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NCT04036292

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

Completed Phase 3 Results posted Last updated 22 November 2021
What this trial tests

Phase 3 trial testing OC-01 (varenicline) nasal spray in Dry Eye Disease (DED) in 758 participants. Completed in 5 February 2021.

Timeline
23 July 2019
Primary endpoint
10 April 2020
5 February 2021

Quick facts

Lead sponsorOyster Point Pharma, Inc.
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingdouble
Primary purposetreatment
Enrollment758
Start date23 July 2019
Primary completion10 April 2020
Estimated completion5 February 2021
Sites22 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Oyster Point Pharma, Inc. — full company profile →

Who can join

22 and older, any sex, with Dry Eye Disease (DED). Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28) Primary · 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.

GroupValue95% CI
OC-01 Low Dose, 0.6 mg/mL123
OC-01 High Dose, 1.2 mg/mL121
Placebo (Vehicle) Nasal Spray70
Mean Change From Baseline in Eye Dryness Score in CAE at Week 4 at 5 Minutes Secondary · 28 Days [Visit 1 (baseline) and Visit 4a (28 days)]

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.

GroupValue95% CI
OC-01 Low Dose, 0.6 mg/mL-10.3± 1.62
OC-01 High Dose, 1.2 mg/mL-9.0± 1.75
Placebo-7.4± 1.74
Mean Change From Baseline in Eye Dryness Score From Baseline to Day 28 Secondary · 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]

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.

GroupValue95% CI
OC-01 Low Dose, 0.6 mg/mL-19.8± 1.54
OC-01 High Dose, 1.2 mg/mL-22.2± 1.61
Placebo-15.4± 1.57
Mean Change Form Baseline in Schirmer's Test Score From Baseline to Day 28 Secondary · 28 Days [Visit 1 (baseline) and Visit 4b (28 days)]

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.

GroupValue95% CI
OC-01 Low Dose, 0.6 mg/mL11.3± 0.61
OC-01 High Dose, 1.2 mg/mL11.5± 0.64
Placebo6.3± 0.61
Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 2 Secondary · 14 Days [Visit 1 (baseline) and Visit 3 (14 days)]

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.

GroupValue95% CI
OC-01 Low Dose, 0.6 mg/mL-16.5± 1.38
OC-01 High Dose, 1.2 mg/mL-17.9± 1.45
Placebo-12.7± 1.41
Mean Change From Baseline in Eye Dryness Score in the Study Eye at Week 1 Secondary · 7 Days [Visit 1 (baseline) and Visit 2 (7 days)]

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.

GroupValue95% CI
OC-01 Low Dose, 0.6 mg/mL-15.7± 1.33
OC-01 High Dose, 1.2 mg/mL-15.4± 1.40
Placebo-13.3± 1.35

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 7. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

OC-01 Low Dose, 0.6 mg/mL
Serious: 5/260 (2%)
Deaths: 0/260
OC-01 High Dose, 1.2 mg/mL
Serious: 12/245 (5%)
Deaths: 2/245
Placebo
Serious: 9/251 (4%)
Deaths: 1/251

Serious adverse events (26 terms)

ReactionSystemOC-01 Low Dose, 0.6 mg/mLOC-01 High Dose, 1.2 mg/mLPlacebo
Coronavirus infectionInfections and infestations
SepsisInfections and infestations
Diabetic gangreneInfections and infestations
GangreneInfections and infestations
OsteomyelitisInfections and infestations
PneumoniaInfections and infestations
Pneumonia bacterialInfections and infestations
Atrial fibrillationCardiac disorders
Atrioventricular block completeCardiac disorders
BradycardiaCardiac disorders
Coronary artery diseaseCardiac disorders
Myocardial infarctionCardiac disorders
Intervertebral disc protrusionMusculoskeletal and connective tissue disorders
Lumbar spinal stenosisMusculoskeletal and connective tissue disorders
Lung neoplasm malignantNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Pancreatic carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Cerebrovascular accidentNervous system disorders
Transient ischemic attackNervous system disorders
Deep vein thrombosisVascular disorders
Hypertensive urgencyVascular disorders
Arnold-Chiari malformationCongenital, familial and genetic disorders
Umbilical herniaGastrointestinal disorders
Cholecystitis acuteHepatobiliary disorders
Humerus fractureInjury, poisoning and procedural complications
Renal haematomaRenal and urinary disorders
Other adverse events (86 terms — click to expand)

ReactionSystemOC-01 Low Dose, 0.6 mg/mLOC-01 High Dose, 1.2 mg/mLPlacebo
SneezingRespiratory, thoracic and mediastinal disorders
CoughRespiratory, thoracic and mediastinal disorders
Throat irritationRespiratory, thoracic and mediastinal disorders
Instillation site irritationGeneral disorders
NasopharyngitisInfections and infestations
conjunctival hyperaemiaEye disorders
Nasal congestionRespiratory, thoracic and mediastinal disorders
Visual acuity reducedEye disorders
Nasal mucosal disorderRespiratory, thoracic and mediastinal disorders
EpistaxisRespiratory, thoracic and mediastinal disorders
RhinorrhoeaRespiratory, thoracic and mediastinal disorders
HeadacheNervous system disorders
Nasal discomfortRespiratory, thoracic and mediastinal disorders
SinusitisInfections and infestations
Upper respiratory tract infectionInfections and infestations
DysgeusiaNervous system disorders
NauseaGastrointestinal disorders
Coronavirus test positiveInvestigations
HypertensionVascular disorders
BlepharitisEye disorders
PingueculaEye disorders
CataractEye disorders
Conjunctival haemorrageEye disorders
Eye irritationEye disorders
Posterior capsule opacificationEye disorders
HordeolumInfections and infestations
Nasal inflammationRespiratory, thoracic and mediastinal disorders
Nasal crustingRespiratory, thoracic and mediastinal disorders
Nasal drynessRespiratory, thoracic and mediastinal disorders
Upper-airway cough syndromeRespiratory, thoracic and mediastinal disorders
Oropharyngeal discomfortRespiratory, thoracic and mediastinal disorders
InfluenzaInfections and infestations
Urinary tract infectionInfections and infestations
BronchitisInfections and infestations
PneumoniaInfections and infestations
MigraineNervous system disorders
DizzinessNervous system disorders
Abdominal pain upperGastrointestinal disorders
OsteoarthritisMusculoskeletal and connective tissue disorders
RashSkin and subcutaneous tissue disorders

Most-reported serious reactions: Coronavirus infection, Sepsis, Diabetic gangrene, Gangrene, Osteomyelitis, Pneumonia, Pneumonia bacterial, Atrial fibrillation.

Data from ClinicalTrials.gov NCT04036292 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. 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.
    Wirta D, Vollmer P, Paauw J, Chiu KH, et al · · 2022 · cited 74× · PMID 34767866 · DOI 10.1016/j.ophtha.2021.11.004
  2. Does Dry Eye Disease Severity Impact Efficacy of Varenicline Solution Nasal Spray on Sign and Symptom Treatment Outcomes?
    Sheppard JD, O'Dell LE, Karpecki PM, Raizman MB, et al · · 2023 · cited 9× · PMID 36728653 · DOI 10.1097/opx.0000000000001986
  3. 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.
    Nijm LM, Zhu D, Hemphill M, Blemker GL, et al · · 2023 · cited 7× · PMID 36401081 · DOI 10.1007/s40123-022-00607-7
  4. Safety, adherence and discontinuation in varenicline solution nasal spray clinical trials for dry eye disease.
    Hauswirth SG, Kabat AG, Hemphill M, Somaiya K, et al · · 2023 · cited 5× · PMID 37096956 · DOI 10.57264/cer-2022-0215
  5. Matching-adjusted indirect comparison of phase 3 clinical trial outcomes: OC-01 (varenicline solution) nasal spray and cyclosporine a 0.05% ophthalmic emulsion for the treatment of dry eye disease.
    Visco DM, Hendrix LH, Sun L, Tam I, et al · · 2022 · cited 5× · PMID 35687793 · DOI 10.18553/jmcp.2022.22005
  6. OC-01 (Varenicline Solution) Nasal Spray for the Treatment of Dry Eye Disease Signs and Symptoms in Subjects with Autoimmune Disease: Integrated Data from ONSET-1 and ONSET-2.
    Schallhorn JM, McGee S, Nau J, Macsai M, et al · · 2023 · cited 3× · PMID 36895950 · DOI 10.2147/opth.s403953
  7. OC-01 (Varenicline Solution) Nasal Spray Demonstrates Consistency of Effect Regardless of Age, Race, Ethnicity, and Artificial Tear Use.
    Epitropoulos AT, Daya SM, Matossian C, Kabat AG, et al · · 2022 · cited 2× · PMID 36249445 · DOI 10.2147/opth.s383091
  8. Corneal Fluorescein Staining in FDA-Reviewed Dry Eye Registrational Clinical Trials: Evidence of Limited Endpoint Responsiveness.
    Dabre Z, Mun C, Sheth T, Kim C, et al · · 2026 · PMID 41493896 · DOI 10.1159/000550166

Verify or expand the search:

Other trials of OC-01 (varenicline) nasal spray

Trials testing the same drug.

Other recruiting trials for Dry Eye Disease (DED)

Currently open trials in the same condition.

Other Oyster Point Pharma, Inc. trials

Trials by the same sponsor.

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04036292.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing