NCT03639844

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Expanded Access Protocol for CaspaCIDe T Cells From An HLA-Partially Matched Related Donor After Negative Selection of TCR αβ+T Cells In Pediatric Patients Affected by Hematological and Other Disorders

• Status: No Longer Available
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Bellicum Pharmaceuticals · Industry
• Sex: All
• Age: 3 Months – 21 Years
• Healthy volunteers: —

Summary

Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.

Detailed description

This is an expanded access protocol of BPX-501 T cells infused after T cell-depleted HSCT in pediatric patients with non-malignant hematologic disorders eligible for treatment on the BP-U-004 study. The purpose of this protocol is to provide access to the CaspaCIDe system combination product (BPX-501 gene modified T cells and rimiducid) to patients on a case by case basis who do not meet the BP-U-004 protocol eligibility criteria. BPX-501 infusion can enhance immune reconstitution with the potential for reducing the severity and duration of severe acute GVHD.

Conditions

• Hurler Syndrome
• Inherited Metabolic Disorder
• Lysosomal Storage Disorder
• Metachromatic Leukodystrophy
• Inborn Errors of Metabolism

Interventions

Type	Name	Description
BIOLOGICAL	rivogenlecleucel	BPX-501 T cells are genetically modified with a suicide safety switch. The cells are infused after T cell-depleted HSCT to potentially enhance immune reconstitution while reducing severity and duration of GVHD.
DRUG	rimiducid	Rimiducid induces activation of the Caspase 9 suicide gene in BPX-501 T cells inducing apoptosis of the modified T cells in case of GVHD

Timeline

• First posted: 2018-08-21
• Last updated: 2020-10-05

Locations

2 sites across 1 country: United States