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Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

Primary Purpose

Cystic Fibrosis

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ELX/TEZ/IVA
IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • 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

Key Exclusion Criteria:

  • History of study drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

  • Medizinische Universität Innsbruck
  • Cliniques Universitaires de Bruxelles Hopital Erasme
  • Universitair Ziekenhuis Brussel - Campus Jette
  • Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital
  • Universitair Ziekenhuis Gent
  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
  • Cliniques Universitaires Saint-Luc
  • University of Alberta Hospital, Edmonton Clinic
  • Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
  • McGill University Health Centre, Glen Site, Montreal Children's Hospital
  • The Hospital for Sick Children
  • British Columbia Children's Hospital
  • St. Paul's Hospital
  • Klinika Detskych Infekcnich Nemoci
  • Fakultni nemocnice v Motole
  • CHU Lyon - Hopital Femme Mere-Enfant
  • Centre Hospitalier Intercommunal Creteil
  • Institut Cœur Poumon, CHU de Lille
  • CHU Marseille - Hopital Nord
  • Hopital Arnaud de Villeneuve
  • Centre Hospitalier Universitaire De Nantes - G. R. Laennec
  • Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
  • Hopital Necker, Enfants Malades
  • Hopital Cochin
  • Hopital Robert Debre
  • Centre Hospitalier Lyon Sud
  • Centre Hospitalier Universitaire De Reims, Hopital Sebastopol
  • CHU de Rennes - Hôpital Sud
  • Centre de Perharidy
  • CHU de Toulouse - Hopital Larre
  • Hopital Bretonneau
  • Charite Paediatric Pulmonology Department
  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
  • Johann Wolfgang Goethe University
  • Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
  • Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
  • Hannover Medical School
  • Medizinische Hochschule Hannover
  • Universitaetsklinikum Jena, Mukoviszidose-Zentrum
  • Universitaetsklinkum Koeln, CF-Studienzentrum
  • Johannes Gutenberg-Universitaet
  • Pneumologisches Studienzentrum Muenchen-West
  • Dr. von Haunersches Kinderspital
  • Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster
  • Klinikum Westbrandenburg (CF)
  • National Koranyi Institute for TBC and Pulmonology
  • Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
  • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
  • Azienda Ospedaliera Universitaria Policlinico G. Martino
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
  • Azienda Ospedaliera Universitaria Federico II (Adults)
  • Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
  • Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
  • Ospedale Pediatrico Bambino Gesu
  • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
  • Academisch Medisch Centrum (Academic Medical Centre)
  • HagaZiekenhuis van den Haag
  • UMC St. Radboud
  • Erasmus Medical Center
  • UMCU, Locatie Wilhelmina Kinderziekenhuis
  • Oslo University Hospital, Department of Paediatric Medicine
  • Pediatric Hospital Polanki named of Maciej Płażyński
  • Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym
  • Hospital de Santa Maria
  • Hospital de Cruces
  • Hospital Saint Joan de Deu
  • Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
  • Hospital Universitario de Jerez de la Frontera
  • Hospital Infantil Universitario Nino Jesus
  • Hospital Universitario 12 de Octubre
  • Hospital Universitario Infantil La Paz
  • Hospital Universitario Ramon y Cajal
  • Hospital Virgen de la Arrixaca
  • Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
  • Hospital Universitario Virgen del Rocio
  • Hospital Universitario y Politecnico La Fe
  • Sahlgrenska Universitetssjukhuset
  • Karolinska Universitetssjukhuset, Huddinge
  • Kinderspital Zuerich

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ELX/TEZ/IVA

Arm Description

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Outcomes

Primary Outcome Measures

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)
Absolute Change in Sweat Chloride (SwCl)
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Absolute Change in Body Mass Index (BMI)
Absolute Change in Weight
Number of Pulmonary Exacerbations (PEx)

Full Information

First Posted
April 8, 2022
Last Updated
August 3, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT05331183
Brief Title
Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
Official Title
A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 23, 2022 (Actual)
Primary Completion Date
April 2025 (Anticipated)
Study Completion Date
April 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
297 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ELX/TEZ/IVA
Arm Type
Experimental
Arm Description
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Intervention Type
Drug
Intervention Name(s)
ELX/TEZ/IVA
Other Intervention Name(s)
VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Intervention Description
Fixed-dose combination (FDC) tablets for oral administration.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Tablets for oral administration.
Primary Outcome Measure Information:
Title
Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
Day 1 up to Week 100
Secondary Outcome Measure Information:
Title
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)
Time Frame
From Baseline up to Week 96
Title
Absolute Change in Sweat Chloride (SwCl)
Time Frame
From Baseline up to Week 96
Title
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Time Frame
From Baseline up to Week 96
Title
Absolute Change in Body Mass Index (BMI)
Time Frame
From Baseline up to Week 96
Title
Absolute Change in Weight
Time Frame
From Baseline up to Week 96
Title
Number of Pulmonary Exacerbations (PEx)
Time Frame
From Baseline up to Week 96

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: 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 Key Exclusion Criteria: History of study drug intolerance in the parent study Other protocol defined Inclusion/Exclusion criteria may apply.
Facility Information:
Facility Name
Medizinische Universität Innsbruck
City
Innsbruck
Country
Austria
Facility Name
Cliniques Universitaires de Bruxelles Hopital Erasme
City
Brussels
Country
Belgium
Facility Name
Universitair Ziekenhuis Brussel - Campus Jette
City
Brussels
Country
Belgium
Facility Name
Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital
City
Edegem
Country
Belgium
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
City
Leuven
Country
Belgium
Facility Name
Cliniques Universitaires Saint-Luc
City
Woluwe-Saint-Lambert
Country
Belgium
Facility Name
University of Alberta Hospital, Edmonton Clinic
City
Edmonton
Country
Canada
Facility Name
Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
City
Montreal
Country
Canada
Facility Name
McGill University Health Centre, Glen Site, Montreal Children's Hospital
City
Montreal
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
British Columbia Children's Hospital
City
Vancouver
Country
Canada
Facility Name
St. Paul's Hospital
City
Vancouver
Country
Canada
Facility Name
Klinika Detskych Infekcnich Nemoci
City
Brno
Country
Czechia
Facility Name
Fakultni nemocnice v Motole
City
Praha 5
Country
Czechia
Facility Name
CHU Lyon - Hopital Femme Mere-Enfant
City
Bron Cedex
Country
France
Facility Name
Centre Hospitalier Intercommunal Creteil
City
Créteil
Country
France
Facility Name
Institut Cœur Poumon, CHU de Lille
City
Lille
Country
France
Facility Name
CHU Marseille - Hopital Nord
City
Marseille
Country
France
Facility Name
Hopital Arnaud de Villeneuve
City
Montpellier Cedex 5
Country
France
Facility Name
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
City
Nantes
Country
France
Facility Name
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
City
Nice
Country
France
Facility Name
Hopital Necker, Enfants Malades
City
Paris Cedex 15
Country
France
Facility Name
Hopital Cochin
City
Paris
Country
France
Facility Name
Hopital Robert Debre
City
Paris
Country
France
Facility Name
Centre Hospitalier Lyon Sud
City
Pierre-Bénite
Country
France
Facility Name
Centre Hospitalier Universitaire De Reims, Hopital Sebastopol
City
Reims
Country
France
Facility Name
CHU de Rennes - Hôpital Sud
City
Rennes
Country
France
Facility Name
Centre de Perharidy
City
Roscoff cedex
Country
France
Facility Name
CHU de Toulouse - Hopital Larre
City
Toulouse
Country
France
Facility Name
Hopital Bretonneau
City
Tours
Country
France
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
City
Essen
Country
Germany
Facility Name
Johann Wolfgang Goethe University
City
Frankfurt
Country
Germany
Facility Name
Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
City
Gießen
Country
Germany
Facility Name
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
City
Halle
Country
Germany
Facility Name
Hannover Medical School
City
Hannover
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
Country
Germany
Facility Name
Universitaetsklinikum Jena, Mukoviszidose-Zentrum
City
Jena
Country
Germany
Facility Name
Universitaetsklinkum Koeln, CF-Studienzentrum
City
Koeln
Country
Germany
Facility Name
Johannes Gutenberg-Universitaet
City
Mainz
Country
Germany
Facility Name
Pneumologisches Studienzentrum Muenchen-West
City
Muenchen
Country
Germany
Facility Name
Dr. von Haunersches Kinderspital
City
Munchen
Country
Germany
Facility Name
Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster
City
Münster
Country
Germany
Facility Name
Klinikum Westbrandenburg (CF)
City
Potsdam
Country
Germany
Facility Name
National Koranyi Institute for TBC and Pulmonology
City
Budapest
Country
Hungary
Facility Name
Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona
City
Ancona
Country
Italy
Facility Name
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
City
Firenze
Country
Italy
Facility Name
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
City
Genova
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Policlinico G. Martino
City
Messina
Country
Italy
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
Milano
Country
Italy
Facility Name
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
City
Milan
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Federico II (Adults)
City
Naples
Country
Italy
Facility Name
Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
City
Orbassano
Country
Italy
Facility Name
Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
City
Potenza
Country
Italy
Facility Name
Ospedale Pediatrico Bambino Gesu
City
Rome
Country
Italy
Facility Name
Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
City
Verona
Country
Italy
Facility Name
Academisch Medisch Centrum (Academic Medical Centre)
City
Amsterdam
Country
Netherlands
Facility Name
HagaZiekenhuis van den Haag
City
Den Haag
Country
Netherlands
Facility Name
UMC St. Radboud
City
Nijmegen
Country
Netherlands
Facility Name
Erasmus Medical Center
City
Rotterdam
Country
Netherlands
Facility Name
UMCU, Locatie Wilhelmina Kinderziekenhuis
City
Utrecht
Country
Netherlands
Facility Name
Oslo University Hospital, Department of Paediatric Medicine
City
Oslo
Country
Norway
Facility Name
Pediatric Hospital Polanki named of Maciej Płażyński
City
Gdańsk
Country
Poland
Facility Name
Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym
City
Łomianki
Country
Poland
Facility Name
Hospital de Santa Maria
City
Lisbon
Country
Portugal
Facility Name
Hospital de Cruces
City
Barakaldo
Country
Spain
Facility Name
Hospital Saint Joan de Deu
City
Barcelona
Country
Spain
Facility Name
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
City
Barcelona
Country
Spain
Facility Name
Hospital Universitario de Jerez de la Frontera
City
Jerez de la Frontera
Country
Spain
Facility Name
Hospital Infantil Universitario Nino Jesus
City
Madrid
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
Country
Spain
Facility Name
Hospital Universitario Infantil La Paz
City
Madrid
Country
Spain
Facility Name
Hospital Universitario Ramon y Cajal
City
Madrid
Country
Spain
Facility Name
Hospital Virgen de la Arrixaca
City
Murcia
Country
Spain
Facility Name
Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari
City
Sabadell
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocio
City
Sevilla
Country
Spain
Facility Name
Hospital Universitario y Politecnico La Fe
City
Valencia
Country
Spain
Facility Name
Sahlgrenska Universitetssjukhuset
City
Göteborg
Country
Sweden
Facility Name
Karolinska Universitetssjukhuset, Huddinge
City
Stockholm
Country
Sweden
Facility Name
Kinderspital Zuerich
City
Zürich
Country
Switzerland

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
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

Learn more about this trial

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

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