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

A Phase 1/ Phase 2 Study Evaluating the Safety and Efficacy of the Investigational New Drug TTHX1114(NM141) on the Regeneration of Corneal Endothelial Cells in Patients With Corneal Endothelial Dystrophies Following Intracameral Delivery


Lead Sponsor: Trefoil Therapeutics, Inc.

Source Trefoil Therapeutics, Inc.
Brief Summary

Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study that includes a non-interventional observational sub-study in which subjects will undergo (standard) ocular assessments

Detailed Description

This is a prospective, multi-center, randomized, masked, vehicle-controlled, dose-escalation study that will include an observational (no intervention) sub-study. Up to 71 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 in the main study. The Observational Sub-study will enroll approximately 25 to 50 subjects.

Overall Status Recruiting
Start Date August 19, 2020
Completion Date December 2021
Primary Completion Date August 2021
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Change in Corneal Endothelial Cell Count 56 Days
Enrollment 22

Intervention Type: Drug

Intervention Name: TTHX1114(NM141)

Description: engineered FGF-1 delivered intracamerally

Intervention Type: Other

Intervention Name: Vehicle (placebo)

Description: Placebo

Arm Group Label: Vehicle (placebo)



Key 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

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Thomas M Tremblay, RN BSN Study Director Trefoil Therapeutics
Overall Contact

Last Name: Thomas M Tremblay, RN BSN

Phone: (415) 305-3491

Email: [email protected]

Facility: Status: Contact:
North Bay Eye Associates, Inc. | Petaluma, California, 94954, United States Recruiting Eduardo Chavez 707-769-2240
Levenson Eye Associates, Inc | Jacksonville, Florida, 32204, United States Recruiting Vontrece Williams 904-701-2417
Chicago Corneal Consultants | Hoffman Estates, Illinois, 60169, United States Recruiting Parag Majmudar, MD
Price Vision Group | Indianapolis, Indiana, 46260, United States Recruiting Marianne Price, PhD 317-814-2990 [email protected]
Tauber Eye Center | Kansas City, Missouri, 64111, United States Recruiting Megan Hefter
Vance Thompson Vision - Omaha | Omaha, Nebraska, 68137, United States Recruiting 402-704-3236
Alterman, Modi and Wolter | Poughkeepsie, New York, 12603, United States Recruiting Sarah Ortiz Piza 204
Vance Thompson Vision - Sioux Falls | Sioux Falls, South Dakota, 57108, United States Recruiting 605-231-9716
Location Countries

United States

Verification Date

December 2020

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 4
Arm Group

Label: Vehicle (placebo)

Type: Placebo Comparator

Description: Placebo weekly x 4

Label: Low dose

Type: Experimental

Description: TTHX1114(NM141) low-dose weekly x 4

Label: Mid-dose

Type: Experimental

Description: TTHX1114(NM141) mid-dose weekly x 4

Label: High-dose

Type: Experimental

Description: TTHX1114(NM141) high-dose weekly x 4

Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Intervention Model Description: Randomized, Masked, Vehicle-controlled, Dose-escalation study

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov

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