Eltrombopag in Children With Idiopathic Aplastic Anemia (Eltroplastic)
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|ClinicalTrials.gov Identifier: NCT03243656|
Recruitment Status : Recruiting
First Posted : August 9, 2017
Last Update Posted : December 31, 2018
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 .
|Condition or disease||Intervention/treatment||Phase|
|Eltrombopag||Drug: Eltrombopag||Phase 4|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Randomized Clinical Trial of the Use of Eltrombopag in Children With Idiopathic Aplastic Anemia|
|Actual Study Start Date :||December 20, 2017|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2020|
No Intervention: control arm
standard immunosuppressive therapy (antilymphocyte globulin and cyclosporine),
Active Comparator: case arm
standard immunosuppressive therapy plus an oral dose of Eltrombopag
An oral thrombopoietin receptor agonist
Other Name: Revolade
- :Platelet count [ Time Frame: from 12 to 16 week ]The hematologic response to eltrombopag in subjects with moderate or severe Aplastic Anemia will be assessed by measuring increase of platelet count from baseline by 20,000/microliter or more (in the absence of platelet transfusion), or no platelet transfusion requirements for 8 weeks.
- Hemoglobin [ Time Frame: from 12 to 16 week ]The hematologic response to eltrombopag in subjects with moderate or severe Aplastic Anemia will be assessed by measuring the following. Increase from baseline by 1.5 gram (g)/ decilitre (dL) or more: When the baseline hemoglobin level is <8 g/dL and no red blood cell (RBC) transfusion at baseline. A decrease of at least 4 units in red blood cell transfusions in the post-treatment 8-week period compared to the pre-treatment 8-week period.
- Neutrophil count [ Time Frame: from 12 to 16 week ]The hematologic response to eltrombopag in subjects with moderate or severe Aplastic Anemia will be assessed by measuring (in the absence of granulocyte colony-stimulating factor (G-CSF) the increase in neutrophil count from baseline by 500/microliter or more.
- The hematological responses [ Time Frame: from 12 to 16 week ]
- Duration of hematologic response Time from the date of the start of response to the date of relapse defined as again meeting criteria for severe or moderate aplastic anemia
- Duration of platelet and blood transfusion independence Transfusion independency is defined when blood and platelet transfusions are not required in a consecutive 8-week period. Transfusion dependency will be defined as at least 2 units of red blood cells or five units of random platelets or equivalent apheresis per month for a period of 8 weeks. The duration of platelet and blood transfusion independence will be evaluated separately.
- The blood & platelet transfusion [ Time Frame: from 12 to 16 week ]
- Percentage of patients who received a blood transfusion Transfusions of red blood cells may be given during study participation as medically necessary. Packed RBCs should be administered for hemoglobin <8 g/dL, or when patients are symptomatic
- Percentage of patients who received platelet transfusion Transfusions of platelets may be given during study participation as medically necessary. In general, platelet transfusions should be administered when the platelet count <10 x 109/L or when significant bleeding occurs
- The toxicity [ Time Frame: from 12 to 16 week ]• Number of participants with adverse events Measure toxicity, using CTCAE associated with assessment of eltrombopag use, dose, and tolerability in patients with moderate to very severe aplastic anemia
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03243656
|Contact: Mohammed M El Gazally, email@example.com|
|Contact: khaled A El sonosy, ass prof.|
|Facility of medicine||Recruiting|
|Assiut, Egypt, 01515|
|Contact: Mohammed Mahmoud El Gazaly, MD 0100 1296603 ext 002 firstname.lastname@example.org|