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)
- Conditions
- Chronic Kidney Disease; Iron Deficiency Anemia
- Interventions
- Registration Number
- NCT03619850
- Lead Sponsor
- AMAG Pharmaceuticals, Inc.
- Brief Summary
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).
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 129
-
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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Ferumoxytol Ferumoxytol - Iron sucrose Iron sucrose -
- Primary Outcome Measures
Name Time Method Hemoglobin change of at least 0.5 g/dL from Baseline to Week 5 35 days Proportion of patients achieving a hemoglobin change of at least 0.5 g/dL during the period 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 (21)
Memorial Healthcare System
🇺🇸Hollywood, Florida, United States
West Virginia University
🇺🇸Morgantown, West Virginia, United States
Polish Mother's Memorial Hospital Research Institute
🇵🇱Łódź, Poland
The Feinstein Institute Medical Research Organization of Northwell Health, Inc.
🇺🇸Lake Success, New York, United States
Akron Nephrology Associates, Inc.
🇺🇸Akron, Ohio, United States
Instituto Mexicano De Trasplantes S.C
🇲🇽Cuernavaca, Mexico
Uniwersytecki Szpital Kliniczny Im. Zamenhofa w Bialystoku
🇵🇱Białystok, Poland
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
🇺🇸New York, New York, United States
Wayne State University
🇺🇸Detroit, Michigan, United States
JM Research, SC
🇲🇽Cuernavaca, Mexico
University Children Hospital
🇵🇱Kraków, Poland
Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK) - I. sz. Gyermekgyogyaszati Klinika
🇭🇺Budapest, Hungary
Klaipeda Children's Hospital
🇱🇹Klaipėda, Lithuania
Specjalistyczne Gabinety Sp. z o.o
🇵🇱Kraków, Poland
University Of Szeged
🇭🇺Szeged, Hungary
Hospital Civil de Guadalajara Fray Antonio Alcalde
🇲🇽Guadalajara, Mexico
Hospital of Lithuanian University of Health Sciences Kauno klinikos
🇱🇹Kaunas, Lithuania
Vsl Vilniaus Universiteto Vaiku Ligonine (VUVL) (Vilnius University Children's Hospital)
🇱🇹Vilnius, Lithuania
Instituto Nacional de Pediatria (INP) (National Institute of Pediatrics)
🇲🇽Mexico City, Mexico
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)
🇺🇸Bronx, New York, United States