A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy in Pediatric Patients Under 2 Years With Acid Sphingomyelinase Deficiency

What this Trial Is About

Researchers are conducting a multicenter, open-label observational study to assess the long-term safety and immune response to olipudase alfa therapy in young children with acid sphingomyelinase deficiency (ASMD), including Niemann-Pick diseases. This study focuses on pediatric patients under 2 years of age, aiming to gather real-world clinical data without altering their prescribed treatments. The study design is hybrid, allowing patients to participate without needing to travel to study sites. The study will observe patients receiving olipudase alfa as part of their routine clinical care. No treatments are administered by the study itself. The total study duration is 5 years, with an enrollment period up to 4 years and follow-up from 1 to 3 years for each participant. Data collected include safety events, immune responses to the drug, and laboratory and vital sign abnormalities over a 3-year period. Participants will provide informed consent through their parents or guardians and be monitored through clinical and laboratory assessments. Researchers will track adverse events, anti-drug antibody presence and levels, and any changes in laboratory tests and vital signs. The study collects data without influencing treatment decisions, aiming to understand long-term outcomes and safety during routine care.

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)