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

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

Recruiting
18-70 years
All
Phase N/A
  • Simplified description (AI rewritten)
  • Technical description (Original)

The goal of this observational study is to characterize long-term disease progression over at least 4 years in at least 1,000 adults with myotonic dystrophy type 1 (DM1).

The main questions this study aims to answer are:

  1. How do clinical measures, such as walking speed, hand function, and muscle strength, change over a multi-year period in people with DM1?
  2. Can long-term changes in slowly progressive measures, like heart rhythms (ECG) and lung function (FVC), be accurately captured and used as biomarkers for the disease over time?
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Study details
    DM1
    Myotonic Dystrophy
    Myotonic Dystrophy 1
    Myotonic Dystrophy Type 1
    Myotonic Dystrophy Type-1
    Myotonic Dystrophy
    Type 1 (DM1)
    Myotonic Muscular Dystrophy

NCT07700225

Virginia Commonwealth University

15 July 2026

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