Summary

Eligibility
for people ages 6-45 (full criteria)
Location
at UCLA
Dates
study started
completion around

Description

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.

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)

Details

This is an open-label study of weight-adjusted 1000 mg tideglusib, once daily for 52 weeks with an open-ended optional extended access period in children and adolescents with a diagnosis of Congenital DM1 who participated in the AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

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 6-45

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
  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. 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

  • University of California, Los Angeles (UCLA) accepting new patients by invitation only
    Los Angeles California 90095 United States
  • Stanford University accepting new patients by invitation only
    Palo Alto California 94304 United States
  • University of Utah Clinical Neurosciences Center accepting new patients
    Salt Lake City Utah 84132 United States
  • Arkansas Children's Hospital accepting new patients
    Little Rock Arkansas 72202 United States

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
AMO Pharma Limited
ID
NCT05004129
Phase
Phase 2/3 Myotonic Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 76 study participants
Last Updated