Christ, Andreas Dominik and Soldermann, Nicolas (2015) An open-label, non-randomized, within-patient dose-finding study followed by a randomized, double-blind placebo controlled study with extension to assess the safety and efficacy of CDZ173 in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency). Part of a CRADA with NIH, thus NIH will be allowed to make the abstract publicly available.

Abstract

Under a Cooperative Research and Development Agreement (CRADA), Novartis Pharmaceuticals Corporation will provide the National Institute of Allergy and Infectious Diseases (NlAID) with CDZ173, a proprietary PI3Kδ inhibitor, for use in a clinical trial to evaluate its safety, pharmacokinetics, tolerability and efficacy in patients with "p110δ activating mutation causing senescent T cells, lymphadenopathy, and immunodeficiency" (PASLl). This study follows the discovery by NIAID that PASLl patients harbor gain of function mutations in the PIK3CD gene (Lucas et al 2014; Angulo et al 20J3; Crank et al 2014). Given the specificity of CDZ173 to selectively inhibit the p110δ subunit of PI3KD, this study will test if CDZ173 can provide effective treatment for PASLl, a newly described disease with a significant unmet medical need.

Item Type:	Article
Date Deposited:	13 Oct 2015 13:11
Last Modified:	13 Oct 2015 13:11
URI:	https://oak.novartis.com/id/eprint/24338