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    Clinical Trial Results:
    A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

    Summary
    EudraCT number
    2016-001004-33
    Trial protocol
    Outside EU/EEA  
    Global end of trial date
    08 Sep 2017

    Results information
    Results version number
    v1(current)
    This version publication date
    07 Jun 2018
    First version publication date
    07 Jun 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VX15-809-115
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT02797132
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, Massachusetts, United States, 022101862
    Public contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, 1 617-341-6777, 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-001582-PIP01-13
    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
    29 Sep 2017
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    08 Sep 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Sep 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the pharmacokinetics (PK) and safety of lumacaftor (LUM) and ivacaftor (IVA) combination therapy in subjects aged 2 through 5 years with cystic fibrosis (CF) homozygous for F508del
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Council on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    13 May 2016
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 7
    Country: Number of subjects enrolled
    United States: 55
    Worldwide total number of subjects
    62
    EEA total number of subjects
    0
    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)
    62
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    -

    Pre-assignment
    Screening details
    The study was conducted in 2 parts, Parts A and B. In Parts A and B, subjects received LUM/IVA based on weight. Subjects in Part A could have participated in Part B.

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

    Arms
    Are arms mutually exclusive
    No

    Arm title
    Part A: LUM 100 mg/IVA 125 mg
    Arm description
    Subjects weighing less than (<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination for 15 days.
    Arm type
    Experimental

    Investigational medicinal product name
    LUM/IVA fixed-dose combination
    Investigational medicinal product code
    VX-809+VX-770
    Other name
    Pharmaceutical forms
    Granules in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Administered orally every 12 hours for 15 days.

    Arm title
    Part A: LUM 150 mg/IVA 188 mg
    Arm description
    Subjects weighing greater than or equal to (>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination for 15 days.
    Arm type
    Experimental

    Investigational medicinal product name
    LUM/IVA fixed-dose combination
    Investigational medicinal product code
    VX-809+VX-770
    Other name
    Pharmaceutical forms
    Granules in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Administered orally every 12 hours for 15 days.

    Arm title
    Part B: LUM 100 mg/IVA 125 mg
    Arm description
    Subjects weighing less than <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination for 24 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    LUM/IVA fixed-dose combination
    Investigational medicinal product code
    VX-809+VX-770
    Other name
    Pharmaceutical forms
    Granules in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Administered orally every 12 hours for 24 weeks.

    Arm title
    Part B: LUM 150 mg/IVA 188 mg
    Arm description
    Subjects weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination for 24 weeks.
    Arm type
    Experimental

    Investigational medicinal product name
    LUM/IVA fixed-dose combination
    Investigational medicinal product code
    VX-809+VX-770
    Other name
    Pharmaceutical forms
    Granules in sachet
    Routes of administration
    Oral use
    Dosage and administration details
    Administered orally every 12 hours 24 weeks.

    Number of subjects in period 1
    Part A: LUM 100 mg/IVA 125 mg Part A: LUM 150 mg/IVA 188 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg
    Started
    4
    8
    19
    41
    Completed
    4
    7
    19
    38
    Not completed
    0
    1
    0
    3
         Adverse Event
    -
    1
    -
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Part A: LUM 100 mg/IVA 125 mg
    Reporting group description
    Subjects weighing less than (<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination for 15 days.

    Reporting group title
    Part A: LUM 150 mg/IVA 188 mg
    Reporting group description
    Subjects weighing greater than or equal to (>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination for 15 days.

    Reporting group title
    Part B: LUM 100 mg/IVA 125 mg
    Reporting group description
    Subjects weighing less than <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination for 24 weeks.

    Reporting group title
    Part B: LUM 150 mg/IVA 188 mg
    Reporting group description
    Subjects weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination for 24 weeks.

    Reporting group values
    Part A: LUM 100 mg/IVA 125 mg Part A: LUM 150 mg/IVA 188 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg Total
    Number of subjects
    4 8 19 41
    Age categorical
    Units: Subjects
    Age continuous
    Units: months
        arithmetic mean (standard deviation)
    27 ± 6 48 ± 11.11 31.6 ± 5.05 49.9 ± 10.63 -
    Gender categorical
    Units: Subjects
        Female
    2 2 9 20 33
        Male
    2 6 10 21 39

    End points

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    End points reporting groups
    Reporting group title
    Part A: LUM 100 mg/IVA 125 mg
    Reporting group description
    Subjects weighing less than (<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination for 15 days.

    Reporting group title
    Part A: LUM 150 mg/IVA 188 mg
    Reporting group description
    Subjects weighing greater than or equal to (>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination for 15 days.

    Reporting group title
    Part B: LUM 100 mg/IVA 125 mg
    Reporting group description
    Subjects weighing less than <14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination for 24 weeks.

    Reporting group title
    Part B: LUM 150 mg/IVA 188 mg
    Reporting group description
    Subjects weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination for 24 weeks.

    Primary: Part A: Pre-dose Concentration (Ctrough) of Lumacaftor (LUM) and Ivacaftor (IVA)

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    End point title
    Part A: Pre-dose Concentration (Ctrough) of Lumacaftor (LUM) and Ivacaftor (IVA) [1] [2]
    End point description
    End point type
    Primary
    End point timeframe
    Day 15
    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: No statistical testing was planned for this endpoint. Therefore, only descriptive data is provided.
    [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: This primary endpoint is only applicable for Part A. Therefore, only Part A arms are included here.
    End point values
    Part A: LUM 100 mg/IVA 125 mg Part A: LUM 150 mg/IVA 188 mg
    Number of subjects analysed
    4
    7
    Units: Nanogram per milliliter (ng/mL)
    arithmetic mean (standard deviation)
        LUM
    8710 ± 3590
    12300 ± 5960
        IVA
    94.0 ± 67.0
    216 ± 185
    No statistical analyses for this end point

    Primary: Part B: Number of Subjects With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

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    End point title
    Part B: Number of Subjects With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [3] [4]
    End point description
    End point type
    Primary
    End point timeframe
    Day 1 up to Week 26
    Notes
    [3] - 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: No statistical testing was planned for this endpoint. Therefore, only descriptive data is provided.
    [4] - 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: This primary endpoint for AEs is only applicable for Part B. Therefore, only Part B arms are included here.
    End point values
    Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg
    Number of subjects analysed
    19
    41
    Units: subjects
        AEs
    19
    40
        SAEs
    2
    2
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Part A: Day 1 up to Day 25; Part B: Day 1 up to Week 26
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    20.0
    Reporting groups
    Reporting group title
    Part A: LUM 100 mg/IVA 125 mg
    Reporting group description
    -

    Reporting group title
    Part B: LUM 100 mg/IVA 125 mg
    Reporting group description
    -

    Reporting group title
    Part B: LUM 150 mg/IVA 188 mg
    Reporting group description
    -

    Reporting group title
    Part A: LUM 150 mg/IVA 188 mg
    Reporting group description
    -

    Serious adverse events
    Part A: LUM 100 mg/IVA 125 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg Part A: LUM 150 mg/IVA 188 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis viral
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Part A: LUM 100 mg/IVA 125 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg Part A: LUM 150 mg/IVA 188 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 4 (100.00%)
    19 / 19 (100.00%)
    40 / 41 (97.56%)
    6 / 8 (75.00%)
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Seasonal allergy
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    0 / 4 (0.00%)
    7 / 19 (36.84%)
    10 / 41 (24.39%)
    0 / 8 (0.00%)
         occurrences all number
    0
    9
    12
    0
    Fatigue
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Chills
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Discomfort
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pain
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Asthenia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Psychiatric disorders
    Enuresis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Sleep terror
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Tearfulness
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Injury, poisoning and procedural complications
    Joint dislocation
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Radial head dislocation
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Investigations
    Respiratory rate increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    0 / 41 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 19 (15.79%)
    5 / 41 (12.20%)
    0 / 8 (0.00%)
         occurrences all number
    0
    3
    5
    0
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    4 / 41 (9.76%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    4
    0
    Forced expiratory volume decreased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Pseudomonas test positive
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 19 (15.79%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    3
    2
    0
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Burkholderia test positive
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    1
    0
    International normalised ratio increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Prothrombin time prolonged
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pulmonary function test decreased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Staphylococcus test positive
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Streptococcus test positive
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Bacterial test positive
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Blood creatinine increased
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Blood iron decreased
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Enterovirus test positive
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respirovirus test positive
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Vitamin D decreased
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Ventricular extrasystoles
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 4 (50.00%)
    12 / 19 (63.16%)
    26 / 41 (63.41%)
    3 / 8 (37.50%)
         occurrences all number
    3
    23
    52
    3
    Rhinorrhoea
         subjects affected / exposed
    0 / 4 (0.00%)
    8 / 19 (42.11%)
    7 / 41 (17.07%)
    2 / 8 (25.00%)
         occurrences all number
    0
    9
    13
    2
    Nasal congestion
         subjects affected / exposed
    1 / 4 (25.00%)
    4 / 19 (21.05%)
    6 / 41 (14.63%)
    0 / 8 (0.00%)
         occurrences all number
    1
    4
    9
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    3 / 41 (7.32%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    3
    0
    Dyspnoea
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    2
    0
    Nasal discharge discolouration
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Productive cough
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Wheezing
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    2
    0
    Epistaxis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Respiration abnormal
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Sinus congestion
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Sputum discoloured
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Tonsillar inflammation
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Upper respiratory tract congestion
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Lymphocytosis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    4 / 41 (9.76%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    6
    0
    Cognitive disorder
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Lethargy
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Eye disorders
    Eye irritation
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Lacrimation increased
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Gastrointestinal disorders
    Faeces discoloured
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 19 (0.00%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Faeces soft
         subjects affected / exposed
    2 / 4 (50.00%)
    0 / 19 (0.00%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    0
    0
    Flatulence
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Vomiting
         subjects affected / exposed
    2 / 4 (50.00%)
    6 / 19 (31.58%)
    11 / 41 (26.83%)
    0 / 8 (0.00%)
         occurrences all number
    2
    7
    13
    0
    Constipation
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 19 (15.79%)
    4 / 41 (9.76%)
    0 / 8 (0.00%)
         occurrences all number
    0
    4
    5
    0
    Diarrhoea
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    4 / 41 (9.76%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    4
    0
    Abdominal pain
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    3 / 41 (7.32%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    3
    0
    Nausea
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    2
    0
    Abdominal pain upper
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Haematochezia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Oral discomfort
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Oral pain
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Post-tussive vomiting
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Steatorrhoea
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    1
    0
    Dyschezia
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Frequent bowel movements
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Renal and urinary disorders
    Urinary incontinence
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hepatobiliary disorders
    Hepatomegaly
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Rash
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 19 (15.79%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    3
    1
    0
    Urticaria
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hyperhidrosis
         subjects affected / exposed
    1 / 4 (25.00%)
    0 / 19 (0.00%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Musculoskeletal pain
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Endocrine disorders
    Hypothyroidism
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    1
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    3
    0
    Dehydration
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    1
    0
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    2 / 41 (4.88%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    3
    1
    Lice infestation
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    0 / 41 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Lower respiratory tract infection viral
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    0 / 41 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 19 (15.79%)
    7 / 41 (17.07%)
    0 / 8 (0.00%)
         occurrences all number
    0
    4
    12
    0
    Ear infection
         subjects affected / exposed
    0 / 4 (0.00%)
    3 / 19 (15.79%)
    4 / 41 (9.76%)
    0 / 8 (0.00%)
         occurrences all number
    0
    5
    6
    0
    Otitis media
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    4 / 41 (9.76%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    5
    0
    Sinusitis
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    3 / 41 (7.32%)
    0 / 8 (0.00%)
         occurrences all number
    0
    3
    4
    0
    Conjunctivitis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Rhinitis
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    2
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    2 / 41 (4.88%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    4
    0
    Cellulitis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Impetigo
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Labyrinthitis
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Pharyngitis streptococcal
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    1
    0
    Viral rash
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Vulvovaginal mycotic infection
         subjects affected / exposed
    0 / 4 (0.00%)
    0 / 19 (0.00%)
    1 / 41 (2.44%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Candida nappy rash
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Gastroenteritis rotavirus
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Hand-foot-and-mouth disease
         subjects affected / exposed
    0 / 4 (0.00%)
    2 / 19 (10.53%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    0
    0
    Influenza
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Oral candidiasis
         subjects affected / exposed
    0 / 4 (0.00%)
    1 / 19 (5.26%)
    0 / 41 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    01 Aug 2016
    - Added a Washout Period - Removed inclusion criterion related to ppFEV1
    13 Apr 2017
    - Included prescription for a short-acting bronchodilator

    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
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