Open Label Treatment of Beremagene Geperpavec (B-VEC)

B-VEC-EX-02

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.

2021-05-25

2023-07-31

2023-07-31

Completed

Early phase I

47

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)

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 47, 'type': 'ACTUAL'}}

2024-04-09