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

• Sponsor: AMAG Pharmaceuticals, Inc. (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01114217
• Collaborator: (none)
• Enrollment: 634
• Locations: 140
• Arm: 1
• Actual Duration (Months): 32.9
• Patients Per Site: 4.5
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

To evaluate the safety and efficacy of ferumoxytol for the episodic treatment of iron deficiency anemia (IDA).

Condition or Disease	Intervention/Treatment	Phase
Iron Deficiency Anemia	Drug: Ferumoxytol	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 634 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase III, Open-Label Extension Trial of the Safety and Efficacy of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia
• Actual Study Start Date: Jul 27, 2010
• Actual Primary Completion Date: Sep 24, 2012
• Actual Study Completion Date: Apr 23, 2013

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Ferumoxytol Participants received ferumoxytol or placebo during AMAG-FER-IDA-301 [NCT01114139]. Participants enrolled in AMAG-FER-IDA-303, a 6-month Extension Study, were evaluated monthly and could receive treatment with ferumoxytol only if they met criteria defined as persistent or recurrent IDA, hemoglobin <11.0 grams per deciliter (g/dL) and transferrin saturation (TSAT) <20% at any evaluation visit, (except study termination visit). Participants who met criteria began a 5-week treatment period (TP) and received 2 doses of ferumoxytol 510 mg intravenously (IV). The first IV 510-mg dose was administered on TP Day 1 (Baseline); the second 2-8 (5±3) days after Dose 1. The first treatment course with ferumoxytol for participants who previously received placebo in AMAG-FER-IDA-301 was considered Course 1; Course 2 included participants who previously received ferumoxytol in AMAG-FER-IDA-301; subsequent treatment courses were serially numbered.

Drug: Ferumoxytol; IV Ferumoxytol; Other Names: Feraheme

Outcome Measures

Primary Outcome Measures

1. Mean Change In Hemoglobin From TP Baseline To TP Week 5 Following The First Course Of Ferumoxytol [TP Baseline (Day 1), TP Week 5]

   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.

Secondary Outcome Measures

1. 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 [TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3]

   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.

2. Percentage Of Participants With An Increase In Hemoglobin ≥2.0 g/dL At Any Time From TP Baseline To TP Week 5 [TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3]

   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.

3. 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 [TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3]

   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.

4. Mean Change In TSAT Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol [TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3]

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

5. 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 [TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3]

   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.

6. Time To Hemoglobin Increase Of ≥2.0 g/dL Or To A Hemoglobin Level Of ≥12.0 g/dL From Baseline [TP Baseline (Day 1) up to TP Week 5 for Courses 1, 2, and 3]

   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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Key Inclusion Criteria include:

1. Participants who completed participation in study AMAG-FER-IDA-301 [NCT01114139]

2. Female participants of childbearing potential who are sexually active must be on an effective method of birth control and agree to remain on birth control until completion of participation in the study

Key Exclusion Criteria include:

1. Experienced a serious adverse event (SAE) related to ferumoxytol in study AMAG-FER-IDA-301

2. Female participants who are pregnant, intend to become pregnant, are breastfeeding, or have a positive serum/urine pregnancy test

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Trial Site	Birmingham	Alabama	United States
2	Clinical Trial Site	Mobile	Alabama	United States
3	Clinical Trial Site	Montgomery	Alabama	United States	36106
4	Clinical Trial Site	Montgomery	Alabama	United States	36116
5	Clinical Trial Site	Phoenix	Arizona	United States	85015
6	Clinical Trial Site	Phoenix	Arizona	United States	85032
7	Clinical Trial Site	Tucson	Arizona	United States	85710
8	Clinical Trial Site	Tucson	Arizona	United States	85712
9	Clinical Trial Site	Anaheim	California	United States
10	Clinical Trial Site	Bakersfield	California	United States
11	Clinical Trial Site	Buena Park	California	United States
12	Clinical Trial Site	Colton	California	United States
13	Clinical Trial Site	Los Angeles	California	United States	90036
14	Clinical Trial Site	Los Angeles	California	United States	90057
15	Clinical Trial Site	Los Angeles	California	United States
16	Clinical Trial Site	Mission Hills	California	United States
17	Clinical Trial Site	Orange	California	United States
18	Clinical Trial Site	San Diego	California	United States	92103
19	Clinical Trial Site	San Diego	California	United States	92121
20	Clinical Trial Site	San Diego	California	United States	92123
21	Clinical Trial Site	Pueblo	Colorado	United States
22	Clinical Trial Site	Bristol	Connecticut	United States
23	Clinical Trial Site	Groton	Connecticut	United States
24	Clinical Trial Site	Boynton Beach	Florida	United States	33426
25	Clinical Trial Site	Boynton Beach	Florida	United States	33472
26	Clinical Trial Site	Clearwater	Florida	United States	33756
27	Clinical Trial Site	Clearwater	Florida	United States	33759
28	Clinical Trial Site	Hialeah	Florida	United States
29	Clinical Trial Site	Holiday	Florida	United States
30	Clinical Trial Site	Inverness	Florida	United States
31	Clinical Trial Site	Margate	Florida	United States
32	Clinical Trial Site	Miami Lakes	Florida	United States
33	Clinical Trial Site	Miami	Florida	United States	33126
34	Clinical Trial Site	Miami	Florida	United States	33135
35	Clinical Trial Site	Miami	Florida	United States	33143
36	Clinical Trial Site	Miami	Florida	United States	33144
37	Clinical Trial Site	Miami	Florida	United States	33175
38	Clinical Trial Site	Naples	Florida	United States
39	Clinical Trial Site	Vero Beach	Florida	United States
40	Clinical Trial Site	West Palm Beach	Florida	United States
41	Clinical Trial Site	Zephyrhills	Florida	United States
42	Clinical Trial Site	Atlanta	Georgia	United States	30308
43	Clinical Trial Site	Atlanta	Georgia	United States	30312
44	Clinical Trial Site	Atlanta	Georgia	United States	30342
45	Clinical Trial Site	Decatur	Georgia	United States
46	Clinical Trial Site	Dublin	Georgia	United States
47	Clinical Trial Site	Sandy Springs	Georgia	United States
48	Clinical Trial Site	Stockbridge	Georgia	United States
49	Clinical Trial Site	Aurora	Illinois	United States
50	Clinical Trial Site	Chicago	Illinois	United States	60616
51	Clinical Trial Site	Skokie	Illinois	United States	60076
52	Clinical Trial Site	Skokie	Illinois	United States
53	Clinical Trial Site	Springfield	Illinois	United States
54	Clinical Trial Site	Wichita	Kansas	United States
55	Clinical Trial Site	New Orleans	Louisiana	United States
56	Clinical Trial Site	Bethesda	Maryland	United States
57	Clinical Trial Site	Hollywood	Maryland	United States
58	Clinical Trial Site	Bay City	Michigan	United States	48706
59	Clinical Trial Site	Bay City	Michigan	United States
60	Clinical Trial Site	Wyoming	Michigan	United States
61	Clinical Trial Site	Kansas City	Missouri	United States
62	Clinical Trial Site	Las Vegas	Nevada	United States
63	Clinical Trial Site	Lawrenceville	New Jersey	United States
64	Clinical Trial Site	Neptune	New Jersey	United States
65	Clinical Trial Site	Plainsboro	New Jersey	United States
66	Clinical Trial Site	Voorhees	New Jersey	United States
67	Clinical Trial Site	Albuquerque	New Mexico	United States
68	Clinical Trial Site	Brooklyn	New York	United States
69	Clinical Trial Site	New York	New York	United States	10038
70	Clinical Trial Site	Raleigh	North Carolina	United States
71	Clinical Trial Site	Winston-Salem	North Carolina	United States
72	Clinical Trial Site	Canton	Ohio	United States
73	Clinical Trial Site	Carlisle	Ohio	United States
74	Clinical Trial Site	Cincinnati	Ohio	United States	45224
75	Clinical Trial Site	Cincinnati	Ohio	United States	45242
76	Clinical Trial Site	Columbus	Ohio	United States	43231
77	Clinical Trial Site	Marion	Ohio	United States	43302
78	Clinical Trial Site	Marion	Ohio	United States
79	Clinical Trial Site	Mentor	Ohio	United States
80	Clinical Trial Site	Middletown	Ohio	United States
81	Clinical Trial Site	Zanesville	Ohio	United States
82	Clinical Trial Site	Norman	Oklahoma	United States
83	Clinical Trial Site	Jenkintown	Pennsylvania	United States
84	Clinical Trial Site	Levittown	Pennsylvania	United States
85	Clinical Trial Site	Columbia	South Carolina	United States
86	Clinical Trial Site	Greer	South Carolina	United States
87	Clinical Trial Site	Myrtle Beach	South Carolina	United States
88	Clinical Trial Site	North Charleston	South Carolina	United States
89	Clinical Trial Site	Rapid City	South Dakota	United States
90	Clinical Trial Site	Arlington	Texas	United States
91	Clinical Trial Site	Dallas	Texas	United States
92	Clinical Trial Site	Houston	Texas	United States	77030
93	Clinical Trial Site	Houston	Texas	United States	77074
94	Clinical Trial Site	Houston	Texas	United States
95	Clinical Trial Site	Laredo	Texas	United States
96	Clinical Trial Site	Longview	Texas	United States
97	Clinical Trial Site	San Antonio	Texas	United States	78205
98	Clinical Trial Site	San Antonio	Texas	United States	78215
99	Clinical Trial Site	San Antonio	Texas	United States	78229
100	Clinical Trial Site	San Antonio	Texas	United States
101	Clinical Trial Site	Orem	Utah	United States
102	Clinical Trial Site	Chesapeake	Virginia	United States
103	Clinical Trial Site	Norfolk	Virginia	United States
104	Clinical Trial Site	Vancouver	British Columbia	Canada
105	Clinical Trial Site	Saint John	New Brunswick	Canada
106	Clinical Trial Site	London	Ontario	Canada
107	Clinical Trial Site	Thornhill	Ontario	Canada
108	Clinical Trial Site	Vaughan	Ontario	Canada
109	Clinical Trial Site	Pointe-Claire	Quebec	Canada
110	Clinical Trial Site	Bekescsaba		Hungary
111	Clinical Trial Site	Gyula		Hungary
112	Clinical Trial Site	Komárom		Hungary
113	Clinical Trial Site	Szekszárd		Hungary
114	Clinical Trial Site	Vác		Hungary
115	Clinical Trial Site	Hyderabad	Andhra Pradesh	India
116	Clinical Trial Site	Secunderabad	Andhra Pradesh	India
117	Clinical Trial Site	Guwahati	Assam	India
118	Clinical Trial Site	Bangalore	Karnataka	India	560002
119	Clinical Trial Site	Bangalore	Karnataka	India	560054
120	Clinical Trial Site	Aurangabad	Maharashtra	India
121	Clinical Trial Site	Nagpur	Maharashtra	India
122	Clinical Trial Site	Nashik	Maharashtra	India
123	Clinical Trial Site	Pune	Maharashtra	India
124	Clinical Trial Site	Jaipur	Rajasthan	India
125	Clinical Trial Site	Chennai	Tamil Nadu	India
126	Clinical Trial Site	Madurai	Tamil Nadu	India
127	Clinical Trial Site	Lucknow	Uttar Pradesh	India	226003
128	Clinical Trial Site	Lucknow	Uttar Pradesh	India
129	Clinical Trial Site	Daugavpils		Latvia
130	Clinical Trial Site	Riga		Latvia	LV-1002
131	Clinical Trial Site	Riga		Latvia	LV-1005
132	Clinical Trial Site	Riga		Latvia	LV-1006
133	Clinical Trial Site	Riga		Latvia	LV-1010
134	Clinical Trial Site	Valmiera		Latvia
135	Clinical Trial Site	Ventspils		Latvia	LV-3601
136	Clinical Trial Site	Ventspils		Latvia
137	Clinical Trial Site	Białystok		Poland
138	Clinical Trial Site	Sopot		Poland
139	Clinical Trial Site	Warszawa		Poland
140	Clinical Trial Site	Wrocław		Poland

Sponsors and Collaborators

• AMAG Pharmaceuticals, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

• Vadhan-Raj S, Ford DC, Dahl NV, Bernard K, Li Z, Allen LF, Strauss WE. Safety and efficacy of ferumoxytol for the episodic treatment of iron deficiency anemia in patients with a history of unsatisfactory oral iron therapy: Results of a phase III, open-label, 6-month extension study. Am J Hematol. 2016 Feb;91(2):E3-5. doi: 10.1002/ajh.24240.

• Responsible Party: AMAG Pharmaceuticals, Inc.
• ClinicalTrials.gov Identifier: NCT01114217
• Other Study ID Numbers: AMAG-FER-IDA-303
• First Posted: May 3, 2010
• Last Update Posted: Apr 21, 2022
• Last Verified: Mar 1, 2022

• Keywords provided by AMAG Pharmaceuticals, Inc.: Iron deficiency anemia; Feraheme; Ferumoxytol; IDA
• Additional relevant MeSH terms: Anemia; Anemia, Iron-Deficiency; Deficiency Diseases; Ferrosoferric Oxide

Study Results

Participant Flow

Recruitment Details	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.

Arm/Group Title	Received Ferumoxytol in IDA-303	Did Not Receive Ferumoxytol in IDA-303
Arm/Group Description	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).	Participants who received ferumoxytol or placebo during AMAG-FER-IDA-301 [NCT01114139] and did not receive ferumoxytol during AMAG-FER-IDA-303.
Period Title: Overall Study
STARTED	337	297
Received at Least 1 Dose of Ferumoxytol	337	0
COMPLETED	262	199
NOT COMPLETED	75	98

Baseline Characteristics

Arm/Group Title	Received Ferumoxytol in IDA-303	Did Not Receive Ferumoxytol in IDA-303	Total
Arm/Group Description	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.	Participants who received ferumoxytol or placebo during AMAG-FER-IDA-301 [NCT01114139] and did not receive ferumoxytol during AMAG-FER-IDA-303.	Total of all reporting groups
Overall Participants	337	297	634
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	46.0 (13.33)	43.8 (13.26)	44.9 (13.33)
Sex: Female, Male (Count of Participants)
Female	304 90.2%	269 90.6%	573 90.4%
Male	33 9.8%	28 9.4%	61 9.6%

Outcome Measures

1. Primary Outcome

Title	Mean Change In Hemoglobin From TP Baseline To TP Week 5 Following The First Course Of Ferumoxytol
Description	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.
Time Frame	TP Baseline (Day 1), TP Week 5

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Ferumoxytol
Arm/Group Description	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.
Measure Participants	151
Mean (Standard Deviation) [g/dL]	2.6 (1.55)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Ferumoxytol
	Comments
	Type of Statistical Test	Other
	Comments
Statistical Test of Hypothesis	p-Value	<0.0001
	Comments
	Method	t-test, 2 sided
	Comments

2. Secondary Outcome

Title	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
Description	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.
Time Frame	TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Ferumoxytol
Arm/Group Description	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.
Measure Participants	337
Course 1	2.6 (1.55)
Course 2	1.5 (1.28)
Course 3	1.1 (1.30)

3. Secondary Outcome

Title	Percentage Of Participants With An Increase In Hemoglobin ≥2.0 g/dL At Any Time From TP Baseline To TP Week 5
Description	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.
Time Frame	TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Ferumoxytol
Arm/Group Description	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.
Measure Participants	337
Course 1	78.8 23.4%
Course 2	43.9 13%
Course 3	37.7 11.2%

4. Secondary Outcome

Title	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
Description	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.
Time Frame	TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Ferumoxytol
Arm/Group Description	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.
Measure Participants	337
Course 1	38.4 11.4%
Course 2	57.0 16.9%
Course 3	40.6 12%

5. Secondary Outcome

Title	Mean Change In TSAT Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol
Description	Mean change in TSAT from TP Baseline to TP Week 5 following each course of ferumoxytol.
Time Frame	TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Ferumoxytol
Arm/Group Description	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.
Measure Participants	337
Course 1	12.8 (10.19)
Course 2	11.7 (12.47)
Course 3	7.5 (9.13)

6. Secondary Outcome

Title	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
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 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.
Time Frame	TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Ferumoxytol
Arm/Group Description	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.
Measure Participants	337
Course 1	6.9 (9.57)
Course 2	4.1 (8.58)
Course 3	1.5 (6.87)

7. Secondary Outcome

Title	Time To Hemoglobin Increase Of ≥2.0 g/dL Or To A Hemoglobin Level Of ≥12.0 g/dL From Baseline
Description	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.
Time Frame	TP Baseline (Day 1) up to TP Week 5 for Courses 1, 2, and 3

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Ferumoxytol
Arm/Group Description	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.
Measure Participants	337
Course 1	27.8
Course 2	30.6
Course 3	30.7

Adverse Events

Adverse Event Reporting Description	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.
Arm/Group Title	Received Ferumoxytol
Arm/Group Description	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.
All Cause Mortality
	Received Ferumoxytol
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Received Ferumoxytol
	Affected / at Risk (%)	# Events
Total	22/337 (6.5%)
Cardiac disorders
Atrial fibrillation	1/337 (0.3%)	1
Cardiac failure congestive	2/337 (0.6%)	2
Gastrointestinal disorders
Colitis ulcerative	2/337 (0.6%)	2
Gastrointestinal haemorrhage	2/337 (0.6%)	2
Small intestinal stenosis	1/337 (0.3%)	1
Vomiting	1/337 (0.3%)	1
General disorders
Chest pain	1/337 (0.3%)	1
Hepatobiliary disorders
Cholecystitis	1/337 (0.3%)	1
Cirrhosis alcoholic	1/337 (0.3%)	1
Infections and infestations
Bacteraemia	1/337 (0.3%)	1
Bronchitis	1/337 (0.3%)	2
Cellulitis	1/337 (0.3%)	1
Laryngitis	1/337 (0.3%)	2
Osteomyelitis	1/337 (0.3%)	1
Injury, poisoning and procedural complications
Head injury	1/337 (0.3%)	1
Incisional hernia	1/337 (0.3%)	1
Post procedural haematoma	1/337 (0.3%)	1
Postoperative fever	1/337 (0.3%)	1
Procedural pain	1/337 (0.3%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Colon cancer metastatic	1/337 (0.3%)	1
Colon cancer stage III	1/337 (0.3%)	1
Colon neoplasm	1/337 (0.3%)	1
Nervous system disorders
Vocal cord paralysis	1/337 (0.3%)	1
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous	2/337 (0.6%)	2
Renal and urinary disorders
Renal failure acute	1/337 (0.3%)	1
Reproductive system and breast disorders
Vaginal haemorrhage	1/337 (0.3%)	1
Respiratory, thoracic and mediastinal disorders
Dyspnoea	1/337 (0.3%)	1
Stridor	1/337 (0.3%)	1
Vocal cord disorder	1/337 (0.3%)	1
Skin and subcutaneous tissue disorders
Hyperhidrosis	1/337 (0.3%)	1
Other (Not Including Serious) Adverse Events
	Received Ferumoxytol
	Affected / at Risk (%)	# Events
Total	93/337 (27.6%)
Gastrointestinal disorders
Abdominal pain	5/337 (1.5%)	6
Constipation	7/337 (2.1%)	8
Diarrhoea	10/337 (3%)	10
Nausea	17/337 (5%)	20
Vomiting	10/337 (3%)	12
General disorders
Fatigue	9/337 (2.7%)	9
Oedema peripheral	6/337 (1.8%)	6
Pyrexia	5/337 (1.5%)	5
Infections and infestations
Nasopharyngitis	10/337 (3%)	10
Sinusitis	5/337 (1.5%)	6
Upper respiratory tract infection	6/337 (1.8%)	6
Urinary tract infection	19/337 (5.6%)	20
Injury, poisoning and procedural complications
Fall	4/337 (1.2%)	9
Metabolism and nutrition disorders
Hypokalaemia	7/337 (2.1%)	7
Musculoskeletal and connective tissue disorders
Arthralgia	6/337 (1.8%)	8
Back pain	9/337 (2.7%)	9
Nervous system disorders
Dizziness	11/337 (3.3%)	11
Headache	24/337 (7.1%)	28
Dysgeusia	4/337 (1.2%)	7
Respiratory, thoracic and mediastinal disorders
Cough	7/337 (2.1%)	8
Dyspnoea	4/337 (1.2%)	4
Vascular disorders
Hypertension	5/337 (1.5%)	5

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.