Extended Dosing With Eltrombopag for Severe Aplastic Anemia
- 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..
- To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts.
- Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia.
- 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.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Extended Dosing With Eltrombopag in Refractory Severe Aplastic Anemia|
- The proportion of drug responders as defined by changes in the platelet count and/or platelet transfusion requirements, hemoglobin levels, number of red blood cell transfusions, or neutrophil counts as measured by IWG criteria. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Hematological response at 3 & 12 months and yearly thereafter; (b) relapse (c) clonal evolution to PNH, clonal chromosomal population in bone marrow, myelodysplasia by morphology, or acute leukemia; (d) survival & (e) health-related qu... [ Time Frame: Indefinite ] [ Designated as safety issue: Yes ]
|Study Start Date:||June 2013|
|Estimated Study Completion Date:||March 2017|
|Estimated Primary Completion Date:||March 2017 (Final data collection date for primary outcome measure)|
Drug: Eltrombopag (promacta)
|Contact: Kinneret S Broder||(301) email@example.com|
|Contact: Ronan G Desmond, M.D.||(301) firstname.lastname@example.org|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL) 800-411-1222 ext TTY8664111010 email@example.com|
|Principal Investigator:||Ronan G Desmond, M.D.||National Heart, Lung, and Blood Institute (NHLBI)|