Extended Dosing With Eltrombopag for Severe Aplastic Anemia

• Status: Active, not recruiting
• Trial end date: 2021-12-31
• Target enrollment: 0
• Participant gender: All

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 should be taken for this type of anemia.

Objectives:

• To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts.

Eligibility:

• Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia.

Design:

• Participants will take eltrombopag by mouth once a day for 6 months.

• Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow samples will be collected at 3 and 6 months. These samples will look at the effects of the study drug on the marrow.

• Participants will continue to take the study drug for as long as it is effective and if the side effects are not severe.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Criteria

• 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- 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.