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 | No |
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 |
|
E.2.3 | Trial contains a sub-study | No |
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 |
|
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 |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 27 |
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 | No |
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 | 0 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 49 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 49 |