Trial Search Results

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

This is a 78-week (approximately a year and a half) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 6 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 III study, as well as, new participants who were unable to participate in the Phase III study, who meet all enrollment criteria.

Stanford is currently accepting patients for this trial.

Lead Sponsor:

Krystal Biotech, Inc.

Stanford Investigator(s):


  • Biological: Open Label Topical Beremagene Geperpavec (B-VEC)


Phase 3


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: 6 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)

Ages Eligible for Study

6 Months - N/A

Genders Eligible for Study


Now accepting new patients

Contact Information

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