Actively Recruiting
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
Led by Virginia Commonwealth University · Updated on 2025-06-11
50
Participants Needed
5
Research Sites
222 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.
CONDITIONS
Official Title
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age from newborn to 3 years 11 months at enrollment
- Diagnosis of congenital myotonic dystrophy with symptoms starting within 30 days of birth such as low muscle tone, feeding or breathing difficulties requiring hospitalization over 72 hours
- Genetic confirmation of an expanded CTG repeat in the DMPK gene in the child or mother (greater than 200 repeats)
- Guardian willing and able to provide consent and comply with study procedures
You will not qualify if you...
- Presence of other non-DM1 illnesses that could interfere with study participation or outcomes
- Significant trauma within the past month
- Internal metal or devices preventing certain study components
- History of bleeding disorder or platelet count below 50,000
- History of reaction to local anesthetic
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
University of California, Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
2
University of Kansas Medical Center
Fairway, Kansas, United States, 66205
Actively Recruiting
3
University of Rochester Medical Center
Rochester, New York, United States, 14642
Actively Recruiting
4
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Actively Recruiting
5
Centro Clinico NeMO
Milan, Italy, 20162
Actively Recruiting
Research Team
R
Ruby Langeslay
CONTACT
J
Jennifer Raymond
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
1
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