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

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)

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.

Study Overview

• Status: Completed
• Conditions: Corneal Edema; Corneal Endothelial Dysfunction
• Intervention / Treatment: Combination product: AURN001; Biological: Corneal Endothelial Cells; Drug: Y27632

• Study Type: Interventional
• Enrollment (Actual): 97
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada, V5Z 1K1 CAN
      • Aurion Biotech site
  • Ontario
    • Oakville, Ontario, Canada, L6H 0J8 CAN
      • Aurion Biotech site
    • Toronto, Ontario, Canada, M5T 3A9 CAN
      • Aurion Biotech site
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • Aurion Biotech site
  • California
    • Los Angeles, California, United States, 90067
      • Aurion Biotech site
    • San Francisco, California, United States, 94158
      • Aurion Biotech site
  • Georgia
    • Atlanta, Georgia, United States, 30339
      • Aurion Biotech site
  • Indiana
    • Indianapolis, Indiana, United States, 46260
      • Aurion Biotech site
  • Kansas
    • Wichita, Kansas, United States, 67206
      • Aurion Biotech site
  • Minnesota
    • Minnetonka, Minnesota, United States, 55305
      • Aurion Biotech site
  • New York
    • Garden City, New York, United States, 11576
      • Aurion Biotech site
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27157
      • Aurion Biotech site
  • Ohio
    • Cincinnati, Ohio, United States, 45242
      • Aurion Biotech site
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • Aurion Biotech site
  • Pennsylvania
    • Bala-Cynwyd, Pennsylvania, United States, 19004
      • Aurion Biotech site
  • South Dakota
    • Sioux Falls, South Dakota, United States, 57108
      • Aurion Biotech site
  • Texas
    • Houston, Texas, United States, 77027
      • Aurion Biotech site
    • Houston, Texas, United States, 77055
      • Aurion Biotech site
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • Aurion Biotech site
  • Washington
    • Seattle, Washington, United States, 98125
      • Aurion Biotech site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AURN001 High; Neltependocel High and Rho-associated protein kinase	Combination product: AURN001; Corneal Endothelial Cells and Y27632
Experimental: AURN001 Medium; Neltependocel Medium and Rho-associated protein kinase	Combination product: AURN001; Corneal Endothelial Cells and Y27632
Experimental: AURN001 Low; Neltependocel Low and Rho-associated protein kinase	Combination product: AURN001; Corneal Endothelial Cells and Y27632
Experimental: ROCK; Rho-associated protein kinase (ROCK)	Drug: Y27632; Y27632
Experimental: Neltependocel; Neltependocel - High	Biological: Corneal Endothelial Cells; Corneal Endothelial Cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Achieving ≥15-letter Improvement in Best-corrected Visual Acuity (BCVA) at Month 6 - Imputed Data	Best-corrected visual acuity was assessed using Early Treatment Diabetic Retinopathy Study(ETDRS) method, standard for vision testing. Standardized Sloan letter charts with 5 letters per line, decreasing by 0.1 logarithm of minimum angle of resolution (LogMAR) per line, were used at 4 meters (or 1 meter if ≤19 letters were read, with +0.75 spherical power added). Each correct letter equaled one point (0.02 LogMAR), with 85 letters (0.0 LogMAR) corresponding to 20/20 Snellen, 70 letters (+0.3) to 20/40, and 35 letters (+1.0) to 20/200. A gain of ≥15 letters (0.3 LogMAR, 3 lines) was clinically meaningful. Data from the study eye only were included in the analysis. Participants read letters aloud, guessing encouraged, and all responses recorded for accuracy. Data presented represent number of participants with available BCVA measurements at Month 6, with those who had missing data, underwent rescue surgery before Month 6, or discontinued study prior to Month 6 imputed as non-responders.	Month 6

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in BCVA at Month 6	Best-corrected visual acuity was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) method, the standard for vision testing. Standardized Sloan letter charts with five letters per line, decreasing by 0.1 logarithm of the minimum angle of resolution (LogMAR) per line, were used at 4 meters (or 1 meter if ≤19 letters were read, with +0.75 spherical power added). Each correct letter equaled one point (0.02 LogMAR), with 85 letters (0.0 LogMAR) corresponding to 20/20 Snellen, 70 letters (+0.3) to 20/40, and 35 letters (+1.0) to 20/200. A gain of ≥15 letters (0.3 LogMAR, 3 lines) was clinically meaningful. Data from the study eye only were included in the analysis. Participants read letters aloud, guessing encouraged, and all responses recorded for accuracy. Baseline was defined as the last measurement prior to the study drug injection on Day 1. Change from baseline values was calculated as follow-up visit minus baseline visit.	Baseline (Day 1) and at Month 6
Change From Baseline in Central Corneal Thickness (CCT) at Month 6	Central corneal thickness was measured by pachymetry which is an essential anatomical marker for detecting the presence or absence of edema and serves as a supportive indicator of treatment efficacy. Baseline was defined as the last measurement prior to the study drug injection on Day 1. Change from baseline values was calculated as follow-up visit minus baseline visit.	Baseline (Day 1) and at Month 6
Percentage of Participants Achieving ≥15-letter Improvement in BCVA at All Timepoints - Observed Data	Best-corrected visual acuity was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) method, the standard for vision testing. Standardized Sloan letter charts with five letters per line, decreasing by 0.1 logarithm of the minimum angle of resolution (LogMAR) per line, were used at 4 meters (or 1 meter if ≤19 letters were read, with +0.75 spherical power added). Each correct letter equaled one point (0.02 LogMAR), with 85 letters (0.0 LogMAR) corresponding to 20/20 Snellen, 70 letters (+0.3) to 20/40, and 35 letters (+1.0) to 20/200. A gain of ≥15 letters (0.3 LogMAR, 3 lines) was clinically meaningful. Data from the study eye only were included in the analysis. Participants read letters aloud, guessing encouraged, and all responses recorded for accuracy. The data presented reflect only participants with non-missing BCVA at each corresponding visit.	At Week 4 and at Months 2, 3, 4.5, 6, 9 and 12
Change From Baseline in BCVA at All Other Timepoints	Best-corrected visual acuity was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) method, the standard for vision testing. Standardized Sloan letter charts with five letters per line, decreasing by 0.1 logarithm of the minimum angle of resolution (LogMAR) per line, were used at 4 meters (or 1 meter if ≤19 letters were read, with +0.75 spherical power added). Each correct letter equaled one point (0.02 LogMAR), with 85 letters (0.0 LogMAR) corresponding to 20/20 Snellen, 70 letters (+0.3) to 20/40, and 35 letters (+1.0) to 20/200. A gain of ≥15 letters (0.3 LogMAR, 3 lines) was clinically meaningful. Data from the study eye only were included in the analysis. Participants read letters aloud, guessing encouraged, and all responses recorded for accuracy. Baseline was defined as the last measurement prior to the study drug injection on Day 1. Change from baseline values was calculated as follow-up visit minus baseline visit.	Baseline (Day 1) and at Week 4, Months 2, 3, 4.5, 6, 9 and 12
Change From Baseline in CCT at All Other Timepoints	Central corneal thickness was measured by pachymetry which is an essential anatomical marker for detecting the presence or absence of edema and serves as a supportive indicator of treatment efficacy. Baseline was defined as the last measurement prior to the study drug injection on Day 1. Change from baseline values was calculated as follow-up visit minus baseline visit.	Baseline (Day 1) and at Weeks 1 and 4, Months 2, 3, 4.5, 6, 9, and 12
Percentage of Participants Achieving ≥10-letter Improvement in BCVA at All Timepoints - Observed Data	Best-corrected visual acuity was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) method, the standard for vision testing. Standardized Sloan letter charts with five letters per line, decreasing by 0.1 logarithm of the minimum angle of resolution (LogMAR) per line, were used at 4 meters (or 1 meter if ≤19 letters were read, with +0.75 spherical power added). Each correct letter equaled one point (0.02 LogMAR), with 85 letters (0.0 LogMAR) corresponding to 20/20 Snellen, 70 letters (+0.3) to 20/40, and 35 letters (+1.0) to 20/200. A gain of ≥15 letters (0.3 LogMAR, 3 lines) was clinically meaningful. Data from the study eye only were included in the analysis. Participants read letters aloud, guessing encouraged, and all responses recorded for accuracy. The data presented reflect only participants with non-missing BCVA at each corresponding visit.	At Week 4 and at Months 2, 3, 4.5, 6, 9 and 12

Collaborators and Investigators

• Sponsor: Aurion Biotech
• Investigators: Study Director: Study Manager, OD, Aurion Biotech

Study record dates

Study Major Dates

• Study Start (Actual): October 18, 2023
• Primary Completion (Actual): October 25, 2024
• Study Completion (Actual): April 15, 2025

Study Registration Dates

• First Submitted: September 11, 2023
• First Submitted That Met QC Criteria: September 11, 2023
• First Posted (Actual): September 18, 2023

Study Record Updates

• Last Update Posted (Estimated): December 17, 2025
• Last Update Submitted That Met QC Criteria: December 16, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Corneal Diseases; Corneal Edema; Y 27632
• Other Study ID Numbers: ABA-1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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