Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Completed · Phase 1/Phase 2 · Has a placebo group

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 Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Key facts

Study ID

NCT05747924

Run by

Avidity Biosciences, Inc.

People needed

90

Starts

2023-04-04

Expected to finish

2026-03-20

Last updated by the study team

2026-07-16

Who can join

Age: 16 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor)
• Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, walking stick or braces)
• At least 1 muscle region suitable for biopsy (testing provided by Sponsor)
• Muscle weakness in both upper and lower body, as determined by Investigator

You may not qualify if…

• Pregnant or intends to become pregnant while on study, or active breastfeeding
• Unwilling or unable to continue to comply with contraceptive requirements
• Body mass index (BMI) >35.0 kg/m2 at Screening
• History of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the study
• History of bleeding disorders, significant keloid, or other skin or muscle conditions (e.g., severe muscle wasting) that, in the opinion of the Investigator, makes the participant unsuitable for serial muscle biopsy
• Anticipated survival less than 2 years
• Blood or plasma donation within 16 weeks of Study Day 1
• Any contraindication to MRI
• Any abnormal lab values, conditions or diseases 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
• Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening

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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