Corneal EdemaPhase 1NCT04894110
Study of Safety and Tolerability of EO2002 in the Treatment of Corneal Edema
A Phase 1 study testing EO2002, EO2002 low dose and EO2002 mid dose in people with Corneal Edema and Corneal Endothelial Dysfunction, run by Emmecell at 8 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 21 years and older
- Conditions the study is for
- Corneal Edema, Corneal Endothelial Dysfunction, Corneal Endothelial Dystrophy
- What is being tested?
- EO2002, EO2002 low dose and EO2002 mid dose
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 8 sitesUnited States
- Study dates
- 22 Jun 2021to 3 Oct 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 15Show fewer
- 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.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Atlanta Location
Atlanta, Georgia, 30339
Des Moines Location
Des Moines, Iowa, 50266
Grand Rapids Location
Grand Rapids, Michigan, 49546
Los Angeles Location
Greater Los Angeles, California, 91208
Miami Location
Miami, Florida, 33136
Kansas City Location
Overland Park, Kansas, 66204
Palm Beach Gardens Location
Palm Beach Gardens, Florida, 33410
San Diego Location
San Diego, California, 92122
About this study
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)
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.
Read the 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
- Being tested
- EO2002, EO2002 low dose, EO2002 mid dose, EO2002 high dose
- Medical subject headings
- Corneal Edema, Corneal Dystrophy, Posterior Polymorphous, 1, Fuchs' Endothelial Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EO2002Biological
EO2002 (magnetic human corneal endothelial cells [HCECs]) with and without endothelial brushing (EB) or Descemet Stripping (DS)
EO2002 low doseBiological
Low dose of EO2002 (magnetic human corneal endothelial cells)
EO2002 mid doseBiological
Mid dose of EO2002 (magnetic human corneal endothelial cells)
EO2002 high doseBiological
High dose of EO2002 (magnetic human corneal endothelial cells)
| Study group | Role | What they receive |
|---|---|---|
| EO2002 treatment - Group 1 | Experimental | Not described Receives: Biological: EO2002 |
| EO2002 treatment - Group 2 - low dose | Active comparator | Not described Receives: Biological: EO2002 low dose |
| EO2002 treatment - Group 2 - mid dose | Active comparator | Not described Receives: Biological: EO2002 mid dose |
| EO2002 treatment - Group 2 - high dose | Active comparator | Not described Receives: Biological: EO2002 high dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 26 Weeks
Secondary outcomes (2)
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
Measured: 26 Weeks
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.
Measured: 26 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2021
- Study start22 Jun 2021
- Primary completion3 Oct 2024
- Study completion3 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Corneal EdemaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- Group 1: Open-Label, Dose-Escalation Study Group 2: Randomized, double masked
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emmecell
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Noelia Kunzevitzky, Study director, Emmecell
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04894110
- Sponsor's protocol ID
- EMME-001
- Status last verified
- Mar 2025
- First submitted
- 11 May 2021
- Last updated
- 5 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04894110
- Acronym / sponsor ID
- EMME-001
- Conditions
- Corneal Edema, Corneal Endothelial Dysfunction, Corneal Endothelial Dystrophy, Fuchs Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- EmmecellIndustry (pharma, biotech, devices)
- Study start
- 22 Jun 2021 (actual)
- Primary completion
- 3 Oct 2024 (actual)
- Study completion
- 3 Oct 2024 (actual)
- First posted
- 20 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- Model description
- Group 1: Open-Label, Dose-Escalation Study Group 2: Randomized, double masked
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 21 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EO2002EO2002 (magnetic human corneal endothelial cells [HCECs]) with and without endothelial brushing (EB) or Descemet Stripping (DS) | Biological | EO2002 treatment - Group 1 |
| EO2002 low doseLow dose of EO2002 (magnetic human corneal endothelial cells) | Biological | EO2002 treatment - Group 2 - low dose |
| EO2002 mid doseMid dose of EO2002 (magnetic human corneal endothelial cells) | Biological | EO2002 treatment - Group 2 - mid dose |
| EO2002 high doseHigh dose of EO2002 (magnetic human corneal endothelial cells) | Biological | EO2002 treatment - Group 2 - high dose |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability of EO2002]
Time frame: 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.
2 secondary outcomes
Effect of EO2002 (+/-EB or DS) on corneal thickness
Time frame: 26 Weeks
Effect of EO2002 (+/-EB or DS) on best corrected visual acuity (BCVA).
Time frame: 26 Weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2025.
- Individual participant dataSharing planned: No
Related trials for Corneal Edema
RecruitingPhase 2
Study of EO2002 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction
Corneal Edema, Fuchs
Active, not recruitingPhase 3
AURN001 Efficacy in Participants With Corneal Edema Secondary to Corneal Endothelial Dysfunction
Corneal Edema, Corneal Endothelial Dysfunction
Active, not recruiting
The Belgian Endothelial Surgical Transplant of the Cornea
Corneal Edema, Corneal Endothelial Disorder
Enrolling by invitationObservational study
The Effect of Topical Rho-kinase Inhibitors on Corneas of Patients With Fuchs Endothelial Corneal Dystrophy
Fuchs Endothelial Corneal Dystrophy, Cataract
Recruiting
Plasma Rich in Growth Factors in Corneal Endothelial Transplantation
Fuchs' Endothelial Dystrophy, Corneal Edema
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Noelia Kunzevitzky study director
Emmecell
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EO2002, EO2002 low dose and EO2002 mid dose in people with Corneal Edema and Corneal Endothelial Dysfunction, run by Emmecell at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability of EO2002] (time frame: 26 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Mar 2025.