Hope for muscle disease: new drug enters final testing phase

NCT ID NCT06411288

First seen Jan 26, 2026 · Last updated May 13, 2026 · Updated 19 times

Summary

This study tests a new medicine called AOC 1001 for people with myotonic dystrophy type 1, a genetic disease that causes muscle weakness and stiffness. About 159 adults with a confirmed diagnosis will receive either the drug or a placebo by IV. The main goal is to see if the drug improves hand function, grip strength, and walking ability.

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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

  • Aarhus University Hospital

    Aarhus N, 8200, Denmark

  • 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

  • Hospital Universitario Donostia

    Donostia / San Sebastian, 20014, Spain

  • 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

    Munich, 80336, Germany

  • Maastricht University Medical Center

    Maastricht, 6229 HX, Netherlands

  • Montreal Neurological Institute

    Montreal, Quebec, H3A 2B4, Canada

  • National Center of Neurology and Psychiatry

    Tokyo, 187-8551, Japan

  • 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

  • Rigshospitalet

    Copenhagen, 2100, Denmark

  • St. Georges University Hospitals NHS Foundation Trust

    London, SW17 0QT, United Kingdom

  • 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 College London Hospital

    London, NW1 2BU, United Kingdom

  • University Research Center of South Florida

    Tampa, Florida, 33612, United States

  • University of Cincinnati Gardner Neuroscience Institute

    Cincinnati, Ohio, 45219, United States

  • University of Colorado

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, 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.