Limbal Stem Cell Deficiency (LSCD) Treatment With Cultivated Stem Cell (CALEC) Graft (CALEC)

January 2, 2025 updated by: Ula Jurkunas, Massachusetts Eye and Ear Infirmary

Safety and Feasibility of Cultivated Autologous Limbal Epithelial Cell Transplantation in the Treatment of Limbal Stem Cell Deficiency (LSCD)

The main aim of the study is to determine the safety and feasibility of a cultivated autologous limbal epithelial cell (CALEC) transplantation in the treatment of limbal stem cell deficiency.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This is an open label, single center study to assess safety, feasibility, and efficacy of Cultivated Autologous Limbal Epithelial Cell (CALEC) grafts in 17 patients with unilateral limbal stem cell deficiency (LSCD).

Participants will have a corneal biopsy in their non-diseased eye, which will provide cells for the creation of the CALEC graft. The CALEC will be made at the Good Manufacturing Practice (GMP) Laboratory, Dana Farber Cancer Institute and transported to Mass. Eye and Ear Infirmary for application to the participant's diseased eye during their standard corneal reconstruction procedure. Subjects will be monitored up to month 18 post-transplant to assess for any delayed adverse events of the product (CALEC) or procedure as well as assessment of the durability of the transplant.

Study Type

Interventional

Enrollment (Actual)

23

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts Eye and Ear Infirmary

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 89 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male or female participants age 18 to <90 years old at time of enrollment
  • Ability of a subject or guardian/legal representative to provide written informed consent and to comply with study assessments for the full duration of the study.
  • Patients with unilateral limbal stem cell deficiency (LSCD) as determined by conjunctivalization of the cornea defined by fibrovascular pannus more than 2 mm from the limbus for greater than or equal to 6 clock hours.

  • Additional optional criteria:

    • Lack of limbal palisades of Vogt for greater than or equal to 9 clock hours
    • Goblet cell presence as defined by impression cytologic criteria

Exclusion Criteria:

  • Corneal or ocular surface infection within 30 days prior to study entry or CALEC transplantation
  • Ocular surface malignancy
  • Uncontrolled diabetes with most recent HgA1c greater than 8.5%
  • Renal Failure with eGFR below 60 mL/min per 1.73 m2
  • Aspartate aminotransferase and alanine aminotransferase levels greater than 3 times institutional upper limit of normal
  • Total bilirubin greater than 2 times institutional upper limit of normal (except patients with known Gilbert's syndrome)
  • Platelet levels less than 100,000 or greater than 450,000 per microliter
  • Hemoglobin levels of less than 11.0 g/dL in men or less than 10.0 g/dL in women
  • Prothrombin time greater than 16 seconds and activated partial thromboplastin time greater than 35 seconds in patients not taking warfarin and an international normalized ratio greater than 3 in patients taking warfarin
  • Inability to tolerate monitored anesthesia
  • HIV infection or AIDS
  • Active Hepatitis B or C
  • Pregnancy (positive test) or lactation
  • Participation in another simultaneous medical investigation or trial
  • Severe cicatricial eye disease
  • Severe dry eye disease as determined by Schirmer's test less than 1mm in at least one eye.
  • Any medical, psychiatric, debilitating disease/disorder or social condition that in the judgment of the investigator would interfere with or serve as a contraindication to adherence to the study protocol or ability to give informed consent.
  • Signs of current infection, including fever and current treatment with antibiotics.
  • History of allo-limbal transplantation
  • Presence of allergy to the CALEC graft or any of the chemical components within its formulation.

Exclusion Based on Donor Eye:

  • Conjunctivalization of the cornea defined by fibrovascular pannus more than 2 mm from the limbus for greater than or equal to 3 clock hours
  • Lack of limbal palisades of Vogt for greater than or equal to 3 clock hours
  • History of allo-limbal transplantation

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cultivated Autologous Limbal Epithelial Cell (CALEC) graft
Participants will have a corneal biopsy in their non-diseased eye, which will provide cells for the creation of the CALEC graft. The CALEC will be made at the Good Manufacturing Practice (GMP) Laboratory, Dana Farber Cancer Institute and transported to Mass. Eye and Ear Infirmary for application to the participant's diseased eye during their standard corneal reconstruction procedure.
Procedure: Biopsy to collect limbal epithelial stem cells that will be cultivated into a graft
Cultivated autologous limbal epithelial cell (CALEC) therapy utilizes a bio-engineered composite of ex vivo expanded autologous corneal epithelial cells and an FDA-approved amniotic membrane (AmnioGraft®, Bio-Tissue, Inc.) to reconstruct the ocular surface. A small biopsy (2-3 mm2) from the patient's contralateral eye serves as a source epithelial (stem) cells that are expanded on the amniotic membrane in culture and the resulting product is surgically transplanted onto the cornea after excision of the fibrovascular pannus.
Other Names:
  • Cultivated Autologous Limbal Epithelial Cell (CALEC)
Biological: Cultivation of Limbal epithelial cells into a graft
A graft is manufactured for transplant
Procedure: CALEC Transplant
Limbal epithelial cells are obtained from the healthy fellow eye and cultivated in a lab for later transplantation into the diseased eye.
Other Names:
  • Conjunctival Limbal Autograft

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Primary Safety Events of Interest
Time Frame: 18 Months

The occurrence of the following adverse events at any time during the 18 months of follow-up in the recipient eye will serve as the primary safety events of interest.

  1. Ocular Infection (defined as endophthalmitis or microbial keratitis [bacterial, fungal, parasitic]:
  2. Corneal Perforation
  3. Graft Detachment ≥50%
18 Months
Manufacturing Feasibility Measures
Time Frame: 18 Months
Each biopsy attempt will be classified as a "feasibility success" if it produced at least one construct that met all of the Quality Control (QC) release criteria. Manufacturing feasibility will be evaluate on a biopsy level (denominator is the total number of biopsies).The number and percentage of biopsy attempts resulting in a feasibility success will be calculated.
18 Months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Measure of Transplant Efficacy
Time Frame: 18 Months
The primary efficacy outcome will be a binary "Complete Success" of the graft defined as improvement in corneal surface integrity
18 Months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Massachusetts Eye and Ear Infirmary

Collaborators

Dana-Farber Cancer Institute

Investigators

  • Principal Investigator: Ula Jurkunas, MD, Massachusetts Eye and Ear Infirmary

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2016

Primary Completion (Actual)

March 31, 2023

Study Completion (Actual)

March 31, 2023

Study Registration Dates

First Submitted

February 9, 2015

First Submitted That Met QC Criteria

October 29, 2015

First Posted (Estimated)

October 30, 2015

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

January 2, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 14-124H

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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