E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Adult patients with hematological malignancies |
|
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 |
The primary objective of this study is to determine the feasibility of umbilical cord blood-derived hematopoietic stem cell transplantation with co-infusion of third party mesenchymal stem cells as assessed by the treatment-related mortality at d100 after transplant. |
|
E.2.2 | Secondary objectives of the trial |
Chimerism at multiple time points Hematopoietic recovery (neutrophil and platelet engraftment) Immune recovery Incidence of acute and chronic graft-versus-host disease (GVHD) Infectious complications Disease free survival Relapse incidence Overall survival
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patient inclusion criteria: Age 15-60 yrs with following diseases Allogeneic stem cell transplantation is the preferred treatment option: High risk acute myeloid leukemia (AML) in first complete remission (CR) High risk acute lymphoblastic leukemia (ALL) in first CR Acute leukemia in second or third remission High risk myelodysplastic syndrome: IPSS Intermediate-2 or high risk Advanced lymphoproliferative disorders Chronic myeloid leukemia Multiple myeloma Informed consent given
|
|
E.4 | Principal exclusion criteria |
Patient exclusion criteria Previous allogeneic transplant Progressive malignant disease Significant organ damage as a contraindication to allotransplantation Creatinine clearance < 60 ml/min AST/ALT > 3x normal value and/or serum bilirubin > 3 mg/dL Cardiac failure (LVEF < 50%) Clinical relevant pulmonary disease: DLCO < 50% normal Significant psychiatric or neurological disorder Uncontrolled viral, fungal or bacterial infection Pregnancy HIV positive
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary objective of this study is to determine the feasibility of umbival cord blood-derived hematopoietic stem cell transplantation with co-infusion of third party mesenchymal stem cells as assessed by the treatment-related mortality at d100 after transplant. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
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
| 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 | 9 |
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 | Information not present in EudraCT |
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
|
The trial will be stopped at any time that there is reasonable evidence that the true rate of day +100 nonrelapse mortality exceeds 0.40. It is the intention to include an initial 20 patients. Operationally, the stopping rule for mortality will be triggered if 4 of 5, 7 of 10, 9 of 15, 12 of 20 patients experience nonrelapse mortality by day +100. |
|
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 |