Clinical trial NCT05274269: Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2022-03-10
Enrollment: 307
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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: Key Inclusion Criteria:

* 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

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 pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
timeFrame: From Baseline Through Week 24
Secondary outcomes:
measure: Absolute Change in Sweat Chloride (SwCl)
timeFrame: From Baseline Through Week 24
measure: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
timeFrame: From Baseline Through Week 24
measure: Absolute Change in Body Mass Index (BMI)
timeFrame: From Baseline at Week 24
measure: Absolute Change in Weight
timeFrame: From Baseline at Week 24
measure: Number of Pulmonary Exacerbations (PEx)
timeFrame: From Baseline Through Week 24
measure: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Day 1 up to Week 28
Other outcomes:
Locations:
city: Innsbruck
country: Austria
facility: Medizinische Universität Innsbruck
city: Brussels
country: Belgium
facility: Cliniques Universitaires de Bruxelles Hopital Erasme
city: Brussels
country: Belgium
facility: Universitair Ziekenhuis Brussel - Campus Jette
city: Edegem
country: Belgium
facility: Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent
city: Leuven
country: Belgium
facility: Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
city: Woluwe-Saint-Lambert
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Edmonton
country: Canada
facility: Stollery Children's Hospital
city: Montreal
country: Canada
facility: Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
city: Montreal
country: Canada
facility: McGill University Health Centre, Glen Site, Montreal Children's Hospital
city: Toronto
country: Canada
facility: The Hospital for Sick Children
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
city: Vancouver
country: Canada
facility: St. Paul's Hospital
city: Brno
country: Czechia
facility: Klinika Detskych Infekcnich Nemoci
city: Praha 5
country: Czechia
facility: Fakultni nemocnice v Motole
city: Bron Cedex
country: France
facility: CHU Lyon - Hopital Femme Mere-Enfant
city: Créteil
country: France
facility: Centre Hospitalier Intercommunal Creteil
city: Lille
country: France
facility: Institut Cœur Poumon, CHU de Lille
city: Marseille
country: France
facility: CHU Marseille - Hopital Nord
city: Montpellier Cedex 5
country: France
facility: Hopital Arnaud de Villeneuve
city: Nantes
country: France
facility: Centre Hospitalier Universitaire De Nantes - G. R. Laennec
city: Nice
country: France
facility: Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
city: Paris Cedex 15
country: France
facility: Hopital Necker, Enfants Malades
city: Paris
country: France
facility: Hopital Cochin
city: Paris
country: France
facility: Hopital Robert Debre
city: Pessac
country: France
facility: Hopital Haut-Leveque - CRCM Adulte
city: Pierre-Bénite
country: France
facility: Centre Hospitalier Lyon Sud
city: Reims
country: France
facility: Centre Hospitalier Universitaire De Reims, Hopital Sebastopol
city: Rennes
country: France
facility: CHU de Rennes - Hôpital Sud
city: Roscoff Cedex
country: France
facility: Centre de Perharidy
city: Toulouse
country: France
facility: CHU de Toulouse - Hopital Larre
city: Tours
country: France
facility: Hopital Bretonneau
city: Berlin
country: Germany
facility: Charite Paediatric Pulmonology Department
city: Erlangen
country: Germany
facility: Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
city: Essen
country: Germany
facility: Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
city: Frankfurt
country: Germany
facility: Johann Wolfgang Goethe University
city: Gießen
country: Germany
facility: Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
city: Halle
country: Germany
facility: Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
city: Hannover
country: Germany
facility: Hannover Medical School
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Jena
country: Germany
facility: Universitaetsklinikum Jena, Mukoviszidose-Zentrum
city: Koeln
country: Germany
facility: Universitaetsklinkum Koeln, CF-Studienzentrum
city: Mainz
country: Germany
facility: Johannes Gutenberg-Universitaet
city: Muenchen
country: Germany
facility: Pneumologisches Studienzentrum Muenchen-West
city: München
country: Germany
facility: Dr. von Haunersches Kinderspital
city: München
country: Germany
facility: Klinikum Innenstadt, University of Munich
city: Münster
country: Germany
facility: Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster
city: Potsdam
country: Germany
facility: Klinikum Westbrandenburg (CF)
city: Tuebingen
country: Germany
facility: Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin
city: Ulm
country: Germany
facility: Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)
city: Würzburg
country: Germany
facility: Universitätsklinikum Würzburg
city: Budapest
country: Hungary
facility: National Koranyi Institute for TBC and Pulmonology
city: Ancona
country: Italy
facility: Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
city: Genova
country: Italy
facility: IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
city: Messina
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico G. Martino
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
city: Milan
country: Italy
facility: Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II (Adults)
city: Orbassano
country: Italy
facility: Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
city: Potenza
country: Italy
facility: Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesu
city: Verona
country: Italy
facility: Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
city: Amsterdam
country: Netherlands
facility: Academisch Medisch Centrum (Academic Medical Centre)
city: Den Haag
country: Netherlands
facility: HagaZiekenhuis van den Haag
city: Nijmegen
country: Netherlands
facility: UMC St. Radboud
city: Rotterdam
country: Netherlands
facility: Erasmus Medical Center
city: Utrecht
country: Netherlands
facility: Universitair Medisch Centrum Utrecht
city: Oslo
country: Norway
facility: Oslo University Hospital, Department of Paediatric Medicine
city: Gdańsk
country: Poland
facility: Pediatric Hospital Polanki named of Maciej Płażyński
city: Rabka-Zdrój
country: Poland
facility: Institute of Tuberculosis and Lung Diseases
city: Łomianki
country: Poland
facility: Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym
city: Lisbon
country: Portugal
facility: Hospital de Santa Maria
city: Porto
country: Portugal
facility: Hospital Sao Joao
city: Barakaldo
country: Spain
facility: Hospital de Cruces
city: Barcelona
country: Spain
facility: Hospital Saint Joan de Deu
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d Hebron
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
city: Jerez De La Frontera
country: Spain
facility: Hospital Universitario de Jerez de la Frontera
city: Madrid
country: Spain
facility: Hospital Infantil Universitario Nino Jesus
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Madrid
country: Spain
facility: Hospital Universitario Infantil La Paz
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Murcia
country: Spain
facility: Hospital Virgen de la Arrixaca
city: Sabadell
country: Spain
facility: Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Valencia
country: Spain
facility: Hospital Universitario y Politecnico La Fe
city: Göteborg
country: Sweden
facility: Sahlgrenska Universitetssjukhuset
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset, Huddinge
city: Zurich
country: Switzerland
facility: Kinderspital Zuerich
Study officials:
References: