A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
NCT ID: NCT06185764
Last Updated: 2025-12-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
36 participants
INTERVENTIONAL
2024-02-20
2026-12-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Study of ATX-01 in Participants With DM1
NCT06300307
Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
NCT06138743
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
NCT02740972
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
NCT03508947
Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1
NCT00577577
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Part A: Single Ascending Dose
Participants will be randomized to receive a single dose of different dose levels of VX-670.
VX-670
Solution for intravenous administration.
Part A: Placebo
Participants will be randomized to receive single dose of placebo matched to VX-670.
Placebo
Solution for intravenous administration.
Part B: Single and Multiple Ascending Dose
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.
VX-670
Solution for intravenous administration.
Part B: Placebo
Participants will be randomized to receive single or multiple doses of placebo matched to VX-670.
Placebo
Solution for intravenous administration.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
VX-670
Solution for intravenous administration.
Placebo
Solution for intravenous administration.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
Exclusion Criteria
18 Years
64 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Stanford Neuromuscular Research
San Carlos, California, United States
University of Florida Clinical Research Center
Gainesville, Florida, United States
University of Kansas Medical Center
Fairway, Kansas, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, United States
Wake Forest Baptist Health
Winston-Salem, North Carolina, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Virginia Commonwealth University (Sanger Hall)
Richmond, Virginia, United States
Wesley Research Institute
Auchenflower, , Australia
Neuroscience Clinical Trials Unit, Alfred Brain
Melbourne, , Australia
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, , Belgium
Hopital de Chicoutimi
Chicoutimi, , Canada
Altasciences Montreal
Montreal, , Canada
McGIll University
Montreal, , Canada
University of Ottawa
Ottawa, , Canada
CHU Research Centre of Quebec
Québec, , Canada
Neuromuscular Reference Center Institute of Myology
Paris, , France
Ludwig Maximilians Universitaet Muenchen
München, , Germany
Centro Clinico NeMO
Milan, , Italy
Maastricht University Medical Center
Maastricht, , Netherlands
Hospital Universitario y Politécnico La Fe
Valencia, , Spain
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, , United Kingdom
Leonard Wolfson Experimental Neurology Centre CRF
London, , United Kingdom
St. George's University Hospital
London, , United Kingdom
Salford Royal Hospital
Salford, , United Kingdom
Royal Hallamshire Hospital
Sheffield, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2023-506028-10-00
Identifier Type: OTHER
Identifier Source: secondary_id
VX23-670-001
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.