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    Clinical Trial Results:
    A 52-week, multicenter, randomized, double-blind, double-dummy, parallel-group, placebo-controlled study of fevipiprant once daily plus standard-of-care (SoC) for reduction of systemic corticosteroids (oral and parenteral) use in patients with severe asthma

    Summary
    EudraCT number
    		 2018-000212-25
    Trial protocol
    		 DE   GB   CZ   SK   ES   BE   FR   GR   BG   HU  
    Global end of trial date
    06 Feb 2020

    Results information
    Results version number
    		 v1
    This version publication date
    29 Jan 2021
    First version publication date
    29 Jan 2021
    Other versions
    		 v2

    Trial information

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    Trial identification
    Sponsor protocol code
    CQAW039A2323
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03629249
    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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    06 Feb 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Feb 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The main purpose of this study was to determine the efficacy of fevipiprant (150 mg and 450 mg once daily), compared with placebo, as add-on to GINA (Global Initiative for Asthma) treatment step 4 or 5 standard-of-care (SoC) asthma therapy in terms of avoidance of systemic corticosteroids (SCS) use over 52 weeks in patients with inadequately controlled severe asthma and high eosinophil counts (eosinophil count at Visit 1 ≥250 cells/ μl) and in the overall patient population regardless of eosinophil counts.
    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. Patients were provided SABA (primary rescue medication such as salbutamol 100 mcg or albuterol 90 mcg) as rescue medication to be used on an ‘as needed basis’ during the study. Nebulized formulation of salbutamol/albuterol was not permitted. Oral corticosteroids (add on rescue medication such as prednisone/prednisolone (or equivalent) tablets) were permitted, as instructed by the treating physician, as part of a written asthma plan for the treatment of acute asthma exacerbations and/or symptoms.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    13 Dec 2018
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Argentina: 153
    Country: Number of subjects enrolled
    Belgium: 8
    Country: Number of subjects enrolled
    Bulgaria: 13
    Country: Number of subjects enrolled
    Chile: 6
    Country: Number of subjects enrolled
    Colombia: 6
    Country: Number of subjects enrolled
    Czechia: 31
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Germany: 106
    Country: Number of subjects enrolled
    United Kingdom: 7
    Country: Number of subjects enrolled
    Greece: 10
    Country: Number of subjects enrolled
    Hungary: 63
    Country: Number of subjects enrolled
    Peru: 17
    Country: Number of subjects enrolled
    Philippines: 22
    Country: Number of subjects enrolled
    Russian Federation: 54
    Country: Number of subjects enrolled
    Slovakia: 17
    Country: Number of subjects enrolled
    South Africa: 15
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    Thailand: 3
    Country: Number of subjects enrolled
    Turkey: 9
    Country: Number of subjects enrolled
    United States: 36
    Country: Number of subjects enrolled
    Vietnam: 14
    Worldwide total number of subjects
    604
    EEA total number of subjects
    262
    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)
    0
    Adults (18-64 years)
    498
    From 65 to 84 years
    106
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants took part in 122 investigative sites in 21 countries.

    Pre-assignment
    Screening details
    		 After the screening, participants went through a Run-in period of 4 or 10 weeks to evaluate maintenance of asthma control and to collect baseline safety data.

    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, Data analyst

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 QAW039 150 mg
    Arm description
    		 QAW039 150 mg once daily orally
    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 orally

    Arm title
    		 QAW039 450 mg
    Arm description
    		 QAW039 450 mg once daily orally
    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 orally

    Arm title
    		 Placebo
    Arm description
    		 Placebo to QAW039 once daily orally
    Arm type
    		 Placebo

    Investigational medicinal product name
    placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Placebo to QAW039 once daily orally

    Number of subjects in period 1
    		QAW039 150 mg QAW039 450 mg Placebo
    Started
    201
    201
    202
    Full Analysis Set (FAS)
    201
    200
    201
    Safety Set (SAF)
    201
    200
    201
    Completed
    0
    0
    0
    Not completed
    201
    201
    202
         Physician decision
    1
    -
    -
         Subject decision
    1
    1
    3
         Adverse event, non-fatal
    1
    1
    -
         Death
    1
    -
    -
         Protocol deviation
    -
    2
    5
         Study terminated by sponsor
    197
    197
    194

    Baseline characteristics

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

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

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo to QAW039 once daily orally

    Reporting group values
    		 QAW039 150 mg QAW039 450 mg Placebo Total
    Number of subjects
    		 201 201 202 604
    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)
    		 0 0 0 0
        Adults (18-64 years)
    		 168 166 164 498
        From 65-84 years
    		 33 35 38 106
        85 years and over
    		 0 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 53.4 ( 12.14 ) 53.0 ( 11.99 ) 52.8 ( 12.81 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 121 136 125 382
        Male
    		 80 65 77 222
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 180 176 174 530
        Black or African American
    		 2 2 2 6
        Asian
    		 10 12 9 31
        American Indian or Alaska Native
    		 3 5 13 21
        Missing
    		 6 6 4 16

    End points

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

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

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo to QAW039 once daily orally

    Primary: Total systemic corticosteroid dose in mg prednisone/prednisolone or equivalent over 52 weeks in the overall population

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    End point title
    		 Total systemic corticosteroid dose in mg prednisone/prednisolone or equivalent over 52 weeks in the overall population
    End point description
    All participants were provided with prednisone/prednisolone (or equivalent) tablets that could be used according to the asthma plan along with an electronic diary (e-Diary/ePEF). Daily use of oral corticosteroids (number of tablets taken in the previous 12 hours) was recorded once in the morning and once in the evening by the subject in the eDiary/PEF device. In case of use of injectable corticosteroids during certain circumstances (eg. hospitalization) the data was collected on the eCRF (electronic case report form). The total systemic corticosteroids (SCS) dose data was aggregated into monthly data for analysis. For the patients discontinuing treatment early, the total SCS dose for 52 weeks was obtained as annualized SCS dose. For example if patient took 120 mg for 3 months then for 12 months patient will be taking 120*12/3=480mg total SCS dose. The mean values over 52 weeks in the overall patient population regardless of peripheral blood eosinophil counts are reported here.
    End point type
    Primary
    End point timeframe
    52 weeks
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    201
    200
    201
    Units: milligrams (mg)
        arithmetic mean (full range (min-max))
    219.67 (0 to 9241.27)
    193.02 (0 to 5184.19)
    223.22 (0 to 9552.69)
    Statistical analysis title
    		 Total SCS dose - overall population
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    402
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.714
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Total SCS dose - overall population
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    401
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.734
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Primary: Total systemic corticosteroid dose in mg prednisone/prednisolone or equivalent over 52 weeks in the subpopulation of patients with high eosinophil count (≥ 250 cells/µl)

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    End point title
    		 Total systemic corticosteroid dose in mg prednisone/prednisolone or equivalent over 52 weeks in the subpopulation of patients with high eosinophil count (≥ 250 cells/µl)
    End point description
    All participants were provided with prednisone/prednisolone (or equivalent) tablets that could be used according to the asthma plan along with an electronic diary (e-Diary/ePEF). Daily use of oral corticosteroids (number of tablets taken in the previous 12 hours) was recorded once in the morning and once in the evening by the subject in the eDiary/PEF device. In case of use of injectable corticosteroids during certain circumstances (eg. hospitalization) the data was collected on the eCRF (electronic case report form). The total systemic corticosteroids (SCS) dose data was aggregated into monthly data for analysis. For the patients discontinuing treatment early, the total SCS dose for 52 weeks was obtained as annualized SCS dose. For example if patient took 120 mg for 3 months then for 12 months patient will be taking 120*12/3=480mg total SCS dose. The mean values over 52 weeks in the subpopulation of patients with high peripheral blood eosinophil count at baseline are reported here.
    End point type
    Primary
    End point timeframe
    52 weeks
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    127
    131
    127
    Units: milligrams (mg)
        arithmetic mean (full range (min-max))
    210.88 (0 to 5352.80)
    185.29 (0 to 5184.19)
    212.66 (0 to 9552.69)
    Statistical analysis title
    		 Total SCS dose - high blood eosinophil count
    Comparison groups
    QAW039 150 mg v Placebo
    Number of subjects included in analysis
    254
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.665
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 Total SCS dose - high blood eosinophil count
    Comparison groups
    QAW039 450 mg v Placebo
    Number of subjects included in analysis
    258
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.91
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: Change from baseline in daytime symptom scores

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    End point title
    		 Change from baseline in daytime symptom scores
    End point description
    All participants were provided with an electronic diary (eDiary/ePEF) to record asthma symptom twice per day. Daytime asthma symptoms were assessed before bed and nighttime asthma symptoms on awakening. The daytime asthma symptom score included 4 questions with a range of response categories for each question from 0 to 6 (0 = totally controlled; 6 = extremely poorly controlled). The questions were equally weighted and the overall score (from 0 to 6) was calculated as the average of the 4 questions with higher values indicating more asthma symptoms. Mean values of daytime asthma symptom scores were calculated over 4-week intervals during the treatment period. The baseline values of daytime asthma symptoms scores were defined as the average score during the run-in period. A negative change from baseline in daytime asthma symptom score is a favorable outcome.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 29-32
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    201
    200
    201
    Units: score on scale
    arithmetic mean (standard deviation)
        Week 1-4 (n = 185, 184, 187)
    -0.12 ( 0.494 )
    -0.12 ( 0.497 )
    -0.09 ( 0.459 )
        Week 5-8 (n= 162, 155, 159)
    -0.21 ( 0.615 )
    -0.25 ( 0.570 )
    -0.17 ( 0.561 )
        Week 9-12 (n= 134, 133, 136)
    -0.29 ( 0.632 )
    -0.36 ( 0.588 )
    -0.17 ( 0.605 )
        Week 13-16 (n= 98, 96, 95)
    -0.32 ( 0.644 )
    -0.37 ( 0.653 )
    -0.17 ( 0.629 )
        Week 17-20 (n= 67, 68, 69)
    -0.30 ( 0.570 )
    -0.35 ( 0.759 )
    -0.12 ( 0.622 )
        Week 21-24 (n= 41, 34, 39)
    -0.31 ( 0.536 )
    -0.49 ( 0.760 )
    -0.07 ( 0.688 )
        Week 25-28 (n= 19, 17, 20)
    -0.13 ( 0.519 )
    -0.65 ( 0.751 )
    -0.14 ( 0.726 )
        Week 29-32 (n= 6, 4, 9)
    -0.54 ( 0.546 )
    -0.83 ( 1.072 )
    -0.20 ( 0.887 )
    No statistical analyses for this end point

    Secondary: Change from baseline in nighttime symptom scores

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    End point title
    		 Change from baseline in nighttime symptom scores
    End point description
    All participants were provided with an electronic diary (eDiary/ePEF) to record asthma symptom twice per day. Daytime asthma symptoms were assessed before bed and nighttime asthma symptoms on awakening. The nighttime asthma symptom score included 1 question with a range of response categories from 0 to 3 (0 = no awakening with asthma symptoms; 3 = awake all night). Mean values of nighttime asthma symptom scores were calculated over 4-week intervals during the treatment period. The baseline values of nighttime asthma symptoms scores were defined as the average score during the run-in period. A negative change from baseline in nighttime asthma symptom score is a favorable outcome.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 29-32
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    201
    200
    201
    Units: score on scale
    arithmetic mean (standard deviation)
        Week 1-4 (n= 185, 185, 188)
    -0.08 ( 0.267 )
    -0.05 ( 0.281 )
    -0.07 ( 0.253 )
        Week 5-8 (n= 163, 156, 159)
    -0.11 ( 0.313 )
    -0.12 ( 0.314 )
    -0.13 ( 0.322 )
        Week 9-12 (n= 136, 132, 135)
    -0.15 ( 0.317 )
    -0.19 ( 0.369 )
    -0.12 ( 0.379 )
        Week 13-16 (n= 101, 100, 96)
    -0.19 ( 0.328 )
    -0.18 ( 0.360 )
    -0.12 ( 0.364 )
        Week 17-20 (n= 70, 69, 69)
    -0.21 ( 0.355 )
    -0.18 ( 0.397 )
    -0.17 ( 0.424 )
        Week 21-24 (n= 43, 37, 41)
    -0.17 ( 0.284 )
    -0.16 ( 0.323 )
    -0.14 ( 0.311 )
        Week 25-28 (n= 20, 16, 20)
    -0.17 ( 0.311 )
    -0.29 ( 0.355 )
    -0.15 ( 0.467 )
        Week 29-32 (n= 8, 6, 9)
    -0.12 ( 0.176 )
    -0.46 ( 0.505 )
    -0.24 ( 0.540 )
    No statistical analyses for this end point

    Secondary: Change from baseline in ACQ-5 total score up to end of treatment visit

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    End point title
    		 Change from baseline in ACQ-5 total score up to end of treatment visit
    End point description
    The Asthma Control Questionnaire (ACQ-5) was completed by the patients at the investigator’s site. Patients were asked to recall how their asthma had been during the previous week and to respond to the symptom questions on a 7-point scale (0=no impairment, 6=maximum impairment). The questions were equally weighted and the ACQ-5 score was the mean of the 5 questions and therefore between 0 (totally controlled) and 6 (severely uncontrolled). The baseline values of ACQ-5 score were defined as the ACQ-5 scores obtained on Day 1. A negative change from baseline in ACQ-5 score is a favorable outcome.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 28
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    201
    200
    201
    Units: score on scale
    arithmetic mean (standard deviation)
        Week 6 (n= 166, 158, 160)
    -0.74 ( 0.852 )
    -0.94 ( 0.870 )
    -0.85 ( 0.926 )
        Week 12 (n= 120, 119, 119)
    -0.97 ( 0.948 )
    -1.11 ( 0.904 )
    -1.05 ( 0.884 )
        Week 20 (n= 60, 58, 53)
    -1.08 ( 0.967 )
    -1.13 ( 1.075 )
    -1.22 ( 0.900 )
        Week 28 (n= 19, 13, 15)
    -1.14 ( 0.943 )
    -1.52 ( 1.079 )
    -1.15 ( 1.150 )
    No statistical analyses for this end point

    Secondary: Change from baseline in AQLQ+12 total score up to end of treatment visit

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    End point title
    		 Change from baseline in AQLQ+12 total score up to end of treatment visit
    End point description
    The Asthma Quality of Life Questionnaire (AQLQ+12) was completed by the patients at the investigator’s site. The AQLQ+12 is a 32-item disease specific questionnaire designed to measure functional impairments that are most important to patients with asthma. Patients were asked to recall their experiences during the previous 2 weeks and to score each of the 32 items on a 7-point scale, where 1 indicates maximal impairment and 7 indicates no impairment. Thus, higher scores indicate better asthma-related quality of life. Each item of the AQLQ+12 was equally weighted and the overall score was the mean score of all 32 items and therefore ranged between 1 and 7. The baseline values of AQLQ+12 score were defined as the AQLQ+12 scores obtained on Day 1. A positive change from baseline in AQLQ+12 score is a favorable outcome.
    End point type
    Secondary
    End point timeframe
    Baseline, up to Week 28
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    201
    200
    201
    Units: score on scale
    arithmetic mean (standard deviation)
        Week 6 (n= 166, 159, 162)
    0.43 ( 0.757 )
    0.43 ( 0.762 )
    0.40 ( 0.854 )
        Week 12 (n= 120, 119, 120)
    0.55 ( 0.884 )
    0.60 ( 0.876 )
    0.52 ( 0.833 )
        Week 20 (n= 60, 58, 53)
    0.64 ( 0.903 )
    0.67 ( 0.883 )
    0.60 ( 0.867 )
        Week 28 (n= 19, 13, 15)
    0.43 ( 0.662 )
    1.03 ( 1.178 )
    0.55 ( 1.053 )
    No statistical analyses for this end point

    Secondary: Percentage of patients requiring ≥ 7.5 mg systemic corticosteroid dose in mg prednisone/prednisolone or equivalent per day continuously for at least 30 days

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    End point title
    		 Percentage of patients requiring ≥ 7.5 mg systemic corticosteroid dose in mg prednisone/prednisolone or equivalent per day continuously for at least 30 days
    End point description
    All participants were provided with prednisone/prednisolone (or equivalent) tablets that could be used according to the asthma plan along with an electronic diary (e-Diary/ePEF) to record medication use. Daily use of oral corticosteroids (the number of tablets taken in the previous 12 hours) was recorded once in the morning and once in the evening by the subject using the eDiary/PEF device. In case of use of injectable corticosteroids during certain circumstances (eg. hospitalization) the data was collected on the eCRF (electronic case report form). The percentage of patients requiring ≥ 7.5 mg systemic corticosteroid dose in mg prednisone/prednisolone (or equivalent) per day continuously for at least 30 days within the on-treatment period is presented in this record.
    End point type
    Secondary
    End point timeframe
    Up to 36 weeks
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    201
    200
    201
    Units: percentage of participants
        number (not applicable)
    0.5
    0
    0
    No statistical analyses for this end point

    Secondary: Percentage of patients with no systemic corticosteroids use

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    End point title
    		 Percentage of patients with no systemic corticosteroids use
    End point description
    All participants were provided with prednisone/prednisolone (or equivalent) tablets that could be used according to the asthma plan along with an electronic diary (e-Diary/ePEF) to record medication use. Daily use of oral corticosteroids (the number of tablets taken in the previous 12 hours) was recorded once in the morning and once in the evening by the subject using the eDiary/PEF device. In case of use of injectable corticosteroids during certain circumstances (eg. hospitalization) the data was collected on the eCRF (electronic case report form). The percentage of patients with no systemic corticosteroids use up to visit on Week 36 is presented in this record.
    End point type
    Secondary
    End point timeframe
    Week 36
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    201
    200
    201
    Units: percentage of participants
        number (not applicable)
    84.6
    82.0
    83.6
    No statistical analyses for this end point

    Secondary: Percentage of patients with prescription of biologic therapy

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    End point title
    		 Percentage of patients with prescription of biologic therapy
    End point description
    As part of the flexible therapy, investigators were allowed to prescribe biologics approved for asthma from randomization visit onwards. Prescription of biologic therapy during the treatment period was recorded. The proportion of patients with prescription of biologic therapy during the on-treatment period is presented in this record.
    End point type
    Secondary
    End point timeframe
    Up to 36 weeks
    End point values
    		 QAW039 150 mg QAW039 450 mg Placebo
    Number of subjects analysed
    201
    200
    201
    Units: percentage of participants
        number (not applicable)
    0.5
    0.5
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events (AEs) are presented from first dose of study treatment until last dose plus 14 days. Serious AEs are presented from first dose of study treatment until last dose plus 30 days, up to maximum duration of 40 weeks.
    Adverse event reporting additional description
    Any sign or symptom that occurs during the study treatment plus 14 days post treatment for adverse events and 30 days post treatment for serious adverse events.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    QAW039 150mg
    Reporting group description
    		 QAW039 150mg

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

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

    Serious adverse events
    		 QAW039 150mg Placebo QAW039 450mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    7 / 201 (3.48%)
    4 / 201 (1.99%)
    5 / 200 (2.50%)
         number of deaths (all causes)
    1
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Investigations
    Hepatic enzyme increased
         subjects affected / exposed
    0 / 201 (0.00%)
    0 / 201 (0.00%)
    1 / 200 (0.50%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Intentional product misuse
         subjects affected / exposed
    0 / 201 (0.00%)
    0 / 201 (0.00%)
    1 / 200 (0.50%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Traumatic fracture
         subjects affected / exposed
    1 / 201 (0.50%)
    0 / 201 (0.00%)
    0 / 200 (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
    Vascular disorders
    Circulatory collapse
         subjects affected / exposed
    1 / 201 (0.50%)
    0 / 201 (0.00%)
    0 / 200 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    Cardiac disorders
    Arteriosclerosis coronary artery
         subjects affected / exposed
    0 / 201 (0.00%)
    0 / 201 (0.00%)
    1 / 200 (0.50%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Transient ischaemic attack
         subjects affected / exposed
    0 / 201 (0.00%)
    0 / 201 (0.00%)
    1 / 200 (0.50%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia macrocytic
         subjects affected / exposed
    0 / 201 (0.00%)
    1 / 201 (0.50%)
    0 / 200 (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
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 201 (0.50%)
    0 / 201 (0.00%)
    0 / 200 (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
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    3 / 201 (1.49%)
    3 / 201 (1.49%)
    0 / 200 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Osteoarthritis
         subjects affected / exposed
    1 / 201 (0.50%)
    0 / 201 (0.00%)
    0 / 200 (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
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    0 / 201 (0.00%)
    0 / 201 (0.00%)
    1 / 200 (0.50%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 201 (0.00%)
    0 / 201 (0.00%)
    1 / 200 (0.50%)
         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
    1 / 201 (0.50%)
    0 / 201 (0.00%)
    0 / 200 (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
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 QAW039 150mg Placebo QAW039 450mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    48 / 201 (23.88%)
    44 / 201 (21.89%)
    45 / 200 (22.50%)
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    39 / 201 (19.40%)
    38 / 201 (18.91%)
    41 / 200 (20.50%)
         occurrences all number
    47
    53
    60
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    13 / 201 (6.47%)
    13 / 201 (6.47%)
    10 / 200 (5.00%)
         occurrences all number
    13
    13
    10

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Dec 2018
    The protocol was amended based on health authority feedback. The in-clinic study visit frequencies were modified to 8 weeks intervals after week 12, interspaced with telephone contacts. Additional collection of hematology, clinical chemistry and urinalysis samples were added to all in-clinic visits.

    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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