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

Recruiting now · Phase 2/Phase 3

Conditions studied: Congenital Myotonic Dystrophy

In brief

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.

Key facts

Study ID

NCT05004129

Run by

AMO Pharma Limited

People needed

76

Starts

2021-08-23

Expected to finish

2026-12-31

Last updated by the study team

2025-05-28

Who can join

Age: 6 and older, up to 45. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:
• Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
• Diagnosis must be genetically confirmed
• Subjects must be male or female aged ≥6 years to ≤45 years at Screening
• Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
• Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)
• Subject's caregiver must be willing and able to support participation for duration of study
• Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
• Subjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:
• Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
• Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations)
• Subject's caregiver must be willing and able to support participation for duration of study
• Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
• Key Exclusion Criteria:
• Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
• New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
• Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
• Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
• Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months other than the AMO-02- MD-2-003 study
• Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or respiratory disease) that may impact the interpretability of the study results
• Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
• University of Iowa Hospitals and Clinics — Iowa City, Iowa, United States (enrolling)
• University of Rochester - Medical Center — Rochester, New York, United States (enrolling)
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States (enrolling)
• University of Utah Clinical Neurosciences Center — Salt Lake City, Utah, United States (enrolling)
• The Bright Alliance — Randwick, New South Wales, Australia (enrolling)
• Children's Hospital of Eastern Ontario — Ottawa, Ontario, Canada (enrolling)
• Lurie's Children's Hospital — Chicago, Illinois, United States (enrolling)
• University of California, Los Angeles (UCLA) — Los Angeles, California, United States
• Stanford University — Palo Alto, California, United States
• Children's Hospital London Health Sciences Centre (LHSC) — London, Ontario, Canada
• Children's Hospital of The King's Daughters — Norfolk, Virginia, United States
• Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program — Richmond, Virginia, United States
• New Zealand Clinical Research (NZCR) — Auckland, New Zealand

Full record on ClinicalTrials.gov

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