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

NCT04440280 | Recruiting Phase 2 FDA Drug

• 1 other identifier: 2020P001909
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

This protocol will investigate whether topical application of N-acetyl cysteine (NAC) eye drops decreases oxidative stress and confers cytoprotection in patients with FECD.

Conditions

Fuchs Endothelial Corneal Dystrophy

Keywords

Fuchs; Fuchs Endothelial Corneal Dystrophy; oxidative stress; N-Acetylcysteine ophthalmic drops; Descemet Membrane Endothelial Keratoplasty; DMEK

Outcome Measures

Primary Outcomes (1)

• Level of H2O2 in the aqueous humor

  The primary endpoint is the level of H2O2 in the aqueous humor obtained at time of DMEK surgery.

  DMEK Surgery occurs 28 to 42 days after participant enrollment

Secondary Outcomes (6)

• Assessment of safety of NAC eye drops in patients with FECD by documenting the number and severity of adverse events.

  Ongoing safety assessment will occur starting on the first day when subjects receive the study drug, through the pre-operative visit, surgery and post-surgical follow-up visits, a period of approximately 6-7 weeks.

• Assessment of tolerability of N-acetyl cysteine drops in patients with FECD by documenting participants' reports of irritation, burning, itchiness or redness in their eyes while taking the study drug

  Tolerability will be assessed after participants have taken study drug for 28 days or earlier, if the participants report difficulties tolerating the drug.

• Change in patient-reported visual disability after NAC administration

  Assessed when subject starts study drug and again, 21 to 28 days later.

• Change in central corneal thickness after NAC administration

  Assessed when subject starts study drug and again, 21 to 28 days later.

• Change in corneal endothelial cell counts after NAC administration

  Assessed when subject starts study drug and again, 21 to 28 days later.

Study Arms (3)

NAC 10% group

ACTIVE COMPARATOR

Subjects in this group will be treated with eye drops containing a 10% solution of N-acetyl cysteine. Topical NAC is a well-tolerated medication that has many applications in ophthalmology including dry eye disease and meibomian gland dysfunction.

Drug: N-acetyl cysteine (NAC) 10% solution

NAC 20% group

ACTIVE COMPARATOR

Subjects in this group will be treated with eye drops containing a 20% solution of N-acetyl cysteine. Topical NAC is a well-tolerated medication that has many applications in ophthalmology including dry eye disease and meibomian gland dysfunction.

Drug: N-acetyl cysteine (NAC) 20% solution

Placebo group

PLACEBO COMPARATOR

Subjects in this group will be treated with a placebo (Visine Tears Dry Eye Relief artificial tears ophthalmic solution.)

Drug: Visine Dry Eye Relief eye drops

Interventions

N-acetyl cysteine (NAC) 10% solution DRUG

N-acetyl cysteine is a sterile, unpreserved eye drop containing 10% (100 mg/mL) of acetylcysteine.

Also known as: acetylcysteine

NAC 10% group

N-acetyl cysteine (NAC) 20% solution DRUG

N-acetyl cysteine is a sterile, unpreserved eye drop containing 20% (200 mg/mL) of acetylcysteine.

Also known as: acetylcysteine

NAC 20% group

Visine Dry Eye Relief eye drops DRUG

Visine Tears Dry Eye Relief artificial tears ophthalmic solution

Placebo group

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

• Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study.
• History of prior intraocular surgery in study eye including cataract, glaucoma and/or retina surgery
• History of other corneal diseases, such as severe dry eye, corneal scars, pseudophakic bullous keratopathy, corneal degenerations, corneal infections
• Use of ocular prescription medications except for lubricants, hyperosmotic agents, or ocular hypotensive agents
• History of ocular surface infection within the past 30 days
• Use of systemic, inhalational, or topical N-Acetylcysteine within the past 30 days
• History of intolerance to topical N-Acetylcysteine
• 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.

Sponsors & Collaborators

• Massachusetts Eye and Ear Infirmary: lead

Study Sites (1)

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, 02114, United States

RECRUITING

MeSH Terms

Conditions

Fuchs' Endothelial Dystrophy

Interventions

Acetylcysteine; Solutions

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins; Pharmaceutical Preparations

Study Officials

• Ula Jurkunas, MD

  Mass Eye and Ear

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Michael Cheung, MSc, CCRP

CONTACT

6175736060; mcheung0@meei.harvard.edu

Ula Jurkunas, MD

CONTACT

617-573-4330; Ula_Jurkunas@meei.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: This is a double-blind trial, where neither the study participant nor the investigators will know to which treatment group the study participant has been assigned.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor, Harvard Medical School

Model Details: Initially, 30 eyes from up to 30 participants will be randomized 1:1 to one of two groups, a group administering N-acetyl cysteine (NAC) 10% and a group administering Visine Tears Dry Eye Relief artificial tears ophthalmic solution (the placebo). After interim analysis of the study with 30 eyes, if a significant difference in the primary endpoint between NAC 10% and placebo is not found, an additional 15 eyes from up to 15 participants will be enrolled to investigate 20% NAC.

Study Record Dates

• First Submitted: June 11, 2020
• First Posted: June 19, 2020
• Study Start: September 16, 2020
• Primary Completion: April 29, 2026
• Study Completion (Estimated): April 30, 2027
• Last Updated: September 29, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)