A Study to Characterize the PK and PD Profile of IV FCM in Pediatric Subjects 1-17 Years Old With IDA
Condition: Iron Deficiency Anemia (IDA)
MeSH terms: Anemia; Anemia, Iron-Deficiency; Iron Deficiencies
Intervention: Ferric Carboxymaltose (FCM)
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Sequential Assignment
Intervention 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)
Enrollment: 35.0
Sex: All
Minimum age: 1 Year
Maximum age: 17 Years
Healthy volunteers: No

Summary:

      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.
    

Eligibility criteria:

        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.