Duchenne Muscular Dystrophy Clinical Trial
Official title:
Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)
Verified date | April 2024 |
Source | Sarepta Therapeutics, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.
Status | Completed |
Enrollment | 4 |
Est. completion date | April 25, 2023 |
Est. primary completion date | April 25, 2023 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 3 Months to 7 Years |
Eligibility | Inclusion Criteria: - Cohort A participants: 3 months to 3 years of age, inclusive - Cohort B participants: 4 to 7 years of age, inclusive - Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. - Ability to cooperate with motor assessment testing. - Cohort A participants: No previous treatment with corticosteroids. - Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study. - Cohorts A & B: A frameshift mutation contained between exons 18 and 58 (inclusive). Exclusion Criteria: - Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits. - Abnormality in protocol-specified diagnostic evaluations or laboratory tests. - Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer. Other inclusion or exclusion criteria could apply. |
Country | Name | City | State |
---|---|---|---|
United States | Nationwide Children's Hospital | Columbus | Ohio |
Lead Sponsor | Collaborator |
---|---|
Sarepta Therapeutics, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Participants With Adverse Events (AEs) | An AE is any untoward medical occurrence in a clinical study participant that does not necessarily have a causal relationship with the study drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of a study drug, whether or not considered related to the study drug. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module. | Up to 5 years | |
Secondary | Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Western Blot | Baseline muscle biopsies with ultrasound guidance were performed pre-treatment and post-treatment (Day 90) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin protein levels in these muscle biopsy samples was determined by Western blot. An increase in protein expression indicates production of the delandistrogene moxeparvovec dystrophin protein. | Baseline, Day 90 | |
Secondary | Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Immunofluorescence (IF) Fiber Intensity | Baseline muscle biopsies with ultrasound guidance were performed pre-treatment and post-treatment (Day 90) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin expression in these muscle biopsy samples was determined using IF. Automated software was used to quantify the intensity of dystrophin expression post-treatment compared to pre-treatment (Percent Normal). The number of muscle fibers expressing micro-dystrophin was quantified by independent trained evaluators. An increase in IF fiber intensity indicates increased delandistrogene moxeparvovec dystrophin expression. | Baseline, Day 90 | |
Secondary | Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by IF Percent Dystrophin Positive Fibers (PDPF) | Baseline muscle biopsies with ultrasound guidance were performed pre-treatment and post-treatment (Day 90) on all participants. The change from baseline in delandistrogene moxeparvovec dystrophin expression in these muscle biopsy samples was determined by IF PDPF. Automated software was used to quantify the intensity of dystrophin expression post-treatment compared to pre-treatment (Percent Normal). The number of muscle fibers expressing micro-dystrophin was quantified by independent trained evaluators. An increase in IF PDPF indicates increased delandistrogene moxeparvovec dystrophin expression. | Baseline, Day 90 | |
Secondary | Change From Baseline at Year 5 in the 100 Meter Timed Test | This assessment measures the time needed to move 100 meters and served as the primary motor outcome measure for this study. A decrease in the time needed to move 100 meters indicates increased motor function. | Baseline, Year 5 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT05575648 -
Dual Task in Duchenne Muscular Dystrophy
|
N/A | |
Terminated |
NCT03907072 -
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
|
Phase 2/Phase 3 | |
Not yet recruiting |
NCT06450639 -
An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT04335942 -
Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor
|
N/A | |
Active, not recruiting |
NCT04906460 -
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT02500381 -
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
|
Phase 3 | |
Enrolling by invitation |
NCT05967351 -
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
|
Phase 3 | |
Recruiting |
NCT03067831 -
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Recruiting |
NCT01834040 -
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Completed |
NCT02246478 -
A Study of TAS-205 for Duchenne Muscular Dystrophy
|
Phase 1 | |
Active, not recruiting |
NCT01772043 -
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
|
N/A | |
Terminated |
NCT01168908 -
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT00758225 -
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
|
Phase 2 | |
Completed |
NCT03680365 -
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
|
||
Recruiting |
NCT03513367 -
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
|
||
Recruiting |
NCT05712447 -
Duchenne Muscular Dystrophy Video Assessment Registry
|
||
Recruiting |
NCT01484678 -
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
|
||
Completed |
NCT03319030 -
Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
|
||
Terminated |
NCT01753804 -
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
|
N/A | |
Completed |
NCT02530905 -
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
|
Phase 1 |