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RECRUITING
NCT05224778

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Sponsor: Virginia Commonwealth University

View on ClinicalTrials.gov

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

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