RecruitingPhase 3NCT05422222

Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age


Sponsor

Vertex Pharmaceuticals Incorporated

Enrollment

210 participants

Start Date

Jun 21, 2022

Study Type

INTERVENTIONAL

Conditions

Summary

The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.


Eligibility

Min Age: 1 YearMax Age: 11 Years

Inclusion Criteria1

  • Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene

Exclusion Criteria3

  • History of solid organ, hematological transplantation, or cancer
  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
  • Lung infection with organisms associated with a more rapid decline in pulmonary status

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Interventions

DRUGVX-121/TEZ/D-IVA

Fixed-dose combination for oral administration.


Locations(38)

Children's Hospital of Orange County

Orange, California, United States

Stanford University Clinical and Translational Research Unit

Palo Alto, California, United States

Children's Hospital of Colorado

Aurora, Colorado, United States

The Emory Clinic / Children's Healthcare of Atlanta at Egleston

Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, United States

The Children's Mercy Hospital

Kansas City, Missouri, United States

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

St Louis, Missouri, United States

Cohen Children's Medical Center

Lake Success, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Nationwide Children's Hospital

Columbus, Ohio, United States

Oregon Health & Science University

Portland, Oregon, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Texas Children's Hospital - Wallace Tower

Houston, Texas, United States

Vermont Lung Center

Colchester, Vermont, United States

American Family Childrens Hospital

Madison, Wisconsin, United States

The Kids Research Institute Australia

Nedlands, Australia

Women's and Children's Hospital

North Adelaide, Australia

The Royal Children's Hospital

Parkville, Australia

Queensland Children's Hospital

South Brisbane, Australia

The Hospital for Sick Children

Toronto, Canada

British Columbia Children's Hospital

Vancouver, Canada

CHU Lyon - Hopital Femme Mere-Enfant

Bron, France

Hopital Necker, Enfants Malades

Paris, France

Charité - Paediatric Pulmonology Department

Berlin, Germany

Universitatsklinikum Essen

Essen, Germany

Medizinische Hochschule Hannover

Hanover, Germany

Erasmus Medical Center / Sophia Children's Hospital

Rotterdam, Netherlands

Starship Children's Hospital

Grafton, New Zealand

Sahlgrenska Universitetssjukhuset

Gothenburg, Sweden

Karolinska University Hospital - Pulmonology

Stockholm, Sweden

Inselspital - Universitaetsspital Bern

Bern, Switzerland

Kinderspital Zurich

Zurich, Switzerland

Children and Young Adults Research Unit

Cardiff, United Kingdom

Great Ormond Street Hospital for Children

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT05422222


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