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 (Escalon)

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

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.

Study Overview

• Status: Completed
• Conditions: Corneal Edema
• Intervention / Treatment: Drug: HCEC-1

• Study Type: Interventional
• Enrollment (Actual): 22
• Phase: Phase 1

Contacts and Locations

Study Locations

• El Salvador
  • San Salvador, El Salvador
    • Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

1. 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
2. Clinical diagnosis of corneal edema secondary to endothelial dysfunction
3. 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:

1. Keratoconus or other conditions of corneal thinning/ectasia
2. Progressive stromal or anterior corneal dystrophies
3. 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
4. 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.
5. Relative Afferent Pupillary Defect (RAPD) in the study eye
6. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Aurion Biotech

Study record dates

Study Major Dates

• Study Start (Actual): March 24, 2022
• Primary Completion (Actual): March 9, 2023
• Study Completion (Actual): March 9, 2023

Study Registration Dates

• First Submitted: March 16, 2022
• First Submitted That Met QC Criteria: March 24, 2022
• First Posted (Actual): April 4, 2022

Study Record Updates

• Last Update Posted (Actual): July 19, 2023
• Last Update Submitted That Met QC Criteria: July 18, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Corneal Diseases; Edema; Corneal Edema
• Other Study ID Numbers: AB-HCEC-1-004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: The study plan and protocol will be shared with the study center and other researchers per request.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No