This phase I study will collect preliminary information on the activity and safety of cLSC. We will investigate the ability to manufacture and transplant cLSC onto the cornea successfully at the time of surgery (feasibility), and have cLSC begin to populate the ocular surface (efficacy) without serious adverse events (safety).
20 patients with severe to total Limbal Stem Cell Deficiency (LSCD) in one eye attributable to injury or ocular surgery. The first 5 eyes will receive the cLSCs to determine the feasibility and safety. Then the subsequent 15 eyes will be randomized into cLSC group and a scleral lens treatment (SCL) control group in a 2:1 ratio in blocks of 3 or 6 using a random number generator.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Transplantation of cLSC
Scleral contact lens device (SCL) will be used to stabilize and improve ocular surface.
University of California
Los Angeles, California, United States
Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation
Safety assessed by incidence of Serious Adverse Events (SAEs) directly related to cLSC: infection and/or corneal perforation.
Time frame: 12 months for all participants
Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria
Feasibility assessed by successful manufacturing of cLSC from biopsy that meets release criteria.
Time frame: 12 months for all participants
Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination
Feasibility assessed by successful cLSC manufacturing without contamination.
Time frame: 12 months for all participants
Changes in the Area of Corneal Epithelial Defect
Evaluate changes in the area of corneal epithelial defect (ED) or lack of ED.
Time frame: At 6 months and 12 months in both cLSC and the control groups
Changes in the Clinical Score
Evaluate changes in the clinical score determined by slit lamp examination using fluorescein staining and confocal imaging.
Time frame: At 6 months and 12 months in both cLSC and the control groups
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