Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: CSB-001 Ophthalmic Solution 0.1%.
- Who it may be relevant to
- Registry conditions: Limbal Stem Cell Deficiency. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
An Open-Label Study Evaluating the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency
Overview
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).
Interventions
- Biological CSB-001 Ophthalmic Solution 0.1%
CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)
Primary outcome measures
- 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]
Secondary outcome measures (2)
- Efficacy as Assessed by Change in Stage of LSCD [Time frame: Day 0 through Week 144]
- Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity [Time frame: Day 0 through Week 144]
Eligibility 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 6 centers
- Loma Linda University Eye Institute — Loma Linda
- Midwest Cornea Associates, LLC — Carmel
- Francis Price Jr, MD — Indianapolis
- Minnesota Eye Consultants — Minnetonka
- Legacy Devers Eye Institute — Portland
- Stuart A. Terry, MD PA — San Antonio
Identifiers
NCT: NCT06452316 · CSB-C23-001