A study of tefinostat for chronic myelomonocytic leukaemia (MONOCLE)

Cancer type:

Blood cancers
Chronic leukaemia




Phase 2

This study looked at a targeted cancer drug called tefinostat for chronic myelomonocytic leukaemia (CMML). 

This study was open for people to join between January 2017 and January 2018. These results were presented at a conference in 2018. 

More about this trial

CMML is a rare type of leukaemia. It causes a build up of a type of white blood cell called monocytes in the bone marrow and blood. The main treatments for CMML are supportive care and chemotherapy. 

Tefinostat is a targeted cancer drug Open a glossary item called a cancer growth blocker. It works by blocking an enzyme that monocytes need to grow and divide.

Researchers thought that tefinostat could help people with CMML. 

The aims of this study were to find:

  • how acceptable tefinostat was for people with CMML
  • how well it worked
  • how it affected quality of life 
  • out more about the side effects

Summary of results

The team found that tefinostat didn’t work as well as they hoped.

About this study
This was a phase 2 study. 21 people took part. 

Everyone took tefinostat once a day. 

The average length of time people took tefinostat was 16 weeks. 

The team were able to look at how well tefinostat worked in 13 people. They found that for:

  • 1 person their CMML had got a bit better (a partial response)
  • 9 people their CMML had not got better or worse (stable disease)
  • 3 people their CMML had got worse (progressive disease)

Side effects
The most common side effects included:

  • a change to how well the kidneys work causing an increased level of creatinine in the blood 
  • tiredness (fatigue)
  • loss of appetite
  • feeling or being sick
  • a drop in a type of blood cells called platelets causing an increased risk of bruising and bleeding
  • constipation or diarrhoea
  • blood and or protein in the urine
  • depression 
  • dizziness

The study team concluded people appeared to be able to take tefinostat without many problems. But it didn’t work very well. And they won’t investigate tefinostat further for people with CMML. 

Sometimes trials show that a treatment isn’t useful for people in a particular situation. But they add to our knowledge and understanding of cancer and how to treat it.

Where this information comes from    
We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (peer reviewed Open a glossary item) but may not have been published in a medical journal.  The figures we quote above were provided by the research 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

Dr Steven Knapper

Supported by

Cardiff University
NIHR Clinical Research Network: Cancer

Freephone 0808 800 4040

Last review date

CRUK internal database number:


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

Around 1 in 5 people take part in clinical trials

3 phases of trials

Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.

Last reviewed:

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