A trial of ADI-PEG 20 for advanced liver cancer

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Liver cancer




Phase 3

This trial is looking at a new drug called ADI-PEG 20 for a type of liver cancer called hepatocellular carcinoma (HCC). It is for people whose cancer has got worse despite having other treatments that reach the whole body (systemic treatments Open a glossary item).

Doctors use a number of different treatments for HCC. But the cancer may get worse or come back and doctors are looking for new treatments to help people in that situation. In this trial, they are looking at a new 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 may stop liver cancer cells growing. The aims of the trial are to

  • See if ADI-PEG 20 helps people who have HCC that has got worse despite having other treatments
  • Learn more about the side effects

Who can enter

You may be able to enter this trial if you

  • Have hepatocellular carcinoma (HCC) that has got worse despite having other treatments that reach the whole body (systemic treatment Open a glossary item), or you had to stop other types of treatment because of bad side effects
  • Have cancer that can be measured on a scan
  • Finished treatments directly to the liver such as chemoembolisation, radiofrequency ablation or alcohol injections into your liver at least 4 weeks ago and at least 1 area of cancer is outside the area of the liver that was treated, or has got at least 20% bigger since the treatment
  • Have normal liver function (Child-Pugh grade A or B)
  • Have recovered from side effects of any other cancer treatment
  • Have satisfactory blood test results
  • Are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
  • Are willing to use reliable contraception during the trial if there is any chance you or your partner could become pregnant
  • Are at least 18 years old

You cannot enter this trial if you

  • Can have another treatment that may cure your cancer
  • Have cancer that has spread to your brain unless it is well controlled and you do not have fits (seizures)
  • Have had major surgery in the last 2 weeks, or have not fully recovered from earlier surgery (liver biopsies are allowed)
  • Have had any other cancer treatment in the last 2 weeks
  • Are taking part in another trial of an experimental drug
  • Have already had ADI-PEG 20
  • Have heart problems that are a cause for concern
  • Have an infection that needs treatment with antibiotics
  • Have another medical condition that the trial team think could affect you taking part in this study
  • Have had another type of cancer unless there are no signs of the disease and the trial doctors don’t think it will affect the outcome of this treatment
  • Are allergic to any drugs which have been treated in the same way as ADI-PEG 20 (they have been pegylated Open a glossary item) - you can check this with your doctor
  • Have ever had fits (seizures)
  • Have had bleeding from the large veins in your stomach or food pipe in the last 3 months, unless this was successfully treated
  • Have a build up of fluid in your tummy (ascites) that cannot be controlled with medication
  • Have had a blood transfusion Open a glossary item, other blood products, erythropoietin Open a glossary item or a growth factor called G-CSF in the last 7 days
  • Have taken alternative traditional medicine such as Chinese herbs in the last 2 weeks – the trial doctors can advise you about this
  • Are pregnant or breastfeeding
  • Are known to be HIV positive

Trial design

This international phase 3 trial will recruit about 600 people all together, including about 50 people in the UK. It is a randomised trial. The people taking part are put into 1 of 2 treatment groups by a computer. Neither you nor your doctor will be able to decide which group you are in. And neither of you will know which group you are in either. This is called a double blind trial.

People in both groups will have all treatment available to help with any cancer symptoms and side effects and to improve their quality of life. Researchers usually call this best supportive care Open a glossary item.

  • People in one group have best supportive care and ADI-PEG 20
  • People in the other group have best supportive care and a dummy drug (placebo Open a glossary item)

There will be twice as many people in the group having ADI-PEG 20 as in the group having the dummy drug.

You have ADI-PEG 20 (or the placebo) as injections into the muscle of your shoulder, buttocks or thigh. You have 1 or 2 injections, once a week for 12 weeks. So you may have up to 24 injections all together.

After 12 weeks of treatment, you have a CT scan or MRI scan to see how the treatment is affecting your cancer. If the scan shows that your cancer has not grown and you don’t have any bad side effects, you may have another 12 weeks of injections. If a scan after the next 12 weeks shows that your cancer is still not growing and you are not having any bad side effects, you can carry on having treatment for as long as it helps you.

Hospital visits

You see the trial team and have some tests before you start treatment. The tests include

The trial team will also get a sample of tissue that was removed when you had surgery or a biopsy Open a glossary item.

You go to hospital every week for 12 weeks. During this time, you have regular blood tests and more ECGs. The doctor will examine you each week and they will ask how you have been feeling and about any medication you have taken.

If you carry on having injections after the first 12 weeks, you will go to hospital once a week for as long as you continue to have treatment.

When you finish treatment, you see the trial team again about a month later.

Side effects

As ADI-PEG 20 is a new drug, there may be some side effects we don’t know about yet. The most common known side effects include

  • Rash, itch or tenderness at the injection site
  • Reddening of your face or other parts of your skin (flushing)
  • Feeling tired (fatigue)
  • Feeling sick
  • Diarrhoea
  • Taste changes
  • Aching muscles or painful joints

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

Questions about cancer? Contact our 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.

Wendy took part in a new trial studying the possible side effect of hearing loss

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"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

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