This study evaluates the gene therapy delandistrogene moxeparvovec (SRP-9001) compared with placebo in children with Duchenne muscular dystrophy (DMD), a progressive muscle disease. Patients with a clinical diagnosis of DMD and a documented dystrophin gene mutation, with a frameshift mutation between exons 18 and 58, were able to take part. The study was carried out in Phase 1/Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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.