corneal lesions with associated moderate to severe limbal stem cell deficiency due to ocular burns MedDRA version: 20.0 Level: LLT Classification code 10011012 Term: Corneal epithelium opacity System Organ Class: 100000004853
Conditions
Sponsors
Holostem Terapie Avanzate S.r.l.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Written informed consent prior to any study-related procedures; 2. Adult male and female patients (=18 years old) - [Five Paediatric patients aged 2 to 17 years will be also enrolled for safety purposes only]; 3. LSCD secondary to unilateral or bilateral physical or chemical ocular burns, with at least 1-2 mm2 of undamaged limbus to harvest stem cells for expansion in culture. LSCD will be considered for inclusion in presence of superficial neo-vascularization invading at least two corneal quadrants with evidence of central corneal (central 6 mm diameter) involvement (including central corneal neo-vascularisation or corneal opacity) according to the independent assessors; 4. Stability of LSCD, defined by a duration of disease of at least 24 months (12 months for minors) at the time of the Screening Visit and as presence of continuum epithelium as per fluorescein staining scored as none or trace; 5. Presence of severe impairment in visual acuity defined by a score after best correction (i.e. Best Corrected Visual Acuity) equal or below 1/10 (or 20/200) at the Snellen chart (legal blindness); 6. Absence of other clinical contraindications to ACLSC transplantation based upon investigator’s judgment; 7. A cooperative attitude to follow up the study procedures (Caregivers in case of minors). 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 73 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 14
Exclusion criteria
Exclusion criteria: 1. LSCD of mild degree (i.e. below 2 quadrants of vascularization invasion), due to a recent burn (less than 24 months before screening for adults and 12 months for minors), or secondary to medical conditions other than burns (i.e. radiotherapy); 2. Severe ocular inflammation according to the Efron Grading Scale for Contact Lens Complications. Patient can be re-screened after appropriate treatment; 3. Presence of eyelids malposition; 4. Conjunctival scarring with fornix shortening; 5. Tear secretion deficiency, determined by Schirmer’s test (<5 mm/ 5 min); 6. Corneal anaesthesia and conjunctival anaesthesia or severe hypoesthesia;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the efficacy of Holoclar® at one year after the first treatment in patients suffering from moderate (vascularization in two-three corneal quadrants with central corneal involvement) to severe (vascularization in four corneal quadrants with central corneal involvement) LSCD with severe visual impairment and secondary to ocular burns, in terms of percentage of patients with a success of transplantation at approximately 12 months from the first Holoclar® treatment.;Secondary Objective: To evaluate the efficacy of one or two treatments with Holoclar at one year after the last treatment. ;Primary end point(s): - Percentage of patients with a success of transplantation (success is defined on the basis of the degree of “superficial corneal neo-vascularization” and “epithelial defects”);Timepoint(s) of evaluation of this end point: 12 months from the first Holoclar treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Percentage of patients with clinical success after one or two ACLSCTs (same definition of success as primary endpoint) ;Timepoint(s) of evaluation of this end point: 12 months after the last treatment with Holoclar. | — |
Countries
Belgium, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom
Contacts
Public ContactClinical Project Manager
Holostem Terapie Avanzate S.r.l.
Outcome results
None listed