A trial looking at chemotherapy before a stem cell transplant for children with acute myeloid leukaemia (MyeChild01)

Cancer type:

Acute leukaemia
Acute myeloid leukaemia (AML)
Blood cancers
Children's cancers
Myelodysplastic syndrome (MDS)




Phase 3

This trial is for children and young people with leukaemia that has a high risk of coming back after treatment. It is open to children and young people with:

This summary is 1 of 3 summaries of the MyeChild01 trial. We have information on:

 Cancer Research UK supports the MyeChild01 trial.

This study is for children and young people who haven’t reached their 18th birthday. We use the term ‘you’ in the summary but if you are a parent we are referring to your child.

More about this trial

When treating AML with chemotherapy there are 2 stages. The 1st stage is to get rid of the leukaemia cells in the bone marrow. This is called induction chemotherapy. The 2nd stage is to stop it coming back. This is called consolidation chemotherapy. 

After each stage doctors do some tests to work out the risk of your leukaemia coming back. Your doctor might suggest you have a stem cell transplant if you are at high risk. 

You have a combination of chemotherapy drugs before the stem cell transplant. This is called conditioning chemotherapy. 

The standard treatment Open a glossary item for this is called myeloablative conditioning (MAC). This works but it has many side effects. 

Reduced intensity conditioning (RIC) is another combination of chemotherapy drugs that doctors might use. 

Research suggests that RIC might be as good as MAC. And because it isn’t as severe as MAC it has fewer side effects. But there haven’t been any large randomised clinical trials to show this. 

In this trial researchers want to compare MAC with RIC. The combination of chemotherapy drugs they will use is: 

The aims of the trial are to find: 

  • if RIC is as good a conditioning chemotherapy as MAC before a stem cell transplant to treat AML 
  • if RIC has fewer side effects

Who can enter

The following bullet points list the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you. 

You may be able to join this trial if all of the following apply. You have one of the following:

And all of the following must apply:

  • you have had induction chemotherapy as part of the MyeChild01 trial or you have had 2 courses of mitoxantrone and cytarabine as induction chemotherapy outside of the MyeChild01 trial
  • you are considered able to have a stem cell transplant
  • you have a matched donor 
  • your heart works well enough, you have a test for this
  • you have satisfactory blood test results
  • you are willing to use reliable contraception if you are sexually active and there is any chance you or your partner could become pregnant
  • you are under 18 years old

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

  • have AML that has come back after treatment (relapsed) or continued to get worse during treatment (refractory) 
  • have acute promyelocytic leukaemia (APL)
  • have chronic myeloid leukaemia (CML) that is in blast crisis
  • have a disease where your bone marrow Open a glossary item can’t make blood cells (bone marrow failure syndromes)
  • have already had treatment with chemotherapy or a biological therapy Open a glossary item that could affect you taking part in this study for example previous treatment with an anthracycline chemotherapy drugs such as doxorubicin and idarubicin 
  • have Down’s syndrome Open a glossary item
  • are pregnant or breastfeeding

Trial design

This is an international phase 3 trial. In the UK the trial team need 60 children and young people to join this part of the MyeChild01 trial. 

This is a randomised trial. People taking part are put into treatment groups by a computer. Neither you nor your doctor can choose which group you are in. 

There are 2 groups:

  • busulfan and cyclophosphamide (MAC)
  • busulfan and fludarabine (RIC)

Diagram for MyeChild01

You have all your treatment as a drip through a central line. You have the treatment  given over 10 days before your transplant. 

Tissue and blood samples
The trial team will ask for extra blood samples and bone marrow samples Open a glossary item. These might be taken at the same time as the routine samples are taken.

They use some of the samples to look at different ways to measure how the leukaemia has responded to treatment. They hope this will help them identify people who are at a greater risk of their leukaemia coming back after treatment. 

Hospital visits

You have all your treatment as an inpatient in the hospital. 

Before starting treatment you have the following tests:

  • a physical examination
  • blood tests
  • a test to see how much oxygen is in your blood 
  • bone marrow test

After you have completed all your treatment you see the doctor:

  • every 3 months for a year 
  • every 6 months in year 2
  • once a year for at least another 3 years

And have the following tests:

  • a physical examination
  • blood tests
  • echocardiogram (ECHO Open a glossary item) 1, 3, 5 and 10 years after diagnosis

Side effects

The side effects of having a stem cell transplant include:

  • a drop in blood cells causing an increased risk of infection, bruising and bleeding 
  • tiredness
  • shortness of breath
  • feeling or being sick
  • sore mouth
  • diarrhoea
  • a change to the way your liver, kidneys, lung and heart works
  • not been able to have children (infertility)
  • graft versus host disease

We have more information on the side effects of a stem cell transplant

Your doctor will talk to you about the side effects and risks of having a stem cell transplant before you agree to take part.


Newcastle upon Tyne

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 Brenda Gibson

Supported by

Cancer Research UK
University of Birmingham

Other information

This is Cancer Research UK trial number CRUK/14/013.

You can read more information about the trial on the MyeChild01 website.

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.

Charlie took part in a trial to try new treatments

A picture of Charlie

“I think it’s really important that people keep signing up to these type of trials to push research forward.”

Last reviewed:

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