Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

  • End date
    Mar 28, 2022
  • participants needed
  • sponsor
    AMO Pharma Limited
Updated on 28 August 2021
myotonic dystrophy


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).


This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.

Condition Muscular Dystrophy, Myotonic Dystrophy
Treatment Placebo, Tideglusib
Clinical Study IdentifierNCT03692312
SponsorAMO Pharma Limited
Last Modified on28 August 2021


Yes No Not Sure

Inclusion Criteria

Male or female children and adolescents aged 6 years and 16 years
Diagnosis of Congenital DM1 (also known as Steinert's disease)
Diagnosis must be genetically confirmed
One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth
Generalized weakness
Respiratory insufficiency
Feeding difficulties
Clubfoot or another musculoskeletal deformity
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
Where a parent or LAR provides consent, there must also be assent from the subject
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/m or greater than 40 kg/m
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
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