Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: BVEC.
- Who it may be relevant to
- Registry conditions: Squamous Cell Carcinoma. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Impact of Collagen VII Gene Therapy on Squamous Cell Carcinoma Recurrence in Recessive Dystrophic Epidermolysis Bullosa Skin
Overview
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
- 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.
Primary outcome measures
- Recurrence rate of Squamous Cell Carcinoma [Time frame: 2 years]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Prevention
Study locations
United States · 1 center
- Stanford University — Redwood City
Italy · 1 center
- Azienda Ospedliero-Universitaia — Bari
Identifiers
NCT: NCT06731933 · IRB-73906 · NCI-2025-09174