RecruitingPhase 1Phase 2NCT06185764

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

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

Sponsor

Vertex Pharmaceuticals Incorporated

Enrollment

52 participants

Start Date

Feb 20, 2024

Study Type

INTERVENTIONAL

Conditions

Myotonic Dystrophy Type 1 (DM1)

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

Min Age: 18 YearsMax Age: 64 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Placebo and a drug called VX-670 for people with myotonic dystrophy type 1 (dm1). The study is currently recruiting participants at 26 locations. People eligible for this study include aged 18 Years to 64 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGVX-670

Solution for intravenous administration.

DRUGPlacebo

Solution for intravenous administration.

Locations(26)

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

View Full Details on ClinicalTrials.gov

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NCT06185764

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