Skip to main content
OpenTrials
Recruiting

NCT Number: NCT06452316

Study to Evaluate 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).

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Limbal Stem Cell Deficiency Corneal Diseases Eye Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Loma Linda University Eye Institute, Loma Linda, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

CSB-001 Ophthalmic Solution 0.1%

Biological

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

Primary outcomes

  1. Safety as Assessed by Adverse Event Reporting

    Time frame: Day 0 through Week 144

    Incidence of ocular and systemic adverse events

  2. Safety as Assessed by Slit-lamp Biomicroscopy

    Time frame: Day 0 through Week 144

    Change from baseline in slit-lamp parameters

  3. 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

Secondary outcomes

  1. Efficacy as Assessed by Change in Stage of LSCD

    Time frame: Day 0 through Week 144

    Change from baseline in LSCD signs based on imaging (white light, blue light with fluorescein and Wratten filter, and AS-OCT)

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

    Time frame: Day 0 through Week 144

    Visual acuity measured using the EDTRS visual acuity chart

Study contacts

Contact information is provided by the study sponsor or research team.

Central Operations Representative

CONTACT

[email protected]

919-354-1299

Sponsors and collaborators

Lead sponsor

Claris Biotherapeutics, Inc.

Industry

Registry information

Official study title

An Open-Label Study Evaluating the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Jun 11, 2024
Registry last updated
Apr 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.