Phase 2 Clinical Trial to Evaluate OC-01 Nasal Spray in Subjects With Neurotrophic Keratopathy

Study Overview
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Brief Summary
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The objective of this study is to evaluate the safety and effectiveness of OC-01 (varenicline) nasal spray as compared to placebo nasal spray for mean change from baseline in corneal fluorescein staining in subjects with Stage 1 (corneal epithelial hyperplasia/punctate keratopathy) neurotrophic keratopathy (NK) in one or both eyes.

Detailed Description
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Protocol OPP-102 is a Phase 2, multicenter, randomized, controlled, double- masked study designed to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray in subjects with NK. Approximately 100 subjects at least 18 years of age with a physicians' diagnosis of Stage 1 NK as defined by the Mackie Criteria and meeting all other study eligibility criteria will be randomized 1:1 and will receive OC-01 (varenicline)/ nasal spray or placebo nasal spray for 8 weeks three times daily (TID) as follows: OC-01 (varenicline) nasal spray, 1.2 mg/mL Placebo (vehicle control) nasal spray All doses will be delivered as a 50 microliter (µL) nasal spray.

Official Title
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A Phase 2, Multicenter, Randomized, Controlled, Double-Masked, Clinical Trial to Evaluate the Efficacy and Safety of OC-01 (Varenicline) Nasal Spray in Subjects With Neurotrophic Keratopathy (the Olympia Study)

Conditions
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Neurotrophic Keratopathy

Intervention / Treatment
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* Drug: OC-01 (varenicline) nasal spray 1.2 mg/ml
* Drug: Placebo (vehicle) nasal spray


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Be at least 18 years of age at Visit 1. Patients with Stage 1 (corneal epithelial hyperplasia/punctate keratopathy) NK in one or both eyes, as defined by the Mackie Criteria. Evidence of decreased corneal sensitivity (≤ 4 cm using the Cochet-Bonnet aesthesiometer) in at least 1 corneal quadrant. Schirmer's test without anesthesia ≥3 mm/ 5 minutes in the affected eye. If a female is of childbearing potential, they must: use an acceptable means of birth control (acceptable methods of contraception include hormonal - oral, implantable, injectable, or transdermal contraceptives, mechanical - spermicide in conjunction with a barrier such as a diaphragm or condom, IUD, or surgical sterilization of partner), and have a negative urine pregnancy test on Day 1. Exclusion Criteria: Have Stage 2 or Stage 3 NK affecting one or both eyes. Have ocular graft versus host disease or Stevens-Johnson syndrome. Have any active ocular infection (COVID-19 conjunctivitis, bacterial, viral, fungal, or protozoal) or active ocular inflammation not related to NK in the affected eye. Be currently receiving autologous serum tears, amniotic membrane, cenegermin, fresh frozen plasma or cord blood derivative tears. Have severe blepharitis and/or severe meibomian gland disease in the study eye.

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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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: OC-01 (varenicline) nasal spray, 1.2 mg/mL<br>OC-01 (varenicline) nasal spray, 1.2 mg/mL | Drug: OC-01 (varenicline) nasal spray 1.2 mg/ml<br>* OC-01<br>|
| Placebo Comparator: Placebo (vehicle control) nasal spray<br>Placebo (vehicle control) nasal spray | Drug: Placebo (vehicle) nasal spray<br>* Placebo (vehicle)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change from baseline in corneal fluorescein staining | Primary Endpoint | 8 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean change from baseline in visual acuity at Week 8 | Secondary Endpoint #1 | 8 weeks |