Status:
RECRUITING
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Conditions:
Myotonic Dystrophy Type 1 (DM1)
Eligibility:
All Genders
18-64 years
Phase:
PHASE1
PHASE2
Brief 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.
Eligibility Criteria
Key Inclusion Criteria:
- Documented clinical diagnosis of DM1 with age of onset greater than (>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100
Key Exclusion Criteria:
- History of any illness or any clinical condition as pre-specified in the protocol
Other protocol defined Inclusion/Exclusion criteria may apply.
Key Trial Info
Start Date :
February 20 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 22 2026
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT06185764
Start Date
February 20 2024
End Date
December 22 2026
Last Update
February 18 2026
Active Locations (26)
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1
Stanford Neuromuscular Research
San Carlos, California, United States, 94070
2
University of Florida Clinical Research Center
Gainesville, Florida, United States, 32608
3
University of Kansas Medical Center
Fairway, Kansas, United States, 66205
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115