Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension Studies.

doi:10.4049/jimmunol.2000326

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Article

Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension Studies.

Journal of immunology (Baltimore, Md. : 1950) - 1 Dec 2020

Diaz Nieves, Juarez Maria, Cancrini Caterina, Heeg Maximilian, Soler-Palacín Pere, Payne Andrew, Johnston Geoffrey I, Helmer Eric, Cain Dionne, Mann Joanne, Yuill Daisy, Conti Francesca, Di Cesare Silvia, Ehl Stephan, Garcia-Prat Marina, Maccari Maria Elena, Martín-Nalda Andrea, Martínez-Gallo Mónica, Moshous Despina, Santilli Veronica, Semeraro Michaela, Simonetti Alessandra, Suarez Felipe, Cavazzana Marina, Kracker Sven

Abstract excerpt

Mutations in two genes can result in activated PI3Kδ syndrome (APDS), a rare immunodeficiency disease with limited therapeutic options. Seletalisib, a potent, selective PI3Kδ inhibitor, was evaluated in patients with APDS1 and APDS2. In the phase 1b study (European Clinical Trials Database 2015-002900-10) patients with genetic and clinical confirmation of APDS1 or APDS2 received 15-25 mg/d seletalisib for 12 wk....

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PMID: 33115853
DOI: 10.4049/jimmunol.2000326

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