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A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)

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ClinicalTrials.gov Identifier: NCT04346108
Recruitment Status : Completed
First Posted : April 15, 2020
Results First Posted : March 22, 2024
Last Update Posted : March 22, 2024
Sponsor:
Information provided by (Responsible Party):
Takeda ( Baxalta now part of Shire )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Primary Immunodeficiency Diseases (PID)
Interventions Biological: Immune Globulin Intravenous (IGIV)
Biological: Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
Enrollment 17
Recruitment Details Participants took part in the study at 8 investigative sites in Japan from 11 August 2020 to 22 December 2021.
Pre-assignment 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.
Arm/Group Title Epoch 1: IGIV 200-600 mg/kg Epoch 2: IGSC (20%) 50-200 mg/kg Epoch 3: IGSC (20%) 100-400 mg/kg
Hide Arm/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. 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. 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.
Period Title: Epoch 1 (13 Weeks)
Started 17 0 0
Completed 17 0 0
Not Completed 0 0 0
Period Title: Epoch 2 (24 Weeks After Epoch 1)
Started 0 17 [1] 0
Completed 0 15 0
Not Completed 0 2 0
Reason Not Completed
Withdrawal by Parent/Guardian             0             1             0
Withdrawal by Subject             0             1             0
[1]
Those participants who received at least 1 dose of study drug (IGIV) in Epoch 1 and entered Epoch 2.
Period Title: Epoch 3 (12 Weeks After Epoch 2)
Started 0 0 7 [1]
Completed 0 0 7
Not Completed 0 0 0
[1]
Those participants who received at least 1 dose of study drug (IGIV or IGSC) in Epoch 1 and Epoch 2 entered Epoch 3.
Arm/Group Title IGIV 200 to 600 mg/kg + IGSC [50 to 200 and 100 to 400] mg/kg
Hide Arm/Group 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.
Overall Number of Baseline Participants 17
Hide Baseline Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 17 participants
31.0  (21.13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
Female
7
  41.2%
Male
10
  58.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
17
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
American Indian or Alaska Native
0
   0.0%
Asian
17
 100.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
0
   0.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Weight  
Mean (Standard Deviation)
Unit of measure:  Kilogram (kg)
Number Analyzed 17 participants
48.16  (18.120)
Height  
Mean (Standard Deviation)
Unit of measure:  Centimeter (cm)
Number Analyzed 17 participants
152.15  (20.076)
Body Mass Index (BMI)   [1] 
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 17 participants
19.88  (3.242)
[1]
Measure Description: BMI is calculated as [weight (kg) / height(m)^2]
1.Primary Outcome
Title Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2
Hide Description Total serum trough levels of IgG antibodies measured during period 2 of Epoch 2 were assessed.
Time Frame Epoch 2 (period 2): Up to 24 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic Analysis Set (PKAS) 1 included all participants in the all-treated set who have had at least 1 evaluable serum IgG concentration value and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Only PKAS 1 participants in Epoch 2 were analyzed for this outcome measure.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 17
Geometric Mean (95% Confidence Interval)
Unit of Measure: grams per liter (g/L)
8.56
(8.03 to 9.12)
2.Primary Outcome
Title Epoch 3: Total Serum Trough Levels of IgG Antibodies
Hide Description Total serum trough levels of IgG antibodies measured during Epoch 3 were assessed.
Time Frame Epoch 3: Up to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 1 included all participants in the all-treated set who have had at least 1 evaluable serum IgG concentration value and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Only PKAS 1 participants in Epoch 3 were analyzed for this outcome measure.
Arm/Group Title Epoch 3: IGSC (20%) 100-400 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: g/L
8.39
(7.89 to 8.91)
3.Secondary Outcome
Title Epoch 1: Total Serum Trough Levels of IgG Antibodies
Hide Description Total serum trough levels of IgG antibodies measured during Epoch 1 were assessed.
Time Frame Epoch 1: Up to Week 13
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 1 included all participants in the all-treated set who have had at least 1 evaluable serum IgG concentration value and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Only PKAS 1 participants in Epoch 1 were analyzed for this outcome measure.
Arm/Group Title Epoch 1: IGIV 200-600 mg/kg
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
Overall Number of Participants Analyzed 17
Geometric Mean (95% Confidence Interval)
Unit of Measure: g/L
8.18
(7.71 to 8.68)
4.Secondary Outcome
Title Epoch 2: Area Under the Curve From Time 0 to Last Interval (AUC0-last) for Total Serum Levels of IgG
Hide Description [Not Specified]
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: g*day/L
58.9
(48.7 to 71.3)
5.Secondary Outcome
Title Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses
Hide Description Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: g*day/L
IgG 1
35.8
(32.4 to 39.5)
IgG 2
23.4
(20.4 to 26.9)
IgG 3
1.32
(0.624 to 2.80)
IgG 4
1.67
(1.10 to 2.53)
6.Secondary Outcome
Title Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG
Hide Description [Not Specified]
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: mL/kg/day
1.93
(1.71 to 2.19)
7.Secondary Outcome
Title Epoch 2: CL/F for Total Serum Levels of IgG Subclasses
Hide Description Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: mL/kg/day
IgG 1
3.18
(2.88 to 3.51)
IgG 2
4.86
(4.18 to 5.66)
IgG 3
99.5
(37.1 to 266)
IgG 4
68.3
(48.3 to 96.6)
8.Secondary Outcome
Title Epoch 2: Maximum Concentration (Cmax) for Total Serum Levels of IgG
Hide Description [Not Specified]
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: g/L
9.08
(7.46 to 11.1)
9.Secondary Outcome
Title Epoch 2: Cmax for Total Serum Levels of IgG Subclasses
Hide Description Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: g/L
IgG 1
5.40
(4.95 to 5.89)
IgG 2
3.51
(3.06 to 4.03)
IgG 3
0.202
(0.0960 to 0.425)
IgG 4
0.256
(0.169 to 0.387)
10.Secondary Outcome
Title Epoch 2: Minimum Concentration (Cmin) for Total Serum Levels of IgG
Hide Description [Not Specified]
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: g/L
7.45
(6.17 to 8.99)
11.Secondary Outcome
Title Epoch 2: Cmin for Total Serum Levels of IgG Subclasses
Hide Description Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: g/L
IgG 1
4.89
(4.34 to 5.51)
IgG 2
3.15
(2.70 to 3.68)
IgG 3
0.181
(0.0861 to 0.381)
IgG 4
0.218
(0.153 to 0.311)
12.Secondary Outcome
Title Epoch 2: Time to Maximum Concentration (Tmax) for Total Serum Levels of IgG
Hide Description [Not Specified]
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Median (Full Range)
Unit of Measure: hours
71.08
(22.82 to 168.23)
13.Secondary Outcome
Title Epoch 2: Tmax for Total Serum Levels of IgG Subclasses
Hide Description Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time Frame Epoch 2: Week 21
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 2 included all participants >=12 years of age in the all-treated set who have had at least 1 evaluable serum IgG concentration value during Epoch 2 and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants with data available for analyses.
Arm/Group Title Epoch 2: IGSC (20%) 50-200 mg/kg
Hide Arm/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.
Overall Number of Participants Analyzed 7
Median (Full Range)
Unit of Measure: hours
IgG 1
71.08
(22.82 to 118.80)
IgG 2
71.08
(0.00 to 164.32)
IgG 3
72.92
(71.08 to 118.80)
IgG 4
71.17
(70.13 to 118.80)
14.Secondary Outcome
Title Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV)
Hide Description Trough levels of specific antibodies to clinically relevant pathogen (Clostridium tetani toxoid and HBV) were assessed in Epoch 1, Epoch 2 and Epoch 3. Data was analyzed per interval in each Epoch for this outcome measure.
Time Frame Epoch 1 (Week 1); Epoch 2 (Week 1, 24); Epoch 3 (Week 1, 13)
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 1 included all participants in the all-treated set who have had at least 1 evaluable serum IgG concentration value and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants available for analyses. Data is represented as per doses received for specific interval in each Epoch for this outcome measure.
Arm/Group Title Epoch 1: IGIV 3-week Interval (Week 1) Epoch 1: IGIV 4-week Interval (Week 1) Epoch 2: IGSC, 20% Weekly (Week 1) Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) Epoch 3: IGSC, 20% Biweekly (Week 1) Epoch 3: IGSC, 20% Biweekly (Week 13)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 3 weeks for up to 13 weeks.
Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 4 weeks for up to 13 weeks.
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.
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 end of study (EOS) (24 weeks) or early termination (ET) after Epoch 1.
Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to EOS (13 weeks) after Epoch 2.
Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to EOS (13 weeks) after Epoch 2.
Overall Number of Participants Analyzed 5 12 17 10 7 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: International units (IU)/mL
Clostridium Tetani Toxoid
0.524
(0.290 to 0.949)
0.602
(0.364 to 0.997)
0.674
(0.468 to 0.970)
1.52
(1.11 to 2.08)
1.97
(1.65 to 2.35)
1.80
(1.35 to 2.39)
Hepatitis B Virus
27.7
(17.8 to 43.0)
35.2
(17.5 to 70.9)
54.1
(33.7 to 87.0)
486
(335 to 706)
645
(452 to 920)
923
(832 to 1020)
15.Secondary Outcome
Title Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB)
Hide Description Trough levels of specific antibodies to clinically relevant pathogens (HIB) were assessed in Epoch 1, Epoch 2, and Epoch 3. Data was analyzed per interval in each Epoch for this outcome measure.
Time Frame Epoch 1 (Week 1); Epoch 2 (Week 1, 24); Epoch 3 (Week 1, 13)
Hide Outcome Measure Data
Hide Analysis Population Description
PKAS 1 included all participants in the all-treated set who have had at least 1 evaluable serum IgG concentration value and have had no major protocol deviations or events that would affect the serum IgG concentration analysis results. Overall number analyzed are the number of participants available for analyses. Data is represented as per doses received for specific intervals in each Epoch for this outcome measure.
Arm/Group Title Epoch 1: IGIV 3-week Interval (Week 1) Epoch 1: IGIV 4-week Interval (Week 1) Epoch 2: IGSC, 20% Weekly (Week 1) Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) Epoch 3: IGSC, 20% Biweekly (Week 1) Epoch 3: IGSC, 20% Biweekly (Week 13)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 3 weeks for up to 13 weeks.
Participants received 200 to 600 mg/kg of Immunoglobulin Intravenous (IGIV) infusion for every 4 weeks for up to 13 weeks.
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.
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 EOS (24 weeks) or ET after Epoch 1.
Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to EOS (13 weeks) after Epoch 2.
Participants who entered to Epoch 3 from Epoch 2 received 100-400 mg/kg of Immune Globulin Subcutaneous (Human) 20% infusion biweekly up to EOS (13 weeks) after Epoch 2.
Overall Number of Participants Analyzed 5 12 17 10 7 7
Geometric Mean (95% Confidence Interval)
Unit of Measure: mg/L
1.58
(0.568 to 4.38)
1.55
(1.11 to 2.18)
2.50
(1.95 to 3.20)
1.77
(1.07 to 2.91)
2.04
(1.74 to 2.39)
1.73
(1.46 to 2.04)
16.Secondary Outcome
Title Health Related Quality of Life: Treatment Preference
Hide 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).
Time Frame Up to approximately 1.5 years
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants of age '2-13 years' and '>=14 years' who received at least 1 dose of study drug (IGIV or IGSC). Number analyzed is the number of participants with data available for analyses at specific timepoints.
Arm/Group Title IGSC: Epoch 2: 2-13 Years IGSC: Epoch 2: >=14 Years IGSC: Epoch 3: 2-13 Years IGSC: Epoch 3: >=14 Years
Hide Arm/Group 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 Epoch 2.
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.
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.
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.
Overall Number of Participants Analyzed 4 6 2 5
Measure Type: Count of Participants
Unit of Measure: Participants
Where do you Prefer to Receive Your Immunoglobulin Therapy: At Hospital 0 0 0 1
Where do you Prefer to Receive Your Immunoglobulin Therapy: At Home 3 1 1 4
Where do you Prefer to Receive Your Immunoglobulin Therapy: No Preference 1 5 1 0
The Frequency of Administration: Like Very Much 2 1 1 4
The Frequency of Administration: Like 1 3 1 1
The Frequency of Administration: No Preference 1 2 0 0
The Frequency of Administration: Dislike Very Much 0 1 0 0
17.Secondary Outcome
Title Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Hide 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.
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Measure Type: Count of Participants
Unit of Measure: Participants
11
  64.7%
16
  94.1%
3
  42.9%
18.Secondary Outcome
Title Number of Participants With Tolerability Events Related to the Infusion of Study Drug
Hide 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 were assessed.
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Measure Type: Count of Participants
Unit of Measure: Participants
Infusions With Infusion Rate Reduced
0
   0.0%
0
   0.0%
0
   0.0%
Infusions That Were Interrupted
0
   0.0%
1
   5.9%
0
   0.0%
Infusions That Were Stopped
0
   0.0%
0
   0.0%
0
   0.0%
Infusions With Infusion Rate Reduced or Interrupted or Stopped
0
   0.0%
1
   5.9%
0
   0.0%
19.Secondary Outcome
Title Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
Hide 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.
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Mean (Standard Deviation)
Unit of Measure: number of infections per year
0.0  (0.00) 0.25  (1.042) 0.0  (0.00)
20.Secondary Outcome
Title Annual Rate of All Infections Per Year
Hide Description Annual rate is the number of participants reporting any infection per year.
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Mean (Standard Deviation)
Unit of Measure: number of infections per year
1.65  (2.479) 2.78  (3.074) 0.00  (0.000)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 1.65
Confidence Interval (2-Sided) 95%
0.73 to 3.15
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 0.00
Confidence Interval (2-Sided) 95%
0.00 to 0.00
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 2.48
Confidence Interval (2-Sided) 95%
1.34 to 4.13
Estimation Comments [Not Specified]
21.Secondary Outcome
Title Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection
Hide 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.
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Median (Full Range)
Unit of Measure: days
0.00
(0.0 to 16.1)
0.00
(0.0 to 17.2)
4.30
(0.0 to 38.7)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 2.60
Confidence Interval (2-Sided) 95%
1.02 to 5.30
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 9.82
Confidence Interval (2-Sided) 95%
2.82 to 23.82
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 2.87
Confidence Interval (2-Sided) 95%
1.37 to 5.18
Estimation Comments [Not Specified]
22.Secondary Outcome
Title Number of Days Participants Were on Antibiotics
Hide 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.
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Median (Full Range)
Unit of Measure: days
0.00
(0.0 to 31.8)
0.00
(0.0 to 34.5)
0.00
(0.0 to 21.5)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 4.01
Confidence Interval 95%
1.46 to 8.54
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 3.07
Confidence Interval (2-Sided) 95%
0.37 to 10.74
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 5.87
Confidence Interval (2-Sided) 95%
2.32 to 11.93
Estimation Comments [Not Specified]
23.Secondary Outcome
Title Number of Participants Hospitalized Due to Illness or Infection
Hide 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.
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 0.00
Confidence Interval (2-Sided) 95%
0.0 to 0.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 0.13
Confidence Interval (2-Sided) 95%
0.03 to 0.35
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 0.00
Confidence Interval (2-Sided) 95%
0.00 to 0.00
Estimation Comments [Not Specified]
24.Secondary Outcome
Title Length of Hospital Stay
Hide 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.
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Median (Full Range)
Unit of Measure: days
0.00
(0.0 to 0.0)
0.00
(0.0 to 17.4)
0.00
(0.0 to 0.0)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 0.00
Confidence Interval (2-Sided) 95%
0.00 to 0.00
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 1.04
Confidence Interval (2-Sided) 95%
0.25 to 2.76
Estimation Comments [Not Specified]
25.Secondary Outcome
Title Number of Acute Physician Visits Due to Illness/Infection
Hide Description Number of acute physician visits is standardized to per year (365.25 days).
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
Arm/Group Title Epoch 1: Immune Globulin Intravenous (IGIV) Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC) Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Hide Arm/Group Description:
Participants received 200 to 600 mg/kg of IGIV infusion for every 3 or 4 weeks for up to 13 weeks.
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.
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.
Overall Number of Participants Analyzed 17 17 7
Mean (Standard Deviation)
Unit of Measure: number of visits per year
1.18  (3.098) 3.30  (6.807) 1.624  (8.121)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 1.18
Confidence Interval (2-Sided) 95%
0.38 to 2.68
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 2.35
Confidence Interval (2-Sided) 95%
0.91 to 4.82
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Epoch 1: Immune Globulin Intravenous (IGIV), Epoch 2: Immune Globulin Subcutaneous 20% Solution (IGSC), Epoch 3: Immune Globulin Subcutaneous 20% Solution (IGSC)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Poisson Estimate
Estimated Value 0.61
Confidence Interval (2-Sided) 95%
0.07 to 2.15
Estimation Comments [Not Specified]
26.Secondary Outcome
Title Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score
Hide 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.
Time Frame Baseline up to end of study (approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included 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). Number analyzed are the number of participants with data available for analysis at the given timepoint.
Arm/Group Title Age 2-7 Years: Epoch 1[IGIV 200-600 mg/kg]+Epoch 2 [IGSC 50-100 mg/kg]+Epoch 3 [IGSC 100-400 mg/kg] Age 8-13 Years: Epoch 1[IGIV 200-600 mg/kg]+Epoch 2 [IGSC 50-100 mg/kg]+Epoch 3 [IGSC 100-400 mg/kg]
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 2 4
Mean (Standard Deviation)
Unit of Measure: score on a scale
IGIV: Epoch 1: Baseline (Week 1) Number Analyzed 2 participants 4 participants
88.04  (13.835) 87.23  (11.820)
IGSC: Epoch 2: Change from Baseline (Week 1) Number Analyzed 2 participants 4 participants
9.78  (15.372) -6.79  (9.977)
IGSC: Epoch 3: Change from Baseline (Week 1) Number Analyzed 1 participants 1 participants
14.13 [1]   (NA) 20.65 [2]   (NA)
IGSC: Epoch 3: Change from Baseline (EOS/ET]) Number Analyzed 2 participants 4 participants
3.26  (7.686) 4.08  (16.374)
[1]
Standard deviation was not estimable for 1 participant
[2]
Standard deviation was not estimable due to 1 participant.
27.Secondary Outcome
Title EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score
Hide 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).
Time Frame Baseline up to end of the study (approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
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). Number analyzed is the number of participants with data available for analyses at specific timepoints
Arm/Group Title Age 2-11 Years: Epoch 1[IGIV 200-600 mg/kg]+Epoch 2 [IGSC 50-100 mg/kg]+Epoch 3 [IGSC 100-400 mg/kg] Age >=12 Years: Epoch 1[IGIV 200-600 mg/kg]+Epoch 2 [IGSC 50-100 mg/kg]+Epoch 3 [IGSC 100-400 mg/kg]
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 4 13
Mean (Standard Deviation)
Unit of Measure: score on a scale
IGIV: Epoch 1: Baseline (Week 1): Health State Index Score Number Analyzed 4 participants 13 participants
0.8080  (0.04619) 0.8217  (0.04544)
IGIV: Epoch 1: Baseline (Week 1): EQ Visual Analogue Scale Number Analyzed 4 participants 13 participants
87.5  (15.55) 77.1  (22.16)
IGSC: Epoch 2: Change from Baseline (Week 1): Health State Index Score Number Analyzed 3 participants 13 participants
-0.0270  (0.12275) 0.0016  (0.03318)
IGSC: Epoch 2: Change from Baseline (Week 1): EQ Visual Analogue Scale Number Analyzed 3 participants 13 participants
-5.0  (5.00) 4.2  (17.15)
IGSC: Epoch 3: Change from Baseline (Week 1): Health State Index Score Number Analyzed 1 participants 6 participants
0.0000 [1]   (NA) 0.0133  (0.03266)
IGSC:Epoch 3: Change from Baseline (Week 1): EQ Visual Analogue Scale Number Analyzed 1 participants 6 participants
0.0 [1]   (NA) -1.3  (12.39)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Health State Index Score Number Analyzed 4 participants 13 participants
0.0400  (0.04619) 0.0229  (0.04566)
IGSC: Epoch 3: Change from Baseline (EOS/ET): EQ Visual Analogue Scale Number Analyzed 4 participants 13 participants
1.3  (6.29) 6.9  (13.21)
[1]
Standard deviation was not estimable for 1 participant
28.Secondary Outcome
Title Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score
Hide 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.
Time Frame Baseline up to end of the study (approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
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). Number analyzed is the number of participants with data available for analyses at specific timepoints.
Arm/Group Title Age 14 Years and Above: IGIV 200 to 600 mg/kg + IGSC (20%) 50 to 200 mg/kg and 100 to 400 mg/kg
Hide Arm/Group 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.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: score on a scale
IGIV: Epoch 1: Baseline (Week 1): Physical Component Summary Score Number Analyzed 11 participants
50.21  (7.282)
IGIV: Epoch 1: Baseline (Week 1): Mental Component Summary Score Number Analyzed 11 participants
50.59  (8.114)
IGSC: Epoch 2: Change from Baseline (Week 1): Physical Component Summary Score Number Analyzed 11 participants
0.10  (7.142)
IGSC: Epoch 2: Change from Baseline (Week 1): Mental Component Summary Score Number Analyzed 11 participants
-0.30  (2.294)
IGSC: Epoch 3: Change from Baseline (Week 1): Physical Component Summary Score Number Analyzed 5 participants
1.81  (8.189)
IGSC: Epoch 3: Change from Baseline (Week 1): Mental Component Summary Score Number Analyzed 5 participants
3.60  (5.352)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Physical Component Summary Score Number Analyzed 10 participants
0.23  (4.649)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Mental Component Summary Score Number Analyzed 10 participants
4.32  (8.185)
29.Secondary Outcome
Title Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score
Hide 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).
Time Frame Baseline up to end of the study (approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All-Treated Set included all enrolled participants of age group ' 2-12 years' and '>=3 years' who received at least 1 dose of study drug (IGIV or IGSC). Number analyzed is the number of participants with data available for analyses at specific timepoints.
Arm/Group Title Age 2-12 Years: Epoch 1[IGIV 200-600 mg/kg]+Epoch 2 [IGSC 50-100 mg/kg]+Epoch 3 [IGSC 100-400 mg/kg] Age >=13 Years: Epoch 1[IGIV 200-600 mg/kg]+Epoch 2 [IGSC 50-100 mg/kg]+Epoch 3 [IGSC 100-400 mg/kg]
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 6 11
Mean (Standard Deviation)
Unit of Measure: score on a scale
IGIV: Epoch 1: Baseline (Week 1): Effectiveness Number Analyzed 6 participants 11 participants
69.44  (14.380) 75.25  (18.817)
IGIV: Epoch 1: Baseline (Week 1): Convenience Number Analyzed 6 participants 11 participants
62.96  (13.907) 65.66  (23.016)
IGIV: Epoch 1: Baseline (Week 1): Global Satisfaction Number Analyzed 6 participants 11 participants
75.00  (17.928) 74.68  (18.173)
IGSC: Epoch 2: Change from Baseline (Week 1): Effectiveness Number Analyzed 5 participants 11 participants
2.22  (9.296) -1.52  (10.568)
IGSC: Epoch 2: Change from Baseline (Week 1): Convenience Number Analyzed 5 participants 11 participants
3.33  (16.942) 5.05  (13.484)
IGSC: Epoch 2: Change from Baseline (Week 1): Global Satisfaction Number Analyzed 5 participants 11 participants
-2.86  (10.833) 1.30  (10.507)
IGSC: Epoch 3: Change from Baseline (Week 1): Effectiveness Number Analyzed 2 participants 5 participants
2.78  (3.928) -10.00  (52.673)
IGSC: Epoch 3: Change from Baseline (Week 1): Convenience Number Analyzed 2 participants 5 participants
13.89  (19.642) 15.56  (12.669)
IGSC: Epoch 3: Change from Baseline (Week 1): Global Satisfaction Number Analyzed 2 participants 5 participants
-7.14  (10.02) 4.29  (29.709)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Effectiveness Number Analyzed 6 participants 11 participants
9.26  (10.924) 6.57  (18.892)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Convenience Number Analyzed 6 participants 11 participants
5.56  (22.222) 9.09  (23.210)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Global Satisfaction Number Analyzed 6 participants 11 participants
7.14  (23.905) 3.90  (23.768)
30.Secondary Outcome
Title Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score
Hide 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.
Time Frame Baseline up to end of the study (approximately 1.5 years)
Hide Outcome Measure Data
Hide Analysis Population Description
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). Number analyzed is the number of participants with data available for analyses at specific timepoints.
Arm/Group Title Age 2-13 Years: Epoch 1[IGIV 200-600 mg/kg]+Epoch 2 [IGSC 50-100 mg/kg]+Epoch 3 [IGSC 100-400 mg/kg] Age >=14 Years: Epoch 1[IGIV 200-600 mg/kg]+Epoch 2 [IGSC 50-100 mg/kg]+Epoch 3 [IGSC 100-400 mg/kg]
Hide Arm/Group 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.
Participants aged >=14 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.
Overall Number of Participants Analyzed 6 11
Mean (Standard Deviation)
Unit of Measure: score on a scale
IGIV: Epoch 1: Baseline (Week 1): Factor 1 Treatment Interference Number Analyzed 6 participants 11 participants
81.48  (14.873) 77.02  (17.963)
IGIV: Epoch 1: Baseline (Week 1): Factor 2 Therapy-Related Problems Number Analyzed 6 participants 11 participants
81.94  (15.516) 79.17  (15.811)
IGIV: Epoch 1: Baseline (Week 1): Factor 3 Therapy Setting Number Analyzed 6 participants 11 participants
77.78  (20.488) 74.75  (23.748)
IGIV: Epoch 1: Baseline (Week 1): Factor 4 Treatment Costs Number Analyzed 6 participants 11 participants
65.28  (24.391) 53.79  (21.847)
IGSC: Epoch 2: Change from Baseline (Week 1): Factor 1 Treatment Interference Number Analyzed 6 participants 11 participants
-8.33  (21.445) -2.53  (14.351)
IGSC: Epoch 2: Change from Baseline (Week 1): Factor 2 Therapy-Related Problems Number Analyzed 6 participants 11 participants
2.78  (9.742) -1.52  (15.841)
IGSC: Epoch 2: Change from Baseline (Week 1): Factor 3 Therapy Setting Number Analyzed 6 participants 11 participants
-6.48  (30.106) 0.51  (11.773)
IGSC: Epoch 2: Change from Baseline (Week 1): Factor 4 Treatment Costs Number Analyzed 6 participants 11 participants
-9.72  (22.618) 0.76  (31.282)
IGSC: Epoch 3: Change from Baseline (Week 1): Factor 1 Treatment Interference Number Analyzed 5 participants 5 participants
4.17  (1.964) 3.33  (4.120)
IGSC: Epoch 3: Change from Baseline (Week 1): Factor 2 Therapy-Related Problems Number Analyzed 5 participants 5 participants
-14.58  (14.731) 1.67  (14.613)
IGSC: Epoch 3: Change from Baseline (Week 1): Factor 3 Therapy Setting Number Analyzed 5 participants 5 participants
19.44  (3.928) 12.22  (16.387)
IGSC: Epoch 3: Change from Baseline (Week 1): Factor 4 Treatment Costs Number Analyzed 5 participants 5 participants
0.00  (0.000) 18.33  (40.139)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 1 Treatment Interference Number Analyzed 6 participants 11 participants
-2.78  (13.494) -0.25  (13.046)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 2 Therapy-Related Problems Number Analyzed 6 participants 11 participants
-1.39  (9.001) 1.14  (9.334)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 3 Therapy Setting Number Analyzed 6 participants 11 participants
2.78  (16.005) 4.55  (16.067)
IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 4 Treatment Costs Number Analyzed 6 participants 11 participants
4.17  (16.672) 14.39  (26.898)
Time Frame From first dose of study drug up to end of study (up to approximately 1.5 years)
Adverse Event Reporting 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.
 
Arm/Group Title Epoch 1: IGIV 200-600 mg/kg Epoch 2: IGSC (20%) 50-200 mg/kg Epoch 3: IGSC (20%) 100-400 mg/kg
Hide Arm/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. 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. 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.
All-Cause Mortality
Epoch 1: IGIV 200-600 mg/kg Epoch 2: IGSC (20%) 50-200 mg/kg Epoch 3: IGSC (20%) 100-400 mg/kg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/17 (0.00%)      0/17 (0.00%)      0/7 (0.00%)    
Hide Serious Adverse Events
Epoch 1: IGIV 200-600 mg/kg Epoch 2: IGSC (20%) 50-200 mg/kg Epoch 3: IGSC (20%) 100-400 mg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/17 (0.00%)      0/17 (0.00%)      0/7 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Epoch 1: IGIV 200-600 mg/kg Epoch 2: IGSC (20%) 50-200 mg/kg Epoch 3: IGSC (20%) 100-400 mg/kg
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   11/17 (64.71%)      16/17 (94.12%)      3/7 (42.86%)    
Eye disorders       
Myopia  1  0/17 (0.00%)  0 1/17 (5.88%)  1 0/7 (0.00%)  0
Gastrointestinal disorders       
Stomatitis  1  3/17 (17.65%)  1/17 (5.88%)  0/7 (0.00%) 
Dental caries  1  1/17 (5.88%)  1/17 (5.88%)  0/7 (0.00%) 
Diarrhoea  1  1/17 (5.88%)  1/17 (5.88%)  0/7 (0.00%) 
Nausea  1  1/17 (5.88%)  1/17 (5.88%)  0/7 (0.00%) 
Constipation  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Gastrooesophageal reflux disease  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Haemorrhoids  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Toothache  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
General disorders       
Injection site swelling  1  0/17 (0.00%)  3/17 (17.65%)  1/7 (14.29%) 
Injection site erythema  1  0/17 (0.00%)  3/17 (17.65%)  1/7 (14.29%) 
Injection site pain  1  0/17 (0.00%)  2/17 (11.76%)  0/7 (0.00%) 
Pyrexia  1  1/17 (5.88%)  1/17 (5.88%)  0/7 (0.00%) 
Vaccination site pain  1  0/17 (0.00%)  2/17 (11.76%)  0/7 (0.00%) 
Administration site discolouration  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Administration site pain  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Administration site swelling  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Chills  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Infusion site bruising  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Infusion site erythema  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Infusion site pain  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Infusion site pruritus  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Infusion site swelling  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Injection site bruising  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Injection site reaction  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Malaise  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Puncture site pain  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Vaccination site joint erythema  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Infections and infestations       
Nasopharyngitis  1  2/17 (11.76%)  1/17 (5.88%)  0/7 (0.00%) 
Conjunctivitis  1  1/17 (5.88%)  2/17 (11.76%)  0/7 (0.00%) 
Gastroenteritis  1  0/17 (0.00%)  2/17 (11.76%)  0/7 (0.00%) 
Sinusitis  1  0/17 (0.00%)  2/17 (11.76%)  0/7 (0.00%) 
Upper respiratory tract infection  1  1/17 (5.88%)  1/17 (5.88%)  0/7 (0.00%) 
Bronchitis  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Cystitis  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Hordeolum  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Impetigo  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Laryngitis  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Oral herpes  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Otitis externa  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Parotitis  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Periodontitis  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Pharyngitis  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Pneumonia  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Pulpitis dental  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Injury, poisoning and procedural complications       
Vaccination complication  1  0/17 (0.00%)  1/17 (5.88%)  1/7 (14.29%) 
Ligament sprain  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Traumatic arthropathy  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Wound  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Investigations       
Blood creatine phosphokinase increased  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Occult blood positive  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Musculoskeletal and connective tissue disorders       
Pain in extremity  1  0/17 (0.00%)  2/17 (11.76%)  0/7 (0.00%) 
Arthritis  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Back pain  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Pain in jaw  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Colon adenoma  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Seborrhoeic keratosis  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Nervous system disorders       
Headache  1  3/17 (17.65%)  3/17 (17.65%)  1/7 (14.29%) 
Orthostatic intolerance  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Sciatica  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Psychiatric disorders       
Insomnia  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Skin and subcutaneous tissue disorders       
Acne  1  1/17 (5.88%)  0/17 (0.00%)  0/7 (0.00%) 
Dermatitis atopic  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Eczema asteatotic  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Miliaria  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Rash pruritic  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Seborrhoeic dermatitis  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
Urticaria  1  0/17 (0.00%)  1/17 (5.88%)  0/7 (0.00%) 
1
Term from vocabulary, MedDRA24.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The first study related publication will be a multi-center publication submitted within 24 months after conclusion or termination of a study at all sites. After such multi site publication, all proposed site publications and presentations will be submitted to sponsor for review 60 days in advance of publication. Site will remove Sponsor confidential information unrelated to study results. Sponsor can delay a proposed publication for another 60 days to preserve intellectual property.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Shire
Phone: +1 866 842 5335
EMail: ClinicalTransparency@shire.com
Layout table for additonal information
Responsible Party: Takeda ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT04346108    
Other Study ID Numbers: TAK-664-3001
JapicCTI-205162 ( Registry Identifier: JapicCTI )
First Submitted: April 10, 2020
First Posted: April 15, 2020
Results First Submitted: October 26, 2022
Results First Posted: March 22, 2024
Last Update Posted: March 22, 2024