Primary and secondary Limbal Stem Cell Deficiencies MedDRA version: 17.1 Level: PT Classification code 10069798 Term: Amniotic membrane graft System Organ Class: 10042613 - Surgical and medical procedures MedDRA version: 17.1 Level: PT Classification code 10072138 Term: Limbal stem cell deficiency System Organ Class: 10015919 - Eye disorders
Conditions
Interventions
Product Name: Limbal Epithelial Cell (LEC) graft
Product Code: LEC
Pharmaceutical Form: Ophthalmic insert
INN or Proposed INN: LEC
Current Sponsor code: LEC
Other descriptive name: Limbal Epithelial C
Sponsors
Antwerp University Hospital (UZA)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Voluntary written informed consent 2. Patients suffering from LSCD IIa and IIb. Those suffering from IIc may be included once inflammation has subsided and cornea can be staged as IIb. 3. Absence of any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule; those conditions should be discussed with the patient before registration in the trial. 4. Women of child-bearing potential should use adequate contraception prior to study entry and for the duration of study participation. Negative serum or urine ß-HCG pregnancy test at screening. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 30
Exclusion criteria
Exclusion criteria: 1. Subjects who are pregnant or lactating 2. Subjects who have sensitivity to drugs that provide local anesthesia 3. Subjects suffering from active infection of the external eye 4. Medical conditions that prohibit the use of systemic immunosuppression (in cases of allogenic transplantation)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the success rate of transplanting non-xenogenic, bioengineered, composite grafts of cultured limbal epithelial stem cells on standardized amniotic membranes – in patients with LSCD. Based on the past experience from our phase I/II monocenter clinical trial, our primary hypothesis is that it will be well-tolerated, and not associated with serious adverse side effects. Assuming a success percentage of 67% based on our previous study, a sample size of 60 patients produces a two-sided 95% confidence interval with a width equal to 0.238. The larger sample size and inclusion of more allogenic transplant recipients, together with a longer follow-up, will help ascertain safety and efficacy of the proposed treatment.;Secondary Objective: The secondary objective of this study is to develop and implement a sub-clinical Ocular Surface Inflammation Monitoring (OSIM) tool based on biomarkers in tear samples. Further we would like to improve intra-operative visibility for the surgeon by introducing FD-OCT assisted pannus dissection prior to limbal stem cell transplantation. We further wish to introduce improved non-contact methods for transplanted corneal epithelial thickness monitoring and graft tracking via FD-OCT assisted follow-up. ;Primary end point(s): We will assess the success rate of transplanted limbal stem cells by a clinical observation of: Short term: 1. Increased visual acuity 2. Absence of conjunctivalization 3. Resolution of corneal vascularization 4. Absence of persistent epithelial defect 5. Decreased pain 6. Decreased photophobia Long term: 1. Prolonged subsequent corneal graft survival time ;Timepoint(s) of evaluation of this end point: Monitoring will be performed according to a fixed time schedule: * First month: weekly * Months 1, 2, 3, 6 * Every six months thereafter until minimun of 2 years follow-up | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Development and implementation of a sub-clinical Ocular Surface Inflammation Monitoring (OSIM) tool based on biomarkers in tear samples 2. Implementation of FD-OCT assisted limbal stem cell transplantation and post operative follow-up 3. Time until complete corneal epithelization is visualised using slit lamp 4. Presence or absence of normal corneal epithelial thickness using FD-OCT 5. Determine evolution of corneal pachymetry over time 6. Determine evolution of corneal epithelial thickness over time 7. In cases where a second operation needs to be preformed for improved visual rehabilitation, for example, penetrating keratoplasty, the removed corneal button will be sent for immunohistopathology in order to provide further insight into disease progression. ;Timepoint(s) of evaluation of this end point: Monitoring will be performed according to a fixed time schedule: * First month: weekly * Months 1, 2, 3, 6 * Every six months thereafter until minimun of 2 years follow-up | — |
Countries
Belgium
Contacts
Public ContactNadia Zakaria
Antwerp University Hospital (UZA)
Outcome results
None listed