A Trial of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia in Pediatric Participants With Chronic Kidney Disease

Phase 3

Terminated

• Conditions: Iron Deficiency Anemia
• Interventions: Drug: Ferumoxytol

• Registration Number: NCT01264679
• Lead Sponsor: AMAG Pharmaceuticals, Inc.

Brief Summary

Study AMAG-FER-CKD-253 is an extension study of the combined AMAG-FER-CKD-251 (NCT01155375) and AMAG-FER-CKD-252 (NCT01155388) studies to evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with ferumoxytol.

Detailed Description

Study AMAG-FER-CKD-251 was a study evaluating the efficacy and safety of intravenous (IV) ferumoxytol in pediatric participants with dialysis-dependent chronic kidney disease (CKD). Study AMAG-FER-CKD-252 was a study evaluating the efficacy and safety of IV ferumoxytol in pediatric participants with nondialysis-dependent chronic kidney disease.

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.

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.

Participants who completed the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies had the option of participating in this extension study.

Study Timeline

• Study First Submitted: 2010-12-20
• Study First Submitted QC: 2010-12-21
• Study First Posted: 2010-12-22
• Study Start: 2011-12-27
• Primary Completion: 2015-04-24
• Study Completion: 2015-04-24
• Results First Submitted: 2017-08-10
• Results First Submitted QC: 2017-10-03
• Results First Posted: 2017-11-06
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-31
• Last Update Posted: 2022-04-28

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 8

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ferumoxytol	Ferumoxytol	When a participant has persistent or recurrent IDA (defined as hemoglobin \<12.0 grams \[g\]/deciliter \[dL\] and with either transferrin saturation \<40% or ferritin \<100 nanograms/milliliter), the participant will begin a 7-week treatment period. Participants will receive 2 IV injections of ferumoxytol 7.0 milligrams (mg) iron/kilogram (maximum of 510 mg/dose), the first dose administered on Day 1 and the second on Days 3 through 9 of the Treatment Period.

Primary Outcome Measures

Name	Time	Method
Mean Change In Hemoglobin From Baseline To Week 5	Baseline, Week 5	Mean changes in hemoglobin following the first course of ferumoxytol 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, and the AMAG-FER-CKD-253 study. 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

Name	Time	Method
Proportion Of Participants With An Increase In Hemoglobin From Baseline To Week 5 And To Week 7	Baseline, Week 5 and Week 7	The proportion of participants with an increase hemoglobin to ≥1.0 g/dL or to ≥12.0 g/dL during the period from Baseline to Week 5 and to Week 7 following each course of ferumoxytol was to be presented. However, 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, and the AMAG-FER-CKD-253 study. 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.
Mean Change In TSAT From Baseline To Week 5 And To Week 7	Baseline, Week 5 and Week 7	Mean changes in TSAT following the first course of ferumoxytol from Baseline to Week 5 and to Week 7 were to be presented. However, 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, and the AMAG-FER-CKD-253 study. 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.

Trial Locations

Locations (1)

AMAG Pharmaceuticals, Inc.; 🇺🇸 Waltham, Massachusetts, United States