Corneal Nerves After Treatment With Cenegermin

NCT04627571 | Completed FDA Drug

• 1 other identifier: 20-000833
• Study Type: observational
• Target: 8
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Neurotrophic Keratitis

Keywords

cenegermin; neurotrophic keratopathy; corneal nerve; corneal sensation

Outcome Measures

Primary Outcomes (1)

• Change in corneal nerve fiber density (CNFD)

  To determine the changes in corneal nerve fiber density (CNFD), assessed by in vivo confocal microscopy.

  1 year

Secondary Outcomes (1)

• Duration of changes in CNFD

  1 year

Other Outcomes (3)

• Presence/absence of Changes in Dry Eye Symptom

  1 year

• Changes in Tear Production

  1 year

• Changes in Tear Characteristics

  1 year

Study Arms (1)

Patient Population

Patients diagnosed with neurotrophic keratopathy.

Drug: Cenegermin Ophthalmic Solution [Oxervate]

Interventions

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.

Patient Population

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of California, Los Angeles: lead
• Dompé Farmaceutici S.p.A: collaborator

Study Sites (1)

University of California, Los Angeles

Los Angeles, California, 90095, United States

Location

Related Publications (7)

• 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.

  PMID: 24672223 BACKGROUND

• Bonini 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.

  PMID: 10889110 BACKGROUND

• Lambiase 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.

  PMID: 22543481 BACKGROUND

• Bonini 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.

  PMID: 29653861 BACKGROUND

• Ting 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.

  PMID: 30683186 BACKGROUND

• Pflugfelder 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.

  PMID: 31585826 BACKGROUND

• Mastropasqua 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.

  PMID: 32387431 BACKGROUND

Related Links

• Related Info

MeSH Terms

Interventions

cenegermin

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 8, 2020
• First Posted: November 13, 2020
• Study Start: May 23, 2022
• Primary Completion: March 25, 2025
• Study Completion: March 25, 2025
• Last Updated: April 1, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)