A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)
Sponsor: Sarepta Therapeutics, Inc.
No open prediction endpoints
Endpoints are classified and published by ProgramSignal analysts.
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Primary Endpoints (CT.gov)
Number of Participants With Adverse Events (AEs)
Time frame: Up to 5 years
Secondary Endpoints
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Western Blot
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by Immunofluorescence (IF) Fiber Intensity
Change From Baseline at Day 90 in Delandistrogene Moxeparvovec Dystrophin Expression as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Eligibility Criteria
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 clinica…
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