Trial Outcomes & Findings for A Trial Comparing Ferumoxytol With Iron Sucrose for the Treatment of Iron Deficiency Anemia (NCT NCT01114204)

NCT ID: NCT01114204

Last Updated: 2022-04-21

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

605 participants

Primary outcome timeframe

Baseline (Day 1) through Week 5

Results posted on

2022-04-21

Participant Flow

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.

Participant milestones

Participant milestones
Measure
Ferumoxytol
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.
Iron Sucrose
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 Study
STARTED
406
199
Overall Study
Received at Least 1 Dose of Study Drug
406
199
Overall Study
COMPLETED
385
191
Overall Study
NOT COMPLETED
21
8

Reasons for withdrawal

Reasons for withdrawal
Measure
Ferumoxytol
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.
Iron Sucrose
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 Study
Adverse Event
3
2
Overall Study
Lost to Follow-up
1
0
Overall Study
Withdrawal by Subject
10
6
Overall Study
Death
1
0
Overall Study
Other-Personal reasons
2
0
Overall Study
Other-Medical monitor request
1
0
Overall Study
Other-Participant request
2
0
Overall Study
Other-Protocol Violation
1
0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Ferumoxytol
n=406 Participants
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.
Iron Sucrose
n=199 Participants
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
n=605 Participants
Total of all reporting groups
Age, Continuous
48.0 years
STANDARD_DEVIATION 14.89 • n=5 Participants
48.9 years
STANDARD_DEVIATION 14.66 • n=7 Participants
48.2 years
STANDARD_DEVIATION 14.81 • n=5 Participants
Sex: Female, Male
Female
342 Participants
n=5 Participants
160 Participants
n=7 Participants
502 Participants
n=5 Participants
Sex: Female, Male
Male
64 Participants
n=5 Participants
39 Participants
n=7 Participants
103 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline (Day 1) through Week 5

Population: ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or iron sucrose) and was based upon randomized treatment assignment.

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.

Outcome measures

Outcome measures
Measure
Ferumoxytol
n=406 Participants
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.
Iron Sucrose
n=199 Participants
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.
Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5
Up to Week 3
291 Participants
117 Participants
Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5
Up to Week 4
327 Participants
145 Participants
Participants Who Achieved A ≥2.0 g/dL Increase In Hemoglobin At Any Time From Baseline To Week 5
Up to Week 5
341 Participants
162 Participants

SECONDARY outcome

Timeframe: Baseline (Day 1), Week 5

Population: ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or iron sucrose) and was based upon randomized treatment assignment.

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.

Outcome measures

Outcome measures
Measure
Ferumoxytol
n=406 Participants
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.
Iron Sucrose
n=199 Participants
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.
Mean Change In Hemoglobin From Baseline To Week 5
2.9 g/dL
Standard Deviation 1.62
2.7 g/dL
Standard Deviation 1.30

SECONDARY outcome

Timeframe: Baseline (Day 1) through Week 5

Population: ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or iron sucrose) and was based upon randomized treatment assignment.

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.

Outcome measures

Outcome measures
Measure
Ferumoxytol
n=406 Participants
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.
Iron Sucrose
n=199 Participants
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.
Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5
Up to Week 3
123 Participants
33 Participants
Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5
Up to Week 4
210 Participants
67 Participants
Participants Achieving A Hemoglobin Level ≥12.0 g/dL At Any Time From Baseline To Week 5
Up to Week 5
271 Participants
96 Participants

SECONDARY outcome

Timeframe: Baseline (Day 1), Week 5

Population: ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or iron sucrose) and was based upon randomized treatment assignment.

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.

Outcome measures

Outcome measures
Measure
Ferumoxytol
n=406 Participants
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.
Iron Sucrose
n=199 Participants
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.
Mean Change In TSAT From Baseline To Week 5
15.7 percentage of saturation
Standard Deviation 16.80
11.9 percentage of saturation
Standard Deviation 14.41

SECONDARY outcome

Timeframe: Baseline (Day 1), Week 5

Population: ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or iron sucrose) and was based upon randomized treatment assignment.

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.

Outcome measures

Outcome measures
Measure
Ferumoxytol
n=399 Participants
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.
Iron Sucrose
n=198 Participants
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.
Mean Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score From Baseline To Week 5
13.1 units on a scale
Standard Deviation 11.78
12.4 units on a scale
Standard Deviation 11.22

SECONDARY outcome

Timeframe: From Baseline (Day 1) up to Week 5

Population: ITT Population: Any randomized participant who had any exposure to study drug (ferumoxytol or iron sucrose) and was based upon randomized treatment assignment.

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.

Outcome measures

Outcome measures
Measure
Ferumoxytol
n=406 Participants
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.
Iron Sucrose
n=199 Participants
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.
Time To Hemoglobin Increase Of ≥2.0 g/dL Or Hemoglobin Value Of ≥12.0 g/dL From Baseline
23.1 days
Interval 15.0 to 24.0
25.2 days
Interval 21.0 to 30.0

Adverse Events

Ferumoxytol

Serious events: 17 serious events
Other events: 95 other events
Deaths: 0 deaths

Iron Sucrose

Serious events: 5 serious events
Other events: 69 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Ferumoxytol
n=406 participants at risk
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.
Iron Sucrose
n=199 participants at risk
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.
Blood and lymphatic system disorders
Anaemia
0.25%
1/406
0.50%
1/199
Blood and lymphatic system disorders
Iron deficiency anaemia
0.25%
1/406
0.00%
0/199
Cardiac disorders
Atrioventricular block second degree
0.25%
1/406
0.00%
0/199
Cardiac disorders
Tachycardia
0.25%
1/406
0.00%
0/199
Gastrointestinal disorders
Gastric ulcer haemorrhage
0.25%
1/406
0.00%
0/199
Gastrointestinal disorders
Gastrointestinal obstruction
0.25%
1/406
0.00%
0/199
Gastrointestinal disorders
Nausea
0.25%
1/406
0.00%
0/199
General disorders
Asthenia
0.25%
1/406
0.00%
0/199
General disorders
Pyrexia
0.00%
0/406
0.50%
1/199
Hepatobiliary disorders
Hepatic cirrhosis
0.25%
1/406
0.00%
0/199
Immune system disorders
Anaphylactic reaction
0.25%
1/406
0.00%
0/199
Infections and infestations
Bartholin's abscess
0.00%
0/406
0.50%
1/199
Infections and infestations
Bronchopneumonia
0.00%
0/406
0.50%
1/199
Infections and infestations
Rectal abscess
0.25%
1/406
0.00%
0/199
Infections and infestations
Viraemia
0.25%
1/406
0.00%
0/199
Injury, poisoning and procedural complications
Road traffic accident
0.25%
1/406
0.00%
0/199
Musculoskeletal and connective tissue disorders
Spinal osteoarthritis
0.25%
1/406
0.00%
0/199
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lymphoma
0.25%
1/406
0.00%
0/199
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Myelodysplastic syndrome
0.00%
0/406
0.50%
1/199
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm progression
0.00%
0/406
0.50%
1/199
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumour haemorrhage
0.25%
1/406
0.00%
0/199
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Uterine leiomyoma
0.29%
1/342
0.00%
0/160
Renal and urinary disorders
Dysuria
0.25%
1/406
0.00%
0/199
Reproductive system and breast disorders
Uterine haemorrhage
0.58%
2/342
0.00%
0/160
Skin and subcutaneous tissue disorders
Angioedema
0.25%
1/406
0.00%
0/199
Skin and subcutaneous tissue disorders
Urticaria
0.25%
1/406
0.00%
0/199
Vascular disorders
Hypertension
0.25%
1/406
0.00%
0/199

Other adverse events

Other adverse events
Measure
Ferumoxytol
n=406 participants at risk
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.
Iron Sucrose
n=199 participants at risk
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.
Gastrointestinal disorders
Nausea
2.7%
11/406
3.5%
7/199
Gastrointestinal disorders
Dry mouth
1.5%
6/406
0.00%
0/199
Gastrointestinal disorders
Abdominal pain
0.99%
4/406
1.5%
3/199
Gastrointestinal disorders
Vomiting
0.74%
3/406
2.0%
4/199
General disorders
Chest discomfort
2.2%
9/406
1.0%
2/199
General disorders
Pyrexia
0.49%
2/406
3.0%
6/199
General disorders
Chills
0.00%
0/406
2.0%
4/199
Infections and infestations
Urinary tract infection
0.49%
2/406
1.5%
3/199
Infections and infestations
Cystitis
0.25%
1/406
1.5%
3/199
Investigations
Alanine aminotransferase increased
0.99%
4/406
2.0%
4/199
Investigations
Aspartate aminotransferase increased
0.74%
3/406
1.5%
3/199
Investigations
White blood cell count decreased
0.74%
3/406
1.5%
3/199
Musculoskeletal and connective tissue disorders
Back pain
1.7%
7/406
0.50%
1/199
Musculoskeletal and connective tissue disorders
Myalgia
1.2%
5/406
0.00%
0/199
Nervous system disorders
Headache
4.7%
19/406
5.5%
11/199
Nervous system disorders
Dizziness
2.2%
9/406
1.5%
3/199
Nervous system disorders
Dysgeusia
2.2%
9/406
6.5%
13/199
Reproductive system and breast disorders
Dysmenorrhoea
0.88%
3/342
1.9%
3/160
Respiratory, thoracic and mediastinal disorders
Cough
0.74%
3/406
2.0%
4/199
Skin and subcutaneous tissue disorders
Pruritus
0.00%
0/406
2.0%
4/199
Vascular disorders
Hypertension
0.99%
4/406
1.5%
3/199
Blood and lymphatic system disorders
Anaemia
0.25%
1/406
1.0%
2/199
Ear and labyrinth disorders
Vertigo
0.49%
2/406
1.0%
2/199
Gastrointestinal disorders
Constipation
0.99%
4/406
1.0%
2/199
General disorders
Fatigue
0.74%
3/406
1.0%
2/199
General disorders
Oedema peripheral
0.74%
3/406
1.0%
2/199
General disorders
Feeling hot
0.49%
2/406
1.0%
2/199
General disorders
Injection site pain
0.00%
0/406
1.0%
2/199
Immune system disorders
Hypersensitivity
0.74%
3/406
1.0%
2/199
Immune system disorders
Drug hypersensitivity
0.49%
2/406
1.0%
2/199
Infections and infestations
Influenza
0.99%
4/406
1.0%
2/199
Infections and infestations
Nasopharyngitis
0.49%
2/406
1.0%
2/199
Injury, poisoning and procedural complications
Fall
0.00%
0/406
1.0%
2/199
Investigations
Lymphocyte count decreased
0.99%
4/406
1.0%
2/199
Investigations
Gamma-glutamyltransferase increased
0.74%
3/406
1.0%
2/199
Investigations
Blood urea decreased
0.49%
2/406
1.0%
2/199
Musculoskeletal and connective tissue disorders
Pain in extremity
0.25%
1/406
1.0%
2/199
Nervous system disorders
Somnolence
0.00%
0/406
1.0%
2/199
Vascular disorders
Hypotension
0.25%
1/406
1.0%
2/199

Additional Information

Medical Information

AMAG Pharmaceuticals, Inc.

Results disclosure agreements

  • Principal investigator is a sponsor employee 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.
  • Publication restrictions are in place

Restriction type: OTHER