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

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.

NCT Number	NCT07700225
Lead Sponsor	Virginia Commonwealth University Collaborators: Dyne Therapeutics, Myotonic Dystrophy Clinical Research Network (DMCRN), Vertex Pharmaceuticals Incorporated
Conditions	DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type-1, Myotonic Dystrophy, Type 1 (DM1), Myotonic Muscular Dystrophy
Enrollment	1,000 participants
Start Date	2026-08
Primary Completion	2032-12 (estimated)
Study Completion	2032-12 (estimated)
Updated on ClinicalTrials.gov	2026-08-06