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

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05331183
• Other study ID #: VX21-445-125
• Secondary ID: 2021-005914-33
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: November 23, 2022
• Est. completion date: April 2027

Study information

• Verified date: May 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 297
• Est. completion date: April 2027
• Est. primary completion date: April 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination (FDC) tablets for oral administration.
• IVA
Tablets for oral administration.

Locations

Country	Name	City	State
Austria	Medizinische Universität Innsbruck	Innsbruck
Belgium	Cliniques Universitaires de Bruxelles Hopital Erasme	Brussels
Belgium	Universitair Ziekenhuis Brussel - Campus Jette	Brussels
Belgium	Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital	Edegem
Belgium	Universitair Ziekenhuis Gent	Gent
Belgium	Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg	Leuven
Belgium	Cliniques Universitaires Saint-Luc	Woluwe-Saint-Lambert
Canada	University of Alberta Hospital, Edmonton Clinic	Edmonton
Canada	Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu	Montreal
Canada	McGill University Health Centre, Glen Site, Montreal Children's Hospital	Montreal
Canada	The Hospital for Sick Children	Toronto
Canada	British Columbia Children's Hospital	Vancouver
Canada	St. Paul's Hospital	Vancouver
Czechia	Klinika Detskych Infekcnich Nemoci	Brno
Czechia	Fakultni nemocnice v Motole	Praha 5
France	CHU Lyon - Hopital Femme Mere-Enfant	Bron Cedex
France	Centre Hospitalier Intercommunal Creteil	Créteil
France	Institut Cœur Poumon, CHU de Lille	Lille
France	CHU Marseille - Hopital Nord	Marseille
France	Hopital Arnaud de Villeneuve	Montpellier Cedex 5
France	Centre Hospitalier Universitaire De Nantes - G. R. Laennec	Nantes
France	Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur	Nice
France	Hopital Cochin	Paris
France	Hopital Robert Debre	Paris
France	Hopital Necker, Enfants Malades	Paris Cedex 15
France	Centre Hospitalier Lyon Sud	Pierre-Bénite
France	Centre Hospitalier Universitaire De Reims, Hopital Sebastopol	Reims
France	CHU de Rennes - Hôpital Sud	Rennes
France	Centre de Perharidy	Roscoff cedex
France	CHU de Toulouse - Hopital Larre	Toulouse
France	Hopital Bretonneau	Tours
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen	Essen
Germany	Johann Wolfgang Goethe University	Frankfurt
Germany	Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin	Gießen
Germany	Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie	Halle
Germany	Hannover Medical School	Hannover
Germany	Medizinische Hochschule Hannover	Hannover
Germany	Universitaetsklinikum Jena, Mukoviszidose-Zentrum	Jena
Germany	Universitaetsklinkum Koeln, CF-Studienzentrum	Koeln
Germany	Johannes Gutenberg-Universitaet	Mainz
Germany	Pneumologisches Studienzentrum Muenchen-West	Muenchen
Germany	Dr. von Haunersches Kinderspital	Munchen
Germany	Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster	Münster
Germany	Klinikum Westbrandenburg (CF)	Potsdam
Hungary	National Koranyi Institute for TBC and Pulmonology	Budapest
Italy	Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona	Ancona
Italy	Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer	Firenze
Italy	IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico	Genova
Italy	Azienda Ospedaliera Universitaria Policlinico G. Martino	Messina
Italy	Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena	Milan
Italy	Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	Milano
Italy	Azienda Ospedaliera Universitaria Federico II (Adults)	Naples
Italy	Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica	Orbassano
Italy	Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo	Potenza
Italy	Ospedale Pediatrico Bambino Gesu	Rome
Italy	Azienda Ospedaliera di Verona - Ospedale Civile Maggiore	Verona
Netherlands	Academisch Medisch Centrum (Academic Medical Centre)	Amsterdam
Netherlands	HagaZiekenhuis van den Haag	Den Haag
Netherlands	UMC St. Radboud	Nijmegen
Netherlands	Erasmus Medical Center	Rotterdam
Netherlands	UMCU, Locatie Wilhelmina Kinderziekenhuis	Utrecht
Norway	Oslo University Hospital, Department of Paediatric Medicine	Oslo
Poland	Pediatric Hospital Polanki named of Maciej Plazynski	Gdansk
Poland	Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddzial Chorób Pluc SZPZOZ im.Dzieci Warszawy w Dziekanowie Lesnym	Lomianki
Portugal	Hospital de Santa Maria	Lisbon
Spain	Hospital de Cruces	Barakaldo
Spain	Hospital Saint Joan de Deu	Barcelona
Spain	Hospital Universitari Vall d´Hebron Servicio de Broncoscopia	Barcelona
Spain	Hospital Universitario de Jerez de la Frontera	Jerez de la Frontera
Spain	Hospital Infantil Universitario Nino Jesus	Madrid
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital Universitario Infantil La Paz	Madrid
Spain	Hospital Universitario Ramon y Cajal	Madrid
Spain	Hospital Virgen de la Arrixaca	Murcia
Spain	Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari	Sabadell
Spain	Hospital Universitario Virgen del Rocio	Sevilla
Spain	Hospital Universitario y Politecnico La Fe	Valencia
Sweden	Sahlgrenska Universitetssjukhuset	Göteborg
Sweden	Karolinska Universitetssjukhuset, Huddinge	Stockholm
Switzerland	Kinderspital Zuerich	Zürich

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Austria,  Belgium,  Canada,  Czechia,  France,  Germany,  Hungary,  Italy,  Netherlands,  Norway,  Poland,  Portugal,  Spain,  Sweden,  Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		Day 1 up to Week 196
Secondary	Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)		From Baseline up to Week 96
Secondary	Part A: Absolute Change in Sweat Chloride (SwCl)		From Baseline up to Week 96
Secondary	Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score		From Baseline up to Week 96
Secondary	Part A: Absolute Change in Body Mass Index (BMI)		From Baseline up to Week 96
Secondary	Part A: Absolute Change in Weight		From Baseline up to Week 96
Secondary	Part A: Number of Pulmonary Exacerbations (PEx)		From Baseline up to Week 96