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First received: October 17, 2022

Clinical Trial: Pediatric Patients Aged 1 to 6 Years With APDS

Study Status: Completed

Recruit Status: RECRUITING

Condition: APDS

Study Type: INTERVENTIONAL


Official Title: An Open-label, Single Arm Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Leniolisib in Pediatric Patients (Aged 1 to 6 Years) With APDS (Activated Phosphoinositide 3-Kinase D

Brief 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 (PI3K?) syndrome (APDS)

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