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Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA

Primary Purpose

Iron Deficiency Anemia

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Ferric carboxymaltose
Ferrous Sulfate
Sponsored by
American Regent, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Iron Deficiency Anemia

Eligibility Criteria

1 Year - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Arkansas Children's Hospital
  • International Research Partners, Inc.
  • ProHealth Research Center
  • South Florida Research Phase I-IV
  • Garden Medical Research, Inc.
  • Miami Clinical Research
  • Riley Hospital for Children,Room 4340
  • Caro Health Plaza
  • Galen Research
  • Tiga Pediatrics, PC
  • Cincinnati Children's Hospital and Medical Center
  • Cincinnati Children's Hospital Medical Center
  • Cook Children's Medical Center
  • Baylor College of Medicine/Texas Children Hospital
  • Tekton Research
  • Aspen Clinical Research

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

Ferric Caroboxymaltose

Oral Ferrous Sulfate

Arm Description

Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.

Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants <12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to <4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to <12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.

Outcomes

Primary Outcome Measures

Change in Hemoglobin g/dL
Change in hemoglobin g/dL from baseline to day 35 will be analyzed using parametric analysis of covariance (ANCOVA). The model will include terms for the randomization strata (hemoglobin and age categories), baseline hemoglobin, as well as treatment group. Baseline hemoglobin will be defined as the last hemoglobin obtained before randomization.

Secondary Outcome Measures

Change in Ferritin µg/L From Baseline to Day 35
Change in ferritin µg/L from baseline to day 35 was analyzed using parametric analysis of covariance (ANCOVA). The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a covariate.
Change in TSAT (%) From Baseline to Day 35
Change in TSAT (%) from baseline to day 35 was analyzed using parametric analysis of covariance (ANCOVA). The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a covariate
Change in Reticulocyte Hemoglobin (Picograms) Content From Baseline to Day 35
Change in reticulocyte hemoglobin (picograms) content from baseline to day 35 was analyzed using a mixed model repeated. The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a reticulocyte hemoglobin content.

Full Information

First Posted
April 17, 2018
Last Updated
May 26, 2022
Sponsor
American Regent, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03523117
Brief Title
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

2. Study Status

Record Verification Date
May 2022
Overall Recruitment Status
Completed
Study Start Date
January 31, 2019 (Actual)
Primary Completion Date
December 22, 2020 (Actual)
Study Completion Date
January 29, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
American Regent, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Deficiency Anemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
79 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Ferric Caroboxymaltose
Arm Type
Active Comparator
Arm Description
Ferric Carboxymaltose - 2 doses (day 0 and day 7) at 15 mg/kg to a maximum single dose of 750 mg (whichever is smaller) up to a maximum of total dose of 1500 mg administered as either an undiluted IV push at a rate of 100 mg (2mL)/minute OR in no more than 250 mL of normal saline and infused over 15 minutes.
Arm Title
Oral Ferrous Sulfate
Arm Type
Active Comparator
Arm Description
Oral Ferrous Sulfate - will receive an age-dependent formulation of oral ferrous sulfate daily for 28 days as follows: participants <12 years of age will receive 6 mg (elemental iron)/kg/day divided into 2 daily doses of an oral liquid formulation, either drops or elixir, and participants ≥12 will receive 2 daily doses of oral tablets. Infants and children (ages 1 to <4 years) will receive oral ferrous sulfate drops, while children (ages ≥4 to <12 years) will receive oral ferrous sulfate elixir. Adolescents (ages ≥12 to 17 years) will receive an oral ferrous sulfate tablet (65 mg of elemental iron/tablet/dose) twice a day (BID). The maximum daily dose for all participants is 130 mg of elemental iron.
Intervention Type
Drug
Intervention Name(s)
Ferric carboxymaltose
Other Intervention Name(s)
Injectafer, Ferinject
Intervention Description
Intravenous iron
Intervention Type
Drug
Intervention Name(s)
Ferrous Sulfate
Other Intervention Name(s)
oral iron
Intervention Description
oral iron therapy
Primary Outcome Measure Information:
Title
Change in Hemoglobin g/dL
Description
Change in hemoglobin g/dL from baseline to day 35 will be analyzed using parametric analysis of covariance (ANCOVA). The model will include terms for the randomization strata (hemoglobin and age categories), baseline hemoglobin, as well as treatment group. Baseline hemoglobin will be defined as the last hemoglobin obtained before randomization.
Time Frame
Baseline to day 35
Secondary Outcome Measure Information:
Title
Change in Ferritin µg/L From Baseline to Day 35
Description
Change in ferritin µg/L from baseline to day 35 was analyzed using parametric analysis of covariance (ANCOVA). The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a covariate.
Time Frame
Baseline to day 35
Title
Change in TSAT (%) From Baseline to Day 35
Description
Change in TSAT (%) from baseline to day 35 was analyzed using parametric analysis of covariance (ANCOVA). The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a covariate
Time Frame
Baseline to day 35
Title
Change in Reticulocyte Hemoglobin (Picograms) Content From Baseline to Day 35
Description
Change in reticulocyte hemoglobin (picograms) content from baseline to day 35 was analyzed using a mixed model repeated. The model included terms for the randomization strata (hemoglobin and age categories), baseline ferritin as a reticulocyte hemoglobin content.
Time Frame
Baseline to day 35

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 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. Screening Hgb <11 g/dL. Screening ferritin ≤300 ng/mL and transferrin saturation (TSAT) <30%. 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. 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. Participants undergoing treatment for inflammatory bowel disease (IBD) must be on stable therapy for at least 8 weeks prior to consent. Exclusion Criteria: Known history of hypersensitivity reaction to any component of FCM. Previous randomization and treatment in this study or any other clinical study of FCM or VIT-45. History of acquired iron overload, hemochromatosis, or other iron accumulation disorders. Chronic kidney disease participants on hemodialysis. 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. Any existing non-viral infection. Known history of positive hepatitis B antigen (HBsAg) or hepatitis C viral antibody (HCV) with evidence of active hepatitis. Known history of positive HIV-1/HIV-2 antibodies (anti-HIV). Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy and vitamin B12 or folic acid deficiency) that has not been corrected. Intravenous iron and /or blood transfusion in the 4 weeks prior to consent. 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 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. Unable to comply with study procedures and assessments
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mark Falone
Organizational Affiliation
American Regent, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
International Research Partners, Inc.
City
Doral
State/Province
Florida
ZIP/Postal Code
33122
Country
United States
Facility Name
ProHealth Research Center
City
Doral
State/Province
Florida
ZIP/Postal Code
33166
Country
United States
Facility Name
South Florida Research Phase I-IV
City
Miami Springs
State/Province
Florida
ZIP/Postal Code
33166
Country
United States
Facility Name
Garden Medical Research, Inc.
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Miami Clinical Research
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Riley Hospital for Children,Room 4340
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Caro Health Plaza
City
Caro
State/Province
Michigan
ZIP/Postal Code
48723
Country
United States
Facility Name
Galen Research
City
Chesterfield
State/Province
Missouri
ZIP/Postal Code
63005
Country
United States
Facility Name
Tiga Pediatrics, PC
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Cincinnati Children's Hospital and Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Cook Children's Medical Center
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76101
Country
United States
Facility Name
Baylor College of Medicine/Texas Children Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Tekton Research
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78240
Country
United States
Facility Name
Aspen Clinical Research
City
Orem
State/Province
Utah
ZIP/Postal Code
84058
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA

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