Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
- Registration Number
- NCT05422222
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 210
- Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene
Key
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A: VX-121/TEZ/D-IVA VX-121/TEZ/D-IVA Participants will receive VX-121/TEZ/D-IVA in the morning. Part B: VX-121/TEZ/D-IVA VX-121/TEZ/D-IVA Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.
- Primary Outcome Measures
Name Time Method Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) From Day 1 up to Day 50 Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) From Day 1 up to Week 28 Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites From Day 1 up to Day 22
- Secondary Outcome Measures
Name Time Method Part B: Absolute Change in Body Mass Index (BMI) From Baseline at Week 24 Part B: Absolute Change in BMI-for-age Z-score From Baseline at Week 24 Part B: Absolute Change in Weight-for-age Z-score From Baseline at Week 24 Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites From Day 1 up to Week 16 Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) From Baseline Through Week 24 Part B: Proportion of Participants With SwCl <30 mmol/L From Baseline Through Week 24 Part B: Number of CF-Related Hospitalizations From Baseline Through Week 24 Part B: Absolute Change in Weight From Baseline at Week 24 Part B: Absolute Change in Weight-for-length From Baseline at Week 24 Part B: Absolute Change in Height From Baseline at Week 24 Part B: Absolute Change in Height-for-age Z-score From Baseline at Week 24 Part B: Absolute Change in Sweat Chloride (SwCl) From Baseline Through Week 24 Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale At Day 1 and Week 24 Part B: Number of Pulmonary Exacerbation (PEx) From Baseline Through Week 24 Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score From Baseline Through Week 24 Part B: Absolute Change in Weight-for-length Z-score From Baseline at Week 24 Part B: Absolute Change in Length From Baseline at Week 24 Part B: Absolute Change in Length-for-age Z-score From Baseline at Week 24 Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L) From Baseline Through Week 24
Trial Locations
- Locations (37)
The Hospital for Sick Children
🇨🇦Toronto, Canada
Charité Universitätsmedizin BerlinX
🇩🇪Berlin, Germany
Starship Children's Hospital
🇳🇿Grafton, New Zealand
Stanford University Clinical and Translational Research Unit
🇺🇸Palo Alto, California, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
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
Washington University School of Medicine / St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Cohen Children's Medical Center
🇺🇸Lake Success, New York, 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
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, 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
British Columbia Children's Hospital
🇨🇦Vancouver, Canada
CHU Lyon - Hopital Femme Mere-Enfant
🇫🇷Bron Cedex, France
Hopital Necker, Enfants Malades
🇫🇷Paris Cedex 15, France
Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
Erasmus Medical Center / Sophia Children's Hospital
🇳🇱Rotterdam, Netherlands
Sahlgrenska Universitetssjukhuset
🇸🇪Göteborg, Sweden
Inselspital - Universitaetsspital Bern
🇨ðŸ‡Bern, Switzerland
Kinderspital Zurich
🇨ðŸ‡Zurich, Switzerland
Children and Young Adults Research Unit
🇬🇧Cardiff, United Kingdom
Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom