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...
Deferasirox is a small molecule studied for transfusional iron overload, a condition in which repeated blood transfusions cause excess iron to accumulate in the body. It has been evaluated in a Phase 2 trial comparing it with deferoxamine in patients with cardiac iron overload, including transfusional hemosiderosis.
Deferasirox is being developed by Novartis AG, which trades under the ticker NVS. The company is the sponsor of the clinical program evaluating the drug in transfusional iron overload.
Deferasirox is in Phase 2 development for transfusional iron overload. It is investigational and has not been approved for this use. One Phase 2 trial has been completed, and no active trials are currently listed.
Deferasirox has been studied in the completed Phase 2 trial NCT00600938, titled Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload. The trial enrolled 197 participants across Canada, China, Cyprus, Egypt, Italy, Lebanon, Taiwan, Thailand, Turkey, the United Arab Emirates, and the United Kingdom.
The Phase 2 trial NCT00600938 was randomized and active-controlled, comparing deferasirox with deferoxamine in patients with transfusional iron overload and cardiac iron overload. It was not double-blind, enrolled 197 participants aged 10 years and older, and did not include healthy volunteers.