NCT06731933

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
16

participants targeted

Target at below P25 for phase_2

Timeline
9mo left

Started Mar 2025

Geographic Reach
2 countries

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress60%
Mar 2025Feb 2027

First Submitted

Initial submission to the registry

December 11, 2024

Completed
2 days until next milestone

First Posted

Study publicly available on registry

December 13, 2024

Completed
4 months until next milestone

Study Start

First participant enrolled

March 28, 2025

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2027

Last Updated

April 20, 2026

Status Verified

April 1, 2026

Enrollment Period

1.8 years

First QC Date

December 11, 2024

Last Update Submit

April 15, 2026

Conditions

Squamous Cell Carcinoma

Keywords

gene therapy

Outcome Measures

Primary Outcomes (1)

  • Recurrence rate of Squamous Cell Carcinoma

    The outcome measure is recurrence rate of SCC following BVEC application at each SCC lesion

    2 years

Study Arms (2)

Treatment with BVEC

ACTIVE COMPARATOR
Drug: BVEC

No treatment with BVEC

NO INTERVENTION

Interventions

BVECDRUG

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.

Also known as: Beremagene Geperpavec
Treatment with BVEC

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Stanford University

Redwood City, California, 94063, United States

RECRUITING

Azienda Ospedliero-Universitaia

Bari, Italy

RECRUITING

MeSH Terms

Conditions

Carcinoma, Squamous Cell

Condition Hierarchy (Ancestors)

CarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeNeoplasmsNeoplasms, Squamous Cell

Study Officials

  • Peter Marinkovich, MD

    Stanford University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Kunju Sridhar

CONTACT

650-721-4902kunju@stanford.edu

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 11, 2024

First Posted

December 13, 2024

Study Start

March 28, 2025

Primary Completion (Estimated)

February 1, 2027

Study Completion (Estimated)

February 1, 2027

Last Updated

April 20, 2026

Record last verified: 2026-04

Locations

US(1)IT(1)