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).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
65
CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)
Loma Linda University Eye Institute
Loma Linda, California, United States
RECRUITINGMidwest Cornea Associates, LLC
Carmel, Indiana, United States
RECRUITINGFrancis Price Jr, MD
Indianapolis, Indiana, United States
RECRUITINGMinnesota Eye Consultants
Minnetonka, Minnesota, United States
RECRUITINGLegacy Devers Eye Institute
Portland, Oregon, United States
RECRUITINGStuart A. Terry, MD PA
San Antonio, Texas, United States
RECRUITINGSafety as Assessed by Adverse Event Reporting
Incidence of ocular and systemic adverse events
Time frame: Day 0 through Week 96
Safety as Assessed by Slit-lamp Biomicroscopy
Change from baseline in slit-lamp parameters
Time frame: Day 0 through Week 96
Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity
Change from baseline using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart
Time frame: Day 0 through Week 96
Efficacy as Assessed by Change in Stage of LSCD
Change from baseline in LSCD signs based on imaging (white light, blue light with fluorescein and Wratten filter, and AS-OCT)
Time frame: Day 0 through Week 96
Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity
Visual acuity measured using the EDTRS visual acuity chart
Time frame: Day 0 through Week 96
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