NCT02231710

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

A Study Evaluating BPX-501 T Cells and AP1903 for Prevention of Graft Versus Host Disease (GVHD) After Haploidentical, Related, T Cell-Depleted Hematopoietic Cell Transplantation for Non-Malignant Diseases

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Bellicum Pharmaceuticals · Industry
• Sex: All
• Age: 4 Months – 55 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.

Detailed description

This is a single arm dose finding study evaluating the safety and efficacy of a BPX 501 infusion (T cells genetically modified with the inducible Caspase 9 suicide gene) of 3x10E6 to 1X10E7 cells/kg followed by a Rimiducid infusion on day 7 after a partially mismatched, related, T cell-depleted hematopoietic cell transplantation (HCT) in patients with non-malignant diseases. The purpose of this clinical trial is to determine the dose of BPX 501 T cell infusion with subsequent planned infusion of Rimiducid which can facilitate engraftment and prevent the occurrence of GVHD.

Conditions

• Primary Immune Deficiency Disorders
• Hemophagocytic Lymphohistiocytosis
• Inherited Bone Marrow Failure Syndrome
• Hemoglobinopathies
• Metabolic Disorders

Interventions

Type	Name	Description
BIOLOGICAL	BPX-501 and Rimiducid	Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7

Timeline

• Start date: 2015-02-01
• Primary completion: 2015-09-01
• Completion: 2018-01-01
• First posted: 2014-09-04
• Last updated: 2024-03-25
• Results posted: 2024-03-25

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study