Clinical trial NCT03523117: Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA
Official title: A Multicenter, Randomized, Active-Controlled Study to Investigate the Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients With Iron Deficiency Anemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Iron Deficiency Anemia
Lead sponsor: American Regent, Inc.
Sponsor class: INDUSTRY
First posted: 2018-05-14
Enrollment: 79
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objective of this study is to demonstrate the efficacy and safety of intravenous ferric carboxymaltose (FCM), compared to oral iron, in pediatric participants who have iron deficiency anemia.

Detailed description: This is a Phase III, multicenter, randomized, active-controlled study that compares the efficacy and safety of FCM to oral iron in pediatric participants with IDA and a documented history of an inadequate response to oral iron therapy at least 8 weeks (56 days) prior to randomization.

Participants who satisfy the inclusion requirements and no exclusion criteria will be eligible to participate in this study and enter into a screening phase to confirm eligibility. Randomization will occur via the Interactive Response Technology (IRT) system in a 1:1 ratio to either Group A, participants receiving FCM, or Group B, participants receiving oral iron (oral solution drops, elixir or oral tablets). Randomization will be stratified by baseline Hgb (\<10, ≥10 g/dL) and age (1 to \<12 years and ≥12 to 17 years).

The oral ferrous sulfate formulation received will be based on the participant's age, such that infants and children (1 to \<4 years of age) will receive ferrous sulfate drops, children (≥4 to \<12 years of age) will receive ferrous sulfate elixir, and adolescents (≥12 to 17 years of age) will receive ferrous sulfate tablets. Participants who experience adverse clinical symptoms due to the oral iron during the treatment phase may have a weight-based dose of ferrous sulfate reduced from 6 mg/kg to 3 mg/kg. If the participant is receiving tablets, the dose will be reduced from one tablet taken twice daily to one tablet per day.

Once randomized, all participants will return for efficacy and safety evaluations, including adverse events and laboratory assessments, on Days 7, 14, 28, and 35. Additional pharmacokinetic sampling and analyses will be performed for participants receiving FCM on Days 0 and 7.

Eligibility criteria: Inclusion Criteria:

1. Male or female participants 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.
2. Screening Hgb \<11 g/dL.
3. Screening ferritin ≤300 ng/mL and transferrin saturation (TSAT) \<30%.
4. Participants must have a documented history of an inadequate response to any oral iron therapy for at least 8 weeks (56 days) prior to randomization.
5. For participants who are receiving an erythropoietin stimulating agent (ESA): stable ESA therapy (+/- 20% of current dose) for at least 8 weeks prior to the qualifying screening visit and no ESA dosing or product changes anticipated for the length of the trial.
6. Participants undergoing treatment for inflammatory bowel disease (IBD) must be on stable therapy for at least 8 weeks prior to consent.

Exclusion Criteria:

1. Known history of hypersensitivity reaction to any component of FCM.
2. Previous randomization and treatment in this study or any other clinical study of FCM or VIT-45.
3. History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
4. Chronic kidney disease participants on hemodialysis.
5. History of significant diseases of the liver, hematopoietic system, cardiovascular system, psychiatric disorder, or other conditions which, on the opinion of the investigator, may place a subject at added risk for participation in the study.
6. Any existing non-viral infection.
7. Known history of positive hepatitis B antigen (HBsAg) or hepatitis C viral antibody (HCV) with evidence of active hepatitis.
8. Known history of positive HIV-1/HIV-2 antibodies (anti-HIV).
9. Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy and vitamin B12 or folic acid deficiency) that has not been corrected.
10. Intravenous iron and /or blood transfusion in the 4 weeks prior to consent.
11. Administration and / or use of an investigational product (drug or device) within 30 days of screening.
12. Alcohol or drug abuse within the past six months.
13. Female participant who is 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.
14. Unable to comply with study procedures and assessments

Primary outcomes:
measure: Change in Hemoglobin g/dL
timeFrame: Baseline to day 35
Secondary outcomes:
measure: Change in Ferritin µg/L From Baseline to Day 35
timeFrame: Baseline to day 35
measure: Change in TSAT (%) From Baseline to Day 35
timeFrame: Baseline to day 35
measure: Change in Reticulocyte Hemoglobin (Picograms) Content From Baseline to Day 35
timeFrame: Baseline to day 35
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Doral
country: United States
facility: International Research Partners, Inc.
state: Florida
city: Doral
country: United States
facility: ProHealth Research Center
state: Florida
city: Miami Springs
country: United States
facility: South Florida Research Phase I-IV
state: Florida
city: Miami
country: United States
facility: Garden Medical Research, Inc.
state: Florida
city: Miami
country: United States
facility: Miami Clinical Research
state: Florida
city: Indianapolis
country: United States
facility: Riley Hospital for Children,Room 4340
state: Indiana
city: Caro
country: United States
facility: Caro Health Plaza
state: Michigan
city: Chesterfield
country: United States
facility: Galen Research
state: Missouri
city: Bronx
country: United States
facility: Tiga Pediatrics, PC
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital and Medical Center
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine/Texas Children Hospital
state: Texas
city: San Antonio
country: United States
facility: Tekton Research
state: Texas
city: Orem
country: United States
facility: Aspen Clinical Research
state: Utah
Study officials:
affiliation: American Regent, Inc.
name: Mark Falone
role: STUDY_DIRECTOR
References: