E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10060930 |
E.1.2 | Term | Acute leukaemia in remission |
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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 |
1. To compare the cumulative incidence of extensive chronic GvHD (cGvHD) in high risk leukemia patients who underwent haploidentical HCT followed by add back strategy of HSV-Tk donor lymphocytes or haploidentical HCT followed by any T cell repletion strategy. 2. To compare the overall survival (OS) in the two treatment arms |
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E.2.2 | Secondary objectives of the trial |
To compare engraftment rate, cumulative incidence of grade II-IV acute GvHD and time to T-cell immune reconstitution in the two treatment arms To compare incidence and duration of infectious episodes and infectious disease mortality in the two treatment arms To compare transplant-related mortality (TRM), cumulative incidence of relapse (CIR), and disease-free survival (DFS) in the two treatment arms To evaluate the acute and long-term toxicity related to the HSV-Tk infusions To assess quality of life (QoL) and Medical Care Utilization (MCU) in both arms |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Age ≥ 18 years Any of the following conditions: - AML and ALL in 1st complete remission (CR) at high risk of relapse based on negative prognostic factors - AML and ALL in 2nd or subsequent CR - secondary AML in CR Absence of HLA matched family or unrelated donor Stable clinical conditions and life expectancy 3 months PS ECOG < 2 Patients must be willing after appropriate evaluation with the investigator to utilize one of the more effective birth control methods during the study and for 3 months after the study is concluded Patients or legal guardians must sign an informed consent indicating that they are aware this is a research study and have been told of its possible benefits and toxic side effects |
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E.4 | Principal exclusion criteria |
Patients with life-threatening condition or complication other than their basic disease. Contraindication to haploidentical HCT as defined by the Investigator Patients with active CNS disease Pregnant or lactating women Exclusion criteria for HSV-Tk infusion: Infections requiring administration of ganciclovir or acyclovir at the time of infusion GvHD requiring systemic immunosuppressive therapy Ongoing systemic immunosuppressive therapy after haploidentical HCT Administration of G-CSF after haploidentical HCT CD3+ cells ≥100 /µl at day of planned experimental infusion after haploidentical HCT. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. To compare the cumulative incidence of extensive chronic GvHD (cGvHD) in high risk leukemia patients who underwent haploidentical HCT followed by add back strategy of HSV-Tk donor lymphocytes or haploidentical HCT followed by any T cell repletion strategy. 2. To compare the overall survival (OS) in the two treatment arms |
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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 | 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 | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
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 | 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 |
T cell repletion strategies |
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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.8.5 | The trial involves multiple Member States | Yes |
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 | Information not present in EudraCT |
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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |