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

Trial ID or NCT#

NCT04917874

Status

recruiting iconRECRUITING

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

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Contact

Sinem Bagci
650-484-6878

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View on ClinicalTrials.gov