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

NCT05422222 · tracked via the Priya Life Science Germany tracker

Sponsor

Vertex Pharmaceuticals Incorporated

Phase

Phase 3

Started

2022-06-21

Last updated

2026-05-28

Condition(s) studied

Cystic Fibrosis

Investigational drug(s) / intervention(s)

VX-121/TEZ/D-IVA →

VX-121/TEZ/D-IVA: Fixed-dose combination for oral administration.

Study 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

Sex

ALL

Min age

1 Year

Max age

11 Years

Healthy volunteers

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.

Primary outcome measure(s)

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

Trial sites (38)

Facility	City	Region
Children's Hospital of Orange County	Orange	California
Stanford University Clinical and Translational Research Unit	Palo Alto	California
Children's Hospital of Colorado	Aurora	Colorado
The Emory Clinic / Children's Healthcare of Atlanta at Egleston	Atlanta	Georgia
Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
Riley Hospital for Children at Indiana University Health	Indianapolis	Indiana
Boston Children's Hospital	Boston	Massachusetts
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
The Children's Mercy Hospital	Kansas City	Missouri
Washington University School of Medicine / St. Louis Children's Hospital	St Louis	Missouri
Cohen Children's Medical Center	Lake Success	New York
Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center	Cleveland	Ohio
Nationwide Children's Hospital	Columbus	Ohio
Oregon Health & Science University	Portland	Oregon
UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
Texas Children's Hospital - Wallace Tower	Houston	Texas
Vermont Lung Center	Colchester	Vermont
American Family Childrens Hospital	Madison	Wisconsin
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

More Vertex Pharmaceuticals Incorporated trials in Germany

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting

A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes Recruiting

A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 Diabetes Active, not recruiting

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) Active, not recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) Active, not recruiting

Other trials for the same condition

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting

Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators Recruiting

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del Active, not recruiting

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older Enrolling by invitation

Development and Validation of a Disease Specific PROM to Assess Abdominal Involvement in Patients With CF (CFAbd-Score) Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05422222 on ClinicalTrials.gov ↗ ← All trials in Germany