Clinical Trial

Pediatric Patients Aged 1 to 6 Years With APDS

Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
This is a 2-part, prospective, open-label, single arm, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and efficacy of leniolisib in at least 15 pediatric patients (aged 1 to 6 years) with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS)
Protocol Amendment History 19 amendments
This ClinicalTrials.gov record has been amended 19 times since 2023-01-12; most recent amendment 2026-06-11.
Status change: Recruiting → Active, Not Recruiting 2025-04-17
Status change: Not Yet Recruiting → Recruiting 2023-10-02
Trial Details
NCT Number NCT05693129
Lead Sponsor Pharming Technologies B.V.
Collaborators: CMIC Co, Ltd. Japan, Labcorp Central Laboratory, Fortrea, Aixial Group
Conditions APDS
Enrollment 16 participants
Start Date 2023-08-30
Primary Completion 2028-07-09 (estimated)
Study Completion 2028-10-28 (estimated)
Updated on ClinicalTrials.gov 2026-06-15