RECRUITING

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

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Conditions

Congenital Myotonic DystrophyTideglusibAMO-02-MD-2-004Myotonic DystrophyDystrophia MyotonicaMyotonia AtrophicaMyotonia DystrophicaMyotonic Dystrophy, CongenitalSteinert DiseaseSteinert Myotonic DystrophySteinert's Disease

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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)

Quick Facts

Study Start:2021-08-23
Study Completion:2025-03-28
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05004129

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.

Ages Eligible for Study:6 Years to 45 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
  2. 2. Diagnosis must be genetically confirmed
  3. 3. Subjects must be male or female aged ≥6 years to ≤45 years at Screening
  4. 4. Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
  5. 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. 6. Subject's caregiver must be willing and able to support participation for duration of study
  7. 7. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
  8. 1. Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
  9. 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)
  10. 3. Subject's caregiver must be willing and able to support participation for duration of study
  11. 4. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
  1. 1. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
  2. 2. New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
  3. 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. 4. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
  5. 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. 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. 7. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry

Contacts and Locations

Principal Investigator

Joseph P Horrigan, MD
STUDY_DIRECTOR
AMO Pharma

Study Locations (Sites)

Arkansas Children's Hospital
Little Rock, Arkansas, 72202
United States
University of California, Los Angeles (UCLA)
Los Angeles, California, 90095
United States
Stanford University
Palo Alto, California, 94304
United States
Lurie's Children's Hospital
Chicago, Illinois, 60611
United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
United States
University of Rochester - Medical Center
Rochester, New York, 14642
United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
United States
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132
United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507
United States
Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program
Richmond, Virginia, 23219
United States

Collaborators and Investigators

Sponsor: AMO Pharma Limited

  • Joseph P Horrigan, MD, STUDY_DIRECTOR, AMO Pharma

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2021-08-23
Study Completion Date2025-03-28

Study Record Updates

Study Start Date2021-08-23
Study Completion Date2025-03-28

Terms related to this study

Keywords Provided by Researchers

  • Tideglusib
  • AMO-02-MD-2-004
  • Congenital Myotonic Dystrophy
  • Myotonic Dystrophy
  • Dystrophia Myotonica
  • Myotonia Atrophica
  • Myotonia Dystrophica
  • Myotonic Dystrophy, Congenital
  • Steinert Disease
  • Steinert Myotonic Dystrophy
  • Steinert's Disease

Additional Relevant MeSH Terms

  • Congenital Myotonic Dystrophy