©2024 Stanford Medicine
A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
Recruiting
I'm InterestedTrial ID: NCT04917874
Purpose
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
Associate Professor of Dermatology
Jennifer Chen, MD
Clinical Professor, Dermatology
Eligibility
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
application
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)
Intervention(s):
biological: Open Label Topical Beremagene Geperpavec (B-VEC)
Recruiting
I'm InterestedContact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Sinem Bagci
650-484-6878