New hope for FSHD: experimental drug AOC 1020 completes early testing

NCT ID NCT05747924

Completed Disease control Sponsor: Avidity Biosciences, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a new medicine called AOC 1020 in 90 adults with facioscapulohumeral muscular dystrophy (FSHD), a genetic condition that causes muscle weakness. The goal was to check if the drug is safe and how the body processes it. Participants received either the drug or a placebo by IV, and researchers measured side effects and signs of muscle improvement.

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Conditions

The condition(s) this trial relates to.

facioscapulohumeral muscular dystrophy facioscapulohumeral muscular dystrophy 1 facioscapulohumeral muscular dystrophy 2 frontometaphyseal dysplasia frontometaphyseal dysplasia 2 muscular dystrophy progressive muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ATROPHIES, FACIOSCAPULOHUMERAL ATROPHY, FACIOSCAPULOHUMERAL DYSTROPHIES, FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHIES, LANDOUZY-DEJERINE DYSTROPHY, FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY, LANDOUZY-DEJERINE FACIO-SCAPULO-HUMERAL DYSTROPHY FACIOSCAPULOHUMERAL ATROPHY FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY 1 FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY 2 FASCIOSCAPULOHUMERAL MUSCULAR DYSTROPHY FASCIOSCAPULOHUMERAL MUSCULAR DYSTROPHY TYPE 1 FASCIOSCAPULOHUMERAL MUSCULAR DYSTROPHY TYPE 2 FMD FMD2 FSH FSH MUSCULAR DYSTROPHY FSHD FSHD1 FSHD2 LANDOUZY DEJERINE DYSTROPHY LANDOUZY-DEJERINE MUSCULAR DYSTROPHY LANDOUZY-DEJERINE SYNDROME MUSCULAR DYSTROPHIES MUSCULAR DYSTROPHY, FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY, LANDOUZY DEJERINE PROGRESSIVE MUSCULAR DYSTROPHY

Contacts and locations

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Locations

Duke University

Durham, North Carolina, 27708, United States
Kansas University Medical Center

Kansas City, Kansas, 66205, United States
Ohio State University

Columbus, Ohio, 43221, United States
Rare Disease Research

Atlanta, Georgia, 30329, United States
Stanford University

Palo Alto, California, 94304, United States
University College London

London, WIT 7HA, United Kingdom
University of California Los Angeles

Los Angeles, California, 90095, United States
University of California San Diego

San Diego, California, 92093, United States
University of Colorado

Denver, Colorado, 80045, United States
University of Florida

Gainesville, Florida, 32608, United States
University of Ottawa

Ottawa, Ontario, K1Y 4E9, Canada
University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States
University of Rochester Medical Center

Rochester, New York, 14642, United States
University of Sheffield

Sheffield, S10 2TN, United Kingdom
University of Texas Southwestern

Dallas, Texas, 75390, United States
University of Washington

Seattle, Washington, 98104, United States
Virginia Commonwealth University

Richmond, Virginia, 23298, United States