Pharming Technologies B.v.

This study tests a drug called leniolisib in 20 people with common variable immunodeficiency (CVID), a condition that weakens the immune system. All participants will receive the drug for 6 months, starting at a low dose and increasing. The main goal is to check if the drug is sa…

This study tests a drug called leniolisib in 12 people aged 12 to 75 with certain primary immunodeficiency disorders that cause immune system problems. The main goal is to see if the drug is safe and tolerable, and to explore how well it works at different doses. Participants wil…

This study tests a drug called leniolisib in children aged 4 to 11 with APDS, a rare genetic condition that causes a faulty immune system. The goal is to see if the drug is safe and can control the disease by blocking an overactive immune signal. About 15 children will take the d…

This study tests a drug called leniolisib in children aged 1 to 6 years who have a rare genetic immune disease called APDS. The goal is to see if the drug is safe and can help control the disease by reducing swollen lymph nodes and other symptoms. About 16 children will take the …