Clinical Trial to Evaluate the Efficacy of OC-02 Nasal Spray on Signs and Symptoms of Dry Eye Disease (The PEARL Study)

• Sponsor: Oyster Point Pharma, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03452397
• Collaborator: (none)
• Enrollment: 165
• Locations: 3
• Arms: 4
• Actual Duration (Months): 1.9
• Patients Per Site: 55
• Patients Per Site Per Month: 28.4

Study Details

Study Description

Brief Summary

The objective of this study is to evaluate the safety and effectiveness of OC-02 Nasal Spray as compared to placebo on signs and symptoms of dry eye disease.

Condition or Disease	Intervention/Treatment	Phase
Dry Eye Disease	Drug: 0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL); Drug: 1.0 % hemigalactarate (0.11% free base) 1.0 % OC-02 Mid Dose (5.5 mg/mL); Drug: 2.0 % hemigalactarate (0.11% free base) 2.0 % OC-02 High Dose (11.1 mg/mL); Drug: Placebo (vehicle) nasal spray	Phase 2

Detailed Description

This was a Phase 2, multicenter, randomized, double-masked, placebo controlled study designed to evaluate the safety and efficacy of OC 02 Nasal Spray in adult participants with dry eye disease. Approximately 160 subjects, at least 22 years of age, with a subject-reported history of dry eye disease and meeting all other study eligibility criteria were planned to be randomized to receive an application of OC-02 or placebo at Visit 1 and Visit 2.

Study Design

• Study Type: Interventional
• Actual Enrollment: 165 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-02 Nasal Spray on Signs and Symptoms of Dry Eye Disease (The PEARL Study)
• Actual Study Start Date: Feb 27, 2018
• Actual Primary Completion Date: Apr 27, 2018
• Actual Study Completion Date: Apr 27, 2018

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: 0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL) 0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL)	Drug: 0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL); 0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL)
Active Comparator: 1.0 % hemigalactarate (0.11% free base) 1.0 % OC-02 Mid Dose (5.5 mg/mL) 1.0 % hemigalactarate (0.11% free base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)	Drug: 1.0 % hemigalactarate (0.11% free base) 1.0 % OC-02 Mid Dose (5.5 mg/mL); 1.0 % hemigalactarate (0.11% free base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)
Active Comparator: 2.0 % hemigalactarate (0.11% free base) 2.0 % OC-02 High Dose (11.1 mg/mL) 2.0 % hemigalactarate (0.11% free base) 2.0 % OC-02 High Dose (11.1 mg/mL)	Drug: 2.0 % hemigalactarate (0.11% free base) 2.0 % OC-02 High Dose (11.1 mg/mL); 2.0 % hemigalactarate (0.11% free base) 2.0 % OC-02 High Dose (11.1 mg/mL)
Placebo Comparator: Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray	Drug: Placebo (vehicle) nasal spray; Placebo (vehicle) nasal spray

Outcome Measures

Primary Outcome Measures

1. Schirmer's Test Score at Day 1 [Day 1 (Pre to Post-Treatment Change)]

   The primary endpoint was the change in anesthetized Schirmer's Test Score (STS) from baseline to Day 1. Change in Schirmer test score pre to post treatment. The Schirmer's test measures the amount of tears produced by placing a paper strip in the eye for 5 minutes and distance of wetting was recorded. Schirmer's test scores from 0-35 mm where a higher score is indicative of a better outcome.

2. Eye Dryness Score at Visit 2 [Day 15 (Pre to Post-Treatment Change)]

   Change in Eye Dryness score from baseline to Day 15. Eye dryness score 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	Louisville	Louisville	Kentucky	United States	40206
2	Andover	Andover	Massachusetts	United States	01810
3	Nashville	Nashville	Tennessee	United States	37210

Sponsors and Collaborators

• Oyster Point Pharma, Inc.

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol - Oct 11, 2017
• Statistical Analysis Plan - May 10, 2018

More Information

Publications

None provided.

• Responsible Party: Oyster Point Pharma, Inc.
• ClinicalTrials.gov Identifier: NCT03452397
• Other Study ID Numbers: OPP-001
• First Posted: Mar 2, 2018
• Last Update Posted: Dec 14, 2021
• Last Verified: May 1, 2019

• 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

Study Results

Participant Flow

Arm/Group Title	0.2% Hemigalactarate (0.11% Free Base) 0.2% OC-02 Mid Dose (1.1 mg/mL)	1% Hemigalactarate (0.11% Free Base) 1% OC-02 Mid Dose (5.5 mg/mL)	2% Hemigalactarate (0.11% Free Base) 2% OC-02 High Dose (11.1 mg/mL)	Placebo (Vehicle) Nasal Spray
Arm/Group Description	0.2% hemigalactarate (0.11% free base) 0.2% OC-02 Mid Dose (1.1 mg/mL)	1% hemigalactarate (0.11% free base) 1% OC-02 Mid Dose (5.5 mg/mL)	2% hemigalactarate (0.11% free base) 2% OC-02 High Dose (11.1 mg/mL)	Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray: Placebo (vehicle) nasal spray
Period Title: Overall Study
STARTED	41	41	41	42
COMPLETED	40	41	39	41
NOT COMPLETED	1	0	2	1

Baseline Characteristics

Arm/Group Title	0.2 % Hemigalactarate (0.11% Free Base) 0.2 % OC-02 Low Dose (1.1 mg/mL)	1.0 % Hemigalactarate (0.11% Free Base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)	2.0 % Hemigalactarate (0.11% Free Base) 2.0 % OC-02 High Dose (11.1 mg/mL)	Placebo (Vehicle) Nasal Spray	Total
Arm/Group Description	0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL)	1.0 % hemigalactarate (0.11% free base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)	2.0 % hemigalactarate (0.11% free base) 2.0 % OC-02 High Dose (11.1 mg/mL)	Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray: Placebo (vehicle) nasal spray	Total of all reporting groups
Overall Participants	41	41	41	42	165
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	64.4 (11.80)	64.0 (11.15)	62.7 (9.30)	64.4 (11.76)	63.9 (10.97)
Sex: Female, Male (Count of Participants)
Female	24 58.5%	29 70.7%	31 75.6%	34 81%	118 71.5%
Male	17 41.5%	12 29.3%	10 24.4%	8 19%	47 28.5%
Race/Ethnicity, Customized (Count of Participants)
Hispanic/Latino	0 0%	1 2.4%	0 0%	2 4.8%	3 1.8%
Not Hispanic/Latino	41 100%	40 97.6%	41 100%	39 92.9%	161 97.6%
Not reported	0 0%	0 0%	0 0%	1 2.4%	1 0.6%
Race/Ethnicity, Customized (Count of Participants)
American Indian Alaskan Native	0 0%	0 0%	0 0%	0 0%	0 0%
Asian	1 2.4%	2 4.9%	0 0%	0 0%	3 1.8%
Black/African American	3 7.3%	2 4.9%	3 7.3%	3 7.1%	11 6.7%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%	0 0%	0 0%
White	37 90.2%	37 90.2%	37 90.2%	39 92.9%	150 90.9%
Multiple races	0 0%	0 0%	1 2.4%	0 0%	1 0.6%
Other	0 0%	0 0%	0 0%	0 0%	0 0%
Region of Enrollment (participants) [Number]
United States	41 100%	41 100%	41 100%	42 100%	165 100%

Outcome Measures

1. Primary Outcome

Title	Schirmer's Test Score at Day 1
Description	The primary endpoint was the change in anesthetized Schirmer's Test Score (STS) from baseline to Day 1. Change in Schirmer test score pre to post treatment. The Schirmer's test measures the amount of tears produced by placing a paper strip in the eye for 5 minutes and distance of wetting was recorded. Schirmer's test scores from 0-35 mm where a higher score is indicative of a better outcome.
Time Frame	Day 1 (Pre to Post-Treatment Change)

Outcome Measure Data

Analysis Population Description

Subjects in the ITT population

Arm/Group Title	0.2 % Hemigalactarate (0.11% Free Base) 0.2 % OC-02 Low Dose (1.1 mg/mL)	1.0 % Hemigalactarate (0.11% Free Base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)	2.0 % Hemigalactarate (0.11% Free Base) 2.0 % OC-02 High Dose (11.1 mg/mL)	Placebo (Vehicle) Nasal Spray
Arm/Group Description	0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL)	1.0 % hemigalactarate (0.11% free base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)	2.0 % hemigalactarate (0.11% free base) 2.0 % OC-02 High Dose (11.1 mg/mL)	Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray: Placebo (vehicle) nasal spray
Measure Participants	41	41	41	42
Least Squares Mean (95% Confidence Interval) [mm]	9.0	17.5	19.6	3.0

2. Primary Outcome

Title	Eye Dryness Score at Visit 2
Description	Change in Eye Dryness score from baseline to Day 15. Eye dryness score 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	Day 15 (Pre to Post-Treatment Change)

Outcome Measure Data

Analysis Population Description

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/Group Title	0.2 % Hemigalactarate (0.11% Free Base) 0.2 % OC-02 Low Dose (1.1 mg/mL)	1 % Hemigalactarate (0.11% Free Base) 1 % OC-02 Mid Dose (5.5 mg/mL)	2 % Hemigalactarate (0.11% Free Base) 2 % OC-02 High Dose (11.1 mg/mL)	Placebo (Vehicle) Nasal Spray
Arm/Group Description	0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL)	1 % hemigalactarate (0.11% free base) 1 % OC-02 Mid Dose (5.5 mg/mL)	2 % hemigalactarate (0.11% free base) 2 % OC-02 High Dose (11.1 mg/mL)	Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray: Placebo (vehicle) nasal spray
Measure Participants	37	41	38	41
Least Squares Mean (95% Confidence Interval) [mm]	-9.4	-17.4	-20.7	-6.5

Adverse Events

Time Frame	Adverse events were collected from the first dose of study drug administration until the final study visit at Visit 2, up to 15 days.
Arm/Group Title	0.2 % Hemigalactarate (0.11% Free Base) 0.2 % OC-02 Low Dose (1.1 mg/mL)		1.0 % Hemigalactarate (0.11% Free Base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)		2.0 % Hemigalactarate (0.11% Free Base) 2.0 % OC-02 High Dose (11.1 mg/mL)		Placebo (Vehicle) Nasal Spray
Arm/Group Description	0.2 % hemigalactarate (0.11% free base) 0.2 % OC-02 Low Dose (1.1 mg/mL)		1.0 % hemigalactarate (0.11% free base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)		2.0 % hemigalactarate (0.11% free base) 2.0 % OC-02 High Dose (11.1 mg/mL)		Placebo (vehicle) nasal spray Placebo (vehicle) nasal spray: Placebo (vehicle) nasal spray
All Cause Mortality
	0.2 % Hemigalactarate (0.11% Free Base) 0.2 % OC-02 Low Dose (1.1 mg/mL)		1.0 % Hemigalactarate (0.11% Free Base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)		2.0 % Hemigalactarate (0.11% Free Base) 2.0 % OC-02 High Dose (11.1 mg/mL)		Placebo (Vehicle) Nasal Spray
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/41 (0%)		0/41 (0%)		0/41 (0%)		0/42 (0%)
Serious Adverse Events
	0.2 % Hemigalactarate (0.11% Free Base) 0.2 % OC-02 Low Dose (1.1 mg/mL)		1.0 % Hemigalactarate (0.11% Free Base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)		2.0 % Hemigalactarate (0.11% Free Base) 2.0 % OC-02 High Dose (11.1 mg/mL)		Placebo (Vehicle) Nasal Spray
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/41 (2.4%)		0/41 (0%)		0/41 (0%)		0/42 (0%)
Metabolism and nutrition disorders
failure to thrive	1/41 (2.4%)		0/41 (0%)		0/41 (0%)		0/42 (0%)
Other (Not Including Serious) Adverse Events
	0.2 % Hemigalactarate (0.11% Free Base) 0.2 % OC-02 Low Dose (1.1 mg/mL)		1.0 % Hemigalactarate (0.11% Free Base) 1.0 % OC-02 Mid Dose (5.5 mg/mL)		2.0 % Hemigalactarate (0.11% Free Base) 2.0 % OC-02 High Dose (11.1 mg/mL)		Placebo (Vehicle) Nasal Spray
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	17/41 (41.5%)		23/41 (56.1%)		24/41 (58.5%)		4/42 (9.5%)
Ear and labyrinth disorders
Tympanic membrane perforation	0/41 (0%)		0/41 (0%)		0/41 (0%)		1/42 (2.4%)
Vertigo	0/41 (0%)		0/41 (0%)		0/41 (0%)		1/42 (2.4%)
Eye disorders
Eye pruritus	0/41 (0%)		0/41 (0%)		1/41 (2.4%)		0/42 (0%)
Keratits	0/41 (0%)		1/41 (2.4%)		0/41 (0%)		0/42 (0%)
Gastrointestinal disorders
Gastrooesphageal reflux disease	0/41 (0%)		0/41 (0%)		0/41 (0%)		1/42 (2.4%)
General disorders
Instillation site irritation	3/41 (7.3%)		4/41 (9.8%)		6/41 (14.6%)		0/42 (0%)
Infections and infestations
Pharyngitis streptococcal	0/41 (0%)		0/41 (0%)		0/41 (0%)		1/42 (2.4%)
Upper respiratory tract infection	0/41 (0%)		0/41 (0%)		1/41 (2.4%)		0/42 (0%)
Nasopharyngitis	2/41 (4.9%)		0/41 (0%)		3/41 (7.3%)		0/42 (0%)
Metabolism and nutrition disorders
Dehydration	1/41 (2.4%)		0/41 (0%)		0/41 (0%)		0/42 (0%)
Failure to thrive	1/41 (2.4%)		0/41 (0%)		0/41 (0%)		0/42 (0%)
Nervous system disorders
Headache	0/41 (0%)		0/41 (0%)		0/41 (0%)		1/42 (2.4%)
Paraesthesia	0/41 (0%)		1/41 (2.4%)		0/41 (0%)		0/42 (0%)
Psychiatric disorders
Acute stress disorder	1/41 (2.4%)		0/41 (0%)		0/41 (0%)		0/42 (0%)
Respiratory, thoracic and mediastinal disorders
Cough	8/41 (19.5%)		10/41 (24.4%)		15/41 (36.6%)		0/42 (0%)
Throat irritation	6/41 (14.6%)		10/41 (24.4%)		10/41 (24.4%)		0/42 (0%)
Sneezing	1/41 (2.4%)		4/41 (9.8%)		1/41 (2.4%)		1/42 (2.4%)
Epistaxis	0/41 (0%)		1/41 (2.4%)		0/41 (0%)		0/42 (0%)

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.