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

NCT04894110 | Completed Phase 1 FDA Drug

• 1 other identifier: EMME-001
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 8

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.

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

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability of EO2002]

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

  26 Weeks

Secondary Outcomes (2)

• Effect of EO2002 (+/-EB or DS) on corneal thickness

  26 Weeks

• Effect of EO2002 (+/-EB or DS) on best corrected visual acuity (BCVA).

  26 Weeks

Study Arms (4)

EO2002 treatment - Group 1

EXPERIMENTAL

Biological: EO2002

EO2002 treatment - Group 2 - low dose

ACTIVE COMPARATOR

Biological: EO2002 low dose

EO2002 treatment - Group 2 - mid dose

ACTIVE COMPARATOR

Biological: EO2002 mid dose

EO2002 treatment - Group 2 - high dose

ACTIVE COMPARATOR

Biological: EO2002 high dose

Interventions

EO2002 BIOLOGICAL

EO2002 (magnetic human corneal endothelial cells \[HCECs\]) with and without endothelial brushing (EB) or Descemet Stripping (DS)

EO2002 treatment - Group 1

EO2002 low dose BIOLOGICAL

Low dose of EO2002 (magnetic human corneal endothelial cells)

EO2002 treatment - Group 2 - low dose

EO2002 mid dose BIOLOGICAL

Mid dose of EO2002 (magnetic human corneal endothelial cells)

EO2002 treatment - Group 2 - mid dose

EO2002 high dose BIOLOGICAL

High dose of EO2002 (magnetic human corneal endothelial cells)

EO2002 treatment - Group 2 - high dose

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• All ocular criteria apply to study eye unless otherwise noted.
• Age ≥ 21 years.
• Phakic or Pseudophakic with a posterior chamber intraocular lens
• Symptomatic corneal edema associated with endothelial dysfunction which may be secondary to Fuchs corneal dystrophy or pseudophakic bullous keratopathy.

You may not qualify if:

• All ocular criteria apply to study eye unless otherwise noted.
• Other corneal disease
• Anterior chamber intraocular lens, Sutured or scleral-fixated intraocular lens.
• Macular disease that in the investigator and/or sponsor's opinion would limit the ability of the subject to demonstrate improvement in BCVA.
• History of refractive surgery.
• Descemet membrane detachment.
• History of uveitis or other ocular inflammatory disease.
• History of incisional glaucoma surgery (e.g.,trabeculectomy, glaucoma drainage implant).
• Intraocular pressure \>21 or \<7 mm Hg
• Prior incisional eye surgery within 3 months prior to study treatment or penetrating or endothelial keratoplasty.
• History of ocular neoplasm.
• ETDRS BCVA in the fellow eye is worse than 35 letters (Snellen equivalent of 20/200).
• 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.
• 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.
• 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.

Sponsors & Collaborators

• Emmecell: lead

Study Sites (8)

Los Angeles Location

Greater Los Angeles, California, 91208, United States

Location

San Diego Location

San Diego, California, 92122, United States

Location

Miami Location

Miami, Florida, 33136, United States

Location

Palm Beach Gardens Location

Palm Beach Gardens, Florida, 33410, United States

Location

Atlanta Location

Atlanta, Georgia, 30339, United States

Location

Des Moines Location

Des Moines, Iowa, 50266, United States

Location

Kansas City Location

Overland Park, Kansas, 66204, United States

Location

Grand Rapids Location

Grand Rapids, Michigan, 49546, United States

Location

MeSH Terms

Conditions

Corneal Edema; Corneal Dystrophy, Posterior Polymorphous, 1; Fuchs' Endothelial Dystrophy

Condition Hierarchy (Ancestors)

Corneal Diseases; Eye Diseases; Corneal Dystrophies, Hereditary; Eye Diseases, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Noelia Kunzevitzky

  Emmecell

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Group 1: Open-Label, Dose-Escalation Study Group 2: Randomized, double masked

Study Record Dates

• First Submitted: May 11, 2021
• First Posted: May 20, 2021
• Study Start: June 22, 2021
• Primary Completion: October 3, 2024
• Study Completion: October 3, 2024
• Last Updated: March 5, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)