Topical Recombinant Human Nerve Growth Factor (Cenegermin) for Neurotrophic Keratopathy: A Multicenter Randomized Vehicle-Controlled Pivotal Trial


Stephen C Pflugfelder 1, Mina Massaro-Giordano 2, Victor L Perez 3, Pedram Hamrah 4, Sophie X Deng 5, Ladan Espandar 6, C Stephen Foster 7, John Affeldt 8, John A Seedor 9, Natalie A Afshari 10, Wendy Chao 11, Marcello Allegretti 11, Flavio Mantelli 11, Reza Dana

Main idea: Cenegermin treatment showed higher rates of corneal healing than vehicle in neurotrophic keratopathy associated with nonhealing corneal defects.


Purpose: To evaluate the efficacy and safety of topical cenegermin (recombinant human nerve growth factor) in patients with neurotrophic keratopathy.

Design: Multicenter, randomized, double-masked, vehicle-controlled trial.

Participants: Patients with neurotrophic persistent epithelial defect with or without stromal thinning.

Methods: The NGF0214 trial, conducted among 11 sites in the United States, randomized 48 patients to cenegermin or vehicle eye drops, 6 drops daily for 8 weeks of masked treatment. Follow-up was 24 weeks. Safety was assessed in all patients who received study drugs. Efficacy was assessed by intention to treat.

Main outcome measures: The primary endpoint was healing of the neurotrophic lesion (persistent epithelial defect or corneal ulcer) after 8 weeks of masked treatment. Masked central readers measured neurotrophic lesions in randomized clinical pictures, then assessed healing status conventionally and conservatively. Secondary variables included corneal healing at 4 weeks of masked treatment (key secondary end point), overall changes in lesion size, rates of disease progression, and changes in visual acuity and corneal sensitivity from baseline to week 8.

Results: Conventional assessment of corneal healing showed statistically significant differences at week 8. Compared to vehicle, cenegermin-treated patients showed significant reductions in lesion size and disease progression rates during masked treatment. Cenegermine was well tolerated; adverse effects were mostly local, mild, and transient. 


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