Actively Recruiting
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
Led by AMO Pharma Limited · Updated on 2025-05-28
76
Participants Needed
14
Research Sites
279 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of 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 voluntary informed consent obtained; assent from participant if required
- Caregiver willing and able to support participation throughout the study
- Willing and able to follow the required food intake restrictions as per protocol
- For those entering after completing prior AMO-02-MD-2-003 study: must have completed that study through visit 11
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 eligibility/baseline visit
- Use of strong CYP3A4 inhibitors within 4 weeks before eligibility/baseline visit
- Concurrent use of drugs metabolized by CYP3A4 with narrow therapeutic windows
- Participation in another investigational drug clinical trial in the last 6 months (except prior AMO-02-MD-2-003 study)
- Existing or past medical conditions (neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine, or respiratory diseases) that may affect study results
- Allergy or hypersensitivity to tideglusib or any components including strawberry allergy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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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