Back to resultsA Trial of Ferumoxytol for the Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease
Primary Purpose
Iron Deficiency Anemia
Status
Terminated
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Ferumoxytol
Oral Iron
Sponsored by
About this trial
This is an interventional treatment trial for Iron Deficiency Anemia focused on measuring Iron deficiency anemia, Feraheme, ferumoxytol, CKD, pediatric, dialysis-dependent, nondialysis-dependent
Eligibility Criteria
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
Sites / Locations
- AMAG Pharmaceuticals, Inc.
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
Ferumoxytol
Oral Iron
Arm Description
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.
Participants will receive oral iron 2.5 mg Fe/kg twice daily (maximum of 100 mg/dose) on Days 1 through 35.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
Full Information
NCT ID
NCT01155375
First Posted
June 28, 2010
Last Updated
March 31, 2022
Sponsor
AMAG Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT01155375
Brief Title
A Trial of Ferumoxytol for the Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease
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 Type
Interventional
2. Study Status
Record Verification Date
March 2022
Overall Recruitment Status
Terminated
Why Stopped
Several factors contributed to significant challenges in enrollment and led the Sponsor to discontinue the AMAG-FER-CKD-251 study as designed.
Study Start Date
October 17, 2011 (Actual)
Primary Completion Date
June 24, 2014 (Actual)
Study Completion Date
June 24, 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AMAG Pharmaceuticals, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief 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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Deficiency Anemia
Keywords
Iron deficiency anemia, Feraheme, ferumoxytol, CKD, pediatric, dialysis-dependent, nondialysis-dependent
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
14 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Ferumoxytol
Arm Type
Experimental
Arm Description
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.
Arm Title
Oral Iron
Arm Type
Active Comparator
Arm Description
Participants will receive oral iron 2.5 mg Fe/kg twice daily (maximum of 100 mg/dose) on Days 1 through 35.
Intervention Type
Drug
Intervention Name(s)
Ferumoxytol
Intervention Description
Experimental: Ferumoxytol
Intervention Type
Drug
Intervention Name(s)
Oral Iron
Other Intervention Name(s)
Ferrous fumarate
Intervention Description
Active Comparator: Oral iron
Primary Outcome Measure Information:
Title
Mean Change In Hemoglobin From Baseline To Week 5
Description
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.
Time Frame
Baseline, Week 5
Secondary Outcome Measure Information:
Title
Pharmacokinetics: Area Under The Curve Of Ferumoxytol
Description
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.
Time Frame
Baseline; 10, 30, 120, and 360 minutes postdose; 24, 48, and 72 hours postdose
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
AMAG Pharmaceuticals, Inc.
City
Waltham
State/Province
Massachusetts
ZIP/Postal Code
02451
Country
United States
12. IPD Sharing Statement
Learn more about this trial
A Trial of Ferumoxytol for the Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease
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