Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation
- Conditions
- Cystic Fibrosis
- Interventions
- Registration Number
- NCT05274269
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 307
- 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
Key
- 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
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo (matched to ELX/TEZ/IVA) Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks. Placebo Placebo (matched to IVA) Participants 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 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. ELX/TEZ/IVA 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.
- Primary Outcome Measures
Name Time Method Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline Through Week 24 FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
- Secondary Outcome Measures
Name Time Method Absolute Change in Sweat Chloride (SwCl) From Baseline Through Week 24 Sweat samples were collected using an approved collection device.
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score From Baseline Through Week 24 The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Absolute Change in Body Mass Index (BMI) From Baseline at Week 24 BMI was defined as weight in kilogram (kg) divided by height in square meter (m\^2).
Absolute Change in Weight From Baseline at Week 24 Number of Pulmonary Exacerbations (PEx) From Baseline Through Week 24 Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Day 1 up to Week 28
Trial Locations
- Locations (90)
McGill University Health Centre, Glen Site, Montreal Children's Hospital
🇨🇦Montreal, Canada
Fakultni nemocnice v Motole
🇨🇿Praha 5, Czechia
Hopital Cochin
🇫🇷Paris, France
Centre Hospitalier Lyon Sud
🇫🇷Pierre-Bénite, France
Hopital Arnaud de Villeneuve
🇫🇷Montpellier Cedex 5, France
Oslo University Hospital, Department of Paediatric Medicine
🇳🇴Oslo, Norway
Cliniques Universitaires de Bruxelles Hopital Erasme
🇧🇪Brussels, Belgium
Universitair Ziekenhuis Brussel - Campus Jette
🇧🇪Brussels, Belgium
HagaZiekenhuis van den Haag
🇳🇱Den Haag, Netherlands
Institute of Tuberculosis and Lung Diseases
🇵🇱Rabka-Zdrój, Poland
Universitair Ziekenhuis Gent
🇧🇪Gent, Belgium
Academisch Medisch Centrum (Academic Medical Centre)
🇳🇱Amsterdam, Netherlands
Hospital Sao Joao
🇵🇹Porto, Portugal
Kinderspital Zuerich
🇨🇭Zurich, Switzerland
Cliniques Universitaires Saint-Luc
🇧🇪Woluwe-Saint-Lambert, Belgium
Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital
🇧🇪Edegem, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
🇮🇹Firenze, Italy
Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
🇮🇹Orbassano, Italy
Hopital Necker, Enfants Malades
🇫🇷Paris Cedex 15, France
Sahlgrenska Universitetssjukhuset
🇸🇪Göteborg, Sweden
Pneumologisches Studienzentrum Muenchen-West
🇩🇪Muenchen, Germany
Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin
🇩🇪Tuebingen, Germany
Klinikum Innenstadt, University of Munich
🇩🇪München, Germany
Stollery Children's Hospital
🇨🇦Edmonton, Canada
The Hospital for Sick Children
🇨🇦Toronto, Canada
St. Paul's Hospital
🇨🇦Vancouver, Canada
Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
🇨🇦Montreal, Canada
British Columbia Children's Hospital
🇨🇦Vancouver, Canada
Klinika Detskych Infekcnich Nemoci
🇨🇿Brno, Czechia
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
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
🇫🇷Nantes, France
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
🇫🇷Nice, France
Hopital Haut-Leveque - CRCM Adulte
🇫🇷Pessac, France
Centre Hospitalier Universitaire De Reims, Hopital Sebastopol
🇫🇷Reims, France
Hopital Robert Debre
🇫🇷Paris, France
CHU de Rennes - Hôpital Sud
🇫🇷Rennes, France
Centre de Perharidy
🇫🇷Roscoff Cedex, France
CHU de Toulouse - Hopital Larre
🇫🇷Toulouse, France
Hopital Bretonneau
🇫🇷Tours, France
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
🇩🇪Essen, Germany
Johann Wolfgang Goethe University
🇩🇪Frankfurt, Germany
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
🇩🇪Halle, Germany
Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
🇩🇪Gießen, 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
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
🇩🇪Erlangen, Germany
Dr. von Haunersches Kinderspital
🇩🇪München, Germany
Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster
🇩🇪Münster, Germany
Klinikum Westbrandenburg (CF)
🇩🇪Potsdam, Germany
Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF)
🇩🇪Ulm, Germany
Universitätsklinikum Würzburg
🇩🇪Würzburg, Germany
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
🇮🇹Ancona, Italy
National Koranyi Institute for TBC and Pulmonology
🇭🇺Budapest, Hungary
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
🇮🇹Genova, Italy
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
🇮🇹Milan, Italy
Azienda Ospedaliera Universitaria Policlinico G. Martino
🇮🇹Messina, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Italy
Ospedale Pediatrico Bambino Gesu
🇮🇹Rome, Italy
Azienda Ospedaliera Universitaria Federico II (Adults)
🇮🇹Naples, Italy
Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
🇮🇹Potenza, Italy
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
🇮🇹Verona, Italy
UMC St. Radboud
🇳🇱Nijmegen, Netherlands
Erasmus Medical Center
🇳🇱Rotterdam, Netherlands
Universitair Medisch Centrum Utrecht
🇳🇱Utrecht, Netherlands
Pediatric Hospital Polanki named of Maciej Płażyński
🇵🇱Gdańsk, Poland
Hospital de Santa Maria
🇵🇹Lisbon, Portugal
Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym
🇵🇱Łomianki, Poland
Hospital Saint Joan de Deu
🇪🇸Barcelona, Spain
Hospital de Cruces
🇪🇸Barakaldo, Spain
Hospital Universitari Vall d Hebron
🇪🇸Barcelona, Spain
Hospital Universitario de Jerez de la Frontera
🇪🇸Jerez De La Frontera, Spain
Hospital Infantil Universitario Nino Jesus
🇪🇸Madrid, Spain
Hospital Universitario Ramon y Cajal
🇪🇸Madrid, Spain
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
🇪🇸Barcelona, Spain
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital Universitario Infantil La Paz
🇪🇸Madrid, Spain
Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
🇪🇸Sabadell, Spain
Hospital Universitario Virgen del Rocio
🇪🇸Sevilla, Spain
Hospital Virgen de la Arrixaca
🇪🇸Murcia, Spain
Hospital Universitario y Politecnico La Fe
🇪🇸Valencia, Spain
Karolinska Universitetssjukhuset, Huddinge
🇸🇪Stockholm, Sweden
Medizinische Universität Innsbruck
🇦🇹Innsbruck, Austria