Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis
Condition: Chronic Anemia; Transfusional Hemosiderosis
MeSH terms: Anemia; Hemochromatosis; Hemosiderosis
Intervention: Deferasirox (ICL670)
Intervention description: The recommended initial daily dose of Deferasirox is 20 mg/kg body weight.
Intervention MeSH terms: Deferasirox
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 102.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: Iron Overload,; Chelation,; MDS,; Chronic Anemia,; Deferasirox,; Transfusional hemosiderosis,

Summary:

      The overall purpose of this trial is to further evaluate the efficacy and safety of
      deferasirox, dosed initially according to the transfusional iron intake, in patients with
      transfusion dependant anemia related to disorders other than β-thalassemia and sickle cell
      disease.

      During the study, the dose will be adjusted based on serum Ferritin.The overall purpose of
      the extension is to allow further treatment of patients who have already completed the core
      study, and to enable collection of long term efficacy and safety data. Patients will continue
      to receive Deferasirox at the dose they received at the end of the core study.
    

Eligibility criteria:

        Inclusion criteria (Core):

          -  Patients with transfusional iron overload due to:

          -  low or intermediate (INT-1) risk Myelodysplastic Syndrome (MDS)determined via
             International Prognosis Scoring System (IPSS) criteria

          -  other congenital or acquired anemias excluding B-thalassemia and sickle cell disease

          -  Lifetime transfusion history of ≥20 unit (approximately 100 mL/kg) of packed red blood
             cells or showing evidence of iron overload (serum ferritin >1000 µg/L).

          -  Able to provide written informed consent

          -  Life expectancy ≥ 12 months If patient was previously treated with deferiprone, a
             washout period of one month should occur before the first dose of deferasirox

        Inclusion criteria (Extension):

          -  Patients completing the planned 12-month core study (CICL670A2204).

          -  Written informed consent obtained from the patient and/or legal guardian on the
             patient's behalf in accordance with national legislation.

        Exclusion criteria (Core and Extension):

          -  Patients with β-thalassemia, sickle cell disease or myelodysplastic syndrome with an
             IPSS score being Intermediate-2 or High.

          -  Patients with serum creatinine > ULN

          -  Patients with ALT(SGPT) levels > 5 x ULN

          -  Significant proteinuria as indicated by a urinary protein/creatinine ratio >0.5 mg/mg
             in a non-first void urine sample on two assessments during the screening period.

          -  History of HIV positive test result , or of clinical or laboratory evidence of active
             Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV
             RNA positive and ALT above the normal range)

          -  Patients on investigational MDS therapies, including lenalidomide, thalidomide,
             azacitidine and arsenic trioxide, must have a ≥ 4 week washout period prior to the
             first dose of study drug.

          -  Patients with systemic uncontrolled hypertension

          -  Patients with unstable cardiac disease not controlled by standard medical therapy

          -  Systemic disease (cardiovascular, renal, hepatic, etc.) which would prevent study
             treatment

          -  Pregnancy (as documented in required screening laboratory test) or breast feeding.

          -  Patients treated with systemic investigational drug within the past 4 weeks or topical
             investigational drug within the past 7 days

          -  Other surgical or medical condition which might significantly alter the absorption,
             distribution, metabolism or excretion of study drug

          -  Patients being considered by the investigator potentially unreliable and/or not
             cooperative with regard to the study protocol

          -  History of hypersensitivity to any of the study drug or excipients

          -  Sexually active pre-menopausal female patients without adequate contraception. Female
             patients must use effective contraception or must have undergone clinically documented
             total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by
             amenorrhea for at least 12 months.

        Other protocol defined inclusion/exclusion criteria may apply.