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    Clinical Trial Results:
    An open-label, single arm study to investigate the safety,pharmacokinetics and pharmacodynamics of repeat doses of inhaled nemiralisib in patients with APDS/PASLI

    Summary
    EudraCT number
    		 2015-004876-31
    Trial protocol
    		 GB  
    Global end of trial date
    04 Jun 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    12 Jun 2021
    First version publication date
    12 Jun 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    204745
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    GlaxoSmithKline
    Sponsor organisation address
    980 Great West Road, Brentford, Middlesex, United Kingdom,
    Public contact
    GSK Response Center, GlaxoSmithKline, 1 8664357343, GSKClinicalSupportHD@gsk.com
    Scientific contact
    GSK Response Center, GlaxoSmithKline, 1 8664357343, GSKClinicalSupportHD@gsk.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
    09 Feb 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    04 Jun 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the safety and tolerability of 84 days repeat dosing of inhaled nemiralisib in patients with activated PI3K delta syndrome (APDS)
    Protection of trial subjects
    Not applicable
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Jul 2016
    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
    United Kingdom: 5
    Worldwide total number of subjects
    5
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    5
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This is an open-label study conducted to investigate safety, pharmacokinetics and pharmacodynamics of repeat doses of inhaled nemiralisib (NEMI) in participants with activated phosphoinositide 3-kinase (PI3K) delta syndrome /p110 delta-activating mutation causing senescent T Cells, lymphadenopathy and immunodeficiency (APDS/PASLI)

    Pre-assignment
    Screening details
    		 Participants received either 1000 mcg NEMI DISKUS or 700 mcg NEMI ELLIPTA or 500 mcg NEMI ELLIPTA. All NEMI DISKUS and NEMI ELLIPTA dose levels were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices. The study had protocol amendments to reflect changes in dose and device administration.

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

    Arms
    Arm title
    		 All NEMI
    Arm description
    		 Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Nemiralisib
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Inhalation powder
    Routes of administration
    Inhalation use
    Dosage and administration details
    Participants received either 1000 mcg NEMI DISKUS or 700 mcg NEMI ELLIPTA or 500 mcg NEMI ELLIPTA. All NEMI DISKUS and NEMI ELLIPTA dose levels were combined as All NEMI treatment group. There was no intent to compare two dose levels or devices.

    Number of subjects in period 1
    		All NEMI
    Started
    5
    Completed
    4
    Not completed
    1
         Adverse event, non-fatal
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    All NEMI
    Reporting group description
    		 Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.

    Reporting group values
    		 All NEMI Total
    Number of subjects
    		 5 5
    Age categorical
    Units: Subjects
        All participants
    		 5 5
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 36.6 ( 12.36 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 3 3
        Male
    		 2 2
    Race/Ethnicity, Customized
    Units: Subjects
        White-White/Caucasian/European Heritage
    		 5 5

    End points

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    End points reporting groups
    Reporting group title
    All NEMI
    Reporting group description
    		 Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.

    Subject analysis set title
    NEMI 1000 mcg Via DISKUS
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants were administered NEMI 1000 mcg using DISKUS DPI once daily in the morning.

    Subject analysis set title
    NEMI 700 mcg Via ELLIPTA
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants were administered NEMI 700 mcg using ELLIPTA DPI once daily in the morning.

    Subject analysis set title
    NEMI 500 mcg Via ELLIPTA
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants were administered NEMI 500 mcg using ELLIPTA DPI once daily in the morning.

    Primary: Number of participants with any serious adverse events (SAEs) and any non-serious adverse events (Non-SAEs)

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    End point title
    		 Number of participants with any serious adverse events (SAEs) and any non-serious adverse events (Non-SAEs) [1]
    End point description
    An adverse event is any untoward medical occurrence in a clinical study participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is defined as any untoward medical occurrence that; results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, is a congenital anomaly/birth defect, other situations as judged by physician. Number of participants with any SAE and non-SAEs are presented. All Subjects Population consisted of all participants who received at least one dose of the study treatment.
    End point type
    Primary
    End point timeframe
    Upto 7.5 months
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [2]
    Units: Participants
        Any non-SAE
    5
        Any SAE
    0
    Notes
    [2] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change From Baseline in Diastolic Blood Pressure (DBP) and Systolic Blood Pressure (SBP)

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    End point title
    		 Change From Baseline in Diastolic Blood Pressure (DBP) and Systolic Blood Pressure (SBP) [3]
    End point description
    SBP and DBP were measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. 99999 indicates that standard deviation could not be calculated as a single participant was analyzed. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [4]
    Units: Millimeters of Mercury (mmHg)
    arithmetic mean (standard deviation)
        DBP, Day 14, n= 5
    1.0 ( 6.44 )
        DBP, Day 28, n= 4
    4.0 ( 6.98 )
        DBP, Day 56, n= 4
    8.5 ( 5.80 )
        DBP, Day 83, n= 4
    2.0 ( 4.40 )
        DBP, Day 84 n= 1
    17.0 ( 99999 )
        SBP, Day 14, n= 5
    -1.0 ( 3.67 )
        SBP, Day 28, n= 4
    5.3 ( 8.54 )
        SBP, Day 56, n= 4
    10.0 ( 9.31 )
        SBP, Day 83, n= 4
    1.5 ( 3.51 )
        SBP, Day 84, n= 1
    16.0 ( 99999 )
    Notes
    [4] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline in Pulse Rate

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    End point title
    		 Change from Baseline in Pulse Rate [5]
    End point description
    Pulse rate was measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. 99999 indicates that standard deviation could not be calculated as a single participant was analyzed. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
    Notes
    [5] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [6]
    Units: Beats per minute
    arithmetic mean (standard deviation)
        Day 14, n= 5
    3.2 ( 7.73 )
        Day 28, n= 4
    2.0 ( 10.86 )
        Day 56, n= 4
    6.8 ( 9.43 )
        Day 83, n= 4
    4.8 ( 7.76 )
        Day 84 n= 1
    7.0 ( 99999 )
    Notes
    [6] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline in Respiratory Rate

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    End point title
    		 Change from Baseline in Respiratory Rate [7]
    End point description
    Respiratory rate was measured in participants in a semi-supine position after 5 minutes rest. . Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. 99999 indicates that standard deviation could not be calculated as a single participant was analyzed. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
    Notes
    [7] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [8]
    Units: Breaths per minute
    arithmetic mean (standard deviation)
        Day 14, n= 5
    0.2 ( 2.28 )
        Day 28, n= 4
    0.8 ( 4.57 )
        Day 56, n= 4
    1.5 ( 5.26 )
        Day 83, n= 4
    1.0 ( 2.45 )
        Day 84 n= 1
    2.0 ( 99999 )
    Notes
    [8] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline in body temperature

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    End point title
    		 Change from Baseline in body temperature [9]
    End point description
    Temperature was measured in participants in a semi-supine position after 5 minutes rest. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. 99999 indicates that standard deviation could not be calculated as a single participant was analyzed. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56, 83 and 84
    Notes
    [9] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [10]
    Units: Degrees celsius
    arithmetic mean (standard deviation)
        Day 14, n= 5
    0.10 ( 0.158 )
        Day 28, n= 4
    0.17 ( 0.350 )
        Day 56, n= 4
    0.07 ( 0.171 )
        Day 83, n= 4
    0.17 ( 0.479 )
        Day 84 n= 1
    0.50 ( 99999 )
    Notes
    [10] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline in electrocardiogram (ECG) mean heart rate

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    End point title
    		 Change from Baseline in electrocardiogram (ECG) mean heart rate [11]
    End point description
    Single 12-lead ECGs were recorded at indicated timepoints using an ECG machine that automatically calculated the heart rate. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
    Notes
    [11] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [12]
    Units: Beats per minute
    arithmetic mean (standard deviation)
        Day 14, n= 5
    -1.0 ( 3.94 )
        Day 28, n= 4
    -0.8 ( 2.99 )
        Day 56, n= 4
    5.3 ( 7.54 )
        Day 83, n= 4
    1.0 ( 2.31 )
    Notes
    [12] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline in PR interval, QRS duration, uncorrected QT interval, QT corrected interval-Fredericia interval (QTcF) and QTc corrected by Bazett’s formula (QTcB)

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    End point title
    		 Change from Baseline in PR interval, QRS duration, uncorrected QT interval, QT corrected interval-Fredericia interval (QTcF) and QTc corrected by Bazett’s formula (QTcB) [13]
    End point description
    Twelve lead ECGs were recorded at indicated timepoints. At each time point, ECG machine automatically measured QRS duration, uncorrected QT interval, QTcF interval and QTcB. Baseline value is defined as latest pre-dose (Day 1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
    Notes
    [13] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [14]
    Units: Milliseconds
    arithmetic mean (standard deviation)
        PR, Day 14, n= 5
    -2.2 ( 9.23 )
        PR, Day 28, n= 4
    -4.8 ( 4.27 )
        PR, Day 56, n= 4
    -3.3 ( 11.18 )
        PR, Day 83, n= 4
    -6.0 ( 11.80 )
        QRS Duration, Day 14, n= 5
    -5.6 ( 7.33 )
        QRS Duration, Day 28, n= 4
    -4.5 ( 11.03 )
        QRS Duration, Day 56, n= 4
    -6.5 ( 8.81 )
        QRS Duration, Day 83, n= 4
    -6.0 ( 7.79 )
        QT Interval, Day 14, n= 5
    -7.2 ( 10.66 )
        QT Interval, Day 28, n= 4
    -2.8 ( 28.89 )
        QT Interval, Day 56, n= 4
    -22.3 ( 16.01 )
        QT Interval, Day 83, n= 4
    -16.3 ( 9.74 )
        QTcB, Day 14, n= 5
    -9.4 ( 4.93 )
        QTcB, Day 28, n= 4
    -4.5 ( 22.55 )
        QTcB, Day 56, n= 4
    -8.3 ( 24.55 )
        QTcB, Day 83, n= 4
    -12.5 ( 8.19 )
        QTcF, Day 14, n= 5
    -8.2 ( 4.32 )
        QTcF, Day 28, n= 4
    -3.5 ( 24.09 )
        QTcF, Day 56, n= 4
    -13.0 ( 19.92 )
        QTcF, Day 83, n= 4
    -14.0 ( 7.79 )
    Notes
    [14] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change From Baseline in Clinical Chemistry Parameters: alanine aminotransferase (ALT), alkaline phosphatase (ALP) and aspartate aminotransferase (AST)

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    End point title
    		 Change From Baseline in Clinical Chemistry Parameters: alanine aminotransferase (ALT), alkaline phosphatase (ALP) and aspartate aminotransferase (AST) [15]
    End point description
    Blood samples were collected for the analysis of clinical parameters including ALT, ALP and AST. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day -1) and at Days 14, 28, 56 and 83
    Notes
    [15] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [16]
    Units: International units per liter (IU/L)
    arithmetic mean (standard deviation)
        ALT, Day 14, n= 5
    1.0 ( 2.12 )
        ALT, Day 28, n= 4
    0.8 ( 1.71 )
        ALT, Day 56, n= 4
    0.0 ( 1.63 )
        ALT, Day 83, n= 4
    1.3 ( 7.50 )
        AST, Day 14, n= 5
    -0.8 ( 1.48 )
        AST, Day 28, n= 4
    0.8 ( 2.22 )
        AST, Day 56, n= 4
    -0.8 ( 4.79 )
        AST, Day 83, n= 4
    -1.0 ( 7.70 )
        ALP, Day 14, n= 5
    2.6 ( 5.77 )
        ALP, Day 28, n= 4
    3.8 ( 5.68 )
        ALP, Day 56, n= 4
    7.5 ( 5.00 )
        ALP, Day 83, n= 4
    6.0 ( 2.94 )
    Notes
    [16] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline in clinical chemistry parameters : Albumin and Total Protein

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    End point title
    		 Change from Baseline in clinical chemistry parameters : Albumin and Total Protein [17]
    End point description
    Blood samples were collected for the analysis of clinical chemistry parameter-albumin and total protein. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day -1) and at Days 14, 28, 56 and 83
    Notes
    [17] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [18]
    Units: Grams per liter
    arithmetic mean (standard deviation)
        Albumin, Day 14, n= 5
    2.6 ( 2.07 )
        Albumin, Day 28, n= 4
    1.3 ( 3.20 )
        Albumin, Day 56, n= 4
    2.8 ( 2.36 )
        Albumin, Day 83, n= 4
    2.0 ( 3.16 )
        Total Protein, Day 14, n= 5
    4.0 ( 1.58 )
        Total Protein, Day 28, n= 4
    1.3 ( 3.59 )
        Total Protein, Day 56, n= 4
    4.0 ( 4.76 )
        Total Protein, Day 83, n= 4
    4.3 ( 2.50 )
    Notes
    [18] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline values in clinical chemistry parameters: sodium, potassium, calcium, glucose and urea

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    End point title
    		 Change from Baseline values in clinical chemistry parameters: sodium, potassium, calcium, glucose and urea [19]
    End point description
    Blood samples were collected for the analysis of clinical parameters including sodium, potassium, calcium, glucose and urea. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day -1) and at Days 14, 28, 56 and 83
    Notes
    [19] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [20]
    Units: Millimoles per liter
    arithmetic mean (standard deviation)
        Sodium, Day 14, n= 5
    -0.2 ( 2.68 )
        Sodium, Day 28, n= 4
    0.5 ( 0.58 )
        Sodium, Day 56, n= 4
    -0.8 ( 2.06 )
        Sodium, Day 83, n= 4
    0.0 ( 1.83 )
        Potassium, Day 14, n= 5
    0.24 ( 0.397 )
        Potassium, Day 28, n= 4
    0.30 ( 0.346 )
        Potassium, Day 56, n= 4
    0.35 ( 0.370 )
        Potassium, Day 83, n= 4
    0.38 ( 0.275 )
        Calcium, Day 14, n= 5
    0.040 ( 0.0990 )
        Calcium, Day 28, n= 4
    0.063 ( 0.0704 )
        Calcium, Day 56, n= 4
    0.133 ( 0.1343 )
        Calcium, Day 83, n= 4
    0.082 ( 0.1325 )
        Glucose, Day 14, n= 5
    -0.64 ( 1.557 )
        Glucose, Day 28, n= 4
    -0.60 ( 1.774 )
        Glucose, Day 56, n= 4
    -0.48 ( 1.578 )
        Glucose, Day 83, n= 4
    -0.68 ( 1.333 )
        Urea, Day 14, n= 5
    4.732 ( 5.8283 )
        Urea, Day 28, n= 4
    8.330 ( 2.6837 )
        Urea, Day 56, n= 4
    2.610 ( 5.1954 )
        Urea, Day 83, n= 4
    2.185 ( 4.1025 )
    Notes
    [20] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline values in clinical chemistry parameters: Direct bilirubin, total bilirubin and creatinine

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    End point title
    		 Change from Baseline values in clinical chemistry parameters: Direct bilirubin, total bilirubin and creatinine [21]
    End point description
    Blood samples were collected for the analysis of clinical chemistry parameters: direct bilirubin, total bilirubin and creatinine. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day -1) and at Days 14, 28, 56 and 83
    Notes
    [21] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [22]
    Units: Micromoles per liter
    arithmetic mean (standard deviation)
        Direct bilirubin, Day 14, n= 5
    -1.0 ( 0.71 )
        Direct bilirubin, Day 28, n= 4
    -1.8 ( 0.96 )
        Direct bilirubin, Day 56, n= 4
    -0.5 ( 1.00 )
        Direct bilirubin, Day 83, n= 4
    -0.5 ( 1.00 )
        Total bilirubin, Day 14, n= 5
    -1.8 ( 1.64 )
        Total bilirubin, Day 28, n= 4
    -3.0 ( 2.45 )
        Total bilirubin, Day 56, n= 4
    -0.8 ( 1.89 )
        Total bilirubin, Day 83, n= 4
    -1.5 ( 2.52 )
        Creatinine , Day 14, n= 5
    -2.0 ( 12.43 )
        Creatinine , Day 28, n= 4
    -4.3 ( 6.08 )
        Creatinine , Day 56, n= 4
    5.0 ( 14.07 )
        Creatinine , Day 83, n= 4
    -2.5 ( 14.93 )
    Notes
    [22] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline values in clinical chemistry parameter: C-Reactive Protein

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    End point title
    		 Change from Baseline values in clinical chemistry parameter: C-Reactive Protein [23]
    End point description
    Blood samples were collected for the analysis of clinical chemistry parameter:C-Reactive Protein. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
    Notes
    [23] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [24]
    Units: Milligrams per liter
    arithmetic mean (standard deviation)
        Day 14, n= 5
    0.56 ( 1.592 )
        Day 28, n= 4
    0.75 ( 1.085 )
        Day 56, n= 4
    2.75 ( 6.222 )
        Day 83, n= 4
    3.90 ( 7.141 )
    Notes
    [24] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline for hematology parameters: basophil, eosinophils, White Blood Cells (WBC), lymphocytes, neutrophils, monocytes and platelets

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    End point title
    		 Change from Baseline for hematology parameters: basophil, eosinophils, White Blood Cells (WBC), lymphocytes, neutrophils, monocytes and platelets [25]
    End point description
    Blood samples were collected for the analysis of hematology parameters including basophils, eosinophils, WBC, lymphocytes, neutrophils, monocytes and platelets at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day -1) and at Days 14, 28, 56 and 83
    Notes
    [25] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [26]
    Units: 10^9 cells per liters
    arithmetic mean (standard deviation)
        Basophils, Day 14, n= 5
    -0.002 ( 0.0045 )
        Basophils, Day 28, n= 4
    0.008 ( 0.0096 )
        Basophils, Day 56, n= 4
    0.008 ( 0.0236 )
        Basophils, Day 83, n= 4
    0.013 ( 0.0222 )
        Eosinophils, Day 14, n= 5
    0.108 ( 0.1542 )
        Eosinophils, Day 28, n= 4
    0.088 ( 0.0650 )
        Eosinophils, Day 56, n= 4
    0.128 ( 0.2241 )
        Eosinophils, Day 83, n= 4
    0.075 ( 0.0810 )
        Lymphocytes, Day 14, n= 5
    0.122 ( 0.2411 )
        Lymphocytes, Day 28, n= 4
    -0.133 ( 0.0918 )
        Lymphocytes, Day 56, n= 4
    -0.105 ( 0.1115 )
        Lymphocytes, Day 83, n= 4
    0.037 ( 0.3140 )
        Monocytes, Day 14, n= 5
    0.096 ( 0.2893 )
        Monocytes, Day 28, n= 4
    0.030 ( 0.0572 )
        Monocytes, Day 56, n= 4
    0.115 ( 0.0614 )
        Monocytes, Day 83, n= 4
    0.065 ( 0.0592 )
        Neutrophils , Day 14, n= 5
    0.334 ( 1.2388 )
        Neutrophils , Day 28, n= 4
    0.575 ( 0.6125 )
        Neutrophils , Day 56, n= 4
    0.553 ( 0.7647 )
        Neutrophils , Day 83, n= 4
    0.653 ( 0.7857 )
        Platelet, Day 14, n= 5
    49.4 ( 52.53 )
        Platelet, Day 28, n= 4
    9.0 ( 25.86 )
        Platelet, Day 56, n= 4
    17.0 ( 31.79 )
        Platelet, Day 83, n= 4
    61.8 ( 43.41 )
        WBC, Day 14, n= 5
    0.656 ( 1.0304 )
        WBC, Day 28, n= 4
    0.568 ( 0.6004 )
        WBC, Day 56, n= 4
    0.698 ( 1.0605 )
        WBC, Day 83, n= 4
    0.845 ( 0.6608 )
    Notes
    [26] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline for hematology parameter: hemoglobin

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    End point title
    		 Change from Baseline for hematology parameter: hemoglobin [27]
    End point description
    Blood samples were collected for the analysis of hematology parameter: hemoglobin at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
    Notes
    [27] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [28]
    Units: Grams per liter
    arithmetic mean (standard deviation)
        Day 14, n= 5
    10.0 ( 5.24 )
        Day 28, n= 4
    8.3 ( 3.77 )
        Day 56, n= 4
    13.5 ( 8.50 )
        Day 83, n= 4
    13.3 ( 5.74 )
    Notes
    [28] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline for hematology parameter: hematocrit

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    End point title
    		 Change from Baseline for hematology parameter: hematocrit [29]
    End point description
    Blood samples were collected for the analysis of hematology parameter: hematocrit at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
    Notes
    [29] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [30]
    Units: Percentage of red blood cells in blood
    arithmetic mean (standard deviation)
        Day 14, n= 5
    0.0320 ( 0.01488 )
        Day 28, n= 4
    0.0333 ( 0.01204 )
        Day 56, n= 4
    0.0518 ( 0.02337 )
        Day 83, n= 4
    0.0465 ( 0.01396 )
    Notes
    [30] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline in hematology parameter: mean corpuscular volume (MCV)

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    End point title
    		 Change from Baseline in hematology parameter: mean corpuscular volume (MCV) [31]
    End point description
    Blood samples were collected for the analysis of hematology parameter: MCV at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
    Notes
    [31] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [32]
    Units: Femtoliters
    arithmetic mean (standard deviation)
        Day 14, n= 5
    1.00 ( 2.187 )
        Day 28, n= 4
    2.33 ( 2.287 )
        Day 56, n= 4
    2.02 ( 2.331 )
        Day 83, n= 4
    1.03 ( 3.407 )
    Notes
    [32] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline in hematology parameter: mean corpuscular Hemoglobin (MCH)

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    End point title
    		 Change from Baseline in hematology parameter: mean corpuscular Hemoglobin (MCH) [33]
    End point description
    Blood samples were collected for the analysis of hematology parameters including MCH at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
    Notes
    [33] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [34]
    Units: Picograms
    arithmetic mean (standard deviation)
        Day 14, n= 5
    0.10 ( 0.485 )
        Day 28, n= 4
    -0.15 ( 0.676 )
        Day 56, n= 4
    -0.43 ( 0.763 )
        Day 83, n= 4
    -0.47 ( 0.525 )
    Notes
    [34] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change from Baseline for hematology parameter: Red Blood Cell Count

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    End point title
    		 Change from Baseline for hematology parameter: Red Blood Cell Count [35]
    End point description
    Blood samples were collected for the analysis of hematology parameter: Blood Cell Count at indicated timepoints. Baseline value is defined as latest pre-dose (Day -1) assessment with a non-missing value, including those from unscheduled visits. Change from Baseline is defined as post-dose visit value minus Baseline value. Only those participants with data available at the specified time points were analyzed (represented by n=X in the category titles).
    End point type
    Primary
    End point timeframe
    Baseline (Day 1 pre-dose) and at Days 14, 28, 56 and 83
    Notes
    [35] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [36]
    Units: 10^12 cells per liter
    arithmetic mean (standard deviation)
        Day 14, n= 5
    0.304 ( 0.1417 )
        Day 28, n= 4
    0.280 ( 0.0673 )
        Day 56, n= 4
    0.488 ( 0.1640 )
        Day 83, n= 4
    0.485 ( 0.1034 )
    Notes
    [36] - All Subjects Population.
    No statistical analyses for this end point

    Primary: Change From Baseline in forced expiratory volume in 1 second (FEV1)

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    End point title
    		 Change From Baseline in forced expiratory volume in 1 second (FEV1) [37]
    End point description
    FEV1 is used to assess pulmonary function using a spirometer at indicated timepoints. Baseline value is defined as the maximum measurement of the planned pre-dose measurements on Day 1, predose. Change from Baseline is defined as post-dose visit value minus Baseline value. Spirometry assessments were performed in accordance with American Thoracic Society (ATS)/European Respiratory Society (ERS) guidelines.
    End point type
    Primary
    End point timeframe
    Baseline (Day 1: pre-dose) and at Day 1: 1 Hour post-dose; Day 2: 1 Hour post-dose; Day 14: Pre-dose; Day 14: 1 Hour post-dose and Day 83: Pre-dose
    Notes
    [37] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: There is no statistical analysis to report
    End point values
    		 All NEMI
    Number of subjects analysed
    5 [38]
    Units: Liters
    arithmetic mean (standard deviation)
        Day 1, 1 Hour post-dose
    0.226 ( 0.2166 )
        Day 2, 1 Hour post-dose
    0.290 ( 0.2884 )
        Day 14, Pre-dose
    -0.165 ( 0.5024 )
        Day 14, 1 Hour post-dose
    0.016 ( 0.4812 )
        Day 83, Pre-dose
    -0.002 ( 0.4584 )
    Notes
    [38] - All Subjects Population.
    No statistical analyses for this end point

    Secondary: Plasma Concentration Following Administration of NEMI

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    End point title
    		 Plasma Concentration Following Administration of NEMI
    End point description
    Blood samples for pharmacokinetic analysis was collected at the indicated time points following administration of NEMI. 99999 indicates that standard deviation could not be calculated as a single participant was analyzed. Only those participants with data available at specified time point were analyzed (represented by n=X in category titles). Pharmacokinetic (PK) Population consisted of all participants in the ‘All Subjects’ population who had at least 1 non-missing PK assessment.
    End point type
    Secondary
    End point timeframe
    Day 1: Pre-dose, 5 minutes, 3 hours and 24 hours post-dose; Day 14 and 83: pre-dose
    End point values
    		 NEMI 1000 mcg Via DISKUS NEMI 700 mcg Via ELLIPTA NEMI 500 mcg Via ELLIPTA
    Number of subjects analysed
    1 [39]
    1
    3
    Units: Picograms per milliliter
    arithmetic mean (standard deviation)
        Day 1: Pre-dose; n=1,1,3
    0.00 ( 99999 )
    0.00 ( 99999 )
    0.00 ( 0.00 )
        Day 1: 5 minutes post-dose; n=1,1,3
    467.00 ( 99999 )
    1524.70 ( 99999 )
    658.27 ( 394.468 )
        Day 1: 3 hours post-dose; n=1,1,3
    895.10 ( 99999 )
    568.60 ( 99999 )
    397.07 ( 172.226 )
        Day 1: 24 hours post-dose; n=1,1,3
    444.40 ( 99999 )
    304.90 ( 99999 )
    198.17 ( 82.442 )
        Day 14: Pre-dose; n=1,1,3
    1396.70 ( 99999 )
    1242.60 ( 99999 )
    750.83 ( 526.337 )
        Day 83: Pre-dose; n=0,1,3
    0 ( 0 )
    1419.60 ( 99999 )
    1525.83 ( 1181.340 )
    Notes
    [39] - PK Population
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Non-serious and serious adverse events were collected up to 7.5 months
    Adverse event reporting additional description
    Non-serious and serious adverse events were collected in the All Subjects population. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group as there was no intent to compare two dose levels or devices.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.0
    Reporting groups
    Reporting group title
    All NEMI
    Reporting group description
    		 Participants were administered with either NEMI 1000 mcg using DISKUS DPI or NEMI 700 or 500 mcg using ELLIPTA DPI once daily in the morning. NEMI DISKUS and NEMI ELLIPTA were combined as All NEMI treatment group

    Serious adverse events
    		 All NEMI
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 5 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 All NEMI
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 5 (100.00%)
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Skin abrasion
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 5 (60.00%)
         occurrences all number
    22
    Migraine
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    General disorders and administration site conditions
    Chest discomfort
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Fatigue
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Feeling hot
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Feeling of body temperature change
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Pain
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Swelling face
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    2 / 5 (40.00%)
         occurrences all number
    2
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    2 / 5 (40.00%)
         occurrences all number
    4
    Abdominal discomfort
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    3
    Abdominal pain
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Abdominal tenderness
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Constipation
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Mouth ulceration
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Bronchospasm
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Cough
         subjects affected / exposed
    3 / 5 (60.00%)
         occurrences all number
    4
    Epistaxis
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Nasal congestion
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Oropharyngeal pain
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Pleuritic pain
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    2
    Wheezing
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    2
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Myalgia
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    3 / 5 (60.00%)
         occurrences all number
    7
    Oral candidiasis
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    2
    Sinusitis
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Fungal skin infection
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Skin bacterial infection
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Product issues
    Product taste abnormal
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Metabolism and nutrition disorders
    Iron deficiency
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Vitamin B complex deficiency
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1
    Vitamin D deficiency
         subjects affected / exposed
    1 / 5 (20.00%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Feb 2016
    Minor change to exploratory efficacy endpoint; Reference to randomisation of subjects removed; Mitigation strategy of the APDS exacerbation risk has been updated; Removal of instruction on the need for precautionary measures (following new non-clinical data supporting the discharge of this risk to humans); Inclusion/Exclusion criteria has been modified; Time and Events Table for Screening and Run-in Period updated; Laboratory tests has been amended; Removal of ‘severity of infection’ as a measurement taken during the screening and critical baseline assessments; Clarification of the period during which details of pregnancies in female subjects will be collected after the start of dosing; Additional text added to clarify which lung lobe will be targeted during BAL procedure; Additional text added to analysis section to clarify the timing of reporting of exploratory biomarker data.
    02 Nov 2016
    Replaced administration of GSK2269557 via the DISKUS device (1000μg) by a comparable dose administered via the ELLIPTA device (700μg); Included patients with APDS1 with new disease-associated mutations and APDS2 with mutations in the PIK3R1 regulatory subunit of class IA phosphoinositide 3 kinases; Administrative changes and clarifications.
    15 Jun 2018
    Replaced administration of nemiralisib in a blend containing 0.6% MgSt, via a dry powder ELLIPTA inhaler (700 μg) by a comparable dose nemiralisib in a blend containing 0.4% magnesium stearate (MgSt) administered via the ELLIPTA device (500μg); Exploratory Phamacodynamics endpoint Volatile Organic Chemicals (VOCs);analysis in breath removed as capabilities not available at study site; Risk assessment table updated with post inhalation cough; Modified inclusion/exclusion criteria; Dose reduction removed as dose strength is 500μg/blister; Time and Event schedule visit window added to Follow Up Visit; Administrative changes, including change from the compound number GSK2269557 to the INN nemiralisib, corrections, relevant updates

    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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