Back to resultsStudy of Safety and Tolerability of EO2002 in the Treatment of Corneal Edema
Primary Purpose
Corneal Edema, Corneal Endothelial Dysfunction, Corneal Endothelial Dystrophy
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
EO2002
EO2002 low dose
EO2002 mid dose
EO2002 high dose
Sponsored by
About this trial
This is an interventional treatment trial for Corneal Edema
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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).
- IOP >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.
Sites / Locations
- San Diego LocationRecruiting
- Denver LocationRecruiting
- Miami LocationRecruiting
- Palm Beach Gardens LocationRecruiting
- Atlanta LocationRecruiting
- Des Moines LocationRecruiting
- Kansas City LocationRecruiting
- Boston LocationRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Active Comparator
Active Comparator
Active Comparator
Arm Label
EO2002 treatment - Group 1
EO2002 treatment - Group 2 - low dose
EO2002 treatment - Group 2 - mid dose
EO2002 treatment - Group 2 - high dose
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Effect of EO2002 (+/-EB or DS) on corneal thickness
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).
Evaluate the effect of a single injection of EO2002 (+/-EB or DS) on best corrected visual acuity (BCVA) as determined by ETDRS letter score.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04894110
Brief Title
Study of Safety and Tolerability of EO2002 in the Treatment of Corneal Edema
Official Title
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)
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
June 22, 2021 (Actual)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
December 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Emmecell
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Model Description
Group 1: Open-Label, Dose-Escalation Study Group 2: Randomized, double masked
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
42 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
EO2002 treatment - Group 1
Arm Type
Experimental
Arm Title
EO2002 treatment - Group 2 - low dose
Arm Type
Active Comparator
Arm Title
EO2002 treatment - Group 2 - mid dose
Arm Type
Active Comparator
Arm Title
EO2002 treatment - Group 2 - high dose
Arm Type
Active Comparator
Intervention Type
Biological
Intervention Name(s)
EO2002
Intervention Description
EO2002 (magnetic human corneal endothelial cells [HCECs]) with and without endothelial brushing (EB) or Descemet Stripping (DS)
Intervention Type
Biological
Intervention Name(s)
EO2002 low dose
Intervention Description
Low dose of EO2002 (magnetic human corneal endothelial cells)
Intervention Type
Biological
Intervention Name(s)
EO2002 mid dose
Intervention Description
Mid dose of EO2002 (magnetic human corneal endothelial cells)
Intervention Type
Biological
Intervention Name(s)
EO2002 high dose
Intervention Description
High dose of EO2002 (magnetic human corneal endothelial cells)
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability of EO2002]
Description
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability of EO2002 (+/- EB or Descemet Stripping)] in subjects with corneal edema at week 26.
Time Frame
26 Weeks
Secondary Outcome Measure Information:
Title
Effect of EO2002 (+/-EB or DS) on corneal thickness
Description
Evaluate the effect of a single injection of EO2002 (+/-EB or DS) on corneal thickness as determined by pachymetry
Time Frame
26 Weeks
Title
Effect of EO2002 (+/-EB or DS) on best corrected visual acuity (BCVA).
Description
Evaluate the effect of a single injection of EO2002 (+/-EB or DS) on best corrected visual acuity (BCVA) as determined by ETDRS letter score.
Time Frame
26 Weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Emmecell Study Contact
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Noelia Kunzevitzky
Organizational Affiliation
Emmecell
Official's Role
Study Director
Facility Information:
Facility Name
San Diego Location
City
San Diego
State/Province
California
ZIP/Postal Code
92122
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
Facility Name
Denver Location
City
Littleton
State/Province
Colorado
ZIP/Postal Code
80120
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
Facility Name
Miami Location
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
Facility Name
Palm Beach Gardens Location
City
Palm Beach Gardens
State/Province
Florida
ZIP/Postal Code
33410
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
Facility Name
Atlanta Location
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30339
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
Facility Name
Des Moines Location
City
Des Moines
State/Province
Iowa
ZIP/Postal Code
50266
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
Facility Name
Kansas City Location
City
Overland Park
State/Province
Kansas
ZIP/Postal Code
66204
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
Facility Name
Boston Location
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02111
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Principal Investigator
Phone
650-769-4232
Email
clinicaltrials@emmecell.com
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Study of Safety and Tolerability of EO2002 in the Treatment of Corneal Edema
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