CARAML
Research type
Research Study
Full title
Phase 1 Study of base edited CAR T cells against AML: deep conditioning ahead of allogeneic stem cell transplantation
IRAS ID
1007053
Contact name
Waseem Qasim
Contact email
Sponsor organisation
Great Ormond Street Hospital NHS Trust
Eudract number
2021-004311-66
ISRCTN Number
ISRCTN14430213
Clinicaltrials.gov Identifier
Research summary
In this phase 1 clinical trial, we are testing an experimental medicine for the first time in children aged 6m up to 16 years with acute myeloid leukaemia (AML), which has come back (relapsed). The new product is made from white blood cells (T cells) collected from a healthy donor and changed so they can kill leukaemia cells. These ‘ready-made’ CAR T cells have been made using a new technique called Base Editing to modify their DNA code and have been given the code name ‘BE CAR-33’. This technique allows them to work after chemotherapy and also disarms them to prevent effects against normal cells. The main purpose of this study is to assess the safety of the ‘BE CAR-33’ therapy and to see if ready-made CAR T cells can get rid of T cell leukaemia ahead of a planned bone marrow transplant that will hopefully prevent the leukaemia from returning. Up to 10 patients will be included in this trial that will take place at Great Ormond Street Hospital. Patients will undergo careful screening to confirm that this treatment is adequate for them. If they join the study, we will need to wipe out their existing immune system with chemotherapy prior to BE CAR-33 infusion, as past research indicates that this improves the ability of T-cells to establish and grow. Patients will then receive a single infusion of the BE CAR-33 cells. They will be closely monitored via blood and bone marrow tests for safety and to check the levels of BE CAR-33 and leukaemia cells. We expect patients to be in hospital for 5 weeks for the BE CAR-33 therapy and the transplant will be scheduled 4 weeks after the end of BE CAR33. Patients will be monitored every month for the first three months and then every 6 months.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
23/LO/0014
Date of REC Opinion
15 Mar 2023
REC opinion
Further Information Favourable Opinion