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    Clinical Trial Results:
    A Phase 2b, randomized, double-blind, placebo-controlled, dose-ranging, multi-center study to evaluate the efficacy and safety of GB001 as maintenance therapy in adult subjects with moderate to severe asthma

    Summary
    EudraCT number
    		 2018-002242-36
    Trial protocol
    		 CZ   DE   BE   AT   ES   GB  
    Global end of trial date
    18 Aug 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Sep 2021
    First version publication date
    04 Sep 2021
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GB001-2001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03683576
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    GB001, Inc, a wholly owned subsidiary of Gossamer Bio, Inc.
    Sponsor organisation address
    3013 Science Park Road, San Diego, United States, 92121
    Public contact
    GB001, Inc., Study Director, GB001, Inc., wholly owned subsidiary of Gossamer Bio Inc., 866 668-4083, ClinicalTrials@gossamerbio.com
    Scientific contact
    GB001, Inc., Study Director, GB001, Inc., wholly owned subsidiary of Gossamer Bio Inc., 866 668-4083, ClinicalTrials@gossamerbio.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
    18 Aug 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Jul 2020
    Global end of trial reached?
    Yes
    Global end of trial date
    18 Aug 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the effect of GB001 (the investigational product) compared to placebo on reducing asthma worsening.
    Protection of trial subjects
    This study was conducted in accordance with consensus ethical principles derived from international guidelines including the Declaration of Helsinki, Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines and International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All applicable local laws and regulations regarding patient safety were also followed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Oct 2018
    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
    Austria: 17
    Country: Number of subjects enrolled
    Belgium: 8
    Country: Number of subjects enrolled
    Canada: 3
    Country: Number of subjects enrolled
    Czechia: 54
    Country: Number of subjects enrolled
    France: 7
    Country: Number of subjects enrolled
    Germany: 43
    Country: Number of subjects enrolled
    Poland: 86
    Country: Number of subjects enrolled
    Spain: 16
    Country: Number of subjects enrolled
    Ukraine: 141
    Country: Number of subjects enrolled
    United Kingdom: 4
    Country: Number of subjects enrolled
    United States: 101
    Worldwide total number of subjects
    480
    EEA total number of subjects
    231
    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)
    408
    From 65 to 84 years
    72
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were recruited from Austria, Belgium, Canada, Czechia, France, Germany, Poland, Spain, Ukraine, United Kingdom, and United States.

    Pre-assignment
    Screening details
    		 The study included a run-in period, during which eligibility for randomization was determined. 731 participants entered the run-in period, 481 of whom were randomized. One participant randomized to GB001 40 mg was never treated and was therefore excluded from the Intent-to-Treat (ITT) and Safety Analysis Populations.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 Placebo QD for 24 weeks
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    once per day (QD) for 24 weeks

    Arm title
    		 GB001 20 mg
    Arm description
    		 GB001 20 mg QD for 24 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    GB001 20 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    GB001 20 mg QD for 24 weeks

    Arm title
    		 GB001 40 mg
    Arm description
    		 GB001 40 mg QD for 24 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    GB001 40 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    GB001 40 mg QD for 24 weeks

    Arm title
    		 GB001 60 mg
    Arm description
    		 GB001 60 mg QD for 24 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    GB001 60 mg
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    GB001 60 mg QD for 24 weeks

    Number of subjects in period 1
    		Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Started
    120
    120
    118
    122
    Completed
    114
    116
    106
    114
    Not completed
    6
    4
    12
    8
         Consent withdrawn by subject
    2
    -
    8
    3
         Adverse event, non-fatal
    3
    -
    -
    4
         Other
    -
    2
    -
    -
         Lost to follow-up
    -
    1
    2
    -
         Lack of efficacy
    1
    -
    1
    1
         Protocol deviation
    -
    1
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo QD for 24 weeks

    Reporting group title
    GB001 20 mg
    Reporting group description
    		 GB001 20 mg QD for 24 weeks

    Reporting group title
    GB001 40 mg
    Reporting group description
    		 GB001 40 mg QD for 24 weeks

    Reporting group title
    GB001 60 mg
    Reporting group description
    		 GB001 60 mg QD for 24 weeks

    Reporting group values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg Total
    Number of subjects
    		 120 120 118 122 480
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 51.5 ( 11.91 ) 52.8 ( 11.81 ) 52.9 ( 13.32 ) 49.9 ( 14.37 ) -
    Gender categorical
    Units: Subjects
        Female
    		 76 86 74 72 308
        Male
    		 44 34 44 50 172

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Placebo QD for 24 weeks

    Reporting group title
    GB001 20 mg
    Reporting group description
    		 GB001 20 mg QD for 24 weeks

    Reporting group title
    GB001 40 mg
    Reporting group description
    		 GB001 40 mg QD for 24 weeks

    Reporting group title
    GB001 60 mg
    Reporting group description
    		 GB001 60 mg QD for 24 weeks

    Primary: Proportion of Participants Who Experience Worsening of Asthma by Week 24

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    End point title
    		 Proportion of Participants Who Experience Worsening of Asthma by Week 24
    End point description
    Proportion of participants who experience worsening of asthma by Week 24 as defined by at least 1 of the following: • On 2 consecutive days, morning (AM) peak expiratory flow (PEF) ≤75% of mean AM PEF measured over the last 7 days of the Run-in • Forced expiratory volume in 1 second (FEV1) < 80% of baseline • Increase in rescue medication use of ≥6 puffs/day on 2 consecutive days compared to mean use over the last 7 days of the Run-in • Increase in Asthma Control Questionnaire 5 (ACQ-5; see Secondary: Change From Baseline to Week 24 in ACQ-5 Score for description) score of ≥0.5 compared to baseline • The occurrence of a severe asthma exacerbation (asthma attack) defined as deterioration of asthma that leads to the use of systemic corticosteroids for at least 3 days, hospitalization, or an Emergency Department visit. Analysis Population Description ITT Population: all participants who were randomized and received at least 1 dose of study treatment
    End point type
    Primary
    End point timeframe
    up to Week 24
    End point values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Number of subjects analysed
    120
    120
    118
    122
    Units: proportion of participants
        number (confidence interval 95%)
    0.658 (0.570 to 0.737)
    0.567 (0.477 to 0.652)
    0.568 (0.478 to 0.654)
    0.557 (0.469 to 0.642)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 20 mg v Placebo
    Number of subjects included in analysis
    240
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1425
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.674
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.398
         upper limit
    		 1.142
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    GB001 40 mg v Placebo
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1482
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.677
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.399
         upper limit
    		 1.149
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    GB001 60 mg v Placebo
    Number of subjects included in analysis
    242
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1086
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    0.651
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.385
         upper limit
    		 1.1

    Secondary: Change From Baseline to Week 24 in ACQ-5 Score

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    End point title
    		 Change From Baseline to Week 24 in ACQ-5 Score
    End point description
    The ACQ-5 is a 5-item questionnaire which has been developed as a measure of the participant's asthma control that can be quickly and easily completed. The questions are designed to be self-completed by the participant. The 5 questions enquire about the frequency and/or severity of symptoms in the prior week (nocturnal awakening, activity limitation, shortness of breath, wheeze). The response options for each of these questions consists of a zero (no impairment/limitation) to 6 (total impairment/limitation) scale. Analysis Population Description ITT Population: all participants who were randomized and received at least 1 dose of study treatment.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Number of subjects analysed
    120
    120
    118
    122
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -0.89 (-1.05 to -0.73)
    -1.04 (-1.20 to -0.89)
    -1.04 (-1.20 to -0.88)
    -1.08 (-1.24 to -0.92)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 20 mg v Placebo
    Number of subjects included in analysis
    240
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1647
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.15
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.36
         upper limit
    		 0.06
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    GB001 40 mg v Placebo
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1737
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.15
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.37
         upper limit
    		 0.07
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    GB001 60 mg v Placebo
    Number of subjects included in analysis
    242
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0879
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.19
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.4
         upper limit
    		 0.03

    Secondary: Change From Baseline to Week 24 in Pre-Bronchodilator FEV1

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    End point title
    		 Change From Baseline to Week 24 in Pre-Bronchodilator FEV1
    End point description
    Pre-albuterol/salbutamol morning FEV1 was measured using electronic spirometry. Analysis Population Description ITT Population: all participants who were randomized and received at least 1 dose of study treatment.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Number of subjects analysed
    120
    120
    118
    122
    Units: litres (L)
        least squares mean (confidence interval 95%)
    0.105 (0.027 to 0.182)
    0.121 (0.041 to 0.200)
    0.146 (0.064 to 0.227)
    0.180 (0.102 to 0.257)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 20 mg v Placebo
    Number of subjects included in analysis
    240
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7718
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.016
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.091
         upper limit
    		 0.123
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    GB001 40 mg v Placebo
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.4562
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.041
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.067
         upper limit
    		 0.149
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    GB001 60 mg v Placebo
    Number of subjects included in analysis
    242
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1631
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.075
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.03
         upper limit
    		 0.18

    Secondary: Time to First Asthma Worsening

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    End point title
    		 Time to First Asthma Worsening
    End point description
    Time to first asthma worsening is defined as the time from the date of the first dose of study treatment to the first date that any of the components of asthma worsening endpoint is met. See Primary: Proportion of Participants Who Experience Worsening of Asthma by Week 24 description for the definition of asthma worsening. Analysis Population Description ITT Population: all participants who were randomized and received at least 1 dose of study treatment. '99999' indicates the value is not estimable due to an insufficient number of observed events.
    End point type
    Secondary
    End point timeframe
    up to Week 24
    End point values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Number of subjects analysed
    120
    120
    118
    122
    Units: weeks
        median (confidence interval 95%)
    10.57 (7.857 to 16.286)
    17.43 (12.143 to 99999)
    17.57 (13.429 to 24.286)
    19.86 (14.857 to 99999)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 20 mg v Placebo
    Number of subjects included in analysis
    240
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0466
    Method
    		 Regression, Cox
    Parameter type
    		 Hazard ratio (HR)
    Point estimate
    0.719
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.519
         upper limit
    		 0.995
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    GB001 40 mg v Placebo
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1222
    Method
    		 Regression, Cox
    Parameter type
    		 Hazard ratio (HR)
    Point estimate
    0.773
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.558
         upper limit
    		 1.071
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    GB001 60 mg v Placebo
    Number of subjects included in analysis
    242
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0304
    Method
    		 Regression, Cox
    Parameter type
    		 Hazard ratio (HR)
    Point estimate
    0.698
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.505
         upper limit
    		 0.967

    Secondary: Annualized Rate of Severe Asthma Exacerbations

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    End point title
    		 Annualized Rate of Severe Asthma Exacerbations
    End point description
    A severe asthma exacerbation is defined as deterioration of asthma that leads to the use of systemic corticosteroids for at least 3 days, hospitalization, or an Emergency Department visit. Analysis Population Description ITT Population: all participants who were randomized and received at least 1 dose of study treatment.
    End point type
    Secondary
    End point timeframe
    up to Week 24
    End point values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Number of subjects analysed
    120
    120
    118
    122
    Units: events/year
        least squares mean (confidence interval 95%)
    0.933 (0.664 to 1.311)
    0.744 (0.517 to 1.070)
    0.698 (0.480 to 1.015)
    0.829 (0.585 to 1.174)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 20 mg v Placebo
    Number of subjects included in analysis
    240
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.3382
    Method
    		 Negative binomial regression model
    Parameter type
    		 Rate ratio
    Point estimate
    0.797
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.501
         upper limit
    		 1.268
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    GB001 40 mg v Placebo
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.2248
    Method
    		 Negative binomial regression model
    Parameter type
    		 Rate ratio
    Point estimate
    0.748
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.469
         upper limit
    		 1.195
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    GB001 60 mg v Placebo
    Number of subjects included in analysis
    242
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.609
    Method
    		 Negative binomial regression model
    Parameter type
    		 Rate ratio
    Point estimate
    0.889
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.565
         upper limit
    		 1.397

    Secondary: Change From Baseline to Week 24 in Post-Bronchodilator FEV1

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    End point title
    		 Change From Baseline to Week 24 in Post-Bronchodilator FEV1
    End point description
    Post-albuterol/salbutamol morning FEV1 was measured using electronic spirometry. Analysis Population Description ITT Population: all participants who were randomized and received at least 1 dose of study treatment.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Number of subjects analysed
    120
    120
    118
    122
    Units: litres (L)
        least squares mean (confidence interval 95%)
    0.012 (-0.064 to 0.088)
    -0.011 (-0.088 to 0.066)
    0.047 (-0.037 to 0.131)
    0.091 (0.015 to 0.166)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 20 mg v Placebo
    Number of subjects included in analysis
    240
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.6645
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    -0.023
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.127
         upper limit
    		 0.081
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    GB001 40 mg v Placebo
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.5288
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.035
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.074
         upper limit
    		 0.144
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    GB001 60 mg v Placebo
    Number of subjects included in analysis
    242
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.1362
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    0.079
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.025
         upper limit
    		 0.182

    Secondary: Change From Baseline to Week 24 in AM PEF

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    End point title
    		 Change From Baseline to Week 24 in AM PEF
    End point description
    AM PEF was measured by participants using an electronic diary. Analysis Population Description ITT Population: all participants who were randomized and received at least 1 dose of study treatment.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 24
    End point values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Number of subjects analysed
    120
    120
    118
    122
    Units: L/minute
        least squares mean (confidence interval 95%)
    8.993 (-1.514 to 19.499)
    15.115 (4.779 to 25.451)
    22.941 (12.042 to 33.839)
    14.581 (4.140 to 25.021)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    GB001 20 mg v Placebo
    Number of subjects included in analysis
    240
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.3957
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    6.122
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -8.007
         upper limit
    		 20.251
    Statistical analysis title
    		 Statistical Analysis 2
    Comparison groups
    GB001 40 mg v Placebo
    Number of subjects included in analysis
    238
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0598
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    13.948
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.578
         upper limit
    		 28.474
    Statistical analysis title
    		 Statistical Analysis 3
    Comparison groups
    GB001 60 mg v Placebo
    Number of subjects included in analysis
    242
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.4376
    Method
    		 ANCOVA
    Parameter type
    		 Mean difference (net)
    Point estimate
    5.588
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -8.522
         upper limit
    		 19.698

    Secondary: Incidence of Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Incidence of Treatment-Emergent Adverse Events (TEAEs)
    End point description
    An adverse event (AE) is any untoward medical occurrence in a participant, whether or not considered related to study treatment. Abnormal laboratory test results or other safety assessments, including those that worsened from baseline, that were considered clinically significant in the medical and scientific judgment of the investigator were to be reported as AEs. Analysis Population Description Safety Population: all participants who received at least 1 dose of study treatment.
    End point type
    Secondary
    End point timeframe
    From first dose of study treatment through Week 28
    End point values
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Number of subjects analysed
    120
    120
    118
    122
    Units: percentage of participants
        number (not applicable)
    65.8
    65.8
    69.5
    68.0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study treatment through Week 28
    Adverse event reporting additional description
    [Not specified]
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 -

    Reporting group title
    GB001 20 mg
    Reporting group description
    		 -

    Reporting group title
    GB001 40 mg
    Reporting group description
    		 -

    Reporting group title
    GB001 60 mg
    Reporting group description
    		 -

    Serious adverse events
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    9 / 120 (7.50%)
    5 / 120 (4.17%)
    5 / 118 (4.24%)
    7 / 122 (5.74%)
         number of deaths (all causes)
    0
    0
    0
    1
         number of deaths resulting from adverse events
    0
    0
    0
    1
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Chronic myeloid leukaemia
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    1 / 122 (0.82%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Small cell lung cancer
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    1 / 122 (0.82%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    Vascular disorders
    Hypertension
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Metrorrhagia
         subjects affected / exposed
    1 / 120 (0.83%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Uterine haemorrhage
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Uterine polyp
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Allergic bronchitis
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Asthma
         subjects affected / exposed
    2 / 120 (1.67%)
    2 / 120 (1.67%)
    2 / 118 (1.69%)
    1 / 122 (0.82%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 2
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumothorax
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    1 / 122 (0.82%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Foot fracture
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    1 / 122 (0.82%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Fracture displacement
         subjects affected / exposed
    1 / 120 (0.83%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Acute myocardial infarction
         subjects affected / exposed
    1 / 120 (0.83%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Left ventricular failure
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    1 / 120 (0.83%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Myasthenia gravis
         subjects affected / exposed
    1 / 120 (0.83%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastrooesophageal reflux disease
         subjects affected / exposed
    0 / 120 (0.00%)
    1 / 120 (0.83%)
    0 / 118 (0.00%)
    0 / 122 (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
    Hepatobiliary disorders
    Liver injury
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    1 / 122 (0.82%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Angioedema
         subjects affected / exposed
    0 / 120 (0.00%)
    1 / 120 (0.83%)
    0 / 118 (0.00%)
    0 / 122 (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
    Renal and urinary disorders
    Ureterolithiasis
         subjects affected / exposed
    0 / 120 (0.00%)
    1 / 120 (0.83%)
    0 / 118 (0.00%)
    0 / 122 (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
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Influenza
         subjects affected / exposed
    1 / 120 (0.83%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 120 (0.83%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    1 / 122 (0.82%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    1 / 120 (0.83%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Sinusitis
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    1 / 118 (0.85%)
    0 / 122 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Mineral metabolism disorder
         subjects affected / exposed
    0 / 120 (0.00%)
    0 / 120 (0.00%)
    0 / 118 (0.00%)
    1 / 122 (0.82%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo GB001 20 mg GB001 40 mg GB001 60 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    45 / 120 (37.50%)
    53 / 120 (44.17%)
    59 / 118 (50.00%)
    55 / 122 (45.08%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 120 (0.83%)
    2 / 120 (1.67%)
    3 / 118 (2.54%)
    13 / 122 (10.66%)
         occurrences all number
    1
    2
    6
    15
    Aspartate aminotransferase increased
         subjects affected / exposed
    2 / 120 (1.67%)
    2 / 120 (1.67%)
    4 / 118 (3.39%)
    13 / 122 (10.66%)
         occurrences all number
    2
    2
    7
    14
    Vascular disorders
    Hypertension
         subjects affected / exposed
    2 / 120 (1.67%)
    3 / 120 (2.50%)
    6 / 118 (5.08%)
    5 / 122 (4.10%)
         occurrences all number
    3
    3
    7
    5
    Nervous system disorders
    Headache
         subjects affected / exposed
    11 / 120 (9.17%)
    14 / 120 (11.67%)
    14 / 118 (11.86%)
    13 / 122 (10.66%)
         occurrences all number
    12
    24
    22
    24
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    3 / 120 (2.50%)
    6 / 120 (5.00%)
    1 / 118 (0.85%)
    4 / 122 (3.28%)
         occurrences all number
    3
    8
    1
    5
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    1 / 120 (0.83%)
    1 / 120 (0.83%)
    2 / 118 (1.69%)
    8 / 122 (6.56%)
         occurrences all number
    2
    1
    2
    11
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    6 / 120 (5.00%)
    4 / 120 (3.33%)
    1 / 118 (0.85%)
    1 / 122 (0.82%)
         occurrences all number
    7
    4
    1
    1
    Nasopharyngitis
         subjects affected / exposed
    19 / 120 (15.83%)
    23 / 120 (19.17%)
    29 / 118 (24.58%)
    17 / 122 (13.93%)
         occurrences all number
    26
    26
    41
    24
    Rhinitis
         subjects affected / exposed
    6 / 120 (5.00%)
    1 / 120 (0.83%)
    2 / 118 (1.69%)
    7 / 122 (5.74%)
         occurrences all number
    8
    1
    2
    8
    Sinusitis
         subjects affected / exposed
    3 / 120 (2.50%)
    4 / 120 (3.33%)
    10 / 118 (8.47%)
    3 / 122 (2.46%)
         occurrences all number
    4
    4
    10
    3
    Upper respiratory tract infection
         subjects affected / exposed
    7 / 120 (5.83%)
    3 / 120 (2.50%)
    8 / 118 (6.78%)
    4 / 122 (3.28%)
         occurrences all number
    7
    4
    9
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 Feb 2019
    Amendment 1 (protocol version 2)
    28 Aug 2019
    Amendment 2 (protocol version 3)
    18 Feb 2020
    Amendment 3 (protocol version 4)
    16 Apr 2020
    Amendment 4 (protocol version 5)

    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
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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