Summary

Eligibility
for people ages 6-16 (full criteria)
Location
at UCLA
Dates
study started
estimated completion
Principal Investigator
by Perry Shieh, MD, PhD (ucla)

Description

Summary

This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).

Official Title

A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM)

Details

This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.

Keywords

Congenital Myotonic Dystrophy Tideglusib AMO-02-MD-2-003 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 6-16

  1. Male or female children and adolescents aged ≥6 years and ≤16 years
  2. Diagnosis of Congenital DM1 (also known as Steinert's disease)
  3. Diagnosis must be genetically confirmed
  4. One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth:
  5. Hypotonia
  6. Generalized weakness
  7. Respiratory insufficiency
  8. Feeding difficulties
  9. Clubfoot or another musculoskeletal deformity
  10. Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
  11. Written, voluntary informed consent must be obtained before any study related procedures are conducted.
  12. Where a parent or LAR provides consent, there must also be assent from the subject
  13. Subject's caregiver must be willing and able to support participation for duration of study
  14. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol

You CAN'T join if...

  1. Not able to walk; (full time wheel chair use)
  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 Screening
  4. Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
  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
  7. Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
  8. Hypersensitivity to tideglusib and its excipients including allergy to strawberry

Locations

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

Lead Scientist at University of California Health

  • Perry Shieh, MD, PhD (ucla)
    HS Clinical Professor, Neurology, Medicine. Authored (or co-authored) 66 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
AMO Pharma Limited
ID
NCT03692312
Phase
Phase 2/3 Myotonic Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 56 study participants
Last Updated