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Stem Cell Therapy

CSB-001 Ophthalmic Solution for Limbal Stem Cell Deficiency

Phase 1
Recruiting
Research Sponsored by Claris Biotherapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
If dosing with Acthar at time of Screening Visit, must have a history of stable dosing for approximately 8 weeks, plan to maintain the same Acthar treatment dose and regimen, and have demonstrated little to no improvement in severity of LSCD
Have an area of the central 5 mm zone of cornea affected, presence of at least one clinical sign observed by slit-lamp examination with white light and/or cobalt blue light, fluorescein, and a Wratten filter, and at least one clinical sign observed with AS-OCT
Must not have
Any active ocular infection (bacterial, viral, fungal, or protozoan) in either eye
Planned ocular surgery on or before Week 20 visit
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 0 through week 48
Awards & highlights
No Placebo-Only Group

Summary

This trial will include participants with a certain eye condition called limbal stem cell deficiency. All participants will be given an experimental drug called CSB-001 in one or both of their eyes. The study

Who is the study for?
This trial is for individuals with limbal stem cell deficiency (LSCD), a condition affecting the eyes. Participants will receive CSB-001 Ophthalmic Solution in one or both eyes if they qualify. Specific eligibility criteria are not provided, but typically include having LSCD without other major eye diseases and being able to follow the study protocol.
What is being tested?
The safety and effectiveness of CSB-001 Ophthalmic Solution at 0.1% concentration are being tested on people with LSCD. The study has two main dosing phases separated by a break, followed by an observation phase to monitor long-term effects.
What are the potential side effects?
Potential side effects are not listed, but ophthalmic solutions like CSB-001 may cause eye irritation, redness, discomfort, or blurred vision temporarily after application.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I've been on a stable Acthar dose for 8 weeks with little improvement in my condition.
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I have a diagnosed eye condition affecting the central part of my cornea.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I do not have any active eye infections.
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I am scheduled for eye surgery before or by my Week 20 visit.
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I had eye surgery less than 30 days ago and my healing is not complete.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 0 through week 48
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 0 through week 48 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Safety as Assessed by Adverse Event Reporting
Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity
Safety as Assessed by Slit-lamp Biomicroscopy
Secondary study objectives
Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity
Efficacy as Assessed by Change in Stage of LSCD

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: CSB-001 QIDExperimental Treatment1 Intervention
One drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s)
Group II: CSB-001 BIDExperimental Treatment1 Intervention
One drop CSB-001 twice daily for two 8-week dosing periods in the study eye(s)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
CSB-001 Ophthalmic Solution 0.1%
2021
Completed Phase 2
~140

Find a Location

Who is running the clinical trial?

Claris Biotherapeutics, Inc.Lead Sponsor
2 Previous Clinical Trials
151 Total Patients Enrolled
~20 spots leftby Aug 2025