protocol_id stringclasses 263
values | section_number stringlengths 1 12 | title stringlengths 1 1.88k | content stringlengths 0 866k | merged_titles listlengths 0 491 |
|---|---|---|---|---|
NCT03717415 | 9.11.2 | Eastern Cooperative Oncology Group Performance Status | 9.11.2 Eastern Cooperative Oncology Group Performance Status Assessments will be summarized overall by time point utilizing continuous descriptive statistics. In addition, the change from baseline will be summarized for continuous parameters. For categorical parameters, the n and percentage will be displayed for each c... | [] |
NCT03717415 | 9.11.3 | Vital Signs | 9.11.3 Vital Signs Assessments will be summarized overall by time point utilizing continuous descriptive statistics. In addition, the change from baseline will be summarized for continuous parameters. | [] |
NCT03717415 | 9.11.4 | Echocardiogram/Multigated Acquisition Scans | 9.11.4 Echocardiogram/Multigated Acquisition Scans Assessments will be summarized overall by time point utilizing continuous descriptive statistics. In addition, the change from baseline will be summarized for continuous parameters. For categorical parameters, the n and percentage will be displayed for each cohort. | [] |
NCT03717415 | 9.11.5 | Ophthalmologic Assessments | 9.11.5 Ophthalmologic Assessments Ophthalmologic assessments will be summarized by n and percent for each cohort. | [] |
NCT03717415 | 9.11.6 | Clinical Laboratory Parameters | 9.11.6 Clinical Laboratory Parameters Assessments will be summarized overall by time point utilizing continuous descriptive statistics. In addition, the change from baseline will be summarized for continuous parameters. For categorical parameters, the n and percentage will be displayed. Shift tables for labs with NCI-C... | [] |
NCT03717415 | 9.12 | Pharmacokinetic Analysis | 9.12 Pharmacokinetic Analysis Pharmacokinetic concentrations will be summarized utilizing continuous descriptive statistics: n, median, mean, standard deviation, minimum, and maximum. Cmax, Cmin, and AUC will also be summarized using geometric mean. | [] |
NCT03717415 | 9.12.1 | Tumor Markers Analysis | 9.12.1 Tumor Markers Analysis Tumor markers will be summarized overall by time points using descriptive statistics for each cohort. In addition, changes from baseline will be summarized. | [] |
NCT03717415 | 9.12.2 | Biomarker and Pharmacodynamic Analysis | 9.12.2 Biomarker and Pharmacodynamic Analysis Biomarker and pharmacodynamic parameters will be summarized graphically and with descriptive statistics (mean, SD, median, min, max). | [] |
NCT03717415 | 9.13 | Pharmacogenomic Analysis | 9.13 Pharmacogenomic Analysis Pharmacogenomic analysis will explore the impact of variations in genes encoding for drug metabolism enzymes and drug transporters on patient's response to study drug. | [] |
NCT03717415 | 9.14 | Procedures for Reporting Deviations to Original Statistical Analysis Plan | 9.14 Procedures for Reporting Deviations to Original Statistical Analysis Plan All deviations from the original SAP will be provided in the final clinical study report. | [] |
NCT03717415 | 10 | QUALITY CONTROL AND QUALITY ASSURANCE | 10 QUALITY CONTROL AND QUALITY ASSURANCE | [] |
NCT03717415 | 10.1 | Study Site Monitoring Visits | 10.1 Study Site Monitoring Visits During study conduct, the Sponsor or its agent will conduct periodic monitoring visits to ensure that the protocol and GCPs are being followed. The monitors will review source documents to confirm that the data recorded on eCRFs is accurate. The Investigator and institution will allow ... | [] |
NCT03717415 | 10.2 | Protocol Compliance | 10.2 Protocol Compliance The Investigator must conduct the study in compliance with the protocol provided by the Sponsor, and given approval/favorable opinion by the IRB and the appropriate regulatory authority(ies). Modifications to the protocol must not be made without agreement between both the Investigator and the ... | [] |
NCT03717415 | 11 | DATA HANDLING AND RECORD KEEPING | 11 DATA HANDLING AND RECORD KEEPING | [] |
NCT03717415 | 11.1 | Electronic Case Report Form | 11.1 Electronic Case Report Form The Sponsor or designee will provide the study sites with secure access to and training on the electronic data capture application sufficient to permit site personnel to enter or correct information in the eCRFs on the patients for which they are responsible. An eCRF is required and mus... | [] |
NCT03717415 | 11.2 | Record Retention | 11.2 Record Retention To enable evaluations and/or audits from regulatory authorities or the Sponsor, the Investigator agrees to keep records, including the identity of all participating patients (sufficient information to link records, e.g., eCRFs and hospital records), all signed ICFs, SAE forms, source documents, de... | [] |
NCT03717415 | 12 | ETHICS | 12 ETHICS | [] |
NCT03717415 | 12.1 | Ethical Conduct of the Study | 12.1 Ethical Conduct of the Study The study will be conducted in accordance with the Declaration of Helsinki on Ethical Principles for Medical Research Involving Human Subjects, adopted by the General Assembly of the World Medical Association. In addition, the study will be conducted in accordance with the protocol, IC... | [] |
NCT03717415 | 12.2 | Patient Information and Consent | 12.2 Patient Information and Consent All parties must ensure protection of patient personal data and must not include patient names on any Sponsor forms, reports, publications, or in any other disclosures, except where required by laws. In case of data transfer, the Sponsor must maintain high standards of confidentiali... | [] |
NCT03717415 | 12.3 | IRB | 12.3 IRB It is the responsibility of the Investigator to have prospective approval of the study protocol, protocol amendments, ICFs, and other relevant documents from the IRB. All correspondence with the IRB must be retained in the Investigator Site File. The only circumstance in which an amendment may be initiated pri... | [] |
NCT03717415 | 12.4 | Patient Confidentiality | 12.4 Patient Confidentiality The Sponsor and designees affirm and uphold the principle of the patient's right to protection against invasion of privacy. Throughout this study, a patient's source data must only be linked to the Sponsor's clinical study database or documentation via a unique identification number. As per... | [] |
NCT03717415 | 12.5 | Reporting of Safety Issues or Serious Breaches of the Protocol or International Conference on Harmonization Good Clinical Practice | 12.5 Reporting of Safety Issues or Serious Breaches of the Protocol or International Conference on Harmonization Good Clinical Practice In the event of any prohibition or restriction imposed (i.e., clinical hold) by an applicable Competent Authority, or if the Investigator is aware of any new information which might in... | [] |
NCT03717415 | 12.6 | Liability and Insurance | 12.6 Liability and Insurance The Sponsor has subscribed to an insurance policy covering, in its terms and provisions, its legal liability for injuries caused to participating persons and arising out of this research performed strictly in accordance with the scientific protocol as well as with applicable law and profess... | [] |
NCT03717415 | 13 | STUDY TERMINATION | 13 STUDY TERMINATION When the Sponsor is aware of information on matters concerning the quality, efficacy, and safety of the study drug, as well as other important information that may affect proper conduct of the clinical study, the Sponsor may discontinue the clinical study and send a written notice of the discontinu... | [] |
NCT03717415 | 13.1 | Criteria for Suspension or Premature Termination of the Study | 13.1 Criteria for Suspension or Premature Termination of the Study Criteria for either temporary suspension or premature termination of the study include: - 1. New information regarding the safety or efficacy of the study drug that indicates a change in the known risk/benefit profile for the compound, such that the ris... | [] |
NCT03717415 | 13.2 | Criteria for Premature Termination or Suspension of Investigational Sites | 13.2 Criteria for Premature Termination or Suspension of Investigational Sites A study site may be terminated prematurely or suspended if the site (including the Investigator) is found to be in significant violation of GCP, protocol, contractual agreement, or is unable to ensure adequate performance of the study. | [] |
NCT03717415 | 13.3 | Procedures for Premature Termination or Suspension of the Study or the Participation of Investigational Site(s) | 13.3 Procedures for Premature Termination or Suspension of the Study or the Participation of Investigational Site(s) In the event that the Sponsor elects to terminate or suspend the study or the participation of an investigational site, a study-specific procedure for early termination or suspension will be provided by ... | [] |
NCT03717415 | 14 | PUBLICATION OF STUDY RESULTS | 14 PUBLICATION OF STUDY RESULTS Any and all scientific, commercial, and technical information disclosed by the Sponsor in this protocol or elsewhere must be considered the confidential and proprietary property of the Sponsor. The Investigator shall hold such information in confidence and shall not disclose the informat... | [] |
NCT03717415 | 15 | REFERENCES | 15 REFERENCES - 1. Hammond MEH, Hayes DF, Dowsett M, Allred DC, Hagerty KL, Badve S, et al. American Society of Clinical Oncology/College of American Pathologists Guideline Recommendations for Immunohistochemical Testing of Estrogen and Progesterone Receptors in Breast Cancer. Arch Pathol Lab Med. 2010;134:907-01. - 2.... | [] |
NCT03734588 | 1 | PROTOCOL SUMMARY | 1 PROTOCOL SUMMARY Protocol Title: Dose-finding study of SPK-8016 gene therapy in patients with hemophilia A to support future evaluations in individuals with FVIII inhibitors. Protocol Number: SPK-8016-101 Sponsor: Spark Therapeutics Development Phase: Phase 1/2a Name of Investigational Product: SPK-8016 Study Indicat... | [
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"Exploratory",
"Study Population:",
"Inclusion Criteria:",
"Exclusion Criteria:",
"iii. APRI >1",
"Planned Number of Participants:",
"Number and Location of Study Sites:",
"Seque... |
NCT03734588 | 2 | INTRODUCTION | 2 INTRODUCTION | [] |
NCT03734588 | 2.1 | Background | 2.1 Background SPK-8016 is an investigational gene therapy medicinal product that contains SPK-8016 is being developed by Spark Therapeutics for the treatment of hemophilia A. This clinical study (SPK-8016-101) will evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with clinically severe hemoph... | [] |
NCT03734588 | 2.1.1 | Hemophilia A | 2.1.1 Hemophilia A Hemophilia A is an X-chromosome linked bleeding disorder that primarily affects 1 in 5,000 male births (Giangrande, 2005; Srivastava, 2013). The severity of disease is characterized by the endogenous level of FVIII measured in the plasma. Severe hemophilia A is defined as a coagulation activity of FV... | [] |
NCT03734588 | 2.1.2 | Clinical Manifestations | 2.1.2 Clinical Manifestations About 70% of newborn babies with hemophilia have a positive family history. When the diagnosis is not suspected based on a positive family history, affected children present with bleeding from the umbilical stump, prolonged bleeding after circumcision, bleeding following intramuscular immu... | [] |
NCT03734588 | 2.1.3 | Current Therapies and Prevention for Hemophilia A | 2.1.3 Current Therapies and Prevention for Hemophilia A | [] |
NCT03734588 | 2.1.3.1 | Current Therapies | 2.1.3.1 Current Therapies There is no available cure for hemophilia A. Factor replacement therapy, purified from human plasma, first became available more than 4 decades ago. Although these FVIII products dramatically improved life expectancy and QoL in the U.S.A. and Western Europe, they also resulted in exposure of i... | [] |
NCT03734588 | 2.1.3.2 | Current Prevention | 2.1.3.2 Current Prevention Chronic arthropathy is the major morbidity of hemophilia resulting from recurrent spontaneous bleeds into the joints. Many studies have shown that, even at high doses, on-demand therapy is not effective in preventing arthropathy (Petrini, 1991; Aledort, 1994). Thus, scheduled protein replacem... | [] |
NCT03734588 | 2.1.4 | Alternative Therapy for Hemophilia A | 2.1.4 Alternative Therapy for Hemophilia A In the case of hemophilia, where a cure is currently not attainable and lifelong therapy is needed, QoL is an essential outcome parameter. All hemophilia prophylaxis studies using health-related quality-of-life (HR-QoL) score as an outcome reported a decreased HR-QoL compared ... | [] |
NCT03734588 | 2.1.4.1 | Factor VIII and Protein | 2.1.4.1 Factor VIII and Protein The human FVIII gene is located on the long arm of the X chromosome, at Xq28 (Poustka, 1991; Freije, 1992). It spans 186 kb and consists of 26 exons separated by 25 introns (Gitschier, 1984; Toole, 1984). Most of the exons that make up the 9 kb mRNA are small (69-262 bp), with the except... | [] |
NCT03734588 | 2.1.4.2 | Biology of Adeno-Associated Virus (AAV) Vectors | 2.1.4.2 Biology of Adeno-Associated Virus (AAV) Vectors AAV is a non-enveloped, replication-defective parvovirus that has not been associated with human disease. AAV vectors are derived from the parent virus by removing all of the viral elements except for the inverted terminal repeats (ITR) and inserting the gene or g... | [] |
NCT03734588 | 2.1.4.3 | Gene Therapy as an Alternative Approach | 2.1.4.3 Gene Therapy as an Alternative Approach As a proposed alternative approach, gene therapy may potentially reduce short-term disability and long-term hemophilic arthropathy, reduce incidence of central nervous system (CNS) bleeding, eliminate the need for indwelling intravenous catheters or frequent factor infusi... | [] |
NCT03734588 | 2.2 | Rationale | 2.2 Rationale The primary aim of hemophilia care is to prevent and treat bleeding due to deficient clotting factors. Both hemophilia A and B can be treated with recombinant factor replacement leading to significant improvement in morbidity and mortality. However, such treatment is extremely costly  A number of measures have been taken to improve the expression and safety of the transgene product compared with the previous vector iterations tested by the Sponsor or by others [\(Figure](#page-37-1) [3\)](#page-37-1).   immediately and/or seek immediate emergency care, depending on the severity of the reaction, if a... | [] |
NCT03734588 | 2.3.1.1 | Allergic Reaction or Anaphylaxis | 2.3.1.1 Allergic Reaction or Anaphylaxis There has been 1 report of symptoms associated with SPK-8011 (rAAV-FVIII) infusion, occurring following SPK-8011 vector infusion at a dose of 2x1012 vg/kg. Nonserious events including vomiting, pyrexia, back pain, and myalgia were reported in 1 participant; events started to res... | [] |
NCT03734588 | 2.3.1.2 | Inhibitor Development | 2.3.1.2 Inhibitor Development There is a possibility that participants infused with SPK-8016 will develop inhibitors to FVIII, although inhibitor development has not occurred in any participants who have received AAV2 hFIX, AAV8-hFIX (Nathwani, 2014), AAV8-hFIX19 or BAX-335 (under National Clinical Trial (NCT) 01687608... | [] |
NCT03734588 | 2.3.1.3 | Elevation of Hepatic Transaminases | 2.3.1.3 Elevation of Hepatic Transaminases None of the 4 enrolled participants in study SPK-8016-101 experienced adverse events of transaminitis related to SPK-8016. Of the 17 participants enrolled in the Sponsor's similar hemophilia A gene therapy trial SPK-8011-101, 5 (29%) reported SPK-8011-related adverse events of... | [] |
NCT03734588 | 2.3.1.4 | Anti-AAV Neutralizing Antibody Development | 2.3.1.4 Anti-AAV Neutralizing Antibody Development As is expected after systemic administration of AAV vectors, all 4 participants (100%) in study SPK-8016-101 who were treated with SPK-8016 developed neutralizing antibodies to the AAV capsid. This risk seems to have no immediate impact on the expression of FVIII produ... | [] |
NCT03734588 | 2.3.1.5 | Bleeding Episodes | 2.3.1.5 Bleeding Episodes The proposed dose levels of SPK-8016 may not be sufficient to raise the vector-derived FVIII:C levels to a therapeutic level for controlling and preventing bleeding episodes when the participants halt their prophylactic (factor or non-factor replacement) treatment after vector administration. ... | [] |
NCT03734588 | 2.3.1.6 | Possible Side Effects from Corticosteroids | 2.3.1.6 Possible Side Effects from Corticosteroids To mitigate the potential for harm due to cytotoxic T-lymphocyte (CTL) response against the capsid and to maintain endogenous FVIII expression, corticosteroids are allowed in the current protocol for participants who develop elevated transaminases and/or declining FVII... | [] |
NCT03734588 | 2.3.1.7 | Risks Associated with Additional Immune Modulating Agents | 2.3.1.7 Risks Associated with Additional Immune Modulating Agents The potential side effects of corticosteroids increase in prevalence as treatment is prolonged. For this reason, inflammatory conditions that respond to corticosteroid monotherapy are frequently treated instead with combined immune modulating agents to a... | [] |
NCT03734588 | 2.3.1.7.5 | Immune Suppressive Medications and Risk of Infections, including COVID-19 Risk | 2.3.1.7.5 Immune Suppressive Medications and Risk of Infections, including COVID-19 Risk Immune suppressive medications including used alone or in combination, may increase the risk of bacterial, mycobacterial, fungal and viral infection. This may include community acquired viruses such as influenza and COVID-19 as wel... | [] |
NCT03734588 | 2.3.2 | Potential Benefits | 2.3.2 Potential Benefits The main purpose of study SPK-8016-101 is to evaluate the safety of SPK-8016 at 3 or 4 different dose levels. It is not known if higher dose levels will raise the activity level of FVIII in humans, but, based on prior clinical experience in hemophilia B and non-clinical experience in NHPs with ... | [] |
NCT03734588 | 2.4 | Rationale | 2.4 Rationale Published data from Nathwani and colleagues (Nathwani, 2014) of an AAV8-mediated selfcomplementary hFIX gene transfer trial for severe hemophilia B demonstrated long-term expression of FIX activity levels with mean (±SD) of 5.1 ±1.7% of normal in 6 participants at the highest dose level of 2x1012vg/kg, ov... | [] |
NCT03734588 | 2.5 | Rationale for Dose and Schedule Selection | 2.5 Rationale for Dose and Schedule Selection In non-clinical studies, conducted in both NHPs and in cell culture, SPK-8016 resulted in increased expression of B domain-deleted FVIII, ranging from 2.6- to 4.4-fold higher in in vivo experiments, and 3.7- to 6.6-fold higher in in vitro experiments. With this promising no... | [] |
NCT03734588 | 3 | OBJECTIVES AND ENDPOINTS | 3 OBJECTIVES AND ENDPOINTS | [] |
NCT03734588 | 3.1 | Primary Objective | 3.1 Primary Objective Primary objectives for this study are: - To evaluate the safety and tolerability of SPK-8016. - To evaluate the efficacy as evidenced by prevention of bleeds and level of FVIII expression with SPK-8016. | [] |
NCT03734588 | 3.2 | Secondary Objectives | 3.2 Secondary Objectives Secondary objectives for this study are: - To evaluate additional parameters associated with SPK-8016 directed FVIII expression and vector shedding. - To characterize the immune response to the vector and transgene product. | [] |
NCT03734588 | 3.3 | Primary Endpoints | 3.3 Primary Endpoints The primary endpoints include the following: - For safety and tolerability: - − Incidence of adverse events, including clinically significant abnormal laboratory values. - − Occurrence of hepatic transaminase elevation requiring immunosuppression. - For efficacy: - − Peak and steady-state FVIII ac... | [] |
NCT03734588 | 3.4 | Secondary Endpoints | 3.4 Secondary Endpoints The secondary endpoints include the following: - Additional kinetic assessments including, but not limited to: - − Time to achieve steady-state FVIII activity levels; - − Vector-shedding of SPK-8016 in bodily fluids. - Incidence of immune responses to AAV capsid protein and BDD-hFVIII transgene. | [] |
NCT03734588 | 3.5 | Exploratory Endpoints | 3.5 Exploratory Endpoints The exploratory endpoints will assess changes from baseline in the following: 36. Joint assessments:  - Number of target joints - Hemophilia Joint Health score - 37. Activities assessments: - Hemophilia Activities List - Change in Level of Activity questionnaire - 38. Q... | [] |
NCT03734588 | 4 | STUDY DESIGN | 4 STUDY DESIGN | [] |
NCT03734588 | 4.1 | Overall Design | 4.1 Overall Design SPK-8016-101 is a Phase 1/2a, open-label, non-randomized, dose escalation study to evaluate the safety, tolerability, and efficacy of a single IV infusion of SPK-8016 in men with clinically severe hemophilia A, and no measurable inhibitor against FVIII. A maximum of 40 evaluable (i.e., dosed) partici... | [] |
NCT03734588 | 4.1.1 | Sequence of Enrollment | 4.1.1 Sequence of Enrollment The dose level for Cohorts 1 & 2 will begin at 1x1012 concomitant medications. The following 2 staggering strategies are used in this study based on the recommendation from The Guidance for Industry: Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy P... | [] |
NCT03734588 | 4.1.1.1 | Dose-Level and Cohort Expansion | 4.1.1.1 Dose-Level and Cohort Expansion Dose-cohort expansion may occur under the following scenarios: Dose Level Expansion - After 2 participants are dosed in a given dose level, it may be possible to expand a cohort to up to 10 participants if there is evidence of FVIII:C increases above 5% of normal in either partic... | [
"Dose Escalation"
] |
NCT03734588 | 4.1.2 | FVIII Incremental Recovery | 4.1.2 FVIII Incremental Recovery If FVIII levels resulting from endogenous FVIII production from transduced hepatocytes begin to decline without evidence of elevation of transaminases, the participant will be evaluated for evidence of anti-FVIII antibody formation. This evaluation will include FVIII inhibitor assays an... | [] |
NCT03734588 | 4.1.3 | Corticosteroids | 4.1.3 Corticosteroids Based on observation and experience from earlier clinical studies of liver-directed AAV gene transfer, including the SJ-UCL trial (Nathwani, 2011b; Nathwani, 2014), the Sponsor's earlier clinical studies , and the Baxalta BAX 335 trial (NCT#01687608), participants may develop an immune response to... | [] |
NCT03734588 | 4.1.3.1 | Corticosteroid Regimen | 4.1.3.1 Corticosteroid Regimen | If transaminases become elevated or the FVIII declines, | |------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------... | [] |
NCT03734588 | 4.1.3.2 | Other Immunomodulatory Considerations | 4.1.3.2 Other Immunomodulatory Considerations | Prolonged courses of high dose oral corticosteroids are undesirable and associated with adverseevents.In the AASLD and the EASL guidelines for the treatment of autoimmune hepatitis, analternative regimen of corticosteroids and steroid sparing agents,, isoutlined for use i... | [] |
NCT03734588 | 4.1.3.4 | Experience with Long Term Immunomodulation | 4.1.3.4 Experience with Long Term Immunomodulation There is extensive experience with corticosteroids and immunosuppressive regimens in hemophilia: first, as a maneuver to eradicate antibodies to factor VIII or IX, clinically termed inhibitors (Nilsson, 1976; Hultin, 1976; Hay, 2006) and second, in the setting of liver... | [] |
NCT03734588 | 4.1.4 | Three to Seven Days Prior to Day 0 | 4.1.4 Three to Seven Days Prior to Day 0  | [] |
NCT03734588 | 4.1.5 | Day -2 | 4.1.5 Day -2  | [] |
NCT03734588 | 4.1.6 | Dosing Period (Days 0, 1) | 4.1.6 Dosing Period (Days 0, 1) | Assessments and procedures to be performed on Dosing Day (Day 0) are described in Table 1. | |-------------------------------------------------------------------------------------------------------| | All participants will administer a prophylactic infusion of 50 IU/kg of their current... | [] |
NCT03734588 | 4.1.7 | Follow-Up Observation Period | 4.1.7 Follow-Up Observation Period Participants who have received a dose of SPK-8016 will report to either the vectoradministration center or follow-up center for follow-up evaluations, according to the protocol assessments for 52 (±2) weeks after the infusion of SPK-8016. During the follow-up observation period, a qua... | [] |
NCT03734588 | 4.2 | Study Duration, Enrollment and Number of Sites | 4.2 Study Duration, Enrollment and Number of Sites | [] |
NCT03734588 | 4.2.1 | Duration of Study Participation | 4.2.1 Duration of Study Participation The study will consist of the following periods - Screening period (up to a maximum of 16 weeks) - Study Intervention: Assignment into cohort with possible pre-infusion immunosuppressive medication or administration of oral immunosuppressive medication, and then study product infus... | [] |
NCT03734588 | 4.2.2 | Total Number of Participants; Sites Projected and Geographic Regions | 4.2.2 Total Number of Participants; Sites Projected and Geographic Regions It is estimated that approximately 50 potential participants may be enrolled for a maximum of 40 evaluable (i.e., dosed) participants. The study is planned to be conducted at approximately 15 study centers (vector-administration centers and/or f... | [] |
NCT03734588 | 4.3 | Study Stopping Rules | 4.3 Study Stopping Rules The Sponsor may terminate this study at any time. The Sponsor, or designee, will notify the Investigator(s) and appropriate regulatory authorities if the study or any given study cohort is suspended, terminated, or completed. If the study or any given study cohort is suspended or terminated, th... | [] |
NCT03734588 | 5 | STUDY POPULATION | 5 STUDY POPULATION Prospective approval of protocol deviations to recruitment and enrollment criteria, also known as protocol waivers or exemptions, is not permitted. | [] |
NCT03734588 | 5.1 | Inclusion Criteria | 5.1 Inclusion Criteria Participants must meet all the following criteria at screening and prior to dosing of SPK-8016 (Day 0) to be eligible for the study: - 1. Be able to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (P... | [] |
NCT03734588 | 5.2 | Exclusion Criteria | 5.2 Exclusion Criteria Participants who meet any of the following criteria at screening or prior to dosing of SPK-8016 (Day 0) are not eligible for the study: - 1. Have active hepatitis B or C. All participants must be screened for both active hepatitis B and C regardless of prior known history. - a. Screening for hepa... | [] |
NCT03734588 | 5.3 | Screen Failures | 5.3 Screen Failures Screen failures are defined as participants who consent to participate in the clinical study but are not subsequently administered study drug. A minimal set of information will be collected for screen failures: demography, screen failure details, eligibility criteria, and, if applicable, any SAE. In... | [] |
NCT03734588 | 5.4 | Enrollment of Participants | 5.4 Enrollment of Participants Each participant will be assigned a unique participant identification (ID) number after providing written informed consent. The participant ID number will be used to identify the participant for the duration of the study. Participant ID numbers will not be reassigned or reused. No partici... | [] |
NCT03734588 | 5.5 | Randomization | 5.5 Randomization This is a non-randomized study; therefore, there is no randomization of patients. | [] |
NCT03734588 | 5.6 | Blinding Procedures | 5.6 Blinding Procedures This is an open-label study; therefore, there is no study blinding.  | [] |
NCT03734588 | 6 | STUDY PROCEDURES/ASSESSMENTS AND SCHEDULE | 6 STUDY PROCEDURES/ASSESSMENTS AND SCHEDULE Planned timepoints for all safety assessments are provided in [Table 1.](#page-25-0) | [] |
NCT03734588 | 6.1.1 | Clinical Safety Assessments | 6.1.1 Clinical Safety Assessments The following clinical assessments will be performed to assess the safety profile of SPK-8016: - Physical examination - Vital signs - AEs - Concomitant therapy and procedures. | [] |
NCT03734588 | 6.1.2 | Laboratory Safety Assessments | 6.1.2 Laboratory Safety Assessments The following laboratory tests will be performed to assess the safety profile of SPK-8016 - Hematology - Clinical chemistry - Urinalysis - Liver Function Tests - Coagulation - Neutralizing antibody development against FVIII by Bethesda assay (FVIII inhibitor) - Neutralizing antibody ... | [] |
NCT03734588 | 6.2 | Additional Assessments | 6.2 Additional Assessments | [] |
NCT03734588 | 6.2.1 | Joint Assessments | 6.2.1 Joint Assessments Baseline clinical status of joints and identification of target joint(s) will be conducted during Screening or pre-vector infusion. Joint health and identification of target joint(s) will be assessed during this study. A target joint is defined as a major joint (e.g., hip, elbow, wrist, shoulder... | [] |
NCT03734588 | 6.2.2 | Hemophilia Joint Health Score | 6.2.2 Hemophilia Joint Health Score Joint assessment will be conducted using a Hemophilia Joint Health Score (HJHS) (See Section [14.4\)](#page-143-0). HJHS will be performed to evaluate the clinical status of the joints at screening or prior to Day 0, Week 26, and Week 52. This assessment is based on the scoring syste... | [] |
NCT03734588 | 6.2.3 | Activity Assessments (Hemophilia Activities List) | 6.2.3 Activity Assessments (Hemophilia Activities List) Participants will complete the Hemophilia Activities List (HAL) questionnaire (See Section [14.1\)](#page-121-1) (van Genderen, 2004; van Genderen, 2006). In the questionnaire, several activities are listed that could be difficult for patients with hemophilia. | [] |
NCT03734588 | 6.2.4 | Participant Questionnaires | 6.2.4 Participant Questionnaires The following questionnaires will be administered at selected visits: - Quality-of-Life (See Section [14.2\)](#page-132-0): The Haem-A-QoL Questionnaire, a hemophiliaspecific QoL tool for hemophilia patients who are 17 years old and above, consists of 46 items covering 10 dimensions to ... | [] |
NCT03734588 | 6.2.5 | Health-economic Assessment | 6.2.5 Health-economic Assessment The following information will be captured during the study as part of a health-economic assessment: - a) Number of hospitalizations (excluding pre-planned hospitalizations documented at screening) - b) Number of hospitalization days - c) Number of emergency room visits - d) Number of p... | [] |
NCT03734588 | 6.2.6 | Archived Bio-samples | 6.2.6 Archived Bio-samples At each time-point where samples are collected for FVIII activity, spare plasma will also be collected. Samples will be archived for testing (if required) of further coagulation assays, or for  clarification of any clinical or laboratory AEs. If a participant provides ... | [] |
NCT03734588 | 6.3 | Clinical Procedures | 6.3 Clinical Procedures The clinical procedures that will be conducted during this study related to the evaluation of population demographics, medical and hemophilia history, safety, and efficacy are provided in [Table 3.](#page-75-1) Table 3: Clinical Procedures: Study Assessments | Assessment | Description | | | | | ... | [] |
NCT03734588 | 6.4 | Screening Period | 6.4 Screening Period All participants must provide written informed consent before any study-specific procedures or assessments are performed. All screening evaluations must be completed and reviewed to confirm that potential participants meet all eligibility criteria. The Investigator will maintain a screening log to ... | [] |
NCT03734588 | 6.4.1 | Screening Assessments | 6.4.1 Screening Assessments The following procedures will be performed during the screening period, which may occur over a period up to a maximum of 16 weeks: - Obtain written informed consent - Review inclusion/exclusion criteria - Review prior and concomitant therapy - Record information on demographic and baseline c... | [] |
NCT03734588 | 6.5 | Three to Seven Days Prior to Day 0 Assessments | 6.5 Three to Seven Days Prior to Day 0 Assessments Laboratory assessments should occur within 3 to 7 days prior to Day 0 for Cohorts 1 and 2. Results must be consistent with inclusion/exclusion criteria. - Liver Function (local lab) - Hematology (local lab) - Immunology (central lab) - Immune Profiling (central lab) - ... | [] |
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