NCT01744223

Safety Study of Gene Modified Donor T-cells Following Partially Mismatched Stem Cell Transplant

A Phase 1/2 Dose Escalation Study Evaluating Safety and Feasibility of BPX-501 T Cells After Partially Mismatched, Related, T Cell-Depleted HSCT (Hematopoietic Stem Cell Transplant)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Bellicum Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate patients with blood cell cancers who are going to have an allogeneic (donor) blood stem cell transplant from a partially matched relative. The research study will test whether immune cells, called T cells, which come from the donor relative and are specially grown in the laboratory and then given back to the patient along with the stem cell transplant (T cell addback), can help the immune system recover faster after the transplant. As a safety measure, these T cells have been "programmed" with a "self-destruct switch" so that if, after they have been given to the patient, the T cells start to react against the tissues (called "graft versus host" disease, GVHD), the T cells can be destroyed.

Detailed description

This is a Phase1/2 dose escalation study evaluating the safety and feasibility of BPX-501 infused after partially mismatched, related (haploidentical), T cell-depleted HSCT. The purpose of this clinical trial is to determine whether BPX-501 infusion can facilitate engraftment, enhance immune reconstitution and potentially improve the graft versus leukemia (GVL) effect, with the potential for reducing the severity and duration of severe acute graft versus host disease (GvHD). The trial will evaluate the treatment of GvHD by the infusion of dimerizer drug (Rimiducid) in those subjects who present with GvHD that does not adequately respond to standard of care therapy.

Conditions

• Acute Lymphoblastic Leukemia
• Acute Myelogenous Leukemia
• Lymphoma
• Myelodysplastic Syndromes

Interventions

Type	Name	Description
BIOLOGICAL	BPX-501 dose 1	Subjects will receive 2x10E5 donor T cells/kg genetically modified with BPZ-1001 retroviral vector containing the iCasp suicide gene (BPX-501) after stem cell transplant.
DRUG	Rimiducid	Dimerizer drug administered by intravenous infusion in those subjects who develop GVHD after infusion of BPX-501 cells.
BIOLOGICAL	BPX-501 dose 2	Subjects will receive 5x10E5 donor T cells/kg genetically modified with BPZ-1001 retroviral vector containing the iCasp suicide gene (BPX-501) after stem cell transplant.
BIOLOGICAL	BPX-501 dose 3	Subjects will receive 1x10E6 donor T cells/kg genetically modified with BPZ-1001 retroviral vector containing the iCasp suicide gene (BPX-501) after stem cell transplant.
BIOLOGICAL	BPX-501 dose 4	Subjects will receive 3x10E6 donor T cells/kg genetically modified with BPZ-1001 retroviral vector containing the iCasp suicide gene (BPX-501) after stem cell transplant.
PROCEDURE	SCT	all subjects will receive an alpha beta depleted donor transplant as part of treatment

Timeline

• Start date: 2013-03-01
• Primary completion: 2019-10-09
• Completion: 2032-10-01
• First posted: 2012-12-06
• Last updated: 2022-07-12

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study