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Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Vehicle (placebo) | Placebo Comparator | Placebo weekly x 4 |
|
| Low dose | Experimental | TTHX1114(NM141) low-dose weekly x 4 |
|
| Mid-dose | Experimental | TTHX1114(NM141) mid-dose weekly x 4 |
|
| High-dose | Experimental | TTHX1114(NM141) high-dose weekly x 4 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TTHX1114(NM141) | Drug | engineered FGF-1 delivered intracamerally |
|
| Measure | Description | Time Frame |
|---|---|---|
| DLTs/ Adverse Reactions | Suspected Adverse Reactions Reported Following Study Drug Adminsitration | Day 90 |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Thomas M Tremblay, RN BSN | Trefoil Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| North Bay Eye Associates, Inc. | Petaluma | California | 94954 | United States | ||
| Levenson Eye Associates, Inc |
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| ID | Title | Description |
|---|---|---|
| FG000 | Vehicle (Placebo) | Placebo weekly x 4 Vehicle (placebo): Placebo |
| FG001 | Low Dose | TTHX1114(NM141) low-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 8, 2020 | Oct 27, 2023 |
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Randomized, Masked, Vehicle-controlled, Dose-escalation study
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| Vehicle (placebo) | Other | Placebo |
|
| 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 |
| FG002 | Mid-dose | TTHX1114(NM141) mid-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally |
| FG003 | High-dose | TTHX1114(NM141) high-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Vehicle (Placebo) | Placebo weekly x 4 Vehicle (placebo): Placebo |
| BG001 | Low Dose | TTHX1114(NM141) low-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally |
| BG002 | Mid-dose | TTHX1114(NM141) mid-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally |
| BG003 | High-dose | TTHX1114(NM141) high-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | DLTs/ Adverse Reactions | Suspected Adverse Reactions Reported Following Study Drug Adminsitration | Safety | Posted | Count of Participants | Participants | Day 90 |
|
|
|
90 days
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Vehicle (Placebo) | Placebo weekly x 4 Vehicle (placebo): Placebo | 0 | 5 | 0 | 5 | 2 | 5 |
| EG001 | Low Dose | TTHX1114(NM141) low-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally | 0 | 7 | 0 | 7 | 1 | 7 |
| EG002 | Mid-dose | TTHX1114(NM141) mid-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally | 0 | 3 | 0 | 3 | 2 | 3 |
| EG003 | High-dose | TTHX1114(NM141) high-dose weekly x 4 TTHX1114(NM141): engineered FGF-1 delivered intracamerally | 0 | 7 | 0 | 7 | 3 | 7 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Conjunctival haemorrhage | Eye disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Foreign body sensation in eyes | Eye disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Iritis | Eye disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Photophobia | Eye disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Punctate keratitis | Eye disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Hand fracture | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
| |
| Lymphoedema | Vascular disorders | MedDRA (Unspecified) | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Thomas M Tremblay, RN, BSN | Trefoil Therapeutics.com | 4153053491 | ttremblay@trefoiltherapeutics.com |
| Prot_001.pdf |
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| ID | Term |
|---|---|
| C562745 | Corneal Dystrophy, Posterior Polymorphous, 1 |
| D005642 | Fuchs' Endothelial Dystrophy |
| ID | Term |
|---|---|
| D003317 | Corneal Dystrophies, Hereditary |
| D003316 | Corneal Diseases |
| D005128 | Eye Diseases |
| D015785 | Eye Diseases, Hereditary |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|