E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
high risk leukemia |
hoog risico ziekte |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
• To investigate the feasibility and safety of administration of donor HA-1 TCR transduced virus-specific T cells after allo-SCT. |
|
E.2.2 | Secondary objectives of the trial |
• To evaluate the persistence of donor HA-1 TCR transduced virus-specific T cells after administration.
• To evaluate whether administration of HA-1 TCR transduced virus-specific T cells after allo-SCT makes patients eligible for standard DLI at 6 months after allo-SCT.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Age 18-75 years
• WHO performance score 0-2
• High-risk leukemia (see appendix D)
• Complete remission (CR) or stable partial remission (PR) (see appendix D)
• HLA-A*0201 positive
• HA-1h positive
• Availability of a suitable donor (see donor criteria)
• Written informed consent |
|
E.4 | Principal exclusion criteria |
• Life expectation < 3 months.
• End stage irreversible multi-system organ failure.
• Pregnant or lactating women.
• Severe psychological disturbances.
• HIV-positivity |
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E.5 End points |
E.5.1 | Primary end point(s) |
• The number of events of acute GvHD, all other adverse events and death.
• The feasibility of generation of HA-1 TCR transduced virus-specific T cells. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
- interim analysis will be performed by the principal investigator after treatment of the first 3, 6 and10 patients
- End of the study is defined as 18 weeks after the first infusion of HA-1 TCR transduced virus-specific T-cells of patient number 20. Subsequently the patients will be followed until death or last follow-up. |
|
E.5.2 | Secondary end point(s) |
• The number of HA-1 TCR transduced virus-specific T cells in blood or bone marrow at different time points.
• The number of patients eligible for standard DLI at 6 months. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- interim analysis will be performed by the principal investigator after treatment of the first 3, 6 and10 patients
- End of the study is defined as 18 weeks after the first infusion of HA-1 TCR transduced virus-specific T-cells of patient number 20. Subsequently the patients will be followed until death or last follow-up. |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 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
|
It is anticipated that the first patient will be recruited in the study from media 2012 onwards. We aim to include 20 patients in a 3 year period. An interim analysis will be performed after treatment of the first 3 patients. End of the study is defined as 18 weeks after the infusion of HA-1 TCR transduced virus-specific T-cells of patient number 20. Subsequently the patients will be followed until death or last follow-up. |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |