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.
Conditions
Interventions
Product Name: Stem cells from non-related or haploidentical donators depleted by means of CliniMACS CD3/CD19
Pharmaceutical Form: Infusion
Other descriptive name: stem cells
Concentration type: range
Sponsors
Johann Wolfgang Goethe-Universität Frankfurt am Main
Eligibility
Sex/Gender
All
Inclusion criteria
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 Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: 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 ;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. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): To evaluate immunoreconstitution after transplantation by assessing lymphocyte subsets | — |
Countries
Germany
Contacts
Public ContactDivision for stem cell transplantat
University hospital frankfurt, Department for children and adolescents
Outcome results
None listed