Trial Outcomes & Findings for A Trial of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia (NCT NCT01114217)

NCT ID: NCT01114217

Last Updated: 2022-04-21

Results Overview

Mean change in hemoglobin from TP Baseline (Day 1) to TP Week 5 following the first dose of ferumoxytol was calculated as: Hemoglobin Change = Hemoglobin (TP Week 5) - Hemoglobin (TP Baseline) TP Baseline was the most recent value measured on/after the screening or the closest monthly evaluation visit prior to Day 1 dosing of Course 1. Change from Baseline used an imputed value of 0 for missing values at the post-baseline visit.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 634 participants
• Primary outcome timeframe: TP Baseline (Day 1), TP Week 5
• Results posted on: 2022-04-21

Participant Flow

Participants who previously enrolled in and completed AMAG-FER-IDA-301 [NCT01114139], received any dose of study drug, and met the inclusion/exclusion criteria were eligible to enroll in this Extension Study AMAG-FER-IDA-303.

Participant milestones

Measure	Received Ferumoxytol in IDA-303 Participants received ferumoxytol or placebo during AMAG-FER-IDA-301 \[NCT01114139\]. Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent iron deficiency anemia (IDA), defined as hemoglobin \<11.0 grams per deciliter (g/dL) and transferrin saturation (TSAT) \<20% at any monthly evaluation visit (with the exception of the Study Termination visit), began a 5-week Treatment Period (TP) and received a total of 2 doses of ferumoxytol 510 milligrams (mg) intravenously (IV). The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 grams (g).	Did Not Receive Ferumoxytol in IDA-303 Participants who received ferumoxytol or placebo during AMAG-FER-IDA-301 \[NCT01114139\] and did not receive ferumoxytol during AMAG-FER-IDA-303.
Overall Study STARTED	337	297
Overall Study Received at Least 1 Dose of Ferumoxytol	337	0
Overall Study COMPLETED	262	199
Overall Study NOT COMPLETED	75	98

Reasons for withdrawal

Measure	Received Ferumoxytol in IDA-303 Participants received ferumoxytol or placebo during AMAG-FER-IDA-301 \[NCT01114139\]. Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent iron deficiency anemia (IDA), defined as hemoglobin \<11.0 grams per deciliter (g/dL) and transferrin saturation (TSAT) \<20% at any monthly evaluation visit (with the exception of the Study Termination visit), began a 5-week Treatment Period (TP) and received a total of 2 doses of ferumoxytol 510 milligrams (mg) intravenously (IV). The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 grams (g).	Did Not Receive Ferumoxytol in IDA-303 Participants who received ferumoxytol or placebo during AMAG-FER-IDA-301 \[NCT01114139\] and did not receive ferumoxytol during AMAG-FER-IDA-303.
Overall Study Adverse Event	5	3
Overall Study Lost to Follow-up	17	33
Overall Study Withdrawal by Subject	21	28
Overall Study Other-Clerical Error	3	0
Overall Study Other-Early termination	6	10
Overall Study Other-Procedure	1	1
Overall Study Other-Pregnancy	2	4
Overall Study Other-Protocol Violation	1	3
Overall Study Other-Lack of Efficacy	1	0
Overall Study Other-Non Compliance	2	0
Overall Study Other-Physician Decision	1	1
Overall Study Other-Sponsor Decision	10	12
Overall Study Other-Study Termination	0	1
Overall Study Other-Study Withdrawn	1	1
Overall Study Other-Site Stopped Communicating	2	0
Overall Study Other-Lost to Follow-up	1	0
Overall Study Other-Withdrawal by Subject	1	1

Baseline Characteristics

A Trial of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia

Baseline characteristics by cohort

Measure	Received Ferumoxytol in IDA-303 n=337 Participants Participants received ferumoxytol or placebo during AMAG-FER-IDA-301 \[NCT01114139\]. Participants in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.	Did Not Receive Ferumoxytol in IDA-303 n=297 Participants Participants who received ferumoxytol or placebo during AMAG-FER-IDA-301 \[NCT01114139\] and did not receive ferumoxytol during AMAG-FER-IDA-303.	Total n=634 Participants Total of all reporting groups
Age, Continuous	46.0 years STANDARD_DEVIATION 13.33 • n=5 Participants	43.8 years STANDARD_DEVIATION 13.26 • n=7 Participants	44.9 years STANDARD_DEVIATION 13.33 • n=5 Participants
Sex: Female, Male Female	304 Participants n=5 Participants	269 Participants n=7 Participants	573 Participants n=5 Participants
Sex: Female, Male Male	33 Participants n=5 Participants	28 Participants n=7 Participants	61 Participants n=5 Participants

PRIMARY outcome

Timeframe: TP Baseline (Day 1), TP Week 5

Population: ITT Population: Participants who received at least 1 dose of ferumoxytol and had evaluable data for hemoglobin at TP Baseline and TP Week 5 in AMAG-FER-IDA-303.

Mean change in hemoglobin from TP Baseline (Day 1) to TP Week 5 following the first dose of ferumoxytol was calculated as: Hemoglobin Change = Hemoglobin (TP Week 5) - Hemoglobin (TP Baseline) TP Baseline was the most recent value measured on/after the screening or the closest monthly evaluation visit prior to Day 1 dosing of Course 1.

Change from Baseline used an imputed value of 0 for missing values at the post-baseline visit.

Outcome measures

Measure

Ferumoxytol n=151 Participants Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.

Mean Change In Hemoglobin From TP Baseline To TP Week 5 Following The First Course Of Ferumoxytol

2.6 g/dL Standard Deviation 1.55

SECONDARY outcome

Timeframe: TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Population: ITT Population: Participants who received at least 1 dose of ferumoxytol and had evaluable data for hemoglobin at TP Baseline and TP Week 5 in AMAG-FER-IDA-303.

Mean change in hemoglobin from TP Baseline to TP Week 5 following each course of ferumoxytol after the first course was calculated for each participant as:

Hemoglobin Change = Hemoglobin (TP Week 5) - Hemoglobin (TP Baseline) The first course of treatment with ferumoxytol for participants who had previously received placebo in AMAG-FER-IDA-301 was considered Course 1. The first course of treatment with ferumoxytol for participants who had previously received ferumoxytol in AMAG-FER-IDA-301 was considered Course 2; subsequent treatment courses were serially numbered.

Outcome measures

Measure	Ferumoxytol n=337 Participants Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.
Mean Change In Hemoglobin Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol After The First Course Course 1	2.6 g/dL Standard Deviation 1.55
Mean Change In Hemoglobin Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol After The First Course Course 2	1.5 g/dL Standard Deviation 1.28
Mean Change In Hemoglobin Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol After The First Course Course 3	1.1 g/dL Standard Deviation 1.30

SECONDARY outcome

Timeframe: TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Population: ITT Population: Participants who received at least 1 dose of ferumoxytol and had evaluable data for hemoglobin at TP Baseline and TP Week 5 in AMAG-FER-IDA-303. Participants with no post-baseline hemoglobin values were classified as not achieving the increase. Percentages are based on the number of participants in each course.

Proportion of participants with an increase in hemoglobin ≥2.0 g/dL at any time from TP Baseline to TP Week 5 following each course of ferumoxytol. The first course of treatment with ferumoxytol for participants who had previously received placebo in AMAG-FER-IDA-301 was considered Course 1. The first course of treatment with ferumoxytol for participants who had previously received ferumoxytol in AMAG-FER-IDA-301 was considered Course 2; subsequent treatment courses were serially numbered.

Outcome measures

Measure	Ferumoxytol n=337 Participants Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.
Percentage Of Participants With An Increase In Hemoglobin ≥2.0 g/dL At Any Time From TP Baseline To TP Week 5 Course 1	78.8 percentage of participants
Percentage Of Participants With An Increase In Hemoglobin ≥2.0 g/dL At Any Time From TP Baseline To TP Week 5 Course 2	43.9 percentage of participants
Percentage Of Participants With An Increase In Hemoglobin ≥2.0 g/dL At Any Time From TP Baseline To TP Week 5 Course 3	37.7 percentage of participants

SECONDARY outcome

Timeframe: TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Population: ITT Population: Participants who received at least 1 dose of ferumoxytol and had evaluable data for hemoglobin at TP Baseline and TP Week 5 in AMAG-FER-IDA-303. Participants with no post-baseline hemoglobin values were classified as not achieving the increase. Percentages are based on the number of participants in each course.

Proportion of participants who achieved a hemoglobin level ≥12.0 g/dL at any time from TP Baseline to TP Week 5 following each course of ferumoxytol. The first course of treatment with ferumoxytol for participants who had previously received placebo in AMAG-FER-IDA-301 was considered Course 1. The first course of treatment with ferumoxytol for participants who had previously received ferumoxytol in AMAG-FER-IDA-301 was considered Course 2; subsequent treatment courses were serially numbered.

Outcome measures

Measure	Ferumoxytol n=337 Participants Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.
Percentage Of Participants Who Achieved A Hemoglobin Level ≥12.0 g/dL At Any Time From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 1	38.4 percentage of participants
Percentage Of Participants Who Achieved A Hemoglobin Level ≥12.0 g/dL At Any Time From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 2	57.0 percentage of participants
Percentage Of Participants Who Achieved A Hemoglobin Level ≥12.0 g/dL At Any Time From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 3	40.6 percentage of participants

SECONDARY outcome

Timeframe: TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Population: ITT Population: Participants who received at least 1 dose of ferumoxytol and had evaluable data for TSAT at TP Baseline and TP Week 5 in AMAG-FER-IDA-303.

Mean change in TSAT from TP Baseline to TP Week 5 following each course of ferumoxytol.

Outcome measures

Measure	Ferumoxytol n=337 Participants Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.
Mean Change In TSAT Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 2	11.7 percentage of saturation Standard Deviation 12.47
Mean Change In TSAT Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 3	7.5 percentage of saturation Standard Deviation 9.13
Mean Change In TSAT Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 1	12.8 percentage of saturation Standard Deviation 10.19

SECONDARY outcome

Timeframe: TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Population: ITT Population: Participants who received at least 1 dose of ferumoxytol and had evaluable FACIT-Fatigue Questionnaire data at TP Baseline and TP Week 5 in AMAG-FER-IDA-303.

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 questionnaire from TP Baseline to TP Week 5 following each course of ferumoxytol was calculated as:

FACIT-Fatigue Score Change = FACIT-Fatigue Score (Week 5) - FACIT-Fatigue Score (Baseline).

TP Baseline was the most recent value measured on/after the screening or the closest monthly evaluation visit prior to Day 1 dosing in each course.

If the TP Week 5 FACIT-Fatigue Score value was missing, the change from TP Baseline was conservatively imputed as zero.

Outcome measures

Measure	Ferumoxytol n=337 Participants Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.
Patient-reported Outcome Measure: Mean Change In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Questionnaire From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 1	6.9 units on a scale Standard Deviation 9.57
Patient-reported Outcome Measure: Mean Change In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Questionnaire From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 2	4.1 units on a scale Standard Deviation 8.58
Patient-reported Outcome Measure: Mean Change In Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue Questionnaire From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol Course 3	1.5 units on a scale Standard Deviation 6.87

SECONDARY outcome

Timeframe: TP Baseline (Day 1) up to TP Week 5 for Courses 1, 2, and 3

Population: ITT Population: Participants who received at least 1 dose of ferumoxytol and had evaluable data for hemoglobin at TP Baseline and TP Week 5 in AMAG-FER-IDA-303.

Days to event was defined as the days from Baseline to the first time the participant met the criteria. Participants without any post-Baseline study visits were not included in this analysis.

The first course of treatment with ferumoxytol for participants who had previously received placebo in AMAG-FER-IDA-301 was considered Course 1. The first course of treatment with ferumoxytol for participants who had previously received ferumoxytol in AMAG-FER-IDA-301 was considered Course 2; subsequent treatment courses were serially numbered.

Outcome measures

Measure	Ferumoxytol n=337 Participants Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.
Time To Hemoglobin Increase Of ≥2.0 g/dL Or To A Hemoglobin Level Of ≥12.0 g/dL From Baseline Course 1	27.8 days Interval 22.0 to 36.0
Time To Hemoglobin Increase Of ≥2.0 g/dL Or To A Hemoglobin Level Of ≥12.0 g/dL From Baseline Course 2	30.6 days Interval 22.0 to 37.0
Time To Hemoglobin Increase Of ≥2.0 g/dL Or To A Hemoglobin Level Of ≥12.0 g/dL From Baseline Course 3	30.7 days Interval 22.0 to Insufficient number of participants with events.

Adverse Events

Received Ferumoxytol

Serious events: 22 serious events

Other events: 93 other events

Deaths: 0 deaths

Serious adverse events

Measure	Received Ferumoxytol n=337 participants at risk Participants received ferumoxytol during AMAG-FER-IDA-301 \[NCT01114139\]. Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.
Cardiac disorders Atrial fibrillation	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Cardiac disorders Cardiac failure congestive	0.59% 2/337 • Number of events 2 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Gastrointestinal disorders Colitis ulcerative	0.59% 2/337 • Number of events 2 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Gastrointestinal disorders Gastrointestinal haemorrhage	0.59% 2/337 • Number of events 2 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Gastrointestinal disorders Small intestinal stenosis	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Gastrointestinal disorders Vomiting	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
General disorders Chest pain	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Hepatobiliary disorders Cholecystitis	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Hepatobiliary disorders Cirrhosis alcoholic	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Bacteraemia	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Bronchitis	0.30% 1/337 • Number of events 2 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Cellulitis	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Laryngitis	0.30% 1/337 • Number of events 2 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Osteomyelitis	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Injury, poisoning and procedural complications Head injury	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Injury, poisoning and procedural complications Incisional hernia	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Injury, poisoning and procedural complications Post procedural haematoma	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Injury, poisoning and procedural complications Postoperative fever	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Injury, poisoning and procedural complications Procedural pain	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Colon cancer metastatic	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Colon cancer stage III	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Colon neoplasm	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Nervous system disorders Vocal cord paralysis	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Pregnancy, puerperium and perinatal conditions Abortion spontaneous	0.59% 2/337 • Number of events 2 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Renal and urinary disorders Renal failure acute	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Reproductive system and breast disorders Vaginal haemorrhage	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Respiratory, thoracic and mediastinal disorders Stridor	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Respiratory, thoracic and mediastinal disorders Vocal cord disorder	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Skin and subcutaneous tissue disorders Hyperhidrosis	0.30% 1/337 • Number of events 1 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.

Other adverse events

Measure	Received Ferumoxytol n=337 participants at risk Participants received ferumoxytol during AMAG-FER-IDA-301 \[NCT01114139\]. Participants enrolled in AMAG-FER-IDA-303 who were found to have persistent or recurrent IDA, defined as hemoglobin \<11.0 g/dL and TSAT \<20% at any monthly evaluation visit, (with the exception of the Study Termination visit), began a 5-week TP and received a total of 2 doses of ferumoxytol 510 mg IV. The first IV 510 mg dose was administered on TP Day 1 (baseline) and second dose 2 to 8 (5±3) days after Dose 1, for a total cumulative dose of 1.02 g.
Gastrointestinal disorders Abdominal pain	1.5% 5/337 • Number of events 6 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Gastrointestinal disorders Constipation	2.1% 7/337 • Number of events 8 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Gastrointestinal disorders Diarrhoea	3.0% 10/337 • Number of events 10 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Gastrointestinal disorders Nausea	5.0% 17/337 • Number of events 20 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Gastrointestinal disorders Vomiting	3.0% 10/337 • Number of events 12 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
General disorders Fatigue	2.7% 9/337 • Number of events 9 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
General disorders Oedema peripheral	1.8% 6/337 • Number of events 6 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
General disorders Pyrexia	1.5% 5/337 • Number of events 5 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Nasopharyngitis	3.0% 10/337 • Number of events 10 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Sinusitis	1.5% 5/337 • Number of events 6 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Upper respiratory tract infection	1.8% 6/337 • Number of events 6 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Infections and infestations Urinary tract infection	5.6% 19/337 • Number of events 20 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Injury, poisoning and procedural complications Fall	1.2% 4/337 • Number of events 9 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Metabolism and nutrition disorders Hypokalaemia	2.1% 7/337 • Number of events 7 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Musculoskeletal and connective tissue disorders Arthralgia	1.8% 6/337 • Number of events 8 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Musculoskeletal and connective tissue disorders Back pain	2.7% 9/337 • Number of events 9 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Nervous system disorders Dizziness	3.3% 11/337 • Number of events 11 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Nervous system disorders Headache	7.1% 24/337 • Number of events 28 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Respiratory, thoracic and mediastinal disorders Cough	2.1% 7/337 • Number of events 8 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Respiratory, thoracic and mediastinal disorders Dyspnoea	1.2% 4/337 • Number of events 4 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Vascular disorders Hypertension	1.5% 5/337 • Number of events 5 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.
Nervous system disorders Dysgeusia	1.2% 4/337 • Number of events 7 The analysis of adverse events focused on treatment-emergent adverse events (TEAEs). A TEAE was defined as an event with an onset date and time on or after the first dosing start date and time, or on or after the first dosing start date if the onset time was missing. Thus, TEAEs by definition were only assessed in participants who received ferumoxytol in this extension study. Participants who did not receive ferumoxytol were not included the analysis of adverse events.

Additional Information

Medical Information

AMAG Pharmaceuticals, Inc.

Email: CTInterest@covispharma.com

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