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

Trefoil Therapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Endothelial Dysfunction; Fuchs Endothelial Corneal Dystrophy; Corneal Endothelial Dystrophy; Pseudophakic Bullous Keratopathy

Treatments: Drug: TTHX1114(NM141); Other: Vehicle (placebo)

Study type: Interventional

Funder types: Industry

Identifiers

NCT04520321

TTHX-001

Details and patient eligibility

About

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

Full description

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.

Enrollment: 22 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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22 participants in 4 patient groups, including a placebo group

Vehicle (placebo)

Placebo Comparator group

Description:

Placebo weekly x 4

Treatment:

Other: Vehicle (placebo)

Low dose

Experimental group

Description:

TTHX1114(NM141) low-dose weekly x 4

Treatment:

Drug: TTHX1114(NM141)

Mid-dose

Experimental group

Description:

TTHX1114(NM141) mid-dose weekly x 4

Treatment:

Drug: TTHX1114(NM141)

High-dose

Experimental group

Description:

TTHX1114(NM141) high-dose weekly x 4

Treatment:

Drug: TTHX1114(NM141)