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

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital London Health Sciences Centre (LHSC)

    London, Ontario, N6A4G5, Canada

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • New Zealand Clinical Research (NZCR)

    Auckland, 1010, New Zealand

  • Newcastle University

    Newcastle upon Tyne, NE2 4HH, United Kingdom

  • Stanford University

    Palo Alto, California, 94304, United States

  • The Bright Alliance

    Randwick, New South Wales, 2031, Australia

  • University of California, Los Angeles (UCLA)

    Los Angeles, California, 90095, United States

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Utah Hospital

    Salt Lake City, Utah, 84112, United States

  • Virginia Commonwealth University - Department of Neurology. Muscular Dystrophy Translational Research Program.

    Richmond, Virginia, 23219, United States

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