A Safety and Efficacy Trial of TTHX1114 in People With CED (OPTIC)

A Phase 2 Clinical Trial to Assess the Safety and Observe the Potential Benefit of TTHX1114 Delivered Via Intra-Cameral (IC) Injection

Expanded access, open label study at a single dose level in patients with CED that in the opinion of the investigators might benefit from TTHX1114

Study Overview

• Status: Completed
• Conditions: Corneal Endothelial Dystrophy
• Intervention / Treatment: Drug: TTHX1114

• Study Type: Interventional
• Enrollment (Actual): 41
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Trefoil Study Site
  • Missouri
    • Washington, Missouri, United States, 30090
      • Trefoil Study Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male or female, 18 years of age or older
• Subjects who are women of childbearing potential (WOCBP) must be using an acceptable method of birth control (See Section 6.4.3)
• Proposed Study Eye that has been diagnosed with an ocular condition that, in the opinion of the Investigator, could possibly benefit from TTHX1114 administration, and has a measurable efficacy endpoint
• Fellow Eye with 20/100 BCVA or better
• No concurrent ocular or medical condition that would impair the assessment of safety and efficacy

Exclusion Criteria:

• Prior exposure to TTHX1114
• Intolerance, hypersensitivity, or significant allergy to any drug compound, food, or other substance (Note: This includes all components and excipients of the study drug)
• Current or recent (e.g., the 28 days prior to Study Day 0) participation in any other, interventional clinical research study

• History of:

  • Ocular cancer (including melanoma)
  • Herpetic keratitis
  • Documented and repeated elevated IOP in either eye
  • Posterior Polymorphous Corneal Dystrophy (PPCD; aka Schlichting dystrophy)
  • Uveitis
• Use of any concomitant medications that may interfere with the assessment of safety and efficacy
• Any other reason (e.g., serious systemic disease or uncontrolled medical condition) that, in the opinion of the Investigator could increase the subject's risk, interfere with the interpretation of the study results, or affect the subject's ability to provide informed consent or comply with the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: TTHX1114 weekly x 5; TTHX1114 via IC injection weekly x 5	Drug: TTHX1114; engineered FGF-1

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Specular Microscopy	Corneal Endothelial Cell Density	Day 28

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pachymetry	Central Corneal Thickness	Day 28

Collaborators and Investigators

• Sponsor: Trefoil Therapeutics, Inc.
• Investigators: Study Director: Thomas Tremblay, Trefoil Therapeutics.com

Study record dates

Study Major Dates

• Study Start (Actual): November 30, 2021
• Primary Completion (Actual): August 29, 2023
• Study Completion (Actual): October 31, 2023

Study Registration Dates

• First Submitted: March 19, 2021
• First Submitted That Met QC Criteria: March 19, 2021
• First Posted (Actual): March 23, 2021

Study Record Updates

• Last Update Posted (Actual): November 7, 2023
• Last Update Submitted That Met QC Criteria: November 6, 2023
• Last Verified: November 1, 2023

More Information

Terms related to this study

• Other Study ID Numbers: TTHX-901

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No