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New hope for muscle disease: experimental drug VX-670 enters human trials

NCT ID NCT06185764

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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 21, 2026 · Updated 2 times

Summary

This early-stage trial tests the safety and tolerability of a new drug called VX-670 in 52 adults with myotonic dystrophy type 1 (DM1), a genetic condition that causes muscle weakness and other problems. Participants receive either VX-670 or a placebo, and researchers will monitor side effects and how the drug moves through the body. The goal is to find a safe dose for future studies, not to cure the disease.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

47 people

The number who actually took part.

Started

Feb 2024

Expected to finish

Feb 2027

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 to 64 years

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: \- Documented clinical diagnosis of DM1 with age of onset greater than (\>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100 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)

Contacts and locations

Locations

  • Altasciences Montreal

    Montreal, Canada

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHU Research Centre of Quebec

    Québec, Canada

  • Centro Clinico NeMO

    Milan, Italy

  • Clinical Research Facility, Queen Elizabeth University Hospital

    Glasgow, United Kingdom

  • Hopital de Chicoutimi

    Chicoutimi, Canada

  • Hospital Universitario y Politécnico La Fe

    Valencia, Spain

  • Leonard Wolfson Experimental Neurology Centre CRF

    London, United Kingdom

  • Ludwig Maximilians Universitaet Muenchen

    München, Germany

  • Maastricht University Medical Center

    Maastricht, Netherlands

  • McGIll University

    Montreal, Canada

  • Neuromuscular Reference Center Institute of Myology

    Paris, France

  • Neuroscience Clinical Trials Unit, Alfred Brain

    Melbourne, Australia

  • Royal Hallamshire Hospital

    Sheffield, United Kingdom

  • Salford Royal Hospital

    Salford, United Kingdom

  • St. George's University Hospital

    London, United Kingdom

  • Stanford Neuromuscular Research

    San Carlos, California, 94070, United States

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven, Belgium

  • University of Florida Clinical Research Center

    Gainesville, Florida, 32608, United States

  • University of Kansas Medical Center

    Fairway, Kansas, 66205, United States

  • University of Ottawa

    Ottawa, Canada

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • Virginia Commonwealth University (Sanger Hall)

    Richmond, Virginia, 23298, United States

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina, 27104, United States

  • Washington University School of Medicine / St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Wesley Research Institute

    Auchenflower, Australia

More trials for these conditions

Other studies related to the condition(s) this trial covers.