Allogeneic Stem Cell Transplantation in Pediatric Patients With Malignant and Non-malignant High-risk Diseases

Study Overview
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Brief Summary
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The aim of the study is to investigate the feasibility and toxicity of allogeneic haploidentical or unrelated transplantation with CD3/CD19 depleted stem cells associated with a reduced or a standard conditioning regimen in pediatric patients with malignant and non-malignant high-risk diseases, for whom allogeneic stem cell transplantation represents the only possible therapy option and no human leukocyte antigen (HLA) compatible related donors are available.

Official Title
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Allogeneic Stem Cell Transplantation With CD3/CD19 Depleted Stem Cells of Related or Unrelated Haploidentical Donors in Pediatric Patients With Malignant and Non-malignant Diseases

Conditions
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Malignant and Non-malignant High Risk Diseases

Intervention / Treatment
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* Other: Hematopoietic stem cell product from haploidentical or unrelated donor CD3/CD19 depleted with CliniMACS


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients aged 0 to 30 years Written informed consent from patient and/or parents or guardian Patients with Karnofsky Index > 60% Malignant disease: acute lymphoblastic leukemia acute myeloid leukemia myelodysplastic syndrome chronic myeloid leukemia according to the standard indications solid tumors (e.g. neuroblastoma recurrence, soft-tissue sarcoma, Ewing's sarcoma, osteosarcoma, hepatoblastoma) . Non malignant disease: acquired anemias (e.g. severe aplastic anemia, particularly severe Evans syndrome) congenital anemias (e.g. thalassemia and sickle cell anemia) Women reliable contraception method when appropriate Exclusion Criteria: Participation in other clinical trials Patients, parents, or guardians unable to understand the nature, the importance and the implications of the procedure Pregnant or nursing women Patients who underwent a stem cell transplantation in the last 250 days Patients with kidney, heart or liver insufficiency

Ages Eligible for Study
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Maximum Age: 30 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

| Intervention/Treatment |
| --- |
|Other: Hematopoietic stem cell product from haploidentical or unrelated donor CD3/CD19 depleted with CliniMACS|The aim is to transplant 7x106 CD34+/kg of recipient body weight.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate engraftment after CD3/CD19 depletion of the graft |  | within 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate immunoreconstitution after transplantation by assessing lymphocyte subsets |  | within 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Allogeneic stem cell transplantation in pediatric patients, CD3/CD19 depleted stem cells, malignant and non-malignant high risk diseases