A trial of azacitidine for low risk myelodysplastic syndrome

Cancer type:

Blood cancers
Myelodysplastic syndrome (MDS)




Phase 3

This trial is looking at azacitidine (also known as Vidaza) for people with low risk myelodysplastic syndrome.

More about this trial

Myelodysplastic syndromes (MDS) are a group of blood disorders that cause a drop in the number of blood cells.  In some people, MDS can develop into acute myeloid leukaemia. This trial is for people with MDS that has a low risk of turning into leukaemia.

If you have MDS, your bone marrow Open a glossary item doesn’t make enough normal blood cells and you may need to have blood transfusions Open a glossary item. If your red blood cells are low, you have a drip containing red blood cells. If your platelets are low, you have a drip containing platelets. This is called supportive care. In this trial, researchers are looking at a drug called azacitidine, alongside supportive care.

Azacitidine is a chemotherapy drug that doctors can already use to treat MDS as an injection given under the skin or as a drip into a vein. But in this trial, they are looking at a new tablet form of azacitidine.

The aims of the trial are to

  • See if the tablet form of azacitidine helps people with low risk MDS and find out how it affects peoples’ quality of life
  • Learn more about what happens to azacitidine tablets in your body

Who can enter

You may be able to enter this trial if you

  • Have myelodysplastic syndrome (MDS) that is classified as lower risk – your doctor can confirm this
  • Have needed to have blood transfusions Open a glossary item at least once every 4 weeks for the last 3 months and have a low number of platelets
  • Have satisfactory results from other blood tests
  • Are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
  • Are at least 18 years old
  • Are willing to use reliable contraception during the trial and for at least 3 months afterwards if there is any chance you or your partner could become pregnant

You cannot enter this trial if you

  • Have MDS that is intermediate risk 2 or high risk – your doctor can advise you about this
  • Have MDS as a result of exposure to a chemical or treatment with chemotherapy or radiotherapy (secondary MDS) unless you finished the anti cancer treatment at least 24 weeks (nearly 6 months) ago
  • Have a type of MDS that could be treated in other ways – the trial doctor can explain this
  • Have already had azacitidine or drugs called lenalidomide or decitabine (or a similar drug) unless you had the last dose at least 24 weeks ago
  • Take drugs to remove iron from your body (chelating agents), unless you have been on a stable dose for at least 8 weeks or your dose is reducing
  • Take steroids Open a glossary item unless you take them for another medical condition and have been on a stable or decreasing dose for at least a week
  • Have had a stem cell transplant
  • Have had drugs that target MDS, drugs that stimulate the growth of blood cells, chemotherapy, a drug called hydroxyurea or an experimental drug in the last 4 weeks
  • Still have side effects from any earlier treatment
  • Have  a low number of red blood cells (anaemia Open a glossary item) or a low number of platelets for reasons other than having MDS
  • Have had any other cancer in the last 3 years, unless it was a very early stage and has been successfully treated – the trial team can advise you about this
  • Have had a heart attack in the last 6 months or have certain other heart problems – the trial team can advise you about this
  • Have any problem with your digestive system Open a glossary item that could affect how you swallow and absorb tablets
  • Have an infection that can’t be controlled with medication
  • Are known to be HIV or hepatitis C positive or have active hepatitis B infection
  • Have any other medical condition or mental illness that the trial team think would stop you taking part, or would make it unsafe for you to do so
  • Are known to be very sensitive to azacitidine or a drug called mannitol
  • Are pregnant or breastfeeding

Trial design

This international phase 3 trial will recruit 386 people, including about 36 at 13 hospitals in the UK.

It is a randomised trial. The people taking part are put into treatment groups by a computer. Neither you nor your doctor will be able to decide which group you are in.  And neither of you will know which group you are in. This is called a double blind trial.

Everybody taking part will continue to have best supportive care including blood transfusions and platelet transfusions.

Half the people taking part have azacitidine tablets. The other half have dummy tablets (placebos Open a glossary item).

trial diagram

You take the azacitidine tablets (or dummy tablets) every day for 3 weeks, followed by a week without treatment. As long as you don’t have bad side effects, you can continue having treatment in this way for as long as it helps you. The trial team will explain exactly how and when to take the tablets.

The researchers will look at blood samples you give during treatment to learn more about how much of the trial drug is absorbed into your bloodstream and how long it stays in your body.

The trial team will ask you to fill out a questionnaire before you start treatment, every 4 weeks during treatment and after you finish treatment. The questionnaire will ask about side effects and how you’ve been feeling.  This is called a quality of life study.

They will also give you a diary card to keep at home. On this you write down

  • The number of tablets you take and when you take them
  • Whether you take other medication to stop you feeling or being sick
  • How much water you drink when you take your tablets

Hospital visits

You see the trial team and have some tests before you start the trial treatment. The tests include

  • Physical examination
  • Blood tests
  • Urine test
  • Heart trace (ECG Open a glossary item)
  • Bone marrow test

The trial team will also take a swab from the inside of your cheek. They will use this sample, along with samples of your blood and bone marrow to look for genetic and chemical changes that might affect how MDS responds to the trial drug. These are called biomarkers Open a glossary item.

You go to hospital

  • Every week for the first 8 weeks of treatment
  • Every 2 weeks for the rest of the 1st year
  • Then once every 4 weeks if you carry on having treatment for longer than a year

You have regular blood tests throughout the trial. You have a bone marrow test after 3 months, 6 months, 1 year and then every 6 months after that.

When you finish the trial treatment, you will see the trial team again 4 weeks later. After that, they will contact you (or your own doctor) by phone every 3 months to see you how you are.

Side effects

The most common side effects of azacitidine include

The doctor will talk to you about all the possible side effects before you agree to take part in the trial.


Sutton in Ashfield

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 P Vyas

Supported by

Experimental Cancer Medicine Centre (ECMC)

Questions about cancer? Contact our information nurses

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.

Wendy took part in a new trial studying the possible side effect of hearing loss

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"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

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