Clinical trial NCT03688802: Efficacy of OC-01 Nasal Spray on Goblet Cell and Meibomian Gland Stimulation
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Dry Eye Disease
Lead sponsor: Oyster Point Pharma, Inc.
Sponsor class: INDUSTRY
First posted: 2018-09-28
Enrollment: 18
Sex: ALL
Minimum age (years): 18
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 in simulating Goblet Cell and Meibomian Gland function in adult subjects with DED.

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

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), 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

Primary outcomes:
measure: Mean Change in Goblet Cell Area
timeFrame: Baseline (pre-treatment), 1 day (post treatment)
measure: Mean Change in Goblet Cell Perimeter
timeFrame: Baseline (pre-treatment), 1 day (post treatment)
measure: Change in Meibomian Gland Area, Upper Lid
timeFrame: Baseline (pre-treatment), 1 day (post treatment)
measure: Mean Change in Meibomian Gland Area, Lower Lid.
timeFrame: Baseline (pre-treatment), 1 day (post treatment)
measure: Mean Change in Meibomian Gland Perimeter, Upper Lid.
timeFrame: Baseline (pre-treatment), 1 day (post treatment)
measure: Mean Change in Meibomian Gland Perimeter, Lower Lid
timeFrame: Baseline (pre-treatment), 1 day (post treatment)
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Tufts
state: Massachusetts
Study officials:
References:
citation: 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.