Limbal Stem Cell DeficiencyPhase 1NCT06452316
Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
A Phase 1 study testing CSB-001 Ophthalmic Solution 0.1% in people with Limbal Stem Cell Deficiency, run by Claris Biotherapeutics, Inc. at 6 sites in United States.
Trial summary
This study will enroll subjects with qualifying limbal stem cell deficiency (LSCD). All subjects will receive CSB-001 investigational drug in either one or both study eyes. The study is comprised of two identical phases (Dosing Phase I and II) of test article dosing separated by a 31- to 40-day period, the Dosing… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Limbal Stem Cell Deficiency
- What is being tested?
- CSB-001 Ophthalmic Solution 0.1%
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 6 sitesUnited States
- Study dates
- 15 May 2024to 31 Dec 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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
- 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.
- 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.
- Sponsor written confirmation of qualifying LSCD diagnosis.
- Inflammation associated with LSCD or other non-infectious inflammation that will not interfere with subject's participation.
Who cannot join
- Any active ocular infection (bacterial, viral, fungal, or protozoan) in either eye.
- Ocular surgery within 30 days of Day 0 where investigator assesses that healing is not considered complete/stable.
- Planned ocular surgery on or before Week 20 visit.
- Presence or history of any ocular or systemic disorder, condition, or procedure that might hinder the efficacy of the study treatment or its evaluation, or could interfere with the interpretation of study results, in the opinion of the investigator.
- Note: Other inclusion/exclusion criteria apply.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
United States6 sites, 6 recruiting
- Recruiting
Midwest Cornea Associates, LLC
Carmel, Indiana, 46290
Study Coordinator
- Recruiting
Francis Price Jr, MD
Indianapolis, Indiana, 46260
Study Coordinator
- Recruiting
Loma Linda University Eye Institute
Loma Linda, California, 92354
Study Coordinator
- Recruiting
Minnesota Eye Consultants
Minnetonka, Minnesota, 55305
Study Coordinator
- Recruiting
Legacy Devers Eye Institute
Portland, Oregon, 97210
Study Coordinator
- Recruiting
Stuart A. Terry, MD PA
San Antonio, Texas, 78212
Study Coordinator
About this study
Official title: An Open-Label Study Evaluating the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
This study will enroll subjects with qualifying limbal stem cell deficiency (LSCD). All subjects will receive CSB-001 investigational drug in either one or both study eyes. The study is comprised of two identical phases (Dosing Phase I and II) of test article dosing separated by a 31- to 40-day period, the Dosing Holiday, where no test article is administered. Dosing Phase II is followed by an observational, noninterventional phase (Observation Phase).
- Conditions
- Limbal Stem Cell Deficiency
- Being tested
- CSB-001 Ophthalmic Solution 0.1%
- Medical subject headings
- Limbal Stem Cell Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CSB-001 Ophthalmic Solution 0.1%Biological
CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)
| Study group | Role | What they receive |
|---|---|---|
| Control | Experimental | Period I: Subjects will be observed (non-interventional) for 20 weeks. Period II: Subjects will be dosed one drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s) separated by >30 days and <40 days (dosing holiday) followed by observation (non-interventional) to Week 48. Period III: Subjects may elect to participate in the Observation Extension Phase (non-interventional) for an additional 36 months to Week 144. Receives: Biological: CSB-001 Ophthalmic Solution 0.1% |
| CSB-001 QID | Experimental | Period I: Subjects will be dosed one drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s) separated by >30 days and <40 days (dosing holiday) followed by observation (non-interventional) to Week 20. Period II: Subject will be observed (non-interventional) for 28 weeks to Week 48. Period III: Subjects may elect to participate in the Observation Extension Phase (non-interventional) for an additional 36 months to Week 144. Receives: Biological: CSB-001 Ophthalmic Solution 0.1% |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety as Assessed by Adverse Event Reporting
Incidence of ocular and systemic adverse events
Measured: Day 0 through Week 144
Safety as Assessed by Slit-lamp Biomicroscopy
Change from baseline in slit-lamp parameters
Measured: Day 0 through Week 144
Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity
Change from baseline using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart
Measured: Day 0 through Week 144
Secondary outcomes (2)
Efficacy as Assessed by Change in Stage of LSCD
Change from baseline in LSCD signs based on imaging (white light, blue light with fluorescein and Wratten filter, and AS-OCT)
Measured: Day 0 through Week 144
Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity
Visual acuity measured using the EDTRS visual acuity chart
Measured: Day 0 through Week 144
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2024
- Study start15 May 2024
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limbal Stem Cell DeficiencyStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Claris Biotherapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06452316
- Sponsor's protocol ID
- CSB-C23-001
- Status last verified
- Aug 2026
- First submitted
- 5 Jun 2024
- Last updated
- 13 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- 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 13 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study Evaluating the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
- ClinicalTrials.gov ID
- NCT06452316
- Acronym / sponsor ID
- CSB-C23-001
- Conditions
- Limbal Stem Cell Deficiency
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Claris Biotherapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 May 2024 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 11 Jun 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will enroll subjects with qualifying limbal stem cell deficiency (LSCD). All subjects will receive CSB-001 investigational drug in either one or both study eyes. The study is comprised of two identical phases (Dosing Phase I and II) of test article dosing separated by a 31- to 40-day period, the Dosing Holiday, where no test article is administered. Dosing Phase II is followed by an observational, noninterventional phase (Observation Phase).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control | Experimental | Biological: CSB-001 Ophthalmic Solution 0.1%Period I: Subjects will be observed (non-interventional) for 20 weeks. Period II: Subjects will be dosed one drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s) separated by >30 days and <40 days (dosing holiday) followed by observation (non-interventional) to Week 48. Period III: Subjects may elect to participate in the Observation Extension Phase (non-interventional) for an additional 36 months to Week 144. |
| CSB-001 QID | Experimental | Biological: CSB-001 Ophthalmic Solution 0.1%Period I: Subjects will be dosed one drop CSB-001 four times daily for two 8-week dosing periods in the study eye(s) separated by >30 days and <40 days (dosing holiday) followed by observation (non-interventional) to Week 20. Period II: Subject will be observed (non-interventional) for 28 weeks to Week 48. Period III: Subjects may elect to participate in the Observation Extension Phase (non-interventional) for an additional 36 months to Week 144. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
- 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.
- Sponsor written confirmation of qualifying LSCD diagnosis.
- Inflammation associated with LSCD or other non-infectious inflammation that will not interfere with subject's participation.
Key exclusion criteria (5 listed)
- Any active ocular infection (bacterial, viral, fungal, or protozoan) in either eye.
- Ocular surgery within 30 days of Day 0 where investigator assesses that healing is not considered complete/stable.
- Planned ocular surgery on or before Week 20 visit.
- Presence or history of any ocular or systemic disorder, condition, or procedure that might hinder the efficacy of the study treatment or its evaluation, or could interfere with the interpretation of study results, in the opinion of the investigator.
- Note: Other inclusion/exclusion criteria apply.
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 |
|---|---|---|
| CSB-001 Ophthalmic Solution 0.1%CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor) | Biological | CSB-001 QID, Control |
Primary outcome
Safety as Assessed by Adverse Event Reporting
Time frame: Day 0 through Week 144
Incidence of ocular and systemic adverse events
Safety as Assessed by Slit-lamp Biomicroscopy
Time frame: Day 0 through Week 144
Change from baseline in slit-lamp parameters
Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity
Time frame: Day 0 through Week 144
Change from baseline using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart
2 secondary outcomes
Efficacy as Assessed by Change in Stage of LSCD
Time frame: Day 0 through Week 144
Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity
Time frame: Day 0 through Week 144
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2026.
- Individual participant dataSharing planned: No
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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.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CSB-001 Ophthalmic Solution 0.1% in people with Limbal Stem Cell Deficiency, run by Claris Biotherapeutics, Inc. at 6 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 4 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 13 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Central Operations Representative (949-354-1299, clinicaltrials@clarisbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety as Assessed by Adverse Event Reporting (time frame: Day 0 through Week 144). The study lists 3 primary outcomes in all. 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 13 Aug 2026.