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Brief Title: TT52CAR19 Therapy for B-cell Acute Lymphoblastic Leukaemia (B-ALL)
Official Title: Phase 1, Open Label Study of CRISPR-CAR Genome Edited T Cells (PBLTT52CAR19) in Relapsed /Refractory B Cell Acute Lymphoblastic Leukaemia
Study ID: NCT04557436
Brief 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.
Detailed Description:
Minimum Age: 6 Months
Eligible Ages: CHILD, ADULT
Sex: ALL
Healthy Volunteers: No
Great Ormond Street Hospital, London, , United Kingdom
Name: Waseem Qasim, Prof
Affiliation: UCL GOSH Institute of Child Health
Role: PRINCIPAL_INVESTIGATOR
Name: Paul Veys, PhD, MD
Affiliation: Great Ormond Street Hospital
Role: STUDY_DIRECTOR
Name: Kanchan Rao, PhD, MD
Affiliation: Great Ormond Street Hospital
Role: STUDY_DIRECTOR
Name: Ajay Vora, Prof, MD
Affiliation: Great Ormond Street Hospital
Role: STUDY_DIRECTOR