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

NCT ID NCT06926621

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 11, 2026 · Updated 4 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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 44 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2025

Expected to finish

Jan 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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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.

Myotonic dystrophy type 1 (DM1)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    15 sites in 8 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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

  • St. Louis Children's Hospital - Neurology

    St Louis, Missouri, 63110, United States

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg - Pulmonology

    Leuven, Belgium

  • Universite Laval - Neurology

    Québec, Canada

  • University of Ottawa

    Ottawa, Canada

  • Virginia Commonwealth University - Sanger Hall

    Richmond, Virginia, 23298, United States

  • Wesley Research Institute

    Auchenflower, Australia

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