Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Congenital Myotonic Dystrophy

In brief

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).

Key facts

Study ID

NCT03692312

Run by

AMO Pharma Limited

People needed

56

Starts

2021-03-03

Expected to finish

2023-04-04

Last updated by the study team

2025-10-08

Who can join

Age: 6 and older, up to 16. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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:
• Hypotonia
• 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

You may not qualify if…

• 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

Where it is running

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

Full record on ClinicalTrials.gov

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