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

May 26, 2026 updated by: Vertex Pharmaceuticals Incorporated

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

297

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Innsbruck, Austria
        • Medizinische Universität Innsbruck - Heilstättenschule Universitätsklinik
  • Belgium
      • Brussels, Belgium
        • CUB Hôpital Erasme
      • Brussels, Belgium
        • UZ Brussel - Campus Jette
      • Edegem, Belgium
        • Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital
      • Ghent, Belgium
        • Universitair Ziekenhuis Gent
      • Leuven, Belgium
        • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
      • Woluwe-Saint-Lambert, Belgium
        • Universite Catholique de Louvain - Pulmonology
  • Canada
      • Edmonton, Canada
        • Stollery Children's Hospital
      • Montreal, Canada
        • McGill University Health Centre, Glen Site, Montreal Children's Hospital
      • Montreal, Canada
        • Le Centre de recherche du CHUM (CRCHUM)
      • Toronto, Canada
        • Hospital for Sick Children - Pulmonology
      • Vancouver, Canada
        • British Columbia Children's Hospital
      • Vancouver, Canada
        • St. Paul's Hospital - Pulmonology
  • Czechia
      • Brno, Czechia
        • Klinika Detskych Infekcnich Nemoci
      • Prague, Czechia
        • Motol University Hospital
  • France
      • Bron, France
        • Hopital Femme Mere-Enfant
      • Créteil, France
        • Centre Hospitalier Intercommunal Creteil - Pulmonology
      • Lille, France
        • Institut Cœur Poumon, CHU de Lille
      • Marseille, France
        • Hôpital Nord
      • Montpellier, France
        • Hopital Arnaud de Villeneuve - Service de Pneumologie et Maladies Respiratoires
      • Nantes, France
        • Hopital Nord Laennec
      • Nice, France
        • CHU de Nice - Hôpital Pasteur 2
      • Paris, France
        • Hopital Cochin - Pulmonology
      • Paris, France
        • Hopital Necker Enfants Malades - Pulmonology
      • Paris, France
        • Hopital Robert Debre - Pulmonology
      • Pierre-Bénite, France
        • Hôpital Lyon Sud - Pulmonology
      • Reims, France
        • Hôpital américain de Reims - Département de pédiatrie A
      • Rennes, France
        • Hôpital Sud - Rennes
      • Roscoff, France
        • Centre de Perharidy, Roscoff
      • Toulouse, France
        • Hopital Larrey
      • Tours, France
        • Hopital Bretonneau
  • Germany
      • Berlin, Germany
        • Charité - Paediatric Pulmonology Department
      • Cologne, Germany
        • University Hospital Cologne (Koeln) - CF-Studienzentrum
      • Essen, Germany
        • Ruhrlandklinik
      • Frankfurt, Germany
        • Klinikum der Johann-Wolfgang-Goethe Universitaet - Pulmonology
      • Giessen, Germany
        • Justus-Liebig-Universität Gießen
      • Halle, Germany
        • Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
      • Hanover, Germany
        • Medizinische Hochschule Hannover
      • Hanover, Germany
        • Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre
      • Jena, Germany
        • Mukoviszidose-Zentrum am Universitätsklinikum Jena
      • Mainz, Germany
        • Johannes Gutenberg-Universitaet
      • München, Germany
        • Dr. von Haunersches Kinderspital - Pulmonology
      • München, Germany
        • Lungenheilkunde Munchen - Pasing
      • Münster, Germany
        • Klinik für Kinder- und Jugendmedizin
      • Potsdam, Germany
        • Klinikum Westbrandenburg (CF)
  • Hungary
      • Budapest, Hungary
        • National Koranyi Institute for TBC and Pulmonology
  • Italy
      • Ancona, Italy
        • Azienda Ospedaliero Universitaria delle Marche
      • Florence, Italy
        • Azienda Ospedaliero Universitaria Ospedale Meyer
      • Genova, Italy
        • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
      • Messina, Italy
        • Policlinico Universitario G. Martino
      • Milan, Italy
        • Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
      • Milan, Italy
        • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
      • Naples, Italy
        • Azienda Ospedaliero Universitaria Federico II Napoli
      • Orbassano, Italy
        • Azienda Ospedaliero Universitaria (AOU) San Luigi Gonzaga
      • Potenza, Italy
        • Azienda Ospedaliera Regionale 'San Carlo', Potenza
      • Rome, Italy
        • IRCSS Ospedale Pediatrico Bambino Gesu - Pulmonology
      • Verona, Italy
        • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
  • Netherlands
      • Amsterdam, Netherlands
        • Amsterdam UMC - locatie AMC - Afdeling Longgeneeskunde
      • Nijmegen, Netherlands
        • Radboud University Medical Center - Pulmonology
      • Rotterdam, Netherlands
        • Erasmus Medisch Centrum - Pulmonology
      • The Hague, Netherlands
        • Hagaziekenhuis
      • Utrecht, Netherlands
        • UMCU, Locatie Wilhelmina Kinderziekenhuis
  • Norway
      • Oslo, Norway
        • Oslo University Hospital, Department of Paediatric Medicine
  • Poland
      • Gdansk, Poland
        • Pediatric Hospital Polanki named of Maciej Płażyński
      • Łomianki, Poland
        • Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym
  • Portugal
      • Lisbon, Portugal
        • Unidade Local de Saúde de Santa Maria, E.P.E. - Pulmonology
  • Spain
      • Barakaldo, Spain
        • Hospital de Cruces
      • Barcelona, Spain
        • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
      • Barcelona, Spain
        • Hospital Saint Joan de Deu
      • Jerez de la Frontera, Spain
        • Hospital Universitario de Jerez de la Frontera
      • Madrid, Spain
        • Hospital Infantil Universitario Niño Jesús
      • Madrid, Spain
        • Hospital Universitario 12 de Octubre - Pulmonology
      • Madrid, Spain
        • Hospital Universitario Ramon y Cajal - Pulmonology
      • Madrid, Spain
        • La Paz Infantil
      • Murcia, Spain
        • Hospital Virgen de la Arrixaca
      • Sabadell, Spain
        • Institut d´Investigacio i Innovacio Parc Tauli
      • Seville, Spain
        • Hospital Universitario Virgen del Rocío
      • Valencia, Spain
        • Hospital Universitario y Politecnico La Fe - Pulmonology
  • Sweden
      • Gothenburg, Sweden
        • Sahlgrenska Universitetssjukhuset - Göteborg CF-center
      • Stockholm, Sweden
        • Karolinska University Hospital - Pulmonology
  • Switzerland
      • Zurich, Switzerland
        • Kinderspital Zuerich

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ELX/TEZ/IVA

Part 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.
Drug: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablets for oral administration.
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor
Drug: IVA
Tablets for oral administration.
Other Names:
  • VX-770
  • ivacaftor

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)
Time Frame: From Baseline up to Week 96
From Baseline up to Week 96
Part A: Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline up to Week 96
From Baseline up to Week 96
Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Time Frame: From Baseline up to Week 96
From Baseline up to Week 96
Part A: Absolute Change in Body Mass Index (BMI)
Time Frame: From Baseline up to Week 96
From Baseline up to Week 96
Part A: Absolute Change in Weight
Time Frame: From Baseline up to Week 96
From Baseline up to Week 96
Part A: Number of Pulmonary Exacerbations (PEx)
Time Frame: From Baseline up to Week 96
From Baseline up to Week 96

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 23, 2022

Primary Completion (Estimated)

April 6, 2027

Study Completion (Estimated)

April 6, 2027

Study Registration Dates

First Submitted

April 8, 2022

First Submitted That Met QC Criteria

April 8, 2022

First Posted (Actual)

April 15, 2022

Study Record Updates

Last Update Posted (Actual)

May 29, 2026

Last Update Submitted That Met QC Criteria

May 26, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX21-445-125
  • 2021-005914-33 (EudraCT Number)
  • 2024-515637-14-00 (Other Identifier: EU Trial (CTIS) Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

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/

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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