A Phase 2 interventional study of VX-670 in Myotonic Dystrophy Type 1 (DM1), sponsored by Vertex Pharmaceuticals Incorporated. Enrolling by invitation at 16 sites in 8 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-14.
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).
126 studies on the registry are indexed under Myotonic Dystrophy; 50 are open to participants now.
This study's planned enrollment of 44 is close to the median of 40 across 67 interventional studies indexed under Myotonic Dystrophy.
Browse Myotonic Dystrophy studies →Vertex Pharmaceuticals Incorporated is the lead sponsor of 249 studies on the registry; 17 are open to participants now.
Of its 106 completed or terminated interventional studies of FDA-regulated products, 53 (50%) have results posted.
Counted across the registry records on this site, refreshed daily.
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/
No publications or documents are linked to this record.
Started Apr 2025 (actual). Primary completion is estimated for Jan 2029, 45 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Vertex Pharmaceuticals Incorporated →