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

Pharming Healthcare

Status and phase

Active, not recruiting

Phase 3

Phase 2

Conditions

Activated PI3Kdelta Syndrome (APDS); PASLI Disease

Treatments

Drug: CDZ173

Study type

Interventional

Funder types

Industry

Identifiers

NCT02859727

2016-000468-41 (EudraCT Number)

CCDZ173X2201E1

Details and patient eligibility

About

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.

Enrollment

37 patients

Sex

All

Ages

12 to 75 years old

Volunteers

No Healthy Volunteers

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