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

AMAG Pharmaceuticals

Status and phase: Terminated; Phase 3

Conditions: Iron Deficiency Anemia

Treatments: Drug: Ferumoxytol

Study type: Interventional

Funder types: Industry

Identifiers

NCT01264679

AMAG-FER-CKD-253

Details and patient eligibility

About

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 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.

Enrollment: 8 patients

Sex: All

Ages: 6 months to 17 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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8 participants in 1 patient group

Ferumoxytol

Experimental group

Description:

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.

Treatment:

Drug: Ferumoxytol