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Dystrophic Epidermolysis Bullosa - Healing Genes
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Dystrophic Epidermolysis Bullosa

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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)


Phase 2

DESCRIPTION:

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.


PATIENT MUST:

  • 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:

  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. Assignment of wounds to treat and medicine for each.
  3. Daily application of assigned drugs to each wound for 90 days.
  4. 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.

Contact:

  1. Peter Marinkovich, M.D. | 650-723-6316  |  [email protected]

SPONSOR INFORMATION:
Krystal Biotech, Inc.

Or go online:
https://clinicaltrials.gov/ct2/show/NCT03536143

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