Actively Recruiting
Impact of Collagen VII Gene Therapy on Squamous Cell Carcinoma Recurrence in Recessive Dystrophic Epidermolysis Bullosa Skin
Led by Stanford University · Updated on 2026-04-20
16
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
Sponsors
S
Stanford University
Lead Sponsor
E
Epidermolysis Bullosa Research Partnership
Collaborating Sponsor
AI-Summary
What this Trial Is About
This research aims to evaluate whether BVEC gene therapy can reduce the recurrence of squamous cell carcinoma (SCC) in skin affected by Recessive Dystrophic Epidermolysis Bullosa (RDEB). The study focuses on restoring collagen VII expression after SCC removal to normalize the tumor environment. Preventing SCC in RDEB patients may help improve their life expectancy. Participants will be assigned either to receive BVEC gene therapy or no treatment. The BVEC drug is given by a home nurse to participants living in the US, while those enrolled in Bari, Italy will not receive the treatment. This is a Phase 2, non-randomized study without masking. During the study, participants will be monitored for up to two years to assess the rate of SCC recurrence. Researchers will collect data on tumor return and overall treatment impact. Participants must provide informed consent and use contraception during the trial. The study is sponsored by Stanford University and is expected to run until February 2027.
CONDITIONS
Brief Title
Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of generalized Recessive Dystrophic Epidermolysis Bullosa (RDEB) confirmed by COL7A1 mutations
- Biopsy-proven squamous cell carcinoma (SCC) present
- Ability to understand and willingness to provide written informed consent
- US participants willing to use beremagene geperpavec (BVEC)
- Age 18 years or older
- Willingness to use an effective method of contraception
You will not qualify if you...
- Inability to travel to the study site for visits
- Pregnancy
- Presence of metastatic SCC or current systemic treatment for SCC
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 2 years
Participants receive BVEC gene therapy administered at home by a nurse if residing in the US; participants in Bari, Italy do not receive BVEC. This treatment aims to prevent recurrence of squamous cell carcinoma in RDEB skin.
Regular home nurse visits for drug administration and periodic study visits
Duration - Up to 2 years
Participants are monitored for recurrence of squamous cell carcinoma over a 2-year period after treatment initiation.
Periodic study visits during follow-up
Trial Site Locations
Total: 2 locations
1
Stanford University
Redwood City, California, United States, 94063
Actively Recruiting
2
Azienda Ospedliero-Universitaia
Bari, Italy
Actively Recruiting
Research Team
K
Kunju Sridhar
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
PREVENTION
Number of Arms
2
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