An Open-Label Extension Study of the Safety and Efficacy of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia in Pediatric Subjects With Chronic Kidney Disease
Trial Snapshot
- Phase
- Phase 3
- Status
- Terminated
- Sponsor
- AMAG Pharmaceuticals, Inc.
- Enrollment
- 8
- Locations
- 1
- Primary Endpoint
- Mean Change In Hemoglobin From Baseline To Week 5
Study Overview
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 Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 6 Months to 17 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
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.
Intervention: Ferumoxytol (Drug)
Outcomes
Primary Outcomes
Mean Change In Hemoglobin From Baseline To Week 5
Time Frame: 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 Outcomes
- Proportion Of Participants With An Increase In Hemoglobin From Baseline To Week 5 And To Week 7(Baseline, Week 5 and Week 7)
- Mean Change In TSAT From Baseline To Week 5 And To Week 7(Baseline, Week 5 and Week 7)