Impact of Collagen VII Gene Therapy on Squamous Cell Carcinoma Recurrence in Recessive Dystrophic Epidermolysis Bullosa Skin
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• 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
Locations
United States
California
Stanford University
RECRUITING
Redwood City
Other Locations
Italy
Azienda Ospedliero-Universitaia
RECRUITING
Bari
Contact Information
Primary
Kunju Sridhar
kunju@stanford.edu
650-721-4902
Time Frame
Start Date: 2025-03-28
Estimated Completion Date: 2027-02
Participants
Target number of participants: 16
Treatments
Active_comparator: Treatment with BVEC
No_intervention: No treatment with BVEC
Related Therapeutic Areas
Sponsors
Collaborators: Epidermolysis Bullosa Research Partnership
Leads: Stanford University