A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy

Condition studied

Duchenne muscular dystrophy

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change from baseline in time to rise velocity at Day 540

Endpoint detail

Change from baseline in stride velocity 95th centile (SV95C) by activity monitoring using the Syde wearable device at Day 540, Change from baseline in 10-meter walk/run velocity at Day 540, Change from baseline in 4-stair climb velocity at Day 540, Change from baseline in absolute NSAA score at Day 540, Cumulative loss of function in North Star Ambulatory Assessment (NSAA) items at Day 540, Change from baseline in microdystrophin protein levels at Day 90, Change from baseline in microdystrophin tissue distribution at Day 90, Change from baseline in % predicted forced vital capacity (FVC) at Day 540, Change from baseline in % predicted peak expiratory flow (PEF) at Day 540, Change from baseline in % predicted forced expiratory volume in 1 second (FEV1) at Day 540, Incidence of treatment-emergent adverse events through Day 540, Incidence of serious adverse events through Day 540, Incidence of adverse events of special interest through Day 540, Incidence of clinically significant changes in ECGs through Day 540, Incidence of clinically significant changes in ECHOs through Day 540, Change from baseline in the PODCI Global score at Day 540.