Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin
Phase 2
Recruiting
- Conditions
- Squamous Cell Carcinoma
- Interventions
- Drug: BVEC
- Registration Number
- NCT06731933
- Lead Sponsor
- Stanford University
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 16
Inclusion Criteria
- Diagnosis of generalized Recessive dystrophic epidermolysis bullosa (RDEB) demonstrated by COL7A1 mutations
- Presence of biopsy proven squamous cell carcinoma (SCC)
- Ability to understand and the willingness to provide written informed consent.
- US based participants are willing to use beremagene geperpavec (BVEC)
- Subject is 18 years or older
- participant willingness to use an effective method of contraception
Exclusion Criteria
- Inability to travel to site for study visit
- Subject is pregnant
- Subject has Metastatic SCC's or is on any current systemic treatment for SCC.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Treatment with BVEC BVEC -
- Primary Outcome Measures
Name Time Method Recurrence rate of Squamous Cell Carcinoma 2 years The outcome measure is recurrence rate of SCC following BVEC application at each SCC lesion
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
How does BVEC-mediated COL7A1 expression modulate the SCC tumor microenvironment in RDEB patients?
What is the comparative efficacy of BVEC gene therapy versus standard SCC excision in RDEB recurrence prevention?
Which biomarkers correlate with COL7A1 gene therapy response in RDEB-associated SCC patients?
What are the potential off-target effects of BVEC-mediated collagen VII expression in RDEB skin?
Are there synergistic combination therapies with BVEC for enhanced SCC prevention in RDEB?
Trial Locations
- Locations (2)
Stanford University
🇺🇸Redwood City, California, United States
Azienda Ospedliero-Universitaia
🇮🇹Bari, Italy