Trial Outcomes & Findings for Study to Evaluate OXERVATE® in Patients With Stage 1 Neurotrophic Keratitis (NCT NCT04485546)

Last Updated: 2025-11-10

Results Overview

Percentage of patients who experienced corneal epithelial healing. Corneal epithelial healing was defined by the central reading center as the absence of persistent epithelial staining abnormalities related to disease.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

37 participants

Primary outcome timeframe

Week 8

Results posted on

2025-11-10

Participant Flow

Participant milestones

Participant milestones
Measure	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj cenegermin-bkbj: Cenegermin-bkbj ophthalmic solution administered as one drop in affected eye(s) every 2 hours 6 times daily for 8 weeks
Overall Study STARTED	37
Overall Study COMPLETED	29
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study to Evaluate OXERVATE® in Patients With Stage 1 Neurotrophic Keratitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj n=37 Participants cenegermin-bkbj: Cenegermin-bkbj ophthalmic solution administered as one drop in affected eye(s) every 2 hours 6 times daily for 8 weeks
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	19 Participants n=5 Participants
Age, Categorical >=65 years	18 Participants n=5 Participants
Age, Continuous	64.6 years STANDARD_DEVIATION 11.89 • n=5 Participants
Sex: Female, Male Female	28 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	35 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	32 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African American	3 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian	1 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	1 Participants n=5 Participants
Region of Enrollment United States	37 participants n=5 Participants
Treated Eyes Study Eye	37 participants n=5 Participants
Treated Eyes Non-Study Eye	15 participants n=5 Participants

PRIMARY outcome

Timeframe: Week 8

Population: Full Analysis Set (FAS) - All enrolled patients who received at least one dose of the study drug. The FAS was used both for efficacy and safety analyses. Observed cases (n=33)

Outcome measures

Outcome measures
Measure	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj n=33 Participants cenegermin-bkbj: Cenegermin-bkbj ophthalmic solution administered as one drop in affected eye(s) every 2 hours 6 times daily for 8 weeks
Corneal Epithelial Healing Yes	28 Participants
Corneal Epithelial Healing No	5 Participants

Adverse Events

OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Study Eye

Serious events: 1 serious events

Other events: 22 other events

Deaths: 0 deaths

OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Non Study Eye

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Systemic

Serious events: 7 serious events

Other events: 16 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Study Eye n=37 participants at risk cenegermin-bkbj: Cenegermin-bkbj ophthalmic solution administered as one drop in affected eye(s) every 2 hours 6 times daily for 8 weeks - Study Eye	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Non Study Eye n=37 participants at risk cenegermin-bkbj: Cenegermin-bkbj ophthalmic solution administered as one drop in affected eye(s) every 2 hours 6 times daily for 8 weeks - Non Study Eye	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Systemic n=37 participants at risk enegermin-bkbj: Cenegermin-bkbj ophthalmic solution administered as one drop in affected eye(s) every 2 hours 6 times daily for 8 weeks - Systemic Conditions
Cardiac disorders Myocardial infarction	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Infections and infestations COVID-19	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Nervous system disorders Syncope	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Gastrointestinal disorders Hiatus Hernia	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Injury, poisoning and procedural complications Incarcerated parastomal hernia	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Infections and infestations Pneumonia bacterial	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Vascular disorders Deep Vein Thrombosis	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Eye disorders Iridocyclitis	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Gastrointestinal disorders Intestinal Haemorrhage	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Infections and infestations COVID-19 Pneumonia	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.
Infections and infestations Pneumonia	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	0.00% 0/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.	2.7% 1/37 • 34 Weeks An AE was defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product that did not necessarily have a causal relationship with this treatment. Safety evaluations were based on adverse event (AE) monitoring, intraocular pressure (IOP), slit lamp exam, and external eye exam.

Other adverse events

Additional Information

Sarah Wilting, OD MS

Dompé Farmaceutici S.p.A

Phone: 833-366-7387

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The agreement is between the Sponsor and the sites, and restricts all use of trial related content and results for the duration of the trial until contractual obligations are superseded. The Sponsor has sole rights to all trial related content and results, and all PIs must gain permission to utilize the results.
Publication restrictions are in place

Restriction type: OTHER