Efficacy of OC-01 Nasal Spray on Goblet Cell and Meibomian Gland Stimulation

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 Nasal Spray as compared to placebo in simulating Goblet Cell and Meibomian Gland function in adult subjects with DED.

Detailed Description
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This was a Phase 2, single-center, randomized, masked (including all subjects, investigators, and study site personnel), placebo-controlled study designed to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray in stimulating goblet cell and Meibomian gland function in in adult subjects with DED. Approximately 45 subjects at least 18 years of age with a physicians' diagnosis of DED and meeting all other study eligibility criteria were planned to be randomized to receive a single application of 0.2% (1.2 mg/mL) OC-01 or placebo

Official Title
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A Single-Center, Randomized, Controlled, Masked Clinical Trial to Evaluate the Efficacy of OC-01 Nasal Spray on Goblet Cell and Meibomian Gland Stimulation (The IMPERIAL Study)

Conditions
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Dry Eye Disease

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


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have used and/or desired to use an artificial tear substitute for dry eye symptoms within 6 months prior to visit 1. Exclusion Criteria: Have had any intraocular surgery (such as cataract surgery), extraocular surgery (such as blepharoplasty) in either eye within three months or refractive surgery (e.g. laser-assisted in-situ keratomileusis, laser epithelial keratomileusis, photorefractive keratectomy or corneal implant) within twelve months of Visit 1 Have a history or presence of any ocular disorder or condition in either eye that would, in the opinion of the Investigator, likely interfere with the interpretation of the study results or participant safety such as significant corneal or conjunctival scarring; pterygium or nodular pinguecula; current ocular infection, conjunctivitis, or inflammation not associated with dry eye; anterior (epithelial) basement membrane corneal dystrophy or other clinically significant corneal dystrophy or degeneration; ocular herpetic infection; evidence of keratoconus; etc. Blepharitis not requiring treatment and mild meibomian gland disease that are typically associated with DED are allowed. Have a systemic condition or disease not stabilized or judged by the Investigator to be incompatible with participation in the study or with the lengthier assessments required by the study (e.g., current systemic infection, uncontrolled autoimmune disease, uncontrolled immunodeficiency disease, history of myocardial infarction or heart disease, etc.) Have a known hypersensitivity to any of the procedural agents or study drug components Have any condition or history that, in the opinion of the investigator, may interfere with study compliance, outcome measures, safety parameters, and/or the general medical condition of the subject

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, one time dosing | Drug: OC-01 (varenicline) nasal spray<br>* OC-01 (varenicline) nasal spray<br>|
| Placebo Comparator: Placebo (vehicle control) nasal spray<br>Placebo (vehicle control) nasal spray, one time dosing | Drug: Placebo (vehicle control) nasal spray<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean Change in Goblet Cell Area | Mean change in Goblet Cell Area with a larger decrease is indicative of a better outcome. | Baseline (pre-treatment), 1 day (post treatment) | 
| Mean Change in Goblet Cell Perimeter | Mean change in Goblet Cell Perimeter. A larger decrease is indicative of a better outcome. | Baseline (pre-treatment), 1 day (post treatment) | 
| Change in Meibomian Gland Area, Upper Lid | Change in Meibomian gland area, upper lid. A larger decrease is indicative of a better outcome | Baseline (pre-treatment), 1 day (post treatment) | 
| Mean Change in Meibomian Gland Area, Lower Lid. | Mean change in Meibomian Gland Area, lower Lid. A larger decrease is indicative of a better outcome. | Baseline (pre-treatment), 1 day (post treatment) | 
| Mean Change in Meibomian Gland Perimeter, Upper Lid. | Chane in Meibomian Gland Perimeter, upper lid. A larger decrease is indicative of a better outcome. | Baseline (pre-treatment), 1 day (post treatment) | 
| Mean Change in Meibomian Gland Perimeter, Lower Lid | Mean change in Meibomian Gland Perimeter, lower lid. A larger decrease is indicative of a better outcome. | Baseline (pre-treatment), 1 day (post treatment) |