Clinical Trials Register

Summary
EudraCT Number:	2011-001865-42
Sponsor's Protocol Code Number:	AMAG-FER-IDA-301
National Competent Authority:	Hungary - National Institute of Pharmacy
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2011-07-18
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Hungary - National Institute of Pharmacy

A.2

EudraCT number

2011-001865-42

A.3

Full title of the trial

A Phase III, Randomized, Double-Blind, Placebo-Controlled Trial
of Ferumoxytol for the Treatment of Iron Deficiency Anemia

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A Phase III, Randomized, Double coded, Placebo-Controlled Trial
of Ferumoxytol for the Treatment of Iron Deficiency Anemia

A.4.1

Sponsor's protocol code number

AMAG-FER-IDA-301

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT01114139

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	AMAG Pharmaceuticals, Inc.
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	AMAG Pharmaceuticals, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	AMAG Pharmaceuticals, Inc.
B.5.2	Functional name of contact point	Ratna Lingamaneni
B.5.3	Address:
B.5.3.1	Street Address	100 Hayden Ave
B.5.3.2	Town/ city	Lexington
B.5.3.3	Post code	MA 02421
B.5.3.4	Country	United States
B.5.4	Telephone number	+1617498 3371
B.5.5	Fax number	+1617812 7854
B.5.6	E-mail	rlingamaneni@amagpharma.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	Yes
D.2.1.1.1	Trade name	Feraheme™
D.2.1.1.2	Name of the Marketing Authorisation holder	AMAG Pharmaceuticals, Inc
D.2.1.2	Country which granted the Marketing Authorisation	United States
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	ferumoxytol
D.3.4	Pharmaceutical form	Solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	ferumoxytol
D.3.9.1	CAS number	722492-56-0
D.3.9.2	Current sponsor code	5128; ferumoxytol Drug Substance
D.3.9.3	Other descriptive name	FERUMOXYTOL
D.3.9.4	EV Substance Code	SUB31284
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	30
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Solution for injection
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Iron deficiency anemia (IDA)

E.1.1.1

Medical condition in easily understood language

Anemia due to lack of iron amount in the blood

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.0
E.1.2	Level	LLT
E.1.2	Classification code	10022976
E.1.2	Term	Iron deficiency anemia secondary to inadequate dietary iron intake
E.1.2	System Organ Class	10005329 - Blood and lymphatic system disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.0
E.1.2	Level	LLT
E.1.2	Classification code	10022975
E.1.2	Term	Iron deficiency anemia secondary to blood loss (chronic)
E.1.2	System Organ Class	10005329 - Blood and lymphatic system disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the efficacy and safety of a 1.02 g course of IV ferumoxytol,
administered as two doses of 510 mg each, compared with placebo (normal
saline) for the treatment of iron deficiency anemia (IDA)

E.2.2

Secondary objectives of the trial

Not Applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Males and females ≥18 years of age
2. Subjects with IDA defined as:
a) hemoglobin <10.0 g/dL
b) TSAT <20%
3. Subjects who have a history of unsatisfactory oral iron therapy or in whom
oral iron cannot be used (includes subjects who remain anemic despite oral
iron therapy, have side effects that preclude the use of oral iron therapy, or
who cannot otherwise take oral iron)
4. Female subjects of childbearing potential who are sexually active must be on
an effective method of birth control for at least 1 month prior to screening
and agree to remain on birth control until completion of participation in the
study; for postpartum female subjects within 1 month of delivery who have
not resumed menses, birth control must be resumed at screening
5. Subject is capable of understanding and complying with the protocol
requirements and available for the duration of the study
6. Subject has been informed of the investigational nature of this study and has
given voluntary written informed consent and, if applicable, Health
Insurance Portability and Accountability Act (HIPAA) or patient protection
authorization in accordance with institutional, local, and national personal
health data protection guidelines.

E.4

Principal exclusion criteria

1. History of allergy to IV iron
2. Allergy to two or more classes of drugs
3. Subjects on dialysis or with an estimated glomerular filtration rate (eGFR)
<30 mL/min/1.73m2
4. Female subjects who are pregnant, intend to become pregnant, are
breastfeeding, or have a positive serum/urine pregnancy test (unless within
the first two weeks postpartum)
5. Hemoglobin ≤7.0 g/dL
6. Serum ferritin >600 ng/mL
7. Parenteral iron therapy within 4 weeks prior to screening, oral iron therapy
within 2 weeks prior to screening, or red blood cell (RBC)/whole blood
transfusion within 2 weeks prior to screening or planned during the study
8. Erythropoiesis-stimulating agent (ESA) therapy initiated, stopped, or dose
changed by >20% within 4 weeks prior to screening, or an anticipated ESA
dose change of >20% during the study
9. Hematologic malignancies (other than lymphomas), hemolysis, untreated
vitamin B12 or untreated folate deficiency, or other known causes of anemia
other than iron deficiency
10. Major surgery or invasive intervention within 4 weeks prior to screening
(with the exception of uncomplicated diagnostic colonoscopy, endoscopy,
and tubal ligation), C-section within 2 weeks prior to screening, organ
transplant within 6 months prior to screening, or any planned surgery or
intervention during the course of the study
11. Recent (within 2 months prior to screening) initiation or change in therapy to
control bleeding (eg, azathioprine or 6-mercaptopurine for inflammatory
bowel disease [IBD], hormonal therapy for abnormal uterine bleeding
[AUB]) or expected to change therapy during the study
12. Active clinically significant infection or acute serious medical illness (with
the exception of cancer) requiring treatment or intervention within 2 weeks
prior to screening
13. Among subjects with cancer , an Eastern Cooperative Oncology Group
(ECOG) Performance Status of >2 or life expectancy <24 weeks; or >6
months of myeloablative chemotherapy (ie, mitomycin C, carboplatin,
nitrosoureas) or radiation therapy to >40% of the bone marrow within the
previous 2 years; or history of or preparation for bone marrow transplant
14. Received another investigational agent within 4 weeks prior to screening, or
planned receipt of an investigational agent not specified by this protocol
during the study period
15. Any other clinically significant medical disease or condition (eg,
uncontrolled hypertension) or subject responsibility that, in the Investigator’s
opinion, may interfere with a subject’s ability to give informed consent,
adhere to the protocol, interfere with assessment of the investigational
product, or serve as a contraindication to the subject’s participation in the
study

E.5 End points

E.5.1

Primary end point(s)

Proportion of subjects achieving a ≥2.0 g/dL increase in hemoglobin at any
time from Baseline to Week 5

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 5

E.5.2

Secondary end point(s)

• Mean change in hemoglobin from Baseline to Week 5
• Proportion of subjects achieving a hemoglobin level ≥12.0 g/dL at any time
from Baseline to Week 5
• Mean change in TSAT from Baseline to Week 5
• Time to hemoglobin increase of ≥2.0 g/dL or to a hemoglobin level of
≥12.0 g/dL from Baseline
• Mean change in Functional Assessment of Chronic Illness Therapy
(FACIT)-Fatigue score from Baseline to Week 5
• Proportion of subjects with AUB, cancer, GI disorders, and postpartum
anemia each achieving a ≥2.0 g/dL increase in hemoglobin at any time from
Baseline to Week 5
• Mean change in hemoglobin from Baseline to Week 5 in subjects with AUB,
cancer, GI disorders, and postpartum anemia

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 5

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Canada

Ecuador

El Salvador

Honduras

India

United States

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

The end of trial is defined as the last visit of the last subject.

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

640

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

160

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

120

F.4.2.2

In the whole clinical trial

800

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Subjects who remain anaemic will be able to receive treatment with ferumoxytol in the accompanying extension study (AMAG-FER-IDA-303) immediately after completion of this study.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2011-09-05

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-08-30

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2012-02-27

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