E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hematopoietic stem cell transplantation in patients with newly diagnoses AML ≤60 years of age in intermediate risk, after first complete response in comparison to standard consolidation chemotherapy
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E.1.1.1 | Medical condition in easily understood language |
AML (newly diagnosed), age 18-60 years, cytogenetic intermediate risk,
first complete response (CR/CRi), stem cell donator available |
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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 | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000886 |
E.1.2 | Term | Acute myeloid leukemia |
E.1.2 | System Organ Class | 100000004864 |
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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 |
To define the role of allogeneic stem cell transplantation in patients ≤60 years with intermediate-risk acute myeloid leukemia in first complete remission. According to the primary hypothesis, four years after randomisation, a 15% survival advantage of allogeneic stem cell transplantation (60% vs. 45%) compared to conventional postremission therapy is assumed. |
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E.2.2 | Secondary objectives of the trial |
- Disease-free survival
- Cumulative incidence of relapse
- Cumulative incidence of non-relapse mortality
- Quality of life
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• AML in first remission
• cytological standard risk, i.e. karyotype not listed under exclusion criteria
• Identification of HLA-identical sibling or HLA-compatible related or unrelated donor (9/10 HLA-alleles matched, high resolution typing for HLA-A, B, Cw, DRB1 and DQB1)
• age: 18 - 60 years
• medically fit for allogeneic stem cell transplantation
• CR / CRi after induction therapy
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E.4 | Principal exclusion criteria |
• core-binding factor leukemia (t(8;21), inv16)
• acute promyelocytic leukemia (t(15;17)
• complex aberrant karyotype
• karyotypes: -7; -5; del5q; t(3;3); t(6;11), t(6;9), 11q aberrations, trisomy 8 ± one single additional aberration, t(9;11)
• pregnancy / nursing
• non-compliance |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy end-point is overall survival from first complete remission (CR) 4 years after randomisation evaluated for each study arm in accordance with intention-to-treat. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
year 1+2: every 3 months
year 3+4: every 6 months |
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E.5.2 | Secondary end point(s) |
Event-free survival
Cumulative incidence of relapse
Cumulative incidence of non-relapse mortality
Quality of Life |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
year 1+2: every 3 months
year 3+4: every 6 months |
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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 | 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 | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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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 | 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 | 30 |
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 | 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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already provided in the protocol |
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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 | 7 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |