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

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).

Study Overview

• Status: Recruiting
• Conditions: Limbal Stem Cell Deficiency
• Intervention / Treatment: Biological: CSB-001 Ophthalmic Solution 0.1%

• Study Type: Interventional
• Enrollment (Estimated): 75
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Central Operations Representative; Phone Number: 919-354-1299; Email: clinicaltrials@clarisbio.com

Study Locations

• United States
  • California
    • Loma Linda, California, United States, 92354
      • Recruiting
      • Loma Linda University Eye Institute
      • Contact: Study Coordinator
  • Indiana
    • Carmel, Indiana, United States, 46290
      • Recruiting
      • Midwest Cornea Associates, LLC
      • Contact: Study Coordinator
    • Indianapolis, Indiana, United States, 46260
      • Recruiting
      • Francis Price Jr, MD
      • Contact: Study Coordinator
  • Minnesota
    • Minnetonka, Minnesota, United States, 55305
      • Recruiting
      • Minnesota Eye Consultants
      • Contact: Study Coordinator
  • Oregon
    • Portland, Oregon, United States, 97210
      • Recruiting
      • Legacy Devers Eye Institute
      • Contact: Study Coordinator
  • Texas
    • San Antonio, Texas, United States, 78212
      • Recruiting
      • Stuart A. Terry, MD PA
      • Contact: Study Coordinator

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Control; 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: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)
Experimental: CSB-001 QID; 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%; CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	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	Day 0 through Week 144

Collaborators and Investigators

• Sponsor: Claris Biotherapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): May 15, 2024
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: June 5, 2024
• First Submitted That Met QC Criteria: June 5, 2024
• First Posted (Actual): June 11, 2024

Study Record Updates

• Last Update Posted (Actual): April 30, 2026
• Last Update Submitted That Met QC Criteria: April 27, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Corneal Diseases; Limbal Stem Cell Deficiency; Pharmaceutical Solutions; Ophthalmic Solutions
• Other Study ID Numbers: CSB-C23-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No