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    Clinical Trial Results:
    A Phase 3, Open-label, Non-controlled, Multi-dose Study to Evaluate the Pharmacokinetics, Safety and Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Subjects with Primary Immunodeficiency Diseases (PID)

    Summary
    EudraCT number
    		 2022-001873-29
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    20 Dec 2021

    Results information
    Results version number
    		 v1
    This version publication date
    06 Jul 2022
    First version publication date
    06 Jul 2022
    Other versions
    		 v2

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    TAK-664-3001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04346108
    WHO universal trial number (UTN)
    		 U1111-1189-8055
    Sponsors
    Sponsor organisation name
    Shire
    Sponsor organisation address
    300 Shire Way, Lexington, United States, MA 02421
    Public contact
    Study Director, Shire, ClinicalTransparency@shire.com
    Scientific contact
    Study Director, Shire, ClinicalTransparency@shire.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
    20 Dec 2021
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Dec 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of this study is to assess serum trough Immunoglobulin globulin G (IgG) concentrations following weekly administration of IGSC, 20% (Epoch 2) and serum trough IgG concentration after biweekly administration of IGSC, 20% in Japanese participants with PID.
    Protection of trial subjects
    All the participants were required to read and sign the Informed Consent Form
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    11 Aug 2020
    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
    Japan: 17
    Worldwide total number of subjects
    17
    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)
    4
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    10
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants took part in the study at 8 investigative sites in Japan from 11 August 2020 to 22 December 2021.

    Pre-assignment
    Screening details
    		 Participants diagnosed with primary immunodeficiency diseases (PID) received immunoglobulin administered intravenously (IGIV) in Epoch 1, followed by immunoglobulin administered subcutaneously (IGSC) 20% in Epoch 2 and then followed by Epoch 3 respectively for up to approximately 50 weeks.

    Period 1
    Period 1 title
    Epoch 1 (13 weeks)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Assessor

    Arms
    Arm title
    		 Epoch 1: IGIV 200-600 mg/kg
    Arm description
    		 Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 3 or 4 weeks for up to 13 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Immune Globulin Intravenous (IGIV)
    Investigational medicinal product code
    Other name
    Immune Globulin Infusion (Human)
    Pharmaceutical forms
    Infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received IGIV infusion.

    Number of subjects in period 1
    		Epoch 1: IGIV 200-600 mg/kg
    Started
    17
    Completed
    17
    Period 2
    Period 2 title
    Epoch 2 (24 weeks after Epoch 1)
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Assessor

    Arms
    Arm title
    		 Epoch 2: IGSC (20%) 50-200 mg/kg
    Arm description
    		 Participants who entered to Epoch 2 from Epoch 1 received 50-200 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion once a week up to approximately 24 weeks after Epoch 1.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
    Investigational medicinal product code
    Other name
    Immune Globulin Infusion (Human)
    Pharmaceutical forms
    Infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received IGSC, 20% SC infusion.

    Number of subjects in period 2
    		Epoch 2: IGSC (20%) 50-200 mg/kg
    Started
    17
    Completed
    15
    Not completed
    2
         Consent withdrawn by subject
    1
         Withdrawal by Parent/Guardian
    1
    Period 3
    Period 3 title
    Epoch 3 (12 weeks after Epoch 2)
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Investigator, Monitor, Assessor, Subject

    Arms
    Arm title
    		 Epoch 3: IGSC (20%) 100-400 mg/kg
    Arm description
    		 Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to approximately 12 weeks after Epoch 2.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
    Investigational medicinal product code
    Other name
    Immune Globulin Infusion (Human)
    Pharmaceutical forms
    Infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received IGSC, 20% SC infusion.

    Number of subjects in period 3 [1]
    		Epoch 3: IGSC (20%) 100-400 mg/kg
    Started
    7
    Completed
    7
    Notes
    [1] - The number of subjects starting the period is not consistent with the number completing the preceding period. It is expected the number of subjects starting the subsequent period will be the same as the number completing the preceding period.
    Justification: Epoch 3 included those participants who completed Epoch 2 and were eligible to enter Epoch 3.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Epoch 1: IGIV 200-600 mg/kg
    Reporting group description
    		 Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 3 or 4 weeks for up to 13 weeks.

    Reporting group values
    		 Epoch 1: IGIV 200-600 mg/kg Total
    Number of subjects
    		 17
    Age Categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 31.0 ( 21.13 ) -
    Gender categorical
    Units: Subjects
        Female
    		 7 7
        Male
    		 10 10
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
        Not Hispanic or Latino
        Unknown or Not Reported
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
        Asian
        Native Hawaiian or Other Pacific Islander
        Black or African American
        White
        More than one race
        Unknown or Not Reported
    Weight
    Units: kilogram (kg)
        arithmetic mean (standard deviation)
    		 ( ) -
    Height
    Units: centimeter (cm)
        arithmetic mean (standard deviation)
    		 ( ) -
    Body Mass Index (BMI)
    BMI is calculated as [weight (kg) / height(m)^2]
    Units: kg/m^2
        arithmetic mean (standard deviation)
    		 ( ) -
    Subject analysis sets

    Subject analysis set title
    Epoch 1: Immune Globulin Intravenous (IGIV)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.

    Subject analysis set title
    Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants who entered to Epoch 2 from Epoch 1 received 50-200 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion once a week up to approximately 24 weeks after Epoch 1.

    Subject analysis set title
    Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to approximately 12 weeks after Epoch 2.

    Subject analysis set title
    Age 2-7 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 2-7 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age 8-13years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 8-13 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age 2-11years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged 2-11 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age ≥12 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged >=12 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age 2-12years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged 2-12 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age ≥13 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged >=13 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age 2-13years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged 2-13 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age ≥14 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged 14 and above years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    IGSC: Epoch 2: 2-13 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 2-13 years received approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch2.

    Subject analysis set title
    IGSC: Epoch 2: >=14 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged >=14 years received approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2.

    Subject analysis set title
    IGSC: Epoch 3: 2-13 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 2-13 years received approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    IGSC: Epoch 3: >=14 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged >=14 years received approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    IGIV: Epoch 1: 3-week interval
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received approximately 200 to 600 mg/kg of IGIV infusion for every 3 for a total of 13 weeks.

    Subject analysis set title
    IGIV: Epoch 1: 4-week interval
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received approximately 200 to 600 mg/kg of IGIV infusion for every 4 for a total of 13 weeks.

    Subject analysis set title
    IGSC: Epoch 3: Biweekly
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2.

    Subject analysis set title
    IGSC: Epoch 2
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received approximately 50 to 200 mg/kg of IGSC infusion, 20% once a week for a total of 24 weeks after Epoch 1.

    Subject analysis set title
    IGIV 200 to 600 mg/kg + IGSC [50 to 200 and 100 to 400] mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks up to 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis sets values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC) Age 2-7 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age 8-13years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age 2-11years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age ≥12 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age 2-12years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age ≥13 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age 2-13years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age ≥14 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg IGSC: Epoch 2: 2-13 years IGSC: Epoch 2: >=14 years IGSC: Epoch 3: 2-13 years IGSC: Epoch 3: >=14 years IGIV: Epoch 1: 3-week interval IGIV: Epoch 1: 4-week interval IGSC: Epoch 3: Biweekly IGSC: Epoch 2 IGIV 200 to 600 mg/kg + IGSC [50 to 200 and 100 to 400] mg/kg
    Number of subjects
    17
    17
    7
    2
    4
    4
    13
    6
    11
    6
    11
    4
    6
    2
    7
    17
    17
    7
    17
    17
    Age Categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    31.0 ( 21.13 )
    Gender categorical
    Units: Subjects
        Female
    7
        Male
    10
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    0
        Not Hispanic or Latino
    17
        Unknown or Not Reported
    0
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0
        Asian
    17
        Native Hawaiian or Other Pacific Islander
    0
        Black or African American
    0
        White
    0
        More than one race
    0
        Unknown or Not Reported
    0
    Weight
    Units: kilogram (kg)
        arithmetic mean (standard deviation)
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    48.16 ( 18.120 )
    Height
    Units: centimeter (cm)
        arithmetic mean (standard deviation)
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    152.15 ( 20.076 )
    Body Mass Index (BMI)
    BMI is calculated as [weight (kg) / height(m)^2]
    Units: kg/m^2
        arithmetic mean (standard deviation)
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    ( )
    19.88 ( 3.242 )

    End points

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    End points reporting groups
    Reporting group title
    Epoch 1: IGIV 200-600 mg/kg
    Reporting group description
    		 Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 3 or 4 weeks for up to 13 weeks.
    Reporting group title
    Epoch 2: IGSC (20%) 50-200 mg/kg
    Reporting group description
    		 Participants who entered to Epoch 2 from Epoch 1 received 50-200 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion once a week up to approximately 24 weeks after Epoch 1.
    Reporting group title
    Epoch 3: IGSC (20%) 100-400 mg/kg
    Reporting group description
    		 Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to approximately 12 weeks after Epoch 2.

    Subject analysis set title
    Epoch 1: Immune Globulin Intravenous (IGIV)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.

    Subject analysis set title
    Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants who entered to Epoch 2 from Epoch 1 received 50-200 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion once a week up to approximately 24 weeks after Epoch 1.

    Subject analysis set title
    Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to approximately 12 weeks after Epoch 2.

    Subject analysis set title
    Age 2-7 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 2-7 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age 8-13years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 8-13 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age 2-11years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged 2-11 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age ≥12 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged >=12 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age 2-12years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged 2-12 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age ≥13 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged >=13 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age 2-13years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged 2-13 years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    Age ≥14 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants aged 14 and above years received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for a total of 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    IGSC: Epoch 2: 2-13 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 2-13 years received approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch2.

    Subject analysis set title
    IGSC: Epoch 2: >=14 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged >=14 years received approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2.

    Subject analysis set title
    IGSC: Epoch 3: 2-13 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 2-13 years received approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    IGSC: Epoch 3: >=14 years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged >=14 years received approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Subject analysis set title
    IGIV: Epoch 1: 3-week interval
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received approximately 200 to 600 mg/kg of IGIV infusion for every 3 for a total of 13 weeks.

    Subject analysis set title
    IGIV: Epoch 1: 4-week interval
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received approximately 200 to 600 mg/kg of IGIV infusion for every 4 for a total of 13 weeks.

    Subject analysis set title
    IGSC: Epoch 3: Biweekly
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2.

    Subject analysis set title
    IGSC: Epoch 2
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received approximately 50 to 200 mg/kg of IGSC infusion, 20% once a week for a total of 24 weeks after Epoch 1.

    Subject analysis set title
    IGIV 200 to 600 mg/kg + IGSC [50 to 200 and 100 to 400] mg/kg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks up to 13 weeks in Epoch 1; followed by approximately 50 to 200 mg/kg of IGSC infusion,20% once a week for a total of 24 weeks after Epoch 1 in Epoch 2; followed by approximately 100 to 400 mg/kg of IGSC infusion, 20% once every two weeks for a total of 12 weeks after Epoch 2 in Epoch 3.

    Primary: Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2

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    End point title
    		 Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2 [1]
    End point description
    Total serum trough levels of IgG antibodies measured during period 2 of Epoch 2 were assessed.
    End point type
    Primary
    End point timeframe
    Epoch 2 (period 2): Up to 24 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: No statistical analyses have been specified for this primary end point.
    End point values
    		 IGIV: Epoch 1: 3-week interval IGIV: Epoch 1: 4-week interval IGSC: Epoch 3: Biweekly IGSC: Epoch 2
    Number of subjects analysed
    0 [2]
    0 [3]
    0 [4]
    0 [5]
    Units: participants
    Notes
    [2] - Data is not available due to delay in measurement of IgG subclasses.
    [3] - Data is not available due to delay in measurement of IgG subclasses.
    [4] - Data is not available due to delay in measurement of IgG subclasses.
    [5] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Primary: Epoch 3: Total Serum Trough Levels of IgG Antibodies

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    End point title
    		 Epoch 3: Total Serum Trough Levels of IgG Antibodies [6]
    End point description
    Total serum trough levels of IgG antibodies measured during Epoch 3 were assessed.
    End point type
    Primary
    End point timeframe
    Epoch 3: Up to Week 12
    Notes
    [6] - 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: No statistical analyses have been specified for this primary end point.
    End point values
    		 IGIV: Epoch 1: 3-week interval IGIV: Epoch 1: 4-week interval IGSC: Epoch 3: Biweekly IGSC: Epoch 2
    Number of subjects analysed
    0 [7]
    0 [8]
    0 [9]
    0 [10]
    Units: participants
    Notes
    [7] - Data is not available due to delay in measurement of IgG subclasses.
    [8] - Data is not available due to delay in measurement of IgG subclasses.
    [9] - Data is not available due to delay in measurement of IgG subclasses.
    [10] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Secondary: Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG and IgG Subclasses

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    End point title
    		 Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG and IgG Subclasses
    End point description
    End point type
    Secondary
    End point timeframe
    Epoch 2: Week 21: Pre-infusion (Day 0), Day 1, 3, 5, and 7
    End point values
    		 IGSC: Epoch 2
    Number of subjects analysed
    0 [11]
    Units: participants
    Notes
    [11] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Secondary: Epoch 2: Area Under the Curve (AUC) for Total Serum Levels of IgG and IgG Subclasses

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    End point title
    		 Epoch 2: Area Under the Curve (AUC) for Total Serum Levels of IgG and IgG Subclasses
    End point description
    End point type
    Secondary
    End point timeframe
    Epoch 2: Week 21: Pre-infusion (Day 0), Day 1, 3, 5, and 7
    End point values
    		 IGSC: Epoch 2
    Number of subjects analysed
    0 [12]
    Units: participants
    Notes
    [12] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Secondary: Epoch 1: Total Serum Trough Levels of IgG Antibodies

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    End point title
    		 Epoch 1: Total Serum Trough Levels of IgG Antibodies
    End point description
    Total serum trough levels of IgG antibodies measured during Epoch 1 were assessed.
    End point type
    Secondary
    End point timeframe
    Epoch 1: Up to Week 13
    End point values
    		 IGIV: Epoch 1: 3-week interval IGIV: Epoch 1: 4-week interval IGSC: Epoch 3: Biweekly IGSC: Epoch 2
    Number of subjects analysed
    0 [13]
    0 [14]
    0 [15]
    0 [16]
    Units: participants
    Notes
    [13] - Data is not available due to delay in measurement of IgG subclasses.
    [14] - Data is not available due to delay in measurement of IgG subclasses.
    [15] - Data is not available due to delay in measurement of IgG subclasses.
    [16] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Secondary: Epoch 2: Maximum Concentration (Cmax) for Total Serum Levels of IgG and IgG Subclasses

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    End point title
    		 Epoch 2: Maximum Concentration (Cmax) for Total Serum Levels of IgG and IgG Subclasses
    End point description
    End point type
    Secondary
    End point timeframe
    Epoch 2: Week 21: Pre-infusion (Day 0), Day 1, 3, 5, and 7
    End point values
    		 IGSC: Epoch 2
    Number of subjects analysed
    0 [17]
    Units: participants
    Notes
    [17] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Secondary: Epoch 2: Minimum Concentration (Cmin) for Total Serum Levels of IgG and IgG Subclasses

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    End point title
    		 Epoch 2: Minimum Concentration (Cmin) for Total Serum Levels of IgG and IgG Subclasses
    End point description
    End point type
    Secondary
    End point timeframe
    Epoch 2: Week 21: Pre-infusion (Day 0), Day 1, 3, 5, and 7
    End point values
    		 IGSC: Epoch 2
    Number of subjects analysed
    0 [18]
    Units: participants
    Notes
    [18] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Secondary: Epoch 2: Time to Maximum Concentration (Tmax) for Total Serum Levels of IgG and IgG Subclasses

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    End point title
    		 Epoch 2: Time to Maximum Concentration (Tmax) for Total Serum Levels of IgG and IgG Subclasses
    End point description
    End point type
    Secondary
    End point timeframe
    Epoch 2: Week 21: Pre-infusion (Day 0), Day 1, 3, 5, and 7
    End point values
    		 IGSC: Epoch 2
    Number of subjects analysed
    0 [19]
    Units: participants
    Notes
    [19] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Secondary: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens

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    End point title
    		 Trough Levels of Specific Antibodies to Clinically Relevant Pathogens
    End point description
    Trough levels of specific antibodies to clinically relevant pathogens (Clostridium tetani toxoid, HIB, HBV) were assessed in Epoch 1, Epoch 2 and Epoch 3.
    End point type
    Secondary
    End point timeframe
    Epoch 1 (Week 1); Epoch 2 (Week 1); Epoch 3 (Week 1, 13)
    End point values
    		 IGIV: Epoch 1: 3-week interval IGIV: Epoch 1: 4-week interval IGSC: Epoch 3: Biweekly IGSC: Epoch 2
    Number of subjects analysed
    0 [20]
    0 [21]
    0 [22]
    0 [23]
    Units: participants
    Notes
    [20] - Data is not available due to delay in measurement of IgG subclasses.
    [21] - Data is not available due to delay in measurement of IgG subclasses.
    [22] - Data is not available due to delay in measurement of IgG subclasses.
    [23] - Data is not available due to delay in measurement of IgG subclasses.
    No statistical analyses for this end point

    Secondary: Number of Participants with Treatment Emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Participants with Treatment Emergent Adverse Events (TEAEs)
    End point description
    TEAEs was defined as adverse events (AEs) with onset after date-time of first dose of study drug, or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP. Any TEAE that is recorded by the investigator as "possibly related" or "probably related" to IP was considered as IGSC, 20%-related AE, and any AE recorded as "unlikely related" or "not related" was considered as unrelated AE. AEs included vital signs, clinical laboratory measurements. All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: participants
    11
    16
    3
    No statistical analyses for this end point

    Secondary: Number of Participants with Tolerability Events Related to the Infusion of Study Drug

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    End point title
    		 Number of Participants with Tolerability Events Related to the Infusion of Study Drug
    End point description
    An infusion is considered tolerable if the infusion rate was not reduced, or the infusion was not interrupted or stopped, due to TEAE related to study drug (IGIV or IGSC) infusion. A tolerability event is considered to have occurred if an infusion was not tolerable in Epoch 1, Epoch 2 and Epoch 3. Number of participants with tolerability events related to infusion of IP will be assessed. All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: participants
        Infusions With Infusion Rate Reduced
    0
    0
    0
        Infusions That Were Interrupted
    0
    1
    0
        Infusions That Were Stopped
    0
    0
    0
        Infusion Rate Reduced/Interrupted/Stopped
    0
    1
    0
    No statistical analyses for this end point

    Secondary: Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)

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    End point title
    		 Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
    End point description
    The ASBI rate was calculated as the mean number of acute serious bacterial infections per participants per year. Annual rate of validated acute serious bacterial infections per participant was assessed. All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: number of infections per year
        arithmetic mean (standard deviation)
    0.0 ( 0.00 )
    0.25 ( 1.042 )
    0.0 ( 0.00 )
    No statistical analyses for this end point

    Secondary: Annual Rate of All Infections Per Year

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    End point title
    		 Annual Rate of All Infections Per Year
    End point description
    Annual rate is the number of participants reporting any infection per year. All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: number of infections per year
        arithmetic mean (standard deviation)
    1.65 ( 2.479 )
    2.78 ( 3.074 )
    0.00 ( 0.000 )
    Statistical analysis title
    		 Epoch 1: IGIV
    Comparison groups
    Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    1.65
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.73
         upper limit
    		 3.15
    Statistical analysis title
    		 Epoch 3: IGSC
    Comparison groups
    Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    0
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0
         upper limit
    		 0
    Statistical analysis title
    		 Epoch 2: IGSC
    Comparison groups
    Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    2.48
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.34
         upper limit
    		 4.13

    Secondary: Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection

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    End point title
    		 Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection
    End point description
    Number of days not able to attend school or work to perform normal daily activities due to illness/infection are standardized per year (365.25 days). The number of days not able to attend school or work to perform normal daily activities due to illness/infection were assessed. All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: days
        median (full range (min-max))
    0.00 (0.0 to 16.1)
    0.00 (0.0 to 17.2)
    4.30 (0.0 to 38.7)
    Statistical analysis title
    		 Epoch 1: IGIV
    Comparison groups
    Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    2.6
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.02
         upper limit
    		 5.3
    Statistical analysis title
    		 Epoch 3: IGSC
    Comparison groups
    Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    9.82
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.82
         upper limit
    		 23.82
    Statistical analysis title
    		 Epoch 2: IGSC
    Comparison groups
    Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    2.87
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.37
         upper limit
    		 5.18

    Secondary: Number of Days Participants Were on Antibiotics

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    End point title
    		 Number of Days Participants Were on Antibiotics
    End point description
    Number of days on antibiotics is defined as the number of days those antibiotics were taken as concomitant medications and is standardized to per year (365.25 days). Antibiotics are defined as any medication under anatomical therapeutic chemical Level 2 therapeutic class “ANTIBACTERIALS FOR SYSTEMIC USE”. If a participant took multiple antibiotics on a single day, that day is counted for only once. Protocol defined prophylactic antibiotics for viral, fungal or protozoal infections (e.g. trimethoprim/sulfamethoxazole twice a week for pneumocystis) which are not treated by immunoglobulin, were excluded from this analysis. All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: days
        median (full range (min-max))
    0.00 (0.0 to 31.8)
    0.00 (0.0 to 34.5)
    0.00 (0.0 to 21.5)
    Statistical analysis title
    		 Epoch 1: IGIV
    Comparison groups
    Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    4.01
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.46
         upper limit
    		 8.54
    Statistical analysis title
    		 Epoch 3: IGSC
    Comparison groups
    Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV)
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    3.07
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.37
         upper limit
    		 10.74
    Statistical analysis title
    		 Epoch 2: IGSC
    Comparison groups
    Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    5.87
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.32
         upper limit
    		 11.93

    Secondary: Number of Participants Hospitalized due to Illness or Infection

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    End point title
    		 Number of Participants Hospitalized due to Illness or Infection
    End point description
    Number of participants with hospitalization are standardized to per year (365.25 days). A hospitalization is counted for a specific epoch only if that hospitalization started during that epoch. All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: participants
    0
    1
    0
    Statistical analysis title
    		 Epoch 1: IGIV
    Comparison groups
    Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    34
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    0
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0
         upper limit
    		 0
    Statistical analysis title
    		 Epoch 3: IGSC
    Comparison groups
    Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    0
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0
         upper limit
    		 0
    Statistical analysis title
    		 Epoch 2: IGSC
    Comparison groups
    Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    0.13
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.03
         upper limit
    		 0.35

    Secondary: Length of Hospital Stay

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    End point title
    		 Length of Hospital Stay
    End point description
    Length of hospital stay per stay is standardized to per year (365.25 days). A hospitalization is counted for a specific epoch only if that hospitalization started during that epoch. All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: days
        median (full range (min-max))
    0.00 (0.0 to 0.0)
    0.00 (0.0 to 17.4)
    0.00 (0.0 to 0.0)
    Statistical analysis title
    		 Epoch 1: IGIV
    Comparison groups
    Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    0
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0
         upper limit
    		 0
    Statistical analysis title
    		 Epoch 3: IGSC
    Comparison groups
    Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    0
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0
         upper limit
    		 0
    Statistical analysis title
    		 Epoch 2: IGSC
    Comparison groups
    Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    1.04
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.25
         upper limit
    		 2.76

    Secondary: Number of Acute Physician Visits due to Illness/Infection

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    End point title
    		 Number of Acute Physician Visits due to Illness/Infection
    End point description
    Number of acute physician visits is standardized to per year (365.25 days). All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to end of study (up to approximately 1.5 years)
    End point values
    		 Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects analysed
    17
    17
    7
    Units: number of visits per year
        arithmetic mean (standard deviation)
    1.18 ( 3.098 )
    3.30 ( 6.807 )
    0.61 ( 1.624 )
    Statistical analysis title
    		 Epoch 1: IGIV
    Comparison groups
    Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    1.18
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.38
         upper limit
    		 2.68
    Statistical analysis title
    		 Epoch 3: IGSC
    Comparison groups
    Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    0.61
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.07
         upper limit
    		 2.15
    Statistical analysis title
    		 Epoch 2: IGSC
    Comparison groups
    Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) v Epoch 1: Immune Globulin Intravenous (IGIV) v Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
    Number of subjects included in analysis
    41
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    Method
    Parameter type
    		 Poisson Estimate
    Point estimate
    2.35
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.91
         upper limit
    		 4.82

    Secondary: Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score

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    End point title
    		 Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score
    End point description
    Peds-QL=generic HR QoL instrument designed specifically for pediatrics has domains as:general health/activities,feelings/emotional,social functioning,school functioning.In this study,2-7 years (parent as observer),8-13 years (participant as observer) for Peds-QL health questionnaire was analyzed.Higher scores=better QOL for all domains.This modular instrument used 5-point scale:0(never) to 4(almost always).Items are reversed scored;linearly transformed to 0-100 scale as follows:0=100,1=75,2=50,3=25,4=0.4 dimensions(physical, emotional, social, & school functioning) are scored.PEDS-QL Total Scale Score has 0-100 scale,higher scores=better HRQoL.All-Treated Set=all enrolled participants of age group ‘2-7 years’ and ‘8-13 years’ who received at least 1 dose of study drug (IGIV or IGSC).‘n’=Number analysed are participants with data available for analysis.End of Study/Early Termination (EOS/ET). 999=Standard deviation was not estimable for 1 participant. CFB=change from baseline.
    End point type
    Secondary
    End point timeframe
    Baseline up to end of study (approximately 1.5 years)
    End point values
    		 Age 2-7 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age 8-13years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Number of subjects analysed
    2
    4
    Units: score on a scale
    arithmetic mean (standard deviation)
        IGIV: Epoch 1: Baseline (Week 1)(n=2,4)
    88.04 ( 13.835 )
    87.23 ( 11.820 )
        IGSC: Epoch 2: CFB (Week 1)(n=2,4)
    9.78 ( 15.372 )
    -6.79 ( 9.977 )
        IGSC: Epoch 3: CFB (Week 1)[n=1,1]
    14.13 ( 999 )
    20.65 ( 999 )
        IGSC: Epoch 3: CFB (EOS/ET)(n=2,4)
    3.26 ( 7.686 )
    4.08 ( 16.374 )
    No statistical analyses for this end point

    Secondary: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score

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    End point title
    		 EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score
    End point description
    EQ-5D-3L health questionnaire=participant answered questionnaire scoring 5 dimensions -mobility,self-care,usual activities, pain/discomfort and anxiety/depression. n this study, 2-11 years (parent as observer),12 years and older (participant as observer) for EQ-5D-3L health questionnaire was analyzed.Health state index score range from 0 (worst health state) to 1 (perfect health state) and 1 reflects the best outcome.EQ visual analogue scale range from 0 to 100, where higher scores indicate better health status.Data is reported as per age groups (2-11 years and >=12 years).All-Treated Set included all enrolled participants of age group ‘2-11 years’ and ‘>=12 years’ who received at least 1 dose of study drug (IGIV or IGSC). ‘n’=Number analysed are participants with data available for analysis. Health State Index Score (HSIS), Q Visual Analogue Scale (EQVAS).999=Standard deviation was not estimable for 1 participant.
    End point type
    Secondary
    End point timeframe
    Baseline up to end of the study (approximately 1.5 years)
    End point values
    		 Age 2-11years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age ≥12 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Number of subjects analysed
    4
    13
    Units: score on a scale
    arithmetic mean (standard deviation)
        IGIV: Epoch 1: Baseline (Week 1): HSIS(n=4,13)
    0.8080 ( 0.04619 )
    0.8217 ( 0.04544 )
        IGIV: Epoch 1: Baseline (Week 1): EQVAS(n=4,13)
    87.5 ( 15.55 )
    77.1 ( 22.16 )
        IGSC: Epoch 2: CFB (Week 1): HSIS (n=3,13)
    -0.0270 ( 0.12275 )
    0.0016 ( 0.03318 )
        IGSC: Epoch 2: CFB (Week 1): EQVAS (n=3,13)
    -5.0 ( 5.00 )
    4.2 ( 17.15 )
        IGSC: Epoch 3:CFB (Week 1): HSIS (n=1,6)
    0.0000 ( 999 )
    0.0133 ( 0.03266 )
        IGSC: Epoch 3:CFB (Week 1): EQVAS (n=1,6)
    0.0 ( 999 )
    -1.3 ( 12.39 )
        IGSC: Epoch 3:CFB (EOS/ET):HSIS(n=4,13)
    0.0400 ( 0.04619 )
    0.0229 ( 0.04566 )
        IGSC: Epoch 3:CFB (EOS/ET): EQVAS(n=4,13)
    1.3 ( 6.29 )
    6.9 ( 13.21 )
    No statistical analyses for this end point

    Secondary: Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score

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    End point title
    		 Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score
    End point description
    SF-36=generic quality-of-life instrument that has been widely used to assess HRQL of participants.In this study, 14 years and older (participant as observer) for SF-36 health questionnaire was analyzed. Generic instruments are used in general populations to assess a wide range of domains applicable to a variety of health states, conditions, and diseases.SF-36=36 items aggregated into 8 multi-item scales (physical functioning, role - physical, bodily pain, general health, vitality, social functioning, role - emotional, and mental health), with scores ranging from 0 to 100.Higher scores=better HRQL. As pre-specified in protocol data is reported for participants with age group of 14 years or older.All-Treated Set included all enrolled participants of age ’14 years and above’ who received at least 1 dose of study drug (IGIV or IGSC).‘n’=Number analysed are participants with data available for analysis.Physical Component Summary Score (PCSS), Mental Component Summary Score (MCSS).
    End point type
    Secondary
    End point timeframe
    Baseline up to end of the study (approximately 1.5 years)
    End point values
    		 Age ≥14 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Number of subjects analysed
    11
    Units: score on a scale
    arithmetic mean (standard deviation)
        IGIV: Epoch 1: Baseline (Week 1): PCSS(n=11)
    50.21 ( 7.282 )
        IGIV: Epoch 1: Baseline (Week 1): MCSS(n=11)
    50.59 ( 8.114 )
        IGSC: Epoch 2: CFB (Week 1): PCSS(n=11)
    0.10 ( 7.142 )
        IGSC: Epoch 2: CFB (Week 1): MCSS(n=11)
    -0.30 ( 2.294 )
        IGSC: Epoch 3: CFB (Week 1): PCSS(n=5)
    1.81 ( 8.189 )
        IGSC: Epoch 3: CFB (Week 1): MCSS (n=5)
    3.60 ( 5.352 )
        IGSC: Epoch 3:CFB (EOS/ET):PCSS(n=10)
    0.23 ( 4.649 )
        IGSC: Epoch 3:CFB (EOS/ET):MCSS(n=10)
    4.32 ( 8.185 )
    No statistical analyses for this end point

    Secondary: Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score

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    End point title
    		 Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score
    End point description
    TSQM=is a global satisfaction scale used to assess the overall level of participant's satisfaction or dissatisfaction with their medications. In this study, 2-12 years (parent as observer), 13 years and older (participant as observer) for TSQM health questionnaire will be analyzed. TSQM-9 is a 9-item, validated, self-administered instrument used to assess participant's satisfaction with medication. The three domains assessed are effectiveness, convenience, and global satisfaction. The score of each of the 3 domains is based on an algorithm to create a score of 0 to 100. Higher score indicated greater satisfaction in that domain. As pre-specified in protocol, data is reported as per age group (2-12 years and >=13 years). All-Treated Set included all enrolled participants of age group ‘ 2-12 years’ and ‘>=13 years’ who received at least 1 dose of study drug (IGIV or IGSC). ‘n’=Number analysed are participants with data available for analysis.
    End point type
    Secondary
    End point timeframe
    Baseline up to end of the study (approximately 1.5 years)
    End point values
    		 Age 2-12years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age ≥13 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Number of subjects analysed
    6
    11
    Units: score on a scale
    arithmetic mean (standard deviation)
        IGIV:Epoch1:Baseline(Week1):Effectiveness(n=6,11)
    69.44 ( 14.380 )
    75.25 ( 18.817 )
        IGIV:Epoch 1:Baseline(Week 1):Convenience(n=6,11)
    62.96 ( 13.907 )
    65.66 ( 23.016 )
        IGIV: Epoch 1: Baseline (Week 1): GS(n=6,11)
    75.00 ( 17.928 )
    74.68 ( 18.173 )
        IGSC: Epoch 2: CFB (Week 1): Effectiveness(n=5,11)
    2.22 ( 9.296 )
    -1.52 ( 10.568 )
        IGSC: Epoch 2: CFB (Week 1): Convenience (n=5,11)
    3.33 ( 16.942 )
    5.05 ( 13.484 )
        IGSC: Epoch 2: CFB (Week 1): GS (n=5,11)
    -2.86 ( 10.833 )
    1.30 ( 10.507 )
        IGSC: Epoch 3: CFB (Week 1): Effectiveness (n=2,5)
    2.78 ( 3.928 )
    -10.00 ( 52.673 )
        IGSC: Epoch 3: CFB (Week 1): Convenience (n=2,5)
    13.89 ( 19.642 )
    15.56 ( 12.669 )
        IGSC: Epoch 3: CFB (Week 1): GS (n=2,5)
    -7.14 ( 10.02 )
    4.29 ( 29.709 )
        IGSC:Epoch 3:CFB (EOS/ET):Effectiveness(n=6,11)
    9.26 ( 10.924 )
    6.57 ( 18.892 )
        IGSC: Epoch 3:CFB (EOS/ET): Convenience (n=6,11)
    5.56 ( 22.222 )
    9.09 ( 23.210 )
        IGSC: Epoch 3: CFB (EOS/ET): GS(n=6,11)
    7.14 ( 23.905 )
    3.90 ( 23.768 )
    No statistical analyses for this end point

    Secondary: Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score

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    End point title
    		 Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score
    End point description
    LQI=self-administered questionnaire developed specifically for participants/legal guardians involved in IGIV treatments.2-13 years (parent as observer),14 years and older (participant as observer) for LQI health questionnaire was analyzed.LQI=15-items, divided into 4 domains: treatment interferences(TI)[6 items],therapy-related problems(TRP)[4 items],therapy setting(TS)[3 items];treatment costs(TC)[2 items].Items are rated on a 7-point Likert-type scale ranging from 1:"Extremely bad" to 7:"Extremely good".Total scores=0 to 100,higher scores=highest possible satisfaction with factors such as independence,therapy convenience,social/school/work activities;health and travel costs.As pre-specified in protocol, data is reported as per age (2-13 years and >=14 years).All-Treated Set included all enrolled participants of age group ‘2-13 years’ and ‘>=14 years’ who received at least 1 dose of study drug (IGIV or IGSC).‘n’=Number analysed are participants with data available for analysis.
    End point type
    Secondary
    End point timeframe
    Baseline up to end of the study (approximately 1.5 years)
    End point values
    		 Age 2-13years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg Age ≥14 years:Epoch 1+2+3[IGIV200-600+IGSC50-100+100-400]mg/kg
    Number of subjects analysed
    6
    11
    Units: score on a scale
    arithmetic mean (standard deviation)
        IGIV:Epoch 1:Baseline(Week 1):Factor 1 TI(n=6,11)
    81.48 ( 14.873 )
    77.02 ( 17.963 )
        IGIV:Epoch1:Baseline(Week1):Factor 1 TRP(n=6,11)
    81.94 ( 15.516 )
    79.17 ( 15.811 )
        IGIV:Epoch 1:Baseline(Week 1):Factor 1 TS(n=6,11)
    77.78 ( 20.488 )
    74.75 ( 23.748 )
        IGIV:Epoch 1:Baseline(Week 1):Factor 1 TC(n=6,11)
    65.28 ( 24.391 )
    53.79 ( 21.847 )
        IGSC: Epoch 2: CFB (Week 1): Factor 1 TI(n=6,11)
    -8.33 ( 21.445 )
    -2.53 ( 14.351 )
        IGSC: Epoch 2: CFB (Week 1): Factor 1 TRP(n=6,11)
    2.78 ( 9.742 )
    -1.52 ( 15.841 )
        IGSC: Epoch 2: CFB (Week 1): Factor 1 TS(n=6,11)
    -6.48 ( 30.106 )
    0.51 ( 11.773 )
        IGSC: Epoch 2: CFB (Week 1): Factor 1 TC(n=6,11)
    -9.72 ( 22.618 )
    0.76 ( 31.282 )
        IGSC: Epoch 3: CFB (Week 1): Factor 1 TI (n=5,5)
    4.17 ( 1.964 )
    3.33 ( 4.120 )
        IGSC: Epoch 3: CFB (Week 1): Factor 1 TRP (n=5,5)
    -14.58 ( 14.731 )
    1.67 ( 14.613 )
        IGSC: Epoch 3: CFB (Week 1): Factor 1 TS (n=5,5)
    19.44 ( 3.928 )
    12.22 ( 16.387 )
        IGSC: Epoch 3: CFB (Week 1): Factor 1 TC (n=5,5)
    0.00 ( 0.000 )
    18.33 ( 40.139 )
        IGSC:Epoch3:CFB(Week13[EOS/ET]):Factor1 TI(n=6,11)
    -2.78 ( 13.494 )
    -0.25 ( 13.046 )
        IGSC:Epoch3:CFB(Week13[EOS/ET]):Factor1TRP(n=6,11)
    -1.39 ( 9.001 )
    1.14 ( 9.334 )
        IGSC:Epoch3:CFB(Week 13[EOS/ET]):Factor1TS(n=6,11)
    2.78 ( 16.005 )
    4.55 ( 16.067 )
        IGSC:Epoch3:CFB(Week 13[EOS/ET]):Factor1TC(n=6,11)
    4.17 ( 16.672 )
    14.39 ( 26.898 )
    No statistical analyses for this end point

    Secondary: Health Related Quality of Life: Treatment Preference

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    End point title
    		 Health Related Quality of Life: Treatment Preference
    End point description
    Treatment preference questionnaire is a self-administered questionnaire developed to assess participants' preference towards the administration of new IGSC therapy. There are 4-items on the questionnaire, which investigate a participant's preference on the clinic/hospital/home setting of receiving the immunoglobulin therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the IGSC treatment. The questionnaire included following categories: Where do you prefer to receive your immunoglobulin therapy, The frequency of administration, As pre-specified in protocol, data is reported as per age (2-13 years and >=14 years).
    End point type
    Secondary
    End point timeframe
    Up to approximately 1.5 years
    End point values
    		 IGSC: Epoch 2: 2-13 years IGSC: Epoch 2: >=14 years IGSC: Epoch 3: 2-13 years IGSC: Epoch 3: >=14 years
    Number of subjects analysed
    4
    6
    2
    7
    Units: participants
        Where do you prefer to receive your IG TH:At home
    3
    1
    1
    4
        The frequency of administration: Like very much
    2
    1
    1
    4
        The frequency of administration: Like
    1
    3
    1
    1
    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 drug up to end of treatment (up to Week 57)
    Adverse event reporting additional description
    At each visit the investigator had to document any occurrence of adverse events and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to study treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Epoch 1: IGIV 200-600 mg/kg
    Reporting group description
    		 Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 3 or 4 weeks for up to 13 weeks.

    Reporting group title
    Epoch 3: IGSC (20%) 100-400 mg/kg
    Reporting group description
    		 Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to approximately 12 weeks after Epoch 2.

    Reporting group title
    Epoch 2: IGSC (20%) 50-200 mg/kg
    Reporting group description
    		 Participants who entered to Epoch 2 from Epoch 1 received 50-200 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion once a week up to approximately 24 weeks after Epoch 1.

    Serious adverse events
    		 Epoch 1: IGIV 200-600 mg/kg Epoch 3: IGSC (20%) 100-400 mg/kg Epoch 2: IGSC (20%) 50-200 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Epoch 1: IGIV 200-600 mg/kg Epoch 3: IGSC (20%) 100-400 mg/kg Epoch 2: IGSC (20%) 50-200 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    11 / 17 (64.71%)
    3 / 7 (42.86%)
    16 / 17 (94.12%)
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Occult blood positive
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Seborrhoeic keratosis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Colon adenoma
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Injury, poisoning and procedural complications
    Vaccination complication
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 7 (14.29%)
    1 / 17 (5.88%)
         occurrences all number
    0
    1
    2
    Ligament sprain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Traumatic arthropathy
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Wound
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 17 (17.65%)
    1 / 7 (14.29%)
    3 / 17 (17.65%)
         occurrences all number
    7
    1
    8
    Orthostatic intolerance
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Sciatica
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    2
    Injection site pain
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    2
    Injection site swelling
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 7 (14.29%)
    3 / 17 (17.65%)
         occurrences all number
    0
    1
    7
    Injection site erythema
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 7 (14.29%)
    3 / 17 (17.65%)
         occurrences all number
    0
    1
    4
    Infusion site erythema
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    4
    Infusion site bruising
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Chills
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Administration site swelling
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Administration site pain
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    3
    Administration site discolouration
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Vaccination site pain
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    2
    Puncture site pain
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Vaccination site joint erythema
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Malaise
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    2
    Injection site reaction
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Injection site bruising
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Infusion site swelling
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    7
    Infusion site pruritus
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    4
    Infusion site pain
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Eye disorders
    Myopia
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Gastrointestinal disorders
    Dental caries
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Stomatitis
         subjects affected / exposed
    3 / 17 (17.65%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    4
    0
    1
    Constipation
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Nausea
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Diarrhoea
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    2
    0
    1
    Toothache
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Haemorrhoids
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Dermatitis atopic
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Eczema asteatotic
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Miliaria
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Urticaria
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Seborrhoeic dermatitis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Rash pruritic
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    2
    Arthritis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Back pain
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    2
    0
    0
    Pain in jaw
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Infections and infestations
    Sinusitis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    3
    Gastroenteritis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    0
    0
    2
    Conjunctivitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    2 / 17 (11.76%)
         occurrences all number
    1
    0
    2
    Nasopharyngitis
         subjects affected / exposed
    2 / 17 (11.76%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    2
    0
    2
    Bronchitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    1
    0
    1
    Cystitis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Hordeolum
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Periodontitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Laryngitis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Oral herpes
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Otitis externa
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Parotitis
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Impetigo
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Pharyngitis
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 7 (0.00%)
    0 / 17 (0.00%)
         occurrences all number
    1
    0
    0
    Pneumonia
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1
    Pulpitis dental
         subjects affected / exposed
    0 / 17 (0.00%)
    0 / 7 (0.00%)
    1 / 17 (5.88%)
         occurrences all number
    0
    0
    1

    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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    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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