A phase I, open-label, dose-escalation, safety, pharmacokinetic/-dynamic and clinical activity study of p38α inhibitor Skepinone-L to enhance therapeutic efficacy of Sorafenib in advanced hepatocellular carcinomas of non-viral origin
This early Phase 1 study is testing a new experimental medicine called Skepinone-L for people with advanced liver cancer (hepatocellular carcinoma). It specifically looks at liver cancer that is not linked to viral hepatitis (such as hepatitis B or C). Participants will take Skepinone-L alongside an existing, standard cancer drug called sorafenib. Because this is the first time Skepinone-L is being given to people, the main goal is to find out what dose is safe to use and to monitor any side effects. Doctors will also check how the body processes the new medicine and whether taking both drugs together helps to slow down or shrink liver tumours better than standard treatment alone. Joining the study is completely voluntary, and your healthcare team will monitor you very closely throughout.
At a glance
What is this study about?
Hepatocellular carcinoma is the most common type of primary liver cancer. While some liver cancers develop after viral infections like hepatitis B or C, others develop from non-viral causes, such as fatty liver disease or alcohol-related liver damage. For people with advanced liver cancer, treatments often aim to slow the cancer down and manage symptoms rather than cure it completely.
Sorafenib is an approved targeted drug commonly used to treat advanced liver cancer. However, over time, cancer cells can stop responding to it. Researchers have developed an experimental drug called Skepinone-L, which blocks a specific protein called p38α that cancer cells may use to survive and resist treatment. In laboratory tests, combining these two medicines helped make the cancer cells more sensitive to treatment.
This study is a Phase 1 'dose-escalation' trial. This means small groups of patients will start on a very low dose of Skepinone-L alongside sorafenib. If that dose is safe, the next group will receive a slightly higher dose. This careful process allows doctors to find the best, safest dose for future treatments while keeping a close eye on patient safety and tumour response.
Key takeaways
- Tests a new medicine (Skepinone-L) combined with standard treatment (sorafenib).
- Open to adults with advanced liver cancer that is not caused by a viral infection.
- Main goal is to find a safe and tolerable dose in humans.
- No placebo is used; all participants receive the active treatment.
- Requires frequent clinic visits and close monitoring by cancer specialists.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with advanced liver cancer (hepatocellular carcinoma) that cannot be cured with surgery alone. The cancer must not be caused by a chronic viral infection such as hepatitis B or hepatitis C.
To take part, participants will need to be well enough to carry out daily activities and have adequate liver, kidney, and bone marrow function. This ensures that taking the study medications is as safe as possible.
People whose liver cancer is caused by an active viral hepatitis infection, or those with other severe medical conditions that make cancer treatment unsafe, will likely not be able to join. Your hospital doctor or oncologist will run simple tests to confirm if this trial is right for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have been diagnosed with advanced liver cancer (hepatocellular carcinoma).
- My liver cancer is not linked to hepatitis B or hepatitis C.
- I am well enough to look after myself and manage light daily tasks.
- I am willing and able to travel to regular hospital appointments for check-ups.
What does participation involve?
Taking part involves regular visits to a specialist hospital clinic. Before starting, you will have safety checks including blood tests, heart traces (ECGs), and imaging scans such as CT or MRI scans. Once enrolled, you will take the study drugs as tablets or capsules and keep a diary of any symptoms or side effects.
Because this is an early-stage study, you will need frequent hospital visits at first—sometimes once or twice a week—so the team can take regular blood samples to see how the drug breaks down in your body. Scans will be repeated every few weeks or months to see how your tumour is responding. You will continue on the study for as long as the treatment helps you and does not cause unacceptable side effects, or until you choose to stop.
Potential risks and benefits
Locations (1)
- —UnverifiedGermany
Common questions
What does 'Phase 1' mean?
A Phase 1 trial is the earliest stage of human research. It focuses mainly on finding out if a new drug is safe, what the right dose is, and what side effects it might cause.
Will I receive a dummy drug (placebo)?
No. This is an open-label trial, which means there is no placebo. Everyone who takes part receives the active study medication alongside sorafenib.
What does 'non-viral' liver cancer mean?
Some liver cancers are caused by hepatitis B or C viruses. 'Non-viral' means your liver cancer was not caused by these infections, but instead by other factors such as fatty liver disease or lifestyle causes.
How often will I need to go to the hospital?
In the first few weeks, visits are frequent (often several times a month) for close monitoring and blood tests. Over time, visits usually become less frequent if you are doing well.
Can I stop taking part if I change my mind?
Yes. Taking part is completely your choice. You can leave the study at any point, and your standard cancer care will continue as normal.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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