Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
Condition: Transfusional Iron Overload; Transfusional Hemosiderosis
MeSH terms: Hemochromatosis; Iron Overload; Hemosiderosis
Intervention: Core Study: Deferasirox; Core Study: Deferoxamine; Extension: deferoxamine to deferasirox; Extension: deferasirox to deferoxamine; Deferasirox; Deferoxamine
Intervention description: 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; 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week; 40 mg/kg deferasirox once daily administered 30 minutes before taking food.; 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 MeSH terms: Deferasirox; Deferoxamine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 197.0
Sex: All
Minimum age: 10 Years
Maximum age: 65 Years
Healthy volunteers: No
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

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.
    

Eligibility criteria:

        Inclusion criteria:

          -  Male or female patients, aged 10 years and above, with β-thalassemia major or DBA or
             sideroblastic anemia on chronic transfusion therapy, having given written consent to
             participate in the study.

          -  Patients with cardiac iron as measured by a myocardial T2* value that is ≥ 6ms but not
             ≥ 20 ms.

          -  Patients with a lifetime history of at least 50 units of red cell transfusions, and
             must be receiving at least ≥10 units/yr of red blood cells transfusions.

          -  Patients with a left ventricular ejection fraction (LVEF) ≥ 56 % as determined by
             cardiovascular magnetic resonance (CMR).

          -  Patients with liver iron content (LIC) value ≥ 3 mg Fe / g dw, as determined by liver
             MRI.

        Exclusion criteria:

          -  Patients with clinical symptoms of cardiac dysfunction.

          -  Patients unable to undergo study assessments including MRI

          -  Patients participating in another clinical trial or receiving an investigational drug.

        Other protocol-defined inclusion/exclusion criteria may apply.