Escalón: 12-Month Safety And Efficacy Clinical Trial Of Human Corneal Endothelial Cell Therapy For Corneal Edema Secondary To Endothelial Dysfunction

To assess the safety and efficacy of a fixed dose of human corneal endothelial cells when administered with varying concentrations of Rho kinase inhibitor via intracameral injection in patients with corneal edema secondary to endothelial dysfunction.

The Escalon study was a single center, randomized, double-masked, parallel group, dose ranging study of Rho kinase inhibitor in 22 subjects with corneal edema in El Salvador. 

The study evaluated low, medium and high concentrations of Rho kinase inhibitor with 1 x 106 cells injected intracamerally in subjects with corneal edema secondary to endothelial disease. Human corneal endothelial cells and Rho kinase inhibitor are co-formulated as a suspension for injection into the eye via a single intracameral injection, in order to restore corneal endothelial function and ultimately improve vision. This study included multiple different surgeons and subjects were followed for one year. 

At the 12-month timepoint after injection of endothelial cells and Rho kinase inhibitor, mean improvement in best corrected visual acuity (BCVA) was 0.66 logMAR and mean improvement in central corneal thickness (CCT) was 131 microns across all three groups. 89% of subjects showed an improvement of 3 lines or more in BCVA. There were no systemic or study eye ocular SAE's. All three treatment groups showed clinically meaningful improvements in CCT and BCVA and no dose related safety or efficacy difference was noted between three different concentrations of Y-27632. 

Subjects treated with human corneal endothelial cells and Rho kinase inhibitor demonstrated biological activity as measured by BCVA and CCT. Therapy was well tolerated with a favorable safety profile at all concentrations of Rho kinase inhibitor. Results of the Escalon study are consistent with the previous three investigator-initiated studies in Japan and two sponsor-initiated studies in El Salvador involving multiple different surgeons. Collectively, these studies have evaluated different formulations of corneal endothelial cells with and without Rho kinase inhibitor in subjects with corneal edema secondary to corneal endothelial dysfunction. In total, 130 subjects have been treated with this therapy.