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

NCT04968379 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: 1VIT19046
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 4

Brief Summary

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.

Conditions

Iron Deficiency, Anaemia

Outcome Measures

Primary Outcomes (7)

• 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

Study Arms (2)

Ferric Carboxymaltose

EXPERIMENTAL

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

Injectafer

EXPERIMENTAL

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

Interventions

Ferric carboxymaltose DRUG

Intravenous

Also known as: Injectafer

Ferric Carboxymaltose; Injectafer

Eligibility Criteria

• Age: Up to 1 Year
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).
• Screening Hb ≥7 g/dL to \<10 g/dL.
• 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

You may not qualify if:

• Known history of hypersensitivity reaction to FCM.
• Body weight \<2.5 kg.
• History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
• Hemodialysis-dependent chronic kidney disease.
• 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.
• Active infection.
• Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy vitamin B12 deficiency, or folic acid deficiency).
• Blood transfusion in the 4 weeks prior to consent.
• Administration of an iron-containing product within 14 days of administration of the study article.
• Administration and / or use of an investigational product (drug or device) within 30 days of screening.
• Current participation in another clinical trial.
• Unable to comply with study procedures and assessments.

Sponsors & Collaborators

• American Regent, Inc.: lead

Study Sites (4)

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Cohen Children's Medical Center

New Hyde Park, New York, 11040, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

St. Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States

Location

MeSH Terms

Conditions

Anemia, Iron-Deficiency

Interventions

ferric carboxymaltose

Condition Hierarchy (Ancestors)

Anemia, Hypochromic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Iron Deficiencies; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Mark Falone, MD

  American Regent

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2020
• First Posted: July 20, 2021
• Study Start: July 21, 2022
• Primary Completion: October 8, 2024
• Study Completion: December 12, 2024
• Last Updated: January 6, 2023

Record last verified: 2023-01

Locations

US (4)