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Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy | Clinical Trials | Clareo Health

RECRUITINGPHASE2/PHASE3

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)

NCT05004129•AMO Pharma Limited

View on ClinicalTrials.gov

Summary

This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

Detailed Description

This is an open-label study of either a weight-adjusted 1000 mg fixed dose or a weight banded fixed dose of tideglusib across a 52-week treatment period with an open-ended optional extended access period. The subjects are children and adolescents with Congenital DM1 who participated in the antecedent AMO-02-MD-2-003 study or individuals with either Congenital or Childhood onset DM1 who are treatment naïve.

Eligibility

Age

6 - 45 years

Sex

ALL

Healthy Volunteers

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
+3 more criteria

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

Locations (14)

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

Lurie's Children's Hospital

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 Clinical Neurosciences Center

Salt Lake City, Utah, United States

Children's Hospital of The King's Daughters

Norfolk, Virginia, United States

Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program

Richmond, Virginia, United States

Key Dates

Start Date

August 23, 2021

Primary Completion Date

December 31, 2026

Completion Date

December 31, 2026

Last Updated

May 28, 2025

Enrollment

ESTIMATED participants

Conditions

Congenital Myotonic Dystrophy

Interventions

Tideglusib

DRUG

Myotonic Dystrophy Family Registry

NCT02398786

Myotonic DystrophyCongenital Myotonic Dystrophy+14 more

View study

COMPLETED

Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy

NCT03059264

Congenital Myotonic Dystrophy

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: May 28, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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