E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
very high risk acute lymphoblastic leukaemia in children and adolescents with indicaton for allogenic hematopoetic stem cell transplantation |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 8.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000844 |
E.1.2 | Term | Acute lymphoblastic leukaemia |
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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 evaluate whether HSCT from matched family or unrelated donors (MD) is equivalent to the HSCT from matched sibling donors (MSD) *To evaluate the efficacy of HSCT from mismatched family or unrelated donors (MMD) as compared to HSCT from MSD/MD. *To determine whether therapy has been carried out according to the main HSCT protocol recommendations. The standardisation of the treatment options during HSCT from different donor types aims at the achievement of an optimal comparison of survival after HSCT with survival after chemotherapy only. *To prospectively evaluate and compare the incidence of acute and chronic GvHD after HSCT from MSD, from MD and from MMD.
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E.2.2 | Secondary objectives of the trial |
*To prospectively evaluate survival and event free survival after HSCT from matched sibling donors (MSD), from matched family or unrelated donors (MD) and from mismatched family or unrelated donors (MMD).
*To reduce transplantation associated mortality by standardising the selection criteria for donors, the stem cell manipulation and supportive measures for allogeneic HSCT.
*To prevent extensive chronic GvHD by close monitoring and by using standardised GvHD- and anti-infectious therapy.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion criteria: *Age at ALL diagnosis (for HSCT indication in CR1) or at ALL relapse diagnosis: ≤18 years *ALL in first, second or any following complete remission *Indication for allogeneic HSCT (see chapter Indications for allogeneic HSCT, page 11) *Signed informed consent of parents or legal guardian of the minor patient (and in certain cases the patient himself/herself) for participation in the study ALL SCT BFM international. *The patient is treated in a hospital participating in the study during the study period. *No evidence of pregnancy *No secondary malignancy *No history of allogeneic or autologous HSCT
All patients who are entered into the study according to these criteria are study patients. Once registered into the study, a patient is only excluded from the study, if the diagnosis ALL turns out to be clearly wrong.
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E.4 | Principal exclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
*event-free and overall survival after allogeneic HSCT *occurrence of acute and chronic Graft-versus-Host-Disease (GvHD) *occurrence and course of late effects after chemotherapy with subsequent allogeneic HSCT |
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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) | 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 |
stem cell transplantation from HLA identical sibling donors vs HLA matched donors vs. HLA mismatched |
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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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 34 |
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 | Information not present in EudraCT |
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 |
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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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time schedule: start of I-BFM pilot phase: 01.01.2007 stop of patient recruitment: 09.09.2009 end of main trial: 09.09.2014 Amendment: 15.03.2008
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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 | 8 |
E.8.9.1 | In the Member State concerned days | 9 |
E.8.9.2 | In all countries concerned by the trial years | 7 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 9 |