NCT03873246

Clinical Trial to Evaluate the Chronic Efficacy of OC-01 Nasal Spray on Signs of Dry Eye Disease (The MYSTIC Study)

Completed Phase 2 Results posted Last updated 8 June 2022

What this trial tests

Phase 2 trial testing OC-01 0.1% 0.6 mg/ml in Dry Eye Disease in 123 participants. Completed in 7 January 2020.

Timeline

18 February 2019

Primary endpoint
7 January 2020

7 January 2020

Quick facts

Lead sponsor	Oyster Point Pharma, Inc.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	123
Start date	18 February 2019
Primary completion	7 January 2020
Estimated completion	7 January 2020
Sites	1 location across Mexico

Drugs / interventions tested

• OC-01 0.1% 0.6 mg/ml — full drug profile →
• OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml) — full drug profile →
• placebo comparator

Conditions studied

• Dry Eye Disease — all drugs for Dry Eye Disease →

Sponsor

Oyster Point Pharma, Inc. — full company profile →

Who can join

22 and older, any sex, with Dry Eye Disease. 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.

Mean Change in Schirmer's Test Score From Baseline to 84 Days Primary · Visit 1 (baseline) and Visit 6 (84 days)

The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.

Group	Value	95% CI
OC-01 0.1%, 0.6 mg/ml	10.8	± 1.29
OC-01 0.2%. 1.2 mg/ml	11.0	± 1.29
Placebo	6.0	± 1.29

Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 6 (Day 84) Secondary · Visit 1 (baseline) and Visit 6 (84 days)

The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.

Group	Value	95% CI
OC-01 0.1%, 0.6 mg/ml	15
OC-01 0.2%, 1.2 mg/ml	20
Placebo	10

Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28) Secondary · Visit 1 (baseline) and Visit 4 (28 Days)

The Schirmer's test measures the amount of tears produced by placing a paper strip in the eye for 5 minutes and the distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.

Group	Value	95% CI
OC-01 0.1%, 0.6 mg/ml	18
OC-01 0.2%. 1.2 mg/ml	17
Placebo	12

Mean Change in Schirmer's Test Score From Baseline to 84 Days (Visit 6) - Fellow Eye Secondary · Visit 1 (baseline) to Visit 6 (84 Days)

The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.

Group	Value	95% CI
OC-01 0.1%, 0.6 mg/ml	7.9	± 1.19
OC-01 0.2%. 1.2 mg/ml	8.7	± 1.18
Placebo	4.8	± 1.18

Mean Change in Schirmer's Test Score From Baseline to 28 Days (Visit 4) - Study Eye Secondary · Visit 1 (baseline) to Visit 4 (28 days)]

The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment of OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.

Group	Value	95% CI
OC-01 0.1%, 0.6 mg/ml	10.2	± 1.42
OC-01 0.2%. 1.2 mg/ml	11.4	± 1.42
Placebo	6.9	± 1.42

Mean Change in Schirmer's Test Score From Baseline to 28 Days (Visit 4) - Fellow Eye Secondary · Visit 1 (baseline) to Visit 4 (28 Days)

The Schirmer's test measures the amount of tears produced by numbing the eye and placing a paper strip in the eye for 5 minutes and distance of wetting was recorded following treatment with OC-01 or placebo. Schirmer's test scores range from 0-35 mm where a higher score is indicative of a better outcome.

Group	Value	95% CI
OC-01 0.1%, 0.6 mg/ml	5.4	± 1.36
OC-01 0.2%. 1.2 mg/ml	9.2	± 1.35
Placebo	6.7	± 1.35

Mean Change in Total Corneal Fluorescein Staining Secondary · Visit 1 (baseline) and Visit 5 (56 days)

A standardized grading system of 0-3 is used for each of the five areas of the cornea (inferior, superior, nasal, temporal, central). Grade 0 will be specified when no staining is present. The total score ranges from 0-15. Lower scores indicate improvement.

Group	Value	95% CI
OC-01 0.1%, 0.6 mg/ml	-3.3	± 0.56
OC-01 0.2%, 1.2 mg/ml	-3.7	± 0.6
Placebo	-3.2	± 0.59

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 6 (84 days).. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

OC-01 0.1%, 0.6 mg/ml

Serious: 0/41 (0%)

Deaths: 0/41

OC-01 0.2%, 1.2 mg/ml

Serious: 0/41 (0%)

Deaths: 0/41

Placebo

Serious: 0/41 (0%)

Deaths: 0/41

Other adverse events (11 terms — click to expand)

Reaction	System	OC-01 0.1%, 0.6 mg/ml	OC-01 0.2%, 1.2 mg/ml	Placebo
Ocular TEAEs	Eye disorders	—	—	—
Visual acuity reduced	Eye disorders	—	—	—
Sneezing	Respiratory, thoracic and mediastinal disorders	—	—	—
Epistaxis	Respiratory, thoracic and mediastinal disorders	—	—	—
Throat irritation	Respiratory, thoracic and mediastinal disorders	—	—	—
Headache	Nervous system disorders	—	—	—
Nausea	Gastrointestinal disorders	—	—	—
Conjunctival hyperaemia	Eye disorders	—	—	—
Eye irritation	Eye disorders	—	—	—
Eyelid oedema	Eye disorders	—	—	—
Hyperaemia	Vascular disorders	—	—	—

Data from ClinicalTrials.gov NCT03873246 adverse events section.

Sponsor's own description

The objective of this study is to evaluate the chronic safety and effectiveness of OC-01 Nasal Spray as compared to placebo on signs of dry eye disease (DED).

Publications & conference data

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

1. A phase II randomized trial to evaluate the long-term (12-week) efficacy and safety of OC-01 (varenicline solution) nasal spray for dry eye disease: The MYSTIC study.
   Quiroz-Mercado H, Hernandez-Quintela E, Chiu KH, Henry E, et al · · 2022 · cited 31× · PMID 34920097 · DOI 10.1016/j.jtos.2021.12.007
2. Tumour- and Non-Tumour-Associated Factors That Modulate Response to PD-1/PD-L1 Inhibitors in Non-Small Cell Lung Cancer.
   Khalil M, Tsao MS. · · 2025 · cited 5× · PMID 40647497 · DOI 10.3390/cancers17132199
3. 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