Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.
A study of tefinostat for chronic myelomonocytic leukaemia (MONOCLE)
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
Tefinostat is a
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)
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
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 (
How to join a clinical trial
Dr Steven Knapper
NIHR Clinical Research Network: Cancer