This is an open label, interventional, non-randomized, phase II trial of TCR alpha/beta and CD19-depeleted allogeneic HCT in pediatric patients with hematologic disease.
This is a single-site, open label, interventional, non-randomized, phase II trial of TCRαβ/CD19 deplete allogeneic HCT as donor source and sole GVHD prophylaxis in pediatric patients with either malignant or non-malignant hematologic disease who are eligable for allogeneic HCT, but lack a HLA-matched sibling donor.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Miltenyi Biotec's CliniMACS Plus Instrument is to be used to TCRαβ CD19 deplete products utilized in this protocol. The CliniMACS Plus is an automated cell separation platform which is functionally closed, maintaining a sterile system for cell depletion and enrichment utilizing a magnetic separation column. Reagents and supplies are to be used for research only but are manufactured and tested under a quality system certified to ISO 13485. Should CD34 selection be required to augment stem cell dose, Miltenyi Biotec's CliniMACS® Plus CD34 Reagent System is FDA approved as a Humanitarian Use Device (HUD). The approved indication was the treatment of patients with acute myeloid leukemia (AML) undergoing myeloablative transplant from matched related allogeneic donors. The CliniMACS® Plus reagent system was approved to obtain an enriched CD34+ cell population for hematopoietic reconstitution without the need for GVHD prophylaxis.
Incidence of severe (grade III-IV) acute graft-versus-host disease (GVHD) at day 100 after infusion of a TCRαβ+/CD19+ negative, peripheral blood stem cell (PBSC) product without additional GVHD prophylaxis.
Grade to be determined using Acute GVHD Staging Scale
Time frame: 5 years
Number of patients with non-engraftment
Defined as the lack of donor-derived neutrophil engraftment
Time frame: 100 days
Number of patients with relapse
Interval from transplant to relapse/recurrence of disease
Time frame: 1 year
Number of treatment-related mortality (TRM)
Defined as death due to regimen-related toxicity or GVD.
Time frame: 1 year
Disease-free Survival (DFS) measured in days
Defined as the minimum time interval from transplant to relapse/recurrence of disease, death or last follow-up.
Time frame: 1 year
Overall Survival (OS) measured in days
Determined at 1 year post-HCT
Time frame: 1 year
Immune Reconstitution
Incidence of absolute CD4+ T-cell count \>400 cells at 1 year post-HCT
Time frame: 1 year
Post-HCT infections
Incidence of bacterial, fungal, and viral infections at day +100
Time frame: 100 days
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.