E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
iron deficiency anemia (IDA) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10022975 |
E.1.2 | Term | Iron deficiency anemia secondary to blood loss (chronic) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10022976 |
E.1.2 | Term | Iron deficiency anemia secondary to inadequate dietary iron intake |
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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 |
The purpose of this study is to evaluate the efficacy and safety of a 1.02 g of IV ferumoxytol, administered as 2 doses of 510 mg each, compared with 1.0 g of iron sucrose, administered as 5 doses of 200 mg each for the treatment of IDA. |
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E.2.2 | Secondary objectives of the trial |
there are no secondary objectives of the trial |
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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
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 |
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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/1.73 square metre
4. Female subjects who are pregnant, intend to become pregnant, are breastfeeding, within 2 weeks postpartum, or have a positive serum/urine pregnancy test
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. Known causes of anemia other than iron deficiency (eg, hemolysis, vitamin B12 or folate deficiency, etc)
10. Major surgery or invasive intervention within 4 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 (ie, histologic diagnosis of nonhematologic malignancy), an Eastern Cooperative Oncology Group (ECOG) Performance Status of >2 or life expectancy <24 weeks; or >2 prior courses/regimens of chemotherapy 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 1. History of allergy to IV iron 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
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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 |
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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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | 10 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 101 |
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 | Information not present in EudraCT |
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 |
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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
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The end of trial is defined as the last visit of the last subject. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |