Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.
A trial of azacitidine with or without vorinostat for acute myeloid leukaemia or high risk myelodysplastic syndrome (RAvVA)
This trial looked at azacitidine and vorinostat for people who can’t have intensive treatment for acute myeloid leukaemia or high risk myelodysplastic syndrome.
More about this trial
Doctors usually treat acute myeloid leukaemia (AML) or high risk myelodysplastic syndrome (MDS) with chemotherapy. If MDS is high risk, it means that there is an increased risk that it will develop into AML. Treatment for AML and MDS can be intensive, and some people aren’t able to have intensive chemotherapy.
When this trial was done, there was no standard treatment for people who weren’t able to have intensive chemotherapy. In this trial, researchers looked at 2 drugs called azacitidine (Vidaza) and vorinostat.
Azacitidine is a chemotherapy drug that doctors were already using to treat AML in people who cannot have intensive chemotherapy.
The aim of the trial was to see if a combination of azacitidine and vorinostat is better than azacitidine alone, for people who can’t have intensive chemotherapy for AML or MDS.
Summary of results
This trial recruited 259 people. This included 217 people with AML and 42 people with MDS.
They were put into 1 of 2 treatment groups at random, and:
- 129 people had azacitidine
- 130 people had azacitidine and vorinostat
The research team looked at how many people’s AML or MDS improved with treatment. It was nearly the same in both groups at just over 4 out of 10 (40%).
They also looked at how long people lived for, and found it was similar in the two groups:
- 9.6 months for those who azacitidine
- 11.0 months for those had has azacitidine and vorinostat
The researchers looked at cells from samples of bone marrow from the people taking part. They looked for specific changes (mutations) in genes in the cells that may help them predict how well treatment will work. They found that if the cells had certain genetic changes, then treatment was less likely to work. The changes were in genes called CDKN2A, TP53 and IDH1.
The treatments in this trial did cause some side effects in:
- 106 out of 129 people (82%) who had azacitidine
- 110 out of 130 people (85%) who had azacitidine and vorinostat
Some of these side effects were mild or short lived, but some were more serious. The most common side effects were a drop in red blood cells, white blood cells and cells which help the blood to clot (platelets).
The research team concluded that the combination of azacitidine and vorinostat was not better than azacitidine alone for AML and high risk MDS. And they found that certain genetic changes that may affect how well treatment will work.
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
Professor Charles Craddock
Bloodwise Trials Acceleration Programme (TAP)
Experimental Cancer Medicine Centre (ECMC)
Medical Research Council (MRC)
NIHR Clinical Research Network: Cancer
Oxford Partnership Comprehensive Biomedical Research Centre
University of Birmingham; Cancer Research UK Clinical Trials Unit