What this study is
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).
What's being tested
- VX-670
Intervention · DRUG
Solution for intravenous administration.
Study details
- 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
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Conditions
- Myotonic Dystrophy Type 1 (DM1)
- Enrolling
- 44 people
- Starts
- 2025-04-28
- Est. completion
- 2029-01-30
- Design
- Open-label, single group, Phase 2
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 18 Years and overSex ALLHealthy volunteers Not acceptedYou may qualify if
- Completed study drug treatment in parent study VX23-670-001 (NCT06185764)
You can't join if
- History of any illness or any clinical condition as pre-specified in the protocol
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (16)
16 openSt. Louis Children's Hospital - Neurology
St Louis, Missouri, United States
Wake Forest Univ School of Medicine - Neurology
Winston-Salem, North Carolina, United States
Virginia Commonwealth University - Sanger Hall
Richmond, Virginia, United States
13 sites outside the US
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
Friedrich Baur Institute
München, Germany
Maastricht UMC
Maastricht, Netherlands
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
Study team
Sponsor
Vertex Pharmaceuticals Incorporated
Study ID VX24-670-101
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.