Long-Term Safety Follow-Up of Prademagene Zamikeracel Treatment for Recessive Dystrophic Epidermolysis Bullosa with Annual Remote Monitoring

What this Trial Is About

This observational study focuses on individuals with Recessive Dystrophic Epidermolysis Bullosa RDEB who were treated with prademagene zamikeracel pz-cel, a gene therapy made from the participants own skin cells and applied surgically to treat wounds. The study aims to evaluate the long-term safety of pz-cel, the durability of wound healing, and the occurrence of any malignancies after treatment in the post-marketing setting. Participants who have received pz-cel treatment will be followed for up to 15 years after their treatment. Assessments will primarily be collected remotely on an annual basis, but if the participants healthcare provider deems it necessary after the first year, some data may be collected during in-person visits. Annual evaluations will include reviewing adverse events, serious adverse events, hospitalizations, evidence of malignancies, potential retroviral infections, wound healing status based on caregiver reports, and any changes in medications or procedures. Throughout the study, participants will provide data annually for up to 15 years post-treatment. Researchers will monitor the number and incidence of treatment-related malignancies, serious adverse events including wound-specific events, and any positive retroviral infection test results. This long-term follow-up aims to understand the safety profile and wound healing durability of pz-cel, with participants contributing data primarily through remote means and occasional in-person assessments as needed.

A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting