Stem Cell Therapy for Limbal Stem Cell Deficiency

Phase 1

Recruiting

• Conditions: Limbal Stem-cell Deficiency
• Interventions: Biological: cLSC; Device: Scleral contact lens (SCL)

• Registration Number: NCT03957954
• Lead Sponsor: University of California, Los Angeles

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.

Study Timeline

• Study First Submitted: 2019-02-21
• Study First Submitted QC: 2019-05-17
• Study First Posted: 2019-05-21
• Study Start: 2020-09-30
• Status Verified: 2023-12
• Last Update Submitted: 2023-12-05
• Last Update Posted: 2023-12-06
• Primary Completion: 2024-04-30
• Study Completion: 2024-05-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cultivated Limbal Stem-Cells (cLSC)	cLSC	One dose of cultivated limbal stem-cells (cLSC), size between 7.6 to 15 mm in the average diameter.
Scleral Contact Lens Device (SCL)	Scleral contact lens (SCL)	Scleral contact lens device (SCL) will be fitted to stabilize and improve ocular surface.

Primary Outcome Measures

Name	Time	Method
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.
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 without Contamination	12 months for all participants	Feasibility assessed by successful cLSC manufacturing without contamination.

Secondary Outcome Measures

Name	Time	Method
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.
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.

Trial Locations

Locations (1)

University of California; 🇺🇸 Los Angeles, California, United States