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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/NCT02392234
Registration number
NCT02392234
Ethics application status
Date submitted
12/03/2015
Date registered
18/03/2015
Date last updated
12/06/2018
Titles & IDs
Public title
A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation
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Scientific title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and 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 Allele With a CFTR Mutation Predicted to Have Residual Function
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Secondary ID [1]
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2014-004788-18
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Secondary ID [2]
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VX14-661-108
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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 - VX-661/Ivacaftor
Treatment: Drugs - Ivacaftor
Treatment: Drugs - Placebo matched to VX-661/ ivacaftor
Treatment: Drugs - Placebo matched to Ivacaftor
Experimental: VX-661/Ivacaftor combination -
Experimental: Ivacaftor monotherapy -
Placebo Comparator: Placebo -
Treatment: Drugs: VX-661/Ivacaftor
Fixed dose combination tablet, oral use
Treatment: Drugs: Ivacaftor
Tablet, oral use
Treatment: Drugs: Placebo matched to VX-661/ ivacaftor
Fixed dose combination tablet, oral use
Treatment: Drugs: Placebo matched to Ivacaftor
Tablet, oral use
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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 Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and 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, Week 4 and Week 8 of each treatment period
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Secondary outcome [1]
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Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8
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Assessment method [1]
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The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.
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Timepoint [1]
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Baseline, Week 4 and Week 8 of each treatment period
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Secondary outcome [2]
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Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [2]
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Timepoint [2]
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Day 1 up to Week 28
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Secondary outcome [3]
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Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8
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Assessment method [3]
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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 [3]
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Baseline, Week 4 and Week 8 of each treatment period
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Secondary outcome [4]
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Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8
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Assessment method [4]
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Baseline, Week 4 and Week 8 of each treatment period
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Secondary outcome [5]
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Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy
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Assessment method [5]
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Timepoint [5]
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Pre-morning dose on Week 8 of each treatment period
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Secondary outcome [6]
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Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy
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Assessment method [6]
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Timepoint [6]
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Pre-morning dose on Week 8 of each treatment period
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Eligibility
Key inclusion criteria
- Heterozygous for F508del-CFTR and a second allele with a CFTR mutation predicted to
have residual function
- Forced Expiratory Volume in 1 Second (FEV1) greater than or equal to (=) 40 percent
(%) and less than or equal to (=) 90% of predicted normal for age, sex, and height
during screening
- Sweat chloride value =60 millimole per liter (mmol/L) during screening OR as
documented in the participant's medical record
- 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
participant
- An acute upper or lower respiratory infection, pulmonary exacerbation
- History of solid organ or hematological transplantation
- Ongoing or prior participation in an investigational drug study (including studies
investigating VX-661, lumacaftor [VX-809], and/or ivacaftor) within 30 days of
screening
- Pregnant and nursing females
- Sexually active participants 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 analysing the results/data
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Intervention assignment
Crossover
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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
1/03/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
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Actual
1/02/2017
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Sample size
Target
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Accrual to date
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Final
248
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Recruitment in Australia
Recruitment state(s)
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- Adelaide
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- Melbourne
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- South Brisbane
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- Westmead
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- Adelaide
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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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United States of America
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Funding & Sponsors
Primary sponsor type
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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
The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination
with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF)
who are heterozygous for F508del-CFTR allele and a second allele with a CFTR mutation
predicted to have residual function.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02392234
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Public notes
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Contacts
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02392234
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