Dry Eye DiseasePhase 2NCT03688802

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

A Phase 2 study testing OC-01 (varenicline) nasal spray in people with Dry Eye Disease, run by Oyster Point Pharma, Inc. at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Dry Eye Disease
What is being tested?: OC-01 (varenicline) nasal spray
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 18 participants
Where: 1 siteUnited States
Study dates: 25 Sep 2018to 30 Sep 2019
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Have used and/or desired to use an artificial tear substitute for dry eye symptoms within 6 months prior to visit 1.

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

United States1 site

Tufts
Boston, Massachusetts, 02111

Other trials in United States

About this study

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)

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.

Read the 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

Conditions: Dry Eye Disease
Being tested: OC-01 (varenicline) nasal spray, Placebo (vehicle control) nasal spray
Medical subject headings: Dry Eye Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

OC-01 (varenicline) nasal sprayDrug
OC-01 (varenicline) nasal spray
Placebo (vehicle control) nasal sprayDrug
Placebo

Study groups in NCT03688802
Study group	Role	What they receive
OC-01 (varenicline) nasal spray, 1.2 mg/mL	Active comparator	OC-01 (varenicline) nasal spray, 1.2 mg/mL, one time dosing Receives: Drug: OC-01 (varenicline) nasal spray
Placebo (vehicle control) nasal spray	Placebo comparator	Placebo (vehicle control) nasal spray, one time dosing Receives: Drug: Placebo (vehicle control) nasal spray

What is being measured

The primary outcome is the main question the study is designed to answer.

Mean Change in Goblet Cell Area
Mean change in Goblet Cell Area with a larger decrease is indicative of a better outcome.
Measured: Baseline (pre-treatment), 1 day (post treatment)
Mean Change in Goblet Cell Perimeter
Mean change in Goblet Cell Perimeter. A larger decrease is indicative of a better outcome.
Measured: Baseline (pre-treatment), 1 day (post treatment)
Change in Meibomian Gland Area, Upper Lid
Change in Meibomian gland area, upper lid. A larger decrease is indicative of a better outcome
Measured: Baseline (pre-treatment), 1 day (post treatment)
Mean Change in Meibomian Gland Area, Lower Lid.
Mean change in Meibomian Gland Area, lower Lid. A larger decrease is indicative of a better outcome.
Measured: Baseline (pre-treatment), 1 day (post treatment)
Mean Change in Meibomian Gland Perimeter, Upper Lid.
Chane in Meibomian Gland Perimeter, upper lid. A larger decrease is indicative of a better outcome.
Measured: Baseline (pre-treatment), 1 day (post treatment)
Mean Change in Meibomian Gland Perimeter, Lower Lid
Mean change in Meibomian Gland Perimeter, lower lid. A larger decrease is indicative of a better outcome.
Measured: Baseline (pre-treatment), 1 day (post treatment)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

25 Sep 201830 Sep 2019

First posted28 Sep 2018
Study start25 Sep 2018
Primary completion30 Sep 2019
Study completion30 Sep 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Dry Eye DiseaseStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 18 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03688802
Sponsor's protocol ID: OPP-005
Status last verified: Nov 2021
First submitted: 26 Sep 2018
Last updated: 29 Dec 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Dieckmann GM, Cox SM, Lopez MJ, Ozmen MC, Yavuz Saricay L, Bayrakutar BN, Binotti WW, Henry E, Nau J, Hamrah P. A Single Administration of OC-01 (Varenicline Solution) Nasal Spray Induces Short-Term Alterations in Conjunctival Goblet Cells in Patients with Dry Eye Disease. Ophthalmol Ther. 2022 Aug;11(4):1551-1561. doi: 10.1007/s40123-022-00530-x. Epub 2022 Jun 2.
PubMed 35653029

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 29 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

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)
ClinicalTrials.gov ID: NCT03688802
Acronym / sponsor ID: OPP-005
Conditions: Dry Eye Disease
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 18 participants (actual)
Lead sponsor: Oyster Point Pharma, Inc.Industry (pharma, biotech, devices)
Study start: 25 Sep 2018 (actual)
Primary completion: 30 Sep 2019 (actual)
Study completion: 30 Sep 2019 (actual)
First posted: 28 Sep 2018 (actual)
Results: Posted 29 Dec 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
OC-01 (varenicline) nasal spray, 1.2 mg/mL	Active comparator	Drug: OC-01 (varenicline) nasal sprayOC-01 (varenicline) nasal spray, 1.2 mg/mL, one time dosing
Placebo (vehicle control) nasal spray	Placebo comparator	Drug: Placebo (vehicle control) nasal sprayPlacebo (vehicle control) nasal spray, one time dosing

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (1 listed)

Have used and/or desired to use an artificial tear substitute for dry eye symptoms within 6 months prior to visit 1.

Key exclusion criteria (5 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
OC-01 (varenicline) nasal sprayOC-01 (varenicline) nasal spray	Drug	OC-01 (varenicline) nasal spray, 1.2 mg/mL
Placebo (vehicle control) nasal sprayPlacebo	Drug	Placebo (vehicle control) nasal spray

Primary outcome

Mean Change in Goblet Cell Area
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Mean change in Goblet Cell Area with a larger decrease is indicative of a better outcome.
Mean Change in Goblet Cell Perimeter
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Mean change in Goblet Cell Perimeter. A larger decrease is indicative of a better outcome.
Change in Meibomian Gland Area, Upper Lid
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Change in Meibomian gland area, upper lid. A larger decrease is indicative of a better outcome
Mean Change in Meibomian Gland Area, Lower Lid.
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Mean change in Meibomian Gland Area, lower Lid. A larger decrease is indicative of a better outcome.
Mean Change in Meibomian Gland Perimeter, Upper Lid.
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Chane in Meibomian Gland Perimeter, upper lid. A larger decrease is indicative of a better outcome.
Mean Change in Meibomian Gland Perimeter, Lower Lid
Time frame: Baseline (pre-treatment), 1 day (post treatment)
Mean change in Meibomian Gland Perimeter, lower lid. A larger decrease is indicative of a better outcome.

Results and safety

Results posted to the registry on 29 Dec 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
OC-01 (Varenicline) Nasal Spray, 1.2 mg/mL	12	11
Placebo (Vehicle Control) Nasal Spray	6	6

Primary outcome: Mean Change in Goblet Cell Area

Time frame: Baseline (pre-treatment), 1 day (post treatment). Mean (Standard Deviation). Unit: um

Measure	OC-01, 1.2 mg/mL n = 7	Placebo n = 3
Value	-38.8 (30.20)	-6.7 (1.94)

Population analysed: 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.

Primary outcome: Mean Change in Goblet Cell Perimeter

Time frame: Baseline (pre-treatment), 1 day (post treatment). Mean (Standard Deviation). Unit: um

Measure	OC-01, 1.2 mg/mL n = 7	Placebo n = 3
Value	-7.7 (5.79)	-1.8 (0.76)

Population analysed: 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.

Primary outcome: Change in Meibomian Gland Area, Upper Lid

Time frame: Baseline (pre-treatment), 1 day (post treatment). Mean (Standard Deviation). Unit: um

Measure	OC-01, 1.2 mg/mL n = 10	Placebo n = 6
Value	11.2 (118.6)	-143.2 (154.8)

Population analysed: 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.

Primary outcome: Mean Change in Meibomian Gland Area, Lower Lid.

Time frame: Baseline (pre-treatment), 1 day (post treatment). Mean (Standard Deviation). Unit: um

Measure	OC-01, 1.2 mg/mL n = 9	Placebo n = 6
Value	-29.9 (88.1)	-12.4 (95.8)

Population analysed: 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.

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).. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
OC-01, 1.2 mg/mL	0 of 12 (0%)	6 of 12 (50%)	0 of 12
Placebo	0 of 6 (0%)	1 of 6 (16.7%)	0 of 6

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 29 Dec 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 35653029Dieckmann GM, Cox SM, Lopez MJ, Ozmen MC, Yavuz Saricay L, Bayrakutar BN, Binotti WW, Henry E, Nau J, Hamrah P. A Single Administration of OC-01 (Varenicline Solution) Nasal Spray Induces Short-Term Alterations in Conjunctival Goblet Cells in Patients with Dry Eye Disease. Ophthalmol Ther. 2022 Aug;11(4):1551-1561. doi: 10.1007/s40123-022-00530-x. Epub 2022 Jun 2.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing OC-01 (varenicline) nasal spray in people with Dry Eye Disease, run by Oyster Point Pharma, Inc. at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 Dec 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Mean Change in Goblet Cell Area (time frame: Baseline (pre-treatment), 1 day (post treatment)). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03688802, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Dec 2021.