A trial of elacytarabine for acute myeloid leukaemia that did not respond to treatment or has come back

This trial is looking at a drug called elacytarabine for people with Acute Myeloid Leukaemia (AML) that has not responded to treatment, or has come back after 2 or 3 other types of treatment. People who are over 65 may have had only 1 other type of treatment if tests show certain changes to the chromosomes in their leukaemia cells (researchers call this adverse cytogenetics).

Doctors usually treat AML with chemotherapy. The aim of the chemotherapy is to get rid of the leukaemia and is called remission induction. But for some people this does not work, or the leukaemia comes back later on. If AML does come back, it is more difficult to treat and doctors are looking for new ways to help people in this situation. In this trial they are looking at a drug called elacytarabine.

Elacytarabine is made up of a drug called cytarabine and a fatty acid. Cytarabine is already used to treat AML, but it is often difficult to get enough of the drug into the leukaemia cells. Researchers hope that having the drug attached to a fatty acid will help to get more of the drug into the cells.

The aims of the trial are to

See if elacytarabine is better than other treatments for people with AML that has not responded or has come back after induction treatment
Learn more about the side effects

Recruitment

Start 17/08/2010

End 30/11/2012

Phase

Phase 3

Who can enter

You can enter this trial if

You have acute myeloid leukaemia and have already had 2 or 3 types of treatment to try to get rid of it (induction treatments) - you may be able to take part if you have only had 1 other type of treatment as long as you are over 65 years old and tests show there are certain changes to the chromosomes in your leukaemia cells (adverse cytogenetics)
You never went into complete remission, or you did respond to the 1st treatment you had but your leukaemia came back within 12 months and you didn’t respond to the 2nd treatment, or your leukaemia has come back within 6 months of having the last treatment you did respond to
More than 5% of your bone marrow is made up of leukaemia cells, or tests show there are leukaemia cells outside your bone marrow
You have already had cytarabine (unless you are over 65 years old)
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 the trial (and also for 3 months afterwards if you are male) if there is any chance that you could become pregnant or father a child
You are at least 18 years old

You cannot enter this trial if you

Have a type of AML called promyelocytic leukaemia
Have had an allergic reaction to cytarabine, or you are allergic to eggs
Have had any other treatment for leukaemia in the last 4 weeks (apart from hydroxyurea)
Have had an experimental drug as part of another clinical trial in the last 2 weeks
Have not recovered from side effects of any other treatment
Have had another type of cancer and the trial doctors think this could affect your taking part in this trial
Have liver or kidney problems that the trial doctors think would make it unsafe for you to join this trial
Have had a heart attack in the last 3 months, have other heart problems, or any other serious medical condition apart from your leukaemia
Are known to be HIV positive
Are pregnant or breastfeeding

Trial design

This is an international phase 3 trial that will recruit up to 380 people. It is a randomised trial which means the people taking part are put into treatment groups by computer. Neither you nor your doctor can decide which group you are in.

Half the people taking part have elacytarabine. Half have another treatment recommended by their doctor. This is called ‘doctors choice’ and is the control group in this trial.

If you are in the control group, your doctor selects your treatment from a group of drugs commonly used to treat AML. You may have 1 drug or a combination or drugs.

If you are in the elacytarabine group, you have the drug through a drip into a vein over 5 days. You have a number of blood tests during and after the treatment. Then about 3 weeks later you have a bone marrow test. If this shows the treatment has helped and you have recovered from any side effects, your doctor may suggest you have more elacytarabine.

You will be asked to fill out a questionnaire when you join the study and a number of times during treatment. The questionnaire will ask about any side effects you have had and about how you have been feeling. This is called a quality of life study.

Hospital visits

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

Physical examination
Blood tests
Bone marrow tests

During the treatment, you see your doctors and have blood tests regularly. You also have more bone marrow tests.

You will see the trial doctors again between a month and 6 weeks after you finish the trial treatment. Then they will continue to look at your blood test results and check how you are once a month. This may carry on until the trial has completely finished.

Side effects

As elacytarabine is quite a new drug, there may be some side effects we don’t know about yet. From earlier trials, doctors know the possible side effects include

A drop in the number of blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness
Fever
Headache
Constipation or diarrhoea
A drop in the level of potassium in your body
Temporary changes to the way your liver works
An allergic reaction to the drug