A trial of romidepsin and azacitidine for acute myeloid leukaemia (ROMAZA)

Cancer type:

Acute leukaemia

Acute myeloid leukaemia (AML)

Blood cancers

Leukaemia

Status:

Results

Phase:

Phase 1

This trial looked at adding romidepsin to azacitidine for people with acute myeloid leukaemia (AML). It was for people who:

couldn’t have standard chemotherapy for AML and
had already had a lot of treatment for AML

The trial was open for people to join between 2013 and 2017. The team published the results in 2021.

More about this trial

Doctors usually treat acute myeloid leukaemia (AML) with chemotherapy. Some people can’t have standard chemotherapy Open a glossary item . So doctors were looking for other treatments to help this group of people.

At the time of this trial, azacitidine was a chemotherapy drug that doctors already used to treat people with AML.

Romidepsin blocks substances in the body called histone deacetylases. Cells need these to divide and grow. Blocking them may stop cancer growing. Drugs that block these enzymes are called histone deacetylase inhibitors or HDAC inhibitors. The doctors running this trial knew from research that other HDAC inhibitors helped people with other types of cancers.

The researchers thought romidepsin and azacitidine could help people with AML.

The main aims of this trial were to find out:

the best dose of romidepsin to have with azacitidine
how safe the combination is
how well the combination of romidepsin and azacitidine works

Summary of results

The trial team found the best dose of romidepsin to have with azacitidine. In this small trial they also found this combination of treatment worked and the side effects weren’t too bad.

Trial design
This was a phase 1 trial. 48 people took part. There were 2 parts. Part 1 looked at the best dose of romidepsin to have with azacitidine. Part 2 tested this dose in more people.

Results
13 people joined part 1. The team found the best dose of romidepsin to have with azacitidine.

35 people joined part 2. Everyone had the best dose found in part 1 of the trial with azacitidine.

The team checked how well treatment worked after 6 cycles of treatment Open a glossary item . They did this for people who had the best dose found in part 1. They found treatment worked for 9 out of 38 people. The leukaemia:

went away in 7 people
went away a little bit in 2 people

Side effects
Most of the side effects were mild but some were more severe.

The most common side effects of treatment included:

a drop in the number of blood cells causing an increased risk of infection, bruising and bleeding, tiredness and breathlessness
feeling or being sick
diarrhoea or constipation
injection site reaction
high temperatures (fever)
a drop in the amount of magnesium in the body

Conclusion
The trial team found the best dose of romidepsin to give with azatadine. They also found that the combination was safe and worked for some people in this small trial.

The team suggest that this combination should be compared to other treatments in a trial.

More detailed information
There is more information about this research in the reference below.

Please note, the information we link to here is not in plain English. It has been written for healthcare professionals and researchers.

Combination romidepsin and azacitidine therapy is well tolerated and clinically active in adults with high-risk acute myeloid leukaemia ineligible for intensive chemotherapy
J Loke and others
British Journal of Haematology, 2022. Volume 196, issue 2, pages 368–373.

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.