Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Core Study: Deferasirox · 1 trial · 2 indications
Non- inferiority in efficacy of deferasirox compared to deferoxamine (DFO) in treating cardiac iron overload as measured by T2\*. A non-inferiority margin of 0.9 (90%) was applied. Due to limitations in performing heart biopsies, T2\* (T2 star), a Magnetic Resonance (MR) relaxation parameter expressed in milliseconds, as is an important tool to noninvasively quantify cardiac iron concentration. Studies have shown that myocardial T2\* evaluations may predict cardiac events, e.g., impaired (\<56%) left ventricular ejection fraction (LVEF) is prevalent among patients with low T2\*: found in 62% of patients with T2\*\<8 ms; 20% with T2\* of 8-12 ms; and in 5% with T2\* \>12 ms (Tanner 2006)
| Arm | Type | Description |
|---|---|---|
| Deferasirox | EXPERIMENTAL | 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day |
| Deferasirox Placebo | ACTIVE_COMPARATOR | 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week |
| Extension: deferoxamine to deferasirox | EXPERIMENTAL | "DFO to ICL" (patients who switched from DFO to deferasirox in extension) |
| Extension: deferasirox to deferoxamine | EXPERIMENTAL | "ICL to DFO" (patients who switched from deferasirox to DFO in extension) |
| Name | Type | Description |
|---|---|---|
| Core Study: Deferasirox | DRUG | 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day |
| Core Study: Deferoxamine | DRUG | 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week |
| Extension: deferoxamine to deferasirox | DRUG | 40 mg/kg deferasirox once daily administered 30 minutes before taking food. |
| Extension: deferasirox to deferoxamine | DRUG | DFO at a target range of 50 mg/kg/day to 60 mg/kg/day via subcutaneous (sc) infusion lasting a period of 8 to 12 hrs administered for 5 to 7 days per week, |
| Deferasirox | DRUG | - |
| Deferoxamine | DRUG | - |
Inclusion criteria: * Male or female patients, aged 10 years and above, with β-thalassemia major or DBA or sideroblastic anemia on chronic transfusion therapy, having given written consent to participate in the study. * Patients with cardiac iron as measured by a myocardial T2\* value that is ≥ 6ms...