Clinical trial NCT04520321: A Phase 1/ Phase 2 Study of TTHX1114(NM141)
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
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Corneal Endothelial Dystrophy
Fuchs Endothelial Corneal Dystrophy
Pseudophakic Bullous Keratopathy
Endothelial Dysfunction
Lead sponsor: Trefoil Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2020-08-20
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Randomized, Masked, Vehicle-controlled, Dose-escalation study
Primary purpose: TREATMENT

Brief summary: Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study

Detailed 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.

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

Primary outcomes:
measure: DLTs/ Adverse Reactions
timeFrame: Day 90
Secondary outcomes:
Other outcomes:
Locations:
city: Petaluma
country: United States
facility: North Bay Eye Associates, Inc.
state: California
city: Jacksonville
country: United States
facility: Levenson Eye Associates, Inc
state: Florida
city: Hoffman Estates
country: United States
facility: Chicago Corneal Consultants
state: Illinois
city: Indianapolis
country: United States
facility: Price Vision Group
state: Indiana
city: Kansas City
country: United States
facility: Tauber Eye Center
state: Missouri
city: Omaha
country: United States
facility: Vance Thompson Vision - Omaha
state: Nebraska
city: Poughkeepsie
country: United States
facility: Alterman, Modi and Wolter
state: New York
city: Sioux Falls
country: United States
facility: Vance Thompson Vision - Sioux Falls
state: South Dakota
Study officials:
affiliation: Trefoil Therapeutics
name: Thomas M Tremblay, RN BSN
role: STUDY_DIRECTOR
References: