Trial Outcomes & Findings for Clinical Trial to Evaluate the Chronic Efficacy of OC-01 Nasal Spray on Signs of Dry Eye Disease (The MYSTIC Study) (NCT NCT03873246)
NCT ID: NCT03873246
Last Updated: 2022-06-08
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
123 participants
Primary outcome timeframe
Visit 1 (baseline) and Visit 6 (84 days)
Results posted on
2022-06-08
Participant Flow
Participant milestones
| Measure |
OC-01 0.1%, 0.6 mg/ml
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
OC-01 0.2%, 1.2 mg/ml
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
vehicle control
placebo comparator: vehicle control
|
|---|---|---|---|
|
Overall Study
STARTED
|
41
|
41
|
41
|
|
Overall Study
COMPLETED
|
36
|
29
|
32
|
|
Overall Study
NOT COMPLETED
|
5
|
12
|
9
|
Reasons for withdrawal
| Measure |
OC-01 0.1%, 0.6 mg/ml
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
OC-01 0.2%, 1.2 mg/ml
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
vehicle control
placebo comparator: vehicle control
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
3
|
3
|
|
Overall Study
Lost to Follow-up
|
4
|
4
|
5
|
|
Overall Study
Protocol Violation
|
0
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
0
|
4
|
1
|
Baseline Characteristics
Clinical Trial to Evaluate the Chronic Efficacy of OC-01 Nasal Spray on Signs of Dry Eye Disease (The MYSTIC Study)
Baseline characteristics by cohort
| Measure |
OC-01 0.1%, 0.6 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
OC-01 0.2%, 1.2 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=41 Participants
vehicle control
placebo comparator: vehicle control
|
Total
n=123 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
51.4 years
STANDARD_DEVIATION 13.18 • n=5 Participants
|
54.2 years
STANDARD_DEVIATION 11.77 • n=7 Participants
|
55.8 years
STANDARD_DEVIATION 26.77 • n=5 Participants
|
53.8 years
STANDARD_DEVIATION 12.75 • n=4 Participants
|
|
Sex: Female, Male
Female
|
32 Participants
n=5 Participants
|
35 Participants
n=7 Participants
|
33 Participants
n=5 Participants
|
100 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
23 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Race, Other
|
41 Participants
n=5 Participants
|
41 Participants
n=7 Participants
|
41 Participants
n=5 Participants
|
123 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
41 Participants
n=5 Participants
|
41 Participants
n=7 Participants
|
41 Participants
n=5 Participants
|
123 Participants
n=4 Participants
|
|
Baseline Schirmer's Test Score
|
5.5 mm
STANDARD_DEVIATION 2.46 • n=5 Participants
|
5.4 mm
STANDARD_DEVIATION 2.47 • n=7 Participants
|
5.3 mm
STANDARD_DEVIATION 2.04 • n=5 Participants
|
5.4 mm
STANDARD_DEVIATION 2.33 • n=4 Participants
|
PRIMARY outcome
Timeframe: Visit 1 (baseline) and Visit 6 (84 days)Population: Subjects in the ITT Population-LOCF population
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.
Outcome measures
| Measure |
OC-01 0.2%, 1.2 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=41 Participants
vehicle control
placebo comparator: vehicle control
|
OC-01 0.1%, 0.6 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
|---|---|---|---|
|
Mean Change in Schirmer's Test Score From Baseline to 84 Days
|
11.0 mm
Standard Error 1.29
|
6.0 mm
Standard Error 1.29
|
10.8 mm
Standard Error 1.29
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Visit 1 (baseline) and Visit 6 (84 days)Population: Subjects in the ITT-LOCF population
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.
Outcome measures
| Measure |
OC-01 0.2%, 1.2 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=41 Participants
vehicle control
placebo comparator: vehicle control
|
OC-01 0.1%, 0.6 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
|---|---|---|---|
|
Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 6 (Day 84)
|
20 Participants
|
10 Participants
|
15 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Visit 1 (baseline) and Visit 4 (28 Days)Population: Subjects in the ITT-LOCF patient population.
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.
Outcome measures
| Measure |
OC-01 0.2%, 1.2 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=41 Participants
vehicle control
placebo comparator: vehicle control
|
OC-01 0.1%, 0.6 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
|---|---|---|---|
|
Percent of Subjects Who Achieve ≥10 mm Improvement in Schirmer's Test Score From Baseline at Visit 4 (Day 28)
|
17 Participants
|
12 Participants
|
18 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Visit 1 (baseline) to Visit 6 (84 Days)Population: Subjects in the ITT-LOCF population
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.
Outcome measures
| Measure |
OC-01 0.2%, 1.2 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=41 Participants
vehicle control
placebo comparator: vehicle control
|
OC-01 0.1%, 0.6 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
|---|---|---|---|
|
Mean Change in Schirmer's Test Score From Baseline to 84 Days (Visit 6) - Fellow Eye
|
8.7 mm
Standard Error 1.18
|
4.8 mm
Standard Error 1.18
|
7.9 mm
Standard Error 1.19
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Visit 1 (baseline) to Visit 4 (28 days)]Population: Subjects in the ITT-LOCF population
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.
Outcome measures
| Measure |
OC-01 0.2%, 1.2 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=41 Participants
vehicle control
placebo comparator: vehicle control
|
OC-01 0.1%, 0.6 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
|---|---|---|---|
|
Mean Change in Schirmer's Test Score From Baseline to 28 Days (Visit 4) - Study Eye
|
11.4 mm
Standard Error 1.42
|
6.9 mm
Standard Error 1.42
|
10.2 mm
Standard Error 1.42
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Visit 1 (baseline) to Visit 4 (28 Days)Population: Subjects in the ITT-LOCF population
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.
Outcome measures
| Measure |
OC-01 0.2%, 1.2 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=41 Participants
vehicle control
placebo comparator: vehicle control
|
OC-01 0.1%, 0.6 mg/ml
n=41 Participants
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
|---|---|---|---|
|
Mean Change in Schirmer's Test Score From Baseline to 28 Days (Visit 4) - Fellow Eye
|
9.2 mm
Standard Error 1.35
|
6.7 mm
Standard Error 1.35
|
5.4 mm
Standard Error 1.36
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Visit 1 (baseline) and Visit 5 (56 days)Population: Subjects in the ITT population
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.
Outcome measures
| Measure |
OC-01 0.2%, 1.2 mg/ml
n=30 Participants
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=31 Participants
vehicle control
placebo comparator: vehicle control
|
OC-01 0.1%, 0.6 mg/ml
n=35 Participants
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
|---|---|---|---|
|
Mean Change in Total Corneal Fluorescein Staining
|
-3.7 units on a scale
Standard Error 0.6
|
-3.2 units on a scale
Standard Error 0.59
|
-3.3 units on a scale
Standard Error 0.56
|
Adverse Events
OC-01 0.1%, 0.6 mg/ml
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
OC-01 0.2%, 1.2 mg/ml
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
OC-01 0.1%, 0.6 mg/ml
n=41 participants at risk
OC-01 (varenicline) nasal spray, 0.6 mg/mL
OC-01 0.1% 0.6 mg/ml: OC-01 (varenicline) nasal spray: 0.1 % (0.6 mg/ml)
|
OC-01 0.2%, 1.2 mg/ml
n=41 participants at risk
OC-01 (varenicline) nasal spray, 1.2 mg/mL
OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml): OC-01 (varenicline) nasal spray: 0.2 % (1.2 mg/ml)
|
Placebo
n=41 participants at risk
vehicle control
placebo comparator: vehicle control
|
|---|---|---|---|
|
Eye disorders
Ocular TEAEs
|
9.8%
4/41 • Number of events 4 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
12.2%
5/41 • Number of events 5 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
9.8%
4/41 • Number of events 4 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Eye disorders
Visual acuity reduced
|
9.8%
4/41 • Number of events 4 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
7.3%
3/41 • Number of events 3 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
7.3%
3/41 • Number of events 3 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Eye disorders
Conjunctival hyperaemia
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
2.4%
1/41 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Eye disorders
Eye irritation
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
2.4%
1/41 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Eye disorders
Eyelid oedema
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
2.4%
1/41 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Vascular disorders
Hyperaemia
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
2.4%
1/41 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
2.4%
1/41 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
4.9%
2/41 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
7.3%
3/41 • Number of events 3 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
4.9%
2/41 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
4.9%
2/41 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
4.9%
2/41 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Nervous system disorders
Headache
|
4.9%
2/41 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
4.9%
2/41 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/41 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
2.4%
1/41 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
4.9%
2/41 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 6 (84 days).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place