Corneal Endothelial DystrophyPhase 1/2INTREPIDNCT04520321
A Phase 1/ Phase 2 Study of TTHX1114(NM141)
A Phase 1/2 study testing TTHX1114(NM141) in people with Corneal Endothelial Dystrophy and Fuchs Endothelial Corneal Dystrophy, run by Trefoil Therapeutics, Inc. at 8 sites in United States.
Trial summary
Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy
- What is being tested?
- TTHX1114(NM141)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- 8 sitesUnited States
- Study dates
- 19 Aug 2020to 18 May 2021
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 16Show fewer
- 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 locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Chicago Corneal Consultants
Hoffman Estates, Illinois, 60169
Price Vision Group
Indianapolis, Indiana, 46260
Levenson Eye Associates, Inc
Jacksonville, Florida, 32204
Tauber Eye Center
Kansas City, Missouri, 64111
Vance Thompson Vision - Omaha
Omaha, Nebraska, 68137
North Bay Eye Associates, Inc.
Petaluma, California, 94954
Alterman, Modi and Wolter
Poughkeepsie, New York, 12603
Vance Thompson Vision - Sioux Falls
Sioux Falls, South Dakota, 57108
About this study
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
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
- Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy, Endothelial Dysfunction
- Being tested
- TTHX1114(NM141), Vehicle (placebo)
- Medical subject headings
- Corneal Dystrophy, Posterior Polymorphous, 1, Fuchs' Endothelial Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TTHX1114(NM141)Drug
engineered FGF-1 delivered intracamerally
Vehicle (placebo)Other
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Vehicle (placebo) | Placebo comparator | Placebo weekly x 4 Receives: Other: Vehicle (placebo) |
| Low dose | Experimental | TTHX1114(NM141) low-dose weekly x 4 Receives: Drug: TTHX1114(NM141) |
| Mid-dose | Experimental | TTHX1114(NM141) mid-dose weekly x 4 Receives: Drug: TTHX1114(NM141) |
| High-dose | Experimental | TTHX1114(NM141) high-dose weekly x 4 Receives: Drug: TTHX1114(NM141) |
What is being measured
The primary outcome is the main question the study is designed to answer.
DLTs/ Adverse Reactions
Suspected Adverse Reactions Reported Following Study Drug Adminsitration
Measured: Day 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2020
- Study start19 Aug 2020
- Primary completion23 Apr 2021
- Study completion18 May 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Corneal Endothelial DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, Masked, Vehicle-controlled, Dose-escalation study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Trefoil Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Thomas M Tremblay, RN BSN, Study director, Trefoil Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04520321
- Sponsor's protocol ID
- TTHX-001
- Acronym
- INTREPID
- Status last verified
- Oct 2022
- First submitted
- 14 Aug 2020
- Last updated
- 30 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04520321
- Acronym / sponsor ID
- INTREPID / TTHX-001
- Conditions
- Corneal Endothelial Dystrophy, Fuchs Endothelial Corneal Dystrophy, Pseudophakic Bullous Keratopathy, Endothelial Dysfunction
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Trefoil Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Aug 2020 (actual)
- Primary completion
- 23 Apr 2021 (actual)
- Study completion
- 18 May 2021 (actual)
- First posted
- 20 Aug 2020 (actual)
- Results
- Posted 30 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- Model description
- Randomized, Masked, Vehicle-controlled, Dose-escalation study
| Arm | Type | Receives |
|---|---|---|
| Vehicle (placebo) | Placebo comparator | Other: Vehicle (placebo)Placebo weekly x 4 |
| Low dose | Experimental | Drug: TTHX1114(NM141)TTHX1114(NM141) low-dose weekly x 4 |
| Mid-dose | Experimental | Drug: TTHX1114(NM141)TTHX1114(NM141) mid-dose weekly x 4 |
| High-dose | Experimental | Drug: TTHX1114(NM141)TTHX1114(NM141) high-dose weekly x 4 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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 (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TTHX1114(NM141)engineered FGF-1 delivered intracamerally | Drug | High-dose, Low dose, Mid-dose |
| Vehicle (placebo)Placebo | Other | Vehicle (placebo) |
Primary outcome
DLTs/ Adverse Reactions
Time frame: Day 90
Suspected Adverse Reactions Reported Following Study Drug Adminsitration
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Corneal Endothelial Dystrophy
Active, not recruitingPhase 3
Descemet Endothelial Thickness Comparison Trial II
Fuchs, Fuchs Dystrophy
RecruitingPhase 2
Study of EO2002 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction
Corneal Edema, Fuchs
RecruitingObservational study
A Study to Test the Diagnostic Potential of Brillouin Microscopy for Corneal Ectasia
Keratoconus, Ectasia
RecruitingPhase 2
DT-168 in Keratoplasty Patients With Fuchs Endothelial Corneal Dystrophy
Fuchs Endothelial Corneal Dystrophy, Fuchs
Active, not recruiting
The Belgian Endothelial Surgical Transplant of the Cornea
Corneal Edema, Corneal Endothelial Disorder
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Thomas M Tremblay, RN BSN study director
Trefoil Therapeutics
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing TTHX1114(NM141) in people with Corneal Endothelial Dystrophy and Fuchs Endothelial Corneal Dystrophy, run by Trefoil Therapeutics, Inc. at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DLTs/ Adverse Reactions (time frame: Day 90). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2023.