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

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Cancer type:

Acute leukaemia
Acute myeloid leukaemia (AML)
Blood cancers




Phase 3

This trial looked at a drug called elacytarabine (pronounced el-ah-site-ar-ah-been) for people with acute myeloid leukaemia (AML). It was for people with AML that had come back or did not respond to treatment.

Doctors usually treat AML with chemotherapy. The aim of chemotherapy is to get rid of the leukaemia.  This is called remission induction Open a glossary item. But for some people it doesn’t work, or the leukaemia comes back later on. If this happens it is more difficult to treat. So doctors are looking for new ways to help people in this situation. In this trial they looked at a drug called elacytarabine.

Elacytarabine is a 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 wanted to see if having the drug attached to a fatty acid helps to get more of the drug into the cells.

The aims of the trial were to find out

  • If elacytarabine is better than other treatments for people with AML that has not responded to induction treatment or has come back afterwards
  • More about the side effects


Summary of results

The researchers found that elacytarabine was not a useful treatment for people with AML that had come back or did not respond to treatment.

381 people took part and

  • Half had elacytarabine
  • Half had 1 of 7 usual AML treatments recommended by their doctor (Doctors call this the (control group)

The researchers looked at

  • How well the treatment worked
  • The length of time before the cancer came back
  • The length of time people lived after joining the trial

They found there was no difference between the people who had elacytarabine and the control group in any of these.

People in the elacytarabine group had more side effects such as liver problems, headaches and high levels of cholesterol and potassium in the blood.

The researchers concluded that elacytarabine was no better than any of the 7 usual AML treatments that people had in this trial.

We have based this summary on information from the team who ran the trial. 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. 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 Francis Giles
Dr Gail Roboz

Supported by

Clavis Pharma
NIHR Clinical Research Network: Cancer

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Last review date

CRUK internal database number:

Oracle 7294

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Cara took part in a clinical trial

A picture of Cara

"I am glad that taking part in a trial might help others on their own cancer journey.”

Last reviewed:

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