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

Study acronym: END-EXT
StatusRecruiting
PhaseNot specified
Started

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Record History

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  1. Primary endpoints: details revised at 1 of 6 entries Show 4 more changesShow less
    • Start date changed from estimate of Sep 2026 to Oct 15, 2026
    • Eligibility criteria expanded, about 8 words
    • Publications: details revised at 12 of 12 entries
    • Study description expanded, about 2 words
  2. Start date: Aug 2026 to Sep 2026v4
  3. Start date: Jul 2026 to Aug 2026v3
  4. Collaborators: 2 to 3v2
  5. Study First Submitted
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT07700225
Lead Sponsor Virginia Commonwealth University
Collaborators: Dyne Therapeutics, Myotonic Dystrophy Clinical Research Network (DMCRN), Vertex Pharmaceuticals Incorporated
Conditions
(from sponsor)		 DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type-1, Myotonic Dystrophy, Type 1 (DM1), Myotonic Muscular Dystrophy
Related terms Muscular Dystrophies, Myotonia, Myotonic Dystrophy, Neuromuscular Diseases
Enrollment 1,000 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov