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Evaluating Tideglusib for Congenital Myotonic Dystrophy

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)

Phase2; Phase3 Interventional AMO Pharma Limited · NCT05004129

This study is testing a new medication called tideglusib to see if it can help children and teenagers with Congenital Myotonic Dystrophy feel better over a year of treatment.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment76 (estimated)
Ages6 Years to 45 Years
SexAll
SponsorAMO Pharma Limited Industry-sponsored
Locations14 sites (Little Rock, Arkansas and 13 other locations)
Trial IDNCT05004129 on ClinicalTrials.gov

What this trial studies

This open-label phase 2/3 study investigates the safety and efficacy of tideglusib in children and adolescents with Congenital Myotonic Dystrophy (DM1). Participants include those who previously took part in an earlier study or are treatment-naïve. The treatment involves a weight-adjusted fixed dose of tideglusib administered over a 52-week period, with an option for extended access. The study aims to assess the drug's impact on the condition's severity and overall patient well-being.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents aged 6 to 45 years with genetically confirmed Congenital or Childhood Onset DM1.

Not a fit: Patients who do not have a confirmed diagnosis of Congenital or Childhood Onset DM1 or those who do not meet the eligibility criteria will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the quality of life for patients with Congenital Myotonic Dystrophy.

How similar studies have performed: Previous studies involving tideglusib have shown promise, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

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:

1. Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
2. Diagnosis must be genetically confirmed
3. Subjects must be male or female aged ≥6 years to ≤45 years at Screening
4. Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
5. 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)
6. Subject's caregiver must be willing and able to support participation for duration of study
7. 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:

1. Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
2. 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)
3. Subject's caregiver must be willing and able to support participation for duration of study
4. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol

Key Exclusion Criteria:

1. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
2. New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
3. Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
4. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
5. 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
6. 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
7. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry

Where this trial is running

Little Rock, Arkansas and 13 other locations

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Congenital Myotonic DystrophyTideglusibAMO-02-MD-2-004Myotonic DystrophyDystrophia MyotonicaMyotonia AtrophicaMyotonia DystrophicaMyotonic Dystrophy, Congenital
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.