New hope for DM1: VX-670 safety trial expands

NCT ID NCT06926621

ENROLLING_BY_INVITATION Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

First seen Nov 14, 2025 · Last updated May 22, 2026 · Updated 27 times

Summary

This study looks at the long-term safety and effects of the drug VX-670 in 44 adults with myotonic dystrophy type 1 (DM1). All participants must have already taken VX-670 in an earlier study. Researchers will monitor side effects and measure drug levels in the blood. This is an open-label extension, meaning everyone gets the active drug.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Locations

Altasciences Montreal

Montreal, Canada
Hospital Universitario y Politecnico La Fe - Neurology

Valencia, Spain
Leonard Wolfson Experimental Neurology Centre CRF

London, United Kingdom
Montreal Neurological Institute-Hospital

Montreal, Canada
Neuroscience Clinical Trials Unit, Alfred Brain

Melbourne, Australia
Queen Elizabeth University Hospital - Neurology

Glasgow, United Kingdom
Royal Hallamshire Hospital - Neurology

Sheffield, United Kingdom
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology

Leuven, Belgium
Universite Laval - Neurology

Québec, Canada
University of Ottawa

Ottawa, Canada
Wesley Research Institute

Auchenflower, Australia

Conditions

Explore the condition pages connected to this study.

MYOTONIC DYSTROPHY TYPE 1 (DM1)