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

Trial ID or NCT#

NCT04917874

Status

not recruiting iconNOT RECRUITING

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)

Eligibility Criteria

Ages Eligible for Study: Older than 2 Months
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. * 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:
  1. * 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)
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Investigator(s)

M. Peter Marinkovich, MD
M. Peter Marinkovich, MD
Dermatologist, Blistering disease specialist, Psoriasis specialist
Associate Professor of Dermatology
Jennifer Chen, MD
Jennifer Chen, MD
Dermatologist, General dermatologist
Clinical Professor, Dermatology

Contact us to find out if this trial is right for you.

Contact

Sinem Bagci
650-484-6878

View on ClinicalTrials.gov