A Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Ferumoxytol, Iron sucrose.
- Who it may be relevant to
- Registry conditions: Chronic Kidney Disease; Iron Deficiency Anemia. Basic parameters: 2 years — 17 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Hungary, Lithuania, Mexico, Poland
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
Overview
Primary Objectives: To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA Secondary Objective: To determine the single-dose pharmacokinetics (PK) and pharmacodynamics (PD) profile of ferumoxytol in pediatric subjects.
Detailed description
This is a Phase 3, randomized, open-label, multicenter, study of the safety (compared to iron sucrose), efficacy, and PK/PD of ferumoxytol (7.0 mg Fe/kg x 2 \[max 510 mg/dose\]) in pediatric subjects with iron deficiency anemia (IDA) and CKD. There will be a total of approximately 125 subjects randomized to treatment in a 2:1 ratio to either ferumoxytol or iron sucrose.
Total subject participation in the study will be up to 7 weeks, which includes a 2-week Screening Period and a 5-week Treatment Period.
Subjects receive the following:
• Two IV infusions of ferumoxytol 7.0 mg Fe/kg (max 510 mg/dose), the first administered on Day 1 and the second 2-8 days later
OR
• Iron sucrose (Venofer®): For Hemodialysis Dependent (HDD) patients: 2 mg Fe/kg, administered on consecutive dialysis sessions, for 10 doses (max 100mg/dose with a total max treatment course of 1000mg) For Non-hemodialysis Dependent (NDD) or Peritoneal Dialysis De pendent(PDD) patients: 4 mg Fe/kg, administered up to 3 times/week, for 5 doses (max 200mg/dose with a total max treatment course of 1000mg).
Interventions
- Drug 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. - Drug 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
- Hemoglobin change of at least 0.5 g/dL from Baseline to Week 5 [Time frame: 35 days]
Secondary outcome measures (2)
- Incidence of Treatment Emergent Adverse Events [Time frame: 49 days]
- Incidence of adverse events of special interest (AESI) [Time frame: 49 days]
Eligibility criteria
Inclusion criteria
- Male or female 2 years to <18 years of age at time of consent
- Has IDA defined as: a) hemoglobin <12.0 g/dL and b) with either transferrin saturation (TSAT) <40% or ferritin <100 ng/mL; or considered to be at risk of development of IDA, i.e., TSAT<20% with falling hemoglobin during the preceding 2 months and a history of hemoglobin <12 g/dL
- Has Chronic Kidney Disease defined as one of the following:
- on chronic hemodialysis;
- receiving chronic peritoneal dialysis;
- estimated glomerular filtration rate (eGFR) of <60 mL/min/1.73 m2;
- has evidence of structural and/or functional abnormalities e.g., persistent albuminuria, abnormal urine sediment, electrolyte and other abnormalities due to tubular disorders for > 3 months.
- For patients other than hemodialysis dependent CKD patients, documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate
- All subjects (female and male) of childbearing potential who are sexually active must be on an effective method of birth control for at least 1 month prior to Day 1 Dosing and agree to remain on birth control until completion of the study
Exclusion criteria
- Known hypersensitivity reaction to any component of ferumoxytol and iron sucrose
- History of allergy to intravenous (IV) iron
- History of multiple drug allergies (>2)
- Low systolic blood pressure (Age 1-9 years <70 + \[age in years x 2\] mmHg, Age 10-17 years <90 mmHg)
- Hemoglobin ≤7.0 g/dL
- Serum ferritin level >600 ng/mL
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 8 centers
- Memorial Healthcare System — Hollywood
- Wayne State University — Detroit
- Children's Mercy Hospital — Kansas City
- The Feinstein Institute Medical Research Organization of Northwell Health, Inc. — Lake Success
- Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) — New York
- Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM) — The Bronx
- Akron Nephrology Associates, Inc. — Akron
- West Virginia University — Morgantown
Mexico · 4 centers
- Instituto Mexicano De Trasplantes S.C — Cuernavaca
- JM Research, SC — Cuernavaca
- Hospital Civil de Guadalajara Fray Antonio Alcalde — Guadalajara
- Instituto Nacional de Pediatria (INP) (National Institute of Pediatrics) — Mexico City
Poland · 4 centers
- Uniwersytecki Szpital Kliniczny Im. Zamenhofa w Bialystoku — Bialystok
- Specjalistyczne Gabinety Sp. z o.o — Krakow
- University Children Hospital — Krakow
- Polish Mother's Memorial Hospital Research Institute — Lodz
Lithuania · 3 centers
- Hospital of Lithuanian University of Health Sciences Kauno klinikos — Kaunas
- Klaipeda Children's Hospital — Klaipėda
- Vsl Vilniaus Universiteto Vaiku Ligonine (VUVL) (Vilnius University Children's Hospital) — Vilnius
Hungary · 2 centers
- Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK) - I. sz. Gyermekgyogyaszati Kli — Budapest
- University Of Szeged — Szeged
Identifiers
NCT: NCT03619850 · AMAG-FER-CKD-354