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

Phase 2
Conditions
Newly Diagnosed Pediatric ITP
Interventions
Drug: standard IVIG product
Drug: IVIG prepared by minipool technology
Registration Number
NCT02669277
Lead Sponsor
Ain Shams University
Brief Summary

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

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:

1. 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

2. 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.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
72
Inclusion Criteria
  1. All newly diagnosed ITP patients (< 2 weeks from onset of the disease), with no platelet enhancing therapy.
  2. Age eligible for study: 1-16 years old
  3. Gender eligible for study: both sexes
  4. Informed consent signed by patient or his legal guardian.
Exclusion Criteria
  1. Patients started on steroid therapy.
  2. Platelet count<10,000/mm3.
  3. Having a life threatening bleeding.
  4. Patients with known or suspected hypersensitivity to immunoglobulins or previous severe side effects to immunoglobulin therapy.
  5. Treatment with immunosuppressive or other immunomodulatory drugs within 3 weeks prior to screening.
  6. Treatment with any other investigational drug within 7 days before study entry.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
group Bstandard IVIG productStandard IVIG single dose 1.0 gm /kg/dose
group CIVIG prepared by minipool technologyminipool IVIG product single dose 1.0 gm /kg/dose
Primary Outcome Measures
NameTimeMethod
bleeding score(SMOG)4 weeks

change of bleeding score(SMOG) at day 3,week1,week 2 and week 4 after presentation

complete blood count4 weeks

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

Secondary Outcome Measures
NameTimeMethod
Number of participants with treatment-related adverse events4 weeks

presence of side effects: anaphylaxis,fever,rash and any others

Trial Locations

Locations (1)

hematology clinic ,pediatrics hospital, Ain Shams University hospital Cairo, Egypt

🇪🇬

Cairo, Egypt

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