Phase 2a Study of Oral SAT-3247 for Boys with Duchenne Muscular Dystrophy Aged 7 to 9 Years Randomized, Double-Blind, Placebo-Controlled Trial to Find the Best Dose and Safety

What this Trial Is About

Researchers are studying SAT-3247 in boys aged 7 to under 10 years with Duchenne Muscular Dystrophy DMD who can walk. This phase 2a trial aims to find the best dose and assess the safety, tolerability, and early effects of SAT-3247 compared to a placebo. The study is randomized, double-blind, and placebo-controlled and takes place globally with up to 51 participants. Participants will take either 60 mg or 120 mg of SAT-3247 or a placebo pill daily for 12 weeks. The 120 mg dose is tested outside the US and Canada, while the 60 mg dose and placebo are studied in all locations. The trial includes a screening period, a baseline visit, and follow-up visits at weeks 1 phone call, 4, 8, and 12. During the study, participants will be monitored with clinical visits and phone calls to assess muscle strength, quality, function, and regeneration. Researchers will track safety and tolerability over the 12 weeks of treatment. The goal is to understand how SAT-3247 affects these outcomes while ensuring participant safety throughout the trial duration.

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients