Clinical trial NCT01155375: 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
Overall status: TERMINATED
Phase: PHASE3
Conditions: Iron Deficiency Anemia
Keywords: Iron deficiency anemia
Feraheme
ferumoxytol
CKD
pediatric
dialysis-dependent
nondialysis-dependent
Lead sponsor: AMAG Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2010-07-01
Enrollment: 14
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Mean Change In Hemoglobin From Baseline To Week 5
timeFrame: Baseline, Week 5
Secondary outcomes:
measure: Pharmacokinetics: Area Under The Curve Of Ferumoxytol
timeFrame: Baseline; 10, 30, 120, and 360 minutes postdose; 24, 48, and 72 hours postdose
Other outcomes:
Locations:
city: Waltham
country: United States
facility: AMAG Pharmaceuticals, Inc.
state: Massachusetts
Study officials:
References: