CompletedNCT02254265Updated Aug 29, 2022Results posted

Phase 2/3 Dose-Ranging Study of the Safety and Efficacy of OTX-101 in the Treatment of Keratoconjunctivitis Sicca

A Phase 2/3 interventional study of OTX-101 0.05% and OTX-101 0.09% in Keratoconjunctivitis Sicca and Dry Eye Disease, sponsored by Sun Pharmaceutical Industries Limited. Completed at 1 site 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 2/3, Interventional, and Treatment

Phase: Phase 2/3
Study type: Interventional
Enrollment: 455
Allocation: Randomized
Ages: 18 Years and older
Sex: All

Study summary

This is a Phase 2/3 study to evaluate the safety and efficacy of 2 different dose concentrations of OTX-101 dosed twice a day in both eyes for 84 days compared to placebo (vehicle) in patients with keratoconjunctivitis sicca (dry eye disease).

Read the detailed description

Keratoconjunctivitis sicca (KCS) is a common multifactorial ophthalmologic disorder of the tears and ocular surface characterized by symptoms of burning, stinging, itching, grittiness, scratchiness, foreign body sensation, dryness, stickiness and tired eye sensation.

This study is the first clinical safety and efficacy study with OTX-101 and is designed to compare two dose concentrations of OTX-101 against vehicle (placebo) in reducing the signs and symptoms of KCS and evaluate safety when dosed twice a day in both eyes over an 84 day period.

Conditions studied

Keratoconjunctivitis Sicca
Dry Eye Disease

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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 455 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

Subjects age 18 years or older on the date of .
All subjects must provide signed written consent prior to participation in any study-related procedures.
Patient-reported history of KCS for a period of at least 6 months.
Clinical diagnosis of bilateral KCS supported by OTX-101-2014-001 study assessments.
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 Screening and Baseline.
Global symptom score ≥ 40 at both Screening and Baseline.
Corrected Snellen VA of better than 20/200 in each eye.
Willing to discontinue use of current dry eye therapy (including artificial tears or ocular lubricants) during the study as of the run-in period.
Female subjects of must have a negative urine pregnancy test at Screening. Women of childbearing potential (ie, women who are not either for one year or surgically sterile) must use an acceptable form of throughout the study.

Exclusion criteria

Exclusion Criteria:

Use of cyclosporine ophthalmic emulsion 0.05% (Restasis®) within 3 months prior to Screening.
Previous treatment failure (lack of efficacy) on cyclosporine ophthalmic emulsion 0.05% (Restasis).
Diagnosed with Sjögren's disease ˃5 years prior to Screening.
Clinical diagnosis of seasonal and perennial allergic conjunctivitis.
Use of systemic and topical medications that are known to cause dry eye within 7 days prior to Screening and throughout the study period. These include the following medications:
- Immunomodulators (permitted if dose is stable for 3 months prior to screening and does not change during the study period)
- Antihistamines (including over-the counter (OTC))
- Cholinergics
- Antimuscarinics
- Tricyclic antidepressants
- Phenothiazines
- Retinoids
Oral omega-3 fatty acids (permitted if dose is stable for 3 months prior to Screening and does not change during the study period)
Use of any topical ophthalmic medications, prescription (including antiglaucoma medications) or OTC (including artificial tears), other than the assigned study medication during the study period.
Current active eye disease other than KCS (i.e., any disease for which topical or systemic ophthalmic medication is necessary).
History of herpes keratitis.
Unstable macular disease (e.g., age-related macular degeneration, diabetic maculopathy). Stable macular disease is defined as no reduction in central VA within 6 months prior to Screening.
Diagnosis of chronic uveitis.
Corneal transplant (e.g., penetrating keratoplasty, lamellar keratoplasty, Descemet's stripping endothelial keratoplasty (DSEK) ).
Corneal refractive surgery (e.g., laser-assisted in situ keratomileusis (LASIK]) photo refractive keratectomy (PRK), limbal relaxing incision (LRI) ) within 6 months prior to screening or postoperative refractive surgery symptoms of dryness that have not resolved.
Cataract surgery within 3 months prior to Screening.
Non-laser glaucoma surgery at any time; glaucoma laser procedures within 3 months prior to Screening.
Presence of punctal plugs or past history of permanent punctal occlusion (e.g., cautery).
Lagophthalmos or eyelid margin irregularity of the study eye whether congenital or acquired.
Presence of conjunctivochalasis (i.e., mechanical blockage of the lower lid punctum by redundant conjunctiva).
Presence of pterygium in the study eye.
Unwilling to discontinue use of contact lenses during the duration of the study.
Preplanned elective surgery or hospitalization during the study period.
-positive.
Unable to reliably report symptoms and history.
Has known or to the study medication(s) or their components.
Has a history or presence of chronic generalized systemic disease that the Investigator feels might increase the risk to the subject or confound the result(s) of the study.
Has a severe/serious ocular condition, or any other unstable medical condition that, in the Investigator's opinion, may preclude study treatment or follow-up.
Women who are pregnant or breastfeeding.
Participation in any drug or device clinical investigation within 30 days prior to entry into this study and/or during the period of study participation.
Previous randomization into this study.

Terms in these criteria, in plain language(8)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Postmenopausal

Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

Study design

Phase: Phase 2 / Phase 3
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 455 participants (actual)

Study arms

Experimental
OTX-101 0.05%
OTX-101 0.05% ophthalmic solution 1 drop in both eyes BID for 84 days
Drug: OTX-101 0.05%
Experimental
OTX-101 0.09%
OTX-101 0.09% ophthalmic solution 1 drop in both eyes BID for 84 days
Drug: OTX-101 0.09%
Placebo comparator
Vehicle
Vehicle of OTX-101 ophthalmic solution 1 drop in both eyes BID for 84 days
Drug: Vehicle

Interventions

DrugOTX-101 0.05%
OTX-101 0.05% Ophthalmic Solution
DrugOTX-101 0.09%
OTX-101 0.09% Ophthalmic Solution
DrugVehicle
Vehicle of OTX-101 Ophthalmic Solution

What researchers measure

Primary outcomes

Conjunctival Staining
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
Time frame: Baseline to 84 days
Global Symptom Score
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
Time frame: Baseline to 84 days

Secondary outcomes

Tear Film Break up Time (TBUT)
Mean change from baseline in TBUT in the study eye from baseline at Day 84
Time frame: Baseline to 84 days
Corneal Staining Score
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
Time frame: Baseline to 84 days
Schirmer's Test
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
Time frame: Baseline to 84 days
Patient Satisfaction
Patient satisfaction with treatment score using 5-point scale (1=extremely dissatisfied to 5=extremely satisfied)
Time frame: Baseline to 84 days

Results

Posted Dec 14, 2018

Participant flow

Participant flow — Overall Study
Milestone	OTX-101 0.05%	OTX-101 0.09%	Vehicle
Started	151	152	152
Completed	142	140	144
Not completed	9	12	8

Outcome measures

PrimaryConjunctival Staining

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

Time frame:: Baseline to 84 days
Reported as:: Mean · score on a scale

Conjunctival Staining
score on a scale	OTX-101 0.05%	OTX-101 0.09%	Vehicle
Conjunctival Staining	-1.9 ± 2.17	-1.8 ± 2.27	-1.2 ± 2.17

PrimaryGlobal Symptom Score

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

Time frame:: Baseline to 84 days
Reported as:: Mean · score on a scale

Global Symptom Score
score on a scale	OTX-101 0.05%	OTX-101 0.09%	Vehicle
Global Symptom Score	-19.78 ± 23.791	-18.86 ± 22.810	-19.16 ± 22.973

SecondaryTear Film Break up Time (TBUT)

Mean change from baseline in TBUT in the study eye from baseline at Day 84

Time frame:: Baseline to 84 days
Reported as:: Mean · seconds

Tear Film Break up Time (TBUT)
seconds	OTX-101 0.05%	OTX-101 0.09%	Vehicle
Tear Film Break up Time (TBUT)	0.43 ± 2.032	0.53 ± 2.590	0.39 ± 2.149

SecondaryCorneal Staining Score

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

Time frame:: Baseline to 84 days
Reported as:: Mean · score on a scale

Corneal Staining Score
score on a scale	OTX-101 0.05%	OTX-101 0.09%	Vehicle
Corneal Staining Score	-1.4 ± 3.23	-1.4 ± 2.87	-0.6 ± 2.76

SecondarySchirmer's Test

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

Time frame:: Baseline to 84 days
Reported as:: Mean · score on a scale

Schirmer's Test
score on a scale	OTX-101 0.05%	OTX-101 0.09%	Vehicle
Schirmer's Test	0.41 ± 1.263	0.37 ± 1.260	-0.08 ± 1.307

SecondaryPatient Satisfaction

Patient satisfaction with treatment score using 5-point scale (1=extremely dissatisfied to 5=extremely satisfied)

Time frame:: Baseline to 84 days
Reported as:: Mean · score on a scale

Patient Satisfaction
score on a scale	OTX-101 0.05%	OTX-101 0.09%	Vehicle
Patient Satisfaction	3.8 ± 1.17	3.7 ± 1.17	3.9 ± 1.08

Adverse events

Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
OTX-101 0.09%	0/152 (0%)	1/152 (0.7%)	37/151 (24.5%)
OTX-101 0.05%	0/151 (0%)	0/151 (0%)	31/152 (20.4%)
Vehicle	0/152 (0%)	4/152 (2.6%)	30/152 (19.7%)

Most frequent serious events

Most frequent serious events
Event	OTX-101 0.09%	OTX-101 0.05%	Vehicle
Peripheral nerve decompressionSurgical and medical procedures	1/152	0/151	0/152
HypokalaemiaMetabolism and nutrition disorders	0/152	0/151	1/152
Humerus fractureInjury, poisoning and procedural complications	0/152	0/151	1/152
Cerebrovascular accidentNervous system disorders	0/152	0/151	1/152
Bipolar disorderPsychiatric disorders	0/152	0/151	1/152

Most frequent other events

Showing 10 of 21

Most frequent other events
Event	OTX-101 0.09%	OTX-101 0.05%	Vehicle
Instillation site painGeneral disorders	23/151	20/152	5/152
Eye pruritusEye disorders	1/151	3/152	4/152
Vision blurredEye disorders	0/151	1/152	4/152
NasopharyngitisInfections and infestations	2/151	1/152	4/152
Eye irritationEye disorders	3/151	2/152	1/152
Lacrimation increasedEye disorders	3/151	2/152	1/152
Vitreous detachmentEye disorders	0/151	3/152	0/152
SinusitisInfections and infestations	0/151	3/152	0/152
Meibomian gland dysfunctionEye disorders	2/151	0/152	1/152
Conjunctival haemorrhageEye disorders	1/151	2/152	2/152

Baseline characteristics

Intent to treat population

Age, Continuous
Age, Continuous(years)	OTX-101 0.05%	OTX-101 0.09%	Vehicle	Total
Mean	61.9 ± 13.27	59.2 ± 14.55	59.3 ± 13.75	60.1 ± 13.90

Sex: Female, Male
Sex: Female, Male(Participants)	OTX-101 0.05%	OTX-101 0.09%	Vehicle	Total
Female	119	122	120	361
Male	32	30	32	94

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	OTX-101 0.05%	OTX-101 0.09%	Vehicle	Total
American Indian or Alaska Native	1	0	0	1
Asian	8	7	3	18
Native Hawaiian or Other Pacific Islander	0	0	2	2
Black or African American	11	13	20	44
White	128	126	120	374
More than one race	3	6	7	16
Unknown or Not Reported	0	0	0	0

Study locations

1 site

Cleveland, Ohio, 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

Not marked actual or estimatedDates not marked actual or estimated

The record gives a start of Sep 2014, a primary completion of May 2015 and a completion of May 2015. It does not say whether these dates are actual or estimated.

Results were first posted in Dec 2018, 3 years 7 months after primary completion. A results delay was requested in Feb 2016.

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

Registered: Sep 29, 2014
First posted: Oct 1, 2014
Start: Sep 2014
Primary completionActual: May 2015
CompletionActual: May 2015
Results delay requested: Feb 13, 2016
Results first posted: Dec 14, 2018
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: NCT02254265
Lead sponsor: Sun Pharmaceutical Industries Limited
Responsible party: Sponsor
First posted: Oct 1, 2014
Start date: Sep 2014
Primary completion: May 2015
Completion: May 2015
Results posted: Dec 14, 2018
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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