Safety, Tolerability and Efficacy of Single Dose Low Cost Minipool Intravenous Immunoglobulins in Moderately Severe Newly Diagnosed ITP

Study Overview
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Brief Summary
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To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks. 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 ≥ 100 x 10^9/L and absence of bleeding (complete response) or platelet count ≥ 50 x 10^9/L and at least 2-fold increase the base line count with absence of bleeding (partial response), within the specified time frame.

Detailed Description
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To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks. 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 ≥ 100 x 10^9/L and absence of bleeding (complete response) or platelet count ≥ 50 x 10^9/L and at least 2-fold increase the base line count with absence of bleeding (partial response), within the specified time frame. Secondary: To assess safety of mini-pool IVIG in patients with newly diagnosed ITP by assessing frequency of adverse events related to mini-pool IgG administration To compare data on efficacy and safety of mini-pool IVIG with data obtained from literature on standard IVIG. Study population: Patients with newly diagnosed ITP attending Hematology clinic Ain Shams Children hospital Sample size: Seventy- two patients will be enrolled. They will be divided into 3 groups: Group A; platelet non enhancing group Group B; group receiving standard IVIG Group C; group receiving IVIG New product 24 patients in each group in a ratio 1:1:1.

Official Title
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Safety, Tolerability and Efficacy of Single Dose Low Cost Minipool Intravenous Immunoglobulins in Moderately Severe Newly Diagnosed ITP: A Prospective Randomized Study

Conditions
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Newly Diagnosed Pediatric ITP

Intervention / Treatment
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* Drug: IVIG prepared by minipool technology
* Drug: standard IVIG product


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: All newly diagnosed ITP patients (< 2 weeks from onset of the disease), with no platelet enhancing therapy. Age eligible for study: 1-16 years old Gender eligible for study: both sexes Informed consent signed by patient or his legal guardian. Exclusion Criteria: Patients started on steroid therapy. Platelet count<10,000/mm3. Having a life threatening bleeding. Patients with known or suspected hypersensitivity to immunoglobulins or previous severe side effects to immunoglobulin therapy. Treatment with immunosuppressive or other immunomodulatory drugs within 3 weeks prior to screening. Treatment with any other investigational drug within 7 days before study entry.

Ages Eligible for Study
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Minimum Age: 12 Months
Maximum Age: 16 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: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| No Intervention: group A<br>no platelet enhancing therapy | |
| Active Comparator: group B<br>Standard IVIG single dose 1.0 gm /kg/dose | Drug: standard IVIG product<br>* to compare efficacy and safety with the minipool IVIG<br>|
| Experimental: group C<br>minipool IVIG product single dose 1.0 gm /kg/dose | Drug: IVIG prepared by minipool technology<br>* Drug: To demonstrate the efficacy of new product IVIG prepared by minipool technology as assessed by Bleeding Score and platelet count at 3 days, 1 week, 2 weeks and 4 weeks.<br>* Other names: IG plasma;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| bleeding score(SMOG) | change of bleeding score(SMOG) at day 3,week1,week 2 and week 4 after presentation | 4 weeks | 
| complete blood count | change of complete blood count for number of platelets,absolute neutrophil count,absolute lymphocyte count and hemoglobin at day 3,week 1,week 2 and week 4 after presentation | 4 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with treatment-related adverse events | presence of side effects: anaphylaxis,fever,rash and any others | 4 weeks |