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

Study Overview
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Brief Summary
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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.

Detailed Description
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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).

Official Title
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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
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Iron Deficiency, Anaemia

Intervention / Treatment
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* Drug: Ferric carboxymaltose


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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.

Ages Eligible for Study
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Maximum Age: 1 Year

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ferric Carboxymaltose<br>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<br>* Intravenous<br>* Other names: Injectafer;|
| Experimental: Injectafer<br>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<br>* Intravenous<br>* Other names: Injectafer;|

What is the study measuring?
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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 |