A trial of epacadostat for women who have a raised level of CA125 after treatment for ovarian, primary peritoneal or fallopian tube cancer

Cancer type:

Ovarian cancer




Phase 2
This trial looked at a drug called epacadostat for women with ovarian cancer who had no signs of cancer after chemotherapy. But who then had a raised level of a marker called CA125 in their blood a few months later. 
The trial was for women who had:
These cancers are all treated in the same way. So, when we use the term ovarian cancer in this summary, we are referring to all 3.

More about this trial

The usual treatment for ovarian cancer is chemotherapy. Having no signs of cancer afterwards, means that there has been a complete response to chemotherapy.

After treatment, women have blood tests to look for a marker called CA125. Having an increased amount of CA125 in the blood may mean that the cancer has come back. But the test isn’t completely reliable. You can have an increased level of CA125, without any other signs or symptoms of the cancer having come back. 

Doctors aren’t sure if women should have more treatment if this happens and what the best treatment is. Some women might have a hormone therapy Open a glossary item called tamoxifen to delay having more chemotherapy. But this isn’t a standard treatment Open a glossary item

In this trial, researchers looked at a drug called epacadostat (INCB024360). It is a type of targeted drug called an immunotherapy. It helps the body’s immune system Open a glossary item attack cancer cells. Doctors thought it might keep the cancer under control for longer. But they weren’t sure so wanted to find out more. 

Some women had tamoxifen and some had epacadostat. The trial compared these 2 drugs to see if epacadostat worked better than tamoxifen.

Summary of results

The trial team found that epacadostat didn’t work better than tamoxifen for women with a raised CA125 after chemotherapy for ovarian cancer. 
This trial was open for women to join between 2013 and 2014. The researchers published the results in 2017. 
The trial team had hoped to find 110 women to take part. But only 42 joined. This was because it was difficult to find women who were suitable and wanted to take part. The trial closed earlier than planned. 
About this trial
This phase 2 trial took place worldwide. 42 women joined. They were put into 1 of 2 treatment groups at random
  • 22 had epacadostat
  • 20 had tamoxifen

The trial team looked at the average length of time women lived without signs of their cancer getting worse. Researchers call this progression free survival. They found this was: 
  • just under 4 months in women who had epacadostat
  • just over 5.5 months in women who had tamoxifen 
Although these numbers look different, this wasn’t statistically significant. This means the difference could have happened by chance. 
The trial was stopped early. This was because it took a long time to find suitable women to join. 
The results suggested that it was very unlikely this trial would show that epacadostat worked better than tamoxifen at stopping the cancer getting worse. 
Side effects
The most common side effect of both groups was tiredness (fatigue). Women who had epacadostat had more skin problems such as rashes and itching. 
The research team concluded that epacadostat didn’t work better than tamoxifen for ovarian cancer. But all trial results help doctors and researchers understand more about different cancers 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

Dr Rebecca Kristeleit

Supported by

Incyte Corporation

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.

Charlie took part in a trial to try new treatments

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

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