Recruiting
Phase 1

CSB-001

Sponsor:

Claris Biotherapeutics, Inc.

Code:

NCT06452316

Conditions

Limbal Stem Cell Deficiency

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

CSB-001 Ophthalmic Solution 0.1%

Study Details

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

Study ID

NCT06452316

Start date

May 15, 2024

Status verified date

Aug, 2026

Completion date

Dec 31, 2027

Anticipated

Primary completion date

Dec 31, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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 Design

Enrollment

75 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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.

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.

Interventions

CSB-001 Ophthalmic Solution 0.1%

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

Primary outcome measure

  • Safety as Assessed by Adverse Event Reporting [ Time Frame: Day 0 through Week 144 ]
  • Safety as Assessed by Slit-lamp Biomicroscopy [ Time Frame: Day 0 through Week 144 ]
  • Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity [ Time Frame: Day 0 through Week 144 ]

Central Contacts and Locations

Central contacts

Central Operations Representative

949-354-1299clinicaltrials@clarisbio.com

Locations

Loma Linda University Eye Institute

Recruiting

Loma Linda, California, United States, 92354

Midwest Cornea Associates, LLC

Recruiting

Carmel, Indiana, United States, 46290

Francis Price Jr, MD

Recruiting

Indianapolis, Indiana, United States, 46260

Minnesota Eye Consultants

Recruiting

Minnetonka, Minnesota, United States, 55305

Legacy Devers Eye Institute

Recruiting

Portland, Oregon, United States, 97210

Stuart A. Terry, MD PA

Recruiting

San Antonio, Texas, United States, 78212

More Information

Sponsor

Claris Biotherapeutics, Inc.

Last update posted

Aug 13, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Claris Biotherapeutics, Inc. on 2026-08-13.