Clinical trial NCT02410213: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Iron Deficiency Anemia (IDA)
Lead sponsor: American Regent, Inc.
Sponsor class: INDUSTRY
First posted: 2015-04-07
Enrollment: 35
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
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.
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Maximum Serum Concentration (Cmax)
timeFrame: prior to dosing and 1, 2, 6, 12, 48 and 72 hours post dosing
Secondary outcomes:
Other outcomes:
Locations:
city: Bydgoszcz
country: Poland
facility: Szpital Uniwersytecki Katedra i Klinika Pediatrii, Hematologii i Onkologii
city: Dębica
country: Poland
facility: Zespół Opieki Zdrowotnej w Dębicy z siedzibą w Dębicy , Oddział Dziecięcy
city: Kraków
country: Poland
facility: Uniwersytecki Szpital Dziecięcy w Krakowie, Oddział Pediatrii i Gastroenterologii (V)
city: Lublin
country: Poland
facility: Klinika Hematologii, Onkologii i Transplantologii Dziecięcej Uniwersytecki Szpital Dziecięcy w Lublinie
city: Lublin
country: Poland
facility: Oddział Ogólnopediatryczny; Uniwersytecki Szpital Dziecięcy w Lublinie
city: Rzeszów
country: Poland
facility: Indywidualna Specjalistyczna Praktyka lekarska z siedzibą w Rzeszowie
city: Szczecin
country: Poland
facility: Klinika Pediatrii, Hematologii i Onkologii Dziecięcej
city: Warszawa
country: Poland
facility: Klinika Gastroenterologii, Hepatologii, Zaburzeń Odżywiania i Pediatrii
city: Ryazan'
country: Russian Federation
facility: 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: Saint Petersburg
country: Russian Federation
facility: State Educational Institution of Higher Professional Education Saint Petersburg State Pediatric Medical Acamy of Ministry of Health and Social Development of the Russian Federation
Study officials:
References: