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

AMAG Pharmaceuticals

Status and phase: Terminated; Phase 3

Conditions: Iron Deficiency Anemia; Nondialysis-dependent Chronic Kidney Disease

Treatments: Drug: Oral Iron; Drug: Ferumoxytol

Study type: Interventional

Funder types: Industry

Identifiers

NCT01155388

AMAG-FER-CKD-252

Details and patient eligibility

About

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).

Full description

Study AMAG-FER-CKD-252 was a study evaluating the efficacy and safety of IV ferumoxytol in pediatric participants with nondialysis-dependent CKD. Study AMAG-FER-CKD-251 (NCT01155375) was a study evaluating the efficacy and safety of IV ferumoxytol in pediatric participants with dialysis-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.

Enrollment: 14 patients

Sex: All

Ages: 6 months to 17 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria for this study include:

1. Males or females 6 months to <18 years of age
2. Nondialysis dependent CKD, including kidney transplant recipients
3. Has iron deficiency anemia defined as: a) hemoglobin level <11.0 grams (g)/deciliter (dL) and b) transferrin saturation level <20%
4. Female participants of childbearing potential who are sexually active must be on an effective method of birth control for at least 1 month prior to Screening and agree to remain on birth control until completion of participation in the study

Key Exclusion Criteria for this study include:

1. History of allergy to either oral or IV iron
2. Allergy to two or more classes of drugs
3. Female participants who are pregnant, intend to become pregnant, are breastfeeding, within 3 months postpartum, or have a positive serum/urine pregnancy test
4. Hemoglobin level ≤7.0 g/dL
5. Serum ferritin level >600 nanograms/milliliter

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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14 participants in 2 patient groups

Ferumoxytol

Experimental group

Description:

Participants will receive 1 of the following 2 ferumoxytol dose regimens: * Four IV injections of ferumoxytol 3.5 mg Fe/kg (maximum of 255 mg/dose) administered on nonconsecutive days within a 14-day period as follows: Day 1 (dose 1), Days 3\* through 10 (dose 2), Days 5 through 12 (dose 3), and Days 7 through 14 (dose 4). \*Participants participating in PK sampling received the second dose on Day 4 after the 72-hour PK sample was collected. * Two IV injections of ferumoxytol 7.0 mg Fe/kg (maximum of 510 mg/dose), the first administered on Day 1 and the second on Days 3 through 9.

Treatment:

Drug: Ferumoxytol

Oral Iron

Active Comparator group

Description:

Participants will receive oral iron: 2.5 mg Fe/kg twice daily (maximum of 100 mg/dose) on Days 1 through 35.

Treatment:

Drug: Oral Iron