DEFENDO Long Term Follow-up Study in Stage 1 NK Patients

NCT05552261 | Completed Results FDA Drug

• 1 other identifier: NGF0122
• Study Type: observational
• Target: 24
• Locations: 1 country
• Sites: 4

Brief Summary

Primary Objective To evaluate the long-term safety and efficacy (healing) of OXERVATE® 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution in Stage 1 neurotrophic keratitis (NK) patients who enrolled in the DEFENDO study. Secondary Objective To evaluate the long-term efficacy of OXERVATE® 0.002% (20 mcg/mL) cenergemin-bkbj ophthalmic solution in terms of corneal sensitivity, Schirmer I test, tear film break-up time (TFBUT), best corrected distance visual acuity (BCDVA), and quality of life at 24 and 30 months post-treatment

Conditions

Neurotrophic Keratitis

Keywords

Stage 1 NK; OXERVATE®; Cenegermin; NK; DEFENDO; NGF0120 (original DEFENDO study); NGF0122 (DEFENDO long-term follow-up study)

Outcome Measures

Primary Outcomes (2)

• Number/Percentage of Patients Who Achieved Corneal Epithelial Healing at NGF0120 Week 8 (EoT NGF0120=Baseline NGF0122) and Remained Healed at Month 24 and Month 30 of the NGF0122 Study

  Corneal epithelial at Month 24 and Month 30 of the NGF0122 study was summarized as a binary goal attainment variable (Yes/No) based on the subset of patients who achieved corneal epithelial healing at Week 8 of the NGF0120, as determined by the Central Reading Center (CRC). Healing was defined as the absence of persistent epithelial staining abnormalities related to disease. The persistence (or worsening) of a staining pattern in the same corneal area was considered a failure in terms of healing.

  At Month 24 and Month 30 of the NGF0122 study

• Number/Percentage of Patients Who Did Not Achieve Corneal Epithelial Healing at NGF0120 Week 8 (EoT NGF0120=Baseline NGF0122) Who Had a Healed Corneal Epithelium at Month 24 and 30 of the NGF0122 Study.

  Corneal epithelial healing at the overall Follow-up Month 24 and Month 30 was summarized as a binary goal attainment variable (Yes/No) based on the subset of patients who did not achieve corneal epithelial healing at Week 8 of the NGF0120, as determined by the Central Reading Center (CRC). Healing was defined as the absence of persistent epithelial staining abnormalities related to disease. Epithelial healing took into consideration the Baseline epithelial staining patterns of each patient and their evolution over time.The persistence (or worsening) of a staining pattern in the same corneal area was considered a failure in terms of healing.

  At Month 24 and Month 30 of the NGF0122 study

Secondary Outcomes (11)

• Number/Percentage of Patients With an Improvement in Corneal Sensitivity at NGF0120 Week 8 (EoT NGF0120=Baseline NGF0122) Who Still Had Improvement in Corneal Sensitivity at Month 24 and 30 of the NGF0122 Study.

  At Month 24 and Month 30 of the NGF0122 study

• Number/Percentage of Patients Who Did Not Improved Corneal Sensitivity at NGF0120 Week 8 (EoT NGF0120=Baseline NGF0122) and Improved Corneal Sensitivity at Month 24 and 30 of the NGF0122 Study.

  At Month 24 and Month 30 of the NGF0122 study

• Mean Change in Corneal Sensitivity From NGF0120 Baseline and NGF0120 Week 8 (EoT NGF0120=Baseline NGF0122) to Month 24 and Month 30 of the NGF0122 Study.

  At Month 24 and Month 30 of the NGF0122 study

• Change From NGF0120 Baseline and NGF0120 Week 8 (EoT NGF0120=Baseline NGF0122) in Schirmer I Scores to Month 24 and Month 30 of the NGF0122 Study.

  At Month 24 and Month 30 of the NGF0122 study

• Change From NGF0120 Baseline and NGF0120 Week 8 (EoT NGF0120=Baseline NGF0122) in TFBUT to Months 24 and 30 of the NGF0122.

  At Month 24 and Month 30 of the NGF0122 study

Study Arms (1)

Group Long-term follow-up

All eligible patients, after completing enrollment in the original DEFENDO Study, were invited to enter the DEFENDO Long-Term Follow-Up Study (all standard of care permitted), according to inclusion and exclusion criteria.

Drug: Cenegemin in the DEFENDO Study

Interventions

Cenegemin in the DEFENDO Study DRUG

Cenegemin as administered in the original DEFENDO Study. Long-term safety and efficacy of OXERVATE® 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution administered in Stage 1 Neurotrophic Keratitis (NK) patients enrolled in the original DEFENDO Study (NGF0120 / NCT04485546). No intervention was performed in this follow-up / extension study.

Also known as: OXERVATE®

Group Long-term follow-up

Eligibility Criteria

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

Study Population

Stage 1 NK

You may qualify if:

• Previously enrolled in the DEFENDO Study.
• Satisfied all Informed Consent requirements. The patient and/or their legal representative read, signed, and dated the IRB approved Informed Consent document before any study-related procedures were performed.
• Had the ability and willingness to comply with study procedures.

You may not qualify if:

• Were participating in another study that involved treating the study eye. Participation in non-ocular studies was acceptable provided that the treatment was not considered to be confounding with the DEFENDO Long-Term Follow-up Study, in the opinion of the Investigator.

Sponsors & Collaborators

• Dompé Farmaceutici S.p.A: lead

Study Sites (4)

New Vision Institute

San Diego, California, 92037, United States

Location

Cincinnati Eye Institute

Edgewood, Kentucky, 41017, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02011, United States

Location

Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Interventions

cenegermin

Results Point of Contact

• Title: Clinical Development & Operations
• Organization: Dompé Farmaceutici S.p.A.

clinicaltrials@dompe.com

+39 02 583831

Study Officials

• Flavio Mantelli, MD, PhD

  Dompé

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2022
• First Posted: September 23, 2022
• Study Start: February 1, 2023
• Primary Completion: April 12, 2024
• Study Completion: April 12, 2024
• Last Updated: November 28, 2025
• Results First Posted: November 28, 2025

Record last verified: 2025-11

Locations

US (4)