Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
- Registration Number
- NCT05331183
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 297
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
History of study drug intolerance in the parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description ELX/TEZ/IVA 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. ELX/TEZ/IVA 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.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Part A: Absolute Change in Weight From Baseline up to Week 96 Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score From Baseline up to Week 96 Part A: Absolute Change in Body Mass Index (BMI) From Baseline up to Week 96 Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1) From Baseline up to Week 96 Part A: Absolute Change in Sweat Chloride (SwCl) From Baseline up to Week 96 Part A: Number of Pulmonary Exacerbations (PEx) From Baseline up to Week 96
Trial Locations
- Locations (81)
Medizinische Universitรคt Innsbruck
๐ฆ๐นInnsbruck, Austria
Cliniques Universitaires de Bruxelles Hopital Erasme
๐ง๐ชBrussels, Belgium
CHU Marseille - Hopital Nord
๐ซ๐ทMarseille, France
Universitair Ziekenhuis Brussel - Campus Jette
๐ง๐ชBrussels, Belgium
Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital
๐ง๐ชEdegem, Belgium
The Hospital for Sick Children
๐จ๐ฆToronto, Canada
Cliniques Universitaires Saint-Luc
๐ง๐ชWoluwe-Saint-Lambert, Belgium
St. Paul's Hospital
๐จ๐ฆVancouver, Canada
Johann Wolfgang Goethe University
๐ฉ๐ชFrankfurt, Germany
Pneumologisches Studienzentrum Muenchen-West
๐ฉ๐ชMuenchen, Germany
Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
๐จ๐ฆMontreal, Canada
Universitair Ziekenhuis Gent
๐ง๐ชGent, Belgium
University of Alberta Hospital, Edmonton Clinic
๐จ๐ฆEdmonton, Canada
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
๐ง๐ชLeuven, Belgium
McGill University Health Centre, Glen Site, Montreal Children's Hospital
๐จ๐ฆMontreal, Canada
British Columbia Children's Hospital
๐จ๐ฆVancouver, Canada
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
๐ฎ๐นGenova, Italy
CHU Lyon - Hopital Femme Mere-Enfant
๐ซ๐ทBron Cedex, France
Klinika Detskych Infekcnich Nemoci
๐จ๐ฟBrno, Czechia
Centre Hospitalier Intercommunal Creteil
๐ซ๐ทCrรฉteil, France
Fakultni nemocnice v Motole
๐จ๐ฟPraha 5, Czechia
Institut Cลur Poumon, CHU de Lille
๐ซ๐ทLille, France
Hopital Arnaud de Villeneuve
๐ซ๐ทMontpellier Cedex 5, France
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
๐ซ๐ทNice, France
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
๐ซ๐ทNantes, France
Hopital Necker, Enfants Malades
๐ซ๐ทParis Cedex 15, France
Hopital Cochin
๐ซ๐ทParis, France
CHU de Rennes - Hรดpital Sud
๐ซ๐ทRennes, France
Centre Hospitalier Universitaire De Reims, Hopital Sebastopol
๐ซ๐ทReims, France
Hopital Robert Debre
๐ซ๐ทParis, France
Centre Hospitalier Lyon Sud
๐ซ๐ทPierre-Bรฉnite, France
Centre de Perharidy
๐ซ๐ทRoscoff cedex, France
CHU de Toulouse - Hopital Larre
๐ซ๐ทToulouse, France
Charite Paediatric Pulmonology Department
๐ฉ๐ชBerlin, Germany
Hopital Bretonneau
๐ซ๐ทTours, France
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
๐ฉ๐ชEssen, Germany
Justus-Liebig-Universitรคt Gieรen Zentrum fรผr Kinderheilkunde und Jugendmedizin
๐ฉ๐ชGieรen, Germany
Universitรคtsklinikum Halle (Saale) / Universitรคtsklinik und Poliklinik fรผr Innere Medizin, Schwerpunkt Pneumologie
๐ฉ๐ชHalle, Germany
Hannover Medical School
๐ฉ๐ชHannover, Germany
Medizinische Hochschule Hannover
๐ฉ๐ชHannover, Germany
Universitaetsklinikum Jena, Mukoviszidose-Zentrum
๐ฉ๐ชJena, Germany
National Koranyi Institute for TBC and Pulmonology
๐ญ๐บBudapest, Hungary
Dr. von Haunersches Kinderspital
๐ฉ๐ชMunchen, Germany
Klinikum Westbrandenburg (CF)
๐ฉ๐ชPotsdam, Germany
Johannes Gutenberg-Universitaet
๐ฉ๐ชMainz, Germany
Universitaetsklinkum Koeln, CF-Studienzentrum
๐ฉ๐ชKoeln, Germany
Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster
๐ฉ๐ชMรผnster, Germany
Ospedale Pediatrico Bambino Gesu
๐ฎ๐นRome, Italy
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
๐ฎ๐นAncona, Italy
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
๐ฎ๐นFirenze, Italy
Azienda Ospedaliera Universitaria Policlinico G. Martino
๐ฎ๐นMessina, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
๐ฎ๐นMilano, Italy
Azienda Ospedaliera Universitaria Federico II (Adults)
๐ฎ๐นNaples, Italy
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
๐ฎ๐นMilan, Italy
Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
๐ฎ๐นOrbassano, Italy
Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
๐ฎ๐นPotenza, Italy
HagaZiekenhuis van den Haag
๐ณ๐ฑDen Haag, Netherlands
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
๐ฎ๐นVerona, Italy
Erasmus Medical Center
๐ณ๐ฑRotterdam, Netherlands
UMCU, Locatie Wilhelmina Kinderziekenhuis
๐ณ๐ฑUtrecht, Netherlands
Academisch Medisch Centrum (Academic Medical Centre)
๐ณ๐ฑAmsterdam, Netherlands
UMC St. Radboud
๐ณ๐ฑNijmegen, Netherlands
Oslo University Hospital, Department of Paediatric Medicine
๐ณ๐ดOslo, Norway
Pediatric Hospital Polanki named of Maciej Pลaลผyลski
๐ต๐ฑGdaลsk, Poland
Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddziaล Chorรณb Pลuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leลnym
๐ต๐ฑลomianki, Poland
Hospital de Cruces
๐ช๐ธBarakaldo, Spain
Hospital de Santa Maria
๐ต๐นLisbon, Portugal
Hospital Saint Joan de Deu
๐ช๐ธBarcelona, Spain
Hospital Universitari Vall dยดHebron Servicio de Broncoscopia
๐ช๐ธBarcelona, Spain
Hospital Universitario 12 de Octubre
๐ช๐ธMadrid, Spain
Hospital Universitario de Jerez de la Frontera
๐ช๐ธJerez de la Frontera, Spain
Hospital Universitario Infantil La Paz
๐ช๐ธMadrid, Spain
Hospital Virgen de la Arrixaca
๐ช๐ธMurcia, Spain
Hospital Universitario Virgen del Rocio
๐ช๐ธSevilla, Spain
Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
๐ช๐ธSabadell, Spain
Hospital Universitario y Politecnico La Fe
๐ช๐ธValencia, Spain
Sahlgrenska Universitetssjukhuset
๐ธ๐ชGรถteborg, Sweden
Karolinska Universitetssjukhuset, Huddinge
๐ธ๐ชStockholm, Sweden
Kinderspital Zuerich
๐จ๐ญZรผrich, Switzerland
Hospital Infantil Universitario Nino Jesus
๐ช๐ธMadrid, Spain
Hospital Universitario Ramon y Cajal
๐ช๐ธMadrid, Spain