Trial Outcomes & Findings for Intravenous Immunoglobulin for PANDAS (NCT NCT01281969)
NCT ID: NCT01281969
Last Updated: 2020-03-17
Results Overview
Active IVIG will be significantly superior to sham IVIG in reducing OC symptoms and providing global relief of neuropsychiatric symptomatology. Total score is reported as the sum of all items and has a range of 0-40. Higher scores indicate more severe symptoms.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
48 participants
Primary outcome timeframe
6 weeks
Results posted on
2020-03-17
Participant Flow
Participant milestones
| Measure |
IVIG
Gamunex Intravenous Immunoglobulin: 2.0 gm/kg total, IV (in the vein), over 2 days
|
Placebo
Placebo: Normal saline, IV (in the vein), over 2 days
|
Screened But Not Randomized
Excluded prior to randomization because child did not meet study entry criteria or child refused consent
|
|---|---|---|---|
|
Overall Study
STARTED
|
18
|
18
|
12
|
|
Overall Study
COMPLETED
|
17
|
18
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
12
|
Reasons for withdrawal
| Measure |
IVIG
Gamunex Intravenous Immunoglobulin: 2.0 gm/kg total, IV (in the vein), over 2 days
|
Placebo
Placebo: Normal saline, IV (in the vein), over 2 days
|
Screened But Not Randomized
Excluded prior to randomization because child did not meet study entry criteria or child refused consent
|
|---|---|---|---|
|
Overall Study
not randomized didn't meet criteria
|
0
|
0
|
7
|
|
Overall Study
AE during LP prior to IVIG admin
|
1
|
0
|
3
|
|
Overall Study
not randomized, child consent not given
|
0
|
0
|
2
|
Baseline Characteristics
Intravenous Immunoglobulin for PANDAS
Baseline characteristics by cohort
| Measure |
IVIG
n=17 Participants
Gamunex Intravenous Immunoglobulin: 2.0 gm/kg total, IV (in the vein), over 2 days
|
Placebo
n=18 Participants
Placebo: Normal saline, IV (in the vein), over 2 days
|
Total
n=35 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
8.99 years
STANDARD_DEVIATION 2.37 • n=99 Participants
|
9.61 years
STANDARD_DEVIATION 2.32 • n=107 Participants
|
9.30 years
STANDARD_DEVIATION 2.32 • n=206 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=99 Participants
|
17 Participants
n=107 Participants
|
33 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=99 Participants
|
16 Participants
n=107 Participants
|
30 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
17 participants
n=99 Participants
|
18 participants
n=107 Participants
|
35 participants
n=206 Participants
|
|
Clinical Global Impressions Severity
Moderate (4)
|
2 participants
n=99 Participants
|
3 participants
n=107 Participants
|
5 participants
n=206 Participants
|
|
Clinical Global Impressions Severity
Marked (5)
|
8 participants
n=99 Participants
|
9 participants
n=107 Participants
|
17 participants
n=206 Participants
|
|
Clinical Global Impressions Severity
Severe (6)
|
7 participants
n=99 Participants
|
4 participants
n=107 Participants
|
11 participants
n=206 Participants
|
|
Clinical Global Impressions Severity
Extreme (7)
|
0 participants
n=99 Participants
|
2 participants
n=107 Participants
|
2 participants
n=206 Participants
|
|
Children's Yale-Brown Obsessive Compulsive Scale Total
|
26.47 units on a scale
STANDARD_DEVIATION 5.14 • n=99 Participants
|
28.78 units on a scale
STANDARD_DEVIATION 3.98 • n=107 Participants
|
27.65 units on a scale
STANDARD_DEVIATION 4.66 • n=206 Participants
|
PRIMARY outcome
Timeframe: 6 weeksActive IVIG will be significantly superior to sham IVIG in reducing OC symptoms and providing global relief of neuropsychiatric symptomatology. Total score is reported as the sum of all items and has a range of 0-40. Higher scores indicate more severe symptoms.
Outcome measures
| Measure |
Group A
n=17 Participants
Gamunex Intravenous Immunoglobulin: 2.0 gm/kg total, IV (in the vein), over 2 days
|
Group B
n=18 Participants
Placebo: Normal saline, IV (in the vein), over 2 days
|
|---|---|---|
|
Children's Yale-Brown Obsessive Compulsive Scale Total Score
|
20.59 units on a scale
Standard Deviation 10.12
|
25.67 units on a scale
Standard Deviation 8.65
|
SECONDARY outcome
Timeframe: 6 weeks1=very much improved, 2=much improved, 3=slightly improved, 4=no change, 5=slightly worse, 6=much worse, 7=very much worse
Outcome measures
| Measure |
Group A
n=17 Participants
Gamunex Intravenous Immunoglobulin: 2.0 gm/kg total, IV (in the vein), over 2 days
|
Group B
n=18 Participants
Placebo: Normal saline, IV (in the vein), over 2 days
|
|---|---|---|
|
Clinical Global Impressions Improvement
|
2.88 units on a scale
Standard Deviation 1.20
|
3.53 units on a scale
Standard Deviation 1.62
|
SECONDARY outcome
Timeframe: 6 weeksDefined as a CGI-I score of 1 or 2 ("much" or "very much" improved) and a decrease in CY-BOCS of at least 30%
Outcome measures
| Measure |
Group A
n=17 Participants
Gamunex Intravenous Immunoglobulin: 2.0 gm/kg total, IV (in the vein), over 2 days
|
Group B
n=18 Participants
Placebo: Normal saline, IV (in the vein), over 2 days
|
|---|---|---|
|
Clinical Responder to Treatment
|
6 participants
|
4 participants
|
SECONDARY outcome
Timeframe: BaselineNon-zero values of antinuclear antibodies are considered "positive" and reflective of an ongoing immune response in the individual. First, the number of participants who were classified at baseline as having "positive" antinuclear antibodies was calculated (see outcome measure data table, which states the number (AKA "count") of participants who had "positive" antinuclear antibodies at baseline). We hypothesized that improvement in the ongoing immune response, and therefore a reduction in antinuclear antibody titers, would mediate the effect of IVIG on OCD symptom improvement. However, because very few participants were classified as "positive" at baseline, it was not appropriate to pursue the original question of whether a decline in antinuclear antibodies (i.e., from "positive" to "negative") was related to symptom improvement.
Outcome measures
| Measure |
Group A
n=17 Participants
Gamunex Intravenous Immunoglobulin: 2.0 gm/kg total, IV (in the vein), over 2 days
|
Group B
n=18 Participants
Placebo: Normal saline, IV (in the vein), over 2 days
|
|---|---|---|
|
The Degree of Treatment Response is Expected to Correlate With the Percentage Reduction in Antinuclear Antibody Titers Following IVIG Administration.
|
8 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: 3 MonthsOutcome measures
Outcome data not reported
Adverse Events
Group A
Serious events: 0 serious events
Other events: 17 other events
Deaths: 0 deaths
Group B
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Group A
n=17 participants at risk
Gamunex Intravenous Immunoglobulin: 2.0 gm/kg total, IV (in the vein), over 2 days
|
Group B
n=18 participants at risk
Placebo: Normal saline, IV (in the vein), over 2 days
|
|---|---|---|
|
General disorders
Headache
|
47.1%
8/17 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
16.7%
3/18 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
|
General disorders
Sore throat
|
5.9%
1/17 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
11.1%
2/18 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
|
General disorders
Stomach or abdominal discomfort
|
17.6%
3/17 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
5.6%
1/18 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
|
General disorders
Nausea (vomiting)
|
23.5%
4/17 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
5.6%
1/18 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
|
General disorders
Muscle/bone/joint pain
|
17.6%
3/17 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
11.1%
2/18 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
|
General disorders
Tiredness/fatigue
|
11.8%
2/17 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
5.6%
1/18 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
|
General disorders
Anxiety
|
11.8%
2/17 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
11.1%
2/18 • AE data were collected 1 week post-infusion and at 6 weeks post infusion.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place