Actively Recruiting
An Open-Label Study Evaluating the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
Led by Claris Biotherapeutics, Inc. · Updated on 2026-04-30
75
Participants Needed
6
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of CSB-001 Ophthalmic Solution 0.1% in people with limbal stem cell deficiency (LSCD), a condition affecting the cornea. This open-label Phase 1 study involves subjects who have a qualifying diagnosis of LSCD confirmed by specific eye examinations. The main goal is to assess safety through adverse event reporting and eye exams, and to evaluate any changes in the severity of LSCD and vision over time. The study treatment is CSB-001, a human recombinant growth factor given as eye drops. Participants will receive one drop in one or both eyes four times daily during two separate 8-week dosing phases. These dosing phases are separated by a 31- to 40-day break with no treatment, called the dosing holiday. After these dosing periods, participants enter an observational non-interventional phase lasting up to Week 48. They may also choose to continue in an extended observation phase for an additional 36 months, up to Week 144. Participants will be monitored throughout the study using slit-lamp biomicroscopy, visual acuity tests, and assessments of LSCD severity. Safety is tracked from the start through Week 144. The study includes detailed ophthalmic exams, adverse event reporting, and visual tests to understand the effects of CSB-001. Total participation may last up to three years if participants join the extension phase.
CONDITIONS
Brief Title
Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Have an area of the central 5 mm zone of cornea affected with at least one clinical sign observed by slit-lamp examination using white light and/or cobalt blue light, fluorescein, and a Wratten filter
- Have at least one clinical sign observed with anterior segment optical coherence tomography (AS-OCT)
- If using Acthar at screening, must have stable dosing for about 8 weeks and plan to keep the same dose and regimen
- Have demonstrated little to no improvement in LSCD severity if dosing with Acthar
- Sponsor's written confirmation of qualifying LSCD diagnosis
- Inflammation related to LSCD or other non-infectious inflammation that does not interfere with participation
You will not qualify if you...
- Any active ocular infection (bacterial, viral, fungal, or protozoan) in either eye
- Ocular surgery within 30 days before Day 0 with incomplete or unstable healing
- Planned ocular surgery on or before Week 20 visit
- Any ocular or systemic condition that might affect study treatment efficacy or result interpretation, as judged by the investigator
- Other inclusion/exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Two 8-week dosing periods with a 30 to 40 day break between, or 20 weeks observation
Participants receive CSB-001 ophthalmic solution 0.1% as one drop four times daily during two 8-week dosing periods separated by a dosing holiday, or are observed without intervention during this time depending on the group assignment.
Regular visits during dosing and observation periods as per study schedule
Duration - Up to 36 months after initial 48 weeks
Participants are observed without intervention following the treatment period to monitor safety and efficacy up to Week 48, with an optional extended observation phase up to 36 months.
Periodic visits during observation extension phase depending on participation
Trial Site Locations
Total: 6 locations
1
Loma Linda University Eye Institute
Loma Linda, California, United States, 92354
Actively Recruiting
2
Midwest Cornea Associates, LLC
Carmel, Indiana, United States, 46290
Actively Recruiting
3
Francis Price Jr, MD
Indianapolis, Indiana, United States, 46260
Actively Recruiting
4
Minnesota Eye Consultants
Minnetonka, Minnesota, United States, 55305
Actively Recruiting
5
Legacy Devers Eye Institute
Portland, Oregon, United States, 97210
Actively Recruiting
6
Stuart A. Terry, MD PA
San Antonio, Texas, United States, 78212
Actively Recruiting
Research Team
C
Central Operations Representative
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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