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

Phase 3

Recruiting

• Conditions: Iron Deficiency Anemia
• Interventions: Drug: ferumoxytol; Drug: Iron sucrose

• Registration Number: NCT03893045
• Lead Sponsor: AMAG Pharmaceuticals, Inc.

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.

Study Timeline

• Study First Submitted: 2019-03-22
• Study First Submitted QC: 2019-03-26
• Study First Posted: 2019-03-28
• Study Start: 2019-09-18
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-09
• Last Update Posted: 2024-08-12
• Primary Completion: 2025-07
• Study Completion: 2025-10

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 75

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

Read More

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

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Ferumoxytol	ferumoxytol	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.
Iron sucrose	Iron 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)

Primary Outcome Measures

Name	Time	Method
Change in Hemoglobin from Baseline to Week 5	35 days	Proportion of subjects achieving a change in hemoglobin from Baseline to Week 5

Secondary Outcome Measures

Name	Time	Method
Incidence of adverse events of special interest (AESI)	49 days	Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity)
Incidence of Treatment Emergent Adverse Events	49 days	Incidence of Treatment Emergent Adverse Events

Trial Locations

Locations (15)

Biomedical Research LLC; 🇺🇸 Miami, Florida, United States

Gwinnett Research Institute; 🇺🇸 Buford, Georgia, United States

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu; 🇵🇱 Wrocław, Poland

Pro Familia Altera Sp. z o.o.; 🇵🇱 Katowice, Poland

Centrum Zdrowia MDM; 🇵🇱 Warsaw, Poland

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

Prywatny Gabinet Lekarski Dr N. med. Jerzy Brzostek; 🇵🇱 Debica, Poland

JSC Saules seimos medicinos centras; 🇱🇹 Kaunas, Lithuania

Klaipeda Children's Hospital; 🇱🇹 Klaipėda, Lithuania

Children's Hospital-Affiliate of Vilnius University Hospital Santariskiu Klinikos; 🇱🇹 Vilnius, Lithuania

Korczowski Bartosz, Gabinet Lekarski; 🇵🇱 Rzeszów, Poland

Sun Research Institute; 🇺🇸 San Antonio, Texas, United States

Optimus U Corporation; 🇺🇸 Miami, Florida, United States

Osrodek Badan Klinicznych In Vivo sp. z o.o.; 🇵🇱 Bydgoszcz, Poland

University of Florida; 🇺🇸 Gainesville, Florida, United States