Monocle

  • Research type

    Research Study

  • Full title

    A phase 2 study of the monocyte-targeted histone deacetylase inhibitor tefinostat (CHR-2845) in chronic myelomonocytic leukaemia (CMML)

  • IRAS ID

    180498

  • Contact name

    Steven Knapper

  • Contact email

    knappers@cardiff.ac.uk

  • Eudract number

    2015-002281-23

  • ISRCTN Number

    ISRCTN17394489

  • Duration of Study in the UK

    3 years, 0 months, 0 days

  • Research summary

    Chronic myelomonocytic leukaemia (CMML) is an uncommon form of cancer that causes a build up of cells called monocytes in the bone marrow and blood. It mainly effects older people and the average length of survival is only 11-17 months. Most patients with CMML are unsuitable for bone marrow transplant therapy and there are currently very few other treatment options. There is a pressing need for new drug treatments
    which can target the abnormal monocytes without causing unacceptable side effects.

    Tefinostat is a new drug, taken by mouth as a capsule, which only becomes active after it is inside monocytes. In laboratory studies, tefinostat was effective against monocytic tumours and, in a small 'phase 1' clinical trial in 18 bone marrow cancer patients, it was shown to target monocytes without causing side effects.

    In this 'phase 2' trial, 40 patients with CMML will be treated at different hospitals in the UK to allow us to test the effectiveness of tefinostat in CMML as well as monitoring for side effects. Blood and bone marrow samples from patients will be tested in the laboratory to help us better understand how tefinostat works and to identify which patients are most likely to benefit in future.

  • REC name

    Wales REC 3

  • REC reference

    15/WA/0391

  • Date of REC Opinion

    15 Jan 2016

  • REC opinion

    Further Information Favourable Opinion