Clinical trial NCT04036292: Evaluation of the Efficacy and Safety of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease
Official title: Multicenter, Randomized, Controlled, Double-Masked, Clinical Trial to Evaluate the Efficacy and Safety of OC-01 (Varenicline) Nasal Spray on Signs and Symptoms of Dry Eye Disease (The ONSET-2 Study)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Dry Eye Disease (DED)
Lead sponsor: Oyster Point Pharma, Inc.
Sponsor class: INDUSTRY
First posted: 2019-07-29
Enrollment: 758
Sex: ALL
Minimum age (years): 22
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: This was a Phase 3, multicenter, randomized, controlled, double-masked (including subjects, Investigators, study site personnel, and Sponsor personnel) study designed to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray 0.6 mg/mL and 1.2 mg/mL in adult participants with DED. The study randomized 758 subjects at least 22 years of age with a physicians' diagnosis of DED and meeting all other study eligibility criteria to receive OC-01 (varenicline) nasal spray or placebo twice daily (BID) for 28 days with three additional long-term follow-up visits at 6 weeks, 6 months, and 12 months. Participants who terminated from the study during the treatment period were asked to complete safety assessments (if the participants agree) prior to study exit. Participants who were terminated early from the study were not replaced

Eligibility criteria: 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) or extraocular surgery 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 dry eye disease 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.

Primary outcomes:
measure: Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28)
timeFrame: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
Secondary outcomes:
measure: Mean Change From Baseline in Eye Dryness Score in CAE at Week 4 at 5 Minutes
timeFrame: 28 Days [Visit 1 (baseline) and Visit 4a (28 days)]
measure: Mean Change From Baseline in Eye Dryness Score From Baseline to Day 28
timeFrame: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
measure: Mean Change Form Baseline in Schirmer's Test Score From Baseline to Day 28
timeFrame: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
measure: Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 2
timeFrame: 14 Days [Visit 1 (baseline) and Visit 3 (14 days)]
measure: Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 1
timeFrame: 7 Days [Visit 1 (baseline) and Visit 2 (7 days)]
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix
state: Arizona
city: Newport Beach
country: United States
facility: New Port Beach
state: California
city: Fort Collins
country: United States
facility: Fort Collins
state: Colorado
city: Waterbury
country: United States
facility: Waterbury
state: Connecticut
city: Delray Beach
country: United States
facility: Delray Beach
state: Florida
city: Indianapolis
country: United States
facility: Indianapolis
state: Indiana
city: Louisville
country: United States
facility: Louisville
state: Kentucky
city: Raynham
country: United States
facility: Raynham
state: Massachusetts
city: Las Vegas
country: United States
facility: Las Vegas
state: Nevada
city: Raleigh
country: United States
facility: Raleigh
state: North Carolina
city: Shelby
country: United States
facility: Shelby
state: North Carolina
city: Fargo
country: United States
facility: Fargo
state: North Dakota
city: Cranberry Township
country: United States
facility: Cranberry Township
state: Pennsylvania
city: Warwick
country: United States
facility: Warwick
state: Rhode Island
city: Sioux Falls
country: United States
facility: Sioux Falls
state: South Dakota
city: Nashville
country: United States
facility: Nashville
state: Tennessee
city: Austin
country: United States
facility: Austin
state: Texas
city: Houston
country: United States
facility: Houston
state: Texas
city: San Antonio
country: United States
facility: San Antonio
state: Texas
city: San Antonio
country: United States
facility: San Antonio
state: Texas
city: Layton
country: United States
facility: Layton
state: Utah
city: Lynchburg
country: United States
facility: Lynchburg
state: Virginia
Study officials:
References:
citation: 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.
citation: Wirta D, Vollmer P, Paauw J, Chiu KH, Henry E, Striffler K, Nau J; ONSET-2 Study Group. Efficacy and Safety of OC-01 (Varenicline Solution) Nasal Spray on Signs and Symptoms of Dry Eye Disease: The ONSET-2 Phase 3 Randomized Trial. Ophthalmology. 2022 Apr;129(4):379-387. doi: 10.1016/j.ophtha.2021.11.004. Epub 2021 Nov 10.