A trial looking at azacitidine for chronic myelomonocytic leukaemia (CMML201)

Cancer type:

Blood cancers
Chronic leukaemia




Phase 2

This trial was done to see if azacitidine is a safe and helpful treatment for chronic myelomonocytic leukaemia (CMML). This trial was supported by Cancer Research UK.

Although chronic myelomonocytic leukaemia (CMML) has leukaemia as part of its name, it is classed by the World Health Organisation (WHO) as a 'myeloproliferative or myelodysplastic neoplasm' rather than as a leukaemia.

Doctors often treat CMML in the same way they would treat myelodysplastic syndrome (MDS) or myeloproliferative disorder (MPD). Treatment sometimes includes chemotherapy.

This trial looked at a chemotherapy drug called azacitidine (Vidaza) for CMML. Earlier clinical trials had shown that azacitidine may be useful for treating MDS. Researchers hoped that it would also be useful for treating CMML.

The aims of this trial were to see

  • How well azacitidine works for CMML
  • What the side effects are

Summary of results

The research team found that azacitidine worked for chronic myelomonocytic leukaemia (CMML), but not as well as they’d hoped.

This trial recruited 30 people with CMML. They had azacitidine injections on 7 days out of every 4 weeks. Each 4 weeks period is called one cycle of treatment.

The research team were able to look at how well the treatment worked in 28 of the 30 people who took part. They found that the CMML had

  • Gone away completely in 2 people
  • Got a bit better in 4 people
  • Stayed the same in 5 people
  • Got worse in 17 people

So the treatment had worked for a few people, but not as many as the trial team had hoped.

Azacitidine did cause some side effects, although they were often mild. The most common side effects were

  • Soreness or redness at the injection site
  • Feeling or being sick
  • A drop in blood cells which can cause an increased risk of infection, bleeding problems, tiredness and breathlessness.

The research team concluded that azacitidine didn’t cause too many side effects, but that it didn’t work as well as they’d hoped for CMML. They say that it shouldn’t be used as standard treatment for CMML, but suggest it is looked at further in other trials.

We have based this summary on information from the team who ran the trial. The information they sent us has been published in a medical journal. The figures we quote above were provided by the trial team. We have not analysed the data ourselves.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Professor David Bowen

Supported by

Cancer Research UK
Celgene Ltd
Experimental Cancer Medicine Centre (ECMC)
National Institute for Health Research Cancer Research Network (NCRN)
The Leeds Teaching Hospitals NHS Trust

Other information

This is Cancer Research UK trial number CRUK/08/034. 

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 3353

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Last reviewed:

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