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A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
Sponsor: Vertex Pharmaceuticals Incorporated
Summary
The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).
Official title: An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type I
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
44
Start Date
2025-04-28
Completion Date
2029-01-30
Last Updated
2026-02-27
Healthy Volunteers
No
Conditions
Interventions
VX-670
Solution for intravenous administration.
Locations (11)
Wesley Research Institute
Auchenflower, Australia
Neuroscience Clinical Trials Unit, Alfred Brain
Melbourne, Australia
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology
Leuven, Belgium
Altasciences Montreal
Montreal, Canada
Montreal Neurological Institute-Hospital
Montreal, Canada
University of Ottawa
Ottawa, Canada
Universite Laval - Neurology
Québec, Canada
Hospital Universitario y Politecnico La Fe - Neurology
Valencia, Spain
Queen Elizabeth University Hospital - Neurology
Glasgow, United Kingdom
Leonard Wolfson Experimental Neurology Centre CRF
London, United Kingdom
Royal Hallamshire Hospital - Neurology
Sheffield, United Kingdom