New drug tested to help kids with rare Muscle-Weakening disease
NCT ID NCT03692312
Summary
This study tested whether a drug called tideglusib could improve symptoms in children and teenagers (ages 6-16) with congenital myotonic dystrophy, a rare genetic muscle disease. Participants were randomly assigned to receive either the active drug or a placebo (a dummy pill) for the duration of the trial. The main goal was to see if tideglusib could improve scores on a doctor's rating scale that measures the severity of the disease's symptoms.
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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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