Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation

Inclusion Criteria

• •1. Written informed consent or assent as appropriate obtained prior to any study-related procedure being performed;
• •2. Patients at least 8 years of age or older at the time of consent or assent;
• •3. Patient with TDT syndrome and iron overload currently receiving iron chelation therapy with Jadenu or Exjade and demonstrating inadequate response assessed with serum ferritin and LIC;
• •4. At least 8 or more blood transfusions in the past year;
• •5. Survival expected of \>12 months;
• •6. Patient previously on dual iron chelation therapy will be transfered to iron chelation monotherapy ,stable dosing with Jadenu or Exjade for ≥1month prior to screening and receiving doses in the maximal dose range per day (e.g., Jadenu: \>21 mg/kg or Exjade: \>30 mg/kg, with specific doses in these ranges prescribed at the physician's discretion);
• •7. Serum ferritin levels that are persistently \>800 mcg/L determined by 2 separate assessments during screening over the previous 2-4 weeks prior to study treatment and not showing a decreasing trend over these weeks OR, an LIC of \>5 mg Fe/g dw measured by MRI in the 52 weeks prior to study entry, OR clearly identified as a poor responder in medical records within 3 months prior to the study
• •8. Compliant with chelation therapy in the 3 months prior to enrollment in the opinion of the Investigator; has taken at least 75% of medication prescribed on a regular basis was taken (Investigator enquiry into patient prescription refill records, preferably 3 months if available, SICT scores); and
• •1. Willing to comply with chelation therapy for the duration of the study;
• •2. The determination of compliance is at the discretion of the investigator.
• •9. Agree not to use other anti-chelating agents concurrently;
• •10. Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0-1;
• •11. Women of childbearing potential (WOCBP) must use an adequate method of birth control (double barrier, e.g. hormonal control and barrier contraception) at least 28 days prior to the first administration of the study drug, during the study and for at least 30 days after the last dose of the study drug;
• •12. Male patients whose partners are WOCBP must use an adequate method of birth control (double barrier control) at least 28 days prior to the first administration of the study drug, during the study and for at least 30 days after the last dose of the study drug; and
• •13. Patient is willing and able to comply with all protocol required visits and assessments.