A trial of ECX chemotherapy for cancer of unknown primary (CUP-ONE)

Cancer type:

Cancer of unknown primary (CUP)

Status:

Results

Phase:

Phase 2

This trial looked at epirubicin, cisplatin and capecitabine for cancer of unknown primary. It also looked at testing new ways to find out where the cancer started. It was supported by Cancer Research UK.

This trial was open for people to join between 2010 and 2014. The trial team first reported results at a conference in 2014, and reported further results in 2019.

More about this trial

Cancers are named according to where in the body they start. This is called the primary site. If cancer spreads to another part of the body, this is called secondary cancer.

Sometimes people have secondary cancers in one or more parts of the body, but doctors are unable to find the primary cancer. This is called cancer of unknown primary (CUP) or unknown primary cancer.

Doctors often use chemotherapy to treat cancer of unknown primary. In this trial, they looked at a combination of 3 chemotherapy drugs called epirubicin, cisplatin and capecitabine. This combination is known as ECX.

The aims of this trial were to find out:

how well ECX works for cancer of unknown primary
more about new ways of testing cancer tissue removed during a biopsy

Summary of results

The trial team concluded that ECX chemotherapy worked for cancer of unknown primary and didn’t cause too many serious side effects.

About this trial
This trial was for people with a type of cancer of unknown primary (CUP) called a carcinoma Open a glossary item . They all had cancer that had spread to one or more parts of the body such as the liver, lungs, bones or brain. They hadn’t had treatment before.

Everyone taking part had the same treatment – a combination of the chemotherapy drugs epirubicin, cisplatin and capecitabine (ECX). They had up to 8 cycles of treatment Open a glossary item .

Results
The research team looked at how many people’s cancer had responded to treatment. They found that the cancer had:

gone away or got smaller in 17 people (31%)
stayed the same or continued to grow in 34 people (62%)

CUP-ONE Graph 1

They weren’t able to assess how well the treatment worked in 4 people (7%).

They also looked at how long it was before the cancer started to grow, and it was nearly 7 months. And when they looked at how long people lived for, they found it was just over 10 months.

The trial team are now analysing all the information they gathered about changes in cells and genetic material. This may take some time, but we will update this page once the results are available.

Side effects
Everyone taking part had at least 1 side effect. Some were mild or short lived, but just under half (46%) had at least one side effect that was more serious.

The most common of these side effects included:

feeling or being sick
diarrhoea
extreme tiredness (fatigue)

We have more information about the side effects of ECX in our Cancer Drugs section.

Testing cancer tissue

The team analysed the samples of cancer tissue taken from people in the trial. They used 2 different methods to find out where in the body their cancer came from.

One method looked for changes in the genes of the cancer cells. This is called a gene assay. The other method looked at particular proteins called antigens present in the cancer. This is called immunohistochemistry Open a glossary item

. The team wanted to find out which method was best at diagnosing where in the body the cancer started.

They found that both methods worked as well as each other when trying to diagnose the primary site.

Conclusion
The trial team concluded that ECX chemotherapy worked for some people with CUP, and didn’t cause too many serious side effects.

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.