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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ferric Caroboxymaltose | Active Comparator | 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. |
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| Oral Ferrous Sulfate | Active Comparator | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ferric carboxymaltose | Drug | Intravenous iron |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline to day 35 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline to day 35 |
| Change in TSAT (%) From Baseline to Day 35 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mark Falone | American Regent, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| International Research Partners, Inc. |
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Phase 3, multicenter, multinational, randomized, active-controlled study that compared 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 at least 8 weeks (56 days) prior to randomization.
Participants who satisfied the inclusion requirements and no exclusionary criteria were eligible to participate in this study and enter into a screening phase to confirm eligibility.
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| ID | Title | Description |
|---|---|---|
| FG000 | Ferric Caroboxymaltose | 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. Ferric carboxymaltose: Intravenous iron |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Sep 26, 2019 | Jan 18, 2022 |
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| Ferrous Sulfate | Drug | oral iron therapy |
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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 |
| Baseline to day 35 |
| 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. | Baseline to day 35 |
| Doral |
| Florida |
| 33122 |
| United States |
| ProHealth Research Center | Doral | Florida | 33166 | United States |
| Garden Medical Research, Inc. | Miami | Florida | 33155 | United States |
| Miami Clinical Research | Miami | Florida | 33155 | United States |
| South Florida Research Phase I-IV | Miami Springs | Florida | 33166 | United States |
| Riley Hospital for Children,Room 4340 | Indianapolis | Indiana | 46202 | United States |
| Caro Health Plaza | Caro | Michigan | 48723 | United States |
| Galen Research | Chesterfield | Missouri | 63005 | United States |
| Tiga Pediatrics, PC | The Bronx | New York | 10467 | United States |
| Cincinnati Children's Hospital and Medical Center | Cincinnati | Ohio | 45229 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76101 | United States |
| Baylor College of Medicine/Texas Children Hospital | Houston | Texas | 77030 | United States |
| Tekton Research | San Antonio | Texas | 78240 | United States |
| Aspen Clinical Research | Orem | Utah | 84058 | United States |
| FG001 | Oral Ferrous Sulfate | 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. Ferrous Sulfate: oral iron therapy |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Ferric Caroboxymaltose | 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. Ferric carboxymaltose: Intravenous iron |
| BG001 | Oral Ferrous Sulfate | 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. Ferrous Sulfate: oral iron therapy |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Intent to Treat (ITT) | Posted | Least Squares Mean | 95% Confidence Interval | g/dL | Baseline to day 35 |
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| Secondary | 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. | intent to treat (ITT) all randomized | Posted | Least Squares Mean | 95% Confidence Interval | µg/L | Baseline to day 35 |
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| Secondary | 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 | Intent to treatment | Posted | Least Squares Mean | 95% Confidence Interval | percentage | Baseline to day 35 |
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| Secondary | 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. | Intent to treat | Posted | Least Squares Mean | 95% Confidence Interval | picograms | Baseline to day 35 |
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Day 1 through Day 35
AE will be elicited by nonspecific questions such as "Have you noticed any problems?" Participants will be encouraged to report AEs at their onset. Any AE spontaneously reported by, elicited from the participant, or observed by the physician or study staff, shall be recorded on the appropriate Adverse Event (AE) page of the eCRF. T
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ferric Caroboxymaltose | 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. Ferric carboxymaltose: Intravenous iron | 0 | 40 | 0 | 40 | 0 | 40 |
| EG001 | Oral Ferrous Sulfate | 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. Ferrous Sulfate: oral iron therapy | 0 | 39 | 0 | 39 | 0 | 39 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Mark A. Falone, MD | American Regent, Inc. | 631.772.3544 | mfalone@americanregent.com |
| Prot_003.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 26, 2019 | Oct 27, 2021 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D018798 | Anemia, Iron-Deficiency |
| ID | Term |
|---|---|
| D000747 | Anemia, Hypochromic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000090463 | Iron Deficiencies |
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C522335 | ferric carboxymaltose |
| C020748 | ferrous sulfate |
| D007501 | Iron |
| ID | Term |
|---|---|
| D019216 | Metals, Heavy |
| D004602 | Elements |
| D007287 | Inorganic Chemicals |
| D028561 | Transition Elements |
| D008670 | Metals |
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| Male |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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