Iron Deficiency AnemiaPhase 3NCT03523117
Multicenter Randomized Active-controlled Study to Investigate Efficacy & Safety of IV FCM in Pediatric Patients With IDA
A Phase 3 study testing Ferric carboxymaltose and Ferrous Sulfate in people with Iron Deficiency Anemia, run by American Regent, Inc. at 16 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Iron Deficiency Anemia
- What is being tested?
- Ferric carboxymaltose and Ferrous Sulfate
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 79 participants
- Where
- 16 sitesUnited States
- Study dates
- 31 Jan 2019to 29 Jan 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 14Show fewer
- 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
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
United States16 sites
Caro Health Plaza
Caro, Michigan, 48723
Galen Research
Chesterfield, Missouri, 63005
Cincinnati Children's Hospital and Medical Center
Cincinnati, Ohio, 45229
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
International Research Partners, Inc.
Doral, Florida, 33122
ProHealth Research Center
Doral, Florida, 33166
Cook Children's Medical Center
Fort Worth, Texas, 76101
Baylor College of Medicine/Texas Children Hospital
Houston, Texas, 77030
Riley Hospital for Children,Room 4340
Indianapolis, Indiana, 46202
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Garden Medical Research, Inc.
Miami, Florida, 33155
Miami Clinical Research
Miami, Florida, 33155
South Florida Research Phase I-IV
Miami Springs, Florida, 33166
Aspen Clinical Research
Orem, Utah, 84058
Tekton Research
San Antonio, Texas, 78240
Tiga Pediatrics, PC
The Bronx, New York, 10467
About this study
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
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.
Read the 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.
- Conditions
- Iron Deficiency Anemia
- Being tested
- Ferric carboxymaltose, Ferrous Sulfate
- Medical subject headings
- Anemia, Iron-Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ferric carboxymaltoseDrug
Also called Injectafer, Ferinject
Intravenous iron
Ferrous SulfateDrug
Also called oral iron
oral iron therapy
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ferric carboxymaltose |
| 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. Receives: Drug: Ferrous Sulfate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline to day 35
Secondary outcomes (3)
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.
Measured: Baseline to day 35
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
Measured: 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.
Measured: Baseline to day 35
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2018
- Study start31 Jan 2019
- Primary completion22 Dec 2020
- Study completion29 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Iron Deficiency AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 79 participants (actual)
Sponsor and investigators
- Lead sponsor
- American Regent, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mark Falone, Study director, American Regent, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03523117
- Sponsor's protocol ID
- 1VIT17044
- Status last verified
- May 2022
- First submitted
- 17 Apr 2018
- Last updated
- 22 Jun 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03523117
- Acronym / sponsor ID
- 1VIT17044
- Conditions
- Iron Deficiency Anemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 79 participants (actual)
- Lead sponsor
- American Regent, Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Jan 2019 (actual)
- Primary completion
- 22 Dec 2020 (actual)
- Study completion
- 29 Jan 2021 (actual)
- First posted
- 14 May 2018 (actual)
- Results
- Posted 22 Jun 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ferric Caroboxymaltose | Active comparator | Drug: Ferric carboxymaltoseFerric 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 | Active comparator | Drug: Ferrous SulfateOral 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. |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (14 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ferric carboxymaltoseAlso: Injectafer, FerinjectIntravenous iron | Drug | Ferric Caroboxymaltose |
| Ferrous SulfateAlso: oral ironoral iron therapy | Drug | Oral Ferrous Sulfate |
Primary outcome
Change in Hemoglobin g/dL
Time frame: Baseline to day 35
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.
3 secondary outcomes
Change in Ferritin µg/L From Baseline to Day 35
Time frame: Baseline to day 35
Change in TSAT (%) From Baseline to Day 35
Time frame: Baseline to day 35
Change in Reticulocyte Hemoglobin (Picograms) Content From Baseline to Day 35
Time frame: Baseline to day 35
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mark Falone study director
American Regent, Inc.
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ferric carboxymaltose and Ferrous Sulfate in people with Iron Deficiency Anemia, run by American Regent, Inc. at 16 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Jun 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Hemoglobin g/dL (time frame: Baseline to day 35). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2022.