UCART19 PALL study (Paediatric Acute Lymphoblastic Leukaemia)
Research type
Research Study
Full title
A phase 1, open label, non-comparative, monocenter study to evaluate the safety and the ability of UCART19 to induce molecular remission in paediatric patients with relapsed /refractory B acute lymphoblastic leukaemia UCART19-PALL study (UCART19-Paediatric Acute Lymphoblastic Leukaemia)
IRAS ID
200331
Contact name
Marta Garcia
Contact email
Sponsor organisation
Servier Research & Development Ltd
Eudract number
2015-004293-15
Duration of Study in the UK
3 years, 2 months, 1 days
Research summary
This is a first trial of an experimental new therapy involving cells from the immune system that have been genetically modified and designed to fight leukaemia. Children whose B-cell acute lymphoblastic leukaemia has returned and there are no treatment options left, will be included.
The cell therapy is called UCART19. The cells are T-cells, which are important white blood cells, involved in fighting infection. They are collected from a healthy donor and then changed in several ways. A receptor is added so that the UCART19 cells can recognise a marker on the B leukaemia cells, an interaction that leads to the death of the leukaemia cell. Other changes include removal of markers that reduce the chance of the patient’s immune cells recognising and killing the foreign UCART19 cell.
The aim of this study is to test the safety of this therapy and to see if the UCART19 cells can get rid of the leukaemia so that we can proceed to a stem cell transplant to hopefully prevent the leukaemia from returning. This trial will only take place at Great Ormond Street Hospital. Up to 10 patients will be included.
Patients will undergo careful screening to confirm that their leukaemia requires treatment. Prior to giving the UCART19 cells we need to wipe out the patient’s existing immune system. Research indicates that this improves the ability of the T-cells to establish and grow. Then a single infusion of the UCART19 cells will be given. The patients will be closely monitored via blood tests and bone marrow tests for safety reasons and to see what is happening to the UCART19 cells and their leukaemia. We expect patients to be in hospital for 2-4 weeks and a transplant to take place 6-12 weeks after UCART19. Patients will be followed for 12 months post transplant.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
16/LO/0440
Date of REC Opinion
24 May 2016
REC opinion
Further Information Favourable Opinion