A trial of a drug called tefinostat for liver cancer

Cancer type:

Liver cancer




Phase 1/2

This trial looked at tefinostat for the most common type of liver cancer called hepatocellular carcinoma (HCC). 

This trial was open for people to join between 2012 and 2017. The team reported the results in 2019.

Cancer Research UK supported this trial.

More about this trial

There are a number of different treatments for cancer that starts in the liver.  But sometimes they stop working and the cancer gets worse. Researchers are trying to improve treatment for people with hepatocellular carcinoma. In this trial they looked at a drug called tefinostat. 

Tefinostat is targeted drug Open a glossary item called a cancer growth blocker. It stops signals that cancer cells use to divide and grow.

Researchers thought that tefinostat could help people with liver cancer. This was the first time people with liver cancer had tefinostat. 

The aims of the trial were to:

  • find the best dose of tefinostat for people with hepatocellular carcinoma (HCC)
  • see how well it works 
  • learn more about the side effects

Summary of results

The team found the best dose of tefinostat to give to people with hepatocellular carcinoma (HCC). 

Trial design
The study took place in the UK. It was a phase 1/2 trial. There were 2 parts to the trial. Part 1 was to find the best dose. Part 2 was to test this dose in more people. 

17 people joined part 1. They took tefinostat once or twice a day. 

The first people taking part had a low dose of tefinostat. The next few people had a higher dose if they didn’t have any side effects. And so on, until the researchers found the best dose to give. This is called a dose escalation study. 

Everyone had treatment for as long as it was working and the side effects weren’t too bad. 

Once the team had found the best dose in part 1, they hoped to test it in more people. 

The trial team found 2 doses of tefinostat to give. They recommended the higher dose for part 2 of the trial. 

Part 2 didn’t go ahead as planned. This was because there had been changes in how trials were set up and designed since this trial first began. For example, trials were run differently and there were newer treatments. The team say they would have had to redesign this trial which would take a long time. So they decided to close the trial earlier than planned. 

Side effects
Everyone had at least 1 side effect.

Some of the more common side effects of tefinostat were:

  • changes to how the liver and kidneys work
  • shortness of breath 
  • diarrhoea or constipation 
  • tummy pain
  • feeling or being sick
  • muscle or joint pain
  • loss of appetite 

The trial team found the best dose of tefinostat to have. The trial closed earlier than planned so they didn’t test this dose in more people. 

Even so, 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. As far as we are aware, the information they sent us has not been reviewed independently (peer reviewed Open a glossary item) or published in a medical journal yet. The figures we quote above were provided by the research team. 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

Cancer Research UK
Chroma Therapeutics
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
Queen Mary University of London

Other information

This is Cancer Research UK trial number CRUKD/12/011.

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.

Over 60,000 cancer patients enrolled on clinical trials in the UK last year.

Last reviewed:

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