A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects
AMAG Pharmaceuticals, Inc.
Summary
This is a Phase 3, randomized, open-label, multicenter, study in male and female pediatric subjects (2 years to \<18 years of age) with IDA, or felt by their clinician to be at risk of developing IDA. This study allows for enrollment of subjects with IDA regardless of etiology, except for CKD subjects (pediatric CKD subjects are being studied in a separate ferumoxytol protocol).
Description
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 t…
Eligibility
- Age range
- 2–17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Male or female 2 years to \<18 years of age at time of consent 2. Has IDA defined as: 1. Hemoglobin (Hgb) \<11.0 g/dL AND 2. Any one or more of the following: * Transferrin saturation (TSAT) \<20% * ferritin \<100 ng/mL 3. 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: 1. Known hypersensitivity reaction to any component of ferumoxytol or iron sucrose 2. History of allergy to intravenous (IV) iron 3. History of ≥2 clinica…
Interventions
- Drugferumoxytol
Each 20 mL single-use vial contains 17 mL of ferumoxytol that consists of iron at a concentration of 30 mg Fe/mL, coated with polyglucose sorbitol carboxymethylether and formulated with mannitol, at a concentration of 44 mg/mL, in a black to reddish brown sterile, aqueous, colloidal, isotonic solution
- DrugIron sucrose
Each mL contains 20 mg of elemental iron as iron sucrose in water for injection. The 5 mL single-use vial contains 100 mg of iron per 5 mL. The drug product contains approximately 30% sucrose (300 mg/mL).
Locations (15)
- Arkansas Children's HospitalLittle Rock, Arkansas
- University of FloridaGainesville, Florida
- Optimus U CorporationMiami, Florida
- Biomedical Research LLCMiami, Florida
- Gwinnett Research InstituteBuford, Georgia
- Sun Research InstituteSan Antonio, Texas