Topical Bercolagene Telserpavec (KB103) Gene Therapy to Restore Functional Collagen VII for the Treatment of Dystrophic Epidermolysis Bullosa (GEM-1)
A Phase II Study of Bercolagene Telserpavec (KB103), a Non-Integrating, Replication-Incompetent Herpes Simplex Virus 1 (HSV-1) Vector Expressing the Human Collagen VII (COL7) Protein, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
Doctors at Stanford University seek participants in a clinical investigation of a new topical gene therapy for Dystrophic Epidermolysis Bullosa (DEB). This investigatory drug is a deactivated form of the herpes simplex virus that is not able to reproduce, but is able to transfer a gene to the skin cells it contacts. It is genetically engineered to transfer a gene that will hopefully help DEB wounds to heal.
Medicine will be applied daily to 3 wounds per patient, but one wound will receive a placebo drug, over a 90 day period.. Throughout the study, wounds will be imaged and biopsied to evaluate effectiveness and safety. Follow up includes 3 months of on-site visits followed by a 3-month at-home imaging period.
- Be 5 years of age or older
- Have at least one wound that is between 10 and 20 cm2 in wound area.
- Not have current evidence or a history of squamous cell carcinoma in the area that will undergo treatment
THE STUDY INVOLVES:
- Prescreening tests to confirm eligibility of the patient to participate.
- Assignment of wounds to treat and medicine for each.
- Daily application of assigned drugs to each wound for 90 days.
- Throughout the study, wounds will be imaged and biopsied to evaluate effectiveness and safety. Follow up includes 3 months of on-site visits followed by a 3-month at-home imaging period.
LOCATIONS AND CONTACTS:
The study site is at Stanford University in Stanford, CA. Map.
- Peter Marinkovich, M.D. | 650-723-6316 | [email protected]
Krystal Biotech, Inc.
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