NCT01263379

Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa

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: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Abeona Therapeutics, Inc · Industry
• Sex: All
• Age: 13 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Epidermolysis Bullosa Dystrophica
• Epidermolysis Bullosa

Interventions

Type	Name	Description
BIOLOGICAL	LZRSE-Col7A1 Engineered Autologous Epidermal Sheets	This trial will create a graft, which we call "LEAES", of the patient's own skin that has been genetically engineered in our lab to express this missing protein.

Timeline

• Start date: 2010-10-05
• Primary completion: 2022-03-09
• Completion: 2022-03-09
• First posted: 2010-12-20
• Last updated: 2023-08-22
• Results posted: 2023-07-10

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study