A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
- Conditions
- Muscular Dystrophy, Duchenne
- Registration Number
- NCT04626674
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active, not recruiting
- Sex
- Male
- Target Recruitment
- 55
Inclusion Criteria:<br><br> - For Cohorts 1-7: Has a definitive diagnosis of DMD based on documented clinical<br> findings and prior genetic testing.<br><br> - Cohort 1: Is ambulatory, and =4 to <8 years of age at the time of Screening.<br><br> - Cohort 2: Is ambulatory, and =8 to <18 years of age at the time of Screening.<br><br> - Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening.<br><br> - Cohort 4: Is ambulatory and =3 to <4 years of age at the time of Screening.<br><br> - Cohort 5a: Is ambulatory and =4 to <9 years of age.<br><br> - Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening.<br><br> - Cohort 6: Is ambulatory, and =2 to <3 years of age at the time of Screening.<br><br> - Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening.<br><br> - Ability to cooperate with motor assessment testing.<br><br> - Cohorts 1, 2, 3, 5, and 7 only: Stable dose equivalent of oral glucocorticoids for<br> at least 12 weeks before screening and the dose is expected to remain constant<br> (except for modifications to accommodate changes in weight) throughout the first<br> year of the study.<br><br> - Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their<br> DMD, in the opinion of the Investigator, and are not receiving steroids at the time<br> of Screening.<br><br> - rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.<br><br> - Genetic mutation inclusion criteria vary by cohort.<br><br>Exclusion Criteria:<br><br> - Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or<br> cognitive delay/impairment that in the opinion of the Investigator creates<br> unnecessary risks for gene transfer.<br><br> - Exposure to gene therapy, investigational medication, or any treatment designed to<br> increase dystrophin expression within protocol-specified time limits.<br><br> - Abnormality in protocol-specified diagnostic evaluations or laboratory tests.<br><br>Other inclusion/exclusion criteria apply.
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Part 1: Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot;Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
- Secondary Outcome Measures
Name Time Method Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion;Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74);Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events (AEs) of Special Interest;Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity;Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF);Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity:;Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF