Last updated on September 2017

Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant

Brief description of study

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).

Detailed Study Description

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 and retain the graft versus leukemia (GVL) effect, with the potential for reducing the severity and duration of severe acute GvHD. The trial will evaluate the treatment of GvHD by the infusion of dimerizer drug (AP1903/rimiducid) in those subjects who present with Grade III-IV acute GVHD, as well as those subjects with Grade II gut/liver GvHD or with Grade I/II GvHD (skin only) who progress or do not respond within 7 days to standard of care treatment.

Clinical Study Identifier: NCT02065869

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Neena Kapoor, M.D.

Children's Hospital Los Angeles
Los Angeles, CA United States
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Rajni Agarwal-Hashmi, MD

Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine
Palo Alto, CA United States
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David Jacobsohn, MD, ScM

Children's National Medical Center
Washington, D.C., United States
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Lakshmanan Krishnamurti, M.D.

Children's Healthcare of Atlanta
Atlanta, GA United States
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Susanne Baumeister, M.D.

Dana-Farber Boston Children's Cancer and Blood Disorders Center
Boston, MA United States
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Kris Mahadeo, MD, MPH

Children's Hospital at Montefiore
The Bronx, NY United States
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Eneida Nemecek, M.D.

Oregon Health Sciences University - Doernbecher Children's Hospital
Portland, OR United States
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Victor M Aquino, M.D.

University of Texas Southwestern-Children's Medical Center
Dallas, TX United States
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Swati Naik, M.D.

Baylor College of Medicine/ Texas Children's Hospital
Houston, TX United States
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Ann E Woolfrey, M.D.

Fred Hutchinson Cancer Research Center
Seattle, WA United States
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