New drug shows promise for rare muscle disease in kids
NCT ID NCT03692312
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a drug called tideglusib against a placebo in 56 children aged 6 to 16 with congenital myotonic dystrophy, a rare genetic muscle disorder. The goal was to see if tideglusib could reduce symptoms like muscle weakness and breathing problems. The trial was completed, but results are not yet publicly available.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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
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