Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

Part of paid clinical trials in Richmond, Virginia.

Sponsor
Virginia Commonwealth University
Study ID
NCT06747884
Status
Recruiting
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Conditions

  • Childhood Myotonic Dystrophy
  • Congenital Myotonic Dystrophy
  • Myotonic Dystrophy

Eligibility Criteria

Sex
ALL
Age
3 Years - 17 Years
Healthy Volunteers
Not accepted

Study Details

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 Dates

Start date
Jun 6, 2025
Status verified
Jul 2025
Primary completion
Jun 30, 2030
Completion
Jun 30, 2030

Study Design

Enrollment
200 participants (estimated)

Arms

  • Arm: Congenital myotonic dystrophy (CDM)
    Children with congenital myotonic dystrophy. No intervention will be administered to either group.
  • Arm: Childhood Myotonic Dystrophy
    Children with childhood myotonic dystrophy. No intervention will be administered to either group.

Primary Outcome Measure

Change in ambulation over 24 months as measured by the 10-Meter Walk/Run Test (10M WRT) [ Time Frame: Baseline, Month 12 and Month 24 visits ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Virginia Commonwealth UniversityRichmondVirginia23298
Shantel Kyles
[email protected]
804-828-3381
Levi Headrick
[email protected]
Nicholas E Johnson, MD, MSCI, FAAN (PRINCIPAL_INVESTIGATOR)

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