Safety, Tolerability, PK and PD of Intravenous Ferric Carboxymaltose in Infants With Iron Deficiency Anemia

An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 Year) With Iron Deficiency Anemia

An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 year) with Iron Deficiency Anemia.

Study Overview

• Status: Withdrawn
• Conditions: Iron Deficiency, Anaemia
• Intervention / Treatment: Drug: Ferric carboxymaltose

Detailed Description

A phase II, open-label, multi-center study with 2 Cohorts to evaluate the safety, tolerance, PK, and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving either a 5.0 mg/kg or 7.5 mg/kg dose of FCM.

Participants will have a screening evaluation within 14 days of the first dose of study drug. A medically supervised environment is required on Day 1 (day of dosing) and for 4 hours post dosing. Participants are allowed to be enrolled if satisfying the inclusion and exclusion criteria. Participants will return to the study site for additional evaluation and sampling on Days 8 (± 2 days), 15 (± 2 days), 22 (± 2 days), and 36 (± 2 days).

• Study Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa
  • New York
    • New Hyde Park, New York, United States, 11040
      • Cohen Children's Medical Center
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19134
      • St. Christopher's Hospital for Children

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 1 year (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Male and female participants 0 to 1 year of age, medically indicated for iron replacement, with his/her parent or legal guardian willing and able to sign the informed consent form approved by the IRB / Independent Ethics Committee (IEC).
2. Screening Hb ≥7 g/dL to <10 g/dL.

3. Infants with any of the following conditions:

   • Heart failure with IDA defined as syndromes of excessive preload, excessive afterload, abnormal rhythm, or decreased contractility
   • Gastrointestinal diseases with acquired short bowel syndrome (due to volvulus, necrotizing enterocolitis from surgical resection or spontaneous intestinal perforation)
   • Gastrointestinal intolerance of oral iron or an unsatisfactory response to oral iron
   • Other conditions associated with IDA which in the opinion of the investigator might benefit from administration of FCM

Exclusion Criteria:

1. Known history of hypersensitivity reaction to FCM.
2. Body weight <2.5 kg.
3. History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
4. Hemodialysis-dependent chronic kidney disease.
5. History of significant diseases of the liver, hematopoietic system, cardiovascular system, or other conditions which, on the opinion of the investigator, may place a participant at added risk for participation in the study.
6. Active infection.
7. Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy vitamin B12 deficiency, or folic acid deficiency).
8. Blood transfusion in the 4 weeks prior to consent.
9. Administration of an iron-containing product within 14 days of administration of the study article.
10. Administration and / or use of an investigational product (drug or device) within 30 days of screening.
11. Current participation in another clinical trial.
12. Unable to comply with study procedures and assessments.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ferric Carboxymaltose; To evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 5.0 mg/kg dose of FCM	Drug: Ferric carboxymaltose; Intravenous; Other Names: Injectafer
Experimental: Injectafer; To evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 7.5 mg/kg dose dose of FCM.	Drug: Ferric carboxymaltose; Intravenous; Other Names: Injectafer

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment-emergent adverse events	Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities	Baseline to Day 36
Change in hemoglobin (Hb): g/dL	determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters	baseline to Days 8, 15, 22, and 36
Change in reticulocytes count: %	determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters	baseline to Days 8, 15, 22, and 36
Evaluate the PD parameters - Change in serum iron: mcg/dL	To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.	baseline to Day 36
Evaluate the PD parameters - Change in serum ferritin: ng/mL	Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.	baseline to Day 36
Evaluate the PD parameters - Change in total iron binding capacity [TIBC]): mcg/dL	Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.	baseline to Day 36
Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL	Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.	baseline to Day 36

Collaborators and Investigators

• Sponsor: American Regent, Inc.
• Investigators: Study Director: Mark Falone, MD, American Regent

Study record dates

Study Major Dates

• Study Start (Actual): July 21, 2022
• Primary Completion (Anticipated): October 8, 2024
• Study Completion (Anticipated): December 12, 2024

Study Registration Dates

• First Submitted: November 2, 2020
• First Submitted That Met QC Criteria: July 8, 2021
• First Posted (Actual): July 20, 2021

Study Record Updates

• Last Update Posted (Estimate): January 6, 2023
• Last Update Submitted That Met QC Criteria: January 5, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Anemia, Hypochromic; Iron Metabolism Disorders; Anemia, Iron-Deficiency; Anemia
• Other Study ID Numbers: 1VIT19046

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No