An observational, multicenter study to evaluate the safety and tolerability of deferasirox in the treatment of pediatric patients with non-transfusion-dependent iron overload (NESO)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Non Interventional Safety

Study drug and medical condition

• Name of medicine:
  EXJADE
• Medical condition to be studied:
  Iron overload

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  40

Study design details

• Main study objective:
  Primary objective: To characterize the long term safety profile of deferasirox in pediatric patients with NTDT with exposure up to 5 years
• Data analysis plan:
  Data will be collected on 40 eligible pediatric patients aged ≥ 10 to <18 years at enrollment with non-transfusion dependent thalassemia treated with deferasirox and will be followed for up to 5 years from the start of deferasirox treatment. Retrospective data collection will be done for patients whom have started deferasirox 12 months or less prior to enrollment, i.e. data will be collected from the start of deferasirox treatment (baseline) and then on for five years.