Cultivated Limbal Epithelial Transplantation (CLET) for Limbal Stem Cell Deficiency (LSCD)

Study Overview
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Brief Summary
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CLET is a published treatment for the management of corneal failure due to extensive LSCD. Due to our previous studies on this novel treatment, the regulatory agency of Spain Agencia Española de Medicamentos y Productos Sanitarios (AEMPS) authorized our institution (IOBA-University of Valladolid) to perform this kind of therapy (CLET) in a case-by-case base following the Special Situation Medicines Policy Procedure in Spain. Upon approval of the permanent authorization patients will be included as specified by AEMPS.The objective of this study is to perform a protocolized treatment and follow up so that results can be reported to the scientific community.

Official Title
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Cultivated Limbal Epithelial Transplantation (CLET) for Limbal Stem Cell Deficiency (LSCD): an Observational Study on Safety and Effectiveness

Conditions
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Limbal Stem-cell Deficiency

Participation Criteria
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Eligibility Criteria
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Total and/or severe LSCD of any etiology in one or both eyes.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients treated with CLET<br>Previously treated with CLET patients are included retrospectively, as the follow-up procedure is already stablished in our centre, and patients treated following standard care or through drugs-in-special-situation request in Spain prospectively. Patients are not treated to be included in the study, only the follow-up variables are taken into account. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Points of improvement in the Single Item Dry Eye Questionnaire (SIDEQ) | The SIDEQ gives a 0-4 score about the presence of dryness, foreign body sensation, burning/stinging, pain, itching, sensitivity to light, and blurred vision, where higher scores indicate higher intensity of the symptom (maximun score: 28). One of the primary outcomes for this work is the improvement in any of the four questionnaires shown here. | 6 months | 
| Points of improvement about the Ocular Surface Disease Index (OSDI) | The OSDI evaluates ocular surface symptoms with 12 questions, and scores >12 indicate abnormal symptomatology, and >32 means severe symptoms (maximum score 100). One of the primary outcomes for this work is the improvement in any of the four questionnaires shown here. | 6 months | 
| Points of improvement in the National Eye Institute 25-item Visual Function Questionnaire (NEI-VFQ25) | The visual function-related aspects of the quality of life were evaluated with the NEI-VFQ25, where higher scores on a 0 to 100 scale indicate better function. One of the primary outcomes for this work is the improvement in any of the four questionnaires shown here. | 6 months | 
| Points of improvement in the Change in Dry Eye Symptoms Questionnaire (CDES-Q) | The CDES-Q evaluates ocular surface symptoms with two questions, CDES-Q1 and CDES-Q2. CDES-Q1 evaluates the change in symptoms occured since the previous examination (same, better or worse). CDES-Q2 evaluates the magnitude of the change in a scale from 0 to 10, where 0 means slightly better/worse and 10 means extremely better/worse. One of the primary outcomes for this work is the improvement in any of the four questionnaires shown here. | 6 months | 
| Percentage of improvement in corneal conjunctivalization | Percentage of corneal area with conjunctivalization (superficial opacity and/or superficial neovascularization) will be measured. | 6 months | 
| Complete absence of persistent epithelial defects | Corneal fluorescein staining has to rule out epithelial breakdown | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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limbal stem cell deficiency, corneal failure, limbal epithelial stem cells, ocular surface, corneal blindness