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

Description

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.

Conditions

Congenital Myotonic Dystrophy

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Study Overview

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Study Details

Study overview

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.

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)

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

Condition
Congenital Myotonic Dystrophy
Intervention / Treatment

-

Contacts and Locations

Little Rock

Arkansas Children's Hospital, Little Rock, Arkansas, United States, 72202

Los Angeles

University of California, Los Angeles (UCLA), Los Angeles, California, United States, 90095

Palo Alto

Stanford University, Palo Alto, California, United States, 94304

Chicago

Lurie's Children's Hospital, Chicago, Illinois, United States, 60611

Iowa City

University of Iowa Hospitals and Clinics, Iowa City, Iowa, United States, 52242

Rochester

University of Rochester - Medical Center, Rochester, New York, United States, 14642

Pittsburgh

University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States, 15213

Salt Lake City

University of Utah Clinical Neurosciences Center, Salt Lake City, Utah, United States, 84132

Norfolk

Children's Hospital of The King's Daughters, Norfolk, Virginia, United States, 23507

Richmond

Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program, Richmond, Virginia, United States, 23219

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 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
  • 1. Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
  • 2. 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 (as required by local regulations)
  • 3. Subject's caregiver must be willing and able to support participation for duration of study
  • 4. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
  • 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

Ages Eligible for Study

6 Years to 45 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

AMO Pharma Limited,

Joseph P Horrigan, MD, STUDY_DIRECTOR, AMO Pharma

Study Record Dates

2025-03-28