Clinical trial NCT03619850: 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)
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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Chronic Kidney Disease; Iron Deficiency Anemia
Keywords: Iron deficiency anemia (IDA)
chronic kidney disease (CKD)
pediatrics
ferumoxytol
iron sucrose
Feraheme
Venofer
Lead sponsor: AMAG Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2018-08-08
Enrollment: 129
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

Eligibility criteria: 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

Primary outcomes:
measure: Hemoglobin change of at least 0.5 g/dL from Baseline to Week 5
timeFrame: 35 days
Secondary outcomes:
measure: Incidence of Treatment Emergent Adverse Events
timeFrame: 49 days
measure: Incidence of adverse events of special interest (AESI)
timeFrame: 49 days
Other outcomes:
measure: Area Under the Curve (AUC)
timeFrame: 35 days
measure: Clearance
timeFrame: 35 days
measure: Distribution and elimination half-lives
timeFrame: 35 days
Locations:
city: Hollywood
country: United States
facility: Memorial Healthcare System
state: Florida
city: Detroit
country: United States
facility: Wayne State University
state: Michigan
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Bronx
country: United States
facility: Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)
state: New York
city: Lake Success
country: United States
facility: The Feinstein Institute Medical Research Organization of Northwell Health, Inc.
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)
state: New York
city: Akron
country: United States
facility: Akron Nephrology Associates, Inc.
state: Ohio
city: Morgantown
country: United States
facility: West Virginia University
state: West Virginia
city: Budapest
country: Hungary
facility: Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK) - I. sz. Gyermekgyogyaszati Klinika
city: Szeged
country: Hungary
facility: University Of Szeged
city: Kaunas
country: Lithuania
facility: Hospital of Lithuanian University of Health Sciences Kauno klinikos
city: Klaipėda
country: Lithuania
facility: Klaipeda Children's Hospital
city: Vilnius
country: Lithuania
facility: Vsl Vilniaus Universiteto Vaiku Ligonine (VUVL) (Vilnius University Children's Hospital)
city: Cuernavaca
country: Mexico
facility: Instituto Mexicano De Trasplantes S.C
city: Cuernavaca
country: Mexico
facility: JM Research, SC
city: Guadalajara
country: Mexico
facility: Hospital Civil de Guadalajara Fray Antonio Alcalde
city: Mexico City
country: Mexico
facility: Instituto Nacional de Pediatria (INP) (National Institute of Pediatrics)
city: Białystok
country: Poland
facility: Uniwersytecki Szpital Kliniczny Im. Zamenhofa w Bialystoku
city: Kraków
country: Poland
facility: Specjalistyczne Gabinety Sp. z o.o
city: Kraków
country: Poland
facility: University Children Hospital
city: Łódź
country: Poland
facility: Polish Mother's Memorial Hospital Research Institute
Study officials:
References: