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

Last updated: July 25, 2025
Sponsor: Avidity Biosciences, Inc.
Overall Status: Active - Recruiting

Phase

3

Condition

Muscular Dystrophy

Treatment

Placebo

AOC-1020

Clinical Study ID

NCT07038200
AOC 1020-CS3
  • Ages 16-70
  • All Genders

Study Summary

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)

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Clinical and genetic diagnosis of FSHD1 or FSHD2

  • Ability to walk independently at pre-specified walking speed (orthoses and anklebraces allowed) for at least 10 meters at screening

  • Adequate muscle strength based on QMT composite score

Exclusion

Exclusion Criteria:

  • Breastfeeding, pregnancy, or intent to become pregnant during the study

  • Unwilling or unable to comply with contraceptive requirements

  • Abnormal lab values, conditions or diseases that would make the participantunsuitable for the study

  • Blood Pressure > 140/90 mmHg at Screening

  • Treatment with another investigational drug or biological agent within 1 month ofScreening or 5 half-lives of the drug, whichever is longer

  • Treatment with an oligonucleotide within 9 months of Screening

Study Design

Total Participants: 200
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 3
Study Start date:
June 10, 2025
Estimated Completion Date:
July 31, 2028

Study Description

The study consists of a Screening Period of up to 6 weeks and 72-week Treatment Period. The anticipated duration is approximately 78 weeks.

Participants will be randomized to receive an intravenous infusion of either del-brax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78.

After completion of the Week 78 visit, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for a period of 12 weeks for safety.

An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Connect with a study center

  • University of California Irvine

    Orange, California 92868
    United States

    Site Not Available

  • Stanford University

    Palo Alto, California 94304
    United States

    Site Not Available

  • University of Colorado

    Denver, Colorado 80045
    United States

    Site Not Available

  • University of Florida

    Gainesville, Florida 32608
    United States

    Active - Recruiting

  • University of Iowa

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • Kansas University Medical Center

    Kansas City, Kansas 66205
    United States

    Active - Recruiting

  • Kennedy Krieger Institute

    Baltimore, Maryland 21205
    United States

    Site Not Available

  • University of Massachusetts

    Worcester, Massachusetts 01655
    United States

    Site Not Available

  • University of Rochester Medical Center

    Rochester, New York 14642
    United States

    Active - Recruiting

  • Duke University

    Durham, North Carolina 27708
    United States

    Site Not Available

  • Ohio State University

    Columbus, Ohio 43221
    United States

    Site Not Available

  • University of Pennsylvania

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Active - Recruiting

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