NCT04485546

Brief 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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
37

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Sep 2020

Geographic Reach
1 country

5 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 21, 2020

Completed
3 days until next milestone

First Posted

Study publicly available on registry

July 24, 2020

Completed
2 months until next milestone

Study Start

First participant enrolled

September 9, 2020

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2021

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 30, 2022

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

June 23, 2023

Completed
Last Updated

November 17, 2025

Status Verified

November 1, 2025

Enrollment Period

1.1 years

First QC Date

July 21, 2020

Results QC Date

May 22, 2023

Last Update Submit

November 10, 2025

Conditions

Neurotrophic Keratitis

Keywords

Stage 1 Neurotrophic KeratitisCorneal DiseaseOcular Disease

Outcome Measures

Primary Outcomes (1)

  • 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.

    Week 8

Study Arms (1)

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

EXPERIMENTAL

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

cenegermin-bkbjDRUG

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

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

San Diego

San Diego, California, 92122, United States

Location

Edgewood

Edgewood, Kentucky, 41017, United States

Location

Boston

Boston, Massachusetts, 02111, United States

Location

Saint Louis

St Louis, Missouri, 63131, United States

Location

Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (2)

  • 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.

    PMID: 27683068BACKGROUND

  • 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.

    PMID: 38175466DERIVED

MeSH Terms

Conditions

Corneal Diseases

Interventions

cenegermin

Condition Hierarchy (Ancestors)

Eye Diseases

Results Point of Contact

Title
Sarah Wilting, OD MS
Organization
Dompé Farmaceutici S.p.A
usmedinfo@dompe.com
833-366-7387

Study Officials

  • Flavio Mantelli, MD, PhD

    Domp Farmaceutici SpA

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 21, 2020

First Posted

July 24, 2020

Study Start

September 9, 2020

Primary Completion

September 30, 2021

Study Completion

March 30, 2022

Last Updated

November 17, 2025

Results First Posted

June 23, 2023

Record last verified: 2025-11

Data Sharing

IPD Sharing
Will not share

Locations

US(5)