Study of Safety and Tolerability of EO2002 in the Treatment of Corneal Edema

Phase 1

Completed

• Conditions: Fuchs Dystrophy; Fuchs Endothelial Corneal Dystrophy; Pseudophakic Bullous Keratopathy; Endothelial Dysfunction; Corneal Edema; Corneal Endothelial Dysfunction; Corneal Endothelial Dystrophy; Bullous Keratopathy; Moderate Corneal Endothelial Decompensation
• Interventions: Biological: EO2002; Biological: EO2002 mid dose; Biological: EO2002 low dose; Biological: EO2002 high dose

• Registration Number: NCT04894110
• Lead Sponsor: Emmecell

Brief Summary

Corneal edema is the most common indication for corneal transplantation, accounting for approximately 70% of penetrating keratoplasty (PK), and 100% of endothelial keratoplasty (EK) procedures annually. There is currently no disease-modifying treatment for corneal edema. Topical treatments like hypertonic saline are not effective on a long-term basis. For those with advanced disease, where edema and vision loss are not controlled by topical treatment, the only option is a corneal transplant. A potential approach to avoidance of the risks of corneal transplantation is to inject cultured human corneal endothelial cells (HCECs) into the anterior chamber of the eye. This approach may avoid surgery by re-populating the inner most aspect of the cornea with functioning endothelial cells. Emmecell has developed a treatment based on technology integrating biocompatible magnetic nanoparticles with cultured HCECs to treat corneal edema in a minimally invasive way. The primary objective of this phase 1, prospective, multi-center, open-label, dose-escalation study is to evaluate the safety and tolerability of 3 doses of EO2002 with and without endothelial brushing (EB) or Descemet Stripping (DS) in eyes with corneal edema secondary to corneal endothelial dysfunction that qualify for surgery involving full-thickness corneal transplantation or EK.

Detailed Description

The study is designed to occur in 2 groups. The objective with the first group is to preliminarily evaluate the safety of 4 escalating doses. The purpose of the second group is to gather additional safety information on the 3 highest doses when administered according to a randomized schedule and evaluated by masked observers.

Group 1: Phase 1 prospective, multi-center, open-label, dose-escalation study designed to evaluate the safety and tolerability of EO2002 with and without endothelial brushing (EB) or Descemet Stripping (DS) in eyes with corneal edema secondary to corneal endothelial dysfunction that qualify for surgery involving full-thickness corneal transplantation or EK. Four doses will be studied in approximately 21 study participants that meet the inclusion/exclusion criteria. Safety and tolerability will be the primary focus for 26 weeks following treatment with EO2002.

Group 2: The three highest doses will be administered to up to 21 subjects in a randomized, double-masked design.

Study Timeline

• Study First Submitted: 2021-05-11
• Study First Submitted QC: 2021-05-16
• Study First Posted: 2021-05-20
• Study Start: 2021-06-22
• Primary Completion: 2024-10-03
• Study Completion: 2024-10-03
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-03
• Last Update Posted: 2025-03-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 42

Inclusion Criteria

All ocular criteria apply to study eye unless otherwise noted.

1. Age ≥ 21 years.
2. Phakic or Pseudophakic with a posterior chamber intraocular lens
3. Symptomatic corneal edema associated with endothelial dysfunction which may be secondary to Fuchs corneal dystrophy or pseudophakic bullous keratopathy.

Exclusion Criteria

All ocular criteria apply to study eye unless otherwise noted.

1. Other corneal disease
2. Anterior chamber intraocular lens, Sutured or scleral-fixated intraocular lens.
3. Macular disease that in the investigator and/or sponsor's opinion would limit the ability of the subject to demonstrate improvement in BCVA.
4. History of refractive surgery.
5. Descemet membrane detachment.
6. History of uveitis or other ocular inflammatory disease.
7. History of incisional glaucoma surgery (e.g.,trabeculectomy, glaucoma drainage implant).
8. Intraocular pressure >21 or <7 mm Hg
9. Prior incisional eye surgery within 3 months prior to study treatment or penetrating or endothelial keratoplasty.
10. History of ocular neoplasm.
11. ETDRS BCVA in the fellow eye is worse than 35 letters (Snellen equivalent of 20/200).
12. Female who is pregnant, nursing, or planning to become pregnant, or who is of childbearing potential and not using a reliable means of contraception during the study.
13. Subject is currently participating in or has participated within the last 3 months in any other clinical trial of an investigational drug by ocular or systemic administration.
14. Any concomitant medical or psychological condition that could interfere with study participation or is otherwise not suitable for entry into the study in the opinion of the investigator.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
EO2002 treatment - Group 1	EO2002	-
EO2002 treatment - Group 2 - mid dose	EO2002 mid dose	-
EO2002 treatment - Group 2 - low dose	EO2002 low dose	-
EO2002 treatment - Group 2 - high dose	EO2002 high dose	-

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability of EO2002]	26 Weeks	Incidence of Treatment-Emergent Adverse Events \[Safety and Tolerability of EO2002 (+/- EB or Descemet Stripping)\] in subjects with corneal edema at week 26.

Secondary Outcome Measures

Name	Time	Method
Effect of EO2002 (+/-EB or DS) on corneal thickness	26 Weeks	Evaluate the effect of a single injection of EO2002 (+/-EB or DS) on corneal thickness as determined by pachymetry
Effect of EO2002 (+/-EB or DS) on best corrected visual acuity (BCVA).	26 Weeks	Evaluate the effect of a single injection of EO2002 (+/-EB or DS) on best corrected visual acuity (BCVA) as determined by ETDRS letter score.

Trial Locations

Locations (8)

Los Angeles Location; 🇺🇸 Greater Los Angeles, California, United States

San Diego Location; 🇺🇸 San Diego, California, United States

Miami Location; 🇺🇸 Miami, Florida, United States

Palm Beach Gardens Location; 🇺🇸 Palm Beach Gardens, Florida, United States

Atlanta Location; 🇺🇸 Atlanta, Georgia, United States

Des Moines Location; 🇺🇸 Des Moines, Iowa, United States

Kansas City Location; 🇺🇸 Overland Park, Kansas, United States

Grand Rapids Location; 🇺🇸 Grand Rapids, Michigan, United States