Actively Recruiting
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
Led by Virginia Commonwealth University · Updated on 2025-07-30
200
Participants Needed
1
Research Sites
260 weeks
Total Duration
On this page
Sponsors
V
Virginia Commonwealth University
Lead Sponsor
N
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.
CONDITIONS
Official Title
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
Who Can Participate
Eligibility Criteria
You may qualify if you...
- For Congenital Myotonic Dystrophy group: Age 5 to 17 years and 11 months at enrollment (exceptions for prior study participants).
- Diagnosis of CDM with symptoms in newborn period and genetic confirmation of expanded CTG repeat in DMPK gene.
- For Childhood Myotonic Dystrophy group: Age 3 to 17 years and 11 months at enrollment (exceptions for prior study participants).
- Diagnosis of ChDM with muscle weakness, cognitive deficits, or myotonia starting after age 1 and before age 10 and genetic confirmation of expanded CTG repeat.
- Written informed consent obtained before study procedures.
You will not qualify if you...
- Any other illness that could interfere with safe testing or affect results interpretation.
- Significant trauma within the past month.
- Presence of internal metal or devices (for DEXA scan).
- Use of blood thinners like warfarin or direct oral anticoagulants.
- Platelet count less than 50,000.
- History of bleeding disorders.
- Participation in another clinical trial with investigational treatment.
- History of adverse reaction to lidocaine if muscle biopsy is involved.
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Actively Recruiting
Research Team
R
Ruby Langeslay, MPH
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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