Gene-Modified Living Skin Graft GMEB-SASS for Treating Recessive Dystrophic Epidermolysis Bullosa RDEB in Children and Adults

What this Trial Is About

Researchers are evaluating a new gene-modified skin graft called GMEB-SASS to help wounds heal in people with Recessive Dystrophic Epidermolysis Bullosa RDEB. This first-in-human trial aims to assess the safety and effectiveness of this graft, which contains the patients own living skin cells genetically modified in the lab to produce a missing protein called type VII collagen. The study includes both adults and children and is divided into two phases to gather safety data first in adults before treating pediatric patients. The GMEB-SASS graft is created by growing the patients skin cells in the lab and using a virus-like tool to correct the genetic defect. The treatment involves two surgical steps first, temporary allogeneic skin grafts are applied to prepare the wound bed, followed by removal and application of the GMEB-SASS graft. The graft is designed to integrate permanently with the patients skin and covers up to 9% of the total body surface area per session, with the number of grafts based on the participants height, weight, and age. Participants will receive wound debridement and grafting procedures and be monitored for up to 12 months. Researchers will assess safety by tracking adverse events and measure pain and itch levels using visual scales at various time points. They will also evaluate how much of the grafted area has healed and use clinical scoring tools to assess disease activity and scarring. The study involves regular follow-ups to monitor healing and safety over one year after the grafting procedure.

GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment