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This study will enroll subjects with stage 2 or 3 neurotrophic keratitis. Subjects will be randomized in a 1:1 ratio to the CSB-001 investigational treatment arm or vehicle control arm. All subjects will dose with the randomized treatment four times daily for 8 weeks (controlled treatment phase). During the controlled treatment phase, subjects will return to the clinic weekly from Day 0 to Week 8, and again at Week 10. Subjects randomized to the vehicle arm who are not healed will have the opportunity to participate in an open-label uncontrolled treatment phase.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CSB-001 Investigational Treatment Arm | Experimental | One drop CSB-001 four times daily for 8 weeks in the study eye |
|
| Vehicle Control Arm | Placebo Comparator | One drop matching vehicle four times daily for 8 weeks in the study eye |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CSB-001 Ophthalmic Solution 0.1% | Biological | CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy as Assessed by Complete Corneal Healing | Percentage of subjects achieving complete corneal healing in study eye as assessed by the Central Reading Center (CRC). Complete corneal healing was defined as absence of corneal staining in the area of the defect/ulcer (i.e., 0 mm lesion) at Week 8 and the absence of corneal staining in the area of the defect/ulcer (i.e., 0 mm lesion) at Week 10. | Week 8 through Week 10 |
| Safety as Assessed by Adverse Event Reporting | Number of participants with ocular and systemic adverse events | Screening (Day 0) through Week 10 |
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Inclusion Criteria:
Exclusion Criteria:
Note: Other inclusion/exclusion criteria apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Eye Care North | Cave Creek | Arizona | 85331 | United States | ||
| Loma Linda University Eye Institute |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35266655 | Derived | Hamrah P, Yavuz Saricay L, Ozmen MC. Cutting Edge: Topical Recombinant Nerve Growth Factor for the Treatment of Neurotrophic Keratopathy-Biologicals as a Novel Therapy for Neurotrophic Keratopathy. Cornea. 2022 Jun 1;41(6):673-679. doi: 10.1097/ICO.0000000000002974. Epub 2022 Feb 2. |
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| ID | Title | Description |
|---|---|---|
| FG000 | CSB-001 Investigational Treatment Arm | One drop CSB-001 four times daily for 8 weeks in the study eye CSB-001 Ophthalmic Solution 0.1%: CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor) |
| FG001 | Vehicle Control Arm |
| 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: CSB-C20-003 Protocol v5 21July2023 Part 1 | Jul 21, 2023 |
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| Vehicle Control | Biological | Matching vehicle control without the drug substance |
|
| Loma Linda |
| California |
| 92354 |
| United States |
| Nvision Clinical Research, LLC | Los Angeles | California | 90013 | United States |
| Global Research Foundation | Los Angeles | California | 90041 | United States |
| UCLA Stein Eye Institute | Los Angeles | California | 90095 | United States |
| LoBue Laser and Eye Medical Center, Inc. | Murrieta | California | 92562 | United States |
| California Eye Specialists Medical Group Inc. | Pasadena | California | 91107 | United States |
| Martel Eye Medical Group | Rancho Cordova | California | 95670 | United States |
| Gordon Schanzlin New Vision Institute | San Diego | California | 92122 | United States |
| Atlantis Eyecare | Torrance | California | 90505 | United States |
| University of Colorado - Dept. of Ophthalmology | Aurora | Colorado | 80045 | United States |
| Vision Institute | Colorado Springs | Colorado | 80907 | United States |
| Corneal Consultants of Colorado | Littleton | Colorado | 80120 | United States |
| Advanced Research | Boynton Beach | Florida | 33437 | United States |
| Bowden Eye and Associates | Jacksonville | Florida | 32256 | United States |
| International Eye Associates, PA | Ormond Beach | Florida | 32174 | United States |
| Bascom Palmer Eye Institute at Palm Beach Gardens | Palm Beach | Florida | 33418 | United States |
| International Research Center | Tampa | Florida | 33603 | United States |
| Eye Consultants of Atlanta | Atlanta | Georgia | 30339 | United States |
| Midwest Cornea Associates, LLC | Carmel | Indiana | 46290 | United States |
| Price Vision Group | Indianapolis | Indiana | 46260 | United States |
| Cincinnati Eye Institute | Edgewood | Kentucky | 41017 | United States |
| Kentucky Eye Institute | Lexington | Kentucky | 40517 | United States |
| Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| Tufts Medical Center | Boston | Massachusetts | 02111 | United States |
| Cornea and Contact Lens Institute of Minnesota | Edina | Minnesota | 55436 | United States |
| Minnesota Eye Consultants | Minnetonka | Minnesota | 55305 | United States |
| Tauber Eye Center | Kansas City | Missouri | 64111 | United States |
| Ophthalmology Associates | St Louis | Missouri | 63131 | United States |
| Metropolitan Eye Research and Surgery Institute | Palisades Park | New Jersey | 07650 | United States |
| SightMD | Babylon | New York | 11702 | United States |
| Cornea Consultants of Albany | Slingerlands | New York | 12159 | United States |
| Duke Eye Center | Durham | North Carolina | 27710 | United States |
| Eye Consultants of North Dakota | Fargo | North Dakota | 58104 | United States |
| The Ohio State University, Department of Ophthalmology and Visual Sciences | Columbus | Ohio | 43212 | United States |
| Devers Eye Institute | Portland | Oregon | 97210 | United States |
| Hazleton Eye Specialists | Hazle Township | Pennsylvania | 18202 | United States |
| Scheie Eye Institute | Philadelphia | Pennsylvania | 19104 | United States |
| UPMC Eye Center | Pittsburgh | Pennsylvania | 15213 | United States |
| Carolina Cataract & Laser Center | Ladson | South Carolina | 29456 | United States |
| Vance Thompson Vision | Sioux Falls | South Dakota | 57108 | United States |
| BLINK Research Center, LLC | Memphis | Tennessee | 38119 | United States |
| University of Texas at Austin; The Mitchel and Shannon Wong Eye Institute | Austin | Texas | 78712 | United States |
| Stuart A. Terry, MD PA | San Antonio | Texas | 78212 | United States |
| Virginia Eye Consultants | Norfolk | Virginia | 23502 | United States |
| Dr. Gregory Moloney | Vancouver | British Columbia | V5Z 1K1 | Canada |
| Precision Cornea Centre | Ottawa | Ontario | K1G 5L1 | Canada |
| Kensington Eye Institute | Toronto | Ontario | M5T 3A9 | Canada |
One drop matching vehicle four times daily for 8 weeks in the study eye Vehicle Control: Matching vehicle control without the drug substance |
| Received CSB-001 in Uncontrolled Investigational Treatment Arm |
|
| COMPLETED |
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| NOT COMPLETED |
|
Intent-to-Treat defined as all subjects who were randomized and had post-baseline efficacy data.
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| ID | Title | Description |
|---|---|---|
| BG000 | CSB-001 Investigational Treatment Arm | One drop CSB-001 four times daily for 8 weeks in the study eye CSB-001 Ophthalmic Solution 0.1%: CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor) |
| BG001 | Vehicle Control Arm | One drop matching vehicle four times daily for 8 weeks in the study eye Vehicle Control: Matching vehicle control without the drug substance |
| BG002 | 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 |
| |||||||||||||||
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Current Neurotrophic Keratitis (NK) Stage | Stage 2 is defined as moderate NK showing a persistent or recurrent epithelial defect. Stage 3 is defined as severe NK showing corneal ulceration involving stromal issues that can lead to severe, possibly irreversible vision loss. | Count of Participants | Participants |
| |||||||||||||||
| Time since Current NK stage Diagnosis | Median | Full Range | months |
|
| 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 | Efficacy as Assessed by Complete Corneal Healing | Percentage of subjects achieving complete corneal healing in study eye as assessed by the Central Reading Center (CRC). Complete corneal healing was defined as absence of corneal staining in the area of the defect/ulcer (i.e., 0 mm lesion) at Week 8 and the absence of corneal staining in the area of the defect/ulcer (i.e., 0 mm lesion) at Week 10. | The full analysis set (FAS) includes all subjects who were randomized and confirmed by the CRC with stage 2 or 3 neurotrophic keratitis at the screening visit. | Posted | Count of Participants | Participants | Week 8 through Week 10 |
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| |||||||||||||||||||||||||||||
| Primary | Safety as Assessed by Adverse Event Reporting | Number of participants with ocular and systemic adverse events | The safety analysis set (SAS) is defined as all subjects who were randomized. | Posted | Count of Participants | Participants | Screening (Day 0) through Week 10 |
|
|
Controlled Treatment Phase: 10 weeks and Uncontrolled Treatment Phase: 10 weeks for a total of 20 weeks
Treatment-emergent AEs (TEAEs) were those that were experienced on or after the day of first dose in the study.
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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 | CSB-001 Investigational Treatment Arm | One drop CSB-001 four times daily for 8 weeks in the study eye CSB-001 Ophthalmic Solution 0.1%: CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor) | 2 | 69 | 8 | 69 | 18 | 69 |
| EG001 | Vehicle Control Arm | One drop matching vehicle four times daily for 8 weeks in the study eye Vehicle Control: Matching vehicle control without the drug substance | 1 | 62 | 7 | 62 | 35 | 62 |
| EG002 | CSB-001 Uncontrolled Investigational Treatment Arm | One drop CSB-001 four times daily for 8 weeks in the study eye CSB-001 Ophthalmic Solution 0.1%: CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor) | 0 | 25 | 1 | 25 | 5 | 25 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neurotrophic keratopathy | Eye disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Retinal detachment | Eye disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (24.0) | Systematic Assessment |
| |
| Osteomyelitis | Infections and infestations | MedDRA (24.0) | Systematic Assessment |
| |
| Pneumonia pseudomonal | Infections and infestations | MedDRA (24.0) | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA (24.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (24.0) | Systematic Assessment |
| |
| Ankle fracture | Injury, poisoning and procedural complications | MedDRA (24.0) | Systematic Assessment |
| |
| Skin laceration | Injury, poisoning and procedural complications | MedDRA (24.0) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (24.0) | Systematic Assessment |
| |
| Malignant neoplasm progression | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (24.0) | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Anemia | Blood and lymphatic system disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Hyponatremia | Metabolism and nutrition disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Low hemoglobin | Investigations | MedDRA (24.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Neurotrophic keratopathy | Eye disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Eye irritation | Eye disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Ocular hyperaemia | Eye disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Visual acuity reduced | Eye disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Conjunctival hyperaemia | Eye disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Corneal oedema | Eye disorders | MedDRA (24.0) | Systematic Assessment |
| |
| Instillation site pain | General disorders | MedDRA (24.0) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (24.0) | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA (24.0) | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Tomi Luan, OD, PhD | Claris Biotherapeutics, Inc. | 619-504-0309 | tomi.luan@clarisbio.com |
| Aug 12, 2025 |
| Prot_000.pdf |
| Prot | Yes | No | No | Study Protocol: CSB-C20-003 Protocol v5 21July2023 Part 2 | Jul 21, 2023 | Sep 17, 2025 | Prot_003.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jun 10, 2024 | Aug 4, 2025 | SAP_002.pdf |
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| ≥ 60 |
|
| Male |
|
| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Stage 3 |
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|