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

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ClinicalTrials.gov Identifier: NCT01114204
Recruitment Status : Completed
First Posted : May 3, 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: None (Open Label);   Primary Purpose: Treatment
Condition Iron Deficiency Anemia
Interventions Drug: Ferumoxytol
Drug: Iron Sucrose
Enrollment 605
Recruitment Details The study was open to enrollment for adult participants with iron deficiency anemia (IDA), defined as hemoglobin <10.0 grams (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 Iron Sucrose
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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period, for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries.
Period Title: Overall Study
Started 406 199
Received at Least 1 Dose of Study Drug 406 199
Completed [1] 385 191
Not Completed 21 8
Reason Not Completed
Adverse Event             3             2
Lost to Follow-up             1             0
Withdrawal by Subject             10             6
Death             1             0
Other-Personal reasons             2             0
Other-Medical monitor request             1             0
Other-Participant request             2             0
Other-Protocol Violation             1             0
[1]
Completed is defined as had study drug and Week 5 visit.
Arm/Group Title Ferumoxytol Iron Sucrose 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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries. Total of all reporting groups
Overall Number of Baseline Participants 406 199 605
Hide Baseline Analysis Population Description
Intent-to-Treat (ITT) Population: Any randomized participant who had any exposure to study drug (ferumoxytol or iron sucrose) and was based upon randomized treatment assignment.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 406 participants 199 participants 605 participants
48.0  (14.89) 48.9  (14.66) 48.2  (14.81)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 406 participants 199 participants 605 participants
Female
342
  84.2%
160
  80.4%
502
  83.0%
Male
64
  15.8%
39
  19.6%
103
  17.0%
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 iron sucrose) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Iron Sucrose
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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period, for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries.
Overall Number of Participants Analyzed 406 199
Measure Type: Count of Participants
Unit of Measure: Participants
Up to Week 3
291
  71.7%
117
  58.8%
Up to Week 4
327
  80.5%
145
  72.9%
Up to Week 5
341
  84.0%
162
  81.4%
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ferumoxytol, Iron Sucrose
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 Non-Inferiority
Comments The 95% confidence interval (CI) was calculated using the large sample assumption. The pre-defined non-inferiority margin for testing the difference between treatment groups was -15%.
Statistical Test of Hypothesis P-Value 0.2833
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 2.58
Confidence Interval (2-Sided) 95%
-3.89 to 9.06
Estimation Comments The treatment difference (ferumoxytol - iron sucrose) 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 5) – Hemoglobin (Baseline).

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.

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 iron sucrose) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Iron Sucrose
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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period, for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries.
Overall Number of Participants Analyzed 406 199
Mean (Standard Deviation)
Unit of Measure: g/dL
2.9  (1.62) 2.7  (1.30)
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 iron sucrose) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Iron Sucrose
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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period, for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries.
Overall Number of Participants Analyzed 406 199
Measure Type: Count of Participants
Unit of Measure: Participants
Up to Week 3
123
  30.3%
33
  16.6%
Up to Week 4
210
  51.7%
67
  33.7%
Up to Week 5
271
  66.7%
96
  48.2%
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).

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 iron sucrose) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Iron Sucrose
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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period, for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries.
Overall Number of Participants Analyzed 406 199
Mean (Standard Deviation)
Unit of Measure: percentage of saturation
15.7  (16.80) 11.9  (14.41)
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 iron sucrose) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Iron Sucrose
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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period, for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries.
Overall Number of Participants Analyzed 399 198
Mean (Standard Deviation)
Unit of Measure: units on a scale
13.1  (11.78) 12.4  (11.22)
6.Secondary Outcome
Title Time To Hemoglobin Increase Of ≥2.0 g/dL Or 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 iron sucrose) and was based upon randomized treatment assignment.
Arm/Group Title Ferumoxytol Iron Sucrose
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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period, for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries.
Overall Number of Participants Analyzed 406 199
Mean (Inter-Quartile Range)
Unit of Measure: days
23.1
(15.0 to 24.0)
25.2
(21.0 to 30.0)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Ferumoxytol Iron Sucrose
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 an IV injection or infusion of iron sucrose 200 mg (10 mL) on Day 1 (Baseline) and on 4 other non-consecutive days over a 14-day period for a total cumulative dose of 1.0 g. Participants receiving their first ever exposure to IV iron sucrose, received a test dose on Day 1 prior to receiving the remainder of the first dose, as prescribed in the package insert for some countries.
All-Cause Mortality
Ferumoxytol Iron Sucrose
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Ferumoxytol Iron Sucrose
Affected / at Risk (%) Affected / at Risk (%)
Total   17/406 (4.19%)   5/199 (2.51%) 
Blood and lymphatic system disorders     
Anaemia  1/406 (0.25%)  1/199 (0.50%) 
Iron deficiency anaemia  1/406 (0.25%)  0/199 (0.00%) 
Cardiac disorders     
Atrioventricular block second degree  1/406 (0.25%)  0/199 (0.00%) 
Tachycardia  1/406 (0.25%)  0/199 (0.00%) 
Gastrointestinal disorders     
Gastric ulcer haemorrhage  1/406 (0.25%)  0/199 (0.00%) 
Gastrointestinal obstruction  1/406 (0.25%)  0/199 (0.00%) 
Nausea  1/406 (0.25%)  0/199 (0.00%) 
General disorders     
Asthenia  1/406 (0.25%)  0/199 (0.00%) 
Pyrexia  0/406 (0.00%)  1/199 (0.50%) 
Hepatobiliary disorders     
Hepatic cirrhosis  1/406 (0.25%)  0/199 (0.00%) 
Immune system disorders     
Anaphylactic reaction  1/406 (0.25%)  0/199 (0.00%) 
Infections and infestations     
Bartholin's abscess  0/406 (0.00%)  1/199 (0.50%) 
Bronchopneumonia  0/406 (0.00%)  1/199 (0.50%) 
Rectal abscess  1/406 (0.25%)  0/199 (0.00%) 
Viraemia  1/406 (0.25%)  0/199 (0.00%) 
Injury, poisoning and procedural complications     
Road traffic accident  1/406 (0.25%)  0/199 (0.00%) 
Musculoskeletal and connective tissue disorders     
Spinal osteoarthritis  1/406 (0.25%)  0/199 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Lymphoma  1/406 (0.25%)  0/199 (0.00%) 
Myelodysplastic syndrome  0/406 (0.00%)  1/199 (0.50%) 
Neoplasm progression  0/406 (0.00%)  1/199 (0.50%) 
Tumour haemorrhage  1/406 (0.25%)  0/199 (0.00%) 
Uterine leiomyoma  1/342 (0.29%)  0/160 (0.00%) 
Renal and urinary disorders     
Dysuria  1/406 (0.25%)  0/199 (0.00%) 
Reproductive system and breast disorders     
Uterine haemorrhage  2/342 (0.58%)  0/160 (0.00%) 
Skin and subcutaneous tissue disorders     
Angioedema  1/406 (0.25%)  0/199 (0.00%) 
Urticaria  1/406 (0.25%)  0/199 (0.00%) 
Vascular disorders     
Hypertension  1/406 (0.25%)  0/199 (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 Iron Sucrose
Affected / at Risk (%) Affected / at Risk (%)
Total   95/406 (23.40%)   69/199 (34.67%) 
Blood and lymphatic system disorders     
Anaemia  1/406 (0.25%)  2/199 (1.01%) 
Ear and labyrinth disorders     
Vertigo  2/406 (0.49%)  2/199 (1.01%) 
Gastrointestinal disorders     
Nausea  11/406 (2.71%)  7/199 (3.52%) 
Dry mouth  6/406 (1.48%)  0/199 (0.00%) 
Abdominal pain  4/406 (0.99%)  3/199 (1.51%) 
Vomiting  3/406 (0.74%)  4/199 (2.01%) 
Constipation  4/406 (0.99%)  2/199 (1.01%) 
General disorders     
Chest discomfort  9/406 (2.22%)  2/199 (1.01%) 
Pyrexia  2/406 (0.49%)  6/199 (3.02%) 
Chills  0/406 (0.00%)  4/199 (2.01%) 
Fatigue  3/406 (0.74%)  2/199 (1.01%) 
Oedema peripheral  3/406 (0.74%)  2/199 (1.01%) 
Feeling hot  2/406 (0.49%)  2/199 (1.01%) 
Injection site pain  0/406 (0.00%)  2/199 (1.01%) 
Immune system disorders     
Hypersensitivity  3/406 (0.74%)  2/199 (1.01%) 
Drug hypersensitivity  2/406 (0.49%)  2/199 (1.01%) 
Infections and infestations     
Urinary tract infection  2/406 (0.49%)  3/199 (1.51%) 
Cystitis  1/406 (0.25%)  3/199 (1.51%) 
Influenza  4/406 (0.99%)  2/199 (1.01%) 
Nasopharyngitis  2/406 (0.49%)  2/199 (1.01%) 
Injury, poisoning and procedural complications     
Fall  0/406 (0.00%)  2/199 (1.01%) 
Investigations     
Alanine aminotransferase increased  4/406 (0.99%)  4/199 (2.01%) 
Aspartate aminotransferase increased  3/406 (0.74%)  3/199 (1.51%) 
White blood cell count decreased  3/406 (0.74%)  3/199 (1.51%) 
Lymphocyte count decreased  4/406 (0.99%)  2/199 (1.01%) 
Gamma-glutamyltransferase increased  3/406 (0.74%)  2/199 (1.01%) 
Blood urea decreased  2/406 (0.49%)  2/199 (1.01%) 
Musculoskeletal and connective tissue disorders     
Back pain  7/406 (1.72%)  1/199 (0.50%) 
Myalgia  5/406 (1.23%)  0/199 (0.00%) 
Pain in extremity  1/406 (0.25%)  2/199 (1.01%) 
Nervous system disorders     
Headache  19/406 (4.68%)  11/199 (5.53%) 
Dizziness  9/406 (2.22%)  3/199 (1.51%) 
Dysgeusia  9/406 (2.22%)  13/199 (6.53%) 
Somnolence  0/406 (0.00%)  2/199 (1.01%) 
Reproductive system and breast disorders     
Dysmenorrhoea  3/342 (0.88%)  3/160 (1.88%) 
Respiratory, thoracic and mediastinal disorders     
Cough  3/406 (0.74%)  4/199 (2.01%) 
Skin and subcutaneous tissue disorders     
Pruritus  0/406 (0.00%)  4/199 (2.01%) 
Vascular disorders     
Hypertension  4/406 (0.99%)  3/199 (1.51%) 
Hypotension  1/406 (0.25%)  2/199 (1.01%) 
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: NCT01114204     History of Changes
Other Study ID Numbers: AMAG-FER-IDA-302
First Submitted: April 29, 2010
First Posted: May 3, 2010
Results First Submitted: March 26, 2018
Results First Posted: June 11, 2018
Last Update Posted: June 11, 2018