Using N-acetyl cysteine eye drops to treat Fuchs Endothelial Corneal Dystrophy

Targeting Reactive Oxygen Species Production as a Novel Therapeutic in Fuch's Endothelial Corneal Dystrophy

Phase 2 Interventional Massachusetts Eye and Ear Infirmary · NCT04440280

This study is testing if N-acetyl cysteine eye drops can help people with Fuchs Endothelial Corneal Dystrophy and cataracts feel better and improve their eye health before surgery.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment45 (estimated)
Ages21 Years and up
SexAll
SponsorMassachusetts Eye and Ear Infirmary Academic / other
Locations1 site (Boston, Massachusetts)
Trial IDNCT04440280 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of topical N-acetyl cysteine (NAC) eye drops in reducing oxidative stress and providing cytoprotection in patients with Fuchs Endothelial Corneal Dystrophy (FECD). The study involves adult participants with advanced FECD and cataracts who are eligible for combined Descemet Membrane Endothelial Keratoplasty (DMEK) and cataract surgery. It is a single center, double-blind, placebo-controlled, randomized trial conducted at Massachusetts Eye and Ear, aiming to identify a pharmacotherapeutic intervention to prevent endothelial cell loss and improve graft survival. The trial compares different concentrations of NAC with a placebo to assess their impact on disease progression.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 21 and older diagnosed with advanced Fuchs Endothelial Corneal Dystrophy and visually significant cataracts who are eligible for DMEK surgery.

Not a fit: Patients with other corneal diseases, prior intraocular surgeries, or those who are pregnant or breastfeeding may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a non-surgical option to slow the progression of Fuchs Endothelial Corneal Dystrophy and improve patient outcomes.

How similar studies have performed: While the use of N-acetyl cysteine in ophthalmology has shown promise in other conditions, this specific application for Fuchs Endothelial Corneal Dystrophy is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

1. Male or female ≥21 years of age at time of surgical evaluation.
2. Diagnosis of advanced FECD and visually significant cataract
3. Indication for DMEK (Descemet Membrane Endothelial Keratoplasty) with concurrent cataract surgery
4. Written informed consent obtained from subject and ability for subject to comply with the requirements of the study.
5. Willingness and ability to adhere to medication regimen

Exclusion Criteria:

1. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study.
2. History of prior intraocular surgery in study eye including cataract, glaucoma and/or retina surgery
3. History of other corneal diseases, such as severe dry eye, corneal scars, pseudophakic bullous keratopathy, corneal degenerations, corneal infections
4. Use of ocular prescription medications except for lubricants, hyperosmotic agents, or ocular hypotensive agents
5. History of ocular surface infection within the past 30 days
6. Use of systemic, inhalational, or topical N-Acetylcysteine within the past 30 days
7. History of intolerance to topical N-Acetylcysteine
8. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.

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Where this trial is running

Boston, Massachusetts

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Fuchs Endothelial Corneal DystrophyFuchsoxidative stressN-Acetylcysteine ophthalmic dropsDescemet Membrane Endothelial KeratoplastyDMEK
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.