transfusion dependent thalassemia (TDT) and non-transfusion dependent thalassemia (NTDT) patients who have iron overload thalassemia, iron overload
Conditions
Interventions
Maximum dose: 40 mg/kg/day for TDT, 30 mg/kg/day for NTDT
Mode of administration: given orally
Duration: 48 weeks
Experimental Drug
Deferasirox GPO
Sponsors
The Government Pharmaceutical Organization (GPO)
Health Systems Research Institute
Eligibility
Sex/Gender
All
Age
2 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Thai patient, Aged more than 2 years old 2. Have a documented diagnosis of TDT or NTDT; TDT patients are those who received blood transfusion at least 6 units (or episodes for children) within 24 weeks period and had no transfusion-free period of more than 45 days 3. Have a confirmed diagnosis of TDT or NTDT 4. Have an iron overload as indicated by LIC value during screening period; LIC > 7 mgFe/gDW for TDT and LIC >5 for NTDT, for those under 10 years old using ferritin > 1,000 and >800 ng/mL for TDT and NTDT, respectively
Exclusion criteria
Exclusion criteria: 1. Have a clinically significant GI disease that could interrupt the absorption of deferasirox or history of surgically removal of intestine 2. Have an active hepatitis B or hepatitis C (ALT > 3x upper limit normal) 3. Have a HIV infection 4. Have a clinically significant psychiatric disorder that could interfere the study protocol 5. Have a history of recent malignancy within 5 years, except for limited stage basal cell carcinoma 6. Currently participate in a clinical study 7. Have a history of hypersensitivity or serious side effect from deferasirox 8. Have an uncontrolled underlying disease that investigator believes that it would affect this study 9. serum creatinine > 1.5x upper limit normal 10. AST or ALT > 3x upper limit normal 11. Total bilirubin > 5x upper limit normal 12 Urinary protein/creatinine ratio (UPCR) > 0.5 mg/mg 13 Absolute neutrophil < 1,000 cells/uL 14. For female participant, be currently pregnancy or have a plan for pregnancy during clinical study or currently breastfeeding 15. For those of reproductive potential, be abstinent or agree to use 2 effective forms of contraception during the study period 16. Cannot perform MRI study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Liver iron concentration (LIC) reduction during 48 weeks at 48 weeks after end of the intervention Magnetic resonance imaging (MRI) for LIC | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety profile 48 weeks treatment period any abnormal laboratory or clinical symptoms that related to the intervention,Serum ferritin reduction at 24 and 48 weeks after end of the intervention Serum ferritin,Pharmacokinetics during 48 weeks treatment period plasma DFX level,LIC reduction during 24 weeks at 24 weeks after end of the intervention MRI for LIC,Cardiac T2* change at 48 weeks after end of the intervention MRI for cardiac T2* | — |
Countries
Thailand
Contacts
Public ContactSupachai Ekwattanakit
Faculty of Medicine Siriraj Hospital, Mahidol University
Outcome results
None listed