Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin

April 15, 2026 updated by: Stanford University

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

16

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Italy
      • Bari, Italy
        • Recruiting
        • Azienda Ospedliero-Universitaia
        • Contact:
        • Principal Investigator:
          • Lucia Lospalluti
  • United States
    • California
      • Redwood City, California, United States, 94063
        • Recruiting
        • Stanford University
        • Contact:
        • Principal Investigator:
          • Peter M Marinkovich, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Treatment with BVEC
Drug: 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.
Other Names:
  • Beremagene Geperpavec
No Intervention: No treatment with BVEC

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Stanford University

Collaborators

Epidermolysis Bullosa Research Partnership

Investigators

  • Principal Investigator: Peter Marinkovich, MD, Stanford University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 28, 2025

Primary Completion (Estimated)

February 1, 2027

Study Completion (Estimated)

February 1, 2027

Study Registration Dates

First Submitted

December 11, 2024

First Submitted That Met QC Criteria

December 11, 2024

First Posted (Actual)

December 13, 2024

Study Record Updates

Last Update Posted (Actual)

April 20, 2026

Last Update Submitted That Met QC Criteria

April 15, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IRB-73906
  • NCI-2025-09174 (Registry Identifier: National Cancer Institute Clinical Trials Reporting Program)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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