A Multicenter, Randomized, Active-Controlled Study to Investigate the Efficacy and Safety of Intravenous Ferric Carboxymaltose in Pediatric Patients With Iron Deficiency Anemia
Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA
Sponsors
Source
American Regent, Inc.
Oversight Info
Has Dmc
Yes
Is Fda Regulated Drug
Yes
Is Fda Regulated Device
No
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.
Overall Status
Recruiting
Start Date
2018-09-12
Completion Date
2021-01-01
Primary Completion Date
2020-01-01
Phase
Phase 3
Study Type
Interventional
Primary Outcome
Measure |
Time Frame |
|
Change in hemoglobin |
Baseline to day 35 |
Secondary Outcome
Measure |
Time Frame |
|
Change in ferritin |
Baseline to day 35 |
|
Change in TSAT |
Baseline to day 35 |
|
Change in reticulocyte hemoglobin content |
Baseline to day 35 |
Enrollment
72
Condition
Intervention
Intervention Type
Drug
Intervention Name
Description
Intravenous iron
Arm Group Label
Ferric Caroboxymaltose
Other Name
Injectafer
Ferinject
Intervention Type
Drug
Intervention Name
Description
oral iron therapy
Arm Group Label
Oral Ferrous Sulfate
Other Name
oral iron
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
Gender
All
Minimum Age
1 Year
Maximum Age
17 Years
Healthy Volunteers
No
Overall Contact
Location
Facility |
Status |
Contact |
Investigator |
|
Arkansas Children's Hospital Little Rock Arkansas 72202 United States |
Recruiting |
Last Name: Shelley Crary, MD Role: Principal Investigator | |
|
International Research Partners, Inc. Doral Florida 33122 United States |
Recruiting |
Last Name: Luis Aponte, MD Role: Principal Investigator | |
|
South Florida Research Phase I-IV Miami Springs Florida 33166 United States |
Recruiting |
Last Name: Maria Jamie, MD Role: Principal Investigator | |
|
Miami Clinical Research Miami Florida 33155 United States |
Recruiting |
Last Name: Keila Hoover, MD Role: Principal Investigator | |
|
Riley Hospital for Children,Room 4340 Indianapolis Indiana 46202 United States |
Recruiting |
Last Name: Kathleen Overholt, MD Role: Principal Investigator | |
|
Caro Health Plaza Caro Michigan 48723 United States |
Recruiting |
Last Name: Naveed Mahfooz, MD Role: Principal Investigator | |
|
Galen Research Chesterfield Missouri 63005 United States |
Recruiting |
Last Name: Alexander Beyzer, MD Role: Principal Investigator | |
|
Cincinnati Children's Hospital and Medical Center Cincinnati Ohio 45229 United States |
Recruiting |
Last Name: Patrick McGann, MD Role: Principal Investigator | |
|
Cook Children's Medical Center Fort Worth Texas 76101 United States |
Recruiting |
Last Name: Timothy L McCavit, MD Role: Principal Investigator | |
|
Baylor College of Medicine/Texas Children Hospital Houston Texas 77030 United States |
Recruiting |
Last Name: Jacquelyn Powers, MD Role: Principal Investigator | |
|
Tekton Research San Antonio Texas 78240 United States |
Recruiting |
Last Name: Olutola Adetona, MD Role: Principal Investigator | |
|
Aspen Clinical Research Orem Utah 84058 United States |
Recruiting |
Last Name: Joshua D Fuller, MD Role: Principal Investigator |
Location Countries
Country
United States
Verification Date
2019-05-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Has Expanded Access
No
Condition Browse
Number Of Arms
2
Intervention Browse
Mesh Term
Iron
Ferric Compounds
Arm Group
Arm Group Label
Ferric Caroboxymaltose
Arm Group Type
Active Comparator
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 Group Label
Oral Ferrous Sulfate
Arm Group Type
Active Comparator
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.
Firstreceived Results Date
N/A
Overall Contact Backup
Patient Data
Sharing Ipd
No
Firstreceived Results Disposition Date
N/A
Study Design Info
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)
Study First Submitted
April 17, 2018
Study First Submitted Qc
May 1, 2018
Study First Posted
May 14, 2018
Last Update Submitted
May 15, 2019
Last Update Submitted Qc
May 15, 2019
Last Update Posted
May 16, 2019
ClinicalTrials.gov processed this data on December 10, 2019
Conditions
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conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.