FV 1183. Long-Term Safety and Efficacy of Intraventricular Enzyme Replacement Therapy in CLN2 Disease: 2-Year Results from an Ongoing Multicenter Extension Study

Background: CLN2 disease, a rare, inherited, pediatric, neurodegenerative lysosomal storage disorder caused by TPP1 deficiency, is characterized by seizures, language and motor function loss, blindness, and early death. A phase 1/2 study (NCT01907087) demonstrated that intracerebroventricular (ICV) infusion of 300 mg cerliponase alfa, a recombinant human TPP1 enzyme, every other week for 48 weeks slowed progression in motor and language function. This extension study (NCT02485899) assesses the long-term safety and efficacy of ICV-administered cerliponase alfa in children with CLN2 disease for up to 240 weeks.