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.

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).

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.