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

NCT ID: NCT00945126

Last Updated: 2018-08-15

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 68 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-12-31
• Study Completion Date: 2017-08-31

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.

Conditions

Malignant and Non-malignant High Risk Diseases

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

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.

Intervention Type: OTHER

Eligibility Criteria

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

• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Johann Wolfgang Goethe University Hospital

OTHER

Sponsor Role: lead

Responsible Party

Peter Bader

Prof. Dr. med. Peter Bader

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Peter Bader, Professor

Role: PRINCIPAL_INVESTIGATOR

University Hospital Frankfurt, Dept. for Children and Adolescents, Division for Stem Cell Transplantation, Goethe University.

Locations

University Hospital Frankfurt, Dept. for Children and Adolescents, Division for Stem Cell Transplantation, Goethe University

Frankfurt am Main, Hesse, Germany

Countries

Germany

Other Identifiers

ZKI-SCT-HAPLO-0106

Identifier Type: -

Identifier Source: org_study_id