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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/NCT02412111
Registration number
NCT02412111
Ethics application status
Date submitted
3/04/2015
Date registered
8/04/2015
Date last updated
8/01/2019
Titles & IDs
Public title
A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor
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Scientific title
A Phase 3, Randomized, Double-Blind, Ivacaftor-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and a Second CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive
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Secondary ID [1]
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VX14-661-109
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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 - Ivacaftor
Treatment: Drugs - Tezacaftor/Ivacaftor
Experimental: Ivacaftor (Run-in Period) - Ivacaftor 150 milligram (mg) tablet orally every 12 hours for 4 weeks.
Experimental: VX-661 + Ivacaftor (Active comparator period) - VX-661 100 mg and ivacaftor 150 mg fixed-dose combination tablet orally once daily in the morning and ivacaftor 150 mg tablet orally once daily in the evening for 8 weeks.
Active Comparator: Ivacaftor monotherapy (Active comparator period) - Ivacaftor 150 mg tablet orally every 12 hours as monotherapy for 8 weeks.
Treatment: Drugs: Ivacaftor
Treatment: Drugs: Tezacaftor/Ivacaftor
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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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Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8
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Assessment method [1]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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Timepoint [1]
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Baseline, Through Week 8
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Secondary outcome [1]
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Relative Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8
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Assessment method [1]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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Timepoint [1]
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Baseline, Through Week 8
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Secondary outcome [2]
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Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline Through Week 8
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Assessment method [2]
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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Timepoint [2]
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Baseline, Through Week 8
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Secondary outcome [3]
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Absolute Change From Baseline in Sweat Chloride Through Week 8
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Assessment method [3]
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Sweat samples were collected using an approved collection device.
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Timepoint [3]
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Baseline, Through Week 8
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Secondary outcome [4]
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Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [4]
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Timepoint [4]
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Baseline up to Week 16
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Secondary outcome [5]
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Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
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Assessment method [5]
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Timepoint [5]
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Predose on Week -2 for Run-in period; Pre-dose on Week 2 for Active comparator period
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Eligibility
Key inclusion criteria
- Heterozygous for F508del-CFTR mutation and a second CFTR allele with a gating defect
that is clinically demonstrated to be ivacaftor responsive
- FEV1 =40% and =90% of predicted normal for age, sex, and height during screening
- Stable CF disease as judged by the investigator.
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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 any comorbidity that, in the opinion of the investigator, might confound
the results of the study or pose an additional risk in administering study drug to the
subject.
- Pregnant and nursing females (females of childbearing potential must have a negative
pregnancy test at Screening and Week -4 Visits).
- Sexually active subjects of reproductive potential who are not willing to follow the
contraception requirements
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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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
1/06/2015
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Date of last participant enrolment
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Actual
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Date of last data collection
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Actual
1/09/2017
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Sample size
Target
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Accrual to date
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Final
156
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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- Chermside
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- Clayton
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- Melbourne
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- South Brisbane
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- Westmead
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Recruitment postcode(s) [1]
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- Chermside
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- Clayton
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- Melbourne
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- South Brisbane
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- Westmead
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Recruitment outside Australia
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Alabama
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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 is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group,
multicenter study of tezacaftor in combination with ivacaftor in subjects aged 12 years and
older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele
with a gating defect that is clinically demonstrated to be ivacaftor responsive.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02412111
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Public notes
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Contacts
Principal investigator
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02412111
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