Clinical trial NCT02669277: Safety, Tolerability and Efficacy of Single Dose Low Cost Minipool Intravenous Immunoglobulins in Moderately Severe Newly Diagnosed ITP
Official title: Safety, Tolerability and Efficacy of Single Dose Low Cost Minipool Intravenous Immunoglobulins in Moderately Severe Newly Diagnosed ITP: A Prospective Randomized Study
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Newly Diagnosed Pediatric ITP
Lead sponsor: Ain Shams University
Sponsor class: OTHER
First posted: 2016-02-01
Enrollment: 72
Sex: ALL
Minimum age (years): 12
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: bleeding score(SMOG)
timeFrame: 4 weeks
measure: complete blood count
timeFrame: 4 weeks
Secondary outcomes:
measure: Number of participants with treatment-related adverse events
timeFrame: 4 weeks
Other outcomes:
Locations:
city: Cairo
country: Egypt
facility: hematology clinic ,pediatrics hospital, Ain Shams University hospital Cairo, Egypt
Study officials:
References: