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A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Sponsor: Vertex Pharmaceuticals Incorporated
Summary
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
Official title: A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1
Key Details
Gender
All
Age Range
18 Years - 64 Years
Study Type
INTERVENTIONAL
Enrollment
44
Start Date
2024-02-20
Completion Date
2026-12-22
Last Updated
2026-02-18
Healthy Volunteers
No
Conditions
Interventions
VX-670
Solution for intravenous administration.
Placebo
Solution for intravenous administration.
Locations (26)
Stanford Neuromuscular Research
San Carlos, California, United States
University of Florida Clinical Research Center
Gainesville, Florida, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, United States
Wake Forest Baptist Health
Winston-Salem, North Carolina, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Virginia Commonwealth University (Sanger Hall)
Richmond, Virginia, United States
Wesley Research Institute
Auchenflower, Australia
Neuroscience Clinical Trials Unit, Alfred Brain
Melbourne, Australia
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
Hopital de Chicoutimi
Chicoutimi, Canada
Altasciences Montreal
Montreal, Canada
McGIll University
Montreal, Canada
University of Ottawa
Ottawa, Canada
CHU Research Centre of Quebec
Québec, Canada
Neuromuscular Reference Center Institute of Myology
Paris, France
Ludwig Maximilians Universitaet Muenchen
München, Germany
Centro Clinico NeMO
Milan, Italy
Maastricht University Medical Center
Maastricht, Netherlands
Hospital Universitario y Politécnico La Fe
Valencia, Spain
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
Leonard Wolfson Experimental Neurology Centre CRF
London, United Kingdom
St. George's University Hospital
London, United Kingdom
Salford Royal Hospital
Salford, United Kingdom
Royal Hallamshire Hospital
Sheffield, United Kingdom