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
Vertex Pharmaceuticals Incorporated
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
- Age range
- 1–11 years
- Sex
- All
- Healthy volunteers
- No
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.
Interventions
- DrugVX-121/TEZ/D-IVA
Fixed-dose combination for oral administration.
Locations (38)
- Children's Hospital of Orange CountyOrange, California
- Stanford University Clinical and Translational Research UnitPalo Alto, California
- Children's Hospital of ColoradoAurora, Colorado
- The Emory Clinic / Children's Healthcare of Atlanta at EglestonAtlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois
- Riley Hospital for Children at Indiana University HealthIndianapolis, Indiana