Open-label, longitudinal, post-authorisation safety study to assess the safety of Cystadrops in pediatric and adult cystinosis patients in long term use (CYT-DS-001)

Study type

• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Descriptive and longitudinal single-arm study

Study drug and medical condition

• Name of medicine:
  CYSTADROPS
• Medical condition to be studied:
  Cystinosis

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • 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 assess whether they are any potential risks to develop high severity degrees of local intolerance with CYstadrops or other serious AEs that could lead to potential consequences for compliance to treatment in the long term
• Outcomes:
  To assess the safety profile of Cystadrops in long term use by measuring the incidence of the safety events collected in the study for all patients, Benefit of Cystadrops by measuring photophobia, description of ophthalmological assessments (CCCS, Crystal thickness and BCVA)
• Data analysis plan:
  Due to the limited number of patients in this rare disease, no formal statistical test will be done. Descriptive analysis will be performed for the total study population (overall analysis) defined as all patients enrolled in the study and that received at least one dose of Cystadrops.