E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
cancer- and chemotherapy-related anemia |
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E.1.1.1 | Medical condition in easily understood language |
cancer- and chemotherapy-related anemia |
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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 | 19.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10002272 |
E.1.2 | Term | Anemia |
E.1.2 | System Organ Class | 100000004851 |
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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 primary objective of this study is to compare the efficacy and safety of ferric carboxymaltose (FCM) versus placebo as monotherapy for maintaining hemoglobin (Hgb) levels in patients with cancer- and chemotherapy-related anemia |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives: - To evaluate the safety and tolerability of FCM * Percentage of adverse events and serious adverse events - To evaluate the percentage of patients with a hemoglobin stabilized throughout the study - To evaluate transfusion rate (The decision of when to transfuse will be at the discretion of the Investigator and treating physicians. However, investigators and treating physicians will be encouraged not to transfuse patients whose measured [Hgb] is greater than 7.0 g/dL in absence of any evidence of inadequate oxygen delivery to organs that could be due to [Hgb].) * Percentage of patients receiving a transfusion - To evaluate quality of life * FACIT-Fatigue scale - To evaluate efficacy response based on hepcidin levels |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects (male or female) ≥ 18 years of age able to give informed consent to the study 2. Subjects with solid tumors that have metastasized 3. Receiving chemotherapy as part of their cancer treatment, with at least 4 weeks of treatment remaining 4. Screening visit central laboratory hemoglobin (Hgb) <11 g/dL, but ≥8 g/dL 5. Ferritin between 100 and 800 ng/mL and transferrin saturation (TSAT) ≤ 35% 6. Patients must have Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2 7. Life expectancy of at least 6 months 8. Demonstrate the ability to understand the requirements of the study, willingness to abide by study restrictions and to return for the required assessments. |
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E.4 | Principal exclusion criteria |
1. Previous participation in a ferric carboxymaltose clinical trial 2. Known hypersensitivity reaction to any component of ferric carboxymaltose 3. Any anemia treatment within 4 weeks before inclusion (oral iron, IV iron, transfusion, or erythropoiesis-stimulating agents) 4. Patients on erythropoiesis-stimulating agents 5. Requiring dialysis for the treatment of chronic kidney disease 6. Any non-viral infection 7. Patients with overt bleeding 8. Known positive hepatitis with evidence of active disease 9. Received an investigational drug within 30 days of screening 10. Alcohol or drug abuse within the past 6 months 11. Hemochromatosis or other iron storage disorders 12. Any other laboratory abnormality, medical condition or psychiatric disorders which in the opinion of the Investigator would put the subject's disease management at risk or may result in the subject being unable to comply with study requirements. 13. Pregnant or actively trying to become pregnant (Female subjects who are of childbearing age must have a negative pregnancy test at screening and be practicing an acceptable method of birth control during the study.). |
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E.5 End points |
E.5.1 | Primary end point(s) |
Percentage of patients with a decrease in Hgb ≥ 0.5 g/dL from baseline (Day 0 value) beginning at Week 3 through Week 18: - If the patient’s Hgb decrease is between 0.5-1.0 g/dL from baseline, two consecutive Hgb values will be needed to qualify as part of the primary endpoint - If the patient’s Hgb decrease is > 1.0 g/dL from baseline, on will be needed to qualify as part of the primary endpoint |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
from baseline (Day 0 value) beginning at Week 3 through Week 18 |
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E.5.2 | Secondary end point(s) |
1. Percentage of patients with avoidance of anemia progression as defined as initiation of other anemia management at any time during the study (i.e. ESAs, blood transfusions, and iron) 2. Percentage of patients with a decrease in Hgb ≥ 0.5 g/dL from their baseline between Day 0 to each study visit 3. Time to a decrease in Hgb ≥ 0.5 g/dL from their baseline between Day 0 to Week 18 4. Percentage of patients requiring a blood transfusion 5. Mean change in Hgb from baseline to week 18 or time of intervention 6. Percentage of patients with Hgb >1 g/dL increase at any time point in the absence of any blood transfusion or ESA treatment 7. Percentage of patients with Hgb >12 at any time point in the absence of any blood transfusion or ESA treatment 8. Time to hemoglobin response defined as ≥ 1 g/dL increase 9. Correlation of change in hemoglobin with Day 0 hepcidin level 10. Total score of the Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-Fatigue) 11. Adverse eventsood transfusion or ESA treatment |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. at any time during the study 2. between Day 0 to each study visit 3. between Day 0 to Week 18 4. at any time during the study 5. whole study duration 6. at any time point 7. at any time point 8. whole study duration 9. Week 2, and either at Week 18 or the time of intervention (e.g. administration of oral or IV Iron, ESAs, or transfusion) 10. whole study duration 11. whole study duration |
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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 | No |
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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
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 |
Bulgaria |
Georgia |
Hungary |
Poland |
United States |
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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
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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 | 4 |
E.8.9.1 | In the Member State concerned days | |
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 |