Leniolisib for immunedysregulation

National Institute Of Allergy And Infectious Diseases

Investigators

Abstract

Clinical protocol titled "An open-label, single-arm extension study to evaluate the long-term safety, tolerability, and efficacy of leniolisib for immune dysregulation in primary immunodeficiency" has been developed in collaboration with Pharming, and its is now at its final stage of IRB approval. We have been enrolling patients since the beginning of 2025 and currently surpassed 50% enrollment An open label extension study to follow this trial has also been approved by IRB. First 5 patients who completed the trial have been rolled over to the open label extension trial using Leniolisib We have developed a flow based PI3K signaling assay to evaluate in vitro response to in vivo treatment, which is being used during this study.