Observational follow-up study to evaluate the long term efficacy of (HOLO-UP)

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

• Medicinal product name:
  HOLOCLAR
• Medical condition to be studied:
  Limbal stem cell deficiency

Population studied

• Age groups:
  • Adults (18 to < 46 years)
  • Adults (46 to < 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.