Clinical Trial: A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Brief Summary: Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.

Detailed Summary:
Sponsor: Novartis

Current Primary Outcome: Liver Iron Concentration (as measured by biopsy) [ Time Frame: at baseline and after 1 yeor of ICL670 treatment ]

Original Primary Outcome: Efficacy of ICL670 treatment based on LIC

Current Secondary Outcome:

  • Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores) [ Time Frame: after 1 year of ICL670 treatment ]
  • Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation [ Time Frame: monthly assessments for 1 year ]
  • Adverse events [ Time Frame: during 1 year ICL670 treatment ]
  • hematology and biochemistry, urinalysis [ Time Frame: monthly assessments for 1 year ICL670 treatment ]
  • ECG and Echocardiography [ Time Frame: 6-monthly for 1 year ICL670 treatment ]


Original Secondary Outcome:

  • Tolerability profile of ICL670
  • Absolute + relative change of LIC and Total Body Iron Excretion (TBIE) rate for subgroups
  • Relationship between LIC and potential surrogate markers
  • Potential of Magnetic Resonance Imaging (MRI)for LIC
  • Quality of life and patient global assessment
  • Drug usage
  • Dose adjustment regimens as dictated by efficacy and safety parameters in comparison to transfusional burdens


Information By: Novartis

Dates:
Date Received: September 12, 2005
Date Started: May 2004
Date Completion:
Last Updated: February 28, 2017
Last Verified: November 2016