Pediatric Patients Aged 4 to 11 Years With APDS

Running, not enrolling · Phase 3

Conditions studied: APDS

In brief

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).

Key facts

Study ID

NCT05438407

Run by

Pharming Technologies B.V.

People needed

15

Starts

2023-02-01

Expected to finish

2026-12-30

Last updated by the study team

2026-05-06

Who can join

Age: 4 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

Where it is running

• University of California Los Angeles — Los Angeles, California, United States
• Stanford University — Standford, California, United States
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States
• National Institutes of Health — Bethesda, Maryland, United States
• Necker Hospital Paris — Paris, France
• Kyoto University Hospital — Kyoto, Japan
• Institute of Science Tokyo Hospital — Tokyo, Japan

Full record on ClinicalTrials.gov

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