{ "generated_at": "2026-07-25T22:24:04.756300+00:00", "model": "claude-opus-4-8", "thresholds": { "min_confidence": 0.55, "min_xcheck_cosine": 0.2 }, "source": { "trials": 719, "therapies": 199, "abstained": 140 }, "classifications": [ { "id": "tdp43", "name": "TDP-43 proteinopathy", "description": "Therapies targeting TDP-43 aggregation, mislocalization, or cryptic splicing (TARDBP, STMN2, UNC13A).", "primary_count": 5, "therapy_count": 7, "trial_count": 5, "therapies": [ { "name": "FB418", "modality": "Small molecule", "target": "TDP-43", "aliases": [], "mechanisms": [ { "class": "TDP-43 proteinopathy", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.488, "evidence": "Pathological aggregates of TDP-43 are implicated in Alzheimer's disease, frontotemporal dementia, and amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT05995782", "title": "A Phase 1, SAD and MAD Study to Evaluate the Safety and Tolerability of FB418", "phase": "PHASE1", "status": "RECRUITING", 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This type of ALS is known as \"SOD1-ALS\"" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2024-08-28", "sponsor": "Regeneron Pharmaceuticals", "url": "https://clinicaltrials.gov/study/NCT06351592" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "AP-101", "modality": "Biologic/Antibody", "target": "Misfolded SOD1", "aliases": [], "mechanisms": [ { "class": "SOD1", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.409, "evidence": "a new antibody-based radiopharmaceutical to detect misfolded SOD1 in amyotrophic lateral sclerosis" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2021-09-02", "sponsor": "AL-S Pharma", "url": "https://clinicaltrials.gov/study/NCT05039099" }, { "nct_id": "NCT03981536", "title": "A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "UNKNOWN", "status_group": "other", "start_date": "2019-10-10", "sponsor": "AL-S Pharma", "url": "https://clinicaltrials.gov/study/NCT03981536" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 1, "total": 2 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "ARO-SOD1", "modality": "Other", "target": "SOD1 mRNA", "aliases": [ "ARO-SOD1 Injection" ], "mechanisms": [ { "class": "SOD1", "role": "primary", "confidence": 0.97, "xcheck_cosine": 0.528, "evidence": "will also evaluate the effect of ARO-SOD1 on SOD1 cerebrospinal fluid (CSF) levels" } ], "trials": [ { "nct_id": "NCT05949294", "title": "Study of AROSOD-1 in Adult Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "WITHDRAWN", "status_group": "terminated", "start_date": "2024-01", "sponsor": "Arrowhead Pharmaceuticals", "url": "https://clinicaltrials.gov/study/NCT05949294" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.97 }, { "name": "AMT-162", "modality": "Gene therapy", "target": "SOD1", "aliases": [ "AMT-162-001" ], "mechanisms": [ { "class": "SOD1", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.479, "evidence": "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." } ], "trials": [ { "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", "status_group": "active", "start_date": "2024-08-01", "sponsor": "UniQure Biopharma B.V.", "url": "https://clinicaltrials.gov/study/NCT06100276" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Pyrimethamine", "modality": "Small molecule", "target": "SOD1 (protein expression)", "aliases": [], "mechanisms": [ { "class": "SOD1", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.457, "evidence": "Pyrimethamine significantly lowers cerebrospinal fluid Cu/Zn superoxide dismutase in amyotrophic lateral sclerosis patients with SOD1 mutations" } ], "trials": [ { "nct_id": "NCT01083667", "title": "SOD1 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"trials": [ { "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", "status_group": "completed", "start_date": "2018-09-10", "sponsor": "Biogen", "url": "https://clinicaltrials.gov/study/NCT03626012" }, { "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", "status_group": "terminated", "start_date": "2020-04-28", "sponsor": "Biogen", "url": "https://clinicaltrials.gov/study/NCT04288856" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 1, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "TPN-101 (censavudine)", "modality": "Small molecule", "target": "Reverse transcriptase (LINE-1 / retrotransposon)", "aliases": [ "TPN-101, 400 mg/day" ], "mechanisms": [ { "class": "C9orf72", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.368, "evidence": "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" } ], "trials": [ { "nct_id": "NCT04993755", "title": "A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-10-01", "sponsor": "Transposon Therapeutics, Inc.", "url": "https://clinicaltrials.gov/study/NCT04993755" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Apilimod (LAM-002A)", "modality": "Small molecule", "target": "PIKfyve kinase", "aliases": [ "Apilimod", "LAM-002A" ], "mechanisms": [ { "class": "C9orf72", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.246, "evidence": "evaluate the safety, tolerability, and biological effect of LAM-002A in adults with C9ORF72-associated ALS (C9ALS)" }, { "class": "Proteostasis / autophagy", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.21, "evidence": "evaluate the safety, tolerability, and biological effect of LAM-002A" } ], "trials": [ { "nct_id": "NCT05163886", "title": "Study of Safety, Tolerability, and Biological Activity of LAM-002A in C9ORF72-Associated Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-12-23", "sponsor": "OrphAI Therapeutics", "url": "https://clinicaltrials.gov/study/NCT05163886" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.65 }, { "name": "Metformin", "modality": "Small molecule", "target": "RAN protein translation (C9orf72 repeat expansion)", "aliases": [], "mechanisms": [ { "class": "C9orf72", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.405, "evidence": "reduces repeat-associated non-canonical start codon ... (RAN) proteins" }, { "class": "Mitochondrial dysfunction", "role": "contributing", "confidence": 0.65, "xcheck_cosine": 0.607, "evidence": "Metformin ameliorates mitochondrial damage induced by C9orf72 poly(GR) via upregulating AKT phosphorylation" } ], "trials": [ { "nct_id": "NCT04220021", "title": "Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-01-10", "sponsor": "University of Florida", "url": "https://clinicaltrials.gov/study/NCT04220021" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 } ] }, { "id": "fus", "name": "FUS", "description": "Therapies targeting FUS aggregation or nuclear mislocalization in FUS-ALS.", "primary_count": 2, "therapy_count": 2, "trial_count": 2, "therapies": [ { "name": "RAG-21", "modality": "Antisense oligonucleotide", "target": "FUS mRNA", "aliases": [], "mechanisms": [ { "class": "FUS", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.666, "evidence": "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" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2025-08", "sponsor": "Beijing Tiantan Hospital", "url": "https://clinicaltrials.gov/study/NCT07080801" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "ION363 (Jacifusen)", "modality": "Antisense oligonucleotide", "target": "FUS mRNA", "aliases": [ "ION363" ], "mechanisms": [ { "class": "FUS", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.449, "evidence": "evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS)" } ], "trials": [ { "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", "status_group": "active", "start_date": "2021-06-14", "sponsor": "Ionis Pharmaceuticals, Inc.", "url": "https://clinicaltrials.gov/study/NCT04768972" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.95 } ] }, { "id": "neuroinflammation", "name": "Neuroinflammation", "description": "Therapies modulating microglia, astrocytes, or immune/inflammatory signaling.", "primary_count": 37, "therapy_count": 49, "trial_count": 53, "therapies": [ { "name": "Masitinib", "modality": "Small molecule", "target": "Tyrosine kinases (CSF1R, c-Kit, Lyn)", "aliases": [ "AB1010", "Masitinib (3.0)", "Masitinib (4.5)", "Masitinib (6.0)", "Masitinib 4.5 mg/kg/day" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.371, "evidence": "Post-paralysis tyrosine kinase inhibition with masitinib abrogates neuroinflammation and slows disease progression in inherited amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT03127267", "title": "Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients", "phase": "PHASE3", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2021-02-02", "sponsor": "AB Science", "url": "https://clinicaltrials.gov/study/NCT03127267" }, { "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", "status_group": "recruiting", "start_date": "2026-01-01", "sponsor": "AB Science", "url": "https://clinicaltrials.gov/study/NCT07174492" }, { "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", "status_group": "completed", "start_date": "2013-04", "sponsor": "AB Science", "url": "https://clinicaltrials.gov/study/NCT02588677" } ], "trial_counts": { "recruiting": 2, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "Cromolyn sodium", "modality": "Small molecule", "target": "Mast cells / microglia", "aliases": [ "ALZT-OP1a", "ALZT-OP1a (cromolyn)", "Cromolyn Sodium (17.1 mg BID)", "Cromolyn Sodium (34.2 mg BID)", "cromoglicate", "cromolyn" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.412, "evidence": "designed to determine whether ALZT-OP1a treatment will positively impact neuro-inflammatory biomarkers and slow down or arrest functional decline" } ], "trials": [ { "nct_id": "NCT07142291", "title": "PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS", "phase": "PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-11-25", "sponsor": "PhenoNet, Inc.", "url": "https://clinicaltrials.gov/study/NCT07142291" }, { "nct_id": "NCT04428775", "title": "A Safety and Biomarker Study of ALZT-OP1a in Subjects With Mild-Moderate ALS Disease", "phase": "PHASE2", "status": "TERMINATED", "status_group": "terminated", "start_date": "2020-09-08", "sponsor": "AZTherapies, Inc.", "url": "https://clinicaltrials.gov/study/NCT04428775" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Methylprednisolone", "modality": "Small molecule", "target": "Glucocorticoid receptor", "aliases": [ "Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.277, "evidence": "evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale" } ], "trials": [ { "nct_id": "NCT07287397", "title": "Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-12-19", "sponsor": "Vector Y Therapeutics", "url": "https://clinicaltrials.gov/study/NCT07287397" }, { "nct_id": "NCT01884571", "title": "Immunosuppression in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-10", "sponsor": "Emory University", "url": "https://clinicaltrials.gov/study/NCT01884571" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Tacrolimus", "modality": "Small molecule", "target": "Calcineurin", "aliases": [ "Tacrolimus 1Mg Cap" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.282, "evidence": "multicenter, 15-month study evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale" } ], "trials": [ { "nct_id": "NCT04302870", "title": "Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2020-02-27", "sponsor": "University of Edinburgh", "url": "https://clinicaltrials.gov/study/NCT04302870" }, { "nct_id": "NCT01884571", "title": "Immunosuppression in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-10", "sponsor": "Emory University", "url": "https://clinicaltrials.gov/study/NCT01884571" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Foralumab", "modality": "Biologic/Antibody", "target": "CD3", "aliases": [ "Foralumab Nasal", "anti-CD3 antibody" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.268, "evidence": "Foralumab is being studied as a possible way to calm the inflammation and immune system activity" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2026-07-25", "sponsor": "Tiziana Life Sciences LTD", "url": "https://clinicaltrials.gov/study/NCT07688239" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "COYA 302", "modality": "Combination", "target": "Regulatory T cells (Treg) / immune modulation", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.327, "evidence": "COYA 302 is an investigational and proprietary biologic combination therapy with a dual immunomodulatory mechanism of action intended to enhance the anti-inflammatory function of" } ], "trials": [ { "nct_id": "NCT07161999", "title": "Study of COYA 302 for the Treatment of ALS", "phase": "PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-10-01", "sponsor": "Coya Therapeutics", "url": "https://clinicaltrials.gov/study/NCT07161999" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Autologous Polyclonal Regulatory T Cells (Treg)", "modality": "Cell therapy", "target": "Regulatory T cell-mediated immune modulation", "aliases": [ "Autologous Human Polyclonal Regulatory T Cells Injection (NP001 Cell Injection)" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.25, "evidence": "Extracellular Vesicles Derived From Ex Vivo Expanded Regulatory T Cells Modulate In Vitro and In Vivo Inflammation" } ], "trials": [ { "nct_id": "NCT06671236", "title": "Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases", "phase": "PHASE1", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2024-11-21", "sponsor": "Novabio Therapeutics", "url": "https://clinicaltrials.gov/study/NCT06671236" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "RAPA-501", "modality": "Cell therapy", "target": "Immune dysregulation / inflammation", "aliases": [ "RAPA-501 Autologous T stem cells", "RAPA-501 autologous hybrid TREG/Th2 cells", "RAPA-501-ALS" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.472, "evidence": "ALS progression is associated with immune dysregulation, including abnormal astrocyte activation, microglial polarization toward the pro-inflammatory M1 phenotype" } ], "trials": [ { "nct_id": "NCT04220190", "title": "RAPA-501 Therapy for ALS", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-01-02", "sponsor": "Rapa Therapeutics LLC", "url": "https://clinicaltrials.gov/study/NCT04220190" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Galunisertib + Nerandomilast Combination", "modality": "Combination", "target": "TGF-\u03b2 receptor I (ALK5) / PDE4B", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.651, "evidence": "A growing body of translational and clinical evidence implicates neuroinflammation, reactive astrocytosis, and maladaptive TGF-\u03b2 signaling as central contributors to disease prog" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2026-06-30", "sponsor": "Gipfel Life Sciences GmbH", "url": "https://clinicaltrials.gov/study/NCT07321860" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "hUC-MSC-sEV-001 (human umbilical cord MSC-derived small extracellular vesicles)", "modality": "Cell therapy", "target": "Neuroinflammatory/complement signaling", "aliases": [ "hUC-MSC-sEV-001 nasal drops" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.281, "evidence": "reduces the progress of amyotrophic lateral sclerosis and the overactivation of complement-coagulation cascade and NF-kB signaling in SOD1(G93A) mice" }, { "class": "Neurotrophic / regenerative", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.456, "evidence": "Mesenchymal stem/stromal cells (MSCs) have been reported to exert neuroprotective effects in models of injury and disease, acting primarily through release of small e" } ], "trials": [ { "nct_id": "NCT06607900", "title": "hUC-MSC-sEV-001 Nasal Drops for Neurodegenerative Diseases", "phase": "PHASE1", "status": "NOT_YET_RECRUITING", "status_group": "recruiting", "start_date": "2025-07-01", "sponsor": "Xuanwu Hospital, Beijing", "url": "https://clinicaltrials.gov/study/NCT06607900" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "SAR443820", "modality": "Small molecule", "target": "RIPK1", "aliases": [ "ACT16970", "DNL788" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.215, "evidence": "RIPK1 is a master regulator of inflammatory signaling and cell death and increased RIPK1 activity is observed in human diseases, including Alzheimer's disease (AD) and amyotrophic lateral sclerosis (ALS)" } ], "trials": [ { "nct_id": "NCT05795907", "title": "Single & Multiple Ascending Dose Study of SAR443820 in Healthy Adult Participants", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-11-30", "sponsor": "Sanofi", "url": "https://clinicaltrials.gov/study/NCT05795907" }, { "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", "status_group": "completed", "start_date": "2021-07-28", "sponsor": "Sanofi", "url": "https://clinicaltrials.gov/study/NCT05797701" }, { "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", "status_group": "completed", "start_date": "2022-02-18", "sponsor": "Sanofi", "url": "https://clinicaltrials.gov/study/NCT05797753" }, { "nct_id": "NCT05237284", "title": "Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "status_group": "terminated", "start_date": "2022-04-13", "sponsor": "Sanofi", "url": "https://clinicaltrials.gov/study/NCT05237284" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 3, "terminated": 1, "other": 0, "total": 4 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "NP001", "modality": "Small molecule", "target": "Macrophage/monocyte activation", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.316, "evidence": "This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation" } ], "trials": [ { "nct_id": "NCT01091142", "title": "Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2010-07", "sponsor": "Neuraltus Pharmaceuticals, Inc.", "url": "https://clinicaltrials.gov/study/NCT01091142" }, { "nct_id": "NCT01281631", "title": "A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-02", "sponsor": "Neuraltus Pharmaceuticals, Inc.", "url": "https://clinicaltrials.gov/study/NCT01281631" }, { "nct_id": "NCT02794857", "title": "Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-08-29", "sponsor": "Neuraltus Pharmaceuticals, Inc.", "url": "https://clinicaltrials.gov/study/NCT02794857" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 3, "terminated": 0, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "RNS60", "modality": "Other", "target": "Immune/glial modulation (charge-stabilized oxygen nanobubbles)", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.262, "evidence": "RNS60 is a novel immune-modulatory agent that has shown neuroprotective effects in amytrophic lateral sclerosis (ALS) preclinical models" } ], "trials": [ { "nct_id": "NCT02525471", "title": "A Pilot Study of RNS60 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2015-10", "sponsor": "Sabrina Paganoni, M.D.", "url": "https://clinicaltrials.gov/study/NCT02525471" }, { "nct_id": "NCT03456882", "title": "The Effect of RNS60 on ALS Biomarkers", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-05-30", "sponsor": "Mario Negri Institute for Pharmacological Research", "url": "https://clinicaltrials.gov/study/NCT03456882" }, { "nct_id": "NCT02988297", "title": "Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "WITHDRAWN", "status_group": "terminated", "start_date": "2026-10", "sponsor": "Revalesio Corporation", "url": "https://clinicaltrials.gov/study/NCT02988297" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 1, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Ibudilast", "modality": "Small molecule", "target": "Phosphodiesterase / macrophage migration inhibitory factor (MIF)", "aliases": [ "MN-166" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.545, "evidence": "Ibudilast attenuates CNS microglial activation and secretion of pro-inflammatory cytokines" } ], "trials": [ { "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", "status_group": "active", "start_date": "2020-05-28", "sponsor": "MediciNova", "url": "https://clinicaltrials.gov/study/NCT04057898" }, { "nct_id": "NCT02238626", "title": "Ibudilast (MN-166) in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2014-09", "sponsor": "MediciNova", "url": "https://clinicaltrials.gov/study/NCT02238626" }, { "nct_id": "NCT02714036", "title": "A Biomarker Study to Evaluate MN-166 in Subjects With Amyotrophic Literal Sclerosis (ALS)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-05-06", "sponsor": "MediciNova", "url": "https://clinicaltrials.gov/study/NCT02714036" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 2, "terminated": 0, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Verdiperstat", "modality": "Small molecule", "target": "Myeloperoxidase (MPO)", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.488, "evidence": "selective, brain-permeable irreversible inhibitor of the myeloperoxidase (MPO) enzyme,1 an abundant peroxidase enzyme in activated myeloid cells, including activated microglia" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.75, "xcheck_cosine": 0.3, "evidence": "MPO catalyzes the production of hypochlorous acid, a key contributor to the oxygen-dependent activity of phagocytes. In systemic and neurological disease states, MPO-induced ox" } ], "trials": [ { "nct_id": "NCT04297683", "title": "HEALEY ALS Platform Trial - Master Protocol", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-06-14", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04297683" }, { "nct_id": "NCT04436510", "title": "HEALEY ALS Platform Trial - Regimen B Verdiperstat", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-07-28", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04436510" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 1, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Thalidomide", "modality": "Small molecule", "target": "TNF-alpha / proinflammatory cytokines", "aliases": [ "Thalidomide (drug)" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.477, "evidence": "ALS tissue is characterized by inflammatory changes... include an accumulation of large numbers of activated microglia and astrocytes. Proinflammatory cytokines, such as tumor necrosis" } ], "trials": [ { "nct_id": "NCT00140452", "title": "Phase II Study Using Thalidomide for the Treatment of ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2005-02", "sponsor": "Dartmouth-Hitchcock Medical Center", "url": "https://clinicaltrials.gov/study/NCT00140452" }, { "nct_id": "NCT00231140", "title": "Pilot-Study of Thalidomide in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "TERMINATED", "status_group": "terminated", "start_date": "2005-12", "sponsor": "Charite University, Berlin, Germany", "url": "https://clinicaltrials.gov/study/NCT00231140" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 1, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "ANX005", "modality": "Biologic/Antibody", "target": "Complement C1q", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.215, "evidence": "multi-center, open-label study of intravenous (IV) ANX005 in participants with ALS" } ], "trials": [ { "nct_id": "NCT04569435", "title": "Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-01-15", "sponsor": "Annexon, Inc.", "url": "https://clinicaltrials.gov/study/NCT04569435" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Glatiramer acetate", "modality": "Peptide", "target": "Immune modulation (MHC class II / T-cell response)", "aliases": [ "40 mg glatiramer acetate" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.315, "evidence": "GA is an immunomodulating drug that has anti inflammatory and neuroprotective properties" } ], "trials": [ { "nct_id": "NCT00326625", "title": "Clinical Trial of Glatiramer Acetate in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-07-27", "sponsor": "Teva Pharmaceutical Industries, Ltd.", "url": "https://clinicaltrials.gov/study/NCT00326625" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Fecal microbiota transplantation", "modality": "Other", "target": "Gut microbiota / microbiota-immunity axis", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.215, "evidence": "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 i" } ], "trials": [ { "nct_id": "NCT03766321", "title": "Fecal Microbiota Transplantation Effect on Amyotrophic Lateral Sclerosis Patients", "phase": "NA", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-07-01", "sponsor": "Azienda Ospedaliero-Universitaria di Modena", "url": "https://clinicaltrials.gov/study/NCT03766321" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "IC14 (atibuclimab)", "modality": "Biologic/Antibody", "target": "CD14", "aliases": [ "Biologic: IC14 (monoclonal antibody against human CD14)", "anti-CD14 antibody", "atibuclimab" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.498, "evidence": "There is a growing understanding of the key role of neuroinflammation in mediating disease progression in ALS" } ], "trials": [ { "nct_id": "NCT03474263", "title": "IC14 for Rapidly Progressive Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "WITHDRAWN", "status_group": "terminated", "start_date": "2019-09-01", "sponsor": "Implicit Bioscience", "url": "https://clinicaltrials.gov/study/NCT03474263" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "Baricitinib", "modality": "Small molecule", "target": "JAK1/JAK2 (Janus kinase)", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.473, "evidence": "people ... with evidence of abnormal inflammatory signaling in cerebrospinal fluid (CSF) at baseline" } ], "trials": [ { "nct_id": "NCT05189106", "title": "Neurodegenerative Alzheimer's Disease and Amyotrophic Lateral Sclerosis (NADALS) Basket Trial", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2022-12-05", "sponsor": "Massachusetts General Hospital", "url": "https://clinicaltrials.gov/study/NCT05189106" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "CK0803", "modality": "Cell therapy", "target": "Regulatory T cells (Treg)", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.233, "evidence": "neurotropic, allogeneic, umbilical cord blood derived T regulatory (Treg) cells" } ], "trials": [ { "nct_id": "NCT05695521", "title": "Regulatory T Cells for Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2023-04-03", "sponsor": "Cellenkos, Inc.", "url": "https://clinicaltrials.gov/study/NCT05695521" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Abatacept + Aldesleukin (IL-2)", "modality": "Combination", "target": "CD80/CD86 costimulation and IL-2 receptor (Treg modulation)", "aliases": [ "Abatacept Injection [Orencia] and Proleukin (aldesleukin)" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.264, "evidence": "the reduction of regulatory T-lymphocyte (Treg) numbers and suppressive function correlates with rapid disease progression" } ], "trials": [ { "nct_id": "NCT06307301", "title": "Study in ALS With Abatacept & IL-2", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-10-28", "sponsor": "The Methodist Hospital Research Institute", "url": "https://clinicaltrials.gov/study/NCT06307301" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Autologous Treg cells + low-dose IL-2", "modality": "Cell therapy", "target": "Regulatory T lymphocytes / immune system", "aliases": [ "Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.472, "evidence": "ALS progression is associated with immune dysregulation, including abnormal astrocyte activation, microglial polarization toward the pro-inflammatory M1 phenotype" } ], "trials": [ { "nct_id": "NCT04055623", "title": "T-regulatory Cells in ALS", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2019-08-07", "sponsor": "The Methodist Hospital Research Institute", "url": "https://clinicaltrials.gov/study/NCT04055623" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "BLZ945", "modality": "Small molecule", "target": "CSF1R", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.273, "evidence": "evaluate safety, tolerability and brain microglia response in participants with Amyotrophic Lateral Sclerosis (ALS) following multiple doses of BLZ945" } ], "trials": [ { "nct_id": "NCT04066244", "title": "Study of Safety and Proof of the Mechanism of BLZ945 in ALS Patients", "phase": "PHASE2", "status": "TERMINATED", "status_group": "terminated", "start_date": "2019-12-30", "sponsor": "Novartis Pharmaceuticals", "url": "https://clinicaltrials.gov/study/NCT04066244" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "HB-adMSCs", "modality": "Cell therapy", "target": "Immunomodulation/neuroprotection", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.495, "evidence": "Stem cells have become a promising tool for the treatment of inflammatory and neurodegenerative conditions" } ], "trials": [ { "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", "status_group": "terminated", "start_date": "", "sponsor": "Hope Biosciences Research Foundation", "url": "https://clinicaltrials.gov/study/NCT04514952" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.65 }, { "name": "Basiliximab", "modality": "Biologic/Antibody", "target": "IL-2 receptor alpha (CD25)", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.277, "evidence": "evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale" } ], "trials": [ { "nct_id": "NCT01884571", "title": "Immunosuppression in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-10", "sponsor": "Emory University", "url": "https://clinicaltrials.gov/study/NCT01884571" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Prednisone", "modality": "Small molecule", "target": "Glucocorticoid receptor", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.277, "evidence": "evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale" } ], "trials": [ { "nct_id": "NCT01884571", "title": "Immunosuppression in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-10", "sponsor": "Emory University", "url": "https://clinicaltrials.gov/study/NCT01884571" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Mycophenolate mofetil", "modality": "Small molecule", "target": "Inosine monophosphate dehydrogenase (IMPDH)", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.282, "evidence": "multicenter, 15-month study evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale" } ], "trials": [ { "nct_id": "NCT01884571", "title": "Immunosuppression in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-10", "sponsor": "Emory University", "url": "https://clinicaltrials.gov/study/NCT01884571" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Withania somnifera (Ashwagandha)", "modality": "Dietary supplement", "target": "Unknown", "aliases": [ "Withania somnifera" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.202, "evidence": "WS has plausible mechanisms for slowing ALS progression because of its effects on inflammation, oxidative stress, autophagy" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.55, "xcheck_cosine": 0.445, "evidence": "because of its effects on inflammation, oxidative stress, autophagy" } ], "trials": [ { "nct_id": "NCT05031351", "title": "NF-\u03baB Inhibition in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2021-10-19", "sponsor": "Sunnybrook Health Sciences Centre", "url": "https://clinicaltrials.gov/study/NCT05031351" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Low-dose interleukin-2 (aldesleukin)", "modality": "Biologic/Antibody", "target": "IL-2 receptor / regulatory T cells", "aliases": [ "1.0 MIU IL-2 per day", "2.0 MIU IL-2 per day" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.448, "evidence": "Low-dose interleukin-2 (ld-IL-2) enhances regulatory T-cell (Treg) function in auto-inflammatory conditions. Neuroinflammation being a pathogenic feature of amyotrophic lateral sclerosis (ALS)" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2015-09", "sponsor": "Centre Hospitalier Universitaire de N\u012bmes", "url": "https://clinicaltrials.gov/study/NCT02059759" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "Intravenous immunoglobulin (IVIG)", "modality": "Biologic/Antibody", "target": "Immune system (immunomodulation)", "aliases": [ "Intravenous immunoglobulin (IVIG), 10% solution for infusion" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.267, "evidence": "evaluate the safety and feasibility of 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Riluzol in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-02", "sponsor": "Charite University, Berlin, Germany", "url": "https://clinicaltrials.gov/study/NCT01277315" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Celecoxib", "modality": "Small molecule", "target": "Cyclooxygenase-2 (COX-2)", "aliases": [ "Celebrex" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.207, "evidence": "To evaluate the efficacy of a fixed-dose combination of two approved drugs, Ciprofloxacin and Celecoxib, as a potential therapeutic treatment for amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT00355576", "title": "Combination Therapy Selection Trial in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-07", "sponsor": "Columbia University", "url": "https://clinicaltrials.gov/study/NCT00355576" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Autologous regulatory T cells (Tregs)", "modality": "Cell therapy", "target": "Immune regulation / neuroinflammation", "aliases": [ "Autologous T-regulatory lymphocytes", "CD4+CD25+ regulatory T cells", "ex vivo expanded Tregs" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.472, "evidence": "ALS progression is associated with immune dysregulation, including abnormal astrocyte activation, microglial polarization toward the pro-inflammatory M1 phenotype" } ], "trials": [ { "nct_id": "NCT03241784", "title": "Ph1 T-Regulatory Cells in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-05-16", "sponsor": "The Methodist Hospital Research Institute", "url": "https://clinicaltrials.gov/study/NCT03241784" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Low-dose Interleukin-2", "modality": "Biologic/Antibody", "target": "IL-2 receptor / regulatory T cells", "aliases": [ "IL-2", "Interleukin-2", "ld-IL2" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.487, "evidence": "Low-dose IL-2 reinvigorates the immunoguardians of neurodegenerative diseases" } ], "trials": [ { "nct_id": "NCT03241784", "title": "Ph1 T-Regulatory Cells in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-05-16", "sponsor": "The Methodist Hospital Research Institute", "url": "https://clinicaltrials.gov/study/NCT03241784" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Wharton's Jelly Mesenchymal Stem Cells (WJMSC)", "modality": "Cell therapy", "target": "Immune modulation / neuroprotection", "aliases": [ "HUMSCs", "Mesenchymal stem cells isolated from Wharton's jelly", "WJMSC", "human umbilical mesenchymal stromal cells" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.425, "evidence": "Transplantation of HUMSCs into the spinal canal of SOD1 mice could ameliorate inflammatory responses, reduce neuronal cell death" }, { "class": "Neurotrophic / regenerative", "role": "contributing", "confidence": 0.65, "xcheck_cosine": 0.637, "evidence": "reduce neuronal cell death, maintain a better mobility and effectively extend their lifespan" } ], "trials": [ { "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", "status_group": "other", "start_date": "2020-12-02", "sponsor": "Polski Bank Komorek Macierzystych JSC (PBKM)", "url": "https://clinicaltrials.gov/study/NCT04651855" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "NurOwn (MSC-NTF cells)", "modality": "Cell therapy", "target": "Neurotrophic factor secretion", "aliases": [ "MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF", "NurOwn\u00ae (MSC-NTF cells)", "Nurown MSC-NTF cells" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.489, "evidence": "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)" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.222, "evidence": "MSCs secrete growth factors and modulate immune system" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2012-12", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT01777646" }, { "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", "status_group": "completed", "start_date": "2014-05", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT02017912" }, { "nct_id": "NCT03280056", "title": "Safety and Efficacy of Repeated Administrations of NurOwn\u00ae in ALS Patients", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-08-28", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT03280056" }, { "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", "status_group": "terminated", "start_date": "", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT04681118" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 3, "terminated": 1, "other": 0, "total": 4 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Granulocyte Colony Stimulating Factor (G-CSF)", "modality": "Biologic/Antibody", "target": "G-CSF receptor", "aliases": [ "Granulocyte Colony Stimulating Factor" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.401, "evidence": "characterized by neuronal degeneration and inflammation in the nerves" } ], "trials": [ { "nct_id": "NCT00397423", "title": "G-CSF Treatment for Amyotrophic Lateral Sclerosis: A RCT Study Assessing Clinical Response", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-12", "sponsor": "Peking University", "url": "https://clinicaltrials.gov/study/NCT00397423" }, { "nct_id": "NCT01825551", "title": "The Effect of GCSF in the Treatment of ALS Patients", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-11", "sponsor": "Tehran University of Medical Sciences", "url": "https://clinicaltrials.gov/study/NCT01825551" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "PrimeC (ciprofloxacin/celecoxib)", "modality": "Combination", "target": "microRNA processing / COX-2 / iron homeostasis", "aliases": [ "Fixed dose combination Ciprofloxacin/Celecoxib" ], "mechanisms": [ { "class": "RNA metabolism", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.255, "evidence": "ALS involves multifaceted pathogenic mechanisms including microRNA dysregulation, iron accumulation, and neuroinflammation" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.328, "evidence": "microRNA dysregulation, iron accumulation, and neuroinflammation" } ], "trials": [ { "nct_id": "NCT04165850", "title": "Open Label Study to Evaluate Ciprofloxacin/Celecoxib Combination in Patients With ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-11-25", "sponsor": "NeuroSense Therapeutics Ltd.", "url": "https://clinicaltrials.gov/study/NCT04165850" }, { "nct_id": "NCT04090684", "title": "Ciprofloxacin/Celecoxib Combination in Patients With ALS", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-12-09", "sponsor": "NeuroSense Therapeutics Ltd.", "url": "https://clinicaltrials.gov/study/NCT04090684" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Mesenchymal stem cells (MSC)", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "Intravenous administration of 1 million of MSC", "Intravenous administration of 2 million of MSC", "Intravenous administration of 4 million of MSC" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.219, "evidence": "MSCs secrete growth factors and modulate immune system" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.75, "xcheck_cosine": 0.222, "evidence": "MSCs secrete growth factors and modulate immune system" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2014-07", "sponsor": "Andalusian Initiative for Advanced Therapies - Fundaci\u00f3n P\u00fablica Andaluza Progreso y Salud", "url": "https://clinicaltrials.gov/study/NCT02290886" }, { "nct_id": "NCT04821479", "title": "Repeated Mesenchymal Stem Cell Injections in ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-01-01", "sponsor": "Hadassah Medical Organization", "url": "https://clinicaltrials.gov/study/NCT04821479" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "contributing", "confidence_here": 0.75 }, { "name": "Rapamycin (Sirolimus)", "modality": "Small molecule", "target": "mTOR", "aliases": [ "Rapamycin" ], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.388, "evidence": "In preclinical studies rapamycin was found to target neuroinflammation, by expanding regulatory T cells, and affecting autophagy, two pillars of amyotrophic lateral sclerosis (ALS) pathogenesis" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.8, "xcheck_cosine": 0.391, "evidence": "rapamycin was found to target neuroinflammation, by expanding regulatory T cells" } ], "trials": [ { "nct_id": "NCT03359538", "title": "Rapamycin Treatment for ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-09-19", "sponsor": "Azienda Ospedaliero-Universitaria di Modena", "url": "https://clinicaltrials.gov/study/NCT03359538" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.8 }, { "name": "Oxaloacetate (Oxaloacetic acid)", "modality": "Small molecule", "target": "TCA cycle intermediate / cellular energy metabolism", "aliases": [ "Oxaloacetic Acid" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.419, "evidence": "Oxaloacetate treatment preserves motor function in SOD1(G93A) mice and normalizes select neuroinflammation-related parameters in the spinal cord" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.528, "evidence": "normalizes select neuroinflammation-related parameters in the spinal cord" } ], "trials": [ { "nct_id": "NCT04204889", "title": "Trial of Oxaloacetate in ALS", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-03-17", "sponsor": "Omar Jawdat", "url": "https://clinicaltrials.gov/study/NCT04204889" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Astaxanthin", "modality": "Dietary supplement", "target": "Reactive oxygen species / oxidative stress pathways", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.294, "evidence": "astaxanthin which has plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti-apoptotic effects" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.374, "evidence": "plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti-apoptotic effects" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2024-06-12", "sponsor": "Duke University", "url": "https://clinicaltrials.gov/study/NCT06429059" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Autologous Adipose-derived Mesenchymal Stromal Cells", "modality": "Cell therapy", "target": "Mesenchymal stromal cells (paracrine neurotrophic/immunomodulatory)", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.346, "evidence": "Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.425, "evidence": "Transplantation of HUMSCs into the spinal canal of SOD1 mice could ameliorate inflammatory responses, reduce neuronal cell death" } ], "trials": [ { "nct_id": "NCT03268603", "title": "Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2017-10-10", "sponsor": "Mayo Clinic", "url": "https://clinicaltrials.gov/study/NCT03268603" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "MSC-NTF cells (NurOwn), intramuscular", "modality": "Cell therapy", "target": "Neurotrophic factor secretion (GDNF, VEGF, etc.)", "aliases": [ "MSC-NTF cells transplantation (IM)", "NurOwn", "autologous MSC-NTF cells IM" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.451, "evidence": "autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF)" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.222, "evidence": "MSCs secrete growth factors and modulate immune system" } ], "trials": [ { "nct_id": "NCT01051882", "title": "Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients.", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-06", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT01051882" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "MSC-NTF cells (NurOwn), intrathecal", "modality": "Cell therapy", "target": "Neurotrophic factor secretion (GDNF, VEGF, etc.)", "aliases": [ "MSC-NTF cells transplantation (IT)", "NurOwn", "autologous MSC-NTF cells IT" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.451, "evidence": "autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF)" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.222, "evidence": "MSCs secrete growth factors and modulate immune system" } ], "trials": [ { "nct_id": "NCT01051882", "title": "Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients.", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-06", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT01051882" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Liposomal resveratrol and curcumin", "modality": "Dietary supplement", "target": "Oxidative stress / inflammatory pathways", "aliases": [ "Liposomed polyphenols resveratrol and curcumin" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.273, "evidence": "it is characterised by loss of oxidative control, excitotoxicity due to excess glutamate and intestinal dysbiosis" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.302, "evidence": "Amyotrophic lateral sclerosis (ALS) is a disease of an inflammatory nature" } ], "trials": [ { "nct_id": "NCT04654689", "title": "Impact of the Combined Treatment of Liposomed Polyphenols With G04CB02 on the ALS Patients", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-11-20", "sponsor": "Fundaci\u00f3n Universidad Cat\u00f3lica de Valencia San Vicente M\u00e1rtir", "url": "https://clinicaltrials.gov/study/NCT04654689" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Granulocyte-Colony Stimulating Factor (G-CSF / Filgrastim)", "modality": "Biologic/Antibody", "target": "G-CSF receptor", "aliases": [ "granulocyte - colony stimulating factor (G-CSF)" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.255, "evidence": "we decided to use G-CSF to mobilise" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.333, "evidence": "ALS) is a neurodegenerative disorder characterized by neuronal degeneration and inflammation in the nerves. G-CSF is a 19.6-kDa hema" } ], "trials": [ { "nct_id": "NCT00298597", "title": "Influence of G-CSF and EPO on Associative Learning and Motor Skills", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-03", "sponsor": "University Hospital Muenster", "url": "https://clinicaltrials.gov/study/NCT00298597" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 } ] }, { "id": "oxidative_stress", "name": "Oxidative stress", "description": "Antioxidants and therapies countering reactive oxygen species and free-radical damage (e.g., edaravone).", "primary_count": 18, "therapy_count": 27, "trial_count": 39, "therapies": [ { "name": "Antioxidants", "modality": "Dietary supplement", "target": "Reactive oxygen species", "aliases": [ "EH301" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.694, "evidence": "the effectiveness of antioxidants can be highlighted, since the disease is directly related to oxidative stress" } ], "trials": [ { "nct_id": "NCT04244630", "title": "Mitochondrial Capacity Boost in ALS (MICABO-ALS) Trial", "phase": "PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2023-11-02", "sponsor": "Dallas VA Medical Center", "url": "https://clinicaltrials.gov/study/NCT04244630" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "N-Acetylcysteine", "modality": "Dietary supplement", "target": "Glutathione precursor / reactive oxygen species", "aliases": [ "Acetylcysteine", "NAC" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.258, "evidence": "a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2025-11-25", "sponsor": "Biogipuzkoa Health Research Institute", "url": "https://clinicaltrials.gov/study/NCT07414212" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Edaravone", "modality": "Small molecule", "target": "Reactive oxygen species (free radical scavenger)", "aliases": [ "FAB122", "MCI-186", "MCI-186 in open label phase", "MT-1186", "Radicava", "Radicut", "edaravone oral suspension", "oral edaravone" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 1.0, "xcheck_cosine": 0.583, "evidence": "Edaravone is a free radical scavenger that slows down functional decline and prevents from disease progression in ALS patients" } ], "trials": [ { "nct_id": "NCT00330681", "title": "Efficacy and Safety Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-05-31", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT00330681" }, { "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", "status_group": "completed", "start_date": "2006-12-31", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT00415519" }, { "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", "status_group": "completed", "start_date": "2007-01-31", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT00424463" }, { "nct_id": "NCT01492686", "title": "Phase 3 Study of MCI-186 for Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-12-31", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT01492686" }, { "nct_id": "NCT04176224", "title": "Clinical Pharmacology Study of Oral Edaravone in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-04-17", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT04176224" }, { "nct_id": "NCT04254913", "title": "Clinical Pharmacology Study of Oral Edaravone in Amyotrophic Lateral Sclerosis Patients With Gastrostomy", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-01-24", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT04254913" }, { "nct_id": "NCT04577404", "title": "Safety Extension Study of Oral Edaravone Administered in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-10-29", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT04577404" }, { "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", "status_group": "completed", "start_date": "2021-10-18", "sponsor": "Ferrer Internacional S.A.", "url": "https://clinicaltrials.gov/study/NCT05178810" }, { "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", "status_group": "completed", "start_date": "2022-10-26", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT05568615" }, { "nct_id": "NCT04569084", "title": "Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS", "phase": "PHASE3", "status": "TERMINATED", "status_group": "terminated", "start_date": "2020-11-13", "sponsor": "Shionogi", "url": "https://clinicaltrials.gov/study/NCT04569084" }, { "nct_id": "NCT05151471", "title": "Efficacy and Safety Extension Study of Oral Edaravone Administered in Subjects With ALS", "phase": "PHASE3", "status": "TERMINATED", "status_group": "terminated", "start_date": "2022-01-11", "sponsor": "Tanabe Pharma America, Inc.", "url": "https://clinicaltrials.gov/study/NCT05151471" }, { "nct_id": "NCT05866926", "title": "Study to Investigate the Long-term Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "TERMINATED", "status_group": "terminated", "start_date": "2023-03-06", "sponsor": "Ferrer Internacional S.A.", "url": "https://clinicaltrials.gov/study/NCT05866926" }, { "nct_id": "NCT03272802", "title": "Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2017-03-16", "sponsor": "Isfahan University of Medical Sciences", "url": "https://clinicaltrials.gov/study/NCT03272802" }, { "nct_id": "NCT04391361", "title": "The Safety and Effectiveness of Cholinergic Receptor Block Therapy in the Treatment of ALS", "phase": "PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2020-11-01", "sponsor": "Ruijin Hospital", "url": "https://clinicaltrials.gov/study/NCT04391361" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 9, "terminated": 3, "other": 2, "total": 14 }, "role_here": "primary", "confidence_here": 1.0 }, { "name": "CuATSM", "modality": "Small molecule", "target": "Copper delivery / ferroptosis (lipid peroxidation)", "aliases": [ "CMD-2016-001", "CMD-2019-001", "Cu(II)(atsm)", "Cu(II)ATSM", "Diacetyl-bis(4-methyl-3-thiosemicarbazonato)copper(II)" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.253, "evidence": "Cu(II) (atsm) inhibits ferroptosis: Implications for treatment of neurodegenerative disease" } ], "trials": [ { "nct_id": "NCT02870634", "title": "Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-11-16", "sponsor": "Collaborative Medicinal Development Pty Limited", "url": "https://clinicaltrials.gov/study/NCT02870634" }, { "nct_id": "NCT04313166", "title": "Treatment Continuation Study for Patients With ALS/MND Who Completed Study CMD-2019-001", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-03-19", "sponsor": "Collaborative Medicinal Development Pty Limited", "url": "https://clinicaltrials.gov/study/NCT04313166" }, { "nct_id": "NCT03136809", "title": "ALS Treatment Extension Study", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2018-01-18", "sponsor": "Collaborative Medicinal Development Pty Limited", "url": "https://clinicaltrials.gov/study/NCT03136809" }, { "nct_id": "NCT04082832", "title": "CuATSM Compared With Placebo for Treatment of ALS/MND", "phase": "PHASE2, PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2019-09-30", "sponsor": "Collaborative Medicinal Development Pty Limited", "url": "https://clinicaltrials.gov/study/NCT04082832" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 2, "total": 4 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Deferiprone", "modality": "Small molecule", "target": "Iron (chelation)", "aliases": [ "FAIR-ALS" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.25, "evidence": "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" } ], "trials": [ { "nct_id": "NCT02880033", "title": "Oxidative Stress and Apoptosis of Energy Metabolism by Deferiprone From the Circulating Lymphocytes", "phase": "NA", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-02", "sponsor": "University Hospital, Lille", "url": "https://clinicaltrials.gov/study/NCT02880033" }, { "nct_id": "NCT02164253", "title": "Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-09", "sponsor": "University Hospital, Lille", "url": "https://clinicaltrials.gov/study/NCT02164253" }, { "nct_id": "NCT03293069", "title": "Conservative Iron Chelation as a Disease-modifying Strategy in Amyotrophic Lateral Sclerosis", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-01-30", "sponsor": "University Hospital, Lille", "url": "https://clinicaltrials.gov/study/NCT03293069" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 3, "terminated": 0, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Tauroursodeoxycholic acid", "modality": "Small molecule", "target": "Bile acid receptor / anti-apoptotic pathways", "aliases": [ "TUDCA", "tauroursodeoxycholic acid (TUDCA)" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.386, "evidence": "the demonstration of antioxidant, antiapoptotic and neuroprotective properties of TUDCA in the central nervous system" }, { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.233, "evidence": "hydrophilic bile acids, particularly tauroursodeoxycholic acid (TUDCA), have shown important anti-apoptotic and neuroprotective activities" } ], "trials": [ { "nct_id": "NCT00877604", "title": "Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2008-06", "sponsor": "Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta", "url": "https://clinicaltrials.gov/study/NCT00877604" }, { "nct_id": "NCT03800524", "title": "Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS", "phase": "PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2019-02-22", "sponsor": "Humanitas Mirasole SpA", "url": "https://clinicaltrials.gov/study/NCT03800524" }, { "nct_id": "NCT05753852", "title": "Open Label Extension of TUDCA-ALS Study", "phase": "PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2021-10-25", "sponsor": "Humanitas Mirasole SpA", "url": "https://clinicaltrials.gov/study/NCT05753852" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 2, "total": 3 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Inosine", "modality": "Small molecule", "target": "Urate (uric acid) elevation / antioxidant", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.483, "evidence": "the measurement of biomarkers of oxidative stress and damage in response to inosine treatment" } ], "trials": [ { "nct_id": "NCT02288091", "title": "A Pilot Study of Inosine in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2015-01", "sponsor": "Massachusetts General Hospital", "url": "https://clinicaltrials.gov/study/NCT02288091" }, { "nct_id": "NCT03168711", "title": "Safety of Urate Elevation in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-10-01", "sponsor": "Massachusetts General Hospital", "url": "https://clinicaltrials.gov/study/NCT03168711" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Tocotrienols", "modality": "Dietary supplement", "target": "Reactive oxygen species / lipid peroxidation", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.444, "evidence": "Oxidative stress, probably interacting with other neurodegenerative processes, is hypothesized to play a leading role in pathogenesis" } ], "trials": [ { "nct_id": "NCT04140136", "title": "The Efficacy and Safety of Vitamin E Mixed Tocotrienols In Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2019-06-17", "sponsor": "University of Malaya", "url": "https://clinicaltrials.gov/study/NCT04140136" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Astaxanthin", "modality": "Dietary supplement", "target": "Reactive oxygen species / oxidative stress pathways", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.294, "evidence": "astaxanthin which has plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti-apoptotic effects" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.374, "evidence": "plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti-apoptotic effects" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2024-06-12", "sponsor": "Duke University", "url": "https://clinicaltrials.gov/study/NCT06429059" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Protandim", "modality": "Dietary supplement", "target": "Nrf2 pathway / antioxidant response", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.344, "evidence": "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" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2024-06-12", "sponsor": "Duke University", "url": "https://clinicaltrials.gov/study/NCT06429059" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Melatonin", "modality": "Dietary supplement", "target": "Reactive oxygen species / autophagy pathways", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.231, "evidence": "Here we review melatonin. We show that it has plausible mechanisms" }, { "class": "Proteostasis / autophagy", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.42, "evidence": "Melatonin and Autophagy in Aging-Related Neurodegenerative Diseases" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2024-06-12", "sponsor": "Duke University", "url": "https://clinicaltrials.gov/study/NCT06429059" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "RT001 (deuterated linoleic acid ethyl ester)", "modality": "Small molecule", "target": "Polyunsaturated fatty acid lipid peroxidation", "aliases": [ "RT001" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.207, "evidence": "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" } ], "trials": [ { "nct_id": "NCT04762589", "title": "RT001 in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-03-10", "sponsor": "Biojiva LLC", "url": "https://clinicaltrials.gov/study/NCT04762589" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Alpha-lipoic acid", "modality": "Dietary supplement", "target": "Mitochondrial enzyme cofactor / antioxidant", "aliases": [ "lipoic acid group" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.352, "evidence": "is a potent antioxidant and has anti-inflammatory effects, which are plausible mechanisms in slowing ALS progression" }, { "class": "Mitochondrial dysfunction", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.398, "evidence": "It serves as an essential cofactor for enzymatic reactions in mitochondrial energy production" } ], "trials": [ { "nct_id": "NCT04518540", "title": "Explore Neuroprotective Effect of Lipoic Acid in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "status_group": "other", "start_date": "2020-09-01", "sponsor": "Second Affiliated Hospital, School of Medicine, Zhejiang University", "url": "https://clinicaltrials.gov/study/NCT04518540" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Liposomal resveratrol and curcumin", "modality": "Dietary supplement", "target": "Oxidative stress / inflammatory pathways", "aliases": [ "Liposomed polyphenols resveratrol and curcumin" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.273, "evidence": "it is characterised by loss of oxidative control, excitotoxicity due to excess glutamate and intestinal dysbiosis" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.302, "evidence": "Amyotrophic lateral sclerosis (ALS) is a disease of an inflammatory nature" } ], "trials": [ { "nct_id": "NCT04654689", "title": "Impact of the Combined Treatment of Liposomed Polyphenols With G04CB02 on the ALS Patients", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-11-20", "sponsor": "Fundaci\u00f3n Universidad Cat\u00f3lica de Valencia San Vicente M\u00e1rtir", "url": "https://clinicaltrials.gov/study/NCT04654689" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Caffeine", "modality": "Small molecule", "target": "Adenosine receptors", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.331, "evidence": "Caffeine mitigates ROS accumulation and attenuates motor neuron degeneration in the wobbler mouse model of amyotrophic lateral sclerosis" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2016-05-31", "sponsor": "Genentech, Inc.", "url": "https://clinicaltrials.gov/study/NCT02655614" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Edaravone (with scopolamine, atropine, dexmedetomidine)", "modality": "Combination", "target": "Free radicals (edaravone); muscarinic/adrenergic receptors (adjuncts)", "aliases": [ "MCI-186", "Scopolamine, atropine, edaravone and dexmedetomidine" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.653, "evidence": "Edaravone Free Radical Scavengers/*therapeutic use" } ], "trials": [ { "nct_id": "NCT04391361", "title": "The Safety and Effectiveness of Cholinergic Receptor Block Therapy in the Treatment of ALS", "phase": "PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2020-11-01", "sponsor": "Ruijin Hospital", "url": "https://clinicaltrials.gov/study/NCT04391361" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "EPI-589", "modality": "Small molecule", "target": "Redox/glutathione system", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.234, "evidence": "potential effects of EPI-589 therapy on known trajectory" } ], "trials": [ { "nct_id": "NCT02460679", "title": "Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-01-14", "sponsor": "PTC Therapeutics", "url": "https://clinicaltrials.gov/study/NCT02460679" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Theracurmin HP (curcumin)", "modality": "Dietary supplement", "target": "Unknown", "aliases": [ "Theracurmin", "Theracurmin HP", "curcumin" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.388, "evidence": "plausible mechanisms for slowing ALS progression including antioxidant, anti-inflammatory, and anti-apoptotic effects" } ], "trials": [ { "nct_id": "NCT04499963", "title": "Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-08-28", "sponsor": "Richard Bedlack, M.D., Ph.D.", "url": "https://clinicaltrials.gov/study/NCT04499963" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Acetyl-L-carnitine", "modality": "Dietary supplement", "target": "Mitochondrial fatty acid metabolism", "aliases": [], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.466, "evidence": "*Acetylcarnitine/therapeutic use/pharmacology *Oxidative Stress/drug effects Mitochondria/drug effects/metabolism" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.65, "xcheck_cosine": 0.585, "evidence": "*Oxidative Stress/drug effects Mitochondria/drug effects/metabolism" } ], "trials": [ { "nct_id": "NCT06126315", "title": "Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-03-26", "sponsor": "Mario Negri Institute for Pharmacological Research", "url": "https://clinicaltrials.gov/study/NCT06126315" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.65 }, { "name": "Kamlanoflast", "modality": "Small molecule", "target": "Unknown", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.559, "evidence": "anti-inflammatory compound, attenuates oxidative stress in an animal model of amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT07396818", "title": "Kamlanoflast In Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "NOT_YET_RECRUITING", "status_group": "recruiting", "start_date": "2026-02", "sponsor": "Inflammasome Therapeutics", "url": "https://clinicaltrials.gov/study/NCT07396818" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Dexpramipexole", "modality": "Small molecule", "target": "Mitochondrial function / bioenergetics", "aliases": [ "(6R)-(+)-4,5,6,7-tetrahydro-N-propyl-2,6-benzothiazolediamine dihydrochloride", "(6R)-pramipexole", "Dexpramipexole (dose 1)", "Dexpramipexole (dose 2)", "Dexpramipexole 150 mg/day", "Dexpramipexole 300 mg/day", "Dexpramipexole 50 mg/day", "KNS-760704" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.243, "evidence": "oral administration of KNS-760704 in patients with ALS" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.394, "evidence": "Plasma and spinal fluid levels of R(+)PPX are monitored, in addition to biochemical markers of oxidative stress" } ], "trials": [ { "nct_id": "NCT00140218", "title": "R(+) Pramipexole in Early Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2005-08", "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "url": "https://clinicaltrials.gov/study/NCT00140218" }, { "nct_id": "NCT00600873", "title": "R(+)PPX High Dose Treatment of ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2007-08", "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "url": "https://clinicaltrials.gov/study/NCT00600873" }, { "nct_id": "NCT00647296", "title": "Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2008-04-09", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT00647296" }, { "nct_id": "NCT00931944", "title": "Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2009-07", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT00931944" }, { "nct_id": "NCT01281189", "title": "Phase 3 Study of Dexpramipexole in ALS", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-03", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01281189" }, { "nct_id": "NCT01424176", "title": "Dexpramipexole Renal PK Study", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-07", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01424176" }, { "nct_id": "NCT01424163", "title": "Dexpramipexole Japanese PK Study", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-08", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01424163" }, { "nct_id": "NCT01449578", "title": "Dexpramipexole SAD/MAD Study", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-11", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01449578" }, { "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", "status_group": "completed", "start_date": "2012-01", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01511029" }, { "nct_id": "NCT01536249", "title": "Dexpramipexole and Cimetidine Drug Drug Interaction (DDI)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-03", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01536249" }, { "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", "status_group": "terminated", "start_date": "", "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "url": "https://clinicaltrials.gov/study/NCT00596115" }, { "nct_id": "NCT01622088", "title": "Phase 3 Extension Study of Dexpramipexole in ALS", "phase": "PHASE3", "status": "TERMINATED", "status_group": "terminated", "start_date": "2012-06", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01622088" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 10, "terminated": 2, "other": 0, "total": 12 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "CNM-Au8", "modality": "Other", "target": "Neuronal bioenergetic/metabolic reactions (NAD+/NADH catalysis)", "aliases": [ "CNMAu8", "Gold Nanocrystals" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.289, "evidence": "an aqueous suspension of gold nanocrystals with a catalytic ability to increase the efficiency of key metabolic reactions while reducing the ROS level" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.75, "xcheck_cosine": 0.548, "evidence": "increase the efficiency of key metabolic reactions while reducing the ROS level" } ], "trials": [ { "nct_id": "NCT04297683", "title": "HEALEY ALS Platform Trial - Master Protocol", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-06-14", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04297683" }, { "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", "status_group": "active", "start_date": "2021-11-13", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT05299658" }, { "nct_id": "NCT04098406", "title": "Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-12-19", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT04098406" }, { "nct_id": "NCT04414345", "title": "HEALEY ALS Platform Trial - Regimen C CNM-Au8", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-07-30", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04414345" }, { "nct_id": "NCT05281484", "title": "Two Intermediate Expanded Access Protocols (EAP) CNMAu8.EAP01 and CNMAu8.EAP02 for ALS", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "status_group": "terminated", "start_date": "", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT05281484" }, { "nct_id": "NCT06408727", "title": "Intermediate Expanded Access Protocol CNMAu8.EAP04", "phase": "Expanded Access", "status": "TEMPORARILY_NOT_AVAILABLE", "status_group": "terminated", "start_date": "", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT06408727" }, { "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", "status_group": "terminated", "start_date": "2020-03", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT03843710" } ], "trial_counts": { "recruiting": 0, "active": 2, "completed": 2, "terminated": 3, "other": 0, "total": 7 }, "role_here": "contributing", "confidence_here": 0.75 }, { "name": "Verdiperstat", "modality": "Small molecule", "target": "Myeloperoxidase (MPO)", "aliases": [], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.488, "evidence": "selective, brain-permeable irreversible inhibitor of the myeloperoxidase (MPO) enzyme,1 an abundant peroxidase enzyme in activated myeloid cells, including activated microglia" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.75, "xcheck_cosine": 0.3, "evidence": "MPO catalyzes the production of hypochlorous acid, a key contributor to the oxygen-dependent activity of phagocytes. In systemic and neurological disease states, MPO-induced ox" } ], "trials": [ { "nct_id": "NCT04297683", "title": "HEALEY ALS Platform Trial - Master Protocol", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-06-14", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04297683" }, { "nct_id": "NCT04436510", "title": "HEALEY ALS Platform Trial - Regimen B Verdiperstat", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-07-28", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04436510" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 1, "terminated": 0, "other": 0, "total": 2 }, "role_here": "contributing", "confidence_here": 0.75 }, { "name": "HK-001 (KCHO-1)", "modality": "Dietary supplement", "target": "Unknown", "aliases": [ "HK-001" ], "mechanisms": [ { "class": "Oxidative stress", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.449, "evidence": "attenuates oxidative stress in an animal model of amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT03651349", "title": "To Determine the Maximum Tolerated Dose (MTD) of HK-001 in Healthy Volunteers", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2021-02-01", "sponsor": "Everfront Biotech Co., Ltd.", "url": "https://clinicaltrials.gov/study/NCT03651349" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.7 }, { "name": "Coenzyme Q10", "modality": "Dietary supplement", "target": "Mitochondrial electron transport chain", "aliases": [ "CoQ10", "ubiquinone" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.426, "evidence": "determine the efficacy and preferred dose of CoQ10 in individuals with ALS" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.286, "evidence": "determine the efficacy and preferred dose of CoQ10 in individuals with ALS" } ], "trials": [ { "nct_id": "NCT00243932", "title": "Clinical Trial of High Dose CoQ10 in ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2005-04", "sponsor": "Columbia University", "url": "https://clinicaltrials.gov/study/NCT00243932" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.7 }, { "name": "MitoQ", "modality": "Small molecule", "target": "Mitochondrial reactive oxygen species (mitochondria-targeted antioxidant)", "aliases": [], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.585, "evidence": "The antioxidant protein Oxr1 influences aspects of mitochondrial morphology" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.8, "xcheck_cosine": 0.276, "evidence": "These general categories are neuroinflammation, oxidative stress, impaired autophagy \\& axonal transport, and mitochondrial dysfunction" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2024-06-12", "sponsor": "Duke University", "url": "https://clinicaltrials.gov/study/NCT06429059" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.8 }, { "name": "Withania somnifera (Ashwagandha)", "modality": "Dietary supplement", "target": "Unknown", "aliases": [ "Withania somnifera" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.202, "evidence": "WS has plausible mechanisms for slowing ALS progression because of its effects on inflammation, oxidative stress, autophagy" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.55, "xcheck_cosine": 0.445, "evidence": "because of its effects on inflammation, oxidative stress, autophagy" } ], "trials": [ { "nct_id": "NCT05031351", "title": "NF-\u03baB Inhibition in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2021-10-19", "sponsor": "Sunnybrook Health Sciences Centre", "url": "https://clinicaltrials.gov/study/NCT05031351" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "contributing", "confidence_here": 0.55 } ] }, { "id": "mitochondrial", "name": "Mitochondrial dysfunction", "description": "Therapies improving mitochondrial bioenergetics, membrane stability, or apoptosis regulation.", "primary_count": 17, "therapy_count": 19, "trial_count": 49, "therapies": [ { "name": "nL-CHCHD-001", "modality": "Antisense oligonucleotide", "target": "CHCHD10 mRNA", "aliases": [], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.479, "evidence": "CHCHD2 and CHCHD10 regulate mitochondrial dynamics and integrated stress response" } ], "trials": [ { "nct_id": "NCT07095686", "title": "Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS", "phase": "PHASE1, PHASE2", "status": "ENROLLING_BY_INVITATION", "status_group": "recruiting", "start_date": "2024-10-02", "sponsor": "n-Lorem Foundation", "url": "https://clinicaltrials.gov/study/NCT07095686" }, { "nct_id": "NCT07423494", "title": "Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)", "phase": "PHASE1, PHASE2", "status": "NOT_YET_RECRUITING", "status_group": "recruiting", "start_date": "2026-03", "sponsor": "n-Lorem Foundation", "url": "https://clinicaltrials.gov/study/NCT07423494" }, { "nct_id": "NCT06392126", "title": "Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS", "phase": "PHASE1, PHASE2", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2024-04-16", "sponsor": "n-Lorem Foundation", "url": "https://clinicaltrials.gov/study/NCT06392126" }, { "nct_id": "NCT06977451", "title": "Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS", "phase": "PHASE1, PHASE2", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2024-06-24", "sponsor": "n-Lorem Foundation", "url": "https://clinicaltrials.gov/study/NCT06977451" } ], "trial_counts": { "recruiting": 2, "active": 2, "completed": 0, "terminated": 0, "other": 0, "total": 4 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Acetyl-L-carnitine", "modality": "Dietary supplement", "target": "Mitochondrial fatty acid metabolism", "aliases": [], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.466, "evidence": "*Acetylcarnitine/therapeutic use/pharmacology *Oxidative Stress/drug effects Mitochondria/drug effects/metabolism" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.65, "xcheck_cosine": 0.585, "evidence": "*Oxidative Stress/drug effects Mitochondria/drug effects/metabolism" } ], "trials": [ { "nct_id": "NCT06126315", "title": "Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS", "phase": "PHASE2, PHASE3", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-03-26", "sponsor": "Mario Negri Institute for Pharmacological Research", "url": "https://clinicaltrials.gov/study/NCT06126315" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "EH301", "modality": "Combination", "target": "NAD+ metabolism (nicotinamide riboside + pterostilbene)", "aliases": [], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.305, "evidence": "a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2025-11-25", "sponsor": "Biogipuzkoa Health Research Institute", "url": "https://clinicaltrials.gov/study/NCT07414212" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Dexpramipexole", "modality": "Small molecule", "target": "Mitochondrial function / bioenergetics", "aliases": [ "(6R)-(+)-4,5,6,7-tetrahydro-N-propyl-2,6-benzothiazolediamine dihydrochloride", "(6R)-pramipexole", "Dexpramipexole (dose 1)", "Dexpramipexole (dose 2)", "Dexpramipexole 150 mg/day", "Dexpramipexole 300 mg/day", "Dexpramipexole 50 mg/day", "KNS-760704" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.243, "evidence": "oral administration of KNS-760704 in patients with ALS" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.394, "evidence": "Plasma and spinal fluid levels of R(+)PPX are monitored, in addition to biochemical markers of oxidative stress" } ], "trials": [ { "nct_id": "NCT00140218", "title": "R(+) Pramipexole in Early Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2005-08", "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "url": "https://clinicaltrials.gov/study/NCT00140218" }, { "nct_id": "NCT00600873", "title": "R(+)PPX High Dose Treatment of ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2007-08", "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "url": "https://clinicaltrials.gov/study/NCT00600873" }, { "nct_id": "NCT00647296", "title": "Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2008-04-09", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT00647296" }, { "nct_id": "NCT00931944", "title": "Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2009-07", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT00931944" }, { "nct_id": "NCT01281189", "title": "Phase 3 Study of Dexpramipexole in ALS", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-03", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01281189" }, { "nct_id": "NCT01424176", "title": "Dexpramipexole Renal PK Study", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-07", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01424176" }, { "nct_id": "NCT01424163", "title": "Dexpramipexole Japanese PK Study", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-08", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01424163" }, { "nct_id": "NCT01449578", "title": "Dexpramipexole SAD/MAD Study", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-11", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01449578" }, { "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", "status_group": "completed", "start_date": "2012-01", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01511029" }, { "nct_id": "NCT01536249", "title": "Dexpramipexole and Cimetidine Drug Drug Interaction (DDI)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-03", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01536249" }, { "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", "status_group": "terminated", "start_date": "", "sponsor": "Bennett, James P., Jr., M.D., Ph.D.", "url": "https://clinicaltrials.gov/study/NCT00596115" }, { "nct_id": "NCT01622088", "title": "Phase 3 Extension Study of Dexpramipexole in ALS", "phase": "PHASE3", "status": "TERMINATED", "status_group": "terminated", "start_date": "2012-06", "sponsor": "Knopp Biosciences", "url": "https://clinicaltrials.gov/study/NCT01622088" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 10, "terminated": 2, "other": 0, "total": 12 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "AMX0035", "modality": "Combination", "target": "Endoplasmic reticulum stress and mitochondrial apoptosis pathways", "aliases": [ "Albrioza", "PB-TURSO", "Relyvrio", "sodium phenylbutyrate and taurursodiol" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.283, "evidence": "evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS" }, { "class": "Proteostasis / autophagy", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.354, "evidence": "extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for ALS" } ], "trials": [ { "nct_id": "NCT05021536", "title": "Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment", "phase": "PHASE3", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2021-10-28", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT05021536" }, { "nct_id": "NCT05286372", "title": "An Intermediate Size Expanded Access Protocol of AMX0035 for ALS", "phase": "Expanded Access", "status": "APPROVED_FOR_MARKETING", "status_group": "completed", "start_date": "", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT05286372" }, { "nct_id": "NCT03127514", "title": "AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-06-22", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT03127514" }, { "nct_id": "NCT03488524", "title": "Open Label Extension Study of AMX0035 in Patients With ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2018-03-29", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT03488524" }, { "nct_id": "NCT04516096", "title": "A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-11-22", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT04516096" }, { "nct_id": "NCT05619783", "title": "Extension Study Evaluating The Safety And Tolerability of AMX0035", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2022-12-29", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT05619783" }, { "nct_id": "NCT04987671", "title": "Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2021-08-05", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT04987671" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 5, "terminated": 0, "other": 1, "total": 7 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "CNM-Au8", "modality": "Other", "target": "Neuronal bioenergetic/metabolic reactions (NAD+/NADH catalysis)", "aliases": [ "CNMAu8", "Gold Nanocrystals" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.289, "evidence": "an aqueous suspension of gold nanocrystals with a catalytic ability to increase the efficiency of key metabolic reactions while reducing the ROS level" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.75, "xcheck_cosine": 0.548, "evidence": "increase the efficiency of key metabolic reactions while reducing the ROS level" } ], "trials": [ { "nct_id": "NCT04297683", "title": "HEALEY ALS Platform Trial - Master Protocol", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-06-14", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04297683" }, { "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", "status_group": "active", "start_date": "2021-11-13", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT05299658" }, { "nct_id": "NCT04098406", "title": "Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-12-19", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT04098406" }, { "nct_id": "NCT04414345", "title": "HEALEY ALS Platform Trial - Regimen C CNM-Au8", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-07-30", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04414345" }, { "nct_id": "NCT05281484", "title": "Two Intermediate Expanded Access Protocols (EAP) CNMAu8.EAP01 and CNMAu8.EAP02 for ALS", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "status_group": "terminated", "start_date": "", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT05281484" }, { "nct_id": "NCT06408727", "title": "Intermediate Expanded Access Protocol CNMAu8.EAP04", "phase": "Expanded Access", "status": "TEMPORARILY_NOT_AVAILABLE", "status_group": "terminated", "start_date": "", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT06408727" }, { "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", "status_group": "terminated", "start_date": "2020-03", "sponsor": "Clene Nanomedicine", "url": "https://clinicaltrials.gov/study/NCT03843710" } ], "trial_counts": { "recruiting": 0, "active": 2, "completed": 2, "terminated": 3, "other": 0, "total": 7 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Creatine", "modality": "Dietary supplement", "target": "Creatine kinase / cellular energy metabolism", "aliases": [ "Creatine monohydrate", "Creatinine" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.357, "evidence": "Recent genetic and biochemical studies implicate free radical toxicity, glutamate excitotoxicity and mitochondrial dysfunction as possible causes of familial AL" } ], "trials": [ { "nct_id": "NCT00005674", "title": "Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis [ALS]", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "", "sponsor": "National Center for Research Resources (NCRR)", "url": "https://clinicaltrials.gov/study/NCT00005674" }, { "nct_id": "NCT00005766", "title": "Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "", "sponsor": "National Center for Research Resources (NCRR)", "url": "https://clinicaltrials.gov/study/NCT00005766" }, { "nct_id": "NCT00355576", "title": "Combination Therapy Selection Trial in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-07", "sponsor": "Columbia University", "url": "https://clinicaltrials.gov/study/NCT00355576" }, { "nct_id": "NCT01257581", "title": "Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-03", "sponsor": "Nazem Atassi", "url": "https://clinicaltrials.gov/study/NCT01257581" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 4, "terminated": 0, "other": 0, "total": 4 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Tauroursodeoxycholic acid", "modality": "Small molecule", "target": "Bile acid receptor / anti-apoptotic pathways", "aliases": [ "TUDCA", "tauroursodeoxycholic acid (TUDCA)" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.386, "evidence": "the demonstration of antioxidant, antiapoptotic and neuroprotective properties of TUDCA in the central nervous system" }, { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.233, "evidence": "hydrophilic bile acids, particularly tauroursodeoxycholic acid (TUDCA), have shown important anti-apoptotic and neuroprotective activities" } ], "trials": [ { "nct_id": "NCT00877604", "title": "Efficacy and Tolerability of Tauroursodeoxycholic Acid in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2008-06", "sponsor": "Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta", "url": "https://clinicaltrials.gov/study/NCT00877604" }, { "nct_id": "NCT03800524", "title": "Safety and Efficacy of TUDCA as add-on Treatment in Patients Affected by ALS", "phase": "PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2019-02-22", "sponsor": "Humanitas Mirasole SpA", "url": "https://clinicaltrials.gov/study/NCT03800524" }, { "nct_id": "NCT05753852", "title": "Open Label Extension of TUDCA-ALS Study", "phase": "PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2021-10-25", "sponsor": "Humanitas Mirasole SpA", "url": "https://clinicaltrials.gov/study/NCT05753852" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 2, "total": 3 }, "role_here": "primary", "confidence_here": 0.65 }, { "name": "Olesoxime", "modality": "Small molecule", "target": "Mitochondrial permeability transition pore (TSPO/VDAC)", "aliases": [ "TRO19622" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.275, "evidence": "TRO19622 330 mg QD as add-on therapy to riluzole 50 mg bid in the treatment of patients suffering from ALS" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2009-04-30", "sponsor": "Hoffmann-La Roche", "url": "https://clinicaltrials.gov/study/NCT00868166" }, { "nct_id": "NCT01285583", "title": "Safety Extension Study of TRO19622 in ALS", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2010-10", "sponsor": "Hoffmann-La Roche", "url": "https://clinicaltrials.gov/study/NCT01285583" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Triheptanoin", "modality": "Small molecule", "target": "Mitochondrial energy metabolism (anaplerotic substrate)", "aliases": [], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.289, "evidence": "Triheptanoin can improve mitochondrial function and energy production and therefore has potential for slowing ALS progression" } ], "trials": [ { "nct_id": "NCT03506425", "title": "A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2018-06-21", "sponsor": "Richard Bedlack, M.D., Ph.D.", "url": "https://clinicaltrials.gov/study/NCT03506425" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "KetoCal (ketogenic diet)", "modality": "Dietary supplement", "target": "Cellular energy metabolism", "aliases": [ "KD", "KetoCal", "ketogenic diet" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.432, "evidence": "these have plausible mechanisms, including augmenting cellular energy balance" } ], "trials": [ { "nct_id": "NCT01016522", "title": "Safety and Tolerability of the Ketogenic Diet in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE3", "status": "TERMINATED", "status_group": "terminated", "start_date": "2009-11", "sponsor": "Johns Hopkins University", "url": "https://clinicaltrials.gov/study/NCT01016522" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.65 }, { "name": "L-Carnitine", "modality": "Dietary supplement", "target": "Mitochondrial fatty acid transport", "aliases": [ "L-Carnitine Injection\uff0c1000mg once daily" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.341, "evidence": "single-arm pilot study to estimate the safety and efficacy L-carnitine in ALS patients with CHCHD10 mutations" } ], "trials": [ { "nct_id": "NCT06849115", "title": "Effects of L-Carnitine in Amyotrophic Lateral Sclerosis Patients With CHCHD10 Mutations", "phase": "PHASE4", "status": "COMPLETED", "status_group": "completed", "start_date": "2024-07-18", "sponsor": "ChaodongWang", "url": "https://clinicaltrials.gov/study/NCT06849115" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Oxaloacetate (Oxaloacetic acid)", "modality": "Small molecule", "target": "TCA cycle intermediate / cellular energy metabolism", "aliases": [ "Oxaloacetic Acid" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.419, "evidence": "Oxaloacetate treatment preserves motor function in SOD1(G93A) mice and normalizes select neuroinflammation-related parameters in the spinal cord" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.528, "evidence": "normalizes select neuroinflammation-related parameters in the spinal cord" } ], "trials": [ { "nct_id": "NCT04204889", "title": "Trial of Oxaloacetate in ALS", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-03-17", "sponsor": "Omar Jawdat", "url": "https://clinicaltrials.gov/study/NCT04204889" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Coenzyme Q10", "modality": "Dietary supplement", "target": "Mitochondrial electron transport chain", "aliases": [ "CoQ10", "ubiquinone" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.426, "evidence": "determine the efficacy and preferred dose of CoQ10 in individuals with ALS" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.286, "evidence": "determine the efficacy and preferred dose of CoQ10 in individuals with ALS" } ], "trials": [ { "nct_id": "NCT00243932", "title": "Clinical Trial of High Dose CoQ10 in ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2005-04", "sponsor": "Columbia University", "url": "https://clinicaltrials.gov/study/NCT00243932" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "MitoQ", "modality": "Small molecule", "target": "Mitochondrial reactive oxygen species (mitochondria-targeted antioxidant)", "aliases": [], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.585, "evidence": "The antioxidant protein Oxr1 influences aspects of mitochondrial morphology" }, { "class": "Oxidative stress", "role": "contributing", "confidence": 0.8, "xcheck_cosine": 0.276, "evidence": "These general categories are neuroinflammation, oxidative stress, impaired autophagy \\& axonal transport, and mitochondrial dysfunction" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2024-06-12", "sponsor": "Duke University", "url": "https://clinicaltrials.gov/study/NCT06429059" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Beta-Hydroxybutyrate Ester (KetoneAid KE4)", "modality": "Dietary supplement", "target": "Cellular energy metabolism / mitochondrial complex I energy deficit", "aliases": [ "Beta Hydroxybutyrate Ester (KetoneAid KE4)" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.431, "evidence": "One potential mechanism of weight loss in ALS is a disturbance of the mitochondrial complex I which causes an energy deficit in affected cells" } ], "trials": [ { "nct_id": "NCT04820478", "title": "Efficacy and Tolerability of Beta Hydroxybutyrate Ester in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "NA", "status": "COMPLETED", "status_group": "completed", "start_date": "2022-04-01", "sponsor": "University of Ulm", "url": "https://clinicaltrials.gov/study/NCT04820478" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Trimetazidine", "modality": "Small molecule", "target": "Fatty acid beta-oxidation (mitochondrial metabolism)", "aliases": [ "TMZ", "Trimetazidine Dihydrochloride" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.515, "evidence": "Trimetazidine Improves Mitochondrial Dysfunction in SOD1(G93A) Cellular Models of Amyotrophic Lateral Sclerosis through Autophagy Activation" }, { "class": "Proteostasis / autophagy", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.496, "evidence": "Trimetazidine Improves Mitochondrial Dysfunction in SOD1(G93A) Cellular Models of Amyotrophic Lateral Sclerosis through Autophagy Activation" } ], "trials": [ { "nct_id": "NCT04788745", "title": "Targeting Metabolic Flexibility in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-06-29", "sponsor": "The University of Queensland", "url": "https://clinicaltrials.gov/study/NCT04788745" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Metformin", "modality": "Small molecule", "target": "RAN protein translation (C9orf72 repeat expansion)", "aliases": [], "mechanisms": [ { "class": "C9orf72", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.405, "evidence": "reduces repeat-associated non-canonical start codon ... (RAN) proteins" }, { "class": "Mitochondrial dysfunction", "role": "contributing", "confidence": 0.65, "xcheck_cosine": 0.607, "evidence": "Metformin ameliorates mitochondrial damage induced by C9orf72 poly(GR) via upregulating AKT phosphorylation" } ], "trials": [ { "nct_id": "NCT04220021", "title": "Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-01-10", "sponsor": "University of Florida", "url": "https://clinicaltrials.gov/study/NCT04220021" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.65 }, { "name": "Alpha-lipoic acid", "modality": "Dietary supplement", "target": "Mitochondrial enzyme cofactor / antioxidant", "aliases": [ "lipoic acid group" ], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.352, "evidence": "is a potent antioxidant and has anti-inflammatory effects, which are plausible mechanisms in slowing ALS progression" }, { "class": "Mitochondrial dysfunction", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.398, "evidence": "It serves as an essential cofactor for enzymatic reactions in mitochondrial energy production" } ], "trials": [ { "nct_id": "NCT04518540", "title": "Explore Neuroprotective Effect of Lipoic Acid in Amyotrophic Lateral Sclerosis", "phase": "NA", "status": "UNKNOWN", "status_group": "other", "start_date": "2020-09-01", "sponsor": "Second Affiliated Hospital, School of Medicine, Zhejiang University", "url": "https://clinicaltrials.gov/study/NCT04518540" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "contributing", "confidence_here": 0.7 } ] }, { "id": "excitotoxicity", "name": "Glutamate excitotoxicity", "description": "Therapies reducing glutamate-mediated excitotoxic motor-neuron injury (e.g., riluzole, AMPA-receptor antagonists).", "primary_count": 10, "therapy_count": 10, "trial_count": 41, "therapies": [ { "name": "Riluzole", "modality": "Small molecule", "target": "Glutamatergic neurotransmission", "aliases": [ "Riluzole (100 mg)", "Riluzole 100mg/day", "Riluzole 50 MG", "Riluzole 50 MG (Part B)", "Riluzole Oral Soluble film (ROSF) 50 mg", "Riluzole tablet" ], "mechanisms": [ { "class": "Glutamate excitotoxicity", "role": "primary", "confidence": 1.0, "xcheck_cosine": 0.41, "evidence": "Riluzole is a benzothiazole derivative that blocks glutamatergic neurotransmission in the CNS, which is thought to exert neuroprotective" } ], "trials": [ { "nct_id": "NCT03127267", "title": "Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients", "phase": "PHASE3", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2021-02-02", "sponsor": "AB Science", "url": "https://clinicaltrials.gov/study/NCT03127267" }, { "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", "status_group": "recruiting", "start_date": "2025-11-25", "sponsor": "Biogipuzkoa Health Research Institute", "url": "https://clinicaltrials.gov/study/NCT07414212" }, { "nct_id": "NCT07142291", "title": "PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS", "phase": "PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-11-25", "sponsor": "PhenoNet, Inc.", "url": "https://clinicaltrials.gov/study/NCT07142291" }, { "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", "status_group": "recruiting", "start_date": "2026-01-01", "sponsor": "AB Science", "url": "https://clinicaltrials.gov/study/NCT07174492" }, { "nct_id": "NCT00542412", "title": "CARE Canadian ALS Riluzole Evaluation", "phase": "PHASE4", "status": "COMPLETED", "status_group": "completed", "start_date": "2001-01", "sponsor": "Sanofi", "url": "https://clinicaltrials.gov/study/NCT00542412" }, { "nct_id": "NCT00353665", "title": "Memantine for Disability in Amyotrophic Lateral Sclerosis (MEDALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2005-07", "sponsor": "University of Lisbon", "url": "https://clinicaltrials.gov/study/NCT00353665" }, { "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", "status_group": "completed", "start_date": "2009-04-30", "sponsor": "Hoffmann-La Roche", "url": "https://clinicaltrials.gov/study/NCT00868166" }, { "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", "status_group": "completed", "start_date": "2011-06", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT01378676" }, { "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", "status_group": "completed", "start_date": "2011-11", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT01486849" }, { "nct_id": "NCT01709149", "title": "Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-10", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT01709149" }, { "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", "status_group": "completed", "start_date": "2013-04", "sponsor": "AB Science", "url": "https://clinicaltrials.gov/study/NCT02588677" }, { "nct_id": "NCT02645461", "title": "Acetylcholine Receptors From Human Muscles as Pharmacological Target for ALS", "phase": "NA", "status": "COMPLETED", "status_group": "completed", "start_date": "2014-01", "sponsor": "University of Roma La Sapienza", "url": "https://clinicaltrials.gov/study/NCT02645461" }, { "nct_id": "NCT02238626", "title": "Ibudilast (MN-166) in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2014-09", "sponsor": "MediciNova", "url": "https://clinicaltrials.gov/study/NCT02238626" }, { "nct_id": "NCT04454840", "title": "Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-05-01", "sponsor": "Peking University Third Hospital", "url": "https://clinicaltrials.gov/study/NCT04454840" }, { "nct_id": "NCT03039673", "title": "MIROCALS: Modifying Immune Response and OutComes in ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-06-19", "sponsor": "Centre Hospitalier Universitaire de N\u012bmes", "url": "https://clinicaltrials.gov/study/NCT03039673" }, { "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", "status_group": "completed", "start_date": "2021-02-24", "sponsor": "Corestemchemon, Inc.", "url": "https://clinicaltrials.gov/study/NCT04745299" }, { "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", "status_group": "completed", "start_date": "2022-11-09", "sponsor": "Corestemchemon, Inc.", "url": "https://clinicaltrials.gov/study/NCT06676423" }, { "nct_id": "NCT05508074", "title": "Treatment Combining Riluzole and IFB-088 in Bulbar Amyotrophic Lateral Sclerosis (TRIALS Protocol)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2022-12-02", "sponsor": "InFlectis BioScience", "url": "https://clinicaltrials.gov/study/NCT05508074" }, { "nct_id": "NCT03537807", "title": "Expanded Access Protocol of BHV-0223 for Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "status_group": "terminated", "start_date": "", "sponsor": "Biohaven Pharmaceuticals, Inc.", "url": "https://clinicaltrials.gov/study/NCT03537807" }, { "nct_id": "NCT00818389", "title": "Study to Investigate the 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"title": "Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2017-03-16", "sponsor": "Isfahan University of Medical Sciences", "url": "https://clinicaltrials.gov/study/NCT03272802" }, { "nct_id": "NCT04950933", "title": "The Treatment of Amyotrophic Lateral Sclerosis With Huollingshengji Granules", "phase": "PHASE2, PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2020-06-01", "sponsor": "Peking University Third Hospital", "url": "https://clinicaltrials.gov/study/NCT04950933" } ], "trial_counts": { "recruiting": 4, "active": 0, "completed": 14, "terminated": 4, "other": 2, "total": 24 }, "role_here": "primary", "confidence_here": 1.0 }, { "name": "Memantine", "modality": "Small molecule", "target": "NMDA receptor", "aliases": [ "Memantine (Ebixa)", "Memantine Hydrochloride Oral Solution" ], "mechanisms": [ { "class": "Glutamate excitotoxicity", 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Autophagy pathway can be activated by mTOR inhibit" } ], "trials": [ { "nct_id": "NCT00214110", "title": "Tamoxifen Therapy in Amyotrophic Lateral Sclerosis [ALS]", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2001-01", "sponsor": "University of Wisconsin, Madison", "url": "https://clinicaltrials.gov/study/NCT00214110" }, { "nct_id": "NCT01257581", "title": "Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-03", "sponsor": "Nazem Atassi", "url": "https://clinicaltrials.gov/study/NCT01257581" }, { "nct_id": "NCT02166944", "title": "Tamoxifen Treatment in Patients With Motor Neuron Disease", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2014-04", "sponsor": "Taipei Medical University Shuang Ho Hospital", "url": "https://clinicaltrials.gov/study/NCT02166944" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 3, "terminated": 0, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "VRG50635", "modality": "Small molecule", "target": "PIKfyve kinase", "aliases": [], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.298, "evidence": "Glycoprotein Non-Metastatic Melanoma Protein B (GPNMB): A Translational Pharmacodynamic Biomarker for PIKfyve Inhibition With VRG50635" } ], "trials": [ { "nct_id": "NCT06286475", "title": "A Study of VRG50635 in Healthy Volunteers", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2024-02-29", "sponsor": "Verge Genomics", "url": "https://clinicaltrials.gov/study/NCT06286475" }, { "nct_id": "NCT06215755", "title": "A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "TERMINATED", "status_group": "terminated", "start_date": "2024-01-15", "sponsor": "Verge Genomics", "url": "https://clinicaltrials.gov/study/NCT06215755" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 1, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Trehalose (SLS-005)", "modality": "Small molecule", "target": "Autophagy induction (mTOR-independent)", "aliases": [ "SLS-005 Trehalose", "Trehalose" ], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.409, "evidence": "Overexpression of the essential Sis1 chaperone reduces TDP-43 effects on toxicity and proteolysis" } ], "trials": [ { "nct_id": "NCT04297683", "title": "HEALEY ALS Platform Trial - Master Protocol", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-06-14", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04297683" }, { "nct_id": "NCT05597436", "title": "Intermediate-Sized Expanded Access Study", "phase": "Expanded Access", "status": "NO_LONGER_AVAILABLE", "status_group": "terminated", "start_date": "", "sponsor": "Seelos Therapeutics, Inc.", "url": "https://clinicaltrials.gov/study/NCT05597436" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 1, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Sodium phenylbutyrate", "modality": "Small molecule", "target": "Histone deacetylase / ER stress chaperone", "aliases": [ "4-PBA", "4-phenylbutyric acid", "NaPB" ], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.286, "evidence": "4-Phenylbutyric Acid (4-PBA) Derivatives Prevent SOD1 Amyloid Aggregation In Vitro" } ], "trials": [ { "nct_id": "NCT00107770", "title": "Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2005-04", "sponsor": "US Department of Veterans Affairs", "url": "https://clinicaltrials.gov/study/NCT00107770" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Rapamycin (Sirolimus)", "modality": "Small molecule", "target": "mTOR", "aliases": [ "Rapamycin" ], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.388, "evidence": "In preclinical studies rapamycin was found to target neuroinflammation, by expanding regulatory T cells, and affecting autophagy, two pillars of amyotrophic lateral sclerosis (ALS) pathogenesis" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.8, "xcheck_cosine": 0.391, "evidence": "rapamycin was found to target neuroinflammation, by expanding regulatory T cells" } ], "trials": [ { "nct_id": "NCT03359538", "title": "Rapamycin Treatment for ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-09-19", "sponsor": "Azienda Ospedaliero-Universitaria di Modena", "url": "https://clinicaltrials.gov/study/NCT03359538" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "ABBV-CLS-7262", "modality": "Small molecule", "target": "eIF2B (integrated stress response modulator)", "aliases": [ "ABBV-CLS-7262 Dose 1", "ABBV-CLS-7262 Dose 2", "CLS-7262", "Regimen F" ], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.265, "evidence": "Regimen F will evaluate the safety and efficacy of a single study drug, ABBV-CLS-7262, in participants with ALS." } ], "trials": [ { "nct_id": "NCT05740813", "title": "HEALEY ALS Platform Trial - Regimen F ABBV-CLS-7262", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2023-03-23", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT05740813" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "IFB-088 (Sephin1 / icergustat)", "modality": "Small molecule", "target": "PPP1R15A (GADD34) / eIF2\u03b1 phosphatase", "aliases": [ "IFB-088 50mg/day" ], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.305, "evidence": "designed to show a signal of efficacy of IFB-088 through ALSFRS-R, MITOS and King's College" } ], "trials": [ { "nct_id": "NCT05508074", "title": "Treatment Combining Riluzole and IFB-088 in Bulbar Amyotrophic Lateral Sclerosis (TRIALS Protocol)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2022-12-02", "sponsor": "InFlectis BioScience", "url": "https://clinicaltrials.gov/study/NCT05508074" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Bosutinib", "modality": "Small molecule", "target": "Src/c-Abl tyrosine kinase", "aliases": [ "Bosutinib (Phase 1 part)", "Bosutinib (Phase 2 part)" ], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.331, "evidence": "c-Abl inhibition could be one of therapeutic options to mitigate the pathology of ALS" }, { "class": "TDP-43 proteinopathy", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.447, "evidence": "c-Abl Regulates the Pathological Deposition of TDP-43 via Tyrosine 43 Phosphorylation" } ], "trials": [ { "nct_id": "NCT04744532", "title": "iPSC-based Drug Repurposing for ALS Medicine (iDReAM) Study", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2019-03-19", "sponsor": "Kyoto University", "url": "https://clinicaltrials.gov/study/NCT04744532" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Trehalose", "modality": "Small molecule", "target": "Autophagy induction (mTOR-independent)", "aliases": [ "SLS-005", "Trehalose injection" ], "mechanisms": [ { "class": "Proteostasis / autophagy", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.205, "evidence": "Regimen 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." } ], "trials": [ { "nct_id": "NCT05136885", "title": "HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2022-02-21", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT05136885" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "AMX0035", "modality": "Combination", "target": "Endoplasmic reticulum stress and mitochondrial apoptosis pathways", "aliases": [ "Albrioza", "PB-TURSO", "Relyvrio", "sodium phenylbutyrate and taurursodiol" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.283, "evidence": "evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS" }, { "class": "Proteostasis / autophagy", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.354, "evidence": "extended treatment with AMX0035 to patients who previously participated in an Amylyx sponsored study of AMX0035 for ALS" } ], "trials": [ { "nct_id": "NCT05021536", "title": "Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment", "phase": "PHASE3", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2021-10-28", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT05021536" }, { "nct_id": "NCT05286372", "title": "An Intermediate Size Expanded Access Protocol of AMX0035 for ALS", "phase": "Expanded Access", "status": "APPROVED_FOR_MARKETING", "status_group": "completed", "start_date": "", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT05286372" }, { "nct_id": "NCT03127514", "title": "AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-06-22", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT03127514" }, { "nct_id": "NCT03488524", "title": "Open Label Extension Study of AMX0035 in Patients With ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2018-03-29", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT03488524" }, { "nct_id": "NCT04516096", "title": "A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-11-22", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT04516096" }, { "nct_id": "NCT05619783", "title": "Extension Study Evaluating The Safety And Tolerability of AMX0035", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2022-12-29", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT05619783" }, { "nct_id": "NCT04987671", "title": "Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2021-08-05", "sponsor": "Amylyx Pharmaceuticals Inc.", "url": "https://clinicaltrials.gov/study/NCT04987671" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 5, "terminated": 0, "other": 1, "total": 7 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Apilimod (LAM-002A)", "modality": "Small molecule", "target": "PIKfyve kinase", "aliases": [ "Apilimod", "LAM-002A" ], "mechanisms": [ { "class": "C9orf72", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.246, "evidence": "evaluate the safety, tolerability, and biological effect of LAM-002A in adults with C9ORF72-associated ALS (C9ALS)" }, { "class": "Proteostasis / autophagy", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.21, "evidence": "evaluate the safety, tolerability, and biological effect of LAM-002A" } ], "trials": [ { "nct_id": "NCT05163886", "title": "Study of Safety, Tolerability, and Biological Activity of LAM-002A in C9ORF72-Associated Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-12-23", "sponsor": "OrphAI Therapeutics", "url": "https://clinicaltrials.gov/study/NCT05163886" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.7 }, { "name": "Melatonin", "modality": "Dietary supplement", "target": "Reactive oxygen species / autophagy pathways", "aliases": [], "mechanisms": [ { "class": "Oxidative stress", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.231, "evidence": "Here we review melatonin. We show that it has plausible mechanisms" }, { "class": "Proteostasis / autophagy", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.42, "evidence": "Melatonin and Autophagy in Aging-Related Neurodegenerative Diseases" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2024-06-12", "sponsor": "Duke University", "url": "https://clinicaltrials.gov/study/NCT06429059" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Trimetazidine", "modality": "Small molecule", "target": "Fatty acid beta-oxidation (mitochondrial metabolism)", "aliases": [ "TMZ", "Trimetazidine Dihydrochloride" ], "mechanisms": [ { "class": "Mitochondrial dysfunction", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.515, "evidence": "Trimetazidine Improves Mitochondrial Dysfunction in SOD1(G93A) Cellular Models of Amyotrophic Lateral Sclerosis through Autophagy Activation" }, { "class": "Proteostasis / autophagy", "role": "contributing", "confidence": 0.7, "xcheck_cosine": 0.496, "evidence": "Trimetazidine Improves Mitochondrial Dysfunction in SOD1(G93A) Cellular Models of Amyotrophic Lateral Sclerosis through Autophagy Activation" } ], "trials": [ { "nct_id": "NCT04788745", "title": "Targeting Metabolic Flexibility in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-06-29", "sponsor": "The University of Queensland", "url": "https://clinicaltrials.gov/study/NCT04788745" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.7 } ] }, { "id": "rna_metabolism", "name": "RNA metabolism", "description": "Therapies targeting broad RNA processing/splicing dysregulation not specific to a single gene above.", "primary_count": 1, "therapy_count": 1, "trial_count": 2, "therapies": [ { "name": "PrimeC (ciprofloxacin/celecoxib)", "modality": "Combination", "target": "microRNA processing / COX-2 / iron homeostasis", "aliases": [ "Fixed dose combination Ciprofloxacin/Celecoxib" ], "mechanisms": [ { "class": "RNA metabolism", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.255, "evidence": "ALS involves multifaceted pathogenic mechanisms including microRNA dysregulation, iron accumulation, and neuroinflammation" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.328, "evidence": "microRNA dysregulation, iron accumulation, and neuroinflammation" } ], "trials": [ { "nct_id": "NCT04165850", "title": "Open Label Study to Evaluate Ciprofloxacin/Celecoxib Combination in Patients With ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-11-25", "sponsor": "NeuroSense Therapeutics Ltd.", "url": "https://clinicaltrials.gov/study/NCT04165850" }, { "nct_id": "NCT04090684", "title": "Ciprofloxacin/Celecoxib Combination in Patients With ALS", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-12-09", "sponsor": "NeuroSense Therapeutics Ltd.", "url": "https://clinicaltrials.gov/study/NCT04090684" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.65 } ] }, { "id": "neurotrophic", "name": "Neurotrophic / regenerative", "description": "Neurotrophic factors, stem-cell, and gene therapies aiming to protect or regenerate motor neurons.", "primary_count": 45, "therapy_count": 47, "trial_count": 60, "therapies": [ { "name": "Pridopidine", "modality": "Small molecule", "target": "Sigma-1 receptor", "aliases": [ "ACR16", "PL101" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.349, "evidence": "Targeting the Sigma-1 Receptor via Pridopidine Ameliorates Central Features of ALS Pathology in a SOD1(G93A) Model" } ], "trials": [ { "nct_id": "NCT07322003", "title": "Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS", "phase": "PHASE3", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2026-02-01", "sponsor": "Prilenia", "url": "https://clinicaltrials.gov/study/NCT07322003" }, { "nct_id": "NCT04297683", "title": "HEALEY ALS Platform Trial - Master Protocol", "phase": "PHASE2, PHASE3", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2020-06-14", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04297683" }, { "nct_id": "NCT04615923", "title": "HEALEY ALS Platform Trial - Regimen D Pridopidine", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2020-12-18", "sponsor": "Merit E. Cudkowicz, MD", "url": "https://clinicaltrials.gov/study/NCT04615923" }, { "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", "status_group": "terminated", "start_date": "", "sponsor": "Prilenia", "url": "https://clinicaltrials.gov/study/NCT06069934" } ], "trial_counts": { "recruiting": 1, "active": 1, "completed": 1, "terminated": 1, "other": 0, "total": 4 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Q-Cells", "modality": "Cell therapy", "target": "Glial progenitor cells (astrocyte replacement)", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.408, "evidence": "Q-Cells\u00ae transplantation in subjects with ALS" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2026-12", "sponsor": "Q Therapeutics, Inc.", "url": "https://clinicaltrials.gov/study/NCT02478450" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "hUC-MSC-derived small extracellular vesicles (hUC-MSC-sEV-001)", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "MSC exosomes nasal drop", "exosomes derived from human umbilical cord blood mesenchymal stem cells for nasal drop", "hUC-MSC-sEV-001" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.212, "evidence": "Intranasal administration of human mesenchymal stromal cell-derived small extracellular vesicles delays disease progression in the SOD1(G93A) mouse model" } ], "trials": [ { "nct_id": "NCT06598202", "title": "Exploring Nasal Drop Therapy With Small Extracellular Vesicles for ALS", "phase": "PHASE1, PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2024-12-01", "sponsor": "Xuanwu Hospital, Beijing", "url": "https://clinicaltrials.gov/study/NCT06598202" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "iPSC-derived motor neuron progenitors (iPSC-MNP)", "modality": "Cell therapy", "target": "Motor neuron replacement", "aliases": [ "iPSC-MNP" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.254, "evidence": "Induced pluripotent stem cells (iPSCs) are a type of induced pluripotent stem cell" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2024-03-13", "sponsor": "Shanghai East Hospital", "url": "https://clinicaltrials.gov/study/NCT06765564" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Nerve Growth Factor", "modality": "Biologic/Antibody", "target": "NGF/TrkA receptor", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.426, "evidence": "Nerve growth factor (NGF) is a protective factor of neural cells" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2024-05", "sponsor": "Beijing Tiantan Hospital", "url": "https://clinicaltrials.gov/study/NCT06391645" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "XS228CN (iPSC-derived motor neuron progenitor cells)", "modality": "Cell therapy", "target": "Motor neuron replacement/support", "aliases": [ "Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.527, "evidence": "Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects with Amyotrophic Lateral Sclerosis" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2025-09-15", "sponsor": "XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.", "url": "https://clinicaltrials.gov/study/NCT07118319" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Debamestrocel (MSC-NTF, NurOwn)", "modality": "Cell therapy", "target": "Neurotrophic factor secretion (autologous MSC-NTF cells)", "aliases": [ "Debamestrocel - MSC-NTF (NurOwn)" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.202, "evidence": "Debamestrocel - MSC-NTF (NurOwn)" } ], "trials": [ { "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", "status_group": "recruiting", "start_date": "2025-06-30", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT06973629" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "NurOwn (MSC-NTF cells)", "modality": "Cell therapy", "target": "Neurotrophic factor secretion", "aliases": [ "MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF", "NurOwn\u00ae (MSC-NTF cells)", "Nurown MSC-NTF cells" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.489, "evidence": "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)" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.222, "evidence": "MSCs secrete growth factors and modulate immune system" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2012-12", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT01777646" }, { "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", "status_group": "completed", "start_date": "2014-05", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT02017912" }, { "nct_id": "NCT03280056", "title": "Safety and Efficacy of Repeated Administrations of NurOwn\u00ae in ALS Patients", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-08-28", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT03280056" }, { "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", "status_group": "terminated", "start_date": "", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT04681118" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 3, "terminated": 1, "other": 0, "total": 4 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "sNN0029 (VEGF165)", "modality": "Biologic/Antibody", "target": "VEGF signaling (VEGF165 growth factor)", "aliases": [ "VEGF165", "sNN0029", "sNN0029-001" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.355, "evidence": "intracerebroventricular administration of sNN0029, containing the growth factor VEGF165, in patients with amyotrophic lateral sclerosis" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2008-12", "sponsor": "Newron Sweden AB", "url": "https://clinicaltrials.gov/study/NCT00800501" }, { "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", "status_group": "terminated", "start_date": "2009-06", "sponsor": "Newron Sweden AB", "url": "https://clinicaltrials.gov/study/NCT01384162" }, { "nct_id": "NCT01999803", "title": "A Safety Study of sNN0029 Administration Via Intracerebroventricular Route to Patients With ALS", "phase": "PHASE1", "status": "TERMINATED", "status_group": "terminated", "start_date": "2014-09", "sponsor": "Newron Sweden AB", "url": "https://clinicaltrials.gov/study/NCT01999803" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 2, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Fasudil", "modality": "Small molecule", "target": "Rho-associated kinase (ROCK)", "aliases": [ "Fasudil (WP-0512)" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.317, "evidence": "The rho kinase (ROCK) inhibitor Fasudil was shown to be neuroprotective, induced axonal regeneration and improved survival and behavioral outcome in models of ALS" } ], "trials": [ { "nct_id": "NCT05218668", "title": "Rho Kinase Inhibitor in Amyotrophic Lateral Sclerosis (REAL)", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2021-12-22", "sponsor": "Woolsey Pharmaceuticals", "url": "https://clinicaltrials.gov/study/NCT05218668" }, { "nct_id": "NCT03792490", "title": "Inhibition of Rho Kinase (ROCK) With Fasudil as Disease-modifying Treatment for ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-02-20", "sponsor": "University Medical Center Goettingen", "url": "https://clinicaltrials.gov/study/NCT03792490" }, { "nct_id": "NCT01935518", "title": "A Clinical Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2013-09", "sponsor": "Peking University Third Hospital", "url": "https://clinicaltrials.gov/study/NCT01935518" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 1, "terminated": 0, "other": 1, "total": 3 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Lenzumestrocel", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "Neuronata-R", "Neuronata-R inj.", "autologous bone marrow-derived mesenchymal stem cells" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.329, "evidence": "Mesenchymal stem cells (MSCs) are a type of adult stem cell with the ability to develop into different types of mesoderm-derived cells" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2021-02-24", "sponsor": "Corestemchemon, Inc.", "url": "https://clinicaltrials.gov/study/NCT04745299" }, { "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", "status_group": "completed", "start_date": "2022-11-09", "sponsor": "Corestemchemon, Inc.", "url": "https://clinicaltrials.gov/study/NCT06676423" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Autologous mesenchymal stem cells", "modality": "Cell therapy", "target": "Unknown", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.602, "evidence": "Boundary Cap Neural Crest Stem Cells Promote Survival of Mutant SOD1 Motor Neurons" } ], "trials": [ { "nct_id": "NCT01142856", "title": "Mesenchymal Stem Cells for Treatment of Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2010-06", "sponsor": "Mayo Clinic", "url": "https://clinicaltrials.gov/study/NCT01142856" }, { "nct_id": "NCT01609283", "title": "A Dose-escalation Safety Trial for Intrathecal Autologous Mesenchymal Stem Cell Therapy in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-05", "sponsor": "Mayo Clinic", "url": "https://clinicaltrials.gov/study/NCT01609283" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Autologous bone marrow mononuclear stem cells", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "Stem Cell", "Stem cell" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.474, "evidence": "The effect of autologous bone marrow mononuclear cells on duration of survival in Amyotrophic Lateral Sclerosis patients" } ], "trials": [ { "nct_id": "NCT02242071", "title": "Cell Therapy for Motor Neuron Disease/Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "WITHDRAWN", "status_group": "terminated", "start_date": "2008-12", "sponsor": "Neurogen Brain and Spine Institute", "url": "https://clinicaltrials.gov/study/NCT02242071" }, { "nct_id": "NCT01984814", "title": "Stem Cell Therapy for Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "WITHDRAWN", "status_group": "terminated", "start_date": "2008-12", "sponsor": "Neurogen Brain and Spine Institute", "url": "https://clinicaltrials.gov/study/NCT01984814" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 2, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.65 }, { "name": "Mesenchymal stem cells (MSC)", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "Intravenous administration of 1 million of MSC", "Intravenous administration of 2 million of MSC", "Intravenous administration of 4 million of MSC" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.219, "evidence": "MSCs secrete growth factors and modulate immune system" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.75, "xcheck_cosine": 0.222, "evidence": "MSCs secrete growth factors and modulate immune system" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2014-07", "sponsor": "Andalusian Initiative for Advanced Therapies - Fundaci\u00f3n P\u00fablica Andaluza Progreso y Salud", "url": "https://clinicaltrials.gov/study/NCT02290886" }, { "nct_id": "NCT04821479", "title": "Repeated Mesenchymal Stem Cell Injections in ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-01-01", "sponsor": "Hadassah Medical Organization", "url": "https://clinicaltrials.gov/study/NCT04821479" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Mesenchymal stem cells", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "mesenchymal stem cell" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.361, "evidence": "stem cell transplantation is safe and can be effective in reduction of disease progression and increase of quality of life" } ], "trials": [ { "nct_id": "NCT02492516", "title": "Intravenous Injection of Adipose Derived Mesenchymal Stem Cell for ALS", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2014-09", "sponsor": "Royan Institute", "url": "https://clinicaltrials.gov/study/NCT02492516" }, { "nct_id": "NCT02116634", "title": "Mesenchymal Stem Cell Injection in Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "WITHDRAWN", "status_group": "terminated", "start_date": "2015-05", "sponsor": "Alzahra Hospital, Iran", "url": "https://clinicaltrials.gov/study/NCT02116634" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 1, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Stem cell therapy", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "Stem Cells" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.404, "evidence": "the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells" } ], "trials": [ { "nct_id": "NCT03067857", "title": "Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2016-09", "sponsor": "Stem Cells Arabia", "url": "https://clinicaltrials.gov/study/NCT03067857" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "sNN0029 (VEGF-A)", "modality": "Biologic/Antibody", "target": "VEGF receptor", "aliases": [ "VEGF165", "sNN0029", "sNN0029 infusion solution" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.226, "evidence": "continuous i.c.v administration of 4 \u03bcg sNN0029/day in patients with ALS" } ], "trials": [ { "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", "status_group": "terminated", "start_date": "2015-01", "sponsor": "Newron Sweden AB", "url": "https://clinicaltrials.gov/study/NCT02269436" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Growth Hormone (Somatropin)", "modality": "Peptide", "target": "Growth hormone receptor / IGF-1 axis", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.374, "evidence": "GH and IGF-1 play an important role as neurotrophic factors within the PNS and CNS" } ], "trials": [ { "nct_id": "NCT00635960", "title": "Growth Hormone in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2007-03", "sponsor": "Federico II University", "url": "https://clinicaltrials.gov/study/NCT00635960" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "HYNR-CS inj (autologous bone marrow-derived stem cells)", "modality": "Cell therapy", "target": "Autologous bone marrow-derived mesenchymal stem cells", "aliases": [ "HYNR-CS inj" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.419, "evidence": "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" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2011-02", "sponsor": "Corestemchemon, Inc.", "url": "https://clinicaltrials.gov/study/NCT01363401" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Autologous Mesenchymal Stem Cells (MSCs)", "modality": "Cell therapy", "target": "Mesenchymal stem cells (paracrine/neurotrophic support)", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.454, "evidence": "The therapeutic plasticity of mesenchymal stem cells (MSCs) may be an attractive therapy to this comple" } ], "trials": [ { "nct_id": "NCT02987413", "title": "Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2015-04-28", "sponsor": "Hospital e Maternidade Dr. Christ\u00f3v\u00e3o da Gama", "url": "https://clinicaltrials.gov/study/NCT02987413" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.65 }, { "name": "AstroRx", "modality": "Cell therapy", "target": "Astrocyte replacement (embryonic stem cell-derived astrocytes)", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.523, "evidence": "intrathecal injection of healthy astrocytes in ALS can potentially compensate for the diseased astrocytes" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2018-04-12", "sponsor": "Kadimastem", "url": "https://clinicaltrials.gov/study/NCT03482050" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Autologous Bone Marrow-Derived Stem Cells", "modality": "Cell therapy", "target": "Motor neuron microenvironment", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.533, "evidence": "the infusion of autologous bone marrow-derived stem cells, in 6 patients with Amyotrophic Lateral Sclerosis" } ], "trials": [ { "nct_id": "NCT01082653", "title": "Safety/Efficacy Study for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "SUSPENDED", "status_group": "terminated", "start_date": "2010-03", "sponsor": "TCA Cellular Therapy", "url": "https://clinicaltrials.gov/study/NCT01082653" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "AlloRx (allogeneic umbilical cord-derived mesenchymal stem cells)", "modality": "Cell therapy", "target": "Motor neuron microenvironment", "aliases": [ "AlloRx" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.474, "evidence": "intrathecal injection of cultured allogeneic adult umbilical cord derived mesenchymal stem cells for the treatment of amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT05003921", "title": "Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cell Intrathecal Injection for ALS", "phase": "PHASE1", "status": "SUSPENDED", "status_group": "terminated", "start_date": "2025-12", "sponsor": "The Foundation for Orthopaedics and Regenerative Medicine", "url": "https://clinicaltrials.gov/study/NCT05003921" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Autologous adipose-derived regenerative cells (ADRC)", "modality": "Cell therapy", "target": "Motor neuron microenvironment (paracrine/regenerative)", "aliases": [ "Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.536, "evidence": "autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT03296501", "title": "Intraspinal Transplantation of Autologous ADRC in ALS Patients", "phase": "PHASE1", "status": "UNKNOWN", "status_group": "other", "start_date": "2015-10-13", "sponsor": "Mossakowski Medical Research Centre Polish Academy of Sciences", "url": "https://clinicaltrials.gov/study/NCT03296501" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "SB-509", "modality": "Gene therapy", "target": "VEGF-A (zinc finger transcription factor activator)", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.235, "evidence": "evaluate the effects of the investigational drug, SB-509 on progression of the disease in subjects with ALS" } ], "trials": [ { "nct_id": "NCT00748501", "title": "Clinical Trial of SB-509 in Subjects With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2008-09", "sponsor": "Sangamo Therapeutics", "url": "https://clinicaltrials.gov/study/NCT00748501" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Human Neural Stem Cells", "modality": "Cell therapy", "target": "Motor neuron microenvironment", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.426, "evidence": "verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model" } ], "trials": [ { "nct_id": "NCT01640067", "title": "Human Neural Stem Cell Transplantation in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-12", "sponsor": "Azienda Ospedaliera Santa Maria, Terni, Italy", "url": "https://clinicaltrials.gov/study/NCT01640067" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Intrathecal Autologous Stem Cell Therapy", "modality": "Cell therapy", "target": "Motor neuron microenvironment", "aliases": [ "Intrathecal autologous stem cell" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.72, "xcheck_cosine": 0.57, "evidence": "Autologous cell therapy in patients with ALS can stimulate neuroplasticity, modifying the neurodegenerative process and stops the clinical progression of disease" } ], "trials": [ { "nct_id": "NCT01933321", "title": "Effect of Intrathecal Administration of Hematopoietic Stem Cells in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2, PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-09", "sponsor": "Hospital Universitario Dr. Jose E. Gonzalez", "url": "https://clinicaltrials.gov/study/NCT01933321" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.72 }, { "name": "Recombinant human erythropoietin", "modality": "Biologic/Antibody", "target": "Erythropoietin receptor", "aliases": [ "recombinant human erythropoietin(rhEPO)" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.271, "evidence": "Erythropoietin is neuroprotective in animal models of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS)." } ], "trials": [ { "nct_id": "NCT03835507", "title": "Randomized, Double-blind, Safety and Efficacy of Recombinant Human Erythropoietin in Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2016-06-20", "sponsor": "Hanyang University Seoul Hospital", "url": "https://clinicaltrials.gov/study/NCT03835507" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "HYNR-CS-Allo (allogeneic bone marrow-derived stem cells)", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "HYNR-CS-Allo" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.377, "evidence": "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" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2012-12", "sponsor": "Hanyang University Seoul Hospital", "url": "https://clinicaltrials.gov/study/NCT01758510" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Filgrastim", "modality": "Biologic/Antibody", "target": "G-CSF receptor", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.505, "evidence": "Granulocyte-colony stimulating factor (G-CSF): an emerging therapeutic approach for amyotrophic lateral sclerosis (ALS)" } ], "trials": [ { "nct_id": "NCT02236065", "title": "Combination Therapy of Cord Blood and G-CSF for Patients With Brain Injury or Neurodegenerative Disorders", "phase": "NA", "status": "COMPLETED", "status_group": "completed", "start_date": "2014-08", "sponsor": "MinYoung Kim, M.D.", "url": "https://clinicaltrials.gov/study/NCT02236065" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "ARGX-119", "modality": "Biologic/Antibody", "target": "MuSK (agonist antibody)", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.65, "xcheck_cosine": 0.338, "evidence": "assess the impact of ARGX-119 on ALS disease outcomes, including muscle function" } ], "trials": [ { "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", "status_group": "active", "start_date": "2024-10-23", "sponsor": "argenx", "url": "https://clinicaltrials.gov/study/NCT06441682" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.65 }, { "name": "Insulin-like Growth Factor 1 (IGF-1)", "modality": "Biologic/Antibody", "target": "IGF-1 receptor", "aliases": [ "Insulin like growth factor, type 1" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.331, "evidence": "determine if insulin-like growth factor-1 (IGF-I) slows the progressive weakness in amyotrophic lateral sclerosis (ALS) patients" } ], "trials": [ { "nct_id": "NCT00035815", "title": "Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS) Trial", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2003-06", "sponsor": "Mayo Clinic", "url": "https://clinicaltrials.gov/study/NCT00035815" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "GSK1223249", "modality": "Biologic/Antibody", "target": "Nogo-A", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.201, "evidence": "The drug works by inhibiting the protein that prevents nerve growth." } ], "trials": [ { "nct_id": "NCT00875446", "title": "First Time in Human Study of GSK1223249 in Amyotrophic Lateral Sclerosis", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2009-05-13", "sponsor": "GlaxoSmithKline", "url": "https://clinicaltrials.gov/study/NCT00875446" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Autologous mesenchymal stem cells (MSC 3P)", "modality": "Cell therapy", "target": "Mesenchymal stromal/stem cells", "aliases": [ "Suspension of human autologous MSC 3P in 1.5 ml" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.345, "evidence": "stem cell research has expanded greatl" } ], "trials": [ { "nct_id": "NCT03828123", "title": "Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-01", "sponsor": "Bioinova, s.r.o.", "url": "https://clinicaltrials.gov/study/NCT03828123" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "NTF001", "modality": "Gene therapy", "target": "Neuron-derived neurotrophic factor (NDNF)", "aliases": [ "NTF001 Injection" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.456, "evidence": "an AAV-mediated human neuron-derived neurotrophic factor gene therapy, in patients with amyotrophic lateral sclerosis (ALS)" } ], "trials": [ { "nct_id": "NCT07618585", "title": "Safety and Efficacy of Intrathecal NTF001 Injection in ALS", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2026-04-01", "sponsor": "Ruijin Hospital", "url": "https://clinicaltrials.gov/study/NCT07618585" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "CNS10-NPC-GDNF", "modality": "Cell therapy", "target": "GDNF (neural progenitor cells engineered to secrete GDNF)", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.97, "xcheck_cosine": 0.582, "evidence": "Human iPSC-derived neural progenitor cells secreting GDNF provide protection in rodent models of ALS" } ], "trials": [ { "nct_id": "NCT05306457", "title": "CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS", "phase": "PHASE1", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2022-05-08", "sponsor": "Cedars-Sinai Medical Center", "url": "https://clinicaltrials.gov/study/NCT05306457" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.97 }, { "name": "Autologous Adipose-derived Mesenchymal Stromal Cells", "modality": "Cell therapy", "target": "Mesenchymal stromal cells (paracrine neurotrophic/immunomodulatory)", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.346, "evidence": "Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.425, "evidence": "Transplantation of HUMSCs into the spinal canal of SOD1 mice could ameliorate inflammatory responses, reduce neuronal cell death" } ], "trials": [ { "nct_id": "NCT03268603", "title": "Intrathecal Autologous Adipose-derived Mesenchymal Stromal Cells for Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "ACTIVE_NOT_RECRUITING", "status_group": "active", "start_date": "2017-10-10", "sponsor": "Mayo Clinic", "url": "https://clinicaltrials.gov/study/NCT03268603" } ], "trial_counts": { "recruiting": 0, "active": 1, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "MSC-NTF cells (NurOwn), intramuscular", "modality": "Cell therapy", "target": "Neurotrophic factor secretion (GDNF, VEGF, etc.)", "aliases": [ "MSC-NTF cells transplantation (IM)", "NurOwn", "autologous MSC-NTF cells IM" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.451, "evidence": "autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF)" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.222, "evidence": "MSCs secrete growth factors and modulate immune system" } ], "trials": [ { "nct_id": "NCT01051882", "title": "Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients.", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-06", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT01051882" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "MSC-NTF cells (NurOwn), intrathecal", "modality": "Cell therapy", "target": "Neurotrophic factor secretion (GDNF, VEGF, etc.)", "aliases": [ "MSC-NTF cells transplantation (IT)", "NurOwn", "autologous MSC-NTF cells IT" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.451, "evidence": "autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF)" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.222, "evidence": "MSCs secrete growth factors and modulate immune system" } ], "trials": [ { "nct_id": "NCT01051882", "title": "Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients.", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2011-06", "sponsor": "Brainstorm-Cell Therapeutics", "url": "https://clinicaltrials.gov/study/NCT01051882" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "GM604", "modality": "Peptide", "target": "Nervous system regulatory signaling pathways", "aliases": [], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.316, "evidence": "GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system" } ], "trials": [ { "nct_id": "NCT01854294", "title": "GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-08", "sponsor": "Genervon Biopharmaceuticals, LLC", "url": "https://clinicaltrials.gov/study/NCT01854294" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Neural progenitor cells engineered to secrete GDNF (CNS10-NPC-GDNF)", "modality": "Cell therapy", "target": "GDNF delivery / astrocyte replacement", "aliases": [ "Stem cell (HPC) implantation" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.492, "evidence": "transplanting cells that have been engineered to produce a growth factor into the spinal cord of patients" } ], "trials": [ { "nct_id": "NCT02943850", "title": "CNS10-NPC-GDNF for the Treatment of ALS", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-04-01", "sponsor": "Cedars-Sinai Medical Center", "url": "https://clinicaltrials.gov/study/NCT02943850" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Granulocyte-Colony Stimulating Factor (G-CSF / Filgrastim)", "modality": "Biologic/Antibody", "target": "G-CSF receptor", "aliases": [ "granulocyte - colony stimulating factor (G-CSF)" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.255, "evidence": "we decided to use G-CSF to mobilise" }, { "class": "Neuroinflammation", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.333, "evidence": "ALS) is a neurodegenerative disorder characterized by neuronal degeneration and inflammation in the nerves. G-CSF is a 19.6-kDa hema" } ], "trials": [ { "nct_id": "NCT00298597", "title": "Influence of G-CSF and EPO on Associative Learning and Motor Skills", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-03", "sponsor": "University Hospital Muenster", "url": "https://clinicaltrials.gov/study/NCT00298597" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Erythropoietin (EPO)", "modality": "Biologic/Antibody", "target": "Erythropoietin receptor", "aliases": [ "erythropoetin (EPO)" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.324, "evidence": "G-CSF) and erythropoetin (EPO) influence associative learning and/or motor skills of patients" } ], "trials": [ { "nct_id": "NCT00298597", "title": "Influence of G-CSF and EPO on Associative Learning and Motor Skills", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-03", "sponsor": "University Hospital Muenster", "url": "https://clinicaltrials.gov/study/NCT00298597" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "VM202 (Engensis)", "modality": "Gene therapy", "target": "Hepatocyte growth factor (HGF)", "aliases": [ "VM202" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.312, "evidence": "safety and tolerability of intramuscular injections of VM202 at different injection sites in people with amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT02039401", "title": "Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2014-03-11", "sponsor": "Helixmith Co., Ltd.", "url": "https://clinicaltrials.gov/study/NCT02039401" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "HYNRCS-Allo inj (allogeneic bone marrow-derived stem cells)", "modality": "Cell therapy", "target": "Unknown", "aliases": [ "HYNRCS-Allo inj", "HYNRCS-Allo-ALS-02 inj" ], "mechanisms": [ { "class": "Neurotrophic / regenerative", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.306, "evidence": "HLA-haplo matched Allogenic Bone Marrow Derived stem cells(\"HYNRCS-Allo-ALS-02 inj\"), through intrathecal delivery" } ], "trials": [ { "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", "status_group": "other", "start_date": "2017-02-01", "sponsor": "Hanyang University Seoul Hospital", "url": "https://clinicaltrials.gov/study/NCT03214146" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "hUC-MSC-sEV-001 (human umbilical cord MSC-derived small extracellular vesicles)", "modality": "Cell therapy", "target": "Neuroinflammatory/complement signaling", "aliases": [ "hUC-MSC-sEV-001 nasal drops" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.281, "evidence": "reduces the progress of amyotrophic lateral sclerosis and the overactivation of complement-coagulation cascade and NF-kB signaling in SOD1(G93A) mice" }, { "class": "Neurotrophic / regenerative", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.456, "evidence": "Mesenchymal stem/stromal cells (MSCs) have been reported to exert neuroprotective effects in models of injury and disease, acting primarily through release of small e" } ], "trials": [ { "nct_id": "NCT06607900", "title": "hUC-MSC-sEV-001 Nasal Drops for Neurodegenerative Diseases", "phase": "PHASE1", "status": "NOT_YET_RECRUITING", "status_group": "recruiting", "start_date": "2025-07-01", "sponsor": "Xuanwu Hospital, Beijing", "url": "https://clinicaltrials.gov/study/NCT06607900" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "contributing", "confidence_here": 0.6 }, { "name": "Wharton's Jelly Mesenchymal Stem Cells (WJMSC)", "modality": "Cell therapy", "target": "Immune modulation / neuroprotection", "aliases": [ "HUMSCs", "Mesenchymal stem cells isolated from Wharton's jelly", "WJMSC", "human umbilical mesenchymal stromal cells" ], "mechanisms": [ { "class": "Neuroinflammation", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.425, "evidence": "Transplantation of HUMSCs into the spinal canal of SOD1 mice could ameliorate inflammatory responses, reduce neuronal cell death" }, { "class": "Neurotrophic / regenerative", "role": "contributing", "confidence": 0.65, "xcheck_cosine": 0.637, "evidence": "reduce neuronal cell death, maintain a better mobility and effectively extend their lifespan" } ], "trials": [ { "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", "status_group": "other", "start_date": "2020-12-02", "sponsor": "Polski Bank Komorek Macierzystych JSC (PBKM)", "url": "https://clinicaltrials.gov/study/NCT04651855" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "contributing", "confidence_here": 0.65 } ] }, { "id": "symptomatic_other", "name": "Symptomatic / Other", "description": "Symptomatic treatments, repurposed drugs, or therapies whose ALS mechanism is unclear or does not fit another class.", "primary_count": 37, "therapy_count": 38, "trial_count": 54, "therapies": [ { "name": "Psilocybin", "modality": "Small molecule", "target": "Serotonin 5-HT2A receptor", "aliases": [ "Psilocybin therapy" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.324, "evidence": "evaluate whether psilocybin therapy can effectively treat depression and psychological distress in adult patients with COPD, ALS, MS, or APD" } ], "trials": [ { "nct_id": "NCT06656702", "title": "Effects of Psilocybin in Patients With Amyotrophic Lateral Sclerosis", "phase": "EARLY_PHASE1", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-04-09", "sponsor": "Johns Hopkins University", "url": "https://clinicaltrials.gov/study/NCT06656702" }, { "nct_id": "NCT06782724", "title": "Psilocybin Therapy for Psychological Distress in Palliative Patients", "phase": "PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-07-01", "sponsor": "University Medical Center Groningen", "url": "https://clinicaltrials.gov/study/NCT06782724" } ], "trial_counts": { "recruiting": 2, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Ultra-high-caloric fatty diet (UHCD)", "modality": "Dietary supplement", "target": "Energy/caloric metabolism", "aliases": [ "Ultra-high-caloric fatty diet", "ultra-high-caloric fatty diet" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.222, "evidence": "Weight loss has been identified as a negative prognostic factor in amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT06280079", "title": "Ultra-high-caloric, Fatty Diet in ALS", "phase": "NA", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2024-06-01", "sponsor": "University of Ulm", "url": "https://clinicaltrials.gov/study/NCT06280079" }, { "nct_id": "NCT04172792", "title": "Safety and Tolerability Ultra-high-caloric Food Supplements in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-11-26", "sponsor": "Albert Christian Ludolph, Prof.", "url": "https://clinicaltrials.gov/study/NCT04172792" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Ranolazine", "modality": "Small molecule", "target": "Late sodium current (persistent Na+ channel)", "aliases": [ "Ranolazine 1000 MG", "Ranolazine 500 MG" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.313, "evidence": "Open-label pilot study of ranolazine for cramps in amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT06527222", "title": "A Study of Ranolazine in ALS", "phase": "PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-04-29", "sponsor": "Swathy Chandrashekhar, MBBS", "url": "https://clinicaltrials.gov/study/NCT06527222" }, { "nct_id": "NCT03472950", "title": "Safety and Efficacy of Ranolazine for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2018-06-11", "sponsor": "University of Kansas Medical Center", "url": "https://clinicaltrials.gov/study/NCT03472950" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "RE104", "modality": "Small molecule", "target": "Serotonin 5-HT2A receptor", "aliases": [ "RE104 for Injection" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.267, "evidence": "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" } ], "trials": [ { "nct_id": "NCT07002034", "title": "RE104 Safety and Efficacy Study in Adjustment Disorder in Cancer and Other Medical Illnesses", "phase": "PHASE2", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-07-30", "sponsor": "Reunion Neuroscience Inc", "url": "https://clinicaltrials.gov/study/NCT07002034" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Isocaloric nutrition (100% of individual caloric requirement)", "modality": "Dietary supplement", "target": "Energy metabolism / body weight", "aliases": [ "100% of individual calory requirement" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.575, "evidence": "various interventional studies targeting the energy deficit in ALS yielded promising results" } ], "trials": [ { "nct_id": "NCT06877143", "title": "Hypercaloric PEG Nutrition in ALS to Sustain Energy Homeostasis", "phase": "NA", "status": "RECRUITING", "status_group": "recruiting", "start_date": "2025-03-01", "sponsor": "University of Ulm", "url": "https://clinicaltrials.gov/study/NCT06877143" } ], "trial_counts": { "recruiting": 1, "active": 0, "completed": 0, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Tirasemtiv", "modality": "Small molecule", "target": "Fast skeletal muscle troponin (troponin activator)", "aliases": [ "250 mg CK-2017357", "500 mg CK-2017357", "CK 2017357", "CK-2017357", "CK-2017357 (Part A)", "CK-2017357 (Part B)" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.469, "evidence": "This study assessed the effect of tirasemtiv versus placebo on respiratory function in patients with ALS." } ], "trials": [ { "nct_id": "NCT01089010", "title": "A Study of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2010-03", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT01089010" }, { "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", "status_group": "completed", "start_date": "2011-06", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT01378676" }, { "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", "status_group": "completed", "start_date": "2011-11", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT01486849" }, { "nct_id": "NCT01709149", "title": "Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-10", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT01709149" }, { "nct_id": "NCT02496767", "title": "Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2015-09-03", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT02496767" }, { "nct_id": "NCT02936635", "title": "A Study for Patients Who Completed VITALITY-ALS (CY 4031)", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-10-17", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT02936635" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 6, "terminated": 0, "other": 0, "total": 6 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "Reldesemtiv", "modality": "Small molecule", "target": "Fast skeletal muscle troponin complex", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.31, "evidence": "Fast skeletal muscle troponin activators (FSTAs) sensitize the sarcomere to calcium and increase muscle force. This mechanism is of potential relevance in amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders that cause weakness and muscle fatigue." } ], "trials": [ { "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", "status_group": "completed", "start_date": "2017-07-24", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT03160898" }, { "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", "status_group": "terminated", "start_date": "2021-08-16", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT04944784" }, { "nct_id": "NCT05442775", "title": "A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)", "phase": "PHASE3", "status": "TERMINATED", "status_group": "terminated", "start_date": "2022-08-04", "sponsor": "Cytokinetics", "url": "https://clinicaltrials.gov/study/NCT05442775" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 2, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "Mexiletine", "modality": "Small molecule", "target": "Voltage-gated sodium channels", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.209, "evidence": "determine if mexiletine is effective for the treatment of muscle cramps in Amyotrophic Lateral Sclerosis (ALS)" } ], "trials": [ { "nct_id": "NCT01811355", "title": "Mexiletine for the Treatment of Muscle Cramps in ALS", "phase": "PHASE4", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-05", "sponsor": "Bjorn Oskarsson, MD", "url": "https://clinicaltrials.gov/study/NCT01811355" }, { "nct_id": "NCT01849770", "title": "Mexiletine in Sporadic Amyotrophic Lateral Sclerosis (SALS)", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-07", "sponsor": "University of Washington", "url": "https://clinicaltrials.gov/study/NCT01849770" }, { "nct_id": "NCT02781454", "title": "Mexiletine in Sporadic Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-10", "sponsor": "University of Washington", "url": "https://clinicaltrials.gov/study/NCT02781454" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 3, "terminated": 0, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "FLX-787", "modality": "Small molecule", "target": "TRPV1/TRPA1 receptors", "aliases": [ "FLX-787-ODT", "FLX-787-ODT (orally disintegrating tablet)", "FLX-787-ODT (orally disintigrating tablet)" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.85, "xcheck_cosine": 0.405, "evidence": "how well FLX-787-ODT works to reduce fasciculations in patients with Amyotrophic Lateral Sclerosis" } ], "trials": [ { "nct_id": "NCT03196375", "title": "A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.", "phase": "PHASE2", "status": "TERMINATED", "status_group": "terminated", "start_date": "2017-07-28", "sponsor": "Flex Pharma, Inc.", "url": "https://clinicaltrials.gov/study/NCT03196375" }, { "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", "status_group": "terminated", "start_date": "2017-11", "sponsor": "Flex Pharma, Inc.", "url": "https://clinicaltrials.gov/study/NCT03338114" }, { "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", "status_group": "terminated", "start_date": "2018-04-05", "sponsor": "Flex Pharma, Inc.", "url": "https://clinicaltrials.gov/study/NCT03334786" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 3, "other": 0, "total": 3 }, "role_here": "primary", "confidence_here": 0.85 }, { "name": "Capsaicin", "modality": "Small molecule", "target": "TRPV1 receptor", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.406, "evidence": "Dysphagia (swallow impairment), dystussia (cough impairment) and respiratory impairment are hallmark features of amyotrophic lateral sclerosis (ALS)" } ], "trials": [ { "nct_id": "NCT02710110", "title": "Respiratory Strength Training in Persons With Amyotrophic Lateral Sclerosis (ALS)", "phase": "EARLY_PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-04", "sponsor": "University of Florida", "url": "https://clinicaltrials.gov/study/NCT02710110" }, { "nct_id": "NCT02710162", "title": "Accurate Screening Tools for Dysphagia in Amyotrophic Lateral Sclerosis (ALS)", "phase": "EARLY_PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2016-04", "sponsor": "University of Florida", "url": "https://clinicaltrials.gov/study/NCT02710162" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Dextromethorphan/Quinidine (Nuedexta)", "modality": "Combination", "target": "NMDA receptor / sigma-1 receptor (dextromethorphan); CYP2D6 (quinidine)", "aliases": [ "AVP-923", "AVP-923-20", "AVP-923-30", "DMQ", "Nuedexta", "dextromethorphan hydrobromide 20 mg and quinidine sulfate 10 mg", "dextromethorphan hydrobromide 30 mg and quinidine sulfate 10 mg" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.234, "evidence": "approved by the Food and Drug Administration (FDA) in 2010 to treat pseudobulbar affect (PBA)" } ], "trials": [ { "nct_id": "NCT00573443", "title": "Safety and Efficacy of AVP-923 in PBA Patients With ALS or MS", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2007-12", "sponsor": "Avanir Pharmaceuticals", "url": "https://clinicaltrials.gov/study/NCT00573443" }, { "nct_id": "NCT01806857", "title": "Clinical Trial Nuedexta in Subjects With ALS", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2013-04", "sponsor": "Center for Neurologic Study, La Jolla, California,", "url": "https://clinicaltrials.gov/study/NCT01806857" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 2, "terminated": 0, "other": 0, "total": 2 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "Pimozide", "modality": "Small molecule", "target": "T-type calcium channels / neuromuscular junction", "aliases": [ "Orap", "Pimozide 2 mg per day", "Pimozide 2mg/day", "Pimozide 2mg/day (current) or 4 mg/day (study initiation)", "Pimozide 4 mg per day", "Pimozide 4mg/day" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.6, "xcheck_cosine": 0.419, "evidence": "a new potential drug for ALS with beneficial effects at the level of NMJs" } ], "trials": [ { "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", "status_group": "other", "start_date": "2015-04", "sponsor": "University of Calgary", "url": "https://clinicaltrials.gov/study/NCT02463825" }, { "nct_id": "NCT03272503", "title": "A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2017-10-27", "sponsor": "University of Calgary", "url": "https://clinicaltrials.gov/study/NCT03272503" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 2, "total": 2 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "Vitamin E", "modality": "Dietary supplement", "target": "Muscle cramps / lipid peroxidation (antioxidant)", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.407, "evidence": "This clinical trial will compare the response of high dose vitamin E supplementation to placebo for treatment of muscular cramps in patients with ALS" } ], "trials": [ { "nct_id": "NCT00372879", "title": "Clinical Trial of Vitamin E to Treat Muscular Cramps in Patients With ALS", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2006-12", "sponsor": "London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's", "url": "https://clinicaltrials.gov/study/NCT00372879" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Botulinum toxin type A", "modality": "Biologic/Antibody", "target": "Neuromuscular junction (SNARE-mediated acetylcholine release)", "aliases": [ "Botox injection" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.98, "xcheck_cosine": 0.361, "evidence": "Botulinum toxin injection is a safe and effective symptomatic treatment for sialorrhea in ALS, significantly improving quality of life" } ], "trials": [ { "nct_id": "NCT01551940", "title": "Toxin Treatment for Amyotrophic Lateral Sclerosis (ALS) Related Sialorrhea", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2012-02", "sponsor": "Hospices Civils de Lyon", "url": "https://clinicaltrials.gov/study/NCT01551940" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.98 }, { "name": "Escitalopram", "modality": "Small molecule", "target": "Serotonin transporter (SERT)", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.436, "evidence": "The purpose of this study is to see if escitalopram (Lexapro) improves symptoms of major depressive disorder in patients who have ALS or MS." } ], "trials": [ { "nct_id": "NCT00965497", "title": "Escitalopram (Lexapro) for Depression MS or ALS", "phase": "PHASE3", "status": "COMPLETED", "status_group": "completed", "start_date": "2009-07", "sponsor": "University of South Carolina", "url": "https://clinicaltrials.gov/study/NCT00965497" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Tricyclic Antidepressants", "modality": "Small molecule", "target": "Monoamine reuptake transporters", "aliases": [ "Tricyclic Antidepressants (\"TCA\")" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.501, "evidence": "Depression is seen in 9-11% of ALS patients and adequate and proper treatment is needed." } ], "trials": [ { "nct_id": "NCT02851914", "title": "SSRIs vs. TCAs for Depression in ALS Patients", "phase": "EARLY_PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2015-07-21", "sponsor": "St. Louis University", "url": "https://clinicaltrials.gov/study/NCT02851914" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Selective Serotonin Reuptake Inhibitors", "modality": "Small molecule", "target": "Serotonin transporter (SERT)", "aliases": [ "Selective Serotonin Uptake Inhibitors (\"SSRI\")" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.501, "evidence": "Depression is seen in 9-11% of ALS patients and adequate and proper treatment is needed." } ], "trials": [ { "nct_id": "NCT02851914", "title": "SSRIs vs. TCAs for Depression in ALS Patients", "phase": "EARLY_PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2015-07-21", "sponsor": "St. Louis University", "url": "https://clinicaltrials.gov/study/NCT02851914" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Dextromethorphan/quinidine (AVP-923, Nuedexta)", "modality": "Combination", "target": "NMDA receptor / sigma-1 receptor (pseudobulbar affect)", "aliases": [ "AVP-923", "dextromethorphan/quinidine" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.301, "evidence": "compare and evaluate the safety of AVP-923 (dextromethorphan/quinidine) for the treatment of emotional lability in ALS patients" } ], "trials": [ { "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", "status_group": "completed", "start_date": "2001-01", "sponsor": "Avanir Pharmaceuticals", "url": "https://clinicaltrials.gov/study/NCT00021697" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "Oxepa (high-fat/high-calorie enteral nutrition)", "modality": "Dietary supplement", "target": "Nutritional/caloric support", "aliases": [ "Oxepa" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.32, "evidence": "long-term use of high fat/high calorie and high calorie diets in people with amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT00983983", "title": "High Fat/High Calorie Trial in Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2009-10", "sponsor": "Massachusetts General Hospital", "url": "https://clinicaltrials.gov/study/NCT00983983" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.8 }, { "name": "Jevity 1.5 (high-calorie enteral nutrition)", "modality": "Dietary supplement", "target": "Nutritional/caloric support", "aliases": [ "Jevity 1.5" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.8, "xcheck_cosine": 0.32, "evidence": "long-term use of high fat/high calorie and high calorie diets in people with amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT00983983", 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"https://clinicaltrials.gov/study/NCT04172792" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Ultra-high-caloric carbohydrate-rich diet", "modality": "Dietary supplement", "target": "Energy/caloric balance", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.382, "evidence": "a high-caloric nutrition might prolong survival in fast-progressing ALS patients" } ], "trials": [ { "nct_id": "NCT04172792", "title": "Safety and Tolerability Ultra-high-caloric Food Supplements in Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE1", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-11-26", "sponsor": "Albert Christian Ludolph, Prof.", "url": "https://clinicaltrials.gov/study/NCT04172792" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, 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"class": "Symptomatic / Other", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.225, "evidence": "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." } ], "trials": [ { "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", "status_group": "completed", "start_date": "1992-10", "sponsor": "National Center for Research Resources (NCRR)", "url": "https://clinicaltrials.gov/study/NCT00004771" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Testosterone", "modality": "Small molecule", "target": "Androgen receptor", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": 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"PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2017-07-13", "sponsor": "Chiba University", "url": "https://clinicaltrials.gov/study/NCT03186040" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.6 }, { "name": "IncobotulinumtoxinA", "modality": "Biologic/Antibody", "target": "Neuromuscular junction (SNARE / acetylcholine release)", "aliases": [ "BTX-A", "Botulinum toxin type A", "Incobotulinum Toxin A", "Xeomin" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.95, "xcheck_cosine": 0.261, "evidence": "BTX-A exerts its principal therapeutic effect through targeted modulation of neuromuscular junction activity" } ], "trials": [ { "nct_id": "NCT01565395", "title": "Incobotulinum Toxin A for Sialorrhea in Parkinson's Disease (PD)/Parkinsonism and Amyotrophic Lateral Sclerosis (ALS)", "phase": "PHASE2", "status": "WITHDRAWN", "status_group": "terminated", "start_date": "2012-03", "sponsor": "Beth Israel Deaconess Medical Center", "url": "https://clinicaltrials.gov/study/NCT01565395" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 1, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.95 }, { "name": "Isocaloric diet", "modality": "Other", "target": "Nutritional status / energy metabolism", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.7, "xcheck_cosine": 0.264, "evidence": "efficacy, safety and tolerability of high caloric diet in amyotrophic lateral sclerosis patients" } ], "trials": [ { "nct_id": "NCT04654689", "title": "Impact of the Combined Treatment of Liposomed Polyphenols With G04CB02 on the ALS Patients", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2021-11-20", "sponsor": "Fundaci\u00f3n Universidad Cat\u00f3lica de Valencia San 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"other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.7 }, { "name": "Salbutamol", "modality": "Small molecule", "target": "Beta-2 adrenergic receptor", "aliases": [], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.75, "xcheck_cosine": 0.475, "evidence": "administration of salbutamol in ALS patients may improve walking capacity related to motor fatigue by enhancing neuromuscular transmission" } ], "trials": [ { "nct_id": "NCT05860244", "title": "Effect of Salbutamol on Walking Capacity in Ambulatory ALS Patients", "phase": "PHASE2", "status": "UNKNOWN", "status_group": "other", "start_date": "2024-09", "sponsor": "Assistance Publique - H\u00f4pitaux de Paris", "url": "https://clinicaltrials.gov/study/NCT05860244" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 0, "terminated": 0, "other": 1, "total": 1 }, "role_here": "primary", "confidence_here": 0.75 }, { "name": "Dextromethorphan/Quinidine", "modality": "Combination", "target": "NMDA receptor / sigma-1 receptor (dextromethorphan); CYP2D6 (quinidine)", "aliases": [ "AVP-923", "DMQ", "Nuedexta", "dextromethorphan HBr and quinidine sulfate" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "primary", "confidence": 0.9, "xcheck_cosine": 0.35, "evidence": "significant improvements in bulbar function as measured by the ALSFRS\u2010R bulbar subscore, quantifiable indices of swallowing efficiency and airway safety, and speech function" } ], "trials": [ { "nct_id": "NCT03883581", "title": "Impact of Nuedexta on Bulbar Physiology and Function in ALS", "phase": "PHASE1, PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2019-07-25", "sponsor": "University of Florida", "url": "https://clinicaltrials.gov/study/NCT03883581" } ], "trial_counts": { "recruiting": 0, "active": 0, "completed": 1, "terminated": 0, "other": 0, "total": 1 }, "role_here": "primary", "confidence_here": 0.9 }, { "name": "Levetiracetam", 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"aliases": [ "creatine" ], "mechanisms": [ { "class": "Symptomatic / Other", "role": "contributing", "confidence": 0.6, "xcheck_cosine": 0.334, "evidence": "whether nine months of administration of creatine monohydrate results in an increase in muscle strength in patients with amyotrophic lateral sclerosis" } ], "trials": [ { "nct_id": "NCT00070993", "title": "Creatine for the Treatment of Amyotrophic Lateral Sclerosis", "phase": "PHASE2", "status": "COMPLETED", "status_group": "completed", "start_date": "2002-12", "sponsor": "National Center for Complementary and Integrative Health (NCCIH)", "url": "https://clinicaltrials.gov/study/NCT00070993" }, { "nct_id": "NCT00069186", "title": "Study of Creatine Monohydrate in Patients With Amyotrophic Lateral Sclerosis", "phase": "PHASE3", "status": "UNKNOWN", "status_group": "other", "start_date": "2003-06", "sponsor": "The Avicena Group", "url": "https://clinicaltrials.gov/study/NCT00069186" } ], "trial_counts": { "recruiting": 0, "active": 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