E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
A serious malignant blood disorder which is fatal if not treated |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
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 |
• Event-free Survival at 3 Years
• Overall Survival at 3 Years
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E.2.2 | Secondary objectives of the trial |
• Remission Induction Rate After 2 Courses of Induction Therapy
• Disease-free Survival
• Mortality
• Time to Marrow Recovery
• Toxicities, Including Infectious Complications |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Infants, Children, Adolescents, and Young Adults (<30 year old) with newly diagnosed acute myeloid leukemia (AML). |
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E.4 | Principal exclusion criteria |
Major exclusion criteria included juvenile myelomonocytic leukemia, Fanconi's anemia, Kostmann syndrome, Shwachman syndrome, or any other known bone marrow failure syndrome, promyelocytic leukemia (M3), secondary or treatment-related AML; prior chemotherapy, radiation therapy, or any antileukemic therapy; other prior treatment for AML. |
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E.5 End points |
E.5.1 | Primary end point(s) |
• Overall survival at 3 years, defined as time from study entry, and from
end of induction course 2 for patients in CR, until death.
• Event-free survival at 3 years defined as time from study entry until
death from any cause, induction failure or any relapse of any type
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|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Overall survival at 3 years, defined as time from study entry, and from
end of induction course 2 for patients in CR, until death.
Event-free survival at 3 years defined as time from study entry until
death from any cause, induction failure or any relapse of any type
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|
E.5.2 | Secondary end point(s) |
• Remission rate (RR) after 2 courses of induction therapy
• Disease-free survival
• Mortality
• Time to marrow recovery
• Toxicities, including infectious complications
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Remission rate (RR) after 2 courses of induction therapy- After 2 courses of induction (I and II) therapy, assessed for up to 10 years;
Disease-free survival: Time from the end of course 3 (Intensification I) to death or relapse; assessed for up to 10 years;
Mortality: During the first three courses of therapy;
Time to marrow recovery: At 25 days after treatment with Induction I, Induction II, and Intensification I;
Toxicities, including infectious complications: From the time therapy is initiated, assessed up to 10 years |
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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 | Yes |
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.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
Australia |
Canada |
New Zealand |
Puerto Rico |
Switzerland |
United States |
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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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|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 14 |