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

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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
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
Novartis Investigative Site
Recruiting
Minsk, 223053
-
Belarus
Czechia
Novartis Investigative Site
Recruiting
Prague 5, 150 00
-
Czechia
Ireland
Novartis Investigative Site
Recruiting
Dublin,
-
Ireland
Italy
Novartis Investigative Site
Recruiting
Palermo, 90127
PA
Italy
Novartis Investigative Site
Recruiting
Brescia, 25123
-
Italy
Netherlands
Novartis Investigative Site
Recruiting
Rotterdam, 3000 CA
-
Netherlands
Russian Federation
Novartis Investigative Site
Recruiting
Moscow, 117198
-
Russian Federation
United Kingdom
Novartis Investigative Site
Recruiting
Belfast, BT9 7AB
-
United Kingdom
Novartis Investigative Site
Recruiting
London, NW3 2PF
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
1-888-669-6682
Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Email: 

Have a question?

Call 1-999-669-6682 or email [email protected]