BVEC Gene Therapy for Squamous Cell Carcinoma in RDEB
This study is looking at how a gene therapy called beremagene geperpavec (BVEC) might help people with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who have had squamous cell carcinoma (SCC) removed. RDEB is a genetic skin condition that makes skin very fragile. The study wants to see if BVEC can help reduce the chances of SCC coming back by improving the skin's environment. To join, you need to be at least 18 years old, have RDEB with specific gene changes (COL7A1 mutations), and have had a biopsy-confirmed SCC. If you live in the US, you must be willing to use BVEC. The main goal is to see how many people have SCC recurrence (cancer coming back) after two years. The study plans to enroll 16 participants, but its current status is unclear.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 16 participants.
- What's involved
- If you are in the US, the study drug, BVEC, will be given by a home nurse at your home. No drug will be applied when you are at the study site.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will measure the recurrence rate of Squamous Cell Carcinoma at 2 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin
At a glance
Conditions
Where it's being run
2 sites across 2 statesStudy leadership
- Peter Marinkovich, MD · PRINCIPAL_INVESTIGATOR · Stanford University
Who to contact
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What this trial measures
- Recurrence rate of Squamous Cell Carcinoma2 years
The outcome measure is recurrence rate of SCC following BVEC application at each SCC lesion