Conditions: Anemia Hemosiderosis
Keywords: β-thalassemia rare chronic anemia iron overload deferasirox chronic anemias transfusional hemosiderosis
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 184
Healthy volunteers: no

Brief summary:
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Patients who successfully completed the main trial may continue in the extension trial to receive chelation therapy with deferasirox for up to 3 years. Extension was prolonged to 4 years.

The objective of this study is to assess the long-term safety and efficacy of deferasirox in these patient groups.

Detailed description:

Arms:
arm 1: Deferasirox daily oral dose between 5-40 mg/kg/day

Interventions: intervention 1: Deferasirox
Intervention descriptions: intervention 1: Deferasirox available as 125 mg, 250 mg or 500 mg tablets

Protocol document: