Primary Immunodeficiency Diseases (PID)
Conditions
Brief summary
In this study, Japanese participants with primary immunodeficiency diseases were treated with Immune Globulin Subcutaneous (Human), 20% solution, (IGSC, 20%). This study will be in 3 parts: Part 1: Infusions with Immunoglobulin Intravenous (IGIV) every 3 or 4 weeks for 13 weeks. Part 2: Participants will switch to weekly subcutaneous infusions with IGSC, 20% for 24 weeks. Part 3: A subset will receive biweekly subcutaneous infusions with IGSC, 20% for 12 weeks. The main aim of the study is to assess base levels of Immunoglobulin globulin G (IgG) levels in the blood of the participants after weekly and biweekly treatment with IGSC, 20% (in Parts 2 and 3 of the study). Their PID will be treated by their doctor according to their doctor's usual clinical practice.
Detailed description
This study consists of 3 treatment parts (Epoch 1, 2, 3). The total evaluation period of the study will be 57 weeks in which screening period is for 2-8 weeks and Epoch 1 is from Week 8 to Week 21, Epoch 2 is from Week 21 to Week 45, Epoch 3 is from Week 45 to Week 57. Each participant will receive IGIV treatment in Epoch 1 for a total of 13 weeks, then switch to weekly subcutaneous (SC) treatment with IGSC, 20% in Epoch 2 for a total of 24 weeks and will continue into Epoch 3 for a total of 12 weeks of biweekly SC treatment with IGSC, 20%. Drug dose in Epoch 2 and Epoch 3 will be adjusted so that it will be an equivalent weekly dose of the dose administered in Epoch 1 and twice the dose administrated in Epoch 2 respectively. Epoch 2 will contain two periods, period 1: dose adjustment period (first 12 weeks) and period 2: evaluation period (second 12 weeks).
Interventions
BIOLOGICALImmune Globulin Intravenous (IGIV)
Participants will receive IGIV infusion.
BIOLOGICALImmune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
Participants will receive IGSC, 20% SC infusion.
Sponsors
Baxalta now part of Shire
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents. * Participants must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement. The diagnosis must be confirmed by the medical director prior to treatment with IGIV. * Participant is 2 years or older at the time of screening. * Written informed consent is obtained from either the participants or the participants legally authorized representative prior to any study-related procedures and study product administration. * Participant has been receiving a consistent dose of IGIV over a period of at least 3 months prior to screening equivalent to approximately 200-600 mg/kg-body weight (BW) per 3- 4 week period, as according to the product package insert * Participant has a serum trough level of IgG \>= 5 gram per liter (g/L) at screening. * Participant has not had a serious bacterial infection within the 3 months prior to screening. * Participant is willing and able to comply with the requirements of the protocol.
Exclusion criteria
* Participant has a known history of or is positive at screening for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2. * Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent): * Persistent alanine aminotransferase (ALT) and aspartate amino transferase (AST) \> 2.5 times the upper limit of normal (ULN) for the testing laboratory * Persistent severe neutropenia (defined as an absolute neutrophil count \[ANC\] \<= 500/milli cubic meter \[mm\^3\]). * Participant has presence of renal function impairment defined by estimated glomerular filtration rate (eGFR) is \<60 milliliter per minute/ 1.73 square meter (mL/min/1.73m\^2). * Participant has been diagnosed with or has a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix), unless the disease-free period prior to screening exceeds 5 years. * Participant is receiving anti-coagulation therapy or has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) within 12 months prior to screening or a history of thrombophilia. * Participant has abnormal protein loss (protein losing enteropathy, nephrotic syndrome). * Participant has anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site. * Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IV immunoglobulin, SC immunoglobulin, and/or Immune Serum Globulin (ISG) infusions. * Participant has immunoglobulin A (IgA) deficiency (IgA less than 0.07 g/L), known anti IgA antibodies, and a history of hypersensitivity. * Participant is on preventative (prophylactic) systemic antibacterial antibiotics at doses sufficient to treat or prevent bacterial infections, and cannot stop these antibiotics at the time of screening. * Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening. * Participant has a bleeding disorder, or a platelet count less than 20,000/ microliter (mcL), or, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of subcutaneous therapy. * Participant has total protein \> 9 gram per deciliter (g/dL) or myeloma, or macroglobulinemia (IgM) or paraproteinemia. * Women of childbearing potential meeting any one of the following criteria: * Participant presents with a positive pregnancy test. * Participant is breast feeding. * Participant intends to begin nursing during the course of the study. * Participant does not agree to employ adequate birth-control measures (e.g. intrauterine device, diaphragm or condom \[for male partner\] with spermicidal jelly or foam, or birth control pills/patches) throughout the course of the study. * Participant has participated in another clinical study and has been exposed to an IP or device within 30 days prior to study enrollment. * Participant is scheduled to participate in another non-observational (interventional) clinical study involving an IP or device during the course of the study. * Participant has severe dermatitis that would preclude adequate sites for safe product administration.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2 | Epoch 2 (period 2): Up to 24 weeks | Total serum trough levels of IgG antibodies measured during period 2 of Epoch 2 were assessed. |
| Epoch 3: Total Serum Trough Levels of IgG Antibodies | Epoch 3: Up to Week 12 | Total serum trough levels of IgG antibodies measured during Epoch 3 were assessed. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses | Epoch 2: Week 21 | Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined. |
| Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG | Epoch 2: Week 21 | — |
| Epoch 2: CL/F for Total Serum Levels of IgG Subclasses | Epoch 2: Week 21 | Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined. |
| Epoch 2: Maximum Concentration (Cmax) for Total Serum Levels of IgG | Epoch 2: Week 21 | — |
| Epoch 2: Cmax for Total Serum Levels of IgG Subclasses | Epoch 2: Week 21 | Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined. |
| Epoch 2: Minimum Concentration (Cmin) for Total Serum Levels of IgG | Epoch 2: Week 21 | — |
| Epoch 2: Cmin for Total Serum Levels of IgG Subclasses | Epoch 2: Week 21 | Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined. |
| Epoch 2: Time to Maximum Concentration (Tmax) for Total Serum Levels of IgG | Epoch 2: Week 21 | — |
| Epoch 2: Tmax for Total Serum Levels of IgG Subclasses | Epoch 2: Week 21 | Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined. |
| Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Epoch 1 (Week 1); Epoch 2 (Week 1, 24); Epoch 3 (Week 1, 13) | 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. |
| Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB) | Epoch 1 (Week 1); Epoch 2 (Week 1, 24); Epoch 3 (Week 1, 13) | 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. |
| Health Related Quality of Life: Treatment Preference | Up to approximately 1.5 years | 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). |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | From first dose of study drug up to end of study (up to approximately 1.5 years) | 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. |
| Epoch 1: Total Serum Trough Levels of IgG Antibodies | Epoch 1: Up to Week 13 | Total serum trough levels of IgG antibodies measured during Epoch 1 were assessed. |
| Annual Rate of Validated Acute Serious Bacterial Infections (ASBI) | From first dose of study drug up to end of study (up to approximately 1.5 years) | 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. |
| Annual Rate of All Infections Per Year | From first dose of study drug up to end of study (up to approximately 1.5 years) | Annual rate is the number of participants reporting any infection per year. |
| Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection | From first dose of study drug up to end of study (up to approximately 1.5 years) | 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. |
| Number of Days Participants Were on Antibiotics | From first dose of study drug up to end of study (up to approximately 1.5 years) | 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. |
| Number of Participants Hospitalized Due to Illness or Infection | From first dose of study drug up to end of study (up to approximately 1.5 years) | 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. |
| Length of Hospital Stay | From first dose of study drug up to end of study (up to approximately 1.5 years) | 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. |
| Number of Acute Physician Visits Due to Illness/Infection | From first dose of study drug up to end of study (up to approximately 1.5 years) | Number of acute physician visits is standardized to per year (365.25 days). |
| Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | Baseline up to end of study (approximately 1.5 years) | 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. |
| EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | Baseline up to end of the study (approximately 1.5 years) | 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). |
| Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | Baseline up to end of the study (approximately 1.5 years) | 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. |
| Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | Baseline up to end of the study (approximately 1.5 years) | 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). |
| Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | Baseline up to end of the study (approximately 1.5 years) | 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. |
| Number of Participants With Tolerability Events Related to the Infusion of Study Drug | From first dose of study drug up to end of study (up to approximately 1.5 years) | 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. |
| Epoch 2: Area Under the Curve From Time 0 to Last Interval (AUC0-last) for Total Serum Levels of IgG | Epoch 2: Week 21 | — |
Countries
Japan
Participant flow
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.
Participants by arm
| Arm | Count |
|---|---|
| IGIV 200 to 600 mg/kg + IGSC [50 to 200 and 100 to 400] mg/kg 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. | 17 |
| Total | 17 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Epoch 2 (24 Weeks After Epoch 1) | Withdrawal by Parent/Guardian | 0 | 1 | 0 |
| Epoch 2 (24 Weeks After Epoch 1) | Withdrawal by Subject | 0 | 1 | 0 |
Baseline characteristics
| Characteristic | IGIV 200 to 600 mg/kg + IGSC [50 to 200 and 100 to 400] mg/kg |
|---|---|
| Age, Continuous | 31.0 years STANDARD_DEVIATION 21.13 |
| Body Mass Index (BMI) | 19.88 kg/m^2 STANDARD_DEVIATION 3.242 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 17 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Height | 152.15 centimeter (cm) STANDARD_DEVIATION 20.076 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 17 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 10 Participants |
| Weight | 48.16 kilogram (kg) STANDARD_DEVIATION 18.12 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 17 | 0 / 17 | 0 / 7 |
| other Total, other adverse events | 11 / 17 | 16 / 17 | 3 / 7 |
| serious Total, serious adverse events | 0 / 17 | 0 / 17 | 0 / 7 |
Outcome results
Primary
Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2
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
Population: 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 | Measure | Value (GEOMETRIC_MEAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies During Period 2 | 8.56 grams per liter (g/L) |
Primary
Epoch 3: Total Serum Trough Levels of IgG Antibodies
Total serum trough levels of IgG antibodies measured during Epoch 3 were assessed.
Time frame: Epoch 3: Up to Week 12
Population: 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 | Measure | Value (GEOMETRIC_MEAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 3: Total Serum Trough Levels of IgG Antibodies | 8.39 g/L |
Secondary
Annual Rate of All Infections Per Year
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Annual Rate of All Infections Per Year | 1.65 number of infections per year | Standard Deviation 2.479 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Annual Rate of All Infections Per Year | 2.78 number of infections per year | Standard Deviation 3.074 |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Annual Rate of All Infections Per Year | 0.00 number of infections per year | Standard Deviation 0 |
95% CI: [0.73, 3.15]
95% CI: [0, 0]
95% CI: [1.34, 4.13]
Secondary
Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Annual Rate of Validated Acute Serious Bacterial Infections (ASBI) | 0.0 number of infections per year | Standard Deviation 0 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Annual Rate of Validated Acute Serious Bacterial Infections (ASBI) | 0.25 number of infections per year | Standard Deviation 1.042 |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Annual Rate of Validated Acute Serious Bacterial Infections (ASBI) | 0.0 number of infections per year | Standard Deviation 0 |
Secondary
Epoch 1: Total Serum Trough Levels of IgG Antibodies
Total serum trough levels of IgG antibodies measured during Epoch 1 were assessed.
Time frame: Epoch 1: Up to Week 13
Population: 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 | Measure | Value (GEOMETRIC_MEAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 1: Total Serum Trough Levels of IgG Antibodies | 8.18 g/L |
Secondary
Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Value (GEOMETRIC_MEAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG | 1.93 mL/kg/day |
Secondary
Epoch 2: Area Under the Curve From Time 0 to Last Interval (AUC0-last) for Total Serum Levels of IgG
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Value (GEOMETRIC_MEAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Area Under the Curve From Time 0 to Last Interval (AUC0-last) for Total Serum Levels of IgG | 58.9 g*day/L |
Secondary
Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses
Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Group | Value (GEOMETRIC_MEAN) |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses | IgG 1 | 35.8 g*day/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses | IgG 2 | 23.4 g*day/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses | IgG 3 | 1.32 g*day/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: AUC0-last for Total Serum Levels of IgG Subclasses | IgG 4 | 1.67 g*day/L |
Secondary
Epoch 2: CL/F for Total Serum Levels of IgG Subclasses
Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Group | Value (GEOMETRIC_MEAN) |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: CL/F for Total Serum Levels of IgG Subclasses | IgG 1 | 3.18 mL/kg/day |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: CL/F for Total Serum Levels of IgG Subclasses | IgG 2 | 4.86 mL/kg/day |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: CL/F for Total Serum Levels of IgG Subclasses | IgG 3 | 99.5 mL/kg/day |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: CL/F for Total Serum Levels of IgG Subclasses | IgG 4 | 68.3 mL/kg/day |
Secondary
Epoch 2: Cmax for Total Serum Levels of IgG Subclasses
Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Group | Value (GEOMETRIC_MEAN) |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Cmax for Total Serum Levels of IgG Subclasses | IgG 1 | 5.40 g/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Cmax for Total Serum Levels of IgG Subclasses | IgG 2 | 3.51 g/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Cmax for Total Serum Levels of IgG Subclasses | IgG 3 | 0.202 g/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Cmax for Total Serum Levels of IgG Subclasses | IgG 4 | 0.256 g/L |
Secondary
Epoch 2: Cmin for Total Serum Levels of IgG Subclasses
Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Group | Value (GEOMETRIC_MEAN) |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Cmin for Total Serum Levels of IgG Subclasses | IgG 1 | 4.89 g/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Cmin for Total Serum Levels of IgG Subclasses | IgG 2 | 3.15 g/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Cmin for Total Serum Levels of IgG Subclasses | IgG 3 | 0.181 g/L |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Cmin for Total Serum Levels of IgG Subclasses | IgG 4 | 0.218 g/L |
Secondary
Epoch 2: Maximum Concentration (Cmax) for Total Serum Levels of IgG
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Value (GEOMETRIC_MEAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Maximum Concentration (Cmax) for Total Serum Levels of IgG | 9.08 g/L |
Secondary
Epoch 2: Minimum Concentration (Cmin) for Total Serum Levels of IgG
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Value (GEOMETRIC_MEAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Minimum Concentration (Cmin) for Total Serum Levels of IgG | 7.45 g/L |
Secondary
Epoch 2: Time to Maximum Concentration (Tmax) for Total Serum Levels of IgG
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Time to Maximum Concentration (Tmax) for Total Serum Levels of IgG | 71.08 hours |
Secondary
Epoch 2: Tmax for Total Serum Levels of IgG Subclasses
Total serum levels of IgG subclasses IgG 1, IgG 2, IgG 3, and IgG 4 were determined.
Time frame: Epoch 2: Week 21
Population: 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 | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Tmax for Total Serum Levels of IgG Subclasses | IgG 1 | 71.08 hours |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Tmax for Total Serum Levels of IgG Subclasses | IgG 2 | 71.08 hours |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Tmax for Total Serum Levels of IgG Subclasses | IgG 3 | 72.92 hours |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Epoch 2: Tmax for Total Serum Levels of IgG Subclasses | IgG 4 | 71.17 hours |
Secondary
EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score
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)
Population: 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 | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGIV: Epoch 1: Baseline (Week 1): Health State Index Score | 0.8080 score on a scale | Standard Deviation 0.04619 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGIV: Epoch 1: Baseline (Week 1): EQ Visual Analogue Scale | 87.5 score on a scale | Standard Deviation 15.55 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 2: Change from Baseline (Week 1): Health State Index Score | -0.0270 score on a scale | Standard Deviation 0.12275 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 2: Change from Baseline (Week 1): EQ Visual Analogue Scale | -5.0 score on a scale | Standard Deviation 5 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 3: Change from Baseline (Week 1): Health State Index Score | 0.0000 score on a scale | — |
| Epoch 2: IGSC (20%) 50-200 mg/kg | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC:Epoch 3: Change from Baseline (Week 1): EQ Visual Analogue Scale | 0.0 score on a scale | — |
| Epoch 2: IGSC (20%) 50-200 mg/kg | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Health State Index Score | 0.0400 score on a scale | Standard Deviation 0.04619 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): EQ Visual Analogue Scale | 1.3 score on a scale | Standard Deviation 6.29 |
| Epoch 1: IGIV 4-week Interval (Week 1) | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): EQ Visual Analogue Scale | 6.9 score on a scale | Standard Deviation 13.21 |
| Epoch 1: IGIV 4-week Interval (Week 1) | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGIV: Epoch 1: Baseline (Week 1): Health State Index Score | 0.8217 score on a scale | Standard Deviation 0.04544 |
| Epoch 1: IGIV 4-week Interval (Week 1) | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 3: Change from Baseline (Week 1): Health State Index Score | 0.0133 score on a scale | Standard Deviation 0.03266 |
| Epoch 1: IGIV 4-week Interval (Week 1) | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGIV: Epoch 1: Baseline (Week 1): EQ Visual Analogue Scale | 77.1 score on a scale | Standard Deviation 22.16 |
| Epoch 1: IGIV 4-week Interval (Week 1) | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Health State Index Score | 0.0229 score on a scale | Standard Deviation 0.04566 |
| Epoch 1: IGIV 4-week Interval (Week 1) | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 2: Change from Baseline (Week 1): Health State Index Score | 0.0016 score on a scale | Standard Deviation 0.03318 |
| Epoch 1: IGIV 4-week Interval (Week 1) | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC:Epoch 3: Change from Baseline (Week 1): EQ Visual Analogue Scale | -1.3 score on a scale | Standard Deviation 12.39 |
| Epoch 1: IGIV 4-week Interval (Week 1) | EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Total Scale Score | IGSC: Epoch 2: Change from Baseline (Week 1): EQ Visual Analogue Scale | 4.2 score on a scale | Standard Deviation 17.15 |
Secondary
Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score
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)
Population: 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 | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | IGIV: Epoch 1: Baseline (Week 1) | 88.04 score on a scale | Standard Deviation 13.835 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | IGSC: Epoch 2: Change from Baseline (Week 1) | 9.78 score on a scale | Standard Deviation 15.372 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | IGSC: Epoch 3: Change from Baseline (Week 1) | 14.13 score on a scale | — |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | IGSC: Epoch 3: Change from Baseline (EOS/ET]) | 3.26 score on a scale | Standard Deviation 7.686 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | IGSC: Epoch 3: Change from Baseline (EOS/ET]) | 4.08 score on a scale | Standard Deviation 16.374 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | IGIV: Epoch 1: Baseline (Week 1) | 87.23 score on a scale | Standard Deviation 11.82 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | IGSC: Epoch 3: Change from Baseline (Week 1) | 20.65 score on a scale | — |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL) Total Scale Score | IGSC: Epoch 2: Change from Baseline (Week 1) | -6.79 score on a scale | Standard Deviation 9.977 |
Secondary
Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score
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)
Population: 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 | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | IGIV: Epoch 1: Baseline (Week 1): Physical Component Summary Score | 50.21 score on a scale | Standard Deviation 7.282 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | IGIV: Epoch 1: Baseline (Week 1): Mental Component Summary Score | 50.59 score on a scale | Standard Deviation 8.114 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Physical Component Summary Score | 0.10 score on a scale | Standard Deviation 7.142 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Mental Component Summary Score | -0.30 score on a scale | Standard Deviation 2.294 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Physical Component Summary Score | 1.81 score on a scale | Standard Deviation 8.189 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Mental Component Summary Score | 3.60 score on a scale | Standard Deviation 5.352 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Physical Component Summary Score | 0.23 score on a scale | Standard Deviation 4.649 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Mental Component Summary Score | 4.32 score on a scale | Standard Deviation 8.185 |
Secondary
Health Related Quality of Life: Treatment Preference
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
Population: 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 | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: At Hospital | 0 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: At Home | 3 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: No Preference | 1 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: No Preference | 1 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Dislike Very Much | 0 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Like Very Much | 2 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Like | 1 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: No Preference | 5 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: No Preference | 2 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: At Home | 1 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Dislike Very Much | 1 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Like Very Much | 1 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: At Hospital | 0 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Like | 3 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: At Hospital | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Like Very Much | 1 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Like | 1 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: No Preference | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: No Preference | 1 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: At Home | 1 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Dislike Very Much | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Dislike Very Much | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: At Home | 4 Participants |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: No Preference | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Like Very Much | 4 Participants |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: Like | 1 Participants |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Health Related Quality of Life: Treatment Preference | The Frequency of Administration: No Preference | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Health Related Quality of Life: Treatment Preference | Where do you Prefer to Receive Your Immunoglobulin Therapy: At Hospital | 1 Participants |
Secondary
Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score
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)
Population: 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 | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGIV: Epoch 1: Baseline (Week 1): Factor 1 Treatment Interference | 81.48 score on a scale | Standard Deviation 14.873 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Factor 1 Treatment Interference | 4.17 score on a scale | Standard Deviation 1.964 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Factor 1 Treatment Interference | -8.33 score on a scale | Standard Deviation 21.445 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Factor 2 Therapy-Related Problems | -14.58 score on a scale | Standard Deviation 14.731 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGIV: Epoch 1: Baseline (Week 1): Factor 3 Therapy Setting | 77.78 score on a scale | Standard Deviation 20.488 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Factor 3 Therapy Setting | 19.44 score on a scale | Standard Deviation 3.928 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Factor 2 Therapy-Related Problems | 2.78 score on a scale | Standard Deviation 9.742 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Factor 4 Treatment Costs | 0.00 score on a scale | Standard Deviation 0 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGIV: Epoch 1: Baseline (Week 1): Factor 2 Therapy-Related Problems | 81.94 score on a scale | Standard Deviation 15.516 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 1 Treatment Interference | -2.78 score on a scale | Standard Deviation 13.494 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Factor 3 Therapy Setting | -6.48 score on a scale | Standard Deviation 30.106 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 2 Therapy-Related Problems | -1.39 score on a scale | Standard Deviation 9.001 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGIV: Epoch 1: Baseline (Week 1): Factor 4 Treatment Costs | 65.28 score on a scale | Standard Deviation 24.391 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 3 Therapy Setting | 2.78 score on a scale | Standard Deviation 16.005 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Factor 4 Treatment Costs | -9.72 score on a scale | Standard Deviation 22.618 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 4 Treatment Costs | 4.17 score on a scale | Standard Deviation 16.672 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Factor 4 Treatment Costs | 0.76 score on a scale | Standard Deviation 31.282 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGIV: Epoch 1: Baseline (Week 1): Factor 1 Treatment Interference | 77.02 score on a scale | Standard Deviation 17.963 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGIV: Epoch 1: Baseline (Week 1): Factor 2 Therapy-Related Problems | 79.17 score on a scale | Standard Deviation 15.811 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGIV: Epoch 1: Baseline (Week 1): Factor 3 Therapy Setting | 74.75 score on a scale | Standard Deviation 23.748 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGIV: Epoch 1: Baseline (Week 1): Factor 4 Treatment Costs | 53.79 score on a scale | Standard Deviation 21.847 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Factor 1 Treatment Interference | -2.53 score on a scale | Standard Deviation 14.351 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Factor 2 Therapy-Related Problems | -1.52 score on a scale | Standard Deviation 15.841 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Factor 3 Therapy Setting | 0.51 score on a scale | Standard Deviation 11.773 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 4 Treatment Costs | 14.39 score on a scale | Standard Deviation 26.898 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Factor 1 Treatment Interference | 3.33 score on a scale | Standard Deviation 4.12 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Factor 2 Therapy-Related Problems | 1.67 score on a scale | Standard Deviation 14.613 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Factor 3 Therapy Setting | 12.22 score on a scale | Standard Deviation 16.387 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Factor 4 Treatment Costs | 18.33 score on a scale | Standard Deviation 40.139 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 1 Treatment Interference | -0.25 score on a scale | Standard Deviation 13.046 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 2 Therapy-Related Problems | 1.14 score on a scale | Standard Deviation 9.334 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Factor 3 Therapy Setting | 4.55 score on a scale | Standard Deviation 16.067 |
Secondary
Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score
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)
Population: 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 | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGIV: Epoch 1: Baseline (Week 1): Effectiveness | 69.44 score on a scale | Standard Deviation 14.38 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGIV: Epoch 1: Baseline (Week 1): Convenience | 62.96 score on a scale | Standard Deviation 13.907 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGIV: Epoch 1: Baseline (Week 1): Global Satisfaction | 75.00 score on a scale | Standard Deviation 17.928 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Effectiveness | 2.22 score on a scale | Standard Deviation 9.296 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Convenience | 3.33 score on a scale | Standard Deviation 16.942 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Global Satisfaction | -2.86 score on a scale | Standard Deviation 10.833 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Effectiveness | 2.78 score on a scale | Standard Deviation 3.928 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Convenience | 13.89 score on a scale | Standard Deviation 19.642 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Global Satisfaction | -7.14 score on a scale | Standard Deviation 10.02 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Effectiveness | 9.26 score on a scale | Standard Deviation 10.924 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Convenience | 5.56 score on a scale | Standard Deviation 22.222 |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Global Satisfaction | 7.14 score on a scale | Standard Deviation 23.905 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Convenience | 9.09 score on a scale | Standard Deviation 23.21 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGIV: Epoch 1: Baseline (Week 1): Effectiveness | 75.25 score on a scale | Standard Deviation 18.817 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Effectiveness | -10.00 score on a scale | Standard Deviation 52.673 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGIV: Epoch 1: Baseline (Week 1): Convenience | 65.66 score on a scale | Standard Deviation 23.016 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Effectiveness | 6.57 score on a scale | Standard Deviation 18.892 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGIV: Epoch 1: Baseline (Week 1): Global Satisfaction | 74.68 score on a scale | Standard Deviation 18.173 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Convenience | 15.56 score on a scale | Standard Deviation 12.669 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Effectiveness | -1.52 score on a scale | Standard Deviation 10.568 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (EOS/ET): Global Satisfaction | 3.90 score on a scale | Standard Deviation 23.768 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Convenience | 5.05 score on a scale | Standard Deviation 13.484 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 3: Change from Baseline (Week 1): Global Satisfaction | 4.29 score on a scale | Standard Deviation 29.709 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9) Score | IGSC: Epoch 2: Change from Baseline (Week 1): Global Satisfaction | 1.30 score on a scale | Standard Deviation 10.507 |
Secondary
Length of Hospital Stay
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Length of Hospital Stay | 0.00 days |
| Epoch 1: IGIV 4-week Interval (Week 1) | Length of Hospital Stay | 0.00 days |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Length of Hospital Stay | 0.00 days |
95% CI: [0, 0]
95% CI: [0.25, 2.76]
Secondary
Number of Acute Physician Visits Due to Illness/Infection
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Acute Physician Visits Due to Illness/Infection | 1.18 number of visits per year | Standard Deviation 3.098 |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Acute Physician Visits Due to Illness/Infection | 3.30 number of visits per year | Standard Deviation 6.807 |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Acute Physician Visits Due to Illness/Infection | 1.624 number of visits per year | Standard Deviation 8.121 |
95% CI: [0.38, 2.68]
95% CI: [0.91, 4.82]
95% CI: [0.07, 2.15]
Secondary
Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection | 0.00 days |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection | 0.00 days |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Days Participants Not Able to Attend School or Work to Perform Normal Daily Activities Due to Illness/Infection | 4.30 days |
95% CI: [1.02, 5.3]
95% CI: [2.82, 23.82]
95% CI: [1.37, 5.18]
Secondary
Number of Days Participants Were on Antibiotics
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Days Participants Were on Antibiotics | 0.00 days |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Days Participants Were on Antibiotics | 0.00 days |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Days Participants Were on Antibiotics | 0.00 days |
95% CI: [1.46, 8.54]
95% CI: [0.37, 10.74]
95% CI: [2.32, 11.93]
Secondary
Number of Participants Hospitalized Due to Illness or Infection
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Participants Hospitalized Due to Illness or Infection | 0 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Participants Hospitalized Due to Illness or Infection | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Participants Hospitalized Due to Illness or Infection | 0 Participants |
95% CI: [0, 0]
95% CI: [0.03, 0.35]
95% CI: [0, 0]
Secondary
Number of Participants With Tolerability Events Related to the Infusion of Study Drug
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions With Infusion Rate Reduced | 0 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions That Were Interrupted | 0 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions That Were Stopped | 0 Participants |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions With Infusion Rate Reduced or Interrupted or Stopped | 0 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions With Infusion Rate Reduced or Interrupted or Stopped | 1 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions With Infusion Rate Reduced | 0 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions That Were Stopped | 0 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions That Were Interrupted | 1 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions With Infusion Rate Reduced or Interrupted or Stopped | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions That Were Interrupted | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions That Were Stopped | 0 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Participants With Tolerability Events Related to the Infusion of Study Drug | Infusions With Infusion Rate Reduced | 0 Participants |
Secondary
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
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)
Population: All-Treated Set included all enrolled participants who received at least 1 dose of study drug (IGIV or IGSC).
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 11 Participants |
| Epoch 1: IGIV 4-week Interval (Week 1) | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 16 Participants |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | 3 Participants |
Secondary
Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB)
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)
Population: 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 | Measure | Value (GEOMETRIC_MEAN) |
|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB) | 1.58 mg/L |
| Epoch 1: IGIV 4-week Interval (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB) | 1.55 mg/L |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB) | 2.50 mg/L |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB) | 1.77 mg/L |
| Epoch 3: IGSC, 20% Biweekly (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB) | 2.04 mg/L |
| Epoch 3: IGSC, 20% Biweekly (Week 13) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogen: Haemophilus Influenzae (HIB) | 1.73 mg/L |
Secondary
Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV)
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)
Population: 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 | Measure | Group | Value (GEOMETRIC_MEAN) |
|---|---|---|---|
| Epoch 2: IGSC (20%) 50-200 mg/kg | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Clostridium Tetani Toxoid | 0.524 International units (IU)/mL |
| Epoch 2: IGSC (20%) 50-200 mg/kg | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Hepatitis B Virus | 27.7 International units (IU)/mL |
| Epoch 1: IGIV 4-week Interval (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Clostridium Tetani Toxoid | 0.602 International units (IU)/mL |
| Epoch 1: IGIV 4-week Interval (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Hepatitis B Virus | 35.2 International units (IU)/mL |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Clostridium Tetani Toxoid | 0.674 International units (IU)/mL |
| Epoch 2: IGSC, 20% Weekly (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Hepatitis B Virus | 54.1 International units (IU)/mL |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Hepatitis B Virus | 486 International units (IU)/mL |
| Epoch 2: IGSC, 20% Weekly (EOS [Week 24]/ET) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Clostridium Tetani Toxoid | 1.52 International units (IU)/mL |
| Epoch 3: IGSC, 20% Biweekly (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Clostridium Tetani Toxoid | 1.97 International units (IU)/mL |
| Epoch 3: IGSC, 20% Biweekly (Week 1) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Hepatitis B Virus | 645 International units (IU)/mL |
| Epoch 3: IGSC, 20% Biweekly (Week 13) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Hepatitis B Virus | 923 International units (IU)/mL |
| Epoch 3: IGSC, 20% Biweekly (Week 13) | Trough Levels of Specific Antibodies to Clinically Relevant Pathogens: Clostridium Tetani Toxoid and Hepatitis B Virus (HBV) | Clostridium Tetani Toxoid | 1.80 International units (IU)/mL |