Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
Status:
ACTIVE_NOT_RECRUITING
Trial end date:
2026-06-30
Target enrollment:
Participant gender:
Summary
Phase 2a trial of SAT-3247 in ambulatory DMD patients aged 7 and \< 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy.