Clinical Trial: Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer
Study Status: Terminated
Recruit Status: Terminated
Study Type: Observational
Official Title: Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer
Brief Summary: Recessive Dystrophic Epidermolysis Bullosa (RDEB) is a severe inherited blistering skin disease caused by absence of type VII collagen. Patients with RDEB develop large, severly painful blisters and open wounds from minor trauma to their skin. In the future, we hope to start a gene transfer study on a specific group of RDEB subjects and we are screening subjects for that potential trial now.
Detailed Summary: This study is a preparation for a gene transfer trial. In the gene transfer trial, we will transfect autologous RDEB keratinocytes with a type VII collagen containing retrovirus vector. We then plan to graft the genetically engineered and corrected autologous keratinocytes back on to wounds of the RDEB subject. We expect the grafts to attach as normal keratinocytes and replace the damaged skin with skin that is able to make type VII collagen and anchor normally to the dermis. We are trying to define the number of subjects 18 or older who meet the criteria for the gene transfer trial. We are offering the subjects the opportunity to be evaluated for the potential trial.
Sponsor: Stanford University
Current Primary Outcome:
- Disease documentation [ Time Frame: 1 year ]Documentation of disease
- documentation of disease [ Time Frame: 1 year ]documentation of disease
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Stanford University
Dates:
Date Received: September 20, 2007
Date Started: August 2007
Date Completion:
Last Updated: May 22, 2015
Last Verified: May 2015
