A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping

Condition studied

Duchenne muscular dystrophy

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change from baseline to Week 54 in TTR velocity

Endpoint detail

1. Change from baseline to Week 54 as per protocol, 2.1 Change from Baseline as per protocol, 2.2 • Change from Baseline (all visits except Week 54) o creatine kinase o 4 stair climb velocity o 10mWRT velocity o SV95C o TTR velocity o NSAA o DMD-QoL o Patient/Caregiver Global Impression of Severity o QMT • Patient/Caregiver Global Impression of Change (all visits except Week 54), 3. • Incidence of TEAEs • Changes in vital signs • Changes in laboratory parameters • Changes in ECGs