A Study to Evaluate Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects
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
1 other identifier
interventional
75
3 countries
15
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Sep 2019
Longer than P75 for phase_3
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 22, 2019
CompletedFirst Posted
Study publicly available on registry
March 28, 2019
CompletedStudy Start
First participant enrolled
September 18, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 1, 2026
April 17, 2026
April 1, 2026
6.8 years
March 22, 2019
April 14, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change in Hemoglobin from Baseline to Week 5
Proportion of subjects achieving a change in hemoglobin from Baseline to Week 5
35 days
Secondary Outcomes (2)
Incidence of Treatment Emergent Adverse Events
49 days
Incidence of adverse events of special interest (AESI)
49 days
Other Outcomes (4)
Area Under the Curve (AUC)
35 days
Clearance
35 days
Distribution
35 days
- +1 more other outcomes
Study Arms (2)
Ferumoxytol
EXPERIMENTALEach 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
ACTIVE COMPARATOREach 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)
Interventions
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
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).
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (15)
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
University of Florida
Gainesville, Florida, 32608, United States
Optimus U Corporation
Miami, Florida, 33125, United States
Biomedical Research LLC
Miami, Florida, 33184, United States
Gwinnett Research Institute
Buford, Georgia, 30519, United States
Sun Research Institute
San Antonio, Texas, 78215, United States
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
Osrodek Badan Klinicznych In Vivo sp. z o.o.
Bydgoszcz, Poland
Prywatny Gabinet Lekarski Dr N. med. Jerzy Brzostek
Dębica, Poland
Pro Familia Altera Sp. z o.o.
Katowice, Poland
Korczowski Bartosz, Gabinet Lekarski
Rzeszów, Poland
Centrum Zdrowia MDM
Warsaw, Poland
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
Wroclaw, Poland
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 22, 2019
First Posted
March 28, 2019
Study Start
September 18, 2019
Primary Completion (Estimated)
July 1, 2026
Study Completion (Estimated)
October 1, 2026
Last Updated
April 17, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will not share