Approval Probability
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Extension: deferoxamine to 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...
Deferoxamine to deferasirox is an investigational small molecule being studied for transfusional iron overload, a condition of excess iron accumulation from repeated blood transfusions. It is in Phase 2 clinical development and is being evaluated for its potential role in managing this hematologic condition.
Deferoxamine to deferasirox is being developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker NVS. The company is conducting clinical research on this asset for transfusional iron overload.
Deferoxamine to deferasirox is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still undergoing clinical trials to evaluate its safety and efficacy for transfusional iron overload.
Deferoxamine to deferasirox has one completed clinical trial, NCT00600938, titled 'Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload.' This Phase 2, randomized, active-controlled study enrolled 197 participants across multiple countries, including Canada, China, Italy, and the United Kingdom.
Deferoxamine to deferasirox is a small molecule designed to address transfusional iron overload. The clinical trial NCT00600938 compares deferasirox to deferoxamine, both of which are iron chelators that bind to excess iron in the body to facilitate its removal, though the specific molecular target of this combination is not detailed.
Deferoxamine to deferasirox is a combination or extension therapy involving two agents: deferoxamine and deferasirox. The clinical trial NCT00600938 evaluates deferasirox as a comparison to deferoxamine, indicating they are distinct treatments being studied for transfusional iron overload.