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 |
Iron deficiency anemia (IDA) |
|
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 | 18.1 |
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 | 100000004851 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10022976 |
E.1.2 | Term | Iron deficiency anemia secondary to inadequate dietary iron intake |
E.1.2 | System Organ Class | 100000004851 |
|
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 safety of 1.020 g of IV ferumoxytol, administered as 2 doses of 510 mg each, compared to 1.500 g of IV FCM, administered as 2 doses of 750 mg each for the treatment of IDA. To assess the incidence of moderate to severe hypersensitivity reactions, including anaphylaxis, and of moderate to severe hypotension with ferumoxytol as compared to FCM |
|
E.2.2 | Secondary objectives of the trial |
-To assess the incidence of the composite safety endpoint of moderate to severe hypersensitivity reactions, serious cardiovascular events, and death for ferumoxytol as compared to FCM
-To assess the incidence of treatment emergent serious adverse events following administration of ferumoxytol as compared to FCM.
|
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Mechanisms of Hypophosphatemia Sub-Study
To explore the mechanisms underlying the increase in urinary excretion of phosphate and hypophosphatemia observed following IV iron usage.
|
|
E.3 | Principal inclusion criteria |
1. Males and females ≥18 years of age
2. Subjects with IDA and in whom intravenous iron treatment is indicated and defined as :
• Subjects with documented hemoglobin <12.0 g/dL for females and <14.0 g/dL for males within 60 days of dosing
And
• Subjects with documented TSAT ≤20% or Ferritin ≤100 ng/mL within 60 days of dosing
3. Documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate (as per oral iron history questionnaire) |
|
E.4 | Principal exclusion criteria |
1. Known hypersensitivity reaction to any component of ferumoxytol or FCM
2. History of allergy to an IV iron
3. History of multiple drug allergies
4. Subjects with dialysis dependent CKD
5. Hemoglobin ≤7.0 g/dL
6. Female subjects who are pregnant, intend to become pregnant, are breastfeeding, have a positive serum/urine pregnancy test or not willing to use effective contraceptive precautions during the study (including females of childbearing potential who are partners of male subjects)
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the incidence of moderate to severe hypersensitivity reactions, including anaphylaxis, and of moderate to severe hypotension. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
-Incidence of the composite safety endpoint of moderate to severe hypersensitivity reactions, including anaphylaxis, serious cardiovascular events, and death.
-Mean change in hemoglobin from Baseline to Week 5
-Mean change in hemoglobin per mg of iron administered
|
|
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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
|
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 | 15 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 48 |
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 |
Hungary |
Latvia |
Lithuania |
Poland |
United States |
|
E.8.7 | Trial has a data monitoring committee | No |
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
|
|
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 | 4 |
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 | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |