NCT06185764PHASE1, PHASE2Recruiting

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

Vertex Pharmaceuticals Incorporated

Start Date

2/20/2024

Completion Date

2/2/2027

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

Age Range: 18 years to 64 years

Key Inclusion Criteria: \- 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 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.

Interventions

DRUG

VX-670

DRUG

Placebo

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Conditions

Myotonic Dystrophy Type 1 (DM1)

Locations

Stanford Neuromuscular Research

San Carlos, California 94070

United States

University of Florida Clinical Research Center

Gainesville, Florida 32608

United States

University of Kansas Medical Center

Fairway, Kansas 66205

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

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

St Louis, Missouri 63110

United States

Wake Forest Baptist Health

Winston-Salem, North Carolina 27104

United States

University of Pennsylvania

Philadelphia, Pennsylvania 19104

United States

Virginia Commonwealth University (Sanger Hall)

Richmond, Virginia 23298

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