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

Phase 2
Completed
Conditions
Iron Deficiency Anemia (IDA)
Interventions
Drug: Ferric Carboxymaltose (FCM)
Registration Number
NCT02410213
Lead Sponsor
American Regent, Inc.
Brief 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.

Detailed Description

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
35
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Ferric Carboxymaltose (FCM)Ferric Carboxymaltose (FCM)FCM at 7.5 mg/kg or 15 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller
Primary Outcome Measures
NameTimeMethod
Maximum Serum Concentration (Cmax)prior to dosing and 1, 2, 6, 12, 48 and 72 hours post dosing

Maximum observed serum concentration; obtained directly from the serum concentration-time profile.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (10)

Indywidualna Specjalistyczna Praktyka lekarska z siedzibą w Rzeszowie

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Rzeszów, Poland

Uniwersytecki Szpital Dziecięcy w Krakowie, Oddział Pediatrii i Gastroenterologii (V)

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Kraków, Poland

State Budgetary Educational Institution of Higher Professional Education "Ryazan State Medical University named after academician I.P. Pavlov" of the Ministry of Health of the Russian Federation

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Ryazan', Russian Federation

Szpital Uniwersytecki Katedra i Klinika Pediatrii, Hematologii i Onkologii

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Bydgoszcz, Poland

Zespół Opieki Zdrowotnej w Dębicy z siedzibą w Dębicy , Oddział Dziecięcy

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Dębica, Poland

Klinika Hematologii, Onkologii i Transplantologii Dziecięcej Uniwersytecki Szpital Dziecięcy w Lublinie

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Lublin, Poland

Klinika Pediatrii, Hematologii i Onkologii Dziecięcej

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Szczecin, Poland

State Educational Institution of Higher Professional Education Saint Petersburg State Pediatric Medical Acamy of Ministry of Health and Social Development of the Russian Federation

🇷🇺

Saint Petersburg, Russian Federation

Oddział Ogólnopediatryczny; Uniwersytecki Szpital Dziecięcy w Lublinie

🇵🇱

Lublin, Poland

Klinika Gastroenterologii, Hepatologii, Zaburzeń Odżywiania i Pediatrii

🇵🇱

Warszawa, Poland

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