Trial Outcomes & Findings for Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency (NCT NCT02806986)
NCT ID: NCT02806986
Last Updated: 2020-06-16
Results Overview
The rate of SBI events per participant per year during IGSC 20% treatment was calculated as the total number of SBI events divided by the total duration of exposure in years across all participants. The 2-sided 98% confidence interval (CI) was determined from a generalized linear model for poisson regression for the log-transformed number of events with log-transformed duration of exposure in years as an offset variable.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
61 participants
Primary outcome timeframe
IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53
Results posted on
2020-06-16
Participant Flow
Participants with primary immunodeficiency (PI) requiring IgG replacement were enrolled. Study has 3 stages: Screening/Previous Regimen Phase, Treatment Stage 1 and 2. Total 62 participants entered Previous Regimen Phase, of which 61 entered in Treatment Stage 1 to receive IGSC 20%. Study was conducted in 8 countries from June 2016 to May 2019.
Participants had no SBI within last 3 months prior to screening and were on immunoglobulin G (IgG) replacement therapy (stable regimen via intravenous \[IV\] or subcutaneous \[SC\] infusion) for ≥3 consecutive months prior to screening. Participants receiving IVIG prior to study entry must have received a dosage of ≥200 mg/kg per infusion.
Participant milestones
| Measure |
IGSC 20%
Following the previous regimen phase (screening), participants were enrolled to receive 13 immune globulin subcutaneous (human), 20% caprylate/chromatography purified (IGSC 20%) infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 milligram per kilogram per week (mg/kg/week) if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and at the Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|
|
Treatment Stage 1
STARTED
|
61
|
|
Treatment Stage 1
COMPLETED
|
60
|
|
Treatment Stage 1
NOT COMPLETED
|
1
|
|
Treatment Stage 2
STARTED
|
60
|
|
Treatment Stage 2
COMPLETED
|
55
|
|
Treatment Stage 2
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
IGSC 20%
Following the previous regimen phase (screening), participants were enrolled to receive 13 immune globulin subcutaneous (human), 20% caprylate/chromatography purified (IGSC 20%) infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 milligram per kilogram per week (mg/kg/week) if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and at the Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|
|
Treatment Stage 1
Adverse Event
|
1
|
|
Treatment Stage 2
Adverse Event
|
3
|
|
Treatment Stage 2
Withdrawal by Subject
|
2
|
Baseline Characteristics
Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
Baseline characteristics by cohort
| Measure |
IGSC 20%
n=61 Participants
Following the previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and at the Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|
|
Age, Continuous
|
27.3 years
STANDARD_DEVIATION 19.97 • n=5 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
42 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
49 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
57 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
2 Participants
n=5 Participants
|
|
Subject Entry Status
Entered on intravenous immune globulin (IVIG)
|
40 Participants
n=5 Participants
|
|
Subject Entry Status
Entered on subcutaneous immune globulin (SCIG)
|
21 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53Population: Analysis was performed on the efficacy evaluable population which included all participants who received at least 1 dose of IGSC 20%.
The rate of SBI events per participant per year during IGSC 20% treatment was calculated as the total number of SBI events divided by the total duration of exposure in years across all participants. The 2-sided 98% confidence interval (CI) was determined from a generalized linear model for poisson regression for the log-transformed number of events with log-transformed duration of exposure in years as an offset variable.
Outcome measures
| Measure |
IGSC 20% Treatment Stage 1
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower). Dose adjustments were permitted in this phase per the study protocol and at the Investigator's discretion.
|
IGSC 20% Treatment Stage 2
n=60 Participants
Following treatment stage 1, participants received 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52. The IGSC 20% dose (mg/kg) remained constant with no dose adjustment permitted in this phase, unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
IGSC 20% Overall
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and the at Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|---|---|
|
Rate of Serious Bacterial Infection (SBI) Per Participant Per Year
|
0 Rate of events per participant per year
Here NA signifies that 98% CI was not estimable due to zero number of events.
|
0.023 Rate of events per participant per year
Interval 0.008 to 0.049
|
0.017 Rate of events per participant per year
Interval 0.006 to 0.036
|
SECONDARY outcome
Timeframe: Previous Regimen Phase: 2 timepoints pre-dose of pIV or pSC between Screening and Baseline (up to 8 weeks). IGSC 20% Phase: Pre-dose of IGSC 20% at Baseline (Week 1), Weeks 2, 5, 9, 13, 17, 18, 20, 24, 28, 32, 36, 40, 44, 48, 52, and at Week 53Population: Analysis was performed on the IgG population which consisted of all participants who received any amount of IGSC 20% and had total IgG concentration data to facilitate the comparison of mean trough IgG concentration during the IGSC 20% phase versus the pre-treatment phase.
Mean trough total IgG concentration during the previous regimen phase was calculated as the average of the trough concentrations at the previous IVIG (pIV)#1 and pIV#2 visits for participants entering the study on a previous IVIG regimen, or at the previous subcutaneous immune globulin (SCIG) (pSC)#1 and baseline/SC#1 visits for participants entering the study on a previous SCIG regimen. Mean trough total IgG concentration during the IGSC 20% phase was calculated as the average of all steady state trough concentrations measured during the IGSC 20% treatment stage 2 at the visits corresponding to Weeks 17, 18, 20, 24, 28, 32, 36, 40, 44, 48, 52 and 53.
Outcome measures
| Measure |
IGSC 20% Treatment Stage 1
n=59 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower). Dose adjustments were permitted in this phase per the study protocol and at the Investigator's discretion.
|
IGSC 20% Treatment Stage 2
n=59 Participants
Following treatment stage 1, participants received 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52. The IGSC 20% dose (mg/kg) remained constant with no dose adjustment permitted in this phase, unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
IGSC 20% Overall
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and the at Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|---|---|
|
Mean Trough Total IgG Concentration
|
947.64 milligram per deciliter (mg/dL)
Standard Deviation 150.262
|
891.37 milligram per deciliter (mg/dL)
Standard Deviation 165.943
|
—
|
SECONDARY outcome
Timeframe: IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53Population: Analysis was performed on the efficacy evaluable population which included all participants who received at least 1 dose of IGSC 20%.
The total number of infections of any kind (serious/non-serious including acute sinusitis, exacerbation of chronic sinusitis, acute otitis media, pneumonia, acute bronchitis, infectious diarrhea, etc.) as determined by the investigator were evaluated. The rate of infection events per participant per year during IGSC 20% treatment was calculated as the total number of infection events divided by the total duration of exposure in years across all participants. The 2-sided 95% CI was determined from a generalized linear model for poisson regression for the log-transformed number of events with log-transformed duration of exposure in years as an offset variable.
Outcome measures
| Measure |
IGSC 20% Treatment Stage 1
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower). Dose adjustments were permitted in this phase per the study protocol and at the Investigator's discretion.
|
IGSC 20% Treatment Stage 2
n=60 Participants
Following treatment stage 1, participants received 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52. The IGSC 20% dose (mg/kg) remained constant with no dose adjustment permitted in this phase, unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
IGSC 20% Overall
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and the at Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
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|---|---|---|---|
|
Rate of Infection of Any Kind Per Participant Per Year
|
2.524 Rate of events per participant per year
Interval 1.72 to 3.547
|
2.353 Rate of events per participant per year
Interval 1.736 to 3.102
|
2.397 Rate of events per participant per year
Interval 1.824 to 3.079
|
SECONDARY outcome
Timeframe: IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53Population: Analysis was performed on the efficacy evaluable population which included all participants who received at least 1 dose of IGSC 20%.
The rate of days on antibiotics per participants per year during IGSC 20% treatment was calculated as the total number of days on antibiotic divided by the total duration of exposure in years across all participants. The 2-sided 95% CI was determined from a generalized linear model for poisson regression for the log transformed number of days with log-transformed duration of exposure in years as an offset variable.
Outcome measures
| Measure |
IGSC 20% Treatment Stage 1
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower). Dose adjustments were permitted in this phase per the study protocol and at the Investigator's discretion.
|
IGSC 20% Treatment Stage 2
n=60 Participants
Following treatment stage 1, participants received 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52. The IGSC 20% dose (mg/kg) remained constant with no dose adjustment permitted in this phase, unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
IGSC 20% Overall
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and the at Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|---|---|
|
Rate of Days on Antibiotics Per Participants Per Year
Prophylactic Antibiotic
|
43.240 Rate of days per participant per year
Interval 24.786 to 69.152
|
44.846 Rate of days per participant per year
Interval 26.197 to 70.755
|
44.432 Rate of days per participant per year
Interval 26.351 to 69.339
|
|
Rate of Days on Antibiotics Per Participants Per Year
Therapeutic Antibiotics
|
13.085 Rate of days per participant per year
Interval 7.777 to 20.387
|
7.451 Rate of days per participant per year
Interval 4.843 to 10.861
|
8.904 Rate of days per participant per year
Interval 5.949 to 12.705
|
SECONDARY outcome
Timeframe: IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53Population: Analysis was performed on the efficacy evaluable population which included all participants who received at least 1 dose of IGSC 20%.
The rate of hospitalization due to infection events per participant per year during IGSC 20% treatment was calculated as the total number of hospitalization due to infection events divided by the total duration of exposure in years across all participants. The 2-sided 95% CI was determined from a generalized linear model for poisson regression for the log-transformed number of events with log-transformed duration of exposure in years as an offset variable.
Outcome measures
| Measure |
IGSC 20% Treatment Stage 1
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower). Dose adjustments were permitted in this phase per the study protocol and at the Investigator's discretion.
|
IGSC 20% Treatment Stage 2
n=60 Participants
Following treatment stage 1, participants received 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52. The IGSC 20% dose (mg/kg) remained constant with no dose adjustment permitted in this phase, unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
IGSC 20% Overall
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and the at Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|---|---|
|
Rate of Hospitalization Due to Infection Per Participants Per Year
|
0 Rate of events per person per year
Here NA signifies that 95% CI was not estimable due to zero number of events.
|
0.023 Rate of events per person per year
Interval 0.01 to 0.044
|
0.017 Rate of events per person per year
Interval 0.008 to 0.033
|
SECONDARY outcome
Timeframe: IGSC 20% Treatment Stage 1: Week 1 to 13; IGSC 20% Treatment Stage 2: Week 14 to 53; IGSC 20% Overall: Week 1 to 53Population: Analysis was performed on the efficacy evaluable population which included all participants who received at least 1 dose of IGSC 20%.
The rate of days of work, school or daily activities missed per participant per year during IGSC 20% treatment was calculated as the total number of days of work/school/daily activities missed divided by the total duration of exposure in years across all participants. The 2-sided 95% CI was determined from a generalized linear model for poisson regression for the log-transformed number of days with log transformed duration of exposure in years as an offset variable.
Outcome measures
| Measure |
IGSC 20% Treatment Stage 1
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower). Dose adjustments were permitted in this phase per the study protocol and at the Investigator's discretion.
|
IGSC 20% Treatment Stage 2
n=60 Participants
Following treatment stage 1, participants received 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52. The IGSC 20% dose (mg/kg) remained constant with no dose adjustment permitted in this phase, unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
IGSC 20% Overall
n=61 Participants
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and the at Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|---|---|
|
Rate of Days of Work/School/Daily Activities Missed Per Participants Per Year Due to Infections and Related Treatment
|
4.118 Rate of days missed per person per year
Interval 2.27 to 6.769
|
5.283 Rate of days missed per person per year
Interval 3.192 to 8.132
|
4.983 Rate of days missed per person per year
Interval 3.064 to 7.572
|
Adverse Events
IGSC 20% Overall
Serious events: 7 serious events
Other events: 49 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
IGSC 20% Overall
n=61 participants at risk
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and the at Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|
|
Injury, poisoning and procedural complications
Joint dislocation
|
1.6%
1/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Cardiac disorders
Aortic valve incompetence
|
1.6%
1/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
1.6%
1/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
General disorders
Medical device site joint pain
|
1.6%
1/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Renal and urinary disorders
Nephrotic syndrome
|
1.6%
1/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Urinary tract infection
|
1.6%
1/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Pneumonia
|
1.6%
1/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
Other adverse events
| Measure |
IGSC 20% Overall
n=61 participants at risk
Following previous regimen phase (screening), participants were enrolled to receive 13 IGSC 20% infusions at weekly intervals from baseline (Week 1) up to Week 13 in treatment stage 1. Participants were infused with IGSC 20% at a 1:1 dose-equivalent regimen from their previous regimen (or a minimum IGSC 20% dose of 100 mg/kg/week if the derived 1:1 dose from the previous regimen was lower) and followed by 39 IGSC 20% infusions at weekly intervals from Week 14 up to Week 52 in treatment stage 2. Dose adjustments were permitted per the study protocol and the at Investigator's discretion in the treatment stage 1. However, dose remained constant in treatment stage 2 unless it was absolutely medically necessary to change the dose, and such change required prior consultation with the Sponsor's Medical Monitor. A final follow-up visit occurred at Week 53.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.8%
9/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Respiratory, thoracic and mediastinal disorders
Headache
|
11.5%
7/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
General disorders
Infusion site erythema
|
16.4%
10/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
General disorders
Infusion site pruritus
|
13.1%
8/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
General disorders
Pyrexia
|
11.5%
7/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
General disorders
Infusion site pain
|
8.2%
5/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
General disorders
Infusion site swelling
|
6.6%
4/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Gastrointestinal disorders
Diarrhoea
|
9.8%
6/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Gastrointestinal disorders
Vomiting
|
8.2%
5/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Gastrointestinal disorders
Nausea
|
6.6%
4/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.2%
5/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.6%
4/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Nasopharyngitis
|
19.7%
12/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Upper respiratory tract infection
|
14.8%
9/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Bronchitis
|
13.1%
8/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Rhinitis
|
11.5%
7/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Sinusitis
|
11.5%
7/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Gastroenteritis
|
9.8%
6/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Lower respiratory tract infection
|
8.2%
5/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Influenza
|
6.6%
4/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
|
Infections and infestations
Viral infection
|
6.6%
4/61 • Treatment-emergent adverse events (TEAEs) were collected from the beginning of treatment administration with IGSC 20% (Baseline/Week 1) until the Final Visit (Week 53). Up to 1 year overall.
TEAEs are presented for the safety population which included all participants who received any amount of IGSC 20%.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Site may publish results from the Study, after providing Sponsor thirty days' notice prior to submitting a manuscript or other materials related to the Study to any outside party. At Sponsors' request, Site will remove any Confidential Information (other than Study results), and Site will upon Sponsors' request, delay publication or presentation for a period of up to one hundred twenty days to allow Sponsor to protect its interests in any Sponsor Inventions.
- Publication restrictions are in place
Restriction type: OTHER