A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Recruiting now · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Myotonic Dystrophy Type 1 (DM1)
In brief
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.
Key facts
- Study ID
- NCT06185764
- Run by
- Vertex Pharmaceuticals Incorporated
- People needed
- 52
- Starts
- 2024-02-20
- Expected to finish
- 2027-02-02
- Last updated by the study team
- 2026-06-08
Who can join
Age: 18 and older, up to 64. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Stanford Neuromuscular Research — San Carlos, California, United States (enrolling)
- University of Florida Clinical Research Center — Gainesville, Florida, United States (enrolling)
- University of Kansas Medical Center — Fairway, Kansas, United States (enrolling)
- Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
- Washington University School of Medicine / St. Louis Children's Hospital — St Louis, Missouri, United States (enrolling)
- Wake Forest Baptist Health — Winston-Salem, North Carolina, United States (enrolling)
- University of Pennsylvania — Philadelphia, Pennsylvania, United States (enrolling)
- Virginia Commonwealth University (Sanger Hall) — Richmond, Virginia, United States (enrolling)
- Wesley Research Institute — Auchenflower, Australia (enrolling)
- Neuroscience Clinical Trials Unit, Alfred Brain — Melbourne, Australia (enrolling)
- Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg — Leuven, Belgium (enrolling)
- Hopital de Chicoutimi — Chicoutimi, Canada (enrolling)
- Altasciences Montreal — Montreal, Canada (enrolling)
- McGIll University — Montreal, Canada (enrolling)
- University of Ottawa — Ottawa, Canada (enrolling)
- CHU Research Centre of Quebec — Québec, Canada (enrolling)
- Neuromuscular Reference Center Institute of Myology — Paris, France (enrolling)
- Ludwig Maximilians Universitaet Muenchen — München, Germany (enrolling)
- Centro Clinico NeMO — Milan, Italy (enrolling)
- Maastricht University Medical Center — Maastricht, Netherlands (enrolling)
- Hospital Universitario y Politécnico La Fe — Valencia, Spain (enrolling)
- Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow, United Kingdom (enrolling)
- Leonard Wolfson Experimental Neurology Centre CRF — London, United Kingdom (enrolling)
- St. George's University Hospital — London, United Kingdom (enrolling)
- Salford Royal Hospital — Salford, United Kingdom (enrolling)
Full record on ClinicalTrials.gov
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