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
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Locations
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AP-HP Hopital Pitie-Salpetriere
Paris, 75013, France
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Aarhus University Hospital
Aarhus N, 8200, Denmark
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Aomori Hospital
Aomori, Aomori, 038-1331, Japan
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Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
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Duke University Medical Center
Durham, North Carolina, 27708, United States
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Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan, 20162, Italy
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Hospital Universitario Donostia
Donostia / San Sebastian, 20014, Spain
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Houston Methodist Neurological Institute
Houston, Texas, 77030, United States
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Indiana University (IU)
Indianapolis, Indiana, 46202, United States
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Kansas University Medical Center
Kansas City, Kansas, 66205, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich, 80336, Germany
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Maastricht University Medical Center
Maastricht, 6229 HX, Netherlands
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Montreal Neurological Institute
Montreal, Quebec, H3A 2B4, Canada
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National Center of Neurology and Psychiatry
Tokyo, 187-8551, Japan
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National Hospital Organization Osaka Toneyama Medical Center
Osaka, 560-8552, Japan
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Ohio State University
Columbus, Ohio, 43221, United States
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Osaka University Hospital
Osaka, 565-0871, Japan
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Rigshospitalet
Copenhagen, 2100, Denmark
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St. Georges University Hospitals NHS Foundation Trust
London, SW17 0QT, United Kingdom
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Stanford University
Stanford, California, 94305, United States
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Stichting Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA, Netherlands
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The Ottawa Hospital
Ottawa, Ontario, K1Y 4E9, Canada
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University College London Hospital
London, NW1 2BU, United Kingdom
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University Research Center of South Florida
Tampa, Florida, 33612, United States
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University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio, 45219, United States
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University of Colorado
Denver, Colorado, 80045, United States
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University of Florida
Gainesville, Florida, 32608, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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University of Washington
Seattle, Washington, 98104, United States
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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
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Wake Forest
Winston-Salem, North Carolina, 27157, United States
Conditions
Explore the condition pages connected to this study.