Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

Recruiting now

Conditions studied: Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy

In brief

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.

Key facts

Study ID

NCT06747884

Run by

Virginia Commonwealth University

People needed

200

Starts

2025-06-06

Expected to finish

2030-06-01

Last updated by the study team

2026-08-06

Who can join

Age: 3 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Any other non-DM1 illness that would interfere with the ability to undergo safe testing or would affect the interpretation of the results, in the opinion of the site investigator
• Significant trauma within the past month
• Internal metal or devices (exclusion for DEXA component)
• Use of anticoagulants, such as warfarin or a direct oral anticoagulant (e.g., dabigatran) due to the increased risk of bleeding with biopsy
• Platelet count <50,000
• History of a bleeding disorder
• Participation in a clinical trial involving an investigational product
• History of adverse reaction to lidocaine (if participating in muscle biopsy)

Where it is running

• Virginia Commonwealth University — Richmond, Virginia, United States (enrolling)
• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States

Full record on ClinicalTrials.gov

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