Nerve Growth Factor for the Treatment of Cornea Disease

Observational Study of Cenegermin-bkbj in the Treatment of Limbal Stem Cell Deficiency Associated With Neurotrophic Keratopathy

To determine the efficacy and safety of nerve growth factor in the treatment of limbal stem cell deficiency (LSCD) associated with neurotrophic cornea.

Study Overview

• Status: Completed
• Conditions: Neurotrophic Keratitis
• Intervention / Treatment: Drug: Cenegermin-Bkbj

Detailed Description

This is a prospective open-label pilot study evaluating the role of cenegermin-bkbj for the treatment of LSCD with associated neurotrophic keratopathy. Patient diagnosed with LSCD with neurotrophic cornea who have failed conventional treatment such as artificial tears are enrolled in the study after a 2 week washout period. Patient are then treated with cenegermin-bkbj over an 8 week period.

• Study Type: Observational
• Enrollment (Actual): 5

Contacts and Locations

Study Locations

• United States
  • California
    • Stanford, California, United States, 94305
      • Stanford University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Adults. Both sexes will be recruited. Any ethnic background, as long as they meet the inclusion criteria.

Description

Inclusion Criteria:

Patients diagnosed with LSCD and neurotrophic cornea confirmed by testing with Cochet-Bonnet aesthesiometer

Exclusion Criteria:

1. Active ocular infection
2. Anticipated need for bandage contact lens, amniotic membrane graft, or tarsorrhaphy during the study period
3. Unable to discontinue the use of contact lens
4. Visual acuity worse than 20/200 in the better eye

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Size of abnormal epithelium	Change in size of abnormal epithelium compared to baseline	Baseline, week 8

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of patients with resolution of epithelial defect if present prior to treatment	Number of patients with resolution of epithelial defect if present prior to treatment (Stage 2) Mackie Classification of neurotrophic cornea at time of enrollment.	Baseline, week 8
Cornea sensation	Cornea sensation measured with Cochet-Bonnet aesthesiometer after treatment compared to screening visit.	Baseline, week 8

Collaborators and Investigators

• Sponsor: Stanford University
• Investigators: Principal Investigator: Christopher N Ta, MD, Stanford University

Publications and helpful links

General Publications

• Arboleda A, Ta CN. Observational study of cenegermin for the treatment of limbal stem cell deficiency associated with neurotrophic keratopathy. Ther Adv Ophthalmol. 2022 Nov 4;14:25158414221134598. doi: 10.1177/25158414221134598. eCollection 2022 Jan-Dec.

Study record dates

Study Major Dates

• Study Start (Actual): October 14, 2020
• Primary Completion (Actual): May 4, 2021
• Study Completion (Actual): May 4, 2021

Study Registration Dates

• First Submitted: September 11, 2020
• First Submitted That Met QC Criteria: September 11, 2020
• First Posted (Actual): September 17, 2020

Study Record Updates

• Last Update Posted (Actual): December 14, 2021
• Last Update Submitted That Met QC Criteria: December 10, 2021
• Last Verified: December 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Corneal Diseases; Keratitis
• Other Study ID Numbers: 57888

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No