REGOBONE_A Randomized Phase II, placebo-controlled, multicenter study evaluating efficacy and safety of regorafenib in patients with metastatic bone sarcomas
Bone sarcomas are rare cancers that start in the bone and can spread to other parts of the body. When standard treatments stop working, doctors need new ways to keep the cancer from growing. This trial is studying a targeted drug called regorafenib, taken as a tablet. Regorafenib works by cutting off the blood supply that tumours need to grow and spread. Adults with advanced bone sarcomas—including osteosarcoma, Ewing sarcoma, chondrosarcoma, and chordoma—will be randomly chosen to receive either regorafenib or a dummy tablet (placebo). Regular scans will check if the treatment keeps tumours stable over several weeks or months. The main goal is to find out if regorafenib can give patients more time without their cancer worsening, while monitoring any side effects closely.
At a glance
What is this study about?
Bone sarcoma is a rare type of cancer that begins in bone tissue. While initial treatments such as surgery or chemotherapy can be successful, the cancer sometimes comes back or spreads to other organs, such as the lungs. When this happens, doctors call it metastatic bone cancer, and finding effective treatments can be very challenging.
This research study, called the REGOBONE trial, looks at an oral medicine called regorafenib (also known as Stivarga). Unlike standard chemotherapy, which attacks all fast-growing cells, regorafenib is a targeted therapy. It blocks specific proteins that cancer cells rely on to build new blood vessels and multiply. By starving tumours of these signals, doctors hope to halt or delay cancer progression.
The trial focuses on several distinct types of bone sarcoma, including osteosarcoma, Ewing sarcoma, chondrosarcoma, and chordoma. Because these cancers are so uncommon, finding high-quality evidence through clinical trials is vital. The findings will help doctors understand whether regorafenib could become a valuable treatment option to keep tumours stable and help patients live longer.
Key takeaways
- Tests regorafenib tablets for adults with bone cancer that has spread.
- Open to patients with osteosarcoma, Ewing sarcoma, chondrosarcoma, or chordoma.
- Compares the active drug against a dummy pill (placebo) to see if it stops tumours growing.
- Treatment is taken as a daily tablet at home, with regular hospital check-ups and scans.
- Participation is entirely voluntary, with close monitoring by specialist cancer teams.
Who may be eligible?
This study is designed for adults aged 18 and over who have been diagnosed with a bone sarcoma that has spread to other parts of the body (metastatic disease).
It includes people with specific bone cancer types, such as osteosarcoma, Ewing sarcoma, chondrosarcoma, or chordoma. In most cases, participants will have already received standard treatments that have stopped keeping the cancer under control.
To take part, you must be well enough to carry out daily activities and be able to swallow tablets. You will also need healthy vital organs, which the study team will check through blood tests and heart monitoring to ensure it is safe for you to join.
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.
- Are you 18 years of age or older?
- Do you have a confirmed bone sarcoma (such as osteosarcoma, Ewing sarcoma, chondrosarcoma, or chordoma)?
- Has your cancer spread to other parts of your body?
- Are you able to swallow tablets daily?
- Are you able to travel to the study hospital for regular check-ups and scans?
What does participation involve?
If you join the trial, you will be asked to take study tablets once a day at home. Because this is a controlled study, a computer will decide whether you receive the active drug (regorafenib) or a dummy pill (placebo). Neither you nor your doctor will know which one you are taking.
You will need to attend hospital visits every few weeks. During these visits, doctors will check your general health, take blood tests, and carry out imaging scans (such as CT or MRI scans) to measure your tumours. You will stay on the study treatment for as long as your cancer remains stable and any side effects are manageable. You can choose to leave the study at any time.
Potential risks and benefits
Locations (1)
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Common questions
What is regorafenib?
Regorafenib (brand name Stivarga) is a targeted cancer treatment taken as a tablet. It works by blocking the signals tumours use to grow blood vessels.
What is a placebo and why is it used?
A placebo is a dummy tablet that looks identical to the active medicine. It helps researchers prove whether the improvements seen are truly caused by the drug.
Will I definitely receive the real medicine?
No. You will be randomly assigned by a computer to receive either regorafenib or the placebo. This gives everyone an equal and fair chance.
How long will I take the study tablets?
You will generally continue taking the tablets for as long as the cancer stays stable and the treatment is not causing unacceptable side effects.
Can I leave the trial if I change my mind?
Yes. You have the right to leave the study at any point, for any reason, and your regular medical team will continue to look after 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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