A Study Assessing KB803 Compared to Matching Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa

Launched by KRYSTAL BIOTECH, INC. · Jun 9, 2025

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ClinConnect Summary

This clinical trial is studying a treatment called B-VEC to see if it can help prevent and treat corneal abrasions, which are painful scratches on the eye's surface, in people with a condition called dystrophic epidermolysis bullosa (DEB). DEB is a rare genetic skin disorder that causes fragile skin and can lead to frequent eye injuries. The study is for both children and adults aged 6 months and older who have been diagnosed with DEB and experience these eye problems regularly. Participants must be able to follow the study rules and provide consent from themselves or their guardians.

If you take part in this trial, you will receive either the B-VEC treatment or a placebo (a substance with no active medicine) during different phases of the study. The study is designed to be double-blind, meaning neither you nor the researchers will know which treatment you are receiving at any given time. This helps ensure the results are fair and unbiased. Participants will be monitored closely to assess the safety and effectiveness of the treatment. It's important to note that there are specific requirements to join, such as not being pregnant or nursing and not having any recent changes in eye treatment. If you think you or someone you know might be eligible and interested, this trial could provide valuable insights into better managing eye health in DEB.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. The subject and/or their parent/legal guardian must provide informed consent/assent and must be able to and willing to follow study procedures and instructions.
• • 2. Age 6 months or older at time of informed consent/assent.
• • 3. Confirmed diagnosis of DEB with a mutation in the COL7A1 gene.
• • 4. Meets minimum corneal abrasion symptom frequency in the NHS study.
• Exclusion Criteria:
• • 1. Initiation of any new treatment regimen or change in treatment for ocular disease during the run-in period except for preservative free topical antibiotics or artificial tears/lubricants associated with standard of care treatment of corneal abrasions.
• • 2. Treatment with an investigational agent or off-label ophthalmic use of an approved product during the run-in period or planned use during the study (Exceptions may be approved by the medical monitor on a case-by-case basis).
• • 3. Any condition that, in the opinion of the Investigator, would impact the completion of all study-related assessments, interfere with the administration of study drug, and/or poses an additional risk to the subject.
• • 4. Women who are pregnant or nursing.
• • 5. Subject who is unwilling to comply with contraception requirements per protocol.
• • 6. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.