A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients

Duchenne muscular dystrophy

The primary efficacy endpoint is defined as the change from baseline in muscle force measurements as determined by dynamometry at Week 12., Safety endpoints include incidence, severity, and relationship to SAT-3247 of adverse events as well as occurrence of clinically significant changes in physical examination, clinical laboratory measures, vital signs, ECG, and C-SSRS

Changes from baseline in intramuscular fat fraction in muscle quantitative magnetic resonance (qMR) in vastus lateralis at Week 12, Changes from baseline in proton muscle transverse relaxation time (T2) in vastus lateralis at Week 12., Changes from baseline in Regeneration Index in open muscle biopsy of the biceps brachii at Week 12, Changes from baseline in function as determined by NSAA assessment at Week 12, Changes from baseline in SV95C at Week 12, Exploratory end point: change from baseline in inflammatory cytokine profile at Week 12, Exploratory end point: change from baseline in creatine kinase at Week 12, Exploratory end point: change from baseline in maximum percent predicted forced vial capacity as measured by spirometry at Week 12, Exploratory end point: change in biceps brachii muscle fiber size and fiber size distribution as determined from histopathology at 12 weeks, Exploratory end point: change in the proportion of embryonic myosin positive fibers as determined from histopathology at 12 weeks., Exploratory end point: change in the number of satellite cells as determined from histopathology at 12 weeks, Exploratory end point: change in endomysial fibrosis and adipose tissue infiltration as determined from histopathology at 12 weeks., Exploratory end point: change in NSAA score over 12 weeks as compared to natural history

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