Leniolisib for Immune Dysregulation in PIDs

Patients ages 12-75 diagnosed with genetically defined PID disorders linked to PI3K signaling. This includes disorders caused by pathogenic variants in SOCS1, PTEN, CTLA4, NFKB1 (variants leading to NFKB pathway activation), FAS (germline or somatic), or RAS-associated leukoproliferative disorder caused by somatic variants in NRAS or KRAS (not juvenile myelomonocytic leukemia \[JMML\]). All subjects participating will receive leniolisib film-coated tablets (FCTs) with a planned dose regimen consisting of a starting dose of 10 mg twice daily (BID) for 4 weeks, followed by 30 mg BID for 4 weeks, and then 70 mg BID for 12 weeks. Leniolisib dose increase at the individual subject level will occur if no safety or tolerability issues have been identified by the Investigator that precludes the planned dose escalation. Subjects not continuing leniolisib treatment outside of the current protocol will be followed up, with the EOS visit planned to occur approximately 28 days after last dose of leniolisib.