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

Running, not enrolling · Phase 2

Conditions studied: FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH

In brief

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

Key facts

Study ID

NCT06547216

Run by

Avidity Biosciences, Inc.

People needed

84

Starts

2024-07-25

Expected to finish

2030-06-01

Last updated by the study team

2026-07-14

Who can join

Age: 16 and older, up to 72. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.
• Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor:
• No significant tolerability issues
• Satisfactory compliance with the AOC 1020-CS1 protocol requirements

You may not qualify if…

• Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding.
• Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2.
• Any new conditions or worsening of existing condition(s) that in the opinion of the Investigator or Sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study. (Note: FSHD progression is not exclusionary, even if the participant no longer has the ability to walk 10 meters without a walker or two canes.)

Where it is running

• University of California Los Angeles — Los Angeles, California, United States
• Stanford University — Palo Alto, California, United States
• University of California San Diego — San Diego, California, United States
• University of Colorado — Denver, Colorado, United States
• University of Florida — Gainesville, Florida, United States
• Rare Disease Research — Atlanta, Georgia, United States
• Kansas University Medical Center — Kansas City, Kansas, United States
• University of Rochester Medical Center — Rochester, New York, United States
• Duke University — Durham, North Carolina, United States
• Ohio State University — Columbus, Ohio, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States
• University of Texas Southwestern — Dallas, Texas, United States
• Virginia Commonwealth University — Richmond, Virginia, United States
• University of Washington — Seattle, Washington, United States
• University of Ottawa — Ottawa, Ontario, Canada
• University College London — London, United Kingdom
• University of Sheffield — Sheffield, United Kingdom

Full record on ClinicalTrials.gov

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