CompletedNCT02688556EmeraldUpdated Aug 29, 2022Results posted

Phase 3 Study of OTX-101 in the Treatment of Keratoconjunctivitis Sicca

A Phase 3 interventional study of cyclosporine and vehicle of OTX-101 in Keratoconjunctivitis Sicca, sponsored by Sun Pharmaceutical Industries Limited. Completed at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-08-29.

Sponsored by Sun Pharmaceutical Industries Limited · Phase 3, Interventional, and Treatment

Phase: Phase 3
Study type: Interventional
Enrollment: 745
Allocation: Randomized
Ages: 18 Years and older
Sex: All

Study summary

This is a randomized, double-masked, vehicle-controlled study of the safety and efficacy of OTX-101 (0.09% cyclosporine nanomicellar solution) in the treatment of keratoconjunctivitis sicca to be conducted at approximately 50 sites.

Conditions studied

Keratoconjunctivitis Sicca

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Keywords

KCS
dry eye
cyclosporine

In context

Keratoconjunctivitis Sicca

85 studies on the registry are indexed under Keratoconjunctivitis Sicca; 1 is open to participants now.

This study's enrollment of 745 is above the median of 82 across 75 interventional studies indexed under Keratoconjunctivitis Sicca.

Browse Keratoconjunctivitis Sicca studies →

Lead sponsor

Sun Pharmaceutical Industries Limited is the lead sponsor of 60 studies on the registry; 5 are open to participants now.

Of its 24 completed or terminated interventional studies of FDA-regulated products, 22 (92%) have results posted.

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Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

History of dry eye syndrome (KCS) for a period of at least 6 months
Clinical diagnosis of bilateral KCS
Lissamine green conjunctival staining sum score of ≥ 3 to ≤ 9 out of a total possible score of 12 (scoring excludes superior zones 2 and 4) in the same eye at both the Screening and Baseline Visits.
Global symptom score (SANDE) ≥ 40 mm at both the Screening and Baseline Visits
Corrected Snellen visual acuity (VA) of better than 20/200 in each eye.

Exclusion criteria

Exclusion Criteria:

Use of cyclosporine ophthalmic emulsion 0.05% (Restasis®) within 3 months prior to the Screening Visit.
Previous treatment failure (lack of efficacy) with cyclosporine ophthalmic emulsion 0.05% (Restasis).
Diagnosis of Sjögren's disease ˃ 5 years prior to the Screening Visit.
Clinical diagnosis or any history of seasonal and/or perennial allergic conjunctivitis.
Use of systemic or topical medications within 7 days prior to the Screening Visit or during the study period that are known to cause dry eye.
Use of any topical ophthalmic medications, prescription (including anti-glaucoma medications) or over the counter (including artificial tears), other than the assigned study medication during the study period.
Current active eye disease other than dry wyw syndrome (i.e., any disease for which topical or systemic ophthalmic medication is necessary).
History of herpes keratitis.
Corneal transplant
Corneal refractive surgery within 6 months prior to the Screening Visit or postoperative refractive surgery symptoms of dryness that have not resolved.
Cataract surgery within 3 months prior to the Screening Visit.

Study design

Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 745 participants (actual)

Study arms

Experimental
OTX-101 0.09%
0.09% cyclosporine nanomicellar ophthalmic solution
Drug: cyclosporine
Placebo comparator
Vehicle
vehicle of OTX-101
Drug: vehicle of OTX-101

Interventions

Drugcyclosporine
Also known as: Seciera
Drugvehicle of OTX-101

What researchers measure

Primary outcomes

Tear Production
Percentage of Eyes with Increase from Baseline of ≥ 10 mm in Schirmer's Test Score
Time frame: Baseline and 12 weeks

Secondary outcomes

Conjunctival Staining
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)
Time frame: Baseline and 12 weeks
Central Corneal Staining
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.
Time frame: Baseline and 12 weeks
Symptom Score
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.
Time frame: Baseline and 12 weeks

Results

Posted Apr 8, 2019

Participant flow

Participant flow — Overall Study
Milestone	OTX-101 0.09%	Vehicle
Started	372	373
Completed	347	361
Not completed	25	12

Outcome measures

PrimaryTear Production

Percentage of Eyes with Increase from Baseline of ≥ 10 mm in Schirmer's Test Score

Time frame:: Baseline and 12 weeks
Reported as:: Number · Percentage of eyes

Tear Production
Percentage of eyes	OTX-101 0.09%	Vehicle
Tear Production	16.6	9.2

SecondaryConjunctival Staining

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)

Time frame:: Baseline and 12 weeks
Reported as:: Mean · score on a scale

Conjunctival Staining
score on a scale	OTX-101 0.09%	Vehicle
Conjunctival Staining	-1.53 ± 1.927	-1.16 ± 2.2064

SecondaryCentral Corneal Staining

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.

Time frame:: Baseline and 12 weeks
Reported as:: Mean · score on a scale

Central Corneal Staining
score on a scale	OTX-101 0.09%	Vehicle
Central Corneal Staining	-0.28 ± 0.533	-0.26 ± 0.588

SecondarySymptom Score

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.

Time frame:: Baseline and 12 weeks
Reported as:: Mean · score on a scale

Symptom Score
score on a scale	OTX-101 0.09%	Vehicle
Symptom Score	-18.8 ± 24.08	-19.1 ± 23.14

Adverse events

Collected over 12 weeks. Non-serious events are listed at a 1% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
OTX-101 0.09%	1/372 (0.3%)	6/372 (1.6%)	130/372 (34.9%)
Vehicle	0/372 (0%)	2/372 (0.5%)	66/372 (17.7%)

Most frequent serious events

Most frequent serious events
Event	OTX-101 0.09%	Vehicle
DeathGeneral disorders	1/372	0/372
PneumoniaInfections and infestations	1/372	0/372
Lung neoplasm malignantNeoplasms benign, malignant and unspecified (incl cysts and polyps)	1/372	0/372
Subdural haematomaInjury, poisoning and procedural complications	1/372	0/372
Spinal column stenosisMusculoskeletal and connective tissue disorders	1/372	0/372
NephrolithiasisRenal and urinary disorders	1/372	0/372
Perforated ulcerGeneral disorders	0/372	1/372
Spinal osteoarthritisMusculoskeletal and connective tissue disorders	0/372	1/372

Most frequent other events

Showing 10 of 14

Most frequent other events
Event	OTX-101 0.09%	Vehicle
Instillation site painGeneral disorders	90/372	16/372
Conjunctival hyperaemiaEye disorders	30/372	18/372
HeadacheNervous system disorders	6/372	2/372
BlepharitisEye disorders	5/372	0/372
Eye irritationEye disorders	3/372	5/372
Eye pruritusEye disorders	1/372	5/372
SinusitisEye disorders	4/372	5/372
Foreign body sensation in eyesEye disorders	1/372	5/372
Instillation site lacrimationGeneral disorders	4/372	0/372
Instillation site reactionEye disorders	4/372	2/372

Baseline characteristics

Intent to treat population

Age, Continuous
Age, Continuous(years)	OTX-101 0.09%	Vehicle	Total
Mean	58.4 ± 14.10	59.5 ± 14.68	59.0 ± 14.40

Sex: Female, Male
Sex: Female, Male(Participants)	OTX-101 0.09%	Vehicle	Total
Female	315	311	626
Male	56	62	118

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	OTX-101 0.09%	Vehicle	Total
American Indian or Alaska Native	1	0	1
Asian	11	12	23
Native Hawaiian or Other Pacific Islander	0	1	1
Black or African American	41	45	86
White	310	305	615
More than one race	8	10	18
Unknown or Not Reported	0	0	0

Study locations

3 sites

Martel Eye Medical Group
Rancho Cordova, California 95670, United States
Cincinnati Eye Institute
Edgewood, Kentucky 41017, United States
Fifth Avenue Eye Associates
New York, New York 10028, United States

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

Timeline

Tracking since Sep 25, 2026

ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Feb 2016 to primary completion in Nov 2016, 9 months.

Results were first posted in Apr 2019, 2 years 5 months after primary completion.

Of 12 phase 3 interventional studies under Keratoconjunctivitis Sicca, half reached primary completion within 14 months of starting (the median); a quarter within 7 and three quarters within 18. This study's 9 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered: Feb 13, 2016
First posted: Feb 23, 2016
StartActual: Feb 2016
Primary completionActual: Nov 2016
CompletionActual: Dec 2016
Results first posted: Apr 8, 2019
Last update: Aug 29, 2022

What these dates mean

Start.: When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.: When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.: When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.: Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.

No changes since tracking began. The registry record was last updated on Aug 29, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT02688556
Lead sponsor: Sun Pharmaceutical Industries Limited
Responsible party: Sponsor
First posted: Feb 23, 2016
Start date: Feb 2016
Primary completion: Nov 2016
Completion: Dec 2016
Results posted: Apr 8, 2019
Last update: Aug 29, 2022

Oversight

Data monitoring committee: No

View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Nov 2021. You cannot join it, but the record below documents what was studied.

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