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)

Chronic Kidney Disease; Iron Deficiency Anemia Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03619850
• Other study ID #: AMAG-FER-CKD-354
• Status: Recruiting
• Phase: Phase 3
• Start date: August 13, 2018
• Est. completion date: August 31, 2023

Study information

• Verified date: July 2023
• Source: AMAG Pharmaceuticals, Inc.
• Contact: Clinical Trial Interest
• Phone: 1-877-374 -4177
• Email: CTInterest[@]covispharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 129
• Est. completion date: August 31, 2023
• Est. primary completion date: August 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Iron-Deficiency
• Chronic Kidney Disease; Iron Deficiency Anemia
• Deficiency Diseases
• Kidney Diseases
• Renal Insufficiency, Chronic

Intervention

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.
• 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).

Locations

Country	Name	City	State
Hungary	Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK) - I. sz. Gyermekgyogyaszati Klinika	Budapest
Hungary	University Of Szeged	Szeged
Lithuania	Hospital of Lithuanian University of Health Sciences Kauno klinikos	Kaunas
Lithuania	Klaipeda Children's Hospital	Klaipeda
Lithuania	Vsl Vilniaus Universiteto Vaiku Ligonine (VUVL) (Vilnius University Children's Hospital)	Vilnius
Mexico	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
Poland	Uniwersytecki Szpital Kliniczny Im. Zamenhofa w Bialystoku	Bialystok
Poland	Specjalistyczne Gabinety Sp. z o.o	Kraków
Poland	University Children Hospital	Kraków
Poland	Polish Mother's Memorial Hospital Research Institute	Lódz
United States	Akron Nephrology Associates, Inc.	Akron	Ohio
United States	Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)	Bronx	New York
United States	Wayne State University	Detroit	Michigan
United States	Memorial Healthcare System	Hollywood	Florida
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	The Feinstein Institute Medical Research Organization of Northwell Health, Inc.	Lake Success	New York
United States	West Virginia University	Morgantown	West Virginia
United States	Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH)	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
AMAG Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Hungary,  Lithuania,  Mexico,  Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Area Under the Curve (AUC)	Pharmacokinetic parameter: Area Under the Curve (AUC)	35 days
Other	Clearance	Pharmacokinetic parameter: clearance	35 days
Other	Distribution and elimination half-lives	Pharmacokinetic parameter: distribution and elimination half-lives	35 days
Primary	Hemoglobin change of at least 0.5 g/dL from Baseline to Week 5	Proportion of patients achieving a hemoglobin change of at least 0.5 g/dL during the period from Baseline to Week 5	35 days
Secondary	Incidence of Treatment Emergent Adverse Events	Incidence of Treatment Emergent Adverse Events	49 days
Secondary	Incidence of adverse events of special interest (AESI)	Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity)	49 days