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

Last updated: July 31, 2024
Sponsor: Vertex Pharmaceuticals Incorporated
Overall Status: Completed

Phase

3

Condition

Cystic Fibrosis

Scar Tissue

Lung Disease

Treatment

Placebo (matched to IVA)

Placebo (matched to ELX/TEZ/IVA)

ELX/TEZ/IVA

Clinical Study ID

NCT05274269
VX21-445-124
2021-005320-38
  • Ages > 6
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  • Participant has a qualifying ELX/TEZ/IVA-responsive CFTR mutation and does not havean exclusionary CFTR mutation

  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=100% of predicted meanfor age, sex, and height

Exclusion

Key Exclusion Criteria:

  • 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 pulmonarystatus

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Total Participants: 307
Treatment Group(s): 4
Primary Treatment: Placebo (matched to IVA)
Phase: 3
Study Start date:
May 09, 2022
Estimated Completion Date:
July 05, 2023

Connect with a study center

  • Medizinische Universität Innsbruck

    Innsbruck,
    Austria

    Site Not Available

  • Cliniques Universitaires de Bruxelles Hopital Erasme

    Brussels,
    Belgium

    Site Not Available

  • Universitair Ziekenhuis Brussel - Campus Jette

    Brussels,
    Belgium

    Site Not Available

  • Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital

    Edegem,
    Belgium

    Site Not Available

  • Universitair Ziekenhuis Gent

    Gent,
    Belgium

    Site Not Available

  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

    Leuven,
    Belgium

    Site Not Available

  • Cliniques Universitaires Saint-Luc

    Woluwe-Saint-Lambert,
    Belgium

    Site Not Available

  • Stollery Children's Hospital

    Edmonton,
    Canada

    Site Not Available

  • University of Alberta Hospital, Edmonton Clinic

    Edmonton,
    Canada

    Site Not Available

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

    Montreal,
    Canada

    Site Not Available

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

    Montreal,
    Canada

    Site Not Available

  • The Hospital for Sick Children

    Toronto,
    Canada

    Site Not Available

  • British Columbia Children's Hospital

    Vancouver,
    Canada

    Site Not Available

  • St. Paul's Hospital

    Vancouver,
    Canada

    Site Not Available

  • Klinika Detskych Infekcnich Nemoci

    Brno,
    Czechia

    Site Not Available

  • Fakultni nemocnice v Motole

    Praha 5,
    Czechia

    Site Not Available

  • CHU Lyon - Hopital Femme Mere-Enfant

    Bron Cedex,
    France

    Site Not Available

  • Centre Hospitalier Intercommunal Creteil

    Créteil,
    France

    Site Not Available

  • Institut Cœur Poumon, CHU de Lille

    Lille,
    France

    Site Not Available

  • CHU Marseille - Hopital Nord

    Marseille,
    France

    Site Not Available

  • CHU Marseille - Hopital Nord

    Marseilles,
    France

    Site Not Available

  • Hopital Arnaud de Villeneuve

    Montpellier Cedex 5,
    France

    Site Not Available

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

    Nantes,
    France

    Site Not Available

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

    Nice,
    France

    Site Not Available

  • Hopital Cochin

    Paris,
    France

    Site Not Available

  • Hopital Robert Debre

    Paris,
    France

    Site Not Available

  • Hopital Necker, Enfants Malades

    Paris Cedex 15,
    France

    Site Not Available

  • Hopital Haut-Leveque - CRCM Adulte

    Pessac,
    France

    Site Not Available

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite,
    France

    Site Not Available

  • Centre Hospitalier Universitaire De Reims, Hopital Sebastopol

    Reims,
    France

    Site Not Available

  • CHU de Rennes - Hôpital Sud

    Rennes,
    France

    Site Not Available

  • Centre de Perharidy

    Roscoff Cedex,
    France

    Site Not Available

  • CHU de Toulouse - Hopital Larre

    Toulouse,
    France

    Site Not Available

  • Hopital Bretonneau

    Tours,
    France

    Site Not Available

  • Charite Paediatric Pulmonology Department

    Berlin,
    Germany

    Site Not Available

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

    Erlangen,
    Germany

    Site Not Available

  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

    Essen,
    Germany

    Site Not Available

  • Johann Wolfgang Goethe University

    Frankfurt,
    Germany

    Site Not Available

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

    Gießen,
    Germany

    Site Not Available

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

    Halle,
    Germany

    Site Not Available

  • Hannover Medical School

    Hannover,
    Germany

    Site Not Available

  • Medizinische Hochschule Hannover

    Hannover,
    Germany

    Site Not Available

  • Universitaetsklinikum Jena, Mukoviszidose-Zentrum

    Jena,
    Germany

    Site Not Available

  • Universitaetsklinkum Koeln, CF-Studienzentrum

    Koeln,
    Germany

    Site Not Available

  • Johannes Gutenberg-Universitaet

    Mainz,
    Germany

    Site Not Available

  • Pneumologisches Studienzentrum Muenchen-West

    Muenchen,
    Germany

    Site Not Available

  • Dr. von Haunersches Kinderspital

    München,
    Germany

    Site Not Available

  • Klinikum Innenstadt, University of Munich

    München,
    Germany

    Site Not Available

  • Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster

    Münster,
    Germany

    Site Not Available

  • Klinikum Westbrandenburg (CF)

    Potsdam,
    Germany

    Site Not Available

  • Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin

    Tuebingen,
    Germany

    Site Not Available

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

    Ulm,
    Germany

    Site Not Available

  • Universitätsklinikum Würzburg

    Würzburg,
    Germany

    Site Not Available

  • National Koranyi Institute for TBC and Pulmonology

    Budapest,
    Hungary

    Site Not Available

  • Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona

    Ancona,
    Italy

    Site Not Available

  • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

    Firenze,
    Italy

    Site Not Available

  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

    Genova,
    Italy

    Site Not Available

  • Azienda Ospedaliera Universitaria Policlinico G. Martino

    Messina,
    Italy

    Site Not Available

  • Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

    Milan,
    Italy

    Site Not Available

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milano,
    Italy

    Site Not Available

  • Azienda Ospedaliera Universitaria Federico II (Adults)

    Naples,
    Italy

    Site Not Available

  • Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica

    Orbassano,
    Italy

    Site Not Available

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

    Potenza,
    Italy

    Site Not Available

  • Ospedale Pediatrico Bambino Gesu

    Rome,
    Italy

    Site Not Available

  • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore

    Verona,
    Italy

    Site Not Available

  • Academisch Medisch Centrum (Academic Medical Centre)

    Amsterdam,
    Netherlands

    Site Not Available

  • HagaZiekenhuis van den Haag

    Den Haag,
    Netherlands

    Site Not Available

  • UMC St. Radboud

    Nijmegen,
    Netherlands

    Site Not Available

  • Erasmus Medical Center

    Rotterdam,
    Netherlands

    Site Not Available

  • Universitair Medisch Centrum Utrecht

    Utrecht,
    Netherlands

    Site Not Available

  • Oslo University Hospital, Department of Paediatric Medicine

    Oslo,
    Norway

    Site Not Available

  • Pediatric Hospital Polanki named of Maciej Płażyński

    Gdańsk,
    Poland

    Site Not Available

  • Institute of Tuberculosis and Lung Diseases

    Rabka-Zdrój,
    Poland

    Site Not Available

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

    Łomianki,
    Poland

    Site Not Available

  • Hospital de Santa Maria

    Lisbon,
    Portugal

    Site Not Available

  • Hospital Sao Joao

    Porto,
    Portugal

    Site Not Available

  • Hospital de Cruces

    Barakaldo,
    Spain

    Site Not Available

  • Hospital Saint Joan de Deu

    Barcelona,
    Spain

    Site Not Available

  • Hospital Universitari Vall d Hebron

    Barcelona,
    Spain

    Site Not Available

  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

    Barcelona,
    Spain

    Site Not Available

  • Hospital Universitario de Jerez de la Frontera

    Jerez De La Frontera,
    Spain

    Site Not Available

  • Hospital Infantil Universitario Nino Jesus

    Madrid,
    Spain

    Site Not Available

  • Hospital Universitario 12 de Octubre

    Madrid,
    Spain

    Site Not Available

  • Hospital Universitario Infantil La Paz

    Madrid,
    Spain

    Site Not Available

  • Hospital Universitario Ramon y Cajal

    Madrid,
    Spain

    Site Not Available

  • Hospital Virgen de la Arrixaca

    Murcia,
    Spain

    Site Not Available

  • Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari

    Sabadell,
    Spain

    Site Not Available

  • Hospital Universitario Virgen del Rocio

    Sevilla,
    Spain

    Site Not Available

  • Hospital Universitario y Politecnico La Fe

    Valencia,
    Spain

    Site Not Available

  • Sahlgrenska Universitetssjukhuset

    Göteborg,
    Sweden

    Site Not Available

  • Karolinska Universitetssjukhuset, Huddinge

    Stockholm,
    Sweden

    Site Not Available

  • Kinderspital Zuerich

    Zurich,
    Switzerland

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.