Stanford University School of Medicine Gene Transfer Study

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

STANFORD UNIVERSITY SCHOOL OF MEDICINE
DEPARTMENT OF DERMATOLOGY

JEAN Y. TANG, M.D., PH.D.
ASSOCIATE PROFESSOR OF DERMATOLOGY
PH: 650.721.7152 FX: 650.724.3823
EMAIL: TANGY [at] STANFORD [dot] EDU

Since you have asked to be part of our database of people contacted about future EB studies, I would like to give you an update on our gene transfer trial for recessive dystrophic EB, entitled: “A Phase 1 Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) using the drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES).”

We have grafted six adult patients and have not had any serious adverse events. The FDA recently granted us permission to start grafting patients, age 13 and over.

We will be creating a skin graft of the subject’s own skin that has been genetically engineered in our lab to express the protein that RDEB skin is usually missing and graft it back on to the participant.

Entrance into the gene transfer study is a two-step process. We will select subjects who first participate in our study of the Characteristics of Patients with Dystrophic EB. We are still recruiting participants for the Characteristics study.

The “Characteristics” study will require a trip to Stanford, during which we will examine you, take some blood, and some skin biopsies. Some of this blood will be sent for genetic testing. We will be able to cover reasonable travel expenses and the cost of the medical procedures. After we have more information about you from the Characteristics study, we will know if you may be a potential candidate for gene transfer.

We are also recruiting subjects age 7 – 12 for the Characteristics study, who would not be eligible for gene transfer right now, but may be able to participate in studies of new treatments in the future.

For more information about the gene transfer study and the Characteristics study, please visit our website: http://med.stanford.edu/dermatology/research/research.html. You can also read more about this study at: http://clinicaltrials.gov/ct2/show/NCT01263379.

If you have any questions, or would like to participate in these studies, please contact me at tangy [at] stanford [dot] edu, or phone 650-721-7152.

Sincerely,

Jean Tang, M.D., Ph.D.