NCT02859727

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)

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 37 (actual)
• Sponsor: Pharming Technologies B.V. · Industry
• Sex: All
• Age: 12 Years – 75 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Activated PI3Kdelta Syndrome (APDS); PASLI Disease

Interventions

Type	Name	Description
DRUG	CDZ173	140 mg/day

Timeline

• Start date: 2016-09-08
• Primary completion: 2025-01-30
• Completion: 2025-01-30
• First posted: 2016-08-09
• Last updated: 2026-03-30
• Results posted: 2026-03-30

Locations

8 sites across 7 countries: United States, Belarus, Czechia, Germany, Italy, Netherlands, Russia

Regulatory

• FDA-regulated drug study