Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects
Status:
Completed
Trial end date:
2017-09-06
Target enrollment:
Participant gender:
Summary
This was an open label, non-randomized, phase II study of eltrombopag in combination with
rabbit ATG/CsA in subjects with moderate or more severe AA who did not received prior
ATG/ALG-based immunosuppressive therapy. The objective was to assess additive effects of
eltorombopag on overall response rate (ORR) at 6 months (Week 26) of treatment with ATG/CsA.
Subjects were assessed at least weekly for safety during the period from the start of ATG/CsA
to 4 weeks after the start of administration of eltrombopag. After that, subjects had visits
every 2 weeks until Week 26. Subjects in whom the treatment was assessed as effective at Week
26 could continued treatment with eltrombopag after 6 months when clinically indicated at the
discretion of the investigator. There were five follow-up visits: at discontinuation of the
treatment of eltrombopag, and Weeks 1, 2, 3, 4 and 26 after treatment discontinuation. As
this study was the first Japanese phase II study in which this product was administered in
combination with ATG/CsA to subjects with naive moderate or more severe AA, the subject
number of this study was determined to be 10 based on the feasibility survey.