NCT04557436

TT52CAR19 Therapy for B-cell Acute Lymphoblastic Leukaemia (B-ALL)

Phase 1, Open Label Study of CRISPR-CAR Genome Edited T Cells (PBLTT52CAR19) in Relapsed /Refractory B Cell Acute Lymphoblastic Leukaemia

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Great Ormond Street Hospital for Children NHS Foundation Trust · Academic / Other
• Sex: All
• Age: 6 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

PBLTT52CAR19 modified T cells are allogenic engineered human T cells (defined as TT52CAR19 +TCRαβ-) prepared for the treatment of CD19+ B cell leukaemia. The cells are from healthy adult volunteer donors and are not HLA-matched. They have been transduced to express and anti-CD19 chimeric antigen receptor (CAR19) using a lentiviral vector that also incorporates CRISPR guides for genome editing of CD52 and TRAC loci in the presence of transiently provided Cas9. Recognition by TT52CAR19 T cells mediates eradication of CD19+ leukaemia and other CD19+ B cells through T cell mediated cytotoxicity. This study aims to apply PBLTT52CAR19 T cells to secure molecular remission in children with relapsed/refractory B-ALL ahead of programmed allogeneic stem cell transplantation. The cells are to be used in a time-limited manner for their anti-leukaemia effects and then depleted by standard pre- transplant conditioning.

Conditions

• B Acute Lymphoblastic Leukemia

Interventions

Type	Name	Description
DRUG	PBLTT52CAR19	gene therapy

Timeline

• Start date: 2020-08-12
• Primary completion: 2024-03-05
• Completion: 2024-03-05
• First posted: 2020-09-21
• Last updated: 2024-03-07

Locations

1 site across 1 country: United Kingdom