A Phase 3 Study to Evaluate AOC 1020 in Participants with FSHD
Research type
Research Study
Full title
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)
IRAS ID
1011931
Contact name
Nirvana Moodley
Contact email
Sponsor organisation
Avidity Biosciences, Inc.
Research summary
Facioscapulohumeral Muscular Dystrophy (FSHD) is a disease that causes muscle weakness in the face, shoulder blades, arms, and legs. People with FSHD have a genetic abnormality that causes DUX4 protein to be present in their muscle when it should not be. The presence of DUX4 protein leads to muscle loss and decreased muscle function. Muscle weakness caused by FSHD worsens over time and can be disabling. Many patients have trouble talking, eating, making facial expressions, reaching and lifting, maintaining posture, and walking. Currently, there are no drugs available to treat FSHD.
The treatment offered in this study, called AOC 1020 (also known as del-brax), is designed to reduce the amount of DUX4.
The main purpose of this study is to measure how efficacy and safety of AOC 1020 in participants with FSHD. Participants will either receive AOC 1020 (the active drug) or placebo by intravenous infusion for a total of 13 doses, given once every 6 weeks. Participants and study staff will not know which treatment (AOC 1020 or placebo) each participant receives.
Throughout the study, participants will be asked to perform tests such as those that measure the strength of arm and leg muscles, and the time it takes to perform short walking assessments. Participants will also answer questions about their health and day to day activities.
Based on the way that AOC 1020 is believed to act (to reduce the amount of DUX4 protein that causes FSHD), study participants who are treated with AOC 1020 may experience improvement in FSHD symptoms. However, as with any clinical study, participants may not benefit directly from study participation. Risks of participating in this study include risks associated with study procedures and potential risks associated with the study drug.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
25/SC/0157
Date of REC Opinion
25 Jun 2025
REC opinion
Further Information Favourable Opinion