A Phase 1/2 Study of AURN001 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction (ABA-1)

Study Overview
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Brief Summary
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The goal of this clinical trial is to compare different doses of AURN001 in patients with corneal edema secondary to corneal endothelial dysfunction. The main questions the clinical trial aims to answer are whether AURN001 effective and safe. Participants will receive a single injection of AURN001. A comparison between low, medium, and high doses of AURN001 against the contribution of each element, cells alone and Y27632 alone, will be conducted to determine the effects on corneal edema.

Official Title
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CLARA: A Phase 1/2 Multi-center, Randomized, Double-Masked, Prospective, Parallel-Arm Study of AURN001 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction (ABA-1)

Conditions
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Corneal Edema, Corneal Endothelial Dysfunction

Intervention / Treatment
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* Combination Product: AURN001


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have a diagnosis of corneal edema secondary to corneal endothelial dysfunction, requiring surgery (full- or partial-thickness endothelial keratoplasty) BCVA between 65 Early Treatment of Diabetic Retinopathy Study (ETDRS) letters (i.e., 0.4 LogMAR or approximate 20/50 Snellen equivalent) and 5 ETDRS letters (i.e., 1.6 LogMAR or approximate 20/800 Snellen equivalent) Exclusion Criteria: Have pre-operative corneal epithelial, sub-epithelial or stromal 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 in the study eye Have history or presence of an ocular disease other than corneal endothelial dysfunction that could affect vision or safety assessments

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 99 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 |
| --- | --- |
| Experimental: AURN001 High<br>Neltependocel High and Rho-associated protein kinase | Combination Product: AURN001<br>* Corneal Endothelial Cells and Y27632<br>|
| Experimental: AURN001 Medium<br>Neltependocel Medium and Rho-associated protein kinase | Combination Product: AURN001<br>* Corneal Endothelial Cells and Y27632<br>|
| Experimental: AURN001 Low<br>Neltependocel Low and Rho-associated protein kinase | Combination Product: AURN001<br>* Corneal Endothelial Cells and Y27632<br>|
| Experimental: Neltependocel - High<br>Neltependocel - High | Combination Product: AURN001<br>* Corneal Endothelial Cells and Y27632<br>|
| Experimental: ROCK<br>Rho-associated protein kinase (ROCK) | Combination Product: AURN001<br>* Corneal Endothelial Cells and Y27632<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| BCVA - 15-letter improvement (3-line gain) | Response, defined as a ≥15-letter improvement (3-line gain) from baseline in best-corrected visual acuity (BCVA) | Month 6 |