Phase 3 Study of Intravenous Del-brax AOC 1020 for Treating Facioscapulohumeral Muscular Dystrophy FSHD

What this Trial Is About

Researchers are evaluating the efficacy and safety of intravenous AOC 1020, called Del-brax, in treating Facioscapulohumeral Muscular Dystrophy FSHD. This randomized, double-blind, placebo-controlled Phase 3 study aims to understand how Del-brax affects muscle function and other symptoms in people with this condition. The study is sponsored by Avidity Biosciences, Inc., and includes participants aged 16 to 70 years with a clinical and genetic diagnosis of FSHD1 or FSHD2. Participants will be randomly assigned to receive either Del-brax or a placebo through intravenous infusions every 6 weeks at the clinical study site. A total of 13 doses will be given over a 78-week active treatment period, concluding with a final assessment at Week 78. After this, participants may choose to join an optional open-label extension study if approved, or undergo a 12-week safety follow-up if they decline. During the study, participants will visit the clinic regularly for infusions and assessments. Researchers will monitor muscle strength and function using tests like Quantitative Muscle Testing QMT, 10-Meter WalkRun Test, Timed Up-and-Go, and patient questionnaires about physical function, fatigue, pain, and quality of life. Safety and tolerability will also be carefully reviewed throughout and after treatment. The total study duration is about 22 months, including screening, treatment, and follow-up periods.

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD