A prospective, randomized, placebo-controlled, double-blind, multicenter phase III registration clinical study to compare toripalimab (JS001) combined with lenvatinib versus placebo combined with lenvatinib as the 1st-line therapy for advanced hepatocellular carcinoma (HCC)
This clinical trial is testing a new treatment combination for advanced hepatocellular carcinoma, the most common type of primary liver cancer. Researchers want to see if adding an immunotherapy drug called toripalimab to a standard cancer medicine called lenvatinib works better than lenvatinib alone. All participants will take standard lenvatinib capsules every day. Alongside this, half of the patients will receive toripalimab through a drip into a vein, while the other half will receive an inactive dummy treatment (placebo) drip. A computer randomly decides who gets which treatment, and neither you nor your doctor will know which drip you are receiving. Doctors will carefully track how long the treatment keeps the cancer from growing and check for any side effects. The main aim is to find out if this combination helps people with advanced liver cancer live longer, healthier lives.
At a glance
What is this study about?
Hepatocellular carcinoma is the most common form of liver cancer. When it reaches an advanced stage, it means the cancer has grown into nearby blood vessels or spread to other parts of the body, making surgery to remove it unsuitable. At this stage, doctors rely on medicines that travel throughout the whole body to control the cancer, slow its spread, and help patients live longer.
Currently, a medicine called lenvatinib is one of the standard first treatments used for advanced liver cancer. Lenvatinib works by blocking signals that cancer cells need to grow and build their own blood supply. This trial aims to find out if combining lenvatinib with an immunotherapy medicine called toripalimab can provide even better results. Toripalimab is designed to help the body's own immune system recognise and attack cancer cells.
In this study, half of the participants will receive lenvatinib plus toripalimab, while the other half will receive lenvatinib plus a dummy medicine called a placebo. Comparing these two groups helps researchers prove whether toripalimab truly adds extra benefit. The main goal is to measure overall survival—how long patients live after starting treatment—while also monitoring cancer shrinkage, symptom control, and safety.
Key takeaways
- The trial tests if combining toripalimab with lenvatinib improves survival in advanced liver cancer.
- All participants receive the standard cancer drug lenvatinib, plus either toripalimab or a placebo drip.
- The study is for adults with advanced liver cancer who have not yet had whole-body medicine for their disease.
- Regular scans and blood tests will closely monitor your health, cancer response, and any side effects.
- Taking part is completely voluntary, and you can withdraw at any point without impacting your care.
Who may be eligible?
This trial is designed for adults aged 18 and over who have been diagnosed with advanced hepatocellular carcinoma (primary liver cancer) that cannot be cured with surgery or local treatments.
To take part, participants must not have received prior systemic drug treatment (such as chemotherapy or targeted medicines) for their advanced liver cancer, as this trial is testing first-line treatment. People must also be well enough in their daily lives to manage regular hospital visits and have reasonably good liver and kidney function.
You may not be eligible if you have certain other serious medical conditions, such as severe autoimmune diseases, uncontrolled heart problems, or active infections that could make immunotherapy unsafe. The study team will review your complete medical history and run tests to make sure joining the trial is safe 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 primary liver cancer (hepatocellular carcinoma).
- My cancer cannot be treated or cured with surgery.
- I have not yet received systemic drug therapy (such as targeted therapy or chemotherapy) for advanced liver cancer.
- I am generally able to get out of bed, look after myself, and attend regular hospital visits.
What does participation involve?
If you decide to take part, you will be randomly assigned by a computer to one of two groups. Everyone will swallow lenvatinib capsules once a day at home. In addition, you will visit the hospital clinic every few weeks to receive either toripalimab or the placebo as an infusion through a drip into your vein. Neither you nor your medical team will know which drip you are getting, ensuring the results are completely unbiased.
During your clinic visits, you will have regular health checks, including physical examinations, blood tests, heart traces (ECGs), and discussions about how you are feeling. You will also have periodic imaging scans (such as CT or MRI scans) every few weeks to see how your liver tumour is responding to the treatment.
You will continue the study treatment for as long as it is helping to control your cancer and any side effects remain manageable. If the cancer grows or side effects become too difficult, the treatment will stop. Afterwards, the study team will continue to check on your health and wellbeing through regular clinic appointments or phone calls.
Potential risks and benefits
Locations (2)
- —UnverifiedPoland
- —UnverifiedItaly
Common questions
What is a placebo, and could I receive one?
A placebo is an inactive dummy treatment that looks identical to the real medicine. In this trial, everyone gets the active medicine lenvatinib, but half also receive toripalimab while the other half receive a placebo drip. This helps doctors see if toripalimab truly makes a difference.
Will I have to pay for the trial medicines?
No, the clinical trial medicines and any extra tests or scans required specifically for the study are provided free of charge.
Can I leave the study if I change my mind?
Yes, you can leave the study at any time for any reason. Choosing to stop will not affect your relationship with your healthcare team or your access to standard NHS care.
How often will I need to visit the hospital?
Visits are typically every few weeks for drip infusions, blood tests, and health reviews, with imaging scans every two to three months. The trial team will give you a full schedule before you start.
What happens if my cancer gets worse during the trial?
If your scans show the cancer has grown, or if your doctor feels the treatment is no longer helping, you will stop the trial medicine. Your doctor will then discuss other standard treatment options with you.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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