"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
A trial looking at treatment for children with acute myeloid leukaemia (AML 17)
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
This trial is looking at the treatment for children with acute myeloid leukaemia (AML). This trial is supported by Cancer Research UK.
There are different parts to this trial that are looking at adult AML and acute promyelocytic leukaemia. We have information about these other parts of the trial listed separately.
This trial is for children and young people up to and including the age of 15. We use the term ‘you’ in this summary, but if you are a parent, we are referring to your child.
We know from research that a group of chemicals called tyrosine kinases sends out signals that tell cells to grow and divide. Some leukaemia cells have a change (
To stop the AML coming back, doctors may use more chemotherapy. But they are not sure which chemotherapy might be the best.
The aims of this trial are to
- Find out the best dose of daunorubicin
- Look at CEP-701 to treat AML
Who can enter
You can enter this trial if you
- Have acute myeloid Leukaemia (AML)
- Are suitable for intensive chemotherapy
- Have satisfactory blood test results
- Are under 16 years of age
You cannot enter this trial if you
- Have acute promyelocytic leukaemia (APL)
- Have already had chemotherapy for your AML (apart from treatment with hydroxycarbamide tablets, or similar low dose treatment, to control your white blood cell count)
- Are in the blast transformation phase
- Have another type of cancer at the same time as your leukaemia or MDS
- Have Down's syndrome
- Are pregnant or breastfeeding
This is a national trial that aims to recruit 2,800 patients with AML and high risk MDS in the UK.
In the induction phase you will have daunorubicin.
After the first cycle of chemotherapy, you have a bone marrow test. This will give the doctor more information about your leukaemia and how it has responded to treatment so far. The doctor will discuss this with you and plan the rest of your treatment. You will be put into 1 of 3 treatment groups.
People in this group will continue on to have daunorubicin, unless your bone marrow test shows you have an abnormal
In this group the leukaemia cells have a change in the gene (mutation) called FLT3. People in this group will be out into 1 of 2 treatment groups. Neither you nor your doctor can choose which group you are in. This is called randomisation. You will have either CEP-701 or a dummy drug (placebo).
CEP-701, and placebo, is a liquid that you take home to have. You can only mix them with the following fruit juices
- V8® 100% vegetable juice
- Orange juice (smooth only)
You cannot have grapefruit juice while taking CEP-701, or the placebo.
In this group, the results of the bone marrow show that there is a high chance your leukaemia may come back after treatment. And the doctor will plan for a bone marrow or stem cell transplant. If you have chemotherapy before your transplant you will have
You have 2 cycles of chemotherapy in the induction phase. After each cycle you have a bone marrow test. If you are in
In the consolidation phase you have 2 courses of high dose Ara-C.
If you take part in this trial, the researchers will ask your permission to store some of your blood and bone marrow samples. These samples will be stored safely and may be used in the future, but for research purposes only. This is because studying these samples may help researchers learn more about leukaemia.
If you don’t wish to have your blood and bone marrow samples stored for research, you don’t have to. You can still take part in the trial.
Before you start treatment you will see the doctor and have various tests. These tests will include
- Blood tests
- Bone marrow tests
You will have a blood test and possibly a bone marrow test before each cycle of chemotherapy.
All the chemotherapy is given through a drip into a vein; you will probably have these as an outpatient. There is a possibility that some people may need to have their chemotherapy as an inpatient, so may stay overnight in hospital.
All chemotherapy has side effects. The most common side effects for the chemotherapy used in this trail are
- A drop in blood cells, causing an increased risk of infection, bruising, bleeding problems, breathlessness and tiredness
- Tiredness (fatigue)
- Hair loss
- Feeling, or being, sick
- Loss of appetite
- Sore mouth
- Loss of fertility
Etoposide can cause a metallic taste in the mouth.
Amsacrine and daunorubicin can turn your urine pink or red. This can start up to 2 hours after having them and can last for up to 2 days.
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.
Professor A K Burnett
Professor N H Russell
Cancer Research UK
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
This is Cancer Research UK trial number CRUK/08/025.