- 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.
- WHAT SUBTYPE OF MDS: Chronic myelomonocytc leukaemia (CMML)
- FOR WHAT LEVEL OF MDS SEVERITY RISK: CMML specific prognostic score (CPSS) Intermediate-2 or high risk
- 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.
- To determine the safety and tolerability of Tefinostat.
- Overall clinical response rate.
- 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
- 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
- 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.
- 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.
- More information:
Please read information and always discuss trial information with your own physician.