Conditions: Transfusional Iron Overload Transfusional Hemosiderosis
Keywords: iron overload cardiac iron haemosiderosis myocardial T2* left ventricular ejection fraction LVEF cardiac dysfunction thalassaemia Diamond Blackfan anemia DBA sideroblastic anemia myelodysplastic syndromes MDS (low and INT-1 risk as per the IPSS for MDS) liver MRI deferasirox deferoxamine ICL670 DFO cardiovascular magnetic resonance imaging
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 197
Healthy volunteers: no

Brief summary:
This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions.

The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a medicine called deferasirox (ICL670) with another medicine called deferoxamine (DFO). The study is aimed at finding out which of the two medicines is the best for treating iron overload in the heart.

Patients will be treated for 12 months (core study phase). Patients who complete the core study phase will be offered to continue their study treatment in a 12 months extension phase. During the core and extension, the effects of treatment on iron overload in the heart and the liver will be evaluated using specific magnetic resonance imaging (MRI) assessments.

Detailed description:

Arms:
arm 1: 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day arm 2: 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week arm 3: "DFO to ICL" (patients who switched from DFO to deferasirox in extension) arm 4: "ICL to DFO" (patients who switched from deferasirox to DFO in extension)

Interventions: intervention 1: Core Study: Deferasirox intervention 2: Core Study: Deferoxamine intervention 3: Extension: deferoxamine to deferasirox intervention 4: Extension: deferasirox to deferoxamine intervention 5: Deferasirox intervention 6: Deferoxamine
Intervention descriptions: intervention 1: 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day intervention 2: 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week intervention 3: 40 mg/kg deferasirox once daily administered 30 minutes before taking food. intervention 4: DFO at a target range of 50 mg/kg/day to 60 mg/kg/day via subcutaneous (sc) infusion lasting a period of 8 to 12 hrs administered for 5 to 7 days per week, intervention 5: None intervention 6: None

Protocol document: