Eltrombopag & Cyclosporine in Children With Sever Aplastic Anemia (NCT03243656) | Clinical Trial Compass
CompletedPhase 4
Eltrombopag & Cyclosporine in Children With Sever Aplastic Anemia
Egypt20 participantsStarted 2017-12-20
Plain-language summary
Aplastic anemia is a rare disorder characterized by pancytopenia and a hypo cellular bone marrow.but,It is very serious disease causing morbidity and mortality.
Aplastic anemia can be treated effectively with haematopoietic stem cell transplantation and immunosuppressive drug regimens but haematopoietic stem cell transplantation has limitations due to its cost and many patient are unsuitable. Immunosuppressive drug has a significant number of patients have persistent cytopenias. Currently, the treatment of these patients is regular transfusion, which are expensive, inconvenient, and associated with serious side effects related to iron overload and transfusion.
Eltrombopag is an oral thrombopoietin mimetic that selectively binds at the transmembrane and juxtamembrane domains of the thrombopoietin receptor, at sites distinct from the binding site of thrombopoietin therefore it does not compete for binding with the native molecule. It promoting thrombopoiesis and release of platelets from mature megakaryocytes. Also, promote other hematopoietic stem cell as well as in thrombopoiesis .
Who can participate
Age range
1 Year – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
Current diagnosis of sever Aplastic anemia
* Diagnosis of sever Aplastic anemia is established if Bone marrow cellularity \<25% or and at least two of the following criteria are met:- (i) absolute neutrophil count less than 0.5 × 109/L, (ii) platelet count less than 20 × 109/L, and (iii) reticulocyte count less than 20 × 109/L
* No, evidence of viral or drug suppression of the marrow, dysplasia, or underproduction anemias secondary to B12, folate, iron or other reversible causes.
* Age equal to 1 years old to 18 years old
* Written informed consent signed by a parent or legal guardian prior to initiation of any study specific procedure.
* Hematopoietic stem cell transplantation is not available or suitable as a treatment option or has been refused by the patient.
* Bone marrow aspirate and biopsy at any time during the 4 weeks prior to first dose of eltrombopag
Exclusion Criteria:
Prior and/or active medical history of:-
* Fanconi anemia (via chromosomal breakage test or growth arrest by flow cytometry). Other known underlying congenital/inherited marrow failure syndromes.
* Symptomatic Paroxysmal Nocturnal Hemoglobinuria
* Other known or suspected underlying primary immunodeficiency
* Any malignancy
* Active infection not responding to appropriate therapy
* Any out of range lab values Creatinine \>2.5 mg/dL× the upper limit of normal, Total bilirubin \>1.5 × the upper limit of normal mg/dL ,Aspartate aminotransferase (AST) or alanine aminotransferase…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
changes in Platelet count (at Baseline, 26 Weeks and up to 52 week)
Timeframe: 52 week
2
Changes in hemoglobin count (at Baseline, 26 Weeks and up to 52 week)
Timeframe: 52 week
3
changes in absolute Neutrophil count (at Baseline, 26 Weeks and up to 52 week)