A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)
Sponsor: Sarepta Therapeutics, Inc. + Hoffmann-La Roche
No open prediction endpoints
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Primary Endpoints (CT.gov)
Part 1: Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
Time frame: Baseline, Week 52 (Part 1)
Secondary Endpoints
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by Western Blot Adjusted by Muscle Content
Part 1: Change From Baseline in Time to Rise From the Floor at Week 52
Part 1: Change From Baseline in Time to Complete 10 Meter Walk/Run (10MWR) at Week 52
Eligibility Criteria
Inclusion Criteria: * Is ambulatory and from 4 to under 8 years of age at time of randomization. * Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. * Ability to cooperate with motor assessment testing. * Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). * rAAVrh74 antibody titers are not elevated as per protocol-specified requirements. * A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45. 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 clinica…
Read full criteria on CT.gov →✦ Analyst Commentary
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