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    Clinical Trial Results:
    A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long-Term Ivacaftor Treatment in Subjects 6 Years of Age and Older with Cystic Fibrosis and a Non-G551D CFTR Mutation

    Summary
    EudraCT number
    2012-000389-39
    Trial protocol
    GB   BE  
    Global end of trial date
    18 Apr 2016

    Results information
    Results version number
    v1(current)
    This version publication date
    02 Nov 2016
    First version publication date
    02 Nov 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VX12-770-112
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT01707290
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, Massachusetts, United States, 02210-1862
    Public contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617-341-677, medicalinfo@vrtx.com
    Scientific contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617-341-6777, medicalinfo@vrtx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-000335-PIP01-08
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    30 Aug 2016
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Apr 2016
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety of long-term ivacaftor treatment in subjects with cystic fibrosis (CF).
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Conference on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    13 Feb 2013
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety
    Long term follow-up duration
    2 Years
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 93
    Country: Number of subjects enrolled
    Belgium: 9
    Country: Number of subjects enrolled
    France: 8
    Country: Number of subjects enrolled
    United Kingdom: 15
    Worldwide total number of subjects
    125
    EEA total number of subjects
    32
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    22
    Adolescents (12-17 years)
    12
    Adults (18-64 years)
    89
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    The study consisted of 2 arms: Ivacaftor arm and Observational arm. The Ivacaftor arm enrolled subjects from Study VX11-770-110 (2012-000387-19), Study VX12-770-111 (2012-000388-26) and Study VX12-770-113. The Observational arm enrolled subjects from Study VX11-770-110 (2012-000387-19) and Study VX12-770-111 (2012-000388-26).

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Not applicable
    Blinding used
    Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Ivacaftor arm
    Arm description
    Subjects who received Ivacaftor 150 milligram (mg) tablet and/or Placebo matched to ivacaftor tablet every 12 hours (q12h) in the previous study VX11-770-110 (Study 110; 2012-000387-19), VX12-770-111 (Study 111; 2012-000388-26) or VX12-770-113 (Study 113); received Ivacaftor 150 mg tablet q12h in this VX12-770-112 (Study 112; 2012-000389-39) up to 104 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    Ivacaftor
    Investigational medicinal product code
    VX-770
    Other name
    Kalydeco
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Ivacaftor 150 mg tablet q12h for 104 weeks.

    Arm title
    Observational arm
    Arm description
    Subjects who received Ivacaftor 150 mg tablet and/or Placebo matched to ivacaftor tablet q12h in the previous Study 110 or Study 111, were observed (did not receive study drug) in this Study 112 for up to 2 years.
    Arm type
    No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    Ivacaftor arm Observational arm
    Started
    121
    4
    Completed
    35
    3
    Not completed
    86
    1
         Adverse Event (AE)
    1
    -
         Physician decision
    1
    -
         Withdrawal of Consent (Not Due to AE)
    4
    -
         Commercial drug available
    60
    1
         Other unspecified
    17
    -
         Lost to follow-up
    3
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Ivacaftor arm
    Reporting group description
    Subjects who received Ivacaftor 150 milligram (mg) tablet and/or Placebo matched to ivacaftor tablet every 12 hours (q12h) in the previous study VX11-770-110 (Study 110; 2012-000387-19), VX12-770-111 (Study 111; 2012-000388-26) or VX12-770-113 (Study 113); received Ivacaftor 150 mg tablet q12h in this VX12-770-112 (Study 112; 2012-000389-39) up to 104 weeks.

    Reporting group title
    Observational arm
    Reporting group description
    Subjects who received Ivacaftor 150 mg tablet and/or Placebo matched to ivacaftor tablet q12h in the previous Study 110 or Study 111, were observed (did not receive study drug) in this Study 112 for up to 2 years.

    Reporting group values
    Ivacaftor arm Observational arm Total
    Number of subjects
    121 4 125
    Age categorical
    Units: Subjects
        6 to 11 years
    21 1 22
        12 to 17 years
    11 1 12
        18 years and over
    89 2 91
    Gender categorical
    Units: Subjects
        Female
    61 3 64
        Male
    60 1 61

    End points

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    End points reporting groups
    Reporting group title
    Ivacaftor arm
    Reporting group description
    Subjects who received Ivacaftor 150 milligram (mg) tablet and/or Placebo matched to ivacaftor tablet every 12 hours (q12h) in the previous study VX11-770-110 (Study 110; 2012-000387-19), VX12-770-111 (Study 111; 2012-000388-26) or VX12-770-113 (Study 113); received Ivacaftor 150 mg tablet q12h in this VX12-770-112 (Study 112; 2012-000389-39) up to 104 weeks.

    Reporting group title
    Observational arm
    Reporting group description
    Subjects who received Ivacaftor 150 mg tablet and/or Placebo matched to ivacaftor tablet q12h in the previous Study 110 or Study 111, were observed (did not receive study drug) in this Study 112 for up to 2 years.

    Subject analysis set title
    Ivacaftor arm: Study 110
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Subjects who received Ivacaftor 150 mg tablet and/or Placebo matched to ivacaftor tablet q12h in the previous Study 110; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks.

    Subject analysis set title
    Ivacaftor arm: Study 111
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Subjects who received Ivacaftor 150 mg tablet and/or Placebo matched to ivacaftor tablet q12h in the previous Study 111; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks.

    Subject analysis set title
    Ivacaftor arm: Study 113
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Subjects who received Ivacaftor 150 mg tablet and/or Placebo matched to ivacaftor tablet q12h in the previous Study 113; received Ivacaftor 150 mg tablet q12h in this Study 112 up to 104 weeks.

    Primary: Number of Subjects With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor arm

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    End point title
    Number of Subjects With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor arm [1] [2]
    End point description
    AE: any untoward medical occurrence in a subject 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 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. TEAEs were defined as adverse events with start date or increased severity on and after the first dose of study drug through Week 108. Safety Set included all subjects who received at least 1 dose of study drug (ivacaftor).
    End point type
    Primary
    End point timeframe
    Day 1 up to Week 108
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive statistics were reported, inferential statistics were not planned for primary endpoint.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Only arms which are applicable to the endpoint are reported.
    End point values
    Ivacaftor arm
    Number of subjects analysed
    121
    Units: subjects
    number (not applicable)
        Subjects with AEs
    117
        Subjects with SAEs
    27
    No statistical analyses for this end point

    Secondary: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104

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    End point title
    Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96, and 104
    End point description
    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, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112 (2012-000389-39). Full Analysis Set (FAS) included all subjects who received at least 1 dose of study drug (ivacaftor). Here, "n" signifies those subjects who were evaluable for this measure at the specified time point for each arm, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)
    End point values
    Ivacaftor arm: Study 110 Ivacaftor arm: Study 111 Ivacaftor arm: Study 113
    Number of subjects analysed
    65
    35
    21
    Units: Percent predicted of FEV1
    arithmetic mean (standard deviation)
        Baseline (n= 65, 35, 21)
    71.8 ± 20.4
    78.3 ± 21.1
    62.8 ± 22.2
        Change at Week 2 (n= 65, 35, 21)
    3.8 ± 8
    4.4 ± 7.7
    4 ± 7.2
        Change at Week 12 (n=63, 33, 21)
    5.4 ± 8.9
    5.5 ± 8.3
    4.9 ± 6.7
        Change at Week 24 (n= 62, 34, 21)
    4.5 ± 8.4
    7.1 ± 8.3
    4.6 ± 8.5
        Change at Week 36 (n= 60, 29, 20)
    4.4 ± 7.1
    7.5 ± 8.2
    4.9 ± 8.2
        Change at Week 48 (n= 58, 19, 20)
    4.4 ± 7.5
    6.4 ± 10.7
    4.9 ± 9
        Change at Week 60 (n= 58, 16, 20)
    4.5 ± 8.4
    8.2 ± 8.3
    4.5 ± 8.8
        Change at Week 72 (n= 50, 13, 18)
    3.6 ± 8.9
    7.8 ± 9.2
    5.9 ± 9.2
        Change at Week 84 (n= 40, 11, 16)
    5.4 ± 13.5
    7.7 ± 7.6
    4.8 ± 7.2
        Change at Week 96 (n= 28, 9, 15)
    4.1 ± 14.6
    9.9 ± 6.4
    5.9 ± 8.1
        Change at Week 104 (n= 20, 9, 12)
    4.6 ± 13.2
    4.9 ± 5.8
    6.3 ± 10
    No statistical analyses for this end point

    Secondary: Absolute Change From Baseline in Body Mass Index (BMI) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104

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    End point title
    Absolute Change From Baseline in Body Mass Index (BMI) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104
    End point description
    BMI was defined as weight in kg divided by height in m^2. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112 (2012-000389-39). FAS included all subjects who received at least 1 dose of study drug (ivacaftor). Here, "n" signifies those subjects who were evaluable for this measure at the specified time point for each arm, respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)
    End point values
    Ivacaftor arm: Study 110 Ivacaftor arm: Study 111 Ivacaftor arm: Study 113
    Number of subjects analysed
    65
    35
    21
    Units: kilogram per square meter (kg/m^2)
    arithmetic mean (standard deviation)
        Baseline (n= 65, 35, 21)
    23.75 ± 5.63
    23.15 ± 5.28
    24.81 ± 5.76
        Change at Week 2 (n= 65, 35, 21)
    0.06 ± 0.43
    0.16 ± 0.47
    0.07 ± 0.43
        Change at Week 12 (n= 64, 35, 21)
    0.32 ± 0.59
    0.39 ± 0.62
    0.22 ± 0.6
        Change at Week 24 (n= 62, 35, 21)
    0.43 ± 0.92
    0.65 ± 0.96
    0.17 ± 0.91
        Change at Week 36 (n= 60, 29, 20)
    0.62 ± 1.04
    0.64 ± 1.07
    0.02 ± 1.27
        Change at Week 48 (n=59, 19, 20)
    0.54 ± 1.39
    0.58 ± 1.22
    0.28 ± 1.26
        Change at Week 60 (n= 58, 16, 20)
    0.72 ± 1.32
    0.25 ± 1.09
    0.38 ± 1.22
        Change at Week 72 (n= 51, 13, 17)
    1.05 ± 1.73
    0.45 ± 1.34
    0.22 ± 1.22
        Change at Week 84 (n= 40, 11, 16)
    0.72 ± 1.51
    0.33 ± 1.42
    0.23 ± 0.92
        Change at Week 96 (n= 28, 9, 16)
    0.49 ± 1.44
    0.1 ± 1.49
    0.52 ± 1.28
        Change at Week 104 (n= 20, 9, 12)
    0.42 ± 1.49
    0.16 ± 1.37
    0.88 ± 1.39
    No statistical analyses for this end point

    Secondary: Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104

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    End point title
    Absolute Change From Baseline in Sweat Chloride at Week 2, 24, 48 and 104
    End point description
    Sweat samples were collected using an approved collection device. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112 (2012-000389-39). FAS included all subjects who received at least 1 dose of study drug (ivacaftor). Here, "Number of subjects analysed" signifies those subjects who were evaluable for this outcome and "n" signifies those subjects who were evaluable for this measure at the specified time point for each arm respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 2, 24, 48 and 104 (Study 112)
    End point values
    Ivacaftor arm: Study 110 Ivacaftor arm: Study 111 Ivacaftor arm: Study 113
    Number of subjects analysed
    59
    33
    21
    Units: millimole per liter (mmol/L)
    arithmetic mean (standard deviation)
        Baseline (n=59, 33, 21)
    60.9 ± 19.4
    80.2 ± 22.8
    55.7 ± 22.2
        Change at Week 2 (n=59, 31, 21)
    -19.3 ± 10.7
    -38.4 ± 27.5
    -4.5 ± 10.9
        Change at Week 24 (n=56, 33, 20)
    -13 ± 18.4
    -39.2 ± 27
    1.1 ± 15.8
        Change at Week 48 (n=50, 19, 19)
    -13.5 ± 16.2
    -40.6 ± 26.1
    -1.9 ± 17.8
        Change at Week 104 (n=15, 9, 11)
    -13.7 ± 22.9
    -32.9 ± 26.8
    5.4 ± 18.1
    No statistical analyses for this end point

    Secondary: Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104

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    End point title
    Absolute Change From Baseline in Respiratory Domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R) at Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104
    End point description
    The CFQ-R is a validated subject reported outcome measuring health related quality of life for subjects with CF. 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. Baseline was defined as the most recent measurement before intake of the first dose of study drug (ivacaftor) in Study 112 (2012-000389-39). FAS included all subjects who received at least 1 dose of study drug (ivacaftor). Here, "n" signifies those subjects who were evaluable for this measure at the specified time point for each arm respectively.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 2, 12, 24, 36, 48, 60, 72, 84, 96 and 104 (Study 112)
    End point values
    Ivacaftor arm: Study 110 Ivacaftor arm: Study 111 Ivacaftor arm: Study 113
    Number of subjects analysed
    65
    35
    21
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline (n= 65, 35, 21)
    68.6 ± 21.6
    72.4 ± 20.3
    65.9 ± 18.8
        Change at Week 2 (n= 65, 35, 21)
    5.5 ± 17.9
    4.5 ± 17.5
    13.2 ± 16.2
        Change at Week 12 (n= 64, 35, 21)
    11.6 ± 18
    5.7 ± 18.5
    13.5 ± 20
        Change at Week 24 (n= 62, 35, 21)
    6.7 ± 19
    3.4 ± 18.7
    8.5 ± 14.9
        Change at Week 36 (n=60, 29, 20)
    12.1 ± 19.3
    5.7 ± 13.8
    7.9 ± 20.5
        Change at Week 48 (n= 59, 19, 20)
    9.5 ± 17.6
    4.4 ± 13.2
    11.4 ± 15.7
        Change at Week 60 (n= 57, 15, 20)
    7.1 ± 18.6
    4.4 ± 10.9
    8.6 ± 19.3
        Change at Week 72 (n= 51, 13, 18)
    10.4 ± 22.2
    5.6 ± 11.8
    8.6 ± 19
        Change at Week 84 (n= 40, 11, 16)
    10.5 ± 19.5
    8.8 ± 14.1
    4.9 ± 14.6
        Change at Week 96 (n= 28, 9, 16)
    8.8 ± 21.1
    0 ± 9.5
    9.4 ± 14.7
        Change at Week 104 (n= 20, 9, 12)
    9.2 ± 14
    1.9 ± 8.8
    19.9 ± 16
    No statistical analyses for this end point

    Secondary: Number of Pulmonary Exacerbations Events

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    End point title
    Number of Pulmonary Exacerbations Events
    End point description
    Pulmonary exacerbation events include those events which require 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. FAS included all subjects who received at least 1 dose of study drug (ivacaftor).
    End point type
    Secondary
    End point timeframe
    Through Week 104
    End point values
    Ivacaftor arm: Study 110 Ivacaftor arm: Study 111 Ivacaftor arm: Study 113
    Number of subjects analysed
    65
    35
    21
    Units: pulmonary exacerbation events
        number (not applicable)
    47
    30
    6
    No statistical analyses for this end point

    Secondary: Number of Subjects With SAEs in Observational arm

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    End point title
    Number of Subjects With SAEs in Observational arm [3]
    End point description
    SAE was defined as a 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. Analysis population included all subjects who were included in the observational arm.
    End point type
    Secondary
    End point timeframe
    up to 2 years
    Notes
    [3] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Only arms which are applicable to the endpoint are reported.
    End point values
    Observational arm
    Number of subjects analysed
    4
    Units: subjects
        number (not applicable)
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 up to Week 108 for Ivacaftor arm; up to 2 years for Observational arm
    Adverse event reporting additional description
    Non-SAEs were not collected for Observational arm. Only SAEs were planned to be collected for the Observational arm.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    15.1
    Reporting groups
    Reporting group title
    Ivacaftor arm
    Reporting group description
    Subjects who received Ivacaftor 150 mg tablet and/or Placebo matched to ivacaftor tablet q12h in the previous study 110 (2012-000387-19) or study 111 (2012-000388-26) or study 113, received Ivacaftor 150 mg tablet q12h in this study 112 (2012-000389-39) up to 104 weeks.

    Reporting group title
    Observational arm
    Reporting group description
    Subjects who received Ivacaftor 150 mg tablet and/or Placebo matched to ivacaftor tablet q12h in the previous study 110 (2012-000387-19) or study 111 (2012-000388-26), were observed (did not receive study drug) in this study 112 (2012-000389-39) for up to 2 years.

    Serious adverse events
    Ivacaftor arm Observational arm
    Total subjects affected by serious adverse events
         subjects affected / exposed
    27 / 121 (22.31%)
    1 / 4 (25.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Investigations
    Influenza A virus test positive
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiomyopathy acute
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Haemoptysis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory failure
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Crohn's disease
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Flatulence
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Renal failure acute
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Angioedema
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Urticaria
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    21 / 121 (17.36%)
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    1 / 34
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis viral
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pharyngitis bacterial
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sinusitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences causally related to treatment / all
    1 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Ivacaftor arm Observational arm
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    117 / 121 (96.69%)
    0 / 4 (0.00%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Hot flush
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Orthostatic hypotension
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pallor
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Seborrhoeic keratosis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Skin papilloma
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Thyroid neoplasm
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Allergy to arthropod bite
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    17 / 121 (14.05%)
    0 / 4 (0.00%)
         occurrences all number
    25
    0
    Fatigue
         subjects affected / exposed
    12 / 121 (9.92%)
    0 / 4 (0.00%)
         occurrences all number
    15
    0
    Chest pain
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Influenza like illness
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Chills
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Feeling of body temperature change
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Oedema peripheral
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Pain
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Asthenia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Device leakage
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Implant site rash
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Local swelling
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vaccination site pain
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    6 / 121 (4.96%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    Insomnia
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Depression
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Abnormal dreams
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Panic attack
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Tobacco abuse
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Drug dependence
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Reproductive system and breast disorders
    Amenorrhoea
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Breast pain
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Dysmenorrhoea
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Menorrhagia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Testicular torsion
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vulvovaginal discomfort
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vulvovaginal pruritus
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Injury, poisoning and procedural complications
    Procedural pain
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Hand fracture
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Contusion
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Laceration
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Muscle strain
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Arthropod bite
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Arthropod sting
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Concussion
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Epicondylitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Fall
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Humerus fracture
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Joint dislocation
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Ligament rupture
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Ligament sprain
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Snake bite
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Skeletal injury
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Soft tissue injury
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Tooth fracture
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Investigations
    Bacterial test positive
         subjects affected / exposed
    7 / 121 (5.79%)
    0 / 4 (0.00%)
         occurrences all number
    7
    0
    Forced expiratory volume decreased
         subjects affected / exposed
    6 / 121 (4.96%)
    0 / 4 (0.00%)
         occurrences all number
    7
    0
    Weight decreased
         subjects affected / exposed
    5 / 121 (4.13%)
    0 / 4 (0.00%)
         occurrences all number
    5
    0
    C-reactive protein increased
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Haemophilus test positive
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Liver function test abnormal
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Alanine aminotransferase increased
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Atypical mycobacterium test positive
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Blood pressure increased
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Body temperature increased
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Hepatic enzyme increased
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Pseudomonas test positive
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Vitamin D decreased
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Weight increased
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Blood bilirubin increased
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Blood calcium decreased
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Blood creatinine increased
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Blood glucose increased
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Blood urea increased
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Crystal urine present
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Electrocardiogram QT prolonged
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Electrocardiogram abnormal
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Influenza A virus test positive
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Flavobacterium test positive
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Klebsiella test positive
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Streptococcus test positive
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Ultrasound biliary tract abnormal
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Urinary sediment present
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Urine analysis abnormal
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vitamin A decreased
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    White blood cells urine positive
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pulmonary function test decreased
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    41 / 121 (33.88%)
    0 / 4 (0.00%)
         occurrences all number
    81
    0
    Sputum increased
         subjects affected / exposed
    22 / 121 (18.18%)
    0 / 4 (0.00%)
         occurrences all number
    27
    0
    Sinus congestion
         subjects affected / exposed
    22 / 121 (18.18%)
    0 / 4 (0.00%)
         occurrences all number
    34
    0
    Oropharyngeal pain
         subjects affected / exposed
    18 / 121 (14.88%)
    0 / 4 (0.00%)
         occurrences all number
    24
    0
    Nasal congestion
         subjects affected / exposed
    17 / 121 (14.05%)
    0 / 4 (0.00%)
         occurrences all number
    20
    0
    Dyspnoea
         subjects affected / exposed
    12 / 121 (9.92%)
    0 / 4 (0.00%)
         occurrences all number
    14
    0
    Respiratory tract congestion
         subjects affected / exposed
    9 / 121 (7.44%)
    0 / 4 (0.00%)
         occurrences all number
    12
    0
    Haemoptysis
         subjects affected / exposed
    12 / 121 (9.92%)
    0 / 4 (0.00%)
         occurrences all number
    25
    0
    Wheezing
         subjects affected / exposed
    9 / 121 (7.44%)
    0 / 4 (0.00%)
         occurrences all number
    10
    0
    Rales
         subjects affected / exposed
    7 / 121 (5.79%)
    0 / 4 (0.00%)
         occurrences all number
    8
    0
    Rhinorrhoea
         subjects affected / exposed
    6 / 121 (4.96%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    Paranasal sinus hypersecretion
         subjects affected / exposed
    5 / 121 (4.13%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    Pleuritic pain
         subjects affected / exposed
    5 / 121 (4.13%)
    0 / 4 (0.00%)
         occurrences all number
    9
    0
    Respiration abnormal
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    7
    0
    Nasal polyps
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    5
    0
    Throat irritation
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Dysphonia
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Nasal mucosal disorder
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Productive cough
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    9
    0
    Upper-airway cough syndrome
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Asthma
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Dyspnoea exertional
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Epistaxis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Increased viscosity of bronchial secretion
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Lung hyperinflation
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Nasal inflammation
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Nasal oedema
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Painful respiration
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pleurisy
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Prolonged expiration
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Respiratory tract irritation
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Rhinitis allergic
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Sinus polyp
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Sneezing
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Sputum discoloured
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Throat tightness
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    23 / 121 (19.01%)
    0 / 4 (0.00%)
         occurrences all number
    38
    0
    Sinus headache
         subjects affected / exposed
    9 / 121 (7.44%)
    0 / 4 (0.00%)
         occurrences all number
    11
    0
    Dizziness
         subjects affected / exposed
    7 / 121 (5.79%)
    0 / 4 (0.00%)
         occurrences all number
    8
    0
    Migraine
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Hypoaesthesia
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Burning sensation
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Carpal tunnel syndrome
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Hyperaesthesia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Hypersomnia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Eye disorders
    Conjunctivitis
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Blepharospasm
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Conjunctival haemorrhage
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Conjunctivitis allergic
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Eye swelling
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Photophobia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vision blurred
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Visual impairment
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vitreous floaters
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    5
    0
    Tinnitus
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Middle ear effusion
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Otorrhoea
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vertigo
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    17 / 121 (14.05%)
    0 / 4 (0.00%)
         occurrences all number
    24
    0
    Diarrhoea
         subjects affected / exposed
    17 / 121 (14.05%)
    0 / 4 (0.00%)
         occurrences all number
    25
    0
    Abdominal pain
         subjects affected / exposed
    12 / 121 (9.92%)
    0 / 4 (0.00%)
         occurrences all number
    18
    0
    Vomiting
         subjects affected / exposed
    12 / 121 (9.92%)
    0 / 4 (0.00%)
         occurrences all number
    13
    0
    Nausea
         subjects affected / exposed
    9 / 121 (7.44%)
    0 / 4 (0.00%)
         occurrences all number
    10
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    7 / 121 (5.79%)
    0 / 4 (0.00%)
         occurrences all number
    7
    0
    Abdominal pain upper
         subjects affected / exposed
    6 / 121 (4.96%)
    0 / 4 (0.00%)
         occurrences all number
    9
    0
    Abdominal distension
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Tooth impacted
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Abdominal discomfort
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Dental caries
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Flatulence
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Steatorrhoea
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Toothache
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Abdominal pain lower
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Barrett's oesophagus
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Colitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Defaecation urgency
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Dental discomfort
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Dyspepsia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Frequent bowel movements
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Gastritis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Palatal disorder
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pancreatitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Proctitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Rectal haemorrhage
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Rectal polyp
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Stomatitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Tooth loss
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Food poisoning
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Nephrolithiasis
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Bile duct stone
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    6 / 121 (4.96%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    Eczema
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Rash erythematous
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Dermatitis contact
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Hyperhidrosis
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Eczema nummular
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Erythema
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Ingrowing nail
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Night sweats
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pruritus
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Psoriasis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Rash generalised
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Rash pruritic
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Skin lesion
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Rosacea
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Subcutaneous nodule
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Acne
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    9 / 121 (7.44%)
    0 / 4 (0.00%)
         occurrences all number
    11
    0
    Back pain
         subjects affected / exposed
    6 / 121 (4.96%)
    0 / 4 (0.00%)
         occurrences all number
    7
    0
    Musculoskeletal pain
         subjects affected / exposed
    5 / 121 (4.13%)
    0 / 4 (0.00%)
         occurrences all number
    11
    0
    Muscle spasms
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Osteopenia
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Pain in extremity
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Myalgia
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Arthritis
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Intervertebral disc protrusion
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Bone pain
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Clubbing
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Joint swelling
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Metatarsalgia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Monarthritis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Musculoskeletal chest pain
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Neck pain
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pain in jaw
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Rotator cuff syndrome
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Tendonitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Vitamin D deficiency
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Dehydration
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Diabetes mellitus
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Dyslipidaemia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Fluid retention
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Impaired fasting glucose
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Hypokalaemia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Increased appetite
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Type 2 diabetes mellitus
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vitamin A deficiency
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    49 / 121 (40.50%)
    0 / 4 (0.00%)
         occurrences all number
    85
    0
    Nasopharyngitis
         subjects affected / exposed
    21 / 121 (17.36%)
    0 / 4 (0.00%)
         occurrences all number
    32
    0
    Sinusitis
         subjects affected / exposed
    18 / 121 (14.88%)
    0 / 4 (0.00%)
         occurrences all number
    35
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    17 / 121 (14.05%)
    0 / 4 (0.00%)
         occurrences all number
    23
    0
    Upper respiratory tract infection
         subjects affected / exposed
    15 / 121 (12.40%)
    0 / 4 (0.00%)
         occurrences all number
    19
    0
    Gastroenteritis viral
         subjects affected / exposed
    9 / 121 (7.44%)
    0 / 4 (0.00%)
         occurrences all number
    9
    0
    Oral candidiasis
         subjects affected / exposed
    8 / 121 (6.61%)
    0 / 4 (0.00%)
         occurrences all number
    13
    0
    Influenza
         subjects affected / exposed
    7 / 121 (5.79%)
    0 / 4 (0.00%)
         occurrences all number
    9
    0
    Gastroenteritis
         subjects affected / exposed
    5 / 121 (4.13%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    Lower respiratory tract infection
         subjects affected / exposed
    5 / 121 (4.13%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    Acute sinusitis
         subjects affected / exposed
    4 / 121 (3.31%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    Vulvovaginal mycotic infection
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    6
    0
    Bacterial disease carrier
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Lower respiratory tract infection bacterial
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Pharyngitis streptococcal
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Respiratory tract infection viral
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Rhinitis
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Tonsillitis
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    3
    0
    Upper respiratory tract infection bacterial
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Urinary tract infection
         subjects affected / exposed
    3 / 121 (2.48%)
    0 / 4 (0.00%)
         occurrences all number
    4
    0
    Bronchopulmonary aspergillosis allergic
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Laryngitis
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Otitis externa
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Pharyngitis bacterial
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Viral infection
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Chronic sinusitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Clostridium difficile colitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Croup infectious
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Diarrhoea infectious
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Ear lobe infection
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Folliculitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Furuncle
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Haemophilus infection
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Herpes zoster
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Infected dermal cyst
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Laryngitis viral
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Lung infection
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Molluscum contagiosum
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Myringitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Nasal abscess
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Oesophagitis bacterial
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Oral herpes
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0
    Otitis media
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Respiratory tract infection bacterial
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pneumonia
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Staphylococcal infection
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Staphylococcal skin infection
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Tooth abscess
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Tooth infection
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vaginitis bacterial
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Viral pharyngitis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Vulvovaginal candidiasis
         subjects affected / exposed
    1 / 121 (0.83%)
    0 / 4 (0.00%)
         occurrences all number
    1
    0
    Pharyngitis
         subjects affected / exposed
    2 / 121 (1.65%)
    0 / 4 (0.00%)
         occurrences all number
    2
    0

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 Mar 2012
    Clarified sample collection, statistical analysis plan and Early Termination visit details; updated follow-up timeframe; corrected pharmacogenomic analysis text.
    05 Dec 2012
    Extended study duration to 2 years; secondary objective was changed to evaluate the post-treatment safety of ivacaftor in subjects enrolled in the observational arm; pharmacokinetic (PK) blood sample collection was added to the ivacaftor arm; ophthalmologic examination was added to the safety endpoints and additional ophthalmologic examinations were added; contraceptive requirements were clarified; included a planned interim analysis for safety and efficacy data after all subjects completed Week 24 Visit.
    01 Apr 2013
    Subjects from Study 113 were offered enrollment in the ivacaftor arm of Study 112; clarified timing of Day 1 Visit in Study 112 with respect to Study 113; clarified that PK blood samples would not be collected from Study 113 subjects.
    29 Jul 2013
    Clarified that subjects from Study 113 must have completed the Study 113 Follow-up Visit and met responder criteria for eligibility to enroll in the Study 112 ivacaftor arm.
    13 Feb 2014
    Pregnancy language was revised; changed protocol to allow subjects to discontinue the study when Kalydeco was commercially-available and reimbursed for that indication in the subject’s country; added optional exploratory hypertonic saline substudy; changed the timing of the planned interim analysis.
    29 Jul 2014
    Added an optional exploratory substudy for subjects with the G970R-CFTR Mutation.
    08 Dec 2014
    Made ophthalmologic examination assessments applicable to all subjects.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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