Children, Teens and Adults with Dystrophic Epidermolysis Bullosa Needed for a Research Study of B-VEC, a Topical Gene Therapy

Recruitment Status: Recruiting


The title of the study is: A Phase III Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB). The Study Sponsor, Krystal Biotech, Inc. is conducting a research study to evaluate the safety and effectiveness of an investigational gene therapy, B-VEC, for the treatment of wounds in people with Dystrophic Epidermolysis Bullosa (DEB).


B-VEC is a topical gene therapy that is intended to correct the underlying cause of DEB by expressing the collagen VII protein that is missing in DEB patients. B-VEC is the first topical gene therapy being developed for DEB and phase I and phase II clinical trials have been completed. B-VEC has been given to both pediatric and adult participants. There were no drug-related safety events reported during or following these clinical trials and it was confirmed that the full-length collagen VII protein is detected following administration. The intent of this study is to further evaluate the safety of B-VEC and determine whether administration improves wound healing as compared to placebo.


  • Ages 6 months and above
  • Clinical diagnosis of Dystrophic Epidermolysis Bullosa including both dominant and recessive forms of DEB
  • Two wounds that are similar in size and location
  • Have not participated in another gene therapy trial in the past 3 months
  • Not currently diagnosed with squamous cell carcinoma or currently receiving chemotherapy or immunotherapy
  • Not pregnant or breastfeeding


The study involves traveling to the research site for 1 visit each week for 24 weeks in a row. Study procedures include a physical exam, vital signs, questionnaire, images, providing urine and blood samples, collection of how much investigational therapy remains on the skin, asking how you are feeling and what medications you are taking and topical investigational therapy application to wounds selected by the Study Doctor. There is also a required safety-follow up visit, 30 days after the last dose of the investigational therapy. At this time, participants will be asked to enroll into a long-term follow up period of an additional five (5) years, with telephone or e-mail contact at least once a year.

Additional details about the study can be found at this link:

B-VEC is not FDA approved for the purposes of this study.


Participants will receive a standard meal reimbursement for themselves and a companion, if applicable, for days they are traveling to the site and for each day of an on-site study visit. Travel expenses including airfare, ride service, hotel, and/or mileage/parking will be arranged or reimbursed for participants and their companion, as applicable.


Dr. Peter Marinkovich's talk about the results of Phase 1/2 trial (2020)