Evaluating Tideglusib for Congenital Myotonic Dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• Lurie's Children's Hospital
  Chicago, Illinois, United States Recruiting
• University of Iowa Hospitals and Clinics
  Iowa City, Iowa, United States Recruiting
• University of Rochester - Medical Center
  Rochester, New York, United States Recruiting
• University of Pittsburgh Medical Center
  Pittsburgh, Pennsylvania, United States Recruiting
• University of Utah Clinical Neurosciences Center
  Salt Lake City, Utah, United States Recruiting

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Assessing Readiness and Endpoints for Pediatric Myotonic Dystrophy

  This study is trying to improve how we understand and measure the effects of congenital and childhood myotonic dystrophy to help create better treatments for kids with these conditions.

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• Assessing clinical endpoints in children with myotonic dystrophy

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  Recruiting at 5 sites Virginia Commonwealth University · Los Angeles, California and 4 other locations · NCT05224778

• Online registry for myotonic dystrophy patients

  This study is creating an online registry for people with myotonic dystrophy to share their experiences and help researchers find better treatments.

  Recruiting Myotonic Dystrophy Foundation · Oakland, California · NCT02398786

• Registry for Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy

  This study is creating a registry for people with myotonic dystrophy or facioscapulohumeral muscular dystrophy to share their health information and connect with researchers working to learn more about these conditions.

  Recruiting University of Rochester · Rochester, New York · NCT00082108

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.