A Phase 3 Double-Blind Study Comparing KB803 Eye Treatment to Placebo for Corneal Abrasions in Patients With Dystrophic Epidermolysis Bullosa

What this Trial Is About

Researchers are evaluating KB803, an ophthalmic suspension containing a modified herpes simplex virus expressing human collagen VII protein, in patients with dystrophic epidermolysis bullosa DEB who experience recurrent corneal abrasions. This Phase 3, double-blind, randomized, placebo-controlled crossover study aims to assess the safety and effectiveness of KB803 compared to a matched placebo in both children and adults with DEB. The study is sponsored by Krystal Biotech, Inc. Participants who have been in the Sponsors natural history study for at least 12 weeks and meet other criteria will be randomly assigned to receive either KB803 or placebo eye drops three times a week for the first 12 weeks. Then, they will switch to the alternate treatment for another 12 weeks. The eye treatments are given at home by a trained person, with monitoring by the study team. During the 24-week study, researchers will monitor safety and tolerability, track the frequency of corneal abrasion symptoms, and assess eye pain. Participants or their guardians will keep weekly symptom diaries and complete monthly questionnaires about symptoms and disease severity. Study staff will regularly check for any side effects or changes in treatment. The total participation lasts 24 weeks with ongoing safety and symptom evaluations.

A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa