RecruitingPhase 3NCT07038200

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

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)


Sponsor

Avidity Biosciences, Inc.

Enrollment

200 participants

Start Date

Jun 10, 2025

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 16 YearsMax Age: 70 Years

Inclusion Criteria3

  • Clinical and genetic diagnosis of FSHD1 or FSHD2
  • Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening
  • Adequate muscle strength based on QMT composite score

Exclusion Criteria6

  • 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 participant unsuitable for the study
  • Blood Pressure > 140/90 mmHg at Screening
  • Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer
  • Treatment with an oligonucleotide within 9 months of Screening

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Interventions

DRUGAOC-1020

Placebo

DRUGPlacebo

Placebo will be administered by intravenous (IV) infusion.


Locations(22)

University of California Irvine

Orange, California, United States

Stanford University

Palo Alto, California, United States

University of Colorado

Denver, Colorado, United States

University of Florida

Gainesville, Florida, United States

University of Iowa

Iowa City, Iowa, United States

Kansas University Medical Center

Kansas City, Kansas, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

University of Massachusetts

Worcester, Massachusetts, 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 Health Science Center at San Antonio

San Antonio, Texas, United States

Virginia Commonwealth University

Richmond, Virginia, United States

University of Calgary - Cumming School of Medicine

Calgary, Alberta, Canada

University of Alberta Hospital

Edmonton, Alberta, Canada

The Ottawa Hospital

Ottawa, Ontario, Canada

Genge Partners Inc

Montreal, Quebec, Canada

National Hospital Organization Osaka Toneyama Medical Center

Osaka, Japan

The University of Osaka Hospital

Osaka, Japan

National Hospital Organization Sendai Nishitaga Hospital

Sendai, Japan

National Center of Neurology and Psychiatry

Tokyo, Japan

View Full Details on ClinicalTrials.gov

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NCT07038200


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