Conditions: Sickle Cell Disease
Keywords: Deferasirox ICL670A Iron chelators Sickle Cell Disease Transfusional Hemosiderosis
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 185
Healthy volunteers: no

Brief summary:
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.

Detailed description:
The treatment period started once the patient completed the core study and signed informed consent. It is continued for up to 4 years. Safety parameters were assessed every 4 weeks. Eye and Ear examinations were performed on a yearly basis. To further investigate the extent of iron overload, serum ferritin, iron, and transferrin were monitored every four weeks. The Program Safety Board monitored the safety of ICL670 during the study to evaluate and categorize any serious case reported in association with ICL670.

Arms:
arm 1: None

Interventions: intervention 1: ICL670
Intervention descriptions: intervention 1: Daily doses of ICL670 were taken orally 30 minutes before breakfast. The doses range from 5-40 mg/kg and were determined based on the patient's trend in serum ferritin over time during the core study (0109) and on the frequency of blood transfusions the patient received. The treatment duration was up to 4 years.

Protocol document: