Actively Recruiting
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Led by Vertex Pharmaceuticals Incorporated · Updated on 2026-02-18
44
Participants Needed
26
Research Sites
148 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented clinical diagnosis of DM1 with age of onset greater than 1 year
- Documented positive genetic test for DM1 with CTG repeat of at least 100
- Age between 18 and 64 years
You will not qualify if you...
- History of any illness or clinical condition as specified in the protocol
- Other protocol defined inclusion/exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 26 locations
1
Stanford Neuromuscular Research
San Carlos, California, United States, 94070
Actively Recruiting
2
University of Florida Clinical Research Center
Gainesville, Florida, United States, 32608
Actively Recruiting
3
University of Kansas Medical Center
Fairway, Kansas, United States, 66205
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, United States, 63110
Actively Recruiting
6
Wake Forest Baptist Health
Winston-Salem, North Carolina, United States, 27104
Actively Recruiting
7
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
Virginia Commonwealth University (Sanger Hall)
Richmond, Virginia, United States, 23298
Actively Recruiting
9
Wesley Research Institute
Auchenflower, Australia
Actively Recruiting
10
Neuroscience Clinical Trials Unit, Alfred Brain
Melbourne, Australia
Actively Recruiting
11
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
Actively Recruiting
12
Hopital de Chicoutimi
Chicoutimi, Canada
Actively Recruiting
13
Altasciences Montreal
Montreal, Canada
Actively Recruiting
14
McGIll University
Montreal, Canada
Actively Recruiting
15
University of Ottawa
Ottawa, Canada
Actively Recruiting
16
CHU Research Centre of Quebec
Québec, Canada
Actively Recruiting
17
Neuromuscular Reference Center Institute of Myology
Paris, France
Actively Recruiting
18
Ludwig Maximilians Universitaet Muenchen
München, Germany
Actively Recruiting
19
Centro Clinico NeMO
Milan, Italy
Actively Recruiting
20
Maastricht University Medical Center
Maastricht, Netherlands
Actively Recruiting
21
Hospital Universitario y Politécnico La Fe
Valencia, Spain
Actively Recruiting
22
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
Actively Recruiting
23
Leonard Wolfson Experimental Neurology Centre CRF
London, United Kingdom
Actively Recruiting
24
St. George's University Hospital
London, United Kingdom
Actively Recruiting
25
Salford Royal Hospital
Salford, United Kingdom
Actively Recruiting
26
Royal Hallamshire Hospital
Sheffield, United Kingdom
Actively Recruiting
Research Team
M
Medical Information
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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