NCT03957954

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).

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
12mo left

Started Sep 2020

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Progress85%
Sep 2020Apr 2027

First Submitted

Initial submission to the registry

February 21, 2019

Completed
3 months until next milestone

First Posted

Study publicly available on registry

May 21, 2019

Completed
1.4 years until next milestone

Study Start

First participant enrolled

September 30, 2020

Completed
6.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 4, 2027

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 30, 2027

Last Updated

February 10, 2026

Status Verified

February 1, 2026

Enrollment Period

6.4 years

First QC Date

February 21, 2019

Last Update Submit

February 6, 2026

Conditions

Limbal Stem-cell Deficiency

Outcome Measures

Primary Outcomes (3)

  • Safety of cLSC Transplant pertaining to Infection and/or Corneal Perforation

    Safety assessed by incidence of Serious Adverse Events (SAEs) directly related to cLSC: infection and/or corneal perforation.

    12 months for all participants

  • Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria

    Feasibility assessed by successful manufacturing of cLSC from biopsy that meets release criteria.

    12 months for all participants

  • Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination

    Feasibility assessed by successful cLSC manufacturing without contamination.

    12 months for all participants

Secondary Outcomes (2)

  • Changes in the Area of Corneal Epithelial Defect

    At 6 months and 12 months in both cLSC and the control groups

  • Changes in the Clinical Score

    At 6 months and 12 months in both cLSC and the control groups

Study Arms (2)

Cultivated Limbal Stem-Cells (cLSC)

EXPERIMENTAL

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

Biological: cLSC

Scleral Contact Lens Device (SCL)

ACTIVE COMPARATOR

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

Device: Scleral contact lens (SCL)

Interventions

cLSCBIOLOGICAL

Transplantation of cLSC

Cultivated Limbal Stem-Cells (cLSC)
Scleral contact lens (SCL)DEVICE

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

Scleral Contact Lens Device (SCL)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male or female ≥18 years of age.
  • Best corrected visual acuity in the affected eye of 20/160 or less.
  • Documentation of a LSCD diagnosis and the central cornea is affected.
  • Absence of lagophthalmos and eyelid abnormality
  • Adequate forniceal depth is ≥ 5 mm.
  • LSCD fails to resolve by surgical treatments of the ocular surface during the previous 6 months of screening visit.
  • If the etiology of LSCD is due to chemical injury, a minimal interval of 1 year since the initial chemical injury is required.
  • A Schirmer test result at 5 minute of ≥5 mm of wetting.
  • Absence of active infectious keratitis in either eye at the Enrollment Visit.
  • Have a life expectancy ≥ 2 years after enrollment.

You may not qualify if:

  • Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study.
  • 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.
  • Exposure keratopathy or lagophthalmos of the study eye.
  • Persistent severe ocular surface inflammation and/or meibomian gland dysfunction
  • Chemical injury occurred less than 12 months ago.
  • Presence of ocular surface tumor.
  • Uncontrolled diabetes with last hemoglobin A1C (HgA1C) \>8.5.
  • Presence of known allergies to any of the cLSC components.
  • Current participation in another simultaneous medical investigation or trial.
  • Unable to be compliant with or complete the requirements of the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of California

Los Angeles, California, 90095, United States

Location

MeSH Terms

Conditions

Limbal Stem Cell Deficiency

Condition Hierarchy (Ancestors)

Corneal DiseasesEye Diseases

Study Officials

  • Sophie Deng, MD, PhD

    Stein Eye Institute UCLA

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

February 21, 2019

First Posted

May 21, 2019

Study Start

September 30, 2020

Primary Completion (Estimated)

February 4, 2027

Study Completion (Estimated)

April 30, 2027

Last Updated

February 10, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations

US(1)