Extension to the Study of Efficacy of CDZ173 in Patients With APDS/PASLI

An Open-label, Non-randomized Extension Study to Evaluate the Long Term Safety, Tolerability, Efficacy and Pharmacokinetics of CDZ173 in Patients With APDS/PASLI (Activated Phosphoinositide 3-kinase Delta Syndrome/p110δ-activating Mutation Causing Senescent T Cells, Lymphadenopathy and Immunodeficiency)

ClinicalTrials.gov Identifier: NCT02859727

Novartis Reference Number: CCDZ173X2201E1

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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 provide long-term CDZ173 treatment, a selective PI3Kδ inhibitor, to the patients with genetically activated PI3Kδ, i.e., patients with APDS/PASLI who participated in the CCDZ173X2201 study or who were treated previously with PI3Kδ inhibitors other than CDZ173. The study is open-label designed to establish the long-term safety, tolerability, efficay and pharmacokinetics of CDZ173 in the target population.

Condition 
Activated PI3Kdelta Syndrome (APDS); PASLI Disease
Phase 
Phase 2
Phase 3
Overall status 
Recruiting
Enrollment count 
42 participants
Start date 
Sep 08, 2016
Completion date 
Sep 01, 2026
Gender 
All
Age(s)
12 Years - 75 Years (Child, Adult, Older Adult)

Interventions

Drug
CDZ173
140 mg/day

Eligibility Criteria

Inclusion Criteria:

Written informed consent must be obtained before any assessment is performed.
Paients must have participated in the study CCDZ173X2201 or were treated previously with PI3Kδ inhibitors other than CDZ173.
Patients who are deemed by the Investigator to benefit from PI3Kδ inhibitor therapy.
Patients or their legal representatives (for patients under the age of 18 years) must be able to communicate well with the Investigator, to understand and comply with the requirements of the study.
Documented APDS/PASLI-associated genetic PI3K delta mutation.

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
Novartis Investigative Site
Recruiting
Bethesda, 20892
Maryland
United States
Czechia
Novartis Investigative Site
Recruiting
Prague 5, 15006
CZE
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

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]