Trial Outcomes & Findings for Phase 2/3 Dose-Ranging Study of the Safety and Efficacy of OTX-101 in the Treatment of Keratoconjunctivitis Sicca (NCT NCT02254265)
NCT ID: NCT02254265
Last Updated: 2022-08-29
Results Overview
Mean change from baseline at day 84 for the lissamine green conjunctival staining score in the designated study eye. The Investigator recorded a score for each area of each eye on a 0 (No punctate stain in zone) to 3 (Densely concentrated micropunctate stain spots) scale
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
455 participants
Primary outcome timeframe
Baseline to 84 days
Results posted on
2022-08-29
Participant Flow
Participant milestones
| Measure |
OTX-101 0.05%
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
OTX-101 0.09%
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
Vehicle
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Overall Study
STARTED
|
151
|
152
|
152
|
|
Overall Study
COMPLETED
|
142
|
140
|
144
|
|
Overall Study
NOT COMPLETED
|
9
|
12
|
8
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 2/3 Dose-Ranging Study of the Safety and Efficacy of OTX-101 in the Treatment of Keratoconjunctivitis Sicca
Baseline characteristics by cohort
| Measure |
OTX-101 0.05%
n=151 Participants
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
OTX-101 0.09%
n=152 Participants
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
Vehicle
n=152 Participants
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
Total
n=455 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
61.9 years
STANDARD_DEVIATION 13.27 • n=5 Participants
|
59.2 years
STANDARD_DEVIATION 14.55 • n=7 Participants
|
59.3 years
STANDARD_DEVIATION 13.75 • n=5 Participants
|
60.1 years
STANDARD_DEVIATION 13.90 • n=4 Participants
|
|
Sex: Female, Male
Female
|
119 Participants
n=5 Participants
|
122 Participants
n=7 Participants
|
120 Participants
n=5 Participants
|
361 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
32 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
32 Participants
n=5 Participants
|
94 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
18 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
11 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
44 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
128 Participants
n=5 Participants
|
126 Participants
n=7 Participants
|
120 Participants
n=5 Participants
|
374 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
16 Participants
n=4 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
|
PRIMARY outcome
Timeframe: Baseline to 84 daysPopulation: Intent to treat population, last observed carried forward approach
Mean change from baseline at day 84 for the lissamine green conjunctival staining score in the designated study eye. The Investigator recorded a score for each area of each eye on a 0 (No punctate stain in zone) to 3 (Densely concentrated micropunctate stain spots) scale
Outcome measures
| Measure |
OTX-101 0.05%
n=151 Participants
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
OTX-101 0.09%
n=152 Participants
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
Vehicle
n=152 Participants
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Conjunctival Staining
|
-1.9 score on a scale
Standard Deviation 2.17
|
-1.8 score on a scale
Standard Deviation 2.27
|
-1.2 score on a scale
Standard Deviation 2.17
|
PRIMARY outcome
Timeframe: Baseline to 84 daysPopulation: Intent to treat population, observed data
Mean change from baseline at day 84 for the global symptom score. The freuency and severy of dry eye and irritation scores were used to calculate symptom score as follows: Frequency of dry eye/irritation based on a scale of 0 (rarely) to 100 (all the time) Severity of dryness or irritation based on a scale of 0 (very mildly) to 100 (very severe). The global symptom score was calculated as the square root of the frequency score times the severity score
Outcome measures
| Measure |
OTX-101 0.05%
n=143 Participants
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
OTX-101 0.09%
n=140 Participants
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
Vehicle
n=144 Participants
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Global Symptom Score
|
-19.78 score on a scale
Standard Deviation 23.791
|
-18.86 score on a scale
Standard Deviation 22.810
|
-19.16 score on a scale
Standard Deviation 22.973
|
SECONDARY outcome
Timeframe: Baseline to 84 daysPopulation: Intent to treat population - observed data
Mean change from baseline in TBUT in the study eye from baseline at Day 84
Outcome measures
| Measure |
OTX-101 0.05%
n=143 Participants
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
OTX-101 0.09%
n=140 Participants
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
Vehicle
n=144 Participants
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Tear Film Break up Time (TBUT)
|
0.43 seconds
Standard Deviation 2.032
|
0.53 seconds
Standard Deviation 2.590
|
0.39 seconds
Standard Deviation 2.149
|
SECONDARY outcome
Timeframe: Baseline to 84 daysPopulation: Intent to treat population, Last observed carried forward approach
Mean Change from Baseline in Corneal Staining Score in the Study Eye. Expanded National Eye Institute Scale for Corneal Staining Score was used to grade each of the 5 areas of the cornea on a 0 (No punctate stain in area) to 4 (Severe diffuse (coalescent) macropunctate stain of the area) scale
Outcome measures
| Measure |
OTX-101 0.05%
n=151 Participants
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
OTX-101 0.09%
n=152 Participants
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
Vehicle
n=152 Participants
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Corneal Staining Score
|
-1.4 score on a scale
Standard Deviation 3.23
|
-1.4 score on a scale
Standard Deviation 2.87
|
-0.6 score on a scale
Standard Deviation 2.76
|
SECONDARY outcome
Timeframe: Baseline to 84 daysPopulation: Intent to treat population (with both eyes averaged)
Change from Baseline in Categorized Schirmer's Test Score. Schirmer's test was performed with strips placed in both eyes at the same time. Strips were removed after 5 minutes and the amount of wetting (in mm) was recorded as scores from 1 to 5. 1: \< 3 mm, 3 - 6 mm, 7 - 10 mm, 11 - 14 mm, and 5: \> 14 mm
Outcome measures
| Measure |
OTX-101 0.05%
n=143 Participants
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
OTX-101 0.09%
n=140 Participants
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
Vehicle
n=144 Participants
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Schirmer's Test
|
0.41 score on a scale
Standard Deviation 1.263
|
0.37 score on a scale
Standard Deviation 1.260
|
-0.08 score on a scale
Standard Deviation 1.307
|
SECONDARY outcome
Timeframe: Baseline to 84 daysPopulation: Intent to treat population (Observed)
Patient satisfaction with treatment score using 5-point scale (1=extremely dissatisfied to 5=extremely satisfied)
Outcome measures
| Measure |
OTX-101 0.05%
n=143 Participants
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
OTX-101 0.09%
n=140 Participants
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
Vehicle
n=144 Participants
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Patient Satisfaction
|
3.8 score on a scale
Standard Deviation 1.17
|
3.7 score on a scale
Standard Deviation 1.17
|
3.9 score on a scale
Standard Deviation 1.08
|
Adverse Events
OTX-101 0.09%
Serious events: 1 serious events
Other events: 37 other events
Deaths: 0 deaths
OTX-101 0.05%
Serious events: 0 serious events
Other events: 31 other events
Deaths: 0 deaths
Vehicle
Serious events: 4 serious events
Other events: 30 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
OTX-101 0.09%
n=151 participants at risk；n=152 participants at risk
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
OTX-101 0.05%
n=152 participants at risk；n=151 participants at risk
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
Vehicle
n=152 participants at risk
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Surgical and medical procedures
Peripheral nerve decompression
|
0.66%
1/152
|
0.00%
0/151
|
0.00%
0/152
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/152
|
0.00%
0/151
|
0.66%
1/152
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
0.00%
0/152
|
0.00%
0/151
|
0.66%
1/152
|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/152
|
0.00%
0/151
|
0.66%
1/152
|
|
Psychiatric disorders
Bipolar disorder
|
0.00%
0/152
|
0.00%
0/151
|
0.66%
1/152
|
Other adverse events
| Measure |
OTX-101 0.09%
n=151 participants at risk；n=152 participants at risk
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.09%: OTX-101 0.09% Ophthalmic Solution
|
OTX-101 0.05%
n=152 participants at risk；n=151 participants at risk
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
OTX-101 0.05%: OTX-101 0.05% Ophthalmic Solution
|
Vehicle
n=152 participants at risk
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Vehicle: Vehicle of OTX-101 Ophthalmic Solution
|
|---|---|---|---|
|
Eye disorders
Eye irritation
|
2.0%
3/151
|
1.3%
2/152
|
0.66%
1/152
|
|
Eye disorders
Lacrimation increased
|
2.0%
3/151
|
1.3%
2/152
|
0.66%
1/152
|
|
Eye disorders
Meibomian gland dysfunction
|
1.3%
2/151
|
0.00%
0/152
|
0.66%
1/152
|
|
Eye disorders
Conjunctival haemorrhage
|
0.66%
1/151
|
1.3%
2/152
|
1.3%
2/152
|
|
Eye disorders
Eye discharge
|
0.66%
1/151
|
1.3%
2/152
|
0.00%
0/152
|
|
Eye disorders
Eye pruritus
|
0.66%
1/151
|
2.0%
3/152
|
2.6%
4/152
|
|
Eye disorders
Vitreous floaters
|
0.66%
1/151
|
0.66%
1/152
|
1.3%
2/152
|
|
Eye disorders
Eye pain
|
0.00%
0/151
|
0.00%
0/152
|
1.3%
2/152
|
|
Eye disorders
Eyelid pain
|
0.00%
0/151
|
1.3%
2/152
|
0.00%
0/152
|
|
Eye disorders
Vision blurred
|
0.00%
0/151
|
0.66%
1/152
|
2.6%
4/152
|
|
Eye disorders
Vitreous detachment
|
0.00%
0/151
|
2.0%
3/152
|
0.00%
0/152
|
|
General disorders
Instillation site pain
|
15.2%
23/151
|
13.2%
20/152
|
3.3%
5/152
|
|
General disorders
Instillation site reaction
|
0.00%
0/151
|
1.3%
2/152
|
0.66%
1/152
|
|
Infections and infestations
Conjunctivitis viral
|
0.00%
0/151
|
0.00%
0/152
|
1.3%
2/152
|
|
Eye disorders
Corneal abrasion
|
0.00%
0/151
|
0.00%
0/152
|
1.3%
2/152
|
|
General disorders
Pyrexia
|
0.00%
0/151
|
0.00%
0/152
|
1.3%
2/152
|
|
Infections and infestations
Sinusitis
|
0.00%
0/151
|
2.0%
3/152
|
0.00%
0/152
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/151
|
0.00%
0/152
|
1.3%
2/152
|
|
Nervous system disorders
Tremor
|
0.00%
0/151
|
0.00%
0/152
|
1.3%
2/152
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.66%
1/151
|
1.3%
2/152
|
1.3%
2/152
|
|
Infections and infestations
Nasopharyngitis
|
1.3%
2/151
|
0.66%
1/152
|
2.6%
4/152
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place