Clinical trial NCT05331183: Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2022-04-15
Enrollment: 297
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Day 1 up to Week 196
Secondary outcomes:
measure: Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)
timeFrame: From Baseline up to Week 96
measure: Part A: Absolute Change in Sweat Chloride (SwCl)
timeFrame: From Baseline up to Week 96
measure: Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
timeFrame: From Baseline up to Week 96
measure: Part A: Absolute Change in Body Mass Index (BMI)
timeFrame: From Baseline up to Week 96
measure: Part A: Absolute Change in Weight
timeFrame: From Baseline up to Week 96
measure: Part A: Number of Pulmonary Exacerbations (PEx)
timeFrame: From Baseline up to Week 96
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: University of Alberta Hospital, Edmonton Clinic
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: 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: 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: Munchen
country: Germany
facility: Dr. von Haunersches Kinderspital
city: Münster
country: Germany
facility: Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster
city: Potsdam
country: Germany
facility: Klinikum Westbrandenburg (CF)
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: UMCU, Locatie Wilhelmina Kinderziekenhuis
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: 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: 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 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: Zürich
country: Switzerland
facility: Kinderspital Zuerich
Study officials:
References: