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

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.

Key Inclusion Criteria for the combined AMAG-FER-CKD-251 and AMAG-FER-CDK-252 studies
include:

1. Males or females 6 months to <18 years of age

2. CKD non-dialysis participants or dialysis-dependent participants who were on
peritoneal dialysis or stable hemodialysis prior to Screening

3. 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)

4. 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:

1. History of allergy to either oral or IV iron

2. Hemoglobin level ≤7.0 g/dL

3. Serum ferritin level >600 ng/mL

4. 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