Phase 2 Study of a New Ophthalmic Formulation of Cyclosporine (Restasis® X) in Patients With Dry Eye Disease

Study Overview
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Brief Summary
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This study will evaluate a new ophthalmic formulation of cyclosporine (Restasis® X) in patients with moderate to severe dry eye disease in two stages. Up to 3 doses will be studied in Stage 2 based on results from Stage 1. No patients participating in Stage 1 will participate in Stage 2 of this study. This study was terminated and Stage 2 of the study was cancelled.

Official Title
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A Phase 2, Multi-center, Vehicle- and Sham-controlled, Randomized Study of RESTASIS® X in Patients With Moderate to Severe Dry Eye Disease

Conditions
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Dry Eye Syndromes

Intervention / Treatment
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* Drug: Vehicle
* Other: Sham
* Drug: Cyclosporine New Ophthalmic Formulation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Moderate to severe dry eye disease in both eyes Best-corrected visual acuity (BCVA) of 20/100 or better in each eye Exclusion Criteria: Use of any cyclosporine preparations within 3 months Use of topical medications, other than artificial tears, in the eyes within 1 month Use of contact lenses in either eye within 1 month Stage 2 only: Participation in Stage 1 of this study

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Stage 1 Cohort 1<br>Vehicle administered to study eye and Sham administered to non-study eye on Day 1. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Other: Sham<br>* Sham administered to non-study eye as per protocol on Day 1<br>|
| Experimental: Stage 1 Cohort 2<br>Cyclosporine New Ophthalmic Formulation Dose A administered to study eye and Vehicle administered to non-study eye on Day 1. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Drug: Cyclosporine New Ophthalmic Formulation<br>* Cyclosporine New Ophthalmic Formulation administered as per protocol<br>* Other names: RESTASIS® X;|
| Experimental: Stage 1 Cohort 3<br>Cyclosporine New Ophthalmic Formulation Dose B administered to study eye and Vehicle administered to non-study eye on Day 1. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Drug: Cyclosporine New Ophthalmic Formulation<br>* Cyclosporine New Ophthalmic Formulation administered as per protocol<br>* Other names: RESTASIS® X;|
| Experimental: Stage 1 Cohort 4<br>Cyclosporine New Ophthalmic Formulation Dose C administered to study eye and Vehicle administered to non-study eye on Day 1. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Drug: Cyclosporine New Ophthalmic Formulation<br>* Cyclosporine New Ophthalmic Formulation administered as per protocol<br>* Other names: RESTASIS® X;|
| Experimental: Stage 1 Cohort 5A<br>Cyclosporine New Ophthalmic Formulation Dose D administered to the study eye and vehicle administered to the non-study eye on Day 1. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Drug: Cyclosporine New Ophthalmic Formulation<br>* Cyclosporine New Ophthalmic Formulation administered as per protocol<br>* Other names: RESTASIS® X;|
| Experimental: Stage 1 Cohort 6A<br>Cyclosporine New Ophthalmic Formulation Dose E administered to study eye and Vehicle administered to non-study eye on Day 1. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Drug: Cyclosporine New Ophthalmic Formulation<br>* Cyclosporine New Ophthalmic Formulation administered as per protocol<br>* Other names: RESTASIS® X;|
| Experimental: Stage 1 Cohort 6B<br>Cyclosporine New Ophthalmic Formulation Dose F administered to study eye and Vehicle administered to non-study eye on Day 1. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Drug: Cyclosporine New Ophthalmic Formulation<br>* Cyclosporine New Ophthalmic Formulation administered as per protocol<br>* Other names: RESTASIS® X;|
| Experimental: Stage 1 Cohort 6C<br>Cyclosporine New Ophthalmic Formulation Dose C administered to study eye and Vehicle administered to non-study eye on Day 1 and retreatment at Week 12 if applicable. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Drug: Cyclosporine New Ophthalmic Formulation<br>* Cyclosporine New Ophthalmic Formulation administered as per protocol<br>* Other names: RESTASIS® X;|
| Experimental: Stage 1 Cohort 6D<br>Cyclosporine New Ophthalmic Formulation Dose F administered to study eye and Vehicle administered to non-study eye on Day 1 and retreatment at Week 12 if applicable. | Drug: Vehicle<br>* Vehicle of cyclosporine administered as per protocol<br>Drug: Cyclosporine New Ophthalmic Formulation<br>* Cyclosporine New Ophthalmic Formulation administered as per protocol<br>* Other names: RESTASIS® X;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. TEAEs are AEs with an onset that occurs after receiving study drug. | First dose of study drug to up to 24 Weeks |