A trial of a drug called tefinostat for liver cancer

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. 

Results
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 

Conclusion
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 ) 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.