Iron Deficiency Anemia
Conditions
Keywords
IDA, anemia, pediatrics, ferumoxytol, iron sucrose
Brief 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).
Detailed 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 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.
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).
Sponsors
AMAG Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
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 clinically significant drug allergies 4. Subjects with CKD (defined as eGFR of \<60 mL/min/1.73 m2 or a requirement for chronic hemodialysis or peritoneal dialysis during Screening) 5. Low systolic blood pressure (BP) (age 1 to 9 years \<70 + \[age in years x 2\] mmHg, age 10 to 17 years \<90 mmHg) 6. Hgb ≤7.0 g/dL 7. Serum ferritin level \>600 ng/mL
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Hemoglobin from Baseline to Week 5 | 35 days | Proportion of subjects achieving a change in hemoglobin from Baseline to Week 5 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment Emergent Adverse Events | 49 days | Incidence of Treatment Emergent Adverse Events |
| Incidence of adverse events of special interest (AESI) | 49 days | Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity) |
Countries
Lithuania, Poland, United States
Contacts
CONTACTClinical Trial Interest
Outcome results
None listed