Actively Recruiting
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)
Led by AMO Pharma Limited · Updated on 2025-05-28
76
Participants Needed
14
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting an open-label phase 2/3 study to evaluate tideglusib in children and adolescents with Congenital or Childhood Onset Myotonic Dystrophy (DM1). This includes those who participated in a previous related study or are treatment-nave. The study aims to assess long-term safety and efficacy in this specific group affected by Congenital DM1. Participants will receive tideglusib orally once daily, either as a weight-adjusted dose of 400 mg, 600 mg, or 1000 mg, or a weight banded fixed dose ranging from 400 mg to 1000 mg. All subjects start at 400 mg for two weeks, then increase to 600 mg for the next two weeks. The treatment period lasts 52 weeks, followed by an optional extended access phase that continues beyond the initial treatment period. During the study, participants will undergo various assessments including safety monitoring through adverse event tracking, and evaluations using the Clinician-Completed Congenital DM1 Rating Scale, Clinical Global Impressions scales, caregiver questionnaires, and physical tests like the 10-meter walk-run test. Blood tests will measure CTG repeats and plasma troponin T levels. These evaluations continue at regular intervals during the treatment and extended access phases, which may last up to 132 weeks or until study closure.
CONDITIONS
Brief Title
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Individuals diagnosed with Congenital or Childhood Onset Myotonic Dystrophy confirmed by genetic testing
- Male or female aged 6 to 45 years at screening
- Clinical Global Impression - Severity (CGI-S) score of 3 or greater at screening
- Written informed consent provided by participant or legally authorized representative with assent as required
- Caregiver willing and able to support participation for the study duration
- Willing and able to follow required food intake restrictions
- For those entering from the previous AMO-02-MD-2-003 study, completion of that study through visit 11 and agreement to consent and caregiver support requirements
You will not qualify if you...
- Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
- New or changed medications or therapies within 4 weeks before baseline visit
- Use of strong CYP3A4 inhibitors within 4 weeks before baseline visit
- Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window
- Participation in other investigational drug trials within the last 6 months except the previous AMO-02-MD-2-003 study
- Existing or past medical conditions (neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine, or respiratory) that could affect study results
- Allergy or hypersensitivity to tideglusib or any of its components including strawberry allergy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 52 weeks
Participants receive weight-adjusted or weight banded tideglusib orally once daily with dose adjustments over the first 4 weeks, continuing treatment for 52 weeks to evaluate safety and efficacy.
Regular visits during treatment period
Duration - Up to 80 weeks (after initial 52 weeks)
Participants may enter an optional extended access period with ongoing safety and efficacy assessments every 8 to 16 weeks until discontinuation or study closure.
Visits every 8 to 16 weeks during extended access
Trial Site Locations
Total: 14 locations
1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
2
University of California, Los Angeles (UCLA)
Los Angeles, California, United States, 90095
Enrolling by Invitation
3
Stanford University
Palo Alto, California, United States, 94304
Enrolling by Invitation
4
Lurie's Children's Hospital
Chicago, Illinois, United States, 60611
Actively Recruiting
5
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
Actively Recruiting
6
University of Rochester - Medical Center
Rochester, New York, United States, 14642
Actively Recruiting
7
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15213
Actively Recruiting
8
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, United States, 84132
Actively Recruiting
9
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
Withdrawn
10
Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program
Richmond, Virginia, United States, 23219
Completed
11
The Bright Alliance
Randwick, New South Wales, Australia, 2031
Actively Recruiting
12
Children's Hospital London Health Sciences Centre (LHSC)
London, Ontario, Canada, N6A 4G5
Enrolling by Invitation
13
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Actively Recruiting
14
New Zealand Clinical Research (NZCR)
Auckland, New Zealand, 1010
Completed
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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