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

Inclusion criteria

• ✓. Written informed consent or assent as appropriate obtained prior to any study-related procedure being performed;
• ✓. Patients at least 8 years of age or older at the time of consent or assent;
• ✓. 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;
• ✓. At least 8 or more blood transfusions in the past year;
• ✓. Survival expected of \>12 months;
• ✓. 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);
• ✓. 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
• ✓. 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