NCT03301168

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

Phase I/II Study of CaspaCIDe® T Cells From an HLA-Partially Matched Family Donor After Negative Selection of TCR αβ+T Cells in Pediatric Patients Affected by Hematological Disorders

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 120 (estimated)
• Sponsor: Bellicum Pharmaceuticals · Industry
• Sex: All
• Age: 1 Month – 26 Years
• Healthy volunteers: Not accepted

Summary

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 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 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.

Conditions

• Acute Lymphoblastic Leukemia
• Leukemia, Acute Myeloid (AML), Child
• Lymphoma, Non-Hodgkin
• Myelodysplastic Syndromes
• Primary Immune Deficiency Disorder
• Osteopetrosis
• Cytopenia
• Hemoglobinopathy in Children
• Anemia, Aplastic

Interventions

Type	Name	Description
BIOLOGICAL	BPX-501 T cells	T cells transduced with CaspaCIDe® safety switch
DRUG	Rimiducid	administered to inactivate BPX-501 cells in the event of GVHD

Timeline

• Start date: 2014-04-01
• Primary completion: 2021-05-11
• Completion: 2034-05-01
• First posted: 2017-10-04
• Last updated: 2022-07-12

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study