Trial Profile

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 Delta Syndrome) Followed by an Open-label Long-term Extension

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 17 Jun 2026

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At a glance

15 Jun 2026 Planned End Date changed from 28 Oct 2026 to 28 Oct 2028.
15 Jun 2026 Planned primary completion date changed from 9 Jul 2026 to 9 Jul 2028.
06 Nov 2025 According to Pharming Group NV media release, company expect to report results from this study in the coming months and if the data are supportive we plan to seek regulatory approval for this younger pediatric population.

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