Four-Year trial tests potential new treatment for debilitating muscle disease
NCT ID NCT07008469
Summary
This study is testing the long-term safety and effectiveness of an investigational drug called del-desiran for adults with myotonic dystrophy type 1 (DM1), a genetic muscle-wasting disease. Participants who completed a previous related study will receive an intravenous infusion of the drug every eight weeks for up to four years. The main goal is to see how well people tolerate the treatment over a long period and if it continues to help control the disease.
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Contacts and locations
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Locations
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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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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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Montreal Neurological Institute
Montreal, Quebec, H3A 2B4, Canada
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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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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 of California, Los Angeles (UCLA)
Los Angeles, California, 90095, 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 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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Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Conditions
Explore the condition pages connected to this study.