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DMCRN-02-001: Assessing Pediatric Endpoints in DM1
Sponsor: Virginia Commonwealth University
Summary
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.
Official title: Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)
Key Details
Gender
All
Age Range
Any - 59 Months
Study Type
OBSERVATIONAL
Enrollment
50
Start Date
2022-08-24
Completion Date
2026-12
Last Updated
2025-06-11
Healthy Volunteers
No
Conditions
Locations (5)
University of California, Los Angeles
Los Angeles, California, United States
University of Kansas Medical Center
Fairway, Kansas, United States
University of Rochester Medical Center
Rochester, New York, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Centro Clinico NeMO
Milan, Italy