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

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the efficacy, tolerability and safety of IgPro10 in the treatment of patients with chronic immune thrombocytopenic purpura (ITP). The main efficacy parameter is the proportion of patients responding to treatment by an increase of platelet count to ≥ 50 x 10^9/L.

Official Title
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An Open-label, Multicenter Study on the Efficacy and Safety of IgPro10 in Patients With Chronic Immune Thrombocytopenic Purpura (ITP)

Conditions
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Immune Thrombocytopenic Purpura

Intervention / Treatment
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* Biological: Immunoglobulin Intravenous (Human)


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Diagnosis of chronic ITP defined by: Failure to find other causes of thrombocytopenia; Platelet count ≤ 150 x 10^9/L over 6 months or response to a previous treatment with subsequent decrease in platelet count even if duration of chronic ITP is less than 6 months Platelet counts ≤ 20 x 10^9/L Key Exclusion Criteria: Planned splenectomy throughout the study period Treatment with IVIG or anti-D immunoglobulin within 3 weeks prior to screening Treatment with immunosuppressive or other immunomodulatory drugs within 3 weeks prior to screening Treatment with intravenous steroids within 10 days prior to screening Change of oral steroid treatment within 15 days prior to screening Patients with known or suspected hypersensitivity to immunoglobulins or previous severe side effects to immunoglobulin therapy Abnormal results in the following laboratory parameters: Hemoglobin < 10 g/dL; Total bilirubin > 1.5 x upper normal limit; ALAT > 2.5 x upper normal limit; ASAT > 2.5 x upper normal limit; Creatinine > 1.5 x upper normal limit; Urea > 1.5 x upper normal limit Positive direct Coombs test Patients with one of the following concomitant diseases Clinical active SLE Known or suspected HIV infection Acute hepatitis Clinically active chronic hepatitis Lymphoproliferative disease Heart failure Grade III or IV according to the New York Heart Association classification Any other concomitant disease that has influence on the clotting system (i.e. hemophilia)

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: IgPro10<br> | Biological: Immunoglobulin Intravenous (Human)<br>* A dose of 1 g IgG per kg body weight (bw) administered on two consecutive days resulting in the total treatment dosage of 2 g IgG per kg bw.<br>* Other names: IgPro10;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Platelet Response | 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. | 7 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Regression of Hemorrhage (Skin) | 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. | up to 29 days | 
| Regression of Hemorrhage (Oral Cavity) | 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. | 29 days | 
| Regression of Hemorrhage (Genitourinary Tract) | 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. | 29 days | 
| Regression of Hemorrhage (Nose) | 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. | 29 days | 
| Regression of Hemorrhage (Internal) | 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. | 29 days | 
| Time to Platelet Response | Median time to reach a platelet count ≥ 50 x 10^9/L. | 29 days | 
| Duration of Platelet Response | The number of days the platelet count remained ≥ 50 x 10^9/L. | up to 29 days | 
| Maximum Platelet Level | Maximum absolute platelet count achieved over the duration of the study. | 29 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Chronic Immune Thrombocytopenic Purpura, Chronic Idiopathic Thrombocytopenic Purpura, Werlhofs Disease, Autoimmune Thrombocytopenia, Immunglobulin Intravenous, Chronic ITP, Platelet count, Thrombocytopenia