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

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)

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.

Study Overview

• Status: Completed
• Conditions: Dry Eye Disease
• Intervention / Treatment: Drug: OC-01 (varenicline) nasal spray; Drug: Placebo (vehicle control) nasal spray

Detailed Description

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

• Study Type: Interventional
• Enrollment (Actual): 18
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02111
      • Tufts

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: OC-01 (varenicline) nasal spray, 1.2 mg/mL; OC-01 (varenicline) nasal spray, 1.2 mg/mL, one time dosing	Drug: OC-01 (varenicline) nasal spray; OC-01 (varenicline) nasal spray
Placebo Comparator: Placebo (vehicle control) nasal spray; Placebo (vehicle control) nasal spray, one time dosing	Drug: Placebo (vehicle control) nasal spray; Placebo

What is the study measuring?

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)

Collaborators and Investigators

• Sponsor: Oyster Point Pharma, Inc.

Publications and helpful links

General Publications

• Dieckmann GM, Cox SM, Lopez MJ, Ozmen MC, Yavuz Saricay L, Bayrakutar BN, Binotti WW, Henry E, Nau J, Hamrah P. A Single Administration of OC-01 (Varenicline Solution) Nasal Spray Induces Short-Term Alterations in Conjunctival Goblet Cells in Patients with Dry Eye Disease. Ophthalmol Ther. 2022 Aug;11(4):1551-1561. doi: 10.1007/s40123-022-00530-x. Epub 2022 Jun 2.

Study record dates

Study Major Dates

• Study Start (Actual): September 25, 2018
• Primary Completion (Actual): September 30, 2019
• Study Completion (Actual): September 30, 2019

Study Registration Dates

• First Submitted: September 26, 2018
• First Submitted That Met QC Criteria: September 27, 2018
• First Posted (Actual): September 28, 2018

Study Record Updates

• Last Update Posted (Actual): December 29, 2021
• Last Update Submitted That Met QC Criteria: November 30, 2021
• Last Verified: November 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Lacrimal Apparatus Diseases; Keratoconjunctivitis; Conjunctivitis; Conjunctival Diseases; Keratitis; Corneal Diseases; Eye Diseases; Dry Eye Syndromes; Keratoconjunctivitis Sicca; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Cholinergic Agents; Nicotinic Agonists; Cholinergic Agonists; Varenicline
• Other Study ID Numbers: OPP-005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No