Efficacy and Safety of Oral E5501 Plus Standard of Care for the Treatment of Thrombocytopenia in Adults With Chronic Immune Thrombocytopenia (Amendment 02)

Summary

Core Study: To demonstrate that the efficacy of avatrombopag (in addition to standard of care) is superior to placebo (in addition to standard of care) for the treatment of adult participants with chronic immune thrombocytopenia (idiopathic thrombocytopenic purpura) (ITP) as measured by cumulative number of weeks of platelet response over 6 months of once daily treatment in adults participants who received at least 1 prior ITP therapy. Extension Phase: To evaluate the safety and tolerability of long-term therapy with avatrombopag in participants with chronic ITP (cITP).

Timeline

Start

2012-02-16

Primary completion

2013-11

Completion

2014-03

Outcome

Met primary endpoint

paper avatrombopag (N = 32) was superior to placebo (N = 17) in the median cumulative number of weeks of platelet response PMID 30191972 ↗

registry MEDIAN Placebo (Core Study): 0 (0 to 2) Weeks MEDIAN Avatrombopag (Core Study): 12.4 (0 to 25) Weeks NCT01438840 ↗

registry p <0.0001; Wilcoxon (Mann-Whitney) NCT01438840 ↗

Publications

• Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Jamieson BD, Tian W, Allen LF. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018 Nov;183(3):479-490. doi: 10.1111/bjh.15573. Epub 2018 Sep 7.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Avatrombopag	Small molecule	20 mg	Oral

Indications