Phase 2 Open-label Extension Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)

A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, and Pharmacokinetics of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

This study will continue to evaluate the safety, tolerability, PK, and efficacy of AOC 1020 in participants who were treated in the randomized, placebo-controlled, Phase 1/2 AOC 1020-CS1 clinical study.

Participants from AOC 1020-CS1 are eligible to enroll in AOC 1020-CS2 if they have satisfactorily completed AOC 1020-CS1. All participants who enroll in AOC 1020-CS2 will receive AOC 1020 regardless of whether they received AOC 1020 or placebo in AOC 1020-CS1. Dosing will occur quarterly (i.e., every 13 weeks).

The total duration of active treatment in AOC 1020-CS2 is approximately 24 months. Once participants have completed active treatment, they will be followed through a 9-month safety follow-up period, which consists of a minimum of quarterly visits (which may be telehealth visits) to monitor for adverse events (AEs), concomitant medications, and pregnancy status (as applicable). Total duration on study is up to approximately 33 months (2 years 9 months), excluding the potential 3-month screening period.