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

Description

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.

Conditions

Cystic Fibrosis

Study Overview

Study Details

Study overview

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.

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

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

Condition
Cystic Fibrosis
Intervention / Treatment

-

Contacts and Locations

Orange

Children's Hospital of Orange County, Orange, California, United States, 92868

Palo Alto

Stanford University, Palo Alto, California, United States, 94304

Aurora

Children's Hospital of Colorado, Aurora, Colorado, United States, 80045

Atlanta

The Emory Clinic / Children's Healthcare of Atlanta at Egleston, Atlanta, Georgia, United States, 30322

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Indianapolis

Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana, United States, 46202

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Minneapolis

Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota, United States, 55404

Kansas City

The Children's Mercy Hospital, Kansas City, Missouri, United States, 64108

Saint Louis

Washington University School of Medicine / St. Louis Children's Hospital, Saint Louis, Missouri, United States, 63110

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

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

Ages Eligible for Study

1 Year to 11 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Vertex Pharmaceuticals Incorporated,

Study Record Dates

2030-06