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Cystic FibrosisPhase 3NCT05274269

Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation

A Phase 3 study testing ELX/TEZ/IVA and IVA in people with Cystic Fibrosis, run by Vertex Pharmaceuticals Incorporated at 90 sites in 15 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study evaluated the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) 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?
Yes, some participants may receive a placebo
How many people
307 participants
Where
90 sitesin 15 countries
Study dates
9 May 2022to 5 Jul 2023
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Participant has a qualifying ELX/TEZ/IVA-responsive CFTR mutation and does not have an exclusionary CFTR mutation
  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=100% of predicted mean for age, sex, and height

Who cannot join

  • History of solid organ or hematological transplantation
  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Other protocol defined Inclusion/Exclusion criteria may apply.

Trial locations and contacts

90 sites in 15 countries.

90 sites in 15 countries

Germany19 sites

  • Charite Paediatric Pulmonology Department

    Berlin

  • Universitaetsklinkum Koeln, CF-Studienzentrum

    Cologne

  • Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital

    Erlangen

  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

    Essen

  • Johann Wolfgang Goethe University

    Frankfurt

  • Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin

    Giessen

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

    Halle

  • Hannover Medical School

    Hanover

  • Medizinische Hochschule Hannover

    Hanover

  • Universitaetsklinikum Jena, Mukoviszidose-Zentrum

    Jena

  • Johannes Gutenberg-Universitaet

    Mainz

  • Dr. von Haunersches Kinderspital

    München

  • Klinikum Innenstadt, University of Munich

    München

  • Pneumologisches Studienzentrum Muenchen-West

    München

  • Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster

    Münster

  • Klinikum Westbrandenburg (CF)

    Potsdam

  • Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin

    Tübingen

  • Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)

    Ulm

  • Universitätsklinikum Würzburg

    Würzburg

Other trials in Germany

France17 sites

  • CHU Lyon - Hopital Femme Mere-Enfant

    Bron

  • Centre Hospitalier Intercommunal Creteil

    Créteil

  • Institut Cœur Poumon, CHU de Lille

    Lille

  • CHU Marseille - Hopital Nord

    Marseille

  • Hopital Arnaud de Villeneuve

    Montpellier

  • Centre Hospitalier Universitaire De Nantes - G. R. Laennec

    Nantes

  • Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur

    Nice

  • Hopital Cochin

    Paris

  • Hopital Necker, Enfants Malades

    Paris

  • Hopital Robert Debre

    Paris

  • Hopital Haut-Leveque - CRCM Adulte

    Pessac

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite

  • Centre Hospitalier Universitaire De Reims, Hopital Sebastopol

    Reims

  • CHU de Rennes - Hôpital Sud

    Rennes

  • Centre de Perharidy

    Roscoff

  • CHU de Toulouse - Hopital Larre

    Toulouse

  • Hopital Bretonneau

    Tours

Other trials in France

Spain13 sites

  • Hospital de Cruces

    Barakaldo

  • Hospital Saint Joan de Deu

    Barcelona

  • Hospital Universitari Vall d Hebron

    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

    Madrid

  • Hospital Universitario Infantil La Paz

    Madrid

  • Hospital Universitario Ramon y Cajal

    Madrid

  • Hospital Virgen de la Arrixaca

    Murcia

  • Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari

    Sabadell

  • Hospital Universitario Virgen del Rocio

    Seville

  • Hospital Universitario y Politecnico La Fe

    Valencia

Other trials in Spain

Italy11 sites

  • Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona

    Ancona

  • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

    Florence

  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

    Genova

  • Azienda Ospedaliera Universitaria Policlinico G. Martino

    Messina

  • Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

    Milan

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan

  • Azienda Ospedaliera Universitaria Federico II (Adults)

    Naples

  • Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica

    Orbassano

  • Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo

    Potenza

  • Ospedale Pediatrico Bambino Gesu

    Rome

  • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore

    Verona

Other trials in Italy

Belgium6 sites

  • Cliniques Universitaires de Bruxelles Hopital Erasme

    Brussels

  • Universitair Ziekenhuis Brussel - Campus Jette

    Brussels

  • Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital

    Edegem

  • Universitair Ziekenhuis Gent

    Ghent

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven

  • Cliniques Universitaires Saint-Luc

    Woluwe-Saint-Lambert

Other trials in Belgium

Canada6 sites

  • Stollery Children's Hospital

    Edmonton

  • Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu

    Montreal

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

    Montreal

  • The Hospital for Sick Children

    Toronto

  • British Columbia Children's Hospital

    Vancouver

  • St. Paul's Hospital

    Vancouver

Other trials in Canada

Netherlands5 sites

  • Academisch Medisch Centrum (Academic Medical Centre)

    Amsterdam

  • UMC St. Radboud

    Nijmegen

  • Erasmus Medical Center

    Rotterdam

  • HagaZiekenhuis van den Haag

    The Hague

  • Universitair Medisch Centrum Utrecht

    Utrecht

Other trials in Netherlands

Poland3 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

  • Institute of Tuberculosis and Lung Diseases

    Rabka-Zdrój

Other trials in Poland

Czechia2 sites

  • Klinika Detskych Infekcnich Nemoci

    Brno

  • Fakultni nemocnice v Motole

    Prague

Other trials in Czechia

Portugal2 sites

  • Hospital de Santa Maria

    Lisbon

  • Hospital Sao Joao

    Porto

Other trials in Portugal

Sweden2 sites

  • Sahlgrenska Universitetssjukhuset

    Gothenburg

  • Karolinska Universitetssjukhuset, Huddinge

    Stockholm

Other trials in Sweden

Austria1 site

  • Medizinische Universität Innsbruck

    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

Switzerland1 site

  • Kinderspital Zuerich

    Zurich

Other trials in Switzerland

About this study

Official title: A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation

This study evaluated the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.

Conditions
Cystic Fibrosis
Being tested
ELX/TEZ/IVA, IVA, Placebo (matched to ELX/TEZ/IVA), Placebo (matched to 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

    Tablet for oral administration.

  • Placebo (matched to ELX/TEZ/IVA)Other

    Placebo matched to ELX/TEZ/IVA for oral administration.

  • Placebo (matched to IVA)Other

    Placebo matched to IVA for oral administration.

Study groups in NCT05274269
Study groupRoleWhat they receive
PlaceboPlacebo comparatorParticipants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
Receives: Other: Placebo (matched to ELX/TEZ/IVA); Other: Placebo (matched to IVA)
ELX/TEZ/IVAExperimentalParticipants 6 to less than (<) 12 years of age and weighing <30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 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. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

    Measured: From Baseline Through Week 24

Secondary outcomes (6)

  1. Absolute Change in Sweat Chloride (SwCl)

    Sweat samples were collected using an approved collection device.

    Measured: From Baseline Through Week 24

  2. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

    Measured: From Baseline Through Week 24

  3. Absolute Change in Body Mass Index (BMI)

    BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).

    Measured: From Baseline at Week 24

  4. Absolute Change in Weight

    Measured: From Baseline at Week 24

  5. Number of Pulmonary Exacerbations (PEx)

    Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.

    Measured: From Baseline Through Week 24

  6. Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Measured: Day 1 up to Week 28

Timeline

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

  1. First posted10 Mar 2022
  2. Study start9 May 2022
  3. Primary completion5 Jul 2023
  4. Study completion5 Jul 2023

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
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
307 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05274269
Sponsor's protocol ID
VX21-445-124
Other IDs
2021-005320-38
Status last verified
Jul 2024
First submitted
1 Mar 2022
Last updated
1 Aug 2024
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/independent-research/clinical-trial-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 90 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 4 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: finished, as of the sponsor's last update on 1 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 90 sites in Germany, France, 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. Some participants in this study may receive a placebo. 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?

Yes. Results are posted in the registry (first posted 1 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) (time frame: From Baseline Through Week 24). 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 NCT05274269.

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 1 Aug 2024.

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