A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Condition studied

Facioscapulohumeral Muscular Dystrophy (FSHD)

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change from Baseline to Week 78 in 10MWRT velocity (m/sec)

Endpoint detail

1.1 Key Secondary Endpoints • Change from Baseline to Week 78 in: o Timed-Up-and-Go (TUG) velocity o NeuroQoL Upper Extremity Function o Quantitative Muscle Testing (QMT) total composite score (PPN), 1.2 Other Secondary Endpoints • Change from Baseline over time in: o Patient-reported Outcomes Measurement Information System instruments (PROMIS) (Physical Function) o PROMIS Fatigue o Worst Pain Numerical Rating Scale (NRS) o Patient Global Impression of Severity (PGI-S)/Patient Global Impression of Change (PGI-C) o Quality of Life in Neurological Disorders (NeuroQoL) Sleep Disturbance o DUX4-regulated plasma KHDC1L o Serum CK