A Study to Characterize the PK and PD Profile of IV FCM in Pediatric Subjects 1-17 Years Old With IDA

Study Overview
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Brief Summary
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This is a Phase II, open-label, non-randomized, multi-center, single arm study to characterize the pharmacokinetic and pharmacodynamics (PK/PD) profile of Ferric Carboxymaltose dosing in pediatric subjects with IDA after receiving either a 7.5 mg/kg or 15 mg/kg dose of Ferric Carboxymaltose.

Detailed Description
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This is a Phase II, open-label, non-randomized, multi-center, single arm study to characterize the pharmacokinetic and pharmacodynamics (PK/PD) profile of Ferric Carboxymaltose dosing in pediatric subjects with IDA after receiving either a 7.5 mg/kg or 15 mg/kg dose of Ferric Carboxymaltose.

Official Title
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A Multi-center, Open-label, Single Arm Study to Characterize the Pharmacokinetics and Pharmacodynamics Profile of Intravenous Ferric Carboxymaltose in Pediatric Subjects 1-17 Years Old With Iron Deficiency Anemia (IDA)

Conditions
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Iron Deficiency Anemia (IDA)

Intervention / Treatment
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* Drug: Ferric Carboxymaltose (FCM)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female subjects 1 to 17 years of age with assent to participation and his/her parent or guardian is willing and able to sign the informed consent approved by the Independent Review Board / Ethics Committee. Screening TSAT < 20% Screening Hemoglobin < 11 g/dL For subjects who are receiving an erythropoietin stimulating agent (ESA): stable ESA therapy (+/- 20% of current dose) for > 8 weeks prior to the qualifying screening visit and no ESA dosing or product changes anticipated for the length of the trial Exclusion Criteria: Known hypersensitivity reaction to any component of Ferric Carboxymaltose. Subject previously randomized and treated in this study or any other clinical study of Ferric Carboxymaltose (FCM or VIT-45). Body mass index (BMI) ≤ 5th percentile for age (see APPENDIX 2) Male or Female subject 1 year of age weighing < 12kg. History of acquired iron overload, hemochromatosis or other iron accumulation disorders. Chronic kidney disease subjects on hemodialysis. Screening Ferritin level > 300ng/mL Subjects with significant severe diseases of the liver, hemopoietic system, cardiovascular system, psychiatric disorder or other conditions which on the opinion of the investigator may place a subject at added risk. Any active infection. Known positive hepatitis B antigen (HBsAg) or hepatitis C viral antibody (HCV) with evidence of active hepatitis. Known positive HIV-1/HIV-2 antibodies (anti-HIV). Anemia due to reasons other than iron deficiency (i.e. hemoglobinopathy). Subjects treated with vitamin B12 or folic acid deficiency are permitted. Intravenous iron and /or blood transfusion in the 4 weeks prior to screening. Immunosuppressive therapy that may lead to anemia (i.e. cyclophosphamide, azathioprine, mycophenolate mofetil). Note steroid therapy is permitted. Administration and / or use of an investigational product (drug or device) within 30 days of screening. Alcohol or drug abuse within the past six months. Female subjects who are pregnant or lactating, or sexually active female who are of childbearing potential not willing to use an acceptable form of contraceptive precautions during the study. Subject is unable to comply with study assessments.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 17 Years

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
Intervention Model: Sequential Assignment
Interventional Model Description: Eligible subjects were enrolled sequentially in Cohort 1 (FCM at 7.5 mg/kg with a maximum single dose of 750 mg) and Cohort 2 (FCM at 15 mg/kg with a maximum single dose of 750 mg). Enrollment in Cohort 2 was initiated only after all subjects in Cohort 1 completed 4 weeks of therapy and a Data and Safety Monitoring Board (DSMB) approved continuation.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ferric Carboxymaltose (FCM)<br>FCM at 7.5 mg/kg or 15 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller | Drug: Ferric Carboxymaltose (FCM)<br> <br> * Other names: Injectafer;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maximum Serum Concentration (Cmax) | Maximum observed serum concentration; obtained directly from the serum concentration-time profile. | prior to dosing and 1, 2, 6, 12, 48 and 72 hours post dosing |