Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency

NCT06452316 | Recruiting Phase 1 FDA Drug

• 1 other identifier: CSB-C23-001
• Study Type: interventional
• Target: 75
• Locations: 1 country
• Sites: 6

Brief 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 Holiday, where no test article is administered. Dosing Phase II is followed by an observational, noninterventional phase (Observation Phase).

Conditions

Limbal Stem Cell Deficiency

Outcome Measures

Primary Outcomes (3)

• Safety as Assessed by Adverse Event Reporting

  Incidence of ocular and systemic adverse events

  Day 0 through Week 144

• Safety as Assessed by Slit-lamp Biomicroscopy

  Change from baseline in slit-lamp parameters

  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

  Day 0 through Week 144

Secondary Outcomes (2)

• Efficacy as Assessed by Change in Stage of LSCD

  Day 0 through Week 144

• Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity

  Day 0 through Week 144

Study Arms (2)

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.

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.

Biological: CSB-001 Ophthalmic Solution 0.1%

Interventions

CSB-001 Ophthalmic Solution 0.1% BIOLOGICAL

CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)

CSB-001 QID; Control

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Claris Biotherapeutics, Inc.: lead

Study Sites (6)

Loma Linda University Eye Institute

Loma Linda, California, 92354, United States

RECRUITING

Midwest Cornea Associates, LLC

Carmel, Indiana, 46290, United States

RECRUITING

Francis Price Jr, MD

Indianapolis, Indiana, 46260, United States

RECRUITING

Minnesota Eye Consultants

Minnetonka, Minnesota, 55305, United States

RECRUITING

Legacy Devers Eye Institute

Portland, Oregon, 97210, United States

RECRUITING

Stuart A. Terry, MD PA

San Antonio, Texas, 78212, United States

RECRUITING

MeSH Terms

Conditions

Limbal Stem Cell Deficiency

Condition Hierarchy (Ancestors)

Corneal Diseases; Eye Diseases

Central Study Contacts

Central Operations Representative

CONTACT

919-354-1299; clinicaltrials@clarisbio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 5, 2024
• First Posted: June 11, 2024
• Study Start: May 15, 2024
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)