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

Primary Purpose

Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
TTHX1114(NM141)
Vehicle (placebo)
Sponsored by
Trefoil Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Corneal Endothelial Dystrophy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • North Bay Eye Associates, Inc.
  • Levenson Eye Associates, Inc
  • Chicago Corneal Consultants
  • Price Vision Group
  • Tauber Eye Center
  • Vance Thompson Vision - Omaha
  • Alterman, Modi and Wolter
  • Vance Thompson Vision - Sioux Falls

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Placebo Comparator

Experimental

Experimental

Experimental

Arm Label

Vehicle (placebo)

Low dose

Mid-dose

High-dose

Arm Description

Placebo weekly x 4

TTHX1114(NM141) low-dose weekly x 4

TTHX1114(NM141) mid-dose weekly x 4

TTHX1114(NM141) high-dose weekly x 4

Outcomes

Primary Outcome Measures

Change in Corneal Endothelial Cell Count
Measured with Specular Microscopy

Secondary Outcome Measures

Full Information

First Posted
August 14, 2020
Last Updated
May 25, 2021
Sponsor
Trefoil Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04520321
Brief Title
A Phase 1/ Phase 2 Study of TTHX1114(NM141)
Acronym
INTREPID
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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
August 19, 2020 (Actual)
Primary Completion Date
April 23, 2021 (Actual)
Study Completion Date
May 18, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Trefoil Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy, Endothelial Dysfunction

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Randomized, Masked, Vehicle-controlled, Dose-escalation study
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
22 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Vehicle (placebo)
Arm Type
Placebo Comparator
Arm Description
Placebo weekly x 4
Arm Title
Low dose
Arm Type
Experimental
Arm Description
TTHX1114(NM141) low-dose weekly x 4
Arm Title
Mid-dose
Arm Type
Experimental
Arm Description
TTHX1114(NM141) mid-dose weekly x 4
Arm Title
High-dose
Arm Type
Experimental
Arm Description
TTHX1114(NM141) high-dose weekly x 4
Intervention Type
Drug
Intervention Name(s)
TTHX1114(NM141)
Intervention Description
engineered FGF-1 delivered intracamerally
Intervention Type
Other
Intervention Name(s)
Vehicle (placebo)
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Change in Corneal Endothelial Cell Count
Description
Measured with Specular Microscopy
Time Frame
56 Days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Thomas M Tremblay, RN BSN
Organizational Affiliation
Trefoil Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
North Bay Eye Associates, Inc.
City
Petaluma
State/Province
California
ZIP/Postal Code
94954
Country
United States
Facility Name
Levenson Eye Associates, Inc
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32204
Country
United States
Facility Name
Chicago Corneal Consultants
City
Hoffman Estates
State/Province
Illinois
ZIP/Postal Code
60169
Country
United States
Facility Name
Price Vision Group
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
Tauber Eye Center
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64111
Country
United States
Facility Name
Vance Thompson Vision - Omaha
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68137
Country
United States
Facility Name
Alterman, Modi and Wolter
City
Poughkeepsie
State/Province
New York
ZIP/Postal Code
12603
Country
United States
Facility Name
Vance Thompson Vision - Sioux Falls
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57108
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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