We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Eltrombopag & Cyclosporine in Children With Sever Aplastic Anemia (Eltroplastic)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03243656
Recruitment Status : Completed
First Posted : August 9, 2017
Last Update Posted : January 18, 2022
Sponsor:
Information provided by (Responsible Party):
Mai Ali Abdelfatah Ahmed, 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 Show detailed description

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual 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 & Cyclosporine in Children With Sever Idiopathic Aplastic Anemia
Actual Study Start Date : December 20, 2017
Actual Primary Completion Date : December 29, 2019
Actual Study Completion Date : January 5, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
No Intervention: historical group
Immunosuppressive therapy (cyclosporine alone ),
Active Comparator: case arm
cyclosporine plus an oral dose of Eltrombopag
Drug: Eltrombopag
An oral thrombopoietin receptor agonist
Other Name: Revolade




Primary Outcome Measures :
  1. changes in Platelet count (at Baseline, 26 Weeks and up to 52 week) [ Time Frame: 52 week ]
    increase of platelet count from baseline by 20,000/microliter or more (in the absence of platelet transfusion), or independence from platelet transfusions for a minimum of 8 weeks in patients who were previously transfusion-dependent.

  2. Changes in hemoglobin count (at Baseline, 26 Weeks and up to 52 week) [ Time Frame: 52 week ]
    measuring the following: Increase from baseline by 1.5 gram g/dL or more when the baseline hemoglobin level is <8.5 g/dL and no red blood cell (RBC) transfusion at baseline. A decrease of at least four units in RBC transfusions in the post-treatment 8-week period compared to the pre-treatment 8-week period..

  3. changes in absolute Neutrophil count (at Baseline, 26 Weeks and up to 52 week) [ Time Frame: 52 week ]
    measuring the increase in the absolute neutrophil count of more than 500 per cubic millimeter

  4. complete response (CR) - 12 month [ Time Frame: 52 weeks ]
    CR defined as all three parameters meet the following criteria : Hb ≥100 g/l, platelet count ≥100 × 109/L, and ANC ≥1 × 109/L. Additionally, patients had to be transfusion and growth factor independent

  5. Partial response (PR) - 12 month [ Time Frame: 52 weeks ]
    PR was defined as blood count no longer meeting Camitta criteria for severs aplastic anemia in case of sever aplastic anemia (SAA): Absolute neutrophil count (ANC) >500/μL Platelet count >20 000/μL Reticulocyte count >20 000/μL


Secondary Outcome Measures :
  1. The hematological responses [ Time Frame: up to to 52 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 toxicity [ Time Frame: Up to 30 days after last dose of study treatment ]
    Number of participants with adverse events Measure toxicity, using CTCAE associated with assessment of eltrombopag use, dose, and tolerability in patients with sever to very severe aplastic anemia

  3. complete response (CR)- 6 month [ Time Frame: 26 week ]
    CR defined as all three parameters meet the following criteria : Hb ≥100 g/l, platelet count ≥100 × 109/L, and ANC ≥1 × 109/L. Additionally, patients had to be transfusion and growth factor independent

  4. Partial response (PR) - 6 month [ Time Frame: 26 week ]

    PR was defined as blood count no longer meeting Camitta criteria for severs aplastic anemia in case of SAA.

    Absolute neutrophil count (ANC) >500/μL Platelet count >20 000/μL Reticulocyte count >20 000/μL


  5. Overall hematologic response (CR + PR) rate - 6 month [ Time Frame: 26 week ]

    Overall hematologic response = patients with complete response + patients with partial response .

    Partial response is defined as blood count no longer meeting Camitta criteria for severs aplastic anemia in case of SAA

    ANC >500/μL Platelet count >20 000/μL Reticulocyte count >20 000/μL

    Complete response is defined as all three parameters meet the following criteria:

    ANC > 1 000/μL Platelet count >100 000/μL Hemoglobin >10 g/L


  6. Overall hematologic response (CR + PR) rate - 12 month [ Time Frame: 52 week ]

    Overall hematologic response = patients with complete response + patients with partial response.

    Partial response is defined as blood count no longer meeting Camitta criteria for severs aplastic anemia in case of SAA

    ANC >500/μL Platelet count >20 000/μL Reticulocyte count >20 000/μL

    Complete response is defined as all three parameters meet the following criteria:

    ANC > 1 000/μL Platelet count >100 000/μL Hemoglobin >10 g/L




Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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 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 (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


Locations
Layout table for location information
Egypt
Facility of medicine
Assiut, Egypt, 71511
Sponsors and Collaborators
Assiut University
Publications:

Layout table for additonal information
Responsible Party: Mai Ali Abdelfatah Ahmed, assistant lecturer, Assiut University
ClinicalTrials.gov Identifier: NCT03243656    
Other Study ID Numbers: MAAhmed
First Posted: August 9, 2017    Key Record Dates
Last Update Posted: January 18, 2022
Last Verified: January 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
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:
Layout table for MeSH terms
Anemia, Aplastic
Anemia
Hematologic Diseases
Bone Marrow Failure Disorders
Bone Marrow Diseases