A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001
Sponsor: Sarepta Therapeutics, Inc.
No open prediction endpoints
Endpoints are classified and published by ProgramSignal analysts.
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Primary Endpoints (CT.gov)
Change From Baseline at Week 12 in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content
Time frame: Baseline, Week 12 (Part 1)
Change From Baseline at Week 48 in NSAA Total Score
Time frame: Baseline, Week 48 (Part 1)
Secondary Endpoints
Change From Baseline at Week 48 in Time to Rise From the Floor
Change From Baseline at Week 48 in Time to Ascend 4 Steps
Change From Baseline at Week 48 in Time of 10-meter Timed Test
Eligibility Criteria
Inclusion Criteria: * Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype. * Indication of symptomatic muscular dystrophy by protocol-specified criteria. * Ability to cooperate with motor assessment testing. * Stable dose equivalent of oral corticosteroids for at least 12 weeks. * A frameshift mutation contained between exons 18 and 58 (inclusive). Exclusion Criteria: * Impaired cardiovascular function on echocardiogram. * Prior or ongoing medical condition on physical examination, electrocardiogram, or laboratory findings that could adversely affect participant safety, compromise completion of follow-up, or impair assessment of study results. * Exposure to another investigational drug or exon skipping medication within 6 months of screening. * Exposure to an investigational or commercial gene therapy product. * Abnormal liver or renal function by protocol-specified criteria. Other inclusion/exclusion criteria apply.
✦ Analyst Commentary
Expert commentary on why this trial matters and what to watch for.
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