Stem cell treatment for eye surface damage

Safety and Feasibility of Cultivated Autologous Limbal Stem Cells (cLSC) for Limbal Stem Cell Deficiency

This study is testing if a new stem cell treatment can help people with severe eye surface damage feel better and improve their vision.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: University of California, Los Angeles (Academic / other)
• Locations: 1 site (Los Angeles, California)
• Trial ID: NCT03957954 on ClinicalTrials.gov

What this trial studies

This phase I study aims to evaluate the safety and effectiveness of cultured limbal stem cells (cLSC) for patients with severe Limbal Stem Cell Deficiency (LSCD) in one eye. The trial will involve 20 patients, with the first 5 receiving cLSC to assess feasibility and safety. The remaining 15 patients will be randomly assigned to receive either cLSC or a scleral lens treatment as a control. The study will monitor the ability of cLSC to populate the ocular surface without serious adverse events.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with severe to total LSCD in one eye that has not responded to previous surgical treatments.

Not a fit: Patients with active infections in either eye or those with conditions that compromise their safety may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could restore vision and improve quality of life for patients suffering from severe eye surface damage.

How similar studies have performed: Other studies have shown promise in using stem cell therapies for ocular surface diseases, indicating potential for success in this approach.

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.

Where this trial is running

Los Angeles, California

• University of California — Los Angeles, California, United States (Recruiting)

Study contacts

• Principal investigator: Sophie Deng, MD, PhD — Stein Eye Institute UCLA
• Study coordinator: Clemence Bonnet, MD, PhD

Conditions Limbal Stem-cell Deficiency