Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT ID: NCT03692312

Last Updated: 2025-10-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 56 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-03-03
• Study Completion Date: 2023-04-04

Brief 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).

Detailed Description

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.

Conditions

Congenital Myotonic Dystrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Tideglusib

Weight adjusted tideglusib, orally, once daily

Group Type: EXPERIMENTAL

Tideglusib

Intervention Type: DRUG

Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily

Placebo

Intervention Type: DRUG

Matching placebo formulation

Placebo

Matching placebo, orally, once daily

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Matching placebo formulation

Interventions

Tideglusib

Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily

Intervention Type: DRUG

Placebo

Matching placebo formulation

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male or female children and adolescents aged ≥6 years and ≤16 years

2. 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

3. Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)

4. 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

5. Subject's caregiver must be willing and able to support participation for duration of study

6. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol

Exclusion Criteria

1. Not able to walk; (full time wheel chair use)

2. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²

3. New or change in medications/therapies within 4 weeks prior to Screening

4. Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline

5. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)

6. 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

7. 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

8. Hypersensitivity to tideglusib and its excipients including allergy to strawberry

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

AMO Pharma Limited

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Joseph P Horrigan, MD

Role: STUDY_DIRECTOR

AMO Pharma

Locations

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

Countries

United States; Australia; Canada; New Zealand; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2016-004623-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AMO-02-MD-2-003

Identifier Type: -

Identifier Source: org_study_id