An efficacy and safety study of cenegermin ophthalmic solution vs. vehicle in the treatment of PCED
Research type
Research Study
Full title
Phase 3, Multicenter, Randomized, Double-Masked, Vehicle-Controlled, Parallel Group Study to Evaluate the Safety and Efficacy of Recombinant Human Nerve Growth Factor Eye Drop Solution in Participants With Persistent Corneal Epithelial Defect (PCED)
IRAS ID
1012938
Contact name
Ahmed Enayetallah
Contact email
Sponsor organisation
Dompé Farmaceutici S.p.A
Research summary
Persistent Corneal Epithelial Defect (PCED) is a rare eye condition where the clear outer layer of the eye's surface (cornea) fails to heal properly for an extended period of time. Symptoms include impaired vision, eye discomfort, and pain. Without proper treatment, PCED can lead to serious complications like eye infections, corneal melting, perforation, and scarring, potentially resulting in vision loss.
Current treatments for PCED, such as surgery, contact lenses, anti-inflammatory drugs, lubricants, and other therapies are often not sufficient to promote corneal healing fully or are burdensome treatments. Thus, there is a need for more effective therapies.
This phase 3 study aims to evaluate whether cenegermin eye drops are safe and effective in treating PCED, as compared to vehicle (placebo) eye drops. Cenegermin is already approved in the EU and US for treating neurotrophic keratitis (another rare eye condition) but is not currently approved anywhere for the treatment of PCED.
The study has three periods: an initial 8-week treatment period (where participants receive either cenegermin or vehicle eye drops 6 times daily), followed by an 8-week extension treatment period (where treatment with cenegermin or vehicle eye drops 6 times daily is assigned based on healing response), and a 24-week follow-up period (where everyone receives artificial tears 4 times daily).
The study will include approximately 150 participants across approximately 83 study sites globally.
This study is sponsored by Dompé Farmaceutici S.p.A., an Italian pharmaceutical company.
REC name
South Central - Hampshire A Research Ethics Committee
REC reference
26/SC/0011
Date of REC Opinion
30 Jan 2026
REC opinion
Further Information Favourable Opinion