A Phase 1/ Phase 2 Study of TTHX1114(NM141)

Phase 1

Completed

• Conditions: Corneal Endothelial Dystrophy; Fuchs Endothelial Corneal Dystrophy; Pseudophakic Bullous Keratopathy; Endothelial Dysfunction
• Interventions: Other: Vehicle (placebo); Drug: TTHX1114(NM141)

• Registration Number: NCT04520321
• Lead Sponsor: Trefoil Therapeutics, Inc.

Brief Summary

Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study

Detailed Description

This is a prospective, multi-center, randomized, masked, vehicle-controlled, dose-escalation study that will include an observational (no intervention) sub-study. Eligible subjects with moderate to severe corneal endothelial dystrophy (defined as Endothelial Cell Density \< 2000 mm\^2) in at least one eye will be enrolled and randomized.

Study Timeline

• Study First Submitted: 2020-08-14
• Study First Submitted QC: 2020-08-18
• Study Start: 2020-08-19
• Study First Posted: 2020-08-20
• Primary Completion: 2021-04-23
• Study Completion: 2021-05-18
• Status Verified: 2022-10
• Results First Submitted: 2022-10-31
• Results First Submitted QC: 2023-10-27
• Last Update Submitted: 2023-10-27
• Results First Posted: 2023-10-30
• Last Update Posted: 2023-10-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

• Fuchs Endothelial Corneal Dystrophy, pseudophakic bullous keratopathy, or endothelial dysfunction/ insufficiency due to surgical intervention diagnosed more than 6 months prior to Study Day 0
• Central endothelial cell count of < 2000 mm^2 in at least one eye as determined by the central reading facility

Key

Exclusion Criteria

• Conditions that would impair examination of the anterior chamber structure
• Documented repeated elevated intra ocular pressure (in either eye)
• Corneal transplant (in either eye)
• Posterior Polymorphous Corneal Dystrophy (PPCD)
• History of uveitis or herpetic keratitis
• Cataract surgery within the past 3 months
• Refractive surgery (in the Study Eye)
• Anterior Chamber IOL placement (in the Study Eye)
• Active extra-ocular inflammation from any non-infectious or infectious cause within the past 6 months
• Expected or planned ocular surgery within the next 3 months
• Use of cytotoxic chemotherapy within the last 1 month
• Treatment with a rho kinase inhibitor within the last 3 months
• Use of cyclosporine ophthalmic emulsion or lifitegrast ophthalmic solution in the last 30 days
• Systemic or ophthalmic corticosteroid use in the 30 days prior to Study Day 0 unless approved by the Medical Monitor
• History of significant allergy, hypersensitivity, or intolerance to any drug compound, food, or other substance
• Unwilling to use birth control

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Vehicle (placebo)	Vehicle (placebo)	Placebo weekly x 4
Low dose	TTHX1114(NM141)	TTHX1114(NM141) low-dose weekly x 4
Mid-dose	TTHX1114(NM141)	TTHX1114(NM141) mid-dose weekly x 4
High-dose	TTHX1114(NM141)	TTHX1114(NM141) high-dose weekly x 4

Primary Outcome Measures

Name	Time	Method
DLTs/ Adverse Reactions	Day 90	Suspected Adverse Reactions Reported Following Study Drug Adminsitration

Trial Locations

Locations (8)

Tauber Eye Center; 🇺🇸 Kansas City, Missouri, United States

Chicago Corneal Consultants; 🇺🇸 Hoffman Estates, Illinois, United States

North Bay Eye Associates, Inc.; 🇺🇸 Petaluma, California, United States

Alterman, Modi and Wolter; 🇺🇸 Poughkeepsie, New York, United States

Price Vision Group; 🇺🇸 Indianapolis, Indiana, United States

Vance Thompson Vision - Omaha; 🇺🇸 Omaha, Nebraska, United States

Vance Thompson Vision - Sioux Falls; 🇺🇸 Sioux Falls, South Dakota, United States

Levenson Eye Associates, Inc; 🇺🇸 Jacksonville, Florida, United States