A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy
A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects
2 other identifiers
interventional
172
4 countries
29
Brief Summary
This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started May 2020
Typical duration for phase_3
29 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
April 22, 2020
CompletedFirst Posted
Study publicly available on registry
April 27, 2020
CompletedStudy Start
First participant enrolled
May 4, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 21, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
December 21, 2022
CompletedResults Posted
Study results publicly available
July 28, 2023
CompletedJuly 28, 2023
July 1, 2023
2.6 years
April 22, 2020
June 20, 2023
July 11, 2023
Conditions
Outcome Measures
Primary Outcomes (2)
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 52
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 86
Study Arms (1)
ELX/TEZ/IVA
EXPERIMENTALPart A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.
Interventions
Fixed-dose combination (FDC) tablet for oral administration.
Eligibility Criteria
You may qualify if:
- Completed study drug treatment in parent study (VX18-445-109); 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
You may not qualify if:
- History of study drug intolerance in parent study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (29)
The Prince Charles Hospital
Chermside, Australia
Institute for Respiratory Health
Nedlands, Australia
Telethon Kids Institute, Perth Children's Hospital
Nedlands, Australia
The Royal Children's Hospital
Parkville, VIC, Australia
Queensland Children's Hospital
South Brisbane, Australia
Universitair Ziekenhuis Gent
Ghent, Belgium
Universitair Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
Charite Paediatric Pulmonology Department
Berlin, Germany
Universitaetsklinkum Koeln, CF-Studienzentrum
Cologne, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, Germany
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
Essen, Germany
Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin
Jena, Germany
Klinikum Innenstadt, University of Munich
München, Germany
Belfast City Hospital
Belfast, United Kingdom
University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
Bristol, United Kingdom
Royal Papworth Hospital NHS Foundation Trust
Cambridge, United Kingdom
Western General Hospital
Edinburgh, United Kingdom
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
Exeter, United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
Leeds General Infirmary
Leeds, United Kingdom
St. James University Hospital
Leeds, United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom
Great Ormond Street Hospital for Sick Children
London, United Kingdom
London and St Bartholomew's Hospital
London, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Nottingham University Hospitals NHS Trust, Queens Medical Center
Nottingham, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth, United Kingdom
Southampton General Hospital
Southampton, United Kingdom
Related Publications (2)
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PMID: 37983082DERIVEDSouthern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
PMID: 33331662DERIVED
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
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 22, 2020
First Posted
April 27, 2020
Study Start
May 4, 2020
Primary Completion
December 21, 2022
Study Completion
December 21, 2022
Last Updated
July 28, 2023
Results First Posted
July 28, 2023
Record last verified: 2023-07
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.