A Study to Evaluate IGSC 20% Biweekly Dosing in Treatment... | NCT04566692

Status Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
IGSC 20%: Treatment-experienced Cohort	Experimental	Participants first received 16 weekly doses of IGSC 20% using a subcutaneous (SC) infusion pump from Week 0 to Week 15. Participants entering study on intravenous immune globulin (IVIG), IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on subcutaneous immunoglobulin (SCIG) received the same milligram/kilogram (mg/kg) equivalent weekly dose as given prior to study entry, without using a dose adjustment factor (DAF). Participants then received 9 biweekly doses of IGSC 20 % (i.e, IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.	Biological: IGSC 20%
IGSC 20%: Treatment-naïve Cohort	Experimental	Treatment-naïve participants received a loading dose of 150 mg/kg/day of IGSC 20% for 5 consecutive days (Week 0, Days 1 to 5) followed by weekly maintenance infusions of 150 mg/kg IGSC 20% starting Week 1 (Day 8) through Week 32. IGSC 20% infusion was administered using an SC infusion pump.	Biological: IGSC 20%

Name	Type	Description	Arm Group Labels	Other Names
IGSC 20%	Biological	SC infusion pump.	IGSC 20%: Treatment-experienced Cohort IGSC 20%: Treatment-naïve Cohort

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Treatment-experienced Cohort: AUC of IGSC 20% Administered Weekly Assessed as: Steady-State AUC of Total IgG Over a Regular Dosing Interval (τ)	Area under the concentration vs. time curve at steady state over the dosing interval (from time 0 to τ), was calculated by a combination of linear and logarithmic trapezoidal methods. The linear trapezoidal method was used for all incremental trapezoids arising from increasing concentrations and the logarithmic trapezoidal method was used for those arising from decreasing concentrations. The dose interval τ was 7 days for participants on weekly IGSC 20% dosing. The data is reported for participants who received weekly dosing.	Predose and post dose at multiple time points after end of infusion up to Week 15
Treatment-experienced Cohort: AUC of IGSC 20% Administered Biweekly Assessed as Steady-State AUC of Total IgG Over a Biweekly Dosing Interval	Area under the concentration vs. time curve at steady state over the dosing interval (from time 0 to τ), was calculated by a combination of linear and logarithmic trapezoidal methods. The linear trapezoidal method was used for all incremental trapezoids arising from increasing concentrations and the logarithmic trapezoidal method was used for those arising from decreasing concentrations. The dose interval τ was 14 days for participants on a biweekly IGSC 20% dosing. AUC (0-14 days) for the biweekly dosing were divided by 2 for comparison with AUC(0-7 days) for the weekly dosing prior to the statistical comparison. The data is reported for participants who received bi-weekly dosing.	Predose and post dose at multiple timepoints after end of infusion up to Week 32

Secondary Outcomes

Measure	Description	Time Frame
Treatment-experienced Cohort: Cmax of Total IgG at Steady State Following IGSC 20% Weekly and Biweekly Administration	The observed maximum total IgG concentration following drug infusion obtained directly from the experimental data without interpolation. The data is reported for participants who received weekly and bi-weekly dosing	Predose and post dose at multiple timepoints after end of infusion up to Week 32

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Inclusion Criteria only for Treatment-Experienced Participants

Participants 18 years to 75 years (inclusive) at screening
Participants with documented and confirmed pre-existing diagnosis of Primary Immunodeficiency (PI) with features of hypogammaglobulinemia requiring Immunoglobulin G (IgG) replacement therapy including but not limited to the following humoral-based immunodeficiency syndromes (example, X-linked agammaglobulinemia, common variable immunodeficiency), and combined immunodeficiency syndromes without lymphocytopenia (example, hyper immunoglobulin M immunodeficiency syndrome).
Participants have not had an Serious bacterial infection (SBI) or been hospitalized for infection of any etiology (example, viral, fungal, parasitic) within the last 3 months prior to screening or during screening.
Participants currently receiving IgG replacement therapy for ≥3 months via Intravenous (IV) or SC infusion. Participants receiving IVIG prior to study must receive a dosage of at least 200 mg/kg per infusion.
Participants whose screening IgG trough levels must be ≥500 milligram per deciliter (mg/dL).
Participants have signed an informed consent form.

Inclusion Criteria only for Treatment-Naïve Participants

Participants 6 years to 75 years (inclusive) at screening.
Participants with documented and confirmed diagnosis of PI with features of hypogammaglobulinemia requiring IgG replacement therapy including but not limited to the following humoral-based immunodeficiency syndromes (example, X-linked agammaglobulinemia, common variable immunodeficiency), and combined immunodeficiency syndromes without lymphocytopenia (example, hyper immunoglobulin M immunodeficiency syndrome).
Participants have never received IgG replacement treatment (ie, no prior immune globulin replacement therapy).
Participants whose screening IgG level must be ≤400 mg/dL.
Participants do not have an SBI nor requires hospitalization for infection of any etiology (example, viral, fungal, parasitic) during screening or at baseline.
Participants have signed an informed consent.

Exclusion Criteria:

Participants with clinical evidence of any significant acute or chronic disease that, in the opinion of the investigator, may interfere with successful completion of the trial or place the participant at undue medical risk.
Participants have had a known serious adverse reaction (AR) to immunoglobulin or any anaphylactic reaction to blood or any blood-derived product.
Participants who have a history of blistering skin disease, clinically significant thrombocytopenia, bleeding disorder, diffuse rash, recurrent skin infections, or other disorders where SC therapy would be contraindicated during the study.
Participants have known isolated IgG subclass deficiency; isolated specific antibody deficiency (SAD) or selective IgG deficiency; or transient hypogammaglobulinemia of infancy.
Participants have known Selective Immunoglobulin A (IgA) Deficiency (with or without antibodies to IgA).
Participants have significant proteinuria (≥3+ or known urinary protein loss >1 gram g/24 hours or nephrotic syndrome), has acute renal failure, is on dialysis, and/or has severe renal impairment on Screening laboratory testing (blood urea nitrogen [BUN] or creatinine more than 2.5 times the upper limit of normal [ULN]).
Participants have screening values of aspartate aminotransferase (AST) or alanine aminotransferase (ALT) levels exceeding more than 2.5 times the ULN for the expected normal range for the testing laboratory.
Participants have hemoglobin <9 gram per deciliter (g/dL) at screening.
Participants have a history (either 1 episode within the year prior to the Screening Visit or 2 previous episodes over a lifetime) of or current diagnosis of thromboembolism (example, myocardial infarction, cerebrovascular accident or transient ischemic attack) or deep venous thrombosis.
Participants are currently receiving anti-coagulation therapy which would make SC administration inadvisable (vitamin K antagonists, nonvitamin K antagonist oral anticoagulants [example, dabigatran etexilate targeting Factor IIa, rivaroxaban, edoxaban, and apixaban targeting Factor Xa], and parenteral anticoagulants [example, fondaparinux]).
Participants currently have a known hyperviscosity syndrome.
Participants have an acquired medical condition that is known to cause secondary immune deficiency, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, chronic or recurrent neutropenia (absolute neutrophil count less than 1000/microliter (μL) [1.0 x10^9/L]), or human immunodeficiency virus infection/acquired immune deficiency syndrome.
Participants have a known previous infection with or clinical signs and symptoms consistent with current hepatitis B virus or hepatitis C virus infection.
Participants are receiving any of the following medications: (a) immunosuppressants including chemotherapeutic agents; (b) immunomodulators; (c) long-term systemic corticosteroids defined as daily dose >1 mg of prednisone equivalent/kg/day for >30 days. Note: Intermittent courses of corticosteroids of not more than 10 days would not exclude a participant. Inhaled or topical corticosteroids are allowed.
Participants (if <18 years of age) have non-controlled arterial hypertension at a level of greater than or equal to the 90th percentile blood pressure (either systolic or diastolic) for their age and height or the adult participant has non-controlled arterial hypertension (systolic blood pressure [SBP] >160 millimeter per mercury (mmHg) and/or diastolic blood pressure [DBP] >100 mmHg).

Facility	Status	City	State	ZIP	Country	Contacts
Clinical Research Center of Alabama		Birmingham	Alabama	35209	United States
Research Solutions of Arizona, PC

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	IGSC 20%: Treatment-experienced Cohort	Participants first received 16 weekly doses of IGSC 20% using a subcutaneous (SC) infusion pump from Week 0 to Week 15. Participants entering study on intravenous immune globulin (IVIG), IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on subcutaneous immunoglobulin (SCIG) received the same milligram/kilogram (mg/kg) equivalent weekly dose as given prior to study entry, without using a dose adjustment factor (DAF). Participants then received 9 biweekly doses of IGSC 20 % (i.e, IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Mar 17, 2020	Jul 14, 2023

ID	Title	Description
BG000	IGSC 20%: Treatment-experienced Cohort	Participants first received 16 weekly doses of IGSC 20% using a SC infusion pump from Week 0 to Week 15. Participants entering study on IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF. Participants then received 9 biweekly doses of IGSC 20 % (i.e, IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.
BG001	IGSC 20%: Treatment-naïve Cohort	Treatment-naïve participants received a loading dose of 150 mg/kg/day of IGSC 20% for 5 consecutive days (Week 0, Days 1 to 5) followed by weekly maintenance infusions of 150 mg/kg IGSC 20% starting Week 1 (Day 8) through Week 32. IGSC 20% infusion was administered using an SC infusion pump.
BG002	Total	Total of all reporting groups

Units	Counts
Participants	BG00027 BG0016 BG00233

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Treatment-experienced Cohort: AUC of IGSC 20% Administered Weekly Assessed as: Steady-State AUC of Total IgG Over a Regular Dosing Interval (τ)

Area under the concentration vs. time curve at steady state over the dosing interval (from time 0 to τ), was calculated by a combination of linear and logarithmic trapezoidal methods. The linear trapezoidal method was used for all incremental trapezoids arising from increasing concentrations and the logarithmic trapezoidal method was used for those arising from decreasing concentrations. The dose interval τ was 7 days for participants on weekly IGSC 20% dosing. The data is reported for participants who received weekly dosing.

PK population included all the participants who had sufficient PK concentration data to have at least one of the PK parameters AUC (0-7 days), Cmax, or Tmax. Overall number analyzed is the number of participants with data available for analysis.

Posted

Geometric Mean

Geometric Coefficient of Variation

hours*milligrams per deciliter (h*mg/dL)

Predose and post dose at multiple time points after end of infusion up to Week 15

ID	Title	Description
OG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.

Units	Counts
Participants	OG00023

Title

Denominators

Categories

Title	Measurements
	OG000167045.65± 20.2

Primary

Treatment-experienced Cohort: AUC of IGSC 20% Administered Biweekly Assessed as Steady-State AUC of Total IgG Over a Biweekly Dosing Interval

Area under the concentration vs. time curve at steady state over the dosing interval (from time 0 to τ), was calculated by a combination of linear and logarithmic trapezoidal methods. The linear trapezoidal method was used for all incremental trapezoids arising from increasing concentrations and the logarithmic trapezoidal method was used for those arising from decreasing concentrations. The dose interval τ was 14 days for participants on a biweekly IGSC 20% dosing. AUC (0-14 days) for the biweekly dosing were divided by 2 for comparison with AUC(0-7 days) for the weekly dosing prior to the statistical comparison. The data is reported for participants who received bi-weekly dosing.

PK population included all the participants who had sufficient PK concentration data to have at least one of the PK parameters AUC (0-7 days), Cmax, or Tmax.

Posted

Geometric Mean

Geometric Coefficient of Variation

h*mg/dL

Predose and post dose at multiple timepoints after end of infusion up to Week 32

ID	Title	Description
OG000	IGSC 20%: Bi-Weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Units	Counts

Secondary

Treatment-experienced Cohort: Cmax of Total IgG at Steady State Following IGSC 20% Weekly and Biweekly Administration

The observed maximum total IgG concentration following drug infusion obtained directly from the experimental data without interpolation. The data is reported for participants who received weekly and bi-weekly dosing

PK population included all the participants who had sufficient PK concentration data to have at least one of the PK parameters AUC (0-7 days), Cmax, or Tmax.

Posted

Geometric Mean

Geometric Coefficient of Variation

milligrams per deciliter (mg/dL)

Predose and post dose at multiple timepoints after end of infusion up to Week 32

ID	Title	Description
OG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.
OG001	IGSC 20%: Bi-weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Secondary

Treatment-experienced Cohort: Tmax of Total IgG at Steady State Given IGSC 20% Weekly and Biweekly

The data is reported for participants who received weekly and bi-weekly dosing

PK population included all the participants who had sufficient PK concentration data to have at least one of the PK parameters AUC (0-7 days), Cmax, or Tmax.

Posted

Median

Full Range

hours

Predose and post dose at multiple timepoints after end of infusion up to Week 32

ID	Title	Description
OG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.
OG001	IGSC 20%: Bi-weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Units	Counts
Participants

Secondary

Treatment-experienced Cohort: Steady-State Mean Trough Concentration of Total IgG Following IGSC 20% Weekly and Biweekly Administration

Mean Trough was calculated as the average of the trough concentrations at Weeks 12, 14 Pre-infusion, and 16 for the Weekly Period; and at Weeks 28, 30 Pre-infusion, and 32 for the Biweekly Period. Mean Trough for a given period was not calculated if at least one trough measure was missing. The data is reported for participants who received weekly and bi-weekly dosing.

IgG population included all participants who received at least 1 dose of IGSC 20%.

Posted

Geometric Mean

Geometric Coefficient of Variation

mg/dL

Predose and post dose at multiple timepoints after end of infusion up to Week 32

ID	Title	Description
OG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.
OG001	IGSC 20%: Bi-weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Secondary

Treatment-experienced Cohort: Individual Trough Concentration of Total IgG Following IGSC 20% Weekly and Biweekly Administration

IgG Trough 2 = For treatment-experienced participants entering the study on IVIG or Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase (HYQVIA), a second IgG trough level was obtained after screening; this was summarized in the visit designated as IgG Trough 2. Week 0 = Participants entering IVIG or HYQVIA, the first IGSC 20% dose was approximately 1 week post last IVIG/HYQVIA dose and so IgG at Week 0 for these participants represented a pre-dose value. For participants entering on all other forms of SCIG, the Week 0 value is true trough.

IgG population included all participants who received at least 1 dose of IGSC 20%. Number analyzed is the number of participants with data available for analysis at specified timepoint.

Posted

Geometric Mean

Geometric Coefficient of Variation

mg/dL

Screening, IgG Trough 2, Weeks 0, 2, 4, 8, 12, 14 (pre-infusion), 15, 16, 20, 24, 28, 30 (pre-infusion) and 32

ID	Title	Description
OG000	IGSC 20%: Treatment-experienced Cohort	Participants first received 16 weekly doses of IGSC 20% using a SC infusion pump from Week 0 to Week 15. Participants entering study on IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF. Participants then received 9 biweekly doses of IGSC 20 % (i.e, IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Secondary

Treatment-naïve Cohorts: Individual Trough Concentration of Total IgG

Week 0 = Participants entering IVIG or HYQVIA, the first IGSC 20% dose was approximately 1 week post last IVIG/HYQVIA dose and so IgG at Week 0 for these participants represented a pre-dose value. For participants entering on all other forms of SCIG, the Week 0 value is true trough.

IgG population included all participants who received at least 1 dose of IGSC 20%. Number analyzed is the number of participants with data available for analysis at specified timepoint.

Posted

Geometric Mean

Geometric Coefficient of Variation

mg/dL

Screening, Weeks 0, 1, 2, 4, 6, 8, 10, 12, 16, 20, 24, 28 and 32

ID	Title	Description
OG000	IGSC 20%: Treatment-naïve Cohort	Treatment-naïve participants received a loading dose of 150 mg/kg/day of IGSC 20% for 5 consecutive days (Week 0, Days 1 to 5) followed by weekly maintenance infusions of 150 mg/kg IGSC 20% starting Week 1 (Day 8) through Week 32. IGSC 20% infusion was administered using an SC infusion pump.

Units	Counts
Participants	OG000

Secondary

Treatment-experienced and Treatment-naïve Cohorts: Number of Participants With Serious Bacterial Infection (SBI)

Efficacy evaluable population included all participants who received at least 1 dose of IGSC 20%.

Posted

Count of Participants

Participants

From screening up to final follow up visit at Week 33

ID	Title	Description
OG000	IGSC 20%: Treatment-experienced Cohort	Participants first received 16 weekly doses of IGSC 20% using a SC infusion pump from Week 0 to Week 15. Participants entering study on IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF. Participants then received 9 biweekly doses of IGSC 20 % (i.e, IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.
OG001	IGSC 20%: Treatment-naïve Cohort	Treatment-naïve participants received a loading dose of 150 mg/kg/day of IGSC 20% for 5 consecutive days (Week 0, Days 1 to 5) followed by weekly maintenance infusions of 150 mg/kg IGSC 20% starting Week 1 (Day 8) through Week 32. IGSC 20% infusion was administered using an SC infusion pump.

Units

Secondary

Treatment-experienced and Treatment-naïve Cohorts: Rate of Events Per Participant Per Year With Infections of Any Kind as Determined by the Investigator

Rate of events per participant per year was calculated as the total number of events divided by the total duration of exposure in years across all participants. All infections of any kind included serious/nonserious including acute sinusitis, exacerbation of chronic sinusitis, acute otitis media, pneumonia, acute bronchitis, infectious diarrhea etc.) as determined by the investigator. The data is reported for participants in the treatment-naive cohort and the participants who received weekly and bi-weekly dosing.

Efficacy evaluable population included all participants who received at least 1 dose of IGSC 20%.

Posted

Number

95% Confidence Interval

rate of events per participant per year

From screening up to final follow up visit at Week 33

ID	Title	Description
OG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.
OG001	IGSC 20%: Bi-Weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Secondary

Treatment-experienced and Treatment-naïve Cohorts: Rate of Events Per Participant Per Year With Validated Infections

Rate of events per participant per year was calculated as the total number of events divided by the total duration of exposure in years across all participants. A validated treatment-emergent infection was documented by positive radiograph, fever (>38°C oral or >39°C rectal), culture, or diagnostic testing for microorganisms e.g., bacterial, viral, fungal, or protozoal pathogens (e.g., rapid streptococcal antigen detection test). Treatment-emergent infection was defined as an infection with onset on or after first infusion start date/time. The data is reported for participants in the treatment-naive cohort and the participants who received weekly and bi-weekly dosing.

Efficacy evaluable population included all participants who received at least 1 dose of IGSC 20%.

Posted

Number

95% Confidence Interval

rate of events per participant per year

From screening up to final follow up visit at Week 33

ID	Title	Description
OG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.
OG001	IGSC 20%: Bi-weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Secondary

Treatment-experienced and Treatment-naïve Cohorts: Rate of Days Per Participant Per Year That Participants Were on Antibiotics

Rate of days per person per year is calculated as the total number of days divided by the total duration of exposure in years across all participants. Antibiotics included prophylactic and therapeutic. The data is reported for participants in the treatment-naive cohort and the participants who received weekly and bi-weekly dosing.

Efficacy evaluable population included all participants who received at least 1 dose of IGSC 20%.

Posted

Number

95% Confidence Interval

rate of days per participant per year

From screening up to final follow up visit at Week 33

ID	Title	Description
OG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.
OG001	IGSC 20%: Bi-weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.
OG002

Secondary

Treatment-experienced and Treatment-naïve Cohorts: Rate of Hospitalizations Per Participant Per Year Due to Infection

Rate of hospitalizations per participant per year was calculated as the total number of events divided by the total duration of exposure in years across all participants. Hospitalization was considered only in cases of hospital admission (including emergency room stay) for equal or more than 24 hours. The data is reported for participants in the treatment-naive cohort and the participants who received weekly and bi-weekly dosing.

Efficacy evaluable population included all participants who received at least 1 dose of IGSC 20%.

Posted

Number

95% Confidence Interval

rate of events per participant per year

From screening up to final follow up visit at Week 33

ID	Title	Description
OG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.
OG001	IGSC 20%: Bi-weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	IGSC 20%: Weekly Dosing	Participants received 16 weekly doses of IGSC 20% from Week 0 to Week 15 using a SC infusion pump. Participants entering study IVIG, IGSC 20% was dosed at 1.37 times the equivalent weekly dose and participants entering on SCIG received the same mg/kg equivalent weekly dose as given prior to study entry, without using a DAF.	27	1	27	4	27
EG001	IGSC 20%: Bi-weekly Dosing	Participants received 9 biweekly doses of IGSC 20 % (i.e., IGSC 20% every 2 weeks) using an SC infusion pump, with the first IGSC 20% dose administered at Week 16 and the final dose given at Week 32.	25	2	25	1	25
EG002	IGSC 20%: Treatment-naïve Cohort	Treatment-naïve participants received a loading dose of 150 mg/kg/day of IGSC 20% for 5 consecutive days (Week 0, Days 1 to 5) followed by weekly maintenance infusions of 150 mg/kg IGSC 20% starting Week 1 (Day 8) through Week 32. IGSC 20% infusion was administered using an SC infusion pump.	6	1	6	3	6

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Headache	Nervous system disorders	MedDRA 23.1	Systematic Assessment	EG0003 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Procedural pain	Injury, poisoning and procedural complications	MedDRA 23.1	Systematic Assessment	EG0001 affected27 at risk EG0011 affected25 at risk EG0021 affected6 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Upper limb fracture	Injury, poisoning and procedural complications	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Plantar fasciitis	Musculoskeletal and connective tissue disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Infusion site reaction	General disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Infusion site scab	General disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Vitamin B12 deficiency	Metabolism and nutrition disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Skin mass	Skin and subcutaneous tissue disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Bartholin's cyst	Reproductive system and breast disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk

ID	Term
D000081207	Primary Immunodeficiency Diseases
D007153	Immunologic Deficiency Syndromes
D007154	Immune System Diseases

ID	Term
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

ID	Term
D005719	gamma-Globulins
D002210	Caprylates

ID	Term
D007136	Immunoglobulins
D007162	Immunoproteins
D001798	Blood Proteins
D011506	Proteins
D000602	Amino Acids, Peptides, and Proteins
D012712	Serum Globulins
D005916	Globulins
D000144	Acids, Acyclic
D002264	Carboxylic Acids
D009930	Organic Chemicals
D005227	Fatty Acids
D008055	Lipids

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Barrett's oesophagus	Gastrointestinal disorders	MedDRA 23.1	Systematic Assessment	EG0001 affected27 at risk EG0010 affected25 at risk EG0020 affected6 at risk
Pancreatitis acute	Gastrointestinal disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0011 affected25 at risk EG0020 affected6 at risk
Compression fracture	Injury, poisoning and procedural complications	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk
Dehydration	Metabolism and nutrition disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0011 affected25 at risk EG0020 affected6 at risk
Back Pain	Musculoskeletal and connective tissue disorders	MedDRA 23.1	Systematic Assessment	EG0000 affected27 at risk EG0010 affected25 at risk EG0021 affected6 at risk