TCRαβ+ and CD19+ Depleted Hematopoietic Stem Cell Transplant From Closely Matched Unrelated Donors or Haploidentical Related Donors for Hematologic Diseases in Children and Young Adults
University of Wisconsin, Madison
Summary
This study is being done to see if the investigators can take peripheral blood stem cells from either an adult family member or a closely matched unrelated donor, and run them through a special lab instrument to remove alpha/beta T cells and B cells and then give them to the patient to treat disease. This is an experimental way of doing a hematopoietic stem cell transplant (HSCT). The investigators want to see if the new stem cells will grow without bad graft vs. host disease (GVHD). This treatment approach is experimental in the United States.
Description
This single institution, phase I clinical trial will determine the safety and feasibility of employing T-cell receptor (TCR) αβ+ and CD19+ (Cluster of Differentiation) depleted hematopoietic stem cell transplantation (HSCT) using peripheral blood stem cells (PBMC) from closely matched unrelated donors or haploidentical donors to treat non-malignant hematologic diseases in children and young adults. Allogeneic hematopoietic stem cell transplantation has become a curative option for children and adolescents with a variety of otherwise fatal conditions. To reduce the incidence and severity of gra…
Eligibility
- Age range
- 0–25 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * No Human leukocyte antigen (HLA) identical sibling available AND * NO HLA matched unrelated donor available OR urgent need of HSCT precludes time necessary to search for suitable HLA matched unrelated donor AND * Haploidentical donor OR closely matched unrelated donor available and willing to undergo mobilization and apheresis * If subject has genetically confirmed inherited bone marrow failure, related donor must be evaluated for this disorder and testing must be negative. * If subject has sickle cell disease, donor must be unaffected or have only sickle cell trait * Pa…
Interventions
- BiologicalTCRαβ+/CD19+ depleted Hematopoietic stem cell (HSC) graft
After undergoing a disease specific conditioning regimen (standard of care), participants will receive peripheral blood stem cell transplant from a haploidentical donor or closely matched unrelated donor, depleted of TCR αβ+ and CD19+ cells using the CliniMACS TCR α/β-biotin and CD19 Systems.
- DeviceCliniMACS® System
The CliniMACS Cell Selection System is based on magnetic-activated cell sorting mechanism. The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures.
Location
- American Family Children's HospitalMadison, Wisconsin