Trial Outcomes & Findings for Dose Ranging Study to Assess the Efficacy and Safety of Voclosporin Ophthalmic Solution in Subjects With Dry Eye Syndrome (NCT NCT04147650)
NCT ID: NCT04147650
Last Updated: 2021-12-10
Results Overview
Number of subjects with a ≥10 mm increase from baseline in STT at Week 4 in the study eye. The STT scale is a measure of tear production with a minimum of 0 mm and no there is no specified maximum for this scale.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
508 participants
Primary outcome timeframe
4 Weeks
Results posted on
2021-12-10
Participant Flow
Participant milestones
| Measure |
0.05% Voclosporin Ophthalmic Solution (VOS)
0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.05% Voclosporin Ophthalmic Solution (VOS): 0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.10% VOS
0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.10% VOS: 0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.20% VOS
0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.20% VOS: 0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
Vehicle Ophthalmic Solution
Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
Vehicle Ophthalmic Solution: Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
127
|
126
|
128
|
127
|
|
Overall Study
Modified Intent to Treat (mITT)
|
120
|
119
|
122
|
121
|
|
Overall Study
COMPLETED
|
116
|
107
|
121
|
120
|
|
Overall Study
NOT COMPLETED
|
11
|
19
|
7
|
7
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Dose Ranging Study to Assess the Efficacy and Safety of Voclosporin Ophthalmic Solution in Subjects With Dry Eye Syndrome
Baseline characteristics by cohort
| Measure |
0.05% Voclosporin Ophthalmic Solution (VOS)
n=127 Participants
0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.05% Voclosporin Ophthalmic Solution (VOS): 0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.10% VOS
n=126 Participants
0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.10% VOS: 0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.20% VOS
n=128 Participants
0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.20% VOS: 0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
Vehicle Ophthalmic Solution
n=127 Participants
Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
Vehicle Ophthalmic Solution: Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
|
Total
n=508 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
64 years
n=93 Participants
|
65 years
n=4 Participants
|
65 years
n=27 Participants
|
65 years
n=483 Participants
|
65 years
n=36 Participants
|
|
Sex: Female, Male
Female
|
96 Participants
n=93 Participants
|
91 Participants
n=4 Participants
|
88 Participants
n=27 Participants
|
97 Participants
n=483 Participants
|
372 Participants
n=36 Participants
|
|
Sex: Female, Male
Male
|
31 Participants
n=93 Participants
|
35 Participants
n=4 Participants
|
40 Participants
n=27 Participants
|
30 Participants
n=483 Participants
|
136 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
7 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
7 Participants
n=27 Participants
|
6 Participants
n=483 Participants
|
25 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
120 Participants
n=93 Participants
|
120 Participants
n=4 Participants
|
121 Participants
n=27 Participants
|
121 Participants
n=483 Participants
|
482 Participants
n=36 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
1 Participants
n=36 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
2 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
1 Participants
n=483 Participants
|
4 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Asian
|
7 Participants
n=93 Participants
|
5 Participants
n=4 Participants
|
9 Participants
n=27 Participants
|
10 Participants
n=483 Participants
|
31 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
1 Participants
n=483 Participants
|
2 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Black or African American
|
17 Participants
n=93 Participants
|
16 Participants
n=4 Participants
|
17 Participants
n=27 Participants
|
22 Participants
n=483 Participants
|
72 Participants
n=36 Participants
|
|
Race (NIH/OMB)
White
|
96 Participants
n=93 Participants
|
103 Participants
n=4 Participants
|
99 Participants
n=27 Participants
|
91 Participants
n=483 Participants
|
389 Participants
n=36 Participants
|
|
Race (NIH/OMB)
More than one race
|
4 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
3 Participants
n=27 Participants
|
2 Participants
n=483 Participants
|
10 Participants
n=36 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
0 Participants
n=36 Participants
|
PRIMARY outcome
Timeframe: 4 WeeksPopulation: Modified intent to treat (mITT) number of subjects achieving ≥10 mm increase improvement at week 4
Number of subjects with a ≥10 mm increase from baseline in STT at Week 4 in the study eye. The STT scale is a measure of tear production with a minimum of 0 mm and no there is no specified maximum for this scale.
Outcome measures
| Measure |
0.05% Voclosporin Ophthalmic Solution (VOS)
n=120 Participants
0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.05% Voclosporin Ophthalmic Solution (VOS): 0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.10% VOS
n=119 Participants
0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.10% VOS: 0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.20% VOS
n=122 Participants
0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.20% VOS: 0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
Vehicle Ophthalmic Solution
n=121 Participants
Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
Vehicle Ophthalmic Solution: Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
|
|---|---|---|---|---|
|
Number of Subjects With a ≥10 mm Increase From Baseline in Schirmer Tear Test (STT)
|
12 participants
|
10 participants
|
13 participants
|
6 participants
|
SECONDARY outcome
Timeframe: Value at 4 Weeks minus value at baselinePopulation: The analysis was performed using all mITT subjects with a baseline eye dryness VAS ≥60 mm. Subjects without a Week 4 eye dryness VAS score were not included in this analysis.
Mean change from baseline in Eye Dryness Visual Analogue Scale (VAS) in subjects with a baseline Eye Dryness VAS score ≥ 60 mm. Eye Dryness Visual Analogue Scale 0-100 mm, where 0 corresponds to "no discomfort" and 100 corresponds to "maximal discomfort."
Outcome measures
| Measure |
0.05% Voclosporin Ophthalmic Solution (VOS)
n=85 Participants
0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.05% Voclosporin Ophthalmic Solution (VOS): 0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.10% VOS
n=84 Participants
0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.10% VOS: 0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.20% VOS
n=86 Participants
0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.20% VOS: 0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
Vehicle Ophthalmic Solution
n=89 Participants
Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
Vehicle Ophthalmic Solution: Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
|
|---|---|---|---|---|
|
Change From Baseline in Eye Dryness
|
-10.6 units on a scale
Standard Deviation 21.80
|
-9.3 units on a scale
Standard Deviation 20.00
|
-5.0 units on a scale
Standard Deviation 17.68
|
-7.0 units on a scale
Standard Deviation 17.59
|
Adverse Events
0.05% Voclosporin Ophthalmic Solution (VOS)
Serious events: 1 serious events
Other events: 29 other events
Deaths: 0 deaths
0.10% VOS
Serious events: 3 serious events
Other events: 42 other events
Deaths: 1 deaths
0.20% VOS
Serious events: 2 serious events
Other events: 44 other events
Deaths: 0 deaths
Vehicle Ophthalmic Solution
Serious events: 3 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
0.05% Voclosporin Ophthalmic Solution (VOS)
n=127 participants at risk
0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.05% Voclosporin Ophthalmic Solution (VOS): 0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.10% VOS
n=126 participants at risk
0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.10% VOS: 0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.20% VOS
n=128 participants at risk
0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.20% VOS: 0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
Vehicle Ophthalmic Solution
n=127 participants at risk
Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
Vehicle Ophthalmic Solution: Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
|
|---|---|---|---|---|
|
Nervous system disorders
Carotid artery stenosis
|
0.00%
0/127 • Day 1 to Week 13
|
0.00%
0/126 • Day 1 to Week 13
|
0.78%
1/128 • Number of events 1 • Day 1 to Week 13
|
0.00%
0/127 • Day 1 to Week 13
|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/127 • Day 1 to Week 13
|
0.00%
0/126 • Day 1 to Week 13
|
0.78%
1/128 • Number of events 1 • Day 1 to Week 13
|
0.00%
0/127 • Day 1 to Week 13
|
|
Metabolism and nutrition disorders
Fluid overload
|
0.00%
0/127 • Day 1 to Week 13
|
0.00%
0/126 • Day 1 to Week 13
|
0.78%
1/128 • Number of events 1 • Day 1 to Week 13
|
0.00%
0/127 • Day 1 to Week 13
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.00%
0/127 • Day 1 to Week 13
|
0.79%
1/126 • Number of events 1 • Day 1 to Week 13
|
0.00%
0/128 • Day 1 to Week 13
|
0.00%
0/127 • Day 1 to Week 13
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/127 • Day 1 to Week 13
|
0.00%
0/126 • Day 1 to Week 13
|
0.00%
0/128 • Day 1 to Week 13
|
0.79%
1/127 • Number of events 1 • Day 1 to Week 13
|
|
Cardiac disorders
Myocardial infarction
|
0.00%
0/127 • Day 1 to Week 13
|
0.79%
1/126 • Number of events 1 • Day 1 to Week 13
|
0.00%
0/128 • Day 1 to Week 13
|
0.00%
0/127 • Day 1 to Week 13
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.00%
0/127 • Day 1 to Week 13
|
0.79%
1/126 • Number of events 1 • Day 1 to Week 13
|
0.00%
0/128 • Day 1 to Week 13
|
0.00%
0/127 • Day 1 to Week 13
|
|
General disorders
Systemic inflammatory response syndrome
|
0.79%
1/127 • Number of events 1 • Day 1 to Week 13
|
0.00%
0/126 • Day 1 to Week 13
|
0.00%
0/128 • Day 1 to Week 13
|
0.00%
0/127 • Day 1 to Week 13
|
|
Infections and infestations
Appendicitis
|
0.00%
0/127 • Day 1 to Week 13
|
0.00%
0/126 • Day 1 to Week 13
|
0.00%
0/128 • Day 1 to Week 13
|
0.79%
1/127 • Number of events 1 • Day 1 to Week 13
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.00%
0/127 • Day 1 to Week 13
|
0.00%
0/126 • Day 1 to Week 13
|
0.00%
0/128 • Day 1 to Week 13
|
0.79%
1/127 • Number of events 1 • Day 1 to Week 13
|
Other adverse events
| Measure |
0.05% Voclosporin Ophthalmic Solution (VOS)
n=127 participants at risk
0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.05% Voclosporin Ophthalmic Solution (VOS): 0.05% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.10% VOS
n=126 participants at risk
0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.10% VOS: 0.10% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
0.20% VOS
n=128 participants at risk
0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
0.20% VOS: 0.20% VOS, in both eyes (OU) twice a day (BID) over 12 weeks
|
Vehicle Ophthalmic Solution
n=127 participants at risk
Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
Vehicle Ophthalmic Solution: Vehicle Ophthalmic Solution, in both eyes (OU) twice a day (BID) over 12 weeks
|
|---|---|---|---|---|
|
General disorders
Instillation site pain
|
22.8%
29/127 • Number of events 30 • Day 1 to Week 13
|
33.3%
42/126 • Number of events 43 • Day 1 to Week 13
|
34.4%
44/128 • Number of events 47 • Day 1 to Week 13
|
6.3%
8/127 • Number of events 10 • Day 1 to Week 13
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place