A Long-term Extension Study to Evaluate the Safety and Efficacy of OXERVATE® 0.002% (20 mcg/mL) Cenegermin-bkbj Ophthalmic Solution in Patients With Stage 1 Neurotrophic Keratitis Who Enrolled in the DEFENDO Study (NGF0120)
Overview
- Phase
- Not Applicable
- Intervention
- Cenegemin in the DEFENDO Study
- Conditions
- Neurotrophic Keratitis
- Sponsor
- Dompé Farmaceutici S.p.A
- Enrollment
- 37
- Locations
- 4
- Primary Endpoint
- 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
- Status
- Completed
- Last Updated
- 5 months ago
Overview
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
Detailed Description
NGF0122 (DEFENDO Long-Term Follow-up) study was a Phase 4, multicenter, open label, long-term follow-up study evaluating safety and efficacy in the patients with Stage 1 Neurotrophic Keratitis (NK) who were enrolled in the NGF0120 (original DEFENDO) study and were treated with OXERVATE® 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution for up to 8 weeks in the NGF0120. After completing enrollment in the NGF0120 Study, patients were invited to enter the NGF0122 Study (all standard of care permitted) in which two additional long-term follow-up visits occurred at 24- and 30-months post-treatment to evaluate long-term clinical outcomes. In the NGF0122, study patients enrolled in the NFG0120 study were evaluated starting from week 8, which corresponds to the end of treatment of the NGF0120 study and is to be considered as the baseline of the NGF0122 study itself. Patients were treated per standard of care including additional OXERVATE® 0.002% if deemed appropriate by the Investigator.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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.
Exclusion Criteria
- •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.
Arms & Interventions
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.
Intervention: Cenegemin in the DEFENDO Study
Outcomes
Primary Outcomes
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
Time Frame: 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.
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.
Time Frame: At Month 24 and Month 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.
Secondary Outcomes
- 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)
- Number/Percentage of Patients Who Achieved a 15-letter Improvement in BCDVA 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) 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 Quality of Life (QoL) Expressed by the IDEEL to Months 24 and 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 Quality of Life Measured With EuroQol 5-Dimension 5-Level (EQ-5D-5L) to Months 24 and 30 of the NGF0122 Study.(At Month 24 and Month 30 of the NGF0122 study)
- Number/Percentage of Subjects Reporting Adverse Events (AEs)(From NGF0120 Week 8=NGF0122 Baseline to Month 30 of the NGF0122 study)
- Frequency of Normal and Clinically Significant Abnormal Findings for Slit-lamp Examination Parameters in the Study Eye at Baseline and Week 8 of the NGF0120 and at Month 24 and 30 of the NGF0122(Baseline and week 8 of the NGF0120, month 24 and 30 of the NGF0122)