Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB 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.

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Key information

Conditions

Squamous Cell Carcinoma Carcinoma Carcinoma, Squamous Cell Neoplasms Neoplasms by Histologic Type Neoplasms, Glandular and Epithelial Neoplasms, Squamous Cell

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Azienda Ospedliero-Universitaia, Bari, Italy

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

Inability to travel to site for study visit
Subject is pregnant
Subject has Metastatic SCC's or is on any current systemic treatment for SCC.

Treatment and study plan

BVEC

Drug

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

Primary outcomes

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

Study contacts

Contact information is provided by the study sponsor or research team.

Kunju Sridhar

CONTACT

[email protected]

650-721-4902

Sponsors and collaborators

Lead sponsor

Stanford University

Other

Collaborators

Epidermolysis Bullosa Research Partnership

Registry information

Official study title

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

Important dates

Study start: 2025
Primary completion: 2027
Study completion: 2027
First posted: Dec 13, 2024
Registry last updated: Apr 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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