Trial Outcomes & Findings for Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects (NCT NCT00634569)
NCT ID: NCT00634569
Last Updated: 2017-02-02
Results Overview
Total number of Bacterial pneumonia, bacteremia or sepsis, osteomyelitis/septic arthritis, visceral abscess or bacterial meningitis
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
24 participants
Primary outcome timeframe
12 months
Results posted on
2017-02-02
Participant Flow
Participant milestones
| Measure |
21-day Dosing
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Overall Study
STARTED
|
14
|
10
|
|
Overall Study
COMPLETED
|
14
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
5
|
Reasons for withdrawal
| Measure |
21-day Dosing
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
4
|
|
Overall Study
Physician Decision
|
0
|
1
|
Baseline Characteristics
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
Baseline characteristics by cohort
| Measure |
21-day Dosing
n=14 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
n=10 Participants
IGIV given every 28-days (300-800 mg/kg)
|
Total
n=24 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
8.6 years
STANDARD_DEVIATION 4.3 • n=99 Participants
|
9.7 years
STANDARD_DEVIATION 4.9 • n=107 Participants
|
9.0 years
STANDARD_DEVIATION 4.53 • n=206 Participants
|
|
Gender
Female
|
4 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Gender
Male
|
10 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
24 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
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 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
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
22 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 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
|
PRIMARY outcome
Timeframe: 12 monthsTotal number of Bacterial pneumonia, bacteremia or sepsis, osteomyelitis/septic arthritis, visceral abscess or bacterial meningitis
Outcome measures
| Measure |
21-day Dosing
n=14 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
n=10 Participants
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Serious Bacterial Infections.
|
1 Total serious bacterial infections
|
0 Total serious bacterial infections
|
SECONDARY outcome
Timeframe: 12 monthsMean Days of school/usual activities missed per subject/year
Outcome measures
| Measure |
21-day Dosing
n=24 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Days of School/Usual Activities Missed Per Year
|
2.0 Days
Standard Deviation 3.33
|
—
|
SECONDARY outcome
Timeframe: 12 monthsMean Days of hospitalization per subject/year
Outcome measures
| Measure |
21-day Dosing
n=24 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Days of Hospitalization Per Year
|
0.2 Days
Standard Deviation 1.04
|
—
|
SECONDARY outcome
Timeframe: 12 monthsMean Number of visits to physician/ER room for acute problems
Outcome measures
| Measure |
21-day Dosing
n=24 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Number of Visits to Physician/ER Room for Acute Problems
|
0 Visits
Standard Deviation 0.0
|
—
|
SECONDARY outcome
Timeframe: 12 monthsOutcome measures
| Measure |
21-day Dosing
n=24 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Other Infections Documented by Fever and Physical Exam or Positive Radiograph.
|
0 Number of other infections
Standard Deviation 0.0
|
—
|
SECONDARY outcome
Timeframe: 12 monthsMean Number of infectious episodes per subject/year
Outcome measures
| Measure |
21-day Dosing
n=24 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Number of Infectious Episodes Per Year
|
0.9 Infectious episodes
Standard Deviation 1.44
|
—
|
SECONDARY outcome
Timeframe: 12 monthsMedian Combined number of days on prophylactic and therapeutic antibiotics
Outcome measures
| Measure |
21-day Dosing
n=24 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Number of Days on Antibiotics (Prophylactic and Therapeutic).
|
72 Days
Standard Deviation 150.45
|
—
|
SECONDARY outcome
Timeframe: 12 monthsTotal Number of Adverse Events
Outcome measures
| Measure |
21-day Dosing
n=24 Participants
IGIV given every 21-days (225-600 mg/kg)
|
28-day Dosing
IGIV given every 28-days (300-800 mg/kg)
|
|---|---|---|
|
Number of Adverse Events
|
159 All Adverse Events
|
—
|
Adverse Events
All Subjects
Serious events: 1 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Subjects
n=24 participants at risk
Intent-to-treat population
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Cellulitis
|
4.2%
1/24 • Number of events 1 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Bronchiectasis
|
4.2%
1/24 • Number of events 1 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
|
Musculoskeletal and connective tissue disorders
Femoral neck fracture
|
4.2%
1/24 • Number of events 1 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
|
Ear and labyrinth disorders
Deafness
|
4.2%
1/24 • Number of events 1 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
Other adverse events
| Measure |
All Subjects
n=24 participants at risk
Intent-to-treat population
|
|---|---|
|
General disorders
Headache
|
41.7%
10/24 • Number of events 31 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
|
Nervous system disorders
Diastolic hypotension
|
20.8%
5/24 • Number of events 20 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
|
Infections and infestations
Pyrexia
|
29.2%
7/24 • Number of events 19 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
|
Nervous system disorders
Hypotension
|
25.0%
6/24 • Number of events 19 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
|
Cardiac disorders
Tachycardia
|
25.0%
6/24 • Number of events 16 • 1 year
Systematic temporal assessment of AEs (by vital sign measurement up to 72 hours post-infusion) occurring over 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place