NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 Mutations research hub

Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations

This study is testing a new medicine called MAS825 for rare conditions like NLRC4-GOF, XIAP deficiency, and CDC42 mutations. It aims to see if the medicine is safe and helps patients feel better, compared to a dummy medicine, over several years.