LTalloCAR
Research type
Research Study
Full title
An observational, long term post-transplant follow-up after allogeneic genome edited lentiviral transduced CAR T cells.
IRAS ID
1009055
Contact name
Waseem Qasim
Contact email
Sponsor organisation
Great Ormond Street Hospital NHS Trust
ISRCTN Number
ISRCTN15432015
Research summary
In this observational study, we are monitoring over the long-term patients who previously received ‘ready-made’ CAR T cells within phase 1 clinical trials.
‘Ready-made’ CAR T cells are universal cellular products that derive from healthy donor white blood cells. As they are created by using special gene editing techniques, they are called "genome-edited allogenic CAR-T cells". Their aim is to fight and reduce leukaemia ahead of bone marrow transplant (BMT) and they are administered to patients with relapsed/refractory acute lymphoblastic or myeloid leukaemia. They are not expected to persist after BMT.
The aim of our study is to monitor long term outcome and adverse effects after ‘ready-made’ CART cells and bone marrow transplant.
To date, three different ‘ready-made’ CAR T have been used in Phase 1 trials at Great Ormond Street Hospital, which are PBLTT52CAR19 for B-cell acute lymphoblastic leukemia, BE-CAR7 for T-cell acute lymphoblastic leukemia and BE-CAR33 for acute myeloid leukemia.
Children previously administered with one of these products and who have completed the treatment study will be eligible to participate in this long-term study.
Up to 20 patients will be included in this study that will take place at Great Ormond Street Hospital
The start of this study will be at the completion of the treatment study, 12 or 24 months after allogenic BMT (depending on which treatment study patients were enrolled).
As long-term persistence CAR T cells beyond SCT is not anticipated, only routine blood tests are required.
Patients will be monitored with yearly visits up to 15 years after BMT. This study will use arrangements already in place in BMT long term follow up. Study visits will match with the planned ones and blood samples will be collected as part of normal routine care.REC name
London - West London & GTAC Research Ethics Committee
REC reference
24/LO/0005
Date of REC Opinion
4 Mar 2024
REC opinion
Further Information Favourable Opinion