Leniolisib for Immune Dysregulation in PIDs

Summary

This study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib. Secondary objectives include assessments of PK/PD, and to explore clinical efficacy measures with administration of three different dose levels of leniolisib.

Timeline

Start

2024-10-21

Primary completion

2026-11

Completion

2026-11

Publications

• Hassan C, Ponstein Y, Hanssen R, Thorneloe KS, Marsh RA, Rao VK. Leniolisib reduced lymphoproliferative disease in murine autoimmune lymphoproliferative syndrome. J Hum Immun. 2025 Nov 17;2(1):e20250125. doi: 10.70962/jhi.20250125. eCollection 2026 Jan 5.

Drugs

Indications