CMML201 version 1.0

• Research type

  Research Study

• Full title

  A phase 2 study of azacitidine in chronic myelomonocytic leukaemia (CMML)

• IRAS ID

  11787

• Contact name

  David Bowen

• Sponsor organisation

  The Leeds Teaching Hospitals NHS Trust

• Eudract number

  2008-006349-23

• ISRCTN Number

  21428905

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  Chronic myelomonocytic leukemia (CMML) is a rare blood cancer with excessive production of blood cells (myeloproliferative or MPD) which are abnormal in appearance (myelodysplastic or MDS) with a poor prognosis and average age at presentation of >70 year old patients. Average survival from diagnosis ranges from 11-17 months. Current treatment comprises standard supportive and mild chemotherapy (usually oral Hydroxycarbamide). This treatment is incapable of clearing the disease temporarily (remission). Even intensive chemotherapy has not been convincingly demonstrated to improve outcome although most patients are not suitable for this intensive approach due to advanced age and other ill-health. It is therefore evident that effective treatment capable of altering the natural history of the disease is currently lacking. Azacitidine, a new drug that works by altering the way that DNA functions (but does not change the DNA code) is the first drug to demonstrate a survival advantage for patients with MDS. Preliminary data suggest that Azacitidine is capable of inducing remission and haematological improvement (HI). Few clinical trials have addressed CMML specifically. We therefore propose to assess whether azacitidine is safe, can be easily tolerated and is effective in patients with newly diagnosed or previously treated CMML in a preliminary (phase 2) study. All eligible patients will receive treatment with at least 6 courses of Azacitidine. Those patients who respond may continue treatment until disease progression or development of unacceptable toxicity. Surplus of blood and bone marrow samples from patients who consent for research studies will also be used in a number of laboratory studies to investigate which patients may respond best to Azacitidine treatment and more about how the treatment works.If the CMML 201 Trial is successful, we will test Azacitidine against the current gold standard treatment of blood transfusions plus hydroxycarbamide in a larger trial.

• REC name

  North East - York Research Ethics Committee

• REC reference

  09/H0903/13

• Date of REC Opinion

  28 Apr 2009

• REC opinion

  Further Information Favourable Opinion