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.
You may not be if
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.
Clareo Health | A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
