Trial Outcomes & Findings for Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary Immunodeficiency (NCT NCT02604810)
NCT ID: NCT02604810
Last Updated: 2019-10-04
Results Overview
The primary PK endpoint (steady-state AUC values) analysis was performed using analysis of variance (ANOVA) using PK data from a total of 49 subjects from the IV phase and 39 subjects from the SC phase.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
53 participants
Primary outcome timeframe
For intravenous infusion, predose, 0,1,3-16 hours and 1,2,3,5,7,14,21 or 28 days (2, 7, 21, or 28 days for pediatric subjects) post-dose and for subcutaneous infusion, pre-dose,1,3,4,5,7 days (3 and 7 days for pediatric subjects) post-dose
Results posted on
2019-10-04
Participant Flow
A total of 61 subjects were screened for participation in this study. Of these 61 subjects, 8 subjects were considered screen failures. A total of 20 investigators in the United States enrolled subjects in this study. The first subject was enrolled in this study on 04 Jan 2016 and the date of the last subject's last study visit was 14 Dec 2017.
One subject was lost to follow-up before the IGIV-C Phase.
Participant milestones
| Measure |
IGIV-C 10% First, IGSC 20% Second
IV dose of Immune Globulin Injection (Human), 10% Caprylate/Chromatography Purified (Grifols)
IGIV-C 10%: IGIV-C 10% infusions every 3 to 4 weeks based on previous IgG regimen
After completing the IGIV-C 10% infusions, subjects continued to receive IGSC 20% infusions.
Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (Grifols)
IGSC 20%: IGSC 20% weekly infusions with dose calculated based on previous IgG regimen
|
|---|---|
|
IGIV-C 10%, 4-5 Weeks
STARTED
|
52
|
|
IGIV-C 10%, 4-5 Weeks
COMPLETED
|
49
|
|
IGIV-C 10%, 4-5 Weeks
NOT COMPLETED
|
3
|
|
IGSC 20%, 24 Weeks
STARTED
|
49
|
|
IGSC 20%, 24 Weeks
COMPLETED
|
42
|
|
IGSC 20%, 24 Weeks
NOT COMPLETED
|
7
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Pharmacokinetics of IGSC 20% in Subjects With Primary Immunodeficiency
Baseline characteristics by cohort
| Measure |
IGIV-C 10% First, IGSC 20% Second
n=53 Participants
IV dose of Immune Globulin Injection (Human), 10% Caprylate/Chromatography Purified (Grifols)
IGIV-C 10%: IGIV-C 10% infusions every 3 to 4 weeks based on previous IgG regimen
After completing the IGIV-C 10% infusions, subjects continued to receive IGSC 20% infusions.
Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (Grifols)
IGSC 20%: IGSC 20% weekly infusions with dose calculated based on previous IgG regimen
|
|---|---|
|
Age, Categorical
<=18 years
|
15 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
33 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
5 Participants
n=5 Participants
|
|
Age, Continuous
|
36.8 years
STANDARD_DEVIATION 21.36 • n=5 Participants
|
|
Sex: Female, Male
Female
|
26 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
27 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
48 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
48 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: For intravenous infusion, predose, 0,1,3-16 hours and 1,2,3,5,7,14,21 or 28 days (2, 7, 21, or 28 days for pediatric subjects) post-dose and for subcutaneous infusion, pre-dose,1,3,4,5,7 days (3 and 7 days for pediatric subjects) post-dosePopulation: The PK population consisted of all subjects who received study drugs and had sufficient and valid total IgG concentration versus time data for either the IV or SC Phase to allow calculation of AUC0-τ,SC or AUC0-τ,IV (the primary PK endpoint).
The primary PK endpoint (steady-state AUC values) analysis was performed using analysis of variance (ANOVA) using PK data from a total of 49 subjects from the IV phase and 39 subjects from the SC phase.
Outcome measures
| Measure |
IGIV-C 10%
n=49 Participants
IV dose of Immune Globulin Injection (Human), 10% Caprylate/Chromatography Purified (Grifols)
IGIV-C 10%: IGIV-C 10% infusions every 3 to 4 weeks based on previous IgG regimen
|
IGSC 20%
n=39 Participants
Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (Grifols)
IGSC 20%: IGSC 20% weekly infusions with dose calculated based on previous IgG regimen
|
|---|---|---|
|
AUC in the IV Phase and SC Phases: Steady-state AUC of Total IgG Over a Regular Dosing Interval
|
207921.5 h*mg/dL
Interval 197865.8 to 218488.2
|
213141.4 h*mg/dL
Interval 200568.6 to 226502.3
|
SECONDARY outcome
Timeframe: For intravenous infusion, pre-dose at Week 1 and Week 3 or Week 4 and for subcutaneous infusion, predose at Weeks 13, 14, 17, and 21Outcome measures
| Measure |
IGIV-C 10%
n=51 Participants
IV dose of Immune Globulin Injection (Human), 10% Caprylate/Chromatography Purified (Grifols)
IGIV-C 10%: IGIV-C 10% infusions every 3 to 4 weeks based on previous IgG regimen
|
IGSC 20%
n=44 Participants
Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (Grifols)
IGSC 20%: IGSC 20% weekly infusions with dose calculated based on previous IgG regimen
|
|---|---|---|
|
Mean Steady-state Trough (Pre-dose) Concentration of Total IgG Following IV Administration of IGIV-C 10% or SC Administration of IGSC 20%
|
957.13 mg/dL
Interval 351.0 to 1920.0
|
1244.84 mg/dL
Interval 651.5 to 2047.5
|
Adverse Events
IGIV-C 10%
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
IGSC 20%
Serious events: 2 serious events
Other events: 35 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
IGIV-C 10%
n=52 participants at risk
IV dose of Immune Globulin Injection (Human), 10% Caprylate/Chromatography Purified (Grifols)
IGIV-C 10%: IGIV-C 10% infusions every 3 to 4 weeks based on previous IgG regimen
|
IGSC 20%
n=49 participants at risk
Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (Grifols)
IGSC 20%: IGSC 20% weekly infusions with dose calculated based on previous IgG regimen
|
|---|---|---|
|
Infections and infestations
Pneumonia bacterial
|
1.9%
1/52 • Number of events 1 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
0.00%
0/49 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Infections and infestations
Sepsis
|
1.9%
1/52 • Number of events 1 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
2.0%
1/49 • Number of events 1 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Injury, poisoning and procedural complications
Animal bite
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
2.0%
1/49 • Number of events 1 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Infections and infestations
Cellulitis
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
2.0%
1/49 • Number of events 1 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
2.0%
1/49 • Number of events 1 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
Other adverse events
| Measure |
IGIV-C 10%
n=52 participants at risk
IV dose of Immune Globulin Injection (Human), 10% Caprylate/Chromatography Purified (Grifols)
IGIV-C 10%: IGIV-C 10% infusions every 3 to 4 weeks based on previous IgG regimen
|
IGSC 20%
n=49 participants at risk
Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (Grifols)
IGSC 20%: IGSC 20% weekly infusions with dose calculated based on previous IgG regimen
|
|---|---|---|
|
Infections and infestations
Sinusitis
|
5.8%
3/52 • Number of events 3 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
18.4%
9/49 • Number of events 10 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
General disorders
Infusion site bruising
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
6.1%
3/49 • Number of events 3 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
General disorders
Infusion site nodule
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
12.2%
6/49 • Number of events 9 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
General disorders
Infusion site pain
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
6.1%
3/49 • Number of events 3 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Infections and infestations
Bronchitis
|
3.8%
2/52 • Number of events 2 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
6.1%
3/49 • Number of events 3 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
6.1%
3/49 • Number of events 3 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
10.2%
5/49 • Number of events 6 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.9%
1/52 • Number of events 1 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
6.1%
3/49 • Number of events 4 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/52 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
6.1%
3/49 • Number of events 3 • During the IV phase, the adverse event data were collected for 4 to 5 weeks. During the SC phase, the adverse event data were collected for 24 weeks.
|
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