Dry Eye Disease
Conditions
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
Interventions
OC-01 (varenicline) nasal spray
DRUGPlacebo (vehicle control) nasal spray
Placebo
Sponsors
Oyster Point Pharma, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Change in Goblet Cell Area | Baseline (pre-treatment), 1 day (post treatment) | Mean change in Goblet Cell Area with a larger decrease is indicative of a better outcome. |
| Mean Change in Goblet Cell Perimeter | Baseline (pre-treatment), 1 day (post treatment) | Mean change in Goblet Cell Perimeter. A larger decrease is indicative of a better outcome. |
| Change in Meibomian Gland Area, Upper Lid | Baseline (pre-treatment), 1 day (post treatment) | Change in Meibomian gland area, upper lid. A larger decrease is indicative of a better outcome |
| Mean Change in Meibomian Gland Area, Lower Lid. | Baseline (pre-treatment), 1 day (post treatment) | Mean change in Meibomian Gland Area, lower Lid. A larger decrease is indicative of a better outcome. |
| Mean Change in Meibomian Gland Perimeter, Upper Lid. | Baseline (pre-treatment), 1 day (post treatment) | Chane in Meibomian Gland Perimeter, upper lid. A larger decrease is indicative of a better outcome. |
| Mean Change in Meibomian Gland Perimeter, Lower Lid | Baseline (pre-treatment), 1 day (post treatment) | Mean change in Meibomian Gland Perimeter, lower lid. A larger decrease is indicative of a better outcome. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| OC-01, 1.2 mg/mL OC-01: OC-01 (varenicline) nasal spray | 12 |
| Placebo Placebo (vehicle) nasal spray: Placebo | 6 |
| Total | 18 |
Baseline characteristics
| Characteristic | OC-01, 1.2 mg/mL | Total | Placebo |
|---|---|---|---|
| Age, Continuous | 60.5 years STANDARD_DEVIATION 13.79 | 61.4 years STANDARD_DEVIATION 13.35 | 63.3 years STANDARD_DEVIATION 13.46 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 11 Participants | 17 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 2 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 9 Participants | 15 Participants | 6 Participants |
| Region of Enrollment United States | 12 participants | 18 participants | 6 participants |
| Schirmer's Test Score | 6.6 mm STANDARD_DEVIATION 4.42 | 5.9 mm STANDARD_DEVIATION 4.25 | 4.5 mm STANDARD_DEVIATION 3.89 |
| Sex: Female, Male Female | 11 Participants | 14 Participants | 3 Participants |
| Sex: Female, Male Male | 1 Participants | 4 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 12 | 0 / 6 |
| other Total, other adverse events | 6 / 12 | 1 / 6 |
| serious Total, serious adverse events | 0 / 12 | 0 / 6 |
Outcome results
Primary
Change in Meibomian Gland Area, Upper Lid
Change in Meibomian gland area, upper lid. A larger decrease is indicative of a better outcome
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Population: Subjects in the ITT population. Only Subjects who had both pre-treatment and post-treatment values were utilized in the change from pre-treatment statistics.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| OC-01, 1.2 mg/mL | Change in Meibomian Gland Area, Upper Lid | 11.2 um | Standard Deviation 118.6 |
| Placebo | Change in Meibomian Gland Area, Upper Lid | -143.2 um | Standard Deviation 154.8 |
Primary
Mean Change in Goblet Cell Area
Mean change in Goblet Cell Area with a larger decrease is indicative of a better outcome.
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Population: Subjects in the ITT population. Only Subjects who had both pre-treatment and post-treatment values were utilized in the change from pre-treatment statistics.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| OC-01, 1.2 mg/mL | Mean Change in Goblet Cell Area | -38.8 um | Standard Deviation 30.2 |
| Placebo | Mean Change in Goblet Cell Area | -6.7 um | Standard Deviation 1.94 |
Primary
Mean Change in Goblet Cell Perimeter
Mean change in Goblet Cell Perimeter. A larger decrease is indicative of a better outcome.
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Population: Subjects in the ITT population. Only Subjects who had both pre-treatment and post-treatment values were utilized in the change from pre-treatment statistics.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| OC-01, 1.2 mg/mL | Mean Change in Goblet Cell Perimeter | -7.7 um | Standard Deviation 5.79 |
| Placebo | Mean Change in Goblet Cell Perimeter | -1.8 um | Standard Deviation 0.76 |
Primary
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.
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Population: Subjects in the ITT population. Only Subjects who had both pre-treatment and post-treatment values were utilized in the change from pre-treatment statistics.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| OC-01, 1.2 mg/mL | Mean Change in Meibomian Gland Area, Lower Lid. | -29.9 um | Standard Deviation 88.1 |
| Placebo | Mean Change in Meibomian Gland Area, Lower Lid. | -12.4 um | Standard Deviation 95.8 |
Primary
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.
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Population: Subjects in the ITT population. Only Subjects who had both pre-treatment and post-treatment values were utilized in the change from pre-treatment statistics.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| OC-01, 1.2 mg/mL | Mean Change in Meibomian Gland Perimeter, Lower Lid | -4 um | Standard Deviation 10.9 |
| Placebo | Mean Change in Meibomian Gland Perimeter, Lower Lid | 4.2 um | Standard Deviation 19.3 |
Primary
Mean Change in Meibomian Gland Perimeter, Upper Lid.
Chane in Meibomian Gland Perimeter, upper lid. A larger decrease is indicative of a better outcome.
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Population: Subjects in the ITT population. Only Subjects who had both pre-treatment and post-treatment values were utilized in the change from pre-treatment statistics.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| OC-01, 1.2 mg/mL | Mean Change in Meibomian Gland Perimeter, Upper Lid. | 6.15 um | Standard Deviation 16.6 |
| Placebo | Mean Change in Meibomian Gland Perimeter, Upper Lid. | -19.2 um | Standard Deviation 22.8 |