Iron Deficiency AnemiaPhase 3NCT01155375
A Trial of Ferumoxytol for the Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease
A Phase 3 study testing Ferumoxytol and Oral Iron in people with Iron Deficiency Anemia, run by AMAG Pharmaceuticals, Inc. at 1 site in United States.
Trial summary
Study evaluating the efficacy and safety of intravenous (IV) ferumoxytol compared with oral iron for the treatment of pediatric participants with chronic kidney disease (CKD). Read more about this study
- Who is it for?
- Ages 6 months to 17 years
- Conditions the study is for
- Iron Deficiency Anemia
- What is being tested?
- Ferumoxytol and Oral Iron
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Oct 2011to 24 Jun 2014
- 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 6 months to 17 years
- All sexes
- No healthy volunteers
- Child
Key Inclusion Criteria for the combined AMAG-FER-CKD-251 and AMAG-FER-CDK-252 studies include:
- Males or females 6 months to <18 years of age
- CKD non-dialysis participants or dialysis-dependent participants who were on peritoneal dialysis or stable hemodialysis prior to Screening
- Had iron deficiency anemia defined as: a) hemoglobin level ≤12.0 grams/deciliter (g/dL) and b) with either transferrin saturation level ≤40% or ferritin level <100 nanograms/milliliter (ng/mL)
- Female participants of childbearing potential who were sexually active must have been on an effective method of birth control for at least 1 month prior to Screening and agreed to remain on birth control until completion of participation in the study
Key Exclusion Criteria for the combined AMAG-FER-CKD-251 and AMAG-FER-CDK-252 studies include:
- History of allergy to either oral or IV iron
- Hemoglobin level ≤7.0 g/dL
- Serum ferritin level >600 ng/mL
- Female participants who were pregnant or intended to become pregnant, or were breastfeeding, were within 3 months postpartum, or had a positive serum pregnancy test
Trial locations and contacts
1 site in 1 country.
United States1 site
AMAG Pharmaceuticals, Inc.
Waltham, Massachusetts, 02451
About this study
Official title: A Randomized, Open-Label, Active-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Ferumoxytol Compared With Oral Iron for the Treatment of Iron Deficiency Anemia in Pediatric Subjects With Chronic Kidney Disease
Study evaluating the efficacy and safety of intravenous (IV) ferumoxytol compared with oral iron for the treatment of pediatric participants with chronic kidney disease (CKD).
Read the detailed description
Study AMAG-FER-CKD-251 was a study evaluating the efficacy and safety of IV ferumoxytol in pediatric participants with dialysis-dependent CKD. Study AMAG-FER-CKD-252 (NCT01155388) was a study evaluating the efficacy and safety of IV ferumoxytol in pediatric participants with nondialysis-dependent CKD. Due to significant challenges with enrollment for both studies, Study AMAG-FER-CKD-252 was combined with Study AMAG-FER-CKD-251 and enrollment continued under Study AMAG-FER-CKD-251. The enrollment number (n=14) includes the number of participants for both AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies, combined.
Participants were enrolled by age cohorts in a stepwise manner following a safety review by the Data Safety Monitoring Board of 1 age cohort prior to enrollment of a subsequent age cohort, with progression from oldest to youngest: Randomization was stratified by the following age cohorts: 12 to <18 years, 6 to <12 years, 2 to <6 years, and 6 months to <2 years.
- Conditions
- Iron Deficiency Anemia
- Being tested
- Ferumoxytol, Oral Iron
- Keywords
- Iron deficiency anemia, Feraheme, ferumoxytol, CKD, pediatric, dialysis-dependent, nondialysis-dependent
- 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.
FerumoxytolDrug
Experimental: Ferumoxytol
Oral IronDrug
Also called Ferrous fumarate
Active Comparator: Oral iron
| Study group | Role | What they receive |
|---|---|---|
| Ferumoxytol | Experimental | Participants will receive 1 of the following 2 ferumoxytol dose regimens:
* Four IV injections of ferumoxytol 3.5 mg Fe/kg (maximum of 255 mg/dose) administered on nonconsecutive days within a 14-day period as follows: Day 1 (dose 1), Days 3* through 10 (dose 2), Days 5 through 12 (dose 3), and Days 7 through 14 (dose 4). *Participants participating in PK sampling received the second dose on Day 4 after the 72-hour PK sample was collected.
* Two IV injections of ferumoxytol 7.0 mg Fe/kg (maximum of 510 mg/dose), the first administered on Day 1 and the second on Days 3 through 9. Receives: Drug: Ferumoxytol |
| Oral Iron | Active comparator | Participants will receive oral iron 2.5 mg Fe/kg twice daily (maximum of 100 mg/dose) on Days 1 through 35. Receives: Drug: Oral Iron |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change In Hemoglobin From Baseline To Week 5
Mean changes in hemoglobin from Baseline to Week 5 were to be presented. Despite efforts to complete the studies as designed, several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this primary outcome measure cannot be summarized nor can the statistical analysis, as described in the protocol, be provided in a way that will provide any significant data based upon the limited study datasets.
Measured: Baseline, Week 5
Secondary outcomes (1)
Pharmacokinetics: Area Under The Curve Of Ferumoxytol
Ferumoxytol concentrations were to be determined using a drug-specific nuclear magnetic resonance assay. Blood samples were to be collected at specified times predose and postdose at the time of the first dose from 6 participants in each age-dose group. Sampling for participants <6 years of age will be minimized to the fewest number of time points required for population PK analysis based on preliminary PK data from the first 2 age cohorts. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this secondary outcome measure cannot be summarized in a way that will provide any significant data based upon the limited study datasets.
Measured: Baseline; 10, 30, 120, and 360 minutes postdose; 24, 48, and 72 hours postdose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2010 (estimated)
- Study start17 Oct 2011
- Primary completion24 Jun 2014
- Study completion24 Jun 2014
Why it stopped: Several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the AMAG-FER-CKD-251 study as designed.
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
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- AMAG Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01155375
- Sponsor's protocol ID
- AMAG-FER-CKD-251
- Status last verified
- Mar 2022
- First submitted
- 28 Jun 2010
- Last updated
- 4 Apr 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-Label, Active-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Ferumoxytol Compared With Oral Iron for the Treatment of Iron Deficiency Anemia in Pediatric Subjects With Chronic Kidney Disease
- ClinicalTrials.gov ID
- NCT01155375
- Acronym / sponsor ID
- AMAG-FER-CKD-251
- Conditions
- Iron Deficiency Anemia
- Keywords
- Iron deficiency anemia, Feraheme, ferumoxytol, CKD, pediatric, dialysis-dependent, nondialysis-dependent
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the AMAG-FER-CKD-251 study as designed.
- Enrolment
- 14 participants (actual)
- Lead sponsor
- AMAG Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 17 Oct 2011 (actual)
- Primary completion
- 24 Jun 2014 (actual)
- Study completion
- 24 Jun 2014 (actual)
- First posted
- 1 Jul 2010 (estimated)
- Results
- Posted 6 Nov 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study evaluating the efficacy and safety of intravenous (IV) ferumoxytol compared with oral iron for the treatment of pediatric participants with chronic kidney disease (CKD).
Detailed description
Study AMAG-FER-CKD-251 was a study evaluating the efficacy and safety of IV ferumoxytol in pediatric participants with dialysis-dependent CKD. Study AMAG-FER-CKD-252 (NCT01155388) was a study evaluating the efficacy and safety of IV ferumoxytol in pediatric participants with nondialysis-dependent CKD. Due to significant challenges with enrollment for both studies, Study AMAG-FER-CKD-252 was combined with Study AMAG-FER-CKD-251 and enrollment continued under Study AMAG-FER-CKD-251. The enrollment number (n=14) includes the number of participants for both AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies, combined.
Participants were enrolled by age cohorts in a stepwise manner following a safety review by the Data Safety Monitoring Board of 1 age cohort prior to enrollment of a subsequent age cohort, with progression from oldest to youngest: Randomization was stratified by the following age cohorts: 12 to <18 years, 6 to <12 years, 2 to <6 years, and 6 months to <2 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ferumoxytol | Experimental | Drug: FerumoxytolParticipants will receive 1 of the following 2 ferumoxytol dose regimens: * Four IV injections of ferumoxytol 3.5 mg Fe/kg (maximum of 255 mg/dose) administered on nonconsecutive days within a 14-day period as follows: Day 1 (dose 1), Days 3* through 10 (dose 2), Days 5 through 12 (dose 3), and Days 7 through 14 (dose 4). *Participants participating in PK sampling received the second dose on Day 4 after the 72-hour PK sample was collected. * Two IV injections of ferumoxytol 7.0 mg Fe/kg (maximum of 510 mg/dose), the first administered on Day 1 and the second on Days 3 through 9. |
| Oral Iron | Active comparator | Drug: Oral IronParticipants will receive oral iron 2.5 mg Fe/kg twice daily (maximum of 100 mg/dose) on Days 1 through 35. |
Who may qualify
- Ages
- 6 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 |
|---|---|---|
| FerumoxytolExperimental: Ferumoxytol | Drug | Ferumoxytol |
| Oral IronAlso: Ferrous fumarateActive Comparator: Oral iron | Drug | Oral Iron |
Primary outcome
Mean Change In Hemoglobin From Baseline To Week 5
Time frame: Baseline, Week 5
Mean changes in hemoglobin from Baseline to Week 5 were to be presented. Despite efforts to complete the studies as designed, several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies. Blood samples were collected, but not run through an analysis to obtain outcome measure data. As such, the data set for this primary outcome measure cannot be summarized nor can the statistical analysis, as described in the protocol, be provided in a way that will provide any significant data based upon the limited study datasets.
1 secondary outcome
Pharmacokinetics: Area Under The Curve Of Ferumoxytol
Time frame: Baseline; 10, 30, 120, and 360 minutes postdose; 24, 48, and 72 hours postdose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Apr 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ferumoxytol and Oral Iron in people with Iron Deficiency Anemia, run by AMAG Pharmaceuticals, Inc. at 1 site 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?
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: stopped early, as of the sponsor's last update on 4 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 6 Nov 2017). 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?
Mean Change In Hemoglobin From Baseline To Week 5 (time frame: Baseline, Week 5). 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 4 Apr 2022.