Efficacy of OC-01 Nasal Spray on Goblet Cell and Meibomian Gland Stimulation

• Sponsor: Oyster Point Pharma, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03688802
• Collaborator: (none)
• Enrollment: 18
• Location: 1
• Arms: 2
• Actual Duration (Months): 12.2
• Patients Per Site Per Month: 1.5

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 in simulating Goblet Cell and Meibomian Gland function in adult subjects with DED.

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

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

Study Design

• Study Type: Interventional
• Actual Enrollment: 18 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Treatment
• Official Title: A Single-Center, Randomized, Controlled, Masked Clinical Trial to Evaluate the Efficacy of OC-01 Nasal Spray on Goblet Cell and Meibomian Gland Stimulation (The IMPERIAL Study)
• Actual Study Start Date: Sep 25, 2018
• Actual Primary Completion Date: Sep 30, 2019
• Actual Study Completion Date: Sep 30, 2019

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: OC-01 (varenicline) nasal spray, 1.2 mg/mL OC-01 (varenicline) nasal spray, 1.2 mg/mL, one time dosing	Drug: OC-01 (varenicline) nasal spray; OC-01 (varenicline) nasal spray
Placebo Comparator: Placebo (vehicle control) nasal spray Placebo (vehicle control) nasal spray, one time dosing	Drug: Placebo (vehicle control) nasal spray; Placebo

Outcome Measures

Primary Outcome Measures

1. 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.

2. 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.

3. 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

4. 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.

5. 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.

6. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 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 (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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Tufts	Boston	Massachusetts	United States	02111

Sponsors and Collaborators

• Oyster Point Pharma, Inc.

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol - Mar 26, 2019
• Statistical Analysis Plan - Jul 1, 2020

More Information

Publications

None provided.

• Responsible Party: Oyster Point Pharma, Inc.
• ClinicalTrials.gov Identifier: NCT03688802
• Other Study ID Numbers: OPP-005
• First Posted: Sep 28, 2018
• Last Update Posted: Dec 29, 2021
• Last Verified: Nov 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; Eye Diseases; Keratoconjunctivitis Sicca; Varenicline

Study Results

Participant Flow

Arm/Group Title	OC-01 (Varenicline) Nasal Spray, 1.2 mg/mL	Placebo (Vehicle Control) Nasal Spray
Arm/Group Description	OC-01 (varenicline) nasal spray, 1.2 mg/mL OC-01 (varenicline) nasal spray: OC-01 (varenicline) nasal spray	Placebo (vehicle control) nasal spray Placebo (vehicle control) nasal spray: Placebo
Period Title: Overall Study
STARTED	12	6
COMPLETED	11	6
NOT COMPLETED	1	0

Baseline Characteristics

Arm/Group Title	OC-01, 1.2 mg/mL	Placebo	Total
Arm/Group Description	OC-01: OC-01 (varenicline) nasal spray	Placebo (vehicle) nasal spray: Placebo	Total of all reporting groups
Overall Participants	12	6	18
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	60.5 (13.79)	63.3 (13.46)	61.4 (13.35)
Sex: Female, Male (Count of Participants)
Female	11 91.7%	3 50%	14 77.8%
Male	1 8.3%	3 50%	4 22.2%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	0 0%	0 0%	0 0%
Not Hispanic or Latino	11 91.7%	6 100%	17 94.4%
Unknown or Not Reported	1 8.3%	0 0%	1 5.6%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%
Asian	1 8.3%	0 0%	1 5.6%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	2 16.7%	0 0%	2 11.1%
White	9 75%	6 100%	15 83.3%
More than one race	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%
Region of Enrollment (participants) [Number]
United States	12 100%	6 100%	18 100%
Schirmer's Test Score (mm) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [mm]	6.6 (4.42)	4.5 (3.89)	5.9 (4.25)

Outcome Measures

1. Primary Outcome

Title	Mean Change in Goblet Cell Area
Description	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)

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	OC-01, 1.2 mg/mL	Placebo
Arm/Group Description	OC-01: OC-01 (varenicline) nasal spray	Placebo: Placebo Vehicle ) nasal spray
Measure Participants	7	3
Mean (Standard Deviation) [um]	-38.8 (30.20)	-6.7 (1.94)

2. Primary Outcome

Title	Mean Change in Goblet Cell Perimeter
Description	Mean change in Goblet Cell Perimeter. A larger decrease is indicative of a better outcome.
Time Frame	Baseline (pre-treatment), 1 day (post treatment)

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	OC-01, 1.2 mg/mL	Placebo
Arm/Group Description	OC-01: OC-01 (varenicline) nasal spray	Placebo: Placebo Vehicle ) nasal spray
Measure Participants	7	3
Mean (Standard Deviation) [um]	-7.7 (5.79)	-1.8 (0.76)

3. Primary Outcome

Title	Change in Meibomian Gland Area, Upper Lid
Description	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)

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	OC-01, 1.2 mg/mL	Placebo
Arm/Group Description	OC-01: OC-01 (varenicline) nasal spray	Placebo: Placebo Vehicle ) nasal spray
Measure Participants	10	6
Mean (Standard Deviation) [um]	11.2 (118.6)	-143.2 (154.8)

4. Primary Outcome

Title	Mean Change in Meibomian Gland Area, Lower Lid.
Description	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)

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	OC-01, 1.2 mg/mL	Placebo
Arm/Group Description	OC-01: OC-01 (varenicline) nasal spray	Placebo: Placebo Vehicle ) nasal spray
Measure Participants	9	6
Mean (Standard Deviation) [um]	-29.9 (88.1)	-12.4 (95.8)

5. Primary Outcome

Title	Mean Change in Meibomian Gland Perimeter, Upper Lid.
Description	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)

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	OC-01, 1.2 mg/mL	Placebo
Arm/Group Description	OC-01: OC-01 (varenicline) nasal spray	Placebo: Placebo Vehicle ) nasal spray
Measure Participants	10	6
Mean (Standard Deviation) [um]	6.15 (16.6)	-19.2 (22.8)

6. Primary Outcome

Title	Mean Change in Meibomian Gland Perimeter, Lower Lid
Description	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)

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	OC-01, 1.2 mg/mL	Placebo
Arm/Group Description	OC-01: OC-01 (varenicline) nasal spray	Placebo: Placebo Vehicle ) nasal spray
Measure Participants	9	6
Mean (Standard Deviation) [um]	-4 (10.9)	4.2 (19.3)

Adverse Events

Time Frame	Adverse events were collected from the time of study drug administration to the last study visit (pre to post treatment 1 day).
Arm/Group Title	OC-01, 1.2 mg/mL		Placebo
Arm/Group Description	OC-01 (varenicline) nasal spray, 1.2 mg/mL		Placebo (vehicle control) nasal spray
All Cause Mortality
	OC-01, 1.2 mg/mL		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/12 (0%)		0/6 (0%)
Serious Adverse Events
	OC-01, 1.2 mg/mL		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/12 (0%)		0/6 (0%)
Other (Not Including Serious) Adverse Events
	OC-01, 1.2 mg/mL		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	6/12 (50%)		1/6 (16.7%)
Eye disorders
TEAE	0/12 (0%)	0	0/6 (0%)	0
Respiratory, thoracic and mediastinal disorders
Sneezing	5/12 (41.7%)		1/6 (16.7%)
Nasal discomfort	2/12 (16.7%)		0/6 (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.