An Open-label, Non-randomized, Within-patient Dose-finding Study Followed by a Randomized, Subject, Investigator and Sponsor-blinded Placebo Controlled Study to Assess the Efficacy and Safety of CDZ173 in Patients With APDS/PASLI
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.
This study is designed to explore CDZ173, a selective PI3Kδ inhibitor, in patients with genetically activated PI3Kδ, i.e., patients with 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.
Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells
Lymphadenopathy and Immunodeficiency (PASLI)
Apr 22, 2020
Aug 24, 2015
Jun 08, 2021
12 Years - 75 Years (Child, Adult, Older Adult)
Part I 10, 30 and 70 mg bid Part II 70 mg bid and matching placebo
Key Inclusion Criteria:
patients who have a documented APDS/PASLI-associated genetic PI3K delta mutation
In Part I and Part II, patients must have nodal and/or extranodal lymphoproliferation, and clinical findings and manifestations compatible with APDS/PASLI such as a history of repeated oto-sino-pulmonary infections and/or organ dysfunction (e.g., lung, liver). Additionally, in part II, patients must have at least one measurable nodal lesion on a CT or MRI scan.
Key Exclusion Criteria:
Any medically significant disease or condition that is unrelated to APDS/PASLI
Other protocol-defined inclusion/exclusion criteria may apply.