Hope for kids with rare muscle disease in drug trial
NCT ID NCT03692312
Summary
This study tested whether a drug called tideglusib could help children and teenagers (ages 6-16) with congenital myotonic dystrophy, a rare genetic muscle disease. Researchers compared tideglusib to a placebo (inactive pill) to see if it could improve symptoms, daily function, and walking speed. The main goal was to see if the drug could reduce the overall severity of the disease.
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
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Children's Hospital London Health Sciences Centre (LHSC)
London, Ontario, N6A4G5, Canada
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Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
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New Zealand Clinical Research (NZCR)
Auckland, 1010, New Zealand
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Newcastle University
Newcastle upon Tyne, NE2 4HH, United Kingdom
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Stanford University
Palo Alto, California, 94304, United States
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The Bright Alliance
Randwick, New South Wales, 2031, Australia
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University of California, Los Angeles (UCLA)
Los Angeles, California, 90095, United States
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University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
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University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, United States
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University of Rochester Medical Center
Rochester, New York, 14642, United States
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University of Utah Hospital
Salt Lake City, Utah, 84112, United States
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Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program.
Richmond, Virginia, 23219, United States
Conditions
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