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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Iron Deficiency Anemia

Intervention / Treatment
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* Drug: ferumoxytol
* Drug: Iron sucrose


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ferumoxytol<br>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. | Drug: ferumoxytol<br>* 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<br>* Other names: Feraheme;|
| Active Comparator: Iron sucrose<br>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) | Drug: Iron sucrose<br>* 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).<br>* Other names: Venofer;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Hemoglobin from Baseline to Week 5 | Proportion of subjects achieving a change in hemoglobin from Baseline to Week 5 | 35 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment Emergent Adverse Events | Incidence of Treatment Emergent Adverse Events | 49 days | 
| Incidence of adverse events of special interest (AESI) | Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity) | 49 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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IDA, anemia, pediatrics, ferumoxytol, iron sucrose