Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT05274269
Other study ID # VX21-445-124
Secondary ID 2021-005320-38
Status Completed
Phase Phase 3
First received
Last updated
Start date May 9, 2022
Est. completion date July 5, 2023

Study information

Verified date July 2023
Source Vertex Pharmaceuticals Incorporated
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study will evaluate 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.


Recruitment information / eligibility

Status Completed
Enrollment 307
Est. completion date July 5, 2023
Est. primary completion date July 5, 2023
Accepts healthy volunteers No
Gender All
Age group 6 Years and older
Eligibility Key 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 Key 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 Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
ELX/TEZ/IVA
Fixed-dose combination (FDC) tablets for oral administration.
IVA
Tablet for oral administration.
Other:
Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration.
Placebo (matched to IVA)
Placebo matched to IVA for oral administration.

Locations

Country Name City State
Austria Medizinische Universität Innsbruck Innsbruck
Belgium 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 Gent
Belgium Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg Leuven
Belgium Cliniques Universitaires Saint-Luc Woluwe-Saint-Lambert
Canada University of Alberta Hospital, Edmonton Clinic 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
Czechia Klinika Detskych Infekcnich Nemoci Brno
Czechia Fakultni nemocnice v Motole Praha 5
France 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 Hopital Arnaud de Villeneuve Montpellier Cedex 5
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 Robert Debre Paris
France Hopital Necker, Enfants Malades Paris Cedex 15
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 Cedex
France CHU de Toulouse - Hopital Larre Toulouse
France Hopital Bretonneau Tours
Germany Charite Paediatric Pulmonology Department Berlin
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 Hannover
Germany Medizinische Hochschule Hannover Hannover
Germany Universitaetsklinikum Jena, Mukoviszidose-Zentrum Jena
Germany Universitaetsklinkum Koeln, CF-Studienzentrum Koeln
Germany Johannes Gutenberg-Universitaet Mainz
Germany Pneumologisches Studienzentrum Muenchen-West Muenchen
Germany Dr. von Haunersches Kinderspital München
Germany Klinikum Innenstadt, University of Munich München
Germany Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster Münster
Germany Klinikum Westbrandenburg (CF) Potsdam
Germany Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin Tuebingen
Germany Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF) Ulm
Germany Universitätsklinikum Würzburg Würzburg
Hungary National Koranyi Institute for TBC and Pulmonology Budapest
Italy Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona Ancona
Italy Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer Firenze
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 Milano
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
Netherlands Academisch Medisch Centrum (Academic Medical Centre) Amsterdam
Netherlands HagaZiekenhuis van den Haag Den Haag
Netherlands UMC St. Radboud Nijmegen
Netherlands Erasmus Medical Center Rotterdam
Netherlands Universitair Medisch Centrum Utrecht Utrecht
Norway Oslo University Hospital, Department of Paediatric Medicine Oslo
Poland Pediatric Hospital Polanki named of Maciej Plazynski Gdansk
Poland Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddzial Chorób Pluc SZPZOZ im.Dzieci Warszawy w Dziekanowie Lesnym Lomianki
Poland Institute of Tuberculosis and Lung Diseases Rabka-Zdrój
Portugal Hospital de Santa Maria Lisbon
Portugal Hospital Sao Joao Porto
Spain 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 Sevilla
Spain Hospital Universitario y Politecnico La Fe Valencia
Sweden Sahlgrenska Universitetssjukhuset Göteborg
Sweden Karolinska Universitetssjukhuset, Huddinge Stockholm
Switzerland Kinderspital Zuerich Zurich

Sponsors (1)

Lead Sponsor Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

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

Outcome

Type Measure Description Time frame Safety issue
Primary Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) From Baseline Through Week 24
Secondary Absolute Change in Sweat Chloride (SwCl) From Baseline Through Week 24
Secondary Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score From Baseline Through Week 24
Secondary Absolute Change in Body Mass Index (BMI) From Baseline at Week 24
Secondary Absolute Change in Weight From Baseline at Week 24
Secondary Number of Pulmonary Exacerbations (PEx) From Baseline Through Week 24
Secondary Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) Day 1 up to Week 28
See also
  Status Clinical Trial Phase
Completed NCT04696198 - Thoracic Mobility in Cystic Fibrosis Care N/A
Completed NCT00803205 - Study of Ataluren (PTC124™) in Cystic Fibrosis Phase 3
Terminated NCT04921332 - Bright Light Therapy for Depression Symptoms in Adults With Cystic Fibrosis (CF) and COPD N/A
Completed NCT03601637 - Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del Phase 3
Terminated NCT02769637 - Effect of Acid Blockade on Microbiota and Inflammation in Cystic Fibrosis (CF)
Recruiting NCT06030206 - Lung Transplant READY CF 2: A Multi-site RCT N/A
Recruiting NCT06012084 - The Development and Evaluation of iCF-PWR for Healthy Siblings of Individuals With Cystic Fibrosis N/A
Recruiting NCT06032273 - Lung Transplant READY CF 2: CARING CF Ancillary RCT N/A
Recruiting NCT06088485 - The Effect of Bone Mineral Density in Patients With Adult Cystic Fibrosis
Recruiting NCT05392855 - Symptom Based Performance of Airway Clearance After Starting Highly Effective Modulators for Cystic Fibrosis (SPACE-CF) N/A
Recruiting NCT04056702 - Impact of Triple Combination CFTR Therapy on Sinus Disease.
Recruiting NCT04039087 - Sildenafil Exercise: Role of PDE5 Inhibition Phase 2/Phase 3
Completed NCT04038710 - Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.
Completed NCT04058548 - Clinical Utility of the 1-minute Sit to Stand Test as a Measure of Submaximal Exercise Tolerance in Patients With Cystic Fibrosis During Acute Pulmonary Exacerbation N/A
Completed NCT03637504 - Feasibility of a Mobile Medication Plan Application in CF Patient Care N/A
Recruiting NCT03506061 - Trikafta in Cystic Fibrosis Patients Phase 2
Completed NCT03566550 - Gut Imaging for Function & Transit in Cystic Fibrosis Study 1
Recruiting NCT04828382 - Prospective Study of Pregnancy in Women With Cystic Fibrosis
Completed NCT04568980 - Assessment of Contraceptive Safety and Effectiveness in Cystic Fibrosis
Recruiting NCT04010253 - Impact of Bronchial Drainage Technique by the Medical Device Simeox® on Respiratory Function and Symptoms in Adult Patients With Cystic Fibrosis N/A