Actively Recruiting
Targeting Reactive Oxygen Species Production as a Novel Therapeutic in Fuch's Endothelial Corneal Dystrophy
Led by Massachusetts Eye and Ear Infirmary · Updated on 2025-09-29
45
Participants Needed
1
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Fuchs Endothelial Corneal Dystrophy (FECD) is a common corneal disorder affecting about 4% of people over 40 in the US. This research evaluates whether applying N-acetyl cysteine (NAC) eye drops can reduce oxidative stress and protect cells in patients with FECD. There are currently no medical treatments to halt FECD progression, and corneal transplantation is the main option. This trial aims to find a drug therapy to prevent cell loss in early to late FECD stages and support graft survival after surgery. This is a single-center, double-blind, placebo-controlled, randomized study involving adult participants with advanced FECD and cataracts who need combined Descemet Membrane Endothelial Keratoplasty (DMEK) and cataract surgery. Participants will be assigned to receive either 10% NAC eye drops, 20% NAC eye drops, or placebo (artificial tears). They will self-administer one drop four times daily in the eye planned for surgery over 28 days before the operation. If no significant difference is found after the initial 30 eyes, an additional 15 eyes will be enrolled for 20% NAC treatment. Participants will complete visual disability questionnaires and tolerability and adherence surveys before and after using the study medication. Corneal measurements and images will be taken. During surgery, aqueous humor fluid and corneal tissue normally discarded will be collected for research. Researchers will measure levels of hydrogen peroxide in the eye fluid, monitor safety and side effects, and assess changes in corneal thickness, cell counts, tomography, and patient-reported vision. The study includes ongoing safety monitoring through surgery and post-operative follow-up over about 6 to 7 weeks.
CONDITIONS
Brief Title
Targeting Reactive Oxygen Species Production as a Novel Therapeutic in Fuch's Endothelial Corneal Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 21 years or older at time of surgical evaluation
- Diagnosis of advanced Fuchs Endothelial Corneal Dystrophy and visually significant cataract
- Indication for Descemet Membrane Endothelial Keratoplasty (DMEK) with concurrent cataract surgery
- Written informed consent obtained and ability to comply with study requirements
- Willingness and ability to follow medication regimen
You will not qualify if you...
- Pregnant, breastfeeding, or unwilling to use birth control during the study
- Prior intraocular surgery in the study eye including cataract, glaucoma, or retina surgery
- History of other corneal diseases such as severe dry eye, corneal scars, pseudophakic bullous keratopathy, corneal degenerations, or corneal infections
- Use of ocular prescription medications except lubricants, hyperosmotic agents, or ocular hypotensive agents
- Ocular surface infection within the past 30 days
- Use of systemic, inhalational, or topical N-acetyl cysteine within the past 30 days
- History of intolerance to topical N-acetyl cysteine
- Any condition that may compromise safety or data quality according to the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 28 days
Participants will self-administer 1 drop of study drug (N-acetyl cysteine or placebo) four times a day in the operative eye for 28 days prior to combined DMEK and cataract surgery. Before and after using the study medication, participants will complete visual disability questionnaires and have corneal measurements and images taken. A tolerability and medication adherence questionnaire will be completed after using the study medication.
2 visits (baseline and pre-operative)
Duration - 1 day
Participants undergo combined Descemet Membrane Endothelial Keratoplasty (DMEK) and cataract surgery. During surgery, aqueous fluid is collected for analysis and corneal tissue removed during transplantation is retained for research.
1 visit (surgery day, in-person)
Duration - 6 to 7 weeks
Participants are monitored for safety and tolerability of the study drug through post-surgical follow-up visits lasting approximately 6 to 7 weeks.
Weekly visits for up to 7 weeks
Trial Site Locations
Total: 1 location
1
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States, 02114
Actively Recruiting
Research Team
M
Michael Cheung, MSc, CCRP
U
Ula Jurkunas, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here