Experimental drug aims to control debilitating muscle disease

NCT ID NCT06411288

Summary

This global Phase 3 trial is testing whether an intravenous drug called AOC 1001 can help control symptoms of myotonic dystrophy type 1, a genetic muscle-wasting disease. About 159 adults with a confirmed diagnosis will receive either the drug or a placebo via infusion every 8 weeks for about a year. Researchers will measure changes in hand function, muscle strength, and walking ability to see if the treatment is effective and safe.

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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.