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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02514473
Registration number
NCT02514473
Ethics application status
Date submitted
23/07/2015
Date registered
3/08/2015
Date last updated
23/10/2017
Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation
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Scientific title
A Phase 3, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
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Secondary ID [1]
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VX14-809-109
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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
Experimental: LUM/IVA - Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h
Placebo comparator: Placebo - Matching placebo q12h
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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 Lung Clearance Index 2.5 (LCI2.5) Through Week 24
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Assessment method [1]
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Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
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Timepoint [1]
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Baseline, Through Week 24
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Secondary outcome [1]
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Average Absolute Change From Baseline in Sweat Chloride at Day 15 and Week 4
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Assessment method [1]
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Sweat samples were collected using an approved collection device. Baseline was defined as the average of the measurements at screening and on Day 1 pre-dose. Change from Baseline in sweat chloride at Day 15 and Week 4 was calculated. The average of the 2 values (Change at Day 15 and Week 4) was reported.
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Timepoint [1]
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Baseline, Day 15 and Week 4
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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 kg divided by 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 Through 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), 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, Through Week 24
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Secondary outcome [4]
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Absolute Change From Baseline in Lung Clearance Index 5.0 (LCI5.0) Through Week 24
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Assessment method [4]
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LCI is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value.
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Timepoint [4]
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Baseline, Through Week 24
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Secondary outcome [5]
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Absolute Change From Baseline in Sweat Chloride at Week 24
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Assessment method [5]
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Sweat samples were collected using an approved collection device.
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Timepoint [5]
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Baseline, Week 24
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Secondary outcome [6]
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Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24
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Assessment method [6]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Wang standards were used to calculate ppFEV1 (for age, gender, race, and height).
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Timepoint [6]
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Baseline, Through Week 24
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Secondary outcome [7]
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Relative Change From Baseline in ppFEV1 Through Week 24
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Assessment method [7]
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FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Wang standards were used to calculate ppFEV1 (for age, gender, race, and height).
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Timepoint [7]
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Baseline, Through Week 24
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Secondary outcome [8]
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Absolute Change From Baseline in BMI-for-age Z-score at Week 24
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Assessment method [8]
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BMI was defined as weight in kg divided by height in m\^2. 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. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score (BMI z-score). The BMI-for-age z-scores were calculated using National Center for Health Statistics growth charts.
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Timepoint [8]
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Baseline, Week 24
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Secondary outcome [9]
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Absolute Change From Baseline in Weight at Week 24
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Assessment method [9]
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Timepoint [9]
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Baseline, Week 24
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Secondary outcome [10]
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Absolute Change From Baseline in Weight-for-age Z-score at Week 24
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Assessment method [10]
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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. Weight, adjusted for age and sex, was analyzed as weight-for-age z-score (weight z-score). The weight-for-age z-scores were calculated using National Center for Health Statistics growth charts.
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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 Height at Week 24
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Assessment method [11]
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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 Height-for-age Z-score at Week 24
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Assessment method [12]
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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. Height, adjusted for age and sex, was analyzed as height-for-age z-score (height z-score). The height-for-age z-scores were calculated using National Center for Health Statistics growth charts.
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Timepoint [12]
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Baseline, Week 24
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Secondary outcome [13]
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Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domains Through Week 24
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Assessment method [13]
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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 [13]
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Baseline, Through Week 24
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Secondary outcome [14]
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Number of Pulmonary Exacerbation Events
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Assessment method [14]
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Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. The number of events were reported.
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Timepoint [14]
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Baseline through Week 24
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Secondary outcome [15]
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Percentage of Participants With At Least 1 Pulmonary Exacerbation Event
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Assessment method [15]
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Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
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Timepoint [15]
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Baseline through Week 24
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Secondary outcome [16]
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Time-to-first Pulmonary Exacerbation
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Assessment method [16]
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Time-to-first pulmonary exacerbation was analyzed using the Kaplan-Meier estimates. Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
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Timepoint [16]
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Baseline through Week 24
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Secondary outcome [17]
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Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [17]
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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, inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or newly developed at or after initial dosing of study drug to Week 28 was considered treatment-emergent.
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Timepoint [17]
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Baseline up to Week 28
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Secondary outcome [18]
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Average Pre-dose Concentration (Ctrough,Ave) and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Ave) For Lumacaftor and Ivacaftor
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Assessment method [18]
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Ctrough,ave is average of individual pre-dose observed concentrations across Week 4 and 24. C3-6h,ave is average of individual 3 to 6 hours post-dose observed concentrations across Day 1, 15 and Week 4. This outcome was not planned to be assessed in Placebo arm.
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Timepoint [18]
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For Ctrough,ave: before morning dose on Week 4 and 24; For C3-6h,ave: 3 to 6 hours after morning dose on Day 1, 15 and Week 4
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Eligibility
Key inclusion criteria
* Subjects who weigh =15 kg without shoes a the Screening Visit
* Subjects with confirmed diagnosis of CF at the Screening Visit.
* Subjects who are homozygous for the F508del CFTR mutation
* Subjects with ppFEV1 of =70 percentage points adjusted for age, sex, and height
* Subjects with a screening LCI2.5 result greater than or equal to 7.5
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Minimum age
6
Years
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Maximum age
11
Years
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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 reviewed at the Screening Visit 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.
* Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
* Clinically significant abnormalities in hemoglobin, liver function, or renal function at the Screening Visit.
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
* History of solid organ or hematological transplantation at the Screening Visit
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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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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
1/07/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/09/2016
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Sample size
Target
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Accrual to date
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Final
206
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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- Herston
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Recruitment hospital [2]
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- New South Wales
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Recruitment hospital [3]
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- Parkville
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Recruitment hospital [4]
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- Subiaco
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Recruitment hospital [5]
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- Westmead
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Recruitment postcode(s) [1]
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- Herston
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Recruitment postcode(s) [2]
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- New South Wales
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Recruitment postcode(s) [3]
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- Parkville
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Recruitment postcode(s) [4]
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- Subiaco
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Recruitment postcode(s) [5]
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- Westmead
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Recruitment outside Australia
Country [1]
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United States of America
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Alabama
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California
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Colorado
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Delaware
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Florida
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Georgia
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Illinois
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Iowa
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Massachusetts
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Minnesota
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Missouri
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Nebraska
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Ohio
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Vermont
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Wisconsin
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Leuven
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Ontario
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Copenhagen
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Bordeaux
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France
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Bron
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France
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Paris Cedex 15
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France
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Paris
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Berlin
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Germany
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Giessen
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Germany
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Hannover
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Germany
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Koeln
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Lothian Region
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West Yorkshire
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Belfast
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London
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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
To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation
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Trial website
https://clinicaltrials.gov/study/NCT02514473
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Trial related presentations / publications
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. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3. Ratjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic S, Milla CE, Robinson PD, Waltz D, Davies JC; VX14-809-109 investigator group. Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. Lancet Respir Med. 2017 Jul;5(7):557-567. doi: 10.1016/S2213-2600(17)30215-1. Epub 2017 Jun 9. Erratum In: Lancet Respir Med. 2017 Aug;5(8):e28. doi: 10.1016/S2213-2600(17)30270-9.
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Public notes
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Contacts
Principal investigator
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Contact person for public queries
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02514473
Download to PDF