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Study identifier: NCT05004129 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

Condition: Congenital Myotonic Dystrophy  ·  Sponsor: AMO Pharma Limited

PhasePhase 2/Phase 3
Planned participants76
Who can joinAll sexes, 6 Years to 45 Years
Healthy volunteersNo

About this study

This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

This description comes directly from the study's public registry record.

Talk to the study team

Contact details are listed per location below or on the registry record.

Always discuss trial participation with your own doctor first.

Locations (14)

Arkansas Children's HospitalLittle Rock, Arkansas, United StatesRecruiting
University of California, Los Angeles (UCLA)Los Angeles, California, United StatesEnrolling By Invitation
Stanford UniversityPalo Alto, California, United StatesEnrolling By Invitation
Lurie's Children's HospitalChicago, Illinois, United StatesRecruiting
University of Iowa Hospitals and ClinicsIowa City, Iowa, United StatesRecruiting
University of Rochester - Medical CenterRochester, New York, United StatesRecruiting
University of Pittsburgh Medical CenterPittsburgh, Pennsylvania, United StatesRecruiting
University of Utah Clinical Neurosciences CenterSalt Lake City, Utah, United StatesRecruiting
Children's Hospital of The King's DaughtersNorfolk, Virginia, United StatesWithdrawn
Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research ProgramRichmond, Virginia, United StatesCompleted
The Bright AllianceRandwick, New South Wales, AustraliaRecruiting
Children's Hospital London Health Sciences Centre (LHSC)London, Ontario, CanadaEnrolling By Invitation

+ 2 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT05004129