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

A Phase III, Randomized, Double-Blind, Placebo-Controlled Trial of Ferumoxytol for the Treatment of Iron Deficiency Anemia

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

Study Overview

• Status: Completed
• Conditions: Iron Deficiency Anemia
• Intervention / Treatment: Drug: Ferumoxytol; Other: Placebo

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 812
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada
      • Clinical Trial Site
  • New Brunswick
    • Saint John, New Brunswick, Canada
      • Clinical Trial Site
  • Ontario
    • London, Ontario, Canada
      • Clinical Trial Site
    • Scarborough, Ontario, Canada
      • Clinical Trial Site
    • Thornhill, Ontario, Canada
      • Clinical Trial Site
    • Vaughan, Ontario, Canada
      • Clinical Trial Site
  • Quebec
    • Pointe-Claire, Quebec, Canada
      • Clinical Trial Site
• Hungary
  • Békéscsaba, Hungary
    • Clinical Trial Site
  • Gyula, Hungary
    • Clinical Trial Site
  • Komárom, Hungary
    • Clinical Trial Site
  • Szekszárd, Hungary
    • Clinical Trial Site
  • Vác, Hungary
    • Clinical Trial Site
• India
  • Andhra Pradesh
    • Hyderabad, Andhra Pradesh, India
      • Clinical Trial Site
    • Secunderabad, Andhra Pradesh, India
      • Clinical Trial Site
  • Andhrapradesh
    • Visakhapatnam, Andhrapradesh, India
      • Clinical Trial Site
  • Assam
    • Guwahati, Assam, India
      • Clinical Trial Site
  • Karnataka
    • Bangalore, Karnataka, India, 560002
      • Clinical Trial Site
    • Bangalore, Karnataka, India
      • Clinical Trial Site
    • Mangalore, Karnataka, India
      • Clinical Trial Site
  • Maharashtra
    • Aurangabad, Maharashtra, India
      • Clinical Trial Site
    • Nagpur, Maharashtra, India
      • Clinical Trial Site
    • Nashik, Maharashtra, India
      • Clinical Trial Site
    • Pune, Maharashtra, India
      • Clinical Trial Site
  • Rajasthan
    • Jaipur, Rajasthan, India, 302001
      • Clinical Trial Site
    • Jaipur, Rajasthan, India, 302013
      • Clinical Trial Site
  • Tamil Nadu
    • Chennai, Tamil Nadu, India, 600096
      • Clinical Trial Site
    • Chennai, Tamil Nadu, India
      • Clinical Trial Site
    • Coimbatore, Tamil Nadu, India
      • Clinical Trial Site
    • Madurai, Tamil Nadu, India
      • Clinical Trial Site
  • Uttar Pradesh
    • Lucknow, Uttar Pradesh, India, 226003
      • Clinical Trial Site
    • Lucknow, Uttar Pradesh, India
      • Clinical Trial Site
• Latvia
  • Daugavpils, Latvia
    • Clinical Trial Site
  • Riga, Latvia, LV-1002
    • Clinical Trial Site
  • Riga, Latvia, LV-1005
    • Clinical Trial Site
  • Riga, Latvia, LV-1006
    • Clinical Trial Site
  • Riga, Latvia, LV-1010
    • Clinical Trial Site
  • Valmiera, Latvia, LV-4201
    • Clinical Trial Site
  • Ventspils, Latvia, LV-3601
    • Clinical Trial Site
  • Ventspils, Latvia
    • Clinical Trial Site
• Poland
  • Białystok, Poland
    • Clinical Trial Site
  • Sopot, Poland
    • Clinical Trial Site
  • Warszawa, Poland, 02-341
    • Clinical Trial Site
  • Warszawa, Poland, 03-580
    • Clinical Trial Site
  • Wrocław, Poland
    • Clinical Trial Site
  • Zgierz, Poland
    • Clinical Trial Site
• United States
  • Alabama
    • Birmingham, Alabama, United States
      • Clinical Trial Site
    • Mobile, Alabama, United States
      • Clinical Trial Site
    • Montgomery, Alabama, United States, 36106
      • Clinical Trial Site
    • Montgomery, Alabama, United States
      • Clinical Trial Site
  • Arizona
    • Green Valley, Arizona, United States
      • Clinical Trial Site
    • Phoenix, Arizona, United States, 85032
      • Clinical Trial Site
    • Phoenix, Arizona, United States
      • Clinical Trial Site
    • Tucson, Arizona, United States, 85710
      • Clinical Trial Site
    • Tucson, Arizona, United States
      • Clinical Trial Site
  • California
    • Alhambra, California, United States
      • Clinical Trial Site
    • Anaheim, California, United States
      • Clinical Trial Site
    • Bakersfield, California, United States
      • Clinical Trial Site
    • Buena Park, California, United States
      • Clinical Trial Site
    • Colton, California, United States
      • Clinical Trial Site
    • Fresno, California, United States
      • Clinical Trial Site
    • Glendale, California, United States
      • Clinical Trial Site
    • Laguna Hills, California, United States, 92653
      • Clinical Trial Site
    • Laguna Hills, California, United States
      • Clinical Trial Site
    • Lakewood, California, United States
      • Clinical Trial Site
    • Los Angeles, California, United States, 90036
      • Clinical Trial Site
    • Los Angeles, California, United States, 90057
      • Clinical Trial Site
    • Los Angeles, California, United States
      • Clinical Trial Site
    • Mission Hills, California, United States
      • Clinical Trial Site
    • Orange, California, United States
      • Clinical Trial Site
    • San Diego, California, United States, 92103
      • Clinical Trial Site
    • San Diego, California, United States, 92123
      • Clinical Trial Site
    • San Diego, California, United States
      • Clinical Trial Site
  • Colorado
    • Pueblo, Colorado, United States
      • Clinical Trial Site
  • Connecticut
    • Bristol, Connecticut, United States
      • Clinical Trial Site
    • Groton, Connecticut, United States
      • Clinical Trial Site
  • Delaware
    • Newark, Delaware, United States
      • Clinical Trial Site
  • Florida
    • Boynton Beach, Florida, United States, 33426
      • Clinical Trial Site
    • Boynton Beach, Florida, United States
      • Clinical Trial Site
    • Clearwater, Florida, United States, 33759
      • Clinical Trial Site
    • Clearwater, Florida, United States
      • Clinical Trial Site
    • Hialeah, Florida, United States
      • Clinical Trial Site
    • Holiday, Florida, United States
      • Clinical Trial Site
    • Inverness, Florida, United States
      • Clinical Trial Site
    • Margate, Florida, United States
      • Clinical Trial Site
    • Miami, Florida, United States, 33126
      • Clinical Trial Site
    • Miami, Florida, United States, 33143
      • Clinical Trial Site
    • Miami, Florida, United States, 33144
      • Clinical Trial Site
    • Miami, Florida, United States, 33175
      • Clinical Trial Site
    • Miami, Florida, United States
      • Clinical Trial Site
    • Miami Lakes, Florida, United States
      • Clinical Trial Site
    • Naples, Florida, United States
      • Clinical Trial Site
    • Ocala, Florida, United States
      • Clinical Trial Site
    • Vero Beach, Florida, United States
      • Clinical Trial Site
    • Wellington, Florida, United States, 33414
      • Clinical Trial Site
    • Wellington, Florida, United States
      • Clinical Trial Site
    • West Palm Beach, Florida, United States
      • Clinical Trial Site
    • Winter Park, Florida, United States
      • Clinical Trial Site
    • Zephyrhills, Florida, United States
      • Clinical Trial Site
  • Georgia
    • Atlanta, Georgia, United States, 30308
      • Clinical Trial Site
    • Atlanta, Georgia, United States, 30342
      • Clinical Trial Site
    • Atlanta, Georgia, United States
      • Clinical Trial Site
    • Columbus, Georgia, United States
      • Clinical Trial Site
    • Decatur, Georgia, United States
      • Clinical Trial Site
    • Dublin, Georgia, United States
      • Clinical Trial Site
    • Rome, Georgia, United States
      • Clinical Trial Site
    • Sandy Springs, Georgia, United States
      • Clinical Trial Site
    • Savannah, Georgia, United States
      • Clinical Trial Site
    • Stockbridge, Georgia, United States
      • Clinical Trial Site
  • Illinois
    • Aurora, Illinois, United States
      • Clinical Trial Site
    • Chicago, Illinois, United States, 60611
      • Clinical Trial Site
    • Chicago, Illinois, United States
      • Clinical Trial Site
    • Evergreen Park, Illinois, United States
      • Clinical Trial Site
    • Skokie, Illinois, United States, 60076
      • Clinical Trial Site
    • Skokie, Illinois, United States
      • Clinical Trial Site
    • Springfield, Illinois, United States
      • Clinical Trial Site
  • Indiana
    • Indianapolis, Indiana, United States
      • Clinical Trial Site
  • Kansas
    • Wichita, Kansas, United States
      • Clinical Trial Site
  • Louisiana
    • New Orleans, Louisiana, United States
      • Clinical Trial Site
  • Maryland
    • Bethesda, Maryland, United States
      • Clinical Trial Site
    • Hollywood, Maryland, United States
      • Clinical Trial Site
    • Prince Frederick, Maryland, United States
      • Clinical Trial Site
  • Massachusetts
    • Waltham, Massachusetts, United States, 02451
      • AMAG Pharmaceuticals, Inc.
  • Michigan
    • Bay City, Michigan, United States, 48706
      • Clinical Trial Site
    • Bay City, Michigan, United States
      • Clinical Trial Site
    • Saginaw, Michigan, United States
      • Clinical Trial Site
    • Wyoming, Michigan, United States
      • Clinical Trial Site
  • Missouri
    • Kansas City, Missouri, United States
      • Clinical Trial Site
  • Nevada
    • Las Vegas, Nevada, United States
      • Clinical Trial Site
  • New Jersey
    • Lawrenceville, New Jersey, United States
      • Clinical Trial Site
    • Neptune, New Jersey, United States
      • Clinical Trial Site
    • Plainsboro, New Jersey, United States
      • Clinical Trial Site
    • Somerville, New Jersey, United States
      • Clinical Trial Site
    • Voorhees, New Jersey, United States
      • Clinical Trial Site
  • New Mexico
    • Albuquerque, New Mexico, United States
      • Clinical Trial Site
  • New York
    • Brooklyn, New York, United States
      • Clinical Trial Site
    • Goshen, New York, United States
      • Clinical Trial Site
    • New York, New York, United States, 10038
      • Clinical Trial Site
    • New York, New York, United States
      • Clinical Trial Site
  • North Carolina
    • Raleigh, North Carolina, United States
      • Clinical Trial Site
    • Wilmington, North Carolina, United States
      • Clinical Trial Site
    • Winston-Salem, North Carolina, United States
      • Clinical Trial Site
  • North Dakota
    • Bismarck, North Dakota, United States
      • Clinical Trial Site
  • Ohio
    • Akron, Ohio, United States
      • Clinical Trial Site
    • Beavercreek, Ohio, United States
      • Clinical Trial Site
    • Canton, Ohio, United States
      • Clinical Trial Site
    • Carlisle, Ohio, United States
      • Clinical Trial Site
    • Cincinnati, Ohio, United States, 45202
      • Clinical Trial Site
    • Cincinnati, Ohio, United States
      • Clinical Trial Site
    • Columbus, Ohio, United States
      • Clinical Trial Site
    • Dayton, Ohio, United States
      • Clinical Trial Site
    • Marion, Ohio, United States, 43302
      • Clinical Trial Site
    • Marion, Ohio, United States
      • Clinical Trial Site
    • Mentor, Ohio, United States
      • Clinical Trial Site
    • Middletown, Ohio, United States
      • Clinical Trial Site
    • Zanesville, Ohio, United States
      • Clinical Trial Site
  • Oklahoma
    • Norman, Oklahoma, United States
      • Clinical Trial Site
  • Pennsylvania
    • Jenkintown, Pennsylvania, United States
      • Clinical Trial Site
    • Levittown, Pennsylvania, United States
      • Clinical Trial Site
  • Rhode Island
    • East Providence, Rhode Island, United States
      • Clinical Trial Site
  • South Carolina
    • Columbia, South Carolina, United States
      • Clinical Trial Site
    • Greer, South Carolina, United States
      • Clinical Trial Site
    • Myrtle Beach, South Carolina, United States
      • Clinical Trial Site
    • North Charleston, South Carolina, United States
      • Clinical Trial Site
    • Orangeburg, South Carolina, United States
      • Clinical Trial Site
  • South Dakota
    • Rapid City, South Dakota, United States
      • Clinical Trial Site
  • Tennessee
    • Nashville, Tennessee, United States
      • Clinical Trial Site
  • Texas
    • Arlington, Texas, United States
      • Clinical Trial Site
    • Dallas, Texas, United States
      • Clinical Trial Site
    • Houston, Texas, United States, 77030
      • Clinical Trial Site
    • Houston, Texas, United States, 77074
      • Clinical Trial Site
    • Houston, Texas, United States
      • Clinical Trial Site
    • Laredo, Texas, United States
      • Clinical Trial Site
    • Longview, Texas, United States
      • Clinical Trial Site
    • San Antonio, Texas, United States, 78205
      • Clinical Trial Site
    • San Antonio, Texas, United States, 78209
      • Clinical Trial Site
    • San Antonio, Texas, United States, 78229
      • Clinical Trial Site
    • San Antonio, Texas, United States, 78258
      • Clinical Trial Site
    • San Antonio, Texas, United States
      • Clinical Trial Site
    • Spring, Texas, United States
      • Clinical Trial Site
  • Utah
    • Orem, Utah, United States
      • Clinical Trial Site
  • Virginia
    • Chesapeake, Virginia, United States
      • Clinical Trial Site
    • Norfolk, Virginia, United States
      • Clinical Trial Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ferumoxytol; 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).	Drug: Ferumoxytol; IV Ferumoxytol; Other Names: Feraheme
Placebo Comparator: Placebo; 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.	Other: Placebo; IV Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5	Participants who achieved a ≥2.0 g/dL increase in hemoglobin at any time from Baseline up to Week 5 are presented. Increase in hemoglobin at any time from Baseline up to Week 5 was calculated for each participant based on: Hemoglobin Change = Hemoglobin (Week X) - Hemoglobin (Baseline), where Week X was any post-Baseline visit up to and including Week 5. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. Participants with no post-Baseline hemoglobin values were classified as not achieving a ≥2.0 g/dL increase. Statistical analysis was performed for data up to Week 5 only.	Baseline (Day 1) through Week 5

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean Change In Hemoglobin From Baseline To Week 5	Mean change in hemoglobin from Baseline to Week 5 was calculated for each participant as: Hemoglobin Change = Hemoglobin (Week X) - Hemoglobin (Baseline), where Week X was any post-Baseline visit up to and including Week 5. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. If the Week 5 hemoglobin value was missing, the change from Baseline was imputed to be zero. Participants without any post-Baseline hemoglobin values were treated as non-responders.	Baseline (Day 1), Week 5
Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5	Participants who achieved a ≥12.0 g/dL hemoglobin level at any time from Baseline up to Week 5 are presented. Increase in hemoglobin at any time from Baseline up to Week 5 was calculated for each participant based on: Hemoglobin Change = Hemoglobin (Week X) - Hemoglobin (Baseline), where Week X was any post-Baseline visit up to and including Week 5. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. Participants without any post-Baseline hemoglobin values were treated as non-responders.	Baseline (Day 1) through Week 5
Mean Change In TSAT From Baseline To Week 5	Mean change in TSAT from Baseline to Week 5 was calculated for each participant as: TSAT Change = TSAT (Week 5) - TSAT (Baseline). TSAT, measured as a percentage, was part of the iron panel laboratory evaluations. Of the transferrin available to bind iron, this value indicates how much serum iron is bound. For example, a value of 20% means that 20% of iron-binding sites of transferrin are being occupied by iron. Baseline was defined as the Day 1 value (prior to injection of study drug). The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. If the Week 5 TSAT value was missing, the change from Baseline was imputed to be zero.	Baseline (Day 1), Week 5
Mean Change In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Score From Baseline To Week 5	The FACIT-Fatigue questionnaire is a 13-item questionnaire designed and validated to specifically assess the presence and impact of treatment on fatigue and related symptoms, such as tiredness, on health-related quality of life in anemic participants with cancer. The questionnaire has 13 items, each measured on a 4-point Likert scale. Scoring ranges from 0 (the most fatigued) to 52 (the least fatigued) points, with higher scores representing better functioning or less fatigue. Mean change in FACIT-Fatigue Score from Baseline to Week 5 was calculated for each participant as: FACIT-Fatigue Score Change = FACIT-Fatigue Score (Week 5) - FACIT-Fatigue Score (Baseline). Baseline was defined as the Day 1 value (prior to first dose of study drug).The screening or most recent value prior to Day 1 was used for any participant with missing Day 1 information. If the Week 5 FACIT-Fatigue Score value was missing, the change from Baseline was imputed to be zero.	Baseline (Day 1), Week 5
Time To Hemoglobin Increase Of ≥2.0 g/dL Or A Hemoglobin Value Of ≥12.0 g/dL From Baseline	The time to hemoglobin increase of ≥2.0 g/dL or hemoglobin value of ≥12.0 g/dL was defined as the days from Baseline (Day 1) to the first time the participant had an increase in hemoglobin of ≥2.0 g/dL or hemoglobin value of ≥12.0 g/dL, and was calculated using a Kaplan-Meier curve. Participants who did not have a hemoglobin increase of ≥2.0 g/dL or to a hemoglobin level ≥12.0 g/dL were censored at their last visit day. Participants without any post-Baseline study visits were not included.	From Baseline (Day 1) up to Week 5

Collaborators and Investigators

• Sponsor: AMAG Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• Vadhan-Raj S, Strauss W, Ford D, Bernard K, Boccia R, Li J, Allen LF. Efficacy and safety of IV ferumoxytol for adults with iron deficiency anemia previously unresponsive to or unable to tolerate oral iron. Am J Hematol. 2014 Jan;89(1):7-12. doi: 10.1002/ajh.23582. Epub 2013 Sep 30.

Study record dates

Study Major Dates

• Study Start (Actual): June 19, 2010
• Primary Completion (Actual): February 27, 2012
• Study Completion (Actual): October 22, 2012

Study Registration Dates

• First Submitted: April 29, 2010
• First Submitted That Met QC Criteria: April 29, 2010
• First Posted (Estimate): April 30, 2010

Study Record Updates

• Last Update Posted (Actual): April 21, 2022
• Last Update Submitted That Met QC Criteria: March 31, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Keywords: Iron deficiency anemia; Feraheme; ferumoxytol; IDA
• Additional Relevant MeSH Terms: Metabolic Diseases; Hematologic Diseases; Nutrition Disorders; Anemia, Hypochromic; Iron Metabolism Disorders; Malnutrition; Anemia, Iron-Deficiency; Anemia; Deficiency Diseases; Hematinics; Pharmaceutical Solutions; Parenteral Nutrition Solutions; Ferrosoferric Oxide
• Other Study ID Numbers: AMAG-FER-IDA-301

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No