Recruiting
Phase 2

COL7A1 Gene Therapy

Sponsor:

Stanford University

Code:

NCT06731933

Conditions

Squamous Cell Carcinoma

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

BVEC

Study Details

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.

Conditions

Squamous Cell Carcinoma

Study ID

NCT06731933

Start date

Mar 28, 2025

Status verified date

Apr, 2026

Completion date

Feb, 2027

Anticipated

Primary completion date

Feb, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

16 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Prevention

Interventions and Outcome Measures

Arms

active comparator: Treatment with BVEC

no intervention: No treatment with BVEC

Interventions

BVEC

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.

Primary outcome measure

  • Recurrence rate of Squamous Cell Carcinoma [ Time Frame: 2 years ]

Central Contacts and Locations

Central contacts

Kunju Sridhar

650-721-4902kunju@stanford.edu

Locations

Stanford University

Recruiting

Redwood City, California, United States, 94063

Contacts

Kunju Sridhar

650-721-4902kunju@stanford.edu

Principal Investigator:

Peter M Marinkovich, MD

More Information

Sponsor

Stanford University

Last update posted

Apr 20, 2026

Last verified

Apr, 2026

Keywords

  • gene therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Stanford University on 2026-04-20.