Trial Outcomes & Findings for Treatment of Chronic Immune Thrombocytopenic Purpura (ITP) With Intravenous Immunoglobulin IgPro10 (NCT NCT00168038)

NCT ID: NCT00168038

Last Updated: 2011-11-23

Results Overview

The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10\^9/L to ≥ 50 x 10\^9/L within the specified time frame.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 58 participants
• Primary outcome timeframe: 7 days
• Results posted on: 2011-11-23

Participant Flow

The study was performed as a multicenter study at 17 centers, 6 in Poland, 4 in the Ukraine, 4 in Russia, 1 in Germany, 1 in Italy and 1 in the United Kingdom (UK).

One enrolled subject was withdrawn prior to receiving treatment due to a non-fatal adverse event. This subject was not included in the analyses.

Participant milestones

Measure	IgPro10 All subjects treated with IgPro10
Overall Study STARTED	57
Overall Study COMPLETED	55
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	IgPro10 All subjects treated with IgPro10
Overall Study Adverse Event	1
Overall Study Therapy failure	1

Baseline Characteristics

Treatment of Chronic Immune Thrombocytopenic Purpura (ITP) With Intravenous Immunoglobulin IgPro10

Baseline characteristics by cohort

Measure	IgPro10 n=57 Participants All subjects treated with IgPro10
Age Continuous	38 years STANDARD_DEVIATION 15 • n=5 Participants
Sex: Female, Male Female	34 Participants n=5 Participants
Sex: Female, Male Male	23 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	57 participants n=5 Participants
Weight	74.0 kg STANDARD_DEVIATION 15.5 • n=5 Participants
Body Mass Index	25.69 kg/m^2 STANDARD_DEVIATION 4.56 • n=5 Participants

PRIMARY outcome

Timeframe: 7 days

Population: Intention to treat (ITT) analysis. The ITT population comprised all subjects who received at least once study medication.

The platelet response rate is defined as the percentage of subjects responding to treatment with an increase of platelet count from ≤ 20 x 10^9/L to ≥ 50 x 10^9/L within the specified time frame.

Outcome measures

Measure	IgPro10 n=57 Participants All subjects treated with IgPro10
Platelet Response	80.7 Percent of participants Interval 69.2 to 89.3

SECONDARY outcome

Timeframe: up to 29 days

Population: The number of participants analyzed represents the number of subjects in the ITT population with skin bleeding at baseline and respective post-baseline assessment.

Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.

Outcome measures

Measure	IgPro10 n=36 Participants All subjects treated with IgPro10
Regression of Hemorrhage (Skin)	31 participants

SECONDARY outcome

Timeframe: 29 days

Population: The number of participants analyzed represents the number of subjects in the ITT population with oral cavity bleeding at baseline and respective post-baseline assessment.

Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.

Outcome measures

Measure	IgPro10 n=11 Participants All subjects treated with IgPro10
Regression of Hemorrhage (Oral Cavity)	11 participants

SECONDARY outcome

Timeframe: 29 days

Population: The number of participants analyzed represents the number of subjects in the ITT population with genitourinary tract bleeding at baseline and respective post-baseline assessment.

Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.

Outcome measures

Measure	IgPro10 n=9 Participants All subjects treated with IgPro10
Regression of Hemorrhage (Genitourinary Tract)	7 participants

SECONDARY outcome

Timeframe: 29 days

Population: The number of participants analyzed represents the number of subjects in the ITT population with nose bleeding at baseline and respective post-baseline assessment.

Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 29 days

Population: The number of participants analyzed represents the number of subjects in the ITT population with internal bleeding at baseline and respective post-baseline assessment.

Number of subjects with a decrease in the severity of bleeding from baseline (prior to first infusion) on at least one post-infusion assessment during the study period (e.g., a change from moderate to mild or a change from mild to none). Regression of hemorrhages was tabulated separately for the organ systems skin, oral cavity, genitourinary tract, nose, and internal.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 29 days

Population: ITT analysis. The ITT population comprised all subjects who received at least once study medication.

Median time to reach a platelet count ≥ 50 x 10^9/L.

Outcome measures

Measure	IgPro10 n=57 Participants All subjects treated with IgPro10
Time to Platelet Response	2.5 days Interval 1.0 to 5.0

SECONDARY outcome

Timeframe: up to 29 days

Population: Analyzed for responders in the ITT population, i.e., only subjects with at least one platelet measurement ≥ 50 x 10\^9/L after start of treatment

The number of days the platelet count remained ≥ 50 x 10^9/L.

Outcome measures

Measure	IgPro10 n=48 Responders All subjects treated with IgPro10
Duration of Platelet Response	15.4 days Interval 8.8 to 21.9

SECONDARY outcome

Timeframe: 29 days

Population: ITT analysis. The ITT population comprised all subjects who received at least once study medication.

Maximum absolute platelet count achieved over the duration of the study.

Outcome measures

Measure	IgPro10 n=57 Participants All subjects treated with IgPro10
Maximum Platelet Level	154 10^9/L Interval 10.0 to 1049.0

Adverse Events

IgPro10

Serious events: 3 serious events

Other events: 52 other events

Deaths: 0 deaths

Serious adverse events

Measure	IgPro10 n=57 participants at risk All subjects treated with IgPro10
Nervous system disorders Dizziness	1.8% 1/57 • Number of events 1 • 28 Days
Infections and infestations Meningitis aseptic	1.8% 1/57 • Number of events 1 • 28 Days
Ear and labyrinth disorders Sudden hearing loss	1.8% 1/57 • Number of events 1 • 28 Days

Other adverse events

Measure	IgPro10 n=57 participants at risk All subjects treated with IgPro10
Nervous system disorders Headache	66.7% 38/57 • Number of events 53 • 28 Days
General disorders Pyrexia	24.6% 14/57 • Number of events 14 • 28 Days
Respiratory, thoracic and mediastinal disorders Epistaxis	19.3% 11/57 • Number of events 22 • 28 Days
Injury, poisoning and procedural complications Contusion	17.5% 10/57 • Number of events 11 • 28 Days
Skin and subcutaneous tissue disorders Petechiae	12.3% 7/57 • Number of events 9 • 28 Days
Gastrointestinal disorders Nausea	10.5% 6/57 • Number of events 8 • 28 Days
Skin and subcutaneous tissue disorders Skin haemorrhage	10.5% 6/57 • Number of events 8 • 28 Days
Gastrointestinal disorders Vomiting	10.5% 6/57 • Number of events 7 • 28 Days
Blood and lymphatic system disorders Anaemia	10.5% 6/57 • Number of events 6 • 28 Days
General disorders Hyperthermia	8.8% 5/57 • Number of events 7 • 28 Days
Investigations Bilirubin conjugated increased	8.8% 5/57 • Number of events 5 • 28 Days
Investigations Blood bilirubin unconjugated increased	8.8% 5/57 • Number of events 5 • 28 Days
Gastrointestinal disorders Mouth haemorrhage	7.0% 4/57 • Number of events 10 • 28 Days
Investigations Coombs direct test positive	7.0% 4/57 • Number of events 5 • 28 Days
Gastrointestinal disorders Gingival bleeding	7.0% 4/57 • Number of events 5 • 28 Days
Blood and lymphatic system disorders Anisocytosis	5.3% 3/57 • Number of events 5 • 28 Days
Investigations Blood lactate dehydrogenase increased	5.3% 3/57 • Number of events 3 • 28 Days
Investigations Coombs test positive	5.3% 3/57 • Number of events 3 • 28 Days
Investigations Haematocrit decreased	5.3% 3/57 • Number of events 3 • 28 Days
Hepatobiliary disorders Hyperbilirubinaemia	5.3% 3/57 • Number of events 3 • 28 Days

Additional Information

Clinical Trial Disclosure Manager

CSL Behring

Email: clinicaltrials@cslbehring.com

Results disclosure agreements

• Principal investigator is a sponsor employee The investigator must provide a copy of any results communication to the sponsor for review at least 30 days prior to public release. The sponsor may request any changes necessary to prevent forfeiture of patent rights to data not in the public domain. For a multi-center study, the investigator must wait (i) at least 1 year after the study is completed at all sites or (ii) until notified by the sponsor that no multi-center publication is planned before seeking publication review.
• Publication restrictions are in place

Restriction type: OTHER