Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation
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
2 other identifiers
interventional
307
15 countries
90
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started May 2022
Shorter than P25 for phase_3
90 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 1, 2022
CompletedFirst Posted
Study publicly available on registry
March 10, 2022
CompletedStudy Start
First participant enrolled
May 9, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 5, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
July 5, 2023
CompletedResults Posted
Study results publicly available
August 1, 2024
CompletedAugust 1, 2024
July 1, 2024
1.2 years
March 1, 2022
July 3, 2024
July 31, 2024
Conditions
Outcome Measures
Primary Outcomes (1)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Baseline Through Week 24
Secondary Outcomes (6)
Absolute Change in Sweat Chloride (SwCl)
From Baseline Through Week 24
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
From Baseline Through Week 24
Absolute Change in Body Mass Index (BMI)
From Baseline at Week 24
Absolute Change in Weight
From Baseline at Week 24
Number of Pulmonary Exacerbations (PEx)
From Baseline Through Week 24
- +1 more secondary outcomes
Study Arms (2)
Placebo
PLACEBO COMPARATORParticipants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks.
ELX/TEZ/IVA
EXPERIMENTALParticipants 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.
Interventions
Fixed-dose combination (FDC) tablets for oral administration.
Eligibility Criteria
You may qualify if:
- Forced expiratory volume in 1 second (FEV1) value \>=40% and \<=100% of predicted mean for age, sex, and height
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (90)
Medizinische Universität Innsbruck
Innsbruck, Austria
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
Ghent, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium
Stollery Children's Hospital
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, Canada
Klinika Detskych Infekcnich Nemoci
Brno, Czechia
Fakultni nemocnice v Motole
Prague, Czechia
CHU Lyon - Hopital Femme Mere-Enfant
Bron, 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, 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 Necker, Enfants Malades
Paris, France
Hopital Robert Debre
Paris, France
Hopital Haut-Leveque - CRCM Adulte
Pessac, 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, France
CHU de Toulouse - Hopital Larre
Toulouse, France
Hopital Bretonneau
Tours, France
Charite Paediatric Pulmonology Department
Berlin, Germany
Universitaetsklinkum Koeln, CF-Studienzentrum
Cologne, Germany
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
Erlangen, 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
Hanover, Germany
Medizinische Hochschule Hannover
Hanover, Germany
Universitaetsklinikum Jena, Mukoviszidose-Zentrum
Jena, Germany
Johannes Gutenberg-Universitaet
Mainz, Germany
Dr. von Haunersches Kinderspital
München, Germany
Klinikum Innenstadt, University of Munich
München, Germany
Pneumologisches Studienzentrum Muenchen-West
München, Germany
Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster
Münster, Germany
Klinikum Westbrandenburg (CF)
Potsdam, Germany
Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin
Tübingen, Germany
Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)
Ulm, Germany
Universitätsklinikum Würzburg
Würzburg, Germany
National Koranyi Institute for TBC and Pulmonology
Budapest, Hungary
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
Ancona, Italy
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
Florence, 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
Milan, 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, Italy
Academisch Medisch Centrum (Academic Medical Centre)
Amsterdam, Netherlands
UMC St. Radboud
Nijmegen, Netherlands
Erasmus Medical Center
Rotterdam, Netherlands
HagaZiekenhuis van den Haag
The Hague, Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
Oslo University Hospital, Department of Paediatric Medicine
Oslo, Norway
Pediatric Hospital Polanki named of Maciej Płażyński
Gdansk, Poland
Institute of Tuberculosis and Lung Diseases
Rabka-Zdrój, Poland
Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym
Łomianki, Poland
Hospital de Santa Maria
Lisbon, Portugal
Hospital Sao Joao
Porto, Portugal
Hospital de Cruces
Barakaldo, Spain
Hospital Saint Joan de Deu
Barcelona, Spain
Hospital Universitari Vall d Hebron
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
Seville, Spain
Hospital Universitario y Politecnico La Fe
Valencia, Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, Sweden
Karolinska Universitetssjukhuset, Huddinge
Stockholm, Sweden
Kinderspital Zuerich
Zurich, Switzerland
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 1, 2022
First Posted
March 10, 2022
Study Start
May 9, 2022
Primary Completion
July 5, 2023
Study Completion
July 5, 2023
Last Updated
August 1, 2024
Results First Posted
August 1, 2024
Record last verified: 2024-07
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing