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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04520321
Recruitment Status : Completed
First Posted : August 20, 2020
Last Update Posted : May 26, 2021
Information provided by (Responsible Party):
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

Condition or disease Intervention/treatment Phase
Corneal Endothelial Dystrophy Fuchs Endothelial Corneal Dystrophy Pseudophakic Bullous Keratopathy Endothelial Dysfunction Drug: TTHX1114(NM141) Other: Vehicle (placebo) Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Randomized, Masked, Vehicle-controlled, Dose-escalation study
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : August 19, 2020
Actual Primary Completion Date : April 23, 2021
Actual Study Completion Date : May 18, 2021

Arm Intervention/treatment
Placebo Comparator: Vehicle (placebo)
Placebo weekly x 4
Other: Vehicle (placebo)

Experimental: Low dose
TTHX1114(NM141) low-dose weekly x 4
Drug: TTHX1114(NM141)
engineered FGF-1 delivered intracamerally

Experimental: Mid-dose
TTHX1114(NM141) mid-dose weekly x 4
Drug: TTHX1114(NM141)
engineered FGF-1 delivered intracamerally

Experimental: High-dose
TTHX1114(NM141) high-dose weekly x 4
Drug: TTHX1114(NM141)
engineered FGF-1 delivered intracamerally

Primary Outcome Measures :
  1. Change in Corneal Endothelial Cell Count [ Time Frame: 56 Days ]
    Measured with Specular Microscopy

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04520321

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United States, California
North Bay Eye Associates, Inc.
Petaluma, California, United States, 94954
United States, Florida
Levenson Eye Associates, Inc
Jacksonville, Florida, United States, 32204
United States, Illinois
Chicago Corneal Consultants
Hoffman Estates, Illinois, United States, 60169
United States, Indiana
Price Vision Group
Indianapolis, Indiana, United States, 46260
United States, Missouri
Tauber Eye Center
Kansas City, Missouri, United States, 64111
United States, Nebraska
Vance Thompson Vision - Omaha
Omaha, Nebraska, United States, 68137
United States, New York
Alterman, Modi and Wolter
Poughkeepsie, New York, United States, 12603
United States, South Dakota
Vance Thompson Vision - Sioux Falls
Sioux Falls, South Dakota, United States, 57108
Sponsors and Collaborators
Trefoil Therapeutics, Inc.
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Study Director: Thomas M Tremblay, RN BSN Trefoil Therapeutics
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Responsible Party: Trefoil Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04520321    
Other Study ID Numbers: TTHX-001
First Posted: August 20, 2020    Key Record Dates
Last Update Posted: May 26, 2021
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Corneal Dystrophies, Hereditary
Fuchs' Endothelial Dystrophy
Corneal Diseases
Eye Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn