NCT00469560

Safety, Tolerability, and Efficacy of Deferasirox in MDS

Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 158 (actual)
• Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis. Patients receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron administered during transfusions and, if needed, to reduce the overload of already present iron. After an screening phase in which patients are evaluated according to eligibility criteria, a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.

Detailed description

It has been widely shown that an appropriate chelating therapy in chronic anemias transfusion dependent can prevent the overstock of iron and can reduce the already existing overstock reducing, then, the co-morbidity and improving survival. In particular, some authors have shown in MDS affected patients undergoing intensive chelating therapy with deferoxamine haematological recovery with a reduction of the need of transfusions. With the present study, we plan to evaluate the safety and efficacy of a therapy with the new oral chelating Deferasirox in MDS patients with transfusional hemosiderosis. This is an open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis. Patients will receive daily oral dosis of Deferasirox in order to eliminate the quantity of iron administered during transfusions and, if needed, to reduce the overload of already present iron. After an screening phase in which patients are evaluated according to eligibility criteria, a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.

Conditions

• Myelodysplastic Syndromes
• Hemosiderosis

Interventions

Type	Name	Description
DRUG	Deferasirox

Timeline

• Start date: 2007-06-01
• Primary completion: 2010-03-01
• Completion: 2013-11-01
• First posted: 2007-05-04
• Last updated: 2016-11-22

Locations

17 sites across 1 country: Italy