Iron Deficiency, AnaemiaPhase 2NCT04968379
Safety, Tolerability, PK and PD of Intravenous Ferric Carboxymaltose in Infants With Iron Deficiency Anemia
A Phase 2 study testing Ferric carboxymaltose in people with Iron Deficiency, Anaemia, run by American Regent, Inc. at 4 sites in United States.
Trial summary
An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 year) with Iron Deficiency Anemia. Read more about this study
- Who is it for?
- Ages Up to 1 year
- Conditions the study is for
- Iron Deficiency, Anaemia
- What is being tested?
- Ferric carboxymaltose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 4 sitesUnited States
- Study dates
- 21 Jul 2022to 12 Dec 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 Up to 1 year
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female participants 0 to 1 year of age, medically indicated for iron replacement, with his/her parent or legal guardian willing and able to sign the informed consent form approved by the IRB / Independent Ethics Committee (IEC).
- Screening Hb ≥7 g/dL to <10 g/dL.
- Infants with any of the following conditions:
- Heart failure with IDA defined as syndromes of excessive preload, excessive afterload, abnormal rhythm, or decreased contractility
- Gastrointestinal diseases with acquired short bowel syndrome (due to volvulus, necrotizing enterocolitis from surgical resection or spontaneous intestinal perforation)
- Gastrointestinal intolerance of oral iron or an unsatisfactory response to oral iron
- Other conditions associated with IDA which in the opinion of the investigator might benefit from administration of FCM
Who cannot join
- Known history of hypersensitivity reaction to FCM.
- Body weight <2.5 kg.
- History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
- Hemodialysis-dependent chronic kidney disease.
- History of significant diseases of the liver, hematopoietic system, cardiovascular system, or other conditions which, on the opinion of the investigator, may place a participant at added risk for participation in the study.
- Active infection.
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- Anemia due to reasons other than iron deficiency (e.g., hemoglobinopathy vitamin B12 deficiency, or folic acid deficiency).
- Blood transfusion in the 4 weeks prior to consent.
- Administration of an iron-containing product within 14 days of administration of the study article.
- Administration and / or use of an investigational product (drug or device) within 30 days of screening.
- Current participation in another clinical trial.
- Unable to comply with study procedures and assessments.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Iowa
Iowa City, Iowa, 52242
Cohen Children's Medical Center
New Hyde Park, New York, 11040
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134
About this study
Official title: An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 Year) With Iron Deficiency Anemia
An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 year) with Iron Deficiency Anemia.
Read the detailed description
A phase II, open-label, multi-center study with 2 Cohorts to evaluate the safety, tolerance, PK, and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving either a 5.0 mg/kg or 7.5 mg/kg dose of FCM.
Participants will have a screening evaluation within 14 days of the first dose of study drug. A medically supervised environment is required on Day 1 (day of dosing) and for 4 hours post dosing. Participants are allowed to be enrolled if satisfying the inclusion and exclusion criteria. Participants will return to the study site for additional evaluation and sampling on Days 8 (± 2 days), 15 (± 2 days), 22 (± 2 days), and 36 (± 2 days).
- Conditions
- Iron Deficiency, Anaemia
- Being tested
- Ferric carboxymaltose
- 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
Intravenous
| Study group | Role | What they receive |
|---|---|---|
| Ferric Carboxymaltose | Experimental | To evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 5.0 mg/kg dose of FCM Receives: Drug: Ferric carboxymaltose |
| Injectafer | Experimental | To evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 7.5 mg/kg dose dose of FCM. Receives: Drug: Ferric carboxymaltose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent adverse events
Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities
Measured: Baseline to Day 36
Change in hemoglobin (Hb): g/dL
determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters
Measured: baseline to Days 8, 15, 22, and 36
Change in reticulocytes count: %
determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters
Measured: baseline to Days 8, 15, 22, and 36
Evaluate the PD parameters - Change in serum iron: mcg/dL
To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Measured: baseline to Day 36
Evaluate the PD parameters - Change in serum ferritin: ng/mL
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Measured: baseline to Day 36
Evaluate the PD parameters - Change in total iron binding capacity [TIBC]): mcg/dL
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Measured: baseline to Day 36
Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Measured: baseline to Day 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2021
- Study start21 Jul 2022
- Primary completion8 Oct 2024 (estimated)
- Study completion12 Dec 2024 (estimated)
Why it stopped: Difficult to recruit and enroll patients for this study in a reasonable amount of time.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Iron Deficiency, AnaemiaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- American Regent, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mark Falone, MD, Study director, American Regent
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04968379
- Sponsor's protocol ID
- 1VIT19046
- Status last verified
- Jan 2023
- First submitted
- 2 Nov 2020
- Last updated
- 6 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 Year) With Iron Deficiency Anemia
- ClinicalTrials.gov ID
- NCT04968379
- Acronym / sponsor ID
- 1VIT19046
- Conditions
- Iron Deficiency, Anaemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Difficult to recruit and enroll patients for this study in a reasonable amount of time.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- American Regent, Inc.Industry (pharma, biotech, devices)
- Study start
- 21 Jul 2022 (actual)
- Primary completion
- 8 Oct 2024 (estimated)
- Study completion
- 12 Dec 2024 (estimated)
- First posted
- 20 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An Open-Label, Multi-Center Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous Ferric Carboxymaltose (FCM) in Infants (0-1 year) with Iron Deficiency Anemia.
Detailed description
A phase II, open-label, multi-center study with 2 Cohorts to evaluate the safety, tolerance, PK, and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving either a 5.0 mg/kg or 7.5 mg/kg dose of FCM.
Participants will have a screening evaluation within 14 days of the first dose of study drug. A medically supervised environment is required on Day 1 (day of dosing) and for 4 hours post dosing. Participants are allowed to be enrolled if satisfying the inclusion and exclusion criteria. Participants will return to the study site for additional evaluation and sampling on Days 8 (± 2 days), 15 (± 2 days), 22 (± 2 days), and 36 (± 2 days).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ferric Carboxymaltose | Experimental | Drug: Ferric carboxymaltoseTo evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 5.0 mg/kg dose of FCM |
| Injectafer | Experimental | Drug: Ferric carboxymaltoseTo evaluate the safety, tolerance, PK and PD profile of intravenous (IV) FCM in infants 0 to 1 year of age with IDA after receiving a 7.5 mg/kg dose dose of FCM. |
Who may qualify
- Ages
- Up to 1 year
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Male and female participants 0 to 1 year of age, medically indicated for iron replacement, with his/her parent or legal guardian willing and able to sign the informed consent form approved by the IRB / Independent Ethics Committee (IEC).
- Screening Hb ≥7 g/dL to <10 g/dL.
- Infants with any of the following conditions:
Key exclusion criteria (12 listed)
- Known history of hypersensitivity reaction to FCM.
- Body weight <2.5 kg.
- History of acquired iron overload, hemochromatosis, or other iron accumulation disorders.
- Hemodialysis-dependent chronic kidney disease.
- History of significant diseases of the liver, hematopoietic system, cardiovascular system, or other conditions which, on the opinion of the investigator, may place a participant 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: InjectaferIntravenous | Drug | Ferric Carboxymaltose, Injectafer |
Primary outcome
Treatment-emergent adverse events
Time frame: Baseline to Day 36
Treatment-emergent clinical laboratory test (clinical chemistry and hematology) abnormalities
Change in hemoglobin (Hb): g/dL
Time frame: baseline to Days 8, 15, 22, and 36
determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters
Change in reticulocytes count: %
Time frame: baseline to Days 8, 15, 22, and 36
determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters
Evaluate the PD parameters - Change in serum iron: mcg/dL
Time frame: baseline to Day 36
To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Evaluate the PD parameters - Change in serum ferritin: ng/mL
Time frame: baseline to Day 36
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Evaluate the PD parameters - Change in total iron binding capacity [TIBC]): mcg/dL
Time frame: baseline to Day 36
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Evaluate the PD parameters - Change in serum transferrin saturation [TSAT]): mg/dL
Time frame: baseline to Day 36
Description: To determine appropriate dosing of FCM in infants from 0 to 1 year of age by evaluating PD parameters.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jan 2023.
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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, MD study director
American Regent
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ferric carboxymaltose in people with Iron Deficiency, Anaemia, run by American Regent, Inc. at 4 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 7 inclusion and 12 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: withdrawn, as of the sponsor's last update on 6 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Treatment-emergent adverse events (time frame: Baseline to Day 36). The study lists 7 primary outcomes in all. 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 6 Jan 2023.