Clinical trial NCT00115349 (phase 2, status: terminated)
Conditions: cardiovascular diseases, heart diseases, beta-thalassemia
ICD codes: A52.00, A52.09, A50.54, Z01.810, Q87.418, Z13.6, R94.30, I11.0, I11.9, I27.1, I51.9, I09.9, I27.9, I01.8, D56.1, D56.2, D56.5, D57.42, D57.44, D57.432, D57.439
Drugs: deferoxamine, deferiprone (l1)
SMILES: CC(=O)N(O)CCCCCNC(=O)CCC(=O)N(O)CCCCCNC(=O)CCC(=O)N(O)CCCCCN, CN1C=CC(=O)C(O)=C1C
Reason stopped: 
    due to low enrollment
  
Eligibility criteria:

        Inclusion Criteria:

          -  Transfusion-dependent beta-thalassemia (eight or more transfusion episodes in the
             previous year)

          -  Left ventricular ejection fraction by MRI less than or equal to 56% by balanced
             steady-state free precession (SSFP) or 63% by spoiled gradient recalled echo (SPGR)

          -  Currently on treatment with subcutaneous or intravenous DFO; participants must be
             willing and able to chelate 7 days per week 12 - 24 hours per day

          -  Serum ferritin greater than 1000 µg/L or ferritin between 500 µg/L and 1000 µg/L and
             cardiac T2* less than 20 ms

        Exclusion Criteria:

          -  Pacemaker, severe claustrophobia, or other contraindications to MRI; severe congestive
             heart failure (New York Heart Association Classification IV); congenital or acquired
             valvular heart disease significant enough to require surgery or medications

          -  Currently receiving treatment for hepatitis; renal insufficiency defined by a
             clinically significant abnormal serum creatinine with a calculated creatinine
             clearance of less than 50 ml/min according to the Cockroft formula

          -  A neutrophil count less than 1.5 x 109/L on two or more occasions at least 4 weeks
             apart within the past year and not associated with an acute viral illness or a
             platelet count less than 80 x 109/L on two or more occasions at least 4 weeks apart
             within the past year

          -  Treatment with L1 or Exjade during the previous 2 weeks or previous adverse experience
             to L1 requiring suspension

          -  Infection with HIV

          -  Active participation in other investigational drug or device studies

          -  Unwilling to consider treatment with DFO at a dose of 50-60 mg/kg 12-24 hours per day
             7 days per week

          -  Women who are pregnant or breast feeding

          -  Systemic infection or cardiovascular, hepatic, renal, pulmonary, or gastrointestinal
             disease that would prevent patients from undergoing any of the study-required
             treatments or procedures or requires treatment with any contraindicated medication(s)

          -  Presence of any other condition that, in the opinion of the investigator, would make
             the patient unsuitable for enrollment or could interfere with the patient's compliance
             with the protocol; may include but is not limited to alcohol or drug abuse

          -  For women of child-bearing potential, an inability or unwillingness to use a highly
             effective method of contraception (e.g., implants, injectables, combined oral
             contraceptives, or some intrauterine devices)