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

Emmecell

Status and phase: Completed; Phase 1

Conditions: Corneal Edema; Moderate Corneal Endothelial Decompensation; Endothelial Dysfunction; Bullous Keratopathy; Fuchs Dystrophy; Corneal Endothelial Dysfunction; Fuchs Endothelial Corneal Dystrophy; Corneal Endothelial Dystrophy; Pseudophakic Bullous Keratopathy

Treatments: Biological: EO2002 low dose; Biological: EO2002 mid dose; Biological: EO2002; Biological: EO2002 high dose

Study type: Interventional

Funder types: Industry

Identifiers

NCT04894110

EMME-001

Details and patient eligibility

About

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.

Full 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.

Enrollment: 42 patients

Sex: All

Ages: 21+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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42 participants in 4 patient groups

EO2002 treatment - Group 1

Experimental group

Treatment:

Biological: EO2002

EO2002 treatment - Group 2 - low dose

Active Comparator group

Treatment:

Biological: EO2002 low dose

EO2002 treatment - Group 2 - mid dose

Active Comparator group

Treatment:

Biological: EO2002 mid dose

EO2002 treatment - Group 2 - high dose

Active Comparator group

Treatment:

Biological: EO2002 high dose