Long-term Follow-up Study for Children Aged 7 to 15 Years Previously in iECURE Protocols Using an Investigational Product

What this Trial Is About

Researchers are conducting a long-term follow-up study lasting 14.5 years to assess the safety and lasting effects of an investigational product IP used in previous iECURE studies. The study also aims to gather ongoing natural disease information from participants who enrolled but were not treated with the IP. Participants continue their usual medical care prescribed by their doctors throughout the study. This observational study does not provide any investigational treatment. Instead, it follows two groups those who received the IP in earlier iECURE studies and those who were enrolled but not dosed. No new drug or therapy is given as part of this follow-up. Participants maintain their prescribed standard care while researchers collect data over time. Participants will be monitored regularly for safety, growth changes, and other health measures over 14.5 years. The study collects information on adverse events, changes in height and weight, urinalysis results, and detailed laboratory tests including vector clearance in blood, saliva, urine, and feces. Researchers also track serious health events related to the disease, hospitalizations, and survival outcomes. This comprehensive follow-up aims to provide valuable long-term data on participants health and treatment effects.

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)