Krystal Biotech (KRYS) announced that the first patient has been dosed in its Phase 3 clinical trial, an intra-patient, double-blind, multicenter, placebo-controlled study with crossover design evaluating KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa patients. KB803 is designed to deliver two copies of the COL7A1 transgene to the corneal epithelium and enable local type VII collagen production in the front of the eye. IOLITE is an intra-patient, double-blind, placebo-controlled, multicenter Phase 3 study with a crossover design to evaluate KB803, administered as an eye drop, for the treatment and prevention of corneal abrasions in DEB patients, 6 months of age or older. Patients seeking to participate in IOLITE must first enroll in the ongoing natural history study and complete a 12-week run-in period, during which they report the number of days that they experience symptoms of corneal abrasions. Subjects meeting the inclusion criteria following the 12-week run-in are eligible to participate in the IOLITE trial. Approximately 16 subjects will be enrolled in the IOLITE study. The sample size was calculated based on the average symptomatic days per month and standard deviation data from subjects enrolled in the natural history study who would be otherwise eligible for IOLITE and provides 90% power to detect an effect size of at least 25%, allowing for a dropout rate up to 20%. Enrolled patients will initially receive either a single eye drop of either placebo or KB803, at a concentration of 109 PFU/mL, to each eye once weekly for 12 weeks. At the conclusion of the first 12 weeks, patients will be switched from placebo to KB803, or vice versa, and continue with once weekly administration for a second 12 week period. IOLITE is designed as a decentralized study and drug administration will occur at the subject’s home by a healthcare provider. The primary study endpoint will be the change in the average number of days per month with corneal abrasion symptoms while receiving KB803 versus placebo. Statistical significance will be analyzed via intra-patient paired measurements to account for potentially expected high inter-patient variability. Safety and secondary efficacy data, including weekly assessments of eye pain and monthly Epidermolysis Bullosa Eye Disease Index questionnaires, will be collected through to the end of the 24-week study period.
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