Neurotrophic KeratitisNCT04627571
Corneal Nerves After Treatment With Cenegermin
An observational study of Neurotrophic Keratitis, run by University of California, Los Angeles at 1 site in United States.
Trial summary
Neurotrophic keratopathy (NK) is a condition in which patients have fewer or complete absence of nerves in the cornea, characterized by the reduced or absent corneal sensation. The lack of nerves in the cornea also result in damages of the cornea and in severe situation the loss of the eye. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Neurotrophic Keratitis
- What is being tested?
- Cenegermin Ophthalmic Solution [Oxervate]
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 23 May 2022to 25 Mar 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients who are 18 years of age or older, who have a persistent corneal epitheliopathy due to neurotrophic keratopathy that is refractory to conventional non-surgical treatments for 2 weeks or more, and who fulfill the inclusion and exclusion criteria detailed above.
Who may be able to join
- Patients ≥18 years of age; AND
- Persistent corneal epitheliopathy that is refractory to treatments for ≥2 weeks; AND
- Evidence of decreased corneal sensitivity, defined as ≤45mm on Cochet-Bonnet esthesiometer, within the area of the persistent corneal epitheliopathy; AND
- Evidence of decreased corneal sensitivity, defined as above, in ≥1 corneal quadrant outside the persistent corneal epithelial defect.
Who cannot join
- Patients with severe neurotrophic keratopathy characterized by corneal stromal ulceration involving over 75% of the total central corneal thickness and impending perforation.
- Patients who have used cenegermin eyedrops, autologous serum eyedrops, plasma-rich plasma eyedrops, or umbilical cord eyedrops for the treatment of neurotrophic keratopathy 8 weeks prior to study recruitment.
- Corneal surgery (including keratoplasty or refractive surgical procedures) within three months before study enrollment.
- Presence of concurrent bacterial or fungal infection.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, Los Angeles
Los Angeles, California, 90095
About this study
Official title: Structural and Functional Changes of Corneal Innervation After Treatment With Cenegermin
Neurotrophic keratopathy (NK) is a condition in which patients have fewer or complete absence of nerves in the cornea, characterized by the reduced or absent corneal sensation. The lack of nerves in the cornea also result in damages of the cornea and in severe situation the loss of the eye.
Cenegermin (trade name Oxervate) is a nerve growth factor eye drops designed to treat NK, and currently, it is the only FDA-approved medication for this purpose.
Read more
Even though cenegermin is effective in the majority of patients, there is a lack of understanding of how cenegermin works in the eye.
In this study, investigators aim to determine the structural and functional effects of cenegermin on the cornea, using non-invasive technologies including in vivo confocal microscopy on study participants with NK over the course of a year.
Read the detailed description
Cenegermin (OxervateTM) is a recombinant human form of nerve growth factor developed by Dompé Farmaceutici S.p.A. for patients who are diagnosed with corneal epithelial defects due to moderate to severe stages of NK.
- Conditions
- Neurotrophic Keratitis
- Being tested
- Cenegermin Ophthalmic Solution [Oxervate]
- Keywords
- cenegermin, neurotrophic keratopathy, corneal nerve, corneal sensation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cenegermin Ophthalmic Solution [Oxervate]Drug
An 8-week course of topical cenegermin eye drops given 6 times daily would be started in the affected eye, in conjunction with the pre-existing medical treatment for neurotrophic keratopathy. Patients will be followed-up at 4 weeks, 6 weeks, 8 weeks, 3 months, 6 months, 9 months and 12 months (±2 weeks at each time point) after treatment. At each visit, patients will be assessed to determine the corneal epithelial defect and corneal sensation.
| Study group | Role | What they receive |
|---|---|---|
| Patient Population | Patients diagnosed with neurotrophic keratopathy. Receives: Drug: Cenegermin Ophthalmic Solution [Oxervate] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in corneal nerve fiber density (CNFD)
To determine the changes in corneal nerve fiber density (CNFD), assessed by in vivo confocal microscopy.
Measured: 1 year
Secondary outcomes (1)
Duration of changes in CNFD
To determine the duration of the changes in corneal nerve fiber density (CNFD), assessed by in vivo confocal microscopy.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2020
- Study start23 May 2022
- Primary completion25 Mar 2025
- Study completion25 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Neurotrophic KeratitisStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Collaborators
- Dompé Farmaceutici S.p.A
- Responsible party
- Principal investigator, Simon Fung, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04627571
- Sponsor's protocol ID
- 20-000833
- Status last verified
- Mar 2025
- First submitted
- 8 Nov 2020
- Last updated
- 1 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Sacchetti M, Lambiase A. Diagnosis and management of neurotrophic keratitis. Clin Ophthalmol. 2014 Mar 19;8:571-9. doi: 10.2147/OPTH.S45921. eCollection 2014.
PubMed 24672223Bonini S, Lambiase A, Rama P, Caprioglio G, Aloe L. Topical treatment with nerve growth factor for neurotrophic keratitis. Ophthalmology. 2000 Jul;107(7):1347-51; discussion 1351-2. doi: 10.1016/s0161-6420(00)00163-9.
PubMed 10889110Lambiase A, Sacchetti M, Bonini S. Nerve growth factor therapy for corneal disease. Curr Opin Ophthalmol. 2012 Jul;23(4):296-302. doi: 10.1097/ICU.0b013e3283543b61.
PubMed 22543481Bonini S, Lambiase A, Rama P, Filatori I, Allegretti M, Chao W, Mantelli F; REPARO Study Group. Phase I Trial of Recombinant Human Nerve Growth Factor for Neurotrophic Keratitis. Ophthalmology. 2018 Sep;125(9):1468-1471. doi: 10.1016/j.ophtha.2018.03.004. Epub 2018 Apr 10. No abstract available.
PubMed 29653861Ting DSJ. Re: Bonini et al.: Phase 2 randomized, double-masked, vehicle-controlled trial of recombinant human nerve growth factor for neurotrophic keratitis (Ophthalmology. 2018;125:1332-1343). Ophthalmology. 2019 Feb;126(2):e14-e15. doi: 10.1016/j.ophtha.2018.09.017. No abstract available.
PubMed 30683186Pflugfelder SC, Massaro-Giordano M, Perez VL, Hamrah P, Deng SX, Espandar L, Foster CS, Affeldt J, Seedor JA, Afshari NA, Chao W, Allegretti M, Mantelli F, Dana R. Topical Recombinant Human Nerve Growth Factor (Cenegermin) for Neurotrophic Keratopathy: A Multicenter Randomized Vehicle-Controlled Pivotal Trial. Ophthalmology. 2020 Jan;127(1):14-26. doi: 10.1016/j.ophtha.2019.08.020. Epub 2019 Aug 26.
PubMed 31585826Mastropasqua L, Lanzini M, Dua HS, D' Uffizi A, Di Nicola M, Calienno R, Bondi J, Said DG, Nubile M. In Vivo Evaluation of Corneal Nerves and Epithelial Healing After Treatment With Recombinant Nerve Growth Factor for Neurotrophic Keratopathy. Am J Ophthalmol. 2020 Sep;217:278-286. doi: 10.1016/j.ajo.2020.04.036. Epub 2020 May 6.
PubMed 32387431
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Structural and Functional Changes of Corneal Innervation After Treatment With Cenegermin
- ClinicalTrials.gov ID
- NCT04627571
- Acronym / sponsor ID
- 20-000833
- Conditions
- Neurotrophic Keratitis
- Keywords
- cenegermin, neurotrophic keratopathy, corneal nerve, corneal sensation
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Collaborators
- Dompé Farmaceutici S.p.A
- Study start
- 23 May 2022 (actual)
- Primary completion
- 25 Mar 2025 (actual)
- Study completion
- 25 Mar 2025 (actual)
- First posted
- 13 Nov 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Neurotrophic keratopathy (NK) is a condition in which patients have fewer or complete absence of nerves in the cornea, characterized by the reduced or absent corneal sensation. The lack of nerves in the cornea also result in damages of the cornea and in severe situation the loss of the eye.
Cenegermin (trade name Oxervate) is a nerve growth factor eye drops designed to treat NK, and currently, it is the only FDA-approved medication for this purpose.
Even though cenegermin is effective in the majority of patients, there is a lack of understanding of how cenegermin works in the eye.
In this study, investigators aim to determine the structural and functional effects of cenegermin on the cornea, using non-invasive technologies including in vivo confocal microscopy on study participants with NK over the course of a year.
Detailed description
Cenegermin (OxervateTM) is a recombinant human form of nerve growth factor developed by Dompé Farmaceutici S.p.A. for patients who are diagnosed with corneal epithelial defects due to moderate to severe stages of NK.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patient Population | – | Drug: Cenegermin Ophthalmic Solution [Oxervate]Patients diagnosed with neurotrophic keratopathy. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients who are 18 years of age or older, who have a persistent corneal epitheliopathy due to neurotrophic keratopathy that is refractory to conventional non-surgical treatments for 2 weeks or more, and who fulfill the inclusion and exclusion criteria detailed above.
Key inclusion criteria (4 listed)
- Patients ≥18 years of age; AND
- Persistent corneal epitheliopathy that is refractory to treatments for ≥2 weeks; AND
- Evidence of decreased corneal sensitivity, defined as ≤45mm on Cochet-Bonnet esthesiometer, within the area of the persistent corneal epitheliopathy; AND
- Evidence of decreased corneal sensitivity, defined as above, in ≥1 corneal quadrant outside the persistent corneal epithelial defect.
Key exclusion criteria (4 listed)
- Patients with severe neurotrophic keratopathy characterized by corneal stromal ulceration involving over 75% of the total central corneal thickness and impending perforation.
- Patients who have used cenegermin eyedrops, autologous serum eyedrops, plasma-rich plasma eyedrops, or umbilical cord eyedrops for the treatment of neurotrophic keratopathy 8 weeks prior to study recruitment.
- Corneal surgery (including keratoplasty or refractive surgical procedures) within three months before study enrollment.
- Presence of concurrent bacterial or fungal infection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cenegermin Ophthalmic Solution [Oxervate]An 8-week course of topical cenegermin eye drops given 6 times daily would be started in the affected eye, in conjunction with the pre-existing medical treatment for neurotrophic keratopathy. Patients will be followed-up at 4 weeks, 6 weeks, 8 weeks, 3 months, 6 months, 9 months and 12 months (±2 weeks at each time point) after treatment. At each visit, patients will be assessed to determine the corneal epithelial defect and corneal sensation. | Drug | Patient Population |
Primary outcome
Change in corneal nerve fiber density (CNFD)
Time frame: 1 year
To determine the changes in corneal nerve fiber density (CNFD), assessed by in vivo confocal microscopy.
1 secondary outcome
Duration of changes in CNFD
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2025.
- Background: PubMed 24672223Sacchetti M, Lambiase A. Diagnosis and management of neurotrophic keratitis. Clin Ophthalmol. 2014 Mar 19;8:571-9. doi: 10.2147/OPTH.S45921. eCollection 2014.
- Background: PubMed 10889110Bonini S, Lambiase A, Rama P, Caprioglio G, Aloe L. Topical treatment with nerve growth factor for neurotrophic keratitis. Ophthalmology. 2000 Jul;107(7):1347-51; discussion 1351-2. doi: 10.1016/s0161-6420(00)00163-9.
- Background: PubMed 22543481Lambiase A, Sacchetti M, Bonini S. Nerve growth factor therapy for corneal disease. Curr Opin Ophthalmol. 2012 Jul;23(4):296-302. doi: 10.1097/ICU.0b013e3283543b61.
- Background: PubMed 29653861Bonini S, Lambiase A, Rama P, Filatori I, Allegretti M, Chao W, Mantelli F; REPARO Study Group. Phase I Trial of Recombinant Human Nerve Growth Factor for Neurotrophic Keratitis. Ophthalmology. 2018 Sep;125(9):1468-1471. doi: 10.1016/j.ophtha.2018.03.004. Epub 2018 Apr 10. No abstract available.
- Background: PubMed 30683186Ting DSJ. Re: Bonini et al.: Phase 2 randomized, double-masked, vehicle-controlled trial of recombinant human nerve growth factor for neurotrophic keratitis (Ophthalmology. 2018;125:1332-1343). Ophthalmology. 2019 Feb;126(2):e14-e15. doi: 10.1016/j.ophtha.2018.09.017. No abstract available.
- Background: PubMed 31585826Pflugfelder SC, Massaro-Giordano M, Perez VL, Hamrah P, Deng SX, Espandar L, Foster CS, Affeldt J, Seedor JA, Afshari NA, Chao W, Allegretti M, Mantelli F, Dana R. Topical Recombinant Human Nerve Growth Factor (Cenegermin) for Neurotrophic Keratopathy: A Multicenter Randomized Vehicle-Controlled Pivotal Trial. Ophthalmology. 2020 Jan;127(1):14-26. doi: 10.1016/j.ophtha.2019.08.020. Epub 2019 Aug 26.
- Background: PubMed 32387431Mastropasqua L, Lanzini M, Dua HS, D' Uffizi A, Di Nicola M, Calienno R, Bondi J, Said DG, Nubile M. In Vivo Evaluation of Corneal Nerves and Epithelial Healing After Treatment With Recombinant Nerve Growth Factor for Neurotrophic Keratopathy. Am J Ophthalmol. 2020 Sep;217:278-286. doi: 10.1016/j.ajo.2020.04.036. Epub 2020 May 6.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurotrophic Keratitis, run by University of California, Los Angeles at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in corneal nerve fiber density (CNFD) (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04627571, which also lists 7 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Apr 2025.