Management of Children With Persistent ITP, A Novel Approach

NCT ID: NCT07068126

Last Updated: 2025-07-16

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-01-01
• Study Completion Date: 2026-03-01

Brief Summary

The goal of this clinical trial is to learn if mini-pool intravenous immunoglobulin (IVIG) is a safe and effective treatment for children with persistent immune thrombocytopenia (ITP). ITP is a condition that causes low platelet levels and increases the risk of bleeding. The main questions this study aims to answer are:

Can mini-pool IVIG raise platelet levels in children with persistent ITP?

Can it reduce bleeding episodes and hospital visits?

What side effects, if any, are seen with this treatment?

There is no comparison group in this study. All participants will receive mini-pool IVIG, which is made from small pools of donated plasma using a cost-effective process.

Participants will:

Receive one dose of mini-pool IVIG through a vein over 6 to 8 hours

Receive follow-up doses every 2 to 4 weeks for up to 5 doses, based on their platelet count

Have regular blood tests and checkups during the study and for 6 months after treatment

Report on bleeding episodes, physical activity, school attendance, and side effects

Detailed Description

Immune thrombocytopenia (ITP) is an autoimmune condition where the immune system destroys platelets, leading to low platelet counts and increased risk of bleeding. Persistent ITP is defined as ongoing thrombocytopenia lasting 3 to 12 months after initial diagnosis. Children with persistent ITP who lose their response to first-line treatments, such as steroids or standard intravenous immunoglobulin (IVIG), have limited therapeutic options, especially in low- and middle-income countries, due to the high cost of commercial IVIG preparations.

Mini-pool IVIG is produced from small pools of plasma collected locally, using a validated process with virus inactivation and IgG purification steps. This method enables safe, cost-effective preparation of IVIG in resource-limited settings. Prior research has shown that mini-pool IVIG is effective and well-tolerated in acute pediatric ITP, but its role as a second-line therapy for persistent ITP has not been evaluated.

This multicenter, prospective clinical trial will enroll 20 children aged 1 to 10 years with persistent ITP at three tertiary care pediatric hematology centers in Egypt. Participants will receive a loading dose of mini-pool IVIG at 1 g/kg, followed by maintenance doses of 0.5 g/kg every 2 to 4 weeks for up to five additional doses, with dose intervals adjusted based on platelet counts.

Throughout the study, participants will undergo regular blood counts, bleeding assessments using the Bleeding Assessment Tool (BAT), and monitoring for infusion-related or delayed adverse events. Data on school attendance, physical activity, and patient or family satisfaction will also be collected.

Responses to therapy will be classified as complete response (CR), response (R), or no response (NR) based on platelet count thresholds and bleeding status, with response duration measured from achievement of CR or R to loss of response. Participants achieving sustained response off therapy (SRoT) or response off therapy (RoT) during the 6-month post-treatment follow-up will be identified to evaluate durability of treatment effects.

This study aims to provide evidence on the safety and efficacy of mini-pool IVIG as a second-line therapy for persistent pediatric ITP, potentially offering an affordable and effective treatment alternative in settings where standard IVIG is inaccessible due to cost.

Conditions

Persistent Immune Thrombocytopenia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Mini-Pool IVIG Treatment Arm

Participants in this arm will receive mini-pool intravenous immunoglobulin (IVIG) as second-line therapy for persistent immune thrombocytopenia (ITP). Treatment includes a loading dose of 1 g/kg infused over 6-8 hours, followed by maintenance doses of 0.5 g/kg given every 2 to 4 weeks for up to five additional doses. The dosing interval is adjusted based on each participant's platelet count. Participants will have regular blood tests and clinical assessments to monitor platelet levels, bleeding episodes, and treatment safety.

Group Type: EXPERIMENTAL

Mini-Pool IVIG

Intervention Type: BIOLOGICAL

Mini-pool intravenous immunoglobulin (IVIG) is a plasma-derived biologic prepared from small pools of locally donated human plasma using a validated, virus-inactivated, closed-system process. Each participant will receive a loading dose of 1 g/kg infused intravenously over 6-8 hours. Maintenance doses of 0.5 g/kg will be given every 2 to 4 weeks for up to five additional doses, with the dosing schedule adjusted based on platelet count. The preparation contains purified IgG and meets safety standards for sterility and viral inactivation.

Interventions

Mini-Pool IVIG

Mini-pool intravenous immunoglobulin (IVIG) is a plasma-derived biologic prepared from small pools of locally donated human plasma using a validated, virus-inactivated, closed-system process. Each participant will receive a loading dose of 1 g/kg infused intravenously over 6-8 hours. Maintenance doses of 0.5 g/kg will be given every 2 to 4 weeks for up to five additional doses, with the dosing schedule adjusted based on platelet count. The preparation contains purified IgG and meets safety standards for sterility and viral inactivation.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Gender: Males and Females
• Persistent ITP according to ASH definition
• No history of treatment with thrombopoietin agonists

Exclusion Criteria

• Previous history of ICH
• Difficult venous access
• Congenital thrombocytopenia, secondary ITP and non-immune thrombocytopenia

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Zagazig University

OTHER_GOV

Sponsor Role: collaborator

Ain Shams University

OTHER

Sponsor Role: collaborator

Assiut University

OTHER

Sponsor Role: lead

Responsible Party

Mervat A. M. Youssef

Principal investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Mervat M A Youssef, Assistant Professor

Role: PRINCIPAL_INVESTIGATOR

Assiut University

Locations

Children's hospital - Assiut University

Asyut, , Egypt

Site Status: RECRUITING

Ain Shams University

Cairo, , Egypt

Site Status: RECRUITING

Zagazig University, Pediatric departement

Zagazig, , Egypt

Site Status: RECRUITING

Countries

Egypt

Central Contacts

Mohsen El Alfy, Professor

Role: CONTACT

Elalfym@hotmail.com

+201000864343

Laila M Sherief, Professor

Role: CONTACT

lamesh25@yahoo.com

+201001891964

Facility Contacts

Dr. Mervat A M Youssef, Assistant Professor

Role: primary

mamuosif2000@aun.edu.eg

+201142606221

Mohsin Elalfy, Professor

Role: primary

Elalfym@hotmail.com

+201000864343

Laila M Sherief, Professor

Role: primary

lamesh25@yahoo.com

+201001891964

References

1. Kohli, R. and S. Chaturvedi, Epidemiology and Clinical Manifestations of Immune Thrombocytopenia. Hamostaseologie, 2019. 39(3): p. 238-249. 2. Pietras, N.M., et al., Immune Thrombocytopenia, in StatPearls. 2024, StatPearls Publishing Copyright © 2024, StatPearls Publishing LLC.: Treasure Island (FL). 3. Thakur, Y., R.J. Meshram, and A. Taksande, Diagnosis and Management of Immune Thrombocytopenia in Paediatrics: A Comprehensive Review. Cureus, 2024. 16(9): p. e69635. 4. Wang, Z., et al., Sustained response off treatment in eltrombopag for children with persistent/chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China. Br J Haematol, 2023. 202(2): p. 422-428. 5. Gotesman, M., et al., Pediatric Immune Thrombocytopenia. Adv Pediatr, 2024. 71(1): p. 229-240. 6. El-Ekiaby, M., et al., Minipool caprylic acid fractionation of plasma using disposable equipment: a practical method to enhance immunoglobulin supply in developing countries. PLoS Negl Trop Dis, 2015. 9(2): p. e0003501. 7. Elalfy, M., et al., A randomized multicenter study: safety and efficacy of mini-pool intravenous immunoglobulin versus standard immunoglobulin in children aged 1-18 years with immune thrombocytopenia. Transfusion, 2017. 57(12): p. 3019-3025. 8. Rodeghiero, F., et al., Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood, 2009. 113(11): p. 2386-93.

Reference Type: BACKGROUND

Provided Documents

Document Type: Study Protocol

View Document

Other Identifiers

IRB:04-2025-300549

Identifier Type: OTHER

Identifier Source: secondary_id

IRB:04-2025-300549

Identifier Type: -

Identifier Source: org_study_id