A phase 2 multicenter, open-labeled study of efficacy and safety of Deferasirox GPO in thalassemia patients with iron overload
- Conditions
- transfusion dependent thalassemia (TDT) and non-transfusion dependent thalassemia (NTDT) patients who have iron overloadthalassemia, iron overload
- Registration Number
- TCTR20240601004
- Lead Sponsor
- The Government Pharmaceutical Organization (GPO)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 160
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
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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method iver iron concentration (LIC) reduction during 48 weeks at 48 weeks after end of the intervention Magnetic resonance imaging (MRI) for LIC
- Secondary Outcome Measures
Name Time Method 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*