E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Bleeding i patients with acute myeloid leukemia |
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E.1.1.1 | Medical condition in easily understood language |
Bleeding i patients with acute myeloid leukemia |
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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 | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10005103 |
E.1.2 | Term | Bleeding |
E.1.2 | System Organ Class | 100000004866 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The purpose of this study is to investigate the efficacy of fibrinogen concentrate as bleeding prophylaxis in patients with thrombocytopenia suffering from AML
Primary objectives The specific objectives are to investigate: a) Whether administration of fibrinogen concentrate beyond platelet transfusions increases clot initiation and clot strength. b) If there is a difference in the fibrin network structure between: 1. Healthy individuals with in vitro induced thrombocytopenia and leukaemia patients with thrombocytopenia undergoing chemotherapy. 2. Baseline, after platelet transfusion and after administration of fibrinogen concentrate in leukaemia patients with thrombocytopenia undergoing chemotherapy.
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients treated for non-promyelocyte AML with intensive chemotherapy at the Department of Haematology, Aarhus University Hospital. 2. Able to give written informed consent 3. Above 18 years old 4. Fertile women must have a negative pregnancy test and agree to use safe anti-conception(hormonal anti-conception or intrauterine device) for 30 days after receiving the study treatment.
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E.4 | Principal exclusion criteria |
1. Known inherited bleeding disorder 2. Known serious thrombophilia(antithrombin deficiency, protein C or S deficiency, homozygous factor V Leiden) 3. Thromboembolic disease within the past 3 months 4. Known allergy to Riastap
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E.5 End points |
E.5.1 | Primary end point(s) |
Improvement in clot initiation (ROTEM, low tissue factor assay) determined as shorter clotting time after in vivo administration of fibrinogen concentrate subsequent to platelet transfusion. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The primary end point will be evaluated after the collection of all data. No interim analyses will be performed. |
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E.5.2 | Secondary end point(s) |
• 30 days mortality • Thromboembolic events
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
The Secondary end points will be evaluated after the collection of all data. No interim analyses will be performed. |
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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 | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
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 | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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 | |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |