Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension

Part of paid clinical trials in Richmond, Virginia.

Sponsor
Virginia Commonwealth University
Study ID
NCT07700225
Status
Recruiting
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Conditions

  • DM1
  • Myotonic Dystrophy
  • Myotonic Dystrophy 1
  • Myotonic Dystrophy Type 1
  • Myotonic Dystrophy Type-1
  • Myotonic Dystrophy, Type 1 (DM1)
  • Myotonic Muscular Dystrophy

Eligibility Criteria

Sex
ALL
Age
18 Years - 70 Years
Healthy Volunteers
Not accepted

Study Details

Myotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy. It is characterized by progressive weakness and myotonia, which preferentially affects the craniofacial, hand, and distal leg muscles. Many patients also experience difficulties with cognition, cardiac arrhythmias, respiratory failure, or cataracts. Currently there is no treatment to slow progression or reverse the symptoms.

Key Dates

First listed
Jul 13, 2026
Start date
Jul 31, 2026
Status verified
Jul 2026
Primary completion
Dec 31, 2032
Completion
Dec 31, 2032

Study Design

Enrollment
1,000 participants (estimated)

Arms

  • Arm: Myotonic Dystrophy Type 1 (DM1) Longitudinal Cohort
    Participants with a clinical or genetic diagnosis of myotonic dystrophy type 1 (DM1) or congenital myotonic dystrophy (CDM). This cohort includes individuals transitioning from the parent study, Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1; NCT03981575), as well as newly enrolled participants.

Primary Outcome Measure

Characterize the long-term disease progression- 10 meter walk/run [ Time Frame: Baseline (0 months), every 12 months over four years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Virginia Commonwealth UniversityRichmondVirginia23298
Jennifer Raymond
[email protected]
804-828-6318
Nicholas Johnson, MD (PRINCIPAL_INVESTIGATOR)

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By research site
Virginia Commonwealth University· Richmond, VA

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