Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin
ConditionSquamous Cell Carcinoma
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorStanford University
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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)
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
BVEC
DrugThe 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.
Treatment groups
16 Participants
are divided into 2 treatment groupsGroup A: Treatment with BVECActive comparator 1 intervention
Group B: No treatment with BVECNo intervention 0 interventions
Trial outcomes
Primary outcomes
1
Recurrence rate of Squamous Cell Carcinoma
The outcome measure is recurrence rate of SCC following BVEC application at each SCC lesion
Time frame
2 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Stanford University
Lead sponsor
Epidermolysis Bullosa Research Partnership
Collaborator
Source ClinicalTrials.gov