Date Updated
EB Type
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Name of Company
Krystal Biotech
Recruitment Status
Active, Not Recruiting
About the Study

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.
 

Trial Entry Criteria
  • Clinical diagnosis of the recessive form of dystrophic epidermolysis bullosa.
  • Age
    • 2 subjects: 18 years old or older,
    • 2 subjects: 5 years old or older.
  • At least one wound that is between 10 and 20 cm2 in wound area.
  • Subjects, who are, in the opinion of the Investigator, able to understand the study, co-operate with the study procedures and are willing to return to the clinic for all the required follow-up visit
  • Please click the link below for exclusion criteria.

Get more information on ClinicalTrials.gov