Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation

In this phase 1 clinical trial, the investigators are testing an experimental medicine in children aged 6 months 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 Acute Myeloid Leukaemia ahead of a planned bone marrow transplant that will hopefully prevent the leukaemia from returning.

NCT Number	NCT05942599
Lead Sponsor	Great Ormond Street Hospital for Children NHS Foundation Trust Collaborators: Wellcome Trust
Conditions	Relapsed Acute Myeloid Leukaemia
Enrollment	10 participants
Start Date	2023-07-21
Primary Completion	2026-06-01 (estimated)
Study Completion	2026-06-01 (estimated)
Updated on ClinicalTrials.gov	2026-01-14