Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
NCT07700225 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 1000
Last updated 2026-08-06
Summary
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.
Conditions
- DM1
- Myotonic Dystrophy
- Myotonic Dystrophy 1
- Myotonic Dystrophy Type 1
- Myotonic Dystrophy Type-1
- Myotonic Dystrophy, Type 1 (DM1)
- Myotonic Muscular Dystrophy
Sponsors & Collaborators
- collaborator INDUSTRY
-
Myotonic Dystrophy Clinical Research Network (DMCRN)
collaborator UNKNOWN -
Vertex Pharmaceuticals Incorporated
collaborator INDUSTRY -
Virginia Commonwealth University
lead OTHER
Principal Investigators
-
Nicholas Johnson, MD · Virginia Commonwealth University
Eligibility
- Min Age
- 18 Years
- Max Age
- 70 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-08-31
- Primary Completion
- 2032-12-31
- Completion
- 2032-12-31
Countries
- United States
Study Locations
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