Clinical Trial to Evaluate the Chronic Efficacy of OC-01 Nasal Spray on Signs of Dry Eye Disease (The MYSTIC Study)

Randomized, Controlled, Single-Masked Clinical Trial to Evaluate the Chronic Efficacy of OC-01 Nasal Spray on Signs of Dry Eye Disease (The MYSTIC Study)

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

Study Overview

• Status: Completed
• Conditions: Dry Eye Disease
• Intervention / Treatment: Drug: OC-01 0.1% 0.6 mg/ml; Drug: OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml); Other: placebo comparator

Detailed Description

Study OPP-004 was a Phase 2, single-center, randomized, masked (including subjects, Investigators, and study site personnel), placebo-controlled study designed to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray in adult subjects with DED. The study planned to randomize approximately 120 subjects at least 22 years of age with a physicians' diagnosis of Dry Eye Disease and meeting all other study eligibility criteria to receive an application of OC-01 or placebo BID for 12 weeks. Subjects who terminated early during the application period were asked to complete safety assessments (if the subjects agree) prior to study exit. Subjects who were terminated early from the study were not replaced.

• Study Type: Interventional
• Enrollment (Actual): 123
• Phase: Phase 2

Contacts and Locations

Study Locations

• Mexico
  • Mexico City, Mexico
    • Mexico City

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 us 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 an 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

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; vehicle control	Other: placebo comparator; vehicle control
Active Comparator: OC-01 0.1%, 0.6 mg/ml; OC-01 (varenicline) nasal spray, 0.6 mg/mL	Drug: OC-01 0.1% 0.6 mg/ml; OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml); Other Names: varenicline
Active Comparator: OC-01 0.2%, 1.2 mg/ml; OC-01 (varenicline) nasal spray, 1.2 mg/mL	Drug: OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml); OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml); Other Names: varenicline

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Change in Schirmer's Test Score From Baseline to 84 Days	The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.	Visit 1 (baseline) and Visit 6 (84 days)

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 6 (Day 84)	The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.	Visit 1 (baseline) and Visit 6 (84 days)
Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28)	The Schirmer's test measures the amount of tears produced by placing a paper strip in the eye for 5 minutes and the distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.	Visit 1 (baseline) and Visit 4 (28 Days)
Mean Change in Schirmer's Test Score From Baseline to 84 Days (Visit 6) - Fellow Eye	The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.	Visit 1 (baseline) to Visit 6 (84 Days)
Mean Change in Schirmer's Test Score From Baseline to 28 Days (Visit 4) - Study Eye	The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment of OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.	Visit 1 (baseline) to Visit 4 (28 days)]
Mean Change in Schirmer's Test Score From Baseline to 28 Days (Visit 4) - Fellow Eye	The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.	Visit 1 (baseline) to Visit 4 (28 Days)
Mean Change in Total Corneal Fluorescein Staining	A standardized grading system of 0-3 is used for each of the five areas of the cornea (inferior, superior, nasal, temporal, central). Grade 0 will be specified when no staining is present. The total score ranges from 0-15. Lower scores indicate improvement.	Visit 1 (baseline) and Visit 5 (56 days)

Collaborators and Investigators

• Sponsor: Oyster Point Pharma, Inc.

Publications and helpful links

General Publications

• Quiroz-Mercado H, Hernandez-Quintela E, Chiu KH, Henry E, Nau JA. A phase II randomized trial to evaluate the long-term (12-week) efficacy and safety of OC-01 (varenicline solution) nasal spray for dry eye disease: The MYSTIC study. Ocul Surf. 2022 Apr;24:15-21. doi: 10.1016/j.jtos.2021.12.007. Epub 2021 Dec 15.

Study record dates

Study Major Dates

• Study Start (Actual): February 18, 2019
• Primary Completion (Actual): January 7, 2020
• Study Completion (Actual): January 7, 2020

Study Registration Dates

• First Submitted: March 11, 2019
• First Submitted That Met QC Criteria: March 11, 2019
• First Posted (Actual): March 13, 2019

Study Record Updates

• Last Update Posted (Actual): June 8, 2022
• Last Update Submitted That Met QC Criteria: March 8, 2022
• Last Verified: March 1, 2022

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-004

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