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

Phase 2

Terminated

• Conditions: Malignant and Non-malignant High Risk Diseases

• Registration Number: NCT00945126
• Lead Sponsor: Johann Wolfgang Goethe University Hospital

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2006-12
• Study First Submitted: 2009-07-21
• Study First Submitted QC: 2009-07-22
• Study First Posted: 2009-07-23
• Primary Completion: 2017-08
• Study Completion: 2017-08
• Status Verified: 2018-08
• Last Update Submitted: 2018-08-13
• Last Update Posted: 2018-08-15

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 68

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
To evaluate engraftment after CD3/CD19 depletion of the graft	within 1 year

Secondary Outcome Measures

Name	Time	Method
To evaluate immunoreconstitution after transplantation by assessing lymphocyte subsets	within 1 year

Trial Locations

Locations (1)

University Hospital Frankfurt, Dept. for Children and Adolescents, Division for Stem Cell Transplantation, Goethe University; 🇩🇪 Frankfurt am Main, Hessen, Germany