Trial Outcomes & Findings for Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia (NCT NCT01349790)
NCT ID: NCT01349790
Last Updated: 2017-06-07
Results Overview
A responder is a study participant with an increase in platelets to ≥ 50x10\^9/L within 7 days after the first infusion, ie, by study Day 8.
COMPLETED
PHASE3
40 participants
Day 1 to Day 8
2017-06-07
Participant Flow
Participant milestones
| Measure |
NewGam
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Overall Study
STARTED
|
40
|
|
Overall Study
COMPLETED
|
31
|
|
Overall Study
NOT COMPLETED
|
9
|
Reasons for withdrawal
| Measure |
NewGam
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Overall Study
Investigator Judgement
|
3
|
|
Overall Study
Death
|
2
|
|
Overall Study
Withdrawal by Patient
|
2
|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
Efficacy and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Primary Immune Thrombocytopenia
Baseline characteristics by cohort
| Measure |
NewGam
n=40 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Age, Continuous
|
36.7 years
STANDARD_DEVIATION 15.34 • n=99 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Day 1 to Day 8Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration.
A responder is a study participant with an increase in platelets to ≥ 50x10\^9/L within 7 days after the first infusion, ie, by study Day 8.
Outcome measures
| Measure |
NewGam
n=36 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Percentage of Responders
|
80.6 Percentage of participants
Interval 63.98 to 91.81
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration.
An alternative responder is a study participant with an increase in platelets to ≥ 30x10\^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.
Outcome measures
| Measure |
NewGam
n=36 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Percentage of Alternative Responders
|
66.7 Percentage of participants
Interval 49.03 to 81.44
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration.
A complete responder is a study participant with an increase in platelets to ≥ 100x10\^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.
Outcome measures
| Measure |
NewGam
n=36 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Percentage of Complete Responders
|
50.0 Percentage of participants
Interval 32.92 to 67.08
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration. Only participants with an alternative response were included in the analysis.
An alternative responder who lost the response is a study participant who met the criterion for an alternative response but who then deteriorated, ie, their platelet count decreased to \< 30x10\^9/L, their platelet count decreased to less than double the baseline count, or bleeding occurred.
Outcome measures
| Measure |
NewGam
n=24 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Percentage of Alternative Responders Who Lost the Response
|
45.8 Percentage of participants
Interval 25.55 to 67.18
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam and who had at least 1 post-baseline measurement of platelet concentration. Only participants with a complete response were included in the analysis.
A complete responder who lost the response is a study participant who met the criterion for a complete response but who then deteriorated, ie, their platelet count decreased to \< 100x10\^9/L or bleeding occurred.
Outcome measures
| Measure |
NewGam
n=18 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Percentage of Complete Responders Who Lost the Response
|
77.8 Percentage of participants
Interval 52.36 to 93.49
|
SECONDARY outcome
Timeframe: Day 1 to Day 8Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only responders were included in the analysis.
A study participant had a response if their platelets increased to ≥ 50x10\^9/L within 7 days after the first infusion, ie, by study Day 8.
Outcome measures
| Measure |
NewGam
n=29 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Time to a Response
|
2.0 Days
Interval 1.0 to 2.0
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only participants with an alternative response were included in the analysis.
A study participant had a response if their platelets increased to ≥ 30x10\^9/L and to at least double the baseline platelet count, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.
Outcome measures
| Measure |
NewGam
n=24 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Time to an Alternative Response
|
1.0 Days
Interval 1.0 to 2.0
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only participants with a complete response were included in the analysis.
A study participant had a complete response if their platelets increased to ≥ 100x10\^9/L, confirmed on at least 2 occasions at least 7 days apart, and absence of bleeding.
Outcome measures
| Measure |
NewGam
n=18 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Time to a Complete Response
|
2.0 Days
Interval 2.0 to 2.0
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only responders were included in the analysis.
The duration of a response was defined as the time from when a response was achieved until the platelet count fell below 50x10\^9/L.
Outcome measures
| Measure |
NewGam
n=29 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Duration of a Response
|
14.0 Days
Interval 10.0 to 17.0
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only participants with an alternative response were included in the analysis.
The duration of an alternative response was defined as the time from when an alternative response was achieved until the platelet count fell below 50x10\^9/L.
Outcome measures
| Measure |
NewGam
n=24 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Duration of an Alternative Response
|
18.5 Days
Interval 17.0 to 20.0
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only participants with a complete response were included in the analysis.
The duration of a complete response was defined as the time from when a complete response was achieved until the platelet count fell below 50x10\^9/L.
Outcome measures
| Measure |
NewGam
n=18 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Duration of a Complete Response
|
12.5 Days
Interval 10.0 to 16.0
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration.
The platelet count at each study visit are presented.
Outcome measures
| Measure |
NewGam
n=36 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Platelet Count by Visit
Baseline
|
8.8 10^9 platelets/L
Standard Deviation 5.76
|
|
Platelet Count by Visit
Day 2
|
41.6 10^9 platelets/L
Standard Deviation 31.41
|
|
Platelet Count by Visit
Day 3
|
115.0 10^9 platelets/L
Standard Deviation 85.58
|
|
Platelet Count by Visit
Day 4
|
154.6 10^9 platelets/L
Standard Deviation 118.04
|
|
Platelet Count by Visit
Day 5
|
168.2 10^9 platelets/L
Standard Deviation 149.89
|
|
Platelet Count by Visit
Day 6
|
179.3 10^9 platelets/L
Standard Deviation 178.64
|
|
Platelet Count by Visit
Day 7
|
206.3 10^9 platelets/L
Standard Deviation 195.75
|
|
Platelet Count by Visit
Day 8
|
186.5 10^9 platelets/L
Standard Deviation 205.44
|
|
Platelet Count by Visit
Day 15
|
88.1 10^9 platelets/L
Standard Deviation 130.07
|
|
Platelet Count by Visit
Day 22/End of treatment
|
41.4 10^9 platelets/L
Standard Deviation 65.48
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration.
The maximum platelet count achieved during the study is presented.
Outcome measures
| Measure |
NewGam
n=36 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Maximum Platelet Count
|
236.9 10^9 platelets/L
Standard Deviation 205.18
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration. Only responders were included in the analysis.
The percentage of responders who achieved a normal platelet count is presented.
Outcome measures
| Measure |
NewGam
n=29 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Percentage of Responders Who Achieved a Normal Platelet Count
|
79.3 Percentage of participants
Interval 60.28 to 92.01
|
SECONDARY outcome
Timeframe: Day 1 to Day 22Population: Full analysis set: All participants enrolled in the study who received at least part of 1 dose of NewGam who had at least 1 post-baseline measurement of platelet concentration.
The percentage of participants with various intensities of overall bleeding, epistaxis (bleeding of the nose), oral bleeding, and skin bleeding graded as none, minor, mild, moderate, or severe at Baseline and Day 22 are reported.
Outcome measures
| Measure |
NewGam
n=36 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Bleeding Intensity
Overall - Total - Day 22
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Overall - None - Baseline
|
36.1 Percentage of participants
|
|
Bleeding Intensity
Overall - None - Day 8
|
91.2 Percentage of participants
|
|
Bleeding Intensity
Overall - None - Day 22
|
72.2 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Moderate - Baseline
|
5.6 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Mild - Baseline
|
27.8 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Mild - Day 22
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Moderate - Baseline
|
5.6 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Moderate - Day 22
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Overall - Total - Baseline
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Overall - Total - Day 8
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Overall - Minor - Baseline
|
38.9 Percentage of participants
|
|
Bleeding Intensity
Overall - Minor - Day 8
|
5.9 Percentage of participants
|
|
Bleeding Intensity
Overall - Minor - Day 22
|
19.4 Percentage of participants
|
|
Bleeding Intensity
Overall - Mild - Baseline
|
5.6 Percentage of participants
|
|
Bleeding Intensity
Overall - Mild - Day 8
|
2.9 Percentage of participants
|
|
Bleeding Intensity
Overall - Mild - Day 22
|
2.8 Percentage of participants
|
|
Bleeding Intensity
Overall - Moderate - Baseline
|
19.4 Percentage of participants
|
|
Bleeding Intensity
Overall - Moderate - Day 8
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Overall - Moderate - Day 22
|
2.8 Percentage of participants
|
|
Bleeding Intensity
Overall - Severe - Baseline
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Overall - Severe - Day 8
|
0 Percentage of participants
|
|
Bleeding Intensity
Overall - Severe - Day 22
|
2.8 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Total - Baseline
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Total - Day 22
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - None - Baseline
|
83.3 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - None - Day 22
|
91.7 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Minor - Baseline
|
8.3 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Minor - Day 22
|
8.3 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Mild - Baseline
|
5.6 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Mild - Day 22
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Moderate - Baseline
|
2.8 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Moderate - Day 22
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Severe - Baseline
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Epistaxis - Severe - Day 22
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Total - Baseline
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Total - Day 22
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - None - Baseline
|
75.0 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - None - Day 22
|
86.1 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Minor - Baseline
|
13.9 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Minor - Day 22
|
8.3 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Mild - Baseline
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Mild - Day 22
|
2.8 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Moderate - Day 22
|
2.8 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Severe - Baseline
|
5.6 Percentage of participants
|
|
Bleeding Intensity
Oral bleeding - Severe - Day 22
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Total - Baseline
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Total - Day 22
|
100.0 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - None - Baseline
|
41.7 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - None - Day 22
|
77.8 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Minor - Baseline
|
25.0 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Minor - Day 22
|
22.2 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Severe - Baseline
|
0.0 Percentage of participants
|
|
Bleeding Intensity
Skin bleeding - Severe - Day 22
|
0.0 Percentage of participants
|
SECONDARY outcome
Timeframe: Day 1 to Day 2Population: Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
The percentage of participants who achieved a platelet count \> 30x10\^9/L within 1 and 2 days after infusion is reported.
Outcome measures
| Measure |
NewGam
n=40 Participants
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L
Day 1
|
55.0 Percentage of participants
|
|
Percentage of Participants Who Achieved a Platelet Count > 30x10^9/L
Day 2
|
77.5 Percentage of participants
|
Adverse Events
NewGam
Serious adverse events
| Measure |
NewGam
n=40 participants at risk
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Blood and lymphatic system disorders
Autoimmune thrombocytopenia
|
7.5%
3/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
2.5%
1/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Gastrointestinal disorders
Dysphagia
|
2.5%
1/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Nervous system disorders
Cerebral haematoma
|
2.5%
1/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Nervous system disorders
Meningitis aseptic
|
2.5%
1/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Infections and infestations
Pneumonia
|
2.5%
1/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.5%
1/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Infections and infestations
Sepsis
|
2.5%
1/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
Other adverse events
| Measure |
NewGam
n=40 participants at risk
Each participant received 1 g/kg NewGam intravenously on 2 consecutive days.
|
|---|---|
|
Nervous system disorders
Headache
|
42.5%
17/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Nervous system disorders
Dizziness
|
7.5%
3/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Blood and lymphatic system disorders
Autoimmune thrombocytopenia
|
15.0%
6/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Blood and lymphatic system disorders
Anaemia
|
12.5%
5/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Blood and lymphatic system disorders
Idiopathic thrombocytopenic purpura
|
5.0%
2/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Gastrointestinal disorders
Nausea
|
15.0%
6/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
4/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
General disorders
Asthenia
|
5.0%
2/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
General disorders
Chills
|
5.0%
2/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
|
General disorders
Pyrexia
|
22.5%
9/40
Safety set: All participants enrolled in the study who received at least part of 1 dose of NewGam.
|
Additional Information
Michael Eppolito, Director, Clinical Operations Immunology and ICU Medicine
Octapharma USA
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place