A Study to Evaluate Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects

A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects

This is a Phase 3, randomized, open-label, multicenter, study in male and female pediatric subjects (2 years to <18 years of age) with IDA, or felt by their clinician to be at risk of developing IDA. This study allows for enrollment of subjects with IDA regardless of etiology, except for CKD subjects (pediatric CKD subjects are being studied in a separate ferumoxytol protocol).

Study Overview

• Status: Recruiting
• Conditions: Iron Deficiency Anemia
• Intervention / Treatment: Drug: ferumoxytol; Drug: Iron sucrose

Detailed Description

Subjects will be randomized to treatment in a 2:1 ratio (ferumoxytol: iron sucrose) and stratified by age group (2 to <6 years; 6 to <12 years; and 12 to <18 years). Subjects will receive one of the following treatment regimens:

• Ferumoxytol: 7 mg Fe/kg IV (maximum 510 mg/dose) x 2 doses, the first dose administered on Day 1 and the second 2 to 8 days later.

OR

• Iron sucrose (Venofer®): 4 mg Fe/kg IV (maximum 200 mg/dose) x 5 doses, the first dose on Day 1 and subsequent doses administered at least once per week and up to 3 times/week. All subjects will be monitored at the study site through at least 1 hour after the completion of each infusion of study drug. Assessment of blood Hgb concentrations, adverse events, and other safety assessments will be performed through study Week 5.

• Study Type: Interventional
• Enrollment (Estimated): 75
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Clinical Trial Interest; Phone Number: 1-877-374 -4177; Email: CTInterest@covispharma.com

Study Locations

• Lithuania
  • Kaunas, Lithuania
    • Not yet recruiting
    • JSC Saules seimos medicinos centras
  • Klaipėda, Lithuania
    • Not yet recruiting
    • Klaipeda Children's Hospital
  • Vilnius, Lithuania
    • Not yet recruiting
    • Children's Hospital-Affiliate of Vilnius University Hospital Santariskiu Klinikos
• Poland
  • Bydgoszcz, Poland
    • Not yet recruiting
    • Osrodek Badan Klinicznych In Vivo sp. z o.o.
  • Debica, Poland
    • Not yet recruiting
    • Prywatny Gabinet Lekarski dr n. med. Jerzy Brzostek
  • Katowice, Poland
    • Not yet recruiting
    • Pro Familia Altera Sp. Z O.O.
  • Rzeszów, Poland
    • Not yet recruiting
    • Korczowski Bartosz, Gabinet Lekarski
  • Warsaw, Poland
    • Not yet recruiting
    • Centrum Zdrowia MDM
  • Wrocław, Poland
    • Not yet recruiting
    • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Not yet recruiting
      • Arkansas Children's Hospital
  • Florida
    • Gainesville, Florida, United States, 32608
      • Not yet recruiting
      • University of Florida
    • Miami, Florida, United States, 33125
      • Recruiting
      • Optimus U Corporation
    • Miami, Florida, United States, 33184
      • Recruiting
      • Biomedical Research LLC
  • Georgia
    • Buford, Georgia, United States, 30519
      • Not yet recruiting
      • Gwinnett Research Institute
  • Texas
    • San Antonio, Texas, United States, 78215
      • Recruiting
      • Sun Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Male or female 2 years to <18 years of age at time of consent

2. Has IDA defined as:

   1. Hemoglobin (Hgb) <11.0 g/dL AND

   2. Any one or more of the following:

      • Transferrin saturation (TSAT) <20%
      • ferritin <100 ng/mL
3. Documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate

Exclusion Criteria:

1. Known hypersensitivity reaction to any component of ferumoxytol or iron sucrose
2. History of allergy to intravenous (IV) iron
3. History of ≥2 clinically significant drug allergies
4. Subjects with CKD (defined as eGFR of <60 mL/min/1.73 m2 or a requirement for chronic hemodialysis or peritoneal dialysis during Screening)
5. Low systolic blood pressure (BP) (age 1 to 9 years <70 + [age in years x 2] mmHg, age 10 to 17 years <90 mmHg)
6. Hgb ≤7.0 g/dL
7. Serum ferritin level >600 ng/mL

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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: 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; Other Names: Feraheme
Active Comparator: 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)	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).; Other Names: Venofer

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Hemoglobin from Baseline to Week 5	Proportion of subjects achieving a change in hemoglobin from Baseline to Week 5	35 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment Emergent Adverse Events	Incidence of Treatment Emergent Adverse Events	49 days
Incidence of adverse events of special interest (AESI)	Incidence of adverse events of special interest (AESI) (hypotension and hypersensitivity)	49 days

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Area Under the Curve (AUC)	Pharmacokinetic parameter: Area Under the Curve (AUC)	35 days
Clearance	Pharmacokinetic parameter: Clearance	35 days
Distribution	Pharmacokinetic parameter: Distribution	35 days
Elimination half-lives	Pharmacokinetic parameter: Elimination half-lives	35 days

Collaborators and Investigators

• Sponsor: AMAG Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): September 18, 2019
• Primary Completion (Estimated): July 1, 2024
• Study Completion (Estimated): October 1, 2024

Study Registration Dates

• First Submitted: March 22, 2019
• First Submitted That Met QC Criteria: March 26, 2019
• First Posted (Actual): March 28, 2019

Study Record Updates

• Last Update Posted (Actual): July 27, 2023
• Last Update Submitted That Met QC Criteria: July 25, 2023
• Last Verified: July 1, 2023

More Information

Terms related to this study

• Keywords: pediatrics; anemia; ferumoxytol; IDA; iron sucrose
• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Nutrition Disorders; Anemia, Hypochromic; Iron Metabolism Disorders; Malnutrition; Anemia, Iron-Deficiency; Anemia; Deficiency Diseases; Iron Deficiencies; Hematinics; Pharmaceutical Solutions; Parenteral Nutrition Solutions; Ferrosoferric Oxide; Ferric Oxide, Saccharated
• Other Study ID Numbers: AMAG-FER-IDA-352

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No