A Study to Evaluate Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects

Subjects will be randomized to treatment in a 2:1 ratio (ferumoxytol: iron sucrose) and stratified by age group (2 to <6 years; 6 to <12 years; and 12 to <18 years). Subjects will receive one of the following treatment regimens:

• Ferumoxytol: 7 mg Fe/kg IV (maximum 510 mg/dose) x 2 doses, the first dose administered on Day 1 and the second 2 to 8 days later.

OR

• Iron sucrose (Venofer®): 4 mg Fe/kg IV (maximum 200 mg/dose) x 5 doses, the first dose on Day 1 and subsequent doses administered at least once per week and up to 3 times/week. All subjects will be monitored at the study site through at least 1 hour after the completion of each infusion of study drug. Assessment of blood Hgb concentrations, adverse events, and other safety assessments will be performed through study Week 5.

Inclusion criteria

• Male or female 2 years to <18 years of age at time of consent
• Has IDA defined as:

• Hemoglobin (Hgb) <11.0 g/dL AND
• Any one or more of the following:

• Transferrin saturation (TSAT) <20%
• ferritin <100 ng/mL
• Documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate

Exclusion criteria

• Known hypersensitivity reaction to any component of ferumoxytol or iron sucrose
• History of allergy to intravenous (IV) iron
• History of ≥2 clinically significant drug allergies
• Subjects with CKD (defined as eGFR of <60 mL/min/1.73 m2 or a requirement for chronic hemodialysis or peritoneal dialysis during Screening)
• Low systolic blood pressure (BP) (age 1 to 9 years <70 + [age in years x 2] mmHg, age 10 to 17 years <90 mmHg)
• Hgb ≤7.0 g/dL
• Serum ferritin level >600 ng/mL