Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer

Epidermolysis Bullosa Dystrophica Clinical Trial

Official title: Recessive Dystrophic Epidermolysis Bullosa Screening for Possible Gene Transfer

Status Terminated

Clinical Trial 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.

Clinical Trial Description

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. ;

Study Design

Observational Model: Cohort, Time Perspective: Cross-Sectional

Related Conditions & MeSH terms

• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica

• NCT number: NCT00533572
• Study type: Observational
• Source: Stanford University
• Status: Terminated
• Phase: N/A
• Start date: August 2007
• Completion date: August 2008