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)
Trial status: Authorised
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
Change from Baseline to Week 78 in 10MWRT velocity (m/sec)
Description
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
Who can participate
Age group: 65+ years, 18-64 years
Gender: Female, Male
Target enrollment: 80 participants
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