Persistent Immune ThrombocytopeniaLMICsNCT07068126
Management of Children With Persistent ITP, A Novel Approach
A study testing Mini-Pool IVIG in people with Persistent Immune Thrombocytopenia, run by Assiut University at 3 sites in Egypt.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 10 years
- Conditions the study is for
- Persistent Immune Thrombocytopenia
- What is being tested?
- Mini-Pool IVIG
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesEgypt
- Study dates
- 1 Jan 2025to 1 Mar 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Gender: Males and Females
- Persistent ITP according to ASH definition
- No history of treatment with thrombopoietin agonists
- Exclusion Criteria:-History of severe drug adverse events to IVIG
- Previous history of ICH
- Difficult venous access
- Congenital thrombocytopenia, secondary ITP and non-immune thrombocytopenia
Who cannot join
None listed.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Egypt3 sites, 3 recruiting
- Recruiting
Children's hospital - Assiut University
Asyut, 71515
Dr. Mervat A M Youssef, Assistant Professor, +201142606221, mamuosif2000@aun.edu.eg
- Recruiting
Ain Shams University
Cairo, 11566
Mohsin Elalfy, Professor, +201000864343, Elalfym@hotmail.com
- Recruiting
Zagazig University, Pediatric departement
Zagazig, 44519
Laila M Sherief, Professor, +201001891964, lamesh25@yahoo.com
About this study
Official title: "The Safety and Efficacy of Mini-Pool IVIG Initiation and Maintenance Therapy for Management of Children With Persistent ITP, A Novel Approach for LMICs"
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?
Read more
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
Read the 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
- Being tested
- Mini-Pool IVIG
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mini-Pool IVIGBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| Mini-Pool IVIG Treatment Arm | Experimental | 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. Receives: Biological: Mini-Pool IVIG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bleeding Frequency and Severity
Number, location, and severity of bleeding episodes assessed using the Bleeding assessed using the ISTH Bleeding Assessment Tool (BAT) score, which evaluates bleeding symptoms across multiple anatomical sites. Each site is scored from 0 to 4, with higher scores indicating more severe bleeding. The total score varies depending on the number and severity of bleeding events.Assessment Tool (BAT) score.
Measured: From enrollment through 6 months post-treatment.
Platelet Count Response
Number of participants achieving complete response (platelet count ≥100×10⁹/L) or response (platelet count ≥30×10⁹/L and at least 2-fold increase from baseline) without bleeding.
Measured: Assessed monthly during treatment and for 6 months after last dose.
Frequency of Hospital Admissions Due to Critical Bleeding
Number of hospital admissions for life-threatening bleeding episodes, excluding admissions solely for IVIG infusion.
Measured: From enrollment through 6 months post-treatment.
Secondary outcomes (4)
Adverse Events
Number and type of infusion-related or delayed adverse events experienced during or after mini-pool IVIG treatment.
Measured: From first infusion through 6 months after the last dose.
School Attendance
Change in days missed from school compared to baseline, as a measure of quality of life.
Measured: From enrollment through 6 months post-treatment.
Patient and Family Satisfaction
Level of satisfaction with mini-pool IVIG treatment as reported by participants and caregivers using a structured satisfaction questionnaire.
Measured: Assessed at the end of treatment and at 6-month follow-up.
Sustained Response Off-Therapy (SRoT/RoT)
number of participants maintaining platelet counts ≥100×10⁹/L (SRoT) or ≥30×10⁹/L without bleeding (RoT) for at least 6 months after completing mini-pool IVIG therapy.
Measured: 6 months after last mini-pool IVIG dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2025
- Study start1 Jan 2025
- Primary completion30 Dec 2025 (estimated)
- Study completion1 Mar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Persistent Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- All enrolled participants will receive mini-pool intravenous immunoglobulin (IVIG) as a second-line treatment for persistent immune thrombocytopenia (ITP). The study follows a single-group assignment model with no control or comparison group. Dosing intervals are adjusted based on each participant's platelet count response.
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Collaborators
- Zagazig University, Ain Shams University
- Responsible party
- Principal investigator, Mervat A. M. Youssef, Assiut University
- Investigators
- Mervat M A Youssef, Assistant Professor, Principal investigator, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07068126
- Sponsor's protocol ID
- IRB:04-2025-300549
- Other IDs
- IRB:04-2025-300549
- Acronym
- LMICs
- Status last verified
- Jul 2025
- First submitted
- 2 Jul 2025
- Last updated
- 16 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- "The Safety and Efficacy of Mini-Pool IVIG Initiation and Maintenance Therapy for Management of Children With Persistent ITP, A Novel Approach for LMICs"
- ClinicalTrials.gov ID
- NCT07068126
- Acronym / sponsor ID
- LMICs / IRB:04-2025-300549
- Conditions
- Persistent Immune Thrombocytopenia
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Collaborators
- Zagazig University, Ain Shams University
- Study start
- 1 Jan 2025 (actual)
- Primary completion
- 30 Dec 2025 (estimated)
- Study completion
- 1 Mar 2026 (estimated)
- First posted
- 16 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- All enrolled participants will receive mini-pool intravenous immunoglobulin (IVIG) as a second-line treatment for persistent immune thrombocytopenia (ITP). The study follows a single-group assignment model with no control or comparison group. Dosing intervals are adjusted based on each participant's platelet count response.
| Arm | Type | Receives |
|---|---|---|
| Mini-Pool IVIG Treatment Arm | Experimental | Biological: Mini-Pool IVIGParticipants 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. |
Who may qualify
- Ages
- 1 year to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Gender: Males and Females
- Persistent ITP according to ASH definition
- No history of treatment with thrombopoietin agonists
- Exclusion Criteria:-History of severe drug adverse events to IVIG
- Previous history of ICH
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mini-Pool IVIGMini-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. | Biological | Mini-Pool IVIG Treatment Arm |
Primary outcome
Bleeding Frequency and Severity
Time frame: From enrollment through 6 months post-treatment.
Number, location, and severity of bleeding episodes assessed using the Bleeding assessed using the ISTH Bleeding Assessment Tool (BAT) score, which evaluates bleeding symptoms across multiple anatomical sites. Each site is scored from 0 to 4, with higher scores indicating more severe bleeding. The total score varies depending on the number and severity of bleeding events.Assessment Tool (BAT) score.
Platelet Count Response
Time frame: Assessed monthly during treatment and for 6 months after last dose.
Number of participants achieving complete response (platelet count ≥100×10⁹/L) or response (platelet count ≥30×10⁹/L and at least 2-fold increase from baseline) without bleeding.
Frequency of Hospital Admissions Due to Critical Bleeding
Time frame: From enrollment through 6 months post-treatment.
Number of hospital admissions for life-threatening bleeding episodes, excluding admissions solely for IVIG infusion.
4 secondary outcomes
Adverse Events
Time frame: From first infusion through 6 months after the last dose.
School Attendance
Time frame: From enrollment through 6 months post-treatment.
Patient and Family Satisfaction
Time frame: Assessed at the end of treatment and at 6-month follow-up.
Sustained Response Off-Therapy (SRoT/RoT)
Time frame: 6 months after last mini-pool IVIG dose.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2025.
- Background1. 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Mervat M A Youssef, Assistant Professor principal investigator
Assiut University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Mini-Pool IVIG in people with Persistent Immune Thrombocytopenia, run by Assiut University at 3 sites in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mohsen El Alfy, Professor (+201000864343, Elalfym@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bleeding Frequency and Severity (time frame: From enrollment through 6 months post-treatment.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07068126, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2025.