Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin

Impact of Collagen VII Gene Therapy on Squamous Cell Carcinoma Recurrence in Recessive Dystrophic Epidermolysis Bullosa Skin

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06731933

Enrollment

Registered

2024-12-13

Start date

2025-03-28

Completion date

2027-02-01

Last updated

2026-04-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Squamous Cell Carcinoma

Keywords

gene therapy

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.

Interventions

DRUGBVEC

The study drug will be administered by home nurse at Subjects home for the participants residing in US. No drug will be applied when at study site. The subjects enrolled in Bari, Italy will not be administered BVEC.

Study design

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

PREVENTION

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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.

Design outcomes

Primary

Measure	Time frame	Description
Recurrence rate of Squamous Cell Carcinoma	2 years	The outcome measure is recurrence rate of SCC following BVEC application at each SCC lesion

Countries

Italy, United States

Contacts

CONTACTKunju Sridhar

kunju@stanford.edu650-721-4902

PRINCIPAL_INVESTIGATORPeter Marinkovich, MD

Stanford University

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 21, 2026