Actively Recruiting
Phase 12 Study of VX-670 to Test Safety and Effects in Adults With Myotonic Dystrophy Type 1
Led by Vertex Pharmaceuticals Incorporated · Updated on 2026-06-08
52
Participants Needed
26
Research Sites
N/A
Total Duration
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and how the body processes and responds to VX-670 in adults with Myotonic Dystrophy Type 1 DM1. This study is a Phase 12 trial that compares different doses of VX-670 to placebo in people diagnosed with DM1 who have a confirmed genetic test showing a specific repeat in their DNA. Participants will be randomly assigned to receive either single or multiple doses of VX-670 or matching placebo. The study has two parts Part A focuses on single ascending doses, while Part B includes both single and multiple ascending doses. The dosing levels in Part B will be based on results from Part A. Both VX-670 and placebo are given as intravenous solutions. During the study, participants will be monitored for adverse events up to 42 days in Part A and 168 days in Part B to assess safety and tolerability. Researchers will measure drug concentrations in blood plasma and muscle at various time points, including baseline, days 15, and 120. Muscle biopsies will be used to analyze changes in gene splicing. The total participation duration varies by study part, with detailed monitoring of drug effects and safety throughout.
CONDITIONS
Brief Title
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Research Team
M
Medical Information
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
