Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells

Study Overview
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Brief Summary
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Feasibility and toxicity of haploidentical transplantation of CD3/CD19 depleted stem cells in combination with a toxicity reduced conditioning regimen or with standard conditioning regimens according to underlying disease.

Official Title
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Haploidentical Stem Cell Transplantation With CD3/CD19 Depleted Stem Cells in Pediatric Patients With Refractory Hematological and Oncological Diseases

Conditions
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Acute Leukemias, Aplastic Anemia, Childhood Solid Tumor

Intervention / Treatment
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* Other: Transplantation of CD3/CD19 depleted stem cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: patients with primary refractory or relapsed disease (including relapse after previous SCT and active disease) AML in complete remission of refractory MDS RAEB-t/secondary AML ALL CML Non-Hodgkin Lymphoma / Hodgkin Lymphoma non malignant diseases (aplastic anemia, thalassemia, SCID) relapsed Neuroblastoma relapsed soft tissue sarcoma (Rhabdomyosarcoma, Ewing sarcoma, PNET soft tissue sarcoma with primary bone metastases or bone marrow in whom no matched donor was available, Exclusion Criteria: < 6 months after previous HSCT active cerebral seizure conditions massive progression of leukemias or solid tumours before planned trp. left ventricular ejection fraction <25% creatinine clearance <40ml/min before conditioning respiratory insufficiency with oxygen demand or DLCO <30% Bilirubin >4mg/dl, GOT/GPT >400 severe infection (HIV, Aspergillosis) pregnancy

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 25 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Transplantation of CD3/CD19 depleted stem cells<br>Patients receive a minimum dosage of 10x10E6/kg bodyweight CD3/CD19 depleted CD34+ stem cells. Maximum dosage of residual T cells will be 1x10E5/kg BW | Other: Transplantation of CD3/CD19 depleted stem cells<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Engraftment rate and transplant related mortality | engraftment rate will be evaluated by leukocyte counts in peripheral blood . Definition of engraftment: >500 neutrophiles/µl blood. Transplant related mortality will be evaluated at day 100 and day 365 post transplant | 365 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Graft versus Host disease and speed of immune reconstitution | Incidence of acute and chronic GvHD will be evaluated according to Glucksberg criteria. Immune recovery will be evaluated by flow cytometry. Percentages and numbers of T, B and NK cells will be measured weekly until day 100, afterwards on day 180 and 365. | 365 days |