Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia
Overview
- Phase
- Phase 2
- Intervention
- Eltrombopag
- Conditions
- Severe Aplastic Anemia
- Sponsor
- University of Utah
- Enrollment
- 13
- Locations
- 1
- Primary Endpoint
- Proportion of Participants With Platelet Response
- Status
- Completed
- Last Updated
- 8 years ago
Overview
Brief Summary
The investigators hypothesis is that eltrombopag given to patients with moderate to very severe aplastic anemia will result in an increase in platelet counts. The investigators hypothesize that in patients with moderate to very severe aplastic anemia, treatment with eltrombopag will lead to fewer platelet transfusions, red blood cell transfusions, and fewer bleeding events. The investigators hypothesize that in patients with moderate to very severe aplastic anemia, eltrombopag will have an acceptable toxicity rate <3%, at doses that result in increased platelet counts. Finally the investigators hypothesize that plasma eltrombopag levels in peripheral blood will correlate with improved platelet counts.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Able to provide written informed consent and any other authorizations required by local law (e.g., Protected Health Information \[PHI\])
- •Have severe or very severe aplastic anemia, or moderate aplastic anemia with platelet counts that have dropped below 20,000/μl
- •Have moderate, severe, or very severe aplastic anemia with moderate bleeding during or after a surgical procedure, (including bone marrow biopsy, lumbar puncture, thoracentesis, paracentesis, port placement, dermal biopsy) or minimal mucocutaneous bleeding otherwise noted
- •Subjects with current or previous exposure to approved medications for the treatment of aplastic anemia will not be excluded; these include but may not be limited to, anti-thymocyte globulin (ATG), cyclosporine, corticosteroids, and G-CSF.
Exclusion Criteria
- •Have diagnosis of Fanconi anemia
- •Have infection not adequately responding to appropriate therapy
- •Have Paroxysmal Nocturnal Hemoglobinuria (PNH) clone size in neutrophils of greater than or equal to 50%
- •Have known HIV positivity
- •Have creatinine and/or blood urea nitrogen (BUN) ≥2 times the upper limit of normal
- •Have serum bilirubin ≥ 1.5 times the upper limit of normal, or ≥4.0 times the upper limit of normal if the patient has been treated with ATG within three weeks of screening.
- •Have AST and/or ALT ≥ 3 times the upper limit of normal
- •Have hypersensitivity to eltrombopag or its components
- •Have chemotherapy given less than or equal to 14 days prior to initiating the study medication. This does not include immunosuppressive agents and growth factor as described above
- •Are female and are nursing or pregnant or are unwilling to take oral contraceptives or refrain from pregnancy if of childbearing potential
Arms & Interventions
Eltrombopag
Single arm study. Dose Escalation.
Intervention: Eltrombopag
Outcomes
Primary Outcomes
Proportion of Participants With Platelet Response
Time Frame: up to 12 weeks
Defined as a stable platelet count of 50,000/μl or more during any 4 week period within the possible 12 weeks while on study,and including maximal platelet counts achieved in patients with moderate to very severe aplastic anemia.
Secondary Outcomes
- Platelet Count Twice Baseline.(Between weeks 1-12.)
- Hematology Labs(12 weeks)
- Number of Patients With AE to Measure Toxicity, Using NCI CTCAE(12 weeks)
- Characterization of the PK Profile of Eltrombopag in Patients With Moderate to Very Severe Aplastic Anemia. Evaluated With AUC, Cmax, Cmin, Tmax.(Weeks 2, 6 and 12)