Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients
Condition: Myelodysplastic Syndrome; Iron Overload
MeSH terms: Myelodysplastic Syndromes; Iron Overload
Intervention: Deferasirox
Intervention description: 20 mg/kg/day over one year in patients with MDS
Intervention MeSH terms: Deferasirox
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 176.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: ICL670; Deferasirox; Iron chelation; Chelator; Desferal

Summary:

      The purpose of this trial is to examine the safety and efficacy of deferasirox in patients
      with Myelodysplastic Syndrome (MDS) and chronic iron overload from blood transfusions.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female patients with low or intermediate (INT-1) risk MDS

          -  Patients can be EITHER naïve to iron chelation OR have had prior treatment with
             deferoxamine (DFO).

          -  Age greater than or equal to 18 years

          -  Availability of transfusion records for the 12 weeks prior to registration

          -  A lifetime minimum of 30 previous packed red blood cell transfusions

          -  Availability of at least three CBC values (pretransfusion) during the 12 weeks prior
             to registration

          -  Serum Ferritin:

        For entry into the screening period, serum ferritin ≥ 1000 ng/mL on at least two occasions,
        at least two weeks apart, during the prior year.

        Serum ferritin ≥ 1000 ng/mL at screening via the central lab.

          -  Life expectancy ≥ 6 months

          -  Sexually active women must use an effective method of contraception, or must have
             undergone clinically documented total hysterectomy and/or oophorectomy, or tubal
             ligation or be postmenopausal (defined as amenorrhea for at least 12 months)

          -  Able to provide written informed consent

        Exclusion Criteria:

          -  Serum creatinine above the upper limit of normal

          -  Alanine aminotransferase (ALT) > 500 U/L during screening

          -  Clinical or laboratory evidence of active Hepatitis B or C

          -  Urinary protein/creatinine ratio > 0.5 mg/mg

          -  History of HIV positive test result (ELISA or Western blot)

          -  Eastern Cooperative Oncology Group (ECOG) Performance Status > 2

          -  Patients with uncontrolled systemic hypertension

          -  Unstable cardiac disease not controlled by standard medical therapy

          -  Patients with a diagnosis of or history of clinically relevant ocular toxicity related
             to iron chelation

          -  Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study
             treatment

          -  Pregnancy or breast feeding

          -  Treatment 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

          -  History of non-compliance to medical regimens or patients who are considered
             potentially unreliable and/or not cooperative