UCART123 for high risk Acute Myeloid Leukaemia
Research type
Research Study
Full title
Phase I, open label dose-escalation study to evaluate the safety, expansion, persistence and clinical activity of multiple infusions of UCART123 (allogeneic engineered T-cells expressing anti-CD123 chimeric antigen receptor) in patients with adverse genetic risk Acute Myeloid Leukaemia
IRAS ID
247301
Contact name
Ghulam Mufti
Contact email
Sponsor organisation
CELLECTIS SA
Eudract number
2018-001018-14
Duration of Study in the UK
3 years, 0 months, 0 days
Research summary
This study will evaluate the safety of delivering two doses of a chimeric antigen receptor T-cell for the treatment of acute myeloid leukaemia (AML). AML is a life-threatening blood cancer. For patients whose leukaemia has bad risk genetic features it is unlikely to be curable with current therapy. A chimeric antigen receptor T-cell is a type of immune cell that has been modified so that it specifically recognises a protein on the surface of an AML cell called CD123. In this study, the chimeric antigen receptor T-cell is referred to as UCART123. Once the modified immune cell (UCART123) recognises and binds to CD123 it is able to kill the AML cell. The use of UCART123 cells is a new way of treating leukaemia where by the immune system is harnessed to kill the leukaemia. Currently UCART123 has been studied in patients who fail to respond or who relapse after multiple traditional chemotherapies. In this trial, we aim to deliver the CAR-T therapy at an earlier time-point before patients may become too unwell from previous treatments. Hence, we propose the administration of UCART123 after a single course of chemotherapy for high risk AML patients. By delivering the therapy early before the patient is too unwell, and after a course of chemotherapy will have removed some of the AML cells, we additionally aim to deliver UCART123 while minimising the complications that may occur in patients who have large numbers of residual leukaemia cells. Patients will be admitted to hospital, treated with one dose of UCART123 and evaluated at 28 days. If there is still evidence of leukaemia present and they have suffered no significant complications from UCART123 they will proceed to a second dose of UCART123. All eligible patients will receive stem cell transplantation at the end of the study.
REC name
North East - York Research Ethics Committee
REC reference
18/NE/0271
Date of REC Opinion
15 Nov 2018
REC opinion
Further Information Favourable Opinion