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Blood, 1 June 2007, Vol. 109, No. 11, pp. 4739-4741.
Prepublished online as a Blood First Edition Paper on February 27, 2007; DOI 10.1182/blood-2006-11-057968.


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HEMATOPOIESIS

Brief Report

Phase 1 clinical study of eltrombopag, an oral, nonpeptide thrombopoietin receptor agonist

Julian M. Jenkins1, Daphne Williams1, Yanli Deng1, Joanne Uhl1, Valerie Kitchen1, David Collins1, and Connie L. Erickson-Miller1

1 GlaxoSmithKline, Collegeville, PA

Eltrombopag (SB-497 115) is a first-in-class, oral, small-molecule, nonpeptide agonist of the thrombopoietin receptor (TpoR), being developed as a treatment for thrombocytopenia of various etiologies. In this phase 1 placebo-controlled clinical trial in 73 healthy male subjects, eltrombopag was administered as once-daily oral capsules for 10 days at doses of 5, 10, 25, 30, 50, and 75 mg. The pharmacokinetics of eltrombopag were dose dependent and linear, and eltrombopag increased platelet counts in a dose-dependent manner. There were no apparent differences in the incidence or severity of adverse events in subjects receiving active or placebo study medication. These observations indicate that eltrombopag is a once-daily, oral TpoR agonist with demonstrated thrombopoietic activity in human subjects, encouraging further studies in patients with thrombocytopenia.


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