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Clinical Trials in the UK / NCT05844449
Enrolling by invitation Phase 3

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

NCT05844449 · tracked via the Priya Life Science UK tracker
Sponsor
Vertex Pharmaceuticals Incorporated
Phase
Phase 3
Started
2023-08-11
Last updated
2026-04-29

Condition(s) studied

Cystic Fibrosis

Investigational drug(s) / intervention(s)

VNZ/TEZ/D-IVA

VNZ/TEZ/D-IVA: Fixed-dose combination tablets or granules for oral administration.

Study summary

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

Eligibility

Sex
ALL
Min age
1 Year
Max age
Healthy volunteers
No
Key Inclusion Criteria: Parts A and B: • Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222) Part B: -Meets at least 1 of the following criteria: * Completed study drug treatment in Part A * Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A Key Exclusion Criteria: * Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug * History of solid organ, hematological transplantation, or cancer * History of drug intolerance in the parent study Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcome measure(s)

Trial sites (37)

FacilityCityRegionStatus
Children's Hospital of Orange County Orange California
Stanford University - Palo Alto - Pulmonology Palo Alto California
Children's Hospital of Colorado - Pulmonology Aurora Colorado
The Emory Clinic - Clifton Road - Pulmonology Atlanta Georgia
Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology Chicago Illinois
JW Riley Hospital for Children - Pulmonology Indianapolis Indiana
Boston Children's Hospital Boston Massachusetts
Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota Minneapolis Minnesota
The Children's Mercy Hospital Kansas City Missouri
St. Louis Children's Hospital - Pulmonology St Louis Missouri
Cohen Children's Medical Center - Lakeville Road Lake Success New York
Cincinnati Children's Hospital Medical Center - Pulmonology Cincinnati Ohio
UH Cleveland Medical Center - Pulmonology Cleveland Ohio
Nationwide Children's Hospital Columbus Ohio
Children's Hospital of Pittsburgh - Pulmonology Pittsburgh Pennsylvania
Texas Children's Hospital - Wallace Tower - Pulmonology Houston Texas
Vermont Lung Center Colchester Vermont
American Family Childrens Hospital Madison Wisconsin
The Kids Research Institute Australia Nedlands Australia
Women's & Children's Hospital North Adelaide Australia
The Royal Children's Hospital Melbourne Parkville Australia
Children's Health Queensland Hospital and Health Service South Brisbane Australia
Hospital for Sick Children - Pulmonology Toronto Canada
British Columbia Children's Hospital Vancouver Canada
Hopital Femme Mere-Enfant Bron France
Hopital Necker Enfants Malades - Pulmonology Paris France
Charite Paediatric Pulmonology Department Berlin Germany
Universitatsklinikum Essen Essen Germany
Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre Hanover Germany
Sophia Children's Hospital Rotterdam Netherlands
Starship Child Health Grafton New Zealand
Sahlgrenska Universitetssjukhuset - Göteborg CF-center Gothenburg Sweden
Karolinska University Hospital - Pulmonology Stockholm Sweden
Inselspital Bern Bern Switzerland
Kinderspital Zurich - Abteilung Pneumologie Zurich Switzerland
Noah's Ark Children's Hospital for Wales Cardiff United Kingdom
Great Ormond Street Hospital for Children London United Kingdom

More Vertex Pharmaceuticals Incorporated trials in the UK

Other trials for the same condition

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 NCT05844449 on ClinicalTrials.gov ↗ ← All trials in the UK