Dry Eye Disease (DED)Phase 3NCT04036292
Evaluation of the Efficacy and Safety of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease
A Phase 3 study testing OC-01 (varenicline) nasal spray in people with Dry Eye Disease (DED), run by Oyster Point Pharma, Inc. at 22 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 22 years and older
- Conditions the study is for
- Dry Eye Disease (DED)
- What is being tested?
- OC-01 (varenicline) nasal spray
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 758 participants
- Where
- 22 sitesUnited States
- Study dates
- 23 Jul 2019to 5 Feb 2021
- Phase
- Phase 3
- 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 22 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) or extraocular surgery 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 dry eye disease 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
22 sites in 1 country.
22 sites in 1 country
United States22 sites
Austin
Austin, Texas, 78731
Cranberry Township
Cranberry Township, Pennsylvania, 16066
Delray Beach
Delray Beach, Florida, 33484
Fargo
Fargo, North Dakota, 58103
Fort Collins
Fort Collins, Colorado, 80528
Houston
Houston, Texas, 77025
Indianapolis
Indianapolis, Indiana, 46290
Las Vegas
Las Vegas, Nevada, 89052
Layton
Layton, Utah, 84041
Louisville
Louisville, Kentucky, 40206
Lynchburg
Lynchburg, Virginia, 24502
Nashville
Nashville, Tennessee, 37210
New Port Beach
Newport Beach, California, 92663
Phoenix
Phoenix, Arizona, 85032
Raleigh
Raleigh, North Carolina, 27603
Raynham
Raynham, Massachusetts, 02767
San Antonio
San Antonio, Texas, 78209
San Antonio
San Antonio, Texas, 78229
Shelby
Shelby, North Carolina, 28150
Sioux Falls
Sioux Falls, South Dakota, 57108
Warwick
Warwick, Rhode Island, 02842
Waterbury
Waterbury, Connecticut, 06708
About this study
Official title: Multicenter, Randomized, Controlled, Double-Masked, Clinical Trial to Evaluate the Efficacy and Safety of OC-01 (Varenicline) Nasal Spray on Signs and Symptoms of Dry Eye Disease (The ONSET-2 Study)
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.
Read the detailed description
This was a Phase 3, multicenter, randomized, controlled, double-masked (including subjects, Investigators, study site personnel, and Sponsor personnel) study designed to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray 0.6 mg/mL and 1.2 mg/mL in adult participants with DED. The study randomized 758 subjects at least 22 years of age with a physicians' diagnosis of DED and meeting all other study eligibility criteria to receive OC-01 (varenicline) nasal spray or placebo twice daily (BID) for 28 days with three additional long-term follow-up visits at 6 weeks, 6 months, and 12 months. Participants who terminated from the study during the treatment period were asked to complete safety assessments (if the participants agree) prior to study exit. Participants who were terminated early from the study were not replaced
- Conditions
- Dry Eye Disease (DED)
- Being tested
- OC-01 (varenicline) nasal spray, Placebo (vehicle) 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) nasal sprayDrug
Placebo (vehicle) nasal spray
| Study group | Role | What they receive |
|---|---|---|
| OC-01 Low Dose, 0.6 mg/mL | Active comparator | OC-01 (varenicline) nasal spray, 0.6 mg/ML Receives: Drug: OC-01 (varenicline) nasal spray |
| OC-01 High Dose, 1.2 mg/mL | Active comparator | OC-01 (varenicline) nasal spray, 1.2 mg/ML Receives: Drug: OC-01 (varenicline) nasal spray |
| Placebo (vehicle) nasal spray | Placebo comparator | Placebo (vehicle) nasal spray Receives: Drug: Placebo (vehicle) nasal spray |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28)
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.
Measured: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
Secondary outcomes (5)
Mean Change From Baseline in Eye Dryness Score in CAE at Week 4 at 5 Minutes
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.
Measured: 28 Days [Visit 1 (baseline) and Visit 4a (28 days)]
Mean Change From Baseline in Eye Dryness Score From Baseline to Day 28
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.
Measured: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
Mean Change Form Baseline in Schirmer's Test Score From Baseline to Day 28
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.
Measured: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 2
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.
Measured: 14 Days [Visit 1 (baseline) and Visit 3 (14 days)]
Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 1
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.
Measured: 7 Days [Visit 1 (baseline) and Visit 2 (7 days)]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2019
- Study start23 Jul 2019
- Primary completion10 Apr 2020
- Study completion5 Feb 2021
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 (3)Papers the sponsor has linked to this study.
- Other trials for Dry Eye Disease (DED)Studies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 758 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oyster Point Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04036292
- Sponsor's protocol ID
- OPP-101
- Status last verified
- Aug 2021
- First submitted
- 25 Jul 2019
- Last updated
- 22 Nov 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Hauswirth SG, Kabat AG, Hemphill M, Somaiya K, Hendrix LH, Gibson AA. Safety, adherence and discontinuation in varenicline solution nasal spray clinical trials for dry eye disease. J Comp Eff Res. 2023 Jun;12(6):e220215. doi: 10.57264/cer-2022-0215. Epub 2023 Apr 25.
PubMed 37096956Nijm LM, Zhu D, Hemphill M, Blemker GL, Hendrix LH, Kabat AG, Gibson AA. Does Menopausal Status Affect Dry Eye Disease Treatment Outcomes with OC-01 (Varenicline Solution) Nasal Spray? A Post Hoc Analysis of ONSET-1 and ONSET-2 Clinical Trials. Ophthalmol Ther. 2023 Feb;12(1):355-364. doi: 10.1007/s40123-022-00607-7. Epub 2022 Nov 18.
PubMed 36401081Wirta D, Vollmer P, Paauw J, Chiu KH, Henry E, Striffler K, Nau J; ONSET-2 Study Group. Efficacy and Safety of OC-01 (Varenicline Solution) Nasal Spray on Signs and Symptoms of Dry Eye Disease: The ONSET-2 Phase 3 Randomized Trial. Ophthalmology. 2022 Apr;129(4):379-387. doi: 10.1016/j.ophtha.2021.11.004. Epub 2021 Nov 10.
PubMed 34767866
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Randomized, Controlled, Double-Masked, Clinical Trial to Evaluate the Efficacy and Safety of OC-01 (Varenicline) Nasal Spray on Signs and Symptoms of Dry Eye Disease (The ONSET-2 Study)
- ClinicalTrials.gov ID
- NCT04036292
- Acronym / sponsor ID
- OPP-101
- Conditions
- Dry Eye Disease (DED)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 758 participants (actual)
- Lead sponsor
- Oyster Point Pharma, Inc.Industry (pharma, biotech, devices)
- Study start
- 23 Jul 2019 (actual)
- Primary completion
- 10 Apr 2020 (actual)
- Study completion
- 5 Feb 2021 (actual)
- First posted
- 29 Jul 2019 (actual)
- Results
- Posted 22 Nov 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 on signs and symptoms of dry eye disease.
Detailed description
This was a Phase 3, multicenter, randomized, controlled, double-masked (including subjects, Investigators, study site personnel, and Sponsor personnel) study designed to evaluate the safety and efficacy of OC-01 (varenicline) nasal spray 0.6 mg/mL and 1.2 mg/mL in adult participants with DED. The study randomized 758 subjects at least 22 years of age with a physicians' diagnosis of DED and meeting all other study eligibility criteria to receive OC-01 (varenicline) nasal spray or placebo twice daily (BID) for 28 days with three additional long-term follow-up visits at 6 weeks, 6 months, and 12 months. Participants who terminated from the study during the treatment period were asked to complete safety assessments (if the participants agree) prior to study exit. Participants who were terminated early from the study were not replaced
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| OC-01 Low Dose, 0.6 mg/mL | Active comparator | Drug: OC-01 (varenicline) nasal sprayOC-01 (varenicline) nasal spray, 0.6 mg/ML |
| OC-01 High Dose, 1.2 mg/mL | Active comparator | Drug: OC-01 (varenicline) nasal sprayOC-01 (varenicline) nasal spray, 1.2 mg/ML |
| Placebo (vehicle) nasal spray | Placebo comparator | Drug: Placebo (vehicle) nasal sprayPlacebo (vehicle) nasal spray |
Who may qualify
- Ages
- 22 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) or extraocular surgery 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 dry eye disease 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 High Dose, 1.2 mg/mL, OC-01 Low Dose, 0.6 mg/mL |
| Placebo (vehicle) nasal sprayPlacebo (vehicle) nasal spray | Drug | Placebo (vehicle) nasal spray |
Primary outcome
Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28)
Time frame: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
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.
5 secondary outcomes
Mean Change From Baseline in Eye Dryness Score in CAE at Week 4 at 5 Minutes
Time frame: 28 Days [Visit 1 (baseline) and Visit 4a (28 days)]
Mean Change From Baseline in Eye Dryness Score From Baseline to Day 28
Time frame: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
Mean Change Form Baseline in Schirmer's Test Score From Baseline to Day 28
Time frame: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]
Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 2
Time frame: 14 Days [Visit 1 (baseline) and Visit 3 (14 days)]
Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 1
Time frame: 7 Days [Visit 1 (baseline) and Visit 2 (7 days)]
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37096956Hauswirth SG, Kabat AG, Hemphill M, Somaiya K, Hendrix LH, Gibson AA. Safety, adherence and discontinuation in varenicline solution nasal spray clinical trials for dry eye disease. J Comp Eff Res. 2023 Jun;12(6):e220215. doi: 10.57264/cer-2022-0215. Epub 2023 Apr 25.
- Derived from this study: PubMed 36401081Nijm LM, Zhu D, Hemphill M, Blemker GL, Hendrix LH, Kabat AG, Gibson AA. Does Menopausal Status Affect Dry Eye Disease Treatment Outcomes with OC-01 (Varenicline Solution) Nasal Spray? A Post Hoc Analysis of ONSET-1 and ONSET-2 Clinical Trials. Ophthalmol Ther. 2023 Feb;12(1):355-364. doi: 10.1007/s40123-022-00607-7. Epub 2022 Nov 18.
- Derived from this study: PubMed 34767866Wirta D, Vollmer P, Paauw J, Chiu KH, Henry E, Striffler K, Nau J; ONSET-2 Study Group. Efficacy and Safety of OC-01 (Varenicline Solution) Nasal Spray on Signs and Symptoms of Dry Eye Disease: The ONSET-2 Phase 3 Randomized Trial. Ophthalmology. 2022 Apr;129(4):379-387. doi: 10.1016/j.ophtha.2021.11.004. Epub 2021 Nov 10.
- 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.
22 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing OC-01 (varenicline) nasal spray in people with Dry Eye Disease (DED), run by Oyster Point Pharma, Inc. at 22 sites 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 22 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 22 Nov 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?
Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28) (time frame: 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]). 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 NCT04036292, which also lists 3 linked publications.
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 22 Nov 2021.