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    Clinical Trial Results:
    A 52-week, multicenter, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of QAW039 when added to existing asthma therapy in patients with uncontrolled severe asthma

    Summary
    EudraCT number
    		 2015-003172-67
    Trial protocol
    		 CZ   SK   ES   IT   NL   GR  
    Global end of trial date
    02 Aug 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    16 Feb 2020
    First version publication date
    16 Feb 2020
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    CQAW039A2314
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02563067
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@novartis.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    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
    02 Aug 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    02 Aug 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    In patients with severe asthma and high eosinophil counts (≥250 cells/μl) receiving standard-of-care asthma therapy: - To demonstrate the efficacy (as measured by rate of moderate-to-severe asthma exacerbations) of at least one dose level of QAW039 (150 mg or 450 mg once daily), compared with placebo, at the end of the 52-week active-treatment period. In all patients with severe asthma receiving standard-of-care asthma therapy: - To demonstrate the efficacy (as measured by rate of moderate-to-severe asthma exacerbations) of at least one dose level of QAW039 (150 mg or 450 mg once daily), compared with placebo, at the end of the 52-week active-treatment period.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    03 Dec 2015
    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
    Argentina: 189
    Country: Number of subjects enrolled
    Canada: 10
    Country: Number of subjects enrolled
    Czech Republic: 56
    Country: Number of subjects enrolled
    Greece: 31
    Country: Number of subjects enrolled
    India: 76
    Country: Number of subjects enrolled
    Israel: 30
    Country: Number of subjects enrolled
    Italy: 70
    Country: Number of subjects enrolled
    Japan: 31
    Country: Number of subjects enrolled
    Lebanon: 9
    Country: Number of subjects enrolled
    Malaysia: 8
    Country: Number of subjects enrolled
    Mexico: 32
    Country: Number of subjects enrolled
    Netherlands: 11
    Country: Number of subjects enrolled
    Russian Federation: 89
    Country: Number of subjects enrolled
    Serbia: 36
    Country: Number of subjects enrolled
    Slovakia: 89
    Country: Number of subjects enrolled
    South Africa: 8
    Country: Number of subjects enrolled
    Spain: 45
    Country: Number of subjects enrolled
    Taiwan: 7
    Country: Number of subjects enrolled
    United States: 50
    Worldwide total number of subjects
    877
    EEA total number of subjects
    302
    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)
    0
    Adolescents (12-17 years)
    33
    Adults (18-64 years)
    699
    From 65 to 84 years
    145
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were recruited from centers in Argentina (18), Canada (5), Czech Republic (7), Greece (6), India (12), Israel (5), Italy (22), Japan (16), Lebanon (3), Malaysia (4), Mexico (3), Netherlands (4), Russian Federation (11), Serbia (4), Slovakia (11), South Africa (3), Spain (13), Taiwan (3) and the United States (19).

    Pre-assignment
    Screening details
    		 The study included a Screening period of up to 2 weeks and a Placebo Run-in period of 2 to 6 weeks, during which eligibility for randomization was determined

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 QAW039 150 mg
    Arm description
    		 QAW039 150 mg once daily
    Arm type
    		 Experimental

    Investigational medicinal product name
    Fevipiprant
    Investigational medicinal product code
    QAW039
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    QAW039 150 mg once daily (one tablet of blinded QAW039 at 150 mg dosage strength to be given together with one tablet blinded placebo to QAW039 450 mg)

    Arm title
    		 QAW039 450 mg
    Arm description
    		 QAW039 450 mg once daily
    Arm type
    		 Experimental

    Investigational medicinal product name
    Fevipiprant
    Investigational medicinal product code
    QAW039
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    QAW039 450 mg once daily (one tablet of blinded QAW039 at 450 mg dosage strength to be given together with one tablet blinded placebo to QAW039 150 mg)

    Arm title
    		 Placebo
    Arm description
    		 Placebo once daily
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Placebo
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo to QAW039 once daily (one tablet blinded placebo to QAW039 150 mg and one tablet blinded placebo to QAW039 450 mg)

    Number of subjects in period 1
    		QAW039 150 mg QAW039 450 mg Placebo
    Started
    296
    294
    287
    Completed
    277
    268
    267
    Not completed
    19
    26
    20
         Adverse event, serious fatal
    -
    1
    1
         Physician decision
    -
    1
    3
         Adverse event, non-fatal
    2
    1
    -
         Technical problems
    -
    1
    -
         Lost to follow-up
    1
    2
    -
         Subject/guardian decision
    14
    20
    16
         Lack of efficacy
    1
    -
    -
         Death after treatment period
    1
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    QAW039 150 mg
    Reporting group description
    		 QAW039 150 mg once daily

    Reporting group title
    QAW039 450 mg
    Reporting group description
    		 QAW039 450 mg once daily

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo once daily

    Reporting group values
    		 QAW039 150 mg QAW039 450 mg Placebo Total
    Number of subjects
    		 296 294 287 877
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0
        Children (2-11 years)
    		 0 0 0 0
        Adolescents (12-17 years)
    		 12 14 7 33
        Adults (18-64 years)
    		 237 232 230 699
        From 65-84 years
    		 47 48 50 145
        85 years and over
    		 0 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 49.8 ± 14.60 49.6 ± 15.25 50.9 ± 14.16 -
    Sex: Female, Male
    Units: Participants
        Female
    		 165 178 177 520
        Male
    		 131 116 110 357
    Race/Ethnicity, Customized
    Units: Subjects
        Caucasian
    		 234 235 227 696
        Black
    		 6 4 1 11
        Asian
    		 44 41 41 126
        Native American
    		 2 2 4 8
        Unknown
    		 2 2 3 7
        Other
    		 8 10 11 29

    End points

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    End points reporting groups
    Reporting group title
    QAW039 150 mg
    Reporting group description
    		 QAW039 150 mg once daily

    Reporting group title
    QAW039 450 mg
    Reporting group description
    		 QAW039 450 mg once daily

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo once daily

    Primary: Rate of moderate-to-severe Asthma Exacerbations during the 52-week treatment period in high eosinophils subpopulation

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    End point title
    		 Rate of moderate-to-severe Asthma Exacerbations during the 52-week treatment period in high eosinophils subpopulation
    End point description
    A severe asthma exacerbation is defined as treatment with ‘rescue’ systemic corticosteroids for greater than or equal to 3 days and hospitalization; or treatment with ‘rescue’ systemic corticosteroids for greater than or equal to 3 days and emergency department visit (greater than 24 hours*); or death due to asthma. A moderate asthma exacerbation is defined as treatment with ‘rescue’ systemic corticosteroids for greater than or equal to 3 days either as an outpatient or in emergency department visits (Emergency department visit less than or equal to 24 hours). The high eosinophils subpopulation consists of all patients with blood eosinophil count ≥ 250 cells/μL at baseline.
    End point type
    Primary
    End point timeframe
    52 weeks
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    198
    196
    193
    Units: Events/year
        least squares mean (confidence interval 95%)
    0.73 (0.57 to 0.94)
    0.76 (0.59 to 0.99)
    1.06 (0.84 to 1.33)
    Statistical analysis title
    		 Logistic Regression
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    391
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.059 [1]
    Method
    		 negative binomial regression model
    Parameter type
    		 Rate Ratio
    Point estimate
    0.69
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.5
         upper limit
    		 0.96
    Notes
    [1] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.
    Statistical analysis title
    		 Logistic Regression
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    389
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.114 [2]
    Method
    		 negative binomial regression model
    Parameter type
    		 Rate Ratio
    Point estimate
    0.72
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.52
         upper limit
    		 1.01
    Notes
    [2] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.

    Primary: Rate of moderate-to-severe Asthma Exacerbations during the 52-week treatment period in overall population

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    End point title
    		 Rate of moderate-to-severe Asthma Exacerbations during the 52-week treatment period in overall population
    End point description
    A severe asthma exacerbation is defined as treatment with ‘rescue’ systemic corticosteroids for greater than or equal to 3 days and hospitalization; or treatment with ‘rescue’ systemic corticosteroids for greater than or equal to 3 days and emergency department visit (greater than 24 hours*); or death due to asthma. A moderate asthma exacerbation is defined as treatment with ‘rescue’ systemic corticosteroids for greater than or equal to 3 days either as an outpatient or in emergency department visits (Emergency department visit less than or equal to 24 hours).
    End point type
    Primary
    End point timeframe
    52 weeks
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    296
    293
    287
    Units: Events/year
        least squares mean (confidence interval 95%)
    0.76 (0.62 to 0.92)
    0.70 (0.57 to 0.87)
    0.93 (0.76 to 1.12)
    Statistical analysis title
    		 Logistic Regression
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    583
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.369 [3]
    Method
    		 negative binomial regression model
    Parameter type
    		 Rate Ratio
    Point estimate
    0.82
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.62
         upper limit
    		 1.07
    Notes
    [3] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.
    Statistical analysis title
    		 Logistic Regression
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    580
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.348 [4]
    Method
    		 negative binomial regression model
    Parameter type
    		 Rate Ratio
    Point estimate
    0.76
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.58
         upper limit
    		 1
    Notes
    [4] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.

    Secondary: Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in high eosinophils subpopulation

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    End point title
    		 Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in high eosinophils subpopulation
    End point description
    AQLQ is a 32-item instrument administered as a self-assessment. AQLQ+12 is a modified version of AQLQ developed to measure functional impairments of participants aged 12-70 years. It is divided into 4 domains: activity limitation, symptoms, emotional function, and environmental stimuli. Participants were asked to recall their experiences during the last 2 weeks and respond to each question on a 7-point scale (1=severe impairment, 7=no impairment), where higher scores indicated "better quality of life." Overall AQLQ+12 score is the mean of all 32 responses.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    198
    196
    193
    Units: units on a scale
        least squares mean (standard error)
    0.73 ± 0.061
    0.71 ± 0.062
    0.58 ± 0.062
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    391
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.369 [5]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.15
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.02
         upper limit
    		 0.32
    Notes
    [5] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    389
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.824 [6]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.13
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.04
         upper limit
    		 0.3
    Notes
    [6] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.

    Secondary: Change from Baseline to Week 52 in Asthma Control Questionnaire-5 (ACQ-5) score in high eosinophils subpopulation

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    End point title
    		 Change from Baseline to Week 52 in Asthma Control Questionnaire-5 (ACQ-5) score in high eosinophils subpopulation
    End point description
    The ACQ-5 is a five-item, self-completed questionnaire, which is used as a measure of asthma control of a participant. The five questions (concerning nocturnal awakening, waking in the morning, activity limitation, shortness of breath and wheeze) enquire about the frequency and/or severity of symptoms over the previous week. The response options for all these questions range from zero (no impairment/limitation) to six (total impairment/ limitation) scale. ACQ-5 score range from 0 to 6. Higher scores indicates worsening of condition.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    198
    196
    193
    Units: units on a scale
        least squares mean (standard error)
    -0.92 ± 0.067
    -0.84 ± 0.068
    -0.75 ± 0.069
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    391
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.369 [7]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.17
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.36
         upper limit
    		 0.01
    Notes
    [7] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    389
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.824 [8]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.09
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.28
         upper limit
    		 0.1
    Notes
    [8] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.

    Secondary: Change from Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 second (FEV1) in high eosinophils subpopulation

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    End point title
    		 Change from Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 second (FEV1) in high eosinophils subpopulation
    End point description
    Forced Expiratory Volume in one second (FEV1) is calculated as the volume of air forcibly exhaled in one second as measured by a spirometer. Baseline is defined as the last available FEV1 measurement taken prior to the first dose of randomized study drug.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    198
    196
    193
    Units: Liter
        least squares mean (standard error)
    0.192 ± 0.0306
    0.162 ± 0.0311
    0.124 ± 0.0313
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    391
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.369 [9]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.068
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.018
         upper limit
    		 0.154
    Notes
    [9] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    389
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.824 [10]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.038
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.048
         upper limit
    		 0.124
    Notes
    [10] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.

    Secondary: Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in overall population

    		 Close Top of page
    End point title
    		 Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in overall population
    End point description
    AQLQ is a 32-item instrument administered as a self-assessment. AQLQ+12 is a modified version of AQLQ developed to measure functional impairments of participants aged 12-70 years. It is divided into 4 domains: activity limitation, symptoms, emotional function, and environmental stimuli. Participants were asked to recall their experiences during the last 2 weeks and respond to each question on a 7-point scale (1=severe impairment, 7=no impairment), where higher scores indicated "better quality of life." Overall AQLQ+12 score is the mean of all 32 responses.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    296
    293
    287
    Units: units on a scale
        least squares mean (standard error)
    0.62 ± 0.050
    0.67 ± 0.050
    0.55 ± 0.051
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    583
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.824 [11]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.07
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.07
         upper limit
    		 0.21
    Notes
    [11] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    580
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.824 [12]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.12
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.02
         upper limit
    		 0.26
    Notes
    [12] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.

    Secondary: Change from Baseline to Week 52 in Asthma Control Questionnaire-5 (ACQ-5) score in overall population

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    End point title
    		 Change from Baseline to Week 52 in Asthma Control Questionnaire-5 (ACQ-5) score in overall population
    End point description
    The ACQ-5 is a five-item, self-completed questionnaire, which is used as a measure of asthma control of a participant. The five questions (concerning nocturnal awakening, waking in the morning, activity limitation, shortness of breath and wheeze) enquire about the frequency and/or severity of symptoms over the previous week. The response options for all these questions range from zero (no impairment/limitation) to six (total impairment/ limitation) scale. ACQ-5 score range from 0 to 6. Higher scores indicates worsening of condition.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    296
    293
    287
    Units: units on a scale
        least squares mean (standard error)
    -0.83 ± 0.055
    -0.77 ± 0.055
    -0.70 ± 0.055
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    583
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.824 [13]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.12
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.28
         upper limit
    		 0.03
    Notes
    [13] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    580
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.824 [14]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.07
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.23
         upper limit
    		 0.08
    Notes
    [14] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.

    Secondary: Change from Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 second (FEV1) in overall population

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    End point title
    		 Change from Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 second (FEV1) in overall population
    End point description
    Forced Expiratory Volume in one second (FEV1) is calculated as the volume of air forcibly exhaled in one second as measured by a spirometer. Baseline is defined as the last available FEV1 measurement taken prior to the first dose of randomized study drug.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 52
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    296
    293
    287
    Units: Liter
        least squares mean (standard error)
    0.153 ± 0.0247
    0.164 ± 0.0249
    0.103 ± 0.0250
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    583
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.369 [15]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.05
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.019
         upper limit
    		 0.119
    Notes
    [15] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.
    Statistical analysis title
    		 ANCOVA
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    580
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.595 [16]
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.061
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.008
         upper limit
    		 0.13
    Notes
    [16] - adjusted p-value, closed testing procedure across the primary and key secondary null hypotheses. Overall type I error rate controlled at two-sided 5%.

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events are presented from first dose of study treatment until last dose of study treatment plus 7 days, up to maximum duration of 56 weeks.
    Adverse event reporting additional description
    Consistent with EudraCT disclosure specifications, Novartis has reported under the Serious adverse events field “number of deaths resulting from adverse events” all those deaths, resulting from serious adverse events that are deemed to be causally related to treatment by the investigator.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.0
    Reporting groups
    Reporting group title
    QAW039 150 mg
    Reporting group description
    		 QAW039 150 mg

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Reporting group title
    QAW039 450 mg
    Reporting group description
    		 QAW039 450 mg

    Serious adverse events
    		 QAW039 150 mg Placebo QAW039 450 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    20 / 296 (6.76%)
    18 / 287 (6.27%)
    20 / 293 (6.83%)
         number of deaths (all causes)
    0
    1
    1
         number of deaths resulting from adverse events
    0
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Malignant melanoma
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ovarian neoplasm
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Parathyroid tumour benign
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal neoplasm
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Squamous cell carcinoma of skin
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Arterial thrombosis
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lymphoedema
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Non-cardiac chest pain
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Uterine enlargement
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Uterine prolapse
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    8 / 296 (2.70%)
    6 / 287 (2.09%)
    6 / 293 (2.05%)
         occurrences causally related to treatment / all
    0 / 9
    0 / 11
    0 / 8
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Atelectasis
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nasal polyps
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    2 / 293 (0.68%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Femur fracture
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Fracture displacement
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hip fracture
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Humerus fracture
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lower limb fracture
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lumbar vertebral fracture
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Radius fracture
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Seroma
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ulna fracture
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Arteriosclerosis coronary artery
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    Nervous system disorders
    Brain hypoxia
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Eye disorders
    Ulcerative keratitis
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Abdominal pain upper
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Inguinal hernia
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Rectal haemorrhage
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Liver disorder
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Acute kidney injury
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal failure
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Gouty arthritis
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    1 / 296 (0.34%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Bronchitis
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Escherichia sepsis
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal viral infection
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 296 (0.68%)
    3 / 287 (1.05%)
    4 / 293 (1.37%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 3
    1 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    Sinusitis
         subjects affected / exposed
    1 / 296 (0.34%)
    0 / 287 (0.00%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Tonsillitis
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 296 (0.00%)
    1 / 287 (0.35%)
    0 / 293 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 296 (0.00%)
    0 / 287 (0.00%)
    1 / 293 (0.34%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 4%
    Non-serious adverse events
    		 QAW039 150 mg Placebo QAW039 450 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    181 / 296 (61.15%)
    180 / 287 (62.72%)
    183 / 293 (62.46%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    19 / 296 (6.42%)
    9 / 287 (3.14%)
    21 / 293 (7.17%)
         occurrences all number
    24
    12
    30
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    129 / 296 (43.58%)
    145 / 287 (50.52%)
    119 / 293 (40.61%)
         occurrences all number
    267
    319
    214
    Rhinitis allergic
         subjects affected / exposed
    13 / 296 (4.39%)
    9 / 287 (3.14%)
    8 / 293 (2.73%)
         occurrences all number
    18
    10
    10
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    13 / 296 (4.39%)
    9 / 287 (3.14%)
    5 / 293 (1.71%)
         occurrences all number
    15
    11
    5
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    37 / 296 (12.50%)
    32 / 287 (11.15%)
    30 / 293 (10.24%)
         occurrences all number
    46
    52
    35
    Influenza
         subjects affected / exposed
    17 / 296 (5.74%)
    13 / 287 (4.53%)
    18 / 293 (6.14%)
         occurrences all number
    19
    15
    21
    Nasopharyngitis
         subjects affected / exposed
    33 / 296 (11.15%)
    26 / 287 (9.06%)
    42 / 293 (14.33%)
         occurrences all number
    40
    33
    48
    Rhinitis
         subjects affected / exposed
    10 / 296 (3.38%)
    13 / 287 (4.53%)
    15 / 293 (5.12%)
         occurrences all number
    10
    14
    20
    Upper respiratory tract infection
         subjects affected / exposed
    18 / 296 (6.08%)
    14 / 287 (4.88%)
    16 / 293 (5.46%)
         occurrences all number
    19
    21
    22
    Upper respiratory tract infection bacterial
         subjects affected / exposed
    10 / 296 (3.38%)
    14 / 287 (4.88%)
    8 / 293 (2.73%)
         occurrences all number
    16
    20
    13
    Viral upper respiratory tract infection
         subjects affected / exposed
    20 / 296 (6.76%)
    24 / 287 (8.36%)
    28 / 293 (9.56%)
         occurrences all number
    27
    31
    29

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    19 Aug 2015
    A literature review identified additional statins that could interact with QAW039, and patients taking these statins were to be excluded from the study: - Text was added that indicated that patients on doses of simvastatin >20 mg, doses of atorvastatin >40 mg, doses of pravastatin >40mg, or doses of pitavastatin >2 mg per day should not be included in the study. - Statin doses less than or equal to these doses as well as other statins were permitted during the study.
    19 May 2016
    - Changed inclusion criterion #2 to allow for the lower age limit in the study to patients aged ≥12 years (or ≥ lower age limit allowed by health authority and/or ethics committee/institutional review board approvals). - Added an exclusion criterion for patients below the 3rd percentile for weight by age for adolescent patients aged 12 to <18 years to ensure malnourished adolescents were excluded. - Added an exclusion criterion for lactose and milk sensitivity since the placebo tablets include lactose - Added an exclusion criterion for patients with a history of conditions other than asthma that could result in elevated eosinophils (e.g., hypereosinophilic syndromes, Churg-Strauss Syndrome, eosinophilic esophagitis) and exclude patients with known parasitic infestation within 6 months prior to Visit 1. - Added country-required local contraception language for certain countries.
    10 Feb 2017
    Primarily updated renal guidelines and liver event and laboratory trigger definitions of Novartis were included. These changes included: - Updated definitions of urine events. - Updated follow-up requirements for liver events and laboratory triggers for ALT and AST (> 3 to ≤ 5 × ULN (patient is asymptomatic)).

    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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