Trial Outcomes & Findings for Evaluation of the Efficacy of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease (NCT NCT03636061)
NCT ID: NCT03636061
Last Updated: 2021-10-25
Results Overview
The primary end point was the change in anesthetized Schirmer's Test Score (STS) from baseline to 28 days in the study eye following treatment with OC-01. Schirmer's test score from 0-35 mm where a higher score is indicative of a better outcome.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
182 participants
Primary outcome timeframe
28 Days [Visit 1 (baseline) and Visit 5 (28 days)]
Results posted on
2021-10-25
Participant Flow
Participant milestones
| Measure |
OC-01 Low Dose, 0.12 mg/ml
OC-01 0.12 mg/ml nasal spray BID for 28 days
|
OC-01 Mid Dose, 0.6 mg/ml
OC-01 0.6 mg/ml nasal spray BID for 28 days
|
OC-01 High Dose, 1.2 mg/ml
OC-01 1.2 mg/ml nasal spray BID for 28 days
|
Placebo
Placebo (vehicle) nasal spray
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
47
|
48
|
44
|
43
|
|
Overall Study
COMPLETED
|
47
|
46
|
40
|
43
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
4
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Evaluation of the Efficacy of OC-01 Nasal Spray on Signs and Symptoms of Dry Eye Disease
Baseline characteristics by cohort
| Measure |
OC-01 Low Dose, 0.12 mg/mL
n=47 Participants
OC-01 0.12 mg/ml nasal spray BID for 28 days
|
OC-01 Mid Dose, 0.6 mg/mL
n=48 Participants
OC-01 0.6 mg/ml nasal spray BID for 28 days
|
OC-01 High Dose, 1.2 mg/mL
n=44 Participants
OC-01 1.2 mg/ml nasal spray BID for 28 days
|
Placebo
n=43 Participants
Vehicle nasal spray
|
Total
n=182 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
64.2 years
STANDARD_DEVIATION 12.7 • n=5 Participants
|
66.5 years
STANDARD_DEVIATION 9.4 • n=7 Participants
|
67.4 years
STANDARD_DEVIATION 10.6 • n=5 Participants
|
64 years
STANDARD_DEVIATION 10.3 • n=4 Participants
|
65.5 years
STANDARD_DEVIATION 10.8 • n=21 Participants
|
|
Sex: Female, Male
Female
|
36 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
35 Participants
n=5 Participants
|
32 Participants
n=4 Participants
|
137 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
45 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
18 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
39 Participants
n=5 Participants
|
45 Participants
n=7 Participants
|
42 Participants
n=5 Participants
|
38 Participants
n=4 Participants
|
164 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
8 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
14 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
42 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
40 Participants
n=4 Participants
|
157 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
47 participants
n=5 Participants
|
48 participants
n=7 Participants
|
44 participants
n=5 Participants
|
43 participants
n=4 Participants
|
182 participants
n=21 Participants
|
|
Schirmer's Test Score
|
5.2 mm
STANDARD_DEVIATION 3.1 • n=5 Participants
|
4.8 mm
STANDARD_DEVIATION 2.7 • n=7 Participants
|
5.5 mm
STANDARD_DEVIATION 3.0 • n=5 Participants
|
4.5 mm
STANDARD_DEVIATION 2.9 • n=4 Participants
|
5.0 mm
STANDARD_DEVIATION 2.9 • n=21 Participants
|
PRIMARY outcome
Timeframe: 28 Days [Visit 1 (baseline) and Visit 5 (28 days)]Population: Subjects in ITT-LOCF population
The primary end point was the change in anesthetized Schirmer's Test Score (STS) from baseline to 28 days in the study eye following treatment with OC-01. Schirmer's test score from 0-35 mm where a higher score is indicative of a better outcome.
Outcome measures
| Measure |
OC-01 Low Dose, 0.12 mg/mL
n=47 Participants
OC-01 0.12 mg/ml nasal spray
|
OC-01 Mid Dose, 0.6 mg/ML
n=46 Participants
OC-01 0.6 mg/ml nasal spray
|
OC-01 High Dose, 1.2 mg/mL
n=40 Participants
OC-01 1.2 mg/ml nasal spray
|
Placebo
n=43 Participants
Vehicle nasal spray
|
|---|---|---|---|---|
|
Mean Change in Schirmer's Test Score From Baseline to 28 Days
|
10.1 score on a scale, mm
Standard Error 1.26
|
11.7 score on a scale, mm
Standard Error 1.27
|
11.0 score on a scale, mm
Standard Error 1.39
|
3.2 score on a scale, mm
Standard Error 1.31
|
SECONDARY outcome
Timeframe: 28 days [Visit 1 (baseline and Visit 5 (28 days)]Population: Subjects in the ITT-LOCF population. The overall number of participants is different because these outcomes were assessed ar different visits 7 days apart and some subjects were not able to attend the Day 21 visit.
Change in Eye Dryness Score from baseline to 28 days. Eye Dryness (EDS) on a Visual Analogue Scale (VAS) from 0 (no discomfort) to 100 (maximum discomfort) millimeters where a lower score is indicative of a better outcome.
Outcome measures
| Measure |
OC-01 Low Dose, 0.12 mg/mL
n=47 Participants
OC-01 0.12 mg/ml nasal spray
|
OC-01 Mid Dose, 0.6 mg/ML
n=46 Participants
OC-01 0.6 mg/ml nasal spray
|
OC-01 High Dose, 1.2 mg/mL
n=40 Participants
OC-01 1.2 mg/ml nasal spray
|
Placebo
n=43 Participants
Vehicle nasal spray
|
|---|---|---|---|---|
|
Change From Baseline in Eye Dryness Score From Baseline to Day 28
|
-11.6 score on a scale, mm
Standard Error 3.63
|
-18.9 score on a scale, mm
Standard Error 3.67
|
-15.6 score on a scale, mm
Standard Error 4.02
|
-5.4 score on a scale, mm
Standard Error 3.8
|
SECONDARY outcome
Timeframe: 21 days [Visit 1 (baseline) and Visit 4 (21 days)]Population: Subjects in the ITT population
Change from baseline to Day 21 in Eye Dryness Score at 5 minutes post treatment in the CAE. Eye Dryness (EDS) on a Visual Analogue Scale (VAS) from 0 (no discomfort) to 100 (maximum discomfort) millimeters where a lower score is indicative of a better outcome.
Outcome measures
| Measure |
OC-01 Low Dose, 0.12 mg/mL
n=44 Participants
OC-01 0.12 mg/ml nasal spray
|
OC-01 Mid Dose, 0.6 mg/ML
n=45 Participants
OC-01 0.6 mg/ml nasal spray
|
OC-01 High Dose, 1.2 mg/mL
n=38 Participants
OC-01 1.2 mg/ml nasal spray
|
Placebo
n=42 Participants
Vehicle nasal spray
|
|---|---|---|---|---|
|
Change From Baseline to Day 21 in Eye Dryness Score at 5 Minutes Post Treatment in the CAE.
|
-8.1 score on a scale, mm
Standard Error 2.7
|
-16.0 score on a scale, mm
Standard Error 2.7
|
-18.4 score on a scale, mm
Standard Error 3.0
|
-4.4 score on a scale, mm
Standard Error 2.8
|
Adverse Events
OC-01 Low Dose, 0.12 mg/mL
Serious events: 0 serious events
Other events: 33 other events
Deaths: 0 deaths
OC-01 Mid Dose, 0.6 mg/mL
Serious events: 1 serious events
Other events: 44 other events
Deaths: 0 deaths
OC-01 High Dose, 1.2 mg/mL
Serious events: 0 serious events
Other events: 41 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
OC-01 Low Dose, 0.12 mg/mL
n=47 participants at risk
OC-01 0.12 mg/ml nasal spray
|
OC-01 Mid Dose, 0.6 mg/mL
n=48 participants at risk
OC-01 0.6 mg/ml nasal spray
|
OC-01 High Dose, 1.2 mg/mL
n=44 participants at risk
OC-01 1.2 mg/ml nasal spray
|
Placebo
n=43 participants at risk
Vehicle nasal spray
|
|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.1%
1/48 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/44 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/43 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
Other adverse events
| Measure |
OC-01 Low Dose, 0.12 mg/mL
n=47 participants at risk
OC-01 0.12 mg/ml nasal spray
|
OC-01 Mid Dose, 0.6 mg/mL
n=48 participants at risk
OC-01 0.6 mg/ml nasal spray
|
OC-01 High Dose, 1.2 mg/mL
n=44 participants at risk
OC-01 1.2 mg/ml nasal spray
|
Placebo
n=43 participants at risk
Vehicle nasal spray
|
|---|---|---|---|---|
|
Eye disorders
Ocular TEAEs
|
2.1%
1/47 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
4.2%
2/48 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.3%
1/44 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
16.3%
7/43 • Number of events 7 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Eye disorders
Visual acuity reduced
|
2.1%
1/47 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.1%
1/48 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/44 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
7.0%
3/43 • Number of events 3 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Eye disorders
Blepharospasm
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/48 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/44 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.3%
1/43 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Eye disorders
Conjunctival deposit
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/48 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/44 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.3%
1/43 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Eye disorders
Conjunctival haemorrhage
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.1%
1/48 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/44 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/43 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Eye disorders
Eyelid oedema
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/48 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.3%
1/44 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/43 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Eye disorders
Visual impairement
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/48 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/44 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.3%
1/43 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Eye disorders
Hordeolum
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/48 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/44 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.3%
1/43 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
61.7%
29/47 • Number of events 29 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
79.2%
38/48 • Number of events 38 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
84.1%
37/44 • Number of events 37 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/43 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
8.5%
4/47 • Number of events 4 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
12.5%
6/48 • Number of events 6 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
25.0%
11/44 • Number of events 11 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/43 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
14.6%
7/48 • Number of events 7 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
20.5%
9/44 • Number of events 9 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/43 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Respiratory, thoracic and mediastinal disorders
Dysaesthesia pharynx
|
10.6%
5/47 • Number of events 5 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
8.3%
4/48 • Number of events 4 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
6.8%
3/44 • Number of events 3 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/43 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal dryness
|
2.1%
1/47 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/48 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/44 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
4.7%
2/43 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
General disorders
Instillation site irritation
|
6.4%
3/47 • Number of events 3 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
16.7%
8/48 • Number of events 8 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
18.2%
8/44 • Number of events 8 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/43 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
|
Nervous system disorders
Headache
|
0.00%
0/47 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
0.00%
0/48 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
4.5%
2/44 • Number of events 2 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
2.3%
1/43 • Number of events 1 • Adverse Events were collected from the first dose of study drug administration until the final study visit at Visit 5 (28 days)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place