Clinical trial NCT02859727: Extension to the Study of Efficacy of CDZ173 in Patients with APDS/PASLI
Official title: 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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Activated PI3Kdelta Syndrome (APDS); PASLI Disease
Keywords: APDS; PASLI; PI3Kdelta; p110delta-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency; Activated PI3Kdelta Syndrome;
Lead sponsor: Pharming Technologies B.V.
Sponsor class: INDUSTRY
First posted: 2016-08-09
Enrollment: 37
Sex: ALL
Minimum age (years): 12
Maximum age (years): 75
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


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.

Primary outcomes:
measure: To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI
timeFrame: 6 years 3 months
Secondary outcomes:
measure: To evaluate the long term efficacy of CDZ173 to modify health-related quality of life in patients with APDS/PASLI
timeFrame: 6 years
measure: To evaluate the long term efficacy of CDZ173 by means of biomarkers reflecting the efficacy of CDZ173 to reduce systemic inflammatory components of the disease in patients with APDS/PASLI
timeFrame: 8 months
measure: To characterize the pharmacokinetics (trough concentration) of CDZ173 in patients with APDS/PASLI
timeFrame: 9 months
measure: • To evaluate the pharmacokinetics and relative bioavailability of CDZ173 film-coated tablets compared to CDZ173 hard-gelatin capsules
timeFrame: up to 6 months
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: Pharming Investigative Site
state: Maryland
city: Minsk
country: Belarus
facility: Pharming Investigative Site
city: Prague 5
country: Czechia
facility: Pharming Investigative Site
state: CZE
city: Dresden
country: Germany
facility: Pharming Investigative Site
city: Brescia
country: Italy
facility: Pharming Investigative Site
state: BS
city: Palermo
country: Italy
facility: Pharming Investigative Site
state: PA
city: Rotterdam
country: Netherlands
facility: Pharming Investigative Site
city: Moscow
country: Russian Federation
facility: Pharming Investigative Site
Study officials:
References: