Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin

NCT ID: NCT06731933

Last Updated: 2025-04-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-03-28

Study Completion Date

2027-02-28

Brief Summary

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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.

Detailed Description

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Conditions

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Squamous Cell Carcinoma

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

Study Groups

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Treatment with BVEC

Group Type ACTIVE_COMPARATOR

BVEC

Intervention Type 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.

No treatment with BVEC

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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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.

Intervention Type DRUG

Other Intervention Names

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Beremagene Geperpavec

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Epidermolysis Bullosa Research Partnership

OTHER_GOV

Sponsor Role collaborator

Stanford University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Peter Marinkovich, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

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Stanford University

Redwood City, California, United States

Site Status RECRUITING

Azienda Ospedliero-Universitaia

Bari, , Italy

Site Status RECRUITING

Countries

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United States Italy

Central Contacts

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Kunju Sridhar

Role: CONTACT

650-721-4902

Facility Contacts

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Kunju Sridhar

Role: primary

650-721-4902

Lucia Lospalluti, MD

Role: primary

080-5592442

Other Identifiers

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IRB-73906

Identifier Type: -

Identifier Source: org_study_id

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