Status:
COMPLETED
Extended Dosing With Eltrombopag for Severe Aplastic Anemia
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Severe Aplastic Anemia (SAA)
Eligibility:
All Genders
2-100 years
Phase:
PHASE2
Brief Summary
Background: \- Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients. However, researchers do not know how long the drug can and s...
Detailed Description
Severe aplastic anemia (SAA) is a life-threatening blood disease that can be successfully treated with immunosuppressive drug regimens or allogeneic stem cell transplantation. However, 20-40% of patie...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Previous diagnosis of refractory severe aplastic anemia and following at least one treatment course of immunosuppression with a regimen containing antithymocyte globulin, alemtuzumab or cyclophosphamide.
- One or more of the following three clinically-significant cytopenias: platelet count less than or equal to 30,000/micro L or platelet-transfusion-dependence (requiring at least 4 platelet transfusions in the 8 weeks prior to study entry); neutrophil count less than 500/micro L; hemoglobin less than 9.0 g/dL or red cell transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior to study entry)
- Age greater than or equal to 2 years old
- Weight \> 12 kg
- EXCLUSION CRITERIA:
- Infection not adequately responding to appropriate therapy
- Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry.
- Creatinine \> 2.5 mg/dL
- Direct Bilirubin \> 2.0 mg/dL
- SGOT or SGPT \>5 times the upper limit of normal
- Hypersensitivity to eltrombopag or its components\<TAB\>
- Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
- Unable to understand the investigational nature of the study or give informed consent
- 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
- Treatment with ATG, cyclophophamide or alemtuzamab within 6 months of study entry.
Exclusion
Key Trial Info
Start Date :
June 28 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 24 2022
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT01891994
Start Date
June 28 2013
End Date
August 24 2022
Last Update
April 25 2023
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892