A Three-part Study of Eltrombopag in Thrombocytopenic Subjects With Myelodysplastic Syndromes or Acute Myeloid Leukemia
Status:
Completed
Trial end date:
2015-12-01
Target enrollment:
Participant gender:
Summary
This was a worldwide, three-part (Part 1: open-label, Part 2: randomized, double-blind, Part
3: extension), multi-center study to evaluate the effect of eltrombopag in subjects with
myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who have thrombocytopenia due
to bone marrow insufficiency from their underlying disease or prior chemotherapy. This
objective was assessed by a composite primary endpoint that consists of the following: the
proportion of ≥Grade 3 hemorrhagic adverse events, or platelet counts <10 Gi/L, or platelet
transfusions. Patients with MDS or AML and Grade 4 thrombocytopenia due to bone marrow
insufficiency from their underlying disease or prior chemotherapy were enrolled in the study.
No low or intermediate-1 risk MDS subjects were enrolled in the study. Subjects must have had
at least one of the following during the 4 weeks prior to enrolment: platelet count <10 Gi/L,
platelet transfusion, or symptomatic hemorrhagic event. Supportive standard of care (SOC),
including hydroxyurea, was allowed as indicated by local practice throughout the study. The
study had 3 sequential parts. Subjects who were enrolled in Part 1 (open-label) cannot be
enrolled in Part 2 of the study (randomized, double-blind); however, subjects who completed
the treatment period for Part 1 or Part 2 (8 and 12 weeks, respectively) continued in Part 3
(extension) if the investigator determined that the subject was receiving clinical benefit on
treatment.