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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03523117
Recruitment Status : Recruiting
First Posted : May 14, 2018
Last Update Posted : February 11, 2020
Information provided by (Responsible Party):
American Regent, Inc.

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.

Condition or disease Intervention/treatment Phase
Iron Deficiency Anemia Drug: Ferric carboxymaltose Drug: Ferrous Sulfate Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 72 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : September 12, 2018
Estimated Primary Completion Date : July 2020
Estimated Study Completion Date : January 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia Iron

Arm Intervention/treatment
Active Comparator: 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.
Drug: Ferric carboxymaltose
Intravenous iron
Other Names:
  • Injectafer
  • Ferinject

Active Comparator: 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.
Drug: Ferrous Sulfate
oral iron therapy
Other Name: oral iron

Primary Outcome Measures :
  1. Change in hemoglobin [ Time Frame: Baseline to day 35 ]
    Change in hemoglobin 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 :
  1. Change in ferritin [ Time Frame: Baseline to day 35 ]
  2. Change in TSAT [ Time Frame: Baseline to day 35 ]
  3. Change in reticulocyte hemoglobin content [ Time Frame: Baseline to day 35 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Year to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03523117

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Contact: James Bambrick, BS 6317723518 ext 61818
Contact: Mark Falone, MD 6317723544 ext 61844

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United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Carol D Pierce    501-364-4440   
Principal Investigator: Shelley Crary, MD         
United States, Florida
International Research Partners, Inc. Recruiting
Doral, Florida, United States, 33122
Contact: Milagros Agosto    305-468-9455   
Principal Investigator: Luis Aponte, MD         
ProHealth Research Center Recruiting
Doral, Florida, United States, 33166
Contact: Hilda Clavijo    305-960-7394   
Principal Investigator: Bernard Ashby, MD         
South Florida Research Phase I-IV Recruiting
Miami Springs, Florida, United States, 33166
Contact: Cynthia Vilches    305-418-0847   
Principal Investigator: Maria Jamie, MD         
Garden Medical Research, Inc. Recruiting
Miami, Florida, United States, 33155
Contact: Claritza Campos    305-846-9303   
Principal Investigator: Juan Miguel Ruiz-Unger         
Miami Clinical Research Recruiting
Miami, Florida, United States, 33155
Contact: Ana Mendez    305-433-6496   
Principal Investigator: Keila Hoover, MD         
United States, Indiana
Riley Hospital for Children,Room 4340 Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Shannon Maraldo    317-948-3395   
Principal Investigator: Kathleen Overholt, MD         
United States, Michigan
Caro Health Plaza Recruiting
Caro, Michigan, United States, 48723
Contact: Sehrish Khurram    443-844-5710   
Principal Investigator: Naveed Mahfooz, MD         
United States, Missouri
Galen Research Recruiting
Chesterfield, Missouri, United States, 63005
Contact: Farzad Qureshi    314-546-5888   
Principal Investigator: Alexander Beyzer, MD         
United States, New York
Tiga Pediatrics, PC Recruiting
Bronx, New York, United States, 10467
Contact: Eston Clare    718-881-8999   
Principal Investigator: Tosan Oruwariye, MD         
United States, Ohio
Cincinnati Children's Hospital and Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Amanda Pfeiffer    513-803-4977   
Principal Investigator: Patrick McGann, MD         
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Wendi Long    513-803-3064   
Principal Investigator: Patrick McGann, MD         
United States, Texas
Cook Children's Medical Center Recruiting
Fort Worth, Texas, United States, 76101
Contact: Liz Miller    682-885-6718   
Principal Investigator: Timothy L McCavit, MD         
Baylor College of Medicine/Texas Children Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Dinu Bogdam    832-824-4825   
Principal Investigator: Jacquelyn Powers, MD         
Tekton Research Recruiting
San Antonio, Texas, United States, 78240
Contact: Megan Malek    210-996-2600   
Principal Investigator: Olutola Adetona, MD         
United States, Utah
Aspen Clinical Research Recruiting
Orem, Utah, United States, 84058
Contact: Nikki Colley    801-753-0081   
Principal Investigator: Joshua D Fuller, MD         
Sponsors and Collaborators
American Regent, Inc.
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Responsible Party: American Regent, Inc. Identifier: NCT03523117    
Other Study ID Numbers: 1VIT17044
First Posted: May 14, 2018    Key Record Dates
Last Update Posted: February 11, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Iron-Deficiency
Hematologic Diseases
Anemia, Hypochromic
Iron Metabolism Disorders
Metabolic Diseases