Clinical trial NCT03957954: Stem Cell Therapy for Limbal Stem Cell Deficiency
Official title: Safety and Feasibility of Cultivated Autologous Limbal Stem Cells (cLSC) for Limbal Stem Cell Deficiency
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Limbal Stem-cell Deficiency
Lead sponsor: University of California, Los Angeles
Sponsor class: OTHER
Collaborators: name: National Eye Institute (NEI)
name: California Institute for Regenerative Medicine (CIRM)
First posted: 2019-05-21
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Main inclusion Criteria:

1. Male or female ≥18 years of age.
2. Best corrected visual acuity in the affected eye of 20/200 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.

Primary outcomes:
measure: Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation
timeFrame: 12 months for all participants
measure: Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria
timeFrame: 12 months for all participants
measure: Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination
timeFrame: 12 months for all participants
Secondary outcomes:
measure: Changes in the Area of Corneal Epithelial Defect
timeFrame: At 6 months and 12 months in both cLSC and the control groups
measure: Changes in the Clinical Score
timeFrame: At 6 months and 12 months in both cLSC and the control groups
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of California
state: California
Study officials:
affiliation: Stein Eye Institute UCLA
name: Sophie Deng, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: