Evaluation of the Efficacy of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease

Multicenter, Randomized, Controlled, Double-Masked Clinical Trial to Evaluate the Efficacy of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease (The ONSET-1 Study)

The objective of this study is to evaluate the safety and effectiveness of OC-01 Nasal Spray as compared to placebo on signs and symptoms of dry eye disease

Study Overview

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

Detailed Description

This was a Phase 2b, multicenter, randomized, double-masked, placebo controlled study designed to evaluate the safety and efficacy of OC 01 nasal spray in adult subjects with DED. The study randomized 182 subjects, at least 22 years of age, who had a physician's diagnosis of dry eye disease and met all other study eligibility criteria to receive an application of OC-01 or placebo twice daily (BID) for 4 weeks.

• Study Type: Interventional
• Enrollment (Actual): 182
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Newport Beach, California, United States, 92663
      • Newport Beach
  • Indiana
    • Indianapolis, Indiana, United States, 46290
      • Indianapolis
  • Massachusetts
    • Andover, Massachusetts, United States, 01810
      • Andover

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 22 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 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
• Masking: DOUBLE

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Placebo (vehicle) nasal spray	Drug: Placebo (vehicle) nasal spray; Placebo
Active Comparator: OC-01 Low Dose, 0.12 mg/mL; OC-01 (varenicline) nasal spray, Low Dose, 0.12 mg/mL	Drug: OC-01 (varenicline) nasal spray; OC-01 (varenicline) nasal spray
Active Comparator: OC-01 Mid Dose, 0.6 mg/mL; OC-01 (varenicline) nasal spray Mid dose, 0.6 mg/mL	Drug: OC-01 (varenicline) nasal spray; OC-01 (varenicline) nasal spray
Active Comparator: OC-01 High Dose, 1.2 mg/mL; OC-01 (varenicline) nasal spray High dose, 1.2 mg/mL	Drug: OC-01 (varenicline) nasal spray; OC-01 (varenicline) nasal spray

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Change in Schirmer's Test Score From Baseline to 28 Days	The primary end point was the change in anesthetized Schirmer's Test Score (STS) from baseline to 28 days in the study eye following treatment with OC-01. Schirmer's test score from 0-35 mm where a higher score is indicative of a better outcome.	28 Days [Visit 1 (baseline) and Visit 5 (28 days)]

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Eye Dryness Score From Baseline to Day 28	Change in Eye Dryness Score from baseline to 28 days. Eye Dryness (EDS) on a Visual Analogue Scale (VAS) from 0 (no discomfort) to 100 (maximum discomfort) millimeters where a lower score is indicative of a better outcome.	28 days [Visit 1 (baseline and Visit 5 (28 days)]
Change From Baseline to Day 21 in Eye Dryness Score at 5 Minutes Post Treatment in the CAE.	Change from baseline to Day 21 in Eye Dryness Score at 5 minutes post treatment in the CAE. Eye Dryness (EDS) on a Visual Analogue Scale (VAS) from 0 (no discomfort) to 100 (maximum discomfort) millimeters where a lower score is indicative of a better outcome.	21 days [Visit 1 (baseline) and Visit 4 (21 days)]

Collaborators and Investigators

• Sponsor: Oyster Point Pharma, Inc.

Publications and helpful links

General Publications

• Nijm LM, Zhu D, Hemphill M, Blemker GL, Hendrix LH, Kabat AG, Gibson AA. Does Menopausal Status Affect Dry Eye Disease Treatment Outcomes with OC-01 (Varenicline Solution) Nasal Spray? A Post Hoc Analysis of ONSET-1 and ONSET-2 Clinical Trials. Ophthalmol Ther. 2023 Feb;12(1):355-364. doi: 10.1007/s40123-022-00607-7. Epub 2022 Nov 18.
• Wirta D, Torkildsen GL, Boehmer B, Hollander DA, Bendert E, Zeng L, Ackermann M, Nau J. ONSET-1 Phase 2b Randomized Trial to Evaluate the Safety and Efficacy of OC-01 (Varenicline Solution) Nasal Spray on Signs and Symptoms of Dry Eye Disease. Cornea. 2022 Oct 1;41(10):1207-1216. doi: 10.1097/ICO.0000000000002941. Epub 2021 Dec 21.

Study record dates

Study Major Dates

• Study Start (Actual): August 15, 2018
• Primary Completion (Actual): September 26, 2018
• Study Completion (Actual): September 26, 2018

Study Registration Dates

• First Submitted: August 15, 2018
• First Submitted That Met QC Criteria: August 15, 2018
• First Posted (Actual): August 17, 2018

Study Record Updates

• Last Update Posted (Actual): October 25, 2021
• Last Update Submitted That Met QC Criteria: October 21, 2021
• Last Verified: August 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-002

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