Eltrombopag for Moderate Aplastic Anemia
Completed · Phase 2
Conditions studied: Moderate Aplastic Anemia, Unilineage Bone Marrow Failure Disorders
In brief
Background: * Moderate aplastic anemia is a blood disease which may require frequent blood and platelet transfusions. Sometimes patients with this disease can be treated with immunosuppressive drugs. Not all patients respond and not all patients are suitable for this treatment. * Thrombopoietin (TPO) is a protein made by the body. The bone marrow needs TPO to produce platelets. TPO may also be able to stimulate bone marrow stem cells to produce red cells and white cells. However, TPO cannot be given by mouth. This has led researchers to develop the drug eltrombopag, which acts in the same way and can be given by mouth. Eltrombopag has been shown to safely increase platelet numbers in healthy volunteers and in patients with other chronic blood diseases, including severe aplastic anemia. Researchers are interested in looking at whether eltrombopag can be given to people with moderate aplastic anemia and significantly low blood cell counts. Objectives: \- To evaluate the safety and effectiveness of eltrombopag in people with moderate aplastic anemia or patients with bone marrow failure and unilineage cytopenia who need treatment for significantly low blood cell counts. Eligibility: \- People at least 2 years of age who have moderate aplastic anemia or bone marrow failure and unilineage cytopenia,and significantly low blood cell counts. Design: * Patients will be screened with a physical examination, medical history, blood tests, a bone marrow biopsy, and an eye exam. * Patients will receive eltrombopag by mouth once a day. * Patients will have weekly blood tests to monitor the effectiveness of the treatment and adjust the dose in response to possible side effects. * Patients may continue to take eltrombopag if their platelet count or hemoglobin increases, their requirement for platelet or blood transfusion decreases after 16 to 20 weeks of treatment, and there have been no serious side effects. Access to the drug will continue until the study is closed. Patients will be asked to return for a follow-up visit 6 months after the last dose of medication.
Key facts
- Study ID
- NCT01328587
- Run by
- National Heart, Lung, and Blood Institute (NHLBI)
- People needed
- 34
- Starts
- 2012-03-13
- Expected to finish
- 2026-01-06
- Last updated by the study team
- 2026-04-23
Who can join
Age: 2 and older, up to 100. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Known diagnosis of Fanconi anemia
- Counts that meet criteria for severe aplastic anemia
- Infection not adequately responding to appropriate therapy
- HIV positivity
- Creatinine > 2.5 mg/dL
- Bilirubin > 2.0 mg/dL, including congenital abnormalities in the bilirubin count
- SGOT or SGPT >5 times the upper limit of normal
- Hypersensitivity to eltrombopag or its components
- Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
- Evidence of an active malignant hematological or clonal disorder, or abnormal cytogenetic studies of the bone marrow performed within 12 weeks of study entry.
- Unable to understand the investigational nature of the study or give informed consent or does not have a legally authorized representative or surrogate that can provide 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 horse or rabbit ATG or Campath within 6 months of study entry.
- Treatment with cytokines such as G-CSF or Erythropoietin.
- Subjects with known cirrhosis in severity that would preclude tolerability of eltrombopag as evidenced by albumin less than 35g/L.
- Life expectancy of less than 3 months
- Patients with an active diagnosis of cancer who have received chemotherapeutic treatment or other specific antineoplastic drugs or radiation therapy within 6 months of study entry.
- Unable to take investigational drug
Where it is running
- National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States
Full record on ClinicalTrials.gov
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