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

Phase 2

Terminated

• Conditions: Activated PI3Kdelta Syndrome (APDS); PASLI Disease
• Interventions: Drug: CDZ173

• Registration Number: NCT02859727
• Lead Sponsor: Pharming Technologies B.V.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-08-04
• Study First Submitted QC: 2016-08-04
• Study First Posted: 2016-08-09
• Study Start: 2016-09-08
• Primary Completion: 2025-01-30
• Study Completion: 2025-01-30
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-10
• Last Update Posted: 2025-03-12

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 37

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CDZ173	CDZ173	140mg/day

Primary Outcome Measures

Name	Time	Method
To evaluate the long term safety and tolerability of CDZ173 in patients with APDS/PASLI	6 years 3 months	All safety parameters (including AEs, physical exam, vital signs, ECG, safety laboratory (hematology, blood chemistry, urinalysis))

Secondary Outcome Measures

Name	Time	Method
To evaluate the long term efficacy of CDZ173 to modify health-related quality of life in patients with APDS/PASLI	6 years	SF-36 (Short Form 36) Survey and WPAI-CIQ (Work Productivity Activity Impairment plus Classroom Impairment Questionnaire), Visual analogue scales for Physician's Global Assessment (PGA) and Patient's Global Assessment (PtGA), patient narratives by Investigator
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	8 months	High sensitivity C-reactive protein (CRP), lactate dehydrogenase (LDH), frequencies of infections and other disease complication
To characterize the pharmacokinetics (trough concentration) of CDZ173 in patients with APDS/PASLI	9 months	Steady-state trough concentration of CDZ173
• To evaluate the pharmacokinetics and relative bioavailability of CDZ173 film-coated tablets compared to CDZ173 hard-gelatin capsules	up to 6 months	PK parameters (including but not limited to AUC0-12,ss and Cmax,ss)

Trial Locations

Locations (1)

Pharming Investigative Site; 🇷🇺 Moscow, Russian Federation