A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa


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Trial ID: NCT04917874


This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB). Participants will be dosed weekly with the topical B-VEC therapy. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study is for those who participated in Phase 3 study, as well as, new participants who were unable to participate in the Phase 3 study, who meet all enrollment criteria.

Official Title

Open Label Treatment of Beremagene Geperpavec (B-VEC)

Stanford Investigator(s)

M. Peter Marinkovich, MD
M. Peter Marinkovich, MD

Associate Professor of Dermatology

Jennifer Chen, MD
Jennifer Chen, MD

Clinical Professor, Dermatology


Inclusion Criteria:

   - Willing and able to give consent/assent

   - Clinical diagnosis of epidermolysis bullosa

   - Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1.

   - Age: 2 months of age and older at the time of informed consent/assent

   - Women of childbearing age must be willing to use reliable birth control method
   throughout the treatment duration and for 3 months following the last treatment

Exclusion Criteria:

   - Diseases or conditions that could interfere with the assessment of safety of the study
   treatment and compliance of the participant with study visits/procedures, as
   determined by the Investigator

   - Pregnant or nursing women

   - Active infection in the area that will undergo administration, that the Investigator
   believes will negatively impact the IP application

   - Known allergy to any of the constituents of the product

   - Concurrent skin transplant or mesh skin grafting; if the participant is currently
   having transplantation or grafting surgeries they must wait until the transplant has
   begun to heal and the graft has absorbed prior to initiating B-VEC therapy

   - Participation in an interventional gene therapy clinical trial within the past three
   (3) months (not including B-VEC administration)


biological: Open Label Topical Beremagene Geperpavec (B-VEC)


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Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Sinem Bagci