A trial of eltrombopag with azacitidine for myelodysplastic syndrome (ELASTIC)

Cancer type:

Blood cancers
Myelodysplastic syndrome (MDS)




Phase 1

This trial is looking at a drug called eltrombopag alongside azacitidine for people with myelodysplastic syndrome (MDS).

Doctors can treat myelodysplastic syndrome (MDS) with azacitidine (pronounced ay-zah-sy-tid-een). This works but doctors are always looking for ways to improve treatment.

Eltrombopag (pronounced el-trom-boe-pag) is a drug that increases the number of cells called platelets Open a glossary item in your blood. We know from research that a drug similar to eltrombopag taken with azacitidine helps people with MDS.

The researchers want to find out if eltrombopag taken with azacitidine can do the same. To do this, they first need to find the best dose to give.

The main aims of this trial are to find out the highest, safest dose of eltrombopag to give with azacitidine and what the side effects are of having these 2 drugs together.

Who can enter

You may be able to join this trial if you are in one of the following situations. You

And all of the following must apply

  • You have a low number of platelets Open a glossary item (your doctor can tell you this)
  • Your other recent blood test results are satisfactory
  • You have had a bone marrow test in the last 8 weeks
  • You are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
  • You are willing to use reliable contraception during treatment and for 3 months afterwards if there is any chance you or your partner could become pregnant
  • You are at least 16 years old

You cannot join this trial if any of these apply. You

  • Have scarring (fibrosis) of your bone marrow Open a glossary item (your doctor can tell you this)
  • Have a large spleen Open a glossary item (your doctor can tell you this)
  • Have already had azacitidine
  • Have had a bone marrow transplant from a donor (allogeneic bone marrow transplant Open a glossary item)
  • Have already had other medications that boost the number of platelets in your blood
  • Have had an experimental drug as part of another clinical trial in the past 6 weeks
  • Have had another cancer in the past 3 years apart from some successfully treated early cancers Open a glossary item (the trial team can tell you more about this)
  • Have liver cirrhosis Open a glossary item
  • Have HIV
  • Have a parent who is from an east Asian country such as China, Japan, Taiwan or Korea

Trial design

This is a phase 1 trial. The researchers need 37 people to join. Everyone will have eltrombopag and azacitidine.

The first few people taking part will have a low dose of eltrombopag with azacitidine. If they don’t have any serious side effects, the next few people will have a higher dose. And so on, until they find the best dose to give. This is called a dose escalation study.

You have azacitidine as an injection under the skin. You have an injection Monday to Friday on the 1st week and then Monday and Tuesday the next week. You have this every 4 weeks. Each 4 week period is called a cycle of treatment.

Eltrombopag is a tablet you take once a day. You start taking it a week before you start azacitidine and continue for at least 9 weeks.

Everyone has 3 cycles of treatment. If your doctor thinks it is helping, you can have another 3 cycles. After this you may continue to have azacitidine only. Your doctor will talk to you about this.

The researchers will ask for a bone marrow test after your 3rd and 6th cycle of azacitidine. This is to see if it is helping you. They will also use some of these bone marrow samples for further research.

They will also ask for some extra blood samples. Most of these will be taken when you give your routine blood samples.

At the start of the trial the team will ask for some finger nail and toe nail clippings. They will use these to get a sample of your normal DNA Open a glossary item. They will compare the DNA in the clippings with any abnormal DNA in your blood and bone marrow sample.

Hospital visits

You see the doctor to have some tests before taking part in this trial. These tests include

  • A physical examination
  • Blood tests
  • Heart trace (ECG Open a glossary item)
  • Bone marrow test if needed
  • Collecting finger and toe nail clippings

During treatment you see the doctor every week for 13 weeks for blood tests. You have 2 more physical examinations and 2 more bone marrow tests during this time.

At the end of treatment you see the doctor for another physical examination and blood tests.

After treatment your doctor will tell you how often they want to see you.

Side effects

The most common side effects of azacitidine are

The most common side effects of eltrombopag are

  • A drop in blood cells causing an increased risk of infection, bruising and bleeding
  • Diarrhoea or constipation
  • Tummy (abdominal) pain
  • Headache
  • Dizziness
  • Tiredness (fatigue)
  • Loss of appetite
  • Chest pain
  • Being aware that your heart is beating more strongly (palpitations)
  • Shortness of breath
  • Flu like symptoms
  • A change to the way your kidneys and liver works
  • Rash and itchiness
  • Hair loss
  • Muscle, joints or bone pain
  • Difficulty sleeping
  • Pins and needles in your hands and feet
  • Dry eyes
  • Changes to your vision
  • Swelling of your hands and feet

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

We have information on azacitidine.



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 Alex Sternberg

Supported by

Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
University of Birmingham

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.

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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