Last year in the UK over 60,000 cancer patients enrolled on clinical trials aimed at improving cancer treatments and making them available to all.
A trial of ADI-PEG 20 for advanced liver cancer
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 (
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
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 (
The trial team looked at the
- 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.
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
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:
- brain tumours
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 (
How to join a clinical trial
Dr David Propper
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer