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

Study Overview
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Brief Summary
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The primary objectives are to evaluate the long-term safety and efficacy of OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution in Stage 1 Neurotrophic Keratitis (NK) patients who enrolled in the DEFENDO Study.

Detailed Description
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This clinical study will be a multi-center, open label, long term follow-up study of the patients who were enrolled in the DEFENDO Study who had Stage 1 Neurotrophic Keratitis (NK) who were treated with OXERVATE™ 0.002% (20 mcg/mL) cenegermin-bkbj ophthalmic solution. The DEFENDO Long-Term Follow-up Study will have follow-up with the patients at month 24-30 post-treatment in the DEFENDO Study: All patients will be evaluated in a real-world setting post the enrollment in the DEFENDO Study through 30 months. Patients will be treated per standard of care as determined by the Investigator and will document topical ophthalmic medications. The original DEFENDO Study duration was for a total of 34 weeks: a screening period of 2 weeks, followed by enrollment in 8 weeks of OXERVATE™ treatment and an Off- Treatment Follow-Up of 6 months. After completing enrollment in the original DEFENDO Study, patients will be invited to enter the DEFENDO Long- Term Follow-up Study (all standard of care is permitted). Two additional long-term follow-up visits will occur at 24- and 30-months to evaluate long-term clinical outcomes.

Official Title
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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

Conditions
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Neurotrophic Keratitis

Intervention / Treatment
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* Drug: Cenegemin in the DEFENDO Study


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Previously enrolled in the DEFENDO Study. Satisfy all Informed Consent requirements. The patient and/or his/her legal representative has read, signed, and dated the IRB approved Informed Consent document before any study-related procedures are performed. Must have the ability and willingness to comply with study procedures. Exclusion Criteria: Participating in another study that involves treating the study eye. a. Participation in non-ocular studies is acceptable provided that the treatment is not considered to be confounding with the DEFENDO Long-Term Follow-up Study, in the opinion of the Investigator.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Group Long-term follow-up<br>All eligible patients, after completing enrollment in the original DEFENDO Study, will be invited to enter the DEFENDO Long-Term Follow-Up Study (all standard of care is permitted), according to inclusion and exclusion criteria. | Drug: Cenegemin in the DEFENDO Study<br>* Cenegemin as administered in the 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 DEFENDO Study (NCT number NCT04485546). No intervention was performed in this follow-up / extension trial.<br>* Other names: OXERVATE™;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Corneal Epithelial Healing | Percentage of patients who have corneal epithelial healing at 24 months and 30 months of the long- term follow-up. | From baseline to 24 months | 
| Corneal Epithelial Healing | Percentage of patients who have corneal epithelial healing at 24 months and 30 months of the long- term follow-up. | From baseline to 30 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Corneal Sensitivity | Percentage of patients who achieved an improvement in corneal sensitivity at 24 months and 30 months of the long-term follow-up. | 24 months and 30 months | 
| Change from Baseline in BCDVA at 24 months and 30 months | Change from Baseline Visit in the DEFENDO Study in Best Corrected Distance Visual Acuity (BCDVA) at 24 months and 30 months of the long-term follow-up. | 24 months and 30 months | 
| Change from Baseline in TFBUT at 24 months and 30 months | Change from the Baseline Visit in the DEFENDO Study in Tear Film Break Up Time (TFBUT) at 24 months and 30 months of the long-term follow-up. | 24 months and 30 months | 
| Percentage of patients that achieve a 15- letter gain in BCDVA | Best Corrected Distance Visual Acuity (BCDVA) | 24 months and 30 months | 
| Change from baseline in Quality of life (QoL) change from baseline | EQ-5D-5L questionnaire will be completed by participants | 24 months and 30 months | 
| Change from baseline in Quality of life (QoL) change from baseline | IDEEL questionnaire will be completed by participants | 24 months and 30 months | 
| Change from baseline in corneal nerve structure (IVCM) at months 24 and 30 | Change from the Baseline Visit in the DEFENDO Study in corneal nerve structure with confocal microscopy | 24 months and 30 months | 
| Change from baseline in Optical Coherence Tomography (OCT) at months 24 and 30 | Evaluate the change from the Baseline Visit in the DEFENDO Study in epithelial thickness with anterior-segment OCT | 24 months and 30 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Stage 1 NK, Oxervate, Cenegermin, NK, DEFENDO