Back to resultsA Study to Characterize the PK and PD Profile of IV FCM in Pediatric Subjects 1-17 Years Old With IDA
Primary Purpose
Iron Deficiency Anemia (IDA)
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Ferric Carboxymaltose (FCM)
Sponsored by
About this trial
This is an interventional treatment trial for Iron Deficiency Anemia (IDA)
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.
Sites / Locations
- Szpital Uniwersytecki Katedra i Klinika Pediatrii, Hematologii i Onkologii
- Zespół Opieki Zdrowotnej w Dębicy z siedzibą w Dębicy , Oddział Dziecięcy
- Uniwersytecki Szpital Dziecięcy w Krakowie, Oddział Pediatrii i Gastroenterologii (V)
- Klinika Hematologii, Onkologii i Transplantologii Dziecięcej Uniwersytecki Szpital Dziecięcy w Lublinie
- Oddział Ogólnopediatryczny; Uniwersytecki Szpital Dziecięcy w Lublinie
- Indywidualna Specjalistyczna Praktyka lekarska z siedzibą w Rzeszowie
- Klinika Pediatrii, Hematologii i Onkologii Dziecięcej
- Klinika Gastroenterologii, Hepatologii, Zaburzeń Odżywiania i Pediatrii
- 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
- State Educational Institution of Higher Professional Education Saint Petersburg State Pediatric Medical Acamy of Ministry of Health and Social Development of the Russian Federation
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Ferric Carboxymaltose (FCM)
Arm Description
FCM at 7.5 mg/kg or 15 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller
Outcomes
Primary Outcome Measures
Maximum Serum Concentration (Cmax)
Maximum observed serum concentration; obtained directly from the serum concentration-time profile.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02410213
Brief Title
A Study to Characterize the PK and PD Profile of IV FCM in Pediatric Subjects 1-17 Years Old With IDA
Official Title
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)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Completed
Study Start Date
February 19, 2015 (Actual)
Primary Completion Date
January 22, 2017 (Actual)
Study Completion Date
June 1, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
American Regent, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Deficiency Anemia (IDA)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
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)
Allocation
N/A
Enrollment
35 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Ferric Carboxymaltose (FCM)
Arm Type
Experimental
Arm Description
FCM at 7.5 mg/kg or 15 mg/kg to a maximum single dose of 750 mg iron, whichever is smaller
Intervention Type
Drug
Intervention Name(s)
Ferric Carboxymaltose (FCM)
Other Intervention Name(s)
Injectafer
Primary Outcome Measure Information:
Title
Maximum Serum Concentration (Cmax)
Description
Maximum observed serum concentration; obtained directly from the serum concentration-time profile.
Time Frame
prior to dosing and 1, 2, 6, 12, 48 and 72 hours post dosing
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
Szpital Uniwersytecki Katedra i Klinika Pediatrii, Hematologii i Onkologii
City
Bydgoszcz
ZIP/Postal Code
85-094
Country
Poland
Facility Name
Zespół Opieki Zdrowotnej w Dębicy z siedzibą w Dębicy , Oddział Dziecięcy
City
Dębica
ZIP/Postal Code
39-200
Country
Poland
Facility Name
Uniwersytecki Szpital Dziecięcy w Krakowie, Oddział Pediatrii i Gastroenterologii (V)
City
Kraków
ZIP/Postal Code
30-663
Country
Poland
Facility Name
Klinika Hematologii, Onkologii i Transplantologii Dziecięcej Uniwersytecki Szpital Dziecięcy w Lublinie
City
Lublin
ZIP/Postal Code
20-093
Country
Poland
Facility Name
Oddział Ogólnopediatryczny; Uniwersytecki Szpital Dziecięcy w Lublinie
City
Lublin
ZIP/Postal Code
20-093
Country
Poland
Facility Name
Indywidualna Specjalistyczna Praktyka lekarska z siedzibą w Rzeszowie
City
Rzeszów
ZIP/Postal Code
35-302
Country
Poland
Facility Name
Klinika Pediatrii, Hematologii i Onkologii Dziecięcej
City
Szczecin
ZIP/Postal Code
71-252
Country
Poland
Facility Name
Klinika Gastroenterologii, Hepatologii, Zaburzeń Odżywiania i Pediatrii
City
Warszawa
ZIP/Postal Code
04-730
Country
Poland
Facility Name
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
City
Ryazan'
ZIP/Postal Code
390029
Country
Russian Federation
Facility Name
State Educational Institution of Higher Professional Education Saint Petersburg State Pediatric Medical Acamy of Ministry of Health and Social Development of the Russian Federation
City
Saint Petersburg
ZIP/Postal Code
194100
Country
Russian Federation
12. IPD Sharing Statement
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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
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