Global Study of Del-desiran for the Treatment of DM1

Last updated: July 28, 2025
Sponsor: Avidity Biosciences, Inc.
Overall Status: Active - Not Recruiting

Phase

3

Condition

Muscular Dystrophy

Treatment

Placebo

AOC 1001 (del-desiran)

Clinical Study ID

NCT06411288
AOC 1001-CS3
  • Ages 16-65
  • All Genders

Study Summary

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1

  • Ability to walk independently (orthoses and ankle braces allowed) for at least 10meters at screening

Exclusion

Key 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

  • Diabetes that is not adequately controlled

  • History of decompensated heart failure within 3 months of screening. Participantswith preexisting pacemaker/ICD are not excluded.

  • Body Mass Index > 35 kg/m2 at Screening

  • Recently treated with an investigational drug or biological agent

  • Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline,whichever is longer, prior to baseline.

Note: Additional protocol defined Inclusion and Exclusion criteria apply

Study Design

Total Participants: 159
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 3
Study Start date:
May 30, 2024
Estimated Completion Date:
September 30, 2026

Study Description

The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks.

Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54.

After completion of Week 54 assessments, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval.

An Independent Data Monitoring Committee (IDMC) comprised of 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

  • The Ottawa Hospital

    Ottawa, Ontario K1Y 4E9
    Canada

    Site Not Available

  • CSSS de Chicoutimi

    Chicoutimi, Quebec G7H 5H6
    Canada

    Site Not Available

  • Montreal Neurological Institute

    Montreal, Quebec H3A 2B4
    Canada

    Site Not Available

  • Aarhus University Hospital

    Aarhus N, 8200
    Denmark

    Site Not Available

  • Rigshospitalet

    Kobenhavn, 2100
    Denmark

    Site Not Available

  • Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone

    Marseille, 13005
    France

    Site Not Available

  • AP-HP Hopital Pitie-Salpetriere

    Paris, 75013
    France

    Site Not Available

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    Munich, 80336
    Germany

    Site Not Available

  • Beaumont Hospital

    Dublin, D09 C562
    Ireland

    Site Not Available

  • Fondazione Serena Onlus - Centro Clinico NeMO Milano

    Milano, 20162
    Italy

    Site Not Available

  • Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore

    Rome, 00168
    Italy

    Site Not Available

  • Aomori Hospital

    Aomori-shi, Aomori 038-1331
    Japan

    Site Not Available

  • National Hospital Organization Osaka Toneyama Medical Center

    Osaka, 560-8552
    Japan

    Site Not Available

  • Osaka University Hospital

    Osaka, 565-0871
    Japan

    Site Not Available

  • National Center of Neurology and Psychiatry

    Tokyo, 187-8551
    Japan

    Site Not Available

  • National Center of Neurology and Psychiatry

    Toyko, 187-8551
    Japan

    Site Not Available

  • Maastricht University Medical Center

    Maastricht, 6229 HX
    Netherlands

    Site Not Available

  • Stichting Radboud Universitair Medisch Centrum

    Nijmegen, 6525 GA
    Netherlands

    Site Not Available

  • Hospital Universitario Donostia

    San Sebastian, 20014
    Spain

    Site Not Available

  • St. Georges University Hospitals NHS Foundation Trust

    London, SW17 0QT
    United Kingdom

    Site Not Available

  • University College London Hospital

    London, NW1 2BU
    United Kingdom

    Site Not Available

  • Royal Hallamshire Hospital

    Sheffield, S10 2JF
    United Kingdom

    Site Not Available

  • University of California, Los Angeles (UCLA)

    Los Angeles, California 90095
    United States

    Site Not Available

  • University of California, San Diego (UCSD)

    San Diego, California 92093
    United States

    Site Not Available

  • Stanford University

    Stanford, California 94305
    United States

    Site Not Available

  • University of Colorado

    Denver, Colorado 80045
    United States

    Site Not Available

  • University of Florida

    Gainesville, Florida 32608
    United States

    Site Not Available

  • University Research Center of South Florida

    Tampa, Florida 33612
    United States

    Site Not Available

  • Indiana University (IU)

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Kansas University Medical Center

    Kansas City, Kansas 66205
    United States

    Site Not Available

  • Kennedy Krieger Institute

    Baltimore, Maryland 21205
    United States

    Site Not Available

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts 02215
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • University of Rochester Medical Center

    Rochester, New York 14642
    United States

    Site Not Available

  • Duke University Medical Center

    Durham, North Carolina 27708
    United States

    Site Not Available

  • Wake Forest

    Winston-Salem, North Carolina 27157
    United States

    Site Not Available

  • University of Cincinnati Gardner Neuroscience Institute

    Cincinnati, Ohio 45219
    United States

    Site Not Available

  • Ohio State University

    Columbus, Ohio 43221
    United States

    Site Not Available

  • University of Pennsylvania

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • University of Texas Southwestern Medical Center

    Dallas, Texas 75390
    United States

    Site Not Available

  • Houston Methodist Neurological Institute

    Houston, Texas 77030
    United States

    Site Not Available

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Site Not Available

  • University of Washington

    Seattle, Washington 98104
    United States

    Site Not Available

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