CompletedPhase 2/3

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

United States56 participantsStarted 2021-03-03

Plain-language summary

This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).

Who can participate

Age range6 Years – 16 Years

SexALL

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Inclusion criteria

✓. Male or female children and adolescents aged ≥6 years and ≤16 years
✓. Diagnosis of Congenital DM1 (also known as Steinert's disease)
✓. Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
✓. Written, voluntary informed consent must be obtained before any study related procedures are conducted.
✓. 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

Exclusion criteria

✕. Not able to walk; (full time wheel chair use)
✕. 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 Screening
✕. Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
✕. 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
✕. Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
✕. Hypersensitivity to tideglusib and its excipients including allergy to strawberry

What they're measuring

Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)

Timeframe: Baseline and week 20

Trial details

NCT IDNCT03692312

SponsorAMO Pharma Limited

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-04-04

Results submitted2025-06-05

View on ClinicalTrials.gov More Congenital Myotonic Dystrophy trials