Limbal Stem-cell Deficiency
Conditions
Brief summary
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).
Detailed description
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.
Interventions
BIOLOGICALcLSC
Transplantation of cLSC
DEVICEScleral contact lens (SCL)
Scleral contact lens device (SCL) will be used to stabilize and improve ocular surface.
Sponsors
University of California, Los Angeles
National Eye Institute (NEI)
California Institute for Regenerative Medicine (CIRM)
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Main inclusion Criteria: 1. Male or female ≥18 years of age. 2. Best corrected visual acuity in the affected eye of 20/160 or less. 3. Documentation of a LSCD diagnosis and the central cornea is affected. 4. Absence of lagophthalmos and eyelid abnormality 5. Adequate forniceal depth is ≥ 5 mm. 6. LSCD fails to resolve by surgical treatments of the ocular surface during the previous 6 months of screening visit. 7. If the etiology of LSCD is due to chemical injury, a minimal interval of 1 year since the initial chemical injury is required. 8. A Schirmer test result at 5 minute of ≥5 mm of wetting. 9. Absence of active infectious keratitis in either eye at the Enrollment Visit. 10. Have a life expectancy ≥ 2 years after enrollment. Main
Exclusion criteria
1. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study. 2. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data. 3. Exposure keratopathy or lagophthalmos of the study eye. 4. Persistent severe ocular surface inflammation and/or meibomian gland dysfunction 5. Chemical injury occurred less than 12 months ago. 6. Presence of ocular surface tumor. 7. Uncontrolled diabetes with last hemoglobin A1C (HgA1C) \>8.5. 8. Presence of known allergies to any of the cLSC components. 9. Current participation in another simultaneous medical investigation or trial. 10. Unable to be compliant with or complete the requirements of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation | 12 months for all participants | Safety assessed by incidence of Serious Adverse Events (SAEs) directly related to cLSC: infection and/or corneal perforation. |
| Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria | 12 months for all participants | Feasibility assessed by successful manufacturing of cLSC from biopsy that meets release criteria. |
| Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination | 12 months for all participants | Feasibility assessed by successful cLSC manufacturing without contamination. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in the Area of Corneal Epithelial Defect | At 6 months and 12 months in both cLSC and the control groups | Evaluate changes in the area of corneal epithelial defect (ED) or lack of ED. |
| Changes in the Clinical Score | At 6 months and 12 months in both cLSC and the control groups | Evaluate changes in the clinical score determined by slit lamp examination using fluorescein staining and confocal imaging. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORSophie Deng, MD, PhD
Stein Eye Institute UCLA
Outcome results
None listed