Long-term safety after Holoclar® implant for restoration of corneal epithelium in patients with limbal stem cell deficiency due to ocular burns: observational study of routine clinical practice (HOLOSIGHT)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Other
• If ‘other’, further details on the scope of the study:
  Effectiveness of risk minimization measures

Non-interventional study

• Non-interventional study design:
  Cohort

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:
  100

Study design details

• Main study objective:
  To evaluate the long-term safety profile of patients treated with Holoclar during a 5-year follow-up period from first ocular implantation under routine clinical conditions
• Outcomes:
  Safety outcomes (AEs and symptoms) will be collected from biopsy to end of observation.AEs include Serious and Non-serious Adverse Events, Adverse Drug Reactions and Adverse Events of Special Interest (AESI). AESI are defined as glaucoma and blepharitis.Symptoms include pain, photophobia, burning, Quality of life, rate of success, visual acuity during 5 years
• Data analysis plan:
  Analyses will include data collected at available observational points, according to clinical practice and clinical judgment. For vital signs, LSCD severity, symptoms, corneal epithelium defects, superficial corneal neovascularization and BCVA at corneal biopsy, if the evaluation is not performed on the same day, the most recent available data at the time will be collected instead. In order to summarize data by time point (e.g. one year after implant, two years after implant, etc.), the nearest available evaluation/ measurement will be considered.Concerning the safety profile, the proportion of patients experiencing at least one adverse event, serious adverse event, AE related to biopsy, surgical procedure, post-implant pharmacological treatment or cell-based product and AESI will be provided, both globally and for the 5 years observational period from the first implantation. The occurrence of symptoms will be described too.