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

Study Overview
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Brief Summary
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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
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Open Label Treatment of Beremagene Geperpavec (B-VEC)

Conditions
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Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa

Intervention / Treatment
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* Biological: Open Label Topical Beremagene Geperpavec (B-VEC)


Participation Criteria
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Eligibility Criteria
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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)

Ages Eligible for Study
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Minimum Age: 2 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: B-VEC<br>Open label B-VEC topical treatment of DEB wounds. | Biological: Open Label Topical Beremagene Geperpavec (B-VEC)<br>* Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To record safety outcomes of participants while on B-VEC. | Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial. | Up to 112 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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DEB, Krystal Biotech