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

NCT ID NCT06185764

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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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
RECRUITING

Montreal, Canada
Boston Children's Hospital
RECRUITING

Boston, Massachusetts, 02115, United States
CHU Research Centre of Quebec
RECRUITING

Québec, Canada
Centro Clinico NeMO
RECRUITING

Milan, Italy
Clinical Research Facility, Queen Elizabeth University Hospital
RECRUITING

Glasgow, United Kingdom
Hopital de Chicoutimi
RECRUITING

Chicoutimi, Canada
Hospital Universitario y Politécnico La Fe
RECRUITING

Valencia, Spain
Leonard Wolfson Experimental Neurology Centre CRF
RECRUITING

London, United Kingdom
Ludwig Maximilians Universitaet Muenchen
RECRUITING

München, Germany
Maastricht University Medical Center
RECRUITING

Maastricht, Netherlands
McGIll University
RECRUITING

Montreal, Canada
Neuromuscular Reference Center Institute of Myology
RECRUITING

Paris, France
Neuroscience Clinical Trials Unit, Alfred Brain
RECRUITING

Melbourne, Australia
Royal Hallamshire Hospital
RECRUITING

Sheffield, United Kingdom
Salford Royal Hospital
RECRUITING

Salford, United Kingdom
St. George's University Hospital
RECRUITING

London, United Kingdom
Stanford Neuromuscular Research
RECRUITING

San Carlos, California, 94070, United States
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
RECRUITING

Leuven, Belgium
University of Florida Clinical Research Center
RECRUITING

Gainesville, Florida, 32608, United States
University of Kansas Medical Center
RECRUITING

Fairway, Kansas, 66205, United States
University of Ottawa
RECRUITING

Ottawa, Canada
University of Pennsylvania
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Virginia Commonwealth University (Sanger Hall)
RECRUITING

Richmond, Virginia, 23298, United States
Wake Forest Baptist Health
RECRUITING

Winston-Salem, North Carolina, 27104, United States
Washington University School of Medicine / St. Louis Children's Hospital
RECRUITING

St Louis, Missouri, 63110, United States
Wesley Research Institute
RECRUITING

Auchenflower, Australia

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