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

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

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.

Study Overview

• Status: Unknown
• Conditions: Newly Diagnosed Pediatric ITP
• Intervention / Treatment: Drug: standard IVIG product; Drug: IVIG prepared by minipool technology

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.

• Study Type: Interventional
• Enrollment (Anticipated): 72
• Phase: Phase 2

Contacts and Locations

Study Locations

• Egypt
  • Cairo, Egypt
    • Recruiting
    • hematology clinic ,pediatrics hospital, Ain Shams University hospital Cairo, Egypt
    • Contact: Mohsen Saleh Elalfy, professor
    • Principal Investigator: Mohsen Saleh Elalfy, professor

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 16 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
No Intervention: group A; no platelet enhancing therapy
Active Comparator: group B; Standard IVIG single dose 1.0 gm /kg/dose	Drug: standard IVIG product; to compare efficacy and safety with the minipool IVIG
Experimental: group C; minipool IVIG product single dose 1.0 gm /kg/dose	Drug: IVIG prepared by minipool technology; 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.; Other Names: IG plasma

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Ain Shams University

Study record dates

Study Major Dates

• Study Start: August 1, 2015
• Primary Completion (Anticipated): February 1, 2016
• Study Completion (Anticipated): February 1, 2016

Study Registration Dates

• First Submitted: January 20, 2016
• First Submitted That Met QC Criteria: January 27, 2016
• First Posted (Estimate): February 1, 2016

Study Record Updates

• Last Update Posted (Estimate): February 1, 2016
• Last Update Submitted That Met QC Criteria: January 27, 2016
• Last Verified: January 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: FMASU R30/2015