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The trial was unable to demonstrate that Tefinostat had any effect in modifying CMML

    1. SUBTYPE OF MDS: Chronic myelomonocytc leukaemia (CMML)
    2. SEVERITY OF MDS: CMML specific prognostic score (CPSS) Intermediate-2 or high risk
    3. NAME OF DRUG: Tefinostat, a histone deacetylase inhibitor that targets monocytes. Tefinostat will be administered orally on a continuous basis for at least 6 cycles each 28 days in length.

  1. Aims and benefits: This is a phase 2 study aimed at determining the effectiveness of Tefinostat in CMML as well as monitoring for side effects. The study is open to patients with CMML-2 and those with CMML-1 who are symptomatic.
    Primary outcomes:

    • To determine the safety and tolerability of Tefinostat.
    • Overall clinical response rate.

    Secondary outcomes:

    • Incidence and duration of response
    • Achievement of red cell and platelet transfusion independence
    • Overall survival
    • Incidence of and time to transformation of CMML to AML
    • Quality of life
  2. Basic inclusion criteria:
    • All patients with CMML-2.
    • Patients with CMML-1 who are symptomatic (includes red cell transfusion dependent, thrombocytopenia with platelets < 50x109/L, bleeding, white cell counts > 50x109/L, weight loss, symptomatic splenomegaly, extramedullary involvement) and/or CPSS Intermediate-2 or high risk.
    • Age > 18 years
    • ECOG performance status 0-2
  3. Basic exclusion criteria:
    • CMML with raised eosinophils.
    • Patients considered suitable for allogeneic stem cell transplant.
    • Previous chemotherapy for CMML except hydroxycarbamide or 5-Azacitidine.
    • Use of experimental drugs or therapy within 28 days of registration.
    • Severe kidney or liver impairment.
    • Seropositive for HIV infection, hepatitis B or C
    • Other malignancy within 3 years other than curatively-treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, organ-confined or treated non-metastatic prostate cancer with negative prostate specific antigen, in situ breast carcinoma after complete surgical resection or superficial transitional cell bladder carcinoma.
  4. Trial sites/locations and name of physician in charge of trial: 
    University hospital of Wales, Cardiff;
    Aberdeen Royal Infirmary, Aberdeen;
    St. James’s University Hospital, Leeds.
  5. More information:

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