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

NCT ID: NCT05331183

Last Updated: 2026-01-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

297 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-23

Study Completion Date

2027-04-06

Brief Summary

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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.

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ELX/TEZ/IVA

Part A: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for 96 weeks.

Part B: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for an additional 96 weeks.

Group Type EXPERIMENTAL

ELX/TEZ/IVA

Intervention Type DRUG

Fixed-dose combination (FDC) tablets for oral administration.

IVA

Intervention Type DRUG

Tablets for oral administration.

Interventions

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ELX/TEZ/IVA

Fixed-dose combination (FDC) tablets for oral administration.

Intervention Type DRUG

IVA

Tablets for oral administration.

Intervention Type DRUG

Other Intervention Names

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VX-445/VX-661/VX-770 elexacaftor/tezacaftor/ivacaftor VX-770 ivacaftor

Eligibility Criteria

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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

Exclusion Criteria

History of study drug intolerance in the parent study
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Medizinische Universität Innsbruck - Heilstättenschule Universitätsklinik

Innsbruck, , Austria

Site Status

CUB Hôpital Erasme

Brussels, , Belgium

Site Status

UZ Brussel - Campus Jette

Brussels, , Belgium

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Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital

Edegem, , Belgium

Site Status

Universitair Ziekenhuis Gent

Ghent, , Belgium

Site Status

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg

Leuven, , Belgium

Site Status

Universite Catholique de Louvain - Pulmonology

Woluwe-Saint-Lambert, , Belgium

Site Status

Stollery Children's Hospital

Edmonton, , Canada

Site Status

Le Centre de recherche du CHUM (CRCHUM)

Montreal, , Canada

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

Montreal, , Canada

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Hospital for Sick Children - Pulmonology

Toronto, , Canada

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British Columbia Children's Hospital

Vancouver, , Canada

Site Status

St. Paul's Hospital - Pulmonology

Vancouver, , Canada

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Klinika Detskych Infekcnich Nemoci

Brno, , Czechia

Site Status

Motol University Hospital

Prague, , Czechia

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Hopital Femme Mere-Enfant

Bron, , France

Site Status

Centre Hospitalier Intercommunal Creteil - Pulmonology

Créteil, , France

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Institut Cœur Poumon, CHU de Lille

Lille, , France

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Hopital Nord

Marseille, , France

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Hopital Arnaud de Villeneuve - Service de Pneumologie et Maladies Respiratoires

Montpellier, , France

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Hôpital Nord Laennec

Nantes, , France

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CHU de Nice - Hôpital Pasteur 2

Nice, , France

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Hopital Cochin - Pulmonology

Paris, , France

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Hopital Necker Enfants Malades - Pulmonology

Paris, , France

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Hopital Robert Debre - Pulmonology

Paris, , France

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Hôpital Lyon Sud - Pulmonology

Pierre-Bénite, , France

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Hôpital américain de Reims - Département de pédiatrie A

Reims, , France

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Hôpital Sud - Rennes

Rennes, , France

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Centre de Perharidy, Roscoff

Roscoff, , France

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Hopital Larrey

Toulouse, , France

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Hopital Bretonneau

Tours, , France

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Charité - Paediatric Pulmonology Department

Berlin, , Germany

Site Status

University Hospital Cologne (Koeln) - CF-Studienzentrum

Cologne, , Germany

Site Status

Ruhrlandklinik

Essen, , Germany

Site Status

Klinikum der Johann-Wolfgang-Goethe Universitaet - Pulmonology

Frankfurt, , Germany

Site Status

Justus-Liebig-Universität Gießen

Giessen, , Germany

Site Status

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

Halle, , Germany

Site Status

Medizinische Hochschule Hannover - Clinic for Pediatric Pneumology, Allergology and Neonatology, CF-Centre

Hanover, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Mukoviszidose-Zentrum am Universitätsklinikum Jena

Jena, , Germany

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Johannes Gutenberg-Universitaet

Mainz, , Germany

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Dr. von Haunersches Kinderspital - Pulmonology

München, , Germany

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Lungenheilkunde Munchen - Pasing

München, , Germany

Site Status

Klinik für Kinder- und Jugendmedizin

Münster, , Germany

Site Status

Klinikum Westbrandenburg (CF)

Potsdam, , Germany

Site Status

National Koranyi Institute for TBC and Pulmonology

Budapest, , Hungary

Site Status

Azienda Ospedaliero Universitaria delle Marche

Ancona, , Italy

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Azienda Ospedaliero Universitaria Ospedale Meyer

Florence, , Italy

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IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

Genova, , Italy

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Policlinico Universitario G. Martino

Messina, , Italy

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Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

Milan, , Italy

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Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

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Azienda Ospedaliero Universitaria Federico II Napoli

Naples, , Italy

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Azienda Ospedaliero Universitaria (AOU) San Luigi Gonzaga

Orbassano, , Italy

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Azienda Ospedaliera Regionale 'San Carlo', Potenza

Potenza, , Italy

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IRCSS Ospedale Pediatrico Bambino Gesu - Pulmonology

Rome, , Italy

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Azienda Ospedaliera di Verona - Ospedale Civile Maggiore

Verona, , Italy

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Amsterdam UMC - locatie AMC - Afdeling Longgeneeskunde

Amsterdam, , Netherlands

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Radboud University Medical Center - Pulmonology

Nijmegen, , Netherlands

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Erasmus Medisch Centrum - Pulmonology

Rotterdam, , Netherlands

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HagaZiekenhuis

The Hague, , Netherlands

Site Status

UMCU, Locatie Wilhelmina Kinderziekenhuis

Utrecht, , Netherlands

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Oslo University Hospital, Department of Paediatric Medicine

Oslo, , Norway

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Pediatric Hospital Polanki named of Maciej Płażyński

Gdansk, , Poland

Site Status

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

Łomianki, , Poland

Site Status

Unidade Local de Saúde de Santa Maria, E.P.E. - Pulmonology

Lisbon, , Portugal

Site Status

Hospital de Cruces

Barakaldo, , Spain

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Hospital Saint Joan de Deu

Barcelona, , Spain

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Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

Barcelona, , Spain

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Hospital Universitario de Jerez de la Frontera

Jerez de la Frontera, , Spain

Site Status

Hospital Infantil Universitario Nino Jesus

Madrid, , Spain

Site Status

Hospital Universitario 12 de Octubre - Pulmonology

Madrid, , Spain

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Hospital Universitario Ramon y Cajal - Pulmonology

Madrid, , Spain

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La Paz Infantil

Madrid, , Spain

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Hospital Virgen de la Arrixaca

Murcia, , Spain

Site Status

Institut d´Investigacio i Innovacio Parc Tauli

Sabadell, , Spain

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Hospital Universitario Virgen del Rocio

Seville, , Spain

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Hospital Universitario y Politecnico La Fe - Pulmonology

Valencia, , Spain

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Sahlgrenska Universitetssjukhuset - Göteborg CF-center

Gothenburg, , Sweden

Site Status

Karolinska University Hospital - Pulmonology

Stockholm, , Sweden

Site Status

Kinderspital Zuerich

Zurich, , Switzerland

Site Status

Countries

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

Other Identifiers

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2021-005914-33

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2024-515637-14-00

Identifier Type: OTHER

Identifier Source: secondary_id

VX21-445-125

Identifier Type: -

Identifier Source: org_study_id

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