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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04362761
Registration number
NCT04362761
Ethics application status
Date submitted
22/04/2020
Date registered
27/04/2020
Date last updated
28/07/2023
Titles & IDs
Public title
A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy
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Scientific title
A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects
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Secondary ID [1]
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2019-003455-11
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Secondary ID [2]
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VX19-445-115
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Cystic fibrosis
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Respiratory
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Other respiratory disorders / diseases
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Oral and Gastrointestinal
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Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
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Inflammatory and Immune System
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Connective tissue diseases
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Inflammatory and Immune System
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Other inflammatory or immune system disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - ELX/TEZ/IVA
Treatment: Drugs - IVA
Experimental: ELX/TEZ/IVA - Part 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.
Treatment: Drugs: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration.
Treatment: Drugs: IVA
Tablet for oral administration.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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Assessment method [1]
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Timepoint [1]
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From Day 1 up to Week 52
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Primary outcome [2]
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Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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Assessment method [2]
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Timepoint [2]
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From Day 1 up to Week 86
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Eligibility
Key inclusion criteria
Key
- 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
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Minimum age
12
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- History of study drug intolerance in parent study
Other protocol defined Inclusion/Exclusion criteria may apply.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
4/05/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
21/12/2022
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Sample size
Target
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Accrual to date
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Final
172
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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The Prince Charles Hospital - Chermside
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Recruitment hospital [2]
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Institute for Respiratory Health - Nedlands
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Recruitment hospital [3]
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Telethon Kids Institute, Perth Children's Hospital - Nedlands
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Recruitment hospital [4]
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The Royal Children's Hospital - Parkville, VIC
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Recruitment hospital [5]
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Queensland Children's Hospital - South Brisbane
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Recruitment postcode(s) [1]
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- Chermside
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Recruitment postcode(s) [2]
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- Nedlands
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Recruitment postcode(s) [3]
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- Parkville, VIC
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Recruitment postcode(s) [4]
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- South Brisbane
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Recruitment outside Australia
Country [1]
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Belgium
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State/province [1]
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Gent
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Belgium
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Leuven
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Germany
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Berlin
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Germany
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Essen
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Germany
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Jena
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Germany
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Koeln
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Germany
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München
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United Kingdom
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Belfast
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United Kingdom
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Birmingham
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Bristol
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United Kingdom
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Cambridge
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Edinburgh
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Exeter
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Glasgow
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United Kingdom
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Leeds
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United Kingdom
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Liverpool
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United Kingdom
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London
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United Kingdom
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Newcastle Upon Tyne
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United Kingdom
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Nottingham
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United Kingdom
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Penarth
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United Kingdom
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State/province [21]
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Southampton
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Vertex Pharmaceuticals Incorporated
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Address
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04362761
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
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Address
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Phone
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Fax
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Email
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04362761
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