Conditions: Thalassemia Sickle Cell Disease Diamond Blackfan Anemia Myelofibrosis
Keywords: Deferasirox Congenital Anemias Anemias Red Blood Cell Disorders Chronic Iron Overload Transfusional Iron Overload Iron Chelators Oral Iron Chelators Thalassemia Sickle Cell Disease Diamond Blackfan Anemia Myelofibrosis ICL670A
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 1683
Healthy volunteers: no

Brief summary:
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.

Detailed description:

Arms:
arm 1: Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Starting dose was determined by the frequency of blood transfusions and recommended initial daily dose of deferasirox is 20 mg/kg body weight for patients receiving blood transfusion, 10 mg/kg for patients receiving less frequent transfusion/exchange transfusion and 30 mg/kg for patients receiving more frequent blood transfusions.

Interventions: intervention 1: Deferasirox
Intervention descriptions: intervention 1: 125 mg, 250 mg and 500 mg tablets. Dosage was calculated based on participant's body weight. Tablets were dispersed in water, orange or apple juice and taken orally once a day.

Protocol document: