Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

NCT05331183 · tracked via the Priya Life Science Italy tracker

Sponsor

Vertex Pharmaceuticals Incorporated

Phase

Phase 3

Started

2022-11-23

Last updated

2026-09-10

Condition(s) studied

Cystic Fibrosis

Investigational drug(s) / intervention(s)

ELX/TEZ/IVA →IVA →

ELX/TEZ/IVA: Fixed-dose combination (FDC) tablets for oral administration.

IVA: Tablets for oral administration.

Study summary

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Eligibility

Sex

ALL

Min age

6 Years

Max age

—

Healthy volunteers

Key Inclusion Criteria: Part A: Completed study drug treatment in parent study or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study Part B: Completed study drug treatment in Part A or had study drug interruption(s) in Part A but completed study visits up to the last scheduled visit of the treatment period of Part A Key Exclusion Criteria: History of study drug intolerance in the parent study Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcome measure(s)

Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) — Day 1 up to Week 196

Trial sites (81)

Facility	City	Region
Medizinische Universität Innsbruck - Heilstättenschule Universitätsklinik	Innsbruck	Austria
CUB Hôpital Erasme	Brussels	Belgium
UZ Brussel - Campus Jette	Brussels	Belgium
Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital	Edegem	Belgium
Universitair Ziekenhuis Gent	Ghent	Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg	Leuven	Belgium
Universite Catholique de Louvain - Pulmonology	Woluwe-Saint-Lambert	Belgium
Stollery Children's Hospital	Edmonton	Canada
Le Centre de recherche du CHUM (CRCHUM)	Montreal	Canada
McGill University Health Centre, Glen Site, Montreal Children's Hospital	Montreal	Canada
Hospital for Sick Children - Pulmonology	Toronto	Canada
British Columbia Children's Hospital	Vancouver	Canada
St. Paul's Hospital - Pulmonology	Vancouver	Canada
Klinika Detskych Infekcnich Nemoci	Brno	Czechia
Motol University Hospital	Prague	Czechia
Hopital Femme Mere-Enfant	Bron	France
Centre Hospitalier Intercommunal Creteil - Pulmonology	Créteil	France
Institut Cœur Poumon, CHU de Lille	Lille	France
Hopital Nord	Marseille	France
Hopital Arnaud de Villeneuve - Service de Pneumologie et Maladies Respiratoires	Montpellier	France
Hôpital Nord Laennec	Nantes	France
CHU de Nice - Hôpital Pasteur 2	Nice	France
Hopital Cochin - Pulmonology	Paris	France
Hopital Necker Enfants Malades - Pulmonology	Paris	France
Hopital Robert Debre - Pulmonology	Paris	France
Hôpital Lyon Sud - Pulmonology	Pierre-Bénite	France
Hôpital américain de Reims - Département de pédiatrie A	Reims	France
Hôpital Sud - Rennes	Rennes	France
Centre de Perharidy, Roscoff	Roscoff	France
Hopital Larrey	Toulouse	France
Hopital Bretonneau	Tours	France
Charité - Paediatric Pulmonology Department	Berlin	Germany
University Hospital Cologne (Koeln) - CF-Studienzentrum	Cologne	Germany
Ruhrlandklinik	Essen	Germany
Klinikum der Johann-Wolfgang-Goethe Universitaet - Pulmonology	Frankfurt	Germany
Justus-Liebig-Universität Gießen	Giessen	Germany
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie	Halle	Germany
Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre	Hanover	Germany
Medizinische Hochschule Hannover	Hanover	Germany
Mukoviszidose-Zentrum am Universitätsklinikum Jena	Jena	Germany

+ 41 more sites — see the full list on the official registry below.

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Other trials for the same condition

A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI 3720931 (Lenticlair™-ON) Active, not recruiting

A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not 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 NCT05331183 on ClinicalTrials.gov ↗ ← All trials in Italy