A trial of azacitidine with or without vorinostat for acute myeloid leukaemia or high risk myelodysplastic syndrome (RAvVA)

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Cancer type:

Acute leukaemia
Acute myeloid leukaemia (AML)
Blood cancers
Leukaemia
Myelodysplastic syndrome (MDS)

Status:

Results

Phase:

Phase 2

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.

Vorinostat is a type of targeted cancer drug called a cancer growth blocker. It stops signals that cancer cells use to divide and grow.

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 showed that the combination of azacitidine and vorinostat was not better than azacitidine alone for acute myeloid leukaemia (AML) or high risk myelodysplastic syndrome (MDS).

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

Results
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.

Side effects
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).

Conclusion
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 (peer reviewed Open a glossary item) and published in a medical journal. The figures we quote above were provided by the trial team who did the research. 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 Charles Craddock

Supported by

Bloodwise Trials Acceleration Programme (TAP)
Celgene
Experimental Cancer Medicine Centre (ECMC)
Medical Research Council (MRC)
Merck
NIHR Clinical Research Network: Cancer
Oxford Partnership Comprehensive Biomedical Research Centre
University of Birmingham; Cancer Research UK Clinical Trials Unit

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Freephone 0808 800 4040

Last review date

CRUK internal database number:

9307

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

Cara took part in a clinical trial

A picture of Cara

"I am glad that taking part in a trial might help others on their own cancer journey.”

Last reviewed:

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