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Active, not recruitingPhase 1/2

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

Key facts

Who runs it
Vertex Pharmaceuticals Incorporated
Where
Boston, Fairway, Gainesville, Philadelphia, Richmond, San Carlos, St Louis, and Winston-Salem, United StatesSee the 8 sitesAlso in 9 other countries, 21 sites
Status
Active, not recruiting
Phase
Phase 1/2
People taking part
47 people enrolled
Started
20 Feb 2024
Expected to end
2 Feb 2027
Type of study
Interventional, 4 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • VX-670
  • Placebo
Record updated
20 August 2026

Who can take part

Age
18 years to 64 years
Sex
Women and men
Healthy volunteers
Cannot take part

The registry criteria, word for word

Show all 2 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • History of any illness or any clinical condition as pre-specified in the protocol; Other protocol defined Inclusion/Exclusion criteria may apply

Where in United States

Also in 9 other countries, 21 sites

  • Boston Children's HospitalBoston, United States
  • University of Kansas Medical CenterFairway, United States
  • University of Florida Clinical Research CenterGainesville, United States
  • University of PennsylvaniaPhiladelphia, United States
  • Virginia Commonwealth University - Sanger HallRichmond, United States
  • Stanford Neuromuscular ResearchSan Carlos, United States
  • Washington University School of Medicine/St. Louis Children's HospitalSt Louis, United States
  • Wake Forest Baptist HealthWinston-Salem, United States

About the trial

What the trial wants to find out

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.

The 4 groups in the trial

  • Part A: Single Ascending DoseTreatment being tested

    Participants will be randomized to receive a single dose of different dose levels of VX-670.

    Treatment: VX-670

  • Part A: PlaceboPlacebo, for comparison

    Participants will be randomized to receive single dose of placebo matched to VX-670.

    Treatment: Placebo

  • Part B: Single and Multiple Ascending DoseTreatment being tested

    Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A.

    Treatment: VX-670

  • Part B: PlaceboPlacebo, for comparison

    Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.

    Treatment: Placebo

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1
Record updated
20 August 2026