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 on signs and symptoms of dry eye disease
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.
Interventions
OC-01 (varenicline) 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
22 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 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 Schirmer's Test Score From Baseline to 28 Days | 28 Days [Visit 1 (baseline) and Visit 5 (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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Eye Dryness Score From Baseline to Day 28 | 28 days [Visit 1 (baseline and Visit 5 (28 days)] | 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. |
| Change From Baseline to Day 21 in Eye Dryness Score at 5 Minutes Post Treatment in the CAE. | 21 days [Visit 1 (baseline) and Visit 4 (21 days)] | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| OC-01 Low Dose, 0.12 mg/mL OC-01 0.12 mg/ml nasal spray BID for 28 days | 47 |
| OC-01 Mid Dose, 0.6 mg/mL OC-01 0.6 mg/ml nasal spray BID for 28 days | 48 |
| OC-01 High Dose, 1.2 mg/mL OC-01 1.2 mg/ml nasal spray BID for 28 days | 44 |
| Placebo Vehicle nasal spray | 43 |
| Total | 182 |
Baseline characteristics
| Characteristic | Total | OC-01 Low Dose, 0.12 mg/mL | OC-01 Mid Dose, 0.6 mg/mL | OC-01 High Dose, 1.2 mg/mL | Placebo |
|---|---|---|---|---|---|
| Age, Continuous | 65.5 years STANDARD_DEVIATION 10.8 | 64.2 years STANDARD_DEVIATION 12.7 | 66.5 years STANDARD_DEVIATION 9.4 | 67.4 years STANDARD_DEVIATION 10.6 | 64 years STANDARD_DEVIATION 10.3 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 18 Participants | 8 Participants | 3 Participants | 2 Participants | 5 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 164 Participants | 39 Participants | 45 Participants | 42 Participants | 38 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 2 Participants | 0 Participants | 1 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 8 Participants | 3 Participants | 4 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 14 Participants | 2 Participants | 4 Participants | 6 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants | 0 Participants | 0 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 157 Participants | 42 Participants | 39 Participants | 36 Participants | 40 Participants |
| Region of Enrollment United States | 182 participants | 47 participants | 48 participants | 44 participants | 43 participants |
| Schirmer's Test Score | 5.0 mm STANDARD_DEVIATION 2.9 | 5.2 mm STANDARD_DEVIATION 3.1 | 4.8 mm STANDARD_DEVIATION 2.7 | 5.5 mm STANDARD_DEVIATION 3 | 4.5 mm STANDARD_DEVIATION 2.9 |
| Sex: Female, Male Female | 137 Participants | 36 Participants | 34 Participants | 35 Participants | 32 Participants |
| Sex: Female, Male Male | 45 Participants | 11 Participants | 14 Participants | 9 Participants | 11 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 47 | 0 / 48 | 0 / 44 | 0 / 43 |
| other Total, other adverse events | 33 / 47 | 44 / 48 | 41 / 44 | 11 / 43 |
| serious Total, serious adverse events | 0 / 47 | 1 / 48 | 0 / 44 | 0 / 43 |
Outcome results
Primary
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.
Time frame: 28 Days [Visit 1 (baseline) and Visit 5 (28 days)]
Population: Subjects in ITT-LOCF population
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| OC-01 Low Dose, 0.12 mg/mL | Mean Change in Schirmer's Test Score From Baseline to 28 Days | 10.1 score on a scale, mm | Standard Error 1.26 |
| OC-01 Mid Dose, 0.6 mg/ML | Mean Change in Schirmer's Test Score From Baseline to 28 Days | 11.7 score on a scale, mm | Standard Error 1.27 |
| OC-01 High Dose, 1.2 mg/mL | Mean Change in Schirmer's Test Score From Baseline to 28 Days | 11.0 score on a scale, mm | Standard Error 1.39 |
| Placebo | Mean Change in Schirmer's Test Score From Baseline to 28 Days | 3.2 score on a scale, mm | Standard Error 1.31 |
Secondary
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.
Time frame: 28 days [Visit 1 (baseline and Visit 5 (28 days)]
Population: Subjects in the ITT-LOCF population. The overall number of participants is different because these outcomes were assessed ar different visits 7 days apart and some subjects were not able to attend the Day 21 visit.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| OC-01 Low Dose, 0.12 mg/mL | Change From Baseline in Eye Dryness Score From Baseline to Day 28 | -11.6 score on a scale, mm | Standard Error 3.63 |
| OC-01 Mid Dose, 0.6 mg/ML | Change From Baseline in Eye Dryness Score From Baseline to Day 28 | -18.9 score on a scale, mm | Standard Error 3.67 |
| OC-01 High Dose, 1.2 mg/mL | Change From Baseline in Eye Dryness Score From Baseline to Day 28 | -15.6 score on a scale, mm | Standard Error 4.02 |
| Placebo | Change From Baseline in Eye Dryness Score From Baseline to Day 28 | -5.4 score on a scale, mm | Standard Error 3.8 |
Secondary
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.
Time frame: 21 days [Visit 1 (baseline) and Visit 4 (21 days)]
Population: Subjects in the ITT population
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| OC-01 Low Dose, 0.12 mg/mL | Change From Baseline to Day 21 in Eye Dryness Score at 5 Minutes Post Treatment in the CAE. | -8.1 score on a scale, mm | Standard Error 2.7 |
| OC-01 Mid Dose, 0.6 mg/ML | Change From Baseline to Day 21 in Eye Dryness Score at 5 Minutes Post Treatment in the CAE. | -16.0 score on a scale, mm | Standard Error 2.7 |
| OC-01 High Dose, 1.2 mg/mL | Change From Baseline to Day 21 in Eye Dryness Score at 5 Minutes Post Treatment in the CAE. | -18.4 score on a scale, mm | Standard Error 3 |
| Placebo | Change From Baseline to Day 21 in Eye Dryness Score at 5 Minutes Post Treatment in the CAE. | -4.4 score on a scale, mm | Standard Error 2.8 |