Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients

Phase 2 Terminated 24 enrolled Weill Medical College of Cornell University Novartis Pharmaceuticals · collab

Non-randomizedSingle-groupOpen-labelTreatment

Summary

The purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.

Timeline

Start: 2009-06
Primary completion: 2017-05-15
Completion: 2017-06-30

Outcome

Outcome not reported

Stopped: “retirement of PI”

Publications

Gerrits AJ, Leven EA, Frelinger AL 3rd, Brigstocke SL, Berny-Lang MA, Mitchell WB, Revel-Vilk S, Tamary H, Carmichael SL, Barnard MR, Michelson AD, Bussel JB. Effects of eltrombopag on platelet count and platelet activation in Wiskott-Aldrich syndrome/X-linked thrombocytopenia. Blood. 2015 Sep 10;126(11):1367-78. doi: 10.1182/blood-2014-09-602573. Epub 2015 Jul 29.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	SB-497115-GR	Unknown	1 mg/kg	Oral