A trial of decitabine and cytarabine for children and teenagers with acute myeloid leukaemia

Cancer type:

Acute leukaemia
Acute myeloid leukaemia (AML)
Blood cancers
Children's cancers




Phase 1/2

This trial looked at decitabine followed by cytarabine for acute myeloid leukaemia (AML). It was for children and teenagers whose AML had come back or treatment had stopped working. 

This trial was open for people to join between 2014 and 2016. The team published the results in 2019. 

More about this trial

Doctors often treat AML with chemotherapy. Sometimes it stops working or the leukaemia comes back. So researchers are always looking for ways to improve treatment for children and teenagers in this situation. 

When this trial was done, doctors already used a drug called decitabine to treat AML in people over 65. Researchers wanted to see if it also worked for people under the age of 18 who have AML. 

Everyone taking part in this trial had decitabine followed by another chemotherapy drug called cytarabine. It was already a usual treatment for leukaemia in children and teenagers at the time of this trial. 

The main aims of the trial were to: 

  • find the best dose of cytarabine to have with decitabine 
  • see how well this combination of treatment works for AML
  • see what the side effects are

Summary of results

The trial team found the best dose of cytarabine to give with decitabine that didn’t cause too many bad side effects. They found that the combination of treatment wasn’t useful for children and teenagers. 

Trial design
This was a phase 1/2 trial. 17 children and teenagers took part. Everyone had decitabine followed by cytarabine. 

Depending on when people joined the trial, some people had a lower dose of cytarabine. If they didn’t have any bad side effects the next few people had a higher dose .

Everyone had treatment in cycles Open a glossary item. Each 4 week period was a cycle of treatment. In each cycle they had:

  • decitabine on days 1 to 5 
  • cytarabine on days 8 to 12 

Of the 17 people who took part:

  • 9 had the lower dose of cytarabine
  • 8 had the higher dose of cytarabine

The team looked at the best dose of cytarabine to give. They found this was the higher dose. 

They also looked at whose leukaemia went away completely or a little bit. They had the results for 8 people who had the higher dose of cytarabine. They found the leukaemia: 

  • went away completely in 1 person
  • went away a little bit in 2 people

The combination of treatment didn’t work as well as the team had hoped. The response to treatment only lasted for a short period of time. So this trial closed earlier than planned. 

Side effects
The team looked at the side effects of having decitabine and cytarabine. 

Everyone taking part had at least one side effect. Some of the side effects were mild and didn’t last long. Some people had more severe side effects. Some of the most common severe side effects included: 

  • a drop in the number of red blood cells (anaemia Open a glossary item)
  • a drop in the number of white blood cells, and some people also had a temperature 
  • changes to how the liver works 

The team found that the side effects of the combination of treatment were manageable.

The trial team found that having decitabine followed by cytarabine was safe. Treatment didn’t work as well as the researchers had hoped so the trial closed early. 

Even so, trial results help doctors and researchers understand more about leukaemias and the best way to treat them. 

Where this information comes from    
We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (peer reviewed Open a glossary item) and published in a medical journal. The figures we quote above were provided by the trial team who did the research. We have not analysed the data ourselves.

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 Pam Kearns

Supported by

Janssen-Cilag International NV
NIHR Clinical Research Network: Cancer

If you have questions about the trial please contact our cancer 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.

Last reviewed:

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