CompletedNCT04485546DEFENDOUpdated Nov 17, 2025Results posted

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

A Phase 4 interventional study of cenegermin-bkbj in Neurotrophic Keratitis, sponsored by Dompé Farmaceutici S.p.A. Completed at 5 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-11-17.

Sponsored by Dompé Farmaceutici S.p.A · Phase 4, Interventional, and Treatment

Phase: Phase 4
Study type: Interventional
Enrollment: 37
Allocation: Not applicable
Ages: 18 Years and older
Sex: All

Study summary

This study is to evaluate the safety and efficacy of OXERVATE® 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution in patients with Stage 1 neurotrophic keratitis (NK).

Read the detailed description

This is a 8-week, multicenter, open label, prospective study with 24 weeks of follow-up to evaluate safety and efficacy of OXERVATE® 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution in patients with Stage 1 neurotrophic keratitis (NK)

Conditions studied

Neurotrophic Keratitis

Browse trials for

Corneal Diseases 130

Keywords

Stage 1 Neurotrophic Keratitis
Corneal Disease
Ocular Disease

In context

Corneal Diseases

130 studies on the registry are indexed under Corneal Diseases; 43 are open to participants now.

This study's enrollment of 37 is above the median of 32 across 78 interventional studies indexed under Corneal Diseases.

Browse Corneal Diseases studies →

Lead sponsor

Dompé Farmaceutici S.p.A is the lead sponsor of 51 studies on the registry; 4 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 18 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Male or female aged ≥ 18 years.
Patients with Stage 1 NK defined by the Mackie criteria

Exclusion criteria

Exclusion Criteria:

Evidence of an active ocular infection (bacterial, viral, protozoal) in either eye.
Have current or history of conditions that may confound the study data including but not limited to Ocular Cicatricial Pemphigoid (OCP), Graft Versus Host Disease (GVHD), neuromyelitis optica, uncontrolled dry eye, and Steven Johnson's syndrome.
History of severe systemic allergy or severe ocular allergy (including seasonal conjunctivitis expected during the subject's participation in the trial) or chronic conjunctivitis and/or keratitis other than dry eye disease.
Patients with severe vision loss with no potential for visual improvement in the study eye, in the opinion of the investigator, or if the subject is deemed legally blind.
Ocular surgery or elective ocular surgery expected during participation in the trial.
Patients with eyelid abnormality that may alter eyelid function including but not limited to Blepharospasm, Cerebrovascular accident, entropion, ectropion, floppy lid syndrome.

Study design

Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 37 participants (actual)

Study arms

Experimental
OXERVATE® 0.002% (20 mcg/mL) cenegermin-bkbj
cenegermin-bkbj ophtalmic solution administered as one drop in affected eye(s) every 2 hours 6 times daily for 8 weeks
Drug: cenegermin-bkbj

Interventions

Drugcenegermin-bkbj
OXERVATE® 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution administered as one drop in the affected eye(s), 6 times a day at 2-hour intervals for 8 weeks, in patients with Stage 1 neurotrophic keratitis.
Also known as: OXERVATE® 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution

What researchers measure

Primary outcomes

Corneal Epithelial Healing
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.
Time frame: Week 8

Results

Posted Jun 23, 2023

Participant flow

Participant flow — Overall Study
Milestone	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
Started	37
Completed	29
Not completed	8

Outcome measures

PrimaryCorneal Epithelial Healing

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.

Time frame:: Week 8
Reported as:: Count of participants · Participants

Corneal Epithelial Healing
Participants	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
No	5
Yes	28

Adverse events

Collected over 34 Weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Study Eye	0/37 (0%)	1/37 (2.7%)	22/37 (59.5%)
OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Non Study Eye	0/37 (0%)	0/37 (0%)	13/37 (35.1%)
OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Systemic	1/37 (2.7%)	7/37 (18.9%)	16/37 (43.2%)

Most frequent serious events

Showing 10 of 12

Most frequent serious events
Event	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Study Eye	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Non Study Eye	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Systemic
Myocardial infarctionCardiac disorders	0/37	0/37	1/37
COVID-19Infections and infestations	0/37	0/37	1/37
SyncopeNervous system disorders	0/37	0/37	1/37
Hiatus HerniaGastrointestinal disorders	0/37	0/37	1/37
Incarcerated parastomal herniaInjury, poisoning and procedural complications	0/37	0/37	1/37
Pneumonia bacterialInfections and infestations	0/37	0/37	1/37
Deep Vein ThrombosisVascular disorders	0/37	0/37	1/37
Respiratory FailureRespiratory, thoracic and mediastinal disorders	0/37	0/37	1/37
IridocyclitisEye disorders	1/37	0/37	0/37
Intestinal HaemorrhageGastrointestinal disorders	0/37	0/37	1/37

Most frequent other events

Showing 10 of 61

Most frequent other events
Event	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Study Eye	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Non Study Eye	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj - Systemic
Eye PainEye disorders	14/37	2/37	0/37
Vision blurredEye disorders	7/37	3/37	0/37
Dry EyeEye disorders	4/37	2/37	0/37
Foreign body sensation in eyesEye disorders	4/37	0/37	0/37
Eye irritationEye disorders	3/37	2/37	0/37
Eyelid painEye disorders	3/37	2/37	0/37
Burning sensationNervous system disorders	3/37	1/37	0/37
Corneal DystrophyCongenital, familial and genetic disorders	2/37	1/37	0/37
Conjunctival hyperaemiaEye disorders	2/37	1/37	0/37
Corneal OedemaEye disorders	2/37	1/37	0/37

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
<=18 years	0
Between 18 and 65 years	19
>=65 years	18

Age, Continuous
Age, Continuous(years)	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
Mean	64.6 ± 11.89

Sex: Female, Male
Sex: Female, Male(Participants)	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
Female	28
Male	9

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
Hispanic or Latino	2
Not Hispanic or Latino	35
Unknown or Not Reported	0

Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
Race — White	32
Race — Black or African American	3
Race — Asian	1
Race — Other	1

Region of Enrollment
Region of Enrollment(participants)	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
United States	37

Treated Eyes
Treated Eyes(participants)	OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj
Participants with Treated Study Eye	37
Participants with Treated Non-Study Eye	15

Study locations

5 sites

San Diego
San Diego, California 92122, United States
Edgewood
Edgewood, Kentucky 41017, United States
Boston
Boston, Massachusetts 02111, United States
Saint Louis
St Louis, Missouri 63131, United States
Philadelphia
Philadelphia, Pennsylvania 19104, United States

References and documents

Publications

Mastropasqua L, Massaro-Giordano G, Nubile M, Sacchetti M. Understanding the Pathogenesis of Neurotrophic Keratitis: The Role of Corneal Nerves. J Cell Physiol. 2017 Apr;232(4):717-724. doi: 10.1002/jcp.25623. Epub 2016 Oct 17. PubMed 27683068 ↗
Hamrah P, Massaro-Giordano M, Schanzlin D, Holland E, Berdy G, Goisis G, Pasedis G, Mantelli F. Phase IV Multicenter, Prospective, Open-Label Clinical Trial of Cenegermin (rhNGF) for Stage 1 Neurotrophic Keratopathy (DEFENDO). Ophthalmol Ther. 2024 Feb;13(2):553-570. doi: 10.1007/s40123-023-00866-y. Epub 2024 Jan 4. PubMed 38175466 ↗

Study documents

Study protocol · May 4, 2021
Statistical analysis plan · May 20, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Nov 17, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT04485546
Lead sponsor: Dompé Farmaceutici S.p.A
Collaborators: Dompé, US, Inc.
Responsible party: Sponsor
First posted: Jul 24, 2020
Start date: Sep 9, 2020
Primary completion: Sep 30, 2021
Completion: Mar 30, 2022
Results posted: Jun 23, 2023
Last update: Nov 17, 2025

Study contacts

Flavio Mantelli, MD, PhD

study chair · Domp Farmaceutici SpA

Oversight

Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Nov 2025. You cannot join it, but the record below documents what was studied.

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