Trial ID or NCT#

NCT01263379

Status

RECRUITING

Purpose

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe inherited blistering skin disease caused by absence of a protein known as type VII collagen. Patients with RDEB develop large, severely painful blisters and open wounds from minor trauma to their skin. This trial will create a graft, which the investigators call "LEAES," of the patient's own skin that has been genetically engineered in the investigators lab to express this missing protein. The purpose of this study is to achieve proof-of-concept for this general approach to cell-based gene therapy in humans and to set the stage for further therapeutic extension in RDEB. The investigators will basically take a subject's own cells, correct them in culture, and then transplant the corrected cells back onto them.

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)

Eligibility Criteria

Ages Eligible for Study: Older than 13 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No

Investigator(s)

Paul A. Khavari, MD, PhD
Dermatologist, Cutaneous oncology specialist, Medical oncologist
Carl J. Herzog Professor in Dermatology in the School of Medicine
Jean Y. Tang MD PhD
General dermatologist, Dermatologic oncologist, Cutaneous oncology specialist
Associate Professor of Dermatology at the Stanford University Medical Center
M. Peter Marinkovich, MD
Dermatologist, Blistering disease specialist, Cutaneous oncology specialist
Associate Professor of Dermatology
H. Peter Lorenz, MD
Plastic and reconstructive surgeon
Professor of Surgery (Plastic and Reconstructive Surgery) at the Stanford University Medical Center
Phuong Khuu
Dermatologist, Pediatric dermatologist
Clinical Assistant Professor, Dermatology
Louise Furukawa
Anesthesiologist
Clinical Associate Professor, Anesthesiology, Perioperative and Pain Medicine

Contact us to find out if this trial is right for you.

CONTACT

Kylie Loutit, MS
(650) 724-1982