An Early Access Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma
This research study is for adults with a type of bile duct cancer called cholangiocarcinoma that has either spread or can't be removed with surgery, and has been treated with other medicines before. The study drug is a tablet called ivosidenib, taken once a day. Doctors want to understand more about how safe and effective it is, and how it impacts patients' quality of life. All eligible participants will receive the study drug. You would continue taking the tablets as long as your doctor sees a benefit and you wish to keep taking part. There will be regular check-ups, including physical exams, blood tests, and scans, throughout your time in the study and potentially for up to 18 months afterwards.
At a glance
What is this study about?
This study is focused on a specific type of cancer called cholangiocarcinoma, which affects the bile ducts. If your bile duct cancer has either spread to other parts of the body or can't be removed by surgery, and you've already had other treatments, this study might be for you. The main goal is to gather more information about a daily tablet called ivosidenib. This medication is already approved in some parts of the world, including the US and EU, for treating advanced cholangiocarcinoma.
The researchers want to confirm that ivosidenib is both safe and effective for patients like you. They will also be looking at how this treatment affects your quality of life. Everyone who joins the study and meets the requirements will receive the ivosidenib tablets. This study is described as 'open-label' and 'single-arm,' meaning that all participants will know what medication they are receiving, and everyone will get the same study drug.
Your participation helps doctors learn more about this treatment, which can improve care for people with cholangiocarcinoma in the future. The study is designed to closely monitor your health and how you respond to the treatment, making sure you are looked after throughout the process.
Key takeaways
- This study is testing a daily tablet called ivosidenib for advanced bile duct cancer.
- It's for patients whose cancer has a specific genetic change (IDH1 mutation) and has been treated before.
- All participants will receive the study drug, not a placebo.
- The study aims to confirm the safety and effectiveness of ivosidenib and its impact on quality of life.
- You'll have regular hospital visits for check-ups and monitoring.
- You can stop participating at any time if you wish, without affecting your care.
Who may be eligible?
To join this study, you need to have a type of bile duct cancer that can't be removed by surgery or has spread. It's important that your cancer has a specific change in a gene called IDH1 (doctors check for this with a special test). You also must have already tried at least one other treatment for your cancer, and any side effects from those previous treatments should have settled down.
There are also some requirements for women who could become pregnant and for men whose partners could become pregnant. They will need to use two reliable forms of contraception (birth control) throughout the study and for about a month after their last dose of the study drug.
However, you won't be able to join if you've already taken a similar IDH1-blocking drug, or if you've had an organ transplant. You also can't have had recent cancer treatments like chemotherapy, radiation, or major surgery within a few weeks of starting the study. Problems like active hepatitis B or C, HIV, or brain tumours needing steroid treatment would also mean you can't take part. Lastly, if you are pregnant or breastfeeding, this study is not suitable 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.
- Do I have bile duct cancer that has spread or can't be removed by surgery?
- Has my doctor confirmed my cancer has an IDH1 genetic change?
- Have I already had at least one other treatment for my cancer?
- Am I willing to use effective birth control if I or my partner could become pregnant?
- Have I avoided other cancer treatments (like chemotherapy) in the last two weeks?
- Am I free from active hepatitis B/C or HIV, and not currently pregnant or breastfeeding?
What does participation involve?
If you join the study, you will take two ivosidenib tablets (a total of 500mg) by mouth once every day. Each 'cycle' of treatment lasts 28 days. You can keep taking the tablets for as long as your doctor believes they are helping you, and as long as you want to continue in the study.
Your first visit will be a 'screening' appointment to make sure the study is right for you. After that, you'll have a study visit on the first day of each new 28-day cycle of treatment. These visits will include checks like an ECG (heart test), a physical examination, tests to see how your tumour is responding, and blood and urine samples.
There will be at least six study visits in total, even if you only complete one treatment cycle. After you stop taking the study drug, you'll have a final check-up within 42 days, and then follow-up visits every six months for up to 18 months to see how you're doing. If the drug becomes widely available at your hospital, you might stop taking it as part of the study, but doctors will still follow your progress.
Potential risks and benefits
Locations (84)
- Erebouni MCVerified postcodeYerevan, Armenia· Completed
- National Center of Oncology of Ra MVerified postcodeYerevan, Armenia· Completed
- Royal brisbane & Women's HospitalVerified postcodeBrisbane, Australia· Completed
- St Vincent's HospitalVerified postcodeFitzroy, Australia· Completed
- St John of God Hospital - Bendat Family Comprehensive Cancer Centre (BFCCC)Verified postcodeSubiaco, Australia· Completed
- Kinghorn Cancer CentreVerified postcodeSydney, Australia· Completed
- The Queen Elizabeth HospitalVerified postcodeWoodville, Australia· Completed
- Medizinische Universitaet GrazVerified postcodeGraz, Austria· Completed
- Ordensklinikum Linz GmbHVerified postcodeLinz, Austria· Completed
- Universitaetsklinik fuer Innere Medizin III, mit Hämatologie, internistischer Onkologie, Hämostaseologie, Infektiologie, Rheumatologie und Onkologisches ZentrumVerified postcodeSalzburg, Austria· Completed
- Medizinische Universitaet Wien Universitaetsklinik fuer Innere Medizin IVerified postcodeVienna, Austria· Completed
- Universite Libre de Bruxelles ULB -Verified postcodeBrussels, Belgium· Completed
Common questions
What is cholangiocarcinoma?
Cholangiocarcinoma is a rare type of cancer that forms in the bile ducts, which are tubes that carry digestive fluid from your liver to your small intestine.
What is ivosidenib?
Ivosidenib is a tablet medication taken by mouth that targets a specific genetic change (mutation) found in some cholangiocarcinoma cells.
Will I get a placebo (dummy pill)?
No, in this study, all eligible patients who join will receive the active study drug, ivosidenib.
How long will I be in the study?
You'll take the study drug as long as it's helping you and you wish to continue. You'll also have follow-up visits for up to 18 months after stopping the treatment.
What is an IDH1 gene mutation?
This is a specific genetic change in your cancer cells that the study drug is designed to target. Your doctors will check for this through a test.
How to find out more
Institut de Recherches Internationales Servier, Clinical Studies Department
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.