A Trial Comparing Ferumoxytol With Placebo for the Treatment of Iron Deficiency Anemia

AMAG Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Iron Deficiency Anemia

Treatments: Other: Placebo; Drug: Ferumoxytol

Study type: Interventional

Funder types: Industry

Identifiers

NCT01114139

AMAG-FER-IDA-301

Details and patient eligibility

About

To evaluate the efficacy and safety of intravenous (IV) ferumoxytol compared with placebo for the treatment of iron deficiency anemia (IDA).

Full description

This Phase III, randomized, double-blind, placebo-controlled, multicenter clinical study evaluated the safety and efficacy of ferumoxytol compared with placebo for the treatment of IDA, specifically in adult patients with IDA who have a history of unsatisfactory oral iron therapy or in whom oral iron could not be used. The effect of ferumoxytol on hemoglobin, iron parameters and patient reported outcomes (PROs) compared with placebo was evaluated. Investigators were blinded to key laboratory parameters that could potentially unblind the treatment arms of the study, eg, hemoglobin [Hgb], hematocrit [Hct], iron, ferritin, total iron binding capacity [TIBC], and transferrin saturation [TSAT], and neither the Investigators nor the subjects were aware of their treatment assignment, hemoglobin or other laboratory values.

Enrollment: 812 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria include:

1. Males and females ≥18 years of age

2. Participants with IDA defined as having:

   1. Hemoglobin <10.0 g/deciliter (dL)
   2. Transferrin saturation <20%

3. Participants who have a history of unsatisfactory oral iron therapy or in whom oral iron cannot be used

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 include:

1. History of allergy to IV iron
2. Allergy to two or more classes of drugs
3. Participants on dialysis or with an estimated glomerular filtration rate <30 mL/minute/1.73 m^2
4. Female participants who are pregnant, intend to become pregnant, are breastfeeding, within 2 weeks postpartum, or have a positive serum/urine pregnancy test
5. Hemoglobin ≤7.0 g/dL
6. Serum ferritin >600 nanograms/mL

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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812 participants in 2 patient groups, including a placebo group

Ferumoxytol

Experimental group

Description:

Participants received a total of 2 doses of IV ferumoxytol 510 milligrams (mg) (17 milliliters \[mL\]). The first IV 510 mg dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose, for a total cumulative dose of 1.02 grams (g).

Treatment:

Drug: Ferumoxytol

Placebo

Placebo Comparator group

Description:

Participants received a total of 2 doses of IV saline (17 mL). The first IV dose was administered on Day 1 (Baseline) and second dose 2 to 8 (5±3) days after the first dose.

Treatment:

Other: Placebo