Hope for DM1: new drug tested for long-term safety

NCT ID NCT07008469

First seen Nov 01, 2025 · Last updated May 08, 2026 · Updated 30 times

Summary

This study looks at the long-term safety and effects of a drug called AOC 1001 in people with myotonic dystrophy type 1 (DM1), a genetic muscle disease. About 230 adults who already took part in earlier AOC 1001 studies will receive the drug intravenously. The main goal is to track any side effects over time.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • AP-HP Hopital Pitie-Salpetriere

    Paris, 75013, France

  • Aomori Hospital

    Aomori, Aomori, 038-1331, Japan

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215, United States

  • Duke University Medical Center

    Durham, North Carolina, 27708, United States

  • Fondazione Serena Onlus - Centro Clinico NeMO Milano

    Milan, 20162, Italy

  • Houston Methodist Neurological Institute

    Houston, Texas, 77030, United States

  • Indiana University (IU)

    Indianapolis, Indiana, 46202, United States

  • Kansas University Medical Center

    Kansas City, Kansas, 66205, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen

    München, 80336, Germany

  • Montreal Neurological Institute

    Montreal, Quebec, H3A 2B4, Canada

  • National Hospital Organization Osaka Toneyama Medical Center

    Osaka, 560-8552, Japan

  • Ohio State University

    Columbus, Ohio, 43221, United States

  • Osaka University Hospital

    Osaka, 565-0871, Japan

  • Stanford University

    Stanford, California, 94305, United States

  • Stichting Radboud Universitair Medisch Centrum

    Nijmegen, 6525 GA, Netherlands

  • The Ottawa Hospital

    Ottawa, Ontario, K1Y 4E9, Canada

  • University Research Center of South Florida

    Tampa, Florida, 33612, United States

  • University of California, Los Angeles (UCLA)

    Los Angeles, California, 90095, United States

  • University of Colorado

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Washington

    Seattle, Washington, 98104, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

  • Wake Forest

    Winston-Salem, North Carolina, 27157, United States

Conditions

Explore the condition pages connected to this study.