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 identification

• PURI:
  https://redirect.ema.europa.eu/resource/47545
• EU PAS number:
  EUPAS5914
• Study ID:
  47545
• Official title and acronym:
  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)
• DARWIN EU® study:
  No
• Study countries:
  Egypt

  France

  Greece

  Lebanon

  Oman

  Saudi Arabia

  Thailand

  Türkiye

  United Arab Emirates

  United States
• Study description:
  This is a pediatric registry in patient with non-transfusion dependent thalassemia who are aged ≥ 10 to <18 years at enrollment and taking deferasirox. 40 patients will be enrolled and evaluated for safety for a 5-years follow-up period.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Novartis Pharmaceuticals

Study timelines

Date when funding contract was signed

Planned: 30/09/2012

Actual: 31/12/2013

Study start date

Planned: 30/06/2014

Actual: 28/07/2014

Data analysis start date

Planned: 06/01/2025

Actual: 08/01/2025

Date of final study report

Planned: 02/05/2025

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Novartis Pharmaceuticals

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 1 (imposed as condition of marketing authorisation)

Other study registration identification numbers and links

CICL670E2422