RECRUITINGPhase 3INTERVENTIONAL
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
About This Trial
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.
Who May Be Eligible (Plain English)
Key Who May Qualify:
- Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene
Key Who Should NOT Join This Trial:
- 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
Other protocol defined Inclusion/Exclusion criteria may apply.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Key Inclusion Criteria:
* Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene
Key Exclusion Criteria:
* 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
Other protocol defined Inclusion/Exclusion criteria may apply.
Treatments Being Tested
DRUG
VX-121/TEZ/D-IVA
Fixed-dose combination for oral administration.
Locations (20)
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