OPTIMISE-FLT3

  • Research type

    Research Study

  • Full title

    OPTIMISE-FLT3 - Optimising Therapy in FLT3-mutated Acute Myeloid Leukaemia

  • IRAS ID

    1008479

  • Contact name

    Chris Shaw

  • Contact email

    resgov@cardiff.ac.uk

  • Sponsor organisation

    Cardiff University

  • ISRCTN Number

    ISRCTN34016918

  • Research summary

    Acute myeloid leukaemia is an aggressive blood cancer and is the commonest form of acute leukaemia in adults, the majority of who will die from the disease. Younger and fitter patients can have treatment aiming to cure the disease with cycles of intensive chemotherapy followed for some patients by stem cell. Survival rates have gradually increased following improvements to chemotherapy, transplantation, better general care measures and new targeted drugs for patients in specific AML sub-groups. This study focusses on a subgroup of AML with mutations in the FLT3 gene, found in about one-third of AML patients. These patients have worse overall outcomes due to increased rates of early disease relapse. Clinical trials recent have identified several promising strategies to improve outcomes for patients with FLT3 AML but these approaches have not yet been combined in a single trial. Currently the standard treatment is standard intensity DA chemotherapy combined with midostaurin. We will compare this standard treatment with two new combinations. One is standard intensity chemotherapy combined both midostaurin and GO; this combination has already undergone pilot testing in the AML19 study and was safe and appeared very effective. The second new combination is intensified chemotherapy (FLAG-Ida) combined with midostaurin & GO. Pilot safety testing of this combination is built into this study. 390 patients who decide to enrol in the study will be randomised to one of the three treatment schedules. They will receive up to 4 cycles of intensive chemotherapy treatment, including stem cell transplant in selected cases; during this time the response of the leukaemia to treatment will be measured by blood & bone marrow tests to see whether patient outcomes are improved in terms of increased survival, reduction in rates of relapse and reduction in the need for stem cell transplant and we will monitor for any increased side effects associated with the intensified treatment schedules.

  • REC name

    East Midlands - Derby Research Ethics Committee

  • REC reference

    24/EM/0220

  • Date of REC Opinion

    22 Aug 2024

  • REC opinion

    Further Information Favourable Opinion