This study evaluates a gene therapy called SGT-003, compared with placebo, in ambulatory males with a confirmed diagnosis of Duchenne muscular dystrophy (DMD). Participants are expected to have a DMD gene mutation, meet certain walking-test criteria, and be on a stable steroid regimen. The study is in Phase 3 and is currently recruiting participants.
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: * Participant is ambulatory. * Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype. * Negative for antibodies against adeno-associated virus serotype 9 (AAV9). * On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (mg/kg/day) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice. * Meet 10-meter walk/run time criteria. * Meet time to rise from supine criteria. * Participant has bodyweight ≤50 kg. Exclusion Criteria: * Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy. * Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment. * Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report. Other Inclusion/Exclusion criteria to be applied as per protocol.