Trial Outcomes & Findings for Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases (NCT NCT03939533)
NCT ID: NCT03939533
Last Updated: 2023-11-07
Results Overview
Mean change from baseline in individual total IgG trough levels in cohort 3 from weekly infusions to end of study (28 weeks) every other week infusions, and for cohort 1 and cohort 2 (weekly infusions) change from baseline through study completion (28 weeks)
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
64 participants
Primary outcome timeframe
Through study completion, up to 28 weeks
Results posted on
2023-11-07
Participant Flow
Subjects with a history of primary immunodeficiency (PI) disease that were currently on a stable does of SCIG treatment were enrolled at 16 research sites across the US between October 2019 and January 2022
Participant milestones
| Measure |
Increased Volume Cohort - Cohort 1
Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
|---|---|---|---|
|
Overall Study
STARTED
|
15
|
15
|
34
|
|
Overall Study
COMPLETED
|
12
|
13
|
30
|
|
Overall Study
NOT COMPLETED
|
3
|
2
|
4
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Monitor Subcutaneous Human Immunoglobulin Administered at Modified Dosing Regimens in Patients With Primary Immunodeficiency Diseases
Baseline characteristics by cohort
| Measure |
Increased Volume Cohort - Cohort 1
n=15 Participants
Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
n=34 Participants
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
Total
n=64 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
51.20 years
STANDARD_DEVIATION 17.27 • n=5 Participants
|
47.88 years
STANDARD_DEVIATION 20.53 • n=7 Participants
|
50.81 years
STANDARD_DEVIATION 18.54 • n=5 Participants
|
50.21 years
STANDARD_DEVIATION 18.49 • n=4 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
48 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
16 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
34 Participants
n=5 Participants
|
61 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Type of PI Disease
Common Variable Immunodeficiency (CVID)
|
14 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
57 Participants
n=4 Participants
|
|
Type of PI Disease
X-linked Agammaglobulinemia (XLA)
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Type of PI Disease
Other
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
6 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Through study completion, up to 28 weeksPopulation: Cohort 3
Mean change from baseline in individual total IgG trough levels in cohort 3 from weekly infusions to end of study (28 weeks) every other week infusions, and for cohort 1 and cohort 2 (weekly infusions) change from baseline through study completion (28 weeks)
Outcome measures
| Measure |
Increased Volume Cohort - Cohort 1
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
n=31 Participants
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
|---|---|---|---|
|
IgG Trough Levels From Baseline to End of Study (28 Weeks)
|
0.144 g/L
Standard Deviation 0.7303
|
0.065 g/L
Standard Deviation 1.1046
|
-0.593 g/L
Standard Deviation 1.0791
|
SECONDARY outcome
Timeframe: Through study completion, up to 28 weeksNumber of subjects who reported SBIs during the study
Outcome measures
| Measure |
Increased Volume Cohort - Cohort 1
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
n=34 Participants
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
|---|---|---|---|
|
Serious Bacterial Infection Rates
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Through study completion, 28 weeksThe amount of days it took for infectious disease occurrence and resolution for subjects
Outcome measures
| Measure |
Increased Volume Cohort - Cohort 1
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
n=34 Participants
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
|---|---|---|---|
|
Time to Resolution of Infections
|
23.5 days
Interval 1.0 to 160.0
|
20.0 days
Interval 11.0 to 85.0
|
16.0 days
Interval 6.0 to 65.0
|
SECONDARY outcome
Timeframe: Through study completion, up to 28 weeksAmount of subjects treated with antibiotics during the study
Outcome measures
| Measure |
Increased Volume Cohort - Cohort 1
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
n=34 Participants
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
|---|---|---|---|
|
Antibiotic Usage
|
10 Participants
|
8 Participants
|
21 Participants
|
SECONDARY outcome
Timeframe: Through study completion, up to 28 weeksTotal number of treatment episodes annualized calculated as the sum of all unique episodes of antibiotics of all subjects from first dose day of cutaquig to last study visit/number of person years exposure
Outcome measures
| Measure |
Increased Volume Cohort - Cohort 1
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
n=15 Participants
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
n=34 Participants
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
|---|---|---|---|
|
Number of Antibiotic Treatment Episodes Annualized
|
4.53 episodes per person year
|
1.96 episodes per person year
|
2.38 episodes per person year
|
Adverse Events
Increased Volume Cohort - Cohort 1
Serious events: 1 serious events
Other events: 13 other events
Deaths: 0 deaths
Increased Infusion Rate Cohort - Cohort 2
Serious events: 1 serious events
Other events: 12 other events
Deaths: 0 deaths
Every Other Week Dosing Cohort - Cohort 3
Serious events: 1 serious events
Other events: 30 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Increased Volume Cohort - Cohort 1
n=15 participants at risk
Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
n=15 participants at risk
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
n=34 participants at risk
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
|---|---|---|---|
|
Infections and infestations
COVID-19
|
0.00%
0/15 • Through study complete, up to 28 weeks
|
0.00%
0/15 • Through study complete, up to 28 weeks
|
2.9%
1/34 • Number of events 1 • Through study complete, up to 28 weeks
|
|
Metabolism and nutrition disorders
Dehydration
|
6.7%
1/15 • Number of events 1 • Through study complete, up to 28 weeks
|
0.00%
0/15 • Through study complete, up to 28 weeks
|
0.00%
0/34 • Through study complete, up to 28 weeks
|
|
Musculoskeletal and connective tissue disorders
Rheumatoid Arthritis
|
0.00%
0/15 • Through study complete, up to 28 weeks
|
6.7%
1/15 • Number of events 1 • Through study complete, up to 28 weeks
|
0.00%
0/34 • Through study complete, up to 28 weeks
|
Other adverse events
| Measure |
Increased Volume Cohort - Cohort 1
n=15 participants at risk
Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Increased Infusion Rate Cohort - Cohort 2
n=15 participants at risk
Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
CUTAQUIG: Human normal immunoglobulin
|
Every Other Week Dosing Cohort - Cohort 3
n=34 participants at risk
Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent \[mg/kg\] weekly dose
CUTAQUIG: Human normal immunoglobulin
|
|---|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
6.7%
1/15 • Number of events 1 • Through study complete, up to 28 weeks
|
0.00%
0/15 • Through study complete, up to 28 weeks
|
8.8%
3/34 • Number of events 4 • Through study complete, up to 28 weeks
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/15 • Through study complete, up to 28 weeks
|
26.7%
4/15 • Number of events 9 • Through study complete, up to 28 weeks
|
5.9%
2/34 • Number of events 5 • Through study complete, up to 28 weeks
|
|
General disorders
Fatigue
|
13.3%
2/15 • Number of events 2 • Through study complete, up to 28 weeks
|
6.7%
1/15 • Number of events 7 • Through study complete, up to 28 weeks
|
2.9%
1/34 • Number of events 1 • Through study complete, up to 28 weeks
|
|
General disorders
Infusion Site Erythema
|
26.7%
4/15 • Number of events 7 • Through study complete, up to 28 weeks
|
53.3%
8/15 • Number of events 54 • Through study complete, up to 28 weeks
|
23.5%
8/34 • Number of events 12 • Through study complete, up to 28 weeks
|
|
General disorders
Infusion Site Pain
|
13.3%
2/15 • Number of events 2 • Through study complete, up to 28 weeks
|
26.7%
4/15 • Number of events 10 • Through study complete, up to 28 weeks
|
8.8%
3/34 • Number of events 7 • Through study complete, up to 28 weeks
|
|
General disorders
Infusion Site Pruritis
|
26.7%
4/15 • Number of events 15 • Through study complete, up to 28 weeks
|
33.3%
5/15 • Number of events 19 • Through study complete, up to 28 weeks
|
17.6%
6/34 • Number of events 10 • Through study complete, up to 28 weeks
|
|
General disorders
Pyrexia
|
6.7%
1/15 • Number of events 1 • Through study complete, up to 28 weeks
|
6.7%
1/15 • Number of events 4 • Through study complete, up to 28 weeks
|
5.9%
2/34 • Number of events 3 • Through study complete, up to 28 weeks
|
|
Infections and infestations
Acute Sinitus
|
13.3%
2/15 • Number of events 2 • Through study complete, up to 28 weeks
|
13.3%
2/15 • Number of events 3 • Through study complete, up to 28 weeks
|
5.9%
2/34 • Number of events 2 • Through study complete, up to 28 weeks
|
|
Ear and labyrinth disorders
Ear Infection
|
13.3%
2/15 • Number of events 2 • Through study complete, up to 28 weeks
|
0.00%
0/15 • Through study complete, up to 28 weeks
|
5.9%
2/34 • Number of events 2 • Through study complete, up to 28 weeks
|
|
Infections and infestations
Sinusitis
|
26.7%
4/15 • Number of events 4 • Through study complete, up to 28 weeks
|
33.3%
5/15 • Number of events 8 • Through study complete, up to 28 weeks
|
17.6%
6/34 • Number of events 9 • Through study complete, up to 28 weeks
|
|
Infections and infestations
Urinary Tract Infection
|
20.0%
3/15 • Number of events 3 • Through study complete, up to 28 weeks
|
6.7%
1/15 • Number of events 1 • Through study complete, up to 28 weeks
|
8.8%
3/34 • Number of events 3 • Through study complete, up to 28 weeks
|
|
Nervous system disorders
Heachache
|
0.00%
0/15 • Through study complete, up to 28 weeks
|
20.0%
3/15 • Number of events 4 • Through study complete, up to 28 weeks
|
20.6%
7/34 • Number of events 16 • Through study complete, up to 28 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place