A Phase 2 interventional study of VX-670 in Myotonic Dystrophy Type 1 (DM1), sponsored by Vertex Pharmaceuticals Incorporated. Enrolling by invitation at 15 sites in 8 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-10.
Sponsored by Vertex Pharmaceuticals Incorporated · Phase 2, Interventional, and Treatment
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).
Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
Participants will receive multiple doses of VX-670.
Drug: VX-670
Solution for intravenous administration.
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From Day 1 up to Week 108
Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
Time frame: From Day 1 up to Week 96
Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma
Time frame: From Day 1 up to Week 96
Plan to share: No — Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
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Vertex Pharmaceuticals Incorporated