Stem Cell Therapy for Limbal Stem Cell Deficiency

University of California, Los Angeles (UCLA)

Status and phase: Enrolling; Phase 1

Conditions: Limbal Stem-cell Deficiency

Treatments: Device: Scleral contact lens (SCL); Biological: cLSC

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT03957954

R01EY021797 (U.S. NIH Grant/Contract)

V001

Details and patient eligibility

About

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).

Full 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.

Enrollment: 20 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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20 participants in 2 patient groups

Cultivated Limbal Stem-Cells (cLSC)

Experimental group

Description:

One dose of cultivated limbal stem-cells (cLSC), size between 7.6 to 15 mm in the average diameter.

Treatment:

Biological: cLSC

Scleral Contact Lens Device (SCL)

Active Comparator group

Description:

Scleral contact lens device (SCL) will be fitted to stabilize and improve ocular surface.

Treatment:

Device: Scleral contact lens (SCL)