A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Last updated: April 21, 2025
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Active - Recruiting

Phase

1/2

Condition

Muscular Dystrophy

Treatment

Placebo

VX-670

Clinical Study ID

NCT06185764
VX23-670-001
2023-506028-10-00
  • Ages 18-64
  • All Genders

Study Summary

The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Documented clinical diagnosis of DM1 with age of onset greater than (>) 1 year ofage and documented positive genetic test for DM1 in the subject with cytosinethymine guanine (CTG) repeat of at least 100

Exclusion

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.

Study Design

Total Participants: 36
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1/2
Study Start date:
February 20, 2024
Estimated Completion Date:
December 31, 2026

Connect with a study center

  • Wesley Research Institute

    Auchenflower,
    Australia

    Active - Recruiting

  • Neuroscience Clinical Trials Unit, Alfred Brain

    Melbourne,
    Australia

    Active - Recruiting

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven,
    Belgium

    Active - Recruiting

  • Hopital de Chicoutimi

    Chicoutimi,
    Canada

    Active - Recruiting

  • Altasciences Montreal

    Mont-Royal,
    Canada

    Site Not Available

  • Altasciences Montreal

    Montreal,
    Canada

    Active - Recruiting

  • McGIll University

    Montreal,
    Canada

    Active - Recruiting

  • University of Ottawa

    Ottawa,
    Canada

    Active - Recruiting

  • CHU Research Centre of Quebec

    Quebec,
    Canada

    Active - Recruiting

  • Neuromuscular Reference Center Institute of Myology

    Paris,
    France

    Active - Recruiting

  • Ludwig Maximilians Universitaet Muenchen

    Muenchen,
    Germany

    Active - Recruiting

  • Centro Clinico NeMO

    Milan,
    Italy

    Active - Recruiting

  • Maastricht University Medical Center

    Maastricht,
    Netherlands

    Active - Recruiting

  • Hospital Universitario y Politécnico La Fe

    Valencia,
    Spain

    Active - Recruiting

  • Clinical Research Facility, Queen Elizabeth University Hospital

    Glasgow,
    United Kingdom

    Active - Recruiting

  • Leonard Wolfson Experimental Neurology Centre CRF

    London,
    United Kingdom

    Active - Recruiting

  • St. George's University Hospital

    London,
    United Kingdom

    Active - Recruiting

  • University College London

    London,
    United Kingdom

    Active - Recruiting

  • Salford Royal Hospital

    Salford,
    United Kingdom

    Active - Recruiting

  • Royal Hallamshire Hospital

    Sheffield,
    United Kingdom

    Active - Recruiting

  • Stanford Neuromuscular Research

    San Carlos, California 94070
    United States

    Active - Recruiting

  • University of Florida Clinical Research Center

    Gainesville, Florida 32608
    United States

    Active - Recruiting

  • University of Kansas Medical Center

    Fairway, Kansas 66205
    United States

    Active - Recruiting

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

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

    Saint Louis, Missouri 63110
    United States

    Active - Recruiting

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina 27104
    United States

    Active - Recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Virginia Commonwealth University (Sanger Hall)

    Richmond, Virginia 23298
    United States

    Active - Recruiting

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