Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin

Stanford University

Status and phase: Enrolling; Phase 2

Conditions: Squamous Cell Carcinoma

Treatments: Drug: BVEC

Study type: Interventional

Funder types: Other

Identifiers

NCT06731933

IRB-73906

Details and patient eligibility

About

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.

Enrollment: 16 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Prevention

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

16 participants in 2 patient groups

Treatment with BVEC

Active Comparator group

Treatment:

Drug: BVEC

No treatment with BVEC

No Intervention group