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DMCRN-02-001: Assessing Pediatric Endpoints in DM1

NCT05224778 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2025-06-11

No results posted yet for this study

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.

Conditions

  • Congenital Myotonic Dystrophy
  • CDM

Sponsors & Collaborators

  • Virginia Commonwealth University

    lead OTHER

Principal Investigators

  • Nicholas E. Johnson, MD · Virginia Commonwealth University

Eligibility

Max Age
59 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-24
Primary Completion
2026-10-31
Completion
2026-12-31

Countries

  • United States
  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05224778 on ClinicalTrials.gov