Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes
3 other identifiers
interventional
297
15 countries
81
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Nov 2022
Typical duration for phase_3
81 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
April 8, 2022
CompletedFirst Posted
Study publicly available on registry
April 15, 2022
CompletedStudy Start
First participant enrolled
November 23, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 6, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 6, 2027
January 21, 2026
January 1, 2026
4.4 years
April 8, 2022
January 20, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
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
Secondary Outcomes (6)
Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)
From Baseline up to Week 96
Part A: Absolute Change in Sweat Chloride (SwCl)
From Baseline up to Week 96
Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
From Baseline up to Week 96
Part A: Absolute Change in Body Mass Index (BMI)
From Baseline up to Week 96
Part A: Absolute Change in Weight
From Baseline up to Week 96
- +1 more secondary outcomes
Study Arms (1)
ELX/TEZ/IVA
EXPERIMENTALPart A: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for 96 weeks. Part B: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for an additional 96 weeks.
Interventions
Fixed-dose combination (FDC) tablets for oral administration.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- History of study drug intolerance in the parent study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (81)
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
Johannes Gutenberg-Universitaet
Mainz, Germany
Dr. von Haunersches Kinderspital - Pulmonology
München, Germany
Lungenheilkunde Munchen - Pasing
München, Germany
Klinik für Kinder- und Jugendmedizin
Münster, Germany
Klinikum Westbrandenburg (CF)
Potsdam, Germany
National Koranyi Institute for TBC and Pulmonology
Budapest, Hungary
Azienda Ospedaliero Universitaria delle Marche
Ancona, Italy
Azienda Ospedaliero Universitaria Ospedale Meyer
Florence, Italy
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
Genova, Italy
Policlinico Universitario G. Martino
Messina, Italy
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
Milan, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
Azienda Ospedaliero Universitaria Federico II Napoli
Naples, Italy
Azienda Ospedaliero Universitaria (AOU) San Luigi Gonzaga
Orbassano, Italy
Azienda Ospedaliera Regionale 'San Carlo', Potenza
Potenza, Italy
IRCSS Ospedale Pediatrico Bambino Gesu - Pulmonology
Rome, Italy
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
Verona, Italy
Amsterdam UMC - locatie AMC - Afdeling Longgeneeskunde
Amsterdam, Netherlands
Radboud University Medical Center - Pulmonology
Nijmegen, Netherlands
Erasmus Medisch Centrum - Pulmonology
Rotterdam, Netherlands
HagaZiekenhuis
The Hague, Netherlands
UMCU, Locatie Wilhelmina Kinderziekenhuis
Utrecht, Netherlands
Oslo University Hospital, Department of Paediatric Medicine
Oslo, Norway
Pediatric Hospital Polanki named of Maciej Płażyński
Gdansk, Poland
Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym
Łomianki, Poland
Unidade Local de Saúde de Santa Maria, E.P.E. - Pulmonology
Lisbon, Portugal
Hospital de Cruces
Barakaldo, Spain
Hospital Saint Joan de Deu
Barcelona, Spain
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona, Spain
Hospital Universitario de Jerez de la Frontera
Jerez de la Frontera, Spain
Hospital Infantil Universitario Nino Jesus
Madrid, Spain
Hospital Universitario 12 de Octubre - Pulmonology
Madrid, Spain
Hospital Universitario Ramon y Cajal - Pulmonology
Madrid, Spain
La Paz Infantil
Madrid, Spain
Hospital Virgen de la Arrixaca
Murcia, Spain
Institut d´Investigacio i Innovacio Parc Tauli
Sabadell, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Hospital Universitario y Politecnico La Fe - Pulmonology
Valencia, Spain
Sahlgrenska Universitetssjukhuset - Göteborg CF-center
Gothenburg, Sweden
Karolinska University Hospital - Pulmonology
Stockholm, Sweden
Kinderspital Zuerich
Zurich, Switzerland
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 8, 2022
First Posted
April 15, 2022
Study Start
November 23, 2022
Primary Completion (Estimated)
April 6, 2027
Study Completion (Estimated)
April 6, 2027
Last Updated
January 21, 2026
Record last verified: 2026-01
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/