This study examines a microdystrophin gene therapy called SGT-001 in adolescents and children with Duchenne muscular dystrophy caused by mutations in the DMD gene. It enrolled patients with a clinical DMD diagnosis and a confirmed dystrophin gene mutation whose cardiac and respiratory function was stable. The trial is in Phase 1/Phase 2 and is active but not currently recruiting.
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 predictive of DMD phenotype * Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants) * Anti-AAV9 antibodies below protocol-specified thresholds * Stable cardiac and pulmonary function * Adolescents: non-ambulatory by protocol-specified criteria * Children: ambulatory by protocol-specified criteria * Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks Exclusion Criteria: * Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results * Abnormal liver function * Abnormal renal function * Clinically significant coagulation abnormalities * Impaired cardiovascular function based on cardiac MRI or ECHO * Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support * Significant spinal deformity or presence of spinal rods * Body mass index ≥ 95th percentile for age * Exposure to another investigational drug within 3 months or 5 half-lives prior to screening * Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening Additional inclusion/exclusion criteria may apply.