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

Ain Shams University

Status and phase: Unknown; Phase 2

Conditions: Newly Diagnosed Pediatric ITP

Treatments: Drug: IVIG prepared by minipool technology; Drug: standard IVIG product

Study type: Interventional

Funder types: Other

Identifiers

NCT02669277

FMASU R30/2015

Details and patient eligibility

About

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.

Full 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.

Enrollment: 72 estimated patients

Sex: All

Ages: 12 months to 16 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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72 participants in 3 patient groups

group A

No Intervention group

Description:

no platelet enhancing therapy

group B

Active Comparator group

Description:

Standard IVIG single dose 1.0 gm /kg/dose

Treatment:

Drug: standard IVIG product

group C

Experimental group

Description:

minipool IVIG product single dose 1.0 gm /kg/dose

Treatment:

Drug: IVIG prepared by minipool technology