Clinical Trial

Disease: Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT04557436)

Disease info:

Leukaemia is cancer of the white blood cells which are responsible for fighting infection. In leukaemia, the bone marrow produces abnormal levels of white blood cells. B-cell acute lymphoblastic leukaemia (B-ALL) is an aggressive (fast-growing) type of leukaemia in which too many B-cell lymphoblasts (immature white blood cells) are found in the bone marrow and blood. It is the most common type of ALL. Also called B-cell acute lymphocytic leukaemia and precursor B-lymphoblastic leukaemia. 

Relapsed refers to when a patient has received active treatment, went off treatment and then the disease came back, whereas refractory refers to disease that is progressing despite active treatment.

 

Frequency:
ALL accounts for less than 1% of all cancers in the U.S., with around 6,540 new cases estimated in the U.S. in 2023. ALL is the most common type of cancer found in children, though it can affect adults too.
Official title:
Phase 1, Open Label Study of CRISPR-CAR Genome Edited T Cells (PBLTT52CAR19) in Relapsed /Refractory B Cell Acute Lymphoblastic Leukaemia
Who:

Principal Investigator: Waseem Qasim, Prof, UCL GOSH Institute of Child Health
Study Director: Paul Veys, PhD, MD, Great Ormond Street Hospital
Study Director: Kanchan Rao, PhD, MD, Great Ormond Street Hospital
Study Director: Ajay Vora, Prof, MD, Great Ormond Street Hospital

Sponsor:

Great Ormond Street Hospital for Children NHS Foundation Trust

Partners:

University College, London

Locations:

United Kingdom, London

Great Ormond Street Hospital, London, United Kingdom

Study start:
Aug. 12, 2020
Enrollment:
10 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene knock-out
Gene:
CD52 molecule, T Cell Receptor alpha and beta locus (TCRαβ)
Delivery method:
Lentivirus (LV) - Ex-vivo
Trial link:
https://clinicaltrials.gov/ct2/show/record/NCT04557436
Other links:

LinkB-cell Acute Lymphoblastic Leukemia Disease InformationLinkB-cell Acute Lymphoblastic Leukemia FrequencyLinkThe Role of Gene Editing Within CAR T-Cell Therapy Development - Interview with Professor Waseem QasimLinkLatest Gene Editing Clinical Trials for Cancer

Note:
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.
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting

Description

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.

Recognition by TT52CAR19 T cells mediates eradication of CD19+ leukaemia and other CD19+ B cells through T cell mediated cytotoxicity.

This is an open label, single-centre, phase I, cohort study using genome edited T cells to bring patients with relapsed or refractory B-cell acute lymphoblastic leukaemia (B-ALL) into remission in anticipation of a haematopoietic stem cell transplant (HSCT) that will hopefully prevent the leukaemia from returning. It involves a single infusion of allogenic T cells transduced with a self-inactivating (SIN) lentiviral vector in up to 10 subjects aged from 6 months to 18 years. The primary objective in this study is to test the safety and secondary objective will test the efficacy of this gene therapy procedure in this population.

Last updated: Dec. 28, 2023
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