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

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

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

Conditions
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Iron Deficiency Anemia

Intervention / Treatment
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* Drug: Ferumoxytol


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria include: Participants who completed participation in study AMAG-FER-IDA-301 [NCT01114139] 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: Experienced a serious adverse event (SAE) related to ferumoxytol in study AMAG-FER-IDA-301 Female participants who are pregnant, intend to become pregnant, are breastfeeding, or have a positive serum/urine pregnancy test

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ferumoxytol<br>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<br>* IV Ferumoxytol<br>* Other names: Feraheme;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | TP Baseline (Day 1), TP Week 5 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3 | 
| Percentage Of Participants With An Increase In Hemoglobin ≥2.0 g/dL At Any Time From TP Baseline To TP Week 5 | 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. | TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 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 | 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. | TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3 | 
| Mean Change In TSAT Following Each Course Of Ferumoxytol From TP Baseline To TP Week 5 Following Each Course Of Ferumoxytol | Mean change in TSAT 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 | 
| 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 | 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. | TP Baseline (Day 1), TP Week 5 for Courses 1, 2, and 3 | 
| Time To Hemoglobin Increase Of ≥2.0 g/dL Or To A Hemoglobin Level Of ≥12.0 g/dL From Baseline | 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. | TP Baseline (Day 1) up to TP Week 5 for Courses 1, 2, and 3 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Iron deficiency anemia, Feraheme, Ferumoxytol, IDA