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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/NCT01807949
Registration number
NCT01807949
Ethics application status
Date submitted
4/03/2013
Date registered
8/03/2013
Date last updated
27/09/2016
Titles & IDs
Public title
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
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Scientific title
A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
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Secondary ID [1]
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VX12-809-104
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Universal Trial Number (UTN)
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Trial acronym
TRANSPORT
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
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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 - Placebo
Treatment: Drugs - Lumacaftor Plus Ivacaftor Combination
Treatment: Drugs - Ivacaftor
Placebo Comparator: Placebo - Placebo matched to lumacaftor (LUM, VX-809) and ivacaftor (IVA, VX-770) tablet every 12 hours (q12h), up to Week 24.
Experimental: LUM 600 mg qd/IVA 250 mg q12h - LUM 600 milligram (mg) plus IVA 250 mg fixed-dose combination (FDC) tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24.
Experimental: LUM 400 mg q12h/ IVA 250 mg q12h - LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24.
Treatment: Drugs: Placebo
Matching placebo tablet
Treatment: Drugs: Lumacaftor Plus Ivacaftor Combination
Fixed dose combination tablet
Treatment: Drugs: Ivacaftor
Film-coated tablet
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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) at Week 24
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Assessment method [1]
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Absolute change from baseline at week 24 was assessed as the average treatment effect at Week 16 and at Week 24. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.
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Timepoint [1]
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Baseline, Week 16 and 24
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Secondary outcome [1]
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Relative Change From Baseline in Percent Predicted FEV1 at Week 24
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Assessment method [1]
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Assessed as the average treatment effect at Week 16 and at Week 24. FEV1 and percent predicted FEV1 are defined in Outcome Measure (OM) 1.
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Timepoint [1]
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Baseline, Week 16 and 24
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Secondary outcome [2]
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Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
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Assessment method [2]
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BMI was defined as weight in kilogram (kg) divided by height*height in square meter (m^2).
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Timepoint [2]
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Baseline, Week 24
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Secondary outcome [3]
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Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
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Assessment method [3]
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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 (for example, coughing, congestion, wheezing), the scaled score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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Timepoint [3]
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Baseline, Week 24
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Secondary outcome [4]
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Percentage of Participants With Response Based on Percent Predicted FEV1
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Assessment method [4]
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A participant was considered as a responder if the participant had >=5% increase from baseline in average percent predicted FEV1 at Week 16 and at Week 24 (relative change). FEV1 and percent predicted FEV1 are defined in OM 1. A participant with a missing average relative change from baseline in percent predicted FEV1 at Week 16 and at Week 24 was considered as a non-responder.
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Timepoint [4]
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Week 16 and 24
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Secondary outcome [5]
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Number of Pulmonary Exacerbation Events
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Assessment method [5]
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The total number of days on study is equal to the Week 24 date or the last dose date (whichever occurred last) minus the first dose date plus 1. The total number of years (48 weeks) on study is equal to the number of days on study divided by 336. Pulmonary exacerbation events per year (48 weeks) are reported.
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Timepoint [5]
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through Week 24
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Secondary outcome [6]
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Absolute Change From Baseline in Weight at Week 24
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Assessment method [6]
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Timepoint [6]
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Baseline, Week 24
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Secondary outcome [7]
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Absolute Change From Baseline in BMI-for-age Z-score at Week 24
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Assessment method [7]
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Z-Score is a statistical measure to evaluate how a single data point compares to a standard. It describes whether a mean was above or below the standard and how unusual the measurement is with range from -infinity to +infinity; 0: same mean, >0: a greater mean, and <0: a lesser mean than the standard. BMI-for-age z-score was calculated by using centers for disease control and prevention (CDC) growth charts for the pediatric population.
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Timepoint [7]
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Baseline, Week 24
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Secondary outcome [8]
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Time-to-First Pulmonary Exacerbation
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Assessment method [8]
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Time to first pulmonary exacerbation was assessed using Cox Regression method. For participants who completed 24 weeks of treatment, participants without a pulmonary exacerbation before treatment completion were considered censored at the time of treatment completion or at the Week 24 Visit (whichever occurred last). For participants who prematurely discontinued study treatment, participants without a pulmonary exacerbation through the Week 24 Visit were considered censored at the time of the Week 24 Visit.
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Timepoint [8]
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through Week 24
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Secondary outcome [9]
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Percentage of Participants With At Least 1 Pulmonary Exacerbation Event
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Assessment method [9]
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Timepoint [9]
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through Week 24
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Secondary outcome [10]
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Absolute Change From Baseline in Euro Quality of Life Scale (EuroQol) 5-Dimension-3 Level (EQ-5D-3L) Index Score at Week 24
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Assessment method [10]
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EQ-5D-3L: participant rated questionnaire to assess health-related quality of life. It consists of EQ-5D descriptive system and EQ-5D Visual Analog Scale (VAS). EQ-5D-3L descriptive system comprises the following 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: no problems (1), some problems (2), and extreme problems (3). The 5 dimensional 3-level systems are converted into a single index utility score. Values for theoretically possible health states are calculated using a regression model and weighted according to the social preferences of the Unites States (US) general population. For this population, the possible EQ-5D-3L index scores ranges from -0.11 (that is, 3 for all 5 dimensions) to 1.0 (that is, 1 for all 5 dimensions), where higher scores indicate a better health state.
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Timepoint [10]
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Baseline, Week 24
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Secondary outcome [11]
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Absolute Change From Baseline in EQ-5D-3L VAS Score at Week 24
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Assessment method [11]
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The EQ-5D-3L VAS records the participant's self-rated health on a vertical, visual analogue scale where the best state a participant can imagine is marked 100 and the worst state a participant can imagine is marked 0, higher scores indicates a better health state.
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Timepoint [11]
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Baseline, Week 24
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Secondary outcome [12]
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Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domain Scores at Week 24
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Assessment method [12]
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The TSQM is a 14-item self-administered questionnaire which measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed to a scale from 0 to 100, where higher scores indicate greater satisfaction.
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Timepoint [12]
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Baseline, Week 24
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Secondary outcome [13]
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Number of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
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Assessment method [13]
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AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or that was newly developed at or after the initial dosing of study drug to 28 days after the last dose of study drug is considered treatment-emergent.
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Timepoint [13]
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up to Week 28
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Secondary outcome [14]
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Pre-dose Concentration (Ctrough), Average Pre-dose Concentration (Ctrough,Avg), 3 to 6 Hours Post-dose Concentration (C3-6h), and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Avg)
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Assessment method [14]
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Ctrough, Ctrough,avg, C3-6h, and C3-6h,avg for lumacaftor, M28 lumacaftor (lumacaftor metabolite), ivacaftor, M1 ivacaftor (ivacaftor metabolite), and M6 ivacaftor (ivacaftor metabolite) were calculated. C3-6h,avg is average of individual 3 to 6 hours post-dose observed concentrations across Day 15, and Weeks 4 and 8 and Ctrough,avg is average of individual pre-dose observed concentrations across Weeks 4, 8, and 16. This outcome was not planned to be assessed in Placebo arm.
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Timepoint [14]
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For C3-6h: 3 to 6 hours after morning dose on Day 1 and 15, Week 4 and 8; For C3-6h,avg 3 to 6 hours after morning dose on Day 15, Week 4 and 8; For Ctrough and Ctrough,avg: before morning dose on Week 4, 8, and 16
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Eligibility
Key inclusion criteria
- Confirmed diagnosis of CF
- Homozygous for the F508del CFTR mutation
- 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
- Willing to remain on a stable CF medication regimen through Week 24 or, if applicable,
the Safety Follow up Visit
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Minimum age
12
Years
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Maximum age
65
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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
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease within 4 weeks before first dose
of study drug
- History of solid organ or hematological transplantation
- History of alcohol or drug abuse in the past year
- Ongoing or prior participation in an investigational drug study (including studies
investigating lumacaftor and/or ivacaftor) within 30 days of screening.
- Use of strong inhibitors, moderate inducers, or strong inducers of Cytochrome P450 3A
(CYP3A) within 14 days before Day 1 of dosing
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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 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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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
1/04/2013
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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
1/04/2014
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Sample size
Target
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Accrual to date
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Final
563
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Recruitment in Australia
Recruitment state(s)
QLD,WA
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Recruitment hospital [1]
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- Brisbane
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Recruitment hospital [2]
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- Chermside
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- Herston
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- South Brisbane
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- Nedlands
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- Subiaco
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- Brisbane
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- Chermside
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- Herston
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- South Brisbane
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- Nedlands
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Recruitment postcode(s) [6]
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- Subiaco
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Recruitment outside Australia
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California
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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
The primary objective of the study was to evaluate the efficacy of lumacaftor in combination
with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF)
who are homozygous for the F508del mutation on the CF transmembrane conductance regulator
(CFTR) gene.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01807949
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01807949
Download to PDF