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

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Blood cancers

Myelodysplastic syndrome (MDS)

Status:

Closed

Phase:

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

Have myelodysplastic syndrome (MDS) that is intermediate 2 risk or high risk group
Have chronic myelomonocytic leukaemia (CMML) with abnormal cells (blasts) making up between 10 and 29 out of every 100 white blood cells (10% to 29%) and the blasts aren’t growing rapidly
Have acute myeloid leukaemia (AML) with abnormal cells making up between 20 and 30 out of every 100 white blood cells (20% to 30%)

And all of the following must apply

You have a low number of platelets (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 (your doctor can tell you this)
Have a large spleen (your doctor can tell you this)
Have already had azacitidine
Have had a bone marrow transplant from a donor (allogeneic bone marrow transplant )
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 (the trial team can tell you more about this)
Have liver cirrhosis
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 )
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

A drop in blood cells causing an increased risk of infection, bruising and bleeding
A reaction or pain at the injection site
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

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