CSB-001 Eye Drops for Neurotrophic Keratitis

No longer recruiting at 48 trial locations

Overseen By Clinical Operations Representative

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Claris Biotherapeutics, Inc.

Must not be taking: Oxervate, Topical treatments

Disqualifiers: Active ocular infection, Herpetic keratitis, others

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests eye drops called CSB-001 to determine their effectiveness in healing neurotrophic keratitis, a condition where the cornea lacks sufficient nerve signals, causing sores or ulcers. Participants will be divided into two groups: one will use the CSB-001 drops, and the other will use placebo drops without active medicine. The trial seeks individuals with stage 2 or 3 neurotrophic keratitis who haven't improved with standard treatments like artificial tears or special contact lenses. Participants will apply the drops four times daily for eight weeks and visit the clinic regularly to monitor progress. The study aims to discover a new method to aid those whose eyes aren't healing with current treatments. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants a chance to be among the first to receive this new treatment.

Do I need to stop my current medications for the trial?

The trial does not specify if you need to stop all current medications, but you can continue oral antibiotics if you're already taking them. You may also use preservative-free antibiotic eye drops and certain glaucoma eye drops during the study.

Is there any evidence suggesting that CSB-001 Eye Drops are likely to be safe for humans?

Research shows that CSB-001, an eye drop treatment for neurotrophic keratitis, is currently being tested for safety and efficacy. Although limited safety information exists from past trials, the treatment remains in the early testing stages. Researchers closely monitor it to ensure safety for participants.

In similar studies, treatments like CSB-001 have generally been well-tolerated, with few serious side effects. However, since CSB-001 is still under investigation, not all side effects may be known. Testing in humans indicates that earlier research suggested its potential promise. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for neurotrophic keratitis?

Unlike the standard of care for neurotrophic keratitis, which often involves lubricating eye drops, antibiotics, or surgery, CSB-001 eye drops use a novel ophthalmic solution that targets nerve healing directly. Researchers are particularly excited because CSB-001 has the potential to repair corneal nerves, which is a new approach compared to current treatments that mainly focus on symptom management. This direct action on nerve regeneration could lead to more effective and quicker healing, offering hope for patients who haven't found relief with existing therapies.

What evidence suggests that CSB-001 Eye Drops could be an effective treatment for neurotrophic keratitis?

Research shows that CSB-001 eye drops, one of the treatments in this trial, might help treat neurotrophic keratitis, a condition where the cornea gets damaged due to nerve issues. This treatment uses a special growth factor to repair and regrow corneal nerves. Studies on similar treatments, such as those using nerve growth factors, have shown they can heal the cornea by addressing the underlying cause of the condition. Early results indicate that patients using these growth factors experienced significant improvements in healing. CSB-001 aims to offer similar benefits with a specific formula designed to enhance corneal repair. Participants in this trial may receive CSB-001 or a vehicle control as part of the study. ^{1 3 4 5 6}

Are You a Good Fit for This Trial?

This trial is for people with moderate to severe neurotrophic keratitis, which affects the cornea of the eye. Participants must have shown no improvement despite standard treatments and be willing to follow study procedures. They can't join if they've used Oxervate recently, have other eye diseases needing treatment during the study (except controlled glaucoma), or any active infections in their eyes.

Inclusion Criteria

I have stage 2 or 3 neurotrophic keratitis.

Pinhole distance visual acuity score ≤ 75 ETDRS letters measured with a LogMAR chart (≥ 0.2 LogMAR, ≤ 20/32 Snellen or worse Snellen or ≤ 0.625 decimal fraction) in the study eye

Subjects must have the ability and willingness to comply with study procedures

Exclusion Criteria

I have an eye condition other than glaucoma that needs treatment.

I used Oxervate for my eye condition within the last 2 months.

I do not have any active eye infections or inflammation unrelated to NK.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

Controlled Treatment

Participants receive randomized treatment or vehicle control four times daily for 8 weeks

8 weeks

Weekly visits from Day 0 to Week 8, and at Week 10

Open-label Uncontrolled Treatment

Participants in the vehicle arm who are not healed may opt into an open-label treatment phase

Not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CSB-001 Ophthalmic Solution 0.1%
• Vehicle Control

Trial Overview

The trial tests CSB-001 Ophthalmic Solution against a placebo control. Participants are randomly chosen to receive either the test medication or placebo, applying it four times daily for 8 weeks with weekly clinic visits. Those on placebo without healing may later try the actual drug in an open-label phase.

How Is the Trial Designed?

Group I: CSB-001 Investigational Treatment Arm Experimental Treatment 1 Intervention

One drop CSB-001 four times daily for 8 weeks in the study eye

Group II: Vehicle Control Arm Placebo Group 1 Intervention

One drop matching vehicle four times daily for 8 weeks in the study eye

Who Is Running the Clinical Trial?

Claris Biotherapeutics, Inc.

Lead Sponsor

Trials

3

Recruited

220+

Published Research Related to This Trial

In a study involving Sprague-Dawley rats, intravitreal injection of hepatocyte growth factor (HGF) significantly protected against degeneration of photoreceptors and retinal pigment epithelium (RPE) after sodium iodate treatment, as evidenced by improved scotopic b-wave thresholds and amplitudes.

HGF-treated eyes showed better preservation of outer retinal structure and larger azide response amplitudes compared to untreated eyes, suggesting its potential as a therapeutic agent for retinal degeneration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Protective effect of hepatocyte growth factor against degeneration of the retinal pigment epithelium and photoreceptor in sodium iodate-injected rats. Ohtaka, K., Machida, S., Ohzeki, T., et al.[2013]

In a rat model of retinal ischemia-reperfusion injury, hepatocyte growth factor (HGF) was found to be upregulated, indicating its potential role in the injury response.

Administering recombinant human HGF intravitreally significantly improved retinal function and reduced cell death, demonstrating its neuroprotective effects against retinal damage.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Expression and neuroprotective effect of hepatocyte growth factor in retinal ischemia-reperfusion injury. Shibuki, H., Katai, N., Kuroiwa, S., et al.[2009]

Hepatocyte growth factor (HGF) levels are significantly elevated in the vitreous fluid of patients with proliferative diabetic retinopathy (PDR) compared to nondiabetic control patients, indicating a potential role in the disease's progression.

Both HGF and vascular endothelial growth factor (VEGF) concentrations are higher in patients with active PDR than in those with quiescent PDR, suggesting that HGF may contribute to neovascularization associated with the active stage of the disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hepatocyte growth factor in vitreous fluid of patients with proliferative diabetic retinopathy and other retinal disorders. Katsura, Y., Okano, T., Noritake, M., et al.[2019]

Patients with retinitis pigmentosa (RP) have significantly higher levels of hepatocyte growth factor (HGF) in their aqueous humor compared to control subjects, with concentrations of 958.75 pg/mL versus 403.52 pg/mL, respectively.

This elevated HGF level in RP patients suggests a potential role for HGF as a neuroprotective treatment, highlighting its importance in future therapeutic strategies for managing RP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aqueous humor levels of hepatocyte growth factor in retinitis pigmentosa. Salom, D., Diaz-Llopis, M., Quijada, A., et al.[2010]

In a study involving 24 New Zealand rabbits undergoing Epi-LASIK, higher levels of hepatocyte growth factor (HGF) in tears were associated with increased corneal haze, indicating a potential biomarker for post-surgical healing.

Rabbits with a deeper ablation of 150 µm exhibited more significant corneal haze and elevated HGF levels compared to those with a 100 µm ablation, suggesting that the depth of corneal ablation influences both healing response and haze formation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Association between hepatocyte growth factor in tears and corneal haze in rabbits early after epipolis laser in situ keratomileusis]. Chen, J., Han, SN., Zou, XL., et al.[2020]

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04909450

Study to Evaluate the Safety and Efficacy of CSB-001 ...

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 ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC9106815/

Topical Recombinant Nerve Growth Factor for the Treatment ...

Recombinant nerve growth factor (rhNGF, Cenegermin) targets the underlying pathogenesis of NK by regenerating corneal nerves and healing the corneal epithelium.

3.

centerwatch.com

centerwatch.com/clinical-trials/listings/NCT04909450/study-to-evaluate-the-safety-and-efficacy-of-csb-001-ophthalmic-solution-01-in-neurotrophic-keratitis-subjects

Study to Evaluate the Safety and Efficacy of CSB-001 ...

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 ...

4.

withpower.com

withpower.com/trial/phase-2-keratitis-7-2021-e6afc

CSB-001 Eye Drops for Neurotrophic Keratitis

This trial tests CSB-001, a new treatment for people with moderate to severe neurotrophic keratitis. The treatment aims to help heal the damaged surface of ...

5.

journals.lww.com

journals.lww.com/ijo/fulltext/2025/04000/neurotrophic_keratopathy__update_in_diagnosis_and.4.aspx

Neurotrophic keratopathy: Update in diagnosis and ...

CSB-001 is a 0.1% ophthalmic solution of human recombinant 5-amino acid deleted hepatocyte growth factor undergoing Phase II clinical trials for the ...

6.

trialfinder.panfoundation.org

trialfinder.panfoundation.org/en-US/trial/listing/499096

Study to Evaluate the Safety and Efficacy of CSB-001 ...

This study will enroll subjects with recently formed corneal scars that resulted from a corneal insult presenting and diagnosed within the ...