A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

NCT06926621 · tracked via the Priya Life Science USA tracker

Sponsor

Vertex Pharmaceuticals Incorporated

Phase

Phase 2

Started

2025-04-28

Last updated

2026-08-10

Condition(s) studied

Myotonic Dystrophy Type 1 (DM1)

Investigational drug(s) / intervention(s)

VX-670

VX-670: Solution for intravenous administration.

Study summary

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).

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Key Inclusion Criteria: • Completed study drug treatment in parent study VX23-670-001 (NCT06185764) 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.

Primary outcome measure(s)

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — From Day 1 up to Week 108

Trial sites (15)

Facility	City	Region
St. Louis Children's Hospital - Neurology	St Louis	Missouri
Virginia Commonwealth University - Sanger Hall	Richmond	Virginia
Wesley Research Institute	Auchenflower	Australia
Neuroscience Clinical Trials Unit, Alfred Brain	Melbourne	Australia
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology	Leuven	Belgium
Altasciences Montreal	Montreal	Canada
Montreal Neurological Institute-Hospital	Montreal	Canada
University of Ottawa	Ottawa	Canada
Universite Laval - Neurology	Québec	Canada
Friedrich Baur Institute	München	Germany
Maastricht UMC	Maastricht	Netherlands
Hospital Universitario y Politecnico La Fe - Neurology	Valencia	Spain
Queen Elizabeth University Hospital - Neurology	Glasgow	United Kingdom
Leonard Wolfson Experimental Neurology Centre CRF	London	United Kingdom
Royal Hallamshire Hospital - Neurology	Sheffield	United Kingdom

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Other trials for the same condition

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 Recruiting

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 Recruiting

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06926621 on ClinicalTrials.gov ↗ ← All trials in the USA