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

NCT ID: NCT04520321

Last Updated: 2023-10-30

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

22 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-08-19

Study Completion Date

2021-05-18

Brief Summary

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

Detailed Description

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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.

Conditions

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Corneal Endothelial Dystrophy Fuchs Endothelial Corneal Dystrophy Pseudophakic Bullous Keratopathy Endothelial Dysfunction

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Randomized, Masked, Vehicle-controlled, Dose-escalation study
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Vehicle (placebo)

Placebo weekly x 4

Group Type PLACEBO_COMPARATOR

Vehicle (placebo)

Intervention Type OTHER

Placebo

Low dose

TTHX1114(NM141) low-dose weekly x 4

Group Type EXPERIMENTAL

TTHX1114(NM141)

Intervention Type DRUG

engineered FGF-1 delivered intracamerally

Mid-dose

TTHX1114(NM141) mid-dose weekly x 4

Group Type EXPERIMENTAL

TTHX1114(NM141)

Intervention Type DRUG

engineered FGF-1 delivered intracamerally

High-dose

TTHX1114(NM141) high-dose weekly x 4

Group Type EXPERIMENTAL

TTHX1114(NM141)

Intervention Type DRUG

engineered FGF-1 delivered intracamerally

Interventions

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TTHX1114(NM141)

engineered FGF-1 delivered intracamerally

Intervention Type DRUG

Vehicle (placebo)

Placebo

Intervention Type OTHER

Eligibility Criteria

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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

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Trefoil Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Thomas M Tremblay, RN BSN

Role: STUDY_DIRECTOR

Trefoil Therapeutics

Locations

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North Bay Eye Associates, Inc.

Petaluma, California, United States

Site Status

Levenson Eye Associates, Inc

Jacksonville, Florida, United States

Site Status

Chicago Corneal Consultants

Hoffman Estates, Illinois, United States

Site Status

Price Vision Group

Indianapolis, Indiana, United States

Site Status

Tauber Eye Center

Kansas City, Missouri, United States

Site Status

Vance Thompson Vision - Omaha

Omaha, Nebraska, United States

Site Status

Alterman, Modi and Wolter

Poughkeepsie, New York, United States

Site Status

Vance Thompson Vision - Sioux Falls

Sioux Falls, South Dakota, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol

View Document

Other Identifiers

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TTHX-001

Identifier Type: -

Identifier Source: org_study_id

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