A Safety and Efficacy Study of Corneal Injection for Endothelial Dysfunction Using Human Corneal Endothelial Cell Therapy in Subjects With Corneal Edema Secondary to Endothelial Dysfunction

Study Overview
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Brief Summary
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The purpose of this study is to assess the safety of a fixed dose of human corneal endothelial cells when administered with varying concentrations of Rho kinase inhibitor (Y-27632) in patients with corneal edema secondary to endothelial dysfunction.

Official Title
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A Phase 1, Double-Masked, Randomized, Safety and Efficacy, Single Center Clinical Evaluation of Corneal Injection for Endothelial Dysfunction Using Human Corneal Endothelial Cell Therapy (HCEC-1) in Adult Subjects With Corneal Edema Secondary to Endothelial Dysfunction

Conditions
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Corneal Edema

Intervention / Treatment
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* Drug: HCEC-1


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Must have given written informed consent (signed and dated by subject or legal guardian), and any authorizations required by local law and be able to comply with all study requirements Clinical diagnosis of corneal edema secondary to endothelial dysfunction Best Corrected Visual Acuity (BCVA) no worse than 20/800 ETDRS (1.6 LogMAR) or better than 20/63 (0.5 LogMAR) in the study eye Key Exclusion Criteria: Keratoconus or other conditions of corneal thinning/ectasia Progressive stromal or anterior corneal dystrophies Pre-operative corneal epithelial, sub-epithelial or stromal staining, scarring or other opacity that is paracentral/central and visually significant, but not suspected to be secondary to corneal endothelial disease with the potential to improve from treatment with HCEC-1 Prior ophthalmic surgery including cataract surgery except as noted above in Inclusion Criterion #4, corneal transplantation, glaucoma (MIGS, iridectomy, trabeculectomy, valve), or vitreo-retinal surgery, aphakia, anterior chamber-IOL or iris claw IOL, multifocal IOL. Relative Afferent Pupillary Defect (RAPD) in the study eye Uncontrolled glaucoma or an IOP greater than 24 mmHg that is not controlled with topical beta blockers alone at the time of the Screening Visit.

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: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Low dose Rho kinase inhibitor Y-27632<br>Low dose Rho kinase inhibitor Y-27632 | Drug: HCEC-1<br>* HCEC-1 = human cultured corneal endothelial cells and Rho kinase Y-27632 inhibitor<br>|
| Active Comparator: Mid dose Rho kinase inhibitor Y-27632<br>Mid dose Rho kinase inhibitor Y-27632 | Drug: HCEC-1<br>* HCEC-1 = human cultured corneal endothelial cells and Rho kinase Y-27632 inhibitor<br>|
| Active Comparator: High dose Rho kinase inhibitor Y-27632<br>High dose Rho kinase inhibitor Y-27632 | Drug: HCEC-1<br>* HCEC-1 = human cultured corneal endothelial cells and Rho kinase Y-27632 inhibitor<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence and severity of Treatment Emergent Ocular Adverse Events (TEAEs) |  | 12 months | 
| Incidence and severity of non-ocular TEAEs |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in Central Corneal Thickness (CCT) as measured by pachymetry |  | 12 months | 
| Change from baseline in Best-Corrected Visual Acuity (BCVA) as measured by LogMAR score |  | 12 months |