NCT04894110

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

A Phase 1, Prospective, Multi-Center, Open-Label, Dose-Escalation Study to Assess the Safety, and Tolerability of EO2002 with and Without Endothelial Brushing or Descemet Stripping in the Treatment of Corneal Edema (EMME-001)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 42 (actual)
• Sponsor: Emmecell · Industry
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Corneal Edema
• Corneal Endothelial Dysfunction
• Corneal Endothelial Dystrophy
• Fuchs Dystrophy
• Fuchs Endothelial Corneal Dystrophy
• Pseudophakic Bullous Keratopathy
• Bullous Keratopathy
• Endothelial Dysfunction
• Moderate Corneal Endothelial Decompensation

Interventions

Type	Name	Description
BIOLOGICAL	EO2002	EO2002 (magnetic human corneal endothelial cells \[HCECs\]) with and without endothelial brushing (EB) or Descemet Stripping (DS)
BIOLOGICAL	EO2002 low dose	Low dose of EO2002 (magnetic human corneal endothelial cells)
BIOLOGICAL	EO2002 mid dose	Mid dose of EO2002 (magnetic human corneal endothelial cells)
BIOLOGICAL	EO2002 high dose	High dose of EO2002 (magnetic human corneal endothelial cells)

Timeline

• Start date: 2021-06-22
• Primary completion: 2024-10-03
• Completion: 2024-10-03
• First posted: 2021-05-20
• Last updated: 2025-03-05

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study