Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients
Condition: Aplastic Anemia
MeSH terms: Anemia; Anemia, Aplastic; Iron Overload
Intervention: ICL670; Chelation; No chelation
Intervention description: ICL670 was supplied in registered packages as 250mg or 500mg dispersible tablets.; Main group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment (immunosupressant - Cyclosporine A) and received chelation with ICL670 (deferasirox).; Comparative group of patients with aplastic anemia and transfusion-dependent iron overload underwent treatment programs of standard immunosuppressive treatment ( immunosupressant -Cyclosporine A)
Intervention MeSH terms: Deferasirox
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 15.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Aplastic anemia; deferasirox; ICL670; renal function; transfusion-dependent iron overload; immunosuppressive treatment; Cyclosporine A; unosuppressive treatment without chelation therapy

Summary:

      Evaluated Exjade efficacy and safety in patients with aplastic anemia and
      transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive
      treatment (Cyclosporine A) , in comparison with a group of patients undergoing treatment
      programs of immunosuppressive treatment (Cyclosporine A) without chelation therapy.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Main diagnosis: aplastic anemia

          -  Absence of severe and/or uncontrolled comorbidities

          -  Confirmed iron overload (serum ferritin ≥ 1000 mkg/L)

          -  Serum creatinine is not higher than the upper limit of normal for the given age

          -  Absence of severe proteinuria. Protein/Creatinine ratio should be < 0.5 mg/mg

          -  Liver enzymes are < 5 ULN

          -  Completion of a scheduled cycle of immunosuppressive treatment program, with no severe
             infectious or generalized hemorrhagic complications

          -  WHO (ECOG) performance status ≤ 2

        Exclusion Criteria:

          -  No signed informed consent form

          -  Patient is under 18 years old

          -  Severe concomitant condition

          -  Severe infectious and generalized haemorrhagic complication following regular planned
             cycle of programmed immune suppressive treatment.

          -  History of increased sensitivity to active substance and any other ingredient of the
             medicinal product.

          -  Creatinine clearance (CC) < 60 ml/min and/or creatinine concentration in blood serum
             is 2 or more times higher than upper limit of age normal by results of 2 tests at
             Visits 1 and 2.

          -  Severe liver disorders (class C by Child-Pugh scale).

          -  Patients with aplastic anaemia in which chelator treatment will be ineffective due to
             rapid progression of the disease.

          -  Significant proteinuria basing on protein creatinine ratio > 1.0 mg/ml in urine sample
             from second urination at Visits 1 and 2 (or as an alternative in 2 of 3 urine samples
             at screening);

          -  Rare hereditary disorders related to galactose intolerance, severe deficit of lactase
             or glucose-galactose malabsorption;

          -  Pregnancy, lactation;

          -  Level of liver enzymes higher than 5 upper limits of age normal at Visits 1 and 2.