E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pediatric patients with malignant and non-malignant high-risk diseases, where the allogeneic stem cell transplantation represents the only option for a curative therapy and where an adequate HLA-identical donator is not available. |
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E.1.1.1 | Medical condition in easily understood language |
Pediatric patients with malignant and non-malignant diseases, where stem cell transplantation represents the only option for a curative therapy and where an adequate donator is not available. |
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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 | Information not present in EudraCT |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Evaluation of the engraftment rate after preparation of the stem cell transplant with CD3/CD19 depletion and administration of approximatelly 7x10E6/kg body weight CD34+ cells. Engraftment is defined as leucocytes >1000µl, neutrophiles >500µl and thrombocytes >20.000/µl on day 28 after transplantation. |
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E.2.2 | Secondary objectives of the trial |
Evaluation of the onset of engraftment (as defined in the main objective).
Evaluation of the reconstitution of the immune system after transplantation by means of characterization of the lymphocyte subpopulations CD 3; CD 4; CD 8; CD 56; CD 14.
Characterization of the toxicity of a myeloablative, toxicity-reducing conditioning with Clofarabine, Etoposide , Cyclophosphamide and fludarabine, thiotepa, melphalane and CAMPATH according to the Common Terminology Criteria for Adverse Events v4.0 (CTCAE).
Characterization of the onset of bacterial, viral and fungal infections defined by means of polymerase chain reaction and evaluation of the clinical outcome defined either by means of polymerase chain reaction or the improvement of clinical symptoms
Evaluation of the relapse-free survival
Evaluation of the GvHD-rate
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients age between 0 and 30 years
Written informed consent from the patient or from at least one of the patient’s parents (person having the care and custody of the child)
Patients with a Karnofsky Index > 60%
A malignant disease:
acute lymphatic leucemia
acute myeloic leucemia
myelodysplastic syndrome
chronic myeloic leucemia according to the standard indications
Solid Tumors (e.g. relapse of a neuroblastom, soft tissue sarcoma, Ewing sarcoma, osteo sarcoma, hepatoblastome
Or a non-malignant disease:
Acquired anemia (e.g. severe aplastic anemia, in particular the severe form of the Evans syndrome)
congenital anemia (e.g. thalassemia; sickle cell anemia)
A reliable contraception has to be observed in sexual reproductive women |
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E.4 | Principal exclusion criteria |
Patient or responsible person (person having the care and custody of the child), who is not able to understand the character and implications of this clinical trial
Pregant or breast-feeding women
Patients with a prior history of a stem cell transplantation within the last 250 days
Renal, cardiac or hepatic insufficiency
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E.5 End points |
E.5.1 | Primary end point(s) |
Evaluation of the engraftment rate after preparation of the stem cell transplant with CD3/CD19 depletion and administration of approximatelly 7x10E6/kg body weight CD34+ cells. Engraftment is defined as leucocytes >1000µl, neutrophiles >500µl and thrombocytes >20.000/µl on day 28 after transplantation. |
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E.5.2 | Secondary end point(s) |
To evaluate immunoreconstitution after transplantation by assessing lymphocyte subsets |
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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 | 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 | No |
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 | No |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 6 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |