ESCALON Trial Tests Corneal Endothelial Cell Therapy for Corneal Edema

Key Takeaways

• Pooled month 12 data showed improvement in central corneal thickness and best-corrected visual acuity after treatment.
• No serious treatment-emergent adverse events were reported in study eyes, and common ocular events were included in the pooled safety summary.
• Data were pooled after no clinically meaningful differences were observed among the 10, 20, and 100 μM Y-27632 groups.

At month 12, 88.9% of treated subjects achieved at least a 0.3 logMAR improvement in best-corrected visual acuity in the ESCALON randomized, double-masked trial. The study evaluated cultured human corneal endothelial cells plus Y-27632 for corneal edema secondary to endothelial dysfunction. No serious treatment-emergent adverse events were reported in study eyes during follow-up. Findings were pooled across the evaluated Y-27632 doses.

In this prospective, randomized, double-masked, parallel-group study, 22 eyes received cultured corneal endothelial cells plus Y-27632. Eligible eyes had bullous keratopathy in 18 cases and Fuchs dystrophy in 4 cases. Investigators performed endothelial polishing followed by a single intracameral injection containing 1 × 10^6 cultured corneal endothelial cells plus 10, 20, or 100 μM Y-27632. The primary endpoint was safety based on ocular and nonocular treatment-emergent adverse events, while efficacy focused on central corneal thickness and best-corrected visual acuity through month 12.

Efficacy was assessed as change from baseline in central corneal thickness and best-corrected visual acuity through month 12. Mean central corneal thickness changed from 697.0 μm at baseline to 571.2 μm at month 12. Mean best-corrected visual acuity changed from 0.995 logMAR at baseline to 0.330 logMAR at month 12. These findings were pooled after investigators found no clinically meaningful differences between dose groups.

Safety reporting focused on the prespecified incidence and severity of ocular and nonocular treatment-emergent adverse events. Study eyes experienced no serious treatment-emergent adverse events. Common study-eye adverse events were posterior capsule opacification in 72.7%, pupillary disorders or dyscoria in 27.3%, and transient intraocular pressure elevations in 22.7%. The 12-month safety findings were pooled across Y-27632 groups.

The authors described cultured corneal endothelial cell transplantation with Y-27632 as safe and efficacious across the evaluated doses. They also characterized the approach as a promising option for corneal edema arising from corneal endothelial dysfunction. Follow-up extended through month 12, the final time point presented. The conclusion was limited to the 22 treated eyes and the pooled 12-month findings.