Clinical Trial

Pediatric Patients Aged 4 to 11 Years With APDS

Active, Not Recruiting Phase 3
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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 4 to 11 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 2022-06-27; most recent amendment 2026-05-05.
Status change: Recruiting → Active, Not Recruiting 2024-03-20
Status change: Not Yet Recruiting → Recruiting 2023-02-16
Trial Details
NCT Number NCT05438407
Lead Sponsor Pharming Technologies B.V.
Collaborators: CMIC Co, Ltd. Japan, Labcorp Central Laboratory, Fortrea, Aixial Group
Conditions APDS
Enrollment 15 participants
Start Date 2023-02-01
Primary Completion 2026-11-30 (estimated)
Study Completion 2026-12-30 (estimated)
Updated on ClinicalTrials.gov 2026-05-06