Study of Efficacy of CDZ173 in Patients With APDS/PASLI

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

ClinicalTrials.gov Identifier: NCT02435173

Novartis Reference Number: CCDZ173X2201

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.

Study Description

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.

Condition

Common Variable Immunodeficiency (CVID) More Specifically Activated PI3Kdelta Syndrome (APDS) p110delta-activating Mutation Causing Senescent T Cells

Lymphadenopathy and Immunodeficiency (PASLI)

Phase

Phase 2

Phase 3

Overall status

Recruiting

Enrollment count

36 participants

Last update

Apr 22, 2020

Start date

Aug 24, 2015

Completion date

Jun 08, 2021

Gender

All

Age(s)

12 Years - 75 Years (Child, Adult, Older Adult)

Interventions

Drug

CDZ173

Part I 10, 30 and 70 mg bid Part II 70 mg bid and matching placebo

Eligibility 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.

Study Locations

United States

National Institute of Health NIH

Recruiting

Bethesda, 20814

Maryland

United States

Belarus