Conditions: Transfusional Iron Overload Beta-Thalassemia
Keywords: Beta-Thalassemia Sickle Cell Anemia Transfusional iron overload Iron Overload Iron Chelation
Allocation: NON_RANDOMIZED | Intervention model: SINGLE_GROUP
Enrollment: 30
Healthy volunteers: no

Brief summary:
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the chronic dosing phase, during which patients will receive an additional 48 weeks of SSP-004184AQ dosing. Two age groups will be studied: 6-\<12, and 12-\<18 years old. The study is designed to initially assess the pharmacokinetics and safety of SSP-004184AQ in older children (adolescents, 12-\<18 years old) and then if deemed safe, in younger children (6-\<12 years old).

Detailed description:
Pharmacokinetic Phase: Patients will receive a single 16 mg/kg dose of SSP-004184AQ in capsule form.

Chronic Dosing Phase: Patients will receive SSP-004184AQ capsules daily for 48 weeks. Doses may range from 8-60 mg/kg/d.

Arms:
arm 1: Oral SSP-004184AQ taken once daily for 48 weeks arm 2: Oral SSP-004184AQ taken once daily for 48 weeks. Starting dose based on transfusion burden and iron overload status. Doses may range from 8-60mg/kg/day depending on clinical response. arm 3: A single dose given in the initial pharmacokinetic phase.

Interventions: intervention 1: SPD602
Intervention descriptions: intervention 1: None

Protocol document: