A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
NCT06185764 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 44
Last updated 2026-02-18
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.
Conditions
- Myotonic Dystrophy Type 1 (DM1)
Interventions
- DRUG
-
VX-670
Solution for intravenous administration.
- DRUG
-
Solution for intravenous administration.
Sponsors & Collaborators
-
Vertex Pharmaceuticals Incorporated
lead INDUSTRY
Study Design
- Allocation
- RANDOMIZED
- Purpose
- TREATMENT
- Masking
- QUADRUPLE
- Model
- SEQUENTIAL
Eligibility
- Min Age
- 18 Years
- Max Age
- 64 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-02-20
- Primary Completion
- 2026-12-22
- Completion
- 2026-12-22
- FDA Drug
- Yes
Countries
- United States
- Australia
- Belgium
- Canada
- France
- Germany
- Italy
- Netherlands
- Spain
- United Kingdom
Study Locations
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