What is the mechanism of action of this treatment?

The most common treatments for Limbal Stem Cell Deficiency (LSCD) include limbal stem cell transplantation and the use of bioengineered corneal epithelial cells. These treatments work by replenishing the depleted or dysfunctional limbal stem cells, which are crucial for maintaining and regenerating the corneal epithelium. For instance, cLSC (Corneal Limbal Stem Cells) therapy involves transplanting cultivated limbal stem cells onto the cornea, promoting the regeneration of a healthy corneal surface. This is vital for LSCD patients as it restores the corneal barrier, improves vision, and prevents further damage or scarring, thereby significantly enhancing their quality of life.

What side effects are associated with this type of intervention?

The most common treatments for Limbal Stem Cell Deficiency, particularly those involving the transplantation of corneal limbal stem cells (cLSC), generally show a favorable safety profile. However, potential side effects can include mild to moderate ocular surface inflammation, infection, and graft rejection. In some cases, patients may experience discomfort, redness, or transient visual disturbances. Long-term safety data are still being gathered, but initial studies suggest that serious adverse events are rare. It is crucial for patients to have regular follow-ups with their ophthalmologist to monitor for any complications.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved treatments for Limbal Stem Cell Deficiency (LSCD) that directly compare to the cLSC (Corneal Limbal Stem Cells) trial. The cLSC trial is investigating the feasibility, efficacy, and safety of transplanting cultivated limbal stem cells onto the cornea to regenerate the ocular surface. While there are treatments under investigation, such as stem cell therapies and regenerative approaches, none have yet received FDA approval specifically for LSCD.

What other types of research exist?

Promising novel alternatives to cLSC for Limbal Stem Cell Deficiency patients include: 1) Autologous serum eye drops, which have shown beneficial effects in ocular surface diseases. 2) Scleral contact lenses, which provide therapeutic benefits for ocular surface disease by protecting the cornea and improving vision. 3) Stem cell therapies, including the use of mesenchymal stem cells (MSCs) and induced pluripotent stem cells (iPSCs), which have shown potential in promoting corneal healing and regeneration. These alternatives are in various stages of research and clinical trials, offering hope for effective treatment options in 2024.

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Stem Cell Therapy

Stem Cell Transplant for Limbal Stem Cell Deficiency

Phase 1

Recruiting

Led By Sophie Deng, MD, PhD

Research Sponsored by University of California, Los Angeles

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Absence of lagophthalmos and eyelid abnormality

Documentation of a LSCD diagnosis and the central cornea is affected

Must not have

Exposure keratopathy or lagophthalmos of the study eye

Presence of ocular surface tumor

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 6 months and 12 months in both clsc and the control groups

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment using lab-grown stem cells to repair severe eye damage in patients who have had injuries or surgeries. The goal is to see if these stem cells can safely and effectively help the eye heal. Stem cell therapy has been increasingly used for treating various diseases, including eye conditions, with limbal epithelial stem transplantation being the only eye-related cell therapy currently in clinical practice.

Who is the study for?

This trial is for adults over 18 with Limbal Stem Cell Deficiency (LSCD) that hasn't improved after surgery in the last 6 months. Participants must have a vision of 20/200 or worse, diagnosed LSCD affecting the central cornea, no eyelid issues, and adequate eye moisture. Not eligible if pregnant, breastfeeding without birth control use during study, had chemical injury within last year, allergic to cLSC components, in another trial currently or have uncontrolled diabetes.

What is being tested?

The study tests cLSC's ability to be manufactured and transplanted onto patients' corneas successfully at surgery time. It aims to see if these cells can populate the ocular surface effectively without causing serious adverse events.

What are the potential side effects?

While specific side effects are not listed here as it's an early phase I trial focusing on feasibility and safety of cLSC transplantation for LSCD treatment; potential risks may include infection risk at surgical site or immune reaction.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My eyelids close completely without any abnormalities.

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My diagnosis is LSCD and it affects the center of my cornea.

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I do not have an active eye infection.

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My eye's forniceal depth is at least 5 mm.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My eye is affected by exposure or cannot fully close.

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I have a tumor on the surface of my eye.

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I have ongoing severe eye inflammation or eyelid gland issues.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 6 months and 12 months in both clsc and the control groups

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 6 months and 12 months in both clsc and the control groups

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 6 months and 12 months in both clsc and the control groups for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Feasibility of cLSC Transplant pertaining to Manufacturing meeting Release Criteria

Feasibility of cLSC Transplant pertaining to Manufacturing without Contamination

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

Secondary study objectives

Changes in the Area of Corneal Epithelial Defect

Changes in the Clinical Score

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Cultivated Limbal Stem-Cells (cLSC)Experimental Treatment1 Intervention

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

Group II: Scleral Contact Lens Device (SCL)Active Control1 Intervention

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

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Limbal Stem Cell Deficiency (LSCD) include limbal stem cell transplantation and the use of bioengineered corneal epithelial cells. These treatments work by replenishing the depleted or dysfunctional limbal stem cells, which are crucial for maintaining and regenerating the corneal epithelium. For instance, cLSC (Corneal Limbal Stem Cells) therapy involves transplanting cultivated limbal stem cells onto the cornea, promoting the regeneration of a healthy corneal surface. This is vital for LSCD patients as it restores the corneal barrier, improves vision, and prevents further damage or scarring, thereby significantly enhancing their quality of life.