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Cystic FibrosisPhase 3NCT05331183

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

A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 81 sites in 15 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 6 years and older
Conditions the study is for
Cystic Fibrosis
What is being tested?
ELX/TEZ/IVA and IVA
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
297 participants
Where
81 sitesin 15 countries
Study dates
23 Nov 2022to 6 Apr 2027
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • History of study drug intolerance in the parent study
  • Other protocol defined Inclusion/Exclusion criteria may apply.

Trial locations and contacts

81 sites in 15 countries.

81 sites in 15 countries

France16 sites

  • Hopital Femme Mere-Enfant

    Bron

  • Centre Hospitalier Intercommunal Creteil - Pulmonology

    Créteil

  • Institut Cœur Poumon, CHU de Lille

    Lille

  • Hopital Nord

    Marseille

  • Hopital Arnaud de Villeneuve - Service de Pneumologie et Maladies Respiratoires

    Montpellier

  • Hôpital Nord Laennec

    Nantes

  • CHU de Nice - Hôpital Pasteur 2

    Nice

  • Hopital Cochin - Pulmonology

    Paris

  • Hopital Necker Enfants Malades - Pulmonology

    Paris

  • Hopital Robert Debre - Pulmonology

    Paris

  • Hôpital Lyon Sud - Pulmonology

    Pierre-Bénite

  • Hôpital américain de Reims - Département de pédiatrie A

    Reims

  • Hôpital Sud - Rennes

    Rennes

  • Centre de Perharidy, Roscoff

    Roscoff

  • Hopital Larrey

    Toulouse

  • Hopital Bretonneau

    Tours

Other trials in France

Germany14 sites

  • Charité - Paediatric Pulmonology Department

    Berlin

  • University Hospital Cologne (Koeln) - CF-Studienzentrum

    Cologne

  • Ruhrlandklinik

    Essen

  • Klinikum der Johann-Wolfgang-Goethe Universitaet - Pulmonology

    Frankfurt

  • Justus-Liebig-Universität Gießen

    Giessen

  • Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie

    Halle

  • Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre

    Hanover

  • Medizinische Hochschule Hannover

    Hanover

  • Mukoviszidose-Zentrum am Universitätsklinikum Jena

    Jena

  • Johannes Gutenberg-Universitaet

    Mainz

  • Dr. von Haunersches Kinderspital - Pulmonology

    München

  • Lungenheilkunde Munchen - Pasing

    München

  • Klinik für Kinder- und Jugendmedizin

    Münster

  • Klinikum Westbrandenburg (CF)

    Potsdam

Other trials in Germany

Spain12 sites

  • Hospital de Cruces

    Barakaldo

  • Hospital Saint Joan de Deu

    Barcelona

  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

    Barcelona

  • Hospital Universitario de Jerez de la Frontera

    Jerez de la Frontera

  • Hospital Infantil Universitario Nino Jesus

    Madrid

  • Hospital Universitario 12 de Octubre - Pulmonology

    Madrid

  • Hospital Universitario Ramon y Cajal - Pulmonology

    Madrid

  • La Paz Infantil

    Madrid

  • Hospital Virgen de la Arrixaca

    Murcia

  • Institut d´Investigacio i Innovacio Parc Tauli

    Sabadell

  • Hospital Universitario Virgen del Rocio

    Seville

  • Hospital Universitario y Politecnico La Fe - Pulmonology

    Valencia

Other trials in Spain

Italy11 sites

  • Azienda Ospedaliero Universitaria delle Marche

    Ancona

  • Azienda Ospedaliero Universitaria Ospedale Meyer

    Florence

  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

    Genova

  • Policlinico Universitario G. Martino

    Messina

  • Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

    Milan

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan

  • Azienda Ospedaliero Universitaria Federico II Napoli

    Naples

  • Azienda Ospedaliero Universitaria (AOU) San Luigi Gonzaga

    Orbassano

  • Azienda Ospedaliera Regionale 'San Carlo', Potenza

    Potenza

  • IRCSS Ospedale Pediatrico Bambino Gesu - Pulmonology

    Rome

  • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore

    Verona

Other trials in Italy

Belgium6 sites

  • CUB Hôpital Erasme

    Brussels

  • UZ Brussel - Campus Jette

    Brussels

  • Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital

    Edegem

  • Universitair Ziekenhuis Gent

    Ghent

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven

  • Universite Catholique de Louvain - Pulmonology

    Woluwe-Saint-Lambert

Other trials in Belgium

Canada6 sites

  • Stollery Children's Hospital

    Edmonton

  • Le Centre de recherche du CHUM (CRCHUM)

    Montreal

  • McGill University Health Centre, Glen Site, Montreal Children's Hospital

    Montreal

  • Hospital for Sick Children - Pulmonology

    Toronto

  • British Columbia Children's Hospital

    Vancouver

  • St. Paul's Hospital - Pulmonology

    Vancouver

Other trials in Canada

Netherlands5 sites

  • Amsterdam UMC - locatie AMC - Afdeling Longgeneeskunde

    Amsterdam

  • Radboud University Medical Center - Pulmonology

    Nijmegen

  • Erasmus Medisch Centrum - Pulmonology

    Rotterdam

  • HagaZiekenhuis

    The Hague

  • UMCU, Locatie Wilhelmina Kinderziekenhuis

    Utrecht

Other trials in Netherlands

Czechia2 sites

  • Klinika Detskych Infekcnich Nemoci

    Brno

  • Motol University Hospital

    Prague

Other trials in Czechia

Poland2 sites

  • Pediatric Hospital Polanki named of Maciej Płażyński

    Gdansk

  • Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym

    Łomianki

Other trials in Poland

Sweden2 sites

  • Sahlgrenska Universitetssjukhuset - Göteborg CF-center

    Gothenburg

  • Karolinska University Hospital - Pulmonology

    Stockholm

Other trials in Sweden

Austria1 site

  • Medizinische Universität Innsbruck - Heilstättenschule Universitätsklinik

    Innsbruck

Other trials in Austria

Hungary1 site

  • National Koranyi Institute for TBC and Pulmonology

    Budapest

Other trials in Hungary

Norway1 site

  • Oslo University Hospital, Department of Paediatric Medicine

    Oslo

Other trials in Norway

Portugal1 site

  • Unidade Local de Saúde de Santa Maria, E.P.E. - Pulmonology

    Lisbon

Other trials in Portugal

Switzerland1 site

  • Kinderspital Zuerich

    Zurich

Other trials in Switzerland

About this study

Official title: 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.

Conditions
Cystic Fibrosis
Being tested
ELX/TEZ/IVA, IVA
Medical subject headings
Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ELX/TEZ/IVADrug

    Also called VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor

    Fixed-dose combination (FDC) tablets for oral administration.

  • IVADrug

    Also called VX-770, ivacaftor

    Tablets for oral administration.

Study groups in NCT05331183
Study groupRoleWhat they receive
ELX/TEZ/IVAExperimentalPart 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.
Receives: Drug: ELX/TEZ/IVA; Drug: IVA

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Measured: Day 1 up to Week 196

Secondary outcomes (6)

  1. Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)

    Measured: From Baseline up to Week 96

  2. Part A: Absolute Change in Sweat Chloride (SwCl)

    Measured: From Baseline up to Week 96

  3. Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

    Measured: From Baseline up to Week 96

  4. Part A: Absolute Change in Body Mass Index (BMI)

    Measured: From Baseline up to Week 96

  5. Part A: Absolute Change in Weight

    Measured: From Baseline up to Week 96

  6. Part A: Number of Pulmonary Exacerbations (PEx)

    Measured: From Baseline up to Week 96

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Apr 2022
  2. Study start23 Nov 2022
  3. Primary completion6 Apr 2027 (estimated)
  4. Study completion6 Apr 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
297 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05331183
Sponsor's protocol ID
VX21-445-125
Other IDs
2021-005914-33, 2024-515637-14-00
Status last verified
Oct 2026
First submitted
8 Apr 2022
Last updated
27 Oct 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: 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/

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 81 sites in 15 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 81 sites in France, Germany, Spain, Italy, Belgium and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05331183.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.

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