This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (graft versus host disease).
This is a Phase 1/2 study evaluating the safety and feasibility of BPX-501 T cells infused after partially mismatched, related, TCR alpha beta T cell depleted hematopoietic stem cell transplant (HSCT) in pediatric patients. The purpose of this clinical trial is to determine whether BPX-501 infusion can enhance immune reconstitution in those patients with hematologic disorders, with the potential for reducing the severity and duration severe acute graft versus host disease (GvHD). The trial will also evaluate the treatment of GvHD by the infusion of dimerizer drug (AP1903/rimiducid) in those subjects who present with GVHD that does not adequately respond to standard of care therapy.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
120
T cells transduced with CaspaCIDe® safety switch
administered to inactivate BPX-501 cells in the event of GVHD
Children's Hospital Los Angeles
Los Angeles, California, United States
Stanford University - Division of Pediatric Stem Cell Transplant & Regenerative Medicine
Palo Alto, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Dana-Farber Boston Children's Cancer and Blood Disorders Center
Boston, Massachusetts, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
Oregon Health Sciences University - Doernbecher Children's Hospital
Portland, Oregon, United States
University of Texas Southwestern-Children's Medical Center
Dallas, Texas, United States
Baylor College of Medicine/ Texas Children's Hospital
Houston, Texas, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States
Adverse Event
Demonstrate safety of BPX-501 MTD
Time frame: Month 24
TRM/NRM
Assess the cumulative incidence of non-relapse/transplant related mortality
Time frame: Day 180, Month 12
Disease-free survival
Disease-free survival rates after transplantation
Time frame: Month 24
Relapse
Cumulative incidence of relapse
Time frame: Month 12
Engraftment
Cumulative incidence of neutrophil and platelet engraftment, primary \& secondary graft failure
Time frame: Month 24
GvHD
Cumulative incidence and severity of acute and chronic GvHD
Time frame: Month 24
Rimiducid Efficacy
Time to resolution of acute or chronic GvHD after administration of rimiducid
Time frame: Month 24
Infection
Rate of infectious complications
Time frame: Month 24
Hospitalizations
Duration of hospitalization and rehospitalization
Time frame: Month 24
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