized to lipoic acid group or control group (75 patients per arm) for 6 courses for about 5 months. Clinical assessment will be done at screen/baseline, 3th course and 6th course. The specific aims are to compare lipoic acid versus control on: motor function and disease progression. During the study period, clinical effect index will be recorded, including bulbar function, motor function, respiratory function, and safety index including blood and urine routine, liver and kidney function, coagulation function.", "interventions": [{"type": "DRUG", "name": "lipoic acid group"}, {"type": "DRUG", "name": "control group"}], "start_date": "2020-09-01", "url": "https://clinicaltrials.gov/study/NCT04518540", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Second Affiliated Hospital,Zhejiang University School of Medicine", "city": "Hangzhou", "state": "Zhejiang", "country": "China", "status": "RECRUITING", "lat": 30.29365, "lon": 120.16142}], "contact_phone": "13646715353", "contact_email": "zhiyingwu@zju.edu.cn", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age range from 20 to 75 (including 20 and 75 years old), regardless of ethnic group or gender;\n2. The subjects should meet the diagnostic criteria for ALS by El Escorial revised criteria: \"Definite ALS\", \"Probable ALS\" and \"Probable, laboratory-supported ALS\".\n3. ALS Functional Rating Scale-Revised (ALSFRS-R 12 items) each item score \u22652 points;\n4. The onset (the symptoms of limbs weakness, muscle atrophy or bulbar involvement ) of the disease is less than 2 years\n5. Baseline breath function: Forced Vital Capacity\u226570% .\n6. Disease Progression Rate FS=(48- ALSFRS-R at \"time of diagnosis\")/duration from onset to diagnosis (month), progression rate FS\u22641;\n\nExclusion Criteria:\n\n1. Combined with one of cerebrovascular disease, spinal cord disease, spinal muscular atrophy, juvenile myoatrophy of distal upper extremity, multifocal motor neuropathy, Kennedy disease, epilepsy, etc;\n2. Severe renal insufficiency: creatinine clearance rate \\<30 mL/min (Cockcroft-Gault formula, urea nitrogen and (or) blood creatinine\\> 1.5 times the upper limit of normal, or other known severe renal insufficiency diseases;\n3. Severe liver damage: ALT, AST\\> 3 times the upper limit of normal, or other known liver diseases such as acute and chronic hepatitis, cirrhosis, etc.;\n4. Obvious tachycardia or bradycardia; patients with acute myocardial infarction or interventional therapy in the past 6 months (patients with grade III-IV according to NYHA classification);\n5. Combined with malignant tumor, blood, digestion or other serious diseases;\n6. Female patients during pregnancy and lactation;\n7. Participated in other clinical trials within 30 days before randomization, or are participating in other clinical trials;", "sex": "ALL", "min_age": "20 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "lipoic acid", "targeting_mechanism": "Antioxidant agent that reduces oxidative stress in neurodegenerative disease.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02118727", "title": "Therapy in Amyotrophic Lateral Sclerosis (TAME)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Kansas Medical Center", "summary": "The purpose of this study is to determine if memantine at up to 20 mg twice a day when used in conjunction with riluzole, can slow down the disease progression of patients with ALS including potentially improving their neuropsychiatric changes, as well as determine if serum biomarkers can be used both as a diagnostic and a prognostic marker in patients with ALS.\n\nFunding Source: FDA - Orphan Products Development (OPD)", "interventions": [{"type": "DRUG", "name": "Memantine"}, {"type": "DRUG", "name": "Placebo (for Memantine)"}], "start_date": "2018-11-07", "url": "https://clinicaltrials.gov/study/NCT02118727", "target_entities": ["NMDA receptor"], "locations": [{"facility": "Phoenix Neurological Associates", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "UC Irvine", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "University of Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Kansas School of Medicine - Wichita", "city": "Wichita", "state": "Kansas", "country": "United States", "status": "", "lat": 37.69224, "lon": -97.33754}, {"facility": "University of Kentucky", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "University of Missouri", "city": "Columbia", "state": "Missouri", "country": "United States", "status": "", "lat": 38.95171, "lon": -92.33407}, {"facility": "CoxHealth", "city": "Springfield", "state": "Missouri", "country": "United States", "status": "", "lat": 37.21533, "lon": -93.29824}, {"facility": "Providence Health Sciences", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Nerve & Muscle Center of Texas", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 18-85\n2. Male or Female\n3. Clinically definite, probable, probable lab-supported, or possible ALS by El Escorial criteria\n4. ALSFRS-R \\> 25\n5. Must be willing to undergo longitudinal blood draws for biomarker analysis\n6. Availability and willingness to complete the study\n7. Capable of providing informed consent and complying with trial procedures\n8. If patients are taking riluzole and/or Radicava, they must be a on a stable dose for at least thirty days prior to the baseline.\n\nExclusion Criteria:\n\n1. Patients with forced vital capacity (FVC) \u2264 60%\n2. History of liver disease\n3. Severe renal failure\n4. History of intolerance to memantine\n5. Onset of weakness for greater than 3 years\n6. Any other co-morbid condition which would make completion of the trial unlikely\n7. If female, pregnant or breast-feeding; or, if of childbearing age, an unwillingness to use birth control.\n8. Taking any investigational medications. If the patient was previously on investigational medications, a 30-day washout period is required before the baseline visit. Non-trial medications are not cause for exclusion.\n9. Unwillingness to provide consent\n\nRemote Inclusion Criteria:\n\n1. Age 18-85\n2. Male or Female\n3. Clinically definite, probable, probable lab-supported, or possible ALS by El Escorial criteria\n4. ALSFRS-R \\> 25\n5. Must be willing to undergo longitudinal blood draws for biomarker analysis. This may be foregone during the screening visit\n6. Availability and willingness to complete the study\n7. Capable of providing informed consent and complying with trial procedures\n8. If patients are taking riluzole and/or Radicava, they must be a on a stable dose for at least thirty days prior to the baseline\n9. Documentation of not clinically significant liver enzymes within the previous 6 months\n\nRemote Exclusion Criteria:\n\n1. Patients with FVC \u2264 60%\\*\n2. History of liver disease\n3. Severe renal failure\n4. History of intolerance to memantine\n5. Onset of weakness for greater than 3 years\n6. Any other co-morbid condition which would make completion of the trial unlikely\n7. If female, pregnant or breast-feeding; or, if of childbearing age, an unwillingness to use birth control.\n8. Taking any investigational medications. If the patient was previously on investigational medications, a 30-day washout period is required before the baseline visit. Non-trial medications are not cause for exclusion.\n9. Unwillingness to provide consent\n\n   * Since FVC cannot be captured during a remote screening visit, and acceptable FVC performed within the previous 90 days is acceptable. If an FVC is not available within the previous 90 days, the subject may be enrolled if the local site PI believes the subject has no significant shortness of breath or respiratory issues.", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Memantine", "targeting_mechanism": "NMDA receptor antagonist that provides neuroprotection.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03068754", "title": "Study of Acthar\u00ae Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mallinckrodt", "summary": "About 213 people with ALS will participate in this study. There will be locations in North and South America.\n\nDuring the first part, participants will be randomly assigned to a group (like by flipping a coin). Out of every 3:\n\n* 2 will get the study drug\n* 1 will get a look-alike with no drug in it (placebo)\n\nDuring the second part, everyone will get the study drug.\n\nParticipation will help doctors find out if Acthar can help or slow down the symptoms of ALS better than placebo.", "interventions": [{"type": "DRUG", "name": "Acthar"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2017-06-22", "url": "https://clinicaltrials.gov/study/NCT03068754", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Neuromuscular Research Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Mayo Clinic - Arizona", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "University of California San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Loma Linda University Health System, Department of Neurology", "city": "Loma Linda", "state": "California", "country": "United States", "status": "", "lat": 34.04835, "lon": -117.26115}, {"facility": "Keck School of Medicine, University of Southern California", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Los Angeles", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Irvine Medical Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of California San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Colorado Springs Neurological Associates", "city": "Colorado Springs", "state": "Colorado", "country": "United States", "status": "", "lat": 38.83388, "lon": -104.82136}, {"facility": "Georgetown University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "George Washington University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "University of Florida - McKnight Brain Institute", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Augusta University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Indiana University-Neuroscience Center of Excellence/Goodman Hall", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Kentucky Chandler Medical Center", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "John Hopkins Outpatient Center", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "University of Massachusetts Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Mercy Health- Saint Mary's", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "Neurology Associates", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "University of Nebraska Medical Center - Physicians Clinical Neurosciences Center", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "", "lat": 41.25626, "lon": -95.94043}, {"facility": "Las Vegas Clinic", "city": "Las Vegas", "state": "Nevada", "country": "United States", "status": "", "lat": 36.17497, "lon": -115.13722}, {"facility": "Jersey Shore University Medical Center", "city": "Neptune City", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.20011, "lon": -74.02792}, {"facility": "Columbia Presbyterian Hospital", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Health Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Temple University School of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Allegheny General Hospital", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Wesley Neurology Clinic", "city": "Cordova", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.15565, "lon": -89.7762}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Texas Neurology, P.A.", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "The Methodist Hospital", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Vermont Medical Center", "city": "Colchester", "state": "Vermont", "country": "United States", "status": "", "lat": 44.54394, "lon": -73.14791}, {"facility": "VCU Medical Center", "city": "Richmond", "state": "Virginia", "country": "United States", "status": "", "lat": 37.55376, "lon": -77.46026}, {"facility": "Swedish Neuroscience Institute", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Medical College of Wisconsin/Froedtert Hospital", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "IADIN", "city": "Ciudad Autonoma de Buenos Aires", "state": "Buenos Aires", "country": "Argentina", "status": "", "lat": null, "lon": null}, {"facility": "STAT Research", "city": "Ciudad Autonoma de Buenos Aires", "state": "Buenos Aires", "country": "Argentina", "status": "", "lat": null, "lon": null}, {"facility": "DIABAID", "city": "Ciudad Autonoma de Buenos Aires", "state": "Buenos Aires", "country": "Argentina", "status": "", "lat": null, "lon": null}, {"facility": "INEBA", "city": "Ciudad Autonoma de Buenos Aires", "state": "Buenos Aires", "country": "Argentina", "status": "", "lat": null, "lon": null}, {"facility": "Hospital Italiano de Buenos Aires", "city": "Ciudad Autonoma de Buenos Aires", "state": "Buenos Aires", "country": "Argentina", "status": "", "lat": null, "lon": null}, {"facility": "Hospital Espa\u00f1ol", "city": "Ciudad Autonoma de Buenos Aires", "state": "Buenos Aires", "country": "Argentina", "status": "", "lat": null, "lon": null}, {"facility": "Hospital Brit\u00e1nico de Buenos Aires", "city": "Ciudad Autonoma de Buenos Aires", "state": "Buenos Aires", "country": "Argentina", "status": "", "lat": null, "lon": null}, {"facility": "ILAIM", "city": "C\u00f3rdoba", "state": "C\u00f3rdoba Province", "country": "Argentina", "status": "", "lat": -31.40648, "lon": -64.18853}, {"facility": "Fundaci\u00f3n Scherbovsky", "city": "Mendoza", "state": "Mendoza Province", "country": "Argentina", "status": "", "lat": -32.88946, "lon": -68.84582}, {"facility": "Instituto de Neurolog\u00eda y Neurorrehabilitaci\u00f3n del Litoral (INNeL )", "city": "Santa Fe", "state": "Santa Fe Province", "country": "Argentina", "status": "", "lat": -31.64881, "lon": -60.70868}, {"facility": "Edmonton Kaye Clinic", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Recherche Sepmus inc", "city": "Greenfield Park", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.48649, "lon": -73.46223}, {"facility": "Centre de recherch\u00e9 du Centre Hospitalier de l'Universite de Montreal (CRCHUM)", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute & Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Centro de Trastornos del Movimiento (CETRAM)", "city": "Santiago", "state": "Santiago Metropolitan", "country": "Chile", "status": "", "lat": -33.45694, "lon": -70.64827}, {"facility": "Clinica D\u00e1vila", "city": "Santiago", "state": "Santiago Metropolitan", "country": "Chile", "status": "", "lat": -33.45694, "lon": -70.64827}, {"facility": "Biomedica Research Group AV Salvador 149, oficina 1101", "city": "Santiago", "state": "", "country": "Chile", "status": "", "lat": -33.45694, "lon": -70.64827}, {"facility": "Centro de Investigaciones Cl\u00ednicas SAS", "city": "Cali", "state": "", "country": "Colombia", "status": "", "lat": 3.43054, "lon": -76.5199}, {"facility": "Clinical Research Institute Saltillo S.A. de C.V.", "city": "Saltillo", "state": "Coahuila", "country": "Mexico", "status": "", "lat": 25.42595, "lon": -100.97963}, {"facility": "Hospital Universitario \"Dr. Jos\u00e9 Eleuterio Gonz\u00e1lez\"", "city": "Monterrey", "state": "Nuevo Le\u00f3n", "country": "Mexico", "status": "", "lat": 25.68435, "lon": -100.31721}, {"facility": "SMIQ BRCR Global M\u00e9xico", "city": "Quer\u00e9taro City", "state": "Quer\u00e9taro", "country": "Mexico", "status": "", "lat": 20.58806, "lon": -100.38806}, {"facility": "Clinical Research Institute S.C.", "city": "San Lucas Tepetlacalco", "state": "Tlalnepantla De Baz", "country": "Mexico", "status": "", "lat": 19.52136, "lon": -99.23257}, {"facility": "Centro Especializado en Investigaci\u00f3n Cl\u00ednica S.C.", "city": "Boca del R\u00edo", "state": "Veracruz", "country": "Mexico", "status": "", "lat": 17.71333, "lon": -94.71028}, {"facility": "Phylasis Clinicas Research", "city": "Mexico City", "state": "", "country": "Mexico", "status": "", "lat": 19.42847, "lon": -99.12766}, {"facility": "FAICIC Clinical Researc", "city": "Veracruz", "state": "", "country": "Mexico", "status": "", "lat": 19.18095, "lon": -96.1429}, {"facility": "Hospital Nivel IV Carlos Alberto Seguin Escobedo", "city": "Arequipa", "state": "", "country": "Peru", "status": "", "lat": -16.39899, "lon": -71.53747}, {"facility": "Hospital Nacional IV Alberto Sabogal Sologuren", "city": "Callao", "state": "", "country": "Peru", "status": "", "lat": -12.05162, "lon": -77.13452}, {"facility": "Hospital Almenara", "city": "Lima", "state": "", "country": "Peru", "status": "", "lat": -12.04318, "lon": -77.02824}, {"facility": "Instituto Neuro Cardiovascular de las Am\u00e9ricas", "city": "Lima", "state": "", "country": "Peru", "status": "", "lat": -12.04318, "lon": -77.02824}, {"facility": "Hospital Nacional Cayetano Heredia", "city": "Lima", "state": "", "country": "Peru", "status": "", "lat": -12.04318, "lon": -77.02824}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Is 18-75 years of age at Screening\n2. Has ALS symptom onset within 2 years prior to Screening\n3. Has forced vital capacity (FVC) no higher than 60% at screening\n4. If taking riluzole, is on a stable dose for 4 weeks before Screening\n\nExclusion Criteria:\n\n1. Has tracheostomy, diaphragm pacing, or an ongoing need for assisted ventilation of any type\n2. Has used any medication within a time period not allowed per protocol\n3. Has history of Type 1 or Type 2 diabetes mellitus, or any clinically significant infection\n4. Used edaravone less than 1 week before Screening\n5. Received any stem cell replacement therapy\n6. Used steroids within a time period not allowed per protocol", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Acthar (corticotropin)", "targeting_mechanism": "Immunomodulatory agent that modulates neuroinflammation in ALS.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03787420", "title": "Development and Needs Assessment and Efficiency of Smart Communication System for Patients With ALS.", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Taipei Veterans General Hospital, Taiwan", "summary": "This project aims to develop a smart communication system for patients with amyotrophic lateral sclerosis (ALS), especially for stage 3 and stage 4 (late stage). Patients with ALS will be able to communicate with outer environment by means of mental control or eye tracking control, which would increase their life quality. This integrated research project includes experts from different domains and proposes a solution for smart communication system.", "interventions": [{"type": "OTHER", "name": "communication system"}], "start_date": "2018-09-01", "url": "https://clinicaltrials.gov/study/NCT03787420", "target_entities": [], "locations": [{"facility": "Cancer Center, Taipei Veterans General Hospital", "city": "Taipei", "state": "", "country": "Taiwan", "status": "RECRUITING", "lat": 25.05306, "lon": 121.52639}], "contact_phone": "+8862-28757296", "contact_email": "cl_chou@vghtpe.gov.tw", "eligibility": {"criteria": "Part one:\n\nInclusion Criteria:\n\n* ALS diagnostic criteria (Brooks et al., 2000).Must have the following characteristics:\n\n  1. Degeneration of motor neurons (LMN) by clinical, electrophysiological or neuropathological evidence.\n  2. Degeneration of upper motor neurons (UMN) is demonstrated by clinical examination.\n  3. According to the medical history or examination, the symptoms or signs gradually disappear in a certain part.\n\nAlso, there are no features:\n\n1. Electrophysiological or pathological evidence of other diseases that may explain signs of LMN and/or UMN degradation\n2. Neuroimaging evidence of other diseases that may explain the observed clinical electrophysiological signs.\n\n   * Use Mandarin as the main language.\n   * Age limits minimum is 20\n\nExclusion Criteria:\n\n* Concomitant other central nervous disorders that interfere with their cognitive function, such as dementia, severe depression, and psychosis.\n* The questionnaire cannot be completed without the assistance of others.\n\nPart two:\n\nInclusion Criteria:\n\n* ALS diagnostic criteria (Brooks et al., 2000).\n* Use Mandarin as the main language.\n* Age limits minimum is 20\n* In everyday oral communication, people who are consciously difficult.\n* Ability to perform equipment wear and system operators.\n\nExclusion Criteria:\n\n* Concomitant other central nervous disorders that interfere with their cognitive function, such as dementia, severe depression, and psychosis.\n* After correction, the visual acuity cannot see the communication board.\n* The scalp is sensitive or there is deep brain stimulation, etc. It is not applicable to brain wave measurement.\n* -The questionnaire cannot be completed without the assistance of others.", "sex": "ALL", "min_age": "20 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01281189", "title": "Phase 3 Study of Dexpramipexole in ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "The purpose of this study is to determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Dexpramipexole"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2011-03", "url": "https://clinicaltrials.gov/study/NCT01281189", "target_entities": ["mitochondrial_function"], "locations": [{"facility": "Barrow Neurological Institute - St. Joseph's Hospital", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of Arkansas for Medical Sciences", "city": "Little Rock", "state": "Arkansas", "country": "United States", "status": "", "lat": 34.74648, "lon": -92.28959}, {"facility": "University of California at San Francisco - Fresno", "city": "Fresno", "state": "California", "country": "United States", "status": "", "lat": 36.74773, "lon": -119.77237}, {"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "University of California, Davis", "city": "Sacramento", "state": "California", "country": "United States", "status": "", "lat": 38.58157, "lon": -121.4944}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "Mayo Clinic - Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami Miller School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of South Florida Medical Center", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Indiana University", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "St. Mary's Health Care", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Mayo Clinic - Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates, P.C.", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "University of Nevada School of Medicine", "city": "Las Vegas", "state": "Nevada", "country": "United States", "status": "", "lat": 36.17497, "lon": -115.13722}, {"facility": "Dartmouth-Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Research Foundation of the State University of New York", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest University", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "ALS Center at Penn", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Drexel University College of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburgh Medical Center", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Vanderbilt University Medical Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Texas Health Sciences Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Prince of Wales Hospital", "city": "Randwick", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.91439, "lon": 151.24895}, {"facility": "Westmead Hospital", "city": "Westmead", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.80383, "lon": 150.98768}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Calvary Health Care Bethlehem", "city": "Melbourne", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.814, "lon": 144.96332}, {"facility": "AZ St-Lucas", "city": "Ghent", "state": "", "country": "Belgium", "status": "", "lat": 51.05, "lon": 3.71667}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Univ of Calgary / Foothills MC", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "CHUM - Hopital Notre Dame", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Mcgill University", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "London Health Sciences Centre", "city": "London", "state": "", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook and Women's College and Health Sciences Centre", "city": "Toronto", "state": "", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "University of British Columbia", "city": "Vancouver", "state": "", "country": "Canada", "status": "", "lat": 49.24966, "lon": -123.11934}, {"facility": "CHRU de Lille - H\u00f4pital Roger Salengro", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU de Limoges - H\u00f4pital Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Centre Hospitalier La Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHU Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nice - H\u00f4pital de l'Archet 1", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "H\u00f4pital La Piti\u00e9 Salp\u00e9tri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Charit\u00e9 - Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Bergmannsheil Gmbh", "city": "Bochum", "state": "", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Medizinische Hochschule Hannover (MHH)", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4tsklinikum Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "University of Ulm, RKU", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Academisch Medisch Centrum", "city": "Amsterdam", "state": "", "country": "Netherlands", "status": "", "lat": 52.37403, "lon": 4.88969}, {"facility": "UMC St. Radboud", "city": "Nijmegen", "state": "", "country": "Netherlands", "status": "", "lat": 51.8425, "lon": 5.85278}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Universitario de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Vall d'Hebron", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital La Paz", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Carlos III", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Sahlgrenska Universitetssjukhuset", "city": "Gothenburg", "state": "", "country": "Sweden", "status": "", "lat": 57.70716, "lon": 11.96679}, {"facility": "Karolinska Universitetssjukhuset, Solna", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Queen Elizabeth Hospital", "city": "Birmingham", "state": "", "country": "United Kingdom", "status": "", "lat": 52.48142, "lon": -1.89983}, {"facility": "Walton Centre for Neurology & Neurosurgery", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "Kings College Hospital NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Newcastle University Hospital - Clinical Ageing Research Unit", "city": "Newcastle", "state": "", "country": "United Kingdom", "status": "", "lat": 54.21804, "lon": -5.88979}, {"facility": "John Radcliffe Hospital", "city": "Oxford", "state": "", "country": "United Kingdom", "status": "", "lat": 51.75222, "lon": -1.25596}, {"facility": "Sheffield Institute for Transnational Neuroscience", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Aged 18 to 80 years old, inclusive, on Day 1.\n* Diagnosis of sporadic or familial ALS.\n* Onset of first ALS symptoms within 24 months prior to Day 1.\n* World Federation of Neurology El Escorial criteria are met for a possible, laboratory-supported probable, probable, or definite ALS diagnosis.\n* Upright slow vital capacity (SVC) of 65% or more at screening.\n* Patients taking or not taking Riluzole are eligible for this study: if a patient has never taken Riluzole, he or she is eligible; if a patient is currently taking Riluzole, he or she must have been on a stable dose for at least 60 days; if a patient has discontinued Riluzole, he or she must have stopped taking it for at least 30 days.\n* Must be able to swallow tablets at the time of study entry.\n\nExclusion Criteria:\n\n* Other medically significant illness.\n* Clinically significant abnormal laboratory values.\n* Pregnant women or women breastfeeding.\n* Prior exposure to dexpramipexole.\n* Currently taking pramipexole or other dopamine agonists.\n\nOther protocol-defined inclusion/exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Dexpramipexole", "targeting_mechanism": "Mitochondrial dysfunction modulator with neuroprotective properties.", "targeting_mechanism_pmid": "35805131", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07533903", "title": "Functional Outcomes and Control Using Synchron BCI - Australia", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Synchron, Inc.", "summary": "Functional Outcomes and Control Using Synchron BCI - Australia (FOCUS-AUS)", "interventions": [{"type": "DEVICE", "name": "Stentrode"}], "start_date": "2026-05-22", "url": "https://clinicaltrials.gov/study/NCT07533903", "target_entities": [], "locations": [{"facility": "The Royal Melbourne Hospital", "city": "Melbourne", "state": "", "country": "Australia", "status": "RECRUITING", "lat": -37.814, "lon": 144.96332}], "contact_phone": "323-796-2476", "contact_email": "clinical@synchron.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to provide informed consent to participate in the study.\n2. Bilateral upper-limb paresis or Amyotrophic lateral sclerosis (ALS) with bilateral upper limb paresis\n3. The underlying condition causing motor impairment must be refractory to treatment and have been present for a minimum of twelve months.\n4. Aged 21 years or older\n5. Life expectancy greater than 12 months post-implantation\n6. Preserved precentral gyrus assessed using CT\n7. Suitable vascular anatomy assessed using CT venography\n8. Suitable anatomy for subcutaneous pocket creation\n9. Able to undergo anesthesia\n10. Willing and able to comply with investigational requirements, including clinical testing visits and training visits in the home.\n11. Caregiver(s) willing and able to facilitate study visits, including visits at the study site and in the home, and BCI use outside of study visits (e.g. device charging)\n12. Patient and caregiver fluent in English\n13. Suitable home environment for BCI training, including an internet connection\n\nExclusion Criteria:\n\n1. Unrealistic expectations regarding the potential benefits of the device.\n2. Active infection or unexplained fever in the 48 hours prior to informed consent\n3. Major psychiatric disorder that may adversely impact the participant's safety or study compliance, including severe depression, psychotic features, personality disorder, severe emotional lability, or substance abuse.\n4. Dementia or cognitive dysfunction that would impact the participant's ability to participate in study activities.\n5. Active implanted device (e.g., deep brain stimulator, cardiac defibrillator, pacemaker, vagal nerve stimulator, spinal cord stimulator, diaphragmatic pacer, etc.).\n6. Known allergy to patient-contacting materials included in the implanted device\n7. Contraindication to angiographic imaging or iodine contrast media.\n8. History of central venous sinus thrombosis.\n9. Recent history of new venous thromboembolic event (in the 6 months prior to implant) or recurrent history of venous thromboembolic disease\n10. Contraindication to antithrombotic therapy.\n11. Participant is at substantially increased risk of infection, including immunocompromised status, recurrent infection, or poorly controlled diabetes mellitus.\n12. Significant risk of non-healing of the subcutaneous pocket incision, including history of chronic non-healing surgical wounds or poorly controlled diabetes mellitus.\n13. Pregnant or breast feeding.\n14. Patients who are currently enrolled in any other clinical trial that would confound interpretation of safety or effectiveness data or may interfere with the ability to meet study requirements.\n15. Any other disease or disorder that could significantly affect participation in the study. Examples may include corrected vision insufficient for viewing computer screens or hearing insufficient for following verbal instructions, which might impact the participant's ability to participate in BCI training and testing.", "sex": "ALL", "min_age": "21 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Stentrode", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00912041", "title": "BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Leigh R. Hochberg, MD, PhD.", "summary": "The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tetraplegia to control a computer cursor and other assistive devices with their thoughts.", "interventions": [{"type": "DEVICE", "name": "Placement of the BrainGate2 sensor(s) into the motor-related cortex"}], "start_date": "2009-05", "url": "https://clinicaltrials.gov/study/NCT00912041", "target_entities": [], "locations": [{"facility": "University of California, Davis", "city": "Sacramento", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 38.58157, "lon": -121.4944}, {"facility": "Stanford University School of Medicine", "city": "Stanford", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.42411, "lon": -122.16608}, {"facility": "Emory University School of Medicine", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "RECRUITING", "lat": 33.749, "lon": -84.38798}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "Providence VA Medical Center", "city": "Providence", "state": "Rhode Island", "country": "United States", "status": "RECRUITING", "lat": 41.82399, "lon": -71.41283}, {"facility": "Baylor College of Medicine", "city": "Houston", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "617-724-9247", "contact_email": "clinicaltrials@braingate.org", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinical diagnosis of spinal cord injury, brainstem stroke, muscular dystrophy, amyotrophic lateral sclerosis or other motor neuron disorders\n* Complete or incomplete tetraplegia (quadriplegia)\n* Must live within a three-hour drive of the Study site\n* (There are additional inclusion criteria)\n\nExclusion Criteria:\n\n* Visual impairment such that extended viewing of a computer monitor would be difficult even with ordinary corrective lenses\n* Chronic oral or intravenous steroids or immunosuppressive therapy\n* Other serious disease or disorder that could seriously affect ability to participate in the study\n* (There are additional exclusion criteria)", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "BrainGate2 sensor", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00563537", "title": "Molecular Imaging Modality by Positron Emission Tomography Using 18F-X : Study of Microglial Activation in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Tours", "summary": "PET imaging of activated microglia offers a tool of investigation of a range of brain diseases where neuroinflammation is a component.\n\nAmyotrophic lateral sclerosis is the most frequent motoneuronal disease in adult.\n\nThis study was designed to explore the feasibility of molecular imaging modality by Positron Emission Tomography using 18F-X as an in vivo marker of activated microglia for the assessment of neuroinflammation in amyotrophic lateral sclerosis.\n\nPET may help in the diagnosis of the disease and, further, may allow assessment of the efficacy of antiinflammatory treatment.", "interventions": [{"type": "RADIATION", "name": "18F-X PET SCAN"}], "start_date": "2007-01", "url": "https://clinicaltrials.gov/study/NCT00563537", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Service de M\u00e9decine Nucl\u00e9aire et Ultrasons - H\u00f4pital Bretonneau", "city": "Tours", "state": "Centre-Val de Loire", "country": "France", "status": "RECRUITING", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "(33) 2.47.47. 97.89", "contact_email": "roussel@med.univ-tours.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* suffering from probable or definite form of amyotrophic lateral sclerosis according to El Escorial criteria. Spinal or bulbar site of the disease.\n* Information and signature of the written consent form\n* French Social Security registration\n\nExclusion Criteria:\n\n* family history of ALS\n* Riluzole treatment before the first PETscan.\n* Psychiatric disorders\n* Evolution of the disease older than 18 months\n* Antiinflammatory or antibiotic treatment in the last month", "sex": "ALL", "min_age": "40 Years", "max_age": "70 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "18F-X PET imaging agent", "targeting_mechanism": "PET imaging of activated microglia as an in vivo marker of microglial activation and neuroinflammation", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07357428", "title": "Connect-One: Early Feasibility Study of Connexus\u00ae Brain-Computer Interface (BCI)", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Paradromics", "summary": "The Connect-One Study is an early feasibility study to obtain preliminary device safety information for the Connexus Brain-Computer Interface (BCI). The Connexus BCI is intended to be used as: (1) an assistive communication device to decode imagined language correlates and speech for patients with impaired communication as a result of severe loss of voluntary motor control; and (2) to provide control of computer devices for individuals with severe loss of voluntary motor control of the upper extremity.", "interventions": [{"type": "DEVICE", "name": "Connexus Brain-Computer Interface"}], "start_date": "2026-03-31", "url": "https://clinicaltrials.gov/study/NCT07357428", "target_entities": [], "locations": [{"facility": "University of California, Davis", "city": "Sacramento", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 38.58157, "lon": -121.4944}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "RECRUITING", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "(512) 559 4120", "contact_email": "clinical-team@paradromics.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Clinical diagnosis of a progressive neuromuscular disease or a neurological injury.\n* Clinical diagnosis of anarthria or severe dysarthria.\n* Wheelchair dependent with severely impaired upper limb function.\n* Has a reliable method of communication and the ability to read and understand the English language.\n* Has a study care partner (e.g. caregiver or multiple caregivers) for the duration of the study.\n* Lives within a 4-hour radius of a study site.\n\nExclusion Criteria:\n\n* Cognitive impairment or psychiatric illness that could impact the ability to comply with study requirements, as determined by the Study Investigator.\n* Co-morbidities or an ongoing chronic medical condition that would impair the ability to comply with study requirements.\n* The presence of another implanted device, like a pacemaker, deep brain stimulator, or implantable pulse generator.\n* Requires, or is expected to require regular MRI scans for on-going medical conditions.\n* In the opinion of the Study Investigator, the patient is not an appropriate candidate for the study, for reasons that could place the patient at undue risk or otherwise result in non-compliance with the study requirements.", "sex": "ALL", "min_age": "22 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Connexus Brain-Computer Interface", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00125203", "title": "Study of Myobloc in the Treatment of Sialorrhea (Drooling) in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The University of Texas Health Science Center at San Antonio", "summary": "The purpose of this study is to determine the safety and efficacy of Myobloc in ALS patients who are having excessive drooling.\n\nThe primary goal of the study is to determine if the patient perceives a benefit from the Myobloc in controlling excessive drooling.", "interventions": [{"type": "DRUG", "name": "Botulinum toxin type B (Myobloc)"}, {"type": "PROCEDURE", "name": "Injection of salivary glands"}], "start_date": "2003-07", "url": "https://clinicaltrials.gov/study/NCT00125203", "target_entities": ["acetylcholine_release"], "locations": [{"facility": "University of Kansas Medical Center/Neurology, 1008 Wescoe", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Carolinas MDA Neuromuscular/ALS Center; Carolinas Medical System; 1540 Garden Terrace", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of probable or definite ALS based on current World Federation of Neurology criteria\n* Between the ages of 21-85, inclusive\n* Sialorrhea refractory to treatment with at least two anticholinergic medications OR has been intolerant of anticholinergic medications due to side effects\n* Capable of giving informed consent\n* Must be able to attend all study visits\n\nExclusion Criteria:\n\n* Patient has any uncontrolled significant medical, psychiatric or neurological disease (other than ALS) over the past 30 days\n* History of ongoing substance abuse\n* History of non-compliance with treatment in other experimental protocols\n* Cannot provide informed consent or comply with evaluation procedures\n* Has received any form of botulinum toxin in the past for any indication\n* Women who are pregnant, lactating, or of child bearing potential not using an adequate form of birth control\n* Currently being treated with coumadin\n* Forced vital capacity (FVC) \\<40% of predicted unless the tidal volume is \\> 600cc, as patients with significant bulbar weakness may show a falsely low FVC due to bulbar muscle spasticity", "sex": "ALL", "min_age": "21 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Botulinum toxin type B (Myobloc)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01641965", "title": "Impact of Early Non Invasive Ventilation in Amyotrophic Lateral Sclerosis (ALS) Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hospital Universitari de Bellvitge", "summary": "Timing of initiating domiciliary no invasive ventilation (NIV) in amyotrophic lateral sclerosis patients remains unclear. The hypothesis of the study is that the early use of NIV, in the initial phase of respiratory muscle weakness, improves the prognosis of ALS patients.\n\nPrincipal objective: To evaluate the impact of early NIV in the survival of ALS patients.\n\nSecondary objectives:To determine the effects from early NIV in the progression of respiratory muscle weakness. To analyze the impact of early NIV in the quality of life of ALS patients. To evaluate the correlation between the FVC and other parameters of respiratory evaluation (maximal inspiratory pressure (MIP), sniff nasal inspiratory pressure (SNP), nocturnal desaturation) and their utility in the early indication of the NIV. To evaluate the tolerance to the early NIV.\n\nMethods: multicentric, randomized, open-label, controlled clinical trial with a parallel treatment design. Patients will be included when their FVC reaches the threshold of the 75% of the predicted value and will be randomized in: Group A: the NIV treatment will begin immediately and Group B: the NIV treatment will be started when patients fulfil at least one of the following criteria: (i) FVC \\< 50% predicted, (ii) orthopnea, and/or (iii) PaCO2 \\> 45 mmHg. Follow-up visits will be done every three months with pulmonary function test, nocturnal pulse oximetry, quality of life and quality of sleep tests, assessment of disease progression (ALSFSR-R scale), tolerance and compliance with NIV.", "interventions": [{"type": "DEVICE", "name": "Home pressure ventilator model Vivo 40 (BREAS Medical AB)"}], "start_date": "2012-04", "url": "https://clinicaltrials.gov/study/NCT01641965", "target_entities": [], "locations": [{"facility": "Hospital Universitari Bellvitge", "city": "L'Hospitalet de Llobregat", "state": "Barcelona", "country": "Spain", "status": "", "lat": 41.35967, "lon": 2.10028}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Participant is willing and able to give informed consent for participation in the study\n* Definite ALS diagnosis according to El Escorial Criteria\n* Ability to understand and perform the pulmonary function test\n* FVC \u2264 75% (with FVC registry \\>75% documented within the six previous months)\n\nExclusion Criteria:\n\n* Major comorbidity (non-related with ALS) that can shorten life expectancy\n* Cognitive impairment that prevents the patient to understand and perform the study procedures including technically acceptable pulmonary function tests (FVC, MIP, SNP, PCF)\n* Patient refusal of NIV treatment\n* Previous respiratory or cardiac diseases with known impaired spirometry\n* Indication of NIV according to standard criteria (PaCO2 \\> 45 mmHg, FVC \\< 50%, orthopnea)\n* ALS with slow disease progression (more than 3 years)\n* Participation in another clinical trial", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01160367", "title": "Trial of Ascertaining Individual Preferences for Loved One's Role in End-of-Life", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "Specific Aims and Hypotheses:\n\nAim 1: To test the effect of the \"Trial of Ascertaining Individual preferences for Loved Ones' Role in End-of-life Decisions\" (TAILORED) Intervention on family decision-making self-efficacy at 8 weeks both with respect to the patient's present situation and in a hypothetical situation in which the patient lacks decision-making capacity.\n\nHypotheses 1a: Family decision-making self-efficacy will be greater at 8 weeks in pairs that have undergone the TAILORED Intervention than in pairs receiving the standard information on advance directives in the patient's present situation.\n\nHypotheses 1b: Family decision-making self-efficacy will be greater at 8 weeks in pairs that have undergone the TAILORED Intervention than in pairs receiving the standard information on advance directives in the hypothetical situation in which the patient lacks decision making capacity.\n\nAim 2: To test the effect of the TAILORED Intervention on family psychological outcomes (depression, caregiver burden, decision making distress).\n\nHypotheses 2a: Depression will be less at 8 weeks in family members who have undergone the TAILORED Intervention than in family members who have received the standard information on advance directives.\n\nHypotheses 2b: Caregiver burden will be less at 8 weeks in family members who have undergone the TAILORED Intervention than in family members who have received the standard information on advance directives.\n\nHypotheses 2c: Decision-making distress will be less at 8 weeks in family members who have undergone the TAILORED Intervention than in family members who have received the standard information on advance directives.\n\nAim 3: To test the effect of the TAILORED Intervention on patient and family satisfaction with family decision-making involvement.\n\nHypothesis 3a: Patient satisfaction with family decision involvement will be greater at 8 weeks in patients who have undergone the TAILORED Intervention than in patients receiving the standard information on advance directives.\n\nHypothesis 3b: Family member satisfaction with decision involvement will be greater at 8 weeks in family members who have undergone the TAILORED Intervention than in family members receiving the standard information on advance directives.\n\nAim 4: To explore family decision-making self-efficacy and perceptions of the TAILORED Intervention.", "interventions": [{"type": "OTHER", "name": "TAILORED Patient Family Decision Making"}, {"type": "OTHER", "name": "standard of care"}], "start_date": "2010-08-26", "url": "https://clinicaltrials.gov/study/NCT01160367", "target_entities": [], "locations": [{"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nPatient Inclusion Criteria\n\n1. Age 18 or older\n2. Speaks and reads English\n3. Diagnosed by a specialist at either Johns Hopkins Medical Institutions or University of Chicago Medical Center as having ALS or stage III or IV GI or pancreatic cancer.\n4. Accompanied to clinic by a family member who may participate in the patient's health care decisions and who patient gives permission to approach for participation in study.\n5. Lives within a 2-hour drive of The Johns Hopkins Medical Institutions or The University of Chicago Medical Center\n\n5.G.2. Family Inclusion Criteria\n\n1. Age 18 or older\n2. Speaks and reads English\n3. Identified by the patient-subject as a family member whom the patient may involve in health care decision making in the present and/or should the patient become too ill to make health care decisions.\n4. Person who the patient-subject has granted investigators permission to approach for participation in this study.\n\nExclusion Criteria:\n\n* Patient Exclusion Criteria\n\n  1. Severe visual impairment that would limit ability to visualize instrument illustrations\n  2. Cognitive impairment indicated by a Short Portable Mental Status Questionnaire adjusted error score of \\>5\n  3. Has no family member who might assist in decision making or family member declines to participate.\n  4. Is not accompanied to the clinic by family member.\n  5. G.4. Family Exclusion Criteria\n\n  <!-- -->\n\n  1. Declines to participate.\n  2. Severe visual impairment that would limit ability to visualize instrument illustrations.\n  3. Cognitive impairment indicated by a Short Portable Mental Status Questionnaire adjusted error score of \\>5", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04788745", "title": "Targeting Metabolic Flexibility in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The University of Queensland", "summary": "MetFlex is an investigator led, open-label, single-arm, Phase 2a trial to determine the safety and tolerability of trimetazidine for the treatment of amyotrophic lateral sclerosis/motor neuron disease (ALS/MND).", "interventions": [{"type": "DRUG", "name": "Trimetazidine Dihydrochloride"}], "start_date": "2021-06-29", "url": "https://clinicaltrials.gov/study/NCT04788745", "target_entities": ["metabolic_flexibility"], "locations": [{"facility": "Royal Brisbane & Women's Hospital", "city": "Brisbane", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.46794, "lon": 153.02809}, {"facility": "University Medical Centre Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "King's College London", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age between 18 and 75 years\n* Signed informed consent prior to the initiation of any study-specific procedures\n* Familial or sporadic ALS/MND, defined as clinically possible, probable, or definite as per the El Escorial criteria\n* Relative TRICALS risk score between -6.0 to -2.0 (75% of patients with ALS/MND)\n* Metabolic index \u2265110%, at the screening visit.\n* The use of riluzole will be permitted during the study. Individuals taking riluzole must be on a stable dose for at least 30 days prior to the baseline visit, or stopped taking riluzole at least 30 days prior to the baseline visit.\n* Ability to swallow tablets\n* Able to lie with torso elevated at a 35\u00b0 angle for 30 minutes without respiratory support\n* Able to give informed consent (as judged by the investigator) and able to comply with all study visits and all study procedures\n* Females must not be able to become pregnant (e.g. post-menopausal, surgically sterile or using highly effective birth control methods) for the duration of the study. Highly effective methods of birth control are those with a failure rate of \\< 1% per year when employed consistently and correctly, e.g. Combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:\n\n  * oral\n  * intravaginal\n  * transdermal\n  * Progestogen-only hormonal contraception associated with inhibition of ovulation:\n  * oral\n  * injectable\n  * implantable\n  * intrauterine device (IUD)\n  * intrauterine hormone-releasing system ( IUS)\n  * vasectomised partner\n* Females of child-bearing potential must have a negative serum pregnancy test at screening and baseline and be non-lactating\n\nExclusion Criteria:\n\n* Unable to provide informed consent\n* History of, or current diagnosis of diabetes or medical condition that impacts whole body energy expenditure (e.g. Hashimoto's, heart disease)\n* Parkinson's disease or parkinsonism, tremor, restless-leg syndrome\n* Safety Laboratory Criteria at screening related to significant kidney disease:\n\n  * Creatinine clearance \\< 50 mL / min (Cockcroft-Gault) based on Cystatin C\n* Tracheostomy or non-invasive ventilation (NIV) use \\> 22 hours per day\n* Inability to swallow tablets\n* Contraindication therapy:\n\n  * Allergy for one of the product's active pharmaceutical ingredients (APIs) or excipients.\n  * Antihypertensive treatment \\[Trimetazidine may cause hypotension\\]\n* Evidence of malignant disease\n* Significant neuromuscular disease other than ALS/MND\n* Ongoing disease that may cause neuropathy\n* Pregnancy or breastfeeding\n* Females actively seeking to become pregnant who are not using an adequate form of contraceptive as detailed in the Inclusion criteria.\n* Deprivation of freedom by administrative or court order", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Trimetazidine Dihydrochloride", "targeting_mechanism": "Trimetazidine improves metabolic flexibility by modulating energy substrate utilization in motor neurons.", "targeting_mechanism_pmid": "34783031", "animal_results": "Trimetazidine treatment extended survival and delayed paralysis onset in SOD1(G93A) ALS mice.", "animal_results_pmid": "34783031", "repurposed_from": "cardiac ischemia", "repurposed_from_pmid": "34783031"}}
{"nct_id": "NCT02479802", "title": "Efficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Instituto Grifols, S.A.", "summary": "Pilot, phase II, prospective, open-label, uncontrolled study of plasma exchange with 5% albumin in 10 subjects having a definite, possible, or probable diagnosis of Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "BIOLOGICAL", "name": "Albumin"}], "start_date": "2014-11", "url": "https://clinicaltrials.gov/study/NCT02479802", "target_entities": [], "locations": [{"facility": "Hospital Universitari Bellvitge", "city": "L'Hospitalet de Llobregat", "state": "Barcelona", "country": "Spain", "status": "", "lat": 41.35967, "lon": 2.10028}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Signed written-informed consent.\n* Subjects over 18 years of age, and less than 70 years old.\n* Subjects with a definite, possible, or probable diagnosis of ALS, according to the revised El Escorial criteria.\n* Subjects having experienced their first ALS symptoms within 18 months before recruitment/consent.\n* FVC \\> 70%\n* Subjects must be medically suitable for study participation and of complying with all planned aspects of the protocol including blood sampling at the time of inclusion in the study.\n\nExclusion Criteria:\n\n* Subjects with a clinically significant preexisting lung disease not attributable to ALS.\n* Subjects with a diagnosis of other neurodegenerative diseases or diseases associated with dysfunction of the motor neurons that can confuse the diagnosis of ALS.\n* Participation in other clinical trials, or the reception of any other investigational drug in the six months prior to the start of the study.\n* Female subjects who are pregnant, currently breastfeeding, or attempting to conceive during the study.\n* Difficult peripheral venous access precluding plasma exchange and inability to implement a viable alternative catheter to make continued performing plasma exchange visits according to protocol\n* Any contraindication for plasma exchange or abnormal coagulation parameters according clinical criteria from apheresis team\n* A history of frequent adverse reactions (serious or otherwise) to blood products.\n* Hypersensitivity to albumin or allergies to any of the components of Albutein.\n* Subjects that can not interrupt treatment with acetylsalicylic acid or oral anticoagulants\n* Plasma creatinine \\> 2mg/dl.\n* Present a history of heart disease including ischemic heart disease or congestive heart failure.\n* Presence of prior conduct disorders requiring pharmacologic intervention, with less than 3 months of stable treatment\n* Any condition that complicates adherence to study protocol (illness with less than one year of expected survival, drug or alcohol abuse, etc.)", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Albumin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05889572", "title": "Safety and Gut Microbiota Analysis of an Oral Microbiotherapy in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "MaaT Pharma", "summary": "The purpose of this pilot study is to assess the safety and tolerability of multiple doses of MaaT033 in ALS patients and to analyze the gut microbiota composition and evolution before considering a larger randomized controlled efficacy study.", "interventions": [{"type": "DRUG", "name": "MaaT033"}], "start_date": "2023-06-08", "url": "https://clinicaltrials.gov/study/NCT05889572", "target_entities": ["gut_microbiota"], "locations": [{"facility": "Centre Hospitalier Universitaire de Lille - CIC", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "H\u00f4pital de la Piti\u00e9-Salp\u00eatri\u00e8re - CIC Neuroscience", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Male or female subjects aged between18 and 80 years\n* ALS meeting the revised El Escorial criteria for possible, probable, laboratory-supported probable, or definite ALS (familial or sporadic)\n* Time since first motor deficit at screening: at least 6 months, up to 24 months\n* Slope of progression of ALS Functional Rating Scale - revised (ALSFRS-R) from date of symptom onset to date of screening test (\u0394FS/number of months) between \\[0.4 and 1.1\\]\n* Able to swallow study treatments (including capsules without opening or chewing them) as per the investigator's assessment\n* SVC (Slow Vital Capacity) equal to or greater than 70% of the predicted normal value for sex, height, and age at the screening visit\n* If taking riluzole, subject must be on a stable dose for \u226530 days\n* Signature of written informed consent by subject\n\nExclusion Criteria:\n\n* Subjects with a non-invasive ventilation, a tracheotomy and /or a gastrostomy\n* Known autoimmune diseases, inflammatory disorders (SLE, Rheumatoid arthritis, connective tissue disorder) or chronic infections (HIV, hepatitis B, or C infection, Tuberculosis)\n* Known hypersensitivity to rifaximin or macrogol or any of its components\n* Known allergy or intolerance to trehalose, maltodextrin or Polyethylene Glycol (PEG)\n* Documented hepatic impairment (Alanine Transaminase/ Aspartate Transaminase \\> 5N)\n* Subject with white blood cells \\< 4000/ mm3; Polynuclear neutrophils \\< 1.5 G/ L\n* Active infection requiring systemic antimicrobial therapy within 2-week prior to screening visit\n* Active infection requiring systemic antimicrobial therapy between screening and baseline\n* Medical condition requiring proton pump inhibitors (PPIs)\n* Gastrointestinal obstruction or perforation\n* Any gastro-intestinal bleeding in the past 3 months\n* Gastric emptying disorders (gastroparesis)\n* Toxic megacolon\n* Severe forms of inflammation of the intestinal tract, including Crohn's disease and ulcerative colitis\n* Severe vital organ dysfunctions unrelated to ALS and not compatible with experimental treatment, as per the investigator's assessment\n* Subjects with negative IgG serology for Epstein Barr virus (EBV)\n* Women of childbearing potential1 without effective contraceptive protection\n* Nursing or pregnant women\n* Any condition that, in the opinion of the investigator, may interfere with full participation in the study, including administration of study drug (and its preparation procedure) and attendance at required study visits; represent a significant risk to the subject; or interfere with the interpretation of study data\n* Enrollment in another trial or expanded access program that may interfere with this study\n* Guardianship/legal protection/curatorship of subjects\n* Vulnerable subjects such as: persons deprived of liberty, persons in intensive care units unable to provide informed consent prior to the procedure", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MaaT033", "targeting_mechanism": "MaaT033 is an oral microbiotherapy that modulates gut microbiota composition to reduce dysbiosis and intestinal inflammation associated with ALS.", "targeting_mechanism_pmid": "28947596", "animal_results": "Dysbiosis and increased intestinal permeability observed in SOD1(G93A) ALS mouse model; restoration of healthy gut microbiota may reduce pro-inflammatory mediators affecting gut-brain communication.", "animal_results_pmid": "28947596", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00330681", "title": "Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "The primary objective of this study is to confirm the efficacy of 60 mg of MCI-186 via intravenous drip once a day in patients with ALS based on the changes in the revised ALS functional rating scale (ALSFRS-R) scores after 24 weeks administration in double-blind, placebo-controlled manner. And in addition, this study will be performed to examine the safety of MCI-186 to ALS patients.", "interventions": [{"type": "DRUG", "name": "MCI-186"}, {"type": "DRUG", "name": "Placebo of MCI-186"}], "start_date": "2006-05-31", "url": "https://clinicaltrials.gov/study/NCT00330681", "target_entities": ["oxidative_stress"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients who are defined as \"definite ALS,\" \"probable ALS\" or \"probable-laboratory-supported ALS,\" met diagnostic criteria revised EL Escorial for Airlie House.\n* Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life.\n* Patients of less than 3 years after the onset of ALS.\n* Patients whose progress of the condition during 12 weeks before administration meet other requirements.\n\nExclusion Criteria:\n\n* Patients judged to be inadequate to participate in this study by their physician, because those patients' general condition deteriorated to the point that they need to be hospitalized for severe hepatic disease, severe heart disease, severe renal disease and so on, or they need to be administered antibiotics to infection.\n* Patients who complain the difficulty in breathing caused by deteriorating the respiratory function.\n* Patients with such complications as Parkinson's disease, schizophrenia, dementia, renal failure, or other severe complication, and patients who have the anamnesis of hypersensitivity to edaravone.\n* Pregnant, lactating, and probably pregnant patients, and patients who want to become pregnant, and patients who can not agree to contraception.\n* Patients who have participated in other trials within 12 weeks before consent, or who are participating in other clinical trials at present.\n* In addition to the above exclusion criteria, patients judged to be inadequate to participate in this study by their physician.", "sex": "ALL", "min_age": "20 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MCI-186", "targeting_mechanism": "Antioxidant that reduces oxidative stress by neutralizing reactive oxygen species and maintaining the antioxidant system balance.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01806857", "title": "Clinical Trial Nuedexta in Subjects With ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Center for Neurologic Study, La Jolla, California,", "summary": "The purpose of this study is to determine whether Nuedexta is effective in the treatment of symptoms (impaired speech, swallowing, and saliva control)associated with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Nuedexta"}, {"type": "DRUG", "name": "Matching Placebo"}], "start_date": "2013-04", "url": "https://clinicaltrials.gov/study/NCT01806857", "target_entities": [], "locations": [{"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Georgetown University Medical Center", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Saint Mary's Health Care", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Neurology Associates, P.C.", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "The Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria\n* Age 18 years or older\n* Exhibits bulbar dysfunction manifested by dysarthria and/or dysphagia, according to PI judgment, exhibits a score of 55 or above on the CNS-Bulbar Function Scale\n* Capable of providing informed consent and following trial procedures\n* Geographic accessibility to the site\n* Women must not be able to become pregnant for the duration of the study and must be willing to be on two contraceptive therapies\n* Slow vital capacity (SVC) measure \u226550% of predicted for gender, height, and age at the screening visit\n* Must be able to swallow capsules throughout the course of the study, according to PI judgment\n* Subjects must not have taken riluzole for at least 30 days or be on a 50mg BID dose of riluzole for at least 30 days prior to randomization (subjects how have never taken riluzole are permitted in the study)\n* Subjects taking anti-sialorrhea medication(s) must be on a stable dose for at least 30 days prior to randomization (anti-sialorrhea na\u00efve subjects are permitted in the study)\n* Must be able to safely swallow at least 30 milliliters (mLs) of water for the water swallowing test\n\nExclusion Criteria:\n\n* Prior use of Nuedexta\u00ae\n* Current use of dextromethorphan, quinidine, quinine, mefloquine or opioids\n* History of quinidine, quinine, or mefloquine-induced thrombocytopenia, hepatitis, or other hypersensitivity reactions\n* History of known sensitivity or intolerability to dextromethorphan\n* Use of an mono amine oxidase inhibitor (MAOI) or within 14 days of stopping an MAOI\n* Prolonged QT interval, congenital long QT syndrome, history suggestive of torsades de pointes, or heart failure\n* Complete atrioventricular (AV) block without implanted pacemaker, or subjects at high risk of complete AV block\n* Concomitant use with drugs that both prolong QT interval and are metabolized by cytochrome P 2D6 (CYP2D6) (i.e., thioridazine or pimozide)\n* Exposure to any other experimental agent (off-label use or investigational) within 30 days prior to Baseline Visit\n* Invasive ventilator dependence, such as tracheostomy\n* Any history of either substance abuse within the past year, unstable psychiatric disease, cognitive impairment, or dementia, according to PI judgment\n* Placement and/or usage of feeding tube\n* Pregnant women or women currently breastfeeding\n* Unable to turn diaphragm pacing device off during swallowing tests\n* Salivatory Botox within 90 days (3 months) of screening\n* Salivatory radiation within 180 days (6 months) of screening", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Nuedexta", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00005674", "title": "Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis [ALS]", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Center for Research Resources (NCRR)", "summary": "The purpose of this study is to evaluate the safety and effectiveness of creatine treatment in amyotrophic lateral sclerosis (ALS). There is currently no known effective treatment for ALS. It is known that nerve cells die in the brains and spinal cords of patients with ALS but the cause of the cell death is unknown. It has been shown that there is overactive nerve activity due to increased levels of a chemical called glutamate and that there is abnormal cellular metabolism along with increased production of substance called \"free radicals.\" Improving cellular metabolism and readjusting the activity of glutamate in the brain may be beneficial to ALS patients.\n\nCreatine is a naturally occurring compound, which improves energy metabolism in cells. Creatine has been given to patients with energy metabolism defects in their muscles, and to athletes. Creatine improves survival in a mouse model of ALS. Three human subjects with ALS have received creatine for up to six months without any side effects. Overall, creatine has been well tolerated and safe.", "interventions": [{"type": "DRUG", "name": "Creatine"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT00005674", "target_entities": ["cellular_energy_metabolism"], "locations": [{"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* A clinical diagnosis of definite, probably or laboratory supported probably ALS, either sporadic or familial ALS according to a modified El Escorial criteria\n* Willing and able to give informed consent\n* FVC greater than or equal to 50% predicted\n* Evidence of abnormality in upper and/or lower extremity motor function (clinical evidence of muscle atrophy and weakness in an upper and/or lower extremity). The patient should have at least 4 or 8 testable upper extremity muscle groups.\n* Subjects may take riluzole. Riluzole must have been at stable doses for at least thirty days prior to baseline visit.\n* If woman of childbearing age, must be non-lactating and surgically sterile or using an effective method of birth control (double barrier or oral contraceptive) and have a negative pregnancy test\n* Disease duration less than five years", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Creatine", "targeting_mechanism": "Cellular energy metabolism enhancer that supports ATP production and mitochondrial function in motor neurons.", "targeting_mechanism_pmid": "29549424", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02313402", "title": "A New Eye-based Communication Device for ALS Patients", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "Eighteen ALS patients will be trained to control a new communication device (Eye On Line: EOL) that permit over smooth eye movements to generate digits, letters, words or drawing at will. The intervention consists in a training program during six visits over 3 weeks on site allowing a gradual acquisition of the eye-writing. The primary objective of the study is to assess the feasibility of the use of EOL device in ALS patients. The EOL device potentially offers a creative and personal means of linguistic and emotional expression in subject with motor disability.", "interventions": [{"type": "DEVICE", "name": "EOL (Eye On Line)"}], "start_date": "2014-06", "url": "https://clinicaltrials.gov/study/NCT02313402", "target_entities": [], "locations": [{"facility": "Groupe Hospitalier Pitie Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion criteria :\n\n* 18 to 65 years old\n* patient with SLA diagnosis\n* patient presenting writing troubles\n* patient with understandable speaking communication\n* patient with health insurance\n\nExclusion criteria :\n\n* patients presenting oculomotricity troubles\n* patients presenting frontotemporal dementia\n* patients presenting a chronic incapacitating disease other than ALS\n* patients presenting epilepsy antecedents\n* patents included in an other clinical study", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "EOL (Eye On Line)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07688239", "title": "The BANYAN Trial, an ALS MyMatch Trial Evaluating Safety, Biomarker Activity, and Microglial Activation of Nasal Foralumab", "phase": "PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Tiziana Life Sciences LTD", "summary": "The goal of this clinical trial is to learn whether a nasal spray medicine called foralumab is safe and easy to tolerate in adults with amyotrophic lateral sclerosis (ALS). ALS is a disease that slowly damages the nerve cells that control movement, causing muscles to grow weaker over time. Foralumab is being studied as a possible way to calm the inflammation and immune system activity that are thought to play a role in ALS.\n\nThe main questions it aims to answer are:\n\n1. Is nasal foralumab safe for people with ALS, and how well do they tolerate it over 12 and 24 weeks of treatment?\n2. Does foralumab change certain markers in the blood and spinal fluid, and lower inflammation in the brain, compared with a placebo?\n\nResearchers will compare people who take foralumab to people who take a placebo (a matching nasal spray with no medicine in it) to see how the two groups differ in safety and in these markers.\n\nAbout 44 adults aged 18 to 75 with ALS will take part. For the first 12 weeks, participants will be randomly assigned so that about 3 in 4 use foralumab and about 1 in 4 use the placebo. Neither the participants nor the study staff will know who is using which during this time (this is called \"double-blind\"). After the first 12 weeks, everyone who continues will use foralumab for the next 12 weeks.\n\nParticipants will:\n\n* Spray the study treatment into both nostrils 3 times a week, on alternate days, in cycles of 2 weeks on treatment followed by 1 week off\n* Learn to use the nasal spray device so they or a caregiver can give most doses at home\n* Take part for about 8.5 months in total, which includes screening, 24 weeks of treatment, and a safety check-in about 4 weeks after the last dose\n* Attend some visits at the study clinic and complete others remotely by phone or video\n* Have study tests that may include blood samples, breathing and muscle-strength checks, and - for those who agree and at certain study centers - spinal fluid samples and brain scans to measure inflammation", "interventions": [{"type": "DRUG", "name": "Foralumab Nasal"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2026-07-25", "url": "https://clinicaltrials.gov/study/NCT07688239", "target_entities": ["neuroinflammation", "immune_activation"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Adults \u226518 years of age and \u226475 years of age with a diagnosis of symptomatic ALS by revised El Escorial Criteria (clinically possible, probable, probable lab-supported, or definite).\n* Participant must be actively enrolled and must have met eligibility criteria for the Healey ALS MyMatch Common Screening Protocol (MCSP) AND determined to have met preliminary eligibility on targeted medical history and review of systems and safety laboratory results required for this trial.\n* Blood neurofilament (NfL) level of greater than 40 pg/mL as measured by the MyMatch Common Screening Protocol specific NfL assay.\n* Ability to provide written informed consent.\n* Less than or equal to 24-months since onset of weakness or spasticity attributed to ALS.\n* Slow vital capacity (SVC) of at least 65% predicted value for gender, height and age at trial screening visit.\n* Participant must not have taken riluzole and/or edaravone for at least 30 days, or be on a stable dose of riluzole and/or edaravone for at least 30 days prior to baseline (riluzole-na\u00efve or edaravone-na\u00efve participants are permitted in the study).\n* Negative urine pregnancy test within 7 days prior to the first dose of study therapy for women of child-bearing potential, defined as a sexually mature woman who has not undergone a hysterectomy or who has not been naturally postmenopausal for at least 24 consecutive months (i.e., who has had menses any time in the preceding 24 consecutive months). Sexually active women of child-bearing potential and male participants must agree to use highly effective methods to avoid pregnancy (oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) throughout the study and for 90 days after the completion of study treatment.\n* Participants must be able to travel to the study site for study visits and complete required study procedures.\n* Participants who are participating in the PET or Lumbar Puncture (LP) outcomes, must be determined to be able to safely tolerate these study procedure(s) in the opinion of the study investigator AND as determined by the targeted medical history and review of systems in the MCSP. Both procedures are highly encouraged.\n* Participants whose immunizations are fully up to date at Screening, according to the assessment of their primary care physician and neurologist.\n\nExclusion Criteria:\n\n* Clinically significant medical condition that would pose a risk to the participant, according to the PI's judgment and examination prior to baseline.\n* Active ENT disease (such as allergic rhinitis, chronic rhinitis, active sinusitis treated within the past 30 days or deemed as clinically relevant, clinically significant deviated nasal septum, nasal polyps) that might interfere with safely administering the drug nasally.\n* Active clinically significant infection or immunocompromised condition.\n* Treatment with chronic immunosuppressants or other immunosuppressants within the past 90 days.\n* Concomitant use of investigational treatments for ALS within 5 half-lives or 30 days prior to the Baseline visit, whichever is longest (high dosage methylcobalamin permitted).\n* Exposure to any gene or cell therapies under investigation for treatment of ALS.\n* Inability to tolerate nasally administered medications.\n* Participant reported history of any vaccines within past 14 days (prior to first dose of foralumab).", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Foralumab Nasal", "targeting_mechanism": "Immunomodulatory antibody that targets CD3 on T cells to reduce neuroinflammation and immune system activation in the CNS.", "targeting_mechanism_pmid": "28872464", "animal_results": "Tyrosine kinase inhibition with masitinib controlled microgliosis, neuroinflammation, and slowed disease progression in SOD1G93A rats, significantly prolonging survival when delivered after paralysis onset.", "animal_results_pmid": "27400786", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01348451", "title": "Human Spinal Cord Derived Neural Stem Cell Transplantation for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuralstem Inc.", "summary": "This is a first-in-human trial of spinal derived stem cells transplanted into the spinal cord of patients with Amyotrophic Lateral Sclerosis (ALS). The goal of the study is to see if the cells and the procedure to transplant them are safe.", "interventions": [{"type": "DEVICE", "name": "surgical implantation"}], "start_date": "2009-01", "url": "https://clinicaltrials.gov/study/NCT01348451", "target_entities": [], "locations": [{"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Have the ability to understand the requirements of the study, provide written informed consent, understand and provide written authorization for the use and disclosure of Protected Health Information (PHI) \\[per Health Insurance Portability and Accountability Act (HIPAA) Privacy Ruling\\] and comply with the study procedures.\n2. Subjects with sporadic or familial ALS diagnosed as laboratory-supported probable,probable or definite according to the World Federation of Neurology El Escorial Criteria (Appendix A), based on examination by the site PI.\n3. Age 18 years or older.\n4. Females must have a negative serum pregnancy test and practice an acceptable method of contraception or be of non-childbearing potential (post-menopausal for at least 2 years or surgically sterile \\[hysterectomy, oophorectomy or surgical sterilization\\]).\n5. Geographic accessibility to the study center and the ability to travel to the clinic for study visits.\n6. Presence of a willing and able caregiver.\n7. Medically able to undergo lumbar or cervical laminectomy as determined by the Investigator, surgeon and anesthesiologist.\n8. Medically able to tolerate immunosuppression regimen consisting of basiliximab, tacrolimus, mycophenolate mofetil, and methylprednisolone as determined by the site Investigator.\n9. Agrees to the visit schedule as outlined in the informed consent.\n10. Not taking riluzole (Rilutek\u00ae) or on a stable dose for \u226530 days.\n11. All required vaccinations current: tetanus/diptheria (TDAP), herpes zoster/shingles(Vostavax\u00ae: within last 10 years and must be prior to surgery), pneumonia (Pneumovax\u00ae),seasonal/H1N1 flu vaccines (as appropriate for season) for Groups B-E.\n\nExclusion Criteria:\n\n1. Etiology of paraplegia or weakness is due to causes other than ALS such as spinal ischemia, traumatic spinal injury, traumatic brain injury, multiple sclerosis, cerebral stroke, cerebral palsy, or infection.\n2. VC \\< 60% predicted normal by standard nomogram at the time of screening and VC \\< 50% predicted normal measured supine for age at the time of surgery.\n3. Current or peak Panel Reactive Antibody (PRA) due to alloantibodies \\> 20% receiving their first allograft.\n4. Any known immunodeficiency syndrome.\n5. Receipt of any investigational drug,device or biologic within 30 days of surgery.\n6. Any concomitant medical disease or condition limiting the safety to participate:\n\n   * Coagulopathy\n   * Active uncontrolled infection\n   * Hypotension requiring vasopressor therapy\n   * Previous spinal surgery at the site of planned transplantation except for anterior cervical dissection fusion (ACDF)\n   * Skin breakdown over the site of surgery\n   * Malignancy (except for non-melanoma skin cancer)\n   * Primary or secondary immune deficiency\n   * Spinal stenosis.\n7. Creatinine \\>1.5, liver function tests (SGOT/SGPT, Bilirubin, Alk Phos) \\> 2x the upper limit of normal, hematocrit/hemoglobin \\< 30/10, total WBC \\< 4000, uncontrolled hypertension (defined as systolic \\>180 or diastolic \\>100) or uncontrolled diabetes(defined as hemoglobin A1C \\>8), evidence of GI bleeding by hemoccult test, positive tuberculosis (TB test: PPD/Mantoux), hepatitis B or C, or human immunodeficiency virus (HIV).\n8. Presence of any of the following conditions:\n\n   * Current drug abuse or alcoholism\n   * Unstable medical conditions\n   * Unstable psychiatric illness including psychosis and untreated major depression within 90 days of screening\n   * Positive blood test for hepatitis B or C.\n9. Any condition that the site PI feels may interfere with participation in the study.\n10. Any condition that the surgeon feels may pose complications for the surgery.\n11. Known hypersensitivity to basiliximab, tacrolimus, mycophenolate mofetil, or methylprednisolone.\n12. Inability to provide informed consent as determined by screening protocol.\n13. Inadequate family or caregiver support as determined by the site PI.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Human spinal cord-derived neural stem cells (neural progenitor cells)", "targeting_mechanism": "Neural progenitor cells transduced to secrete GDNF, which provides neuroprotective support to motor neurons through glial differentiation and growth factor delivery.", "targeting_mechanism_pmid": "36064599", "animal_results": "Human neural progenitor cells transduced with GDNF differentiated to astrocytes and protected spinal motor neurons in animal models.", "animal_results_pmid": "36064599", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02588677", "title": "Masitinib in Combination With Riluzole for the Treatment of Patients Suffering From Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AB Science", "summary": "The objective is to compare the efficacy and safety of masitinib in combination with riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Masitinib (4.5)"}, {"type": "DRUG", "name": "Riluzole"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Masitinib (3.0)"}], "start_date": "2013-04", "url": "https://clinicaltrials.gov/study/NCT02588677", "target_entities": ["receptor_tyrosine_kinase"], "locations": [{"facility": "Hospital Carlos III", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\nMain inclusion criteria:\n\n1. Familial or sporadic ALS\n2. Patient diagnosed with probable of definite ALS\n3. Patient treated with a stable dose of riluzole (100 mg/day) for at least 30 days prior to screening\n\nExclusion Criteria:\n\n1\\. Patient who underwent tracheostomy and/or gastrostomy", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Masitinib", "targeting_mechanism": "Tyrosine kinase inhibitor that targets microgliosis and neuroinflammation by controlling aberrant glial cell activation.", "targeting_mechanism_pmid": "27400786", "animal_results": "Masitinib abrogated neuroinflammation, controlled microgliosis and aberrant glial cells, and significantly prolonged survival when delivered after paralysis onset in SOD1G93A rats.", "animal_results_pmid": "27400786", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01020331", "title": "Memantine Therapy in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Phoenix Neurological Associates, LTD", "summary": "Tau, a protein in the cerebrospinal fluid CSF is believed to be elevated in amyotrophic lateral sclerosis (ALS) patients. The investigators believe that Tau is truly a marker of increased neuronal death from any disease process. It is been shown that Memantine can inhibit and reverse the abnormal hyperphosphorylation of Tau and therefore the investigators are looking at the efficacy of Memantine at 10 mg twice a day (BID) to see if disease progression correlates with possible changes in Tau in ALS patients based on ALS Functional Rating Scale (ALSFRS) scores.", "interventions": [{"type": "DRUG", "name": "Memantine"}], "start_date": "2005-06", "url": "https://clinicaltrials.gov/study/NCT01020331", "target_entities": ["glutamate_excitotoxicity"], "locations": [{"facility": "Phoenix Neurological Associates, LTD", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria\n\n1. Age 18-85\n2. Male or Female\n3. Clinically definite ALS by El Escorial criteria\n4. Elevated levels of Tau in CSF\n\nExclusion Criteria:\n\n1. Patients with FVC below 1.5 L or who require respiratory assistance\n2. History of liver disease\n3. Severe renal failure\n4. History of intolerance to Riluzole or Memantine\n5. Any other co morbid condition which would make completion of trial unlikely\n6. If female, pregnant or breast-feeding; or, if of childbearing age, an unwillingness to use birth control.\n7. Taking any trial medications. Non-trial medications are not cause for exclusion.\n8. Unwillingness to provide consent", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Memantine", "targeting_mechanism": "NMDA receptor antagonist that inhibits and reverses abnormal hyperphosphorylation of tau, a cerebrospinal fluid marker of neuronal death.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07478172", "title": "Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Missouri-Columbia", "summary": "This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD). Due to motor unit impairments, NMD patients often cannot tolerate traditional exercise. WB-EMS bypasses voluntary activation limits by directly stimulating muscle contractions. Up to 50 adults with conditions like ALS, SMA, and MG will undergo 20-minute supervised WB-EMS sessions (1-2 times weekly for 4-8 weeks) using the Katalyst system. Outcomes include neural excitability (TMS), motor unit behavior (EMG, NCS), functional tests (walk, balance, strength), and patient-reported fatigue, pain, and quality of life. Strict safety monitoring and exclusion criteria are in place. This study will provide preliminary data on WB-EMS as a potential exercise modality for NMD.", "interventions": [{"type": "DEVICE", "name": "Whole-body Electrical Muscle Stimulation Exercise"}], "start_date": "2026-03-10", "url": "https://clinicaltrials.gov/study/NCT07478172", "target_entities": [], "locations": [{"facility": "NextGen Precision Health Building, Clinical and Translational Science Unit", "city": "Columbia", "state": "Missouri", "country": "United States", "status": "RECRUITING", "lat": 38.95171, "lon": -92.33407}], "contact_phone": "573-884-2596", "contact_email": "kristina.kelly@health.missouri.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Age 18 or older\n* Diagnosed with one or more of the following neuromuscular conditions: Amyotrophic lateral sclerosis, primary lateral sclerosis, progressive muscle atrophy, spinal muscular atrophy, postpolio syndrome, inclusion body myositis, pompedisease, fascioscapulohumeral muscular dystrophy, charcot marie tooth disease, chronic inflammatory demyelinating polyneuropathy, hereditary spastic paraplegia, myasthenia gravis, lambert-eaton myasthenic syndrome, postural orthostatic tachycardia syndrome, mitochondrial myopathy, nemaline myopathy, centronuclear myopathy, lumbar radiculopathy, non-specific low back pain.\n* Ability to stand for approximately 15 minutes continuously with or without an assistive device (i.e. the length of time to stand to take a shower, complete meal preparation, wait in line at the bank, etc.)\n* At least some anti-gravity strength in major muscle groups as assessed by manual muscle testing (i.e. 2+/5 strength or better)\n* Medical clearance to participate in an exercise program\n* Ability to provide informed consent\n* Ability to conform to the requirements of the study (i.e. attendance at assessment and intervention visits, maintain current level of non-study physical activity for the duration of the study, no intention to relocate mid-study)\n\nExclusion Criteria:\n\n* Diagnosed with one of the following neuromuscular conditions: Becker's muscular dystrophy, Duchenne muscular dystrophy, limb-girdle muscular dystrophy, myotonic dystrophy type 1 or 2, Freidrich's ataxia, any other NMD with known or suspected cardiac involvement or muscle fiber structural integrity defects.\n* Concurrent participation in another interventional research study\n* Unable to tolerate 15 minutes of continuous standing with or without an assistive device\n* Presence of a pacemaker, metal implants, or other implanted medical devices that could impact participant safety during WB-EMS intervention\n* Presence of cochlear implant, cortical stimulator, deep brain stimulator, ventriculoperitoneal shunt, recent skull defect, seizure in the past 12 months while taking anti-epilepsy medication, or previous serious adverse event with TMS, which could impact participant safety during TMS testing\n* Presence of unstable acute or chronic disease (i.e. renal failure, rheumatologic disease, cardia arrhythmia, neoplasm, uncontrolled hypertension)\n* Known pregnancy at time of screening; verbal screening will occur throughout the study.\n* Presence of a terminal disease (i.e. receiving hospice services)\n* Current or previous use of any drugs known to influence muscle mass or performance within 6 months; these may include but are not limited to anabolic steroids, IGF01, growth hormone, replacement androgen therapy, anti-androgen therapy\n* Presence of an additional neurologic conditions affecting somatosensory or motor function/control (i.e. Parkinson's disease, Multiple Sclerosis, h/o stroke, TBI, SCI, ataxia, apraxia, hemiplegia, etc.)\n* Musculoskeletal condition or surgery in the past year that would confound results of exercise interventions (i.e. TKA, THA, RTC repair, spinal fusion)\n* Other medical conditions, signs, or symptoms that would interfere with study conductor interpretation of results as determined by an investigator", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Whole-body electrical muscle stimulation (WB-EMS) exercise", "targeting_mechanism": "Non-pharmacological intervention that directly stimulates muscle contractions by bypassing voluntary activation limits, compensating for motor unit impairments.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03508453", "title": "IC14 for Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Implicit Bioscience", "summary": "Fifty patients with amyotrophic lateral sclerosis that is progressing rapidly will be randomized to receive either the monoclonal antibody IC14 or placebo to be given intravenously over two hours twice weekly for 12 weeks. Blood and urine tests will be done to measure biomarkers in order to evaluate clinical response and to monitor for safety. Other evaluations include patient questionnaires about function, quality of life and mental function; pulmonary function test; and sniff nasal pressure.", "interventions": [{"type": "BIOLOGICAL", "name": "IC14"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2021-08-15", "url": "https://clinicaltrials.gov/study/NCT03508453", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Signed informed consent prior to initiation of any study-specific procedures.\n2. Familial or sporadic MND defined as clinically possible, probable, or definite by Awaji-Shima Consensus Recommendations.\n3. Rapidly progressive MND as defined by a decline of 3 or more points in the ALSFRS-R score during the prior 3 months.\n4. First symptoms of MND within 3 years of informed consent.\n5. Age between 18 and 75 years at time of informed consent.\n6. Seated Forced Vital Capacity (FVC) \u2265 65% of predicted value.\n7. Not taking riluzole or edaravone or on a stable dose of riluzole or edaravone for at least 3 months prior to screening visit.\n8. Adequate bone marrow reserve, renal and liver function:\n\n   * absolute neutrophil count \u2265 1.5 x 109/L\n   * lymphocyte count \\< 6.0 x 109/L\n   * platelet count \u2265 150 x 109/L\n   * hemoglobin \u2265 110 g/L\n   * eGFR \u2265 40 mL/min/1.73 m2\n   * ALT and/or AST \u2264 2x ULN\n   * total bilirubin \u2264 1.5x ULN\n   * serum albumin \u2265 28 g/L\n9. Females of childbearing potential should be using and committed to continue using one of the following acceptable birth control methods:\n\n   * Sexual abstinence (inactivity) for 1 month prior to screening through study completion; or\n   * Intrauterine device (IUD) in place for at least 3 months prior to study through study completion; or\n   * Stable hormonal contraception for at least 3 months prior to study through study completion; or\n   * Surgical sterilization (vasectomy) of male partner at least 6 months prior to study.\n10. To be considered of non-childbearing potential, females should be surgically sterilized (bilateral tubal ligation, hysterectomy, or bilateral oophorectomy at least 2 months prior to study) or be post-menopausal and at least 3 years since last menses.\n11. Males with female partners of childbearing potential must use contraception through study completion.\n12. Able to give informed consent and able to comply with all study visits and all study procedures.\n\nExclusion Criteria:\n\n1. Dependence on mechanical ventilation, defined as being unable to lay supine without it, unable to sleep without it, or continuous daytime use; presence of tracheostomy at screening; or presence of diaphragm pacing system at screening.\n2. Treatment with a drug or device within the last 30 days that has not received regulatory approval.\n3. Treatment within 12 months with immunomodulator or immunosuppressant agent (including but not limited to cyclophosphamide, cyclosporine, interferon-\u03b1, interferon-\u03b2-1a, rituximab, alemtuzumab, azathioprine, etanercept, infliximab, adalimumab, certolizumab, golimumab, anakinra, rilonacept, secukinumab, tocilizumab, mycophenolate mofetil, methotrexate, haematopoietic stem cell transplantation, anti-sense drugs, gene therapy, cell-depleting agents, total lymphoid irradiation). Treatment with intravenous immunoglobulin within 2 months or dimethyl fumarate within 3 months. Non-steroidal anti-inflammatory drugs are acceptable.\n4. Known active current or history of recurrent bacterial, viral, fungal, mycobacterial or other opportunistic infections; or major episode of infection requiring hospitalization or treatment with IV antibiotics within 4 weeks.\n5. Live-attenuated vaccines within 30 days before dosing. Subjects must agree to forego live-attenuated vaccines throughout the study, including 12 weeks after the last dose of study drug.\n6. History of severe allergic or anaphylactic reactions to human, humanized or murine monoclonal antibodies.\n7. Presence of any of the following clinical conditions:\n\n   * History of one or more of the following: cardiac insufficiency (New York Heart Association \\[NYHA\\] III/IV), uncontrolled cardiac arrhythmias, unstable ischemic heart disease, or uncontrolled hypertension (systolic blood pressure \\> 170 mmHg or diastolic blood pressure \\> 110 mmHg).\n   * History of venous thromboembolic disease within 12 months, myocardial infarction, or cerebrovascular accident.\n   * Unstable pulmonary, renal, hepatic, endocrine or hematologic disease.\n   * Autoimmune disease, mixed connective tissue disease, scleroderma, polymyositis, or significant systemic involvement secondary to rheumatoid arthritis.\n   * Evidence of active malignant disease, malignancies diagnosed within the previous 5 years, or breast cancer diagnosed within the previous 5 years (except skin cancers other than melanoma).\n   * History of human immunodeficiency virus infection or other immunodeficiency illness.\n   * Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days.\n   * History of drug abuse (not including marijuana use) or alcoholism within the past 12 months.\n   * Significant neuromuscular disease other than MND.\n   * Other ongoing disease that may cause neuropathy, such as toxin exposure, dietary deficiency, uncontrolled diabetes, hyperthyroidism, cancer, systemic lupus erythematosus or other connective diseases, infection with HIV, hepatitis B virus (HBV), or hepatitis C (HCV), Lyme disease, multiple myeloma, Waldenstr\u00f6m's macroglobulinemia, amyloid, and hereditary neuropathy.\n8. Pregnancy or breastfeeding.\n9. Deprivation of freedom by administrative or court order.", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "IC14", "targeting_mechanism": "Monoclonal antibody targeting complement component C5a receptor to reduce neuroinflammation and glial activation in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04244630", "title": "Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Dallas VA Medical Center", "summary": "The purpose of this research is to investigate the validity of a previous clinical trial named EH301, which showed beneficial effects of anti-oxidant therapies in patients with amyotrophic lateral sclerosis (ALS). If validated by this study, providing over-the-counter anti-oxidants would be a simple, low risk, low-cost approach to significantly slow or stop the progression of ALS, for which currently no effective treatment exists. It is currently thought that oxidative stress is a major cause of ALS. The study investigators are therefore planning to expand the original scope of the previous trial by including anti-oxidants at high doses that were not previously used. All of these compounds are considered safe.", "interventions": [{"type": "COMBINATION_PRODUCT", "name": "Antioxidants"}], "start_date": "2023-11-02", "url": "https://clinicaltrials.gov/study/NCT04244630", "target_entities": ["oxidative_stress"], "locations": [{"facility": "VA North Texas Health Care System", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. A clinical diagnosis by a study investigator of laboratory-supported probable, probable, or definite ALS, according to a modified El Escorial criterion (Appendix 2).\n2. 21 to 80 years of age inclusive.\n3. If patients are taking riluzole for ALS, they must be on a stable dose for at least thirty days prior to the baseline visit.\n4. Willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).\n\nExclusion Criteria:\n\n1. Diagnosis of other neurodegenerative diseases (Parkinson disease, Alzheimer disease, etc.).\n2. Clinically significant history of unstable medical illness (unstable angina, advanced cancer, etc.) over the last 30 days.\n3. Infection with the human immunodeficiency virus (HIV)\n4. Limited mental capacity such that the patient cannot provide written informed consent or comply with evaluation procedures.\n5. History of recent alcohol or drug abuse or noncompliance with treatment or other experimental protocols. 6 Receipt of any investigational drug within the past 30 days.", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Antioxidants", "targeting_mechanism": "Reduces oxidative stress by countering reactive oxygen species imbalance in motor neurons.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05442775", "title": "A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "The purpose of this study is to assess the long-term safety and tolerability of reldesemtiv in patients with ALS who have successfully completed dosing in the Phase 3 clinical trial, CY 5031 (also known as COURAGE-ALS)", "interventions": [{"type": "DRUG", "name": "Reldesemtiv"}], "start_date": "2022-08-04", "url": "https://clinicaltrials.gov/study/NCT05442775", "target_entities": ["neuromuscular_junction"], "locations": [{"facility": "St. Joseph's Hospital & Medical Center - Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California Irvine - ALS & Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center - Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Hospital Anschutz Outpatient Pavilion", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "George Washington Medical Faculty Associates", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "University of Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Mayo Clinic Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida - Carol and Frank Morsani Center for Advanced Health Care", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Indiana University IU Health Neuroscience Center", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "The University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins Outpatient Center", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Michigan Medicine", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Hospital", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Neurology Associates", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University Institute for Human Performance", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Atrium Health Neuroscience Institute", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Texas Neurology, P.A.", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Virginia Commonwealth University", "city": "Henrico", "state": "Virginia", "country": "United States", "status": "", "lat": 36.59264, "lon": -78.61611}, {"facility": "Froedtert Hospital - Department of Neurology", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "Concord Repatriation General Hospital", "city": "Concord", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.84722, "lon": 151.10381}, {"facility": "Royal Brisbane and Women's Hospital, Neurology Department", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "The Perron Institute", "city": "Nedlands", "state": "", "country": "Australia", "status": "", "lat": -31.98184, "lon": 115.8073}, {"facility": "Uz Leuven Gasthuisberg Department of Neurology", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "University of Calgary - Heritage Medical Research Clinic", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "McMaster University", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "Ottawa Hospital Research Institute", "city": "Ottawa", "state": "Ontario", "country": "Canada", "status": "", "lat": 45.41117, "lon": -75.69812}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "University of Saskatchewan", "city": "Saskatoon", "state": "Saskatchewan", "country": "Canada", "status": "", "lat": 52.13238, "lon": -106.66892}, {"facility": "CHU de Quebec-Universite Laval", "city": "Qu\u00e9bec", "state": "", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "RSCI Education and Research Center Beaumount Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "IRCCS Istituto Auxologico Italiano Ospedale San Luca U.O. Neurologia e Stroke Unit", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "AOU Citta della Salute e della Scienza di Torino Universita degli Studi di Torino P.O. Mollinette - Dipartimento de Neuroscienze \"Rita Levi Montalcini\"", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "UMC Utrecht Department of Neurology, ALS Center", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Universitario y Politecnico La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Studieenheten, Akademiskt Specialistcentrum", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Able to comprehend and willing to sign an ICF and willing to comply with all study procedures and restrictions for the duration specified in the Schedule of Activities. If non-written consent is given, a Legal Designee of the patient must sign the ICF form.\n* Completed dosing in CY 5031\n\nExclusion Criteria:\n\n* Has taken investigational study drug (other than reldesemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater\n* Presence on Day 1 of any medically significant cardiac, pulmonary, gastrointestinal, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data.\n* Use of a strong cytochrome P450 (CYP) 3A4 inhibitor within 7 days prior to first dose of reldesemtiv in CY 5032 or a strong CYP3A4 inducer within 14 days prior to first dose of reldesemtiv in CY 5032\n* Use of a medication that is an OCT1/OCT2 substrate within 7 days prior to first dose of reldesemtiv in CY 5032\n* Currently participating in another trial, managed access program, open label extension, early access program, or through the right to try act is receiving an investigational drug or received an investigational drug or device within 30 days (or 5 half-lives for drugs, whichever is longer) prior to Day 1. Patients also cannot be taking outside of a clinical trial certain investigational drugs (which includes drugs, supplements, and nutraceuticals) that are currently being studied or have been studied for the treatment of ALS.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Reldesemtiv", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03168711", "title": "Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "This is a multi-center, 20-week study of inosine treatment.\n\nStudy Objectives and Endpoints The primary objective of the study is to determine the safety and tolerability of oral administration of inosine (administered daily) dosed to moderately elevate serum urate over 20 weeks.\n\nThe primary outcome measures will be\n\n1. Safety, as measured by adverse events\n2. Tolerability, defined as the ability of subjects to complete the entire 20-week study.\n\nAs an exploratory objective, we will test the feasibility and utility of a smartphone application for monitoring symptoms and disease progression in patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Inosine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2017-10-01", "url": "https://clinicaltrials.gov/study/NCT03168711", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Holy Cross Hospital", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 18-85.\n2. Sporadic or familial ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria (Appendix 1).\n3. Slow vital capacity (SVC) \u2265 60% of predicted for age, height, and gender at the Screening Visit.\n4. Capable of providing informed consent and following trial procedures.\n5. Serum urate \\< 5.5 mg/dL at screening (i.e. below the population median serum urate levels).\n6. Women must not be able to become pregnant (e.g. post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and 3 months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device (IUD) in place for \u2265 3 months, barrier method in conjunction with spermicide, or another adequate method.\n7. Is able and willing to participate in the Mobile app study procedures.\n\nExclusion Criteria:\n\n1. History of urolithiasis.\n2. Urine pH \\< 5.5 at screening (as acidic urine is a major determinant of uric acid urolithiasis).\n3. History of gout.\n4. History of stroke or myocardial infarction.\n5. History of symptomatic coronary artery disease (e.g. angina pectoris) or symptomatic peripheral arterial disease within 1 year prior to Screening.\n6. Symptomatic congestive heart failure with a documented ejection fraction below 45%.\n7. Poorly controlled arterial hypertension (SBP\\>160mmHg or DBP\\>100mmHg at Screening).\n8. Women who are pregnant or lactating.\n9. The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the subject to provide informed consent, according to Site Investigator judgment, or a history of active substance abuse within the prior year.\n10. Anything that, in the opinion of the Site Investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.\n11. Use of the following within 30 days prior to Screening: inosine, allopurinol, probenecid, more than 300mg vitamin C daily (note that a subject may take a standard multivitamin up to one tablet or capsule daily). Use of thiazides is permissible as long as the subject is on a stable dose from 1 week prior to Screening.\n12. Known hypersensitivity or intolerability to inosine.\n13. Renal insufficiency as defined by eGFR \\< 60 mL/min/1.73m2 at the time of screening.", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Inosine", "targeting_mechanism": "Elevates serum urate to provide neuroprotection against oxidative stress in motor neurons.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05597436", "title": "Intermediate-Sized Expanded Access Study", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Seelos Therapeutics, Inc.", "summary": "This expanded access protocol is to provide access to the investigational product, SLS-005, to participants with ALS who are not eligible to participate in clinical trials.", "interventions": [{"type": "DRUG", "name": "Trehalose"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT05597436", "target_entities": ["protein_folding"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "University of California, San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "George Washington University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Holy Cross Hospital - Phil Smith Neuroscience Institute", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "Nova Southeastern University", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "Saint Alphonsus Regional Medical Center", "city": "Boise", "state": "Idaho", "country": "United States", "status": "", "lat": 43.6135, "lon": -116.20345}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Maryland School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Sean M. Healey & AMG Center for ALS Neurological Clinical Research Institute, Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Beth Israel Deaconess Medical Center", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Spectrum Health/Corewell Health", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "University of Minnesota", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Lehigh Valley Hospital", "city": "Allentown", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.60843, "lon": -75.49018}, {"facility": "Thomas Jefferson University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Texas Neurology, P.A.-Neal Site:769", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Virginia Commonwealth University Clinical Research Unit", "city": "Richmond", "state": "Virginia", "country": "United States", "status": "", "lat": 37.55376, "lon": -77.46026}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Sporadic or familial ALS.\n2. Age 18 years or older.\n3. Cohort 1: Patients who do not qualify for any reasonably accessible ongoing clinical trial.\n4. Cohort 2: Patients who have completed the open label extension (OLE) period of Regimen E of the HEALEY ALS Platform Trial and are not eligible for enrollment in another treatment regimen of the platform study.\n5. Capable of providing informed consent and complying with study procedures, in the Site Investigator's (SI's) opinion.\n6. Participants have established care with a physician at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP.\n7. Participants must have a life expectancy of at least 6 months in SI's opinion.\n\nExclusion Criteria:\n\n1. Current diagnosis or healthcare professional-recommended treatment (medication, exercise or diet) of diabetes mellitus.\n2. Screening glucose \\>=140 mg/dl.\n3. Known hypersensitivity to trehalose.\n4. Current use of oral trehalose.\n5. Inability for participant to return to site for weekly drug administration, until approved for home infusions.\n6. Screening body weight \\>144 kilograms.\n7. Participant with a history of any clinically significant or unstable medical condition or lab abnormality based on the SI's judgment that may interfere with assessment of the study objectives, with safety or full participation.\n8. Females who are pregnant or nursing or who plan to get pregnant during the course of the EAP.\n9. Females of child-bearing potential, or males, who are unwilling or unable to use highly effective methods of birth control.\n10. Use of investigational treatments for ALS (as part of participation in a clinical trial or another EAP) within 5 half-lives (if known) or 30 days (whichever is longer) prior to the Screening Visit.\n11. Permanent assisted ventilation (PAV), defined as more than 22 hours per day of noninvasive or invasive mechanical ventilation for more than seven consecutive days. The date of onset of PAV is the first day of the seven days.\n12. Active cancer or history of cancer, except for the following: basal cell carcinoma or successfully treated squamous cell carcinoma of the skin, cervical carcinoma in situ, prostatic carcinoma in situ, or other malignancies curatively treated and with no evidence of disease recurrence for at least 3 years.\n13. Presence of unstable psychiatric disease, cognitive impairment, dementia, or substance abuse that would impair ability of the participant to provide informed consent, in the SI's opinion.\n14. Patients who chose to take experimental medications and/or supplements, and that is the only reason they are not eligible for trials, won't be eligible for the EAP.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": null, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Trehalose", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03573466", "title": "Presymptomatic Neuromuscular Junction Defects and Compensatory Mechanisms in ALS", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "Denervation of neuromuscular junctions (NMJs) and initial compensatory reinnervation is the earliest pathological event in various motor neuron disease models, occurring far before motor symptom onset. In patients harboring genetic mutations responsible for Amyotrophic Lateral Sclerosis (ALS), identification of early, pre-symptomatic, NMJ pathological events and compensatory mechanisms could lead to the development of new treatments to prevent motor functional impairment.\n\nThe aims of our study are thus:\n\n1. To investigate and characterize early, presymptomatic, defects of NMJ morphology in pre-manifest C9ORF72 or SOD1 mutation carriers;\n2. To investigate and quantify reinnervation at the level of NMJs in these subjects;\n3. To identify muscle molecular dysregulated pathways involved in the development of NMJ alterations and the development / maintenance of compensatory collateral reinnervation.", "interventions": [{"type": "PROCEDURE", "name": "Muscle biopsy"}], "start_date": "2019-04-10", "url": "https://clinicaltrials.gov/study/NCT03573466", "target_entities": [], "locations": [{"facility": "Piti\u00e9-Salp\u00eatri\u00e8re Hospital", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Subjects harboring a mutation in C9ORF72 or SOD1 gene\n* Without any functional motor complaint and clinical motor deficit at neurological examination\n* Aged 30 to 70 (inclusive).\n* Patients covered by the social insurance system\n\nExclusion criteria:\n\n* Any motor deficit\n* Any significant cognitive or psychiatric impairment leading to limitation in decision making capacity\n* Inability to give informed consent\n* Patient unable to contact or to be contacted by the investigator in case of emergency\n* Women who are pregnant or nursing, or without effective contraceptive method\n* Concomitant medication contraindicating muscle biopsy (platelet suppressive agents, oral anticoagulant therapy)\n* Medical condition contraindicating muscle biopsy (hypocoagulative disease, allergy to anaesthetic drugs, acute intermittent porphyria)", "sex": "ALL", "min_age": "30 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "Neuromuscular junction denervation and compensatory reinnervation occur before motor symptom onset in ALS mouse models (SOD1G93A).", "animal_results_pmid": "30320556", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02116634", "title": "Mesenchymal Stem Cell Injection in Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Alzahra Hospital, Iran", "summary": "Whether the mesenchymal injection on ALS patients is effective or not?", "interventions": [{"type": "BIOLOGICAL", "name": "mesenchymal stem cell"}], "start_date": "2015-05", "url": "https://clinicaltrials.gov/study/NCT02116634", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Neurosciences Research Center", "city": "Isfahan", "state": "", "country": "Iran", "status": "", "lat": 32.65246, "lon": 51.67462}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. sporadic ALS according to escorial criteria\n2. onset of disease with spinal cord involvement, Less than 3 years of disease onset with disease progression at 6 past months\n3. mild to moderate spinal and bulbar disability,at least having score3 in swallowing, 2 in chewing and waking in ALS-FRS and FVC(functional vital capacity) equal or more than 50% of prediction amount\n4. normal polysomnography\n5. Signed consent form\n\nExclusion Criteria:\n\n1. pregnancy or lactation,\n2. vascular disease,diabetes, systemic disease as cancer, autoimmune , liver or hematologic disease\n3. Hospitalization due to serious illness in the last two months\n4. survival time less than two years\n5. Hypersensitivity to any component used in the cell culture", "sex": "ALL", "min_age": "18 Years", "max_age": "60 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "mesenchymal stem cell", "targeting_mechanism": "Stem cells differentiate into support cells such as astrocytes, oligodendrocytes, or microglia, which produce growth factors and anti-inflammatory cytokines, provide nutrients, and buffer excessive glutamate to support degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Majority of preclinical stem cell studies have been performed in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1), demonstrating potential benefit of stem cell therapy for ALS.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07467746", "title": "Research of Traditional Chinese Medicine Oral Preparation of C. Cicadae in the Treatment of ALS Patients With Elevated Plasma Sphingolipids", "phase": "PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The Third Xiangya Hospital of Central South University", "summary": "Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease characterized by the degeneration of motor neurons, leading to progressive muscle weakness and functional decline. This study is designed as a randomized, double-blind, placebo-controlled clinical trial to evaluate the efficacy and safety of an oral preparation of C. cicadae in patients with sporadic ALS and elevated plasma sphingolipid (SL) levels. Efficacy will be assessed primarily by changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score and plasma SL levels.Participants who meet the eligibility criteria and provide written informed consent will be randomly assigned in a 1:1 ratio to either the C. cicadae treatment group or the placebo group. The treatment group will receive oral C. cicadae at a dose of 0.1 g/kg/day (dry weight), administered in three divided doses per day. The placebo group will receive a matched placebo with a similar appearance and odor, administered according to the same schedule. A total of approximately 84 participants will be enrolled. The intervention period will be 6 months, and participants will be followed for a total of 9 months.", "interventions": [{"type": "DRUG", "name": "C. cicadae"}, {"type": "DRUG", "name": "Control"}], "start_date": "2026-04", "url": "https://clinicaltrials.gov/study/NCT07467746", "target_entities": ["sphingolipid_metabolism"], "locations": [], "contact_phone": "+8618975172668", "contact_email": "zhangruxu@vip.163.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients with ALS diagnosed according to the revised El Escorial criteria.\n* Significantly elevated plasma levels of key SL molecules (e.g., Cer(d18:0/24:0), Cer(d18:1/22:0), and other relevant molecules), meeting the predefined cut-off values for metabolic stratification.\n* Time from disease onset to enrollment \u226424 months\n* For participants receiving riluzole and/or edaravone, the dose must have been stable for at least 30 days prior to enrollment.\n* Male or non-pregnant, non-lactating female patients, aged \\> 18 and \u2264 80 years old.\n* Voluntarily participate in clinical trials, sign informed consent, and be able to understand and abide by research procedures.\n\nExclusion Criteria:\n\n* Presence of peripheral neuropathy or motor neuron injury attributable to other clearly defined etiologies and sufficient to interfere with disease classification in this study, including but not limited to vitamin deficiency, toxic neuropathy, drug- or chemotherapy-related neuropathy, alcoholic neuropathy, paraneoplastic syndrome, autoimmune neuropathy, and infection-related neuropathy;\n* Severe hepatic or renal dysfunction that may affect the safety evaluation of the investigational product or the interpretation of metabolomics results;\n* Severe cardiopulmonary dysfunction, active infection, active malignancy, or other major systemic diseases that, in the opinion of the investigator, may significantly affect prognosis assessment or completion of follow-up;\n* Women who are pregnant or breastfeeding, or who plan to become pregnant during the study period;\n* Participation in another interventional clinical trial within 30 days prior to enrollment, or being within the washout period of another investigational product;\n* Inability to comply with clinical assessments, sample collection, or follow-up procedures;\n* Persistent high dependence on noninvasive ventilation (\\>16 hours/day), or advanced respiratory failure as judged by the investigator, such that the participant is unable to complete oral intervention and efficacy evaluation;\n* Severe dysphagia, gastrointestinal dysfunction, or other conditions rendering the participant unable to tolerate oral administration;\n* Known allergy to C. cicadae preparations, fungal products, or any of their excipients;\n* Concomitant diseases or conditions that may substantially affect motor function assessment and thereby interfere with evaluation of the primary endpoint;\n* Any other condition that, in the opinion of the investigator, makes the participant unsuitable for study participation.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "C. cicadae", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00812851", "title": "Randomized Placebo-Controlled Crossover Trial With THC (Delta 9-Tetrahydrocannabinol) for the Treatment of Cramps in Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cantonal Hospital of St. Gallen", "summary": "Many patients with ALS experience cramps during the course of the disease. Frequently, cramps occur as the first symptom of the disease, months before the patients notice weakness and wasting. Cramp severity varies from mild, without affecting daily activities and sleep, to disabling, where almost any voluntary muscle activity induces long standing, severely painful cramping. ALS patients who smoke herbal cannabis (marijuana) or drink hemp tea report lessening of cramps and fasciculations. Although, various medications, such as magnesium, quinine sulfate, lioresal, dantrolene, clonazepam, diphenylhydantoin and gabapentin are used for the treatment of cramps in ALS so far, no medication has been of proven benefit. However, a recent pilot study with THC in ALS showed symptomatic effects in \"spasms\", fasciculations, insomnia and appetite. The aim of the proposed study is to determine the tolerability, safety and efficacy of THC in the treatment of cramps in ALS. The hypothesis is that THC will lessen cramps in ALS.", "interventions": [{"type": "DRUG", "name": "Dronabinol"}], "start_date": "2005-04", "url": "https://clinicaltrials.gov/study/NCT00812851", "target_entities": ["Cannabinoid receptor"], "locations": [{"facility": "Kantonsspital St.Gallen", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients must be at least 18 years of age and have full legal capacity\n* Patients must voluntarily give written informed consent\n* Patients diagnosed with possible, probable laboratory supported, probable or definite ALS according to the revised El Escorial criteria (Brooks 2000)\n* Patients must score severity of cramps on the VAS 5 or more\n* Patients must be able to communicate and report adverse events by phone\n* Patients must have laboratory parameters within the following limits: Creatinine, Bilirubin,Transaminases less than 3x upper limit of normal\n* Patients may take any medication for the treatment of ALS (ALS -specific and -symptomatic) but may not change this medication during the study period\n* Patients must not have cannabis or cannabinoids for at least one month prior to the study and agree not to use it at all during the study. They have to have a negative urinary test for cannabinoids at baseline\n* Pre-menopausal females must provide negative pregnancy test within fourteen days before beginning of study participation and have to apply adequate (barrier) birth-control methods\n* Patients must agree not to drive a vehicle or use dangerous machines during the entire study period\n\nExclusion Criteria:\n\n* Patients who are not willing or able to sign the consent form. Doubt of investigator concerning compliance of the patient\n* Patients who have a history of failure to respond to, or had significant adverse effects from or hypersensitivity to THC or any cannabinoid\n* Patients who have significant concomitant illness(-es), or acute, uncontrolled infections, which might make evaluation of treatment and side effects difficult\n* Patients with a history of significant psychiatric disorder, explicitly of schizophrenia\n* Patients who are current drug abusers, including alcohol abusers\n* Patients with severe coronary artery disease or hemodynamically relevant ECG-documented arrhythmia\n* Pregnancy or breast feeding", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Dronabinol", "targeting_mechanism": "Cannabinoids exert anti-excitotoxicity, anti-oxidant, and anti-inflammatory effects in motor neuron disease.", "targeting_mechanism_pmid": "30520038", "animal_results": "Preclinical studies in mice models of ALS using cannabinoid formulations have been published, demonstrating effects consistent with anti-excitotoxicity, anti-oxidant, and anti-inflammatory mechanisms.", "animal_results_pmid": "30520038", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03984708", "title": "New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways.", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Tours", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects central and peripheral motor neurons. None of the clinical trials conducted have been clearly successful and the disease remains incurable, putting patients' vital prognosis at risk in the medium term. An alteration of the basal metabolism leading to hypermetabolism has been described in several articles in the literature. The causes of this hypermetabolism and the precise exploration of the metabolic pathways involved are still poorly understood. The fibroblasts of ALS patients may be the site of some metabolic disturbances in this disease with a hypothetical specific basal metabolic profile. These cells are adapted to different metabolic explorations such as omnic approaches. Superficial skin biopsy followed by fibroblast culture can provide a considerable biobank. This cellular richness will allow us, in ALS patients and their controls, to perform metabolomic and lipidomic approaches, as well as the quantification transcriptomic approach.\"", "interventions": [{"type": "OTHER", "name": "Samples"}, {"type": "OTHER", "name": "Indirect calorimetry"}, {"type": "OTHER", "name": "Electrical bioimpedance"}], "start_date": "2020-01-27", "url": "https://clinicaltrials.gov/study/NCT03984708", "target_entities": ["energy_metabolism"], "locations": [{"facility": "Neurology Department, University Hospital, Limoges", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Neurology Department, University Hospitla, Tours", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Case group selection criteria:\n\nInclusion Criteria:\n\n* Age \u2265 18 years and \u2265 75 years\n* ALS according to the El Escorial criteria\n* Diagnosis of ALS \\< 6 months\n* Symptoms onset \\< 2 years\n* Patients affiliated to social security scheme\n* Informed consent signed by the patient\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding women\n* Contraindication to biopsy\n* Contraindication to local anesthesia\n* Treatment with oral or injectable anticoagulants, antiplatelet (except aspirin)\n* Unbalanced Diabetes\n* Systemic corticosteroid treatment\n* Dermatological diseases of the fibroblast\n* Skin cancer\n* Protection measure for guardianship or curatorship\n\nControl group selection criteria:\n\nInclusion Criteria:\n\n* Age \u2265 18 years and \u2265 75 years\n* No neuronal disease\n* Patients affiliated to social security scheme\n* Informed consent signed by the patient\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding women\n* Contraindication to biopsy\n* Treatment with oral or injectable anticoagulants, antiplatelet (except aspirin)\n* Unbalanced Diabetes\n* Systemic corticosteroid treatment\n* Dermatological diseases of the fibroblast\n* Skin cancer\n* Protection measure for guardianship or curatorship", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06094205", "title": "Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Leigh R. Hochberg, MD, PhD.", "summary": "The goal of this study is to improve our understanding of speech production, and to translate this into medical devices called intracortical brain-computer interfaces (iBCIs) that will enable people who have lost the ability to speak fluently to communicate via a computer just by trying to speak.", "interventions": [{"type": "DEVICE", "name": "BrainGate Neural Interface System"}], "start_date": "2023-10-16", "url": "https://clinicaltrials.gov/study/NCT06094205", "target_entities": [], "locations": [{"facility": "University of California, Davis", "city": "Sacramento", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 38.58157, "lon": -121.4944}], "contact_phone": "617-724-9247", "contact_email": "lhochberg@mgh.harvard.edu", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Between 18 and 80 years of age\n* Clinical diagnosis of spinal cord injury, brainstem stroke, muscular dystrophy, amyotrophic lateral sclerosis or other motor neuron disorders\n* Complete or incomplete tetraplegia (quadriplegia)\n* Must live within a three-hour drive of the Study site and geographically stable for at least 15 months after enrollment.\n\n(There are additional inclusion criteria)\n\nExclusion Criteria:\n\n* Visual impairment such that extended viewing of a computer monitor would be difficult even with ordinary corrective lenses\n* Chronic oral or intravenous steroids or immunosuppressive therapy\n* Other serious disease or disorder that could seriously affect ability to participate in the study\n\n(There are additional exclusion criteria)", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07023835", "title": "Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Zydus Therapeutics Inc.", "summary": "Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis", "interventions": [{"type": "DRUG", "name": "50 mg Usnoflast"}, {"type": "DRUG", "name": "75 mg Usnoflast"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2025-09-17", "url": "https://clinicaltrials.gov/study/NCT07023835", "target_entities": ["usnoflast"], "locations": [{"facility": "Zydus US015", "city": "La Jolla", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 32.84727, "lon": -117.2742}, {"facility": "Zydus US008", "city": "Orange", "state": "California", "country": "United States", "status": "NOT_YET_RECRUITING", "lat": 33.78779, "lon": -117.85311}, {"facility": "Zydus US013", "city": "San Francisco", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.77493, "lon": -122.41942}, {"facility": "Zydus US005", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "RECRUITING", "lat": 41.66121, "lon": -72.77954}, {"facility": "Zydus US012", "city": "Tampa", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 27.94752, "lon": -82.45843}, {"facility": "Zydus US007", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "RECRUITING", "lat": 33.749, "lon": -84.38798}, {"facility": "Zydus US010", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "Zydus US006", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "RECRUITING", "lat": 42.33143, "lon": -83.04575}, {"facility": "Zydus US014", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "RECRUITING", "lat": 40.8, "lon": -96.66696}, {"facility": "Zydus US003", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "RECRUITING", "lat": 36.09986, "lon": -80.24422}, {"facility": "Zydus US009", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 40.44062, "lon": -79.99589}, {"facility": "Zydus US001", "city": "Dallas", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 32.78306, "lon": -96.80667}, {"facility": "Zydus US002", "city": "Houston", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 29.76328, "lon": -95.36327}, {"facility": "Zydus US004", "city": "Richmond", "state": "Virginia", "country": "United States", "status": "RECRUITING", "lat": 37.55376, "lon": -77.46026}, {"facility": "Zydus US011", "city": "Seattle", "state": "Washington", "country": "United States", "status": "RECRUITING", "lat": 47.60621, "lon": -122.33207}, {"facility": "Zydus 200", "city": "Caulfield South", "state": "Australia", "country": "Australia", "status": "RECRUITING", "lat": -37.89562, "lon": 145.02597}, {"facility": "Zydus 201", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": -33.86785, "lon": 151.20732}, {"facility": "Zydus 202", "city": "Southport", "state": "", "country": "Australia", "status": "NOT_YET_RECRUITING", "lat": -27.96724, "lon": 153.39796}, {"facility": "Zydus 110", "city": "Leuven", "state": "Belgium", "country": "Belgium", "status": "NOT_YET_RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "Zydus 101", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "NOT_YET_RECRUITING", "lat": 43.70643, "lon": -79.39864}, {"facility": "Zydus 100", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "ACTIVE_NOT_RECRUITING", "lat": 46.81228, "lon": -71.21454}, {"facility": "Zydus 124", "city": "Clermont-Ferrand", "state": "France", "country": "France", "status": "NOT_YET_RECRUITING", "lat": 45.77969, "lon": 3.08682}, {"facility": "Zydus 122", "city": "Limoges", "state": "France", "country": "France", "status": "NOT_YET_RECRUITING", "lat": 45.83362, "lon": 1.24759}, {"facility": "Zydus 123", "city": "Montpellier", "state": "France", "country": "France", "status": "RECRUITING", "lat": 43.61093, "lon": 3.87635}, {"facility": "Zydus 125", "city": "Nice", "state": "France", "country": "France", "status": "RECRUITING", "lat": 43.70313, "lon": 7.26608}, {"facility": "Zydus 120", "city": "Paris", "state": "France", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}, {"facility": "Zydus 121", "city": "Tours", "state": "France", "country": "France", "status": "RECRUITING", "lat": 47.39484, "lon": 0.70398}, {"facility": "Zydus 130", "city": "Dresden", "state": "Germany", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.05089, "lon": 13.73832}, {"facility": "Zydus 133", "city": "Hanover", "state": "Germany", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 52.37052, "lon": 9.73322}, {"facility": "Zydus 134", "city": "Jena", "state": "Germany", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 50.92878, "lon": 11.5899}, {"facility": "Zydus 131", "city": "L\u00fcbeck", "state": "Germany", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 53.86893, "lon": 10.68729}, {"facility": "Zydus 132", "city": "Rostock", "state": "Germany", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 54.0887, "lon": 12.14049}, {"facility": "Zydus 135", "city": "Ulm", "state": "Germany", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "Zydus 140", "city": "Dublin", "state": "Ireland", "country": "Ireland", "status": "NOT_YET_RECRUITING", "lat": 53.33306, "lon": -6.24889}, {"facility": "Zydus 154", "city": "Genova", "state": "Italy", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 45.21604, "lon": 11.87211}, {"facility": "Zydus 153", "city": "Milan", "state": "Italy", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 45.46427, "lon": 9.18951}, {"facility": "Zydus 151", "city": "Modena", "state": "Italy", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 44.64783, "lon": 10.92539}, {"facility": "Zydus 152", "city": "Naples", "state": "Italy", "country": "Italy", "status": "RECRUITING", "lat": 40.85216, "lon": 14.26811}, {"facility": "Zydus 150", "city": "Brescia", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.53558, "lon": 10.21472}, {"facility": "Zydus 160", "city": "Utrecht", "state": "Netherlands", "country": "Netherlands", "status": "RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "Zydus 170", "city": "Krakow", "state": "Poland", "country": "Poland", "status": "NOT_YET_RECRUITING", "lat": 50.06143, "lon": 19.93658}, {"facility": "Zydus 171", "city": "Warsaw", "state": "Poland", "country": "Poland", "status": "NOT_YET_RECRUITING", "lat": 52.22977, "lon": 21.01178}, {"facility": "Zydus 180", "city": "Alicante", "state": "Spain", "country": "Spain", "status": "NOT_YET_RECRUITING", "lat": 38.34517, "lon": -0.48149}, {"facility": "Zydus 181", "city": "Barcelona", "state": "Spain", "country": "Spain", "status": "NOT_YET_RECRUITING", "lat": 41.38879, "lon": 2.15899}, {"facility": "Zydus 182", "city": "M\u00e1laga", "state": "Spain", "country": "Spain", "status": "NOT_YET_RECRUITING", "lat": 36.72016, "lon": -4.42034}, {"facility": "Zydus 183", "city": "Valencia", "state": "Spain", "country": "Spain", "status": "RECRUITING", "lat": 39.47391, "lon": -0.37966}, {"facility": "Zydus 191", "city": "Malm\u00f6", "state": "Sweden", "country": "Sweden", "status": "RECRUITING", "lat": 55.60587, "lon": 13.00073}, {"facility": "Zydus 190", "city": "Stockholm", "state": "Sweden", "country": "Sweden", "status": "NOT_YET_RECRUITING", "lat": 59.32938, "lon": 18.06871}, {"facility": "Zydus 192", "city": "Ume\u00e5", "state": "Sweden", "country": "Sweden", "status": "NOT_YET_RECRUITING", "lat": 63.82842, "lon": 20.25972}], "contact_phone": "6094534751", "contact_email": "fshaikh@zydustherapeutics.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of probable or definite Amyotrophic lateral sclerosis, according to the revised version of the El Escorial World Federation of Neurology criteria\n* Time since onset of first symptom of Amyotrophic lateral sclerosis \u226424 months. Date of Amyotrophic lateral sclerosis symptom onset. For the purposes of this study, the date of symptom onset will be defined as the date the subject first had symptoms of their disease, i.e., limb weakness, dysarthria, dysphagia, shortness of breath, or fasciculations, from the screening visit.\n* Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised score of \u226535 at screening.\n* Slow vital capacity: \u226560% of predicted capacity at the screening visit.\n* Be able to swallow capsules.\n* Either not currently receiving riluzole/sodium phenylbutyrate and taurursodiol/tofersen or on a stable dose of riluzole/sodium phenylbutyrate and taurursodiol/tofersen for at least 4 weeks before the screening visit. Subjects receiving riluzole/sodium phenylbutyrate and taurursodiol/tofersen are expected to remain on the same dose throughout the duration of the study.\n* Either not currently receiving edaravone or on edaravone treatment. Subjects receiving edaravone must have completed at least 1 cycle of treatment before the screening visit and are expected to continue with a stable dose of edaravone treatment throughout the duration of the study.\n* Capable of providing informed consent and complying with study procedures in the opinion of the investigator\n\nExclusion Criteria:\n\n* Presence of unstable psychiatric disease, cognitive impairment, dementia, or substance abuse that would impair the ability of the subject to provide informed consent, in the opinion of the investigator.\n* Serious illness (e.g., pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with intravenous antibiotics, antivirals, or antifungals within 4 weeks of screening; chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the judgment of the investigator.\n* Active herpes zoster infection within 2 months prior to the screening visit.\n* Any medical condition that promotes suicidal attempt or behavior within 6 months prior to the screening visit and in the opinion of the investigator might interfere with subject's participation in the study or is a risk for a suicide attempt.\n* History of unstable or severe cardiac, pulmonary, oncological, hepatic, or renal disease or active cancer or another medically significant illness other than Amyotrophic lateral sclerosis, precluding safe participation of subject in this study in the opinion of the investigator.\n* Known allergy, sensitivity, or intolerance to Investigational product or excipients.\n* Subjects who have taken concomitant medications that are substrates of drug metaboliz-ing enzymes (Cytochrome P450 1A2 and/or Cytochrome P450 2B6) within 7 days or 5 half-lives of the medication (whichever is longer) before the first dose of Investigational product and throughout the study.\n* Use of any steroids, colchicine, or anti-IL-1 inhibitors within 7 days or 5 half-lives of the medication (whichever is longer) prior to the first dose of Investigational product administration.\n* Use of any investigational drug concurrently or within 4 weeks or 5 half-lives (whichever is longer) prior to the first dose of Investigational product administration.\n* Any clinically significant condition and/or laboratory significant value that would prevent the subject from participating in the study in the opinion of the investigator.\n* Received a live vaccine within 14 days before the screening visit or planning to receive during the study duration.\n* Subjects who have received stem cell or gene therapy for Amyotrophic lateral sclerosis at any time in the past.\n* Following laboratory test values at screening:\n\n  1. Alanine aminotransferase or Aspartate aminotransferase values \\>3.0 \u00d7 Upper Limit of Normal\n  2. Bilirubin \\>1.5 \u00d7 Upper Limit of Normal unless the subject has documented Gilbert's syndrome (isolated bilirubin \\>1.5 \u00d7 Upper Limit of Normal is acceptable if bilirubin is fractionated, and direct bilirubin is \\<35%)\n  3. Estimated glomerular filtration rate (eGFR) \\<60 mL/min/1.73 m2\n* For those participating in the optional Cerebrospinal fluid collection, contraindications to lumbar puncture including but not limited to lumbar scoliosis, coagulopathy, infection at site of puncture, or use of anticoagulants.\n* Subjects with history of epilepsy within 6 months of screening visit.\n* Surgery within last 3 months or planned major surgery within next 3 months from the date of screening (other than minor cosmetic surgery and minor dental surgery).\n* Use or intended use of any medications/products known to alter drug absorption, metabolism, or elimination processes, including St. John's Wort, within 4 weeks of screening and up to end of study. Use of such medication will be considered on a case-by-case basis as per the opinion of the investigator and/or independent medical monitor.\n* Receiving an elemental diet or parenteral nutrition.\n* Received blood transfusion within 3 months prior to screening.\n* Subjects with Human immunodeficiency virus, hepatitis B, hepatitis C, coronary artery disease, or active gastrointestinal condition that might interfere with drug absorption.\n* Inability to be venipunctured or those not able to tolerate venous puncture.\n* Employee of the investigator or study site, with direct involvement in the proposed study or other studies under the direction of that investigator or study site, as well as family members of employees of investigator or the investigator.\n* Any condition not mentioned in any of above criteria that, as per the investigator, would hinder participation of the subject in the study. This may include, but not limited to, considerations of safety, compliance, or other factors that could impact the integrity of the study or the well-being of the subject.\n* If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of Investigational product. If male of reproductive capacity, unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of Investigational product.\n\nFor Open Label Extension\n\nInclusion Criteria:\n\n* Completion in the randomized, double blind Usnoflast study (main study).\n* Subjects who elect to continue treatment after completion of Usnoflast phase 2b study must enrol in the OLE within 28 days of the completion of Week 36 visit of the main study.\n* Provide a new informed consent to enter the OLE phase.\n\nExclusion Criteria:\n\n* Discontinued IP prematurely in the double-blind phase of the study for reasons other than tracheostomy or permanent-assisted ventilation.\n* Treatment with or use of any restricted medications.\n* Any ongoing AE that, in the opinion of the site investigator, is clear contraindication to the IP.\n* Unstable cardiac or other life-threatening disease emergent during the randomized, double-blind study\n* Any major medical history or other evidence of severe illness or any other conditions that would make the subject, in the opinion of the investigator, unsuitable for the study.\n* If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or of child-bearing potential and unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of IP. If male of reproductive capacity, unwilling to use effective contraception during the study and for at least 1 month after administration of last dose of IP.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Usnoflast", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06169696", "title": "EMPOWER Early Feasibility Study: Non-invasive BCI to Control a Wheelchair for People With Paralysis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Synaptrix Labs Inc.", "summary": "Neuralis is an innovative assistive technology designed for individuals with severe neuromuscular conditions, enabling wheelchair control through EEG signals. This study aims to assess the safety, feasibility, and efficacy of Neuralis in restoring mobility and independence.\n\nThe device is a discreet EEG headset which specializes in decoding signals from visual cortex, allowing users to initiate precise wheelchair movements through focused attention. This research seeks to demonstrate Neuralis' potential in revolutionizing assistive technology by offering a non-invasive, user-friendly solution for individuals facing motor impairments, ultimately enhancing their quality of life.", "interventions": [{"type": "DEVICE", "name": "Intervention/Treatment"}], "start_date": "2024-11", "url": "https://clinicaltrials.gov/study/NCT06169696", "target_entities": [], "locations": [{"facility": "Synaptrix Labs", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "5107099964", "contact_email": "aryan@synaptrix-labs.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Severe quadriparesis\n2. Able to give consent\n3. Appropriate candidate for device\n4. Able and willing to access all clinical testing and not impeded by geographical location\n5. Proficient in English\n6. Have a study partner\n\nExclusion Criteria:\n\n1. History of seizures\n2. History of epilepsy\n3. Psychiatric or psychological disorder\n4. No study partner or caregiver\n5. Unable to provide evidence of COVID vaccination", "sex": "ALL", "min_age": "21 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Neuralis", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07224269", "title": "Effect of Terazosin on ATP Levels in People With Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Iowa", "summary": "This will be a single center, randomized, double-blind, placebo-controlled pilot study to assess the safety and tolerability of terazosin (TZ) at a dose of 5 milligrams (mg) per os (PO) daily for patients with amyotrophic lateral sclerosis (ALS). The primary outcome of this study is to determine whether TZ increases adenosine triphosphate (ATP) levels in ALS. The investigators will measure adverse outcomes, safety, and tolerability of taking TZ. Procedures include blood draws, spirometry, fluorodeoxyglucose-positron emission tomography (FDG-PET) scans, questionnaires, and physical examinations. TZ will be titrated up to 5 mg PO daily. This is a pilot study and is not powered to assess efficacy of this medication. The investigators' hope is that this study will guide future studies of this (and similar) medications for the disease modification of ALS. This study also aims to learn more about how patients produce and use energy and if TZ can help to reverse energy deficits that appear in ALS.", "interventions": [{"type": "DRUG", "name": "Terazosine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2026-08-12", "url": "https://clinicaltrials.gov/study/NCT07224269", "target_entities": ["atp_production"], "locations": [{"facility": "University of Iowa Health Care", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "RECRUITING", "lat": 41.66113, "lon": -91.53017}], "contact_phone": "319-356-8744", "contact_email": "emily-anderson@uiowa.edu", "eligibility": {"criteria": "INCLUSION CRITERIA\n\n* Ages 18 - 80 years old\n* Diagnosed with ALS based on Gold Coast Criteria\n* ALS symptom onset within 36 months at enrollment\n* Slow vital capacity (SVC) greater than or equal to 60%\n* Riluzole use-Never taken or taking a stable dose for at least 4 weeks prior to screening visit or will refrain from starting for the duration of the study\n* Edaravone use-Never taken or completed at least one cycle (typically 14 days) prior to screening visit or will refrain from starting for the duration of the study\n* Must have the ability to swallow pills at the time of the screening visit, and in the principle investigator's opinion, have the ability to swallow pills for the duration of the study\n* Willing to use highly effective contraception for the duration of the trial treatment and for a duration of 80 days after the last dose.\n\nEXCLUSION CRITERIA\n\n* Orthostatic hypotension at screening is defined as decrease in BP \\> 20 mmHg systolic or \\> 10 mmHg diastolic and HR increase \\<20 bpm on transition from supine to sitting or from sitting to standing\n* Known allergy or previous adverse reaction to terazosin or related compound\n* Current use of terazosin or concurrent use of doxazosin, alfuzosin, prazosin, or tamsulosin at the time of screening visit or within the 3 months prior to baseline visit\n* Pregnancy or breastfeeding women\n* Taking therapeutic anticoagulant medication (i.e. warfarin, DOAC's, full dose Lovenox or heparin)\n* Liver function blood tests (ALT or AST) more than twice the upper limit of normal\n* Hemoglobin \\< 11.0 g/dL\n* Traumatic brain injury or post-traumatic stress disorder\n* Presence of a confounding acute or unstable medical, psychiatric, or orthopedic condition\n* Noncompliant or sporadic use of medications that modulate the central nervous system\n* Uncontrolled major depression or bipolar affective disorder, or other mental health disorders that are, in the opinion of the PI, sufficiently severe to increase risk of experiencing an Adverse Drug Reaction (ADR)\n* Current suicidal ideation as measured by question 2 of the Columbia-Suicide Severity Rating Scale (C-SSRS)\n* Participants with insufficient decisional capacity to provide written informed consent determined by the primary investigator.\n* Noncompliant or sporadic use of antihypertensive medications\n* Unable to lie supine and still for 60 minutes for the duration of the study\n* Currently taking part in another clinical trial with an investigational medicinal product or having taken part in one in the three months prior to screening visit\n* Current diagnosis of diabetes (type 1 or type 2) or healthcare professional-recommended treatment (medication, exercise or diet) of diabetes mellitus\n* Screening visit glucose \\>140 mg/dl", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Terazosin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04762589", "title": "RT001 in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biojiva LLC", "summary": "RT001-014 is a Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects with Amyotrophic Lateral Sclerosis", "interventions": [{"type": "DRUG", "name": "RT001"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-03-10", "url": "https://clinicaltrials.gov/study/NCT04762589", "target_entities": ["rt001"], "locations": [{"facility": "University of Tartu", "city": "Tartu", "state": "", "country": "Estonia", "status": "", "lat": 58.38062, "lon": 26.72509}, {"facility": "Riga Stradins Universtiy", "city": "Riga", "state": "", "country": "Latvia", "status": "", "lat": 56.946, "lon": 24.10589}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Karolinska", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Male or female subject with age 20 years to 75 years at the time of signed consent\n2. Patients who are defined as \"definite ALS,\" \"probable ALS\" or \"probable-laboratory-supported ALS,\" met diagnostic criteria revised EL Escorial for Airlie House.\n3. ALSFRS-R \\> 20\n4. Patients who can eat a meal, excrete, or move with oneself alone, and do not need assistance in everyday life\n5. Patients of less than 3 years after the onset of ALS\n6. Subject has an identified, reliable, study partner (e.g., caregiver, family member, social worker, or friend)\n7. If patients are duly capable of study consent but are unable to sign by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation\n\nExclusion Criteria:\n\n1. Received treatment with other experimental therapies within the last 30 days prior to the first dose\n2. Previously received treatment with RT001\n3. Refusal to discontinue fish oils or other oil-based supplements for the duration of the study (Screening till last study procedure completed)\n4. SVC \\< 70 at screening\n5. Subject has a feeding tube or the need for a feeding tube is anticipated within the first 24 weeks after enrollment\n6. Subject resides at a skilled nursing or dementia care facility, or admission to such a facility is planned during the study period\n7. Evidence of any clinically significant neurological disorder other than ALS\n8. The subject has a history of or currently has schizophrenia, schizoaffective disorder or bipolar disorder according to DSM-V or ICD-10 criteria\n9. The subject has a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect of ALS on respiratory function\n10. Subject has had a significant illness or infection requiring medical intervention in the past 30 days\n11. Female who is breastfeeding or has a positive pregnancy test\n12. Male participant or female participant of childbearing potential, who is sexually active and unwilling/unable to use a medically acceptable and effective double barrier birth control method throughout the study\n13. Unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to return for visits as scheduled\n14. History, within the last 2 years, of alcohol abuse or physical opioid dependence", "sex": "ALL", "min_age": "20 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "RT001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06645197", "title": "An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University Third Hospital", "summary": "This is a multicenter, open-label, single-arm investigator-initiated clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of SNUG01 in patients with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "SNUG01"}], "start_date": "2024-10-16", "url": "https://clinicaltrials.gov/study/NCT06645197", "target_entities": ["snug01"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}, {"facility": "Fujian Medical University Union Hospital", "city": "Fujian", "state": "Fuzhou", "country": "China", "status": "", "lat": 28.92651, "lon": 106.12972}, {"facility": "JiangSu Provincce Hospital", "city": "Nanjing", "state": "Jiangsu", "country": "China", "status": "", "lat": 32.06167, "lon": 118.77778}, {"facility": "West China Hospital of Sichuan University", "city": "Chengdu", "state": "Sichuan", "country": "China", "status": "", "lat": 30.66667, "lon": 104.06667}, {"facility": "Second Affiliated Hospital Zhejiang University School of Medicine", "city": "Hangzhou", "state": "Zhejiang", "country": "China", "status": "", "lat": 30.29365, "lon": 120.16142}], "contact_phone": "15710013912", "contact_email": "neurbj@126.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1.Able to understand and voluntarily sign the informed consent form, the informed consent form must be signed before performing any clinical trail procedures.\n\n2.18\\~80 years old (including 18 and 80 years old), both male and female. 3.Must have been diagnosed with clinically probable ALS, clinically possible laboratory-supported ALS, or clinically definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria.\n\n4.Less than or equal to 36 months (inclusive) after the onset of symptoms of amyotrophic lateral sclerosis.\n\n5.Body Mass Index (BMI) \u226519 kg/m2. 6.The forced vital capacity (FVC) in the screening period is greater than or equal to 70% of the estimated vital capacity.\n\n7.Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score \u2265 26 during the screening period, with scores of \u2265 4 in the three respiratory items (dyspnea, orthopnea, and respiratory insufficiency) on the ALSFRS-R.\n\n8\\. Adequate organ function: Hematological: Neutrophil count\u22651.5 \u00d7 10\u2079/L, platelet count\u2265 100 \u00d7 10\u2079/L, Hemoglobin \u226590 g/L. Renal: Creatinine \u22641.5\u00d7ULN or creatinine clearance (CCr) \u226550 ml/min using the Cockcroft-Gault formula.\n\nHepatic: Total bilirubin (TBIL), alanine aminotransferase (ALT), aspartate aminotransferase (AST)\u22642 \u00d7 ULN, alkaline phosphatase (ALP) \u2264 3 \u00d7 ULN.\n\nCoagulation: International Normalized Ratio (INR), Prothrombin Time (PT), Activated Partial Thromboplastin Time (APTT) \u22641.5 \u00d7 ULN.\n\n9.Subjects are not currently receiving riluzole or have been receiving a stable dose of riluzole for at least 4 weeks prior to the screening visit. Subjects treated with riluzole are expected to remain on the same dose throughout the study period.\n\n9.Subjects are not currently treated with edaravone or are being treated with the approved standard regimen of edaravone. Subjects being treated with edaravone must have completed at least 1 cycle of treatment prior to the Screening Visit and are expected to continue treatment with edaravone throughout the study period.\n\n10.Women of childbearing age must have a negative blood pregnancy test during the screening period and be non-lactating. Female subjects of childbearing age (women of childbearing age include premenopausal women and women within 2 years after menopause, except those who have undergone bilateral tubal ligation, complete oophorectomy or hysterectomy.) and male subjects whose partners are women of childbearing age must Agree to use effective contraceptive methods throughout the study period, such as abstinence, double barrier contraceptive methods, condoms, intrauterine devices and other non-drug contraceptive measures, and are not allowed to donate sperm or eggs.\n\nExclusion Criteria:\n\n1. Serum anti-AAV9 neutralizing antibody (Nab) titer \\&gt; 1:100 at the time of screening.\n2. There are contraindications to lumbar puncture during the screening period (including but not limited to skin infection at the administration site and signs or symptoms of increased intracranial pressure), receiving any active intrathecal therapy, and having implants for drainage of cerebrospinal fluid (CSF). Inserted shunt, presence of implanted central nervous system (CNS) cannula, or any condition that prevents CSF collection.\n3. There are other diseases related to motor neuron dysfunction (progressive bulbar palsy, primary lateral sclerosis, cervical spondylosis, lumbar spondylosis, etc., idiopathic inflammatory myopathy), which may confuse or cover up the diagnosis of ALS.\n4. Previously required invasive ventilation or tracheotomy due to ALS disease, or currently using non-invasive ventilation support with an average of \u226516 hours/day.\n5. Previous history of gene therapy, hematopoietic stem cell transplantation, and solid organ transplantation.\n6. The patient has poorly controlled acute or chronic respiratory diseases, including but not limited to: chronic obstructive pulmonary disease, severe asthma, severe pneumonia, active tuberculosis.\n7. Those who have been implanted or the researchers estimate that they will need to implant a diaphragmatic pacing system during the study period.\n8. Any thromboembolic events occurred within 6 months before the first administration, such as deep vein thrombosis, pulmonary arteriovenous embolism, jugular vein embolism, etc..\n9. Received another drug for the treatment of ALS disease (including but not limited to sodium phenylbutyrate (PB), taurine diol (TURSO), tauroursodeoxycholic acid (TUDCA) within 4 weeks before the first dose ) or ursodeoxycholic acid (UDCA), biologics, etc. except riluzole and edaravone).\n10. Autoimmune disease (e.g., rheumatoid arthritis, systemic lupus erythematosus, inflammatory bowel disease, scleroderma, inflammatory myopathies, mixed connective tissue disease, overlap syndrome, etc.) within 30 days prior to the Screening Period, or ongoing immune-related therapy (e.g., corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab, leflunomide, hydroxychloroquine, interleukin 2 antagonists, etc.), except for intranasal, inhaled, ocular, topical topical, intra-articular corticosteroid therapy, or physiologic replacement therapy with corticosteroids.\n11. Suffering from active or uncontrolled infection (including but not limited to: infectious pneumonia, sepsis, herpes zoster infection) within 4 weeks before the first administration, or chronic bacterial infection that is considered unacceptable according to the investigator\\&#39;s judgment ( medical history such as tuberculosis).\n12. Have undergone major surgery within 4 weeks before the first administration, or have not recovered from previous treatment-related adverse events (AE) to \u2264 grade 1 (CTCAE 5.0), except alopecia.\n13. Participated in another clinical study within 4 weeks before the first administration, unless it is an observational (non-intervention) clinical study.\n14. Any febrile illness occurred within 14 days before the first administration.\n15. Have been vaccinated within 14 days before the first dose.\n16. Patients with poorly controlled hypertension (refers to resting blood pressure after treatment: systolic blood pressure (SBP) \\&gt; 160 mmHg or diastolic blood pressure (DBP) \\&gt; 100 mmHg).\n17. Arterial oxygen saturation and/or finger pulse oxygen saturation \\&lt;93% after treatment with oxygen therapy or noninvasive respiratory support (\\&lt;16 hrs/day) at the time of screening, as determined by the investigator.\n18. Hypersensitivity to prednisolone, other glucocorticoids or their excipients.\n19. Positive hepatitis B surface antigen (HBsAg) or positive hepatitis B core antibody (anti-HBcAb) with peripheral blood HBV DNA titer \u2265 1.0E+02 IU/mL.; positive hepatitis C virus (HCV) antibody with positive HCV RNA results; positive human immunodeficiency virus (HIV) antibody; or positive treponemal antibody for syphilis.\n20. The following cardiovascular and cerebrovascular diseases, including but not limited to:\n\n    QTc interval (corrected using Fridericia formula) average value: males QTc \\&gt; 450 ms, females QTc \\&gt; 470 ms.\n\n    Echocardiogram showing left ventricular ejection fraction (LVEF) \\&lt; 50% or below the lower limit of normal (whichever is higher).\n\n    History of heart failure of class III-IV (NYHA classification). Myocardial infarction, unstable angina, poorly controlled arrhythmia, cerebrovascular accident or transient ischemic attack occurred within 6 months before the first administration;\n21. Mental illness that may affect compliance with the test requirements: unstable mental illness, cognitive dysfunction, dementia or alcohol dependence, etc.\n22. Other conditions that the researcher thinks are not suitable for participating in this study.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "SNUG01", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03828123", "title": "Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Bioinova, s.r.o.", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that targets motor neurons. Prognosis is invariably fatal within 3-5 years since manifestation of the disease. Despite improved understanding of the mechanisms underlying ALS, the treatment remains essentially only supportive and focused on symptoms relief. Over the past few years, stem cell research has expanded greatly as a tool for developing new therapies to treat incurable diseases. Stem cell therapy has been shown as promising in several animal ALS models and human clinical trials.", "interventions": [{"type": "BIOLOGICAL", "name": "Suspension of human autologous MSC 3P in 1.5 ml"}], "start_date": "2012-01", "url": "https://clinicaltrials.gov/study/NCT03828123", "target_entities": ["neuroinflammation"], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. established diagnosis of definite ALS according to El Escorial criteria\n2. riluzole naive or stable dose for at least 2 months,\n3. life expectancy more than 2 years\n4. patients able to provide written informed consent.\n\nExclusion Criteria:\n\n1. FVC less than 70%\n2. in case of primary bulbar paralysis less than 15 points on Norris bulbar scale,\n3. less than 15 points on Norris spinal scale,\n4. pregnancy, breastfeeding\n5. coagulopathy,\n6. skin infection at the site of bone marrow aspiration or application of the cell product,\n7. gastrostomy,\n8. any significant medical condition that would compromise the safety of the patient (e.g. recent myocardial infarction, congestive heart failure, renal failure, liver failure, cancer, systemic infection, recurrent thromboembolic disease .....),\n9. alcohol or drug abuse\n10. cancer.\n11. women of childbearing potential not using effective contraception (established oral contraception, intrauterine device, ligation of the uterine tube) including proven contraceptive measures taken by their sexual partners\n12. fertile men not using proven contraceptive measures including effective contraception of their partner (established oral contraception, intrauterine device, ligation of the uterine tube)", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Autologous multipotent mesenchymal stromal cells (MSCs)", "targeting_mechanism": "MSCs reduce neuroinflammation and provide neuroprotection in ALS through modulation of immune responses and inflammatory signaling in the central nervous system.", "targeting_mechanism_pmid": "27938483", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00326625", "title": "Clinical Trial of Glatiramer Acetate in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Teva Pharmaceutical Industries, Ltd.", "summary": "Teva is developing 40 mg/ml Glatiramer Acetate (GA) Injection , administered once daily under the skin, for the treatment of ALS. The study drug is a higher dose formulation of Copaxone\u00ae (20 mg/ml GA), a marketed medication, approved for the treatment of relapsing-remitting multiple sclerosis. GA is an immunomodulating drug that has anti inflammatory and neuroprotective properties, which are believed to be of therapeutic value in ALS. The study treatment duration is 1 year (52 weeks).", "interventions": [{"type": "DRUG", "name": "40 mg glatiramer acetate"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2006-07-27", "url": "https://clinicaltrials.gov/study/NCT00326625", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Teva Benelux", "city": "Haarlem", "state": "", "country": "Belgium", "status": "", "lat": null, "lon": null}, {"facility": "Teva Benelux", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Teva France", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Teva Germany", "city": "M\u00f6rfelden-Walldorf", "state": "", "country": "Germany", "status": "", "lat": 49.99472, "lon": 8.58361}, {"facility": "Teva Israel", "city": "Tel Aviv", "state": "", "country": "Israel", "status": "", "lat": 32.08088, "lon": 34.78057}, {"facility": "Teva Italy", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Teva UK", "city": "Aylesbury", "state": "", "country": "United Kingdom", "status": "", "lat": 51.81665, "lon": -0.81458}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Diagnosis of definite or probable ALS in accordance with the El-Escorial criteria.\n2. Subject has experienced his/her first ALS symptoms within 3 years prior to the screening visit.\n3. Slow VC test equal or greater than 70% of the predicted value.\n4. The sum of the 3 respiratory items on the ALSFRS-R must total at least 10 points.\n5. Stable dose of riluzole for at least 8 weeks prior to screening.\n6. Age - 18-70 (inclusive).\n\nExclusion Criteria:\n\n1. The use of invasive or non-invasive ventilation.\n2. Subject having undergone gastrostomy.\n3. Subject with any clinically significant or unstable medical condition.\n4. Subjects participating in any other clinical trial (within 12 weeks prior to screening and thereafter).\n5. Additional criteria per protocol.", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Glatiramer acetate", "targeting_mechanism": "Glatiramer acetate is an immunomodulating drug that reduces inflammation and provides neuroprotection through modulation of immune responses in the CNS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "relapsing-remitting multiple sclerosis", "repurposed_from_pmid": "28872464"}}
{"nct_id": "NCT01091142", "title": "Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuraltus Pharmaceuticals, Inc.", "summary": "Primary objectives: To assess the safety and tolerability of ascending doses of NP001 compared to placebo in subjects with ALS.\n\nSecondary objective: To explore the effects of NP001 on biomarkers potentially relevant to ALS.", "interventions": [{"type": "DRUG", "name": "NP001"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2010-07", "url": "https://clinicaltrials.gov/study/NCT01091142", "target_entities": ["neuroinflammation"], "locations": [{"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Kansas Medical Center - Landon Center on Aging", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Department of Neurology; University of Kentucky Medical Center", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Males and females, age 21 years - 75 years\n* Subjects must be in generally sound health as appropriate for their ALS diagnosis.\n* Subjects must have a clinical diagnosis of laboratory-supported probable or definite ALS, according to modified El Escorial criteria.\n* Subjects receiving riluzole must be on a stable dose for at least 30 days prior to enrollment.\n* Women of childbearing age must be non-lactating and surgically sterile or using an effective method of birth control and have a negative pregnancy test prior to dosing with study medication.\n* Subjects must understand the study and be willing to adhere to protocol requirements as evidenced by provision of written informed consent.\n* Subject must be willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).\n* Subjects must be willing to have an intravenous infusion.\n* Subjects must have suitable veins for IV access as determined by examination.\n\nExclusion Criteria:\n\n* Subjects should not require nor are expected to require life sustaining interventions for the next six months or longer. (e.g. invasive ventilation).\n* Subjects must not have:\n\n  * presence of a tracheotomy or invasive ventilation. Nocturnal non-invasive ventilation system (e.g. C-PAP) is allowed.\n  * a diagnosis of neurologic disease known to mimic the muscle atrophy or weakness seen in ALS including MS, muscular dystrophy, spinal stenosis, peripheral neuropathy, inherited neuropathies or neuromuscular diseases, foramen magnum or brainstem tumor, or toxic conditions.\n  * an active pulmonary disease under treatment including uncontrolled asthma, chronic obstructive pulmonary disease (pneumonia, bronchitis, etc.), pulmonary fibrosis, pulmonary infection in the last 2 months, or history of aspiration that may expose the subject to increased risk by participating in this trial as determined by the Investigator.\n  * a history of unstable medical illness in the 3 months prior to screening including any emergent hospitalizations.\n  * renal disease based on screening estimated creatinine clearance (eCcr) \\< 50 mL/minute (Cockcroft Gault estimate using ideal body weight) where:\n  * evidence of elevated alanine aminotransferase greater than 3 times the upper limit of normal.\n  * evidence of anemia, thrombocytopenia, or neutropenia (screening hematocrit \\<33%, platelet count \\< lower limit of normal for the site laboratory, or neutrophil count less than 1,500/mm3).\n  * any condition that requires periodic red blood cell transfusions, erythropoietin or any blood dyscrasias undergoing active treatment in the past year.\n  * clinical laboratory parameters that are clinically significant in the opinion of the Investigator.\n  * systolic blood pressure in excess of 160 mmHg nor less than 100 mmHG or a diastolic blood pressure above 98 mmHg.\n  * a history of G6PD deficiency (Glucose-6-phosphate dehydrogenase deficiency) determined by subject report.\n  * a current history of hepatitis or HIV determined by subject report.\n* Subjects must not be using systemic immunosuppressants including steroids and chemotherapeutic agents. Inhaled steroids, eye drops and local topical use are permitted with concurrence of Medical Monitor.\n* Subjects must not have a hematologic disorder such as autoimmune anemia, or hemolytic anemia of any type including paroxysmal nocturnal hemoglobinuria or myoglobinuria.\n* Subjects must not have a history of unexplained jaundice determined by subject report.\n* Subjects must not have received IV Immunoglobulin (IG) within 30 days of the planned initial dose of study drug.\n* Subjects must not be participating in another drug study or have participated in a drug study within the last 30 days prior to enrollment. Observational trials with no intervention are acceptable provided permission for the other study Sponsor is obtained in writing.\n* Subjects must not have any other condition which in the Investigator's opinion would put the subject at risk by participating in this study.", "sex": "ALL", "min_age": "21 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NP001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03293069", "title": "Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Lille", "summary": "The alteration of iron metabolism is reported in animal models of amyotrophic lateral sclerosis (ALS) as well as in sporadic and genetic forms (SOD1 and C9orf72) of ALS. The high iron concentration of the brain, due to its high energy demand (high oxygen consumption), makes motor neurons particularly vulnerable to energy deficit and oxidative stress. Post-mortem examinations and MRI scans in patients with ALS have found signs of iron accumulation in the central motor tract; and a high level of serum ferritin, which is a marker of iron levels, is associated with a lower prognosis. In ALS mouse models, the use of iron chelators has demonstrated neuroprotection and increased life expectancy, suggesting that elimination of excess iron from the brain can prevent neuronal loss and, consequently, a slow progression of the disease. Conservative chelation of iron refers to a modality whereby much of the iron that binds to the chelator is redistributed in the body rather than exhausted. Using a chelator, deferiprone, with this feature, in a safety pilot study, a very good safety profile was observed. Deferiprone eliminated excess iron from brain regions, reduced oxidative damage and cell death associated with regional iron deposits with no apparent negative impact on the iron levels needed. Now, the efficacy of this new therapeutic modality of neuroprotection is being evaluated in a randomized, double-blind, placebo-controlled, multicenter study.", "interventions": [{"type": "DRUG", "name": "Deferiprone"}, {"type": "DRUG", "name": "Placebo Oral Tablet"}], "start_date": "2019-01-30", "url": "https://clinicaltrials.gov/study/NCT03293069", "target_entities": ["iron_metabolism", "oxidative_stress"], "locations": [{"facility": "Chr Angers", "city": "Angers", "state": "", "country": "France", "status": "", "lat": 47.47156, "lon": -0.55202}, {"facility": "Chru Brest", "city": "Brest", "state": "", "country": "France", "status": "", "lat": 48.39029, "lon": -4.48628}, {"facility": "Hopital Pierre Wertheimer - Hcl - Bron", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "Chu Cote de Nacre - Caen", "city": "Caen", "state": "", "country": "France", "status": "", "lat": 49.18585, "lon": -0.35912}, {"facility": "Chu de Clermont-Ferrand", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "", "lat": 45.77969, "lon": 3.08682}, {"facility": "H\u00f4pital Roger Salengro, CHU", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "C H U Dupuytren Limoges", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Aphm Hopital La Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Chu de Nancy", "city": "Nancy", "state": "", "country": "France", "status": "", "lat": 48.68439, "lon": 6.18496}, {"facility": "Chu de Nice Hopital Pasteur", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Hu Pitie Salpetriere Aphp", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Hopital de Hautepierre", "city": "Strasbourg", "state": "", "country": "France", "status": "", "lat": 48.58392, "lon": 7.74553}, {"facility": "Chu de Bordeaux - Talence", "city": "Talence", "state": "", "country": "France", "status": "", "lat": 44.80849, "lon": -0.58915}, {"facility": "Chu Toulouse", "city": "Toulouse", "state": "", "country": "France", "status": "", "lat": 43.60426, "lon": 1.44367}, {"facility": "Chu de Tours", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Categorized as; possible, laboratory supported probable, probable, or definite ALS (revised El Escorial criteria)\n* Spinal and bulbar forms of ALS\n* Duration of the disease of less than 18 months since the first symptoms of motor deficit or amyotrophy (isolated cramps or fasciculation will not be considered).\n* Duration of the disease of less than 6 months since the diagnosis\n* An upright slow vital capacity \u2265 75% of the predicted value for age, height, and sex or at least one test on inspiratory pressure \u2265 60% of the predicted value for age, height, and sex which could be either maximal inspiratory pressure or a SNIFF test. (The best predictive test is sniff test but sometime patients are not able to do it.) (In case of a limit abnormal value for one of them, it will be recommended that patient re-assessment occurs three months later).\n* A mild functional handicap score for ALSFRS-R \u226536\n* An upright slow vital capacity \\> 70% of the predicted value for age, height, and sex and\n* Able to swallow (required for oral treatment)\n* Patients weight included between 40 kg and 130 kg\n* If the patient is under riluzole, it has to be for at least 1 month before inclusion with stable dose (to rule out the principal risk of hepatitis)\n\nExclusion Criteria:\n\n* Patients with high frequency of comorbidity or vital risks that may reasonably impair life expectancy\n* Progressing axis I psychiatric disorders (psychosis, hallucinations, substance addiction, bipolar disorder, severe depression, suicidal ideation), in accordance with the Diagnostic and Statistical Manual of Mental Disorders. Before entry into the study, exclusion or stabilization of conditions must occur for patients suffering from mild or moderate depressive episodes (not in remission) or severe and uncontrolled anxiety.\n* Dementia according to the Diagnostic and Statistical Manual of Mental Disorders\n* Exposure to any other experimental drug up to 30 days before day 1\n* Due to the risk of agranulocytosis (estimated at 2%) caused by the Investigational Medicinal Products (IMPs) and the unknown mechanism by which this agranulocytosis is induced, combining Deferiprone with other medicinal products known to cause agranulocytosis (as described in the IB) will not be allowed. Such medicinal products include clozapine as well as some NSAIDs (e.g. Phenylbutazone or Metamizole), antithyroid agents, sulfonamide antibiotics or methotrexate.\n* A history of relapsing neutropenia\n* Patients with agranulocytosis or with a history of agranulocytosis.\n* Hypersensitivity to Deferiprone\n* Patients with anaemia (regardless of latter aetiology) or a history of another haematological disease. Haemochromatosis is not an exclusion criterion if controlled.\n* Pregnant or breastfeeding women or women of childbearing potential not taking highly effective contraception.\n* Kidney or liver failure.\n* Inability to provide informed consent.\n* Participation in another clinical trial within 1 month prior to inclusion in the study\n* Patients under trusteeship", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Deferiprone", "targeting_mechanism": "Deferiprone is an iron chelator that reduces iron accumulation and associated oxidative stress in motor neurons, protecting against ferroptosis and iron-mediated neurodegeneration.", "targeting_mechanism_pmid": "30266679", "animal_results": "Iron accumulation and alteration of iron metabolism is reported in animal models of amyotrophic lateral sclerosis (ALS) including SOD1 and other genetic forms of ALS.", "animal_results_pmid": "30266679", "repurposed_from": "iron overload disorders", "repurposed_from_pmid": "30266679"}}
{"nct_id": "NCT07698496", "title": "Evaluation of the Feasibility and Efficacy of a Chronic Brain-computer Interface for Speech Rehabilitation in Patients With Locked-in-syndrome (LIS)", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Grenoble", "summary": "Eighteen million people worldwide are affected by speech disorders. Locked-in syndrome (LIS) represents the most extreme form of communication disability resulting from motor impairment. In France, the Association du Locked-In Syndrome (ALIS) reports approximately 500 individuals with LIS, most of whom live at home. The quality of life of people with LIS depends strongly on their ability to communicate, and speech synthesis is the mode of communication restoration most desired by individuals with LIS. Several surveys have shown that improving communication abilities in these individuals leads to a significant improvement in their quality of life as well as that of their caregivers.\n\nNatural speech allows the production of an average of 150 words per minute. Non-invasive communication methods, whether based on residual motor function (eye-blink code) or on brain-machine or brain-computer interfaces (Brain-Computer Interface, BCI) using scalp electroencephalographic (EEG) signals, involve a high cognitive load and have low efficiency (spelling only a few letters per minute). Invasive BCIs for speech rehabilitation aim to overcome the limitations of non-invasive devices (cognitive overload and slow speech rate). The intention to act (the act of speaking) is predicted by an algorithm based on the direct decoding of neuronal activity from the sensorimotor cortex (the area where articulatory muscles are represented).\n\nTo date, studies testing speech rehabilitation BCIs in humans remain rare. A subdural electrocorticographic (ECoG) invasive BCI enabled speech decoding (words and sentences from a limited repertoire) for chronic use (2 years). Real-time control of an on-screen cursor allowing spelling of up to 90 letters per minute (equivalent to text messaging) has also been achieved using an intracortical invasive BCI (Utah Array). Very recently, up to 60 words per minute were produced using an intracortical invasive BCI (Utah Array) implanted in the ventral premotor cortex, although with a connector potentially contaminated by acoustic audio feedback. Furthermore, these devices still rely on transcutaneous connectors, which may be sources of infection and prevent routine daily-life use.\n\nIn summary, there is currently no fully implantable, wireless invasive \"speech BCI\" with real-time speech synthesis suitable for long-term home use. The present study will use an intracranial extradural invasive BCI combined with a speech synthesizer, with the aim of developing a communication tool suitable for everyday use. More specifically, the SpeechBCI protocol will propose two complementary BCI approaches in the same subject: a speech BCI (primary objective, BCI\\_PAROLE device) and a cursor BCI (secondary objective). Both BCIs will use the WIMAGINE intracranial epidural system, enabling ECoG signal acquisition with a very limited risk of infection and brain injury and providing signals that are more stable over time compared with intracortical or subdural ECoG devices that retain transcutaneous connectors. These systems will allow long-term and ecological use, as the WIMAGINE implant is wireless and offers excellent long-term signal stability. The WIMAGINE implant has already been successfully tested for controlling an exoskeleton in a tetraplegic subject (operational for over 6 years) and very recently for controlling walking in real-life conditions via a spinal cord stimulator in a paraplegic individual.\n\nThe BCI\\_PAROLE device will integrate a speech synthesizer providing real-time auditory feedback to the speaker. The BCI-CURSEUR device will allow the subject to control an on-screen cursor to access various communication functionalities (web access, emails, chats, etc.). This will provide a complementary communication solution to real-time speech production.\n\nThe hypothesis of this stydy is that the intention to speak (attempted speech) will be decoded by the BCI\\_PAROLE device in individuals with LIS because, as with limb paralysis, paralysis of articulatory and phonatory muscles does not prevent the corresponding cortical map from producing specific signals. Similarly, the BCI-CURSEUR device will decode the intention to move a cursor and perform actions on a computer screen.\n\nNeuronal electrical signals will be recorded bilaterally from the ventral motor cortex (representation of lips, cheeks, tongue, palate, and larynx-the vocal tract) and from the dorsal part of the motor cortex (larynx and hand, the latter for controlling a two-degree-of-freedom cursor), using a total of 128 electrodes (64 on each cerebral hemisphere). The implant will be optimally positioned over speech motor areas using preoperative functional imaging in order to optimize speech decoding by the BCI\\_PAROLE device.", "interventions": [{"type": "DEVICE", "name": "Chronic speech BCI"}], "start_date": "2026-10-10", "url": "https://clinicaltrials.gov/study/NCT07698496", "target_entities": [], "locations": [{"facility": "H\u00f4pital Raymond Poincar\u00e9 Garches", "city": "Garches", "state": "", "country": "France", "status": "", "lat": 48.84226, "lon": 2.18232}, {"facility": "Centre hospitalier Grenoble", "city": "Grenoble", "state": "", "country": "France", "status": "", "lat": 45.17869, "lon": 5.71479}, {"facility": "Centre hospitalier Nimes", "city": "N\u00eemes", "state": "", "country": "France", "status": "", "lat": 43.83665, "lon": 4.35788}, {"facility": "Centre hospitalier saint Etienne", "city": "Saint-Etienne", "state": "", "country": "France", "status": "", "lat": 45.43389, "lon": 4.39}], "contact_phone": "(33) 456520389", "contact_email": "lafontanell@chu-grenoble.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\na. Male or female between 18 and 65 years old b. French speaking person c. Person in locked in syndrome with a severe speech disorder following either: i. A subcortical stroke ii. Amyotrophic Lateral Sclerosis. d. Person with stable clinical state (esp. regarding respiratory state) e. Person able to read on a screen (sufficient eye movement control) f. Negative plasma pregnancy test for women of childbearing potential\\* g. Highly effective or at least acceptable\\*\\* contraception for women of childbearing potential.\n\nh. Persistence of localizing signals of language function proven by functional MRI.\n\ni. Person able to perform a simple BCI task with MEG j. Person with a neuropsychological profile evaluated by psychiatrist as compatible with sustain BCI training k. Informed consent to participate in the obtained using their usual means and code of communication (residual vocalizations, eye-blinking code, pictograms, eye tracking, alphabet chart, etc.), in the presence of their trusted person or curator who is accustomed to communicating with them using this means.\n\nl. Person affiliated to the French social security system or beneficiary of such a system\n\nExclusion Criteria:\n\nPatients with any of the following criteria cannot be included in this investigation:\n\n1. Severe cognitive disorders assessed after a neuropsychological evaluation\n2. Deafness\n3. Continuous assisted ventilation\n4. Contraindication to intracranial surgery\n5. Contra-indication to MRI (1.5T), CT-scan or their related contrast agent injection or MEG examinations.\n6. Anatomical brain MRI showing structural abnormalities in the cortical areas of language\n7. Functional impairment of language cortical areas on MRI\n8. Auditory evoked potential showing hearing impairment\n9. Person with a skull shape incompatible with one WIMAGINE implant on each hemisphere\n10. Persons referred to in Articles L1125-7 of the Public Health Code and article 64 of MDR(corresponding to all protected persons: pregnant women, women in labor, breastfeeding mothers, minors, persons deprived of liberty by judicial or administrative decision.", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Chronic brain-computer interface (BCI)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05951556", "title": "Telehealth Implementation of Brain-Computer Interface", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shirley Ryan AbilityLab", "summary": "Determine if Telehealth intervention can allow/empower a caregiver (who is untrained) to effectively implement and utilize a Brain-Computer Interface for communication with a participant who is \"Locked in\" following progression of Amyotrophic Lateral Sclerosis and other conditions.", "interventions": [{"type": "DEVICE", "name": "Use of BCI"}], "start_date": "2021-09-30", "url": "https://clinicaltrials.gov/study/NCT05951556", "target_entities": [], "locations": [{"facility": "ShirleyRyan AbilityLab", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "RECRUITING", "lat": 41.85003, "lon": -87.65005}], "contact_phone": "312 238 2997", "contact_email": "ehitchcock@sralab.org", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis leading to severe communication impairment\n\nExclusion Criteria:\n\n\\-", "sex": "ALL", "min_age": "16 Years", "max_age": "100 Years", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Brain-computer interface (BCI) enables communication by detecting brain signals and transforming them into useful commands to compensate for motor neuron loss.", "targeting_mechanism_pmid": "32034787", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04768972", "title": "FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)", "phase": "PHASE3", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ionis Pharmaceuticals, Inc.", "summary": "The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).", "interventions": [{"type": "DRUG", "name": "ION363"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-06-14", "url": "https://clinicaltrials.gov/study/NCT04768972", "target_entities": ["FUS"], "locations": [{"facility": "University of California San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Stanford University Medical Center", "city": "Palo Alto", "state": "California", "country": "United States", "status": "", "lat": 37.44188, "lon": -122.14302}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "The Ohio State University Wexner Medical Center", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "UZ Leuven", "city": "Leuven", "state": "VL-Brabant", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "PSEG Centro de Pesquisa Clinica S.A.", "city": "S\u00e3o Paulo", "state": "", "country": "Brazil", "status": "", "lat": -23.5475, "lon": -46.63611}, {"facility": "Montreal Neurological Institute", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Universitaetsmedizin Rostock", "city": "Rostock", "state": "", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Universit\u00e4tsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "St. James Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Citta della Salute e della Scienza di Torino - Ospedale le Molinette", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Toho University Omori Medical Center", "city": "Tokyo", "state": "", "country": "Japan", "status": "", "lat": 35.6895, "lon": 139.69171}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Linden sp\u00f3lka z ograniczona odpowiedzialnoscia sp\u00f3lka komandytow", "city": "Krakow", "state": "", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}, {"facility": "Seoul National University Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Hanyang University Seoul Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Hospital Universitari de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "University Hospital of Umea", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "Kantonsspital St. Gallen", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "Taipei Veterans General Hospital (VGHTP)", "city": "Taipei", "state": "", "country": "Taiwan", "status": "", "lat": 25.05306, "lon": 121.52639}, {"facility": "King's College Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria for Part 1:\n\n1. Participants must be \u226510 years of age at the time of informed consent and have signs or symptoms consistent with an ALS disease (in the opinion of the Investigator).\n2. Genetic mutation in FUS confirmed by a testing laboratory that is Clinical Laboratory Improvement Amendments (CLIA) certified and European Conformity (CE)-marked, or equivalent. Mutations must be reviewed and approved by a variant classification committee.\n3. Upright (sitting position) slow vital capacity (SVC) is \u2265 50% of predicted value (as adjusted for sex, age, and height) OR if SVC is \\< 50% of predicted value, must be 10 to 30 years of age (inclusive) at the time of informed consent AND had ALS symptom onset within 12 months before the time of informed consent.\n4. Participants taking edaravone, riluzole, Relyvrio (sodium phenylbutyrate/taurursodiol combination, called Albrioza in Canada), sodium phenylbutyrate, or tauroursodeoxycholic acid (TUDCA, also known as taurursodiol or urosodiol) must be on a stable dose for \u2265 28 days prior to Day 1, and willing to continue on that dose throughout the duration of the study, unless the Investigator determines that it should be discontinued for medical reasons, in which case it may not be restarted during the study.\n5. Stable concomitant medications and nutritional support for at least 1 month prior to Study Day 1. Concomitant medications or nutritional support that have not been stable for at least 1 month prior to Study Day 1 may be allowed in consultation with the Sponsor Medical Monitor or designee.\n6. Females must not be pregnant or lactating. Males and females must be willing to following protocol-specified contraception requirements, or be surgically sterile, or be post-menopausal (females).\n7. Has an informant/caregiver who, in the Investigator's judgment, has frequent and sufficient contact with the participant as to be able to provide accurate information about the participant's cognitive and functional abilities throughout the study. In addition, a patient who is \\< 18 years old must have a trial partner (parent, caregiver, or other) who is reliable, competent, at least 18 years of age, and willing to accompany the patient to all trial visits.\n\nInclusion Criteria for Part 2:\n\n1. Completed, or rescued from, Part 1, or\n2. Enrolled and received at least 1 dose of ION363 in the Investigator-initiated study program\n3. Patient meeting Criteria #1-2 is otherwise suitable for study participation, in the opinion of the Investigator\n\nExclusion Criteria for Part 1:\n\n1. Requiring permanent ventilation (\\> 22 hours of mechanical ventilation \\[invasive or noninvasive\\] per day for \\> 21 consecutive days) and/or tracheostomy.\n2. Any known genetic variant (other than those in the FUS gene) that is pathogenic or likely to be pathogenic for the ALS-frontotemporal dementia (FTD) spectrum of disease.\n3. Positive test result for:\n\n   1. Human immunodeficiency virus (HIV)\n   2. Hepatitis C (HCV), unless previously treated and has been serum/plasma HCV RNA negative for at least 6 months after the end of treatment\n   3. Hepatitis B (HBV) by HBV surface antigen test, unless currently on nucleotide/nucleoside analogue treatment\n4. Clinically significant abnormalities in medical history (e.g., previous acute coronary syndrome within 3 months before Screening, major surgery within 2 months before Screening) or physical examination.\n5. Uncontrolled hypertension (blood pressure \\[BP\\] \\> 160/100 millimeters of mercury \\[mm Hg\\]).\n6. Malignancy within 1 year before Screening, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated. Participants with a history of other malignancies that have been treated with curative intent and which have not recurred within 6 months may also be eligible per Investigator judgement.\n7. Obstructive hydrocephalus\n8. Known significant brain or spinal disease that would interfere with the lumbar puncture (LP) process, CSF circulation or safety assessment, including tumors or abnormalities by magnetic resonance imaging (MRI) or computed tomography, subarachnoid hemorrhage, suggestion of raised intracranial pressure on MRI or ophthalmic examination, spinal stenosis or curvature, Chiari malformation, syringomyelia, tethered spinal cord syndrome and connective tissue disorders such as Ehlers-Danlos syndrome and Marfan syndrome.\n9. Concurrent participation in any other interventional clinical study.\n10. Previous or current treatment with an oligonucleotide (including small interfering RNA \\[siRNA\\], tofersen). This exclusion criterion does not apply to COVID-19 vaccinations, which are allowed.\n11. Treatment with another investigational drug, biological agent, or device within 1 month before Screening, or 5 half-lives of investigational agent, whichever is longer.\n12. History of gene therapy or cell transplantation or any other experimental brain surgery.\n13. Anticipated need, in the opinion of the Investigator, for administration of any antiplatelet or anticoagulant medication that cannot be safely paused before and/or after an LP procedure according to local or institutional guidelines and/or Investigator determination after consultation with the appropriate treating physician. Low-dose aspirin (\u2264 100 mg/day, administered as monotherapy) is permitted and may be continued through the LP procedure.\n14. Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion or could interfere with the individual participating in or completing the study, in the opinion of the Investigator.", "sex": "ALL", "min_age": "10 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ION363", "targeting_mechanism": "ION363 targets FUS (fused in sarcoma) mutations that impair RNA binding and dynamic RNA interaction, causing aberrant phase separation and RNA processing defects in ALS.", "targeting_mechanism_pmid": "31630970", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05003921", "title": "Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Intrathecal Injection for ALS", "phase": "PHASE1", "status": "SUSPENDED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The Foundation for Orthopaedics and Regenerative Medicine", "summary": "This trial will study the safety and efficacy of intrathecal injection of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of amyotrophic lateral sclerosis", "interventions": [{"type": "BIOLOGICAL", "name": "AlloRx"}], "start_date": "2025-12", "url": "https://clinicaltrials.gov/study/NCT05003921", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Medical Surgical Associates Center", "city": "St John's", "state": "", "country": "Antigua and Barbuda", "status": "", "lat": 17.12096, "lon": -61.84329}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of Amyotrophic Lateral Sclerosis.\n* Understanding and willingness to sign a written informed consent document\n\nExclusion Criteria:\n\n* Active infection\n* Active cancer\n* Chronic multisystem organ failure\n* Pregnancy\n* Anticoagulation medicine use\n* Clinically significant Abnormalities on pre-treatment laboratory evaluation\n* Medical condition that would (based on the opinion of the investigator) compromise patient's safety.\n* Previous organ transplant\n* Hypersensitivity to sulfur\n* Continued drug abuse\n* Pre-menopausal women not using contraception", "sex": "ALL", "min_age": "", "max_age": "", "healthy_volunteers": false, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "AlloRx", "targeting_mechanism": "Cultured allogeneic adult umbilical cord-derived mesenchymal stem cells delivered intrathecally to target neuroinflammation and support motor neuron survival in ALS.", "targeting_mechanism_pmid": "29132389", "animal_results": "Bone marrow-derived mesenchymal stem cells (BM-MSCs) showed potential benefit in ALS rodent models with varying delivery methods, amounts of injected cells, timing of intervention, and differentiation states.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04098406", "title": "Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Clene Nanomedicine", "summary": "The objective of this trial was to assess the efficacy, safety, and PK/PD effects of CNM-Au8 as a disease-modifying agent for the treatment of ALS by utilizing electrophysiological measures to detect preservation of motor neuron function. The primary endpoint was the mean change in the average difference between active treatment and placebo from Baseline through Week 36 evaluated by electromyography.", "interventions": [{"type": "DRUG", "name": "CNM-Au8"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2019-12-19", "url": "https://clinicaltrials.gov/study/NCT04098406", "target_entities": ["oxidative_stress"], "locations": [{"facility": "University of Sydney Brain and Mind Centre", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}, {"facility": "Westmead Hospital", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to understand and give written informed consent.\n2. Male or female patients aged 30 years or greater (inclusive) and less than 80 years of age at the time of Screening.\n3. Patients whose conditions are defined as possible or probable or definite ALS per the diagnostic criteria by Awaji-Shima criteria as determined by a neurologist sub- specialising in ALS (e.g., the Principal Investigator by study site).\n4. For patients taking riluzole, stable dosing of riluzole over the prior 30-days from Screening.\n5. At the time of Screening either disease duration less than or equal to 24-months from symptom onset, or disease duration less than or equal to 12-months from diagnosis.\n6. Forced vital capacity (FVC) 60% of predicted value as adjusted for gender, height, and age at the Screening Visit.\n7. Patient who has established care with a neurologist at one of the specialized ALS clinics involved in the study and will maintain this clinical care throughout the study. If a patient is referred from a third party (neurologist or a State based ALS organization) they must be willing to transfer care to the neurologist participating in the study.\n\nFollowing completion of the 36-week randomized placebo controlled treatment period, interested participants must meet the following inclusion criteria to enroll in the open-label extension:\n\n1. Participants must have completed the randomized placebo controlled Treatment Period without compliance issues\n2. Able to understand and give written informed consent to participant in the open-label extension.\n3. If referred from a third party (neurologist or a State based ALS organization), participant agrees to maintain transfer of care to a neurologist participating in the study.\n\nExclusion Criteria:\n\n1. Patients will be excluded from the study if they meet any of the following criteria:\n2. At Screening patients who utilize, or in the Investigator's judgment will be imminently dependent upon:\n\n   1. Non-invasive ventilation \\> 22 hours per day, or\n   2. Tracheostomy Note: If the patient requires non-invasive ventilation postrandomisation, they will be allowed to continue in the study.\n3. Patients with Familial ALS (e.g., 2 or more family members with ALS or motor neuron disease)\n4. Patients with a history of carpal tunnel syndrome, polyneuropathy, or in the investigators judgement diseases that could induce polyneuropathy and interfere with electromyography (EMG) recordings.\n5. Patients with too severe atrophy of the Abductor Digiti Minimi (ADM), Abductor Pollicis Brevis (APB), Biceps Brachii (BB), or Tibialis Anterior (TA) muscles in the least clinically affected hand and leg, respectively, to allow for reliable EMG recordings.\n6. Patient with a history of significant other major medical conditions based on the Investigator's judgment.\n7. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.\n8. Patient with clinically significant abnormalities in haematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with study participation.\n9. Patients with clinically significant hepatic or renal dysfunction or clinical laboratory findings that would limit the interpretability of change in liver or kidney function, or those with low platelet counts (\\< 150 x 109 per liter) or eosinophilia (absolute eosinophil count of \u2265 500 eosinophils per microliter) at Screening.\n10. Patient participating in any other investigational drug trial or using investigational drug (within 12 weeks prior to screening and thereafter).\n11. Females who are pregnant or nursing or who plan to get pregnant during the course of this clinical trial or within 6 months of the end of this trial.\n12. Females of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control.\n13. Active inflammatory condition or autoimmune disorder.\n14. Positive screen for drugs of abuse.\n15. History of gold allergy.\n16. Patient is considered a suicide risk in the opinion of the Investigator, has previously made a suicide attempt, or is currently demonstrating active suicidal ideation. Subjects with intermittent passive suicidal ideation are not necessarily excluded based on the assessment of the Investigator.\n\nFollowing completion of the 36-week randomized placebo controlled treatment period, interested participants will be excluded from participating in the open-label extension phase if they meet any of the following criteria:\n\n1. Lack of treatment compliance during the randomized placebo controlled Treatment Period.\n2. Positive pregnancy test at the Week 36 visit, or, females who plan to get pregnant during the course of this extension or within 6 months of the end of this extension.\n3. Based on the investigator's judgment, patients who may have difficulty complying with the protocol and/or any study procedures.\n4. Patient with clinically significant abnormalities in hematology, blood chemistry, ECG, or physical examination identified during the W36 visit which according to Investigator may interfere with continued participation.\n5. Patients with clinically significant hepatic or renal dysfunction or clinical laboratory.\n\n   findings that would limit the interpretability of change in liver or kidney function, or those with low platelet counts (\\< 150 x 109 per liter) or eosinophilia (absolute eosinophil count of \u2265 500 eosinophils per microliter) at the Week 36 visit.\n6. Patient is considered a suicide risk in the opinion of the Investigator, has previously made a suicide attempt, or is currently demonstrating active suicidal ideation. Subjects with intermittent passive suicidal ideation are not necessarily excluded based on the assessment of the Investigator.", "sex": "ALL", "min_age": "30 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CNM-Au8", "targeting_mechanism": "CNM-Au8 acts as a nanocatalyst to reduce oxidative stress and preserve motor neuron function by modulating the antioxidant system and mitochondrial bioenergetics.", "targeting_mechanism_pmid": "34198557", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03481348", "title": "Pharyngeal Electrical Stimulation in Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Ulm", "summary": "During the course of ALS most patients develop swallowing deficits. In this pilot study we investigate if dysphagia in ALS can be improved by Pharyngeal Electrical Stimulation (PES). PES is Communaut\u00e9 Europ\u00e9enne (CE-) certificated and has been approved for treatment of neurological, oropharyngeal dysphagia. During PES, electrical stimuli are applied at the pharynx via a nasogastral tube with the aim of triggering reorganization processes in damaged brain structures. There is evidence of a positive effect of PES in Stroke and Multiple Sclerosis patients.", "interventions": [{"type": "DEVICE", "name": "Pharyngeal Electrical Stimulation"}], "start_date": "2018-02-01", "url": "https://clinicaltrials.gov/study/NCT03481348", "target_entities": [], "locations": [{"facility": "University of Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* possible, probable or definite ALS according to the revised El Escorial criteria (Brooks et al. 2000)\n* age \\>18 years\n* able to understand all information and to give full consent according to good clinical practice (GCP)\n* moderate ot severe dysphagia, defined by a mean value (all consistencies) of 4\n\nExclusion Criteria:\n\n* concurrent participation in another interventional trial\n* tracheostomy\n* severe psychiatric disorder or clinically manifest dementia\n* pulmonal or cardial disorder which constitutes a risk when inserting the tube into the pharynx\n* permanent cardiac pacemaker or defibrillator", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Pharyngeal Electrical Stimulation", "targeting_mechanism": "Electrical stimuli applied via nasogastric tube at the pharynx to trigger reorganization processes in damaged brain structures and improve swallowing function in ALS-related dysphagia.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03174938", "title": "The Swedish BioFINDER 2 Study", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Skane University Hospital", "summary": "The Swedish BioFINDER 2 study is a new study that will launch in 2017 and extends the previous cohorts of BioFINDER 1 study (www.biofinder.se). BioFINDER 1 is used e.g. to characterize the role of beta-amyloid pathology in early diagnosis of Alzheimer's disease (AD) using amyloid-PET (18F-Flutemetamol) and A\u03b2 analysis in cerebrospinal fluid samples. The BioFINDER 1 study has resulted in more than 40 publications during the last three years, many in high impact journals, and some the of the results have already had important implications for the diagnostic work-up patients with AD in the clinical routine practice.\n\nThe original BioFINDER 1 cohort started to include participants in 2008. Since then there has been a rapid development of biochemical and neuroimaging technologies which enable novel ways to the study biological processes involved in Alzheimer's disease in living people. There has also been a growing interest in the earliest stages of AD and other neurodegenerative diseases. With the advent of new tau-PET tracers there is now an opportunity to elucidate the role of tau pathology in the pathogenesis of AD and other tauopathies. The Swedish BioFINDER 2 study has been designed to complement the BioFINDER 1 study and to e.g. address issues regarding the role of tau pathology in different dementias and in preclinical stages of different dementia diseases. Further, the clinical assessments and MRI methods have been further optimized compared to BioFINDER 1. Detailed assessments of motor aspects and dual task performance, which is part of a sub-study named Motor-ACT: \"Motor aspects and activities in relation to cognitive decline and brain pathologies, has been added to further optimize assessment of motor function.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "Flutemetamol F18 Injection"}, {"type": "DIAGNOSTIC_TEST", "name": "[18F]-RO6958948"}, {"type": "DIAGNOSTIC_TEST", "name": "Elecsys (Roche) Abeta42, Ttau and Ptau"}, {"type": "DIAGNOSTIC_TEST", "name": "Lumipulse (Fujirebio) Abeta42, Ttau and Ptau"}], "start_date": "2017-05-15", "url": "https://clinicaltrials.gov/study/NCT03174938", "target_entities": [], "locations": [{"facility": "Memory Clinic, Hospital of \u00c4ngelholm", "city": "\u00c4ngelholm", "state": "", "country": "Sweden", "status": "RECRUITING", "lat": 56.2428, "lon": 12.86219}, {"facility": "Memory Clinic, Sk\u00e5ne University Hospital", "city": "Malm\u00f6", "state": "", "country": "Sweden", "status": "RECRUITING", "lat": 55.60587, "lon": 13.00073}], "contact_phone": "+46 40 33 10 00", "contact_email": "erik.stomrud@med.lu.se", "eligibility": {"criteria": "COHORT A: Cognitively healthy younger individuals (40-65 years of age) INCLUSION CRITERIA\n\n* Age 40-65 years\n* Absence of cognitive symptoms as assessed by a physician with special interest in cognitive disorders.\n* MMSE score 27-30 at screening visit.\n* Do not fulfill the criteria for MCI or any dementia according to DSM-V.\n* Speaks and understands Swedish to the extent that an interpreter is not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Significant neurological or psychiatric illness.\n* Refusing lumbar puncture, MRI or PET.\n\nCOHORT B: Cognitively healthy elderly individuals (66-100 years of age) INCLUSION CRITERIA\n\n* Age 66-100 years\n* Absence of cognitive symptoms as assessed by a physician with special interest in cognitive disorders.\n* MMSE score 26-30 at screening visit.\n* Do not fulfill the criteria for MCI or any dementia according to DSM-V.\n* Speaks and understands Swedish to the extent that an interpreter is not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Significant neurological or psychiatric illness.\n* Refusing lumbar puncture, MRI or PET.\n\nCOHORT C: Subjective cognitive decline and mild cognitive impairment INCLUSION CRITERIA\n\n* Age 40-100 years.\n* Referred to the memory clinics due to cognitive symptoms experienced by the patient and/or informant. These symptoms do not have to be memory complaints, but could also be executive, visuospatial, language, praxis, psychomotor or social cognitive complaints.\n* MMSE score of 24 - 30 points.\n* Do not fulfill the criteria for any dementia (major neurocognitive disorder) according to DSM-V.\n* The medical doctor (after clinical assessments, cognitive testing, CSF analyses and structural brain imaging) believes the cognitive complaints are caused by an incipient neurocognitive disorder of any sort. This is defined as any case fulfilling the criteria above (i.e. both SCD and MCI) with an abnormal CSF A\u03b242/40 ratio, which is strongly associated with brain A\u03b2 pathology and prodromal Alzheimer's disease. Further, cases with MCI (=minor neurocognitive impairment) due to either Parkinson's disease, Lewy body disease, vascular neurocognitive disorder or frontotemporal dementia (please see Appendix below for clinical criteria and references) can also be included.\n* Speaks and understands Swedish to the extent that an interpreter is not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Refusing lumbar puncture, MRI or PET.\n\nCOHORT D: Dementia due to Alzheimer's disease INCLUSION CRITERIA\n\n* Age 40-100 years.\n* Referred to the memory clinics due to cognitive symptoms experienced by the patient and/or informant. These symptoms do not have to be memory complaints, but could also be executive, visuospatial, language, praxis or psychomotor complaints.\n* MMSE score of 12-26 points.\n* Fulfill the criteria for dementia (major neurocognitive disorder) due to Alzheimer's disease (DSM-V).\n* Speaks and understands Swedish to the extent that an interpreter was not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Refusing lumbar puncture, MRI or PET.\n\nCOHORT E: Other dementias INCLUSION CRITERIA\n\n* Age 40-100 years.\n* Fulfill the criteria for dementia (major neurocognitive disorder) due to FTD, PDD, DLB or subcortical VaD alternatively the criteria for PD, PSP, MSA, CBS or ALS.\n* Speaks and understands Swedish to the extent that an interpreter was not necessary for the patient to fully understand the study information and cognitive tests.\n\nEXCLUSION CRITERIA\n\n* Significant unstable systemic illness or organ failure, such as terminal cancer, that makes it difficult to participate in the study.\n* Current significant alcohol or substance misuse.\n* Refusing lumbar puncture, MRI or PET.", "sex": "ALL", "min_age": "20 Years", "max_age": "100 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06877143", "title": "Hypercaloric PEG Nutrition in ALS to Sustain Energy Homeostasis", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Ulm", "summary": "Weight loss is a known negative prognostic factor in amyotrophic lateral sclerosis (ALS). Over the last years, various interventional studies targeting the energy deficit in ALS yielded promising results; however,it is still unclear which kind of nutrition or nutritional supplement is most beneficial. Moreover, there is lack of evidence regarding interventions in patients with a PEG in later disease stages.In a pilot study conducted in 2013, it was demonstrated that body weight can be stabilized in ALS by applying either a fat-rich or carbohydrate-rich high-caloric food supplement. In 2014, Wills et al. conducted a placebo-controlled randomized controlled pilot study, which indicated that a carbohydrate-rich, hypercaloric diet, consisting in 125% of estimated energy requirements as determined by indirect calorimetry, in patients fed via percutaneous endoscopic gastrostomy was safe and well tolerated. Moreover, these patients showed longer survival than patients fed with a fat-rich, hypercaloric diet or an isocaloric diet . Hypercaloric, high-carbohydrate diet also showed beneficial effects on body weight and Body Mass Index . Although these results were promising, the low number of patients (n=24) was a severe limiting factor of this study. The aim of this study is to investigate the effect of a hypercaloric PEG nutrition, consisting of 120% of estimated calorie requierements, compared to an isocaloric nutrition. Individual energy requirement is determined by performing indirect calorimetry and activity questionnaire. The investigators hypothsize, that a hypercaloric PEG nutrition slows down disease progression as measured by neurofilament light chains (NfL) in serum after 6 months compared to placebo. Power calculation relies on the results of the lipids and calories for ALS (LIPCAL-ALS) study which tested the effect of an oral high-caloric fatty nutritional supplement in ALS. The study revealed that NfL serum values declined significantly in the intervention group while remaining stable in the placebo group over the course of the study. Assuming a similar effect size, we calculated that 76 patients had to be included in the current trial.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "100% of individual calory requirement"}, {"type": "DIETARY_SUPPLEMENT", "name": "120% of individual calory requirement"}], "start_date": "2025-03-01", "url": "https://clinicaltrials.gov/study/NCT06877143", "target_entities": ["energy_metabolism"], "locations": [{"facility": "Ulm Universita, Department of Neurology", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "RECRUITING", "lat": 48.39841, "lon": 9.99155}], "contact_phone": "0049 731 177 5374", "contact_email": "christine.herrmann@uni-ulm.de", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Possible, probable (clinically or laboratory) or definite ALS according to the revised version of the El Escorial World Federation of Neurology criteria 1\n* Loss of ALS functional rating scale revised (ALSFRS-R) of \u2265 0.33 points per month since onset (first paresis) based on the formula: (48 - Score at Screening Visit) / (Months between Onset and Screening Visit)\n* Nutrition via PEG\n* Age \u226518 years\n* Intake of a stable dose of riluzole for at least 4 weeks, or no riluzole\n* Capable of thoroughly understanding all information given and giving full informed consent according to GCP\n\nExclusion Criteria:\n\n* Previous participation in another interventional study within the preceding 4 weeks\n* Absence of adequate social support and cooperation, or personal motivation (in the judgment of the investigator) to complete the study satisfactorily\n* Pregnancy or breast-feeding females\n* Evidence of a major psychiatric disorder or clinically evident dementia", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02891629", "title": "Safety and Feasibility of the EyeControl Device", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Eyefree Assisting Communication Ltd", "summary": "The EyeControl device is an eye movement-based communication device in the form of wearable glasses with connected infrared cam-era that tracks the pupil and translates blinks and movements into commands.This study is aimed to demonstrate the safety and feasibility of the EyeControl device in healthy volunteers, and ALS patients in early stages.", "interventions": [{"type": "DEVICE", "name": "EyeControl device"}], "start_date": "2016-09", "url": "https://clinicaltrials.gov/study/NCT02891629", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Subjects 18 to 65 years old\n2. Subject with understandable speaking communication\n3. Subject fluent in Hebrew (speech and writing skills)\n\n   Additional inclusion criteria for Stage 2 of the study:\n4. Subjects with early stage ALS diagnosis - whose speech capability is unaffected\n\nExclusion Criteria:\n\n1. Subjects with glasses or contact lenses\n2. Subjects with eye conditions such as Ptosis, Strabismus And Crossed Eyes\n3. Medical history of epilepsy\n4. Subjects who according to investigator's judgement, unable to comply with the requirements of this protocol\n5. Pregnant or lactating women", "sex": "ALL", "min_age": "18 Years", "max_age": "65 Years", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "EyeControl device", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04414345", "title": "HEALEY ALS Platform Trial - Regimen C CNM-Au8", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.\n\nRegimen C will evaluate the safety and efficacy of a single study drug, CNM-Au8, in participants with ALS.", "interventions": [{"type": "DRUG", "name": "CNM-Au8"}, {"type": "DRUG", "name": "Matching Placebo"}], "start_date": "2020-07-30", "url": "https://clinicaltrials.gov/study/NCT04414345", "target_entities": ["cnm_au8"], "locations": [{"facility": "Healey Center for ALS at Mass General", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\nExclusion Criteria:\n\n* The following exclusion criterion is in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).\n\n  1. History of allergy to gold, gold salts, or colloidal gold preparations.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CNM-Au8", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01994109", "title": "Efficacy and Safety Study of MYOBLOC\u00ae in the Treatment of Sialorrhea in Adult Subjects", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Solstice Neurosciences, LLC, a subsidiary of US WorldMeds, LLC", "summary": "This study will evaluate the efficacy and safety of MYOBLOC in the treatment of Sialorrhea (drooling), which can be a symptom of many disease conditions. MYOBLOC will be injected directly into the salivary glands. MYOBLOC has been shown in previous trials to safely decrease saliva production, thereby demonstrating its potential as a safe and effective treatment for troublesome sialorrhea.", "interventions": [{"type": "DRUG", "name": "MYOBLOC"}, {"type": "OTHER", "name": "PLACEBO"}], "start_date": "2013-11", "url": "https://clinicaltrials.gov/study/NCT01994109", "target_entities": ["botulinum_toxin"], "locations": [{"facility": "", "city": "Loma Linda", "state": "California", "country": "United States", "status": "", "lat": 34.04835, "lon": -117.26115}, {"facility": "", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "", "city": "National City", "state": "California", "country": "United States", "status": "", "lat": 32.67811, "lon": -117.0992}, {"facility": "", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "", "city": "Boca Raton", "state": "Florida", "country": "United States", "status": "", "lat": 26.35869, "lon": -80.0831}, {"facility": "", "city": "Port Charlotte", "state": "Florida", "country": "United States", "status": "", "lat": 26.97617, "lon": -82.09064}, {"facility": "", "city": "Carmel", "state": "Indiana", "country": "United States", "status": "", "lat": 39.97837, "lon": -86.11804}, {"facility": "", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "", "city": "Elkridge", "state": "Maryland", "country": "United States", "status": "", "lat": 39.21261, "lon": -76.71358}, {"facility": "", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "", "city": "Edison", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.51872, "lon": -74.4121}, {"facility": "", "city": "Albany", "state": "New York", "country": "United States", "status": "", "lat": 42.65258, "lon": -73.75623}, {"facility": "", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "", "city": "Cincinnati", "state": "Ohio", "country": "United States", "status": "", "lat": 39.12711, "lon": -84.51439}, {"facility": "", "city": "Tulsa", "state": "Oklahoma", "country": "United States", "status": "", "lat": 36.15398, "lon": -95.99277}, {"facility": "", "city": "Port Royal", "state": "South Carolina", "country": "United States", "status": "", "lat": 32.37896, "lon": -80.69265}, {"facility": "", "city": "Cordova", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.15565, "lon": -89.7762}, {"facility": "", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "", "city": "Kirkland", "state": "Washington", "country": "United States", "status": "", "lat": 47.68149, "lon": -122.20874}, {"facility": "", "city": "Tacoma", "state": "Washington", "country": "United States", "status": "", "lat": 47.25288, "lon": -122.44429}, {"facility": "", "city": "Irkutsk", "state": "Irkutsk Oblast", "country": "Russia", "status": "", "lat": 52.29566, "lon": 104.29076}, {"facility": "", "city": "Vsevolozhsk", "state": "Leningradskaya Oblast'", "country": "Russia", "status": "", "lat": 60.01512, "lon": 30.67314}, {"facility": "", "city": "Saint Petersburg", "state": "Petrodvorets", "country": "Russia", "status": "", "lat": 59.93863, "lon": 30.31413}, {"facility": "", "city": "Krasnoyarsk", "state": "", "country": "Russia", "status": "", "lat": 56.03742, "lon": 92.93136}, {"facility": "", "city": "Dnipropetrovsk", "state": "", "country": "Ukraine", "status": "", "lat": 48.46664, "lon": 35.04066}, {"facility": "", "city": "Ivano-Frankivsk", "state": "", "country": "Ukraine", "status": "", "lat": 48.92312, "lon": 24.71248}, {"facility": "", "city": "Kharkiv", "state": "", "country": "Ukraine", "status": "", "lat": 49.98177, "lon": 36.25475}, {"facility": "", "city": "Lviv", "state": "", "country": "Ukraine", "status": "", "lat": 49.83826, "lon": 24.02324}, {"facility": "", "city": "Rivne", "state": "", "country": "Ukraine", "status": "", "lat": 50.62036, "lon": 26.23695}, {"facility": "", "city": "Uzhhorod", "state": "", "country": "Ukraine", "status": "", "lat": 48.6242, "lon": 22.2947}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Seeking treatment for troublesome sialorrhea for at least 3 months that is occurring secondary to any disorder or related to any cause\n* Investigator sites will review entire list of inclusion criteria with potential subjects\n\nExclusion Criteria:\n\n* Any known prior exposure to botulinum toxin type B, or known adverse reaction or sensitivity to botulinum toxin type A, or known sensitivity to any of the MYOBLOC solution components.\n* Prior botulinum toxin treatment to the salivary glands at any time\n* Investigator sites will review entire list of exclusion criteria with potential subjects", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "MYOBLOC", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05923905", "title": "Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients", "phase": "PHASE4", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University Third Hospital", "summary": "This is a randomized double-blind controlled exploratory clinical study to evaluate the efficacy and safety of FB1006 in the treatment of amyotrophic lateral sclerosis (ALS) patients.", "interventions": [{"type": "DRUG", "name": "FB1006"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2023-01-18", "url": "https://clinicaltrials.gov/study/NCT05923905", "target_entities": ["fb1006"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "RECRUITING", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "+86-10-82265791", "contact_email": "scdoctor@qq.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. World Federation of Neurology modified El Ecorial criteria for diagnosis of patients with laboratory support probable, clinically probable, or definite sporadic and familial amyotrophic lateral sclerosis (ALS)\n2. Age 18 to 80 years old\n3. ALS duration no longer than 18 months(from day of onset)\n4. Patient 's ALSFRS-R total scored \u226527\uff0cEach single item is scored at least 2\uff08dyspnoea, orthopnea and respiratory insufficiency \u22653\uff09\n5. Forced vital capacity (FVC%) no less than 70% of predicted normal for gender, height and age\n6. According to brain function AI analysis in accordance with depressive EEG characteristics\n7. Women and men of childbearing potential should use medically acceptable contraception\n8. Voluntarily participate, and sign an informed consent form\n\nExclusion Criteria:\n\n1. Patients with dementia or severe neurological, psychiatric or systemic disease that is poorly controlled or may interfere with the conduct of the trial or the results of the trial\n2. Pregnant women and lactating women\n3. Suicide attempt or attempted suicide\n4. Combined with other neurological diseases similar to ALS symptoms, or affecting the evaluation of drug efficacy, such as cervical spondylotic myelopathy, lumbar spondylosis, dementia, etc.\n5. Patients with history of spinal surgery after ALS onset\n6. ALT or AST \\> 2 times ULN\uff0ccreatinine clearance \\< 60 mL/min/1.73m2 (MDRD)\n7. Patients who are allergic to the investigational product\n8. Having participated in other clinical studies within 3 months before randomization\n9. Patients that the investigator considers unsuitable for participation in the study", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "FB1006", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07413718", "title": "Inspiratory Muscle Training in Amiotrophyc Lateral Sclerosis", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Ramon Llull", "summary": "Amyotrophic Lateral Sclerosis (ALS) progressively damages the nerve cells responsible for voluntary muscle movement. Over time, this leads to weakness in different muscles such as those used for movement or breathing. Breathing problems are one of the main causes of complications and reduced survival in people with ALS. This happens because the inspiratory muscles-those that help draw air into the lungs-gradually lose strength.\n\nThe study has the aim to explore the benefits of training inspiratory muscles in ALS patients in order to maintain the setrength of these muscles for as long as possible and look the impact on respiratory function.", "interventions": [{"type": "DEVICE", "name": "Inspiratory muscle training"}, {"type": "DEVICE", "name": "Sham training"}], "start_date": "2025-09-01", "url": "https://clinicaltrials.gov/study/NCT07413718", "target_entities": ["respiratory_muscle_function"], "locations": [{"facility": "ADELA Gipuzkoa", "city": "Donostia / San Sebastian", "state": "Gipuzka", "country": "Spain", "status": "RECRUITING", "lat": 43.31283, "lon": -1.97499}], "contact_phone": "+34666580946", "contact_email": "jordi.gestos@gmail.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients diagnosed with spinal ALS\n* men and women\n* diagnosis date less than two years ago, according to the \"El Escorial\" criteria (Appendix 2)\n* PIM above the lower limit of normal\n* Preserved lung function (FVC \u2265 80%, FEV1 \u2265 80%, FEV1/FVC \u2265 80%) and normal values in supine position\n\nExclusion Criteria:\n\n* Patients with signs of respiratory muscle weakness (MIP and MEP below the LLN and abnormal decubitus tests19-21)\n* Nocturnal hypoventilation\n* Inability to perform the measurement tests\n* Inability to understand and perform the exercises\n* Any contraindication to the use of IMT. Severe psychiatric illness.", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00036413", "title": "A 12-week, Multicenter, Safety and Dose-ranging Study of 3 Oral Doses of TCH346 in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Novartis Pharmaceuticals", "summary": "This study is the first to be performed in Amyotrophic Lateral Sclerosis (ALS) patients with the novel compound TCH346. Its purpose is to evaluate the safety and clinical effects of 3 dose levels of TCH 346 compared to placebo in patients with a clinical diagnosis of laboratory-supported probable, probable or definite ALS. The study will require patients to visit the study center a total of at least 7 times over the course of up to 14 weeks. The study consists of 2 phases: A screening phase (up to 2 weeks) when patients will be evaluated for eligibility to participate in the study, and a double-blind treatment phase (12 weeks) when patients will receive daily doses of either TCH346 or placebo and will be evaluated for clinical effects. In addition, patients eligible to participate in this study will be required to have 3 magnetic resonance spectroscopic (MRS) scans. The MRS is a non-invasive, painless, \"brain scan\". The MRS will require traveling to a designated center in Montreal, Canada, which is very experienced in performing such MRS scans in ALS patients.", "interventions": [{"type": "DRUG", "name": "TCH346"}], "start_date": "2002-01", "url": "https://clinicaltrials.gov/study/NCT00036413", "target_entities": ["tch346"], "locations": [{"facility": "Neurological Institute", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "* clinical diagnosis of laboratory-supported probable, probable, or definite ALS;\n* have sporadic or familial ALS;\n* have shown ALS symptom onset for no more than 3 yrs., inclusive, prior to randomization;\n* FVC of \\>60%;\n* ability to tolerate MRS evaluation", "sex": "ALL", "min_age": "40 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "TCH346", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03214146", "title": "Safety/Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in ALS", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hanyang University Seoul Hospital", "summary": "The purpose of this study is to evaluate the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells(\"HYNRCS-Allo-ALS-02 inj\"), through intrathecal delivery for the repeated treatment after 6 months of first treatment in patients with amyotrophic lateral sclerosis(ALS).\n\nThis study is an open label, single-dose study to assess the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells(\"HYNRCS-Allo-ALS-02 inj\")", "interventions": [{"type": "BIOLOGICAL", "name": "HYNRCS-Allo inj"}], "start_date": "2017-02-01", "url": "https://clinicaltrials.gov/study/NCT03214146", "target_entities": ["stem_cell_therapy"], "locations": [{"facility": "Hanyang University Seoul Hospital, Cell Therapy Center for Neurologic Disorders", "city": "Seoul", "state": "Haengdang-dong, Seongdong-gu", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Patients between 25 and 80 years old\n* Patients diagnosed as 'Possible with lab-supported' or 'Possible' or 'Probable' or 'Definite' ALS according to the World Federation of Neurology El Escorial criteria\n* Patients whose duration of disease is within 5 years from the first diagnosis\n* Patients with ALSFRS-R score within 21 to 46 at screening\n* Patients who can visit to a hospital by walk personally or by protector's help\n* Patients who provide the written consent by oneself or his/her legal representative\n* Patients who has HLA-haplo matched Bone marrow donor\n\nExclusion Criteria:\n\n* Patients who doesn't appropriate to the diagnostic criteria of ALS\n* Patients who doesn't have HLA-haplo-matched bone marrow donor\n* Patients suspected of adverse effect after stem cell injection(patients suspected of malignant tumor, risk group of psychogenic shock, patients with serious hypertension)\n* Patients with ALSFRS-R score below 21 at screening\n* Patients performed Tracheostomy at screening\n* Patients with suspected 20% or less of Forced vital capacity(FVC) at screening\n* Patients who doesn't agree with written consent form by oneself of his/her legal representative\n* Patients who have taken any other drug for clinical trial within the past 3 months at screening entry\n* Patients with epilepsy\n* Patients with severe medical disease\n* Pregnant woman, lactating woman, female patients who has a pregnancy planning or who doesn't agree with adoption of contraception methods proper medically, male patients who doesn't agree with adoption of contraception methods proper to his partner during participating this study\n* Patients with hemorrhagic tendency at screening\n* Patients with a known history of hypersensitivity/allergy to penicillin and streptomycin\n* Patients with severe psychotic diseases (such as alzheimer, schizophrenia excepts slight cognitive dysfunction and secondary emotional disorder)", "sex": "ALL", "min_age": "25 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "HYNRCS-Allo inj", "targeting_mechanism": "Allogenic bone marrow derived stem cells delivered intrathecally to provide neuroprotection and promote motor neuron survival.", "targeting_mechanism_pmid": "31189354", "animal_results": "Preclinical stem cell studies performed in mouse and rat ALS models expressing mutant superoxide dismutase 1 demonstrated that stem cell types could be viable options for ALS therapy.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01835782", "title": "Determining the Safety of L-serine in ALS", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Phoenix Neurological Associates, LTD", "summary": "The purpose of this study is to determine the safety of L-Serine in subjects with Amyotrophic Lateral Sclerosis (ALS) at varied doses.", "interventions": [{"type": "DRUG", "name": "L-Serine"}], "start_date": "2013-01", "url": "https://clinicaltrials.gov/study/NCT01835782", "target_entities": ["serine_synthesis_pathway"], "locations": [{"facility": "Phoenix Neurological Associates", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 18-85\n2. Male or Female\n3. Clinically diagnosed with probable or definite ALS based on El Escorial criteria\n4. ALSFRS-R \\> 25\n5. Able to provide informed consent to and comply with all medical procedures\n\nExclusion Criteria:\n\n1. Outside age range of 18-85\n2. Subjects with forced vital capacity (FVC) below 60%\n3. Evidence of any motor neuron disease for over 3 years", "sex": "ALL", "min_age": "18 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "L-Serine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07290062", "title": "A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Insmed Gene Therapy LLC", "summary": "The primary objective of this dose-finding study is to evaluate the safety, tolerability and pharmacodynamics of single dose of INS1202 via IT administration in participants \u2265 18 to \\<80 years of age with ALS who carry superoxide dismutase type 1 (SOD1) mutations or harbor no known ALS-related genetic mutation.", "interventions": [{"type": "GENETIC", "name": "INS1202"}], "start_date": "2026-01-09", "url": "https://clinicaltrials.gov/study/NCT07290062", "target_entities": ["SOD1"], "locations": [{"facility": "USA004", "city": "La Jolla", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 32.84727, "lon": -117.2742}, {"facility": "USA002", "city": "Palo Alto", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.44188, "lon": -122.14302}, {"facility": "USA003", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.29038, "lon": -76.61219}, {"facility": "USA009", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.26259, "lon": -71.80229}, {"facility": "USA001", "city": "Columbia", "state": "Missouri", "country": "United States", "status": "RECRUITING", "lat": 38.95171, "lon": -92.33407}, {"facility": "USA007", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 39.96118, "lon": -82.99879}, {"facility": "USA006", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 39.95238, "lon": -75.16362}], "contact_phone": "1-844-446-7633", "contact_email": "medicalinformation@insmed.com", "eligibility": {"criteria": "Key Inclusion Criteria: -\n\n* Participant with body mass index (BMI) \u226518 kilograms per square meter (kg/m\\^2).\n* Participant with symptomatic ALS as diagnosed by Gold Coast diagnostic criteria.\n* Sporadic ALS cohorts: Negative testing for known monogenic mutations associated with familial ALS.\n* SOD1-ALS (Cohorts 2 and 3 only): Confirmed pathogenic SOD1 mutation, with negative testing for other genetic mutations associated with familial ALS.\n* Any polymorphism or mutation in the coding region will require additional review by the Sponsor to determine compatibility with the study intervention.\n* Baseline ALSFRS-R \u2265 24.\n* ALS disease duration \u2264 42 months.\n\nKey Exclusion Criteria: -\n\n* Previous treatment for ALS with cellular or gene therapies.\n* Any investigational medication or treatment (for ALS or other condition).\n\nNote: Other protocol-defined inclusion/exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "79 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "INS1202", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04468191", "title": "Fatigue in Patients With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cara Donohue", "summary": "Expiratory muscle strength training (EMST) is an emerging palliative intervention for prolonging pulmonary and swallow function in patients with amyotrophic lateral sclerosis (PALS), but it is unknown whether EMST may result in detrimental immediate to short-term fatigue because there is no way to measure fatigue non-invasively. This study will determine the immediate to short-term impact of EMST on objective respiratory and swallow function, whether subjective ratings of dyspnea and fatigue map to objective decompensation of respiratory and swallow function, and the ability to monitor fatigue of the respiratory and swallowing musculature non-invasively. Findings from this research study will provide preliminary evidence regarding optimal timing for PALS to complete EMST and will provide PALS and clinicians increased capabilities to monitor fatigue non-invasively.", "interventions": [{"type": "DEVICE", "name": "Experimental expiratory muscle strength training (EMST)"}, {"type": "DEVICE", "name": "Sham expiratory muscle strength training (EMST)"}], "start_date": "2021-02-10", "url": "https://clinicaltrials.gov/study/NCT04468191", "target_entities": [], "locations": [{"facility": "University of Pittsburgh Medical Center Presbyterian Hospital", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of ALS defined as possible, probable, or definite by a neurologist using the El Escorial criteria\n* FVC\\>65% predicted\n* adequate cognition as defined by a score of \\>10 on the ALS Cognitive Behavioral Screen\n* adequate labial seal for completing pulmonary function tests and expiratory muscle strength training (EMST)\n* on a regular/thin liquid diet\n* no allergies to barium\n* not oxygen-dependent\n* no tracheostomy/ mechanical ventilation\n* no history of other neurological or respiratory disorders\n* no history of smoking\n* no history of head and neck cancer or other major head/neck surgery or radiation therapy.\n\nExclusion Criteria:\n\n* FVC\\<65% predicted\n* inadequate cognition as defined by a score of \\<10 on the ALS Cognitive Behavioral Screen -inadequate labial seal for completing pulmonary function tests and expiratory muscle strength training (EMST)\n* not on a regular/thin liquid diet\n* allergies to barium\n* oxygen-dependent\n* presence of tracheostomy/dependent on mechanical ventilation\n* history of other neurological or respiratory disorders\n* history of smoking\n* history of head and neck cancer or other major head/neck surgery or radiation therapy.", "sex": "ALL", "min_age": "18 Years", "max_age": "100 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Expiratory muscle strength training (EMST)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05299372", "title": "Telemonitoring in NIV MND (OptNIVent)", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Liverpool University Hospitals NHS Foundation Trust", "summary": "This is a feasibility study of telemonitoring system for people with MND/ALS, who are on NIV, via a call centre operated by a local clinical commissioning group.", "interventions": [{"type": "OTHER", "name": "Telemonitoring via Careportal\u00ae"}], "start_date": "2022-04-25", "url": "https://clinicaltrials.gov/study/NCT05299372", "target_entities": [], "locations": [], "contact_phone": "0151529", "contact_email": "hikari.ando@nhs.net", "eligibility": {"criteria": "Inclusion Criteria (Patients):\n\n* Confirmed diagnosis of MND with respiratory muscle weakness\n* Adults who are capable of informed consent\n* Patients for whom we anticipate survival of 6 months or more\n* Able to communicate with an interviewer. Alternative communication methods will be used as required in order to mitigate communication difficulties.\n\nExclusion Criteria (Patients):\n\n* Patients who have declined NIV", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06392126", "title": "Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS", "phase": "PHASE1, PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "n-Lorem Foundation", "summary": "This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10", "interventions": [{"type": "DRUG", "name": "nL-CHCHD-001"}], "start_date": "2024-04-16", "url": "https://clinicaltrials.gov/study/NCT06392126", "target_entities": ["CHCHD10"], "locations": [{"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Informed consent/assent provided by the participant (when appropriate), and/or participant's parent(s) or legally authorized representative(s).\n* Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records.\n* Genetically confirmed neurological disorder.\n\nExclusion Criteria:\n\n* Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures.\n* Use of an investigational medication within less than 5 half-lives of the drug at enrollment", "sex": "MALE", "min_age": "48 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "nL-CHCHD-001", "targeting_mechanism": "Antisense oligonucleotide targeting CHCHD10 to modulate expression of the pathogenic protein variant", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01449578", "title": "Dexpramipexole SAD/MAD Study", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "This Phase 1 study will explore the safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.", "interventions": [{"type": "DRUG", "name": "Dexpramipexole"}, {"type": "DRUG", "name": "Dexpramipexole Placebo"}], "start_date": "2011-11", "url": "https://clinicaltrials.gov/study/NCT01449578", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Research Site", "city": "Overland Park", "state": "Kansas", "country": "United States", "status": "", "lat": 38.98223, "lon": -94.67079}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Must give written informed consent.\n* Adult males/females aged 18 to 55 years inclusive and between 19 and 30 kg/m2 body mass index (BMI), inclusive at screening.\n* Subjects who are healthy as determined by prestudy medical history, physical examination and 12-lead ECG.\n* Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment.\n* Normal systemic blood pressure defined as a systolic blood pressure of 90 to 140 mmHg and a diastolic blood pressure of 50 to 90 mmHg.\n\nExclusion Criteria:\n\n* History of cardiovascular disease (e.g., hypertension, arrhythmia, heart failure, Long QT Syndrome, or other conditions/diseases causing prolongation of the QT/QTc interval).\n* A prolongation of QT/QTc interval (e.g., repeated demonstration of a QT/QTc interval \\>450 ms before study treatment administration) at screening, admission or pre-dose on Day 1.\n* Any clinically important abnormalities in resting ECG that may interfere with the interpretation of QTc interval changes at screening, admission or pre-dose on Day 1.\n* Prior exposure to dexpramipexole.\n* Treatment with pramipexole or any dopamine agonist within 1 year.\n* Treatment with another investigational drug or approved therapy for investigational use within 30 days, or 5 half-lives (whichever is longer), or in follow up for any other drug, biologic, or device study.\n* Currently active infection or serious infection (e.g., pneumonia, septicemia) within the 2 months prior to Day -2 as determined by the Investigator.\n* Female subjects who are pregnant, currently breastfeeding, or attempting to conceive during the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "55 Years", "healthy_volunteers": true, "std_ages": ["ADULT"]}, "mechanism_summary": {"compound": "Dexpramipexole", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00140218", "title": "R(+) Pramipexole in Early Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "summary": "The hypothesis of this study is that treatment with R(+) pramipexole at 30 mg/day will alter the slope of decline in ALS functional rating scale over the course of 6 months. ALS patients at an early stage of disease will be observed for 3 months after enrollment and then treated with drug for 6 months.", "interventions": [{"type": "DRUG", "name": "R(+) pramipexole dihydrochloride monohydrate"}], "start_date": "2005-08", "url": "https://clinicaltrials.gov/study/NCT00140218", "target_entities": ["oxidative_stress"], "locations": [{"facility": "David Lacomis MD", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "University of Virginia", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* established diagnosis of ALS FVC\\>60% of predicted not being ventilated no difficulty swallowing ambulatory (can use assistance devices)\n\nExclusion Criteria:\n\n* ALS duration \\>3 years advanced ALS with survival predicted \\<6 months dementia (MMSE\\<22) prior exposure to R(+) pramipexole orthostatic hypotension \\>30 mmHg history of psychosis or hallucinations abnormal baseline safety lab values", "sex": "ALL", "min_age": "21 Years", "max_age": "85 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "R(+) pramipexole dihydrochloride monohydrate", "targeting_mechanism": "Dopamine agonist with antioxidant and neuroprotective properties targeting oxidative stress in motor neurons", "targeting_mechanism_pmid": "34663413", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03377309", "title": "Safety and Tolerability of Perampanel in Amyotrophic Lateral Sclerosis Patients", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "American University of Beirut Medical Center", "summary": "Amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, is a fatal progressive neurodegenerative disease affecting motor cortex, brainstem and spinal cord leading to motor neuron death. It is a devastating disease of the anterior and lateral corticospinal tracts with approximately 3 years mean duration from symptoms onset to death, one-fifth survival at 5 years and only 10% may make it to 10 years.\n\nAmong the neuronal death pathways, excitotoxicity mechanism is considered to be the foremost-involved mechanism. AMPA receptors are thought to be the prime mediator of the fast excitation in spinal motor neurons, where they are expressed ubiquitously. AMPA receptor antagonist was able to prevent this acute degeneration in previous animal studies.\n\nThe investigators aim to study the tolerability and safety of the novel AMPA antagonist, perampanel, in patients diagnosed with ALS. Perampanel \\[2-(2-oxo-1-phenyl-5- pyridin-2-yl-1,2-dihydropyridin-3-yl) benzonitrile\\] with its selective non-competitive AMPA antagonism, was recently approved for epilepsy. Various long-term trials studying perampanel in epilepsy showed favorable tolerability profile and most common side effects were mainly: dizziness, headache and somnolence. All patients presenting to Neurology clinics at AUBMC diagnosed with Amyotrophic Lateral Sclerosis, will be considered for the study. Investigators will obtain informed consents from all patients who agree to be enrolled in this study in accordance with institutional review board (IRB) requirements. Patients of both genders and over 18 years old who meet the El Escorial criteria for possible, probable or definite ALS and fit the inclusion criteria will be recruited. Subjects should not be started on riluzole for the past 30 days or stable on a dose of riluzole for at least 30 days prior to the screening process.\n\nIn titration phase, perampanel dose will be increase by 2mg/day increments every one week to reach a maximum dose of 8 mg/day; reaching the maximum dose in four weeks. Treatment phase will be followed by washout period during which, dose will be tapered by 2mg/day every 5 days (over total of 15 days).", "interventions": [{"type": "DRUG", "name": "Fycompa"}], "start_date": "2019-12-01", "url": "https://clinicaltrials.gov/study/NCT03377309", "target_entities": ["GRIN2B"], "locations": [{"facility": "Johnny S. Salameh", "city": "Beirut", "state": "", "country": "Lebanon", "status": "", "lat": 33.89332, "lon": 35.50157}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Amyotrophic Lateral Sclerosis (ALS) volunteers must be diagnosed within the 3 years prior to participation as having possible, probable, or definite ALS, either sporadic or familial according to modified El Escorial criteria\n* Age 18-80, able to provide informed consent, and comply with study procedures\n* Participants must not have started riluzole for at least 30 days, or be on a stable dose of riluzole for at least 30 days, prior to screening (riluzole-na\u00efve participants are permitted in the study)\n* Slow VC test equal to or greater than 50% of the predicted value\n\nExclusion Criteria:\n\n* The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair ability of the participant to provide informed consent\n* Exposure to any experimental agent within 30 days of entry or at any time during the trial or enrollment in another research study within 30 days of or during this trial\n* Women who are breastfeeding, who are pregnant or are planning to become pregnant\n* Renal insufficiency as defined by a serum creatinine \\> 1.5 times the upper limit of normal\n* Hepatic insufficiency or abnormal liver function (AST and/or ALT greater than 3 times the upper limit of the normal range)\n* Slow VC test less than 50% of the predicted value\n* ECG finding of QTc prolongation \\> 450 ms\n* Patients who had already undergone tracheostomy", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Fycompa (perampanel)", "targeting_mechanism": "AMPA receptor antagonist that blocks excitotoxic neuronal death pathways in motor neurons", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06126315", "title": "Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mario Negri Institute for Pharmacological Research", "summary": "Phase II/III multicenter, randomized, double-blind, placebo-controlled trial on acetyl-L-carnitine (ALCAR) in subjects living with amyotrophic lateral sclerosis (ALS). Primary study aim: The clinical objective consists of assessing the efficacy of ALCAR (two different dosages will be tested: 1.5g/day and 3g/day) on the progression of functional disability (loss of self-sufficiency), as measured by the ALSFRS-R scale. Secondary study aims: 1. The effect of ALCAR treatment on different clinical aspects: functional decline as measured by ALSFRS-R total score; the decline of forced vital capacity (FVC); quality of life as measured by ALSAQ-40 scale; cognitive function as measured by Edinburgh Cognitive and Behavioural ALS Screen (ECAS) scale; survival (being alive and without tracheostomy). 2. To measure the effects of ALCAR treatment on disease biomarkers potentially involved in the drug's mechanisms of action. These include PGC-1 alpha, 3-nitrotyrosine (3-NT), acetyl cyclophilin A (acetyl-PPIA), neurofilament light chain (NFL), creatine kinase (CK), Musclin/osteocrin, MyomiRNA (MiR-206), Uric acid, Matrix metalloproteinase-9 (MMP-9), Monocyte Chemoattractant Protein-1 (MCP-1), 4-Hydroxynonenal (HNE). 3. The tolerability and safety of ALCAR treatment by identifying unexpected adverse events.\n\nStudy population: 246 subjects will be enrolled on one Australian and ten Italian ALS sites.\n\nInclusion criteria: subjects aged 18+ years with a diagnosis of ALS according to Gold Coast Criteria; disease duration \\<24 months; satisfactory bulbar and spinal function (self-sufficiency evaluated by a score 3+ on the ALSFRS-R for swallowing, cutting food and handling utensils, and walking); satisfactory respiratory function (FVC \u226580% of predicted); documented progression of symptoms as measured by the ALSFRS-R scale. Disease progression rate (DFS) must be\\>= 0.33. DFS =(48- ALSFRS-R at screening)/months from onset to screening, treatment with Riluzole in the last four weeks. Exclusion criteria: antecedent polio infection; other motor neuron disease; involvement of other systems possibly determining a functional impairment; other severe clinical conditions; unwillingness or inability to take riluzole; previous use of ALCAR for any reason; inability to understand and comply with the study requirements, and to give written informed consent personally or via their legally authorized representative.\n\nAll eligible participants will be randomized to receive ALCAR (1,5 or 3 g/day) or placebo in addition to riluzole 50 mg b.i.d. Permuted block (with a block size of 6), 1:1:1 centralized randomization scheme will be used. The overall treatment duration will be 48 weeks. After enrolment, each participant will be followed up until death. Eligible subjects will be seen after 4, 12, 24, 36 and 48 weeks. At each visit, a general assessment will be made, including vital signs, body mass index (BMI), neurological examination (including quantitative and qualitative evaluation of the motor system), comorbidity, concomitant treatments and adverse events. Blood samples will be collected at baseline -Day 1 (randomization)-, 4, 12, 24, 36 and 48 weeks to test biomarkers. Functional disability will be assessed at each visit using the ALS-FRS-R scale. The respiratory function will be assessed using a spirometer to measure FVC before starting treatment (baseline visit) and at 4, 12, 24, 36 and 48 weeks. Cognitive function will be evaluated at baseline, weeks 24 and 48, using ECAS scale. Health-related quality of life, measured by the ALSAQ-40, will be tested at baseline, 24 and 48 weeks. Compliance will be tested by the local investigators, counting unused packages at each follow-up visit. Pre-planned statistical analyses will be done on Intention-to-treat and Per-protocol (PP) populations. The statistical plan will include descriptive statistics and a comparison of the proportions of self-sufficient participants at week 48 using the chi-square or Fisher's exact test for the primary endpoint. Secondary endpoints measured by numerical scores obtained from clinical scales will be analyzed using repeated measures mixed models, while biomarkers using repeated measures ANOVA. Time-to-event endpoints, such as survival and the probability of remaining self-sufficient over 48 weeks, will be analyzed with Kaplan-Meier curves. The number of adverse events and serious adverse events after 48 weeks will be compared between treatment arms.", "interventions": [{"type": "DRUG", "name": "Acetyl-l-carnitine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2025-03-26", "url": "https://clinicaltrials.gov/study/NCT06126315", "target_entities": ["mitochondrial_function"], "locations": [{"facility": "Concord Hospital", "city": "Sydney", "state": "", "country": "Australia", "status": "RECRUITING", "lat": -33.86785, "lon": 151.20732}, {"facility": "Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII, Dipartimento di Neurologia", "city": "Bergamo", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.69601, "lon": 9.66721}, {"facility": "Fondazione Serena ONLUS Centro Clinico NEMO Brescia", "city": "Brescia", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.53558, "lon": 10.21472}, {"facility": "Istituto Auxologico Italiano, IRCCS Dipartimento di Neurologia", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "Fondazione Serena ONLUS centro clinico NEMO", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "AOU di Modena Nuovo Ospedale Civile S. Agostino Estense di Modena - Ospedale di Baggiovara", "city": "Modena", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 44.64783, "lon": 10.92539}, {"facility": "Azienda Ospedaliera Universitaria Federico II Di Napoli", "city": "Naples", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 40.85216, "lon": 14.26811}, {"facility": "Azienda Ospedaliera Universitaria \"Luigi Vanvitelli\", Dipartimento di Scienze mediche e chirurgiche avanzate", "city": "Naples", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 40.85216, "lon": 14.26811}, {"facility": "Azienda Ospedaliero-Universitaria Maggiore della Carit\u00e0", "city": "Novara", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.44694, "lon": 8.62118}, {"facility": "Azienda Ospedale-Universit\u00e0 di Padova, Unit\u00e0 di Neurologia Clinica", "city": "Padova", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 44.38225, "lon": 11.14261}, {"facility": "A.S.P. Palermo, Villa delle Ginestre Hospital", "city": "Palermo", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 38.1166, "lon": 13.3636}, {"facility": "Fondazione Mondino Istituto Neurologico Nazionale a Carattere Scientifico IRCCS", "city": "Pavia", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.19205, "lon": 9.15917}, {"facility": "Fondazione Serena ONLUS-Centro Clinico NEMO Trento", "city": "Pergine Valsugana", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 46.06434, "lon": 11.23758}, {"facility": "Azienda Ospedaliera di Perugia", "city": "Perugia", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 43.1122, "lon": 12.38878}, {"facility": "Azienda Ospedaliero Universitaria Pisana", "city": "Pisa", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 43.70853, "lon": 10.4036}, {"facility": "Azienda Ospedaliero Universitaria Pisana, Dipartimento di Medicina Clinica e Sperimentale", "city": "Pisa", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 43.70853, "lon": 10.4036}, {"facility": "San Camillo Forlanini Hospital, Center for Neuromuscolar and Neurological Rare Diseases, Unit of Neurology and Neurophysiopathology", "city": "Roma", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 44.99364, "lon": 11.10642}, {"facility": "Azienda Ospedaliero-Universitaria Policlinico Umberto I - Universit\u00e0 di Roma \"La Sapienza\"", "city": "Roma", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 44.99364, "lon": 11.10642}, {"facility": "Fondazione Serena ONLUS - Centro Clinico NeMO Ancona", "city": "Torrette", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 40.53485, "lon": 15.01974}], "contact_phone": "00390239014605", "contact_email": "elisabetta.pupillo@marionegri.it", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 18+;\n2. ALS diagnosis according to the Gold Coast Criteria;\n3. Disease duration \\< 24 months from symptom onset, as indicated by limb weakness or bulbar symptoms, at the randomization/baseline visit\\*;\n4. Self-sufficiency \\[Satisfactory bulbar and spinal function (score 3+ on the ALSFRS-R for swallowing, cutting food and handling utensils, and walking)\\];\n5. Satisfactory respiratory function (FVC \u226580% of predicted);\n6. Documented progression of symptoms as measured by the ALSFRS-R scale. Disease progression rate (DFS) must be\\>= 0.33. DFS =(48- ALSFRS-R at screening)/months from onset to screening.\n7. Ability to understand and comply with the study requirements;\n8. Ability to give written informed consent personally or, as an alternative, via a legally authorized representative;\n9. Treatment with riluzole 50 mg twice/day for at least 4 weeks prior to randomization visit;\n10. Intact cognitive function, again determined by the Principal Investigator.\n\n    * The qualifying first symptoms of ALS are limited to manifestations of weakness in extremity, bulbar, or respiratory muscles. Cramps, fasciculations, or fatigue should not be taken in isolation as a first symptom of ALS.\n\nExclusion Criteria:\n\n1. Antecedent polio infection or other active infection;\n2. Motor neuron disease (MND) other than ALS;\n3. Involvement of other systems possibly determining a functional impairment (as measured by the endpoints) for the entire duration of the study;\n4. Other severe clinical conditions (e.g., cardiovascular disorders, neoplasms) with an impact on survival or functional disability in the next 12 months;\n5. Previous use of ALCAR for any reason;\n6. Poor compliance with previous treatments;\n7. Other experimental treatments in the three months prior to the screening visit (if a subject is receiving another experimental drug, a 3-month wash-out period before participating in the present clinical trial will be required);\n8. Women who are lactating or able to become pregnant (e.g. who are not post-menopausal, surgically sterile, or using inadequate birth control) and men unable to practice contraception for the duration of the treatment and three months after its completion;\n9. Inability to understand and comply with the study requirements;\n10. Unwillingness or inability to take riluzole.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Acetyl-L-carnitine (ALCAR)", "targeting_mechanism": "Enhances mitochondrial bioenergetics and ATP production to support neuronal energy metabolism and reduce mitochondrial dysfunction", "targeting_mechanism_pmid": "35805131", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06147843", "title": "French-German Cohort Study to Determine Factors Associated With Weight Loss in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Limoges", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease. Studies have shown the importance of weight loss at the time of diagnosis and during the progression of the disease. However, the pathophysiological mechanisms behind weight loss remain unknown. Identifying these mechanisms could make it possible to propose an effective therapeutic strategy against weight loss for ALS patients, which could improve their survival and quality of life. In this context, the investigators are proposing an innovative multidisciplinary project aimed at structuring a large Franco-German cohort to identify the markers associated with weight loss in ALS.\n\nParticipants will undergo high quality standard care for ALS patients. In addition, participants will be asked to respond different questionnaires and blood samples will be taken for analysis to identify biological markers.", "interventions": [{"type": "OTHER", "name": "Blood sample"}], "start_date": "2024-09-17", "url": "https://clinicaltrials.gov/study/NCT06147843", "target_entities": [], "locations": [{"facility": "CHU de Lille", "city": "Lille", "state": "", "country": "France", "status": "RECRUITING", "lat": 50.63391, "lon": 3.05512}, {"facility": "Limoges University Hospital", "city": "Limoges", "state": "", "country": "France", "status": "RECRUITING", "lat": 45.83362, "lon": 1.24759}, {"facility": "H\u00f4pital de la Timone", "city": "Marseille", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.29695, "lon": 5.38107}, {"facility": "H\u00f4pital Gui Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nice", "city": "Nice", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.70313, "lon": 7.26608}, {"facility": "H\u00f4pital de la Piti\u00e9 Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}, {"facility": "CHU de Tours", "city": "Tours", "state": "", "country": "France", "status": "RECRUITING", "lat": 47.39484, "lon": 0.70398}, {"facility": "Klinik f\u00fcr Medizinische Hochschule Hannover Carl-Neuberg-Str. 1", "city": "Hanover", "state": "Allemagne", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 52.37052, "lon": 9.73322}, {"facility": "Klinik f\u00fcr Neurologie, Universit\u00e4ts- und Rehabilitationskliniken Ulm (RKU", "city": "Ulm", "state": "Allemagne", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 48.39841, "lon": 9.99155}], "contact_phone": "05 55 05 15 69", "contact_email": "philippe.couratier@chu-limoges.fr", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Incident cases included at the time of diagnosis with a definite, probable, probable laboratory-supported, or possible ALS according to El Escorial revised criteria and Gold Coast criteria for early diagnosis.\n* Incident ALS cases identified and followed-up in the participant ALS \\& Other Motor Neuron Diseases Referral Centres: seven in France and two in Germany.\n* Patients who signed the informed consent form.\n* Adults aged \\>18 years old\n\nExclusion Criteria:\n\n* Inability to understand the requirements of the protocol.\n* Cognitive inability to sign and comprehend the informed consent form.\n* Patients who will not accept Riluzole therapy during their follow-up.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04798378", "title": "NuroSleeve Powered Brace & Stimulation System to Restore Arm Function", "phase": "NA", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Thomas Jefferson University", "summary": "The purpose of this study is to investigate if a person with weakness or paralysis in one or both arms, can use the NuroSleeve combined powered arm brace (orthosis) and muscle stimulation system to help restore movement in one arm sufficient to perform daily activities. This study could lead to the development of a product that could allow people with arm weakness or arm paralysis to use the NuroSleeve and similar devices to improve arm health and independent function.", "interventions": [{"type": "DEVICE", "name": "Neurosleeve"}], "start_date": "2020-04-16", "url": "https://clinicaltrials.gov/study/NCT04798378", "target_entities": [], "locations": [{"facility": "Nemours Children's Hospital", "city": "Wilmington", "state": "Delaware", "country": "United States", "status": "", "lat": 39.74595, "lon": -75.54659}, {"facility": "Thomas Jefferson University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\u2022 Must be 4 years or older\n\n* Must have weakness in one or both arms such that flexion or extension of the wrist, elbow or shoulder are 3/5 or less on the Manual Muscle Testing Scale\n* The etiology of weakness is due to a neurological disease or injury or orthopedic condition that occurred 6 or more months ago\n* Participant is willing to comply with trial instructions\n* Adult participant is able to provide informed consent prior to enrollment in the study, and for children, child is able to provide assent and designated caregiver (parent or guardian) is able to provide informed consent\n* The participant is fluent in English and, if the participant were a child, at least one parent/guardian were fluent in English\n* Medically stable and living at home in the community.\n* No joint contracture, spasticity or other limitations to range of motion in the affected lower limb(s) precluding the operation of a wearable, powered orthotic device on the arm\n* Sufficient sitting balance to sit in a chair\n* No condition (e.g., severe arthritis, central pain) that would interfere with movement of the legs, ability to understand verbal commands and cooperate with test procedures.\n* No condition that would pose a risk to the application of electrical current to the body (e.g., skin conditions or skin breakdown)\n\nExclusion Criteria:\u2022 Visual impairment such that following visually-guided instructions would be challenging even with ordinary corrective lenses\n\n* Orthopedic conditions of either arm that would affect performance on study\n* Untreated psychiatric or neurologic disturbances that would affect motivation and trial participation\n* Excessive pain in one or both of the arms (\\> 5 on a 10-point visual analog scale)\n* Excessive spasticity at one or both arms, as defined as a score of \\> 2 on the Modified Ashworth Spasticity Scale\n* Advice from any of the participant's health providers that upper extremity powered orthotics or electrical stimulation were contra-indicated\n* Presence of an implanted medical device in the body (such as cardiac pacemaker, implanted defibrillator, metallic device)\n* Metal implants or exposed metal in the weak or paralyzed arm\n* Lack of access to internet or wireless coverage to enable telemedicine-guided sessions\n* Any history of seizure or epilepsy (only an exclusion criterion for those seeking to undergo optional transcranial magnetic stimulation)\n* Currently taking the medication bupropion (only an exclusion criterion for those seeking to undergo optional transcranial magnetic stimulation)\n* Any history of prior neurosurgical procedure (only an exclusion criterion for those seeking to undergo optional transcranial magnetic stimulation)\n* Known or suspected skull defect (only an exclusion criterion for those seeking to undergo optional transcranial magnetic stimulation)\n* Any history of alcohol or other substance use\n* Other conditions or circumstances that, in the opinion of the investigators, would preclude safe and/or effective participation, including severe skin conditions, and/or other sequelae that may be contraindicated for using a powered orthotic or using electrical stimulation, as well as personal circumstances", "sex": "ALL", "min_age": "4 Years", "max_age": "", "healthy_volunteers": true, "std_ages": ["CHILD", "ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NuroSleeve (powered orthosis with muscle stimulation system)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04090684", "title": "Ciprofloxacin/Celecoxib Combination in Patients With ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "NeuroSense Therapeutics Ltd.", "summary": "This is an open label, off label study, to provide interested ALS patients with Ciprofloxacin/Celecoxib fixed dose combination, while assessing safety and tolerability, routine disease progression measures (ALSFRS-R and Vital Capacity).", "interventions": [{"type": "DRUG", "name": "Fixed dose combination Ciprofloxacin/Celecoxib"}], "start_date": "2019-12-09", "url": "https://clinicaltrials.gov/study/NCT04090684", "target_entities": ["neuroinflammation", "COX2"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to comprehend and willing to sign an Informed Consent Form (ICF)\n2. Males or females between the ages of 18 and 75 years of age, inclusive\n3. Diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria) less than 5 years prior to baseline\n4. Patients may be on Riluzole and/or Edaravone; 30 days of stable use is required to make safety assessments more reliable\n5. Upright Forced Vital Capacity (FVC) \u2265 50% of predicted for age, height and sex at screening\n6. Patient is able to swallow tablets/ capsules\n7. A caregiver (if one is needed)\n8. Female patients must be post-menopausal (\u2265 1 year) OR sterilized, OR if of childbearing potential (i.e., females who have had their first period unless they are anatomically and physiologically incapable to become pregnant), must have a negative pregnancy test, and agree to use contraceptive drugs or devices (e.g., diaphragm plus spermicide, or oral contraceptives) for the duration of the study and 10 weeks after the last treatment dose AND require male partners to use a condom during sexual intercourse\n\nExclusion Criteria:\n\n1. A past history of adverse reaction/hypersensitivity to either NSAIDs, celecoxib or fluoroquinolones, ciprofloxacin\n2. Any known clinically significant abnormal gastric mucosal initial gastroscopic of an erosion, ulcer or tumor or/and GI disorder\n3. Known history of impaired renal function.\n4. Known or suspected congestive heart and/or coronary heart disease, previous history of myocardial infarction, uncontrolled arterial hypertension, or rhythm abnormalities requiring permanent treatment\n5. Known history of QT/QTc prolongation, Torsade de pointes (TdP) (e.g. heart failure, hypokalemia, family history of Long QT syndrome) and the use of concomitant medications that prolong the QT/QTc interval.\n6. Known or suspected diagnosis or family history of epilepsy\n7. Presence at screening of any medically significant cardiac, pulmonary, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety data, including, but not limited to:\n\n   1. Mean systolic blood pressure \\>180 mm Hg; mean diastolic blood pressure \\>100 mm Hg (measurements taken after few min rest) that persist on 3 successive measurements taken at least 2 minutes apart\n   2. NYHA Class II or greater congestive heart failure\n   3. Chronic obstructive pulmonary disease or asthma requiring daily use of bronchodilator medications\n   4. Poorly controlled or brittle diabetes mellitus\n   5. Cognitive impairment, related to ALS or otherwise, sufficient to impair the patient's ability to understand and/or comply with study procedures and provide informed consent\n8. Female who is pregnant or breastfeeding or with intention of becoming pregnant during the course of the study\n9. Any impairment or social circumstance that, in the opinion of the Investigator, would render the patient not suitable to participate in the study\n10. Patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study\n11. Patient is participating in (or plans to participate in) any other investigational drug trial, or plans to be exposed to any other investigational agent, device and/or procedure, from 30 days prior to Screening through study completion", "sex": "ALL", "min_age": "18 Years", "max_age": "75 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "Ciprofloxacin/Celecoxib fixed dose combination", "targeting_mechanism": "Reduction of neuroinflammation through antibiotic and anti-inflammatory mechanisms.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01605006", "title": "Humanitarian Device Exemption Post-Approval Study of NeuRx Diaphragm Pacing System for Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Synapse Biomedical", "summary": "This post-approval study will follow 60 participants who have ALS, documented chronic hypoventilation, and bilateral phrenic nerve function, and who undergo the surgical implantation procedure to receive the NeuRx Diaphragm Pacing System device. Participants who are successfully implanted with the device will use it for daily diaphragm conditioning sessions. Participants will be followed for at least two years (until the last enrolled participant reaches the 2-year follow-up visit). Safety and probable benefit outcome measures will be assessed.", "interventions": [{"type": "DEVICE", "name": "NeuRx Diaphragm Pacing System (DPS)"}], "start_date": "2012-07", "url": "https://clinicaltrials.gov/study/NCT01605006", "target_entities": [], "locations": [{"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Medical Center -- Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Denver", "city": "Denver", "state": "Colorado", "country": "United States", "status": "", "lat": 39.73915, "lon": -104.9847}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Nebraska Medical Center", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "", "lat": 41.25626, "lon": -95.94043}, {"facility": "Stony Brook University", "city": "Stony Brook", "state": "New York", "country": "United States", "status": "", "lat": 40.92565, "lon": -73.14094}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "University Hospitals Case Medical Center", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Providence St. Vincent Medical Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Age 21 or older.\n2. Participants with familial or sporadic ALS diagnosed as laboratory-supported probable, probable, or definite according to the World Federation of Neurology El Escorial criteria.\n3. Bilateral phrenic nerve function clinically acceptable as demonstrated by bilateral diaphragm movement with fluoroscopic sniff test or with EMG recordings and nerve conduction times.\n4. Chronic hypoventilation was documented by at least one of the following:\n\n   * FVC less than 50% predicted, or\n   * \\|MIP\\| less than 60 cmH2O, or\n   * PaCO2 greater than or equal to 45 mmHg, or\n   * Nocturnal SaO2 less than or equal to 88% for at least five continuous minutes\n5. Suitable surgical candidate.\n6. Negative pregnancy test in female participants of childbearing potential.\n7. Informed consent from patient or designated representative.\n\nExclusion Criteria:\n\n1. Underlying cardiac or pulmonary disease that would increase the risk of general anesthesia.\n2. Underlying pulmonary diseases that were present prior to ALS that would affect pulmonary tests independent of ALS.\n3. Uncontrolled excessive secretions.\n4. FVC less than 45% predicted at time of surgery.\n5. Preexisting implanted electrical device such as pacemaker or cardiac defibrillator.\n6. Pre-existing diaphragm abnormality such as a hiatal hernia or paraesophageal hernia of abdominal contents going into the thoracic cavity.", "sex": "ALL", "min_age": "21 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "NeuRx Diaphragm Pacing System (DPS)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03883581", "title": "Impact of Nuedexta on Bulbar Physiology and Function in ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Florida", "summary": "Nuedexta is FDA approved for the treatment of pseudobulbar affect in ALS patients and anecdotal reports of improvements in speech, salivation or swallowing have been reported. However, no prospective study has been conducted to comprehensively examine and determine the physiologic impact of Nuedexta on both speech and swallowing physiology in a large group of ALS individuals. These data are needed in order to provide evidence-based guidance to the management of bulbar dysfunction in ALS.", "interventions": [{"type": "DRUG", "name": "dextromethorphan HBr and quinidine sulfate"}], "start_date": "2019-07-25", "url": "https://clinicaltrials.gov/study/NCT03883581", "target_entities": ["pseudobulbar_affect"], "locations": [{"facility": "Phil Smith Neuroscience Institute at Holy Cross Hospital", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of probable-definite ALS (El-Escorial Criterion);\n* ALSFRS-R Bulbar subscale score \\<10\n* Bamboo oral reading speaking rate \\<140 words per minute\n* No allergies to barium sulfate.\n\nExclusion Criteria:\n\n* Treatment for sialorrhea within the past 3 months that includes either Botox or radiation treatment\n* Participation in another disease modifying study targeting bulbar or cough function\n* Use of invasive mechanical ventilation/presence of tracheostomy\n* Advanced frontotemporal dementia or significant cognitive dysfunction\n* Nil per oral status for feeding (i.e., NPO, nothing by mouth)\n* Previously prescribed Nuedexta. Additionally, if participants are taking Riluzole or other medications to control sialorrhea, they must be on a stable dose for at least 30 days prior to enrollment in the current study.", "sex": "ALL", "min_age": "18 Years", "max_age": "90 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "dextromethorphan HBr and quinidine sulfate", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01486849", "title": "Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "A Phase II, double-blind, randomized, placebo-controlled ascending dose titration study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects of multiple ascending doses of CK-2017357 to an individual patient maximum tolerated dose (MTD), using a within-patient twice daily (BID) dose-titration regimen in ALS patients on 50 mg riluzole once daily (QD).", "interventions": [{"type": "DRUG", "name": "CK-2017357"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Riluzole 50 MG"}], "start_date": "2011-11", "url": "https://clinicaltrials.gov/study/NCT01486849", "target_entities": ["muscle_contractility"], "locations": [{"facility": "University of California at San Francisco, Fresno Campus, Central California Neurological Institute", "city": "Fresno", "state": "California", "country": "United States", "status": "", "lat": 36.74773, "lon": -119.77237}, {"facility": "Coordinated Clinical Research", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "University of California at Irvine, ALS and Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "Massachusetts General Hospital, Neurology Clinical Trials Unit", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Cornell Faculty, Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Duke University School of Medicine, Division of Neurology", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Ohio State University, Department of Neurology", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Texas Health Science Center, Department of Neurology", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Able to comprehend and willing to sign an Informed Consent Form (ICF)\n2. Males or females 18 years of age or older\n3. A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria)\n4. Maximum voluntary grip strength in at least one hand between 10 \\& 40 pounds (females) and 10 \\& 60 pounds (males)\n5. Able to swallow tablets with water\n6. Currently taking and tolerating a stable dose of 50 mg BID riluzole\n7. Willing and able to reduce daily dose of riluzole to 50mg QD for 5 weeks\n8. Not currently taking or willing and able to remain off theophylline-containing medications during study participation\n9. Patient has a caregiver who is capable of observing and reporting patient status\n10. Upright Slow Vital Capacity (SVC) \\>50% of predicted for age, height, and sex\n11. Able to perform pulmonary function tests\n\nExclusion Criteria:\n\n1. Life expectancy \\<3 months\n2. Receipt of investigational study drug within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing\n3. Any prior treatment with CK-2017357\n4. Any use of non-invasive positive pressure ventilation (NIPPV), such as Continuous Positive Airway Pressure (CPAP) or Bilevel Positive Airway Pressure (BiPAP)\n\nOther protocol-defined inclusion/exclusion criteria may apply.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "CK-2017357", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04952155", "title": "Low Dose IL-2 in the Treatment of Immune-associated ALS Syndrome", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Peking University Third Hospital", "summary": "The purpose of this study was to evaluate the efficacy and safety of low-dose IL-2 in the treatment of immunorelated ALS syndrome.", "interventions": [{"type": "DRUG", "name": "IL-2"}], "start_date": "2020-01-01", "url": "https://clinicaltrials.gov/study/NCT04952155", "target_entities": ["IL-2"], "locations": [{"facility": "Peking University Third Hospital", "city": "Beijing", "state": "Beijing Municipality", "country": "China", "status": "RECRUITING", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "+86 13701023871", "contact_email": "dsfan@sina.com", "eligibility": {"criteria": "Inclusion Criteria:\n\n* 18-70 years old;\n* Clinically diagnosed with ALS syndrome, i.e., with ALS -like manifestations, consisting of a combination of upper and/or lower motor neuron damage;\n* significant abnormalities with rheumatoid immune-related indicators, or diagnoses of immune-mediated ALS syndrome, including but not limited to multifocal motor neuropathy (MMN), Lewis-Sumner syndrome, and other ALS-like syndromes with an immune background that cannot be clearly classified;\n* Poor treatment with conventional hormones or gamma globulin;\n* Permitted concomitant treatment: oral prednisone or equivalent doses of other glucocorticoids (\u22641.0mg/kg/d); Oral routine dose of immunosuppressants or immunomodulators, such as cyclophosphamide, tacrolimus, etc.; Routine oral doses such as too much force; Doses and types of accompanying therapeutic drugs should not be changed from the trial enrollment to the end of follow-up.\n* For women of reproductive age, contraception for at least 2 weeks at the time of enrolment and negative urine HCG;\n* Reasonable and effective contraceptive measures should be taken by subjects of childbearing age from the time of trial enrollment to the end of follow-up;\n* Signed informed consent.\n\nExclusion Criteria:\n\n* Allergic or intolerance to IL2;\n* Receive non-standard treatment or use of excessive dose of glucocorticoids or gamma globulin intravenously within 2 months before enrollment;\n* Vaccination within 6 months before enrolment or between enrolment and the end of follow-up;\n* Peripheral venous white blood cells \\< 2000/mm3, lymphocytes \\< 600/mm3, platelets \\< 80,000 /mm3;\n* Complicated with severe infection or inflammation, such as bacteremia, sepsis, etc.;\n* Complicated blood system diseases, infectious diseases (hepatitis, HIV, tuberculosis, etc.), mental diseases, dementia, severe hypotension, substance abuse history, malignant tumor history, organ transplantation history, etc.;\n* Severe liver, kidney, lung or heart dysfunction: heart failure (\u2265NYHA grade III), renal insufficiency (creatinine clearance \u226430ml/min), abnormal liver function (3 times the upper limit of normal \\>);\n* Pregnant and lactating women;\n* Currently participating in other clinical studies or using other investigational drugs.", "sex": "ALL", "min_age": "18 Years", "max_age": "70 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "IL-2", "targeting_mechanism": "Modulation of adaptive immune response through interleukin-2 signaling to treat immune-associated ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00070993", "title": "Creatine for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Center for Complementary and Integrative Health (NCCIH)", "summary": "Creatine is a naturally occurring chemical involved in the production of energy in muscle. Abnormalities in creatine have been linked to the progression of degenerative neuromuscular diseases such as amyotrophic lateral sclerosis (ALS, or Lou Gehrig's Disease). This study will test whether taking creatine can improve the symptoms of ALS.", "interventions": [{"type": "DRUG", "name": "creatine monohydrate"}], "start_date": "2002-12", "url": "https://clinicaltrials.gov/study/NCT00070993", "target_entities": ["cellular_energy_metabolism"], "locations": [{"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Rush-Presbyterian St. Luke's Medical Center", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of New Mexico - Medical Center", "city": "Albuquerque", "state": "New Mexico", "country": "United States", "status": "", "lat": 35.08449, "lon": -106.65114}, {"facility": "University of Texas Health and Science Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria\n\n* Diagnosis of probable or definite ALS\n* At least 5 of 10 testable upper extremity muscle groups (shoulder and elbow extensors/flexors and grip) of Medical Research Council (MRC) grade 4 or better\n* At least 5 years from onset of symptoms\n\nExclusion Criteria\n\n* Requires tracheostomy ventilation\n* History of renal disease", "sex": "ALL", "min_age": "21 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "creatine monohydrate", "targeting_mechanism": "Creatine enhances energy production in muscle by supporting ATP synthesis and cellular energy homeostasis in motor neurons.", "targeting_mechanism_pmid": "29549424", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03892863", "title": "Repetitive Transcranial Magnetic Stimulation as Therapy for Depression in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Jagiellonian University", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by progressive loss of central and peripheral motor neurons. ALS leads to death usually within 3 to 5 years from the onset of the symptoms. Available treatment can prolong the disease duration but cannot modify the disease course. Depression is a frequent complication of ALS, which further decreases quality of life and the available data concerning effectivity of antidepressant drugs are conflicting. Repetitive Transcranial Magnetic Stimulation (rTMS) is a noninvasive method of modulation of brain plasticity with confirmed antidepressive effect. The purpose of this study is to compare the effectiveness of rTMS in improving the depression in patients with ALS with placebo stimulation. Intervention will include 10 daily sessions. In each session 3000 magnetic pulses will be administered over the left dorsolateral prefrontal cortex. Assessment depression severity will be made before and after therapy, as well as two and four weeks later.", "interventions": [{"type": "DEVICE", "name": "active repetitive transcranial magnetic stimulation"}, {"type": "DEVICE", "name": "sham repetitive transcranial magnetic stimulation"}], "start_date": "2019-11-15", "url": "https://clinicaltrials.gov/study/NCT03892863", "target_entities": [], "locations": [{"facility": "Jagiellonian University Medical College, Department of Neurology", "city": "Krakow", "state": "", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Diagnosis of definite or probable ALS according to el Escorial criteria (Brooks et al. 2000)\n* Depression defined as the score in Beck's Depression Inventory \u226514\n* Mini-Mental State Examination score \u226526\n\nExclusion Criteria:\n\n* Psychiatric symptoms, which may negatively influence patient's tolerance and adherence to therapy\n* Respiratory insufficiency and other complications od advanced stages of ALS, which may compromise patient's ability to undergo the study procedure\n* Contraindications for rTMS as listed by the Guidelines of the International Federation of Clinical Neurophysiology (Rossi et al. 2009) i.e. seizure in the past, epilepsy, presence of magnetic material in the reach of magnetic field, pregnancy, likelihood to get pregnant, intracranial electrodes, cardiac pacemaker or intracardiac lines, frequent syncopes", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "repetitive transcranial magnetic stimulation", "targeting_mechanism": "Repetitive transcranial magnetic stimulation modulates upper motor neuron excitability and corticomotor function.", "targeting_mechanism_pmid": "37068329", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06119087", "title": "Mechanical Insufflation in the Philadelphia Amyotrophic Lateral Sclerosis Cohort (MI-PALS) Study", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Pennsylvania", "summary": "The goal of this clinical trial is to learn how doing mechanical insufflation (MI) using a mechanical insufflator-exsufflator (MI-E) device affects breathing in early amyotrophic lateral sclerosis (ALS). This will be a single-center, single-arm study of MI in 20 patients with ALS at Penn.\n\nBased on prior research, we believe that 6-months of MI may slow decline in cough strength, measured as peak cough flow (PCF).\n\nParticipants will perform MI using a device designed for mechanical insufflation-exsufflation (MI-E) known as the BiWaze Cough system. The BiWaze Cough is used for mucus clearance . It is connected to tubing and mouthpiece (or mask). The device will use programmed pressure and timing settings. An insufflation includes inflating the lungs for a maximal size inhalation before exhaling. The daily routine for the device includes 5 sets of 5 insufflations twice daily.\n\nResearchers will compare how use of MI in early ALS affects peak cough flow compared to 20 subjects who did not use MI in early ALS.", "interventions": [{"type": "DEVICE", "name": "Mechanical insufflation"}], "start_date": "2024-09-12", "url": "https://clinicaltrials.gov/study/NCT06119087", "target_entities": [], "locations": [{"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n1. Provision of signed and dated informed consent form.\n2. Stated willingness to comply with all study procedures and availability for the duration of the study.\n3. Age \u226518 years.\n4. Diagnosed with amyotrophic lateral sclerosis using the Gold Coast Criteria.\n5. Have an able and willing caregiver to assist with mechanical insufflation on a daily basis.\n6. Willingness and ability to participate in study procedures.\n\nExclusion Criteria:\n\n1. Age \\<18 years old.\n2. Inability to perform a cough peak flow or spirometry manuever\n3. Current use of non-invasive ventilation (NIV), bi-level positive pressure ventilation, or \"Bi-PAP\" or physician prescribing NIV on day of potential enrollment.\n4. Current use of MI-E (also known as a \"cough assist device\") for airway clearance. Please note that patients can start use of a MI-E device subsequent to enrollment while currently being followed for the study.\n5. Active enrollment in hospice.\n6. Current tracheostomy.\n7. Presence of cognitive dysfunction that would impair ability to complete study procedures, as determined by neurology attending physician.\n8. Absence of an able and willing caregiver to assist with MI twice daily as specified in the protocol.\n9. Pregnancy\n10. Medical history of any of the following:\n\n    1. Recent hemoptysis\n    2. Recent barotrauma\n    3. History of emphysema of any kind (including bullous emphysema)\n    4. History of or known susceptibility to pneumothorax\n    5. History of or known susceptibility to pneumomediastinum\n    6. Chronic obstructive pulmonary disease\n    7. Uncontrolled asthma (defined as recent exacerbation requiring corticosteroids in the previous 30 days)\n    8. Symptomatic cardiomyopathy (heart failure) with left ventricular ejection fraction less than 50%\n    9. History of right heart failure or pulmonary hypertension\n11. Current smoker or tobacco use within the last 30 days.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "mechanical insufflation", "targeting_mechanism": "Mechanical insufflation augments respiratory muscle function by using a mechanical insufflator-exsufflator device to assist breathing and cough clearance.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02487407", "title": "Effects of ODM-109 on Respiratory Function in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Orion Corporation, Orion Pharma", "summary": "In the double-blind, cross-over part of the study, ODM-109 capsules and placebo capsules for ODM-109 will be administered for 2 weeks separated by a 19-23 days wash-out period. During each treatment period of the double-blind cross-over part, there will be a baseline visit (day 1) and 2 visits (5 \u00b1 2 and 14 \u00b1 2 days) after the start of study treatment. After completing the 3rd treatment period, the subjects will continue in the open-label follow-up part for 6 months. During the open-label follow-up, visits will be at 1, 3 and 6 months. An end-of-study visit will take place 14-25 days after the last study treatment administration for each subject. The study duration will be about 13-14 weeks for the double-blind cross-over part, and about 9-10 months for the entire study including the 6 months open-label follow-up.\n\nThe number of randomised study subjects is planned to be approximately 54 in cross-over comparison. The maximum number of subjects will not exceed 70.\n\nPrimary objective is to investigate the efficacy of oral ODM-109 on respiratory function in patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "ODM-109"}, {"type": "DRUG", "name": "Placebo for ODM-109"}], "start_date": "2015-07", "url": "https://clinicaltrials.gov/study/NCT02487407", "target_entities": ["respiratory_function"], "locations": [{"facility": "Charite Universitatsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Medical School Hannover", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "University Clinical Jena", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "University Hospital of Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "University Medical Centre Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Royal Sussex County Hospital", "city": "Brighton", "state": "", "country": "United Kingdom", "status": "", "lat": 50.82838, "lon": -0.13947}, {"facility": "The Walton Centre", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "London Kings College Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Royal London Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "University of Sheffield", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion Criteria:\n\n* Written informed consent (IC) for participation in the study will be obtained from the subject (or from the subject's next of kin, caregiver, or other legally acceptable representative in case the study subject him/herself cannot sign the IC due to severe muscle weakness).\n* Age of at least 18 years.\n* Male or female subjects with diagnosis of laboratory supported probable, probable or definite ALS according to El Escorial revised criteria (Brooks BR et al., 2000). Full electromyogram (EMG) report available compatible with ALS according to an experienced neurophysiologist.\n* Ability to swallow the study treatment capsules.\n* An upright (sitting position) SVC between 60-90% of the predicted value for age, height and sex at screening visit.\n* Normal oxygen saturation during daytime (measure of \u2265 95% when steady state has been reached with a reliable read) in sitting position measured by pulse oximetry.\n* Disease duration from symptom onset (defined by first muscle weakness or dysarthria) of 12-48 months.\n* Using riluzole. The dose must have been stable for at least 4 weeks prior to screening at a dose of 50 mg b.i.d.\n\nExclusion Criteria:\n\n* Subject in whom other causes of neuromuscular weakness have not been excluded.\n* Subject with a diagnosis of another neurodegenerative disease (e.g. Parkinson's or Alzheimer's disease).\n* Assisted ventilation or gastrostomy of any type during the preceding 3 months prior to screening or predicted to be required within the randomised, double-blind cross-over part of the study.\n* Recorded diagnosis or evidence of major psychiatric diagnosis, significant cognitive impairment or clinically evident dementia.\n* Any major surgery within 1 month before the screening visit or patients who are scheduled for any major surgery during the planned study period.\n* Potassium \\< 3.7 mmol/l or \\> 5.5 mmol/l at screening.\n* Creatinine \\> 170 \u03bcmol/l at screening or on dialysis.\n* Blood haemoglobin \\< 10 g/dl at screening.\n* Clinically significant hepatic impairment at the discretion of the investigator.\n* Women of reproductive age without a negative pregnancy test and without a commitment to using an acceptable method of barrier or hormonal contraception (e.g. condoms, diaphragms, oral contraceptives and long acting progestin agents), if sexually active during the study, and for 1 month after the last dose of the study treatment. Women who are postmenopausal (1 year since last menstrual cycle), surgically sterilised or who have undergone a hysterectomy are considered not to be reproductive and can be included.\n* Known hypersensitivity to levosimendan.\n* Administration of levosimendan within 30 days prior to screening visit.\n* Patients with history of botulinum toxin treatment for any reason.\n* Patients with known history of human immunodeficiency virus infection.\n* History of significant arrhythmias or other cardiac events\n* Any other clinically significant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, neurological or psychiatric disorder or any other major concurrent illness that in the opinion of the investigator could interfere with the interpretation of the study results or constitute a health risk for the subject if he/she took part in the study.\n* Blood donation or loss of significant amount of blood within 60 days prior to screening.\n* Participation in a clinical trial with any experimental treatment within 30 days prior to the screening visit or previous participation in the present study.\n* Any other condition that in the opinion of the investigator could interfere with the interpretation of the study results or constitute a health risk for the subject if he/she took part in the study.", "sex": "ALL", "min_age": "18 Years", "max_age": "", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "ODM-109", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03809845", "title": "Serial Fasciculation Measurements in Motor Neurone Disease", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "King's College Hospital NHS Trust", "summary": "Patients with motor neurone disease (MND) typically experience relentless motor decline and die within three years of symptom onset from respiratory muscle weakness. There are currently no effective therapies and the discovery of novel therapies is hampered by the lack of a sensitive disease biomarker. Consequently, there is a huge drive to discover novel biomarkers, which can reliably track disease progression over time. These can then be incorporated into clinical drug trials to expedite effective drug discovery.\n\nMuscle fasciculations represent the hyperexcitability of diseased motor neurons and are almost universally present from the early stages of MND. The investigators predict that the site, frequency and shape of fasciculations might provide a sensitive measure of disease progression in an individual.\n\nIn order to calibrate this technique, the investigators will conduct a 12-month longitudinal study, recruiting 24 patients from the King's College Hospital Motor Nerve Clinic, comprising a mixture of patients with MND and those with benign fasciculation syndrome. Patients in this latter group have fasciculations but do not develop weakness and have normal lifespans. They are therefore an optimal control group. At each visit, the investigators will take resting HDSEMG recordings from all four limbs and perform standard clinical measures of disease progression. The investigators will also monitor the decline in motor unit number using a newly validated neurophysiological technique, called Motor Unit Number Index (MUNIX).", "interventions": [{"type": "DEVICE", "name": "High-density surface electromyography"}], "start_date": "2017-07-06", "url": "https://clinicaltrials.gov/study/NCT03809845", "target_entities": [], "locations": [{"facility": "King's College Hospital NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "eligibility": {"criteria": "Inclusion criteria for MND patients:\n\n(i) Aged between 40 and 80 years of age inclusive, at the time of signing the informed consent.\n\n(ii) Diagnosed with MND by a neurologist with expertise in MND.(20) For subjects with bulbar onset there must be objective limb involvement of at least one limb.\n\n(iii) Diagnosed with MND within 24 months of symptom onset. (iv) Subjects must be ambulatory (i.e. must not be confined to a wheelchair). (v) Male and female subjects (vi) Capable of giving signed informed consent (vii) Capable and willing to comply with the requirements of the protocol (either by themselves or with assistance).\n\nInclusion criteria for Benign Fasciculation Syndrome (BFS) patients:\n\n(i) Aged between 18 and 80 years of age inclusive, at the time of signing the informed consent.\n\n(ii) Diagnosed with BFS by a neurologist with expertise in motor nerve disorders.\n\n(iii) Male and female subjects (vi) Capable of giving signed informed consent (vii) Capable and willing to comply with the requirements of the protocol (either by themselves or with assistance).\n\nExclusion criteria for MND patients:\n\n(i) Neurological (other than the subject's MND) or non-neurological co-morbidities (e.g. joint disease, respiratory disease) which limit mobility.\n\n(ii) Clinically significant cognitive impairment in the opinion of the investigator or lacking capacity in accordance with the Mental Capacity Act (2005).\n\n(iii) Regionally restricted forms of MND, or other atypical variants:\n\n* Isolated corticobulbar pattern of MND with normal ambulation\n* Primary lateral sclerosis\n* Signs of chronic partial denervation restricted to a single limb\n* MND or parkinsonism dementia complex (iv) Subjects requiring mechanical ventilation (non-invasive ventilation for sleep apnoea is allowed).\n\n  (v) Historical or current evidence of clinically significant uncontrolled disease which, in the opinion of the chief investigator, would put the safety of the subject at risk through participation or impact the study assessments or endpoints.\n\n(vi) Presence of an active implantable cardiac medical device (e.g., pacemaker or implantable cardioverter-defibrillator) or at a high risk for needing external defibrillation.\n\n(vii) History of skin hypersensitivity to adhesives. (viii) Current participation in a clinical trial which in the opinion of the chief investigator might impact the objectives of this study.\n\nExclusion criteria for Benign Fasciculation Syndrome patients:\n\n(i) Significant diagnostic uncertainty, whereby motor neurone disease remains a possible differential diagnosis.\n\n(ii) Historical or current evidence of clinically significant uncontrolled disease which, in the opinion of the chief investigator, would put the safety of the subject at risk through participation or impact the study assessments or endpoints.\n\n(iii) Presence of an active implantable cardiac medical device (e.g., pacemaker or implantable cardioverter-defibrillator) or at a high risk for needing external defibrillation.\n\n(iv) History of skin hypersensitivity to adhesives. (v) Current participation in a clinical trial which in the opinion of the chief investigator might impact the objectives of this study.", "sex": "ALL", "min_age": "18 Years", "max_age": "80 Years", "healthy_volunteers": false, "std_ages": ["ADULT", "OLDER_ADULT"]}, "mechanism_summary": {"compound": "high-density surface electromyography", "targeting_mechanism": "High-density surface electromyography is a non-invasive diagnostic and monitoring tool that measures motor unit activity to track motor neuron degeneration.", "targeting_mechanism_pmid": "28043769", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07660614", "title": "A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Leal Therapeutics, Inc", "summary": "This study will examine what happens when patients with amyotrophic lateral sclerosis (ALS) are given an investigational medication (study drug) known as LTX-002. Specifically, the researchers will be looking at safety, tolerability (if someone has any side effects from the drug), pharmacokinetics (what the body does to the study drug) and pharmacodynamics (what the study drug does to the body). The study will also investigate the effect of the drug on indicators of the severity of ALS, such as markers in blood and in the cerebrospinal fluid (the fluid that surrounds the brain and spinal cord, CSF) and on measures of the participant's ability to move, speak, and breathe.", "interventions": [{"type": "DRUG", "name": "LTX-002"}, {"type": "OTHER", "name": "Placebo (aCSF)"}], "start_date": "2026-04-29", "url": "https://clinicaltrials.gov/study/NCT07660614", "target_entities": [], "locations": [{"facility": "University Hospital Schleswig-Holstein, Campus L\u00fcbeck", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 53.86893, "lon": 10.68729}, {"facility": "Istituto Neurologico \"Carlo Besta\"", "city": "Milan", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 45.46427, "lon": 9.18951}, {"facility": "AOU Citt\u00e0 della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 44.88856, "lon": 11.99138}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "Karolinska Universitetssjukhuset", "city": "Stockholm", "state": "", "country": "Sweden", "status": "RECRUITING", "lat": 59.32938, "lon": 18.06871}], "contact_phone": "267-503-4800", "contact_email": "clinical.trials@lealtx.com", "mechanism_summary": {"compound": "LTX-002", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06643481", "title": "A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Novartis Pharmaceuticals", "summary": "This is a multicenter, randomized, double-blind, placebo-controlled, parallel group Phase II study to evaluate the efficacy and safety of VHB937 in participants with early-stage ALS (within 2 years of ALS symptoms onset). The study comprises a core double-blind (DB) 40-week treatment period followed by an open label extension (OLE).", "interventions": [{"type": "BIOLOGICAL", "name": "VHB937"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2024-10-17", "url": "https://clinicaltrials.gov/study/NCT06643481", "target_entities": [], "locations": [{"facility": "University of California San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Loma Linda University Health", "city": "Loma Linda", "state": "California", "country": "United States", "status": "", "lat": 34.04835, "lon": -117.26115}, {"facility": "Keck Medical Center USC", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "UC San Francisco Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Orlando Health Clinical Trials", "city": "Orlando", "state": "Florida", "country": "United States", "status": "", "lat": 28.53834, "lon": -81.37924}, {"facility": "Emory University School of Medicine", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "University Of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Nebraska Medical Center", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "", "lat": 41.25626, "lon": -95.94043}, {"facility": "Lange Neurology PC", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Rochester Medical Center", "city": "Rochester", "state": "New York", "country": "United States", "status": "", "lat": 43.15478, "lon": -77.61556}, {"facility": "Atrium Health", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke University Health System", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Univ of Cincinnati Medical Center", "city": "Cincinnati", "state": "Ohio", "country": "United States", "status": "", "lat": 39.12711, "lon": -84.51439}, {"facility": "The Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "AMR Knoxville", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Nerve and Muscle Center of Texas", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Novartis Investigative Site", "city": "North Ryde", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.79677, "lon": 151.12436}, {"facility": "Novartis Investigative Site", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Novartis Investigative Site", "city": "Caulfield South", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.89562, "lon": 145.02597}, {"facility": "Novartis Investigative Site", "city": "Southport", "state": "", "country": "Australia", "status": "", "lat": -27.96724, "lon": 153.39796}, {"facility": "Novartis Investigative Site", "city": "Leuven", "state": "Vlaams Brabant", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Novartis Investigative Site", "city": "Li\u00e8ge", "state": "", "country": "Belgium", "status": "", "lat": 50.63373, "lon": 5.56749}, {"facility": "Novartis Investigative Site", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Novartis Investigative Site", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Novartis Investigative Site", "city": "Beijing", "state": "", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}, {"facility": "Novartis Investigative Site", "city": "Aalborg", "state": "", "country": "Denmark", "status": "", "lat": 57.048, "lon": 9.9187}, {"facility": "Novartis Investigative Site", "city": "Kobenhavn N V", "state": "", "country": "Denmark", "status": "", "lat": null, "lon": null}, {"facility": "Novartis Investigative Site", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "Novartis Investigative Site", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Novartis Investigative Site", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Novartis Investigative Site", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Novartis Investigative Site", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "Novartis Investigative Site", "city": "Mannheim", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 49.4891, "lon": 8.46694}, {"facility": "Novartis Investigative Site", "city": "Munich", "state": "Bavaria", "country": "Germany", "status": "", "lat": 48.13743, "lon": 11.57549}, {"facility": "Novartis Investigative Site", "city": "W\u00fcrzburg", "state": "Bavaria", "country": "Germany", "status": "", "lat": 49.79391, "lon": 9.95121}, {"facility": "Novartis Investigative Site", "city": "Rostock", "state": "Mecklenburg-Vorpommern", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Novartis Investigative Site", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Novartis Investigative Site", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Novartis Investigative Site", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "", "lat": 53.86893, "lon": 10.68729}, {"facility": "Novartis Investigative Site", "city": "M\u00fcnster", "state": "", "country": "Germany", "status": "", "lat": 51.96236, "lon": 7.62571}, {"facility": "Novartis Investigative Site", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Novartis Investigative Site", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Novartis Investigative Site", "city": "Milan", "state": "MI", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Novartis Investigative Site", "city": "Modena", "state": "MO", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Novartis Investigative Site", "city": "Pisa", "state": "PI", "country": "Italy", "status": "", "lat": 43.70853, "lon": 10.4036}, {"facility": "Novartis Investigative Site", "city": "Torino", "state": "TO", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Novartis Investigative Site", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Novartis Investigative Site", "city": "Krakow", "state": "Poland", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}, {"facility": "Novartis Investigative Site", "city": "Bydgoszcz", "state": "", "country": "Poland", "status": "", "lat": 53.1235, "lon": 18.00762}, {"facility": "Novartis Investigative Site", "city": "Krakow", "state": "", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}, {"facility": "Novartis Investigative Site", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Novartis Investigative Site", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Novartis Investigative Site", "city": "Yangsan", "state": "Gyeongsangnam-do", "country": "South Korea", "status": "", "lat": 35.34199, "lon": 129.03358}, {"facility": "Novartis Investigative Site", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Novartis Investigative Site", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Novartis Investigative Site", "city": "Santiago Compostela", "state": "A Coruna", "country": "Spain", "status": "", "lat": null, "lon": null}, {"facility": "Novartis Investigative Site", "city": "L'Hospitalet de Llobregat", "state": "Barcelona", "country": "Spain", "status": "", "lat": 41.35967, "lon": 2.10028}, {"facility": "Novartis Investigative Site", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Novartis Investigative Site", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Novartis Investigative Site", "city": "Malm\u00f6", "state": "", "country": "Sweden", "status": "", "lat": 55.60587, "lon": 13.00073}, {"facility": "Novartis Investigative Site", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Novartis Investigative Site", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "Novartis Investigative Site", "city": "Basel", "state": "", "country": "Switzerland", "status": "", "lat": 47.55839, "lon": 7.57327}, {"facility": "Novartis Investigative Site", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "Novartis Investigative Site", "city": "Sheffield", "state": "South Yorkshire", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}, {"facility": "Novartis Investigative Site", "city": "Farnborough", "state": "", "country": "United Kingdom", "status": "", "lat": 51.29424, "lon": -0.75565}, {"facility": "Novartis Investigative Site", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Novartis Investigative Site", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Novartis Investigative Site", "city": "Stoke-on-Trent", "state": "", "country": "United Kingdom", "status": "", "lat": 53.00415, "lon": -2.18538}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "VHB937", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04849065", "title": "Clinical Trial on the Use of Cell Therapy in the Treatment of Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia", "summary": "Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease", "interventions": [{"type": "DRUG", "name": "MNC (Mononuclear cells)"}, {"type": "DRUG", "name": "Placebo / Saline"}], "start_date": "2021-05-01", "url": "https://clinicaltrials.gov/study/NCT04849065", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Hospital Clinico Universitario Virgen de la Arrixaca", "city": "Murcia", "state": "", "country": "Spain", "status": "", "lat": 37.98704, "lon": -1.13004}], "contact_phone": "968 36 95 00", "contact_email": "miguelblanquer@um.es", "mechanism_summary": {"compound": "MNC (Mononuclear cells)", "targeting_mechanism": "Autologous bone marrow mononuclear cells to reduce motor unit loss and support motor neuron function through provision of neuroprotective factors and modulation of neuroinflammation.", "targeting_mechanism_pmid": "37433768", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03626012", "title": "A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogen", "summary": "The primary objective of this study is to evaluate the safety and tolerability of BIIB078 in adults with C9ORF72-Amyotrophic Lateral Sclerosis (ALS). The secondary objectives of this study are to evaluate the pharmacokinetic profile of BIIB078 and to evaluate the effects of BIIB078 on clinical function. As the first-in-human study, the study enrolls a small number of participants in each cohort. Every participant in a cohort is treated with the same dose or placebo. The study is designed to evaluate and confirm the safety of each dose before enrolling and exposing new participants to a higher dose in the next cohort.", "interventions": [{"type": "DRUG", "name": "BIIB078"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2018-09-10", "url": "https://clinicaltrials.gov/study/NCT03626012", "target_entities": ["C9orf72"], "locations": [{"facility": "Research Site", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Research Site", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "Research Site", "city": "Palo Alto", "state": "California", "country": "United States", "status": "", "lat": 37.44188, "lon": -122.14302}, {"facility": "Research Site", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Research Site", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Research Site", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Research Site", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Research Site", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Research Site", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Research Site", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Research Site", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Research Site", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}, {"facility": "Research Site", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Research Site", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Research Site", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Research Site", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Research Site", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Research Site", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "Research Site", "city": "London", "state": "Greater London", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Research Site", "city": "Sheffield", "state": "South Yorkshire", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}, {"facility": "Research Site", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "BIIB078", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03268603", "title": "Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mayo Clinic", "summary": "The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a total of 4 injections over 12 months.\n\nMesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine.\n\nThe use of mesenchymal stem cells is considered investigational, which means it has not been approved by the Food and Drug Administration (FDA) for routine clinical use. However, the FDA has allowed the use of mesenchymal stem cells in this research study.", "interventions": [{"type": "DRUG", "name": "Autologous Adipose-derived Mesenchymal Stromal Cells"}], "start_date": "2017-10-10", "url": "https://clinicaltrials.gov/study/NCT03268603", "target_entities": [], "locations": [{"facility": "Mayo Clinic", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Mayo Clinic in Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Autologous Adipose-derived Mesenchymal Stromal Cells", "targeting_mechanism": "Mesenchymal stem cells differentiate into support cells and produce neuroprotective growth factors and anti-inflammatory cytokines to support degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "ASC-exosomes ameliorate disease progression in SOD1(G93A) murine model of ALS.", "animal_results_pmid": "32455791", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06280079", "title": "Ultra-high-caloric, Fatty Diet in ALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Ulm", "summary": "This study aims at evaluating efficacy and tolerability of an ultra-high-caloric, fatty diet (UFD) compared to placebo in patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Ultra-high-caloric fatty diet"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2024-06-01", "url": "https://clinicaltrials.gov/study/NCT06280079", "target_entities": ["metabolic_dysfunction"], "locations": [{"facility": "RWTH Aachen", "city": "Aachen", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 50.77664, "lon": 6.08342}, {"facility": "Charit\u00e9 Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 52.52437, "lon": 13.41053}, {"facility": "University Clinic Bochum", "city": "Bochum", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.48165, "lon": 7.21648}, {"facility": "University Clinic Bonn", "city": "Bonn", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 50.73438, "lon": 7.09549}, {"facility": "Technical University Dresden", "city": "Dresden", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.05089, "lon": 13.73832}, {"facility": "University Clinic Erlangen", "city": "Erlangen", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 49.59099, "lon": 11.00783}, {"facility": "Alfried Krupp Krankenhaus Essen", "city": "Essen", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.45657, "lon": 7.01228}, {"facility": "University Clinic G\u00f6ttingen", "city": "G\u00f6ttingen", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.53443, "lon": 9.93228}, {"facility": "University Clinic Halle", "city": "Halle", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.48158, "lon": 11.97947}, {"facility": "Hannover Medical School", "city": "Hanover", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 52.37052, "lon": 9.73322}, {"facility": "University Clinic Jena", "city": "Jena", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 50.92878, "lon": 11.5899}, {"facility": "DRK Clinic Kassel", "city": "Kassel", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.31667, "lon": 9.5}, {"facility": "Klinikum Kempten", "city": "Kempten", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 49.96729, "lon": 7.93702}, {"facility": "University Clinic Leipzig", "city": "Leipzig", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.33962, "lon": 12.37129}, {"facility": "University Clinic L\u00fcbeck", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 53.86893, "lon": 10.68729}, {"facility": "Diakonissenkrankenhaus Mannheim", "city": "Mannheim", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 49.4891, "lon": 8.46694}, {"facility": "Technical University Munich", "city": "Munich", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 48.13743, "lon": 11.57549}, {"facility": "University Clinic M\u00fcnster", "city": "M\u00fcnster", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 51.96236, "lon": 7.62571}, {"facility": "University Clinic Regensburg", "city": "Regensburg", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 49.01513, "lon": 12.10161}, {"facility": "University Clinic Rostock", "city": "Rostock", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 54.0887, "lon": 12.14049}, {"facility": "University of Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "DKD HELIOS Clinic Wiesbaden", "city": "Wiesbaden", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 50.08601, "lon": 8.24435}, {"facility": "University Clinic W\u00fcrzburg", "city": "W\u00fcrzburg", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 49.79391, "lon": 9.95121}], "contact_phone": "+497311775285", "contact_email": "johannes.dorst@uni-ulm.de", "mechanism_summary": {"compound": "Ultra-high-caloric fatty diet", "targeting_mechanism": "Compensates for hypermetabolism and weight loss by providing high-caloric nutritional support to offset increased energy expenditure and maintain body weight.", "targeting_mechanism_pmid": "29706605", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06266403", "title": "Evaluating Verbal Communication in Structured Interactions: Theoretical and Clinical Implications", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Penn State University", "summary": "The goal of this clinical trial is to learn about the effect of communicative interaction on verbal communication in people with amyotrophic lateral sclerosis (ALS) and age-matched speakers.\n\nThe question is, What are the effects of communicative interaction on verbal communication in people with ALS?\n\nParticipants will read words and sentences while they are in a solo setting and interactive setting.", "interventions": [{"type": "BEHAVIORAL", "name": "Structured Communicative Interaction"}, {"type": "BEHAVIORAL", "name": "Clear Speech"}, {"type": "BEHAVIORAL", "name": "Unstructured communicative interaction"}, {"type": "BEHAVIORAL", "name": "Clear Speech Structured Communicative Interaction"}], "start_date": "2024-11-05", "url": "https://clinicaltrials.gov/study/NCT06266403", "target_entities": [], "locations": [{"facility": "Speech Core, Pennsylvania State University", "city": "University Park", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 40.80201, "lon": -77.85639}], "contact_phone": "814-867-3373", "contact_email": "ajo150@psu.edu", "mechanism_summary": {"compound": "Structured Communicative Interaction", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06284161", "title": "QCT in ALS Diagnosis, Mechanistic Understanding and Follow-up", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Clermont-Ferrand", "summary": "Multidisciplinary management of amyotrophic lateral sclerosis (ALS) can significantly increase survival but also improve the quality of life of patients. The evaluation of cortical-spinal motor neuron damage is currently based only on the assessment of clinical data. However, the alteration of the central motor pathway and conduction can be identified and quantified by different techniques using motor-evoked potentials (MEP). The combined quadriceps test (QCT) has been developed to assess central and peripheral motor pathway conduction. This test allows to quantify central and peripheral part of a mixed disorder, and to detect physiological hyporeflexia or hyperreflexia which, in the case of suspected ALS, can lead to interpretation problems.\n\nThe evolution of the QCT parameters during the course of pathology will lead to determine the preponderance of an initial central involvement, but also its extension throughout the pathology. The study of these parameters as well as the clinical course of the disease could reveal a correlation between peripheral and central involvement. This link would provide arguments in favor of pathophysiological hypotheses of disease onset and progression. From a prognostic point of view and depending on the quantification of central and peripheral involvement, the QCT would make it possible to characterize the different ALS phenotypes. This phenotypic characterization would help identify prognostic factors at diagnosis.\n\nThe investigators propose a cohort study with the exploration of central motor neuron damage by QCT during the course of ALS in order to provide arguments for a better mechanistic understanding and follow-up of this disease with a poor prognosis.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "Quadriceps Combined Test"}], "start_date": "2022-06-28", "url": "https://clinicaltrials.gov/study/NCT06284161", "target_entities": [], "locations": [{"facility": "CHU de Clermont-Ferrand", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "RECRUITING", "lat": 45.77969, "lon": 3.08682}], "contact_phone": "+33473754963", "contact_email": "promo_interne_drci@chu-clermontferrand.fr", "mechanism_summary": {"compound": "Quadriceps Combined Test", "targeting_mechanism": "Uses motor-evoked potentials (MEP) to detect and quantify cortical-spinal motor neuron damage and assess central motor pathway alteration and conduction.", "targeting_mechanism_pmid": "32676769", "animal_results": "Dynamic neuromuscular remodeling and denervation of neuromuscular junctions precedes motor-unit loss in SOD1G93A mouse models of ALS, observed prior to symptom onset.", "animal_results_pmid": "30320556", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06450691", "title": "Modeling Amyotrophic Lateral Sclerosis With Fibroblasts", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "Amyotrophic Lateral Sclerosis (ALS) is the most common motor neuron disease in adults. This longitudinal study involves three cohorts of participants: patients with sporadic or hereditary ALS, asymptomatic individuals carrying pathogenic mutations responsible for ALS, and control subjects. In this study, a skin biopsy and blood sampling will be performed at the initial visit (M0), then at M12 (+/- 2 months) for patients, and at M36 (+/- 12 months) for asymptomatic carriers of pathogenic mutations. The aim of this research is to model ALS pathology using fibroblasts derived from the patients' skin biopsies.", "interventions": [{"type": "PROCEDURE", "name": "biopsy"}], "start_date": "2025-08-28", "url": "https://clinicaltrials.gov/study/NCT06450691", "target_entities": [], "locations": [{"facility": "CIC Neurosciences", "city": "Paris", "state": "", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "33142162472", "contact_email": "mariadelmar.amador@aphp.fr", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "Motor neuron degeneration occurs with progressive loss of neuromuscular junctions and denervation in SOD1G93A mouse models, and V1-interneuron connections onto fast motor neurons are lost as disease progresses.", "animal_results_pmid": "30320556", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06782724", "title": "Psilocybin Therapy for Psychological Distress in Palliative Patients", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Medical Center Groningen", "summary": "The goal of this clinical trial is to evaluate whether psilocybin therapy can effectively treat depression and psychological distress in adult patients with COPD, ALS, MS, or APD who have at least 6 months life expectancy. The main questions it aims to answer are:\n\n* Can psilocybin therapy safely reduce depressive symptoms compared to low-dose control?\n* Will the therapeutic effects be rapid and sustained over a 6-month period?\n\nResearchers will compare patients receiving two escalating doses of psilocybin (15mg followed by 25mg) against those receiving two low doses (1mg) to see if the higher doses lead to greater improvements in depression, anxiety, demoralization, and quality of life.\n\nParticipants will:\n\n* Attend three preparation sessions with psychotherapists (1-2 hours each)\n* Undergo two supervised psilocybin dosing sessions (6-8 hours each)\n* Complete five integration therapy sessions following the dosing sessions\n* Participate in follow-up assessments at 6 weeks, 3 months, and 6 months\n* Have access to a digital care platform and peer support groups during the 6-month follow-up period\n* Optional: Control group participants may receive one high-dose psilocybin session (25mg) after the initial study period", "interventions": [{"type": "DRUG", "name": "Psilocybin therapy"}], "start_date": "2025-07-01", "url": "https://clinicaltrials.gov/study/NCT06782724", "target_entities": ["depression_psychological_distress"], "locations": [{"facility": "National Institute of Mental Health", "city": "Prague", "state": "", "country": "Czechia", "status": "RECRUITING", "lat": 50.08804, "lon": 14.42076}, {"facility": "Bispebjerg Hospital", "city": "Copenhagen", "state": "", "country": "Denmark", "status": "RECRUITING", "lat": 55.67594, "lon": 12.56553}, {"facility": "University Medical Center Groningen", "city": "Groningen", "state": "Provincie Groningen", "country": "Netherlands", "status": "RECRUITING", "lat": 53.21917, "lon": 6.56667}, {"facility": "Champalimaud Foundation", "city": "Lisbon", "state": "", "country": "Portugal", "status": "RECRUITING", "lat": 38.72509, "lon": -9.1498}], "contact_phone": "050 361 2008", "contact_email": "r.a.schoevers@umcg.nl", "mechanism_summary": {"compound": "Psilocybin therapy", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01650818", "title": "Aerobic Exercise Training in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fondazione Salvatore Maugeri", "summary": "The purpose of this study is to compare the safety and the effects of moderate-intensity aerobic endurance training to those of an usual physical therapy intervention on exercise capacity and quality of life in patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "OTHER", "name": "Aerobic exercise training"}, {"type": "OTHER", "name": "Standard physical therapy (Stretching/Range-of-motion)"}], "start_date": "2012-01", "url": "https://clinicaltrials.gov/study/NCT01650818", "target_entities": [], "locations": [{"facility": "Fondazione Salvatore Maugeri, IRCCS - Scientific Institute of Veruno", "city": "Veruno", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.68887, "lon": 8.52863}], "contact_phone": "+39-0322-884723", "contact_email": "fabrizio.pisano@fsm.it", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07743268", "title": "Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS", "phase": "PHASE1, PHASE2", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "n-Lorem Foundation", "summary": "This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.", "interventions": [{"type": "DRUG", "name": "nL-TARDB-006"}], "start_date": "2026-02-19", "url": "https://clinicaltrials.gov/study/NCT07743268", "target_entities": ["TARDBP"], "locations": [{"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Columbia University, Irving Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "nL-TARDB-006", "targeting_mechanism": "Antisense oligonucleotide targeting TARDBP to reduce pathogenic TDP-43 protein expression.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04436510", "title": "HEALEY ALS Platform Trial - Regimen B Verdiperstat", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.\n\nRegimen B will evaluate the safety and efficacy of a single study drug, verdiperstat, in participants with ALS.", "interventions": [{"type": "DRUG", "name": "Matching Placebo"}, {"type": "DRUG", "name": "Verdiperstat"}], "start_date": "2020-07-28", "url": "https://clinicaltrials.gov/study/NCT04436510", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Healey Center for ALS at Mass General", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Verdiperstat", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07321860", "title": "This Study Evaluates the Safety, Target Engagement, and Preliminary Efficacy of Galunisertib (TGF-\u03b2R1/ALK5 Inhibitor)Combined With Nerandomilast (PDE4 Inhibitor) in GREM2-positive ALS, a Biomarker-defined Subgroup Hypothesized to Reflect Heightened TGF-\u03b2/SMAD-driven Astrocytic and Fibrotic Signaling", "phase": "PHASE2, PHASE3", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Gipfel Life Sciences GmbH", "summary": "Amyotrophic lateral sclerosis (ALS) is a relentlessly progressive neurodegenerative disorder characterized by loss of upper and lower motor neurons, leading to muscle weakness, respiratory decline, and eventual mortality. A growing body of translational and clinical evidence implicates neuroinflammation, reactive astrocytosis, and maladaptive TGF-\u03b2 signaling as central contributors to disease progression. Elevated levels of Gremlin-2 (GREM2) have been identified as a marker of dysregulated TGF-\u03b2-linked astrocytic activity and fibrotic gene programs in some ALS patients, and preclinical data suggest that attenuating these pathways may mitigate glial toxicity and improve neuronal survival.\n\nGalunisertib, a selective ATP-competitive TGF-\u03b2 receptor type I (TGF-\u03b2R1/ALK5) inhibitor, has been developed to block SMAD2/3 phosphorylation and TGF-\u03b2-mediated transcriptional programs. Meanwhile, nerandomilast, a selective PDE4B inhibitor, elevates intracellular cAMP in immune and glial cells, shifting pro-inflammatory signaling toward resolution and antagonizing secondary fibrotic and inflammatory cascades. Preclinical models show that PDE4 inhibition and TGF-\u03b2 pathway blockade concurrently reduce maladaptive glial phenotypes and fibrotic mediators.\n\nThis study investigates the combination of galunisertib + nerandomilast in ALS patients with elevated GREM2, hypothesizing that dual targeting of TGF-\u03b2-mediated astrocytic reactivity and PDE4B-regulated inflammatory signaling will translate into slowing of disease progression and favorable pharmacodynamic effects on central biomarkers of neuroinflammation and neurodegeneration.", "interventions": [{"type": "DRUG", "name": "Galunisertib + Nerandomilast Combination"}], "start_date": "2026-06-30", "url": "https://clinicaltrials.gov/study/NCT07321860", "target_entities": ["tgf_signaling", "PDE4"], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Galunisertib + Nerandomilast", "targeting_mechanism": "Dual inhibition of TGF-\u03b2 receptor 1 (ALK5) and phosphodiesterase 4 (PDE4) to reduce TGF-\u03b2/SMAD-driven astrocytic and fibrotic signaling in GREM2-positive ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04856982", "title": "A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation", "phase": "PHASE3", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogen", "summary": "The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Tofersen"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-05-17", "url": "https://clinicaltrials.gov/study/NCT04856982", "target_entities": ["SOD1"], "locations": [{"facility": "HonorHealth Neurology", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "University of California San Diego Medical Center", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "California Pacific Medical Center Research Institute", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Holy Cross Hospital", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "University of Miami School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "The Emory Clinic", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital, MA", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Columbia University Medical center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Austin Neuromuscular Center", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Macquarie University Hospital", "city": "Macquarie Park", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.78105, "lon": 151.12757}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Hospital Sao Paulo", "city": "S\u00e3o Paulo", "state": "S\u00e3o Paulo", "country": "Brazil", "status": "", "lat": -23.5475, "lon": -46.63611}, {"facility": "PSEG Centro de Pesquisa Clinica", "city": "S\u00e3o Paulo", "state": "", "country": "Brazil", "status": "", "lat": -23.5475, "lon": -46.63611}, {"facility": "University of Calgary", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Genge Partners", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Groupe Hospitalier Pitie-Salpetriere", "city": "Paris", "state": "Paris", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Universitaetsklinikum Ulm", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Azienda Ospedaliero-Universitaria Citt\u00e0 della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Kagoshima University Hospital", "city": "Kagoshima", "state": "Kagoshima-ken", "country": "Japan", "status": "", "lat": 31.56667, "lon": 130.55}, {"facility": "University of Tokyo Hospital", "city": "Bunky\u014d City", "state": "Tokyo-To", "country": "Japan", "status": "", "lat": 35.5331, "lon": 139.4217}, {"facility": "NeuroProtect Sp. z o.o.", "city": "Warsaw", "state": "Masovian Voivodeship", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Centrum Medyczne NeuroProtect", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Hanyang University Seoul Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Hospital Universitari i Politecnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "University Hospital of Umea", "city": "Ume\u00e5", "state": "V\u00e4sterbotten County", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "Norrlands Universitetssjukhus", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "University of Sheffield", "city": "Sheffield", "state": "South Yorkshire", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Tofersen", "targeting_mechanism": "Antisense oligonucleotide that reduces mutant superoxide dismutase 1 (SOD1) protein expression.", "targeting_mechanism_pmid": "37975798", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03377868", "title": "Optic Coherence Tomography in Patients With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "American University of Beirut Medical Center", "summary": "Amyotrophic Lateral Sclerosis (ALS) is an adult-onset, devastating, neurodegenerative disease characterized by the loss of cortical, brain stem, and spinal motor neurons. Visual evoked potentials studies in patients with ALS suggest visual pathway involvement. Optic coherence tomography (OCT) is a non-invasive cross-sectional imaging modality measuring the optical reflections in biological tissues. The main objective of this observational cohort study is to explore the correlation between changes on OCT retinal parameters and and clinical disability as measured by the ALS Functional Rating Scale (ALS-FRS-r) in patients with ALS at baseline, 3 and 6 months. A secondary objective is to explore the correlation between changes in retinal OCT parameters and pulmonary function tests (FVC and FEV1) in this cohort of patients with ALS. A parallel cohort of healthy age and sex matched subjects will participate as controls to obtain reference values of their retinal layers' thickness at baseline, 3 and 6 months.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "Optic coherence tomography"}, {"type": "DIAGNOSTIC_TEST", "name": "Pulmonary function test"}], "start_date": "2018-01-03", "url": "https://clinicaltrials.gov/study/NCT03377868", "target_entities": [], "locations": [], "contact_phone": "+9611350000", "contact_email": "am132@aub.edu.lb", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05621213", "title": "Satisfaction of Patients With Amyotrophic Lateral Sclerosis Regarding Home Assisted Teleconsultation", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AGIR \u00e0 Dom", "summary": "Satisfaction of patients with amyotrophic lateral sclerosis under non-invasive ventilation regarding home assisted teleconsultation", "interventions": [{"type": "OTHER", "name": "Teleconsultation assisted by a physiotherapist or nurse"}], "start_date": "2023-01-13", "url": "https://clinicaltrials.gov/study/NCT05621213", "target_entities": [], "locations": [{"facility": "CHU Grenoble Alpes", "city": "Grenoble", "state": "France", "country": "France", "status": "", "lat": 45.17869, "lon": 5.71479}, {"facility": "CHU Montpellier", "city": "Montpellier", "state": "France", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01746381", "title": "Non-invasive Ventilation in Amyotrophic Lateral Sclerosis (ALS) Using the iVAPS Mode", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Marta Kaminska", "summary": "Home non-invasive ventilation (NIV) is the standard-of-care as initial therapy for patients with ALS with worsening symptoms or deteriorating respiratory function, and has been recommended by the American Academy of Neurology (AAN) practice parameter for ALS.\n\nThis study will compare the current standard BiST mode of ventilation with the new iVAPS mode. The main study hypothesis is that the iVAPS mode, initiated in a single daytime trial, will result in a reduction of the number of respiratory therapist interventions and changes in ventilator settings as compared with the standard BiST mode. This will be assessed over a period of one year. In addition this study will test whether the iVAPS mode is superior to BiST mode with respect to: comfort and ease of use; improvement in nighttime and daytime symptoms of hypoventilation; compliance (hours used per day); physiologic parameters (daytime and overnight oxygen saturation and transcutaneous CO2 level).", "interventions": [{"type": "DEVICE", "name": "Non-invasive home ventilation"}], "start_date": "2014-02", "url": "https://clinicaltrials.gov/study/NCT01746381", "target_entities": [], "locations": [{"facility": "Montreal Chest Institute", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07674667", "title": "FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Trace Neuroscience, Inc.", "summary": "The FUNCtion Amyotrophic Lateral Sclerosis (ALS) trial is a randomized, double-blind, placebo-controlled Phase 1/2 trial to evaluate the safety and tolerability of TRCN-1023 in adults living with ALS. TRCN-1023 is an investigational medicine given as a single injection into the fluid surrounding the spine (intrathecal injection). The trial will also assess how the body processes the drug and whether it shows early signs of benefit over 24 weeks.", "interventions": [{"type": "DRUG", "name": "TRCN-1023"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2026-08-20", "url": "https://clinicaltrials.gov/study/NCT07674667", "target_entities": ["UNC13A"], "locations": [{"facility": "TUM Klinikum Rechts der Isar", "city": "Munich", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 48.13743, "lon": 11.57549}, {"facility": "University Medical Center Rostock", "city": "Rostock", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 54.0887, "lon": 12.14049}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "University of Sheffield", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "+1 415-390-9509", "contact_email": "Clinicaltrials@traceneuro.com", "mechanism_summary": {"compound": "TRCN-1023", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07571200", "title": "A Master Protocol (OLMP): A Study of LY4256984 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Eli Lilly and Company", "summary": "Study OLMP is a master protocol that will support a collection of individual sub studies that share key design components. Participants from the originator study OWAA (NCT07100119) will be assigned to the appropriate study treatment group: Sporadic Amyotrophic Lateral Sclerosis OL01 (NCT07571174). The studies aim to evaluate the safety and tolerability of different treatments in participants with Amyotrophic Lateral Sclerosis (ALS) that will last at least 96 weeks.", "interventions": [{"type": "DRUG", "name": "LY4256984"}], "start_date": "2026-05-14", "url": "https://clinicaltrials.gov/study/NCT07571200", "target_entities": [], "locations": [{"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "NOT_YET_RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "University Hospital - London Health Sciences Centre", "city": "London", "state": "", "country": "Canada", "status": "NOT_YET_RECRUITING", "lat": 42.98339, "lon": -81.23304}, {"facility": "McGill University Health Centre", "city": "Montreal", "state": "", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}, {"facility": "Sunnybrook Research Institute", "city": "Toronto", "state": "", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}, {"facility": "Universit\u00e4tsklinikum Schleswig-Holstein", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 53.86893, "lon": 10.68729}, {"facility": "Universit\u00e4tsmedizin Rostock", "city": "Rostock", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 54.0887, "lon": 12.14049}, {"facility": "Universitaetsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "NOT_YET_RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "NOT_YET_RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Universitari de Bellvitge", "city": "L'Hospitalet de Llobregat", "state": "", "country": "Spain", "status": "NOT_YET_RECRUITING", "lat": 41.35967, "lon": 2.10028}, {"facility": "Hospital Universitari i Politecnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "NOT_YET_RECRUITING", "lat": 39.47391, "lon": -0.37966}], "contact_phone": "1-317-615-4559", "contact_email": "LillyTrials@Lilly.com", "mechanism_summary": {"compound": "LY4256984", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04691011", "title": "New Magnetic Resonance Imaging Biomarkers in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique Hopitaux De Marseille", "summary": "Amyotrophic lateral sclerosis (ALS) is a disabling and rapidly progressive neurodegenerative disorder. There is no treatment that significantly slows progression. Our project aims to find new biomarkers in MRI at three levels: cerebral, medullary and muscular. These markers could allow an earlier diagnosis of the disease by showing more specific lesions of ALS and to quantify these lesions to measure the progression of the disease. This study will use advanced Magnetic Resonance Imaging (MRI) techniques High field (3T) and very high field (7T) MRI. Results from neurological and electrophysiological tests will be compared to the MRI. Subjects will be recruited from ALS center of Marseille, France. MRI will be done on ALS patients at baseline, at 3 month and at 6 month intervals.", "interventions": [{"type": "OTHER", "name": "Muscular MRI"}, {"type": "OTHER", "name": "Electrophysiological exam"}, {"type": "OTHER", "name": "Brain MRI"}, {"type": "OTHER", "name": "Spinal cord MRI"}], "start_date": "2021-06-16", "url": "https://clinicaltrials.gov/study/NCT04691011", "target_entities": [], "locations": [{"facility": "Shahram Attarian", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04393467", "title": "Transcranial Static Magnetic Field Stimulation (tSMS) in Amyotrophic Lateral Sclerosis (ALS).", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Campus Bio-Medico University", "summary": "This study aims to evaluate safety and efficacy of tSMS in ALS patients and to obtain preliminary data about the effects of tSMS on cortical excitability.\n\nTo this purpose, 40 ALS patients will be recruited and randomized to real or sham tSMS. After at least 3 months follow-up, they will undergo tSMS, daily for 120 min, at home, for 6 consecutive months.\n\nClinical status will be tested before, during and after the stimulation period. Moreover, cortical excitability will be tested by transcranial magnetic stimulation (TMS) before and after the stimulation period.", "interventions": [{"type": "DEVICE", "name": "tSMS"}, {"type": "DEVICE", "name": "sham tSMS"}], "start_date": "2020-05-31", "url": "https://clinicaltrials.gov/study/NCT04393467", "target_entities": [], "locations": [{"facility": "Department of Neurology and Laboratory of Neuroscience, Istituto Auxologico Italiano, IRCCS", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.46427, "lon": 9.18951}, {"facility": "Neurology Unit, Campus Biomedico University", "city": "Rome", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 41.89193, "lon": 12.51133}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03613571", "title": "A Study to Evaluate the Safety, Tolerability and Efficacy of ILB in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "TikoMed AB", "summary": "This is a Phase 2a single-centre, open single-arm study in patients with Amyotrophic Lateral Sclerosis (ALS) of intermediate progression rate. Eligible subjects will be administered weekly doses of ILB. A total of 5 subcutaneous (s.c.) doses will be administered at the study clinic.\n\nThe study consists of 10 visits; One 2-part screening visit, 5 ILB administration visits, and 3 follow-up visits. Each individual patient's study participation will be 4 months, including the screening and follow-up visits. Fifteen patients are planned to be included.\n\nThe primary objective of the study is to evaluate the safety and tolerability of ILB in patients diagnosed with ALS.\n\nILB is a solution for subcutaneous (s.c.) injection in saline solution. The dose administered will depend on the subject's body weight at the second study visit, prior to the first ILB administration.\n\nNo formal sample size calculation has been performed for this study. The proposed sample size is considered sufficient in this early phase 2 development to provide adequate information on the patients. Categorical data will be presented as counts and percentages. Continuous data will be summarised using descriptive statistics.", "interventions": [{"type": "DRUG", "name": "ILB"}], "start_date": "2018-08-15", "url": "https://clinicaltrials.gov/study/NCT03613571", "target_entities": ["ilb"], "locations": [{"facility": "Sahlgrenska University Hospital", "city": "Gothenburg", "state": "", "country": "Sweden", "status": "", "lat": 57.70716, "lon": 11.96679}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "ILB", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02236065", "title": "Combination Therapy of Cord Blood and G-CSF for Patients With Brain Injury or Neurodegenerative Disorders", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "MinYoung Kim, M.D.", "summary": "This open label trial is conducted to investigate the efficacy and safety of the combination therapy of allogeneic umbilical cord blood (UCB) and granulocyte-colony stimulating factor (G-CSF) for patients with brain injury or neurodegenerative disorders.", "interventions": [{"type": "PROCEDURE", "name": "Umbilical cord blood therapy"}, {"type": "BIOLOGICAL", "name": "Filgrastim"}], "start_date": "2014-08", "url": "https://clinicaltrials.gov/study/NCT02236065", "target_entities": ["neuroinflammation", "stem_cell_transplantation"], "locations": [{"facility": "CHA Bundang Medical Center, CHA University", "city": "Seongnam-si", "state": "Gyeonggi-do", "country": "South Korea", "status": "", "lat": 37.43861, "lon": 127.13778}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Umbilical cord blood and Filgrastim", "targeting_mechanism": "Stem cell therapy modulating neuroinflammation to promote neuroprotection and tissue repair.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06810219", "title": "Augmented Reality BCI Longitudinal Study for Persons With Late Stage ALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cognixion", "summary": "The goal of this study is refine the usability of a BCI based communication platform.\n\nThe study will take place in the greater Los Angeles area and will enroll up to 10 participants with late stage ALS. Each subject will receive a Cognixion Axon-R augmented reality brain computer interface and associated communication software. The study duration is 3 months for each participant.\n\nThe key questions that will be addressed in this study are:\n\n1. How quickly can participants learn and gain confidence with a pure BCI interface.\n2. How effective are alternate input modalities including eye tracking for this use case.\n3. Identify the extent to which generative AI based personalization impacts the communication quality.\n\nKey measures include:\n\nITR - information transfer rate SUS - system usability scale", "interventions": [{"type": "DEVICE", "name": "Cognixion ONE"}], "start_date": "2025-01-20", "url": "https://clinicaltrials.gov/study/NCT06810219", "target_entities": [], "locations": [{"facility": "Cognixion", "city": "Santa Barbara", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 34.42083, "lon": -119.69819}], "contact_phone": "8053200774", "contact_email": "chris@cognixion.com", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06656702", "title": "Effects of Psilocybin in Patients With Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "This study aims to study the feasibility of psilocybin therapy for patients with Amyotropic Lateral Sclerosis (ALS) with depressed mood. The secondary objective is to assess its impact on depression, quality of life, hopelessness, and functional status in this patient population.", "interventions": [{"type": "DRUG", "name": "Psilocybin"}], "start_date": "2025-04-09", "url": "https://clinicaltrials.gov/study/NCT06656702", "target_entities": ["psilocybin", "neuroinflammation"], "locations": [{"facility": "Johns Hopkins Center for Psychedelic and Consciousness Research", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "410-502-0495", "contact_email": "emosmil1@jhmi.edu", "mechanism_summary": {"compound": "Psilocybin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06844370", "title": "Home Versus Hospital Based NIV Care in MND", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Papworth Hospital NHS Foundation Trust", "summary": "Non-invasive ventilation (NIV) is commonly offered to people with Motor Neurone Disease (MND) who have breathing difficulties. It improves their quality of life and can prolong life by 6 months or more. It is initially used at night and typically set up during a hospital admission. By the time that they develop respiratory failure and need to start NIV, however, most patients require wheelchairs or have other significant health problems. Repeated travel to hospitals is increasingly difficult with increasing disability. It is possible to start and monitor NIV treatment at home. This may be more convenient for selected patients, though starting NIV is quite complex; it is not known if home treatment is as safe and effective as hospital-based treatment.\n\nTo establish this, 60 patients with MND who have indications for NIV will be recruited. They will be randomly allocated to a home-based treatment (home NIV set up plus home visits supported by telemonitoring) or hospital-based care (inpatient NIV set up plus outpatient NIV monitoring) and followed up at 1, 4 and 7 months. Alongside measures of treatment effectiveness, assessment of patient and carer preferences, quality of life, and cost-effectiveness will be undertaken.\n\nIn the additional qualitative part on this study, interviews with patients who took part in the main study and their carers will be conducted to understand in more depth their perspective on what makes for a good or bad experience with NIV, how the environment (home vs hospital) influences their NIV experience and what personal factors determine NIV use. Findings from the interviews will inform the design of a truly patient-centred NIV service.", "interventions": [{"type": "OTHER", "name": "Non-invasive ventilation"}], "start_date": "2024-03-06", "url": "https://clinicaltrials.gov/study/NCT06844370", "target_entities": [], "locations": [{"facility": "Royal Paworth Hospital", "city": "Cambridge", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 52.2, "lon": 0.11667}], "contact_phone": "01223639619", "contact_email": "dariusz.wozniak@nhs.net", "mechanism_summary": {"compound": "Non-invasive ventilation", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00860951", "title": "P300 Brain Computer Interface Keyboard to Operate Assistive Technology", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "The purpose of this research is to develop tools enable people who are paralyzed to operate technology and access computers. These tools are called brain computer interfaces (BCIs). BCIs would let a person use brain signals to operate technology.", "interventions": [{"type": "DEVICE", "name": "Brain Computer Interface Keyboard"}], "start_date": "2008-09", "url": "https://clinicaltrials.gov/study/NCT00860951", "target_entities": [], "locations": [{"facility": "University of Michigan Direct Brain Interface Project", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Wadsworth Center", "city": "Albany", "state": "New York", "country": "United States", "status": "", "lat": 42.65258, "lon": -73.75623}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Brain Computer Interface Keyboard", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04866771", "title": "Remotely Supervised tDCS for Slowing ALS Disease Progression", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Illinois at Chicago", "summary": "Most ALS care is centered on patient support and symptom management, making rehabilitation an integral aspect for slowing disease progression, prolonging life span, and increasing quality of life. Brain stimulation has been increasingly explored as a promising neuromodulatory tool to prime motor function in several neurological disorders. We propose a novel mechanism using remotely supervised brain stimulation to preserve motor function in individuals with ALS. This project will also aim to explore the effectiveness of brain stimulation on upper and lower motor neuron mechanisms in individuals with ALS.", "interventions": [{"type": "OTHER", "name": "Transcranial Direct Current Stimulation (tDCS)"}, {"type": "OTHER", "name": "Sham tDCS + anodal tDCS"}], "start_date": "2021-08-27", "url": "https://clinicaltrials.gov/study/NCT04866771", "target_entities": [], "locations": [{"facility": "Brain Plasticity Lab", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Transcranial Direct Current Stimulation (tDCS)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07473765", "title": "Virtual Reality for Anxiety Management in Persons With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Horizon Health Network", "summary": "Virtual Reality (VR) is gaining traction as a new and innovative leisure to augment healthcare services. Several benefits of the leisure experience, such as distraction and full sensory immersion, have demonstrated a potential to significantly impact the field of healthcare through pain reduction, anxiety reduction, and is seen as an innovative approach to motor learning. Persons with ALS (pwALS) have a high prevalence of anxiety over the course of their illness, which has a negative impact on their quality of life, and the quality of life of those closest to them. The use of VR for anxiety management and subsequent quality of life improvement has yet to be explored in the ALS population.\n\nFor individuals with ALS, VR can be both (1) an escape from the reality of living day to day with a progressive fatal diagnosis; and (2) the opportunity to potentially improve anxiety, both of which are linked to the quality of life of individuals living with ALS. Our hypothesis is that a simple and accessible home VR-guided relaxation exercise program can improve subjective anxiety symptoms in a person living with ALS and subsequently improve quality of life.", "interventions": [{"type": "BEHAVIORAL", "name": "Virtual Reality Guided Breathing Exercise"}], "start_date": "2026-04-01", "url": "https://clinicaltrials.gov/study/NCT07473765", "target_entities": [], "locations": [], "contact_phone": "506-447-4379", "contact_email": "shane.a.mccullum@horizonnb.ca", "mechanism_summary": {"compound": "Virtual Reality Guided Breathing Exercise", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01640067", "title": "Human Neural Stem Cell Transplantation in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Azienda Ospedaliera Santa Maria, Terni, Italy", "summary": "Primary aim of the trial\n\n1. to verify safety and tolerability of expanded human fetal neural stem cells\n2. to verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model\n3. to recognize each change in patient's quality of life\n\nSecondary aim of the trial\n\n1. assessment of the impact of human neural stem cells transplantation on the disability of ALS in a clinically significant and measurable way\n2. Assessment of the impact of human neural stem cells transplantation on mortality (all causes)", "interventions": [{"type": "BIOLOGICAL", "name": "Human Neural Stem Cells"}], "start_date": "2011-12", "url": "https://clinicaltrials.gov/study/NCT01640067", "target_entities": ["neural_stem_cell_transplantation"], "locations": [{"facility": "Azienda Ospedaliera Santa Maria", "city": "Terni", "state": "Terni", "country": "Italy", "status": "", "lat": 42.56335, "lon": 12.64329}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Human Neural Stem Cells", "targeting_mechanism": "Neural stem cells differentiate into support cells such as astrocytes, oligodendrocytes, or microglia that produce growth factors and anti-inflammatory cytokines to support degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Human neural progenitor cells transduced with GDNF differentiated to astrocytes and protected spinal motor neurons in animal models.", "animal_results_pmid": "36064599", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07454733", "title": "Do Video Recordings of Multidisciplinary Clinics Improve Quality of Life for People With ALS and Their Caregivers?", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Trustees of Dartmouth College", "summary": "Amyotrophic lateral sclerosis (ALS) is a fatal, rare neurodegenerative disease affecting 30,000 people in the United States. The gold standard of care for people with ALS is multidisciplinary clinics (MDC). In these multidisciplinary clinics, which occur every 3 to 4 months, people with ALS see up to 12 different healthcare providers (e.g., speech therapy, physical therapy, the ALS doctor). These clinics can last from three to five hours, and across these three to five hours people with ALS and their caregivers receive a lot of information that is critical to the care and quality of life for people with ALS. However, this information can be difficult to remember given the large amount of information that is conveyed. The current standard for providing take-home information about the visit is to provide patients with a written after-visit summary and access to their doctor's notes about the visit, typically through the patient portal. This study tests whether providing participants with video recordings of their MDC visits improves their quality of life and the quality of life of their caregivers. The study will enroll 400 pairs of people with ALS and their caregivers from eight different sites in the United States. Half of the participants in the study will receive their after-visit summary notes (the NOTES condition) and the other half of the participants will receive both their summary notes, but will also receive video recordings of their MDC visits that they can watch on their own at home (the VIDEO condition). The study will last for 12 months, with participants receiving NOTES or VIDEO at each of their regularly-scheduled MDCs during the 12 months. The study will test whether caregiver and patient participants in the VIDEO condition experience better quality of life than those in the NOTES condition at 1 month, 6 months, and 12 months from study enrollment. The results of this study will help determine what is the most effective approach to communicating MDC information to people with ALS and their caregivers.", "interventions": [{"type": "BEHAVIORAL", "name": "Video recording"}, {"type": "BEHAVIORAL", "name": "Notes Instruction"}], "start_date": "2026-06-01", "url": "https://clinicaltrials.gov/study/NCT07454733", "target_entities": [], "locations": [{"facility": "Mayo Clinic Scottsdale", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "Mayo Clinic Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Dartmouth-Hitchcock Health", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}, {"facility": "Penn State Health Milton S. Hersey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "University of Pittsburgh Medical Center", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "University of Wisconsin Health", "city": "Madison", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.07305, "lon": -89.40123}], "contact_phone": "603-646-7016", "contact_email": "paul.j.barr@dartmouth.edu", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07410806", "title": "HEALEY ALS Platform Trial - Regimen I NUZ-001", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.\n\nRegimen I will evaluate the safety and efficacy of a single study drug, NUZ-001, in participants with ALS.", "interventions": [{"type": "DRUG", "name": "NUZ-001"}, {"type": "DRUG", "name": "Matching placebo"}], "start_date": "2026-02-25", "url": "https://clinicaltrials.gov/study/NCT07410806", "target_entities": ["nuz_001"], "locations": [{"facility": "Healey Center for ALS at Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "NUZ-001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04820478", "title": "Efficacy and Tolerability of Beta Hydroxybutyrate Ester in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Ulm", "summary": "Weight loss is a known negative prognostic factor in amyotrophic lateral sclerosis (ALS). One potential mechanism of weight loss in ALS is a disturbance of the mitochondrial complex I which causes an energy deficit in affected cells. Over the last years, various interventional studies targeting the energy deficit in ALS yielded promising results; however,it is still unclear which kind of nutrition or nutritional supplement is most beneficial. Ketone bodies represent a logical therapeutic option in ALS as ketone bodies are an extremely high-energetic substrate which yields the double amount of adenosine triphosphate (ATP) per mole compared to glucose. The human liver is able to synthesize ketone bodies (beta-hydroxybutyrate, acetone, and aceto-acetate) from fat in times of glucose shortage, for example after a prolonged period of fasting. This metabolic shift is the underlying principle of the ketogenic diet, a carbohydrate-free, fat-rich diet which has been successfully tested in other neurodegenerative diseases such as Alzheimer's and Parkinson's disease. In the ALS mouse model, a ketogenic diet was associated with a slower decline of motor function. However, a ketogenic diet is difficult to implement in ALS as it requires a long-term change of eating habits, which is difficult to achieve due to progressive dysphagia, fast worsening of general condition, and limited survival. Therefore, the direct administration of ketone bodies yields a more realistic alternative in ALS as it is easy to apply and allows to maintain the usual eating habits. In this study, we hypothesize that the administration of 3 x 10 g beta hydroxybutyrate ester per day (in addition to normal food intake and the standard medication of 2 x 50 mg riluzole) slows down disease progression as measured by neurofilament light chains (NfL) in serum after 6 months compared to placebo. Power calculation relies on the results of the lipids and calories for ALS (LIPCAL-ALS) study which tested the effect of a high-caloric fatty nutritional supplement in ALS. The study revealed that NfL serum values declined significantly in the intervention group while remaining stable in the placebo group over the course of the study. Assuming a similar effect size for ketone bodies, we calculated that 76 patients had to be included in the current trial.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Beta Hydroxybutyrate Ester (KetoneAid KE4)"}, {"type": "DIETARY_SUPPLEMENT", "name": "Placebo"}], "start_date": "2022-04-01", "url": "https://clinicaltrials.gov/study/NCT04820478", "target_entities": ["mitochondrial_complex_i"], "locations": [{"facility": "University of Ulm", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Beta Hydroxybutyrate Ester (KetoneAid KE4)", "targeting_mechanism": "Beta hydroxybutyrate ester provides an alternative energy substrate to address mitochondrial complex I dysfunction and energy deficit in ALS cells.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05053035", "title": "A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Alector Inc.", "summary": "A phase 2 double-blind, placebo-controlled study of AL001 in participants with C9orf72-associated ALS.", "interventions": [{"type": "DRUG", "name": "AL001"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-09-02", "url": "https://clinicaltrials.gov/study/NCT05053035", "target_entities": ["C9orf72"], "locations": [{"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "AL001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05107349", "title": "Cell Signaling, Reinnervation and Metabolism in Kennedy Disease and Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "Amyotrophic lateral sclerosis (ALS), is a rapidly progressive neurodegenerative disorder, usually leading to death from respiratory failure in 3-5 years. Riluzole, the only drug currently available, only modestly prolongs survival and does not improve muscle strength or function. In ALS, loss of functional motor neurons is initially compensated for by collateral reinnervation and strength is preserved. In the majority of ALS patients, as the disease progresses, compensation fails leading to progressive muscle weakness. Conversely, in long-term ALS survivors, slow functional decline is correlated with their ability to maintain a successful compensatory response to denervation over time. Compensatory collateral reinnervation is thus essential for functional motor preservation and survival, and elucidation of the molecular mechanisms involved is crucial to help identify new therapeutic targets. Energy metabolism and glucose homeostasis modifications also influence disease clinical course but the mechanisms by which they contribute to the progression of ALS are unknown. Weight loss is an independent negative prognostic factor for survival and, by contrast, ALS risk and progression are decreased in individuals with high body mass index and non-insulin-dependent diabetes mellitus. Insulin shares many common steps in its signaling pathways with insulin-like growth factor 1 (IGF-1), and is thus at the interface between glucose homeostasis regulation and maintenance of muscle mass. However, the contribution of insulin signaling to preservation of muscle innervation and function in ALS has never been investigated.\n\nWith this study, we aim to determine the role of insulin signaling pathways in maintenance of collateral reinnervation and muscle function in ALS. We will also investigate the link with the disease-modifying effect of metabolic and glucose homeostasis perturbations, by identifying the contribution of metabolic profiles to preservation of skeletal muscle innervation and motor function in patients with ALS. For this purpose, we will determine the whole-body and skeletal muscle metabolic profiles of 20 patients with ALS and correlate these results to collateral reinnervation ability quantified on muscle biopsy specimens. For each patient, we will use both clinical and electrophysiological methods to evaluate motor function and motor neuron loss over time. Body composition, insulin secretion, insulin resistance level and serum concentrations of IGF-1 axis components will be determined. A motor point muscle biopsy will be performed for morphological analysis of neuromuscular junctions and quantification of innervation by confocal microscopy. Activation of insulin/IGF-1 canonical signaling pathways and metabolic pathways of glucose homeostasis will be quantified in muscle specimens. Skeletal muscle and whole-body metabolic parameters will be analyzed together and correlated with clinical assessment of motor function, electrophysiological data, and innervation quantification results. For comparison, 10 healthy subjects of similar age and 10 patients with spinal and bulbar muscular atrophy - a slowly progressive motor neuron disorder with maintenance of effective collateral reinnervation - will be used as controls. This study will be the first to address the question of the contribution of insulin signaling pathways and metabolic profiles in maintenance of muscle reinnervation and function in ALS patients. The molecular mechanisms identified will be new targets for future treatments promoting compensatory reinnervation and slowing disease progression in ALS. Ultimately, this translational project could have a significant therapeutic impact in disorders with muscle denervation and collateral reinnervation as a compensatory mechanism, such as spinal muscle atrophy or peripheral neuropathies.", "interventions": [{"type": "PROCEDURE", "name": "Muscle biopsy"}], "start_date": "2022-12-12", "url": "https://clinicaltrials.gov/study/NCT05107349", "target_entities": [], "locations": [{"facility": "Piti\u00e9-Salp\u00eatri\u00e8re Hospital", "city": "Paris", "state": "", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "0142162467", "contact_email": "nathalie.cormand@aphp.fr", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00877604", "title": "Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta", "summary": "The preclinical rationale for tauroursodeoxycholic acid (TUDCA) use in treating patients with amyotrophic lateral sclerosis (ALS) stems from the demonstration of antioxidant, antiapoptotic and neuroprotective properties of TUDCA in the central nervous system (CNS), both in vitro and in vivo models.\n\nThis protocol is meant for assessing if the addition of TUDCA to the conventional therapy can improve the therapeutic outcome in patients affected by ALS.\n\nSafety will be assessed for all subjects, for the entire duration of the study. 30 patients affected by ALS with site of onset in the limbs will be recruited.\n\nAll enrolled subjects will continue receiving riluzole at the same regimen as before entering the trial. Based on an appropriate random code, subjects will be divided into two groups of equal size treated, after a lead-in period of 3 months, by oral route with TUDCA at the dose 2 g daily for 1 year or with identical placebo by oral route at the same dosing schedule, under double-blind conditions.\n\nEvery concomitant and/or supportive therapy will be admitted.\n\nEvaluation criteria:\n\nEfficacy. The proportion of responder patients in the two treatment groups was the primary outcome measure of the study. Responder patients were defined as those subjects showing an improvement of at least 15% in the ALSFRS-R (2) slope during the treatment period as compared to the lead-in period. This threshold was chosen based according to the consensus conference on designing and implementing clinical trials in ALS (3).\n\nOther parameters will include ALSFRS-R at study end, FVC%, the SF-36 quality of life rating scale, time to tracheotomy from starting of study medication dosing (if appropriate), survival Time from starting of study medication dosing (if appropriate), Medical Research Council scores for right and left muscle groups.\n\nSafety. Incidence, severity and type of adverse events; changes in clinical laboratory findings.", "interventions": [{"type": "DRUG", "name": "tauroursodeoxycholic acid (TUDCA)"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2008-06", "url": "https://clinicaltrials.gov/study/NCT00877604", "target_entities": ["oxidative_stress", "apoptosis"], "locations": [{"facility": "Fondazione IRCCS Istituto neurologico Carlo Besta", "city": "Milan", "state": "Milan", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "tauroursodeoxycholic acid (TUDCA)", "targeting_mechanism": "Antioxidant, antiapoptotic and neuroprotective agent acting in the central nervous system", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07636538", "title": "Auto-calibrating System for Upper Limb Disability Assessment, Neurological and Occupational Rehabilitation", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Pavia", "summary": "This interventional, multicenter, low-intervention clinical trial aims to evaluate the usability, feasibility, safety, and preliminary clinical impact of a robotic rehabilitation system designed for upper limb rehabilitation in adults with neurological disorders, including Parkinson's disease (PD), Amyotrophic Lateral Sclerosis (ALS), post-stroke sequelae, and Mild Cognitive Impairment (MCI).\n\nThe system under study combines a collaborative robot (cobot), inertial sensors, and a graphical user interface capable of supporting reaching exercises, trajectory tracking activities, and cognitive exergames, while also enabling automatic acquisition and visualization of patient performance data.\n\nThe main questions the study aims to answer are:\n\nIs the investigational robotic rehabilitation system usable and feasible in neurological patients undergoing upper limb rehabilitation? Is the use of the device safe for both patients and healthcare operators? Does the addition of robotic-assisted rehabilitation to conventional therapy improve upper limb motor performance, cognitive function, and quality of life compared with conventional rehabilitation alone? Do movement measurements collected by the system correlate with standard clinical assessment scales?\n\nResearchers will compare conventional rehabilitation therapy plus robotic-assisted rehabilitation with conventional rehabilitation therapy alone to evaluate the impact of the device on motor, cognitive, and psychosocial outcomes.\n\nThirty participants will be randomized into two parallel treatment groups. Both groups will receive 12 sessions of conventional rehabilitation therapy lasting 60 minutes each, three times per week. Participants assigned to the experimental group will additionally receive robotic-assisted rehabilitation sessions of up to 30 minutes supervised by rehabilitation staff.\n\nParticipants will undergo:\n\nBaseline collection of demographic and clinical information; Motor, cognitive, and activities of daily living assessments using standardized clinical scales; Conventional rehabilitation therapy sessions; Robotic-assisted upper limb rehabilitation exercises, including task-oriented and trajectory-tracking activities (experimental group only); Monitoring of vital parameters and adverse events during device use; Final evaluation of usability, psychosocial impact, patient satisfaction, motor and cognitive outcomes, and safety.", "interventions": [{"type": "DEVICE", "name": "Auto-calibrating System for Upper Limb disability Assessment, neurological and occupational Rehabilitation"}, {"type": "OTHER", "name": "conventional therapy"}], "start_date": "2026-06", "url": "https://clinicaltrials.gov/study/NCT07636538", "target_entities": [], "locations": [], "contact_phone": "+393348941743", "contact_email": "cira.fundaro@icsmaugeri.it", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04293484", "title": "Cortico-Spinal tDCS as Rehabilitative Intervention in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a motor neuron disease, which is a group of neurological disorders that selectively affect motor neurons, the cells that control voluntary muscles of the body. The disorder causes muscle weakness and atrophy throughout the body due to the degeneration of the upper and lower motor neurons. Current drugs approved for ALS treatment only modestly slow disease progression.\n\nTranscranial direct current stimulation (tDCS) is a non-invasive technique, which has been demonstrated to modulate cerebral excitability in several neurodegenerative disorders and modulate intracortical connectivity measures.\n\nIn this randomized, double-blind, sham-controlled study followed by an open-label phase, the investigators will evaluate whether a repetition of two-weeks' treatment with bilateral motor cortex anodal tDCS and spinal cathodal tDCS, after a six months interval, may further outlast clinical improvement in patients with amyotrophic lateral sclerosis and can modulate intracortical connectivity, at short and long term.", "interventions": [{"type": "DEVICE", "name": "Anodal bilateral motor cortex and cathodal spinal tDCS"}, {"type": "DEVICE", "name": "Sham bilateral motor cortex and sham spinal tDCS"}], "start_date": "2018-03-12", "url": "https://clinicaltrials.gov/study/NCT04293484", "target_entities": ["motor_cortex_stimulation"], "locations": [{"facility": "AO Spedali Civili", "city": "Brescia", "state": "BS", "country": "Italy", "status": "", "lat": 45.53558, "lon": 10.21472}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Transcranial direct current stimulation (tDCS)", "targeting_mechanism": "Non-invasive neuromodulation technique that applies direct electrical current to the motor cortex and spinal cord to modulate neural activity", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03280056", "title": "Safety and Efficacy of Repeated Administrations of NurOwn\u00ae in ALS Patients", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Brainstorm-Cell Therapeutics", "summary": "This study will evaluate the safety and efficacy of repeated administration of NurOwn\u00ae (MSC-NTF cells) therapy, which is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patient's own bone marrow, propagated ex vivo and induced to secrete Neurotrophic factors (NTFs).\n\nThe autologous NurOwn\u00ae (MSC-NTF cells) are back-transplanted into the patient intrathecally by standard lumbar puncture where neurons and glial cells are expected to take up the neurotrophic factors secreted by the transplanted cells", "interventions": [{"type": "BIOLOGICAL", "name": "NurOwn\u00ae (MSC-NTF cells)"}, {"type": "OTHER", "name": "Placebo"}, {"type": "OTHER", "name": "Bone Marrow aspiration"}], "start_date": "2017-08-28", "url": "https://clinicaltrials.gov/study/NCT03280056", "target_entities": ["neurotrophic_factors"], "locations": [{"facility": "University of California Irvine Alpha Stem Cell Clinic", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "NurOwn\u00ae (MSC-NTF cells)", "targeting_mechanism": "Autologous bone marrow-derived mesenchymal stromal cells engineered to secrete neurotrophic factors that provide neuroprotective support to degenerating motor neurons", "targeting_mechanism_pmid": "", "animal_results": "Stem cell therapy in preclinical mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1) demonstrated potential benefit and served as the stimulus for human trials", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00560287", "title": "Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis", "phase": "PHASE4", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fondazione Salvatore Maugeri", "summary": "Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive pulmonary disorders is still controversial, while most of the studies performed in restrictive thoracic disorders (RTD), and in particular in neuromuscular patients, suggested alleviation of the symptoms of chronic hypoventilation in the short term, and in two small studies survival was prolonged.\n\nIn the terminal phase of the disease, when the respiratory muscles became weaker it is very likely that the operators need to frequently adjust the level of inspiratory pressure in an attempt to guarantee an adequate tidal volume, so that alveolar hypoventilation may be avoided.\n\nTheoretically the use of a volume assisted ventilation may overpass this problem of frequent variations of the settings, since the provision of a fixed tidal volume may always guarantee and adequate alveolar ventilation.\n\nThe primary aims of this multicenter randomized study are to evaluate the clinical efficacy, the patients' tolerance and quality of life and the frequency of changing settings in a group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV with Pressure Support Ventilation or Volume Assisted Ventilation.", "interventions": [{"type": "DEVICE", "name": "Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers"}, {"type": "DEVICE", "name": "Non invasive ventilation"}], "start_date": "2008-01", "url": "https://clinicaltrials.gov/study/NCT00560287", "target_entities": [], "locations": [{"facility": "Fondazione S.Maugeri", "city": "Pavia", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.19205, "lon": 9.15917}, {"facility": "Respiratory Unit FSM", "city": "Pavia", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 45.19205, "lon": 9.15917}], "contact_phone": "0382 592", "contact_email": "snava@fsm.it", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Non-invasive mechanical ventilation maintains adequate ventilation and alleviates symptoms of chronic hypoventilation in ALS patients with respiratory failure.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02166944", "title": "Tamoxifen Treatment in Patients With Motor Neuron Disease", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Taipei Medical University Shuang Ho Hospital", "summary": "The aim of this study is to survey the effect of Tamoxifen in motor neuron disease (MND) patients, amyotrophic lateral sclerosis (ALS) with regular riluzole usage. TDP-43 is related to ALS. Increased the ubiquitinated or phosphorylated TDP-43 can cause animal model of ALS, and TDP43 can be degraded either by proteasome or autophagy pathway system. Autophagy pathway can be activated by mTOR inhibition, resulting in ameliorating TDP-43 accumulation and rescue in motor function in animal model. Tamoxifen had shown ability of enhance both proteasome and autophagy pathway, therefore the investigators assume that Tamoxifen probably can ameliorate TDP-43 accumulation and inclusion body formation in ALS.", "interventions": [{"type": "DRUG", "name": "tamoxifen 40 mg daily for one year"}], "start_date": "2014-04", "url": "https://clinicaltrials.gov/study/NCT02166944", "target_entities": ["TARDBP", "MTOR"], "locations": [{"facility": "Po-Chih Chen", "city": "New Taipei City", "state": "", "country": "Taiwan", "status": "", "lat": 25.06199, "lon": 121.45703}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "tamoxifen", "targeting_mechanism": "Tamoxifen activates autophagy through mTOR inhibition to promote degradation of ubiquitinated or phosphorylated TDP-43 and ameliorate TDP-43 accumulation in motor neurons.", "targeting_mechanism_pmid": "", "animal_results": "TDP-43 transgenic mouse models show autophagy pathway activation and reduction of TDP-43 pathology with rescue of motor function.", "animal_results_pmid": "29463850", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02306590", "title": "Efficacy, Safety and Tolerability of High Lipid and Calorie Supplementation in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Ulm", "summary": "The primary objective of the trial is to investigate the survival time (the time from randomization until death or date of end of the study) compared between control group and experimental group.\n\nThis is a prospective, multicenter, randomized, stratified, parallel-group, double-blind trial comparing placebo with high caloric fatty diet for drinking as add-on therapy to 100 mg riluzole in amyotrophic lateral sclerosis (ALS) in 200 enrolled patients. For entry, the El Escorial Criteria for the diagnosis of ALS will be used. The patients have to be stable on riluzole at least 4 weeks prior to randomization.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "Calogen"}, {"type": "DIETARY_SUPPLEMENT", "name": "Placebo"}], "start_date": "2015-02", "url": "https://clinicaltrials.gov/study/NCT02306590", "target_entities": [], "locations": [{"facility": "Department of Neurology, University of Ulm", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Department of Neurology, University of Wuerzburg", "city": "W\u00fcrzburg", "state": "Bavaria", "country": "Germany", "status": "", "lat": 49.79391, "lon": 9.95121}, {"facility": "Department of Neurology, Medical School Hannover", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Department of Neurology, University of Rostock", "city": "Rostock", "state": "Mecklenburg-Vorpommern", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Department of Neurology, TU Dresden", "city": "Dresden", "state": "Saxony", "country": "Germany", "status": "", "lat": 51.05089, "lon": 13.73832}, {"facility": "Department of Neurology, University of Halle-Wittenberg", "city": "Halle", "state": "Saxony-Anhalt", "country": "Germany", "status": "", "lat": 51.48158, "lon": 11.97947}, {"facility": "Department of Neurology, University of Jena", "city": "Jena", "state": "Thuringia", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Department of Neurology, Humboldt University", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Neurologische Universit\u00e4tsklinik Bergmannsheil", "city": "Bochum", "state": "", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Department f\u00fcr Neurologie - Klinik f\u00fcr Schlafmedizin und Neuromuskul\u00e4re Erkrankungen Universit\u00e4tsklinikum M\u00fcnster", "city": "M\u00fcnster", "state": "", "country": "Germany", "status": "", "lat": 51.96236, "lon": 7.62571}, {"facility": "Department of Neurology, DKD HELIOS Klinik", "city": "Wiesbaden", "state": "", "country": "Germany", "status": "", "lat": 50.08601, "lon": 8.24435}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Calogen", "targeting_mechanism": "High-calorie, high-lipid nutritional supplementation to provide enhanced energy support as add-on therapy in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01051882", "title": "Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients.", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Brainstorm-Cell Therapeutics", "summary": "The study will evaluate the safety, tolerability and therapeutic effects (preliminary efficacy) of injection of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF), as a possible treatment for patients with Amyotrophic Lateral Sclerosis (ALS) at the early and progressive disease stages.", "interventions": [{"type": "BIOLOGICAL", "name": "MSC-NTF cells transplantation (IM)"}, {"type": "BIOLOGICAL", "name": "MSC-NTF cells transplantation (IT)"}], "start_date": "2011-06", "url": "https://clinicaltrials.gov/study/NCT01051882", "target_entities": ["neurotrophic_factors"], "locations": [{"facility": "Hadassah Medical Organization", "city": "Jerusalem", "state": "", "country": "Israel", "status": "", "lat": 31.76904, "lon": 35.21633}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "MSC-NTF cells", "targeting_mechanism": "Autologous mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF) support motor neuron survival through trophic support and anti-inflammatory effects.", "targeting_mechanism_pmid": "34890069", "animal_results": "SOD1G93A transgenic mouse models show stem cell transplantation increases lifespan, delays disease onset, and reduces motor neuron loss.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06429059", "title": "ROAR-DIGAP: A Widely Inclusive, Largely Virtual Pilot Trial Utilizing DIGAP (Deep Integrated Genomics Analysis Platform) To Personalize Treatments", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Duke University", "summary": "GenieUs developed an analysis platform that will be tested to separate study participants with ALS into four categories based on blood work. These general categories are neuroinflammation, oxidative stress, impaired autophagy \\& axonal transport, and mitochondrial dysfunction. Once a disease category is established, participants in this study will receive one of four individualized supplements for 6 months and we will determine whether these are slowing ALS progression: Astaxanthin will be given for the category of neuroinflammation, Protandim for oxidative stress, Melatonin for impaired autophagy and MitoQ for mitochondrial dysfunction. During the first 3 months, participants will have routine monitoring and in months 3 through 9 they will receive the assigned supplement.", "interventions": [{"type": "DRUG", "name": "Astaxanthin"}, {"type": "DRUG", "name": "Protandim"}, {"type": "DRUG", "name": "Melatonin"}, {"type": "DRUG", "name": "MitoQ"}], "start_date": "2024-06-12", "url": "https://clinicaltrials.gov/study/NCT06429059", "target_entities": ["neuroinflammation", "oxidative_stress", "autophagy_impairment", "axonal_transport_defect", "mitochondrial_dysfunction"], "locations": [{"facility": "Duke University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Astaxanthin, Protandim, Melatonin, MitoQ", "targeting_mechanism": "Personalized supplement therapy targeting specific disease pathways: Astaxanthin and Melatonin target oxidative stress; Protandim targets antioxidant systems; MitoQ targets mitochondrial dysfunction to slow ALS progression.", "targeting_mechanism_pmid": "35805131", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05340660", "title": "mGluR5 Imaging in ALS Using PET", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Nathalie Braun", "summary": "In ALS models, it was shown that receptors, that bind an important messenger substance (glutamate) in the brain, are increased. In this research project, the investigators want to use a specific radioactive substance to find out whether these receptors are more detectable in people with ALS than in healthy people and increase over the course of the disease.", "interventions": [{"type": "RADIATION", "name": "[ 18 F]PSS232"}], "start_date": "2022-04", "url": "https://clinicaltrials.gov/study/NCT05340660", "target_entities": ["GRM5", "glutamate_excitotoxicity"], "locations": [{"facility": "Neuromuscular Center/ALS Clinic, Cantonal Hospital St. Gallen", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "RECRUITING", "lat": 47.42391, "lon": 9.37477}], "contact_phone": "+41 71 494 35 81", "contact_email": "nathalie.braun@kssg.ch", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "mGluR5 imaging to detect increased glutamate receptor levels in ALS", "targeting_mechanism_pmid": "", "animal_results": "In ALS models, mGluR5 receptors that bind glutamate are increased.", "animal_results_pmid": "37895110", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03604822", "title": "Music Therapy Protocol to Support Bulbar and Respiratory Functions in ALS", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Alisa Apreleva", "summary": "This study evaluates potential of music therapy treatment to support breathing, speech, swallow and cough of persons with amyotrophic lateral sclerosis (ALS). Music therapy is the clinical use of music and its elements to enhance human health and wellbeing. Application of music therapy principles in neurorehabilitation allow to treat cognitive, sensory, and motor dysfunctions.", "interventions": [{"type": "PROCEDURE", "name": "Music therapy"}], "start_date": "2017-09-27", "url": "https://clinicaltrials.gov/study/NCT03604822", "target_entities": [], "locations": [{"facility": "ALS Moscow Centre", "city": "Moscow", "state": "", "country": "Russia", "status": "", "lat": 55.75204, "lon": 37.61781}, {"facility": "Cambridge Institute for Music Therapy Research (CIMTR)", "city": "Cambridge", "state": "Cambridgeshire", "country": "United Kingdom", "status": "", "lat": 52.2, "lon": 0.11667}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Music therapy principles applied in neurorehabilitation to treat motor dysfunctions via cognitive and sensory engagement", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01363882", "title": "Polysomnography-directed Noninvasive Ventilation in Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Columbia University", "summary": "Use of noninvasive ventilation (NIV, also known colloquially as \"Bipap\") has been associated in some studies with improvement in pulmonary function, quality of life and survival. NIV is typically applied during sleep, and without the benefit of sleep study to determine the optimal settings. The investigators have shown that when NIV is used in this fashion, failure of nocturnal oxygenation and ventilation is prominent. This study is randomizing patients to standard application of NIV vs application guided by use of sleep study data to determine the effect of titrated therapy on pulmonary function, quality of life and survival.", "interventions": [{"type": "OTHER", "name": "Sleep study-guided adjustment of NIV"}, {"type": "OTHER", "name": "Standard initiation of NIV"}], "start_date": "2008-02", "url": "https://clinicaltrials.gov/study/NCT01363882", "target_entities": [], "locations": [{"facility": "Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Sleep study-guided adjustment of noninvasive ventilation (NIV) to optimize nocturnal oxygenation and ventilation", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02496767", "title": "Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "This study assessed the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.", "interventions": [{"type": "DRUG", "name": "Tirasemtiv"}, {"type": "DRUG", "name": "Placebo tablets"}], "start_date": "2015-09-03", "url": "https://clinicaltrials.gov/study/NCT02496767", "target_entities": ["ACTA1"], "locations": [{"facility": "St. Joseph's Hospital & Medical Center - Barrow Neurology Clinics", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "University of California Davis Medical Center", "city": "Sacramento", "state": "California", "country": "United States", "status": "", "lat": 38.58157, "lon": -121.4944}, {"facility": "Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Stanford Hospital and Clinics", "city": "Stanford", "state": "California", "country": "United States", "status": "", "lat": 37.42411, "lon": -122.16608}, {"facility": "University of Colorado Hospital Anschutz Outpatient Pavilion", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "George Washington University Medical Center", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Carol and Frank Morsini Center for Advanced Health Care - University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "The Emory Clinic", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Georgia Regents University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Northwestern University Feinberg School of Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Indiana University", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Memorial Medical Center", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "University of Michigan Hospital and Health System", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Health System", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Saint Louis University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Barnes-Jewish Hospital", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Dartmouth Hitchcock Medical Center Dept of Neurology", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Neurological Institute Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Neurosciences Institute: Neurology - Charlotte", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke Neurological Disorders Clinic", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest University Health Sciences", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Ohio State University Wexner Medical Center", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence Brain and Spine Institute ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Oregon Health and Science Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "The Penn Comprehensive Neuroscience Center", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Temple University School of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Vanderbilt University Medical Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Baylor College of Medicine", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Texas Health Science Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Virgina Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "West Virginia University Department of Neurology", "city": "Morgantown", "state": "West Virginia", "country": "United States", "status": "", "lat": 39.62953, "lon": -79.9559}, {"facility": "Froedtert Memorial Lutheran Hospital, Department of Neurology", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "UZ Leuven - Campus Gasthuisberg", "city": "Leuven", "state": "Vlaams Brabant", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "University of Calgary", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Edmonton Kaye Clinic", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "QE II Health Sciences Centre, NHI Site", "city": "Halifax", "state": "Nova Scotia", "country": "Canada", "status": "", "lat": 44.64269, "lon": -63.57688}, {"facility": "McMaster University Medical Centre", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Notre-Dame Hospital/CHUM", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "CHU de Quebec - Universite Laval Hopital de l'Enfant-Jesus", "city": "Qu\u00e9bec", "state": "", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Hopital R. Salengro, CHRU Lille", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "CHU Dupuytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Hopital de la Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Hopital Gui de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "CHU de Nice - Hopital Pasteur 2", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Hopital de la Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Bretonneau University Hospital", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "University of Ulm, Department of Neurology", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Hannover Medical School, Department of Neurology", "city": "Hanover", "state": "Lower Saxony", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Charite Campus Virchow-Klinikum, Neurology Department", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Clinical Research Centre, Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "IRCCS Istituto Auxologico Italiano - U.O. Neurologia", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Dipartimento di Neuroscienze \"Rita Levi Moltalcini\" A.O.U. Citta della Salute e della Scienza di Torino P.O. \"Molinette\"", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Santa Maria-Centro Hospitalar Lisboa Norte", "city": "Lisbon", "state": "", "country": "Portugal", "status": "", "lat": 38.72509, "lon": -9.1498}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Derriford Hospital", "city": "Plymouth", "state": "Devon", "country": "United Kingdom", "status": "", "lat": 50.37153, "lon": -4.14305}, {"facility": "Walton Centre for Neurology and Neurosurgery", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "Clinical Research Centre, Royal London Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Kings College Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Tirasemtiv", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05742828", "title": "Virtual Seating Coach on Power Wheelchairs of Persons With Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Wake Forest University Health Sciences", "summary": "A literature review was completed related to the topic of use of the Virtual Seating Coach (VSC) device with clients with Amyotrophic Lateral Sclerosis (ALS) with no results. The VSC components are FDA approved and Health Insurance Portability and Accountability Act (HIPPA) compliant, which have been used for many years by clinicians to achieve therapy goals of repositioning and best practice of utilizing power wheelchair seat functioning on a frequent basis. The VSC is typically not covered by insurance, but with clinical documentation, it has the potential for reimbursement. There is conflicting and vague information in the literature with regards to the prevalence/types of wounds and prevalence of pain in this population.", "interventions": [{"type": "BEHAVIORAL", "name": "Virtual Seating Coach"}], "start_date": "2017-09-20", "url": "https://clinicaltrials.gov/study/NCT05742828", "target_entities": [], "locations": [{"facility": "Atrium Health - Neurosciences Institute", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Virtual Seating Coach (VSC) device to optimize power wheelchair seating positioning and repositioning", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00580593", "title": "Trial of Early Noninvasive Ventilation for Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "The goal of this trial is to determine the feasibility of conducting a randomized, double-blind, placebo-controlled trial of nocturnal noninvasive positive pressure ventilation in persons with amyotrophic lateral sclerosis with an forced vital capacity greater than or equal to 50 percent.", "interventions": [{"type": "DEVICE", "name": "BiPAP\u00ae S/T System"}, {"type": "OTHER", "name": "sham-NIPPV"}], "start_date": "2007-04", "url": "https://clinicaltrials.gov/study/NCT00580593", "target_entities": [], "locations": [{"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Noninvasive positive pressure ventilation (BiPAP) aims to support respiratory function in ALS patients with compromised respiratory muscles.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03545451", "title": "Neurofeedback Rehabilitation Based on Motor Imaging in Patients in the Immobilization Phase", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "As the prevalence of motor impairment increases with age, the proportion of the population affected by physical limitations is likely to increase in the coming years, considering that one in three people will be over 60 in 2050 (compared to one in five in 2005, INSEE projections). The possibility of reducing recovery time and / or improving the improvement of motor deficits is today a public health issue. The possibility of developing new therapeutic tools using innovative motor imaging rehabilitation technologies is an opportunity to offer rehabilitation adapted to specific disorders, personalized in relation to the patient's performance, and in continuity with the therapist. In this research project, we will use the principle of neurofeedback rehabilitation (EEG) based on motor imaging with a brain-computer interface. Feedback will consist of therapeutic video games.Here, we will test the feasibility of such approach in 10 healthy subjects, 10 patients with amyotrophic lateral sclerosis and 10 patients with uppel shoulder surgery.", "interventions": [{"type": "DEVICE", "name": "Videogames"}], "start_date": "2018-10-03", "url": "https://clinicaltrials.gov/study/NCT03545451", "target_entities": [], "locations": [{"facility": "Hopital PITIE SALPETRIERE", "city": "Paris", "state": "", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "01 42 16 57 72", "contact_email": "jean-christophe.corvol@aphp.fr", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Videogame-based neurofeedback rehabilitation using motor imaging to enhance motor recovery and motor function.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01945853", "title": "Magnetic Resonance Imaging (MRI) in Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "This is a pilot study to identify the degree of grey and white matter involvement in patients with Amyotrophic Lateral Sclerosis (ALS) utilizing non-invasive techniques. The imaging to be utilized will be the 7 Tesla (7T) magnetic resonance imaging (MRI) of the brain. These results will be correlated to the ALS Functional Rating Scale - Revised (ALSFRS-R) score to assess if any changes in MRI can be predictive in the disability of the ALS patients at baseline and at 6 month intervals. The participants will be asked to return every 6 months for a neurological examination, ALSFRS-R assessment, measurement of the vital capacity and MRI as outlined above to monitor progression of the disease.", "interventions": [{"type": "DEVICE", "name": "7 Tesla MRI"}], "start_date": "2013-07", "url": "https://clinicaltrials.gov/study/NCT01945853", "target_entities": [], "locations": [{"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "7 Tesla MRI is a non-invasive imaging technique used to identify grey and white matter involvement in ALS and assess changes predictive of disability.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01083667", "title": "SOD1 Inhibition by Pyrimethamine in Familial Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Weill Medical College of Cornell University", "summary": "The objective of this study will be to evaluate the safety, tolerability and effect on SOD1 levels by pyrimethamine in patients with familial amyotrophic lateral sclerosis.", "interventions": [{"type": "DRUG", "name": "Pyrimethamine"}], "start_date": "2009-11", "url": "https://clinicaltrials.gov/study/NCT01083667", "target_entities": ["SOD1"], "locations": [{"facility": "Weill Cornell Medical Center/New York Presbyterian Hospital", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Universit\u00e4ts- und Rehabilitationskliniken Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Milano Neurological Institute", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Umea University", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Pyrimethamine", "targeting_mechanism": "Pyrimethamine inhibits SOD1 protein levels in familial ALS.", "targeting_mechanism_pmid": "29751510", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06172621", "title": "Spinal Cord Associative Plasticity for ALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "VA Office of Research and Development", "summary": "Veterans are at higher risk than non-Veterans of falling ill with amyotrophic lateral sclerosis (ALS).\n\nThe investigators have shown that synchronized stimulation over the brain and cervical spinal cord can temporarily strengthen weakened nerve circuits between the brain and hand muscles in people with ALS.\n\nThe current proposal will take the next step of individualizing this intervention, then applying it repetitively in an attempt to achieve direct clinical benefit on hand strength and function.\n\nFollowing an initial 2-3 month period of optimizing the intervention for each individual, the investigators will compare the effects of two-week programs of paired brain-spinal stimulation with or without hand exercises.", "interventions": [{"type": "PROCEDURE", "name": "Spinal Cord Associative Plasticity (SCAP)"}, {"type": "PROCEDURE", "name": "Upper extremity task-oriented exercise"}], "start_date": "2024-04-01", "url": "https://clinicaltrials.gov/study/NCT06172621", "target_entities": ["motor_circuit_plasticity"], "locations": [{"facility": "James J. Peters VA Medical Center, Bronx, NY", "city": "The Bronx", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 40.84985, "lon": -73.86641}], "contact_phone": "(718) 584-9000", "contact_email": "francisco.castano@va.gov", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Spinal cord associative plasticity (SCAP) uses synchronized stimulation over the brain and cervical spinal cord to strengthen weakened nerve circuits between the brain and hand muscles.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03359538", "title": "Rapamycin Treatment for ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Azienda Ospedaliero-Universitaria di Modena", "summary": "In the last years research has pointed out potential mechanisms of pathogenesis in ALS including lack of degradation of abnormally accumulated proteins inside motor neurons, and an unbalanced function of the immune system leading to the prevalence of a neurotoxic function over neuroprotection. These two mechanisms contribute to ALS progression hence representing important therapeutic targets to modify disease expression.\n\nWith a phase II clinical trial the investigators aim to study the biological response in ALS treated with Rapamycin, to obtain predictive information for a larger study.\n\nEight Italian Centres will enroll 63 patients; treatment will be double blinded to patients and physicians, and will last 18 weeks.Follow up will be carried out for 36 months (total duration: 54 weeks).", "interventions": [{"type": "DRUG", "name": "Rapamycin"}, {"type": "DRUG", "name": "Placebo Oral Tablet"}], "start_date": "2017-09-19", "url": "https://clinicaltrials.gov/study/NCT03359538", "target_entities": ["MTOR", "autophagy_impairment", "neuroinflammation"], "locations": [{"facility": "Centro Sla, Irccs A.O.U. S.Martino Ist, Genova", "city": "Genova", "state": "", "country": "Italy", "status": "", "lat": 45.21604, "lon": 11.87211}, {"facility": "Centro Clinico Nemo, Fondazione Serena Onlus, Milano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Centro Sla, Irccs Istituto Carlo Besta, Milano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Centro Sla, Ospedale Civile S. Agostino Estense, A.O.U. Modena", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Centro Sla, A.O.U. Maggiore Della Carita', Novara", "city": "Novara", "state": "", "country": "Italy", "status": "", "lat": 45.44694, "lon": 8.62118}, {"facility": "Centro Sla, Universita' Di Padova", "city": "Padova", "state": "", "country": "Italy", "status": "", "lat": 44.38225, "lon": 11.14261}, {"facility": "Centro Sla, Universita' Di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Rapamycin", "targeting_mechanism": "Rapamycin activates autophagy to enhance degradation of abnormally accumulated proteins and modulates immune function to restore neuroprotection over neurotoxic inflammation in ALS.", "targeting_mechanism_pmid": "34057020", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00244244", "title": "A Multicenter, Dose Ranging Safety and Pharmacokinetics Study of Arimoclomol in ALS", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "CytRx", "summary": "The primary purpose of this study is to evaluate the safety and tolerability of arimoclomol in ALS patients following 90 days of dosing. In addition, the amount of arimoclomol in blood and cerebrospinal fluid will be measured.", "interventions": [{"type": "DRUG", "name": "arimoclomol"}], "start_date": "2005-10", "url": "https://clinicaltrials.gov/study/NCT00244244", "target_entities": ["heat_shock_response"], "locations": [{"facility": "University of California, Irvine Medical Center", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "University of Miami School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Hennepin Faculty Associates/Berman Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Penn State Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University College of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Texas Health Science Center at San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Arimoclomol", "targeting_mechanism": "Arimoclomol acts as a heat shock response inducer to enhance clearance of misfolded proteins, particularly TDP-43, and improve protein homeostasis in motor neurons.", "targeting_mechanism_pmid": "26936937", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06700304", "title": "CAN-PRIME: Precise Robotically Implanted Brain-Computer Interface for the Control of External Devices", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuralink Corp", "summary": "The CAN-PRIME Study is to test the safety and functionality of Neuralink's N1 Implant and R1 Robot in people who have difficulty moving their arms and legs (tetraparesis or tetraplegia). The N1 Implant is a small, wireless device placed in the skull. It connects to tiny threads inserted into the brain by the R1 Robot, which is a machine designed to carefully place these threads. This study will help researchers learn how well the implant and robot work and if they are safe for use.", "interventions": [{"type": "DEVICE", "name": "N1 Implant"}, {"type": "DEVICE", "name": "R1 Robot"}], "start_date": "2024-11-20", "url": "https://clinicaltrials.gov/study/NCT06700304", "target_entities": [], "locations": [{"facility": "University Health Network", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}], "contact_phone": "(877) 398-4465", "contact_email": "clinical-team-ct@neuralink.com", "mechanism_summary": {"compound": "N1 Implant", "targeting_mechanism": "The N1 Implant is a wireless brain-computer interface device that records neural signals to enable direct control of external devices for patients with motor paralysis.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02286011", "title": "Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Red de Terapia Celular", "summary": "The purpose of this study is to evaluate the safety of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis by a prospective, single-center, randomized, parallel, double-blind, placebo-controlled phase I clinical trial.", "interventions": [{"type": "BIOLOGICAL", "name": "MNC (Mononuclear cells)"}, {"type": "OTHER", "name": "Saline"}], "start_date": "2014-11", "url": "https://clinicaltrials.gov/study/NCT02286011", "target_entities": ["neuroprotection"], "locations": [{"facility": "Clinical Universitary Hospital Virgen de la Arrixaca", "city": "El Palmar", "state": "Murcia", "country": "Spain", "status": "", "lat": 37.93939, "lon": -1.16095}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "MNC (Mononuclear cells)", "targeting_mechanism": "Autologous bone marrow-derived mononuclear cells differentiate into supportive glial cells and produce neurotrophic factors and anti-inflammatory cytokines to protect degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Bone marrow-derived mesenchymal stem cells expressing Ngn1 administered intravenously in SOD1G93A mice increased lifespan by 3 days, delayed disease onset by 5 days, and reduced motor neuron loss.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02039401", "title": "Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Helixmith Co., Ltd.", "summary": "The purpose of this study is to determine the safety and tolerability of intramuscular injections of VM202 at different injection sites in people with amyotrophic lateral sclerosis.", "interventions": [{"type": "BIOLOGICAL", "name": "VM202"}], "start_date": "2014-03-11", "url": "https://clinicaltrials.gov/study/NCT02039401", "target_entities": ["angiogenesis"], "locations": [{"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "VM202", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05819931", "title": "Breathing With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Toulouse", "summary": "The study aims to evaluate the effect of mechanical insufflator-exsufflator on the respiratory functions of Amyotrophic Lateral Sclerosis (ALS) patients evaluated via peak expiratory flow on cough (PEFC) measurements. The evolution of their PEFC is monitored to see if the curative management can have a positive impact on the latter.", "interventions": [{"type": "DEVICE", "name": "Mechanical In-Exsufflator treatment"}], "start_date": "2023-08-29", "url": "https://clinicaltrials.gov/study/NCT05819931", "target_entities": [], "locations": [{"facility": "SLA Center - Purpan University Hospital Toulouse, FRANCE", "city": "Toulouse", "state": "Occitanie", "country": "France", "status": "RECRUITING", "lat": 43.60426, "lon": 1.44367}], "contact_phone": "+33561775570", "contact_email": "lagarde.t@chu-toulouse.fr", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00231140", "title": "Pilot-Study of Thalidomide in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Charite University, Berlin, Germany", "summary": "Neuroinflammation has recently emerged as a significant contributor to motor neuron damage. ALS tissue is characterized by inflammatory changes that are observed in both sporadic and familial ALS and in the ALS superoxide dismutase 1 (SOD1) transgenic mouse model. They include an accumulation of large numbers of activated microglia and astrocytes.\n\nProinflammatory cytokines, such as tumor necrosis factor (TNF-), are robustly upregulated in ALS. The receptor for tumor necrosis factor- (TNF-R1) is elevated at late presymptomatic as well as symptomatic phases of disease. TNF acts as a principal driver for neuroinflammation in ALS, while several co-stimulating cytokines and chemokines act to potentiate the TNF effects \\[4-6\\].\n\nWe propose an investigational therapy of ALS with oral administration of thalidomide. The rationale for this study is based on the anti-inflammatory properties of thalidomide through the modulation of inflammatory cytokines such as TNF. The primary aim of the trial is to determine whether treatment with thalidomide is safe and well tolerated in conjunction with riluzole and whether patients with ALS can tolerate daily doses of up to 400 mg. The trial is designed as feasibility study in planning for a larger phase IIb/III trial of efficacy.", "interventions": [{"type": "DRUG", "name": "Thalidomide (drug)"}], "start_date": "2005-12", "url": "https://clinicaltrials.gov/study/NCT00231140", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Charite University Hospital, Berlin, Germany", "city": "Berlin", "state": "State of Berlin", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Thalidomide", "targeting_mechanism": "Reduces tumor necrosis factor-\u03b1 (TNF-\u03b1) and modulates neuroinflammatory responses in ALS through inhibition of TNF-R1 signaling and suppression of activated microglia and astrocytes.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05866926", "title": "Study to Investigate the Long-term Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ferrer Internacional S.A.", "summary": "A multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis", "interventions": [{"type": "DRUG", "name": "FAB122"}], "start_date": "2023-03-06", "url": "https://clinicaltrials.gov/study/NCT05866926", "target_entities": ["fab122"], "locations": [{"facility": "Hospital Universitari de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "FAB122", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03520517", "title": "Open-label Study to Evaluate Safety, Tolerability and PK of BHV-0223 in ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biohaven Pharmaceuticals, Inc.", "summary": "Phase 1, open-label study of BHV-0223 in ALS.", "interventions": [{"type": "DRUG", "name": "BHV-0223"}], "start_date": "2018-02-02", "url": "https://clinicaltrials.gov/study/NCT03520517", "target_entities": ["bhv_0223"], "locations": [{"facility": "Holy Cross Neuroscience Research Institute", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "Somnos/Neurology Associates Clinical Research", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Neurosciences Institute, Neurology - Charlotte", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Wesley Neurology Clinic", "city": "Cordova", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.15565, "lon": -89.7762}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "BHV-0223", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03186040", "title": "Open-label Clinical Trial of Lacosamide in ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Chiba University", "summary": "Lacosamide is administered for patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Lacosamide"}], "start_date": "2017-07-13", "url": "https://clinicaltrials.gov/study/NCT03186040", "target_entities": ["lacosamide"], "locations": [{"facility": "Chiba University Hospital", "city": "Chiba", "state": "", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Lacosamide", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00069186", "title": "Study of Creatine Monohydrate in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The Avicena Group", "summary": "The purpose of this study is to determine whether nine months of administration of creatine monohydrate results in an increase in muscle strength in patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Creatine Monohydrate"}], "start_date": "2003-06", "url": "https://clinicaltrials.gov/study/NCT00069186", "target_entities": ["oxidative_stress"], "locations": [{"facility": "University of California, San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Kansas", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of New Mexico", "city": "Albuquerque", "state": "New Mexico", "country": "United States", "status": "", "lat": 35.08449, "lon": -106.65114}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Oregon Health Sciences University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Texas Health Science Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Creatine Monohydrate", "targeting_mechanism": "Creatine enhances cellular energy metabolism and reduces oxidative stress in motor neurons.", "targeting_mechanism_pmid": "34198557", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02238626", "title": "Ibudilast (MN-166) in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "MediciNova", "summary": "This is a single center, randomized, double-blind, placebo-controlled, 6-month study designed to evaluate the safety, tolerability and clinical responsiveness of MN-166/ibudilast (60 mg/day) when administered as an adjunct to riluzole (100 mg/day) in 60 subjects with ALS.\n\nThis study will consist of two treatment arms, MN-166 and matching placebo. Randomization will occur in a 2:1 ratio (MN- 166: placebo).\n\nDuration of Treatment: Screening Phase: up to 3 months; Double-blind Phase: 6 months; Open-label Phase 6 months (for placebo subjects only); Follow-up Phase: 2 weeks after last dose.\n\nDuring treatment phase, subjects return to the clinic at Months 3 and 6 and will be telephoned by staff at Months 1,2,4, and 5 to collect information about side effects and new or concomitant medications.\n\nAll subjects (subjects who complete the Double-blind Phase and subjects who complete the Open-label Phase) or prematurely discontinue will return for a follow-up visit approximately 2 weeks after the last dose of study drug to assess adverse event status and to document concomitant medications.\n\nSafety will be assessed by monitoring and recording all treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and discontinuations due to TEAEs. Additional assessments will include regular monitoring of hematology, blood chemistry, and urine values, regular measurement of vital signs, ECGs, medical history, physical and neurological examinations.", "interventions": [{"type": "DRUG", "name": "Placebo (for MN-166)"}, {"type": "DRUG", "name": "MN-166"}, {"type": "DRUG", "name": "riluzole"}], "start_date": "2014-09", "url": "https://clinicaltrials.gov/study/NCT02238626", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Carolinas Healthcare System, Dept. of Neurology", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "MN-166 (Ibudilast)", "targeting_mechanism": "Ibudilast reduces neuroinflammation by inhibiting microglia and astrocyte activation in the CNS.", "targeting_mechanism_pmid": "28872464", "animal_results": "AAV9-treated SOD1G93A mice showed delayed paralysis onset and extended survival compared to untreated SOD1G93A controls, remaining able to climb and hang from structures at >200 days while untreated mice were paralyzed by 135 days.", "animal_results_pmid": "26891182", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00958048", "title": "Effects of Nocturnal Non-invasive Ventilation in Patients With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Taiwan University Hospital", "summary": "Specific aims:\n\nAim 1. To determine the incidence of hypoventilation in Amyotrophic Lateral Sclerosis (ALS) patients.\n\nAim 2. To identify the clinical characteristics and risk factors associated .\n\nAim 3. To determine the effect of early intervention with nocturnal NIV on the prognosis of ALS patients.", "interventions": [{"type": "DEVICE", "name": "non-invasive ventilation"}], "start_date": "2009-01", "url": "https://clinicaltrials.gov/study/NCT00958048", "target_entities": [], "locations": [{"facility": "Peilin Lee", "city": "Taipei", "state": "", "country": "Taiwan", "status": "", "lat": 25.05306, "lon": 121.52639}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "non-invasive ventilation", "targeting_mechanism": "Non-invasive ventilation (NIV) provides mechanical respiratory support to compensate for progressive motor neuron loss affecting the diaphragm and accessory respiratory muscles.", "targeting_mechanism_pmid": "29990478", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00298597", "title": "Influence of G-CSF and EPO on Associative Learning and Motor Skills", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital Muenster", "summary": "In the study we want to prove whether the subcutaneous application of granulocyte-stimulating factor (G-CSF) and erythropoetin (EPO) influence associative learning and/or motor skills of patients, who suffer from chronic stroke or amyotrophic lateral sclerosis. The study hypothesis is that G-CSF and EPO improve associative learning and/or motor skills.", "interventions": [{"type": "DRUG", "name": "granulocyte - colony stimulating factor (G-CSF)"}, {"type": "DRUG", "name": "erythropoetin (EPO)"}], "start_date": "2006-03", "url": "https://clinicaltrials.gov/study/NCT00298597", "target_entities": ["g_csf", "epo"], "locations": [{"facility": "Department of neurology, University Hospital of Muenster", "city": "M\u00fcnster", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 51.96236, "lon": 7.62571}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "granulocyte-colony stimulating factor (G-CSF) and erythropoietin (EPO)", "targeting_mechanism": "G-CSF and EPO promote hematopoiesis and neuronal protection through neurotrophic and neuroprotective mechanisms to support motor neuron function.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05035823", "title": "COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis", "phase": "NA", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Synchron Medical, Inc.", "summary": "The Synchron motor neuroprosthesis (MNP) is intended to be used in subjects with severe motor impairment, unresponsive to medical or rehabilitative therapy and a persistent functioning motor cortex. The purpose of this research is to evaluate safety and feasibility.\n\nThe MNP is a type of implantable brain computer interface which bypasses dysfunctional motor neurons. The device is designed to restore the transmission of neural signal from the cerebral cortex utilized for neuromuscular control of digital devices, resulting in a successful execution of non-mechanical digital commands.", "interventions": [{"type": "DEVICE", "name": "Motor Neuroprosthesis (MNP)"}], "start_date": "2022-04-27", "url": "https://clinicaltrials.gov/study/NCT05035823", "target_entities": [], "locations": [{"facility": "University at Buffalo Neurosurgery (UBNS)", "city": "Buffalo", "state": "New York", "country": "United States", "status": "", "lat": 42.88645, "lon": -78.87837}, {"facility": "Mount Sinai Health System", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Pittsburgh Medical Center", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Motor Neuroprosthesis (MNP)", "targeting_mechanism": "The implantable brain\u2013computer interface device bypasses dysfunctional motor neurons by directly translating neural signals from the motor cortex to control external digital devices.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07138014", "title": "FHND1002 for ALS Treatment: Phase 2", "phase": "PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.", "summary": "This is a Phase II clinical trial evaluating the effectiveness and safety of an investigational drug, FHND1002 granules, in adults with Amyotrophic Lateral Sclerosis (ALS).\n\nThe main goals are:\n\nTo determine if FHND1002 can slow the progression of ALS compared to a placebo.\n\nTo assess the safety and tolerability of two different doses of FHND1002 (100mg and 200mg) in ALS patients.\n\nApproximately 180 participants will be randomly assigned (like flipping a coin) to one of three groups:\n\nFHND1002 100mg once daily\n\nFHND1002 200mg once daily\n\nPlacebo (an inactive substance) once daily Assignment will consider disease severity (ALSFRS-R score) and where symptoms started (Limb vs. Bulbar). Participants can continue taking stable doses of approved ALS medications (like riluzole or edaravone) or be on no medication.\n\nThe study consists of:\n\nA Screening Period (up to 4 weeks).\n\nA Double-Blind Treatment Period (48 weeks).\n\nDuring the 48-week treatment period:\n\nParticipants will take their assigned granules orally once daily (with or without food).\n\nThey will attend clinic visits at Weeks 2, 4, 12, 24, 36, and 48 for safety checks.\n\nEffectiveness will be measured at Weeks 12, 24, 36, and 48 using standard ALS assessments, including the ALS Functional Rating Scale-Revised (ALSFRS-R), breathing tests (FVC%), and quality of life/questionnaires (ROADS, ALSAQ-5).", "interventions": [{"type": "DRUG", "name": "FHND1002 100mg"}, {"type": "DRUG", "name": "FHND1002 200mg"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2025-10-01", "url": "https://clinicaltrials.gov/study/NCT07138014", "target_entities": [], "locations": [], "contact_phone": "010-82265159", "contact_email": "dsfan@sina.com", "mechanism_summary": {"compound": "FHND1002", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07002034", "title": "RE104 Safety and Efficacy Study in Adjustment Disorder in Cancer and Other Medical Illnesses", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Reunion Neuroscience Inc", "summary": "The purpose of this study is to determine if treatment with a single dose of RE104 for Injection reduces depressive symptoms or depressive symptoms mixed with anxiety symptoms in participants with Adjustment Disorder due to cancer or other illnesses such as Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS), Parkinson's Disease (PD) or Idiopathic Pulmonary Fibrosis (IPF) as compared to active-placebo.", "interventions": [{"type": "DRUG", "name": "RE104 for Injection"}], "start_date": "2025-07-30", "url": "https://clinicaltrials.gov/study/NCT07002034", "target_entities": [], "locations": [{"facility": "UAB, Psychiatry and Behavioral Neurology", "city": "Birmingham", "state": "Alabama", "country": "United States", "status": "RECRUITING", "lat": 33.52066, "lon": -86.80249}, {"facility": "University of Arizona", "city": "Tucson", "state": "Arizona", "country": "United States", "status": "RECRUITING", "lat": 32.22174, "lon": -110.92648}, {"facility": "Kadima Neuropsychiatry Institute", "city": "San Diego", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 32.71571, "lon": -117.16472}, {"facility": "Providence Medical Foundation", "city": "Santa Rosa", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 38.44047, "lon": -122.71443}, {"facility": "University of Colorado", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "RECRUITING", "lat": 39.72943, "lon": -104.83192}, {"facility": "University of South Florida, Department of Psychiatry and Behavioral Neuroscience", "city": "Tampa", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "RECRUITING", "lat": 33.749, "lon": -84.38798}, {"facility": "Hawaii Pacific Neuroscience", "city": "Honolulu", "state": "Hawaii", "country": "United States", "status": "RECRUITING", "lat": 21.30694, "lon": -157.85833}, {"facility": "Rush University Medical Center", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "RECRUITING", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Chicago Medical Center", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "RECRUITING", "lat": 41.85003, "lon": -87.65005}, {"facility": "The University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "RECRUITING", "lat": 39.11417, "lon": -94.62746}, {"facility": "LSU Health Shreveport", "city": "Shreveport", "state": "Louisiana", "country": "United States", "status": "RECRUITING", "lat": 32.52515, "lon": -93.75018}, {"facility": "Sunstone Therapies, PC", "city": "Rockville", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.084, "lon": -77.15276}, {"facility": "Sheppard Pratt", "city": "Towson", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.4015, "lon": -76.60191}, {"facility": "Dana Farber Cancer Institute", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Massachusetts Chan Medical Center", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.26259, "lon": -71.80229}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "RECRUITING", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Health", "city": "Novi", "state": "Michigan", "country": "United States", "status": "RECRUITING", "lat": 42.48059, "lon": -83.47549}, {"facility": "University of Nebraska Medical Center", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "RECRUITING", "lat": 41.25626, "lon": -95.94043}, {"facility": "University of New Mexico, School of Medicine", "city": "Albuquerque", "state": "New Mexico", "country": "United States", "status": "RECRUITING", "lat": 35.08449, "lon": -106.65114}, {"facility": "Roswell Park Center Institute", "city": "Buffalo", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 42.88645, "lon": -78.87837}, {"facility": "NYU Langone Health", "city": "New York", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of North Carolina at Chapel Hill", "city": "Chapel Hill", "state": "North Carolina", "country": "United States", "status": "RECRUITING", "lat": 35.9132, "lon": -79.05584}, {"facility": "Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 41.4995, "lon": -81.69541}, {"facility": "The Ohio State University, Department of Psychiatry", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "RECRUITING", "lat": 39.96118, "lon": -82.99879}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 39.95238, "lon": -75.16362}, {"facility": "Alliance for Multispecialty Research Clinical", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "RECRUITING", "lat": 35.96064, "lon": -83.92074}, {"facility": "Dell Medical School, University of Texas at Austin", "city": "Austin", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 30.26715, "lon": -97.74306}, {"facility": "Cedar Clinical Research Inc.", "city": "Draper", "state": "Utah", "country": "United States", "status": "RECRUITING", "lat": 40.52467, "lon": -111.86382}, {"facility": "UVA Center for Psychiatric Clinical Research", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "RECRUITING", "lat": 38.02931, "lon": -78.47668}, {"facility": "Seattle Neuropsychiatric Treatment Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "RECRUITING", "lat": 47.60621, "lon": -122.33207}, {"facility": "University of Wisconsin at Madison", "city": "Madison", "state": "Wisconsin", "country": "United States", "status": "RECRUITING", "lat": 43.07305, "lon": -89.40123}], "contact_phone": "1-888-880-REUN", "contact_email": "info@reunionneuro.com", "mechanism_summary": {"compound": "RE104", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01363401", "title": "Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Corestemchemon, Inc.", "summary": "The purpose of this study is to evaluate the safety and efficacy of autologous bone marrow-derived stem cells(\"HYNR-CS inj\"), through intrathecal delivery for the treatment in patients with ALS.\n\nThis study consists of 2 steps. First step is a safety study of the intrathecal(IT) injection of \"HYNR-CS inj\" in 8 patients with ALS. In this phase 1 study, AE, laboratory test, physical examination, vital signs, Electrocardiogram, and Chest X-Ray examination were evaluated in terms of safety.\n\nSecond step is to compare the efficacy and safety between test group and control group of total 64 patients with ALS.", "interventions": [{"type": "BIOLOGICAL", "name": "HYNR-CS inj"}, {"type": "OTHER", "name": "Control group"}], "start_date": "2011-02", "url": "https://clinicaltrials.gov/study/NCT01363401", "target_entities": ["stem_cell_therapy"], "locations": [{"facility": "Hanyang University Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "HYNR-CS inj (autologous bone marrow-derived stem cells)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03811301", "title": "[BrainConnexion] - Neurodevice Phase I Trial", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Neuroscience Institute", "summary": "This study aims to evaluate the safety of a wireless implantable neurodevice microsystem in tetraplegic patients, as well as the efficacy of the electrodes for long-term recording of neural activities and the successful control of an external device.", "interventions": [{"type": "DEVICE", "name": "BrainConnexion"}], "start_date": "2017-11-21", "url": "https://clinicaltrials.gov/study/NCT03811301", "target_entities": [], "locations": [{"facility": "National Neuroscience Institute", "city": "Singapore", "state": "", "country": "Singapore", "status": "", "lat": 1.28967, "lon": 103.85007}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07616050", "title": "Evaluation of the Impact of Virtual Park on Training Motivation in Adult Patients", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Marta Mondellini", "summary": "The purpose of this clinical study is to evaluate the feasibility, usability, and motivational impact of VirtualPark, a virtual reality-based dual-task rehabilitation system, in adults with neurological and age-related conditions.\n\nVirtualPark is a virtual reality application designed to deliver cognitive exercises during cycling training using a commercially available ergometer (THERA-Trainer Tigo). The system integrates physical and cognitive tasks in simulated real-life environments.\n\nThe intervention integrates motor and cognitive training tasks targeting domains such as attention, inhibition, working memory, and navigation.\n\nThis is a prospective, multicenter, randomized, cross-over pilot study. It will compare cycling training performed with and without virtual reality. Participants will complete both intervention conditions over a 4-week period separated by a wash-out phase with standard rehabilitation activities. The order of conditions will be randomized.\n\nThe study will assess motivation during rehabilitation training, usability and user experience of the system, as well as exploratory effects on cognitive and motor performance, functional abilities, perceived exertion, and safety.\n\nThe study will enroll adult participants (\u226518 years) with conditions such as stroke, Parkinson's disease, multiple sclerosis, amyotrophic lateral sclerosis, mild cognitive impairment, spinal cord injury, and frail older adults.", "interventions": [{"type": "DEVICE", "name": "Dual-task training with Virtual Reality"}, {"type": "DEVICE", "name": "Cycling training"}], "start_date": "2026-06", "url": "https://clinicaltrials.gov/study/NCT07616050", "target_entities": [], "locations": [{"facility": "Istituti a Carattere Scientifico Maugeri Bari", "city": "Bari", "state": "Bari", "country": "Italy", "status": "", "lat": 41.12066, "lon": 16.86982}, {"facility": "Istituti a Carattere Scientifico Maugeri Telese", "city": "Telese Terme", "state": "Benevento", "country": "Italy", "status": "", "lat": 41.21752, "lon": 14.52681}, {"facility": "Centro di Riabilitazione Villa Beretta", "city": "Costa Masnaga", "state": "Lecco", "country": "Italy", "status": "", "lat": 45.76963, "lon": 9.27632}, {"facility": "Istituti a Carattere Scientifico Maugeri Milano Camaldoli", "city": "Milan", "state": "Milano", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Istituti a Carattere Scientifico Maugeri Montescano", "city": "Montescano", "state": "Pavia", "country": "Italy", "status": "", "lat": 45.03196, "lon": 9.28366}, {"facility": "Istituti a Carattere Scientifico Maugeri Pavia", "city": "Pavia", "state": "Pavia", "country": "Italy", "status": "", "lat": 45.19205, "lon": 9.15917}, {"facility": "Universit\u00e0 di Pisa", "city": "Pisa", "state": "Pisa", "country": "Italy", "status": "", "lat": 43.70853, "lon": 10.4036}], "contact_phone": "0039.0341.2350202", "contact_email": "marta.mondellini@cnr.it", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06714396", "title": "A Study Exploring the PK/PD Relationship of QRL-101 in Adults With ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "QurAlis Corporation", "summary": "This Phase 1 randomized, placebo-controlled, double-blind study will evaluate the PK/PD relationship of single doses of QRL-101 in 12 ALS participants. The study will also assess safety and tolerability in participants receiving either QRL-101 or placebo.", "interventions": [{"type": "DRUG", "name": "QRL-101"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2024-11-11", "url": "https://clinicaltrials.gov/study/NCT06714396", "target_entities": [], "locations": [{"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "Netherlands", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "QRL-101", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04695210", "title": "Virtual Peer-to-peer Support Programme for Carers of MND", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "King's College London", "summary": "Background/scope There is growing recognition that family caregiving is a serious public health issue requiring supportive interventions. Family caregivers play an essential role in sustaining a stable environment enabling individuals with motor neurone disease (MND) that are technology dependent to live at home. The family caregivers can experi\u00acence exceptional burden and significant decline in psychological wellbeing due to MND's rapid and pro\u00acgressive nature with profoundly debilitating effects and intensive support needs. Dependence on assistive technology adds an additional level of complexity to family caregiving due to the need to learn how to operate and troubleshoot medical devices, train other caregivers, and negotiate appointments with new specialties within the healthcare system.\n\nDespite the recognized impact of caregiving for individuals with MND, data are scarce as to effective interventions that provide direct practical and psychosocial supports. Difficulty accessing support may increase psychological distress. As the burden of caring increases due to disease progression and increasing technology dependence, access to existing informal support networks may diminish. Online peer support using virtual modalities is a flexible and low cost form of support. Peers, people who have experienced the same health problem and have similar characteristics as support recipients, can be a key source of emotional, informational, and affirmational support. Peer support improves psychological well-being of caregivers of people with conditions such as dementia, cancer, and brain injury. Although peer support programmes for family caregivers of people with MND exist, data as to their efficacy are limited. Therefore, we have developed an online peer support programme, completed beta and usability testing and now propose to test the effect on caregiver psychological wellbeing and caregiver burden.\n\nAim/research question(s) Overall aim: to determine the efficacy of a 12-week online peer support programme on family caregiver psychological health and caregiver burden.\n\nPrimary research question:\n\nWhat is the effect of the online peer support programme on psychological distress measured using the Hospital Anxiety and Depression Scale (HADS)?\n\nSecondary research questions:\n\n1. What is the effect on positive affect, caregiver burden, caregiving mastery, caregiving personal gain, and coping?\n2. How do participants use the programme (fidelity and reach)?\n3. What is the perceived usability and acceptability?\n\nMethods The investigators will conduct a parallel group randomised controlled trial with participants allocated to 12-week access to the online peer support programme or a usual care control group. The investigators will enrol family caregivers of an individual with MND who is referred for consideration or receiving any of the following\n\n1. assisted ventilation\n2. cough assist\n3. gastroscopy and enteral feeding\n\ni.e., entering King's clinical staging Stage 4A: nutritional support; or Stage 4B: respiratory support \\[51\\]:\n\nThe 12-week peer-to-peer support programme entails:\n\n1. audio, video, or text private messaging;\n2. synchronous weekly chat;\n3. asynchronous discussion forum; and\n4. informational resources.\n\nThe investigators will collect demographic and caregiving data including the Caregiver Assistance Scale and Caregiving Impact Scale, and caregiver measures (HADS, Positive and Negative Affect Schedule, Zarit Burden Interview, Pearlin Mastery Scale, Personal Gain Scale, Brief COPE) at baseline and programme completion.\n\nThe investigators will download use of online peer support programme features, assess usability, and conduct semi-structured interviews to explore acceptability using the Theoretical Framework of Acceptability.\n\nTo test for a medium size effect (d=0.5), at 5% level of significance (2-sided) with power 80%, 64 participants are required in each arm (128 total). Adjusting for 20% attrition requires 154 participants.\n\nProposed findings The proposed study will demonstrate the effect of a online peer support programme on psychological distress, positive affect, caregiving burden, mastery, personal gain and coping. Data on programme fidelity will enable the investigators to objectively assess acceptability and interpret study results. Data on usability and acceptability will inform future scalability of the online peer support programme outside of the trial both nationally and internationally, and to other family caregiver populations.", "interventions": [{"type": "BEHAVIORAL", "name": "Virtual peer-to-peer support"}], "start_date": "2022-06-07", "url": "https://clinicaltrials.gov/study/NCT04695210", "target_entities": [], "locations": [{"facility": "Bedfordshire Hospitals NHS Foundation Trust", "city": "Bedford", "state": "", "country": "United Kingdom", "status": "", "lat": 52.13459, "lon": -0.46632}, {"facility": "Airedale NHS Foundation Trust", "city": "Bradford", "state": "", "country": "United Kingdom", "status": "", "lat": 53.79391, "lon": -1.75206}, {"facility": "University Hospitals Sussex NHS Foundation Trust", "city": "Brighton", "state": "", "country": "United Kingdom", "status": "", "lat": 50.82838, "lon": -0.13947}, {"facility": "Pilgrims Hospice", "city": "Canterbury", "state": "", "country": "United Kingdom", "status": "", "lat": 51.27904, "lon": 1.07992}, {"facility": "Coventry Community Specialist Palliative Care Team", "city": "Coventry", "state": "", "country": "United Kingdom", "status": "", "lat": 52.40656, "lon": -1.51217}, {"facility": "University Hospitals Coventry and Warwickshire NHS Trust", "city": "Coventry", "state": "", "country": "United Kingdom", "status": "", "lat": 52.40656, "lon": -1.51217}, {"facility": "Ninewells Hospital", "city": "Dundee", "state": "", "country": "United Kingdom", "status": "", "lat": 56.46913, "lon": -2.97489}, {"facility": "Royal Devon University Healthcare NHS Foundation Trust", "city": "Exeter", "state": "", "country": "United Kingdom", "status": "", "lat": 50.7236, "lon": -3.52751}, {"facility": "Phyllis Tuckwell Hospice", "city": "Farnham", "state": "", "country": "United Kingdom", "status": "", "lat": 51.21444, "lon": -0.80054}, {"facility": "Medway Community Healthcare", "city": "Gillingham", "state": "", "country": "United Kingdom", "status": "", "lat": 51.38914, "lon": 0.54863}, {"facility": "Kingston Hospital NHS Foundation", "city": "Kingston upon Thames", "state": "", "country": "United Kingdom", "status": "", "lat": 51.41259, "lon": -0.2974}, {"facility": "The Leicestershire & Rutland Organisation for the Relief of Suffering - LOROS", "city": "Leicester", "state": "", "country": "United Kingdom", "status": "", "lat": 52.6386, "lon": -1.13169}, {"facility": "The Walton Centre NHS Foundation Trust", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "Guy's and St Thomas' NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Lancashire Teaching Hospitals NHS Foundation Trust", "city": "Many Locations", "state": "", "country": "United Kingdom", "status": "", "lat": null, "lon": null}, {"facility": "Marie Curie Hospice", "city": "Many Locations", "state": "", "country": "United Kingdom", "status": "", "lat": null, "lon": null}, {"facility": "Nottinghamshire Healthcare NHS Foundation Trust", "city": "Many Locations", "state": "", "country": "United Kingdom", "status": "", "lat": null, "lon": null}, {"facility": "Swansea Bay University Health Board", "city": "Many Locations", "state": "", "country": "United Kingdom", "status": "", "lat": null, "lon": null}, {"facility": "Royal Stoke University Hospital/University Hospitals North", "city": "Multiple Locations", "state": "", "country": "United Kingdom", "status": "", "lat": null, "lon": null}, {"facility": "University Hospitals Plymouth NHS trust", "city": "Plymouth", "state": "", "country": "United Kingdom", "status": "", "lat": 50.37153, "lon": -4.14305}, {"facility": "Sheffield Teaching Hospitals NHS Foundation Trust", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}, {"facility": "Severn Hospice", "city": "Shrewsbury", "state": "", "country": "United Kingdom", "status": "", "lat": 52.71009, "lon": -2.75208}, {"facility": "St Margaret's Hospice", "city": "Taunton", "state": "", "country": "United Kingdom", "status": "", "lat": 51.01494, "lon": -3.10293}, {"facility": "Hounslow and Richmond Community Healthcare", "city": "Teddington", "state": "", "country": "United Kingdom", "status": "", "lat": 51.42233, "lon": -0.33053}, {"facility": "Compton Care", "city": "Wolverhampton", "state": "", "country": "United Kingdom", "status": "", "lat": 52.58547, "lon": -2.12296}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07077668", "title": "Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation", "phase": "EARLY_PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Beijing Tiantan Hospital", "summary": "This study primarily evaluates the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting.", "interventions": [{"type": "DRUG", "name": "A sterile aqueous solution of RAG - 17 preparation (sodium salt)"}], "start_date": "2025-07", "url": "https://clinicaltrials.gov/study/NCT07077668", "target_entities": ["SOD1"], "locations": [], "contact_phone": "13911666571", "contact_email": "yilong528@gmail.com", "mechanism_summary": {"compound": "RAG-17", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07071935", "title": "A Clinical Trial of Early Ventilation in Amyotrophic Lateral Sclerosis (EVENT ALS)", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Pennsylvania", "summary": "Amyotrophic lateral sclerosis (ALS) is a disease that causes weakness of the muscles of the body. The disease can eventually lead to severe breathing problems, which is the most common cause of death from ALS. The treatment for breathing is non-invasive ventilation (NIV). It is a machine that helps a person breathe by pushing air in and out of their lungs through a mask worn over the face. Research has shown that NIV can improve the quality of life and survival of someone with ALS. Unfortunately, NIV is not equally beneficial for everyone. The investigators do not yet know the best time or method for starting NIV in ALS. Europe and Canada allow starting NIV much earlier in ALS than the United States. Current recommendations for starting NIV are based on the opinion of experts rather than large research studies. Medical insurance companies will not cover NIV until significant breathing weakness occurs. After NIV is started, there is no evidence-based guidance on the best way to adjust NIV to benefit patients as much as possible. Some patients have difficulty tolerating NIV, but it is not clear how to identify these individuals ahead of time.\n\nThe investigators have created a new prediction tool that can identify patients at high risk of breathing problems within the next 6 months. This may help the study team identify who is more likely to benefit from starting NIV early. The investigators have published a paper that shows that NIV helps people with ALS live longer. This paper also showed that patients get more benefit with use NIV for at least 4 hours per day. The investigators published another paper that measured a gas called carbon dioxide (CO2), which goes high if someone's breathing is weakened. This paper showed that patients with ALS may live longer when CO2 levels are lowered using NIV. The investigators also have data suggesting that certain characteristics may predict who is less likely to use NIV at least 4 hours per day.\n\nIn this study, the investigators will collect pilot data on starting early NIV in individuals with ALS who do not yet meet insurance criteria for covering NIV. The research team will first use their previously published prediction tool to identify patient risk. Then, subjects would be randomized to start early NIV or to usual care. The usual care group would eventually start NIV as would occur if the participants were not in the study.\n\nThe purpose of this study is to collect data to help the investigators plan a larger randomized clinical trial. This study has 4 objectives. First, the project aims to identify individuals who would benefit from earlier NIV. The research team will use the original prediction tool to identify risk of severe breathing problems within the next 6 months. Second, the project aims to show that it is feasible to start NIV early. Third, the project aims to gather data on the effect of randomization on symptoms, CO2 levels, and outcomes. Fourth, the project aims to identify traits that may make someone less likely to use NIV.", "interventions": [{"type": "DEVICE", "name": "Non-invasive ventilation"}], "start_date": "2026-06-11", "url": "https://clinicaltrials.gov/study/NCT07071935", "target_entities": [], "locations": [{"facility": "Penn State Hershey ALS Clinic", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 40.28592, "lon": -76.65025}, {"facility": "Penn Comprehensive ALS Center at Pennsylvania Hospital", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 39.95238, "lon": -75.16362}, {"facility": "Thomas Jefferson University Weinberg ALS Center", "city": "Philadelphi", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": null, "lon": null}], "contact_phone": "215-829-3053", "contact_email": "jason.ackrivo@pennmedicine.upenn.edu", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03981536", "title": "A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AL-S Pharma", "summary": "Single ascending doses of AP-101 will be administered by intravenous (IV) infusion", "interventions": [{"type": "DRUG", "name": "AP-101"}], "start_date": "2019-10-10", "url": "https://clinicaltrials.gov/study/NCT03981536", "target_entities": [], "locations": [{"facility": "London Health Sciences Centre, University Hospital", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook Health Sciences Centre, Toronto", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute & Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "AP-101", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03293394", "title": "Rehabilitative Trial With tDCS in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a motor neuron disease, which is a group of neurological disorders that selectively affect motor neurons, the cells that control voluntary muscles of the body. The disorder causes muscle weakness and atrophy throughout the body due to the degeneration of the upper and lower motor neurons. Current drugs approved for ALS treatment only modestly slow disease progression.\n\nTranscranial direct current stimulation (tDCS) is a non-invasive technique, which has been demonstrated to modulate cerebral excitability in several neurodegenerative disorders and modulate intracortical connectivity measures.\n\nIn this randomized, double-blind, sham-controlled study, the investigators will evaluate whether a two-weeks' treatment with bilateral motor cortex anodal tDCS and spinal cathodal tDCS can improve symptoms in patients with amyotrophic lateral sclerosis and modulate intracortical connectivity, at short and long term.", "interventions": [{"type": "DEVICE", "name": "Anodal bilateral motor cortex and cathodal spinal tDCS"}, {"type": "DEVICE", "name": "Sham bilateral motor cortex and sham spinal tDCS"}], "start_date": "2017-10-02", "url": "https://clinicaltrials.gov/study/NCT03293394", "target_entities": ["motor_cortex_excitability"], "locations": [{"facility": "AO Spedali Civili", "city": "Brescia", "state": "BS", "country": "Italy", "status": "", "lat": 45.53558, "lon": 10.21472}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Transcranial direct current stimulation (tDCS) is a non-invasive neuromodulation technique applied to motor cortex and spinal cord to modulate neuronal activity.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01254539", "title": "Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia", "summary": "The purpose of this clinical trial is to assess the feasibility and the security of the intraspinal and intrathecal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients.", "interventions": [{"type": "PROCEDURE", "name": "Laminectomy and bone marrow stem cells transplantation"}, {"type": "PROCEDURE", "name": "Intrathecal infusion of autologous bone marrow stem cells"}, {"type": "PROCEDURE", "name": "Intrathecal infusion of placebo (saline solution)."}], "start_date": "2010-10", "url": "https://clinicaltrials.gov/study/NCT01254539", "target_entities": ["neuroprotection_and_neuroregeneration_via_stem_cell_transplantation"], "locations": [{"facility": "Hospital Universitario Virgen de la Arrixaca", "city": "El Palmar", "state": "Murcia", "country": "Spain", "status": "", "lat": 37.93939, "lon": -1.16095}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Autologous bone marrow stem cells", "targeting_mechanism": "Stem cells differentiate into support cells such as astrocytes and oligodendrocytes, which may benefit degenerating motor neurons by producing growth factors and anti-inflammatory cytokines, providing nutrients and buffering excessive glutamate.", "targeting_mechanism_pmid": "32043626", "animal_results": "Stem cell therapies have been demonstrated to have potential benefit in preclinical studies, with the majority performed in mouse and rat ALS models expressing mutant superoxide dismutase 1.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06889857", "title": "Safety and Efficacy of Intravenous Administration of SHED-CM for ALS", "phase": "EARLY_PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hitonowa Medical", "summary": "This study evaluates the safety and efficacy of Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media (SHED-CM) in patients with Amyotrophic Lateral Sclerosis (ALS), using the Japanese version of the revised ALS Functional Rating Scale (ALSFRS-R) as an indicator.", "interventions": [{"type": "BIOLOGICAL", "name": "The study drug is SHED-CM manufactured by U-Factor"}], "start_date": "2024-04-05", "url": "https://clinicaltrials.gov/study/NCT06889857", "target_entities": ["neuroprotection_via_stem_cell_secretome"], "locations": [{"facility": "Hitonowa Medical", "city": "Chiyoda City", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.68449, "lon": 139.75056}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "SHED-CM (Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01825551", "title": "The Effect of GCSF in the Treatment of ALS Patients", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Tehran University of Medical Sciences", "summary": "The aim of this study is to evaluate the effect off Granulocyte Colony Stimulating Factor (GCSF) in the treatment of Amyotrophic Lateral Sclerosis (ALS) patients.", "interventions": [{"type": "DRUG", "name": "Granulocyte Colony Stimulating Factor"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2012-11", "url": "https://clinicaltrials.gov/study/NCT01825551", "target_entities": ["monocyte_macrophage_differentiation_and_neuroprotection"], "locations": [{"facility": "Iranian Neurology Research Center of Tehran University of Medical Sciences", "city": "Tehran", "state": "Tehran Province", "country": "Iran", "status": "", "lat": 35.69439, "lon": 51.42151}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Granulocyte Colony Stimulating Factor (GCSF)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05979688", "title": "Yogic Breathing Exercise for People With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Alabama at Birmingham", "summary": "The aim of this study is to understand how well a 6-week virtual yogic breathing exercise program (YBEP) will improve breathing, speech, and emotional well-being in people with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "BEHAVIORAL", "name": "yogic breathing exercise"}], "start_date": "2023-11-01", "url": "https://clinicaltrials.gov/study/NCT05979688", "target_entities": [], "locations": [{"facility": "University of Alabama at Birmingham", "city": "Birmingham", "state": "Alabama", "country": "United States", "status": "", "lat": 33.52066, "lon": -86.80249}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Yogic breathing exercise is a behavioral intervention designed to improve respiratory muscle function and ventilation in ALS patients.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04997954", "title": "EMERALD TRIAL Open Label Extension Study", "phase": "PHASE4", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Gold Coast Hospital and Health Service", "summary": "EMERALD OLE trial is an open-label extension of the EMERALD trial. Long term tolerability and safety of the MediCabilis CBD oil has not been extensively studied. EMERALD OLE aims to establish data on the prolonged used of the study drug product.\n\nAll participants who completed the EMERALD trial will be offered to enter EMERALD OLE. Participants will be taking the active drug MediCabilis CBD oil for 6 months.", "interventions": [{"type": "DRUG", "name": "MediCabilis CBD oil"}], "start_date": "2021-05-17", "url": "https://clinicaltrials.gov/study/NCT04997954", "target_entities": ["cb1_and_cb2_receptors"], "locations": [{"facility": "Gold Coast Hospital and Health Service", "city": "Gold Coast", "state": "Queensland", "country": "Australia", "status": "", "lat": -28.00029, "lon": 153.43088}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "MediCabilis CBD oil", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00561366", "title": "A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALS", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "CytRx", "summary": "Arimoclomol is a small molecule that upregulates \"molecular chaperones\" in cells under stress. Arimoclomol extends survival by five weeks when given both pre-symptomatically and at disease onset in a mutant superoxide dismutase (SOD1) transgenic mouse model of ALS. Furthermore, it has been demonstrated to have neuroprotective and neuroregenerative effects in other rat models of nerve damage. Molecular chaperone proteins are critical in the cellular response to stress and protein misfolding. Recent data suggest that the SOD1 mutation responsible for ALS in some patients with familial disease reduces the availability of a variety of molecular chaperones, and thus weakens their ability to respond to cellular stress. Protein misfolding and consequent aggregation may play a role in the pathogenesis of both the familial and sporadic forms of ALS. Therapeutic agents such as arimoclomol that improve cellular chaperone response to protein misfolding may be helpful in ALS.", "interventions": [{"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Arimoclomol"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT00561366", "target_entities": ["heat_shock_proteins_molecular_chaperones"], "locations": [{"facility": "University of California Los Angeles - Tier 2 Site", "city": "Pacific Palisades", "state": "California", "country": "United States", "status": "", "lat": 34.04806, "lon": -118.52647}, {"facility": "University of California - San Francisco - Tier 2 Site", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Health Sciences Center - Tier 2 Site", "city": "Denver", "state": "Colorado", "country": "United States", "status": "", "lat": 39.73915, "lon": -104.9847}, {"facility": "University of Miami - Tier 2 Site", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Emory University - Tier 2 site", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern University, Dept. of Neurology - Tier 2 Site", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas Medical Center - Tier 2 site", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "John Hopkins University - Tier 2 Site", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital - Tier 1 Site", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Baystate Medical Center - Tier 2 Site", "city": "Springfield", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.10148, "lon": -72.58981}, {"facility": "Saint Louis University, Neuromuscular Div. - Tier 2 Site", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Washington University - Tier 2 Site", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "BryanLGH Medical Center - Tier 2 Site", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Upstate Clinical Research, LLC - Tier 2 Site", "city": "Albany", "state": "New York", "country": "United States", "status": "", "lat": 42.65258, "lon": -73.75623}, {"facility": "Mount Sinai School of Medicine - Tier 2 Site", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University Medical Center - Tier 2 site", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Downstate Medical Center - Tier 1 Site", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Duke University Medical Center - Tier 1 Site", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest University School of Medicine -Tier 2 Site", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Cleveland Clinic Foundation -Tier 2 site", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Providence ALS Center - Tier 2 Site", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Pennsylvania State University School of Medicine - Tier 2 Site", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University College of Medicine - Tier 1 Site", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburgh Medical Center - Tier 2 Site", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Vanderbilt University Medical Center - Tier 2", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology, PA - Tier 2 Site", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Texas Health Science Center - Tier 2 Site", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Vermont, College of Medicine - Tier 2", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "University of Virginia - Tier 2 Sites", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "Virginia Mason Clinic - Tier 2 Site", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Medical College of Wisconsin - Tier 2 Site", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "University of British Columbia, Gordon and Leslie Diamond Health Care Centre - Tier 2 Site", "city": "Vancouver", "state": "British Columbia", "country": "Canada", "status": "", "lat": 49.24966, "lon": -123.11934}, {"facility": "London Health Science Center - Tier 2 Site", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "University of Toronto, Sunnybrook Health Sciences Centre - Tier 2 Site", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute - Tier 2", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Arimoclomol", "targeting_mechanism": "Arimoclomol upregulates molecular chaperones in cells under stress.", "targeting_mechanism_pmid": "", "animal_results": "Arimoclomol extends survival by five weeks when given both pre-symptomatically and at disease onset in a mutant superoxide dismutase (SOD1) transgenic mouse model of ALS.", "animal_results_pmid": "31900865", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00107770", "title": "Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "US Department of Veterans Affairs", "summary": "The purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this medication without major side effects.", "interventions": [{"type": "DRUG", "name": "sodium phenylbutyrate"}], "start_date": "2005-04", "url": "https://clinicaltrials.gov/study/NCT00107770", "target_entities": ["protein_aggregation_and_cellular_stress"], "locations": [{"facility": "VA Medical Center, Iowa City", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "VA Medical Center, Lexington", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "VA Maryland Health Care System, Baltimore", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Edith Nourse Rogers Memorial Veterans Hospital, Bedford", "city": "Bedford", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.49065, "lon": -71.27617}, {"facility": "VA Medical Center, Jamaica Plain Campus", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "VA Medical Center, Syracuse", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "VA Medical Center, Durham", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "VA Medical Center, Cincinnati", "city": "Cincinnati", "state": "Ohio", "country": "United States", "status": "", "lat": 39.12711, "lon": -84.51439}, {"facility": "VA Pittsburgh Health Care System", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Michael E. DeBakey VA Medical Center (152)", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "sodium phenylbutyrate", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04947436", "title": "ALS and Airway Clearance (ALSAC) Therapy", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The University of Texas Health Science Center at San Antonio", "summary": "Patients will receive one of three respiratory therapy interventions for airway clearance assistance: 1) High frequency chest wall oscillation (HFCWO) and mechanical insufflation/exsufflation (MIE), 2) HFCWO or 3) MIE. The study period will be six months and include three clinic visits, baseline and follow-up visits at 3 and 6 months, and 6 monthly home visits by the respiratory therapist.", "interventions": [{"type": "DEVICE", "name": "High Frequency Chest Wall Oscillation"}, {"type": "DEVICE", "name": "Mechanical insufflation/exsufflation"}], "start_date": "2012-01-25", "url": "https://clinicaltrials.gov/study/NCT04947436", "target_entities": [], "locations": [{"facility": "University of Texas Health San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04326283", "title": "Trial of Safety, Tolerability and Efficacy of Trametinib (SNR1611) in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Genuv Inc.", "summary": "The purpose of this study is to evaluate the safety, tolerability and efficacy of trametinib (SNR1611) in the treatment of amyotrophic lateral sclerosis.", "interventions": [{"type": "DRUG", "name": "Trametinib (0.5 mg)"}, {"type": "DRUG", "name": "Trametinib (1 mg)"}, {"type": "DRUG", "name": "Riluzole (100 mg)"}], "start_date": "2020-04-02", "url": "https://clinicaltrials.gov/study/NCT04326283", "target_entities": ["mapk_erk_pathway"], "locations": [{"facility": "Inje University Busan Paik Hospital", "city": "Busan", "state": "", "country": "South Korea", "status": "", "lat": 35.10168, "lon": 129.03004}, {"facility": "Asan Medical Center", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Korea University Anam Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Samsung Medical Center", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}, {"facility": "Severance Hospital, Yonsei University Health System", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Trametinib", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00875446", "title": "First Time in Human Study of GSK1223249 in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "GlaxoSmithKline", "summary": "The drug being tested in this study is GSK1223249. It is being developed by GlaxoSmithKline to treat symptoms in patients with Amyotrophic Lateral Sclerosis (ALS).\n\nThe drug works by inhibiting the protein that prevents nerve growth.\n\nThis will be the first time the drug will be given to man. The trial is expected to involve approximately 76 patients. The study objective is to investigate the tolerability, safety and the way the body handles GSK1223249 after a range of single doses or repeat dose escalation in patients with ALS.", "interventions": [{"type": "DRUG", "name": "PLACEBO"}, {"type": "DRUG", "name": "GSK1223249"}], "start_date": "2009-05-13", "url": "https://clinicaltrials.gov/study/NCT00875446", "target_entities": ["nerve_growth_factor_pathway"], "locations": [{"facility": "GSK Investigational Site", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "GSK Investigational Site", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "GSK Investigational Site", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "GSK Investigational Site", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "GSK Investigational Site", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "GSK Investigational Site", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "GSK Investigational Site", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "GSK Investigational Site", "city": "Verona", "state": "Veneto", "country": "Italy", "status": "", "lat": 45.43854, "lon": 10.9938}, {"facility": "GSK Investigational Site", "city": "Birmingham", "state": "", "country": "United Kingdom", "status": "", "lat": 52.48142, "lon": -1.89983}, {"facility": "GSK Investigational Site", "city": "Cambridge", "state": "", "country": "United Kingdom", "status": "", "lat": 52.2, "lon": 0.11667}, {"facility": "GSK Investigational Site", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "GSK Investigational Site", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "GSK1223249", "targeting_mechanism": "Inhibits the protein that prevents nerve growth.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06891716", "title": "[18F]ACI-19626 PET in TDP-43 Proteinopathies", "phase": "EARLY_PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AC Immune SA", "summary": "The goal of this clinical trial is to test whether we can reliably and safely measure the accumulation of pathological protein TDP-43 \\[involved in rare forms of dementia such as frontotemporal dementia (FTD) and in amyotrophic lateral sclerosis (ALS)\\] using a new positron emission tomography (PET) tracer called \\[18F\\]ACI-19626. Both healthy people and people with (suspected) TDP-43 accumulation will participate to this trial.\n\nThe main questions it aims to answer are:\n\n* whether \\[18F\\]ACI-19626 is safe and well tolerated when injected into participants\n* whether \\[18F\\]ACI-19626 reliably detects abnormal TDP-43 in the brain using PET technique.\n* whether there are differences in the amount of this protein between people with diseases related to TDP-43 accumulation in the brain and people without these diseases.\n\nParticipants will:\n\n* Visit the clinic to consent to their participation and to ensure they are eligible (physical and neurological examinations, questionnaires, blood and urine tests, ECG and MRI in some cases).\n* Visit the clinic to receive the tracer \\[18F\\]ACI-19626 intravenously and be scanned in a PET scanner, during which blood will be collected.\n* Receive a phone call from the clinic 2 to 4 days after the PET scan to report any symptoms and side-effects that they may be having.\n\nSome of the participants may be asked to come again to the clinic for a second PET scan, allowing the researchers to determine if the measurements with the first PET scan are stable and reproducible.", "interventions": [{"type": "OTHER", "name": "[18F]ACI-19626"}], "start_date": "2025-01-21", "url": "https://clinicaltrials.gov/study/NCT06891716", "target_entities": ["TARDBP"], "locations": [{"facility": "Amsterdam UMC", "city": "Amsterdam", "state": "", "country": "Netherlands", "status": "RECRUITING", "lat": 52.37403, "lon": 4.88969}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "[18F]ACI-19626", "targeting_mechanism": "A positron emission tomography (PET) tracer designed to measure the accumulation of pathological protein TDP-43.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07080801", "title": "Study on the Safety and Efficacy of RAG-21 in the Treatment of Amyotrophic Lateral Sclerosis Patients With FUS Gene Mutations", "phase": "EARLY_PHASE1", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Beijing Tiantan Hospital", "summary": "Amyotrophic lateral sclerosis (ALS) is a chronic progressive neurodegenerative disease that remains incurable, with limited existing therapies or drugs available. Familial ALS can be caused by mutations in various genes. In Asia, mutations in the FUS gene are relatively common among early-onset familial ALS patients. Reducing the levels of toxic FUS protein may be an effective therapeutic approach for such ALS patients without causing side effects.\n\nRAG-21 is a small interfering ribonucleic acid (siRNA) with a molecular weight of 20 kDa. Through the RNA interference mechanism, it targets the FUS gene, recognizes the corresponding mRNA, and mediates its degradation, thereby downregulating FUS gene expression and reducing toxic FUS protein levels. Accordingly, this project plans to conduct a single-center, dose-escalation clinical study aimed at evaluating the safety, tolerability, and pharmacokinetics of intrathecal bolus administration of RAG-21 in ALS patients carrying FUS gene mutations.", "interventions": [{"type": "DRUG", "name": "RAG-21"}], "start_date": "2025-08", "url": "https://clinicaltrials.gov/study/NCT07080801", "target_entities": ["FUS"], "locations": [], "contact_phone": "13521588395", "contact_email": "liuxinru0826@163.com", "mechanism_summary": {"compound": "RAG-21", "targeting_mechanism": "A small interfering RNA that reduces the levels of toxic FUS protein in FUS-mutant ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01777646", "title": "Autologous Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Brainstorm-Cell Therapeutics", "summary": "The study will evaluate the safety, tolerability and therapeutic effects of transplantation of escalating doses of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF), in patients with amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "BIOLOGICAL", "name": "MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF"}], "start_date": "2012-12", "url": "https://clinicaltrials.gov/study/NCT01777646", "target_entities": ["neurotrophic_factors"], "locations": [{"facility": "Hadassah Medical Organization", "city": "Jerusalem", "state": "", "country": "Israel", "status": "", "lat": 31.76904, "lon": 35.21633}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "MSC-NTF cells (autologous mesenchymal bone marrow stromal cells secreting neurotrophic factors)", "targeting_mechanism": "Autologous mesenchymal stromal cells engineered to secrete neurotrophic factors that provide neuroprotective support to degenerating motor neurons.", "targeting_mechanism_pmid": "31054608", "animal_results": "In SOD1G93A mice, hBM-MSC expressing Ngn1 (a similar mesenchymal stromal cell approach) administered intravenously showed increased lifespan of 3 days, delayed disease onset of 5 days, and reduced motor neuron loss.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05595850", "title": "A Mindful Community for People With ALS and Their Primary Caregivers", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Harvard University", "summary": "The psychological impact of ALS on patients and caregivers is high, significantly affecting their quality of life (QOL). Despite this impact, there is not much research about psychological interventions that could reduce psychological distress and improve QOL.\n\nThe efficacy of mindfulness-based treatments for the improvement of QOL was previously demonstrated by the investigator's group. Despite preliminary positive results, treatment efficacy tends to weaken over time. The investigators believe that a robust solution to maintain efficacy is to maximize the utilization of technology and emerging social platforms, establishing a \"mindful community\" to promote and continuously reinforce mindfulness.\n\nThis project's primary aims are 1) to develop a \"mindful\" online community of people with ALS and their caregivers, and 2) to test its efficacy in QOL improvement. This two-part intervention consists of 1) optimizing the investigator's prior e-learning platform with a three-week program including cognitive exercises, videos and lectures to increase participants' mindfulness; and 2) involving participants in a \"mindfulness community\" within a social sharing forum. Assessments will be performed before and immediately post-treatment as well as 3- and 6-months post-program comparing subjects undergoing the intervention to a control group.", "interventions": [{"type": "BEHAVIORAL", "name": "Mindfulness"}, {"type": "BEHAVIORAL", "name": "Mindful Learning"}], "start_date": "2020-05-15", "url": "https://clinicaltrials.gov/study/NCT05595850", "target_entities": [], "locations": [{"facility": "Harvard University", "city": "Cambridge", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.3751, "lon": -71.10561}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04809675", "title": "Effectiveness of Two Oral Appliances for Managing Oral Self-biting Injuries in Patients With ALS", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Barcelona", "summary": "This study assesses to determine the most effective type of device on the degree of satisfaction of patients with amyotrophic lateral sclerosis for managing oral self-biting injuries. Thirty-one patients with amyotrophic lateral sclerosis will wear two devices, a hard occlusal splint (HOS) and a flexible customized mouthguard (FCM), for two weeks each one. The sequence will be randomized to obtain one-half of the participants starting the first week wearing the HOS, and the other half wearing the FCM. The participants will rate the degree of satisfaction with the device and the degree of improvement or worsening of oral self-biting injuries in a 10-point scale. They will also rate the degree of change in their quality of life because of changes in their oral self-biting injuries. Finally they will rate the compliance and report the adverse effects.", "interventions": [{"type": "DEVICE", "name": "Hard occlusal splint"}, {"type": "DEVICE", "name": "Flexible customized mouthguard"}], "start_date": "2019-11-29", "url": "https://clinicaltrials.gov/study/NCT04809675", "target_entities": [], "locations": [{"facility": "Nina Riera-Punet", "city": "Barcelona", "state": "L'Hospitalet de Llobregat", "country": "Spain", "status": "RECRUITING", "lat": 41.38879, "lon": 2.15899}], "contact_phone": "934035555", "contact_email": "ninariera@ub.edu", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02424669", "title": "Neuroinflammation in Amyotrophic Lateral Sclerosis - Mechanisms and Therapeutic Perspectives: a Translational Pilot Study Among ALS Patients", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique Hopitaux De Marseille", "summary": "Amyotrophic Lateral Sclerosis (ALS) is the most common motor neuron diseases. It is considered as a rare disease with a prevalence of about 8 per 100,000 persons. Initiating in mid-life by progressive paralysis, it evolves rapidly into a generalized muscle wasting that leads irrevocably to death within 2 or 5 years of clinical onset.\n\nSince there is no cure for ALS, the management of the disease is supportive and palliative. Riluzole is the only drug that has been shown to extend survival by about three months. The identification of biomarkers sensitive to the progression of the disease might enhance the diagnostic and provide new drug targets.\n\nDysfunction of the immune system is a pathological hallmark of ALS. Increased levels of interferon gamma (IFNgamma) were found in the serum and cerebrospinal fluid (CSF) of ALS patients. However, the cell origin as well as the pathogenic influence of this peripheral source of IFNg is unknown. Thus, IFNgamma might have a role in the pathogenic process of ALS and might be a potential biomarker of the disease.", "interventions": [{"type": "OTHER", "name": "ALS Functional rating Scale-revised (ALS FRS-R)"}, {"type": "OTHER", "name": "slow vital capacity"}, {"type": "OTHER", "name": "Blood sample"}, {"type": "OTHER", "name": "Cerebrospinal Fluid (CSF) sample"}], "start_date": "2015-05", "url": "https://clinicaltrials.gov/study/NCT02424669", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Assistance Publique H\u00f4pitaux de Marseille", "city": "Marseille", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.29695, "lon": 5.38107}], "contact_phone": "", "contact_email": "joelle.micallef@ap-hm.fr", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "Investigating neuroinflammation as a mechanism in ALS pathogenesis and therapeutic target.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01521728", "title": "Trial of Resistance and Endurance Exercise in Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "The first questions asked by patients with a new diagnosis of Amyotrophic Lateral Sclerosis (ALS) often include: \"Does exercise help slow the progression of the disease?\", \"Is there any harm in exercising?\", or \"What type of exercise (endurance or resistance) is most appropriate?\" At this time, however, there is a lack of answers for people who suffer from an illness that affects their strength above all else. Yet the beneficial effects of exercise in both healthy people as well as people with other diseases have been extensively studied and resulted in recommendations about the types of exercise that are beneficial. In this study the investigators will ask participants with ALS to exercise in one of three ways: weightlifting (resistance exercise), stationary bicycling (endurance exercise), and range of motion exercise (the current \"standard of care\" for ALS patients). The investigators will use several different types of tests to determine whether one type of exercise is tolerated better and is safer than another. The investigators will also collect information about how the body responds to exercise in ALS. This study will help in the development of a larger national study to understand how exercise can be combined with other treatments to potentially improve strength and alter the course of the disease.", "interventions": [{"type": "OTHER", "name": "Resistance Exercise"}, {"type": "OTHER", "name": "Endurance Exercise"}, {"type": "OTHER", "name": "Stretching/Range-of-Motion"}], "start_date": "2012-01", "url": "https://clinicaltrials.gov/study/NCT01521728", "target_entities": [], "locations": [{"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04499963", "title": "Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Richard Bedlack, M.D., Ph.D.", "summary": "This will be a 6-month, widely inclusive, virtual, single-center, open-label pilot trial utilizing a historical control group.", "interventions": [{"type": "DRUG", "name": "Theracurmin HP"}], "start_date": "2020-08-28", "url": "https://clinicaltrials.gov/study/NCT04499963", "target_entities": ["curcumin"], "locations": [{"facility": "Duke University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Theracurmin HP", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02881489", "title": "Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Warmia and Mazury", "summary": "The goal of this study is to investigate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells administration in the individuals with diagnosed amyotrophic lateral sclerosis.", "interventions": [{"type": "OTHER", "name": "Biological: Cell-based therapy"}], "start_date": "2015-11", "url": "https://clinicaltrials.gov/study/NCT02881489", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Autologous bone marrow mesenchymal stem cells", "targeting_mechanism": "Mesenchymal stem cells differentiate into support cells that produce growth factors and anti-inflammatory cytokines to benefit degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Preclinical stem cell studies in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1) demonstrated potential benefit of stem cell therapy.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03613038", "title": "A Systematic Investigation of Phonetic Complexity Effects on Articulatory Motor Performance in Progressive Dysarthria", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Missouri-Columbia", "summary": "The goal is to improve the fundamental knowledge about articulatory motor performance in people with Lou Gehrig's disease (also known as ALS) and Parkinson's disease (PD), in order to develop more sensitive assessments for progressive speech loss, which may lead to the improved timing of speech therapies.", "interventions": [{"type": "BEHAVIORAL", "name": "Phonetic complexity effects on speech motor performance"}], "start_date": "2017-07-15", "url": "https://clinicaltrials.gov/study/NCT03613038", "target_entities": [], "locations": [{"facility": "University of Kansas Medical Center", "city": "Fairway", "state": "Kansas", "country": "United States", "status": "", "lat": 39.02223, "lon": -94.6319}, {"facility": "University of Missouri-Columbia", "city": "Columbia", "state": "Missouri", "country": "United States", "status": "", "lat": 38.95171, "lon": -92.33407}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05683860", "title": "Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)", "phase": "PHASE1, PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Wave Life Sciences USA, Inc.", "summary": "This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the study, participants must have successfully completed Phase 1b/2a WVE-004-001 study.", "interventions": [{"type": "DRUG", "name": "WVE-004 10 mg Q12W"}], "start_date": "2022-12-14", "url": "https://clinicaltrials.gov/study/NCT05683860", "target_entities": ["C9orf72"], "locations": [{"facility": "Erasmus MC", "city": "Rotterdam", "state": "", "country": "Netherlands", "status": "", "lat": 51.9225, "lon": 4.47917}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "University of Oxford - Nuffield Department of Clinical Neurosciences", "city": "Oxford", "state": "", "country": "United Kingdom", "status": "", "lat": 51.75222, "lon": -1.25596}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "WVE-004", "targeting_mechanism": "WVE-004 targets the C9orf72 hexanucleotide repeat expansion through antisense oligonucleotide-mediated reduction of C9orf72 repeat transcripts in C9-ALS/FTD patients.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05995782", "title": "A Phase 1, SAD and MAD Study to Evaluate the Safety and Tolerability of FB418", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "1ST Biotherapeutics, Inc.", "summary": "The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics and pharmacodynamics of oral doses of FB418 in healthy adult subjects and healthy elderly subjects.", "interventions": [{"type": "DRUG", "name": "FB418"}], "start_date": "2023-12-20", "url": "https://clinicaltrials.gov/study/NCT05995782", "target_entities": [], "locations": [{"facility": "Seoul National University", "city": "Seoul", "state": "", "country": "South Korea", "status": "RECRUITING", "lat": 37.566, "lon": 126.9784}], "contact_phone": "+82-31-895-4677", "contact_email": "info@1stbio.com", "mechanism_summary": {"compound": "FB418", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07142291", "title": "PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "PhenoNet, Inc.", "summary": "The purpose of this study is to test the effects of PHENOGENE-1A, which is the treatment under investigation in this study. This research will investigate if PHENOGENE-1A can help people with ALS by measuring their function using the ALS Functional Rating Scale Revised (ALSFRS-R), measuring lung function using pulmonary function tests (PFTs), such as forced vital capacity (FVC), and measuring neuro-inflammatory biomarkers in the blood.", "interventions": [{"type": "DRUG", "name": "Cromolyn Sodium (34.2 mg BID)"}, {"type": "DRUG", "name": "Cromolyn Sodium (17.1 mg BID)"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Riluzole (100 mg)"}], "start_date": "2025-11-25", "url": "https://clinicaltrials.gov/study/NCT07142291", "target_entities": ["mast_cell_activation"], "locations": [{"facility": "Honor Health Neurology - Bob Bove Neuroscience Institute", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "RECRUITING", "lat": 33.50921, "lon": -111.89903}, {"facility": "University of California San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 32.84727, "lon": -117.2742}, {"facility": "Sutter Health - California Pacific Medical Center Research Institute", "city": "San Francisco", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 30.33218, "lon": -81.65565}, {"facility": "Lange Neurology", "city": "New York", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 40.71427, "lon": -74.00597}, {"facility": "NEUROHK s.r.o.", "city": "Hradec Kr\u00e1lov\u00e9", "state": "", "country": "Czechia", "status": "RECRUITING", "lat": 50.20923, "lon": 15.83277}, {"facility": "Fakultni Nemocnice Hradec Kralove", "city": "Hradec Kr\u00e1lov\u00e9", "state": "", "country": "Czechia", "status": "RECRUITING", "lat": 50.20923, "lon": 15.83277}, {"facility": "Thomayer University Hospital - Fakultni Thomayerova nemocnice", "city": "Prague", "state": "", "country": "Czechia", "status": "RECRUITING", "lat": 50.08804, "lon": 14.42076}, {"facility": "Charit\u00e9 Centrum f\u00fcr Neurologie, Neurochirurgie und Psychiatrie", "city": "Berlin", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 52.52437, "lon": 13.41053}, {"facility": "DIAKOVERE Henriettenstift - Klinik f\u00fcr Neurologie und Klinische Neurophysiologie", "city": "Hanover", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universitaetsklinikum Schleswig-Holstein", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 53.86893, "lon": 10.68729}, {"facility": "Michalski i Partnerzy Lekarze Sp\u00f3\u0142ka Partnerska", "city": "Krakow", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 50.06143, "lon": 19.93658}, {"facility": "SP ZOZ Szpital Uniwersytecki w Krakowie", "city": "Krakow", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 50.06143, "lon": 19.93658}, {"facility": "Centrum Medyczne NeuroProtect (NeuroProtect Medical Center)", "city": "Warsaw", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 52.22977, "lon": 21.01178}, {"facility": "City Clinic Research", "city": "Warsaw", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 52.22977, "lon": 21.01178}, {"facility": "University Clinical Center of Serbia", "city": "Belgrade", "state": "", "country": "Serbia", "status": "RECRUITING", "lat": 44.80401, "lon": 20.46513}, {"facility": "Hospital General Universitario Dr. Balmis de Alicante", "city": "Alicante", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 38.34517, "lon": -0.48149}, {"facility": "Barcelona Sea Hospital (Hospital Del Mar De Barcelona)", "city": "Barcelona", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario Vall D Hebron", "city": "Barcelona", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario Ramon Y Cajal", "city": "Madrid", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Regional Universitario De Malaga", "city": "M\u00e1laga", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 36.72016, "lon": -4.42034}, {"facility": "Hospital Universitario Virgen Del Rocio", "city": "Seville", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 37.38283, "lon": -5.97317}], "contact_phone": "+1 (617) 784-0490", "contact_email": "delmaleh@phenonet.us", "mechanism_summary": {"compound": "Cromolyn Sodium", "targeting_mechanism": "Cromolyn sodium acts as a mast cell stabilizer and neuroprotective agent.", "targeting_mechanism_pmid": "", "animal_results": "Cromolyn sodium delayed disease onset and demonstrated neuroprotection in the SOD1(G93A) mouse model of amyotrophic lateral sclerosis.", "animal_results_pmid": "31776380", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05136885", "title": "HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.\n\nRegimen E will evaluate the safety and efficacy of a single study drug, SLS-005 (Trehalose injection, 90.5 mg/mL for intravenous infusion) in participants with ALS.", "interventions": [{"type": "DRUG", "name": "SLS-005"}, {"type": "DRUG", "name": "Matching Placebo"}], "start_date": "2022-02-21", "url": "https://clinicaltrials.gov/study/NCT05136885", "target_entities": ["autophagy_impairment"], "locations": [{"facility": "Healey Center for ALS at Mass General", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "SLS-005 (Trehalose)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04681118", "title": "Expanded Access Protocol: Repeated Administration of Nurown\u00ae (Autologous MSC-NTF Cells) for the Treatment of ALS", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Brainstorm-Cell Therapeutics", "summary": "Expanded Access for treatment with investigational product MSC-NTF cells(NurOwn\u00ae) for participants who completed all scheduled treatments and follow-up assessments in the BCT-002-US study", "interventions": [{"type": "BIOLOGICAL", "name": "NurOwn (MSC-NTF cells)"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT04681118", "target_entities": ["neurotrophic_factors"], "locations": [{"facility": "University of California Irvine Alpha Stem Cell Clinic", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "UMass Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "NurOwn (MSC-NTF cells)", "targeting_mechanism": "Mesenchymal stem cells secreting neurotrophic factors to support motor neuron survival and function", "targeting_mechanism_pmid": "", "animal_results": "Preclinical stem cell studies in mouse and rat ALS models expressing mutant superoxide dismutase 1 demonstrated the potential benefit of stem cell therapy for ALS", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00116558", "title": "Early Treatment of Amyotrophic Lateral Sclerosis (ALS) With Nutrition and Non-Invasive Positive Pressure Ventilation", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Edward Kasaraskis", "summary": "The purposes of the study are to determine the energy balance and evaluate the nutritional status of patients with ALS, and to investigate the use of NIPPV as respiratory support to treat patients with ALS.", "interventions": [{"type": "DEVICE", "name": "Early NIPPV"}, {"type": "DEVICE", "name": "Standard NIPPV"}], "start_date": "2004-08-01", "url": "https://clinicaltrials.gov/study/NCT00116558", "target_entities": [], "locations": [{"facility": "University of Colorado", "city": "Denver", "state": "Colorado", "country": "United States", "status": "", "lat": 39.73915, "lon": -104.9847}, {"facility": "University of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of Kentucky", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Henry Ford Hospital", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Beth Israel", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Pennsylvania State University", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Texas-San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Vermont", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01771640", "title": "Intrathecal Transplantation of Mesenchymal Stem Cell in Patients With ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Royan Institute", "summary": "ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). ALS is the most common of the five motor neuron diseases.Riluzole (Rilutek) is the only treatment that has been found to improve survival but only to a modest extent. It lengthens survival by several months, and may have a greater survival benefit for those with a bulbar onset. It also extends the time before a person needs ventilation support.Stem cell transplantation is a new hopeful way to improve the patients conditions and reduce the period of disabilities.", "interventions": [{"type": "BIOLOGICAL", "name": "intrathecal injection"}], "start_date": "2013-08", "url": "https://clinicaltrials.gov/study/NCT01771640", "target_entities": ["neurotrophic_factors"], "locations": [{"facility": "Royan Institute", "city": "Tehran", "state": "", "country": "Iran", "status": "", "lat": 35.69439, "lon": 51.42151}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Mesenchymal stem cells", "targeting_mechanism": "Intrathecal injection of mesenchymal stem cells to support motor neuron survival and function", "targeting_mechanism_pmid": "", "animal_results": "In SOD1 rat lumbar spinal cord, intrathecal injection of neural progenitor cells secreting GDNF at various doses showed dose-dependent delivery feasibility and efficacy assessment at disease onset", "animal_results_pmid": "36064599", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04894240", "title": "A Study of Monepantel in Individuals With Motor Neurone Disease", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neurizon Therapeutics Limited", "summary": "Amyotrophic lateral sclerosis/ Motor Neurone Disease (ALS/MND) is a rare and invariably fatal neurological disease. ALS/MND has a terribly high burden on patients, family and carers, and carries great socioeconomic burden. Current best treatment options are expensive and attempt to control disease progression and manage symptoms while offering no cure. Better treatments are wanting.\n\nMonepantel is a well-known veterinary drug, registered as a livestock wormicide in 39 countries. The industry collaborator, PharmAust Ltd, has found that monepantel shows off-target activity, inhibiting a cellular signaling system controlled by mammalian target of rapamycin (mTOR). This stops cancer growth and reduces protein accumulation in diseased cells. PharmAust has already tested monepantel in humans and pet dogs in Phase I and II anti-cancer clinical trials, respectively, in Australia. Data from these trials show that monepantel treatment associates with an exceptionally high safety profile, mTOR signaling inhibition and anticancer activity.\n\nAbnormal protein accumulation within motor neurons of the brain associates with the cause of ALS/MND. Inhibition of the mTOR signaling pathway slows disease progression in certain preclinical models of ALS/MND and is suggested to provide synergy with the ALS/MND standard-of-care drug, riluzole. An alternative mTOR inhibitor, rapamycin, is currently the subject of an ALS/MND clinical trial in humans investigating control of disease progression. Monepantel has a different structure to rapamycin and an apparently better safety profile.\n\nThis Phase I Clinical Trial hypothesis is that monepantel administration to individuals living with ALS/MND will safely reduce disease associated protein accumulation in motor neurons and provide therapeutic benefit. To test this hypothesis, the safety and tolerability of oral monepantel administration and markers of efficacy will be tested in individuals living with ALS/MND in a dose escalating Phase I/II Clinical Trial. To mitigate risk, only patients with sporadic and certain known familial types of ALS will be eligible. To further mitigate risk, the monepantel starting dose will be reduced a calculated five-fold compared to that already used in human cancer patients and already demonstrated to be safe and effective as an mTOR inhibitor. Dependent upon incremental outcomes, three higher doses may then be tested, each for minimally 28 days with a duration at the optimal dose of at least six months.", "interventions": [{"type": "DRUG", "name": "Monepantel"}], "start_date": "2022-06-28", "url": "https://clinicaltrials.gov/study/NCT04894240", "target_entities": ["Myostatin"], "locations": [{"facility": "Macquarie University", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.86785, "lon": 151.20732}, {"facility": "Calvary Health Care Bethlehem", "city": "Melbourne", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.814, "lon": 144.96332}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Monepantel", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01123200", "title": "An In-home Study of Brain Computer Interfaces", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "The investigators are developing a tool to help people who are severely paralyzed. This tool is called a brain-computer interface (BCI). BCIs can connect to computers or other electronic devices.\n\nThis study allows a person with ALS to communicate, control their wheelchair tilt and perform other tasks using a BCI, thus increasing their independence.", "interventions": [{"type": "DEVICE", "name": "Brain Computer Interface for Wheelchair Tilt Control"}], "start_date": "2010-01", "url": "https://clinicaltrials.gov/study/NCT01123200", "target_entities": [], "locations": [{"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05753852", "title": "Open Label Extension of TUDCA-ALS Study", "phase": "PHASE3", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Humanitas Mirasole SpA", "summary": "This study will provide extended access to patients and assess longer-term outcomes on patients who have completed the TUDCA-ALS study.", "interventions": [{"type": "DRUG", "name": "Tauroursodeoxycholic Acid"}], "start_date": "2021-10-25", "url": "https://clinicaltrials.gov/study/NCT05753852", "target_entities": ["apoptosis"], "locations": [{"facility": "Katholieke Universiteit Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "ACTIVE_NOT_RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "Centre Hospitalier Universitaire de Bordeaux", "city": "Bordeaux", "state": "", "country": "France", "status": "RECRUITING", "lat": 44.84124, "lon": -0.58046}, {"facility": "Centre Hospitalier Universitaire Limoges", "city": "Limoges", "state": "", "country": "France", "status": "RECRUITING", "lat": 45.83362, "lon": 1.24759}, {"facility": "Centre Hospitalier Universitaire de Montpellier", "city": "Montpellier", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.61093, "lon": 3.87635}, {"facility": "Centre Hospitalier Regional Universitaire de Tours", "city": "Tours", "state": "", "country": "France", "status": "RECRUITING", "lat": 47.39484, "lon": 0.70398}, {"facility": "Charit\u00e9 - Universit\u00e4tsmedizin Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 52.52437, "lon": 13.41053}, {"facility": "Universit\u00e4tsklinikum Carl Gustav Carus Dresden", "city": "Dresden", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 51.05089, "lon": 13.73832}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universit\u00e4tsklinikum Jena", "city": "Jena", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 50.92878, "lon": 11.5899}, {"facility": "Universit\u00e4t Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "ACTIVE_NOT_RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "Trinity College Dublin", "city": "Dublin", "state": "", "country": "Ireland", "status": "ACTIVE_NOT_RECRUITING", "lat": 53.33306, "lon": -6.24889}, {"facility": "IRCCS Istituto Auxologico Italiano", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "NEuroMuscular Omnicentre. Fondazione Serena Onlus", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.78235, "lon": 12.59836}, {"facility": "AOU Universit\u00e0 degli Studi della Campania \"Luigi Vanvitelli\"", "city": "Naples", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 40.85216, "lon": 14.26811}, {"facility": "IRCCS Istituto Clinico Humanitas", "city": "Rozzano", "state": "", "country": "Italy", "status": "ACTIVE_NOT_RECRUITING", "lat": 45.38193, "lon": 9.1559}, {"facility": "Azienda Ospedaliera Santa Maria di Terni", "city": "Terni", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 42.56335, "lon": 12.64329}, {"facility": "AOU Citt\u00e0 della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "ACTIVE_NOT_RECRUITING", "lat": 44.88856, "lon": 11.99138}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "ACTIVE_NOT_RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "The Walton Centre NHS Foundation Trust", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "ACTIVE_NOT_RECRUITING", "lat": 53.41058, "lon": -2.97794}, {"facility": "Plymouth Hospitals NHS Trust", "city": "Plymouth", "state": "", "country": "United Kingdom", "status": "WITHDRAWN", "lat": 50.37153, "lon": -4.14305}, {"facility": "Salford Royal NHS Foundation Trust", "city": "Salford", "state": "", "country": "United Kingdom", "status": "WITHDRAWN", "lat": 53.48771, "lon": -2.29042}, {"facility": "University of Sheffield", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 53.38297, "lon": -1.4659}, {"facility": "Royal Stoke University Hospital", "city": "Stoke", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 53.25, "lon": -2.86667}], "contact_phone": "+39-0282246418", "contact_email": "alberto.albanese@humanitas.it", "mechanism_summary": {"compound": "Tauroursodeoxycholic Acid", "targeting_mechanism": "Tauroursodeoxycholic acid exerts anti-apoptotic and neuroprotective activities by inhibiting apoptosis in neurodegenerative diseases.", "targeting_mechanism_pmid": "35659112", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04505358", "title": "Evaluate PU-AD in Subjects With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Samus Therapeutics, Inc.", "summary": "This is a multicenter, Phase 2a, randomized, double-blind, placebo-controlled pilot study to assess the biological activity, safety and pharmacokinetics of PU-AD compared to placebo in ALS. It will be conducted in approximately 20 sites in the US. Approximately 30 subjects will be enrolled in this study; subjects will be randomized 3:2 to receive either PU-AD 30 mg or matching placebo qd, added onto any current stable background treatment.", "interventions": [{"type": "DRUG", "name": "PU-AD"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2023-01", "url": "https://clinicaltrials.gov/study/NCT04505358", "target_entities": ["neuroinflammation"], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "PU-AD", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03694132", "title": "Brain Excitability and Connectivity in Sensory-motor Pathways in ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Institut National de la Sant\u00e9 Et de la Recherche M\u00e9dicale, France", "summary": "The main objective is to determine the origin of somatosensory alteration in patients with ALS and to evaluate its impact on brain activity by coupling different imaging modalities and indirect electrophysiology.\n\nThe secondary objective is to evaluate whether the observed functional changes in MEG / EEG and functional MRI correlate with structural lesions revealed with diffusion MRI (anatomo-functional connectivity of the brain).", "interventions": [{"type": "DEVICE", "name": "functional MRI"}, {"type": "DEVICE", "name": "structural MRI"}, {"type": "DEVICE", "name": "EEG/MEG"}], "start_date": "2018-11-26", "url": "https://clinicaltrials.gov/study/NCT03694132", "target_entities": [], "locations": [{"facility": "Hopital Pitie-Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03487263", "title": "Dose-Escalation, Safety and Pharmacokinetic Study of IC14 in Motor Neurone Disease", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Implicit Bioscience", "summary": "Ten patients with motor neurone disease (MND, also known as amyotrophic lateral sclerosis or ALS) will be successively enrolled to one of two dose levels of IC14 (human chimeric monoclonal anti-CD14) intravenously for four doses. Patients must be within 3 years of MND diagnosis and have adequate respiratory function. Safety, tolerability, immunogenicity, and PK/PD will be measured. To evaluate feasibility of the endpoints, additional endpoints of ALSFRS-R, respiratory function tests, disease biomarkers and patient-reported outcomes will be measured.", "interventions": [{"type": "BIOLOGICAL", "name": "IC14"}], "start_date": "2017-10-01", "url": "https://clinicaltrials.gov/study/NCT03487263", "target_entities": ["CD14"], "locations": [{"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "IC14", "targeting_mechanism": "IC14 is a human chimeric monoclonal antibody targeting CD14.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06782958", "title": "Safety, Tolerability, and Pharmacokinetics of FHND1002 Granules in Healthy Adults", "phase": "PHASE1", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.", "summary": "The goal of this clinical trial is to evaluate the safety, tolerability, and pharmacokinetic (PK) profile of FHND1002 granules in healthy adult volunteers. The study will also assess how a high-fat meal affects the PK characteristics of FHND1002.\n\nThe main questions this study aims to answer are:\n\nWhat are the safety and tolerability of FHND1002 granules when administered as single or multiple doses? What are the PK parameters of FHND1002, and what metabolites can be identified in humans?\n\nParticipants will:\n\nTake FHND1002 granules or a placebo once daily, either as a single dose or for 7 consecutive days.\n\nAttend regular clinic visits for checkups, tests, and blood sample collection. Undergo assessments, including monitoring for adverse events, physical exams, vital signs, ECGs, and laboratory tests.", "interventions": [{"type": "DRUG", "name": "50mg FHND1002"}, {"type": "DRUG", "name": "100mg FHND1002"}, {"type": "DRUG", "name": "150 mg FHND1002"}, {"type": "DRUG", "name": "200 mg FHND1002\uff08fasting\uff09"}, {"type": "DRUG", "name": "250mg FHND1002"}, {"type": "DRUG", "name": "200mg FHND1002(postprandial)"}], "start_date": "2024-07-05", "url": "https://clinicaltrials.gov/study/NCT06782958", "target_entities": [], "locations": [{"facility": "The first hospital of Lanzhou University", "city": "Lanzhou", "state": "Gansu", "country": "China", "status": "", "lat": 36.05701, "lon": 103.83987}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "FHND1002", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07174492", "title": "Efficacy and Safety of Masitinib in Combination With SoC Versus Placebo in the Treatment of ALS Patients", "phase": "PHASE3", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "AB Science", "summary": "The objective is to compare the efficacy and safety of masitinib in combination with riluzole versus matched placebo in combination with riluzole for the treatment of Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "Masitinib 4.5 mg/kg/day"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Riluzole (100 mg)"}], "start_date": "2026-01-01", "url": "https://clinicaltrials.gov/study/NCT07174492", "target_entities": ["C-KIT", "PDGFRA"], "locations": [{"facility": "Aiginition Hospital", "city": "Athens", "state": "", "country": "Greece", "status": "", "lat": 37.98376, "lon": 23.72784}], "contact_phone": "+33(0)147200014", "contact_email": "clinical@ab-science.com", "mechanism_summary": {"compound": "Masitinib", "targeting_mechanism": "Tyrosine kinase inhibitor that abrogates neuroinflammation and microgliosis", "targeting_mechanism_pmid": "27400786", "animal_results": "Masitinib significantly prolonged survival in SOD1G93A rats when delivered after paralysis onset, controlling microgliosis, neuroinflammation, and the emergence/expansion of aberrant glial cells", "animal_results_pmid": "27400786", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04875949", "title": "Anti-Cholinergic Receptors Antibodies, Autonomic Profile and Dysautonomia Symptoms in PAF, ALS and POTS (DISAUT-AB)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Istituto Clinico Humanitas", "summary": "Anti alfa-3 and alfa-7 ganglionic cholinergic receptors (anti-AChRs) antibodies (Abs) plasma removal by plasmapheresis (1,2) acutely improved dysautonomia symptoms in case reports with Pure Autonomic Failure (PAF) (3). We shall assess the prevalence of anti-AChRs Ab and the relationship among Ab titer, cardiovascular autonomic profile and symptoms in neurodegenerative diseases characterized by similar dysautonomia symptoms such as PAF, Amyotrophic Lateral Sclerosis (ALS) and Postural Orthostatic Tachycardia Syndrome (POTS) (4). Ab positive patients will undergo selective immunoabsorption once a week up to achievement of Ab titer lower than 65% of baseline followed by immunosuppressive therapy with prednisone. Both Ab positive and negative groups will undergo anti-AChR Abs, autonomic profile and dysautonomia symptoms assessment, every 4 months up to 3 years. Evidence of correlation among reduced Ab titer and autonomic profile and symptoms improvement may result in new effective therapy.", "interventions": [{"type": "PROCEDURE", "name": "Immunoabsorption/plasmapheresis procedure"}], "start_date": "2016-04", "url": "https://clinicaltrials.gov/study/NCT04875949", "target_entities": ["cholinergic_receptors"], "locations": [{"facility": "Humanitas Research Hospital", "city": "Rozzano", "state": "", "country": "Italy", "status": "", "lat": 45.38193, "lon": 9.1559}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05981040", "title": "Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ZYIL1 in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Zydus Lifesciences Limited", "summary": "ZYIL1 is expected to show benefit in patients with Amyotrophic Lateral Sclerosis (ALS). The present study aims to determine the safety, tolerability, pharmacokinetics, and pharmacodynamics of ZYIL1 when administered to subjects with ALS.", "interventions": [{"type": "DRUG", "name": "ZYIL1 capsules 25 mg and 50 mg Placebo"}, {"type": "DRUG", "name": "ZYIL1 capsules 50 mg and 25 mg Placebo"}, {"type": "DRUG", "name": "ZYIL1 capsules 25 mg and ZYIL1 capsules 50 mg"}, {"type": "DRUG", "name": "Matching placebo 25 mg and Matching placebo 50 mg"}], "start_date": "2023-11-09", "url": "https://clinicaltrials.gov/study/NCT05981040", "target_entities": [], "locations": [{"facility": "Zydus Hospitals & Healthcare Research", "city": "Ahmedabad", "state": "Gujarat", "country": "India", "status": "", "lat": 23.02579, "lon": 72.58727}, {"facility": "Rhythm heart institute", "city": "Vadodara", "state": "Gujarat", "country": "India", "status": "", "lat": 22.29941, "lon": 73.20812}, {"facility": "BrainS Super Speciality Hospital", "city": "Bengaluru", "state": "Karnataka", "country": "India", "status": "", "lat": 12.97194, "lon": 77.59369}, {"facility": "KIMS-Kingsway Hospitals", "city": "Nagpur", "state": "Maharashtra", "country": "India", "status": "", "lat": 21.14631, "lon": 79.08491}, {"facility": "Surya Multispecialty Hospital", "city": "Nashik", "state": "Maharashtra", "country": "India", "status": "", "lat": 19.99727, "lon": 73.79096}, {"facility": "CIMET's Inamdar Multispeciality Hospital", "city": "Pune", "state": "Maharashtra", "country": "India", "status": "", "lat": 18.51957, "lon": 73.85535}, {"facility": "Sir Ganga Ram Hospital", "city": "New Delhi", "state": "", "country": "India", "status": "", "lat": 28.62137, "lon": 77.2148}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "ZYIL1", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07295990", "title": "Tongue-strengthening Exercises in People With ALS.", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Nova Southeastern University", "summary": "This study is testing a tongue exercise program for people living with ALS to see if it can help support speech and swallowing. All participants will receive the treatment, and researchers will measure changes over time by comparing each person's results to their own earlier results.\n\nPeople who join the study will have two in-person visits and four weekly telehealth sessions with a speech-language pathologist. During these sessions, participants will practice tongue resistance exercises, complete speech and swallowing tasks, and answer surveys about their experience. They will also use a small device at home to measure tongue strength and swallowing.\n\nThe exercise program involves pressing the tongue against a device several times a day, five days per week, for five weeks. Researchers want to learn if this program is safe, practical, and helpful for people with ALS.", "interventions": [{"type": "PROCEDURE", "name": "Isometric Lingual Strength Exercises"}], "start_date": "2026-05-01", "url": "https://clinicaltrials.gov/study/NCT07295990", "target_entities": [], "locations": [{"facility": "Nova Southeastern University", "city": "Davie", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.06287, "lon": -80.2331}], "contact_phone": "17863994053", "contact_email": "rw602@nova.edu", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06441682", "title": "A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "argenx", "summary": "This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function. The study consists of 2 periods: a treatment period when participants will receive one of three ARGX-119 doses or placebo and an extension period when all participants will receive the same dose of ARGX-119. Participation in the study will last up to approximately 100 weeks.\n\nThe study was terminated early following review of interim data indicating that continuation was unlikely to demonstrate a clinically meaningful treatment effect. The decision was made to minimize unnecessary participant burden. This decision is not related to safety concerns.\n\nEnd-of-study and Safety-Follow-Up visits are ongoing for the participants of this trial", "interventions": [{"type": "BIOLOGICAL", "name": "ARGX-119"}, {"type": "OTHER", "name": "Placebo"}], "start_date": "2024-10-23", "url": "https://clinicaltrials.gov/study/NCT06441682", "target_entities": ["neuroinflammation"], "locations": [{"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Kaye Edmonton Clinic", "city": "Edmonton", "state": "", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Aarhus Universitets Hospital", "city": "Aarhus", "state": "", "country": "Denmark", "status": "", "lat": 56.15674, "lon": 10.21076}, {"facility": "Bispebjerg University Hospital", "city": "Copenhagen", "state": "", "country": "Denmark", "status": "", "lat": 55.67594, "lon": 12.56553}, {"facility": "H\u00f4pital La Piti\u00e9 Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "CHU Bretonneau", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Akademiskt specialistcentrum Karolinska Institutet", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "ARGX-119", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05633459", "title": "A Study Evaluating the Safety and Tolerability of QRL-201 in ALS", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "QurAlis Corporation", "summary": "The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS", "interventions": [{"type": "DRUG", "name": "Multiple ascending doses of QRL-201"}, {"type": "DRUG", "name": "Multiple ascending doses of Placebo"}, {"type": "DRUG", "name": "QRL-201"}, {"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "QRL-201"}], "start_date": "2022-12-16", "url": "https://clinicaltrials.gov/study/NCT05633459", "target_entities": ["qrl_201"], "locations": [{"facility": "Universitaire Ziekenhuizen Leuven (UZ Leuven)", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "University of Calgary", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Sunnybrook Health Science Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "CHUM - Hopital Notre-Dame", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute-Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Deutsches Zentrum f\u00fcr Neurodegenerative Erkrankungen e. V. (DZNE)", "city": "Bonn", "state": "North Rhine-Westphalia", "country": "Germany", "status": "", "lat": 50.73438, "lon": 7.09549}, {"facility": "Charit\u00e9 Research Organisation", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "University Hospital Schleswig-Holstein (UKSH) Campus L\u00fcbeck, Department for Neurology/ Precision Neurology", "city": "L\u00fcbeck", "state": "", "country": "Germany", "status": "", "lat": 53.86893, "lon": 10.68729}, {"facility": "Universit\u00e4tsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "St James's Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "The University of Sheffield, Royal Hallamshire Hospital", "city": "Sheffield", "state": "United Kingdom", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}, {"facility": "Kings College Hospital NHS Foundation Trust", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "National Hospital for Neurology and Neurosurgery", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "QRL-201", "targeting_mechanism": "Transplantation of human neural progenitor cells secreting GDNF to provide neuroprotection to motor neurons.", "targeting_mechanism_pmid": "36064599", "animal_results": "In SOD1 rat models, injections of CNS10-NPC-GDNF at doses of 10,000 to 250,000 cells were well-tolerated without clogging the delivery cannula, providing the basis for dose escalation in human trials.", "animal_results_pmid": "36064599", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02478450", "title": "Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells\u00ae) Into Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Q Therapeutics, Inc.", "summary": "This study is a non-randomized, open-label, partially blinded, sequential cohort, dose-escalation study designed to obtain preliminary data on the safety, tolerability, and early efficacy of Q-Cells\u00ae transplantation in subjects with ALS. Following an initial cohort receiving cell transplants unilaterally in the lumbar spinal cord, subsequent cohorts will receive escalating doses transplanted unilaterally in cervical spinal cord. Subjects and outcome measure assessors will be blinded to side of treatment. The study will be conducted at sites with extensive clinical experience with the care of patients with ALS.", "interventions": [{"type": "BIOLOGICAL", "name": "Q-Cells"}], "start_date": "2026-12", "url": "https://clinicaltrials.gov/study/NCT02478450", "target_entities": ["glial_restricted_progenitor_cells"], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Q-Cells (human glial restricted progenitor cells, hGRPs)", "targeting_mechanism": "Transplantation of human glial restricted progenitor cells into the spinal cord to support motor neuron survival and function.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04391361", "title": "The Safety and Effectiveness of Cholinergic Receptor Block Therapy in the Treatment of ALS", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ruijin Hospital", "summary": "Thirty cases of amyotrophic lateral sclerosis patients were recruited from the neurology department of Ruijin Hospital, the pain department and the encephalopathy center of Luwan Branch of Ruijin Hospital. After the informed consent was signed, they were divided into a trial group and a control group. Each group contains 15 cases. The patients in the control group was treated with edaravone dissolved in saline during hospitalization, while the patients in the trial group was treated with edaravone, scopolamine, atropine and dexmedetomidine. Both groups of subjects were treated for 7 days within 3 weeks, followed by a buffer period of 3 weeks for observation, which was one treatment course. The total treatment protocol contains 3 treatment courses (or 18 weeks). Patients with amyotrophic lateral sclerosis were evaluated before treatment and 6, 12, 18, 24, 36, 48 weeks after treatment. The observations include whether the functional scores of patients with amyotrophic lateral sclerosis, Norris amyotrophic lateral sclerosis score, amyotrophic lateral sclerosis self-score, forced expiratory volume in one second, partial pressure of oxygen and maximum displacement of the hyoid were superior to those before treatment, and whether the partial pressure of carbon dioxide was inferior to those before treatment. Study hypothesis: Cholinergic receptor blocking therapy for amyotrophic lateral sclerosis is safe and effective in improving motor function and delaying disease progression in patients with amyotrophic lateral sclerosis.", "interventions": [{"type": "DRUG", "name": "Scopolamine, atropine, edaravone and dexmedetomidine"}, {"type": "DRUG", "name": "Edaravone"}], "start_date": "2020-11-01", "url": "https://clinicaltrials.gov/study/NCT04391361", "target_entities": ["cholinergic_signaling", "oxidative_stress"], "locations": [{"facility": "Department of neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine", "city": "Shanghai", "state": "Shanghai Municipality", "country": "China", "status": "", "lat": 31.22222, "lon": 121.45806}], "contact_phone": "64370045", "contact_email": "jly0520@hotmail.com", "mechanism_summary": {"compound": "scopolamine and atropine (cholinergic receptor blockers) plus edaravone and dexmedetomidine", "targeting_mechanism": "Edaravone is a free radical scavenger that quenches hydroxyl radicals and inhibits lipid peroxidation to reduce oxidative stress in ALS.", "targeting_mechanism_pmid": "38474192", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "edaravone was initially developed for acute cerebral infarction", "repurposed_from_pmid": "38474192"}}
{"nct_id": "NCT00790582", "title": "A Multi-Center Controlled Screening Trial of Safety and Efficacy of Lithium Carbonate in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Forbes Norris MDA/ALS Research Center", "summary": "This is a Phase II screening study of lithium carbonate in ALS. The purpose of this study is to find out if lithium carbonate is safe to be used in people with ALS and if it can slow the progression of the disease. Since there is no placebo in this study, all patients will be taking lithium carbonate.", "interventions": [{"type": "DRUG", "name": "lithium carbonate"}], "start_date": "2008-05", "url": "https://clinicaltrials.gov/study/NCT00790582", "target_entities": ["lithium_carbonate"], "locations": [{"facility": "Mayo Clinic", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "UCLA Neuromuscular Research Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "UC Irvine MDA/ALS & Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Kansas University Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Washington University Department of Neurology", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Providence ALS Clinic", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Pennsylvania Neurological Institute", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Utah Clinical Neurosciences Center", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "lithium carbonate", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07491562", "title": "Evaluation of a Structurally Suitable Neck Exoskeleton in Patients With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Utah", "summary": "The investigators will evaluate a new powered neck exoskeleton in patients with amyotrophic lateral sclerosis (ALS) to understand whether this device allows adequate head range of motion and achieves satisfaction from users. In this small cross-sectional device feasibility study, participants will be enrolled from the ALS clinic at the University of Utah Hospital. After obtaining written consent, participants will perform tasks using the neck exoskeleton. Tasks include computerized tracking tasks and simulated activities of daily living. Breaks will be added between tasks to avoid fatigue. Head-neck kinematics will be recorded, and range of motion will be computed. Participants will also report their satisfaction of the device.", "interventions": [{"type": "DEVICE", "name": "powered neck exoskeleton"}], "start_date": "2024-09-06", "url": "https://clinicaltrials.gov/study/NCT07491562", "target_entities": [], "locations": [{"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "RECRUITING", "lat": 40.76078, "lon": -111.89105}], "contact_phone": "801-585-2536", "contact_email": "haohan.zhang@utah.edu", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00956488", "title": "Supported Treadmill Ambulation Training (STAT) for Patients Diagnosed With Amyotrophic Lateral Aclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Wake Forest University Health Sciences", "summary": "The purpose of this study is to find out if supervised exercise training using a treadmill with partial weight support is safe and has an impact on gait and function of persons with Amyotrophic lateral sclerosis.", "interventions": [{"type": "BEHAVIORAL", "name": "Treadmill Exercise"}], "start_date": "2008-09", "url": "https://clinicaltrials.gov/study/NCT00956488", "target_entities": [], "locations": [{"facility": "Carolinas ALS Clinical Resarch Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03113630", "title": "Virtual Task in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Sao Paulo", "summary": "Thirty individuals with ALS (18 men and 12 women, mean age 59 years, range 44-74 years), and 30 healthy controls matched for age and gender, participated. Individuals with ALS and from the control group were randomly divided into three groups, each using a different communication device systems (Kinect\u00ae, Leap Motion Controller\u00ae or touchscreen) to perform two task phases (acquisition and retention). Performance was then explored in a third phase (transfer) by switching devices (two transfers); so that, all groups had contact with all communication interfaces.", "interventions": [{"type": "DEVICE", "name": "Acquisition on TouchScreen"}, {"type": "DEVICE", "name": "Acquisition on Kinect"}, {"type": "DEVICE", "name": "Acquisition on LeapMotion"}, {"type": "DEVICE", "name": "Acquisition on TouchScreen Control Group"}, {"type": "DEVICE", "name": "Acquisition on Kinect Control Group"}, {"type": "DEVICE", "name": "Acquisition on LeapMotion Control Group"}], "start_date": "2016-02-02", "url": "https://clinicaltrials.gov/study/NCT03113630", "target_entities": [], "locations": [{"facility": "Escola de Artes,Ciencias e Humanidades da Universidade d Sao Paulo", "city": "S\u00e3o Paulo", "state": "S\u00e3o Paulo", "country": "Brazil", "status": "", "lat": -23.5475, "lon": -46.63611}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03085706", "title": "Transplantation of Autologous Peripheral Blood Mononuclear Cells for Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The First Affiliated Hospital of Dalian Medical University", "summary": "To assess the safety of peripheral blood mononuclear cell transplantation into the subarachnoid space for the treatment of amyotrophic lateral sclerosis.", "interventions": [{"type": "BIOLOGICAL", "name": "PBMC autotransplantation"}], "start_date": "2010-10", "url": "https://clinicaltrials.gov/study/NCT03085706", "target_entities": ["neuroinflammation"], "locations": [{"facility": "The First Affiliated Hospital of Dalian Medical University", "city": "Dalian", "state": "Liaoning", "country": "China", "status": "", "lat": 38.91222, "lon": 121.60222}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Peripheral blood mononuclear cells (PBMC)", "targeting_mechanism": "Modulation of neuroinflammation through immune cell transplantation into the subarachnoid space.", "targeting_mechanism_pmid": "37433768", "animal_results": "Preclinical stem cell studies in mouse and rat ALS models expressing mutant superoxide dismutase 1 demonstrated potential benefit of cell-based therapy for ALS.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06249867", "title": "A Study to Assess the Safety, Tolerability, and Pharmacology of Darifenacin in Patients With ALS", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Oliver Blanchard", "summary": "Amyotrophic lateral sclerosis (ALS) is a progressive neurological disorder characterized by selective death of upper and lower motor neurons, which leads to severe disability and fatal outcomes. One of the major hallmarks of ALS is the denervation of neuromuscular junctions (NMJs), which is one of the earliest events seen in ALS patients and mouse models of ALS. Under healthy conditions, glial cells called Perisynaptic Schwann Cells (PSCs) have a key role in regulating the stability and maintenance of NMJs, but they only participate in NMJ repair once denervation occurs. Denervation and the subsequent decline in synaptic activity triggers a loss of muscarinic acetylcholine receptors (mAChRs) in the PSC, and the resulting decrease in mAChR-mediated gene expression drives the \"repair mode\" of the PSC. In assessing the NMJ under conditions of ALS, a scarcity of process extensions in PSCs was observed for months prior to disease onset in the superoxide dismutase 1 (SOD1) mouse model of ALS, indicating inadequate glial repair. Collectively, these preclinical findings support the hypothesis that dampening glial mAChRs will restore the anticipated \"repair\" response of PSCs in the NMJ. Hence, the use of a selective M3 muscarinic receptor antagonist, Darifenacin, as a disease-modifying therapeutic in familial and sporadic ALS could improve NMJ function, resulting in a beneficial impact on the autonomy and quality of life of ALS patients.\n\nThe purpose of the current Phase 2 trial is therefore to test the safety, tolerability, and pharmacology of Darifenacin in patients with ALS. Specifically, 30 eligible subjects between 18 and 85 years of age will take 7.5 mg of darifenacin or placebo daily (by mouth) for two weeks followed by an increased dose of 15 mg for the next 22 weeks. The trial will evaluate the effects of this medication on several outcome measures including patient safety, physical and neurological function, muscle strength, depression levels, and NMJ innervation of patients with ALS. Detailed clinical assessments will be conducted at regular intervals throughout the study in order to achieve these objectives.", "interventions": [{"type": "DRUG", "name": "Darifenacin 7.5 MG Extended Release Oral Tablet"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2024-11-08", "url": "https://clinicaltrials.gov/study/NCT06249867", "target_entities": ["Muscarinic acetylcholine receptor"], "locations": [{"facility": "Ottawa Hospital Research Institute", "city": "Ottawa", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 45.41117, "lon": -75.69812}, {"facility": "Montreal Neurological Institute", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "514-396-2401", "contact_email": "nisha.pulimood@mcgill.ca", "mechanism_summary": {"compound": "Darifenacin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "Adeno-associated virus-delivered artificial microRNA extended survival and delayed paralysis in SOD1G93A mouse model of ALS.", "animal_results_pmid": "26891182", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03836716", "title": "Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "ZevraDenmark", "summary": "A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the ORARIALS-01 trial.", "interventions": [{"type": "DRUG", "name": "Arimoclomol"}], "start_date": "2019-09-19", "url": "https://clinicaltrials.gov/study/NCT03836716", "target_entities": ["heat_shock_response"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "UC Irvine Health ALS and Neuromuscular Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "University of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Providence Brain & Spine Institute", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "Catholic University Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universitaetsklinikum Ulm - Klinik fuer Neurologie", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Instituti Clinica Scientifici Maugeri", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Centrum Medyczne NeuroProtect", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Citi Clinic", "city": "Warsaw", "state": "", "country": "Poland", "status": "", "lat": 52.22977, "lon": 21.01178}, {"facility": "Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Carlos III - Hospital Universitario La Paz, ALS Unit", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Ume\u00e5 University Hospital", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "Leonard Wolfson Experimental Neurology Centre", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Arimoclomol", "targeting_mechanism": "Heat shock response modulation to enhance TDP-43 clearance.", "targeting_mechanism_pmid": "26936937", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04931862", "title": "Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)", "phase": "PHASE1, PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Wave Life Sciences USA, Inc.", "summary": "This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients with C9orf72-associated ALS or FTD. To participate in the study, patients must have a documented mutation (GGGGCC \\[G4C2\\] repeat expansion) in the first intronic region of the C9orf72 gene and be diagnosed with ALS or FTD.", "interventions": [{"type": "DRUG", "name": "WVE-004"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-06-28", "url": "https://clinicaltrials.gov/study/NCT04931862", "target_entities": ["C9orf72"], "locations": [{"facility": "Macquarie University", "city": "North Ryde", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.79677, "lon": 151.12436}, {"facility": "The Wesley Hospital", "city": "Brisbane", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.46794, "lon": 153.02809}, {"facility": "Perron Institute", "city": "Nedlands", "state": "Western Australia", "country": "Australia", "status": "", "lat": -31.98184, "lon": 115.8073}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "McGill University Health Center - Research Institute", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "St James Hospital - Ireland", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Erasmus University MC", "city": "Rotterdam", "state": "", "country": "Netherlands", "status": "", "lat": 51.9225, "lon": 4.47917}, {"facility": "Universitair Medisch Centrum Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Auckland City Hospital", "city": "Auckland", "state": "", "country": "New Zealand", "status": "", "lat": -36.84853, "lon": 174.76349}, {"facility": "New Zealand Brain Research Institute", "city": "Christchurch", "state": "", "country": "New Zealand", "status": "", "lat": -43.53333, "lon": 172.63333}, {"facility": "Karolinska University Hospital", "city": "Solna", "state": "", "country": "Sweden", "status": "", "lat": 59.36004, "lon": 18.00086}, {"facility": "University of Cambridge", "city": "Cambridge", "state": "", "country": "United Kingdom", "status": "", "lat": 52.2, "lon": 0.11667}, {"facility": "University College London Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "King's College Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "University of Oxford - Nuffield Department of Clinical Neurosciences", "city": "Oxford", "state": "", "country": "United Kingdom", "status": "", "lat": 51.75222, "lon": -1.25596}, {"facility": "University of Sheffield", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "WVE-004", "targeting_mechanism": "Antisense oligonucleotide targeting C9orf72 GGGGCC repeat expansion to reduce pathogenic dipeptide repeat protein production.", "targeting_mechanism_pmid": "27768896", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04055623", "title": "T-regulatory Cells in ALS", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "The Methodist Hospital Research Institute", "summary": "This study is a randomized, placebo-controlled, phase 2a trial to study the biological activity, safety, and tolerability of regulatory T Lymphocytes (Tregs) taken and expanded outside of the body and returned back to the same person whose Treg were removed, given back by IV (intravenously) and in combination with low-dose IL-2 in people with Amyotrophic Lateral Sclerosis (ALS).", "interventions": [{"type": "BIOLOGICAL", "name": "Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections"}, {"type": "OTHER", "name": "Monthly placebo infusions + 3 times per week placebo injections"}, {"type": "BIOLOGICAL", "name": "Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections"}], "start_date": "2019-08-07", "url": "https://clinicaltrials.gov/study/NCT04055623", "target_entities": ["neuroinflammation", "IL2"], "locations": [{"facility": "Massachusetts General Hospital Neurological Clinical Research Institute", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Houston Methodist Hospital", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "autologous regulatory T cells (Tregs) with low-dose interleukin-2", "targeting_mechanism": "Expansion and reinfusion of regulatory T cells combined with low-dose IL-2 to enhance Treg-mediated suppression of neuroinflammation in ALS.", "targeting_mechanism_pmid": "32651161", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05974579", "title": "Safety and Dosimetry of a New Radiotracer to Detect Misfolded SOD1 Associated With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Universit\u00e9 de Sherbrooke", "summary": "Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a rare neurodegenerative disease resulting in loss, primarily, of the motor neurons in the motor cortex, brainstem and spinal cord. It currently affects 3 of every 100,000 people in the US.\n\nCurrently, there is no diagnostic tool for ALS, resulting in misdiagnosis and significant disease progression before formal diagnosis. An imaging test for early detection of ALS and for monitoring disease progression would have significant diagnostic and prognostic value.\n\nPET imaging with an appropriate radiotracer has great potential as a biomarker for ALS given that it would permit visualization of central nervous system (CNS) pathology in individuals living with the disease.\n\nTo that extent, the primary goal of this phase I study is evaluating the safety and biodistribution of the new tracer \\[89Zr\\]Zr-DFO-AP-101 in healthy volunteers and ALS patients.", "interventions": [{"type": "DRUG", "name": "89Zr-DFO-AP-101"}], "start_date": "2023-11-23", "url": "https://clinicaltrials.gov/study/NCT05974579", "target_entities": ["SOD1"], "locations": [{"facility": "CIUSSS de l'Estrie-CHUS Hospital", "city": "Sherbrooke", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.40008, "lon": -71.89908}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "89Zr-DFO-AP-101", "targeting_mechanism": "Radiotracer designed to detect and image misfolded SOD1 protein associated with ALS for diagnostic and disease monitoring purposes.", "targeting_mechanism_pmid": "29751510", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03272503", "title": "A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Calgary", "summary": "This study will look at whether Pimozide may help to slow the progression of Amyotrophic Lateral Sclerosis.\n\n100 people from several Canadian centres with ALS who have provided their consent will be randomly assigned into one of 2 groups. The first group will receive a dose of up to 2mg of Pimozide per day and the second group will receive placebo (lactose tablets). Subjects will be assigned randomly (like by a flip of a coin) to receive either Pimozide 2 mg per day or placebo tablets. There will be a fifty-fifty chance of receiving Pimozide or placebo.\n\nParticipants will be on study medication up to 22 weeks, and on study up to 26 weeks. There are 8 clinic visits and 1 phone visit over the course of the Treatment Phase of the study. The second phase which is Observational, is optional with follow-up for up to 5 years from the end of the Treatment Phase.", "interventions": [{"type": "DRUG", "name": "Pimozide 2mg/day (current) or 4 mg/day (study initiation)"}, {"type": "DRUG", "name": "Placebo Oral Tablet"}], "start_date": "2017-10-27", "url": "https://clinicaltrials.gov/study/NCT03272503", "target_entities": ["dopamine_receptor"], "locations": [{"facility": "Dr. Lawrence Korngut -South Health Campus", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "RECRUITING", "lat": 51.05011, "lon": -114.08529}, {"facility": "Dr. Wendy Johnston - University of Alberta", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "RECRUITING", "lat": 53.55014, "lon": -113.46871}, {"facility": "Dr. Colleen O'Connell - Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "RECRUITING", "lat": 45.94541, "lon": -66.66558}, {"facility": "Dr. John Turnbull McMaster University/Hamilton Health Services", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 43.25011, "lon": -79.84963}, {"facility": "Dr. Christen Shoesmith - London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 42.98339, "lon": -81.23304}, {"facility": "Dr. Ariel Breiner -Ottawa Hospital Research Institute", "city": "Ottawa", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 45.41117, "lon": -75.69812}, {"facility": "Dr. Lorne Zinman Sunnybrook Research Institute", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}, {"facility": "Dr. Sandrine Larue - Reserche Sepmus Inc.", "city": "Greenfield Park", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.48649, "lon": -73.46223}, {"facility": "Dr. Genevieve Matte", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}], "contact_phone": "403-210-7006", "contact_email": "Pimozide2@ucalgary.ca", "mechanism_summary": {"compound": "Pimozide", "targeting_mechanism": "Neuroleptic that stabilizes neuromuscular transmission by modulating calcium channel activity and preventing motor neuron dysfunction.", "targeting_mechanism_pmid": "29202456", "animal_results": "Pimozide stabilized neuromuscular transmission in multiple disease models including Caenorhabditis elegans and zebrafish.", "animal_results_pmid": "29202456", "repurposed_from": "antipsychotic medication", "repurposed_from_pmid": "29202456"}}
{"nct_id": "NCT01995903", "title": "Developing a Discrimination Model to Diagnose ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "To combine several brain imaging techniques to develop a new diagnostic test to help with earlier diagnosis of amyotrophic lateral sclerosis.", "interventions": [{"type": "DIAGNOSTIC_TEST", "name": "MRI(magnetic resonance imaging)"}], "start_date": "2012-04", "url": "https://clinicaltrials.gov/study/NCT01995903", "target_entities": [], "locations": [{"facility": "University of Michigan Hospital", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04719403", "title": "Determining Feasibility and Acceptability of Sharing Video Recordings With Patients With ALS and Caregivers", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Dartmouth-Hitchcock Medical Center", "summary": "Our objective in the proposed project is to: (a) operationalize and determine the feasibility and acceptability of a trial where clinic multi-disciplinary clinic (MDC) visits are audio/video recorded and shared with patients with ALS and their caregivers; (b) gather preliminary data examining the impact of routinely adding audio/video recordings of clinic visits to UC on self-management ability and other behavioral, health and health services outcomes at baseline (T0) and other regular interviews from enrollment (T1= 1 Week, T2= 3 Months); and (c) identify factors pertinent to the acceptability of our study protocol and the audio/video recording of visits.", "interventions": [{"type": "OTHER", "name": "HealthPAL"}], "start_date": "2021-05-12", "url": "https://clinicaltrials.gov/study/NCT04719403", "target_entities": [], "locations": [{"facility": "Dartmouth-Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04288856", "title": "Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB078 Administered to Previously Treated Adults C9ORF72-Associated Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Biogen", "summary": "The primary objective is to evaluate the long-term safety and tolerability of BIIB078 in participants with chromosome 9 open reading frame 72-amyotrophic lateral sclerosis (C9ORF72-ALS).\n\nThe secondary objective is to evaluate the pharmacokinectic (PK) of BIIB078 in participants with C9ORF72-ALS.", "interventions": [{"type": "DRUG", "name": "BIIB078"}], "start_date": "2020-04-28", "url": "https://clinicaltrials.gov/study/NCT04288856", "target_entities": ["C9orf72"], "locations": [{"facility": "Research Site", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Research Site", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "Research Site", "city": "Palo Alto", "state": "California", "country": "United States", "status": "", "lat": 37.44188, "lon": -122.14302}, {"facility": "Research Site", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Research Site", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Research Site", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Research Site", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Research Site", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Research Site", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Research Site", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Research Site", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Research Site", "city": "Knoxville", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.96064, "lon": -83.92074}, {"facility": "Research Site", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Research Site", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Research Site", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Research Site", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Research Site", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Research Site", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "Research Site", "city": "London", "state": "Greater London", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Research Site", "city": "London", "state": "Greater London", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Research Site", "city": "Sheffield", "state": "South Yorkshire", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "BIIB078", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07539662", "title": "Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "ENROLLING_BY_INVITATION", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Institute of Hematology & Blood Diseases Hospital, China", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive and fatal neurodegenerative disorder. Its global prevalence is approximately 0.73-1.89 per 100,000 individuals. In China, there are about 200,000 ALS patients, with approximately 25,000 new cases diagnosed annually. Microglia, the resident immune cells of the central nervous system (CNS), rapidly transition from a resting state to a pro-inflammatory phenotype (M1) in ALS. This activation leads to the release of a large number of inflammatory factors (such as TNF-\u03b1, IL-1\u03b2, IL-6, NO, ROS) and chemokines (such as MCP-1/CCL2), and triggers the NLRP3 inflammasome. Furthermore, systemic immune dysregulation plays a significant role in the pathogenesis of ALS. ALS patients exhibit reduced numbers of regulatory T cells (Tregs), alterations of activated CD8+ T cell infiltrates, and a shift in the helper T cell (Th1/Th2) balance towards the pro-inflammatory Th1 phenotype. In recent years, therapeutic strategies targeting novel pathways such as neuroinflammation, immune dysregulation, and energy metabolism have emerged, including the infusion of Tregs, mesenchymal stem cells (MSCs), and neural stem cells. However, these approaches have still failed to halt disease progression \\[NCT05695521, NCT03280056, NCT06973629, NCT02290886\\]. Recent research suggests that hematopoietic stem cell transplantation (HSCT) may disrupt the activation cycle between astrocytes and microglia, alleviate chronic inflammatory states in the CNS, partially mitigate mitochondrial dysfunction, and thereby slow neurodegeneration. Unrelated umbilical cord blood transplantation offers advantages such as low HLA-matching requirements, a lower risk of graft-versus-host disease (GVHD), a potent graft-versus-leukemia (GVL) effect, and immediate availability. Investigators plan to conduct an exploratory clinical trial to evaluate the safety and efficacy of umbilical cord blood transplantation for ALS patients. The preliminary plan is to enroll 8 adult subjects. Following successful neutrophil engraftment (defined as an absolute neutrophil count \u22650.5\u00d710\u2079/L for three consecutive days) and confirmation of complete donor chimerism. The trial will focus on assessing transplantation-related complications and patient tolerance. A 3-month post-transplantation follow-up will be conducted for a comprehensive evaluation of safety and efficacy for ALS patients.", "interventions": [{"type": "PROCEDURE", "name": "Mobilization Regimen"}, {"type": "PROCEDURE", "name": "Conditioning Regimen"}, {"type": "PROCEDURE", "name": "GVHD Prophylaxis"}, {"type": "PROCEDURE", "name": "umbilical cord blood"}, {"type": "PROCEDURE", "name": "Rescue"}], "start_date": "2025-12-29", "url": "https://clinicaltrials.gov/study/NCT07539662", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Institute of Hematology & Blood Diseases Hospital", "city": "Tianjin", "state": "", "country": "China", "status": "", "lat": 39.14222, "lon": 117.17667}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "umbilical cord blood", "targeting_mechanism": "modulation of microglia from pro-inflammatory (M1) state to reduce neuroinflammation in the central nervous system", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07571486", "title": "Therapeutic Approach of Repeated Transient Blood-brain Barrier Opening in Amyotrophic Lateral Sclerosis.", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "This is proof-of-concept, single-arm, single-center study to assess the safety and explore the efficacy of repeated US transient disruptions of the blood-brain barrier (BBB) in Amyotrophic Lateral Sclerosis (ALS).\n\nPhase 1:\n\nThe primary objective is to assess the safety of ultrasound induced BBB opening in the upper motor neuron area and adjacent supplementary motor area in adult patients with ALS, as assessed by adverse events frequency and severity during study (incidence of AE summarized by system organ class and/or preferred term and severity) based on the Common Terminology Criteria for Adverse Events, version 5.0 A run-in period of 12 weeks between inclusion and baseline will take place for each patient in order to evaluate precisely disease progression rate, disease severity and to collect concomitant medication. After this run-in period, the patient will be implanted with the SC4 device (baseline visit). The first sonication session will be performed two weeks after implantation. A total of 9 sonications, with no concomitant drug administration, will be performed over a period of 24 weeks.\n\nPhase 2a:\n\nBased on the safety outcome of the Phase 1, an expansion cohort will open to assess the first signal of efficacy of the US transient disruptions of the BBB in ALS. The primary objective will be to assess the first signal of efficacy of the procedure on disease progression over 26 weeks evaluated by the change from baseline to week 26 of neurofilament light (NfL) levels in blood.The Phase 2a will continuously include 11 additional patients. Patients will be treated according to the same schedule as in phase 1", "interventions": [{"type": "DEVICE", "name": "SonoCloud-4 (SC4) device"}], "start_date": "2026-07-15", "url": "https://clinicaltrials.gov/study/NCT07571486", "target_entities": ["blood_brain_barrier_permeability"], "locations": [{"facility": "H\u00f4pital Piti\u00e9-Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "1 42 16 24 72", "contact_email": "gaelle.bruneteau@aphp.fr", "mechanism_summary": {"compound": "SonoCloud-4 (SC4) device", "targeting_mechanism": "ultrasound-induced transient disruption of the blood-brain barrier to enable therapeutic delivery to motor neurons", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00005766", "title": "Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "National Center for Research Resources (NCRR)", "summary": "The objective of this study is to determine whether creatine slows disease progression in subjects with amyotrophic lateral sclerosis (ALS). ALS is a progressive uniformly lethal neurodegenerative disorder for which there is no known cure. Recent genetic and biochemical studies implicate free radical toxicity, glutamate excitotoxicity and mitochondrial dysfunction as possible causes of familial ALS (FALS) and sporadic ALS (SALS). It has been hypothesized that in ALS there may be involvement of oxidative free radical damage and impaired mitochondrial energy metabolism that could in turn lead to excitotoxic cell death. Creatine, an agent that improves mitochondrial function, has been shown to be neuroprotective in animal models of ALS and Huntington's disease.\n\nThis study is a double-blind, randomized, placebo-controlled trial of the safety and efficacy of creatine in patients with ALS enrolled at sites distributed throughout the United States, including Northeast ALS (NEALS) sites. The study will provide preliminary data on the safety and efficacy of creatine in ALS. If creatine slows disease progression in ALS and is well tolerated, a phase 3 study with survival as the primary outcome measure will be initiated.\n\n114 eligible subjects will be randomized to receive treatment for 6 months of (1) active creatine or (2) placebo. After randomization, subjects will be followed prospectively for 6 months. The primary outcome measure for the study is the change in upper extremity motor function after 6 months of experimental therapy as tested with the Tufts Quantitative Neuromuscular Exam. Strength in eight arm muscles will be measured (bilateral shoulder and elbow flexion and extension). Secondary outcome measures include grip strength, motor unit number estimates (MUNE), the ALS functional rating score-revised (ALSFRS-R), and rate of change of a well established biochemical marker of oxidative damage to DNA (8OH2'dG levels in urine), and the safety and tolerability of creatine.", "interventions": [{"type": "DRUG", "name": "Creatinine"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT00005766", "target_entities": ["oxidative_stress", "mitochondrial_dysfunction", "glutamate_excitotoxicity"], "locations": [{"facility": "University of Vermont", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Creatinine", "targeting_mechanism": "mitochondrial support and reduction of free radical toxicity, glutamate excitotoxicity, and mitochondrial dysfunction", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03519880", "title": "A Pilot Study of the Utility of 3D Printed Masks for ALS Subjects", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Michigan", "summary": "Non-invasive ventilation (NIV) is an important therapy for patients with a number of neurological diseases. Specifically, NIV has been shown to be an effective treatment for people with amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease), which is a fatal, non-curable, progressive disease of the motor neurons. However, due to changes in facial structure associated with the disease, many ALS patients find that traditional NIV masks don't fit well. In this study, investigators will perform a feasibility study on NIV mask interfaces which are custom designed for each ALS patient and then manufactured via 3D printing.", "interventions": [{"type": "DEVICE", "name": "Custom Mask Interface"}], "start_date": "2017-03-14", "url": "https://clinicaltrials.gov/study/NCT03519880", "target_entities": [], "locations": [{"facility": "Michigan Medicine", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06834269", "title": "Transcranial Static Magnetic Stimulation (tSMS) and Potential Theranostic Biomarkers in Amyotrophic Lateral Sclerosis.", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Campus Bio-Medico University", "summary": "The objective of the present study is to assess the efficacy of tSMS in ALS patients. This will be achieved by monitoring:\n\n* levels of NF-L and other potential innovative biomarkers,\n* clinical progression, trough ALSFRS-R. After at least three-month follow-up, participants will be recruited to undergo biemispheric tSMS for two daily sessions of 120 minutes each, at home, for 12 months. Together with clinical status, which will be evalueted each three months, blood and urine samples will be collected before the start of the tSMS administration (M0) and during the treatment (M3, M6, M9, M12), to detect potential theranostic biomarkers. In a subgroup of patients, ad additional blood and urine sample will be collected 3 months before M0 (M-3).\n\nMoreover, cortical excitability will be tested through transcranial magnetic stimulation (TMS) before and after the tSMS stimulation period.", "interventions": [{"type": "DEVICE", "name": "Transcranial magnetic stimulation (tSMS)"}], "start_date": "2024-12-02", "url": "https://clinicaltrials.gov/study/NCT06834269", "target_entities": ["motor_cortex_excitability"], "locations": [{"facility": "Fondazione Policlinico Campus Bio-Medico", "city": "Roma", "state": "RM", "country": "Italy", "status": "RECRUITING", "lat": 44.99364, "lon": 11.10642}], "contact_phone": "06 22541 1220", "contact_email": "v.dilazzaro@policlinicocampus.it", "mechanism_summary": {"compound": "Transcranial static magnetic stimulation (tSMS)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01786603", "title": "Rasagiline in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Richard Barohn, MD", "summary": "ALS is a disorder that weakens motor strength and lung function. Rapid loss of motor neurons in the brain and spinal cord of ALS patients causes the symptoms of increasing weakness and loss of muscle function. Motor neurons are responsible for sending signals to muscles in our bodies to trigger movement. While there are drugs to help relieve symptoms of ALS, there is no cure for ALS.\n\nRasagiline is a drug with possible neuroprotective characteristics. Neuroprotective means that the nervous system may be protected against weakening. It is known that rasagiline has possible neuroprotective characteristics, but the effectiveness of rasagiline for patients with ALS has not been tested. Rasagiline is approved for the treatment of Parkinson's disease.\n\nRasagiline for treatment of ALS is not approved by the U.S. Food and Drug Administration (FDA) and is investigational. Investigational drugs are studied to find out if they are safe and effective in the treatment of diseases or conditions.\n\nBy doing this study, researchers hope to learn if rasagiline is safe and slows disease progression in patients with ALS.\n\nFunding Source - FDA OOPD (FDA Orphan Products Division).", "interventions": [{"type": "DRUG", "name": "Rasagiline"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2013-11-21", "url": "https://clinicaltrials.gov/study/NCT01786603", "target_entities": ["Monoamine oxidase"], "locations": [{"facility": "Phoenix Neurological Associates", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California - Irvine", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "St. Louis University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "University of Nebraska", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "", "lat": 41.25626, "lon": -95.94043}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Oregon Health and Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "UT Southwestern Medical Center", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Rasagiline", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04297683", "title": "HEALEY ALS Platform Trial - Master Protocol", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Merit E. Cudkowicz, MD", "summary": "The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.", "interventions": [{"type": "DRUG", "name": "Zilucoplan"}, {"type": "DRUG", "name": "Verdiperstat"}, {"type": "DRUG", "name": "CNM-Au8"}, {"type": "DRUG", "name": "Pridopidine"}, {"type": "DRUG", "name": "SLS-005 Trehalose"}, {"type": "DRUG", "name": "ABBV-CLS-7262"}, {"type": "DRUG", "name": "DNL343"}, {"type": "DRUG", "name": "NUZ-001"}], "start_date": "2020-06-14", "url": "https://clinicaltrials.gov/study/NCT04297683", "target_entities": ["Complement C5", "Myeloperoxidase", "Huntingtin", "Neurofilament light chain", "immunomodulation"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Mayo Clinic Scottsdale", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "University of Arkansas for Medical Sciences", "city": "Little Rock", "state": "Arkansas", "country": "United States", "status": "", "lat": 34.74648, "lon": -92.28959}, {"facility": "UC San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "Loma Linda University Health", "city": "Loma Linda", "state": "California", "country": "United States", "status": "", "lat": 34.04835, "lon": -117.26115}, {"facility": "Kaiser Permanente Los Angeles Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of Southern California", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California, Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Forbes Norris MDA/ALS Research Center, California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of California, San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "Yale University", "city": "New Haven", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.30815, "lon": -72.92816}, {"facility": "Georgetown University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "George Washington University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Nova Southeastern University", "city": "Davie", "state": "Florida", "country": "United States", "status": "", "lat": 26.06287, "lon": -80.2331}, {"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Augusta University", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Saint Alphonsus Regional Medical Center", "city": "Boise", "state": "Idaho", "country": "United States", "status": "", "lat": 43.6135, "lon": -116.20345}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Chicago", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Indiana University Health", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Fairway", "state": "Kansas", "country": "United States", "status": "", "lat": 39.02223, "lon": -94.6319}, {"facility": "University of Kentucky", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Ochsner Health System", "city": "New Orleans", "state": "Louisiana", "country": "United States", "status": "", "lat": 29.95465, "lon": -90.07507}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Beth Israel Deaconess Medical Center", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Lahey Hospital and Medical Center", "city": "Burlington", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.50482, "lon": -71.19561}, {"facility": "University of Massachusetts Medical School", "city": "North Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.31676, "lon": -71.81757}, {"facility": "University of Michigan", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Health System", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Spectrum Health/Corewell Health", "city": "Grand Rapids", "state": "Michigan", "country": "United States", "status": "", "lat": 42.96336, "lon": -85.66809}, {"facility": "Essentia Health", "city": "Duluth", "state": "Minnesota", "country": "United States", "status": "", "lat": 46.78327, "lon": -92.10658}, {"facility": "University of Minnesota Medical School", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Mayo Clinic - Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "University of Missouri Health Care", "city": "Columbia", "state": "Missouri", "country": "United States", "status": "", "lat": 38.95171, "lon": -92.33407}, {"facility": "Saint Louis University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates, P.C./Somnos Clinical Research", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "University of Nebraska Medical Center", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "", "lat": 41.25626, "lon": -95.94043}, {"facility": "Dartmouth-Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}, {"facility": "Hackensack University Medical Center", "city": "Hackensack", "state": "New Jersey", "country": "United States", "status": "", "lat": 40.88593, "lon": -74.04347}, {"facility": "Dent Neurologic Institute", "city": "Amherst", "state": "New York", "country": "United States", "status": "", "lat": 42.97839, "lon": -78.79976}, {"facility": "Mount Sinai", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Rochester", "city": "Rochester", "state": "New York", "country": "United States", "status": "", "lat": 43.15478, "lon": -77.61556}, {"facility": "Stony Brook University Hospital", "city": "Stony Brook", "state": "New York", "country": "United States", "status": "", "lat": 40.92565, "lon": -73.14094}, {"facility": "SUNY Upstate", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "University of North Carolina", "city": "Chapel Hill", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.9132, "lon": -79.05584}, {"facility": "Atrium Health", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest Health Science", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "University of Cincinnati", "city": "Cincinnati", "state": "Ohio", "country": "United States", "status": "", "lat": 39.12711, "lon": -84.51439}, {"facility": "Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "The Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "OhioHealth Research Institute", "city": "Westerville", "state": "Ohio", "country": "United States", "status": "", "lat": 40.12617, "lon": -82.92907}, {"facility": "Providence Brain and Spine Institute ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Lehigh Valley Health Network", "city": "Allentown", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.60843, "lon": -75.49018}, {"facility": "Penn State Hershey", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Jefferson Weinberg ALS Center, Thomas Jefferson University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pennsylvania", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Lewis Katz School of Medicine at Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburg Medical Center", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Vanderbilt University Medical Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Austin Neuromuscular Clinic", "city": "Austin", "state": "Texas", "country": "United States", "status": "", "lat": 30.26715, "lon": -97.74306}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Baylor College of Medicine", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Houston Methodist", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "UTHSCSA", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Virginia", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "Virginia Commonwealth University", "city": "Henrico", "state": "Virginia", "country": "United States", "status": "", "lat": 36.59264, "lon": -78.61611}, {"facility": "Swedish Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "Medical College of Wisconsin", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Zilucoplan, Verdiperstat, CNM-Au8, Pridopidine, SLS-005 Trehalose, ABBV-CLS-7262, DNL343, NUZ-001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07294144", "title": "Tofersen in Non-SOD1 ALS", "phase": "PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Washington University School of Medicine", "summary": "The goal of this clinical trial is to evaluate whether tofersen is safe and effective in adults with non-SOD1 ALS. Tofersen is currently approved by the U.S. Food and Drug Administration to treat SOD1-ALS. The main questions it aims to answer are:\n\n* Does tofersen lower the levels of neurofilament light chain (NfL) in the blood and CSF of adult participants with non-SOD1 ALS?\n* Is tofersen safe and tolerable for adult participants with non-SOD1 ALS?\n* Does tofersen affect other measurements such as clinical outcomes and quality-of-life measures in participants with non-SOD1 ALS?\n\nParticipants will :\n\n* Receive 100mg tofersen via lumbar puncture for 24 weeks. The doses are at the following time points: Weeks 0, 2, 4, 8, 12, 16, 20, and 24.\n* Complete 2 follow-up visits following the end of the dosing period at Weeks 28 and 32.\n* Complete a variety of questionnaires and outcome measurements such as strength and breathing testing.", "interventions": [{"type": "DRUG", "name": "Tofersen"}], "start_date": "2025-12-29", "url": "https://clinicaltrials.gov/study/NCT07294144", "target_entities": ["Neurofilament light chain"], "locations": [{"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "RECRUITING", "lat": 33.749, "lon": -84.38798}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "Washington University ALS Center", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "RECRUITING", "lat": 38.62727, "lon": -90.19789}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Tofersen", "targeting_mechanism": "Antisense oligonucleotide that reduces SOD1 messenger RNA and protein expression.", "targeting_mechanism_pmid": "37975798", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03067857", "title": "Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Stem Cells Arabia", "summary": "Herein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.", "interventions": [{"type": "BIOLOGICAL", "name": "Stem Cells"}], "start_date": "2016-09", "url": "https://clinicaltrials.gov/study/NCT03067857", "target_entities": ["neuroprotection"], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Autologous bone marrow-derived stem cells", "targeting_mechanism": "Stem cells differentiate into support cells (astrocytes, oligodendrocytes, microglia) that produce growth factors and anti-inflammatory cytokines, provide nutrients, and buffer excessive glutamate to benefit degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Preclinical stem cell studies demonstrated potential benefit of stem cell therapy for ALS; the majority of preclinical studies were performed in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1).", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06396260", "title": "Structural and Functional Networks in ALS: An Insight Into Pseudobulbar Affect", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Aalborg University Hospital", "summary": "The investigators aim to elucidate characteristics of structural and functional brain connectivity in patients with amyotrophic lateral sclerosis (ALS) and pseudobulbar affect (PBA) using diffusion kurtosis imaging (DKI) and magnetoencephalography (MEG).", "interventions": [{"type": "OTHER", "name": "Diffusion Kurtosis Imaging (DKI)"}, {"type": "OTHER", "name": "Resting State Magnetoencephalography"}], "start_date": "2024-05-26", "url": "https://clinicaltrials.gov/study/NCT06396260", "target_entities": [], "locations": [{"facility": "Aalborg University hospital", "city": "Aalborg", "state": "Northern Jutland", "country": "Denmark", "status": "RECRUITING", "lat": 57.048, "lon": 9.9187}], "contact_phone": "+4597660000", "contact_email": "c.steenkjaer@rn.dk", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03892382", "title": "Repetitive Transcranial Magnetic Stimulation as Therapy for Apathy in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Jagiellonian University", "summary": "Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by progressive loss of central and peripheral motor neurons. ALS leads to death usually within 3 to 5 years from the onset of the symptoms. Available treatment can prolong the disease duration but cannot modify the disease course. Apathy is a frequent complication of ALS, affecting up to 30% of patients and affecting negatively the survival. Repetitive Transcranial Magnetic Stimulation (rTMS) is a noninvasive method of modulation of brain plasticity with confirmed beneficial effect on apathy in several neurologic and psychiatric conditions. The purpose of this study is to compare the effectiveness of rTMS in improving the apathy in patients with ALS with placebo stimulation.", "interventions": [{"type": "DEVICE", "name": "rTMS"}], "start_date": "2019-11-15", "url": "https://clinicaltrials.gov/study/NCT03892382", "target_entities": ["motor_cortex_excitability"], "locations": [{"facility": "Jagiellonian University Medical College, Department of Neurology", "city": "Krakow", "state": "", "country": "Poland", "status": "", "lat": 50.06143, "lon": 19.93658}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "rTMS (repetitive transcranial magnetic stimulation)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00982150", "title": "Extension Study of Talampanel for Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Teva Branded Pharmaceutical Products R&D, Inc.", "summary": "This will be an open label treatment extension phase in patients with ALS who have previously participated in the double blind, placebo-controlled ALS-TAL-201 study. This study will make talampanel treatment available to all subjects who completed the double blind placebo-controlled phase of ALS-TAL-201 study and where the investigator and patient consider it to be in the patient's interest to receive talampanel 50mg three times daily (tid). It will also enable the exploration of long-term safety and tolerability of talampanel 50mg tid.", "interventions": [{"type": "DRUG", "name": "Talampanel"}], "start_date": "2009-09", "url": "https://clinicaltrials.gov/study/NCT00982150", "target_entities": ["AMPA receptor"], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Talampanel", "targeting_mechanism": "AMPA receptor antagonist that blocks glutamate signaling through AMPA receptors to reduce excitotoxic motor neuron injury.", "targeting_mechanism_pmid": "27350567", "animal_results": "AMPA receptor antagonist perampanel robustly rescues ALS pathology in sporadic ALS model mice (AR2 conditional ADAR2 knockout mice).", "animal_results_pmid": "27350567", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06711510", "title": "Predict to Prevent PGRN Disease", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "Progranulin (GRN or PGRN) mutations are among the most common genetic causes of frontotemporal lobar degeneration (FTLD). With the advent of gene-specific therapeutic interventions, an accurate knowledge of the presymptomatic phase of the disease is of utmost importance. Increases of plasma neurofilament light chain (NfL) levels are good predictors of phenoconversion in presymptomatic carriers. However their increase rates remain partially elucidated insofar, with many confounding factors. Another point which deserves further precision is the definition of the biological onset of the disease, via the identification of markers of intraneuronal accumulation of TDP-43 protein. PREVENT-PGRN aims aims at studying the trajectory of plasma NfL changes in presymptomatic GRN mutation carriers in comparison with healthy controls, in partnership with the GENFI-QBS study. Additionally, other disease-related biomarkers, namely associated with TDP-43 pathology, will be investigated in this study, at the presymptomatic and clinical phase.", "interventions": [{"type": "OTHER", "name": "Clinical, behavioral and cognitive evaluation"}, {"type": "OTHER", "name": "blood sampling"}, {"type": "OTHER", "name": "brain MRI"}, {"type": "OTHER", "name": "olfactory swab sampling with nasal brush"}, {"type": "OTHER", "name": "skin punch biopsy"}, {"type": "OTHER", "name": "lumbar puncture for CSF sampling"}], "start_date": "2025-01", "url": "https://clinicaltrials.gov/study/NCT06711510", "target_entities": ["GRN"], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03954600", "title": "A Study to Assess the Safety, Tolerability and PK of NPT520-34 in Healthy Subjects", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuropore Therapies Inc.", "summary": "To evaluate the PK, safety and tolerability of orally administered NPT520-34 in healthy subjects at single and multiple doses that may be therapeutically relevant.", "interventions": [{"type": "DRUG", "name": "NPT520-34 (125 mg)"}, {"type": "DRUG", "name": "Placebos (125 mg)"}], "start_date": "2019-05-05", "url": "https://clinicaltrials.gov/study/NCT03954600", "target_entities": ["TAU", "neuroinflammation"], "locations": [{"facility": "Celerion, Inc", "city": "Tempe", "state": "Arizona", "country": "United States", "status": "", "lat": 33.41477, "lon": -111.90931}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "NPT520-34", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03411863", "title": "Cervical Electrical Stimulation for ALS", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "VA Office of Research and Development", "summary": "Veterans are at higher risk than non-Veterans of falling ill with amyotrophic lateral sclerosis (ALS). ALS causes degeneration of motor neurons in both the brain and the spinal cord. Evidence from studies in people with spinal cord injury suggests that activating spared nerve circuits with electromagnetic stimulation improves nerve transmission.\n\nWith this goal, the investigators have developed a novel method of noninvasive cervical (neck) electrical stimulation (CES). In this study, the investigators will investigate CES for its potential to strengthen nerve circuits to the hands in ALS.\n\nTo the investigators' knowledge, electrical spinal stimulation for ALS has never been tested previously. This study will be performed in two stages: First, basic experiments will be performed to better understand how CES interacts with other types of electrical and magnetic stimulations over the brain and peripheral nerves. Second, experiments will be performed to determine the types of CES that can facilitate active arm and hand movements.\n\nThese experiments will improve understanding of electrical stimulation in ALS, and may set the table for future treatments.\n\nBoth United States Veterans and non-Veterans are eligible to participate in this study.", "interventions": [{"type": "DEVICE", "name": "CES at rest"}, {"type": "DEVICE", "name": "CES plus active hand or wrist movements"}], "start_date": "2018-01-04", "url": "https://clinicaltrials.gov/study/NCT03411863", "target_entities": ["motor_neuron_circuits"], "locations": [{"facility": "James J. Peters VA Medical Center, Bronx, NY", "city": "The Bronx", "state": "New York", "country": "United States", "status": "", "lat": 40.84985, "lon": -73.86641}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07401121", "title": "Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Celosia Therapeutics Pty Ltd", "summary": "This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.", "interventions": [{"type": "DRUG", "name": "AAV9 Gene therapy"}], "start_date": "2026-01-12", "url": "https://clinicaltrials.gov/study/NCT07401121", "target_entities": ["SMN1"], "locations": [{"facility": "Macquarie University Hospital", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": -33.86785, "lon": 151.20732}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "CTx1000", "targeting_mechanism": "AAV9-delivered gene therapy targeting motor neuron degeneration in ALS.", "targeting_mechanism_pmid": "33839324", "animal_results": "In AAV9-amiRSOD1 treatment, a 50% increase in median survival and preservation of motor function were achieved in SOD1G93A transgenic ALS mice, with spinal subpial delivery of AAV9 demonstrating widespread gene silencing and blockade of motoneuron degeneration in adult symptomatic SOD1G37R ALS mice.", "animal_results_pmid": "26891182", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03272802", "title": "Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Isfahan University of Medical Sciences", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that causes the death of 30,000 affected individual every year. Complex nature and unknown pathogenesis of this disease are 2 major reasons for failure of therapeutic interventions. Edaravone is a free radical scavenger that slows down functional decline and prevents from disease progression in ALS patients. FDA newly approved this drug in these patients (2017/5/5). In this study, investigators aimed to assess the treatment effect of this newly approved drug in patients with ALS in a representative Iranian population.", "interventions": [{"type": "DRUG", "name": "Edaravone"}, {"type": "DRUG", "name": "Riluzole"}], "start_date": "2017-03-16", "url": "https://clinicaltrials.gov/study/NCT03272802", "target_entities": ["oxidative_stress"], "locations": [{"facility": "EMG Department, Alzahra Hospital", "city": "Isfahan", "state": "", "country": "Iran", "status": "", "lat": 32.65246, "lon": 51.67462}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Edaravone", "targeting_mechanism": "Free radical scavenger that quenches hydroxyl radicals and inhibits lipid peroxidation.", "targeting_mechanism_pmid": "38474192", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "acute cerebral infarction", "repurposed_from_pmid": "38474192"}}
{"nct_id": "NCT01565395", "title": "Incobotulinum Toxin A for Sialorrhea in Parkinson's Disease (PD)/Parkinsonism and Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Beth Israel Deaconess Medical Center", "summary": "The purpose of this study is to evaluate the safety and efficacy of Incobotulinum Toxin A (Xeomin\u00ae) injections into the parotid and submandibular glands in patients with Parkinson's Disease/Parkinsonism and Amyotrophic Lateral Sclerosis (ALS) with troublesome sialorrhea.", "interventions": [{"type": "DRUG", "name": "Incobotulinum Toxin A"}, {"type": "DRUG", "name": "placebo"}], "start_date": "2012-03", "url": "https://clinicaltrials.gov/study/NCT01565395", "target_entities": ["acetylcholine"], "locations": [{"facility": "Beth Israel Deaconess Medical Center", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Incobotulinum Toxin A", "targeting_mechanism": "Botulinum toxin that inhibits acetylcholine release at the neuromuscular junction to reduce salivary gland secretion.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06391645", "title": "Nerve Growth Factor Encapsulated With 2-methacryloyloxyethyl Phosphorylcholine Nanocapsules in the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2, PHASE3", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Beijing Tiantan Hospital", "summary": "Amyotrophic lateral sclerosis (ALS) is one of the most lethal neurodegenerative diseases, with most patients dying from respiratory failure 3-5 years after the onset. The purpose of this study is to explore the efficacy and safety of nerve growth factor (NGF) encapsulated with 2-methacryloyloxyethyl phosphorylcholine (MPC) nanocapsules in the treatment of ALS patients.", "interventions": [{"type": "DRUG", "name": "Nerve Growth Factor"}], "start_date": "2024-05", "url": "https://clinicaltrials.gov/study/NCT06391645", "target_entities": ["NGF"], "locations": [], "contact_phone": "13521588395", "contact_email": "liuxinru0826@163.com", "mechanism_summary": {"compound": "Nerve Growth Factor encapsulated with 2-methacryloyloxyethyl phosphorylcholine nanocapsules", "targeting_mechanism": "Nerve growth factor (NGF) promotes neuronal survival and growth to support motor neuron function.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05632757", "title": "Anticipated Patient and Caregiver Burden", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique Hopitaux De Marseille", "summary": "Amyotrophic lateral sclerosis (ALS) is a degenerative neurological disease that causes progressive motor disability and is life threatening within a few years. The severity of the disease, the progressive loss of autonomy that leads to dependence on family and caregivers, and the lack of effective treatment sometimes leads patients to a loss of hope and to dark thoughts. The prevalence of suicidal ideation is high, with more than one third of people with ALS experiencing it. The psychological suffering of patients is often associated with that of their caregivers. The evaluation of the patients' feeling of being a burden has rarely been addressed in previous studies in ALS on the notion of burden. In this work, the investigators wish to evaluate the patient's ideas of death by also taking into account the caregiver's burden and the patient's feeling of being a burden. They wish to better understand this difficult experience by refocusing the study on the patient himself, which has rarely been addressed in studies on ALS and the notion of burden. By working on the caregiver's burden, both from the caregiver's point of view and as perceived by the patient, the investigators hope to find avenues of intervention and define actions that could help patients and their families and improve the quality of life of the patient-caregiver couple.", "interventions": [{"type": "BEHAVIORAL", "name": "Psychological assessments"}], "start_date": "2023-06-22", "url": "https://clinicaltrials.gov/study/NCT05632757", "target_entities": [], "locations": [{"facility": "Service Maladies neuromusculaires et SLA", "city": "Marseille", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.29695, "lon": 5.38107}], "contact_phone": "0491386578", "contact_email": "annie.verschueren@ap-hm.fr", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00035815", "title": "Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS) Trial", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mayo Clinic", "summary": "The purpose of this multicenter study is to determine if insulin-like growth factor-1 (IGF-I) slows the progressive weakness in amyotrophic lateral sclerosis (ALS) patients. Study participants will be followed for 2 years once enrolled. They will receive either placebo or the active IGF-I. Examinations will take place at approximately 6-month intervals.", "interventions": [{"type": "DRUG", "name": "Insulin like growth factor, type 1"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2003-06", "url": "https://clinicaltrials.gov/study/NCT00035815", "target_entities": ["IGF1"], "locations": [{"facility": "Mayo Clinic in Scottsdale", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic in Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Indiana University", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Michigan Medical Center", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Hospital", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "University of Mississippi", "city": "Jackson", "state": "Mississippi", "country": "United States", "status": "", "lat": 32.29876, "lon": -90.18481}, {"facility": "Beth Israel Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Rochester Medical Center", "city": "Rochester", "state": "New York", "country": "United States", "status": "", "lat": 43.15478, "lon": -77.61556}, {"facility": "University of Cincinnati", "city": "Cincinnati", "state": "Ohio", "country": "United States", "status": "", "lat": 39.12711, "lon": -84.51439}, {"facility": "Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Ohio State University", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "University of Pennsylvania, Pennsylvania Hospital", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Methodist Hospital", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "West Virginia University", "city": "Morgantown", "state": "West Virginia", "country": "United States", "status": "", "lat": 39.62953, "lon": -79.9559}, {"facility": "Froedtert and Medical College Clinics", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "University of Puerto Rico", "city": "San Juan", "state": "", "country": "Puerto Rico", "status": "", "lat": 18.46633, "lon": -66.10572}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Insulin-like growth factor-1", "targeting_mechanism": "Insulin-like growth factor-1 (IGF-I) promotes motor neuron survival and slows progressive weakness through growth factor signaling.", "targeting_mechanism_pmid": "", "animal_results": "Intramuscular delivery of scAAV9-hIGF1 prolonged survival in the hSOD1(G93A) ALS mouse model via upregulation of D-amino acid oxidase.", "animal_results_pmid": "27995572", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04089696", "title": "Validation of the \"ExSpiron\u00a9\" in Patients With ALS", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Medical Center Groningen", "summary": "Non-invasive ventilation (NIV) in patients diagnosed with amyotrophic lateral sclerosis (ALS) is nowadays common practice to provide comfort at the end stage of the disease. As complaints vary there is the need of a non-invasive device to measure respiratory volume to objectify complaints.\n\nThe ExSpiron\u00a9 is a device for non-invasive monitoring of respiratory volume. The validation of this monitor in patients with ALS is the aim of this study.\n\nThe hypothesis is that the ExSpiron\u00a9 delivers a valid assessment of respiratory volume in patients with ALS", "interventions": [{"type": "DEVICE", "name": "ExSprion"}], "start_date": "2025-09", "url": "https://clinicaltrials.gov/study/NCT04089696", "target_entities": [], "locations": [], "contact_phone": "+31 50 3613200", "contact_email": "p.j.wijsktra@umcg.nl", "mechanism_summary": {"compound": "ExSpiron", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07322003", "title": "Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS", "phase": "PHASE3", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Prilenia", "summary": "The goal of this clinical trial is to learn if the drug pridopidine works to treat amyotrophic lateral sclerosis in adults. It will also help to learn about the safety of pridopidine. The main question it aims to answer is:\n\nDoes pridopidine slow disease progression of ALS?\n\nResearchers will compare pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works to treat ALS.\n\nParticipants will:\n\nTake pridopidine or a placebo by mouth every day for 48 weeks. Afterwards, all participants will take pridopidine for another 48 weeks.\n\nVisit the clinic once every 1-3 months for checkups and tests", "interventions": [{"type": "DRUG", "name": "Pridopidine"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2026-02-01", "url": "https://clinicaltrials.gov/study/NCT07322003", "target_entities": ["Sigma-1 receptor"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "RECRUITING", "lat": 33.44838, "lon": -112.07404}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "RECRUITING", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado CU Anschutz Neurology", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "RECRUITING", "lat": 39.72943, "lon": -104.83192}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "RECRUITING", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern University", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "RECRUITING", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas", "city": "Fairway", "state": "Kansas", "country": "United States", "status": "RECRUITING", "lat": 39.02223, "lon": -94.6319}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "RECRUITING", "lat": 39.29038, "lon": -76.61219}, {"facility": "Sean M. Healey & AMG Center for ALS", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "RECRUITING", "lat": 42.35843, "lon": -71.05977}, {"facility": "Washington University", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "RECRUITING", "lat": 38.62727, "lon": -90.19789}, {"facility": "Somnos Clinical Research", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "RECRUITING", "lat": 40.8, "lon": -96.66696}, {"facility": "Eleanor and Lou Gehrig ALS Center at Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "RECRUITING", "lat": 40.71427, "lon": -74.00597}, {"facility": "Neuroscience Department at LKSM at Temple University", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "RECRUITING", "lat": 39.95238, "lon": -75.16362}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 32.78306, "lon": -96.80667}, {"facility": "Baylor College of Medicine; McNair Medical Campus", "city": "Houston", "state": "Texas", "country": "United States", "status": "RECRUITING", "lat": 29.76328, "lon": -95.36327}, {"facility": "Swedish Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "RECRUITING", "lat": 47.60621, "lon": -122.33207}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "RECRUITING", "lat": 47.60621, "lon": -122.33207}, {"facility": "UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "Genge Partners", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}, {"facility": "University of Alberta", "city": "Edmonton", "state": "", "country": "Canada", "status": "RECRUITING", "lat": 53.55014, "lon": -113.46871}, {"facility": "McMaster University Medical Centre", "city": "Hamilton", "state": "", "country": "Canada", "status": "NOT_YET_RECRUITING", "lat": 43.25011, "lon": -79.84963}, {"facility": "CHU Lille", "city": "Lille", "state": "", "country": "France", "status": "RECRUITING", "lat": 50.63391, "lon": 3.05512}, {"facility": "H\u00f4pitaux Universitaires de Marseille Timone", "city": "Marseille", "state": "", "country": "France", "status": "NOT_YET_RECRUITING", "lat": 43.29695, "lon": 5.38107}, {"facility": "Centre Hospitalier Universitaire de Nice", "city": "Nice", "state": "", "country": "France", "status": "RECRUITING", "lat": 43.70313, "lon": 7.26608}, {"facility": "Hospitalier Piti\u00e9-Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}, {"facility": "Centre hospitalier r\u00e9gional universitaire de de Tours", "city": "Tours", "state": "", "country": "France", "status": "RECRUITING", "lat": 47.39484, "lon": 0.70398}, {"facility": "ALS outpatient department at Charit\u00e9 - university medicine Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 52.52437, "lon": 13.41053}, {"facility": "ALS outpatient department at Charit\u00e9 - university medicine Berlin", "city": "Berlin", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 52.52437, "lon": 13.41053}, {"facility": "ALS Clinic Bonn University", "city": "Bonn", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 50.73438, "lon": 7.09549}, {"facility": "University Hospital Jena", "city": "Jena", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 50.92878, "lon": 11.5899}, {"facility": "Universit\u00e4tsklinikum Schleswig-Holstein", "city": "M\u00fcnchen", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 51.60698, "lon": 13.31243}, {"facility": "University of Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "Trinity College Dublin", "city": "Dublin", "state": "", "country": "Ireland", "status": "RECRUITING", "lat": 53.33306, "lon": -6.24889}, {"facility": "Hadassah Medical Center", "city": "Jerusalem", "state": "", "country": "Israel", "status": "RECRUITING", "lat": 31.76904, "lon": 35.21633}, {"facility": "Tel Aviv Sourasky Medical Center", "city": "Tel Aviv", "state": "", "country": "Israel", "status": "RECRUITING", "lat": 32.08088, "lon": 34.78057}, {"facility": "University of Campania \"Luigi Vanvitelli\"", "city": "Caserta", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 41.07262, "lon": 14.33231}, {"facility": "Centro Clinico NEMO Fondazione Serena Onlus - ASST Niguarda Hospital", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.46427, "lon": 9.18951}, {"facility": "Istituto Auxologico Italiano Ospedale San Luca", "city": "Milan", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 45.46427, "lon": 9.18951}, {"facility": "Azienda Ospedaliero Universitaria Di Modena", "city": "Modena", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 44.64783, "lon": 10.92539}, {"facility": "University of Padova", "city": "Padova", "state": "", "country": "Italy", "status": "RECRUITING", "lat": 44.38225, "lon": 11.14261}, {"facility": "University of Torino", "city": "Torino", "state": "", "country": "Italy", "status": "NOT_YET_RECRUITING", "lat": 44.88856, "lon": 11.99138}, {"facility": "UMC Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "RECRUITING", "lat": 52.09083, "lon": 5.12222}, {"facility": "Centrum Medyczne Neuromed", "city": "Bydgoszcz", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 53.1235, "lon": 18.00762}, {"facility": "Linden sp. z o. o. sp. k.", "city": "Krakow", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 50.06143, "lon": 19.93658}, {"facility": "City Clinic Research Sp. z o.o.", "city": "Warsaw", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 52.22977, "lon": 21.01178}, {"facility": "Hospital del Mar", "city": "Barcelona", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 41.38879, "lon": 2.15899}, {"facility": "University Hospital of Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario La Paz - Carlos III", "city": "Madrid", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 40.4165, "lon": -3.70256}, {"facility": "Clinical Hospital Santiago de Compostela", "city": "Santiago de Compostela", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 42.88052, "lon": -8.54569}, {"facility": "Hospital Universitari i Polit\u00e8cnic La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "RECRUITING", "lat": 39.47391, "lon": -0.37966}, {"facility": "Studieenheten Akademiskt specialistcentrum", "city": "Stockholm", "state": "", "country": "Sweden", "status": "RECRUITING", "lat": 59.32938, "lon": 18.06871}, {"facility": "Ume\u00e5 University Hospital", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "RECRUITING", "lat": 63.82842, "lon": 20.25972}, {"facility": "King's College Hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 51.50853, "lon": -0.12574}, {"facility": "MDN Centre Queen Square Institute of Neurology", "city": "London", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 51.50853, "lon": -0.12574}, {"facility": "Royal Stoke University Hospital", "city": "Stoke-on-Trent", "state": "", "country": "United Kingdom", "status": "RECRUITING", "lat": 53.00415, "lon": -2.18538}], "contact_phone": "857-574-5755", "contact_email": "MedInfo@prilenia.com", "mechanism_summary": {"compound": "Pridopidine", "targeting_mechanism": "Pridopidine is a sigma-1/2 receptor modulator for the treatment of neurodegenerative disorders.", "targeting_mechanism_pmid": "37166702", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06849115", "title": "Effects of L-Carnitine in Amyotrophic Lateral Sclerosis Patients With CHCHD10 Mutations", "phase": "PHASE4", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "ChaodongWang", "summary": "The study was designed as a single-center, open-label, single-arm pilot study to estimate the safety and efficacy L-carnitine in ALS patients with CHCHD10 mutations.", "interventions": [{"type": "DRUG", "name": "L-Carnitine Injection\uff0c1000mg once daily"}], "start_date": "2024-07-18", "url": "https://clinicaltrials.gov/study/NCT06849115", "target_entities": ["CHCHD10", "mitochondrial_dysfunction"], "locations": [{"facility": "Xuanwu Hospital, Capital Medical University", "city": "Beijing", "state": "China", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "L-Carnitine", "targeting_mechanism": "L-Carnitine improves mitochondrial function and energy production in the context of CHCHD10-associated mitochondrial dysfunction.", "targeting_mechanism_pmid": "38002924", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06100276", "title": "Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)", "phase": "PHASE1, PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "UniQure Biopharma B.V.", "summary": "This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.", "interventions": [{"type": "DRUG", "name": "AMT-162"}], "start_date": "2024-08-01", "url": "https://clinicaltrials.gov/study/NCT06100276", "target_entities": ["SOD1"], "locations": [{"facility": "Barrow Neurological Institute", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California Irvine", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic Florida", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Winship Cancer Institute of Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern University Feinberg School of Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas Medical Center", "city": "Fairway", "state": "Kansas", "country": "United States", "status": "", "lat": 39.02223, "lon": -94.6319}, {"facility": "Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Mayo Clinic Rochester", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Columbia University Irving Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "University of Pennsylvania School of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Norrlands Universitetssjukhus", "city": "Ume\u00e5", "state": "Vasterbottens Ian", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "AMT-162", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01906658", "title": "A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mallinckrodt", "summary": "This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral Sclerosis (ALS). The study will also investigate the mean rate of change in the ALSFRS-R total score as an exploratory endpoint to help design future studies.\n\nThis study will enroll up to 40 patients and include an optional 28-week open-label extension period plus a 3-week treatment taper and 1-week follow up period. After completion of Week 8, patients enrolled in a treatment group that is considered safe and tolerable at that time have the option to continue into the open-label extension period. A 3-week treatment taper and a follow-up visit are planned for all patients enrolled in the study, beginning either at Week 8 or at Week 36 if a patient continues into the optional open-label extension period.", "interventions": [{"type": "DRUG", "name": "Repository corticotropin injection"}], "start_date": "2013-07", "url": "https://clinicaltrials.gov/study/NCT01906658", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Questcor Investigational Site", "city": "Birmingham", "state": "Alabama", "country": "United States", "status": "", "lat": 33.52066, "lon": -86.80249}, {"facility": "Questcor Investigational Site", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Questcor Investigational Site", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Questcor Investigational Site", "city": "Stanford", "state": "California", "country": "United States", "status": "", "lat": 37.42411, "lon": -122.16608}, {"facility": "Questcor Investigational Site", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Questcor Investigational Site", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Questcor Investigational Site", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Questcor Investigational Site", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Questcor Investigational Site", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Questcor Investigational Site", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Questcor Investigational Site", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Questcor Investigational Site", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Questcor Investigational Site", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Questcor Investigational Site", "city": "Memphis", "state": "Tennessee", "country": "United States", "status": "", "lat": 35.14953, "lon": -90.04898}, {"facility": "Questcor Investigational Site", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Questcor Investigational Site", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Questcor Investigational Site", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Repository corticotropin injection (Acthar)", "targeting_mechanism": "Acthar modulates neuroinflammation to reduce the chronic inflammatory response that contributes to motor neuron degeneration.", "targeting_mechanism_pmid": "28872464", "animal_results": "CSF1R blockade slows ALS progression by reducing microgliosis and invasion of macrophages into peripheral nerves in SOD1 transgenic mice.", "animal_results_pmid": "27174644", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03506425", "title": "A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Richard Bedlack, M.D., Ph.D.", "summary": "The causes of ALS are largely unknown. However, mitochondrial dysfunction, resulting in impaired energy production, oxidative stress and apoptosis, may play a key role in ALS progression. Triheptanoin can improve mitochondrial function and energy production and therefore has potential for slowing ALS progression. Indeed, triheptanoin slowed motor neuron loss and delayed the onset of weakness in a mutant SOD1 model of ALS. This pilot trial will determine if Triheptanoin is safe tolerable, alters biomarkers of brain energy metabolism and oxidative stress, and slows functional decline in people with ALS.", "interventions": [{"type": "DRUG", "name": "Triheptanoin"}], "start_date": "2018-06-21", "url": "https://clinicaltrials.gov/study/NCT03506425", "target_entities": ["mitochondrial_dysfunction", "oxidative_stress"], "locations": [{"facility": "Duke University", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Triheptanoin", "targeting_mechanism": "Triheptanoin improves mitochondrial function and energy production to reduce oxidative stress and motor neuron loss.", "targeting_mechanism_pmid": "38002924", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03948178", "title": "Effects of Oral Levosimendan on Respiratory Function in Patients With Amyotrophic Lateral Sclerosis (ALS): Open-Label Extension", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Orion Corporation, Orion Pharma", "summary": "This study provides an opportunity for subjects in the REFALS (3119002; NCT03505021) study to continue treatment with oral levosimendan. The study will also provide more information about long-term safety and effectiveness of oral levosimendan in patients with ALS.\n\nThis is an open-label study, so that all eligible subjects that complete the double-blind REFALS study (48-weeks of treatment) will have the opportunity to receive oral levosimendan treatment. The primary objective, in addition to continuing treatment for subjects enrolled in the REFALS study, is to evaluate long-term safety of oral levosimendan in ALS patients. Another important objective is to explore long-term effectiveness of oral levosimendan in the treatment of patients with ALS.\n\nThis study is open only to patients taking part in the REFALS study.", "interventions": [{"type": "DRUG", "name": "Levosimendan"}], "start_date": "2019-06-26", "url": "https://clinicaltrials.gov/study/NCT03948178", "target_entities": ["cardiac_contractility"], "locations": [{"facility": "Neuromuscular research Centre and Neuromuscular Clinic of Arizona", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "University of California San Diego", "city": "La Jolla", "state": "California", "country": "United States", "status": "", "lat": 32.84727, "lon": -117.2742}, {"facility": "University of California Irvine Medical Center", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "Georgetown University", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "The George Washington Medical Faculty Associates", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "Holy Cross Hospital Neuroscience Institute", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "", "lat": 26.12231, "lon": -80.14338}, {"facility": "University of Florida McKnight Brain Institute", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Mayo Clinic Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of South Florida/USF Health", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Augusta University, Medical Centre", "city": "Augusta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.47097, "lon": -81.97484}, {"facility": "Northwestern University Feinberg School of Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Chicago", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins Hospital", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "University of Michigan, Michigan Medicine University Hospital", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Health Partners Speciality Center", "city": "Saint Paul", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.94441, "lon": -93.09327}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Hospital for Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Columbia Presbyterian Hospital", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Neurosciences Institute - Neurology Charlotte", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Wake Forest University Baptist Medical Center", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "The Ohio State University Wexner Medical center", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Oregon Health and Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Providence Brain and Spine Institute", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": 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"Australia", "status": "", "lat": -33.88965, "lon": 151.17642}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Flinders Medical Centre", "city": "Bedford Park", "state": "South Australia", "country": "Australia", "status": "", "lat": -35.02204, "lon": 138.56815}, {"facility": "Calvary Health Care Bethlehem", "city": "Parkdale", "state": "Victoria", "country": "Australia", "status": "", "lat": -37.99187, "lon": 145.08128}, {"facility": "Perron Institute for Neurological and Translational Science", "city": "Nedlands", "state": "Western Australia", "country": "Australia", "status": "", "lat": -31.98184, "lon": 115.8073}, {"facility": "Medizinische Universitat Innsbruck", "city": "Innsbruck", "state": "Tyrol", "country": "Austria", "status": "", "lat": 47.26266, "lon": 11.39454}, {"facility": "Salzqammergut-klinikum Vocklabruck, Neurologie", "city": "V\u00f6cklabruck", "state": "Upper Austria", "country": "Austria", "status": "", "lat": 48.00279, "lon": 13.65652}, {"facility": "Medizinische Universitat wein Universitatsklinik ffur Neurologie", "city": "Wein", "state": "", "country": "Austria", "status": "", "lat": null, "lon": null}, {"facility": "Algemeen Ziekenhuis St Lucas Gent", "city": "Ghent", "state": "", "country": "Belgium", "status": "", "lat": 51.05, "lon": 3.71667}, {"facility": "Universitair Ziekenhuis Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Centre Hospitalier Regional de la Vitadelle", "city": "Li\u00e8ge", "state": "", "country": "Belgium", "status": "", "lat": 50.63373, "lon": 5.56749}, {"facility": "Alberta Health Services-Neuromuscular Clinic", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta, Division of Neurology", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Stan Cassidy Centre for Rehabilitation", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "McMaster University Medical Centre", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Centre Hospitalier Affilie Universitaire de Quebec", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Helsinki University Central Hospital, Neurology Outpatients Clinic", "city": "Helsinki", "state": "", "country": "Finland", "status": "", "lat": 60.16952, "lon": 24.93545}, {"facility": "Turku University Hospital", "city": "Turku", "state": "", "country": "Finland", "status": "", "lat": 60.45148, "lon": 22.26869}, {"facility": "Centre Hospitalier Universitaire de Limoges Service de Neurologie", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Hopital Gui de Chauliac Service de Neurologie", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Hopital Pasteur Centre de reference des Malades Neuromusculaires et SLA", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Charite Universitatmedizin Berlin- Campus Virchow-Klinikum", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Medizinische Hochschule Hannover", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Universitatsklinikum Jena, Klinik fur Neurologie", "city": "Jena", "state": "", "country": "Germany", "status": "", "lat": 50.92878, "lon": 11.5899}, {"facility": "Universitatsklinikum Munster, Institut fur Schalfmedizin und Neuromuskalaire Erkrankungen", "city": "M\u00fcnster", "state": "", "country": "Germany", "status": "", "lat": 51.96236, "lon": 7.62571}, {"facility": "Universitatsmedizin Rostock, Klinik und Poliklinik fuer Neurologie", "city": "Rostock", "state": "", "country": "Germany", "status": "", "lat": 54.0887, "lon": 12.14049}, {"facility": "Universitatsklinikum Ulm, Poliklinik fur Neurologie", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Deutsche Klinik fur Daignostik", "city": "Wiesbaden", "state": "", "country": "Germany", "status": "", "lat": 50.08601, "lon": 8.24435}, {"facility": "Beaumont Hospital, Clinical Research Centre", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "Azienda Policlinico San Martino", "city": "Genova", "state": "", "country": "Italy", "status": "", "lat": 45.21604, "lon": 11.87211}, {"facility": "ICS Maugeri Spa S UO Riabilitazione Nurologica", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 45.46427, "lon": 9.18951}, {"facility": "Azienda Ospedaliera Universitaria-maggiore della Carita di Novara", "city": "Novara", "state": "", "country": "Italy", "status": "", "lat": 45.44694, "lon": 8.62118}, {"facility": "Azienda Ospedaliero Universitaria Pisana Ospedale Santa Chiara", "city": "Pisa", "state": "", "country": "Italy", "status": "", "lat": 43.70853, "lon": 10.4036}, {"facility": "Policlinico Umberto I di Roma Clinica Neurologica", "city": "Rome", "state": "", "country": "Italy", "status": "", "lat": 41.89193, "lon": 12.51133}, {"facility": "Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Univeritair Medisch Centrum Utrech", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital Universitari de Bellvitge", "city": "Barcelona", "state": "", "country": "Spain", "status": "", "lat": 41.38879, "lon": 2.15899}, {"facility": "Hospital Universitario de Basurto", "city": "Bilbao", "state": "", "country": "Spain", "status": "", "lat": 43.26271, "lon": -2.92528}, {"facility": "Hospital Universitario Reina Sofia Servicio Neurologia", "city": "C\u00f3rdoba", "state": "", "country": "Spain", "status": "", "lat": 37.89155, "lon": -4.77275}, {"facility": "Hospital San Rafael", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}, {"facility": "Hospital Universitario y Politecnico de La Fe", "city": "Valencia", "state": "", "country": "Spain", "status": "", "lat": 39.47391, "lon": -0.37966}, {"facility": "Karlstad Central Hospital Neurology and Rehabilitation", "city": "Karlstad", "state": "", "country": "Sweden", "status": "", "lat": 59.3793, "lon": 13.50357}, {"facility": "Karolinska University Horpital Huddinge Neurology Clinic", "city": "Stockholm", "state": "", "country": "Sweden", "status": "", "lat": 59.32938, "lon": 18.06871}, {"facility": "Norrlanda University Hospital Neuro-huvud-hals-centrum Vasterbotten", "city": "Ume\u00e5", "state": "", "country": "Sweden", "status": "", "lat": 63.82842, "lon": 20.25972}, {"facility": "The Walton Centre NHs Foundation Trust, Neurology and Neurosurgery", "city": "Liverpool", "state": "", "country": "United Kingdom", "status": "", "lat": 53.41058, "lon": -2.97794}, {"facility": "Barts Health NHS Trust Royal London hospital", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Levosimendag", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02464748", "title": "Telehealth in Motor Neurone Disease", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sheffield Teaching Hospitals NHS Foundation Trust", "summary": "Motor neurone disease (MND) is a rare but debilitating neurological condition that causes paralysis of the body's muscles leading to severe disability and eventually death. Patients often struggle to travel the long distances to specialist clinics to receive the care they require whilst this expert care is often unavailable in the community. Telehealth has the potential to enable a specialist team to monitor the health and wellbeing of patients and their carers whilst they are at home. This could improve the patient's health, improve the quality of life of both patients and their carers, and lead to more effective use of health resources.\n\nThis is a randomised controlled pilot study that will involve 40 patients who are cared for by the Sheffield Motor Neurone Disease care centre and their main informal carer (a total of 80 participants). Half of the participants will use the telehealth system for a minimum of six months and maximum of eighteen months and information will be collected from patients, carers and their care team. This will include collecting clinical outcome measures, health resource use and the opinions and experience of using the system. All participants will continue to receive their usual care.\n\nThis is a pilot study. It aims to determine the feasibility and acceptability of the telehealth system to patients, carers and their health care providers. It also aims to determine how a larger trial could successfully evaluate the clinical and cost-effectiveness of the system.", "interventions": [{"type": "OTHER", "name": "TiM telehealth arm"}], "start_date": "2014-09", "url": "https://clinicaltrials.gov/study/NCT02464748", "target_entities": [], "locations": [{"facility": "Sheffield Institute for Translational Neurosciences", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03160898", "title": "A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Cytokinetics", "summary": "The purpose of this study was to assess the effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.", "interventions": [{"type": "DRUG", "name": "Reldesemtiv"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2017-07-24", "url": "https://clinicaltrials.gov/study/NCT03160898", "target_entities": ["skeletal_muscle_function"], "locations": [{"facility": "St. Joseph's Hospital and Medical Center - Barrow Neurological Clinics", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Cedars-Sinai Medical Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California Irvine", "city": "Orange", "state": "California", "country": "United States", "status": "", "lat": 33.78779, "lon": -117.85311}, {"facility": "Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Stanford Hospital and Clinics", "city": "Stanford", "state": "California", "country": "United States", "status": "", "lat": 37.42411, "lon": -122.16608}, {"facility": "University of Colorado Hospital Anschutz Outpatient Pavilion", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "Hospital for Special Care", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "", "lat": 41.66121, "lon": -72.77954}, {"facility": "George Washington University Medical Faculty Associates", "city": "Washington D.C.", "state": "District of Columbia", "country": "United States", "status": "", "lat": 38.89511, "lon": -77.03637}, {"facility": "University of Florida", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Mayo Clinic", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "Carol & Frank Morsani Center for Advanced Healthcare - University of South Florida", "city": "Tampa", "state": "Florida", "country": "United States", "status": "", "lat": 27.94752, "lon": -82.45843}, {"facility": "Emory Clinic", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Duchossois Center for Advanced Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "IU Health Neuroscience Center of Excellence", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Iowa Hospitals and Clinics", "city": "Iowa City", "state": "Iowa", "country": "United States", "status": "", "lat": 41.66113, "lon": -91.53017}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University - Outpatient Center", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School", "city": "Worcester", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.26259, "lon": -71.80229}, {"facility": "Michigan Medicine", "city": "Ann Arbor", "state": "Michigan", "country": "United States", "status": "", "lat": 42.27756, "lon": -83.74088}, {"facility": "Henry Ford Health System", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Hennepin County Medical Center", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}, {"facility": "Saint Louis University, Department of Neurology", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Neurology Associates, P.C.", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Hospital For Special Surgery", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "Neurological Institute, Columbia University Medical Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Neurosciences Institute, Neurology - Charlotte", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke Neurological Disorders Clinic", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest School of Medicine", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "The Ohio State University Wexner Medical Center", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Providence Brain and Spine Institute ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Oregon Health & Science University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Milton S. Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Temple University School of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Vanderbilt University Medical Center - Clinical Research Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "Texas Neurology", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "Houston Methodist Hospital", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "UTHSCSA Medical Arts and Research Center", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Vermont Medical Center", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "VCU Health - Ambulatory Care Center (ACC)", "city": "Richmond", "state": "Virginia", "country": "United States", "status": "", "lat": 37.55376, "lon": -77.46026}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "West Virginia University, Dept. of Neurology", "city": "Morgantown", "state": "West Virginia", "country": "United States", "status": "", "lat": 39.62953, "lon": -79.9559}, {"facility": "Froedtert Memorial Lutheran Hospital", "city": "Milwaukee", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.0389, "lon": -87.90647}, {"facility": "Brain and Mind Centre, The University of Sydney", "city": "Camperdown", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.88965, "lon": 151.17642}, {"facility": "Department of Neurology, Westmead Hospital", "city": "Westmead", "state": "New South Wales", "country": "Australia", "status": "", "lat": -33.80383, "lon": 150.98768}, {"facility": "Royal Brisbane and Women's Hospital", "city": "Herston", "state": "Queensland", "country": "Australia", "status": "", "lat": -27.44453, "lon": 153.01852}, {"facility": "Flinders Medical Centre", "city": "Bedford Park", "state": "South Australia", "country": "Australia", "status": "", "lat": -35.02204, "lon": 138.56815}, {"facility": "The Perron Institute for Neurological and Translation Science", "city": "Nedlands", "state": "Western Australia", "country": "Australia", "status": "", "lat": -31.98184, "lon": 115.8073}, {"facility": "University of Calgary, Heritage Medical Research Center", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "Edmonton Kaye Clinic", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "McMaster University Medical Centre", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "London Health Sciences Centre University Hospital", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook Health Science Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "Centre de recherche du Centre Hospitalier de l'Universite de Montreal", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Saskatoon City Hospital", "city": "Saskatoon", "state": "Saskatchewan", "country": "Canada", "status": "", "lat": 52.13238, "lon": -106.66892}, {"facility": "CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus", "city": "Qu\u00e9bec", "state": "", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Beaumont Hospital", "city": "Dublin", "state": "", "country": "Ireland", "status": "", "lat": 53.33306, "lon": -6.24889}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Hospital San Rafael Servicio de Neurologia", "city": "Madrid", "state": "", "country": "Spain", "status": "", "lat": 40.4165, "lon": -3.70256}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Reldesemtiv (CK-2127107)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03693781", "title": "Colchicine for Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Azienda Ospedaliero-Universitaria di Modena", "summary": "The study evaluates the effects of two different Colchicine doses (0.01mg/kg/day or 0.005 mg/kg/day) compared to placebo in Amyotrophic Lateral Sclerosis (ALS) patients. Disease progression as defined by changes in ALSFRS-r is the primary outcome measure. Other measures of clinical progression and survival, together with safety and tolerability of Colchicine in ALS patients will be assessed.", "interventions": [{"type": "DRUG", "name": "Colchicine 1 MG Oral Tablet"}, {"type": "DRUG", "name": "Colchicine 1 MG Oral Tablet"}, {"type": "DRUG", "name": "Placebo Oral Tablet"}], "start_date": "2019-04-10", "url": "https://clinicaltrials.gov/study/NCT03693781", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Centro Sla, University of Bari", "city": "Bari", "state": "", "country": "Italy", "status": "", "lat": 41.12066, "lon": 16.86982}, {"facility": "Centro Sla, Istituto Auxologico Italiano, University of Milano, Milano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Irccs Carlo Besta", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Irccs St. Raffaele Institute of Milano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Centro Sla, Ospedale Civile S. Agostino Estense, A.O.U. Modena", "city": "Modena", "state": "", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "Universit\u00e0 della Campania Gianluigi Vanvitelli", "city": "Naples", "state": "", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}, {"facility": "Als Centre, \"C. Mondino\" National Neurological Institute, University of Pavia", "city": "Pavia", "state": "", "country": "Italy", "status": "", "lat": 45.19205, "lon": 9.15917}, {"facility": ", Neuromuscular Omnicentre Centre, Rome, Catholic University, Rome", "city": "Roma", "state": "", "country": "Italy", "status": "", "lat": 44.99364, "lon": 11.10642}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Colchicine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06513546", "title": "A Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of PLL001 in ALS Patients", "phase": "PHASE1, PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "PLL TX AUSTRALIA PTY LTD", "summary": "FIH, Phase 1/2, multi-centre, randomised, double-blind, placebo controlled study with an optional open-label dosing extension to assess the safety, tolerability, efficacy, and Pharmacodynamics (PD) of single or multiple (up to 48 weeks QD) subcutaneous (SC) doses of PLL001 compared to placebo in subjects diagnosed with ALS.", "interventions": [{"type": "DRUG", "name": "PLL001 or placebo daily subcutaneous injections"}], "start_date": "2026-04-08", "url": "https://clinicaltrials.gov/study/NCT06513546", "target_entities": ["neuroinflammation"], "locations": [], "contact_phone": "+61 (0)429 300 705", "contact_email": "Tina.Soulis@alithialifesciences.com", "mechanism_summary": {"compound": "PLL001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03482050", "title": "A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Kadimastem", "summary": "This is a study of transplantation of Astrocytes derived from human embryonic stem cells, in patients with Amyotrophic Lateral Sclerosis (ALS).\n\nThere will be no change in the routine ALS treatment of the patients enrolled into the study. Treatment will be administered in addition to the appropriate standard of care treatment.\n\nThe study hypothesis is that transplantation of Astrocyte(AstroRx) cells can compensate for the malfunctioning of patients' own astrocytes by restoring physiological capabilities like the reuptake of excessive glutamate, reducing oxidative stress, reducing other toxic compounds, as well as by secreting different neuroprotective factors", "interventions": [{"type": "BIOLOGICAL", "name": "AstroRx"}], "start_date": "2018-04-12", "url": "https://clinicaltrials.gov/study/NCT03482050", "target_entities": ["neurodegeneration"], "locations": [{"facility": "Hadassah Ein Kerem Medical Center", "city": "Jerusalem", "state": "", "country": "Israel", "status": "", "lat": 31.76904, "lon": 35.21633}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "AstroRx", "targeting_mechanism": "Astrocyte-derived cell transplant to restore physiological capacity of malfunctioning astrocytes in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01016522", "title": "Safety and Tolerability of the Ketogenic Diet in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "This research is being done to see if the ketogenic diet (which is high in fat and low in carbohydrates) is safe and tolerable in amyotrophic lateral sclerosis (ALS) patients who are fed through a gastrostomy tube. This is not a study to see if ketogenic diets are effective in the treatment of ALS.", "interventions": [{"type": "DIETARY_SUPPLEMENT", "name": "KetoCal"}], "start_date": "2009-11", "url": "https://clinicaltrials.gov/study/NCT01016522", "target_entities": ["metabolic_dysfunction_ketone_metabolism"], "locations": [{"facility": "Johns Hopkins ALS Clinic", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "KetoCal", "targeting_mechanism": "Unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04406675", "title": "Social Cognition in Patients With Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University Hospital, Angers", "summary": "Amyotrophic Lateral Sclerosis, also known as Charcot disease, is a neurodegenerative disease evidenced by gradual paralysis of the muscles involved in voluntary motor function. The clinical hallmark of Amyotrophic Lateral Sclerosis is the combination of upper and lower motor neuron signs and symptoms. The most recent studies suggest that up to 50% of Amyotrophic Lateral Sclerosis patients demonstrate mild to moderate cognitive disturbance. Impaired social cognition, including a deficit in the recognition of facial emotions and the identification of vocal prosody, is recognized as a part of the cognitive phenotype of Amyotrophic Lateral Sclerosis, with crucial implications for patients' and caregivers' training. However, studies remain scarce and the data acquired must be supported. The evolution of these manifestations during the disease is still poorly understood.\n\nIn this study the investigators aim to assess the social cognition capacities of patients with Amyotrophic Lateral Sclerosis compared to healthy matched control subjects.", "interventions": [{"type": "OTHER", "name": "neuropsychological test"}], "start_date": "2020-09-21", "url": "https://clinicaltrials.gov/study/NCT04406675", "target_entities": [], "locations": [{"facility": "CHU Angers", "city": "Angers", "state": "Angers", "country": "France", "status": "", "lat": 47.47156, "lon": -0.55202}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04749433", "title": "Study of [11C]CPPC to Assess the Safety and Tolerability in Patients With ALS", "phase": "PHASE1", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Johns Hopkins University", "summary": "The goal of this study is to evaluate the safety of using the \\[5-cyano-N-(4-(4-\\[11C\\]Methylpiperazin-1-yl)-2-(Piperidin-1-yl)Phenyl)Furan-2-carboxamide\\] (\\[11C\\]CPPC) radiotracer in positron emission tomography (PET) imaging of people with amyotrophic lateral sclerosis (ALS). The investigators are also interested to see whether use of this radiotracer reveals imaging differences between patients with ALS and healthy patients.", "interventions": [{"type": "DRUG", "name": "[11C]CPPC Injection"}], "start_date": "2021-09-01", "url": "https://clinicaltrials.gov/study/NCT04749433", "target_entities": ["Sigma-1 receptor"], "locations": [{"facility": "Johns Hopkins Outpatient Center", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "[11C]CPPC", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00160004", "title": "The Effect of Intensive Controlled Exercise in the Early Stages of Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Radboud University Medical Center", "summary": "Amyotrophic Lateral Sclerosis (ALS) is a progressive neurological disorder characterized by amongst others asymmetric muscle weakness, respiratory insufficiency and spasticity. The disease is usually fatal within 2-3 years and until now there is no cure. ALS patients are usually supported by a multidisciplinary team. One of the members of this team is the physical therapist. The aim of physical therapy might be to enhance or to preserve cardiovascular fitness and muscle strength. Some authors suggest, however, that a moderate to high intensive exercise programme might lead to overuse weakness (an undesired fast progression of muscle weakness). The primary objective of this study is therefore to investigate whether regular moderate to high intensity exercise program in ALS can maintain or optimize cardiorespiratory fitness and muscle strength. A secondary objective is to investigate whether such a programme leads to overuse weakness and if there is a positive influence on patient's disability, fatigue and quality of life.", "interventions": [{"type": "BEHAVIORAL", "name": "Intensive Controlled Exercise"}], "start_date": "2006-03", "url": "https://clinicaltrials.gov/study/NCT00160004", "target_entities": [], "locations": [{"facility": "Department of Allied health Occupations, Radboud University Nijmegen Medical Centre", "city": "Nijmegen", "state": "Gelderland", "country": "Netherlands", "status": "", "lat": 51.8425, "lon": 5.85278}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02463825", "title": "A Registry-Based Clinical Trial of Pimozide in Patients With Neuromuscular Junction Transmission Dysfunction Due to ALS", "phase": "PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Calgary", "summary": "Amyotrophic lateral sclerosis (ALS) is a neuromuscular disease that results in rapid decline in normal muscle function and tone leading to difficulties with mobility, eating, drinking, breathing, sleeping, and communicating. The disease is progressive and no cure currently exists. Most people diagnosed with ALS succumb within 3 to 5 years. The only approved treatment to slow the progression of ALS is called Rilutek\u00ae (riluzole) which has only a modest effect and has been shown to increase survival by a few months.\n\nMuscular dysfunction present in people with ALS is caused by nerve breakdown and a dysfunction in the communication between the muscles and the nerves. The area where these communications occur is called the neuromuscular junction. Some recent studies have focused on using different medications to enhance communication at the neuromuscular junction with the goal of improving muscle function as a result. This approach is unproven but may help to slow the progression of the disease.\n\nPimozide is a medication that has been demonstrated to enhance communication at the neuromuscular junction in fish and mice. This study will look at whether Pimozide may help to slow the progression of ALS and how much medication needs to be taken to have an effect.", "interventions": [{"type": "DRUG", "name": "Pimozide 2 mg per day"}, {"type": "DRUG", "name": "Pimozide 4 mg per day"}, {"type": "DRUG", "name": "Placebo (Lactose tablet)"}], "start_date": "2015-04", "url": "https://clinicaltrials.gov/study/NCT02463825", "target_entities": ["neuromuscular_junction_transmission"], "locations": [{"facility": "South Health Campus", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Pimozide", "targeting_mechanism": "Blocks calcium channels to stabilize neuromuscular transmission.", "targeting_mechanism_pmid": "29202456", "animal_results": "Pimozide rescues neuromuscular transmission defects in C. elegans and zebrafish ALS models and stabilizes NMJ function in mice.", "animal_results_pmid": "29202456", "repurposed_from": "antipsychotic medication", "repurposed_from_pmid": "29202456"}}
{"nct_id": "NCT01259050", "title": "Safety Study of High Doses of Zinc in ALS Patients", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Phoenix Neurological Associates, LTD", "summary": "The purpose of this study is to determine the safety of Zinc given at 90mg/d in conjunction with 2mg/d of copper in ALS patients.", "interventions": [{"type": "DRUG", "name": "Zinc and Copper"}], "start_date": "2010-10", "url": "https://clinicaltrials.gov/study/NCT01259050", "target_entities": ["zinc", "copper"], "locations": [{"facility": "Phoenix Neurological Associates", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Zinc and Copper", "targeting_mechanism": "Copper supplementation to restore copper dyshomeostasis and support SOD1 function in motor neurons.", "targeting_mechanism_pmid": "27357743", "animal_results": "Endogenous copper in the central nervous system fails to meet the elevated requirement for copper in mutant SOD1 mouse models of ALS.", "animal_results_pmid": "27357743", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03020797", "title": "A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Stony Brook University", "summary": "This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved by the FDA for treatment of seizures in patients with epilepsy. In this study, perampanel will be used off-label for adults with ALS at an oral medication dose on the low end of the recommended dose range for epilepsy. This study will consist of two treatments arms: perampanel and matching placebo randomized at a 1:1 ratio. Subjects will receive medication for 9 months.", "interventions": [{"type": "DRUG", "name": "Perampanel"}, {"type": "DRUG", "name": "Placebo Oral Tablet"}], "start_date": "2017-01-12", "url": "https://clinicaltrials.gov/study/NCT03020797", "target_entities": ["glutamate_excitotoxicity"], "locations": [{"facility": "Stony Brook University Medical Center", "city": "Stony Brook", "state": "New York", "country": "United States", "status": "", "lat": 40.92565, "lon": -73.14094}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Perampanel", "targeting_mechanism": "AMPA receptor antagonist that reduces glutamate excitotoxicity in motor neurons.", "targeting_mechanism_pmid": "29453960", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04514952", "title": "Individual Patient Expanded Access IND of Autologous HBadMSCs for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Hope Biosciences Research Foundation", "summary": "The drug for this submission is Hope Biosciences' autologous, adipose-derived culture-expanded mesenchymal stem cells (HB-adMSCs) for the treatment of a single patient with Amyotrophic Lateral Sclerosis (ALS). Stem cells have become a promising tool for the treatment of inflammatory and neurodegenerative conditions, including autoimmune diseases, traumatic brain injury, Parkinson's disease, and Alzheimer's disease.", "interventions": [{"type": "BIOLOGICAL", "name": "HB-adMSCs"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT04514952", "target_entities": ["neuroinflammation"], "locations": [{"facility": "River Oaks Hospital and Clinics", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "HB-adMSCs", "targeting_mechanism": "Autologous adipose-derived mesenchymal stem cells that modulate neuroinflammation and provide neuroprotection through paracrine effects.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03516994", "title": "Reducing Disparities in the Quality of Advance Care Planning for Older Adults", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Duke University", "summary": "This study compares the effectiveness of two different approaches to advance care planning among older African Americans and older Whites living in the community. The two approaches are a structured approach with an advance care planning conversation led by a trained person using Respecting Choices (First Steps) and a patient-driven approach which includes a Five Wishes advance care planning form written in plain language. The study will determine which approach is more effective at increasing advance care planning within each racial group and reducing differences between the two groups in advance care planning.", "interventions": [{"type": "BEHAVIORAL", "name": "Respecting Choices First Steps"}, {"type": "BEHAVIORAL", "name": "Five Wishes Form"}], "start_date": "2018-08-01", "url": "https://clinicaltrials.gov/study/NCT03516994", "target_entities": [], "locations": [{"facility": "University of Alabama at Birmingham", "city": "Birmingham", "state": "Alabama", "country": "United States", "status": "", "lat": 33.52066, "lon": -86.80249}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "University of South Carolina", "city": "Columbia", "state": "South Carolina", "country": "United States", "status": "", "lat": 34.00071, "lon": -81.03481}, {"facility": "University of Texas Southwestern", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04987671", "title": "Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Amylyx Pharmaceuticals Inc.", "summary": "The purpose of this study is to evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS.", "interventions": [{"type": "DRUG", "name": "AMX0035"}], "start_date": "2021-08-05", "url": "https://clinicaltrials.gov/study/NCT04987671", "target_entities": ["neuroinflammation", "oxidative_stress"], "locations": [{"facility": "Norman Fixel Institute for Neurological Diseases", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "AMX0035", "targeting_mechanism": "Combination agent that targets oxidative stress and neuroinflammation pathways in motor neuron degeneration.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01160263", "title": "Study of Dopamine and Serotonin Transporters in Patients With Amyotrophic Lateral Sclerosis and Controls", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "The purpose of this study is to study the transporters of serotonin and dopamine in ALS patients in relation with the clinical phenotype, i.e., patients without stiffness, patients with pyramidal stiffness, patients with mixed (pyramidal and extra pyramidal) stiffness.\n\nFor such a goal the investigators will use SPECT to compare the binding of two specific tracers in ALS patients and in matched healthy controls.", "interventions": [{"type": "DRUG", "name": "SPECT : 123 I-FP-CIT (DATSCAN) and 123I-ADAM"}], "start_date": "2012-10", "url": "https://clinicaltrials.gov/study/NCT01160263", "target_entities": ["Dopamine transporter", "Serotonin transporter"], "locations": [{"facility": "Salp\u00eatri\u00e8re Hospital", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Bretonneau Hospital", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00613899", "title": "Feasibility of Telesurveillance and Home Cough Assistance for Amyotrophic Lateral Patients (ALS)", "phase": "PHASE4", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Fondazione Salvatore Maugeri", "summary": "The investigators want to test feasibility of a structured program of telesurveillance and home cough assistance for ALS patients.", "interventions": [{"type": "OTHER", "name": "telesurveillance"}], "start_date": "2007-10", "url": "https://clinicaltrials.gov/study/NCT00613899", "target_entities": [], "locations": [{"facility": "Michele Vitacca", "city": "Lumezzane", "state": "BS", "country": "Italy", "status": "", "lat": 45.64789, "lon": 10.26487}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03679975", "title": "Riluzole Oral Soluble Film (ROSF) Swallowing Safety in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Aquestive Therapeutics", "summary": "The primary objective is to evaluate the effect, if any, of a single 50 mg dose of Riluzole Oral Soluble Film (ROSF) on swallowing safety in individuals with amyotrophic lateral sclerosis.", "interventions": [{"type": "DRUG", "name": "Riluzole Oral Soluble film (ROSF) 50 mg"}], "start_date": "2018-04-04", "url": "https://clinicaltrials.gov/study/NCT03679975", "target_entities": ["riluzole"], "locations": [{"facility": "University of Florida Center for Movement Disorders & Neuroscience", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Riluzole Oral Soluble Film (ROSF) 50 mg", "targeting_mechanism": "Riluzole is a benzothiazole derivative that blocks glutamatergic neurotransmission in the CNS by decreasing presynaptic glutamate release and increasing glutamate reuptake, exerting neuroprotective effects.", "targeting_mechanism_pmid": "32847483", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03755167", "title": "A Follow up Study to Protocol 101/2 - Continued Treatment by IPL344 IV", "phase": "PHASE2", "status": "SUSPENDED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Immunity Pharma Ltd.", "summary": "This is a prospective, open-label, follow up study to protocol 101/2 - continued treatment by IPL344 IV administered once a day in up to 15 participants with ALS.\n\nThe study is designed to determine the safety, tolerability and initial efficacy of IPL344, administered once a day, by IV infusion for up to 36 months", "interventions": [{"type": "DRUG", "name": "IPL344"}], "start_date": "2018-12-09", "url": "https://clinicaltrials.gov/study/NCT03755167", "target_entities": ["ipl344"], "locations": [{"facility": "Hadassah Medical Center/Neuromuscular / EMG service and ALS / Motor Neuron Disease Clinic", "city": "Jerusalem", "state": "", "country": "Israel", "status": "", "lat": 31.76904, "lon": 35.21633}, {"facility": "Hadassah Medical Center -Motor Neuron Disease Clinic", "city": "Jerusalem", "state": "", "country": "Israel", "status": "", "lat": 31.76904, "lon": 35.21633}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "IPL344", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07193953", "title": "Intravenous Immunoglobulin (IVIG) and Blood-Brain Barrier Disruption in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sunnybrook Health Sciences Centre", "summary": "The goal of this study is to evaluate the safety and feasibility of IVIg administration in conjunction with primary motor cortex BBB opening using the Next Generation Dome Helmet (NGDH) FUS in adult participants with ALS.", "interventions": [{"type": "DEVICE", "name": "Next Generation Dome Helmet Focused Ultrasound"}, {"type": "DRUG", "name": "Intravenous immunoglobulin (IVIG), 10% solution for infusion"}, {"type": "DRUG", "name": "Definity\u00ae Vial for (Perflutren Lipid Microsphere) Injectable Suspension"}], "start_date": "2025-04-15", "url": "https://clinicaltrials.gov/study/NCT07193953", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Sunnybrook Health Sciences Centre", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Intravenous immunoglobulin (IVIG)", "targeting_mechanism": "IVIG administration in conjunction with blood-brain barrier opening via focused ultrasound to address neuroinflammation in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06676423", "title": "Evaluate the Safety of Neuronata-R\u00ae Inj. Suspended With HypoTHermosol\u00ae FRS (HTS-FRS) in Patients With ALS", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Corestemchemon, Inc.", "summary": "This clinical trial is a single-center, open-label, and phase I clinical trial to Evaluate the Safety of Neuronata-R\u00ae Inj. suspended with HypoTHermosol\u00ae FRS (HTS-FRS) in Patients with Amyotrophic Lateral Sclerosis.", "interventions": [{"type": "BIOLOGICAL", "name": "Lenzumestrocel"}, {"type": "DRUG", "name": "Riluzole"}], "start_date": "2022-11-09", "url": "https://clinicaltrials.gov/study/NCT06676423", "target_entities": ["riluzole"], "locations": [{"facility": "Hanyang university hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Lenzumestrocel", "targeting_mechanism": "Transplantation of human neural progenitor cells secreting GDNF to provide neurotrophic support and neuroprotection to degenerating motor neurons.", "targeting_mechanism_pmid": "36064599", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05151471", "title": "Efficacy and Safety Extension Study of Oral Edaravone Administered in Subjects With ALS", "phase": "PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Shionogi", "summary": "To evaluate and compare the efficacy of two dosing regimens of oral edaravone in subjects with amyotrophic lateral sclerosis (ALS), based on the time from the randomization date in Study MT-1186-A02 to at least a 12-point decrease in Revised ALS Functional Rating Score (ALSFRS-R) or death, whichever happens first, over the course of the study or until oral edaravone is commercially available in that country", "interventions": [{"type": "DRUG", "name": "MT-1186"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2022-01-11", "url": "https://clinicaltrials.gov/study/NCT05151471", "target_entities": ["oxidative_stress"], "locations": [{"facility": "Woodland Research Northwest, LLC", "city": "Rogers", "state": "Arkansas", "country": "United States", "status": "", "lat": 36.33202, "lon": -94.11854}, {"facility": "UF Health Cancer Center/Clinical Trials Office", "city": "Gainesville", "state": "Florida", "country": "United States", "status": "", "lat": 29.65163, "lon": -82.32483}, {"facility": "Emory University - School of Medicine", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Northwestern University Feinberg School of Medicine", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Neurology Associates, P.C - Lincoln", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "University Of Pittsburgh Medical Center", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Nerve And Muscle Center Of Texas", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "University of Washington Medical Center", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}, {"facility": "West Virginia University School of Medicine (WVUSoM) - Movement Disorder Clinic", "city": "Morgantown", "state": "West Virginia", "country": "United States", "status": "", "lat": 39.62953, "lon": -79.9559}, {"facility": "University of Alberta - Walter C Mackenzie Health Sciences Centre (WCM)", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "Health Science Center Mcmaster University", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "London Health Sciences Centre - University Hospital", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "CHU de Quebec-Hopital-Enfant-Jesus", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "Universitaetsklinikum Wuerzburg", "city": "W\u00fcrzburg", "state": "", "country": "Germany", "status": "", "lat": 49.79391, "lon": 9.95121}, {"facility": "National Hospital Organization Higashinagoya National Hospital", "city": "Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "Nagoya University Hospital", "city": "Nagoya", "state": "Aichi-ken", "country": "Japan", "status": "", "lat": 35.18147, "lon": 136.90641}, {"facility": "National Hospital Organization Chibahigashi National Hospital", "city": "Chiba", "state": "Chiba", "country": "Japan", "status": "", "lat": 35.6, "lon": 140.11667}, {"facility": "Murakami Karindoh Hospital", "city": "Fukuoka", "state": "Fukuoka", "country": "Japan", "status": "", "lat": 33.6, "lon": 130.41667}, {"facility": "Fukushima Medical University Hospital", "city": "Fukushima", "state": "Fukushima", "country": "Japan", "status": "", "lat": 37.75, "lon": 140.46667}, {"facility": "Hiroshima University Hospital", "city": "Hiroshima", "state": "Hiroshima", "country": "Japan", "status": "", "lat": 34.4, "lon": 132.45}, {"facility": "National Hospital Organization Iou National Hospital", "city": "Kanazawa", "state": "Ishikawa-ken", "country": "Japan", "status": "", "lat": 36.6, "lon": 136.61667}, {"facility": "Kagawa University Hospital", "city": "Kita-gun", "state": "Kagawa-ken", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Yokohama City University Hospital", "city": "Yokohama", "state": "Kanagawa", "country": "Japan", "status": "", "lat": 35.43333, "lon": 139.65}, {"facility": "National Hospital Organization Kumamoto Saishun Medical Center", "city": "K\u014dshi", "state": "Kumamoto", "country": "Japan", "status": "", "lat": 32.89271, "lon": 130.77567}, {"facility": "National Hospital Organization Utano National Hospital", "city": "Kyoto", "state": "Kyoto", "country": "Japan", "status": "", "lat": 35.02107, "lon": 135.75385}, {"facility": "Tohoku University Hospital", "city": "Sendai", "state": "Miyagi", "country": "Japan", "status": "", "lat": 38.26667, "lon": 140.86667}, {"facility": "Niigata University Medical & Dental Hospital", "city": "Niigata", "state": "Niigata", "country": "Japan", "status": "", "lat": 37.92259, "lon": 139.04125}, {"facility": "Kansai Electric Power Hospital", "city": "Osaka", "state": "Osaka", "country": "Japan", "status": "", "lat": 34.69379, "lon": 135.50107}, {"facility": "National Hospital Organization Osaka Toneyama Medical Center", "city": "Toyonaka", "state": "Osaka", "country": "Japan", "status": "", "lat": 34.78244, "lon": 135.46932}, {"facility": "National Hospital Organization Shizuoka Institute of Epilepsy and Neurological Disorders", "city": "Shizuoka", "state": "Shizuoka", "country": "Japan", "status": "", "lat": 34.98333, "lon": 138.38333}, {"facility": "Tokyo Metropolitan Neurological Hospital", "city": "Fuch\u016b", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.67452, "lon": 139.48216}, {"facility": "Teikyo University Hospital", "city": "Itabashi-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": null, "lon": null}, {"facility": "Toho University Omori Medical Center", "city": "\u014cta-ku", "state": "Tokyo", "country": "Japan", "status": "", "lat": 35.56126, "lon": 139.71605}, {"facility": "Saitama Neuropsychiatric Institute", "city": "Saitama", "state": "", "country": "Japan", "status": "", "lat": 35.90807, "lon": 139.65657}, {"facility": "Hanyang University Medical Center", "city": "Wangsimni-ro", "state": "Seongdong-gu", "country": "South Korea", "status": "", "lat": null, "lon": null}, {"facility": "Chefarzt Muskelzentrum/ALS Clinic Kantonsspital St.Gallen Muskelzentrum/ALS Clinic", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "MT-1186 (oral edaravone)", "targeting_mechanism": "Edaravone blocks oxidative stress and free radical generation to exert neuroprotective effects in ALS.", "targeting_mechanism_pmid": "38473944", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06351592", "title": "First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Regeneron Pharmaceuticals", "summary": "This study is researching an experimental drug called ALN-SOD (called \"study drug\"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as \"SOD1-ALS\". This is the first time that ALN-SOD will be given to people.\n\nThe aim of the study is to see how safe and tolerable the study drug is.\n\nThe study is looking at several other research questions, including:\n\n* The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF)\n* How much study drug is in the blood and in the CSF, at different times\n* Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)\n* What effects the study drug has on ALS symptoms", "interventions": [{"type": "DRUG", "name": "ALN-SOD"}, {"type": "OTHER", "name": "Diluent"}, {"type": "DRUG", "name": "Placebo (PB)"}], "start_date": "2024-08-28", "url": "https://clinicaltrials.gov/study/NCT06351592", "target_entities": ["SOD1"], "locations": [{"facility": "Concord Repatriation General Hospital", "city": "Concord", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": -33.84722, "lon": 151.10381}, {"facility": "Macquarie University", "city": "Sydney", "state": "New South Wales", "country": "Australia", "status": "RECRUITING", "lat": -33.86785, "lon": 151.20732}, {"facility": "Sunshine Coast University Hospital", "city": "Birtinya", "state": "Queensland", "country": "Australia", "status": "RECRUITING", "lat": -26.74322, "lon": 153.11913}, {"facility": "KU Leuven", "city": "Leuven", "state": "Vlaams-Brabant", "country": "Belgium", "status": "RECRUITING", "lat": 50.87959, "lon": 4.70093}, {"facility": "University of Alberta Hospital, Edmonton, Division of Neurology", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "RECRUITING", "lat": 53.55014, "lon": -113.46871}, {"facility": "University Hospital - London Health Sciences Centre", "city": "London", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 42.98339, "lon": -81.23304}, {"facility": "Sunnybrook Research Institute", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "RECRUITING", "lat": 43.70643, "lon": -79.39864}, {"facility": "Montreal Neurological Institute and Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "RECRUITING", "lat": 45.50884, "lon": -73.58781}, {"facility": "Universitaetsklinikum Ulm AoR", "city": "Ulm", "state": "Baden-Wurttemberg", "country": "Germany", "status": "RECRUITING", "lat": 48.39841, "lon": 9.99155}, {"facility": "Hokkaido University Hospital", "city": "Sapporo", "state": "Hokkaido", "country": "Japan", "status": "RECRUITING", "lat": 43.06667, "lon": 141.35}, {"facility": "Tokushima University Hospital", "city": "Tokushima", "state": "Tokushima", "country": "Japan", "status": "RECRUITING", "lat": 34.06667, "lon": 134.56667}, {"facility": "Toho University Omori Medical Center", "city": "\u014cta-ku", "state": "Tokyo", "country": "Japan", "status": "RECRUITING", "lat": 35.56126, "lon": 139.71605}, {"facility": "Kyoto University Hospital", "city": "Kyoto", "state": "", "country": "Japan", "status": "RECRUITING", "lat": 35.02107, "lon": 135.75385}, {"facility": "Szpital Uniwersytecki w Krakowie, Oddzial Kliniczny Neurologii", "city": "Krakow", "state": "Lesser Poland Voivodeship", "country": "Poland", "status": "RECRUITING", "lat": 50.06143, "lon": 19.93658}, {"facility": "Medical University of Silesia", "city": "Zabrze", "state": "Silesian Voivodeship", "country": "Poland", "status": "RECRUITING", "lat": 50.32492, "lon": 18.78576}, {"facility": "MTZ Clinical Research powered by Pratia", "city": "Warsaw", "state": "", "country": "Poland", "status": "RECRUITING", "lat": 52.22977, "lon": 21.01178}, {"facility": "Hanyang University Seoul Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "RECRUITING", "lat": 37.566, "lon": 126.9784}, {"facility": "Seoul National University Hospital", "city": "Seoul", "state": "", "country": "South Korea", "status": "RECRUITING", "lat": 37.566, "lon": 126.9784}, {"facility": "Noorlands University Hospital-Department of Neurology", "city": "Ume\u00e5", "state": "Noorland", "country": "Sweden", "status": "RECRUITING", "lat": 63.82842, "lon": 20.25972}, {"facility": "Karolinska University Hospital, Karolinska Institutet-ALS Center-Department of Clinical Neuroscience", "city": "Huddinge", "state": "Stockholm County", "country": "Sweden", "status": "RECRUITING", "lat": 59.23705, "lon": 17.98192}, {"facility": "Kaohsiung Chang Gung Memorial Hospital", "city": "Kaohsiung City", "state": "", "country": "Taiwan", "status": "RECRUITING", "lat": 22.61626, "lon": 120.31333}, {"facility": "Taipei Veterans General Hospital", "city": "Taipei", "state": "", "country": "Taiwan", "status": "RECRUITING", "lat": 25.05306, "lon": 121.52639}], "contact_phone": "844-734-6643", "contact_email": "clinicaltrials@regeneron.com", "mechanism_summary": {"compound": "ALN-SOD", "targeting_mechanism": "ALN-SOD is an RNA interference agent targeting mutant SOD1 mRNA to reduce expression of pathogenic SOD1 protein in SOD1-ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05884034", "title": "Effects of a Self-care Educational Program Via Telerehabilitation in Caregivers", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Universidade Federal do Rio Grande do Norte", "summary": "The purpose of this study is to evaluate the effects of a self-care educational program via telerehabilitation on the quality of life, burden, stress, pain, and depression of caregivers of people with ALS.", "interventions": [{"type": "OTHER", "name": "Telerehabilitation"}, {"type": "OTHER", "name": "Education"}], "start_date": "2023-06", "url": "https://clinicaltrials.gov/study/NCT05884034", "target_entities": [], "locations": [{"facility": "Federal University of Rio Grande do Norte", "city": "Natal", "state": "Rio Grande do Norte", "country": "Brazil", "status": "", "lat": -5.795, "lon": -35.20944}], "contact_phone": "+55 84 8117-5502", "contact_email": "Raquel.lindquist@ufrn.br", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01142856", "title": "Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Mayo Clinic", "summary": "The purpose of this study is to determine the safety of injecting mesenchymal stem cells through intraspinal delivery for the treatment of ALS.", "interventions": [{"type": "BIOLOGICAL", "name": "autologous mesenchymal stem cells"}], "start_date": "2010-06", "url": "https://clinicaltrials.gov/study/NCT01142856", "target_entities": [], "locations": [{"facility": "Mayo Clinic", "city": "Rochester", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.02163, "lon": -92.4699}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "autologous mesenchymal stem cells", "targeting_mechanism": "Mesenchymal stem cells differentiate into support cells and produce growth factors and anti-inflammatory cytokines to provide neuroprotection and trophic support to degenerating motor neurons.", "targeting_mechanism_pmid": "32043626", "animal_results": "Preclinical studies in mouse and rat ALS models expressing mutant superoxide dismutase 1 (SOD1) demonstrated the potential benefit of mesenchymal stem cell transplantation, serving as stimulus for human trials.", "animal_results_pmid": "31189354", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05281484", "title": "Three Intermediate Expanded Access Protocols (EAP) CNMAu8.EAP01, CNMAu8.EAP02, and CNMAu8.EAP06 for ALS", "phase": "Expanded Access", "status": "AVAILABLE", "study_type": "EXPANDED_ACCESS", "is_expanded_access": true, "sponsor": "Clene Nanomedicine", "summary": "The primary objective of the intermediate expanded access protocol is to provide access to the investigational product, CNM-Au8, to up to 300 people living with ALS (pALS).\n\nNo formal clinical hypotheses are being evaluated with concurrent controls.\n\nSecondary objectives include assessment of the safety of CNM-Au8 treatment in pALS. Safety will be assessed through the frequency of serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) assessed as 'severe', discontinuations due to TEAEs, and laboratory abnormalities assessed as clinically significant during routine clinical monitoring (as applicable).", "interventions": [{"type": "DRUG", "name": "CNM-Au8"}], "start_date": "", "url": "https://clinicaltrials.gov/study/NCT05281484", "target_entities": ["cnm_au8"], "locations": [{"facility": "Barrow Neurological Institute (Enrollment is full, not recruiting)", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "AVAILABLE", "lat": 33.44838, "lon": -112.07404}, {"facility": "UC Irvine (Enrollment is full, not recruiting)", "city": "Orange", "state": "California", "country": "United States", "status": "AVAILABLE", "lat": 33.78779, "lon": -117.85311}, {"facility": "Sutter Health (Enrollment is full, not recruiting)", "city": "San Francisco", "state": "California", "country": "United States", "status": "AVAILABLE", "lat": 37.77493, "lon": -122.41942}, {"facility": "Hospital for Special Care (Enrollment is full, not recruiting)", "city": "New Britain", "state": "Connecticut", "country": "United States", "status": "AVAILABLE", "lat": 41.66121, "lon": -72.77954}, {"facility": "Nova Southeastern University (Enrollment is full, not recruiting)", "city": "Davie", "state": "Florida", "country": "United States", "status": "AVAILABLE", "lat": 26.06287, "lon": -80.2331}, {"facility": "Northwestern (Enrollment is full, not recruiting)", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "AVAILABLE", "lat": 41.85003, "lon": -87.65005}, {"facility": "University of Kansas (Enrollment is full, not recruiting)", "city": "Fairway", "state": "Kansas", "country": "United States", "status": "AVAILABLE", "lat": 39.02223, "lon": -94.6319}, {"facility": "Massachusetts General Hospital (Enrollment is full, not recruiting)", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "AVAILABLE", "lat": 42.35843, "lon": -71.05977}, {"facility": "Henry Ford Health Systems (Enrollment is full, not recruiting)", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "AVAILABLE", "lat": 42.33143, "lon": -83.04575}, {"facility": "University of Nebraska Medical Center (Enrollment is full, not recruiting)", "city": "Omaha", "state": "Nebraska", "country": "United States", "status": "AVAILABLE", "lat": 41.25626, "lon": -95.94043}, {"facility": "DUKE University Medical Center", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "AVAILABLE", "lat": 35.99403, "lon": -78.89862}, {"facility": "Providence Health (Enrollment is full, not recruiting)", "city": "Portland", "state": "Oregon", "country": "United States", "status": "AVAILABLE", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Health (Enrollment is full, not recruiting)", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "AVAILABLE", "lat": 40.28592, "lon": -76.65025}, {"facility": "Jefferson Hospital (Enrollment is full, not recruiting)", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "AVAILABLE", "lat": 39.95238, "lon": -75.16362}, {"facility": "Texas Neurology (Enrollment is full, not recruiting)", "city": "Dallas", "state": "Texas", "country": "United States", "status": "AVAILABLE", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Washington (Enrollment is full, not recruiting)", "city": "Seattle", "state": "Washington", "country": "United States", "status": "AVAILABLE", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "(801)676-9695", "contact_email": "eap@clene.com", "mechanism_summary": {"compound": "CNM-Au8", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01424176", "title": "Dexpramipexole Renal PK Study", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "This is a multicenter, open-label, single-dose, PK and safety study in subjects with various stages of renal impairment.", "interventions": [{"type": "DRUG", "name": "Dexpramipexole (dose 1)"}, {"type": "DRUG", "name": "Dexpramipexole (dose 2)"}], "start_date": "2011-07", "url": "https://clinicaltrials.gov/study/NCT01424176", "target_entities": ["dexpramipexole"], "locations": [{"facility": "Research Site", "city": "Orlando", "state": "Florida", "country": "United States", "status": "", "lat": 28.53834, "lon": -81.37924}, {"facility": "Research Site", "city": "Brooklyn Center", "state": "Minnesota", "country": "United States", "status": "", "lat": 45.07608, "lon": -93.33273}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Dexpramipexole", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00021697", "title": "Safety/Efficacy of AVP-923 in the Treatment of Emotional Lability (Uncontrolled Crying & Laughing) in Patients With ALS", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Avanir Pharmaceuticals", "summary": "The purpose of this study is to compare and evaluate the safety of AVP-923 (dextromethorphan/quinidine) for the treatment of emotional lability in ALS patients.", "interventions": [{"type": "DRUG", "name": "AVP-923"}], "start_date": "2001-01", "url": "https://clinicaltrials.gov/study/NCT00021697", "target_entities": ["dextromethorphan"], "locations": [{"facility": "Loma Linda University Dept. of Neurology", "city": "Loma Linda", "state": "California", "country": "United States", "status": "", "lat": 34.04835, "lon": -117.26115}, {"facility": "UCLA School of Medicine Dept. of Neurology", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "University of California, San Francisco", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Health Sciences", "city": "Denver", "state": "Colorado", "country": "United States", "status": "", "lat": 39.73915, "lon": -104.9847}, {"facility": "University of Miami Dept. of Neurology", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Northwestern Medical School", "city": "Chicago", "state": "Illinois", "country": "United States", "status": "", "lat": 41.85003, "lon": -87.65005}, {"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Columbia-Presbyterian Center Neurological Institute", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "State University of New York", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Medical Center Carolinas Neuromuscular/ALS-MDA Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Wake Forest University", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Cleveland Clinic Foundation", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "MCP-Hahnemann University Dept. of Neurology", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Penn Neurological Institute", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Texas Health Science Center @ San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Wisconsin ALS Clinical Research Center", "city": "Madison", "state": "Wisconsin", "country": "United States", "status": "", "lat": 43.07305, "lon": -89.40123}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "AVP-923 (dextromethorphan/quinidine)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06207591", "title": "Investigation on the Cortical Communication System", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "UMC Utrecht", "summary": "The goal of this clinical trial is to demonstrate communication through a brain implant in people in locked-in state, i.e. people with severe paralysis and communication problems.\n\nThe main questions it aims to answer are efficient and stable control of Brain-Computer interface (BCI) functions for communication with attempted hand movements and operation of a keyword-based speech BCI.\n\nParticipants will be implanted with four electrode grids, with in total 128 electrodes, on the surface of the brain and a connector on the skull. Participation includes visits of researchers for recording and training at home, 2-3 times per week for one year. Extension of participation after one year is possible.\n\nIf successful, the participant will be able to use the BCI at home independently, without the presence of a researcher.", "interventions": [{"type": "DEVICE", "name": "ECoG (electrocorticography) sensing"}], "start_date": "2023-12-15", "url": "https://clinicaltrials.gov/study/NCT06207591", "target_entities": [], "locations": [{"facility": "University Medical Center", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "RECRUITING", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "+31887555121", "contact_email": "neuroprothese@umcutrecht.nl", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06769620", "title": "A First in Human Study of ORT247 in Healthy Volunteers", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Orthogonal Neuroscience Inc.", "summary": "This is a single center, double-blinded, randomized, placebo controlled single ascending dose clinical study, with the primary purpose of evaluating the safety, tolerability, pharmacokinetics (PK), and immunohistochemistry of escalating intravenous doses of ORT247 in healthy volunteers.", "interventions": [{"type": "DRUG", "name": "ORT247"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2022-06-06", "url": "https://clinicaltrials.gov/study/NCT06769620", "target_entities": ["ort247"], "locations": [{"facility": "Worldwide Clinical Trials", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "ORT247", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00818389", "title": "Study to Investigate the Safety and Efficacy of Lithium in Volunteers With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Massachusetts General Hospital", "summary": "The purpose of this study is to compare the effectiveness of lithium combined with riluzole to riluzole combined with placebo in people with amyotrophic lateral sclerosis.", "interventions": [{"type": "DRUG", "name": "Lithium Carbonate"}, {"type": "DRUG", "name": "Riluzole"}, {"type": "DRUG", "name": "placebo"}], "start_date": "2009-01", "url": "https://clinicaltrials.gov/study/NCT00818389", "target_entities": ["lithium", "riluzole"], "locations": [{"facility": "Phoenix Neurological Assoc., 1331 N. 7th Street, Suite 350", "city": "Phoenix", "state": "Arizona", "country": "United States", "status": "", "lat": 33.44838, "lon": -112.07404}, {"facility": "Cedars-Sinai ALS Center, Neurology Specialty Clinic, 8730 Alden Drive, Thalians, E 245", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "UCSF ALS Center, University of California San Francisco, Neurology, Box 0114, UCSF", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic-Jacksonville, Neurology Department, 4500 San Pablo Road", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "University of Miami, Miller School of Medicine, 1150 NW 14th Street, Suite 609 (SCs are suite 701)", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "Indiana University, Department of Neurology, 1050 Wishard Blvd, RG 6", "city": "Indianapolis", "state": "Indiana", "country": "United States", "status": "", "lat": 39.76838, "lon": -86.15804}, {"facility": "University of Kentucky Medical Center, BAMC, Department of Neurology, Room A307, 1101 Veteran's Drive", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Johns Hopkins University, Department of Neurology, 600 N. Wolfe St, Meyer 6-181", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital, 149 13th St, Room 2266", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "Wayne State University, Department of Neurology, 4201 St. Antoine, 8C UHC", "city": "Detroit", "state": "Michigan", "country": "United States", "status": "", "lat": 42.33143, "lon": -83.04575}, {"facility": "Hennepin County Medical Center, Dept of Neurology, 701 Part Ave S, P5-200", "city": "Minneapolis", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.97997, "lon": -93.26384}, {"facility": "Washington University, 660 S. Euclid Ave., Box 8111 Neurology", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Columbia Univ Med Ctr, Eleanor and Lou Gehrig ALS/MDA Center, 710 West 168th St, 9th Floor", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University, 750 E Adams St, 6610UH", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Duke University Medical Center, Box 3333", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Wake Forest University, ALS Center, Paul Sticht Center, Ground Floor, Medical Center Blvd", "city": "Winston-Salem", "state": "North Carolina", "country": "United States", "status": "", "lat": 36.09986, "lon": -80.24422}, {"facility": "Ohio State University, Neuromuscular Division, 1654 Uphan Drive, 417 Means Hall", "city": "Columbus", "state": "Ohio", "country": "United States", "status": "", "lat": 39.96118, "lon": -82.99879}, {"facility": "Penn State Hershey Medical Center, Department of Neurology, H037, Pennsylvania State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University College of Medicine, 245 North 15th Street", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "Texas Neurology, PA, 6301 Gaston Ave, Suite 400 West Tower", "city": "Dallas", "state": "Texas", "country": "United States", "status": "", "lat": 32.78306, "lon": -96.80667}, {"facility": "University of Vermont, Department of Neurology, 89 Beaumont Drive, Given Bldg, Room C-225", "city": "Burlington", "state": "Vermont", "country": "United States", "status": "", "lat": 44.47588, "lon": -73.21207}, {"facility": "University of Virginia, Department of Neurology, 3100 Hospital Drive", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Calgary, Area 3, University of Calgary Medical Clinic, 3350 Hospital Drive NW Foothills Hosp. Grounds", "city": "Calgary", "state": "Alberta", "country": "Canada", "status": "", "lat": 51.05011, "lon": -114.08529}, {"facility": "University of Alberta, Division of Neurology, Dept of Medicine, 2E3.17 Walter C. MacKenzie Health Sciences Center", "city": "Edmonton", "state": "Alberta", "country": "Canada", "status": "", "lat": 53.55014, "lon": -113.46871}, {"facility": "University of British Columbia, GF Strong Rehab Centre, 4255 Laurel Street", "city": "Vancouver", "state": "British Columbia", "country": "Canada", "status": "", "lat": 49.24966, "lon": -123.11934}, {"facility": "University of Manitoba", "city": "Winnipeg", "state": "Manitoba", "country": "Canada", "status": "", "lat": 49.8844, "lon": -97.14704}, {"facility": "University of New Brunswick, The Stan Cassidy Centre for Rehabilitation, 800 Priestman St.", "city": "Fredericton", "state": "New Brunswick", "country": "Canada", "status": "", "lat": 45.94541, "lon": -66.66558}, {"facility": "Dalhousie University, Capital District Health Authority, Queen Elizabeth II Health Sciences Centre, P.O. Box 9000, Summer Street", "city": "Halifax", "state": "Nova Scotia", "country": "Canada", "status": "", "lat": 44.64269, "lon": -63.57688}, {"facility": "McMaster University, McMaster University Medical Centre, Hamilton Health Sciences, 1200 Main Street West, Room 4U7, Box 2000", "city": "Hamilton", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.25011, "lon": -79.84963}, {"facility": "Queen's University, The Adult Neuromuscular Clinic, PCCC, St. Mary's of the Lake Hospital Site, Department of Physical Medicine and Rehabilitation, 340 Union Street, Postal Bldg 3600", "city": "Kingston", "state": "Ontario", "country": "Canada", "status": "", "lat": 44.22976, "lon": -76.48098}, {"facility": "University of Western Ontario, Department of Clinical Neurological Sciences, Motor Neuron Disease Clinic, 339 Windermere Road, Box 5339", "city": "London", "state": "Ontario", "country": "Canada", "status": "", "lat": 42.98339, "lon": -81.23304}, {"facility": "University of Ottawa, The Rehabilitation Centre, 505 Smyth Road", "city": "Ottawa", "state": "Ontario", "country": "Canada", "status": "", "lat": 45.41117, "lon": -75.69812}, {"facility": "University of Toronto, Sunnybrook Health Sciences Centre, ALS/Neuromuscular Clinic - SCIL, Room UG-35, 2075 Bayview Ave", "city": "Toronto", "state": "Ontario", "country": "Canada", "status": "", "lat": 43.70643, "lon": -79.39864}, {"facility": "University of Montreal, CHUM (Centre Hospitalier de l'Universit\u00e9 de Montr\u00e9al) Notre-Dame Hospital 1560,Sherbrooke east street", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "McGill University, Montreal Neurological Hospital, 3801 University, Room 205", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "Laval University, CHA-Enfant-Jesus Hospital, 1401, 18th Street", "city": "Qu\u00e9bec", "state": "Quebec", "country": "Canada", "status": "", "lat": 46.81228, "lon": -71.21454}, {"facility": "University of Saskatchewan, Saskatoon City Hospital, 701 Queen Street, Room 7717 - 7th Floor", "city": "Saskatoon", "state": "Saskatchewan", "country": "Canada", "status": "", "lat": 52.13238, "lon": -106.66892}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Lithium Carbonate", "targeting_mechanism": "Lithium inhibits glycogen synthase kinase-3 (GSK-3), reducing neuroinflammation and promoting neuroprotection in ALS.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03034317", "title": "NeuRx Diaphram Pacing System (DPS) Use in Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "WITHDRAWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Synapse Biomedical", "summary": "The purpose of this research study is to collect more information about the use, safety, and effectiveness of the NeuRx DPS\u00ae in ALS patients.", "interventions": [{"type": "DEVICE", "name": "NeuRx DPS"}], "start_date": "2017-02-02", "url": "https://clinicaltrials.gov/study/NCT03034317", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "NeuRx DPS (Diaphragm Pacing System)", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06609213", "title": "Oral Intake of Enteral Nutrition Formula Preceding Placement and Feeding Via GTube and Its Impact on Formula Intolerance in pALS", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Andrea Charvet", "summary": "The main objective of the proposed study is to evaluate if oral intake of EN formula preceding Gtube placement will impact tolerance upon placement and feeding via Gtube in pALS. This single arm intervention study all participants will receive the intervention and researchers will utilize validated indicators combined with clinical expertise to assess gastrointestinal symptoms of feeding intolerance before and after the intervention.\n\nThe main questions this study aims to answer are:\n\n1. Wil participants meeting a greater percentage of their estimated nutritional needs at baseline present a slower disease progression rate and a lower incidence of GI symptoms of feeding intolerance when feeding via Gtube?\n2. Will there be significant change in feeding intolerance when oral intake of enteral nutrition formula precedes feeding via Gtube?\n\nThis proposed study consists of three stages, as follows:\n\n1. Pre-Intervention: The lead in period of one-week preceding intervention phase I will be timed to initiate 3 weeks before the scheduled Gtube placement procedure. Patients will be advised to maintain their usual food and beverage intake. Dietary intake and GI symptoms data will be collected by research personnel.\n2. Phase I: Dietary intake data collected from the pre-intervention stage will be averaged and used to determine the number of cartons of enteral nutrition formula needed to meet the participants estimated nutritional needs. For two weeks +- 2 days participants will be directed to drink the number of cartons of a pre-selected enteral nutrition formula to meet their estimated nutritional needs when combined to their current oral dietary intake. A plant based EN formula (Kate Farms 1.4 Standard) commonly prescribed for pALS was selected to be provided to all patients in the study to keep this variable constant. Weekly data collection of dietary intake and GI symptoms will be ongoing.\n3. Phase II: At the end of phase I, patients will undergo a Gtube placement at their selected medical facility. For the following two weeks +- 2 days participants will be directed to feed via Gtube the same number of cartons of the enteral nutrition formula used orally on phase I and make no changes to their current oral intake.", "interventions": [{"type": "OTHER", "name": "Medical Food"}], "start_date": "2025-01-10", "url": "https://clinicaltrials.gov/study/NCT06609213", "target_entities": [], "locations": [{"facility": "Nova Southeastern University, Cathy J, Husman ALS Center", "city": "Fort Lauderdale", "state": "Florida", "country": "United States", "status": "RECRUITING", "lat": 26.12231, "lon": -80.14338}], "contact_phone": "954-262-6901", "contact_email": "acharve1@nova.edu", "mechanism_summary": {"compound": "Medical Food", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01281631", "title": "A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Neuraltus Pharmaceuticals, Inc.", "summary": "This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS.", "interventions": [{"type": "DRUG", "name": "NP001"}, {"type": "DRUG", "name": "NP001"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2011-02", "url": "https://clinicaltrials.gov/study/NCT01281631", "target_entities": ["np001"], "locations": [{"facility": "Mayo Clinic, Scottsdale", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "UC, Irvine", "city": "Irvine", "state": "California", "country": "United States", "status": "", "lat": 33.66946, "lon": -117.82311}, {"facility": "UCLA", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "Mayo Clinic, Jacksonville", "city": "Jacksonville", "state": "Florida", "country": "United States", "status": "", "lat": 30.33218, "lon": -81.65565}, {"facility": "The Emory Clinic", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "University of Kansas Medical Center, Landon Center on Aging", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "University of Kentucky, Department of Neurology", "city": "Lexington", "state": "Kentucky", "country": "United States", "status": "", "lat": 37.98869, "lon": -84.47772}, {"facility": "Massachusetts General Hospital", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "Columbia University", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University, Syracuse", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Carolinas Medical Center", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Duke University, Dept of Neurology", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}, {"facility": "Cleveland Clinic", "city": "Cleveland", "state": "Ohio", "country": "United States", "status": "", "lat": 41.4995, "lon": -81.69541}, {"facility": "Providence ALS Center", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Methodist Hospital Research Institute, Methodist Neurologic Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}, {"facility": "Providence Saint Peter Hospital", "city": "Centralia", "state": "Washington", "country": "United States", "status": "", "lat": 46.71621, "lon": -122.9543}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "NP001", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05795907", "title": "Single & Multiple Ascending Dose Study of SAR443820 in Healthy Adult Participants", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Sanofi", "summary": "This is a Phase 1, single-center study conducted in 2 parts:\n\nPart 1a, single ascending dose (SAD-TDU16519): Double-blind, randomized, placebo-controlled sequential ascending single oral doses including up to 6 cohorts. Each cohort will include 8 participants (6 receiving SAR443820 and 2 placebo).\n\nPart 1b (TDU16519): - Open label, single SAR443820 dose in one or two separated cohort(s) for SAR443820 measurements in CSF and in plasma.\n\nPart 2, multiple ascending dose (MAD -TDR16520): Double-blind, randomized, placebo-controlled, sequential ascending repeated oral doses for 14 days, including up to 4 cohorts. Each cohort will include 10 participants (8 receiving SAR443820 and 2 placebo).", "interventions": [{"type": "DRUG", "name": "SAR443820"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2020-11-30", "url": "https://clinicaltrials.gov/study/NCT05795907", "target_entities": ["sar443820"], "locations": [{"facility": "Prism Research-Site Number:8400001", "city": "Saint Paul", "state": "Minnesota", "country": "United States", "status": "", "lat": 44.94441, "lon": -93.09327}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "SAR443820", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06363357", "title": "The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Seoul National University Hospital", "summary": "The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder.\n\nThe main questions it aims to answer are:\n\n* What is the impact of the muscle-mimicking, fabric-type shoulder orthosis on upper limb functional movements in patients with neuromuscular disorder?\n* Are there observable differences in upper limb function when the shoulder orthosis is worn versus when it is not?\n\nParticipants will:\n\n* Receive education on how to wear and use the shoulder orthosis.\n* Undergo evaluations, including assessment of upper limb performance, shoulder muscle strength testing, active range of motion measurements, assessment of functional workspace, goal attainment scale evaluation, surface electromyography, physiological measurements such as blood pressure and heart rate, fatigue assessment, and assessment for any musculoskeletal or skin-related issues.\n\nResearchers will compare neuromuscular disorder patients before and while wearing and operating the shoulder orthosis to see if there are any significant effects on variables such as upper limb function, range of motion, functional workspace, goal attainment scale, and surface electromyography.", "interventions": [{"type": "DEVICE", "name": "Shoulder orthosis"}], "start_date": "2024-04-20", "url": "https://clinicaltrials.gov/study/NCT06363357", "target_entities": [], "locations": [{"facility": "Seoul National University Hospital", "city": "Seoul", "state": "Jongno-gu", "country": "South Korea", "status": "", "lat": 37.566, "lon": 126.9784}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT04821479", "title": "Repeated Mesenchymal Stem Cell Injections in ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Hadassah Medical Organization", "summary": "An open-label, single-center clinical trial to evaluate the safety and efficacy of repeated intrathecal administrations of autologous bone marrow derived mesenchyme stem cells in ALS patients. The study includes 20 subjects (age: 20-70) with definite diagnosis of ALS and ALS-FRS-R score of at least 20 and disease-duration of less than 3 years. The treatment protocol includes four intrathecal injections of MSC, at intervals of 3 months between the injections.\n\nThe primary endpoints are safety and tolerability. Several efficacy measures are assessed as secondary endpoints.", "interventions": [{"type": "BIOLOGICAL", "name": "Mesenchymal stem cells (MSC)"}], "start_date": "2016-01-01", "url": "https://clinicaltrials.gov/study/NCT04821479", "target_entities": ["neuroinflammation"], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Mesenchymal stem cells (MSC)", "targeting_mechanism": "Bone marrow-derived mesenchymal stem cells provide trophic support and anti-inflammatory effects by differentiating into support cells and producing growth factors and anti-inflammatory cytokines.", "targeting_mechanism_pmid": "32043626", "animal_results": "Bone marrow-derived MSCs showed potential benefit in mouse and rat ALS models expressing mutant superoxide dismutase 1 through various delivery methods, cell amounts, and differentiation states.", "animal_results_pmid": "29132389", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00647296", "title": "Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "This was a 2-part study of dexpramipexole in patients with ALS.\n\nPart 1 was a randomized, placebo-controlled, multi-center study to evaluate the safety, tolerability, and clinical effects of oral administration of 3 dosage levels of dexpramipexole vs. placebo for 12 weeks.\n\nPart 2 was a randomized, double-blind, 2-arm, parallel group, extension study evaluating the safety, tolerability, and clinical effects of oral administration of 2 dosage levels of dexpramipexole for up to 72 weeks.", "interventions": [{"type": "DRUG", "name": "Placebo"}, {"type": "DRUG", "name": "Dexpramipexole 50 mg/day"}, {"type": "DRUG", "name": "Dexpramipexole 150 mg/day"}, {"type": "DRUG", "name": "Dexpramipexole 300 mg/day"}], "start_date": "2008-04-09", "url": "https://clinicaltrials.gov/study/NCT00647296", "target_entities": ["mitochondrial_function"], "locations": [{"facility": "University of Arkansas for Medical Sciences", "city": "Little Rock", "state": "Arkansas", "country": "United States", "status": "", "lat": 34.74648, "lon": -92.28959}, {"facility": "UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center", "city": "Los Angeles", "state": "California", "country": "United States", "status": "", "lat": 34.05223, "lon": -118.24368}, {"facility": "The Forbes Norris MDA/ALS Research Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "University of Colorado Health Sciences Center", "city": "Aurora", "state": "Colorado", "country": "United States", "status": "", "lat": 39.72943, "lon": -104.83192}, {"facility": "University of Miami Miller School of Medicine", "city": "Miami", "state": "Florida", "country": "United States", "status": "", "lat": 25.77427, "lon": -80.19366}, {"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}, {"facility": "Johns Hopkins University School of Medicine", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusettes General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Bryan LGH Medical Center East", "city": "Lincoln", "state": "Nebraska", "country": "United States", "status": "", "lat": 40.8, "lon": -96.66696}, {"facility": "Columbia University, Lou Gehrig MDA/ALS Research Center", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}, {"facility": "SUNY Upstate Medical University", "city": "Syracuse", "state": "New York", "country": "United States", "status": "", "lat": 43.04812, "lon": -76.14742}, {"facility": "Oregon Health Sciences University", "city": "Portland", "state": "Oregon", "country": "United States", "status": "", "lat": 45.52345, "lon": -122.67621}, {"facility": "Penn State Hershey Medical Center", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}, {"facility": "Drexel University College Of Medicine", "city": "Philadelphia", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 39.95238, "lon": -75.16362}, {"facility": "University of Pittsburgh School of Medicine", "city": "Pittsburgh", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.44062, "lon": -79.99589}, {"facility": "Vanderbilt University Medical Center", "city": "Nashville", "state": "Tennessee", "country": "United States", "status": "", "lat": 36.16589, "lon": -86.78444}, {"facility": "University of Texas Health Sciences Center of San Antonio", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "University of Utah", "city": "Salt Lake City", "state": "Utah", "country": "United States", "status": "", "lat": 40.76078, "lon": -111.89105}, {"facility": "University of Virginia Health System", "city": "Charlottesville", "state": "Virginia", "country": "United States", "status": "", "lat": 38.02931, "lon": -78.47668}, {"facility": "University of Washington", "city": "Seattle", "state": "Washington", "country": "United States", "status": "", "lat": 47.60621, "lon": -122.33207}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Dexpramipexole", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01854294", "title": "GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Genervon Biopharmaceuticals, LLC", "summary": "GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system. Neurological diseases are multisystem, multifactorial, and single target drugs are ineffective. Genervon's Master Regulators play a significant role in embryonic/fetal nervous system development and are potent disease modification drug candidates modulating many pathways including inflammation, apoptotic, and hypoxia. The study drug is an regulatory peptide with a sequence identical to one of the active sites of human Motoneuronotrophic Factor and is manufactured by solid phase synthesis. Pre-clinical research indicates it to be a neuro-protective agent in animal models of ALS, motorneuron diseases, PD, other neuro-degenerative diseases and stroke. GM604 controls and modulates over many known and significant ALS genes with positive effects interactively and dynamically through multiple pathways, and up to twenty-two biological processes, including neuro-protection, neurogenesis, neural development, neuronal signaling, neural transport, and other processes. GM6 is not a cocktail of drugs, but one master regulator peptide drug that functions through multiple pathways. Genervon hypothesized that studying the biomarkers of protein expressions of these ALS genes such as superoxide dismutase 1 (SOD1) and the protein expression of substances such as tau, neurofilament - heavy (NF-H), Cystatin C which were indications of degeneration of neuron in the CSF collected from ALS patients will provide information of the possible GM604's mechanisms of action in treating ALS. 1. This pilot trial is designed to test proof of principle, i.e. determine if a 2-week IV bolus treatment with this agent can (1) change ALS protein expression (target biomarkers and efficacy biomarkers) after treatment (2) have preliminary effects measures of ALS disease clinical progression.\n\nStudy Objectives are:\n\n1. To test the safety and tolerability of GM604 in a population of ALS patients.\n2. To test for changes in ALS biomarkers before and after treatment.\n3. To determine preliminary effects of injections of GM604 on measures of ALS disease biomarkers and clinical progression", "interventions": [{"type": "DRUG", "name": "GM604"}, {"type": "DRUG", "name": "Placebo comparator"}], "start_date": "2013-08", "url": "https://clinicaltrials.gov/study/NCT01854294", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Columbia Medical Center NY", "city": "New York", "state": "New York", "country": "United States", "status": "", "lat": 40.71427, "lon": -74.00597}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "GM604", "targeting_mechanism": "GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that modulates multiple pathways including inflammation, apoptotic, and hypoxia signaling.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06365216", "title": "ALS Phase II Study of NX210c", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Axoltis Pharma", "summary": "This study will investigate the efficacy, safety, tolerability and pharmacokinetics (PK) of multiple intravenous infusions of NX210c, at two dose levels, in patients with Amyotrophic lateral sclerosis (ALS).", "interventions": [{"type": "DRUG", "name": "NX210c"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2024-10-25", "url": "https://clinicaltrials.gov/study/NCT06365216", "target_entities": ["GLT1"], "locations": [{"facility": "Centre Hospitalier Regional Et Universitaire De Brest", "city": "Brest", "state": "France", "country": "France", "status": "", "lat": 48.39029, "lon": -4.48628}, {"facility": "Centre Hospitalier Universitaire De Caen Normandie", "city": "Caen", "state": "France", "country": "France", "status": "", "lat": 49.18585, "lon": -0.35912}, {"facility": "H\u00f4pital La Timone - APHM", "city": "Marseille", "state": "France", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "CHRU De Nancy", "city": "Nancy", "state": "France", "country": "France", "status": "", "lat": 48.68439, "lon": 6.18496}, {"facility": "Centre Hospitalier Universitaire d'Angers", "city": "Angers", "state": "", "country": "France", "status": "", "lat": 47.47156, "lon": -0.55202}, {"facility": "Centre Hospitalier Universitaire De Bordeaux", "city": "Bordeaux", "state": "", "country": "France", "status": "", "lat": 44.84124, "lon": -0.58046}, {"facility": "CHU de Lyon HCL", "city": "Bron", "state": "", "country": "France", "status": "", "lat": 45.73865, "lon": 4.91303}, {"facility": "Centre Hospitalier Universitaire de Clermont-Ferrand - H\u00f4pital Gabriel Montpied", "city": "Clermont-Ferrand", "state": "", "country": "France", "status": "", "lat": 45.77969, "lon": 3.08682}, {"facility": "Centre Hospitalier Universitaire de Lille", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Centre Hospitalier Et Universitaire de Limoges", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Centre Hospitalier Universitaire de Montpellier", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Centre Hospitalier Universitaire de Nantes - H\u00f4pital Nord Laennec", "city": "Nantes", "state": "", "country": "France", "status": "", "lat": 47.21725, "lon": -1.55336}, {"facility": "Centre Hospitalier Universitaire de Nice", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "H\u00f4pital de la Piti\u00e9 Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Centre Hospitalier Universitaire de Rennes", "city": "Rennes", "state": "", "country": "France", "status": "", "lat": 48.11109, "lon": -1.67431}, {"facility": "Centre Hospitalier Regional Universitaire de Tours", "city": "Tours", "state": "", "country": "France", "status": "", "lat": 47.39484, "lon": 0.70398}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "NX210c", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07813858", "title": "Colchicine Effect on Amyotrophic Lateral Sclerosis Patients", "phase": "PHASE2", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Azienda Ospedaliero-Universitaria di Modena", "summary": "The goal of this clinical trial is to evaluate whether low-dose colchicine can slow disease progression in patients with amyotrophic lateral sclerosis (ALS), a progressive and fatal neurodegenerative disorder affecting motor neurons.\n\nThe study is designed to answer whether patients receiving colchicine show a slower decline in functional status, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), over a 30-week double-blind treatment period compared to patients receiving placebo. Additional questions include whether colchicine has an effect on respiratory function, disability progression, quality of life, and overall survival.\n\nResearchers will compare participants receiving colchicine at a dose of 0.005 mg/kg/day with those receiving placebo, both in addition to standard-of-care therapy with riluzole, to assess potential differences in disease progression.\n\nParticipants will be randomly assigned in a 2:1 ratio to colchicine or placebo. They will take the assigned study medication for 30 weeks during a double-blind phase and then continue into a 36-week open-label extension phase, during which all participants will receive colchicine while remaining blinded to their initial treatment assignment. Throughout the study, participants will undergo regular clinical evaluations, including assessments of motor and respiratory function, functional disability, and quality of life, for a total follow-up period of up to 66 weeks. Blood samples will also be collected to investigate biological markers of neurodegeneration and inflammation.", "interventions": [{"type": "DRUG", "name": "Colchicine 0.5 MG Oral Tablet"}, {"type": "DRUG", "name": "Placebo Oral Tablet"}], "start_date": "2026-10", "url": "https://clinicaltrials.gov/study/NCT07813858", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Centro Clinico Nemo", "city": "Milan", "state": "Milano", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "CENTRO SLA, Ospedale Civile di Baggiovara, AOU Modena", "city": "Modena", "state": "Modena", "country": "Italy", "status": "", "lat": 44.64783, "lon": 10.92539}, {"facility": "CENTRO SLA, AOU Universit\u00e0 Degli Studi Della Campania Luigi Vanvitelli", "city": "Naples", "state": "Napoli", "country": "Italy", "status": "", "lat": 40.85216, "lon": 14.26811}, {"facility": "CENTRO SLA, AOU Maggiore Della Carit\u00e0", "city": "Novara", "state": "Novara", "country": "Italy", "status": "", "lat": 45.44694, "lon": 8.62118}, {"facility": "CENTRO SLA, Fondazione Istituto Neurologico Nazionale Casimiro Mondino IRCCS", "city": "Pavia", "state": "Pavia", "country": "Italy", "status": "", "lat": 45.19205, "lon": 9.15917}], "contact_phone": "059-3961640", "contact_email": "gianferrari.giulia@aou.mo.it", "mechanism_summary": {"compound": "Colchicine 0.5 MG", "targeting_mechanism": "Reduces neuroinflammation by modulating inflammatory responses in neurodegenerative disease.", "targeting_mechanism_pmid": "28872464", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05279755", "title": "A Study to Evaluate the Safety and Pharmacokinetics of Single and Multiple Doses of Prosetin in Healthy Volunteers and Participants With ALS", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "ProJenX", "summary": "The primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with ALS.", "interventions": [{"type": "DRUG", "name": "prosetin"}, {"type": "DRUG", "name": "placebo"}], "start_date": "2022-02-26", "url": "https://clinicaltrials.gov/study/NCT05279755", "target_entities": ["SOD1"], "locations": [{"facility": "Massachusetts General Hospital", "city": "Boston", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.35843, "lon": -71.05977}, {"facility": "Worldwide Clinical Trials Early Phase Services", "city": "San Antonio", "state": "Texas", "country": "United States", "status": "", "lat": 29.42412, "lon": -98.49363}, {"facility": "The Neuro - Montr\u00e9al Neurological Institute-Hospital", "city": "Montreal", "state": "Quebec", "country": "Canada", "status": "", "lat": 45.50884, "lon": -73.58781}, {"facility": "University Medical Center Utrecht", "city": "Utrecht", "state": "Utrecht", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "prosetin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00694941", "title": "A Phase II Multi-centre, Extension Study to Investigate the Long Term Safety of ONO-2506PO in Patients Diagnosed With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ono Pharmaceutical Co., Ltd.", "summary": "The objective of this study is to investigate the long term safety of ALS patients taking ONO-2506PO.", "interventions": [{"type": "DRUG", "name": "ONO-2506PO"}], "start_date": "2008-06", "url": "https://clinicaltrials.gov/study/NCT00694941", "target_entities": ["neuroinflammation"], "locations": [{"facility": "Prof. Maloteaux, UCL Saint-Luc", "city": "Brussels", "state": "", "country": "Belgium", "status": "", "lat": 50.85045, "lon": 4.34878}, {"facility": "Prof. Wim Robberecht, UZ Leuven", "city": "Leuven", "state": "", "country": "Belgium", "status": "", "lat": 50.87959, "lon": 4.70093}, {"facility": "Prof. Alain Destee, Hopital Roger Salengro - Clinique Neurologique, Neurologie A", "city": "Lille", "state": "", "country": "France", "status": "", "lat": 50.63391, "lon": 3.05512}, {"facility": "Prof. Philippe Couratier, Hopital Duruytren", "city": "Limoges", "state": "", "country": "France", "status": "", "lat": 45.83362, "lon": 1.24759}, {"facility": "Prof. Jan Pouget, Hopital de la Timone", "city": "Marseille", "state": "", "country": "France", "status": "", "lat": 43.29695, "lon": 5.38107}, {"facility": "Prof. William Camu, Hopital de Chauliac", "city": "Montpellier", "state": "", "country": "France", "status": "", "lat": 43.61093, "lon": 3.87635}, {"facility": "Prof. Claude Desnuelle, Hopital 1-Archet 1", "city": "Nice", "state": "", "country": "France", "status": "", "lat": 43.70313, "lon": 7.26608}, {"facility": "Prof. Vincent Meininger, Hopital LaPitie Salpetriere", "city": "Paris", "state": "", "country": "France", "status": "", "lat": 48.85341, "lon": 2.3488}, {"facility": "Dr. Thomas Meyer, Charite Campus Virchow, ALS Ambulanz", "city": "Berlin", "state": "", "country": "Germany", "status": "", "lat": 52.52437, "lon": 13.41053}, {"facility": "Prof. Torsten Grehl, Neurologische Ambulanz Universitatsklinik Bergmannsheil", "city": "Bochum", "state": "", "country": "Germany", "status": "", "lat": 51.48165, "lon": 7.21648}, {"facility": "Professor Dieter Heuss, Poliklinik der Universitat Erlangen-Nurnberg, Neurologische Klinik", "city": "Erlangen", "state": "", "country": "Germany", "status": "", "lat": 49.59099, "lon": 11.00783}, {"facility": "Prof. Stephan Zierz, Martin Luther Universitat Halle-Wittenberg, Klinikum der Medizinischen Fakultat, Universitatsklinik und Poliklinik fur Neurologie", "city": "Halle", "state": "", "country": "Germany", "status": "", "lat": 51.48158, "lon": 11.97947}, {"facility": "Prof. Reinhard Dengler, Medizinische Hochschule Hannover, Neurologische Klinik", "city": "Hanover", "state": "", "country": "Germany", "status": "", "lat": 52.37052, "lon": 9.73322}, {"facility": "Prof Gian Domenico Borasio, Interdisziplinares Zentrum fur Palliativmedizin", "city": "M\u00fcnchen", "state": "", "country": "Germany", "status": "", "lat": 51.60698, "lon": 13.31243}, {"facility": "Prof. Albert Ludolph, Klinik und Poliklinikfur Neurologie der Universitat Ulm-Univeritatsklinikum Ulm", "city": "Ulm", "state": "", "country": "Germany", "status": "", "lat": 48.39841, "lon": 9.99155}, {"facility": "Dr. Berthold Schrank, Deutsche Klinik fur Diagnostik, Fachbereich Neurologie", "city": "Wiesbarden", "state": "", "country": "Germany", "status": "", "lat": null, "lon": null}, {"facility": "Prof. Vincenzo Silani, Dipartimento di Neurologia e Laboratorio di Neuroscienze - Universita di Milano - IRCCS - Istituto Auxologico Italiano", "city": "Milan", "state": "", "country": "Italy", "status": "", "lat": 42.78235, "lon": 12.59836}, {"facility": "Dr. Gabriele Mora, Divisione di Neuroriabilitazione II - Fondazione Salvatore Maugeri - IRCCS", "city": "Pavia", "state": "", "country": "Italy", "status": "", "lat": 45.19205, "lon": 9.15917}, {"facility": "Prof. Adriano Chio, Dipartimento di Neuroscienze - Divisione di Neurologia II - Azienda Ospedaliera S. Giovanni Battista - Molinette", "city": "Torino", "state": "", "country": "Italy", "status": "", "lat": 44.88856, "lon": 11.99138}, {"facility": "Prof Marianne de Visser, Academic Medical Centre (AMC) Amsterdam - Dept of Neurology", "city": "Amsterdam", "state": "", "country": "Netherlands", "status": "", "lat": 52.37403, "lon": 4.88969}, {"facility": "Prof. Leonard H Van Den Berg, University Medical Center Utrecht", "city": "Utrecht", "state": "", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}, {"facility": "Dr. Markus Weber, Kantonsspital St. Gallen, Muskelzentrum/ALS Clinic", "city": "Sankt Gallen", "state": "", "country": "Switzerland", "status": "", "lat": 47.42391, "lon": 9.37477}, {"facility": "Prof. Nigel Leigh, Academic Neuroscience Centre", "city": "London", "state": "", "country": "United Kingdom", "status": "", "lat": 51.50853, "lon": -0.12574}, {"facility": "Prof. Douglas Mitchell, Royal Preston Hospital", "city": "Preston", "state": "", "country": "United Kingdom", "status": "", "lat": 53.76282, "lon": -2.70452}, {"facility": "Dr. Chris McDermott, Royal Hallamshire Hospital", "city": "Sheffield", "state": "", "country": "United Kingdom", "status": "", "lat": 53.38297, "lon": -1.4659}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "ONO-2506PO", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05358431", "title": "Stratification of Presymptomatic Amyotrophic Lateral Sclerosis: the Development of Novel Imaging Biomarkers", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "summary": "Amyotrophic lateral sclerosis (ALS) is a relentlessly progressive neurodegenerative disorder with no effective disease-modifying therapies at present. The disease is sporadic in 90 % of the ALS patients. Up to 40 % familial ALS cases and up to 25% of familial frontotemporal dementia (FTD) are caused by autosomal dominant GGGGCC hexanucleotide repeat expansions in the C9orf72 gene. The presymptomatic phase of the disease represents a unique opportunity to evaluate mechanisms of disease propagation, characterise patterns of anatomical spread, validate staging systems and appraise the comparative sensitivity profile of emerging imaging modalities. Very few spinal cord imaging studies currently exist in ALS despite their potential to characterise both the lower and upper motor neuron components of the disease. This prospective longitudinal study of asymptomatic and symptomatic c9orf72 hexanucleotide carriers will use a purpose-designed spinal and brain imaging protocol and comprehensive clinical, genetic, electrophysiological and neuropsychological profiling. Newly developed imaging techniques such as spinal cord NODDI, spinal fMRI, quantitative thoracic cord imaging will be implemented in addition to established spinal cord and brain imaging techniques.", "interventions": [{"type": "OTHER", "name": "Neuroimaging and electrophysiology"}], "start_date": "2022-07-21", "url": "https://clinicaltrials.gov/study/NCT05358431", "target_entities": ["C9orf72"], "locations": [{"facility": "ICM, GH Piti\u00e9-Salp\u00eatri\u00e8re", "city": "Paris", "state": "", "country": "France", "status": "RECRUITING", "lat": 48.85341, "lon": 2.3488}], "contact_phone": "1.42.16.24.71", "contact_email": "pierre-francois.pradat@aphp.fr", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01041222", "title": "Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Ionis Pharmaceuticals, Inc.", "summary": "This study will test the safety, tolerability and pharmacokinetics of single doses of ISIS 333611 administered into the spinal canal as 12 hour infusions.", "interventions": [{"type": "DRUG", "name": "ISIS 333611"}], "start_date": "2010-01", "url": "https://clinicaltrials.gov/study/NCT01041222", "target_entities": ["SOD1"], "locations": [{"facility": "Johns Hopkins University", "city": "Baltimore", "state": "Maryland", "country": "United States", "status": "", "lat": 39.29038, "lon": -76.61219}, {"facility": "Massachusetts General Hospital-East, Neurology Clinical Trials Unit", "city": "Charlestown", "state": "Massachusetts", "country": "United States", "status": "", "lat": 42.37787, "lon": -71.062}, {"facility": "Washington University School of Medicine", "city": "St Louis", "state": "Missouri", "country": "United States", "status": "", "lat": 38.62727, "lon": -90.19789}, {"facility": "Methodist Neurological Institute", "city": "Houston", "state": "Texas", "country": "United States", "status": "", "lat": 29.76328, "lon": -95.36327}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "ISIS 333611", "targeting_mechanism": "Antisense oligonucleotide targeting SOD1 mRNA to reduce superoxide dismutase 1 protein levels in SOD1 mutant familial ALS.", "targeting_mechanism_pmid": "29751510", "animal_results": "Artificial microRNA targeting SOD1 extends survival and delays paralysis in SOD1 mouse models of ALS.", "animal_results_pmid": "26891182", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT01511029", "title": "Study to Evaluate the QTC Interval in Healthy Volunteers Dosed With Dexpramipexole (QTC = Electrocardiogram (ECG) Interval Measured From the Onset of the QRS Complex to the End of the T Wave Corrected for Heart Rate)", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Knopp Biosciences", "summary": "To evaluate whether dexpramipexole prolongs the QTc interval when orally administered to healthy volunteers.", "interventions": [{"type": "DRUG", "name": "Dexpramipexole"}, {"type": "DRUG", "name": "Dexpramipexole"}, {"type": "DRUG", "name": "Dexpramipexole Placebo"}, {"type": "DRUG", "name": "Moxifloxacin"}], "start_date": "2012-01", "url": "https://clinicaltrials.gov/study/NCT01511029", "target_entities": ["dexpramipexole"], "locations": [{"facility": "Research Site", "city": "Overland Park", "state": "Kansas", "country": "United States", "status": "", "lat": 38.98223, "lon": -94.67079}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Dexpramipexole", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03580616", "title": "Tolerability and Efficacy of L-Serine in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Elijah W. Stommel", "summary": "The purpose of this study is to determine the tolerability of L-Serine oral doses for ALS patients and assess preliminary indications of efficacy", "interventions": [{"type": "DRUG", "name": "L-Serine"}], "start_date": "2018-10-24", "url": "https://clinicaltrials.gov/study/NCT03580616", "target_entities": ["serine_metabolism"], "locations": [{"facility": "Dartmouth-Hitchcock Medical Center", "city": "Lebanon", "state": "New Hampshire", "country": "United States", "status": "", "lat": 43.64229, "lon": -72.25176}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "L-Serine", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05297487", "title": "Evaluation of the Early Use of the Pressure Relaxer in the Respiratory Impairment of Patients With Amyotrophic Lateral Sclerosis: Multicenter Randomized Controlled Study.", "phase": "NA", "status": "NOT_YET_RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Centre Hospitalier Universitaire de la R\u00e9union", "summary": "Amyotrophic lateral sclerosis (ALS) is a rare and serious neurodegenerative disease causing degeneration of motor neurons. . It leads to a progressive paralysis of the muscles involved in voluntary motricity. In France, its incidence is 2.5/100,000 inhabitants per year.\n\nThe death of patients is mainly caused by a progressive attack of the respiratory muscles. Indeed, the thorax is no longer actively mobilized to the maximum amplitude, it will lose its flexibility. A restrictive syndrome sets in followed by alveolar hypoventilation. Bronchial congestion may be concomitant.\n\nManagement is then based on non-invasive ventilation (NIV). This step, which is difficult for patients to accept psychologically, must be delayed as much as possible. However, to date, there are no precise recommendations on preventing the appearance of this restrictive syndrome and on slowing down the deterioration of lung function in patients.\n\nThe pressure relaxer (RLX) is an instrumental aid allowing on the one hand to mobilize the thorax thanks to hyper insufflations, and on the other hand to increase the effectiveness of the cough. The use of this device in physiotherapy is part of the HAS recommendations to promote decluttering.\n\nHowever, we believe that RLX in patients with ALS, through the pulmonary alveolar recruitment it induces, could be relevant at an earlier phase, for the prevention of the decline in pulmonary functions: the restrictive syndrome, bronchial congestion and alveolar hypoventilation. So ultimately, the quality of life and survival of these patients would be improved.\n\nIt is in this context that this multicenter randomized controlled study RELAX'SLA takes place in order to evaluate the effects of the early use of the pressure relaxer on the respiratory impairment of patients with ALS.", "interventions": [{"type": "DEVICE", "name": "early use of intermittent positive pressure breathing"}], "start_date": "2025-07-01", "url": "https://clinicaltrials.gov/study/NCT05297487", "target_entities": [], "locations": [], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT06181526", "title": "Safety and Preliminary Efficacy of Aleeto in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Beijing Tiantan Hospital", "summary": "This study is a single-center, randomized, double-blind, placebo parallel-controlled, dose-escalation clinical study. The aim of this study was to evaluate the safety, tolerability, and preliminary effect of Aleeto in adult patients with ALS, and to provide an appropriate dose for the future clinical trial.", "interventions": [{"type": "DRUG", "name": "Aleeto"}, {"type": "DEVICE", "name": "intravenous injection"}, {"type": "DEVICE", "name": "intrathecal injection"}], "start_date": "2023-12-15", "url": "https://clinicaltrials.gov/study/NCT06181526", "target_entities": ["aleeto"], "locations": [{"facility": "Beijing Tiantan Hospital", "city": "Beijing", "state": "", "country": "China", "status": "", "lat": 39.9075, "lon": 116.39723}], "contact_phone": "13911666571", "contact_email": "yilong528@gmail.com", "mechanism_summary": {"compound": "Aleeto", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT02709330", "title": "ALS Reversals - Lunasin Regimen", "phase": "PHASE2", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Richard Bedlack, M.D., Ph.D.", "summary": "This is a 12-month, widely inclusive, largely virtual, single-center, open-label pilot trial utilizing a historical control group. Participants will receive a Lunasin regimen and will be asked to register for an account of PatientsLikeMe website, where after the initial in-clinic visit, they will be asked to enter specific data.", "interventions": [{"type": "DRUG", "name": "Lunasin Regimen"}, {"type": "OTHER", "name": "Historical control"}], "start_date": "2016-04", "url": "https://clinicaltrials.gov/study/NCT02709330", "target_entities": ["lunasin"], "locations": [{"facility": "Duke Medicine / Neurology", "city": "Durham", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.99403, "lon": -78.89862}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Lunasin", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07380204", "title": "Quantification of Hsp90 in the Human Brain", "phase": "NA", "status": "RECRUITING", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Universitaire Ziekenhuizen KU Leuven", "summary": "This study tests the radiolabeled molecule (\"tracer\"), \\[\u00b9\u00b9C\\]HSP990, using positron emission tomography (PET) imaging to assess whether it can be used to measure levels of Heat Shock Protein 90 (Hsp90). The protein Hsp90 plays an important role in how proteins in the brain fold into their three-dimensional structure and how this protein helps maintain cellular homeostasis. Since neurodegenerative diseases such as Alzheimer's disease (AD), Parkinson's disease (PD), and amyotrophic lateral sclerosis (ALS) are characterized by disrupted three-dimensional protein folding resulting in protein aggregation, we also aim to measure Hsp90 levels in patients with these conditions.\n\n\\[\u00b9\u00b9C\\]HSP990 is a promising tracer for this purpose and has already been extensively tested in animal models with safe and favorable results. The investigator now aims to evaluate this tracer in the human brain in healthy volunteers as well as in patients with Parkinson's disease, Alzheimer's disease and amyotrophic lateral sclerosis. The investigator expects that Hsp90 protein levels will be present at reduced concentrations in patients, possibly in different brain regions depending on the distribution of the disease-causing proteins associated with these disorders.\n\nSince the discovery of the important role of Hsp90 in neurodegenerative diseases, several candidate drugs targeting Hsp90 have been developed in recent years. The imaging method used in this study may support the development of Hsp90-targeting medications by enabling measurement of Hsp90 levels in the brain and assessment of the effects of these drugs.", "interventions": [{"type": "OTHER", "name": "[11C]HSP990 PET dosimetry"}, {"type": "OTHER", "name": "[11C]HSP990 PET test-retest"}, {"type": "OTHER", "name": "[11C]HSP990 simplified scan protocol"}], "start_date": "2024-09-04", "url": "https://clinicaltrials.gov/study/NCT07380204", "target_entities": ["HSP90"], "locations": [{"facility": "UZ Leuven", "city": "Leuven", "state": "Vlaam-Brabant", "country": "Belgium", "status": "RECRUITING", "lat": 50.87959, "lon": 4.70093}], "contact_phone": "+32 16 34 37 15", "contact_email": "koen.vanlaere@uzleuven.be", "mechanism_summary": {"compound": "[11C]HSP990", "targeting_mechanism": "A positron emission tomography (PET) imaging tracer for quantifying Heat Shock Protein 90 (Hsp90), a molecular chaperone involved in protein folding and cellular homeostasis in the brain.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05006352", "title": "A Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL343 in Participants With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Denali Therapeutics Inc.", "summary": "This is a Phase 1b, multicenter, randomized, placebo-controlled, double-blind study of 28 days, followed by an 18-month open-label extension, designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL343 in participants with amyotrophic lateral sclerosis (ALS)", "interventions": [{"type": "DRUG", "name": "DNL343"}, {"type": "DRUG", "name": "Placebo"}], "start_date": "2021-08-11", "url": "https://clinicaltrials.gov/study/NCT05006352", "target_entities": ["dnl343"], "locations": [{"facility": "HonorHealth", "city": "Scottsdale", "state": "Arizona", "country": "United States", "status": "", "lat": 33.50921, "lon": -111.89903}, {"facility": "University of California at San Diego", "city": "San Diego", "state": "California", "country": "United States", "status": "", "lat": 32.71571, "lon": -117.16472}, {"facility": "California Pacific Medical Center", "city": "San Francisco", "state": "California", "country": "United States", "status": "", "lat": 37.77493, "lon": -122.41942}, {"facility": "PPD Orlando", "city": "Orlando", "state": "Florida", "country": "United States", "status": "", "lat": 28.53834, "lon": -81.37924}, {"facility": "Emory University", "city": "Atlanta", "state": "Georgia", "country": "United States", "status": "", "lat": 33.749, "lon": -84.38798}, {"facility": "Atrium Health Neurosciences Institute", "city": "Charlotte", "state": "North Carolina", "country": "United States", "status": "", "lat": 35.22709, "lon": -80.84313}, {"facility": "Centre for Human Drug Research (CHDR)", "city": "Leiden", "state": "South Holland", "country": "Netherlands", "status": "", "lat": 52.15833, "lon": 4.49306}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "DNL343", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT00136110", "title": "Trial of Sodium Valproate in Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "UMC Utrecht", "summary": "The purpose of this study is to determine whether the use of sodium valproate is effective in slowing the disease progression in Amyotrophic Lateral Sclerosis.", "interventions": [{"type": "DRUG", "name": "Sodium Valproate"}], "start_date": "2005-04", "url": "https://clinicaltrials.gov/study/NCT00136110", "target_entities": ["HDAC"], "locations": [{"facility": "UMC Utrecht", "city": "Utrecht", "state": "Utrecht", "country": "Netherlands", "status": "", "lat": 52.09083, "lon": 5.12222}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "Sodium Valproate", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT07680361", "title": "Study of Home Use of the Jaco Robotic Manipulation Device by Individuals With Tetraplegia", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Association APPROCHE", "summary": "For all individuals deprived of active and functional motor control of the upper limbs to perform grasping tasks, the restoration of a grasping function becomes a priority.\n\nThe population concerned is mainly represented by so-called functional tetraplegic individuals.\n\nIn this context, new technologies, and more particularly robotics, appear as a solution for substitution and compensation of motor impairment of the upper limb.\n\nAssistive robotic manipulation today relies on three robot concepts that have led to commercially available products.\n\nIt is the robotic arms mounted on wheelchairs that have benefited the most from technological advances in assistive robotics over the past 15 years and from a more advanced industrial transfer than all other robotic devices. The two main ideas underlying their development are to offer, with a single robotic solution, what several technical and human aids could provide, and to allow the user to take along the \"substitute\" for their missing or impaired effector-namely the arm-into an unknown and non-configured environment.\n\nThe JACO arm, marketed by a Canadian company (KINOVA), now stands out on the market as one of the most promising arms in terms of functional contribution and ergonomics.\n\nNo published study has ever prospectively examined home uses related to this type of manipulator arm. Only an oral communication reports on this, suggesting, in 7 users, an impact in terms of functional gain and quality of life.\n\nThe idea that the use of the Jaco arm could reduce the time of professional caregiver intervention.\n\nThe present study aims to report on the potential uses of the Jaco robot at home, its appropriation in daily life, and its impact on psycho-social-family balance.", "interventions": [{"type": "DEVICE", "name": "Use of the JACO robotic arm for a period of 2 months"}], "start_date": "2022-03-30", "url": "https://clinicaltrials.gov/study/NCT07680361", "target_entities": [], "locations": [{"facility": "Centre Bouffard Vercelli", "city": "Perpignan", "state": "", "country": "France", "status": "", "lat": 42.69764, "lon": 2.89541}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "JACO robotic arm", "targeting_mechanism": "An assistive robotic manipulation device designed to restore and compensate for loss of active motor control of upper limbs in individuals with functional tetraplegia.", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03214224", "title": "Remote Pulmonary Function Testing in Amyotrophic Lateral Sclerosis (Pilot)", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Milton S. Hershey Medical Center", "summary": "The specific objective of this study is to validate the practice of remote pulmonary function testing (rPFT) conducted in the home through the use of connected mobile health devices and the Penn State Hershey ALS Telemanagement program.", "interventions": [{"type": "DEVICE", "name": "remote pulmonary function testing"}, {"type": "DEVICE", "name": "standard pulmonary function testing"}], "start_date": "2017-11-01", "url": "https://clinicaltrials.gov/study/NCT03214224", "target_entities": [], "locations": [{"facility": "Hershey Medical Center ALS Clinic", "city": "Hershey", "state": "Pennsylvania", "country": "United States", "status": "", "lat": 40.28592, "lon": -76.65025}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "unknown", "repurposed_from_pmid": ""}}
{"nct_id": "NCT03201991", "title": "ALS Study Determining Various Biomarkers and Strength Comparison After Exercise", "phase": "NA", "status": "COMPLETED", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "University of Kansas Medical Center", "summary": "The purpose of this study is to determine the muscle strength of a muscle in the thigh after 12 weeks of home exercise.", "interventions": [{"type": "OTHER", "name": "Resistance Exercise Program"}], "start_date": "2017-05-01", "url": "https://clinicaltrials.gov/study/NCT03201991", "target_entities": [], "locations": [{"facility": "University of Kansas Medical Center", "city": "Kansas City", "state": "Kansas", "country": "United States", "status": "", "lat": 39.11417, "lon": -94.62746}], "contact_phone": "", "contact_email": "", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}
{"nct_id": "NCT05716074", "title": "The Effect of Low-\u0130ntensity Combined Exercises in Patients With Early Stage ALS.", "phase": "NA", "status": "UNKNOWN", "study_type": "INTERVENTIONAL", "is_expanded_access": false, "sponsor": "Istanbul University - Cerrahpasa", "summary": "The aim of this study is to investigate the effects of low-intensity combined exercises on balance, fatigue and quality of life applied to patients with ALS.", "interventions": [{"type": "OTHER", "name": "Supervised exercise"}, {"type": "OTHER", "name": "Home exercise"}], "start_date": "2022-11-15", "url": "https://clinicaltrials.gov/study/NCT05716074", "target_entities": [], "locations": [{"facility": "Istanbul University-Cerrahpasa", "city": "Istanbul", "state": "Bakirkoy", "country": "Turkey (T\u00fcrkiye)", "status": "RECRUITING", "lat": 41.01384, "lon": 28.94966}], "contact_phone": "+905422175730", "contact_email": "etarakci@iuc.edu.tr", "mechanism_summary": {"compound": "unknown", "targeting_mechanism": "unknown", "targeting_mechanism_pmid": "", "animal_results": "unknown", "animal_results_pmid": "", "repurposed_from": "not repurposed", "repurposed_from_pmid": ""}}