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
2 other identifiers
interventional
210
10 countries
38
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jun 2022
Longer than P75 for phase_3
38 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 13, 2022
CompletedFirst Posted
Study publicly available on registry
June 16, 2022
CompletedStudy Start
First participant enrolled
June 21, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 30, 2030
May 28, 2026
May 1, 2026
8 years
June 13, 2022
May 27, 2026
Conditions
Outcome Measures
Primary Outcomes (3)
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
From Day 1 up to Day 22
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
Secondary Outcomes (19)
Part B: Absolute Change in Sweat Chloride (SwCl)
From Baseline Through 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: Drug Acceptability Assessment Using Modified Facial Hedonic Scale
At Day 1 and Week 24
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
From Baseline Through Week 24
Part B: Number of Pulmonary Exacerbation (PEx)
From Baseline Through Week 24
- +14 more secondary outcomes
Study Arms (2)
Part A: VX-121/TEZ/D-IVA
EXPERIMENTALParticipants will receive VX-121/TEZ/D-IVA in the morning.
Part B: VX-121/TEZ/D-IVA
EXPERIMENTALParticipants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.
Interventions
Fixed-dose combination for oral administration.
Eligibility Criteria
You may qualify if:
- Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (38)
Children's Hospital of Orange County
Orange, California, 92868, United States
Stanford University Clinical and Translational Research Unit
Palo Alto, California, 94304, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
The Emory Clinic / Children's Healthcare of Atlanta at Egleston
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Cohen Children's Medical Center
Lake Success, New York, 11042, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Texas Children's Hospital - Wallace Tower
Houston, Texas, 77030, United States
Vermont Lung Center
Colchester, Vermont, 05446, United States
American Family Childrens Hospital
Madison, Wisconsin, 53792, 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
Related Publications (1)
Hoppe JE, Kasi AS, Pittman JE, Jensen R, Thia LP, Robinson P, Tirakitsoontorn P, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Salinas DB, Zhu J, Chen YC, Rodriguez-Romero V, Sosnay PR, Davies G; VX21-121-105 Study Group. Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med. 2025 Mar;13(3):244-255. doi: 10.1016/S2213-2600(24)00407-7. Epub 2025 Jan 2.
PMID: 39756425DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 13, 2022
First Posted
June 16, 2022
Study Start
June 21, 2022
Primary Completion (Estimated)
June 30, 2030
Study Completion (Estimated)
June 30, 2030
Last Updated
May 28, 2026
Record last verified: 2026-05
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/