Trial Outcomes & Findings for Evaluation of the Efficacy and Safety of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease (NCT NCT04036292)

Last Updated: 2021-11-22

Results Overview

The primary endpoint was the percentage of subjects who achieve ≥10 mm improvement in Schirmer's Test Score from baseline to 28 days in the study eye following treatment with OC-01. Schirmer's test scores from 0-35 mm where a higher score is indicative of a better outcome.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

758 participants

Primary outcome timeframe

28 Days [Visit 1 (baseline) and Visit 4b (28 days)]

Results posted on

2021-11-22

Participant Flow

Participant milestones

Participant milestones
Measure	OC-01 Low Dose 0.6 mg/mL BID for 28 Days OC-01 (varenicline) nasal spray, 0.6 mg/ML BID for 28 days	OC-01 High Dose 1.2 mg/mL BID for 28 Days OC-01 (varenicline) nasal spray 1.2 mg/mL BID for 28 days	Placebo BID for 28 Days Placebo (vehicle) nasal spray BID for 28 days
Overall Study STARTED	260	246	252
Overall Study COMPLETED	239	212	228
Overall Study NOT COMPLETED	21	34	24

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Evaluation of the Efficacy and Safety of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	OC-01 Low Dose 0.6 mg/mL BID for 28 Days n=260 Participants OC-01 (varenicline) nasal spray, 0.6 mg/ML BID for 28 days OC-01 (varenicline) nasal spray: OC-01 (varenicline) nasal spray	OC-01 High Dose 1.2 mg/mL BID for 28 Days n=246 Participants OC-01 (varenicline) nasal spray, 1.2 mg/ML BID for 28 days OC-01 (varenicline) nasal spray: OC-01 (varenicline) nasal spray	Placebo (Vehicle) Nasal Spray BID for 28 Days n=252 Participants Placebo (vehicle) nasal spray BID for 28 days Placebo (vehicle) nasal spray: Placebo (vehicle) nasal spray	Total n=758 Participants Total of all reporting groups
Age, Continuous	59.6 years STANDARD_DEVIATION 12.76 • n=5 Participants	58.4 years STANDARD_DEVIATION 13.03 • n=7 Participants	58.4 years STANDARD_DEVIATION 13.29 • n=5 Participants	58.8 years STANDARD_DEVIATION 13.02 • n=4 Participants
Sex: Female, Male Female	194 Participants n=5 Participants	181 Participants n=7 Participants	201 Participants n=5 Participants	576 Participants n=4 Participants
Sex: Female, Male Male	66 Participants n=5 Participants	65 Participants n=7 Participants	51 Participants n=5 Participants	182 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	27 Participants n=5 Participants	37 Participants n=7 Participants	36 Participants n=5 Participants	100 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	233 Participants n=5 Participants	209 Participants n=7 Participants	216 Participants n=5 Participants	658 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race/Ethnicity, Customized Race · American Indian or Alaska Native	1 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants	9 Participants n=4 Participants
Race/Ethnicity, Customized Race · Asian	11 Participants n=5 Participants	7 Participants n=7 Participants	5 Participants n=5 Participants	23 Participants n=4 Participants
Race/Ethnicity, Customized Race · Black or African American	27 Participants n=5 Participants	35 Participants n=7 Participants	29 Participants n=5 Participants	91 Participants n=4 Participants
Race/Ethnicity, Customized Race · Native Hawaiian or other Pacific Islander	2 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	3 Participants n=4 Participants
Race/Ethnicity, Customized Race · White	219 Participants n=5 Participants	200 Participants n=7 Participants	211 Participants n=5 Participants	630 Participants n=4 Participants
Race/Ethnicity, Customized Race · Other	0 Participants n=5 Participants	2 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Region of Enrollment United States	260 participants n=5 Participants	246 participants n=7 Participants	252 participants n=5 Participants	758 participants n=4 Participants
Baseline Schirmer's Test Score (mm)	5.1 mm STANDARD_DEVIATION 2.95 • n=5 Participants	5.4 mm STANDARD_DEVIATION 2.93 • n=7 Participants	4.9 mm STANDARD_DEVIATION 2.89 • n=5 Participants	5.1 mm STANDARD_DEVIATION 2.93 • n=4 Participants

PRIMARY outcome

Timeframe: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]

Population: Subjects in the ITT-LOCF population

Outcome measures

Outcome measures
Measure	OC-01 Low Dose, 0.6 mg/mL n=260 Participants OC-01 (varenicline) nasal spray, 0.6 mg/ML OC-01 (varenicline) nasal spray	OC-01 High Dose, 1.2 mg/mL n=246 Participants OC-01 (varenicline) nasal spray, 1.2 mg/ML OC-01 (varenicline) nasal spray	Placebo (Vehicle) Nasal Spray n=252 Participants Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray
Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28)	123 Participants	121 Participants	70 Participants

SECONDARY outcome

Timeframe: 28 Days [Visit 1 (baseline) and Visit 4a (28 days)]

Population: Subjects in the mITT-2 population

Change in Eye Dryness Score Eye from baseline in CAE at 4 Weeks at 5 minutes post treatment. Eye dryness score on a Visual Analogue Scale (VAS) from 0 (no dryness) to 100 (maximum dryness) millimeters where a lower score is indicative of a better outcome.

Outcome measures

Outcome measures
Measure	OC-01 Low Dose, 0.6 mg/mL n=187 Participants OC-01 (varenicline) nasal spray, 0.6 mg/ML OC-01 (varenicline) nasal spray	OC-01 High Dose, 1.2 mg/mL n=171 Participants OC-01 (varenicline) nasal spray, 1.2 mg/ML OC-01 (varenicline) nasal spray	Placebo (Vehicle) Nasal Spray n=169 Participants Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray
Mean Change From Baseline in Eye Dryness Score in CAE at Week 4 at 5 Minutes	-10.3 mm Standard Error 1.62	-9.0 mm Standard Error 1.75	-7.4 mm Standard Error 1.74

SECONDARY outcome

Timeframe: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]

Population: Subjects in the ITT-LOCF population

Change in Eye Dryness Score Eye from baseline to 28 days. Eye dryness score on a Visual Analogue Scale (VAS) from 0 (no dryness) to 100 (maximum dryness) millimeters where a lower score is indicative of a better outcome.

Outcome measures

Outcome measures
Measure	OC-01 Low Dose, 0.6 mg/mL n=255 Participants OC-01 (varenicline) nasal spray, 0.6 mg/ML OC-01 (varenicline) nasal spray	OC-01 High Dose, 1.2 mg/mL n=242 Participants OC-01 (varenicline) nasal spray, 1.2 mg/ML OC-01 (varenicline) nasal spray	Placebo (Vehicle) Nasal Spray n=248 Participants Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray
Mean Change From Baseline in Eye Dryness Score From Baseline to Day 28	-19.8 mm Standard Error 1.54	-22.2 mm Standard Error 1.61	-15.4 mm Standard Error 1.57

SECONDARY outcome

Timeframe: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]

Population: Subjects in the ITT-LOCF population

Change in Schirmer test score from baseline to Day 28. Schirmer's test scores from 0-35 mm where a higher score is indicative of a better outcome.

Outcome measures

Outcome measures
Measure	OC-01 Low Dose, 0.6 mg/mL n=251 Participants OC-01 (varenicline) nasal spray, 0.6 mg/ML OC-01 (varenicline) nasal spray	OC-01 High Dose, 1.2 mg/mL n=235 Participants OC-01 (varenicline) nasal spray, 1.2 mg/ML OC-01 (varenicline) nasal spray	Placebo (Vehicle) Nasal Spray n=248 Participants Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray
Mean Change Form Baseline in Schirmer's Test Score From Baseline to Day 28	11.3 mm Standard Error 0.61	11.5 mm Standard Error 0.64	6.3 mm Standard Error 0.61

SECONDARY outcome

Timeframe: 14 Days [Visit 1 (baseline) and Visit 3 (14 days)]

Population: Subjects in the ITT-LOCF population

Chang in Eye Dryness Score Eye from baseline to 28 days. Eye dryness score on a Visual Analogue Scale (VAS) from 0 (no dryness) to 100 (maximum dryness) millimeters where a lower score is indicative of a better otucome.

Outcome measures

Outcome measures
Measure	OC-01 Low Dose, 0.6 mg/mL n=255 Participants OC-01 (varenicline) nasal spray, 0.6 mg/ML OC-01 (varenicline) nasal spray	OC-01 High Dose, 1.2 mg/mL n=239 Participants OC-01 (varenicline) nasal spray, 1.2 mg/ML OC-01 (varenicline) nasal spray	Placebo (Vehicle) Nasal Spray n=248 Participants Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray
Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 2	-16.5 mm Standard Error 1.38	-17.9 mm Standard Error 1.45	-12.7 mm Standard Error 1.41

SECONDARY outcome

Timeframe: 7 Days [Visit 1 (baseline) and Visit 2 (7 days)]

Population: Subjects in the ITT population

Outcome measures

Outcome measures
Measure	OC-01 Low Dose, 0.6 mg/mL n=254 Participants OC-01 (varenicline) nasal spray, 0.6 mg/ML OC-01 (varenicline) nasal spray	OC-01 High Dose, 1.2 mg/mL n=239 Participants OC-01 (varenicline) nasal spray, 1.2 mg/ML OC-01 (varenicline) nasal spray	Placebo (Vehicle) Nasal Spray n=248 Participants Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray
Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 1	-15.7 mm Standard Error 1.33	-15.4 mm Standard Error 1.40	-13.3 mm Standard Error 1.35

Adverse Events

OC-01 Low Dose, 0.6 mg/mL

Serious events: 5 serious events

Other events: 253 other events

Deaths: 0 deaths

OC-01 High Dose, 1.2 mg/mL

Serious events: 12 serious events

Other events: 243 other events

Deaths: 2 deaths

Placebo

Serious events: 9 serious events

Other events: 158 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Jeffrey Nau

Oyster Point Pharma, Inc.

Phone: 609-382-9035

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	OC-01 Low Dose, 0.6 mg/mL n=260 participants at risk OC-01 (varenicline) nasal spray, 0.6 mg/ML	OC-01 High Dose, 1.2 mg/mL n=245 participants at risk OC-01 (varenicline) nasal spray, 1.2 mg/ML	Placebo n=251 participants at risk Placebo (vehicle) nasal spray
Infections and infestations Coronavirus infection	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Infections and infestations Sepsis	0.38% 1/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Infections and infestations Diabetic gangrene	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Infections and infestations Gangrene	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Infections and infestations Osteomyelitis	0.38% 1/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Infections and infestations Pneumonia	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Infections and infestations Pneumonia bacterial	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Cardiac disorders Atrial fibrillation	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Cardiac disorders Atrioventricular block complete	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Cardiac disorders Bradycardia	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Cardiac disorders Coronary artery disease	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Cardiac disorders Myocardial infarction	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Musculoskeletal and connective tissue disorders Intervertebral disc protrusion	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Musculoskeletal and connective tissue disorders Lumbar spinal stenosis	0.38% 1/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lung neoplasm malignant	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Pancreatic carcinoma	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Nervous system disorders Cerebrovascular accident	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Nervous system disorders Transient ischemic attack	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Vascular disorders Deep vein thrombosis	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Vascular disorders Hypertensive urgency	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Congenital, familial and genetic disorders Arnold-Chiari malformation	0.38% 1/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Gastrointestinal disorders Umbilical hernia	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Hepatobiliary disorders Cholecystitis acute	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Injury, poisoning and procedural complications Humerus fracture	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.41% 1/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Renal and urinary disorders Renal haematoma	0.00% 0/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.40% 1/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7
Surgical and medical procedures Hip surgery	0.38% 1/260 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/245 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7	0.00% 0/251 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7