Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow
This study is looking into a new treatment for children and adults with a serious type of cancer called high-risk neuroblastoma. This is for patients whose cancer in their bones or bone marrow hasn't fully responded to earlier treatments. The study uses two medications: Naxitamab, which is a specially designed antibody that targets cancer cells, and GM-CSF. Participants will receive these treatments for up to 101 weeks, which is about two years. After the treatment period, doctors will continue to check on participants for up to five years. The goal is to find out if this combination of medicines can be an effective way to help manage this complex condition.
En un coup d'œil
De quoi parle cette étude ?
This study is for children and adults dealing with a specific type of cancer called high-risk neuroblastoma. Neuroblastoma is a cancer that typically starts in nerve cells outside the brain. In this study, we're focusing on patients whose cancer is particularly stubborn, meaning it hasn't fully cleared up or responded well to standard treatments, especially when it's found in the bones or bone marrow. We know this can be a very challenging time, and researchers are always looking for better ways to help.
The main idea behind this study is to test a new treatment approach. It involves two medicines: Naxitamab and GM-CSF. Naxitamab is a special type of drug, sometimes called a 'monoclonal antibody,' that's designed to find and attack certain features on cancer cells. GM-CSF is a medicine that can help stimulate your body's immune system, which might help Naxitamab work better or help your body fight off infections. By combining these two treatments, doctors hope to find a more effective way to manage this difficult form of neuroblastoma.
The study aims to understand how well this treatment works and if it can improve outcomes for patients. It's a 'Phase 2' study, which means researchers are carefully evaluating the treatment's safety and effectiveness in a group of patients. If successful, this research could pave the way for a new treatment option for those facing high-risk neuroblastoma.
Points clés
- This study is for children and adults with high-risk neuroblastoma in their bones or bone marrow.
- It tests a new combination of medicines, Naxitamab and GM-CSF.
- The treatment phase lasts up to two years, with follow-up for five years.
- It aims to find better ways to treat neuroblastoma that hasn't fully responded to previous therapies.
- La participation est volontaire, et vous pouvez vous retirer à tout moment.
Qui peut être éligible ?
To join this study, you need to have a diagnosis of high-risk neuroblastoma in your bones or bone marrow. This study is for people whose cancer hasn't completely gone away or is still present after previous treatments. This includes situations where the cancer might be stable, has shown a small improvement, or has partially responded to earlier treatments.
There are also some important points that might mean you can't join. For example, if you've had other anti-cancer treatments, like chemotherapy, within three weeks before the study starts, you wouldn't be able to participate right away. Also, if your neuroblastoma is only outside of your bones and bone marrow, this study wouldn't be suitable for you. You also need to be generally stable and have good organ function, as assessed by the medical team.
The study is open to both children and adults, from one year old upwards. Doctors will explain all criteria in detail and help you understand if this study might be right for you.
Cet étude pourrait-elle vous convenir ?
Répondez à ces questions rapides pour voir si vous êtes éligible. Ceci est un guide uniquement — l'équipe de recherche prend la décision finale.
- Do I have a diagnosis of high-risk neuroblastoma?
- Is my neuroblastoma in my bones or bone marrow?
- Has my neuroblastoma not fully responded to previous treatments?
- Have I avoided other anti-cancer treatments for at least three weeks before the study starts?
- Are my major organs generally healthy (excluding hearing)?
- Am I at least one year old?
En quoi consiste la participation ?
If you decide to take part in this study, the treatment period will last for up to 101 weeks, which is about two years. Before each treatment cycle begins, you'll receive a medicine called GM-CSF for 5 days. Then, the main medication, Naxitamab, will be given on days 1, 3, and 5 of that same cycle. You'll get Naxitamab through a drip into your vein.
These treatment cycles will usually happen every four weeks. If your cancer responds well, you might continue with six cycles of this schedule. After that, if the doctors think it's still beneficial, the cycles might become less frequent, happening every eight weeks, until the 101-week mark. Throughout this time, you'll have regular hospital visits for treatment and check-ups to see how you're doing and how the treatment is working. After the treatment finishes, you'll enter a long-term follow-up period, where doctors will continue to monitor your health for up to five years after your very first treatment.
Risques et avantages potentiels
Emplacements (20)
- Riley Hospital for ChildrenCode postal vérifiéIndianapolis, États-Unis
- Memorial Sloan Kettering Cancer CenterCode postal vérifiéNew York, États-Unis
- The Hospital for Sick ChildrenCode postal vérifiéToronto, Canada
- RigshospitaletCode postal vérifiéCopenhague, Danemark
- Centre Médical Universitaire de Hambourg-EppendorfCode postal vérifiéHambourg, Allemagne
- Johannes Gutenberg-UniversitätCode postal vérifiéMainz, Allemagne
- University Hospital RegensburgCode postal vérifiéRegensburg, Germany
- Hong Kong Children's HospitalCode postal vérifiéHong Kong, Hong Kong
- Queen Mary HospitalCode postal vérifiéHong Kong, Hong Kong
- Tata Memorial CentreCode postal vérifiéMumbai, India
- Giannina Gaslini HospitalCode postal vérifiéGenoa, Italy
- Fondazione IRCCS Istituto Nazionale dei TumoriCode postal vérifiéMilan, Italie
Questions fréquentes
What is high-risk neuroblastoma?
High-risk neuroblastoma is a serious type of cancer that usually starts in nerve cells outside the brain and can be challenging to treat, often needing more intensive therapies.
What are Naxitamab and GM-CSF?
Naxitamab is a special drug that targets cancer cells, and GM-CSF is a medicine that helps boost your body's immune system, potentially helping the cancer treatment work better.
Combien de temps serai-je impliqué dans l'étude ?
The treatment part of the study lasts for up to 101 weeks (about two years), and you'll be monitored by doctors for up to five years after your first treatment.
Puis-je quitter l'étude si je change d'avis ?
Yes, absolutely. Your participation is completely voluntary, and you can choose to leave the study at any time without affecting your medical care.
Will I have to stay in the hospital?
The Naxitamab treatment is given as a drip, so you will need to attend the hospital for these sessions. Your team will advise on whether they require you to stay overnight.
Comment en savoir plus
Parlez toujours à votre médecin généraliste ou spécialiste avant de décider de participer à une étude.
Discussion
Discussion communautaire
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