Trial Outcomes & Findings for Phase 3 Study of OTX-101 in the Treatment of Keratoconjunctivitis Sicca (NCT NCT02688556)
NCT ID: NCT02688556
Last Updated: 2022-08-29
Results Overview
Percentage of Eyes with Increase from Baseline of ≥ 10 mm in Schirmer's Test Score
COMPLETED
PHASE3
745 participants
Baseline and 12 weeks
2022-08-29
Participant Flow
One subject in OTX-101 0.09% group, was never treated with study medication and is not included in any of analysis sets Additionally, because the subjects in the ITT analysis set were analyzed as randomized, one subject who erroneously received OTX-101 0.09%, was included in the Vehicle group for purposes of efficacy analysis.
Participant milestones
| Measure |
OTX-101 0.09%
0.09% cyclosporine nanomicellar ophthalmic solution
|
Vehicle
vehicle of OTX-101
|
|---|---|---|
|
Overall Study
STARTED
|
372
|
373
|
|
Overall Study
COMPLETED
|
347
|
361
|
|
Overall Study
NOT COMPLETED
|
25
|
12
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 3 Study of OTX-101 in the Treatment of Keratoconjunctivitis Sicca
Baseline characteristics by cohort
| Measure |
OTX-101 0.09%
n=371 Participants
0.09% cyclosporine nanomicellar ophthalmic solution
|
Vehicle
n=373 Participants
vehicle of OTX-101
|
Total
n=744 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
58.4 years
STANDARD_DEVIATION 14.10 • n=5 Participants
|
59.5 years
STANDARD_DEVIATION 14.68 • n=7 Participants
|
59.0 years
STANDARD_DEVIATION 14.40 • n=5 Participants
|
|
Sex: Female, Male
Female
|
315 Participants
n=5 Participants
|
311 Participants
n=7 Participants
|
626 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
56 Participants
n=5 Participants
|
62 Participants
n=7 Participants
|
118 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
11 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
41 Participants
n=5 Participants
|
45 Participants
n=7 Participants
|
86 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
310 Participants
n=5 Participants
|
305 Participants
n=7 Participants
|
615 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
8 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and 12 weeksPopulation: Intent to treat population
Percentage of Eyes with Increase from Baseline of ≥ 10 mm in Schirmer's Test Score
Outcome measures
| Measure |
OTX-101 0.09%
n=371 Participants
0.09% cyclosporine nanomicellar ophthalmic solution
|
Vehicle
n=373 Participants
vehicle of OTX-101
|
|---|---|---|
|
Tear Production
|
16.6 Percentage of eyes
|
9.2 Percentage of eyes
|
SECONDARY outcome
Timeframe: Baseline and 12 weeksPopulation: Intent to treat population
change from baseline in total conjunctival staining score (lissamine green, modified National Eye Institute scale) at 12 weeks. Conjunctival Lissamine Green Staining Grades ranged from 0 (No punctate stain in zone) to 3 (Densely concentrated micropunctate stain spots)
Outcome measures
| Measure |
OTX-101 0.09%
n=371 Participants
0.09% cyclosporine nanomicellar ophthalmic solution
|
Vehicle
n=373 Participants
vehicle of OTX-101
|
|---|---|---|
|
Conjunctival Staining
|
-1.53 score on a scale
Standard Deviation 1.927
|
-1.16 score on a scale
Standard Deviation 2.2064
|
SECONDARY outcome
Timeframe: Baseline and 12 weeksPopulation: Intent to treat population
change from baseline in central corneal staining score (fluorescein, modified NEI/FDA scale) at 12 weeks. The Expanded National Eye Institute (NEI)/Industry Workshop 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 0.09%
n=341 Participants
0.09% cyclosporine nanomicellar ophthalmic solution
|
Vehicle
n=373 Participants
vehicle of OTX-101
|
|---|---|---|
|
Central Corneal Staining
|
-0.28 score on a scale
Standard Deviation 0.533
|
-0.26 score on a scale
Standard Deviation 0.588
|
SECONDARY outcome
Timeframe: Baseline and 12 weeksPopulation: Intent to treat population
change from baseline in modified Symptom Assessment in Dry Eye (SANDE) score at 12 weeks. A modified SANDE instrument was used to evaluate dry eye symptoms at each visit. Subjects were asked to indicate: 1. frequency of dry and irritated eyes on a scale of 0 (rarely) to 100 (all the time); and 2. severity of dry eyes on a scale of 0 (very mild) to 100 (severe) The global symptom score is the square root of the frequency score times the severity score and will be completed at each visit. (range 0 to 100) Negative change from baseline indicates improvement.
Outcome measures
| Measure |
OTX-101 0.09%
n=371 Participants
0.09% cyclosporine nanomicellar ophthalmic solution
|
Vehicle
n=373 Participants
vehicle of OTX-101
|
|---|---|---|
|
Symptom Score
|
-18.8 score on a scale
Standard Deviation 24.08
|
-19.1 score on a scale
Standard Deviation 23.14
|
Adverse Events
OTX-101 0.09%
Vehicle
Serious adverse events
| Measure |
OTX-101 0.09%
n=372 participants at risk
0.09% cyclosporine nanomicellar ophthalmic solution.
A total of 744 subjects were included in the Safety population, with 372 subjects in the OTX-101 0.09% group and 372 subjects in the Vehicle group. The only difference between the ITT and the Safety populations is that Subject 14-003 was analyzed in the Safety population according to the treatment received, which was OTX-101 0.09%
|
Vehicle
n=372 participants at risk
vehicle of OTX-101
A total of 744 subjects were included in the Safety population, with 372 subjects in the OTX-101 0.09% group and 372 subjects in the Vehicle group. The only difference between the ITT and the Safety populations is that Subject 14-003 was analyzed in the Safety population according to the treatment received, which was OTX-101 0.09%
|
|---|---|---|
|
General disorders
Death
|
0.27%
1/372 • Number of events 1 • 12 weeks
|
0.00%
0/372 • 12 weeks
|
|
Infections and infestations
Pneumonia
|
0.27%
1/372 • Number of events 1 • 12 weeks
|
0.00%
0/372 • 12 weeks
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung neoplasm malignant
|
0.27%
1/372 • Number of events 1 • 12 weeks
|
0.00%
0/372 • 12 weeks
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
0.27%
1/372 • Number of events 1 • 12 weeks
|
0.00%
0/372 • 12 weeks
|
|
Musculoskeletal and connective tissue disorders
Spinal column stenosis
|
0.27%
1/372 • Number of events 1 • 12 weeks
|
0.00%
0/372 • 12 weeks
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.27%
1/372 • Number of events 1 • 12 weeks
|
0.00%
0/372 • 12 weeks
|
|
General disorders
Perforated ulcer
|
0.00%
0/372 • 12 weeks
|
0.27%
1/372 • Number of events 1 • 12 weeks
|
|
Musculoskeletal and connective tissue disorders
Spinal osteoarthritis
|
0.00%
0/372 • 12 weeks
|
0.27%
1/372 • Number of events 1 • 12 weeks
|
Other adverse events
| Measure |
OTX-101 0.09%
n=372 participants at risk
0.09% cyclosporine nanomicellar ophthalmic solution.
A total of 744 subjects were included in the Safety population, with 372 subjects in the OTX-101 0.09% group and 372 subjects in the Vehicle group. The only difference between the ITT and the Safety populations is that Subject 14-003 was analyzed in the Safety population according to the treatment received, which was OTX-101 0.09%
|
Vehicle
n=372 participants at risk
vehicle of OTX-101
A total of 744 subjects were included in the Safety population, with 372 subjects in the OTX-101 0.09% group and 372 subjects in the Vehicle group. The only difference between the ITT and the Safety populations is that Subject 14-003 was analyzed in the Safety population according to the treatment received, which was OTX-101 0.09%
|
|---|---|---|
|
Eye disorders
Conjunctival hyperaemia
|
8.1%
30/372 • 12 weeks
|
4.8%
18/372 • 12 weeks
|
|
Eye disorders
Blepharitis
|
1.3%
5/372 • 12 weeks
|
0.00%
0/372 • 12 weeks
|
|
Eye disorders
Eye irritation
|
0.81%
3/372 • 12 weeks
|
1.3%
5/372 • 12 weeks
|
|
Eye disorders
Eye pruritus
|
0.27%
1/372 • 12 weeks
|
1.3%
5/372 • 12 weeks
|
|
General disorders
Instillation site pain
|
24.2%
90/372 • 12 weeks
|
4.3%
16/372 • 12 weeks
|
|
General disorders
Instillation site lacrimation
|
1.1%
4/372 • 12 weeks
|
0.00%
0/372 • 12 weeks
|
|
Eye disorders
Instillation site reaction
|
1.1%
4/372 • 12 weeks
|
0.54%
2/372 • 12 weeks
|
|
Eye disorders
Sinusitis
|
1.1%
4/372 • 12 weeks
|
1.3%
5/372 • 12 weeks
|
|
Eye disorders
Urinary tract infection
|
1.1%
4/372 • 12 weeks
|
0.54%
2/372 • 12 weeks
|
|
Nervous system disorders
Headache
|
1.6%
6/372 • 12 weeks
|
0.54%
2/372 • 12 weeks
|
|
Eye disorders
Foreign body sensation in eyes
|
0.27%
1/372 • 12 weeks
|
1.3%
5/372 • 12 weeks
|
|
Eye disorders
Conjunctival haemorrhage
|
0.54%
2/372 • 12 weeks
|
0.27%
1/372 • 12 weeks
|
|
Eye disorders
Posterior capsule opacification
|
0.54%
2/372 • 12 weeks
|
0.54%
2/372 • 12 weeks
|
|
Eye disorders
Punctate keratitis
|
0.54%
2/372 • 12 weeks
|
0.81%
3/372 • 12 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place