CompletedNCT04520321INTREPIDUpdated Oct 30, 2023Results posted

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

A Phase 1/2 interventional study of TTHX1114(NM141) and Vehicle (placebo) in Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy and Pseudophakic Bullous Keratopathy, sponsored by Trefoil Therapeutics, Inc.. Completed at 8 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-10-30.

Sponsored by Trefoil Therapeutics, Inc. · Phase 1/2, Interventional, and Treatment

Phase: Phase 1/2
Study type: Interventional
Enrollment: 22
Allocation: Randomized
Ages: 18 Years and older
Sex: All

Study summary

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

Read the 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.

Conditions studied

Corneal Endothelial Dystrophy
Fuchs Endothelial Corneal Dystrophy
Pseudophakic Bullous Keratopathy
Endothelial Dysfunction

Browse trials for

Fuchs' Endothelial Dystrophy 92

In context

Fuchs' Endothelial Dystrophy

92 studies on the registry are indexed under Fuchs' Endothelial Dystrophy; 27 are open to participants now.

This study's enrollment of 22 is below the median of 50 across 62 interventional studies indexed under Fuchs' Endothelial Dystrophy.

Browse Fuchs' Endothelial Dystrophy studies →

Lead sponsor

Trefoil Therapeutics, Inc. is the lead sponsor of 4 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
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 use in the 30 days prior to Study Day 0 unless approved by the Medical Monitor
History of significant allergy, , or intolerance to any drug compound, food, or other substance
Unwilling to use

Terms in these criteria, in plain language(3)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Study design

Phase: Phase 1 / Phase 2
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 22 participants (actual)

Study arms

Placebo comparator
Vehicle (placebo)
Placebo weekly x 4
Other: Vehicle (placebo)
Experimental
Low dose
TTHX1114(NM141) low-dose weekly x 4
Drug: TTHX1114(NM141)
Experimental
Mid-dose
TTHX1114(NM141) mid-dose weekly x 4
Drug: TTHX1114(NM141)
Experimental
High-dose
TTHX1114(NM141) high-dose weekly x 4
Drug: TTHX1114(NM141)

Interventions

DrugTTHX1114(NM141)
engineered FGF-1 delivered intracamerally
OtherVehicle (placebo)
Placebo

What researchers measure

Primary outcomes

DLTs/ Adverse Reactions
Suspected Adverse Reactions Reported Following Study Drug Adminsitration
Time frame: Day 90

Results

Posted Oct 30, 2023

Participant flow

Participant flow — Overall Study
Milestone	Vehicle (Placebo)	Low Dose	Mid-dose	High-dose
Started	5	7	3	7
Completed	5	7	3	7
Not completed	0	0	0	0

Outcome measures

PrimaryDLTs/ Adverse Reactions

Suspected Adverse Reactions Reported Following Study Drug Adminsitration

Time frame:: Day 90
Reported as:: Count of participants · Participants

DLTs/ Adverse Reactions
Participants	Vehicle (Placebo)	Low Dose	Mid-dose	High-dose
DLTs/ Adverse Reactions	0	0	0	0

Adverse events

Collected over 90 days. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Vehicle (Placebo)	0/5 (0%)	0/5 (0%)	2/5 (40%)
Low Dose	0/7 (0%)	0/7 (0%)	1/7 (14.3%)
Mid-dose	0/3 (0%)	0/3 (0%)	2/3 (66.7%)
High-dose	0/7 (0%)	0/7 (0%)	3/7 (42.9%)

Most frequent other events

Showing 10 of 13

Most frequent other events
Event	Vehicle (Placebo)	Low Dose	Mid-dose	High-dose
Eye painEye disorders	0/5	0/7	1/3	1/7
Sinus congestionRespiratory, thoracic and mediastinal disorders	0/5	0/7	1/3	0/7
Conjunctival haemorrhageEye disorders	1/5	0/7	0/3	0/7
Foreign body sensation in eyesEye disorders	1/5	0/7	0/3	0/7
MalaiseGeneral disorders	1/5	0/7	0/3	0/7
DyspnoeaRespiratory, thoracic and mediastinal disorders	1/5	0/7	0/3	0/7
LymphoedemaVascular disorders	1/5	0/7	0/3	0/7
Eye irritationEye disorders	0/5	0/7	0/3	1/7
IritisEye disorders	0/5	0/7	0/3	1/7
PhotophobiaEye disorders	0/5	1/7	0/3	1/7

Baseline characteristics

Age, Continuous
Age, Continuous(years)	Vehicle (Placebo)	Low Dose	Mid-dose	High-dose	Total
Mean	76.6 ± 11.8	75.7 ± 7.8	81.3 ± 11.0	68.9 ± 11.1	74.5 ± 10.5

Sex: Female, Male
Sex: Female, Male(Participants)	Vehicle (Placebo)	Low Dose	Mid-dose	High-dose	Total
Female	5	5	2	7	19
Male	0	2	1	0	3

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Vehicle (Placebo)	Low Dose	Mid-dose	High-dose	Total
Hispanic or Latino	0	0	0	0	0
Not Hispanic or Latino	5	7	3	7	22
Unknown or Not Reported	0	0	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Vehicle (Placebo)	Low Dose	Mid-dose	High-dose	Total
American Indian or Alaska Native	5	7	3	7	22
Asian	0	0	0	0	0
Native Hawaiian or Other Pacific Islander	0	0	0	0	0
Black or African American	0	0	0	0	0
White	0	0	0	0	0
More than one race	0	0	0	0	0
Unknown or Not Reported	0	0	0	0	0

Study locations

8 sites

North Bay Eye Associates, Inc.
Petaluma, California 94954, United States
Levenson Eye Associates, Inc
Jacksonville, Florida 32204, United States
Chicago Corneal Consultants
Hoffman Estates, Illinois 60169, United States
Price Vision Group
Indianapolis, Indiana 46260, United States
Tauber Eye Center
Kansas City, Missouri 64111, United States
Vance Thompson Vision - Omaha
Omaha, Nebraska 68137, United States
Alterman, Modi and Wolter
Poughkeepsie, New York 12603, United States
Vance Thompson Vision - Sioux Falls
Sioux Falls, South Dakota 57108, United States

References and documents

Study documents

Study protocol ↗ · Sep 8, 2020

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Oct 30, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT04520321
Lead sponsor: Trefoil Therapeutics, Inc.
Responsible party: Sponsor
First posted: Aug 20, 2020
Start date: Aug 19, 2020
Primary completion: Apr 23, 2021
Completion: May 18, 2021
Results posted: Oct 30, 2023
Last update: Oct 30, 2023

Study contacts

Thomas M Tremblay, RN BSN

study director · Trefoil Therapeutics

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Oct 2022. You cannot join it, but the record below documents what was studied.

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Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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Studies of the drug tested here: TTHX1114

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Other studies from this sponsor

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