Stopped: This study was terminated due to treatment stop resulting in an inability to draw conclusions from the data. Evaluation of nonclinical rat findings is ongoing.
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).
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Maximum Observed Plasma Concentration (Cmax) of SPD602 After a Single Oral Dose
Timeframe: Day 1 and up to 24 hours post-dose
Time of Maximum Observed Plasma Concentration Sampled During a Dosing Interval (Tmax) of SPD602 After a Single Oral Dose
Timeframe: Day 1 and up to 24 hours post-dose
Area Under The Plasma Concentration-Time Curve (AUC) From The Time of Dosing to The Last Measurable Concentration (AUClast) of SPD602 After a Single Oral Dose
Timeframe: Day 1 and up to 24 hours post-dose
Terminal Half-life (t1/2) of SPD602 After a Single Oral Dose
Timeframe: Day 1 and up to 24 hours post-dose
Renal Clearance (CLr) of SPD602 After a Single Oral Dose
Timeframe: Day 1 and up to 24 hours post-dose
Amount Excreted Into Urine (Ue) of SPD602 After a Single Oral Dose
Timeframe: Day 1 and up to 24 hours post-dose
Fraction Of Orally Administered Drug Excreted Unchanged In Urine (fe) of SPD602 After a Single Oral Dose
Timeframe: Day 1 and up to 24 hours post-dose
Change From Baseline in Liver Iron Concentration (LIC) Assessed by FerriScan R2 Magnetic Resonance Imaging (MRI)
Timeframe: Baseline, 24 weeks, and 48 weeks
Change From Baseline in LIC Adjusted by Transfusional Iron Intake And Assessed by FerriScan R2 MRI
Timeframe: Baseline, 24 weeks, and 48 weeks