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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
Sponsor:
Information provided by (Responsible Party):
MaiAli, Assiut University

Brief 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 .


Condition or disease Intervention/treatment Phase
Eltrombopag Drug: Eltrombopag Phase 4

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

Drug Information available for: Eltrombopag

Arm Intervention/treatment
No Intervention: control arm
standard immunosuppressive therapy (antilymphocyte globulin and cyclosporine),
Active Comparator: case arm
standard immunosuppressive therapy plus an oral dose of Eltrombopag
Drug: Eltrombopag
An oral thrombopoietin receptor agonist
Other Name: Revolade




Primary Outcome Measures :
  1. :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.

  2. 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.

  3. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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

  3. 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



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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

Current diagnosis of sever and moderate 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
  • Moderate aplastic anemia is defined as bone marrow cellularity <50 percent and depression of at least two out of three blood counts below the normal values: criteria are met:- (i) absolute neutrophil count less than 1200/mm3, (ii) platelet count less than 70,000/mm3, and (iii) anemia with hemoglobin less than or equal to 8.5 g/dL and absolute reticulocyte count less than or equal to 60,000/mm3 in transfusion-dependent patients but not fulfilling the criteria of sever aplastic anemia
  • 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 (ALT) >2.5 × the upper limit of normal
  • Hypersensitivity to eltrombopag or its components
  • Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to tolerate protocol therapy, or that death within 7-10 days is likely.

Information from the National Library of Medicine

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


Contacts
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Contact: Mohammed M El Gazally, prof. 00201001296603 hamdyghazally@yahoo.com
Contact: khaled A El sonosy, ass prof.

Locations
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Egypt
Facility of medicine Recruiting
Assiut, Egypt, 01515
Contact: Mohammed Mahmoud El Gazaly, MD    ‭0100 1296603‬ ext 002    hamdyghazally@yahoo.com   
Sponsors and Collaborators
Assiut University

Publications:

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Responsible Party: MaiAli, assistant lecturer, Assiut University
ClinicalTrials.gov Identifier: NCT03243656     History of Changes
Other Study ID Numbers: MAAhmed
First Posted: August 9, 2017    Key Record Dates
Last Update Posted: December 31, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Additional relevant MeSH terms:
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Anemia, Aplastic
Anemia
Hematologic Diseases
Bone Marrow Diseases