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

NCT ID: NCT05274269

Last Updated: 2024-08-01

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

307 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-05-09

Study Completion Date

2023-07-05

Brief Summary

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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.

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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo

Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.

Group Type PLACEBO_COMPARATOR

Placebo (matched to ELX/TEZ/IVA)

Intervention Type OTHER

Placebo matched to ELX/TEZ/IVA for oral administration.

Placebo (matched to IVA)

Intervention Type OTHER

Placebo matched to IVA for oral administration.

ELX/TEZ/IVA

Participants 6 to less than (\<) 12 years of age and weighing \<30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks.

Group Type EXPERIMENTAL

ELX/TEZ/IVA

Intervention Type DRUG

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

IVA

Intervention Type DRUG

Tablet for oral administration.

Interventions

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ELX/TEZ/IVA

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

Intervention Type DRUG

IVA

Tablet for oral administration.

Intervention Type DRUG

Placebo (matched to ELX/TEZ/IVA)

Placebo matched to ELX/TEZ/IVA for oral administration.

Intervention Type OTHER

Placebo (matched to IVA)

Placebo matched to IVA for oral administration.

Intervention Type OTHER

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

* 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

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
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

Innsbruck, , Austria

Site Status

Cliniques Universitaires de Bruxelles Hopital Erasme

Brussels, , Belgium

Site Status

Universitair Ziekenhuis Brussel - Campus Jette

Brussels, , Belgium

Site Status

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

Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, , Belgium

Site Status

Stollery Children's Hospital

Edmonton, , Canada

Site Status

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

Montreal, , Canada

Site Status

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

Montreal, , Canada

Site Status

The Hospital for Sick Children

Toronto, , Canada

Site Status

British Columbia Children's Hospital

Vancouver, , Canada

Site Status

St. Paul's Hospital

Vancouver, , Canada

Site Status

Klinika Detskych Infekcnich Nemoci

Brno, , Czechia

Site Status

Fakultni nemocnice v Motole

Prague, , Czechia

Site Status

CHU Lyon - Hopital Femme Mere-Enfant

Bron, , France

Site Status

Centre Hospitalier Intercommunal Creteil

Créteil, , France

Site Status

Institut Cœur Poumon, CHU de Lille

Lille, , France

Site Status

CHU Marseille - Hopital Nord

Marseille, , France

Site Status

Hopital Arnaud de Villeneuve

Montpellier, , France

Site Status

Centre Hospitalier Universitaire De Nantes - G. R. Laennec

Nantes, , France

Site Status

Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur

Nice, , France

Site Status

Hopital Cochin

Paris, , France

Site Status

Hopital Necker, Enfants Malades

Paris, , France

Site Status

Hopital Robert Debre

Paris, , France

Site Status

Hopital Haut-Leveque - CRCM Adulte

Pessac, , France

Site Status

Centre Hospitalier Lyon Sud

Pierre-Bénite, , France

Site Status

Centre Hospitalier Universitaire De Reims, Hopital Sebastopol

Reims, , France

Site Status

CHU de Rennes - Hôpital Sud

Rennes, , France

Site Status

Centre de Perharidy

Roscoff, , France

Site Status

CHU de Toulouse - Hopital Larre

Toulouse, , France

Site Status

Hopital Bretonneau

Tours, , France

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

Universitaetsklinkum Koeln, CF-Studienzentrum

Cologne, , Germany

Site Status

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

Erlangen, , Germany

Site Status

Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

Essen, , Germany

Site Status

Johann Wolfgang Goethe University

Frankfurt, , Germany

Site Status

Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin

Giessen, , Germany

Site Status

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

Halle, , Germany

Site Status

Hannover Medical School

Hanover, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Universitaetsklinikum Jena, Mukoviszidose-Zentrum

Jena, , Germany

Site Status

Johannes Gutenberg-Universitaet

Mainz, , Germany

Site Status

Dr. von Haunersches Kinderspital

München, , Germany

Site Status

Klinikum Innenstadt, University of Munich

München, , Germany

Site Status

Pneumologisches Studienzentrum Muenchen-West

München, , Germany

Site Status

Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster

Münster, , Germany

Site Status

Klinikum Westbrandenburg (CF)

Potsdam, , Germany

Site Status

Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin

Tübingen, , Germany

Site Status

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

Ulm, , Germany

Site Status

Universitätsklinikum Würzburg

Würzburg, , Germany

Site Status

National Koranyi Institute for TBC and Pulmonology

Budapest, , Hungary

Site Status

Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona

Ancona, , Italy

Site Status

Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

Florence, , Italy

Site Status

IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

Genova, , Italy

Site Status

Azienda Ospedaliera Universitaria Policlinico G. Martino

Messina, , Italy

Site Status

Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena

Milan, , Italy

Site Status

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

Azienda Ospedaliera Universitaria Federico II (Adults)

Naples, , Italy

Site Status

Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica

Orbassano, , Italy

Site Status

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

Potenza, , Italy

Site Status

Ospedale Pediatrico Bambino Gesu

Rome, , Italy

Site Status

Azienda Ospedaliera di Verona - Ospedale Civile Maggiore

Verona, , Italy

Site Status

Academisch Medisch Centrum (Academic Medical Centre)

Amsterdam, , Netherlands

Site Status

UMC St. Radboud

Nijmegen, , Netherlands

Site Status

Erasmus Medical Center

Rotterdam, , Netherlands

Site Status

HagaZiekenhuis van den Haag

The Hague, , Netherlands

Site Status

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Site Status

Oslo University Hospital, Department of Paediatric Medicine

Oslo, , Norway

Site Status

Pediatric Hospital Polanki named of Maciej Płażyński

Gdansk, , Poland

Site Status

Institute of Tuberculosis and Lung Diseases

Rabka-Zdrój, , Poland

Site Status

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

Łomianki, , Poland

Site Status

Hospital de Santa Maria

Lisbon, , Portugal

Site Status

Hospital Sao Joao

Porto, , Portugal

Site Status

Hospital de Cruces

Barakaldo, , Spain

Site Status

Hospital Saint Joan de Deu

Barcelona, , Spain

Site Status

Hospital Universitari Vall d Hebron

Barcelona, , Spain

Site Status

Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

Barcelona, , Spain

Site Status

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

Madrid, , Spain

Site Status

Hospital Universitario Infantil La Paz

Madrid, , Spain

Site Status

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Site Status

Hospital Virgen de la Arrixaca

Murcia, , Spain

Site Status

Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari

Sabadell, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Hospital Universitario y Politecnico La Fe

Valencia, , Spain

Site Status

Sahlgrenska Universitetssjukhuset

Gothenburg, , Sweden

Site Status

Karolinska Universitetssjukhuset, Huddinge

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

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2021-005320-38

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX21-445-124

Identifier Type: -

Identifier Source: org_study_id

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