Study type

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Safety study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Other

Non-interventional study design, other

Long-term Follow-up study
Study drug and medical condition

Name of medicine

HOLOCLAR

Medical condition to be studied

Limbal stem cell deficiency
Population studied

Age groups

  • Adult and elderly population (≥18 years)
    • Adults (18 to < 65 years)
      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)
      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)

Estimated number of subjects

70
Study design details

Main study objective

To collect long term follow-up clinical data on patients who received transplantation of autologous cultured limbal stem cells (ACLSCT) to confirm efficacy and safety of the treatment in restoring a normal corneal epithelium in patients suffering from limbal stem cell deficiency (LSCD) secondary to ocular burns.

Outcomes

% of patients with successful ACLSCT outcome, defined as CNV assessed by Independent Assessors in less than 2 quadrants and totally absent in central cornea as well as absence of mild to severe epithelial defects, % of patients with successful ACLSCT outcome defined as CNV assessed by investigator in less than 2 quadrants and totally absent in central cornea as well as absence of mild to severe epithelial defects, % of patients with ACLSCT outcome considered a success by investigator based on overall clinical status, % of patients by superficial corneal neo-vascularization score, Nr of related AE

Data analysis plan

General descriptive statistics for numeric variables will include the n (number of observed values), the mean, the standard deviation, the median, the minimum, and the maximum values. For categorical variables, the number and percent of subjects with a specific level of the variable will be presented.