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NCT03619850PHASE3Recruiting

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)

AMAG Pharmaceuticals, Inc.

Start Date

8/13/2018

Completion Date

8/31/2026

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).

Eligibility Criteria

Age Range: 2 years to 17 years

Inclusion Criteria: 1. Male or female 2 years to \<18 years of age at time of consent 2. 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 3. Has Chronic Kidney Disease defined as one of the following: 1. on chronic hemodialysis; 2. receiving chronic peritoneal dialysis; 3. estimated glomerular filtration rate (eGFR) of \<60 mL/min/1.73 m2; 4. 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. 4. 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 5. 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: 1. Known hypersensitivity reaction to any component of ferumoxytol and iron sucrose 2. History of allergy to intravenous (IV) iron 3. History of multiple drug allergies (\>2) 4. Low systolic blood pressure (Age 1-9 years \<70 + \[age in years x 2\] mmHg, Age 10-17 years \<90 mmHg) 5. Hemoglobin ≤7.0 g/dL 6. Serum ferritin level \>600 ng/mL

Interventions

DRUG

Ferumoxytol

DRUG

Iron sucrose

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Conditions

Chronic Kidney Disease; Iron Deficiency Anemia

Locations

Memorial Healthcare System

Hollywood, Florida 33021

United States

Wayne State University

Detroit, Michigan 48201

United States

Children's Mercy Hospital

Kansas City, Missouri 64108

United States

The Feinstein Institute Medical Research Organization of Northwell Health, Inc.

Lake Success, New York 11042

United States

Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)

New York, New York 10029

United States

Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)

The Bronx, New York 10467

United States

Akron Nephrology Associates, Inc.

Akron, Ohio 44302

United States

West Virginia University

Morgantown, West Virginia 26506

United States

Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK) - I. sz. Gyermekgyogyaszati Klinika

Budapest,

Hungary

University Of Szeged

Szeged,

Hungary

Hospital of Lithuanian University of Health Sciences Kauno klinikos

Kaunas,

Lithuania

Klaipeda Children's Hospital

Klaipėda,

Lithuania

Vsl Vilniaus Universiteto Vaiku Ligonine (VUVL) (Vilnius University Children's Hospital)

Vilnius,

Lithuania

Instituto Mexicano De Trasplantes S.C

Cuernavaca,

Mexico

JM Research, SC

Cuernavaca,

Mexico

Hospital Civil de Guadalajara Fray Antonio Alcalde

Guadalajara,

Mexico

Instituto Nacional de Pediatria (INP) (National Institute of Pediatrics)

Mexico City,

Mexico

Uniwersytecki Szpital Kliniczny Im. Zamenhofa w Bialystoku

Bialystok,

Poland

Specjalistyczne Gabinety Sp. z o.o

Krakow,

Poland

University Children Hospital

Krakow,

Poland

Polish Mother's Memorial Hospital Research Institute

Lodz,

Poland