Bethesda, Maryland 20814


Purpose:

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.


Study summary:

This study is designed to explore CDZ173, a selective PI3Kδ inhibitor, in patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)I. 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.


Criteria:

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.


NCT ID:

NCT02435173


Primary Contact:

Principal Investigator
Koneti V Rao, MD
National Institutes of Health (NIH)

Novartis Pharmaceuticals
Phone: 1-888-669-6682
Email: novartis.email@novartis.com


Backup Contact:

Novartis Pharmaceuticals
Phone: +41613241111


Location Contact:

Bethesda, Maryland 20814
United States

Sharon Webster
Phone: 301-496-2771
Email: Sharon.webster@nih.gov

Site Status: Recruiting


Data Source: ClinicalTrials.gov

Date Processed: November 18, 2017

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