Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
Condition: Beta-thalassemia; Iron Overload
MeSH terms: Thalassemia; beta-Thalassemia; Iron Overload
Intervention: Deferasirox
Intervention description: Oral deferasirox 30mg/kg/day once per day for 77 weeks.
Intervention MeSH terms: Deferasirox
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 30.0
Sex: All
Minimum age: 10 Years
Healthy volunteers: No
Keywords: Iron Chelation; Deferasirox; Chelator; Desferal; beta-thalassemia; Iron overload

Summary:

      The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in
      beta-thalassemia patients with deferasirox treatment.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female β-thalassemia outpatients on chronic transfusion therapy (defined as >
             8 transfusions per year)

          -  Lifetime minimum of 100 previous packed red blood cell transfusions

          -  Patients currently on chelation therapy will require a one day wash out prior to the
             first dose of study drug

          -  Age ≥ 10 years

          -  Sexually active females of childbearing potential must have a negative serum or urine
             pregnancy test and use an effective method of contraception, or must have undergone
             clinically documented total hysterectomy.

        Exclusion Criteria:

          -  Ejection Fraction < 56 % measured using steady-state free precession imaging by MRI

          -  Contraindication to MRI, including cardiac pacemaker, brain aneurysm clip, implanted
             neurostimulator, insulin pump, cochlear implant, metal slivers in the eyes,
             intrauterine device or any other MRI incompatible metal implants or intractable
             claustrophobia

          -  Abnormal laboratory values as defined by the protocol

          -  Clinical or laboratory evidence of active Hepatitis B or Hepatitis C

          -  History of HIV positive test result (ELISA or Western blot)

          -  Uncontrolled systemic hypertension

          -  Second or third degree A-V block

          -  Life-threatening arrhythmias, including sustained ventricular tachycardia and aborted
             sudden death, within the last year

          -  History of cardiac conditions or unstable cardiac disease not controlled by standard
             medical therapy

          -  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 (documented negative pregnancy test required for study
             entry)

          -  Patients enrolled in an ongoing clinical trial of deferasirox (ICL670) cannot be
             withdrawn in order to participate in this study

          -  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 any drug

          -  History of non-compliance to medical regimens or patients who are considered
             potentially unreliable and/or not cooperative

          -  Other inclusion/exclusion criteria may apply