Treatment With Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide After Intensive Therapy for People With High-Risk Neuroblastoma (NBL)
This study is for children and young people (up to age 30) with a types of cancer called high-risk neuroblastoma. These patients have already had strong treatments like chemotherapy, stem cell transplants, and radiation. The study wants to find out if adding two chemotherapy drugs (irinotecan and temozolomide) to a standard immune-boosting treatment (dinutuximab, sargramostim, and isotretinoin) can help stop the cancer from coming back or getting worse. Immune-boosting treatments help your body fight cancer cells. The study will check if this combination is safe and if it leads to better long-term results than the standard treatment alone.
At a glance
Results
Results from this study
Posted August 2024Results have been published for this study.
What is this study about?
This study is designed for children and young people who have been diagnosed with a type of cancer called high-risk neuroblastoma or ganglioneuroblastoma. These cancers can be quite challenging to treat. Before joining this study, patients will have already gone through very strong treatments, which usually include high doses of chemotherapy, followed by stem cell transplants (where healthy stem cells are put back into the body to help it recover), and radiation therapy. These initial treatments aim to remove as much of the cancer as possible.
The main goal of this particular study is to see if adding two chemotherapy drugs (irinotecan and temozolomide) to a current standard treatment can help prevent the cancer from returning or worsening after these intensive initial therapies. The standard treatment already involves three important medicines: Dinutuximab (an immunotherapy that helps the immune system target cancer cells without harming healthy ones), Sargramostim (which helps the body make more infection-fighting white blood cells), and Isotretinoin (which helps cancer cells mature and stop growing). The study team believes that combining these standard immune-boosting drugs with the two chemotherapy drugs might be even more effective.
Researchers will carefully monitor the patients to understand a few key things. Firstly, they want to make sure the new combination of drugs is safe and well-tolerated. Secondly, they will look at how well the treatment works in preventing the cancer from coming back or getting worse. This is a "Phase II" study, which means it's testing a new approach after initial safety has been shown, focusing on how well it works and any side effects.
Key takeaways
- This study is for children and young people with high-risk neuroblastoma.
- It tests if adding two chemotherapy drugs to standard immune-boosting treatment works better.
- Patients must have already completed intensive initial cancer treatments.
- The study aims to improve long-term outcomes and prevent cancer recurrence.
- It is a Phase II study, focusing on how well the treatment works and its safety.
- Regular medical tests and close monitoring will be part of participation.
Who may be eligible?
This study is for children and young people up to 30 years old who have been diagnosed with high-risk neuroblastoma or ganglioneuroblastoma. They must have already received a full course of intensive treatment for their cancer, including chemotherapy, stem cell transplants, and radiation, and their cancer must not have gotten worse or come back after these treatments.
There are specific details about your original diagnosis that your doctor will check. For example, it depends on whether a specific gene change (called MYCN amplification) was found in your cancer cells, your age at diagnosis, and other features of your tumour. Your overall health and ability to do daily activities should be good, and your doctors will check this using a special score.
Your doctor will discuss all the specific criteria to confirm if this study is a suitable option for you based on your medical history and test results.
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 30 years old or younger?
- Have you been diagnosed with high-risk neuroblastoma or ganglioneuroblastoma?
- Have you already finished your main, intensive cancer treatments (chemotherapy, stem cell transplants, radiation)?
- Has your cancer not returned or gotten worse after those intensive treatments?
- Are you generally in good health other than your cancer (as judged by your doctor)?
- Does your original cancer diagnosis meet specific criteria regarding gene changes or age at diagnosis?
What does participation involve?
If you join this study, you will receive a combination of medicines over several cycles. This includes: * **Temozolomide** (a chemotherapy drug) given by mouth or feeding tube daily for 5 days. * **Irinotecan** (another chemotherapy drug) given into a vein over 90 minutes daily for 5 days. * **Dinutuximab** (an immunotherapy drug) given into a vein over 10-20 hours daily for 4 days. * **Sargramostim** (to boost white blood cells) given as an injection under the skin or into a vein daily for 7 days. * **Isotretinoin** (to help cancer cells mature) given by mouth twice daily for 14 days.
Before and during the study, you'll have several tests to check your health and how the treatment is working. These may include scans like MRI (Magnetic Resonance Imaging), CT (Computed Tomography), MIBG (Iobenguane I-123) scans, and PET (Positron Emission Tomography) scans. You may also have blood and bone marrow tests. These tests help the doctors see the cancer and how your body is responding to the treatment. The total duration of your participation in the study, including how many cycles of treatment you receive and the follow-up period, will be clearly explained by your study team.
Potential risks and benefits
Locations (77)
- Banner Children's at DesertVerified postcodeMesa, United States
- Arkansas Children's HospitalVerified postcodeLittle Rock, United States
- Kaiser Permanente Downey Medical CenterVerified postcodeDowney, United States
- Children's Hospital Los AngelesVerified postcodeLos Angeles, United States
- Kaiser Permanente-OaklandVerified postcodeOakland, United States
- Children's Hospital of Orange CountyVerified postcodeOrange, United States
- UCSF Medical Center-Mission BayVerified postcodeSan Francisco, United States
- Children's Hospital ColoradoVerified postcodeAurora, United States
- Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical CenterVerified postcodeDenver, United States
- Connecticut Children's Medical CenterVerified postcodeHartford, United States
- Alfred I duPont Hospital for ChildrenVerified postcodeWilmington, United States
- Children's National Medical CenterVerified postcodeWashington D.C., United States
Common questions
What is high-risk neuroblastoma?
It's a type of cancer that starts in certain nerve cells, usually in young children, and is considered 'high-risk' based on how likely it is to grow or spread.
What is immunotherapy?
Immunotherapy helps your body's own immune system find and fight cancer cells more effectively.
Will I still receive standard treatment if I join this study?
Yes, this study combines two existing chemotherapy drugs with the standard immune-boosting therapy that's already shown to help high-risk neuroblastoma.
What is a 'Phase II' study?
A Phase II study looks at how well a treatment works and whether it's safe for patients, after earlier studies have shown it's safe to test.
What if the treatment isn't working for me?
Your doctors will regularly monitor your health and the cancer's response. They will discuss any changes or concerns with you and your family.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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