RDEB
Clinical Trial (Recessive Dystrophic Epidermolysis Bullosa)

Trial Summary

Official Title

A Phase 1/2A Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Using the Drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)

Status

Active, not recruiting

Condition

Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa

About the Trial

Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa

The research project involves gene transfer into keratinocytes, which are the majority of the cells in the outer layer of skin. In this gene transfer trial we plan to biopsy some skin tissue, grow the cells in a skin cell culture (sterile dishes with special fluid that allows cells to grow and multiply) and then infect the cells with a virus that we have genetically engineered to insert the correct type VII collagen gene. The cells should then make type VII collagen.

The process of inserting the correct type VII collagen gene into cells is called "gene transfer." The virus used is called a "retrovirus." The virus is made so that it only delivers the type VII collagen gene and it should not spread to other parts of the body. During the study we will check for growth of the virus. 

After cells have received gene transfer, we will grow the cells in culture into a sheet of cells that look like a plastic film. We plan to graft the sheet to wounds. Grafting means we will take cells from the culture and stitch them to the patient's skin.

Detailed Info

Brief Summary

This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen. The corrected cells will be transplanted back to the patient.

Study Phase

Phase 1/Phase 2

Study Type

Interventional

Interventions
  • Biological: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
Sponsor

Stanford University

Enrollment

For more information on enrollment into the current EB-101 trial, please visit clinicaltrials.gov for location information.

Locations

The following cities have EB-101 clinical trial sites. Please check back often as locations will be updated.

Stanford University School of Medicine
Stanford, California 94305
United States
Status:

The safety and efficacy of the investigational use of this product has not been determined. There is no guarantee that the investigational use listed will be filed with and/or approved for marketing by a regulatory agency.