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

A Phase III, Open-Label Extension Trial of the Safety and Efficacy of Ferumoxytol for the Episodic Treatment of Iron Deficiency Anemia

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01114217

Enrollment

634

Registered

2010-05-03

Start date

2010-07-27

Completion date

2013-04-23

Last updated

2022-04-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Iron Deficiency Anemia

Keywords

Iron deficiency anemia, Feraheme, Ferumoxytol, IDA

Brief summary

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

Interventions

DRUGFerumoxytol

IV Ferumoxytol

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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.
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.
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.
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.
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.
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.

Countries

Canada, Hungary, India, Latvia, Poland, United States

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.

Participants by arm

Arm	Count
Received Ferumoxytol in IDA-303 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.	337
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.	297
Total	634

Withdrawals & dropouts

Period	Reason	FG000	FG001
Overall Study	Adverse Event	5	3
Overall Study	Lost to Follow-up	17	33
Overall Study	Other-Clerical Error	3	0
Overall Study	Other-Early termination	6	10
Overall Study	Other-Lack of Efficacy	1	0
Overall Study	Other-Lost to Follow-up	1	0
Overall Study	Other-Non Compliance	2	0
Overall Study	Other-Physician Decision	1	1
Overall Study	Other-Pregnancy	2	4
Overall Study	Other-Procedure	1	1
Overall Study	Other-Protocol Violation	1	3
Overall Study	Other-Site Stopped Communicating	2	0
Overall Study	Other-Sponsor Decision	10	12
Overall Study	Other-Study Termination	0	1
Overall Study	Other-Study Withdrawn	1	1
Overall Study	Other-Withdrawal by Subject	1	1
Overall Study	Withdrawal by Subject	21	28

Baseline characteristics

Characteristic	Received Ferumoxytol in IDA-303	Did Not Receive Ferumoxytol in IDA-303	Total
Age, Continuous	46.0 years STANDARD_DEVIATION 13.33	43.8 years STANDARD_DEVIATION 13.26	44.9 years STANDARD_DEVIATION 13.33
Sex: Female, Male Female	304 Participants	269 Participants	573 Participants
Sex: Female, Male Male	33 Participants	28 Participants	61 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	93 / 337
serious Total, serious adverse events	22 / 337

Outcome results

Primary

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

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

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.

Arm	Measure	Value (MEAN)	Dispersion
Ferumoxytol	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

p-value: <0.0001t-test, 2 sided

Secondary

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

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.