The Study Sponsor, Krystal Biotech, Inc. is conducting a Phase III research study to determine if B-VEC, a topical gene therapy, can effectively promote and maintain wound healing of DEB patient wounds, following repeat application and to evaluate the change from baseline in Patient Reported Outcomes.
Children, teens and adults with RDEB or DDEB are eligible for participation. Patients are enrolled upon obtaining consent and meeting entry criteria. Participants must have at least one pair of matching wounds that are similar in size and location. The wounds in a pair will be randomized, such than one wound in the pair will receive B-VEC and the other will receive placebo. Additional unmatched wounds may be selected to receive B-VEC. Subjects will be on-trial for approximately 6 months, with single weekly visits to the clinical site.
During the study the Investigator will complete physical exams, administer questionnaires, image and assess the study wounds, monitor your vitals and lab specimens as well as asking you how you are feeling. Additionally, they will monitor changes in your medications, as well as applying B-VEC and placebo gel to the study wounds once a week.
- Clinical diagnosis of the recessive or dominant form of dystrophic epidermolysis bullosa
- Age: 6 months or older at the time of consent
- At least two wounds that are similar in size and location
- 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 visits
- Please click the link below for exclusion criteria.