Nerve growth factor therapy in stage 2 and 3 neurotrophic keratopathy: A prospective, open-label clinical trial

Abstract

Background: Neurotrophic keratopathy (NK) is a rare degenerative corneal disease characterized by reduced corneal sensitivity and impaired epithelial healing, frequently resulting in persistent epithelial defects and corneal ulcers. Conventional therapies provide symptomatic relief but do not restore corneal innervation, highlighting a substantial unmet clinical need.

Objective: To evaluate the efficacy and safety of cenegermin, a recombinant human nerve growth factor, in patients with stage 2 and stage 3 NK.

Methods: This prospective, single-arm, open-label clinical trial enrolled eight patients who received cenegermin (20 mcg/mL) administered six times daily for 8 weeks, with a 48-week follow-up period. Corneal ulcer area, corneal sensitivity, and nerve parameters were assessed throughout the study.

Results: By week 8, 75% of patients achieved corneal healing, increasing to 100% by week 56. Ulcer area significantly decreased (-5.32 mm² at week 8), central corneal sensitivity improved by 22.5 mm, and main nerve density increased by 3.1 mm/mm² at week 8. The most frequently reported adverse events were mild eye pain, foreign body sensation, and tearing.

Conclusions: Cenegermin demonstrated substantial efficacy and a favorable safety profile in patients with advanced NK. It was effective, safe, and well tolerated, presenting a promising therapeutic option for NK.

Keywords

cenegermin / neurotrophic keratitis / neurotrophic keratopathy / recombinant human nerve growth factor / rhNGF

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