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