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Limbal Stem Cell DeficiencyPhase 1/2CALECNCT02592330

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

A Phase 1/2 study testing Biopsy to collect limbal epithelial stem cells that will be cultivated into a graft, Cultivation of Limbal epithelial cells into a graft and CALEC Transplant in people with Limbal Stem Cell Deficiency, run by Massachusetts Eye and Ear Infirmary at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 89 years
Conditions the study is for
Limbal Stem Cell Deficiency
What is being tested?
Biopsy to collect limbal epithelial stem cells that will be cultivated into a graft, Cultivation of Limbal epithelial cells into a graft and CALEC Transplant
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
23 participants
Where
1 siteUnited States
Study dates
1 Aug 2016to 31 Mar 2023
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 89 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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)
Show all 23Show fewer
  • 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.
  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114

Other trials in United States

About this study

Official title: 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.

Read the 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.

Being tested
Biopsy to collect limbal epithelial stem cells that will be cultivated into a graft, Cultivation of Limbal epithelial cells into a graft, CALEC Transplant
Keywords
LSCD treatment, Limbal Stem Cell Deficiency, CALEC, Cultivated Autologous Limbal Epithelial Cell Transplantation, Corneal epithelial stem cells, Corneal scarring, Corneal opacity, Chemical injury eye, Thermal injury eye, Autologous stem cell, Epithelial defect, Corneal cloudiness, Ocular burn, Ocular injury, Autograft, Conjunctival Limbal Autograft, LSCD research, Chronic contact lens wear, Chronic keratoconjunctivitis, Corneal conjunctivalization
Medical subject headings
Limbal Stem Cell Deficiency, Corneal Injuries, Corneal Opacity, Eye Injuries, Corneal Neovascularization

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biopsy to collect limbal epithelial stem cells that will be cultivated into a graftProcedure or surgery

    Also called Cultivated Autologous Limbal Epithelial Cell (CALEC)

    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.

  • Cultivation of Limbal epithelial cells into a graftBiological

    A graft is manufactured for transplant

  • CALEC TransplantProcedure or surgery

    Also called Conjunctival Limbal Autograft

    Limbal epithelial cells are obtained from the healthy fellow eye and cultivated in a lab for later transplantation into the diseased eye.

Study groups in NCT02592330
Study groupRoleWhat they receive
Cultivated Autologous Limbal Epithelial Cell (CALEC) graftExperimentalParticipants 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.
Receives: Procedure: Biopsy to collect limbal epithelial stem cells that will be cultivated into a graft; Biological: Cultivation of Limbal epithelial cells into a graft; Procedure: CALEC Transplant

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Primary Safety Events of Interest

    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%

    Measured: 18 Months

  2. Manufacturing Feasibility Measures

    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.

    Measured: 18 Months

Secondary outcomes (1)

  1. Measure of Transplant Efficacy

    The primary efficacy outcome will be a binary "Complete Success" of the graft defined as improvement in corneal surface integrity

    Measured: 18 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Oct 2015 (estimated)
  2. Study start1 Aug 2016
  3. Primary completion31 Mar 2023
  4. Study completion31 Mar 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
All subjects will receive the study intervention, a stem cell graft cultivated from their own cells.
Masking
None (open label)
Enrolment
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02592330
Sponsor's protocol ID
14-124H
Acronym
CALEC
Status last verified
Jan 2025
First submitted
9 Feb 2015
Last updated
15 Jan 2025
Data monitoring committee
Yes
Individual data sharing
No

Publications

  • Yavuz Saricay L, Kaufman AR, Johns LK, Yin J, Samarakoon L, Ayala AR, Maguire M, Parekh M, Hernandez Rodriguez DE, Daley H, Dana R, Armant M, Ritz J, Jurkunas UV. Central Cornea Changes on Anterior Segment OCT and In Vivo Confocal Microscopy After Autologous Limbal Epithelial Cell Transplantation. Cornea. 2026 Apr 1;45(4):436-442. doi: 10.1097/ICO.0000000000003865. Epub 2025 Mar 28.

    PubMed 40152603

  • Jurkunas UV, Kaufman AR, Yin J, Ayala A, Maguire M, Samarakoon L, Johns LK, Parekh M, Li S, Gauthier A, Negre H, Shaw KL, Hernandez Rodriguez DE, Daley H, Dana R, Armant M, Ritz J. Cultivated autologous limbal epithelial cell (CALEC) transplantation for limbal tem cell deficiency: a phase I/II clinical trial of the first xenobiotic-free, serum-free, antibiotic-free manufacturing protocol developed in the US. Nat Commun. 2025 Mar 4;16(1):1607. doi: 10.1038/s41467-025-56461-1.

    PubMed 40038272

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Biopsy to collect limbal epithelial stem cells that will be cultivated into a graft, Cultivation of Limbal epithelial cells into a graft and CALEC Transplant in people with Limbal Stem Cell Deficiency, run by Massachusetts Eye and Ear Infirmary at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Primary Safety Events of Interest (time frame: 18 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02592330, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jan 2025.

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