Children, Teens and Adults with Recessive Dystrophic Epidermolysis Bullosa are Needed for a Research Study of KB103, a Topical Gene Therapy

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Krystal Biotech is currently conducting a Phase II research study to evaluate the safety and effectiveness of an investigational topical gene therapy, KB103 (bercolagene telserpavec), for the treatment of people with Recessive Dystrophic Epidermolysis Bullosa, or RDEB.

This study is being conducted to determine if topical KB103 can safely and effectively promote healing of DEB patient wounds (primary endpoint) and to assess change from baseline in Investigator Global Assessments and Patient Reported Outcomes (secondary endpoint).

Children, teens and adults that have RDEB may be eligible for participation.  Patients are enrolled upon obtaining consent and meeting entry criteria. Three wounds are selected per subject; two will receive KB103 and one will receive placebo.  Throughout the study, wounds will imaged and biopsied for safety and efficacy analyses. Subjects will be on-trial for approximately 6 months: 3 months of on-site visits followed by a 3-month at-home imaging period.

For more information about the study please visit our link (KB103-001) or contact us by email at patientadvocacy [at] krystalbio [dot] com