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

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)

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.

Study Overview

• Status: Completed
• Conditions: Iron Deficiency Anemia (IDA)
• Intervention / Treatment: Drug: Ferric Carboxymaltose (FCM)

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.

• Study Type: Interventional
• Enrollment (Actual): 35
• Phase: Phase 2

Contacts and Locations

Study Locations

• Poland
  • Bydgoszcz, Poland, 85-094
    • Szpital Uniwersytecki Katedra i Klinika Pediatrii, Hematologii i Onkologii
  • Dębica, Poland, 39-200
    • Zespół Opieki Zdrowotnej w Dębicy z siedzibą w Dębicy , Oddział Dziecięcy
  • Kraków, Poland, 30-663
    • Uniwersytecki Szpital Dziecięcy w Krakowie, Oddział Pediatrii i Gastroenterologii (V)
  • Lublin, Poland, 20-093
    • Klinika Hematologii, Onkologii i Transplantologii Dziecięcej Uniwersytecki Szpital Dziecięcy w Lublinie
  • Lublin, Poland, 20-093
    • Oddział Ogólnopediatryczny; Uniwersytecki Szpital Dziecięcy w Lublinie
  • Rzeszów, Poland, 35-302
    • Indywidualna Specjalistyczna Praktyka lekarska z siedzibą w Rzeszowie
  • Szczecin, Poland, 71-252
    • Klinika Pediatrii, Hematologii i Onkologii Dziecięcej
  • Warszawa, Poland, 04-730
    • Klinika Gastroenterologii, Hepatologii, Zaburzeń Odżywiania i Pediatrii
• Russian Federation
  • Ryazan', Russian Federation, 390029
    • 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
  • Saint Petersburg, Russian Federation, 194100
    • State Educational Institution of Higher Professional Education Saint Petersburg State Pediatric Medical Acamy of Ministry of Health and Social Development of the Russian Federation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SEQUENTIAL
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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	Drug: Ferric Carboxymaltose (FCM); Other Names: Injectafer

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: American Regent, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 19, 2015
• Primary Completion (Actual): January 22, 2017
• Study Completion (Actual): June 1, 2017

Study Registration Dates

• First Submitted: March 27, 2015
• First Submitted That Met QC Criteria: April 1, 2015
• First Posted (Estimate): April 7, 2015

Study Record Updates

• Last Update Posted (Actual): July 25, 2022
• Last Update Submitted That Met QC Criteria: July 21, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Anemia, Hypochromic; Iron Metabolism Disorders; Anemia, Iron-Deficiency; Anemia
• Other Study ID Numbers: 1VIT13036