Conditions: Transfusional Iron Overload β-thalassemia Major Pediatric Rare Anemia
Keywords: β-thalassemia major iron overload deferasirox pediatric rare anemia
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 40
Healthy volunteers: no

Brief summary:
In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation therapy with deferasirox for up to 4 years.

Detailed description:

Arms:
arm 1: Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.

Interventions: intervention 1: Deferasirox
Intervention descriptions: intervention 1: Deferasirox in children from 1 to 18 years old was given orally once daily, 30 minutes prior to breakfast. An initial daily dose of 10 mg/kg was used during the 1-year core study. In this 4-year extension study dose modifications of ± 5 or 10 mg/kg were based on safety parameters and on increasing or decreasing Liver Iron Concentration (LIC), and serum ferritin. Deferasirox was available as 125 mg, 250 mg and 500 mg tablets.

Protocol document: