E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
ACUTE MYELOID LEUKEMIA PATIENTS |
|
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 | 10000880 |
E.1.2 | Term | Acute myeloid leukaemia |
|
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 |
To evaluate 1. Overall survival (OS) at 2 years. Event for OS in both arms is death and patients are censored at the date of last contact if alive. 2. Disease-free survival (DFS) at 2 years. Events for DFS in both arms are death and first relapse (either AML or MDS recurrence). 3. Changes in quality of life from diagnosis in both arms. |
|
E.2.2 | Secondary objectives of the trial |
To evaluate 1. Number and length of hospitalizations in the 2 arms in the 2-year post-remission period. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Age 61 years or more 2. Newly diagnosed AML with > 30% myeloid marrow blasts, either ``de novo`` or evolving from a MDS not previously treated with chemotherapeutic agents. 3. Absence of central nervous system involvement 4. No contraindications for intensive chemotherapy, defined as: a) prior congestive heart failure requiring treatment and/or left ventricular systolic ejection fraction below the normal range; b) a creatinine or bilirubin level more than twice the upper limit of normal, except if AML-related; c) a PS score of > 2; d) uncontrolled severe infection. 5. Informed consent. |
|
E.4 | Principal exclusion criteria |
1. Age ≤ 60 years 2. Newly diagnosed AML with < 30% myeloid marrow blasts 3. Previously treated AML 4. Central nervous system involvement 5. Prior congestive heart failure requiring treatment and/or left ventricular systolic ejection fraction below the normal range; 6. A creatinine or bilirubin level more than twice the upper limit of normal, except if AML-related; 7. A PS score of > 2; 8. Uncontrolled severe infection |
|
E.5 End points |
E.5.1 | Primary end point(s) |
1. Differences in OS at 2 years between 5-Aza and placebo arms. 2. Differences in DFS at 2 years between 5-Aza and placebo arms. 3. Differences in changes in QoL scores from diagnosis between 5-Aza and placebo arms. |
|
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 | Yes |
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) | No |
E.8.2.2 | Placebo | Yes |
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 | 11 |
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 | Information not present in EudraCT |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |