High Risk Neuroblastoma Study 1.8 of SIOP-Europe (SIOPEN)
This study, run by a European group called SIOPEN, aims to find the most effective treatments for children and young people (up to 21 years old) with high-risk neuroblastoma. This is a type of cancer that starts in certain nerve cells. Researchers are testing various treatment steps, such as strong chemotherapy, surgery, radiation, and newer immune-boosting drugs. They want to see if combining these treatments in different ways leads to better outcomes. The study has previously compared different chemotherapy methods and is now focusing on the best way to give immunotherapy to help the body fight the cancer. The overall goal is to improve the long-term health and survival rates for these patients.
At a glance
What is this study about?
This study is designed to help children and young people who have a type of cancer called neuroblastoma. Specifically, it focuses on those with a 'high-risk' form of the disease. This means their cancer is more aggressive or has spread, or it has certain genetic features that make it harder to treat. The study aims to find the best way to combine different treatments to give these children the best possible chance of recovery.
The treatment journey in the study is quite intensive. It typically starts with strong chemotherapy to shrink the tumour. This is followed by collecting the patient's own blood stem cells, which are like 'repair kits' for the bone marrow. Doctors then try to remove the main tumour with surgery. After this, even stronger chemotherapy is given, followed by giving the patient back their stem cells. Radiation therapy is also used to treat the original tumour site.
Finally, immunotherapy is a key part of the study. This uses special drugs to help the body's own immune system fight the cancer cells. Researchers are particularly interested in finding the best way to give these immune-boosting treatments. Over the years, the study has already helped doctors understand which chemotherapy combinations work best, and it continues to explore new approaches to improve long-term results for these young patients.
Key takeaways
- This study investigates treatments for high-risk neuroblastoma in children and young adults.
- It combines chemotherapy, surgery, radiation, and immunotherapy.
- The focus is on finding the most effective treatment combinations to improve patient outcomes.
- The study includes a comprehensive treatment plan followed by long-term monitoring.
- It aims to improve understanding and treatment of aggressive forms of neuroblastoma.
Who may be eligible?
This study is looking for children and young people up to 21 years old who have been diagnosed with neuroblastoma. Your doctors must have confirmed the type of cancer you have using standard tests.
You would be considered for this study if your neuroblastoma is classified as 'high-risk'. This includes certain stages of the disease, or if the cancer cells show a specific genetic change called 'MYCN amplification'. For children over 12 months old, Stage 4 neuroblastoma is also considered high-risk, even without this gene change.
It's important that you or your child haven't received much treatment for neuroblastoma before joining, typically no more than one cycle of specific chemotherapy. Also, if you're a female able to have children, you'll need a negative pregnancy test and agree to use effective birth control. Your doctor will make sure all criteria are met before you can 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.
- Is the person under 21 years old?
- Has neuroblastoma been diagnosed by a doctor?
- Is the neuroblastoma considered 'high-risk' (e.g., specific stages, MYCN gene change)?
- Has the person received very little previous treatment (e.g., no more than one cycle of specific chemotherapy)?
- Are you able to provide consent for participation (or is a legal guardian ready to)?
- If female and able to have children, are you willing to use birth control and have a negative pregnancy test?
What does participation involve?
If you join this study, your treatment journey will be closely managed by your medical team. It starts with intensive chemotherapy, followed by a procedure to collect your own blood stem cells. You will then have surgery to remove the main tumour, more strong chemotherapy, and then receive your stored stem cells back. Radiation therapy will be given to the area where the tumour was.
A key part of the study involves receiving special immunotherapy drugs, often given alongside another drug called isotretinoin. Doctors will carefully monitor your progress throughout these stages, including regular check-ups and tests. While the study has had different treatment 'randomizations' over time (meaning different groups received slightly varied treatments), your doctors will follow the most up-to-date plan. You will be followed up for at least 5 years after your treatment.
Potential risks and benefits
Locations (126)
- Women and Children´s HospitalVerified postcodeAdelaide, Australia· Recruiting
- Lady Cilento Children´s HospitalVerified postcodeBrisbane, Australia· Recruiting
- John Hunter Children's HospitalVerified postcodeNewcastle, Australia· Recruiting
- Royal Children's Hospital MelbourneVerified postcodeParkville, Australia· Recruiting
- Sydney Children's HospitalVerified postcodeSydney, Australia· Recruiting
- Children´s Hospital WestmeadVerified postcodeWestmead, Australia· Recruiting
- St. Anna KinderspitalVerified postcodeVienna, Austria· Recruiting
- Univ.-Klinik für Kinder- und Jugendheilkunde GrazVerified postcodeGraz, Austria· Recruiting
- Univ.Klinik f. Kinder-u. Jugendheilkunde InnsbruckVerified postcodeInnsbruck, Austria· Recruiting
- Landes- Kinderklinik LinzVerified postcodeLinz, Austria· Recruiting
- St. Johanns Spital LKH SalzburgVerified postcodeSalzburg, Austria· Recruiting
- Cliniques universitaires St-LucVerified postcodeBrussels, Belgium· Recruiting
Common questions
What is neuroblastoma?
Neuroblastoma is a rare type of cancer that mostly affects young children. It starts in certain nerve cells, often in the tummy or chest area.
What does 'high-risk' neuroblastoma mean?
High-risk means the cancer is more serious or aggressive, often because it has spread or has certain genetic features that make it harder to treat successfully.
What is chemotherapy and immunotherapy?
Chemotherapy uses strong medicines to kill cancer cells throughout the body. Immunotherapy uses drugs to help your body's own immune system find and destroy cancer cells.
Why is this study important?
This study helps doctors learn the best ways to combine powerful treatments to improve the chances of survival and long-term health for children with serious neuroblastoma.
Will I get to choose my treatment?
Parts of the study have involved randomly assigning patients to slightly different treatment groups to compare which is best. Your doctors will explain how this works for your specific situation.
How to find out more
Ruth L Ladenstein, MD, MBA, cPM
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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