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

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ClinicalTrials.gov Identifier: NCT01114139
Recruitment Status : Completed
First Posted : April 30, 2010
Results First Posted : June 11, 2018
Last Update Posted : June 11, 2018
Sponsor:
Information provided by (Responsible Party):
AMAG Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Iron Deficiency Anemia
Interventions Drug: Ferumoxytol
Other: Placebo
Enrollment 812

Recruitment Details The study was open to enrollment for adult participants with iron deficiency anemia (IDA), defined as hemoglobin <10.0 gram (g)/deciliter (dL) and transferrin saturation (TSAT) <20%, and a history of unsatisfactory oral iron therapy or in whom oral iron could not be used.
Pre-assignment Details  
Arm/Group Title Ferumoxytol Placebo
Hide Arm/Group Description Participants received a total of 2 doses of intravenous (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 g. 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.
Period Title: Overall Study
Started 609 203
Received at Least 1 Dose of Study Drug 608 200
Completed [1] 569 187
Not Completed 40 16
Reason Not Completed
Adverse Event             3             2
Lost to Follow-up             15             3
Withdrawal by Subject             17             6
Other-Missed 1 visit             1             0
Other-Protocol Violation             1             0
Other-Out of window for study drug dose             1             0
Other-Participant falsified records             0             1
Other-Participant went on hospice             0             1
Other-Physician changed treatment             1             0
Other-Withdrew prior to receiving dose             1             3
[1]
Completed is defined as had study drug and Week 5 visit.
Arm/Group Title Ferumoxytol Placebo Total
Hide Arm/Group Description Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 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 g. 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. Total of all reporting groups
Overall Number of Baseline Participants 608 200 808
Hide Baseline Analysis Population Description
Intent-To-Treat (ITT) Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 608 participants 200 participants 808 participants
44.8  (13.82) 46.0  (13.58) 45.1  (13.76)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 608 participants 200 participants 808 participants
Female
542
  89.1%
178
  89.0%
720
  89.1%
Male
66
  10.9%
22
  11.0%
88
  10.9%
1.Primary Outcome
Title Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5
Hide Description

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.

Time Frame Baseline (Day 1) through Week 5
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Placebo
Hide Arm/Group Description:
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 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 g.
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.
Overall Number of Participants Analyzed 608 200
Measure Type: Count of Participants
Unit of Measure: Participants
Up to Week 2
252
  41.4%
5
   2.5%
Up to Week 3
386
  63.5%
7
   3.5%
Up to Week 4
460
  75.7%
8
   4.0%
Up to Week 5
493
  81.1%
11
   5.5%
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ferumoxytol, Placebo
Comments Participants who achieved a ≥2.0 g/dL increase in hemoglobin from Baseline up to Week 5 were analyzed. Statistical comparison was performed for data up to Week 5 only. 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.
Type of Statistical Test Superiority
Comments The 95% confidence interval was calculated using the large sample assumption.
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments The p-value is the result of the Cochran-Mantel-Haenszel test, adjusted for Baseline hemoglobin level and underlying condition.
Method of Estimation Estimation Parameter Treatment Difference
Estimated Value 75.59
Confidence Interval (2-Sided) 95%
71.15 to 80.02
Estimation Comments The treatment difference (ferumoxytol - placebo) was expressed as a percentage.
2.Secondary Outcome
Title Mean Change In Hemoglobin From Baseline To Week 5
Hide Description

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.

Time Frame Baseline (Day 1), Week 5
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Placebo
Hide Arm/Group Description:
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 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 g.
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.
Overall Number of Participants Analyzed 608 200
Mean (Standard Deviation)
Unit of Measure: g/dL
2.6  (1.52) 0.1  (0.89)
3.Secondary Outcome
Title Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5
Hide Description

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.

Time Frame Baseline (Day 1) through Week 5
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Placebo
Hide Arm/Group Description:
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 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 g.
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.
Overall Number of Participants Analyzed 608 200
Measure Type: Count of Participants
Unit of Measure: Participants
Up to Week 2
33
   5.4%
3
   1.5%
Up to Week 3
131
  21.5%
5
   2.5%
Up to Week 4
240
  39.5%
5
   2.5%
Up to Week 5
307
  50.5%
6
   3.0%
4.Secondary Outcome
Title Mean Change In TSAT From Baseline To Week 5
Hide Description

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.

Time Frame Baseline (Day 1), Week 5
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Placebo
Hide Arm/Group Description:
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 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 g.
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.
Overall Number of Participants Analyzed 608 200
Mean (Standard Deviation)
Unit of Measure: percentage of saturation
11.4  (15.06) 0.4  (5.81)
5.Secondary Outcome
Title Mean Change In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Score From Baseline To Week 5
Hide Description

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.

Time Frame Baseline (Day 1), Week 5
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Placebo
Hide Arm/Group Description:
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 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 g.
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.
Overall Number of Participants Analyzed 608 200
Mean (Standard Deviation)
Unit of Measure: units on a scale
11.7  (11.73) 6.8  (9.51)
6.Secondary Outcome
Title Time To Hemoglobin Increase Of ≥2.0 g/dL Or A Hemoglobin Value Of ≥12.0 g/dL From Baseline
Hide Description 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.
Time Frame From Baseline (Day 1) up to Week 5
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or placebo) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Placebo
Hide Arm/Group Description:
Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 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 g.
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.
Overall Number of Participants Analyzed 608 200
Mean (Inter-Quartile Range)
Unit of Measure: Days
23.5
(15.0 to 29.0)
42.5 [1] 
(44 to NA)
[1]
Insufficient number of participants with events.
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Ferumoxytol Placebo
Hide Arm/Group Description Participants received a total of 2 doses of IV ferumoxytol 510 mg (17 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 g. 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.
All-Cause Mortality
Ferumoxytol Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Ferumoxytol Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   16/608 (2.63%)   6/200 (3.00%) 
Blood and lymphatic system disorders     
Anaemia  1/608 (0.16%)  3/200 (1.50%) 
Cardiac disorders     
Supraventricular Tachycardia  1/608 (0.16%)  0/200 (0.00%) 
Gastrointestinal disorders     
Colitis Ulcerative  1/608 (0.16%)  0/200 (0.00%) 
Gastrointestinal Haemorrhage  1/608 (0.16%)  0/200 (0.00%) 
Vomiting  1/608 (0.16%)  0/200 (0.00%) 
General disorders     
Chest Pain  1/608 (0.16%)  0/200 (0.00%) 
Disease Progression  1/608 (0.16%)  0/200 (0.00%) 
Immune system disorders     
Anaphylactic Reaction  1/608 (0.16%)  0/200 (0.00%) 
Hypersensitivity  3/608 (0.49%)  0/200 (0.00%) 
Infections and infestations     
Gastroenteritis  1/608 (0.16%)  0/200 (0.00%) 
Gastroenteritis Viral  1/608 (0.16%)  0/200 (0.00%) 
Lobar Pneumonia  1/608 (0.16%)  0/200 (0.00%) 
Septic Shock  1/608 (0.16%)  0/200 (0.00%) 
Metabolism and nutrition disorders     
Hyperkalaemia  1/608 (0.16%)  0/200 (0.00%) 
Musculoskeletal and connective tissue disorders     
Intervertebral Disc Protrusion  1/608 (0.16%)  0/200 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Lung Neoplasm Malignant  0/608 (0.00%)  1/200 (0.50%) 
Small Intestine Carcinoma  1/608 (0.16%)  0/200 (0.00%) 
Nervous system disorders     
Convulsion  0/608 (0.00%)  1/200 (0.50%) 
Hypoaesthesia  1/608 (0.16%)  0/200 (0.00%) 
Renal and urinary disorders     
Nephrolithiasis  1/608 (0.16%)  0/200 (0.00%) 
Reproductive system and breast disorders     
Dysfunctional Uterine Bleeding  0/542 (0.00%)  1/178 (0.56%) 
Respiratory, thoracic and mediastinal disorders     
Dyspnoea  1/608 (0.16%)  0/200 (0.00%) 
Epistaxis  1/608 (0.16%)  0/200 (0.00%) 
1
Term from vocabulary, MedDRA (13.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 1%
Ferumoxytol Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   198/608 (32.57%)   56/200 (28.00%) 
Gastrointestinal disorders     
Abdominal Pain  11/608 (1.81%)  5/200 (2.50%) 
Abdominal Pain Upper  7/608 (1.15%)  1/200 (0.50%) 
Constipation  10/608 (1.64%)  3/200 (1.50%) 
Diarrhoea  17/608 (2.80%)  6/200 (3.00%) 
Nausea  28/608 (4.61%)  5/200 (2.50%) 
Toothache  5/608 (0.82%)  3/200 (1.50%) 
Vomiting  12/608 (1.97%)  2/200 (1.00%) 
General disorders     
Fatigue  12/608 (1.97%)  3/200 (1.50%) 
Oedema Peripheral  10/608 (1.64%)  1/200 (0.50%) 
Pyrexia  7/608 (1.15%)  1/200 (0.50%) 
Infections and infestations     
Nasopharyngitis  16/608 (2.63%)  4/200 (2.00%) 
Upper Respiratory Tract Infection  8/608 (1.32%)  2/200 (1.00%) 
Urinary Tract Infection  17/608 (2.80%)  6/200 (3.00%) 
Injury, poisoning and procedural complications     
Skin Laceration  0/608 (0.00%)  3/200 (1.50%) 
Metabolism and nutrition disorders     
Hypokalaemia  9/608 (1.48%)  0/200 (0.00%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  9/608 (1.48%)  5/200 (2.50%) 
Back Pain  10/608 (1.64%)  2/200 (1.00%) 
Muscle Spasms  7/608 (1.15%)  2/200 (1.00%) 
Pain in Extremity  7/608 (1.15%)  1/200 (0.50%) 
Nervous system disorders     
Dizziness  24/608 (3.95%)  7/200 (3.50%) 
Dysgeusia  9/608 (1.48%)  1/200 (0.50%) 
Headache  35/608 (5.76%)  12/200 (6.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough  7/608 (1.15%)  1/200 (0.50%) 
Dyspnoea  9/608 (1.48%)  4/200 (2.00%) 
Skin and subcutaneous tissue disorders     
Pruritus  3/608 (0.49%)  3/200 (1.50%) 
Rash  12/608 (1.97%)  0/200 (0.00%) 
Vascular disorders     
Hypertension  9/608 (1.48%)  0/200 (0.00%) 
Hypotension  8/608 (1.32%)  1/200 (0.50%) 
1
Term from vocabulary, MedDRA (13.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
If there is no multi-site publication within 18 months after the Study has been completed or terminated at all Study sites, and all data have been received by Sponsor, the Site, and SMO shall have the right to publish its results from the Study for non-commercial purposes, if submitted to Sponsor for review 60 days prior to submission of publication. Publication must remove all confidential information and may be delayed by up to 180 days to allow Sponsor to protect its interests.
Results Point of Contact
Name/Title: Medical Information
Organization: AMAG Pharmaceuticals, Inc.
Phone: +1-877-411-2510
Responsible Party: AMAG Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01114139     History of Changes
Other Study ID Numbers: AMAG-FER-IDA-301
First Submitted: April 29, 2010
First Posted: April 30, 2010
Results First Submitted: March 26, 2018
Results First Posted: June 11, 2018
Last Update Posted: June 11, 2018