Evaluating a new eye treatment for 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

Phase 1 Interventional Claris Biotherapeutics, Inc. · NCT06452316

This study is testing a new eye drop treatment for people with Limbal Stem Cell Deficiency to see if it is safe and helps improve their condition.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment75 (estimated)
Ages18 Years and up
SexAll
SponsorClaris Biotherapeutics, Inc. Industry-sponsored
Locations6 sites (Loma Linda, California and 5 other locations)
Trial IDNCT06452316 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety and efficacy of CSB-001, an ophthalmic solution, in patients suffering from Limbal Stem Cell Deficiency (LSCD). Participants will receive the investigational drug in one or both eyes over two dosing phases, separated by a period without treatment. The study includes an observational phase following the dosing phases to monitor outcomes. The trial is designed to gather data on the drug's effectiveness and safety profile in this specific patient population.

Who should consider this trial

Good fit: Ideal candidates are individuals diagnosed with Limbal Stem Cell Deficiency who exhibit specific clinical signs and have not shown improvement with current treatments.

Not a fit: Patients with active ocular infections or those who have had recent ocular surgery may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve vision and quality of life for patients with Limbal Stem Cell Deficiency.

How similar studies have performed: While there have been studies on similar treatments for LSCD, the specific approach of using CSB-001 is novel and has not been extensively tested.

Eligibility criteria

Show full inclusion / exclusion criteria
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.

Where this trial is running

Loma Linda, California and 5 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Limbal Stem Cell Deficiency
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.