Vertex tests long-term safety of VX-670 for muscle disease

NCT ID NCT06926621

First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 times

Summary

This study tests the long-term safety and effectiveness of an experimental drug called VX-670 in adults with myotonic dystrophy type 1 (DM1). Participants who completed a previous VX-670 study can join. The drug is given through a vein, and researchers will monitor side effects and how the drug works in the body over time.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

VX-670 (given intravenously)

What this could lead to

If successful, this could support VX-670 as a long-term treatment to control myotonic dystrophy type 1 symptoms.

What could go wrong

This is an early Phase 2 extension study with only 44 participants, so results may not apply broadly. Long-term safety and effectiveness are still unproven.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

hereditary disease inherited neurodegenerative disorder muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy musculoskeletal system disorder Myotonia myotonic dystrophy myotonic dystrophy type 1 myotonic syndrome nervous system disorder neurodegenerative disease neuromuscular disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Altasciences Montreal

    Montreal, Canada

  • Friedrich Baur Institute

    München, Germany

  • Hospital Universitario y Politecnico La Fe - Neurology

    Valencia, Spain

  • Leonard Wolfson Experimental Neurology Centre CRF

    London, United Kingdom

  • Maastricht UMC

    Maastricht, Netherlands

  • 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