unilateral total limbal stem cell deficiency
Conditions
Interventions
Product Name: ex vivo expanded autologous limbal stem cells
Product Code: N/A
Pharmaceutical Form: Living tissue equivalent
INN or Proposed INN: Autologous stem cells
Sponsors
The Newcastle upon Tyne Hospitals NHS Foundation Trust
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Patient has provided written informed consent for participation in the study prior to any study specific procedures; • Patients must be 18 years of age or older and consent to have their data included in the database for research purposes; • Patients must be prepared and able to complete questionnaires; • Diagnosis of unilateral total LSCD (confirmed by impression cytology), with normal B scan ultrasound & electrophysiology; • No other ocular abnormality in recipient eye(s); • Women of child bearing potential must be using adequate contraception for duration of study and have a negative baseline pregnancy test as part of screening (post-consent). Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: • Significant co-morbidity in which compliance with the study procedures would not be expected e.g. suspected insufficient cognitive ability to perform the tests (assessed using the 11-item Telephone Interview for Cognitive Status instrument); • Dry eye and eyelid abnormality in the affected eye; • Previous surgery to ocular surface of healthy contralateral donor eye; • Abnormal corneal impression cytology in healthy contralateral donor eye. • Pregnancy, or women planning to become pregnant within next 12 months, or women who are breast feeding; • Participating in other investigational study within 30 days prior to study entry (defined as date of enrolment/baseline visit into study); • Previous participation in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): 1. Reversal of LSCD by impression cytology; 2. Ocular surface reconstruction (i.e., corneal re-epithelisation); 3. Improvement in patient’s reported ocular surface pain.;Main Objective: To evaluate the safety and efficacy (or capacity to produce a desired effect) of ex vivo expanded autologous Limbal Stem Cells transplantation (transplant derived from the same patient, animal free and grown ex vivo or outside the body) for the treatment of unilateral total Limbal Stem Cell Deficiency (a severe corneal disease resulting in painful blindness). The product is manufactured according to GMP guidelines and under a Quality Management System.;Secondary Objective: 1. To evaluate the impact on quality of life of unilateral total Limbal Stem Cell Deficiency and changes after transplantation; 2. To evaluate the management of Limbal Stem Cell Deficiency in relation to clinical, economic and humanistic outcomes; 3. To evaluate the reversal of LSCD by impression cytology; 4. To evaluate an Optical Coherence Tomography (OCT) pattern as a means of predicting visual outcome after cultured LSC transplantation and the potential need for secondary intervention (e.g. corneal transplantation) after transplantation; 5. To evaluate the pre-corneal tear film in patients with unilateral LSCD before and after limbal stem cell transplantation; 6. To evaluate improvement in LogMAR visual acuity; 7. To evaluate reversal of central corneal vascularisation; 8. To evaluate reduction in corneal opacity; 9. To evaluate corneal/limbal epithelial changes by confocal microscopy; 10.To evaluate the molecular changes in the corneal tear film, particularly cytokines a;Timepoint(s) of evaluation of this end point: 1. Reversal of LSCD by impression cytology - 6 month & 12 month post-op 2. Ocular surface reconstruction (i.e., corneal re-epithelisation) - all visits post-op up to 12 months 3. Improvement in patient’s reported ocular surface pain - 12 weeks, 6 month & 12 month post-op S | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Improvement in LogMAR Visual acuity; 2. Reversal in central corneal vascularisation; 3. Reduction in corneal opacity; 4. Improvement in patient’s reported outcomes; 5. Presence of complications; 6. Corneal opacity assessment by anterior segment OCT; 7. Corneal/limbal epithelial assessment by confocal microscopy.;Timepoint(s) of evaluation of this end point: 1. Improvement in LogMAR Visual acuity - all visits post-op up to 12 months 2. Reversal in central corneal vascularisation - all visits post-op up to 12 months 3. Reduction in corneal opacity - all visits post-op up to 12 months 4. Improvement in patient’s reported outcomes - 12 weeks, 6 month & 12 month post-op 5. Presence of complications - all visits post-op up to 12 months 6. Corneal opacity assessment by anterior segment OCT - 2 weeks, 4 weeks, 8 weeks, 12 weeks, 6 months & 12 months post-op 7. Corneal/limbal epithelial assessment by confocal microscopy - 6 months & 12 months post-op Some of the above procedures/investigations will also continue after 12 months post-op, as per routine clinical practice. | — |
Countries
United Kingdom
Outcome results
None listed