Stem Cell Therapy for Limbal Stem Cell Deficiency

NCT ID: NCT03957954

Last Updated: 2025-09-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-09-30

Study Completion Date

2027-04-30

Brief Summary

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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

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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.

Conditions

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Limbal Stem-cell Deficiency

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cultivated Limbal Stem-Cells (cLSC)

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

Group Type EXPERIMENTAL

cLSC

Intervention Type BIOLOGICAL

Transplantation of cLSC

Scleral Contact Lens Device (SCL)

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

Group Type ACTIVE_COMPARATOR

Scleral contact lens (SCL)

Intervention Type DEVICE

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

Interventions

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cLSC

Transplantation of cLSC

Intervention Type BIOLOGICAL

Scleral contact lens (SCL)

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

Intervention Type DEVICE

Eligibility Criteria

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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.

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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Eye Institute (NEI)

NIH

Sponsor Role collaborator

California Institute for Regenerative Medicine (CIRM)

OTHER

Sponsor Role collaborator

University of California, Los Angeles

OTHER

Sponsor Role lead

Responsible Party

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Sophie Deng, MD, PhD

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Sophie Deng, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Stein Eye Institute UCLA

Locations

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University of California

Los Angeles, California, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Clemence Bonnet, MD, PhD

Role: CONTACT

310-825-6232

Sheyla Gonzalez, PhD

Role: CONTACT

310-825-6232

Facility Contacts

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Clemence Bonnet, MD

Role: primary

310-825-6232

Other Identifiers

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R01EY021797

Identifier Type: NIH

Identifier Source: secondary_id

View Link

V001

Identifier Type: -

Identifier Source: org_study_id

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