Limbal Stem Cell Deficiency of the Eye MedDRA version: 20.0 Level: PT Classification code 10063581 Term: Stem cell transplant System Organ Class: 10042613 - Surgical and medical procedures
Conditions
Interventions
Product Name: CAOMECS
Product Code: Not Applicable
Pharmaceutical Form: Living tissue equivalent
INN or Proposed INN: CAOMECS (Cultured Autologous Oral
Sponsors
Cellseed France S.A.R.L.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion criteria for all patients: 1. Age =2 years to 74 years 2. Males or females with bilateral or unilateral total LSCD due to one of the following causes: a) Chemical burns b) Thermal burns c) Contact lens wear d) Surgery of the ocular surface e) Stevens-Johnson syndrome and other inflammatory disease under stable condition f) Aniridia 3. Documented conjunctivalization of the corneal surface, measured by fluorescein staining 4. Stable disease, i.e. history of LSCD for at least 6 months 5. Clinical signs indicative of conjunctivalisation: a) Superficial blood vessels on the corneal surface b) Loss of epithelial transparency or persistent epithelial defect 6. Healthy oral mucosa 7. Absence from tobacco and alcohol (7 days before the biopsy) 8. Regular tooth brushing (at least twice daily) 9. Ability to comply with the protocol 10. Covered by a social security system 11. Signed informed consent form, ability to understand the study procedures, and contractual capability. Applicable to patient or legal carer (including parent) Special inclusion criteria for patients between 18 and 74 years of age (adults): 12. Multiple surgeries in the limbal region Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 65 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 12
Exclusion criteria
Exclusion criteria: Exclusion criteria for all patients: 1. Acute systemic infection 2. Acute ocular inflammation in the previous 6 months 3. Previous neoplastic/cancer disease 4. Severe dry eye confirmed by a Schirmer test 5. Lyell-Syndrome, epidermolysis bullosa 6. Total symblepharon 7. Medical history of hypersensitivity or allergy to bovine or murine derived materials 8. Pregnant and lactating patients, positive urine pregnancy test in women of childbearing potential 9. Any systemic infectious disease as diagnosed by serology tests such as syphilis, HIV-1, HIV-2, hepatitis B or C, or HTLV-1 infection at screening and at the day of the biopsy 10. Current or previous (within 30 days of enrolment) treatment with another investigational drug or participation in another clinical study 11. Previous participation of the patient in this study 12. Evidence of any other medical conditions (such as psychiatric illness, physical examination or laboratory findings) that may interfere with the planned treatment, affect patient compliance or place the patient at high risk of treatment-related complications 13. Employees of the sponsor or patients who are employees or relatives of the investigator 14. Genetic conditions such as ectodermal dysplasia or multiple endocrine neoplasia Special exclusion criteria for patients =2 and <18 years of age (children): 15. Multiple surgeries in the limbal region
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To restore the ocular surface epithelium by preventing recurrent conjunctivalization and neovacularization up to 12 months post transplantation.; Secondary Objective: Presence of a stable coreal epithilium and absence of conjunctivalisation at each follow-up visit at month 24 and 36. Prevention of recurrent neovacularization at each follow-up visit at month 24 and 36. To reduce the amount of punctate epithelial keratophathy. To increase the transparency of the epithelium at each follow-up visit. To improve visual acuity from Baseline to each follow-up visit. To improve photophobia, watering, and pain symptoms from Baseline to each follow-up visit. Change in patients' quality of life from Baseline to each follow-up visit (assessed by VFQ25 questionnaire). ; Primary end point(s): 1. Presence of a stable epithelium on cornea and absence of conjunctivalization in the visual axis at month 12 (success is defined as the absence of goblet cells, indicative of corneal phenotype) assessed by delayed fluorescein staining and impression cytology 2. Extent of neovascularization at month 12 (success is defined as a reduction of =50% compared to Baseline Criteria for evaluation - children As for primary endpoint No.1, assessments are only performed if investigator considered them feasible. Other than this item, the primary endpoint No.2 for adult patients will be applied for children. ;Timepoint(s) of evaluation of this end point: Month 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Presence of a stable corneal epithelium and absence of conjunctivalization in the visual axis at each follow-up visit at month 24 and 36 assessed by delayed fluorescein staining and impression cytology 2. Extent of neovascularization at each follow-up visit at month 24 and 36 (success is defined as a reduction of =50% as compared to Baseline) 3. Punctate epithelial keratopathy at each follow-up visit 4. Improvement of visual acuity from Baseline to each follow-up visit 5. Change in quality of life assessed by the National Eye Institute Visual Functioning Questionnaire 25 (VFQ25) from Baseline to each follow-up visit (a modified VFQ25 will be used for children and for unilateral cases) 6. Presence of a transparent epithelium in the visual axis at each follow-up visit 7. Improvement of photophobia from Baseline to each follow-up visit 8. Improvement of watering from Baseline to each follow-up visit 9. Improvement of pain from Baseline to each follow-up visit 10. Suitability and risk status for a corneal graft (lamellar or full thickness) at month 12 Criteria for evaluation - children As for secondary endpoint No. 1 and 4, assessments are only performed if investigator considered them feasible. Other than these items, the secondary endpoint No. 2, 3, 5 for adult patients will be applied for children. ;Timepoint(s) of evaluation of this end point: Month 12, 24, 36 | — |
Countries
Austria, Poland, Spain, United Kingdom
Contacts
Public ContactClinical Trials Information
CellSeed France S.A.R.L.
Outcome results
None listed