Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant (NCT03301168)
🩺 Plain-English Summary (8th-Grade Level)
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).
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (15 items) and avoid the listed exclusions (9 items).
Location: Children's Hospital Los Angeles — Los Angeles, California
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous allograft at the time of inclusion
- Subject receiving an immunosuppressive treatment for GVHD treatment due to a previous allograft at the time of inclusion
- Dysfunction of liver (ALT/AST \> 5 times normal value, or bilirubin \> 3 times normal value), or of renal function (creatinine clearance \< 30 mL / min)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \< 40%)
- Current active infectious disease (including positive HIV serology or viral RNA)
- Serious concurrent uncontrolled medical disorder
- Pregnant or breastfeeding subject
- For subjects who have received more than 1 x 10E5 alpha/beta T cells/kg with the graft infusion the clinical trial site must contact the sponsor for approval to be eligible to receive BPX-501 infusion.
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (10 Sites)
- Children's Hospital Los Angeles — Los Angeles, California
- Stanford University - Division of Pediatric Stem Cell Transplant & Regenerative Medicine — Palo Alto, California
- Children's National Medical Center — Washington D.C., District of Columbia
- Children's Healthcare of Atlanta — Atlanta, Georgia
- Dana-Farber Boston Children's Cancer and Blood Disorders Center — Boston, Massachusetts
- Children's Hospital at Montefiore — The Bronx, New York
- Oregon Health Sciences University - Doernbecher Children's Hospital — Portland, Oregon
- University of Texas Southwestern-Children's Medical Center — Dallas, Texas
- Baylor College of Medicine/ Texas Children's Hospital — Houston, Texas
- Fred Hutchinson Cancer Research Center — Seattle, Washington
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.