A trial of ADI-PEG 20 for advanced liver cancer

Cancer type:

Liver cancer




Phase 3

This trial looked at a drug called ADI-PEG 20 for liver cancer that had got worse after treatment. 

It was open to people with a type of liver cancer called hepatocellular carcinoma (HCC). And who had treatment that reached the whole body (systemic treatments) Open a glossary item.

This trial was open for people to join between 2011 and 2015. These results were published in 2018.

More about this trial

Doctors use a number of different treatments for HCC. But the cancer might get worse or come back. They are looking for new treatments to help people in this situation. In this trial, they are looked at a drug called ADI-PEG 20.

Liver cancer cells need an amino acid Open a glossary item called arginine to survive and grow.  ADI-PEG 20 breaks down arginine. So it might stop liver cancer cells growing. 

The aims of the trial were to:

  • see if ADI-PEG 20 helped people who have HCC that had got worse despite having other treatments
  • learn more about the side effects

Summary of results

The team found that ADI-PEG 20 didn’t help people with liver cancer that had got worse after treatment.

About this trial
This was an international phase 3 trial

635 people joined the trial worldwide. 56 people were from the UK.

It was a randomised trial. Everyone was put into 1 of 2 groups at random. Neither they nor their doctor chose which group they were in. 

  • 424 people had ADI-PEG 20 and best supportive care
  • 211 people had a best supportive care and a dummy drug (placebo Open a glossary item)

The trial team looked at the median Open a glossary item time that people lived. They found it was:

  • just under 8 months (7.8) for people who had ADI-PEG 20 and best supportive care
  • just under 7½ months (7.4) for people who had best supportive care and the dummy drug

They also looked at the median time people had no sign of their cancer getting worse. It was the same in both groups, just over 2½ months (2.6).

ADI-PEG 20 works by reducing the amount of the amino acid arginine in the body. For some people this reduction in arginine continued during the trial. For others if didn’t. The team compared how long people in these groups lived. They found that people where the arginine was reduced lived longer. 

Side effects
There was no difference in the side effects reported in both groups. The most common side effects included:

  • tiredness (fatigue)
  • loss of appetite
  • feeling sick
  • tummy (abdominal) pain
  • swollen legs and arms
  • high temperature 
  • cough

The team concluded that ADI-PEG 20 with best supportive care was acceptable as a treatment people with liver after initial treatment. But it didn’t extend their lives. 

Researchers are now looking at ways to lengthen the time arginine is reduced and improve the way ADI-PEG 20 works. 

The trial team are now doing clinical trials looking at ADI-PEG 20 with chemotherapy for the following cancers:

  • mesothelioma
  • sarcoma
  • brain tumours
  • leukaemia 

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 David Propper

Supported by

Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
Polaris Group

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.

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