Data Studio

MSOB x393

DATE: January 17, 2018
TIME: 1:30 - 3:00 pm
TITLE: Clinical trial design for gene transfer therapy of recessive dystrophic epidermolysis bullosa
Jean Tang, Associate Professor, Dermatology



Recessive dystrophic epidermolysis bullosa (RDEB) is a devastating, often fatal, inherited blistering disorder caused by mutation in the COL7A1 gene encoding type VII collagen. Chronic wounds in RDEB patients are unique in that wounds are typically large and last several months to years and can have a dynamic surface area, with areas of healing and re-blistering.  A phase 1/2a study that evaluates the safety and wound outcomes following genetically corrected autologous epidermal grafts has been successfully completed in 7 adult subjects (N=42 grafts). We are currently working on the phase 3 study protocol in which we will enroll 10 RDEB subjects (N=60 grafts) to determine the wound healing outcome of genetically corrected autologous epidermal grafts versus intra-individual control wounds (untreated wounds) in patients aged 6 and older.  The goal of this Data Studio is to discuss the different statistical approaches to analyzing the unique and complex wound healing process in RDEB patients.

Background readings:

J D Fine. Inherited epidermolysis bullosa: past, present, and future.  Annals of the New York Academy of Sciences 2010; 1194:213-222.

Z Siprashvili Z, et al.  Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa. JAMA 2016; 316:1808-1817.