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    Clinical Trial Results:
    Study of immune deficiency patients treated with subcutaneous immunoglobulin (IgPro20, Hizentra®) on weekly and biweekly schedules

    Summary
    EudraCT number
    		 2015-004977-34
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    30 Jan 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    09 Aug 2018
    First version publication date
    09 Aug 2018
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    IgPro20_4005
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02711228
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    CSL Behring, LLC
    Sponsor organisation address
    1020 First Avenue, King of Prussia, United States, 19406
    Public contact
    Trial Registration Coordinator, CSL Behring, LLC, clinicaltrials@cslbehring.com
    Scientific contact
    Trial Registration Coordinator, CSL Behring, LLC, clinicaltrials@cslbehring.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    01 Mar 2018
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Jan 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    • To determine tolerability and safety of biweekly IgPro20 injection regimen • To assess the pharmacokinetics of weekly and biweekly IgPro20 injections
    Protection of trial subjects
    This study was carried out in accordance with the International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) guidelines and standard operating procedures for clinical research and development at CSL Behring.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    15 Mar 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
    Canada: 25
    Worldwide total number of subjects
    25
    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)
    8
    Adolescents (12-17 years)
    7
    Adults (18-64 years)
    9
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Subjects were recruited between 15Mar2016 and 11Oct2016 from the Investigators' clinical practices.

    Pre-assignment
    Screening details
    		 Patients who have a documented diagnosis of primary immune deficiency (PID) and secondary immune deficiency (SID), who were on a stable dosing regimen of immunoglobulin (IgG) replacement therapy at screening.

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

    Arms
    Arm title
    		 IgPro20 (Hizentra)
    Arm description
    		 Commercially available Hizentra was used for this study at doses that were consistent with the approved weekly and biweekly Hizentra therapy regimens for patients with immune deficiency.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Hizentra, Subcutaneous Immune Globulin (Human) (SCIg)
    Investigational medicinal product code
    IgPro20
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Commercially available Hizentra was used for this study at doses that were consistent with the approved weekly and biweekly Hizentra therapy regimens for patients with immune deficiency. In Part 1, all subjects were observed for 12 weeks on a weekly Hizentra home infusion treatment regimen. In Part 2, subjects were observed for up to 52 weeks on a biweekly Hizentra home infusion treatment regimen. Part 2 of the study began immediately after the end of Part 1, with the first biweekly Hizentra infusion occurring 2 weeks after the last weekly infusion of Hizentra.

    Number of subjects in period 1
    		IgPro20 (Hizentra)
    Started
    25
    Completed
    16
    Not completed
    9
         Adverse event, non-fatal
    1
         Travel abroad
    2
         Refuse to continue to do 8 infusion sites
    1
         Patient schedule
    1
         Mother unable to come
    2
         Subject choice
    1
         Child does not accept to have 4 infusion sites
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall Trial Total
    Number of subjects
    		 25 25
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 8 8
        Adolescents (12-17 years)
    		 7 7
        Adults (18-64 years)
    		 9 9
        From 65-84 years
    		 1 1
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 23.6 ( 17.93 ) -
    Gender categorical
    Units: Subjects
        Female
    		 11 11
        Male
    		 14 14

    End points

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    End points reporting groups
    Reporting group title
    IgPro20 (Hizentra)
    Reporting group description
    		 Commercially available Hizentra was used for this study at doses that were consistent with the approved weekly and biweekly Hizentra therapy regimens for patients with immune deficiency.

    Subject analysis set title
    All Treated Subjects Analysis Set (ATS)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    All Treated Subjects Analysis Set (ATS): all subjects in the Enrolled analysis set who receive at least one dose of Hizentra, regardless of treatment regimen.

    Subject analysis set title
    Pharmacokinetic Analysis Set (PK)
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Pharmacokinetic Analysis Set (PK): all subjects in the PK subset who have at least 1 post-infusion sample taken and analyzed for 1 of the 2 PK parts

    Subject analysis set title
    Modified ITT Analysis Set (MITT)
    Subject analysis set type
    		 Modified intention-to-treat
    Subject analysis set description
    Modified ITT Analysis Set (MITT): all subjects in the ITT analysis set who receive at least 1 dose of biweekly Hizentra and have at least 1 QoL questionnaire filled.

    Primary: Annualized Rate of Treatment Emergent Adverse Events (TEAEs) (ATS)

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    End point title
    		 Annualized Rate of Treatment Emergent Adverse Events (TEAEs) (ATS) [1]
    End point description
    The annualized rate of TEAEs was calculated per subject as the number of TEAEs in the respective regimen divided by the days treated in the respective treatment regimen multiplied by 365.25.
    End point type
    Primary
    End point timeframe
    During biweekly treatment period, up to approximately 52 weeks
    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: Only descriptive statistics were used for this endpoint.
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    24
    Units: Rate per subject
    arithmetic mean (standard deviation)
        Local TEAEs
    0.18 ( 0.509 )
        Any TEAEs
    4.14 ( 4.787 )
    No statistical analyses for this end point

    Primary: Area under the concentration-time curve [AUC(0-t )] for IgPro20 (PK)

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    End point title
    		 Area under the concentration-time curve [AUC(0-t )] for IgPro20 (PK) [2]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 7 days after infusion during the weekly treatment period and up to 14 days after infusion during the biweekly treatment periods.
    Notes
    [2] - 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: Only descriptive statistics were used for this endpoint.
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    17
    Units: h*g/L
    arithmetic mean (standard deviation)
        Weekly (n=17)
    1770.62 ( 279.119 )
        Biweekly (n=15)
    3683.26 ( 655.535 )
    No statistical analyses for this end point

    Primary: Maximal serum IgG concentration of IgPro20 (Cmax) for IgPro20 (PK)

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    End point title
    		 Maximal serum IgG concentration of IgPro20 (Cmax) for IgPro20 (PK) [3]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 7 days after infusion during the weekly treatment period and up to 14 days after infusion during the biweekly treatment periods
    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: Only descriptive statistics were used for this endpoint.
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    17
    Units: g/L
    arithmetic mean (standard deviation)
        Weekly (n=17)
    11.09 ( 1.659 )
        Biweekly (n=15)
    11.97 ( 2.024 )
    No statistical analyses for this end point

    Primary: Time to maximal serum IgG concentration (Tmax) for IgPro20 (PK)

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    End point title
    		 Time to maximal serum IgG concentration (Tmax) for IgPro20 (PK) [4]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 7 days after infusion during the weekly treatment period and up to 14 days after infusion during the biweekly treatment periods.
    Notes
    [4] - 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: Only descriptive statistics were used for this endpoint.
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    17
    Units: days
    median (full range (min-max))
        Weekly (n=17)
    2.02 (0 to 5.1)
        Biweekly (n=15)
    3.02 (2.0 to 7.1)
    No statistical analyses for this end point

    Primary: Trough serum IgG concentration (Ctrough) for IgPro20 (PK)

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    End point title
    		 Trough serum IgG concentration (Ctrough) for IgPro20 (PK) [5]
    End point description
    End point type
    Primary
    End point timeframe
    Up to 7 days after infusion during the weekly treatment period and up to 14 days after infusion during the biweekly treatment periods.
    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: Only descriptive statistics were used for this endpoint.
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    17
    Units: g/L
    arithmetic mean (standard deviation)
        Weekly (n=17)
    10.32 ( 1.573 )
        Biweekly (n=15)
    10.13 ( 1.940 )
    No statistical analyses for this end point

    Secondary: Annualized rate of infections per subject (ATS)

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    End point title
    		 Annualized rate of infections per subject (ATS)
    End point description
    End point type
    Secondary
    End point timeframe
    During weekly (up to 6 weeks) and biweekly treatment periods (up to 52 weeks)
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    25
    Units: Rate per subject
    arithmetic mean (standard deviation)
        Weekly (n=25)
    2.43 ( 3.146 )
        Biweekly (n=24)
    1.22 ( 1.641 )
    No statistical analyses for this end point

    Secondary: Quality of life using the SF-36 short form during weekly regimen (MITT)

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    End point title
    		 Quality of life using the SF-36 short form during weekly regimen (MITT)
    End point description
    End point type
    Secondary
    End point timeframe
    Week 6
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    9
    Units: scores
    arithmetic mean (standard deviation)
        Physical summary score
    51.45 ( 6.480 )
        Mental summary score
    51.27 ( 7.807 )
    No statistical analyses for this end point

    Secondary: Quality of life using the SF-36 short form during biweekly regimen (MITT)

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    End point title
    		 Quality of life using the SF-36 short form during biweekly regimen (MITT)
    End point description
    End point type
    Secondary
    End point timeframe
    Week 52
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    8
    Units: scores
    arithmetic mean (standard deviation)
        Physical score
    51.25 ( 7.056 )
        Mental score
    49.20 ( 8.727 )
    No statistical analyses for this end point

    Secondary: Quality of life using the CHQ-PF28 (Child Health Questionnaire Parent Form 28): for age < 10 years during weekly regimen (MITT)

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    End point title
    		 Quality of life using the CHQ-PF28 (Child Health Questionnaire Parent Form 28): for age < 10 years during weekly regimen (MITT)
    End point description
    End point type
    Secondary
    End point timeframe
    Week 6
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    6
    Units: Scores
    arithmetic mean (standard deviation)
        Physical Summary Score
    49.82 ( 4.631 )
        Psychosocial summary score
    41.99 ( 18.248 )
    No statistical analyses for this end point

    Secondary: Quality of life using the CHQ-PF28 (Child Health Questionnaire Parent Form 28): for age < 10 years during biweekly regimen (MITT)

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    End point title
    		 Quality of life using the CHQ-PF28 (Child Health Questionnaire Parent Form 28): for age < 10 years during biweekly regimen (MITT)
    End point description
    End point type
    Secondary
    End point timeframe
    Week 52
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    3
    Units: Scores
    arithmetic mean (standard deviation)
        Physical summary score
    43.98 ( 17.308 )
        Psychosocial summary score
    37.38 ( 5.336 )
    No statistical analyses for this end point

    Secondary: Quality of Life using the CHQ-CF87 (Child Health Questionnaire Child Form 87): for age ≥ 10 years during weekly regimen (MITT)

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    End point title
    		 Quality of Life using the CHQ-CF87 (Child Health Questionnaire Child Form 87): for age ≥ 10 years during weekly regimen (MITT)
    End point description
    End point type
    Secondary
    End point timeframe
    Week 6
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    9
    Units: scores
    arithmetic mean (standard deviation)
        Global health
    84.44 ( 15.501 )
        Physical functioning
    100 ( 0 )
        Role/Social limitations-physical
    98.77 ( 3.704 )
        Bodily pain
    76.67 ( 15.811 )
        Behavior
    89.12 ( 11.606 )
        Global behavior
    84.44 ( 15.501 )
        Mental health
    81.77 ( 13.166 )
        Self esteem
    81.35 ( 15.338 )
        General health perceptions
    73.01 ( 18.478 )
        Change in health
    4 ( 1 )
        Family activities
    92.59 ( 20.706 )
        Family cohesion
    84.44 ( 15.501 )
        Role/Social limitations-emotional
    98.77 ( 3.704 )
        Role/Social limitations-behavioral
    100 ( 0 )
    No statistical analyses for this end point

    Secondary: Quality of Life using the CHQ-CF87 (Child Health Questionnaire Child Form 87): for age ≥ 10 years during biweekly regimen (MITT)

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    End point title
    		 Quality of Life using the CHQ-CF87 (Child Health Questionnaire Child Form 87): for age ≥ 10 years during biweekly regimen (MITT)
    End point description
    End point type
    Secondary
    End point timeframe
    Week 52
    End point values
    		 IgPro20 (Hizentra)
    Number of subjects analysed
    5
    Units: Scores
    arithmetic mean (standard deviation)
        Global health
    75 ( 13.693 )
        Physical functioning
    98.52 ( 3.313 )
        Role/Social limitations-physical
    100 ( 0 )
        Bodily pain
    78 ( 20.494 )
        Behavior
    88.88 ( 16.516 )
        Global behavior
    86 ( 16.355 )
        Mental health
    85.31 ( 16.850 )
        Self esteem
    87.14 ( 15.330 )
        General health perceptions
    65.83 ( 9.247 )
        Change in health
    3.4 ( 0.894 )
        Family activities
    95 ( 11.18 )
        Family cohesion
    89 ( 17.464 )
        Role/Social limitations-emotional
    97.78 ( 4.969 )
        Role/Social limitations-behavioral
    97.78 ( 4.969 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    1 year, 4 months
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    IgPro20 (Hizentra)
    Reporting group description
    		 Commercially available Hizentra was used for this study at doses that were consistent with the approved weekly and biweekly Hizentra therapy regimens for patients with immune deficiency.

    Serious adverse events
    		 IgPro20 (Hizentra)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 25 (4.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 25 (4.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 IgPro20 (Hizentra)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    21 / 25 (84.00%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    5 / 25 (20.00%)
         occurrences all number
    5
    Migraine
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Paraesthesia
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    General disorders and administration site conditions
    Injection site bruising
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Injection site pain
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    3 / 25 (12.00%)
         occurrences all number
    3
    Nausea
         subjects affected / exposed
    3 / 25 (12.00%)
         occurrences all number
    3
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    4 / 25 (16.00%)
         occurrences all number
    4
    Tendonitis
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Infections and infestations
    Ear infection
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2
    Nasopharyngitis
         subjects affected / exposed
    9 / 25 (36.00%)
         occurrences all number
    9
    Sinusitis
         subjects affected / exposed
    3 / 25 (12.00%)
         occurrences all number
    3
    Upper respiratory tract infection
         subjects affected / exposed
    4 / 25 (16.00%)
         occurrences all number
    4
    Urinary tract infection
         subjects affected / exposed
    2 / 25 (8.00%)
         occurrences all number
    2

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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    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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