Study of Efficacy of CDZ173 in Patients With APDS/PASLI
Status:
Completed
Trial end date:
2021-08-16
Target enrollment:
Participant gender:
Summary
This study is designed to explore CDZ173, a selective PI3Kδ inhibitor, in patients with
genetically activated PI3Kδ, i.e., patients with Activated phosphoinositide 3-kinase delta
syndrome/ p110δ-activating mutation causing senescent T cells, lymphadenopathy and
immunodeficiency (APDS/PASLI). The study consists of two parts: Part I is the open label part
designed to establish the safety and pharmacokinetics of CDZ173 in the target population, as
well as to select the optimal dose to be tested in part II. Part II is designed to assess
efficacy and safety of CDZ173 in this population.