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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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

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MYOTONIC DYSTROPHY TYPE 1 (DM1)