Summary

Eligibility
for people ages 6-17 (full criteria)
Location
at UCLA
Dates
study started
estimated completion

Description

Summary

This is an open-label extension phase 2/3 study for children and adolescents with Congenital Myotonic Dystrophy (Congenital DM1) who participated in and completed the preceding AMO-02-MD-2-003 study.

Official Title

A 52-Week, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital DM1 (REACH CDM X)

Details

This is an open-label extension study of weight-adjusted 1000 mg tideglusib, once daily for 52 weeks in children and adolescents with a diagnosis of Congenital DM1 who have completed the AMO-02-MD-2-003 study.

Keywords

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

Eligibility

You can join if…

Open to people ages 6-17

  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

You CAN'T join if...

  1. Subjects who discontinued prematurely from the antecedent AMO-02-MD-2-003 study
  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 Eligibility/Baseline Visit
  4. Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
  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 other than the AMO-02- MD-2-003 study
  7. 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
  8. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry

Locations

  • University of California, Los Angeles (UCLA)
    Los Angeles California 90095 United States
  • Stanford University
    Palo Alto California 94304 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
AMO Pharma Limited
ID
NCT05004129
Phase
Phase 2/3 Myotonic Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 56 study participants
Last Updated