A Phase 1/2 interventional study of Biopsy to collect limbal epithelial stem cells that will be cultivated into a graft and Cultivation of Limbal epithelial cells into a graft in Limbal Stem Cell Deficiency, sponsored by Massachusetts Eye and Ear Infirmary. Completed at 1 site in United States. Open to participants aged 18 Years to 89 Years. Per ClinicalTrials.gov, last updated 2025-01-15.
Sponsored by Massachusetts Eye and Ear Infirmary · Phase 1/2, Interventional, and Treatment
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.
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.
40 studies on the registry are indexed under Limbal Stem Cell Deficiency; 15 are open to participants now.
This study's enrollment of 23 is above the median of 20 across 25 interventional studies indexed under Limbal Stem Cell Deficiency.
Browse Limbal Stem Cell Deficiency studies →Massachusetts Eye and Ear Infirmary is the lead sponsor of 99 studies on the registry; 23 are open to participants now.
Of its 13 completed or terminated interventional studies of FDA-regulated products, 6 (46%) have results posted.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria:
Additional optional criteria:
Exclusion Criteria:
Exclusion Based on Donor Eye:
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 · Biological: Cultivation of Limbal epithelial cells into a graft · Procedure: CALEC Transplant
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.
Also known as: Cultivated Autologous Limbal Epithelial Cell (CALEC)
A graft is manufactured for transplant
Limbal epithelial cells are obtained from the healthy fellow eye and cultivated in a lab for later transplantation into the diseased eye.
Also known as: Conjunctival Limbal Autograft
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%
Time frame: 18 Months
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.
Time frame: 18 Months
Measure of Transplant Efficacy
The primary efficacy outcome will be a binary "Complete Success" of the graft defined as improvement in corneal surface integrity
Time frame: 18 Months
| Milestone | Cultivated Autologous Limbal Epithelial Cell (CALEC) Graft | Control Conjunctival Limbal Autograft (CLAU) |
|---|---|---|
| Started | 16 | 1 |
| Completed | 14 | 1 |
| Not completed | 2 | 0 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Withdrew: Failed biopsy | 1 | 0 |
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%
| Participants | CALEC | CLAU (Control) |
|---|---|---|
| Number of Participants with Ocular Infections | 1 | 0 |
| Number of Participants with Corneal Perforations | 0 | 0 |
| Number of Participants with Graft Detachments | 0 | 0 |
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.
| Biopsies | CALEC |
|---|---|
| Manufacturing Feasibility Measures | 14 |
The primary efficacy outcome will be a binary "Complete Success" of the graft defined as improvement in corneal surface integrity
| Participants | CALEC | CLAU |
|---|---|---|
| Complete Success : 3 Months Post Transplant | 7 | 1 |
| Complete Success : 12 Months Post Transplant | 11 | 0 |
| Complete Success : 18 Months Post Transplant | 10 | 1 |
Collected over 18 Months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| CALEC | 0/16 (0%) | 1/16 (6.3%) | 16/16 (100%) |
| CLAU | 0/1 (0%) | 0/1 (0%) | 1/1 (100%) |
| Event | CALEC | CLAU |
|---|---|---|
| VomitingGastrointestinal disorders | 1/16 | — |
| Event | CALEC | CLAU |
|---|---|---|
| Eye IrritationEye disorders | 0/16 | 1/1 |
| Eye ItchingEye disorders | 3/16 | 1/1 |
| Eye PainEye disorders | 12/16 | 1/1 |
| Eye RednessEye disorders | 4/16 | 1/1 |
| Eyelid DisorderEye disorders | 1/16 | 1/1 |
| Foreign Body SensationEye disorders | 4/16 | 1/1 |
| Subconjunctival HemorrhageEye disorders | 12/16 | 1/1 |
| Corneal Epithelium DefectEye disorders | 15/16 | 1/1 |
| Eye IrritationEye disorders | 1/16 | 1/1 |
| Eye ItchingEye disorders | 1/16 | 1/1 |
Participants receiving a CALEC transplant, or the standard of care treatment (CLAU)
| Age, Continuous(Years) | CALEC | CLAU (Control) | Total |
|---|---|---|---|
| Median | 42.0 (24.0 to 78.0) | 53.0 (53.0 to 53.0) | 46.0 (24.0 to 78.0) |
| Sex: Female, Male(Participants) | CALEC | CLAU (Control) | Total |
|---|---|---|---|
| Female | 1 | 1 | 2 |
| Male | 13 | 0 | 13 |
| Race (NIH/OMB)(Participants) | CALEC | CLAU (Control) | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 1 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 12 | 1 | 13 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Region of Enrollment(Participants) | CALEC | CLAU (Control) | Total |
|---|---|---|---|
| United States | 14 | 1 | 15 |
| Epithelial Integrity(Participants) | CALEC | CLAU (Control) | Total |
|---|---|---|---|
| Count of participants | 4 | 0 | 4 |
| Epithelial Integrity(units on a scale) | CALEC | CLAU (Control) | Total |
|---|---|---|---|
| Median | 15.0 (10.0 to 15.0) | 10.0 (10.0 to 10.0) | 15.0 (10.0 to 15.0) |
| Neovascularization(Neovascular Area (% of total area)) | CALEC | CLAU (Control) | Total |
|---|---|---|---|
| Median | 7.5 (0.5 to 24.2) | 3.6 (3.6 to 3.6) | 7.2 (0.5 to 24.2) |
| Ocular Surface Disease Index Score(units on a scale) | CALEC | CLAU (Control) | Total |
|---|---|---|---|
| Median | 40.6 (6.8 to 100.0) | 95.5 (95.5 to 95.5) | 41.7 (6.8 to 100.0) |
1 further baseline measures are reported on the registry.
Documents are hosted by the registry — open the source record to download them.
Plan to share: No
This study is completed, as verified in Jan 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Massachusetts Eye and Ear Infirmary