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

Overview

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.

Full Title of Study: “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”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: April 24, 2015

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.

Interventions

  • Drug: Ferumoxytol
    • IV Ferumoxytol

Arms, Groups and Cohorts

  • Experimental: 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.

Clinical Trial Outcome Measures

Primary Measures

  • 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 Measures

  • Proportion Of Participants With An Increase In Hemoglobin From Baseline To Week 5 And To Week 7
    • Time Frame: 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
    • Time Frame: 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.

Participating in This Clinical Trial

Key Inclusion Criteria include: 1. Participants who had completed participation in the combined AMAG-FER-CKD-251 and AMAG-FER-CKD-252 studies within 4 weeks of screening 2. Female participants of childbearing potential who are sexually active must be on an effective method of birth control and agree to remain on birth control until completion of participation in the study 3. Participant and/or legal guardian is capable of understanding and complying with the protocol requirements and is available for the duration of the study Key Exclusion Criteria include: 1. Experienced a serious adverse event related to IV iron therapy in the combined AMAG-FER-CKD-251 and AMAG-FER- CKD-252 studies 2. Hemoglobin level ≤7 g/dL

Gender Eligibility: All

Minimum Age: 6 Months

Maximum Age: 17 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • AMAG Pharmaceuticals, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.