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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/NCT02544451
Registration number
NCT02544451
Ethics application status
Date submitted
1/09/2015
Date registered
9/09/2015
Date last updated
24/05/2021
Titles & IDs
Public title
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
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Scientific title
A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
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Secondary ID [1]
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VX15-809-110
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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 - LUM/IVA
Treatment: Drugs - LUM/IVA
Experimental: Treatment Period 1: LUM/IVA to LUM/IVA -
Experimental: Treatment Period 1: Placebo (PBO) to LUM/IVA -
No Intervention: Treatment Period 1: Observational Cohort -
Experimental: Treatment Period 2: LUM/IVA -
Treatment: Drugs: LUM/IVA
Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age).
LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older).
Treatment: Drugs: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
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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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Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [1]
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Timepoint [1]
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Day 1 up to Week 100
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Secondary outcome [1]
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Absolute Change in Lung Clearance Index (LCI) 2.5
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Assessment method [1]
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LCI 2.5 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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From Parent Study Baseline at Week 96
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Secondary outcome [2]
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Absolute Change in Sweat Chloride
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Assessment method [2]
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Sweat samples were collected using an approved collection device.
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Timepoint [2]
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From Parent Study Baseline at Week 96
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Secondary outcome [3]
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Absolute Change in Body Mass Index (BMI)
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Assessment method [3]
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BMI was defined as weight in kilograms divided by height in square meter (m^2).
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Timepoint [3]
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From Parent Study Baseline at Week 96
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Secondary outcome [4]
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Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
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Assessment method [4]
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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 [4]
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From Parent Study Baseline at Week 96
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Secondary outcome [5]
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Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)
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Assessment method [5]
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Timepoint [5]
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Day 1 up to Week 100
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Secondary outcome [6]
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Absolute Change in LCI 5.0
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Assessment method [6]
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LCI 5.0 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 [6]
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From Parent Study Baseline at Week 96
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Secondary outcome [7]
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Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
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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.
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Timepoint [7]
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From Parent Study Baseline at Week 96
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Secondary outcome [8]
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Relative Change in ppFEV1
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Assessment method [8]
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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 [8]
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From Parent Study Baseline at Week 96
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Secondary outcome [9]
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Absolute Change in BMI-for-age Z-score
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Assessment method [9]
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BMI was defined as weight in kilograms divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
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Timepoint [9]
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From Parent Study Baseline at Week 96
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Secondary outcome [10]
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Absolute Change in Weight
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Assessment method [10]
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Timepoint [10]
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From Parent Study Baseline at Week 96
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Secondary outcome [11]
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Absolute Change in Weight-for-age Z-score
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Assessment method [11]
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z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
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Timepoint [11]
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From Parent Study Baseline at Week 96
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Secondary outcome [12]
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Absolute Change in Height
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Assessment method [12]
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Timepoint [12]
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From Parent Study Baseline at Week 96
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Secondary outcome [13]
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Absolute Change in Height-for-age Z-score
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Assessment method [13]
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z-score is a statistical measure to describe whether a mean was above or below the standard. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean.
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Timepoint [13]
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From Parent Study Baseline at Week 96
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Secondary outcome [14]
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Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score
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Assessment method [14]
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The TSQM measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed in the total domain score, which ranges from 0 to 100, where higher scores indicate greater satisfaction.
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Timepoint [14]
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From Parent Study Baseline at Week 96
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Secondary outcome [15]
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Time-to-first Pulmonary Exacerbation
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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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From Parent Study Baseline through Week 96
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Secondary outcome [16]
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Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event
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Assessment method [16]
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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 [16]
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From Parent Study Baseline through Week 96
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Secondary outcome [17]
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Number of Pulmonary Exacerbation Events Per Patient-year
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Assessment method [17]
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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 [17]
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From Parent Study Baseline through Week 96
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Secondary outcome [18]
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Rate of Change in LCI 2.5
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Assessment method [18]
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Rate of change analysis evaluates the change in LCI 2.5 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
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Timepoint [18]
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Day 15 after first dose of LUM/IVA through Week 96
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Secondary outcome [19]
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Rate of Change in LCI 5.0
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Assessment method [19]
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Rate of change analysis evaluates the change in LCI 5.0 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
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Timepoint [19]
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Day 15 after first dose of LUM/IVA through Week 96
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Secondary outcome [20]
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Rate of Change in ppFEV1
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Assessment method [20]
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Rate of change analysis evaluates the change in ppFEV1 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable.
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Timepoint [20]
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Day 15 after first dose of LUM/IVA through Week 96
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Secondary outcome [21]
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Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
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Assessment method [21]
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Timepoint [21]
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Day 1 up to Week 168
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Eligibility
Key inclusion criteria
Subjects entering the Treatment Cohort must meet both of the following criteria:
- Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not
permanently discontinue treatment
- Willing to remain on a stable CF medication through the Safety Follow-up Visit.
Subjects entering the Observational Cohort must meet 1 of the following criteria:
- Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study
drug treatment and the Week 26 Safety Follow up in Study 011B.
- Received at least 4 weeks of study drug and completed visits up to Week 24 of Study
109 or Week 26 of Study 011B.
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Minimum age
6
Years
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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
Exclusion Criteria (Treatment Cohort Only):
- History of any comorbidity or laboratory abnormality 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 (e.g., cirrhosis with portal hypertension).
- Pregnant and nursing females.
- Sexually active subjects of reproductive potential who are not willing to follow the
contraception requirements.
- History of drug intolerance in the prior study that would pose an additional risk to
the subject in the opinion of investigator
- History of poor compliance with study drug and/or procedure in the previous study as
deemed by the investigator.
- Participation in an investigational drug trial (including studies investigating
lumacaftor and/or ivacaftor).
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised 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
Open (masking not used)
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Who is / are masked / blinded?
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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
1/08/2015
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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
1/04/2020
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Sample size
Target
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Accrual to date
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Final
246
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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- Parkville
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Recruitment hospital [2]
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- Herston
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Recruitment hospital [3]
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- New Lambton Heights
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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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- Parkville
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Recruitment postcode(s) [2]
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- Herston
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Recruitment postcode(s) [3]
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- New Lambton Heights
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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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State/province [1]
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Alabama
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United States of America
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Arizona
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Delaware
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United States of America
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Florida
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United States of America
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Georgia
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Illinois
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Indiana
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Iowa
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Massachusetts
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United States of America
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Minnesota
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Missouri
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Nebraska
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Country [15]
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United States of America
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New Hampshire
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Country [16]
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United States of America
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New York
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North Carolina
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Ohio
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South Carolina
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Texas
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Utah
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Vermont
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Virginia
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Washington
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Wisconsin
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Belgium
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Brussels
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Belgium
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Leuven
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Canada
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British Columbia
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Canada
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Ontario
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Canada
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Quebec
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Country [33]
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Denmark
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Copenhagen
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Country [34]
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France
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Cedex
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Country [35]
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France
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Bordeaux Cedex
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Country [36]
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France
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Paris Cedex 15
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Country [37]
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France
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Paris
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Germany
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Berlin
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Germany
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Giessen
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Germany
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Hanover
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Germany
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Koeln
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Sweden
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Stockholm
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United Kingdom
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West Yorkshire
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United Kingdom
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Belfast
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United Kingdom
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Edinburgh
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United Kingdom
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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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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic
fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator
(CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233).
Study 110 is designed to evaluate the safety and efficacy of long term treatment of
lumacaftor in combination with ivacaftor.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02544451
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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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Address
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Country
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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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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02544451
Download to PDF