BVEC: The study drug will be administered by home nurse at Subjects home for the participants residing in US. No drug will be applied when at study site. The subjects enrolled in Bari, Italy will not be administered BVEC.
Study summary
The study objective is to see if BVEC induced C7 expression in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin following Squamous cell carcinoma (SCC) excision will normalize the invasive tumor microenvironment and reduce tumor recurrence.
Prevention of SCC's in the RDEB subjects will increase their life span.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Diagnosis of generalized Recessive dystrophic epidermolysis bullosa (RDEB) demonstrated by COL7A1 mutations
2. Presence of biopsy proven squamous cell carcinoma (SCC)
3. Ability to understand and the willingness to provide written informed consent.
4. US based participants are willing to use beremagene geperpavec (BVEC)
5. Subject is 18 years or older
6. participant willingness to use an effective method of contraception
Exclusion Criteria:
1. Inability to travel to site for study visit
2. Subject is pregnant
3. Subject has Metastatic SCC's or is on any current systemic treatment for SCC.
Primary outcome measure(s)
Recurrence rate of Squamous Cell Carcinoma — 2 years The outcome measure is recurrence rate of SCC following BVEC application at each SCC lesion
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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