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

• Sponsor: Oyster Point Pharma, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03636061
• Collaborator: (none)
• Enrollment: 182
• Locations: 3
• Arms: 4
• Actual Duration (Months): 1.4
• Patients Per Site: 60.7
• Patients Per Site Per Month: 44

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Dry Eye Disease	Drug: OC-01 (varenicline) nasal spray; Drug: Placebo (vehicle) nasal spray	Phase 2

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 182 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: Multicenter, Randomized, Controlled, Double-Masked Clinical Trial to Evaluate the Efficacy of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease (The ONSET-1 Study)
• Actual Study Start Date: Aug 15, 2018
• Actual Primary Completion Date: Sep 26, 2018
• Actual Study Completion Date: Sep 26, 2018

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: OC-01 Low Dose, 0.12 mg/mL OC-01 (varenicline) nasal spray, Low Dose, 0.12 mg/mL	Drug: OC-01 (varenicline) nasal spray; OC-01 (varenicline) nasal spray
Active Comparator: OC-01 Mid Dose, 0.6 mg/mL OC-01 (varenicline) nasal spray Mid dose, 0.6 mg/mL	Drug: OC-01 (varenicline) nasal spray; OC-01 (varenicline) nasal spray
Active Comparator: OC-01 High Dose, 1.2 mg/mL OC-01 (varenicline) nasal spray High dose, 1.2 mg/mL	Drug: OC-01 (varenicline) nasal spray; OC-01 (varenicline) nasal spray
Placebo Comparator: Placebo Placebo (vehicle) nasal spray	Drug: Placebo (vehicle) nasal spray; Placebo

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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.

2. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 22 Years and Older
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Newport Beach	Newport Beach	California	United States	92663
2	Indianapolis	Indianapolis	Indiana	United States	46290
3	Andover	Andover	Massachusetts	United States	01810

Sponsors and Collaborators

• Oyster Point Pharma, Inc.

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol - Jul 11, 2018
• Statistical Analysis Plan - Oct 4, 2018

More Information

Publications

None provided.

• Responsible Party: Oyster Point Pharma, Inc.
• ClinicalTrials.gov Identifier: NCT03636061
• Other Study ID Numbers: OPP-002
• First Posted: Aug 17, 2018
• Last Update Posted: Oct 25, 2021
• Last Verified: Aug 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Dry Eye Syndromes; Keratoconjunctivitis Sicca; Eye Diseases; Varenicline

Study Results

Participant Flow

Arm/Group Title	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
Arm/Group Description	OC-01 0.12 mg/ml nasal spray BID for 28 days	OC-01 0.6 mg/ml nasal spray BID for 28 days	OC-01 1.2 mg/ml nasal spray BID for 28 days	Placebo (vehicle) nasal spray
Period Title: Overall Study
STARTED	47	48	44	43
COMPLETED	47	46	40	43
NOT COMPLETED	0	2	4	0

Baseline Characteristics

Arm/Group Title	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	Total
Arm/Group Description	OC-01 0.12 mg/ml nasal spray BID for 28 days	OC-01 0.6 mg/ml nasal spray BID for 28 days	OC-01 1.2 mg/ml nasal spray BID for 28 days	Vehicle nasal spray	Total of all reporting groups
Overall Participants	47	48	44	43	182
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	64.2 (12.7)	66.5 (9.4)	67.4 (10.6)	64 (10.3)	65.5 (10.8)
Sex: Female, Male (Count of Participants)
Female	36 76.6%	34 70.8%	35 79.5%	32 74.4%	137 75.3%
Male	11 23.4%	14 29.2%	9 20.5%	11 25.6%	45 24.7%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	8 17%	3 6.3%	2 4.5%	5 11.6%	18 9.9%
Not Hispanic or Latino	39 83%	45 93.8%	42 95.5%	38 88.4%	164 90.1%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	1 2.1%	1 2.3%	0 0%	2 1.1%
Asian	3 6.4%	4 8.3%	0 0%	1 2.3%	8 4.4%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	1 2.3%	0 0%	1 0.5%
Black or African American	2 4.3%	4 8.3%	6 13.6%	2 4.7%	14 7.7%
White	42 89.4%	39 81.3%	36 81.8%	40 93%	157 86.3%
More than one race	0 0%	0 0%	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%	0 0%
Region of Enrollment (participants) [Number]
United States	47 100%	48 100%	44 100%	43 100%	182 100%
Schirmer's Test Score (mm) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [mm]	5.2 (3.1)	4.8 (2.7)	5.5 (3.0)	4.5 (2.9)	5.0 (2.9)

Outcome Measures

1. Primary Outcome

Title	Mean Change in Schirmer's Test Score From Baseline to 28 Days
Description	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)]

Outcome Measure Data

Analysis Population Description

Subjects in ITT-LOCF population

Arm/Group Title	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
Arm/Group Description	OC-01 0.12 mg/ml nasal spray	OC-01 0.6 mg/ml nasal spray	OC-01 1.2 mg/ml nasal spray	Vehicle nasal spray
Measure Participants	47	46	40	43
Least Squares Mean (Standard Error) [score on a scale, mm]	10.1 (1.26)	11.7 (1.27)	11.0 (1.39)	3.2 (1.31)

2. Secondary Outcome

Title	Change From Baseline in Eye Dryness Score From Baseline to Day 28
Description	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)]

Outcome Measure Data

Analysis Population Description

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/Group Title	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
Arm/Group Description	OC-01 0.12 mg/ml nasal spray	OC-01 0.6 mg/ml nasal spray	OC-01 1.2 mg/ml nasal spray	Vehicle nasal spray
Measure Participants	47	46	40	43
Least Squares Mean (Standard Error) [score on a scale, mm]	-11.6 (3.63)	-18.9 (3.67)	-15.6 (4.02)	-5.4 (3.8)

3. Secondary Outcome

Title	Change From Baseline to Day 21 in Eye Dryness Score at 5 Minutes Post Treatment in the CAE.
Description	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)]

Outcome Measure Data

Analysis Population Description

Subjects in the ITT population

Arm/Group Title	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
Arm/Group Description	OC-01 0.12 mg/ml nasal spray	OC-01 0.6 mg/ml nasal spray	OC-01 1.2 mg/ml nasal spray	Vehicle nasal spray
Measure Participants	44	45	38	42
Least Squares Mean (Standard Error) [score on a scale, mm]	-8.1 (2.7)	-16.0 (2.7)	-18.4 (3.0)	-4.4 (2.8)

Adverse Events

Time Frame	Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
Arm/Group Title	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
Arm/Group Description	OC-01 0.12 mg/ml nasal spray		OC-01 0.6 mg/ml nasal spray		OC-01 1.2 mg/ml nasal spray		Vehicle nasal spray
All Cause Mortality
	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
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/47 (0%)		0/48 (0%)		0/44 (0%)		0/43 (0%)
Serious Adverse Events
	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
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/47 (0%)		1/48 (2.1%)		0/44 (0%)		0/43 (0%)
Blood and lymphatic system disorders
Anaemia	0/47 (0%)	0	1/48 (2.1%)	1	0/44 (0%)	0	0/43 (0%)	0
Other (Not Including Serious) Adverse Events
	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
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	33/47 (70.2%)		44/48 (91.7%)		41/44 (93.2%)		11/43 (25.6%)
Eye disorders
Ocular TEAEs	1/47 (2.1%)	1	2/48 (4.2%)	2	1/44 (2.3%)	1	7/43 (16.3%)	7
Visual acuity reduced	1/47 (2.1%)	1	1/48 (2.1%)	1	0/44 (0%)	0	3/43 (7%)	3
Blepharospasm	0/47 (0%)	0	0/48 (0%)	0	0/44 (0%)	0	1/43 (2.3%)	1
Conjunctival deposit	0/47 (0%)	0	0/48 (0%)	0	0/44 (0%)	0	1/43 (2.3%)	1
Conjunctival haemorrhage	0/47 (0%)	0	1/48 (2.1%)	1	0/44 (0%)	0	0/43 (0%)	0
Eyelid oedema	0/47 (0%)	0	0/48 (0%)	0	1/44 (2.3%)	1	0/43 (0%)	0
Visual impairement	0/47 (0%)	0	0/48 (0%)	0	0/44 (0%)	0	1/43 (2.3%)	1
Hordeolum	0/47 (0%)	0	0/48 (0%)	0	0/44 (0%)	0	1/43 (2.3%)	1
General disorders
Instillation site irritation	3/47 (6.4%)	3	8/48 (16.7%)	8	8/44 (18.2%)	8	0/43 (0%)	0
Nervous system disorders
Headache	0/47 (0%)	0	0/48 (0%)	0	2/44 (4.5%)	2	1/43 (2.3%)	1
Respiratory, thoracic and mediastinal disorders
Sneezing	29/47 (61.7%)	29	38/48 (79.2%)	38	37/44 (84.1%)	37	0/43 (0%)	0
Cough	4/47 (8.5%)	4	6/48 (12.5%)	6	11/44 (25%)	11	0/43 (0%)	0
Throat irritation	0/47 (0%)	0	7/48 (14.6%)	7	9/44 (20.5%)	9	0/43 (0%)	0
Dysaesthesia pharynx	5/47 (10.6%)	5	4/48 (8.3%)	4	3/44 (6.8%)	3	0/43 (0%)	0
Nasal dryness	1/47 (2.1%)	1	0/48 (0%)	0	0/44 (0%)	0	2/43 (4.7%)	2

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.