A trial of naxitamab and GM-CSF for high risk neuroblastoma

Cancer type:

Children's cancers




Phase 2

This trial is looking at naxitamab with GM-CSF to treat neuroblastoma Open a glossary item  that has continued to grow during treatment or has come back afterwards. 

The trial is mainly for children and young people. We use the term 'you' in this summary, but if you are a parent, we are referring to your child.

More about this trial

Sometimes neuroblastoma comes back or treatment stops working. So doctors are looking for treatments to help this group of people.

In this trial they are looking at a new drug called naxitamab. It is an immunotherapy. It targets a protein on the surface of neuroblastoma cells called GD2. This helps the immune system Open a glossary item recognise and attack cancer cells.    

In this trial everyone also gets GM-CSF Open a glossary item. This is a drug called a growth factor. Doctors sometimes use it to increase the number of white blood cells Open a glossary item in the body. 

The main aims of the trial are to find out:

  • how well naxitamab and GM-CSF work
  • how safe it is to have naxitamab and GM-CSF together
  • how treatment affects quality of life

Who can enter

The following bullet points are a summary of the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you. 

Who can take part

You may be able to join this trial if all of the following apply. You:

  • have high risk neuroblastoma 
  • have had treatment for neuroblastoma but it stopped working or the neuroblastoma has come back in the bone or bone marrow Open a glossary item. If you have it only in the bone then it must be outside the area you had any radiotherapy to. If it is only in the bone marrow then it must be in more than 5%. 
  • have acceptable blood test results
  • are well enough to take part (Karnofsky or Lansky score of at least 50)
  • are at least 1 year old

Who can’t take part

Neuroblastoma related
You cannot join this trial if any of these apply. You: 

  • have neuroblastoma that shows up on a scan and is outside the bone and bone marrow 
  • have neuroblastoma that is getting worse quickly 
  • had neuroblastoma in the brain or spinal cord within 6 months of the first dose of GM-CSF
  • have had treatment with a drug called omburtamab within 6 months of the first dose of GM-CSF
  • have had a stem cell transplant with somebody else’s cells (allogeneic transplant Open a glossary item) or you have had a donor lymphocyte infusion (DLI) within 6 months of the first dose of GM-CSF. Your doctor will know this. 
  • had a stem cell top up within 2 months of the first dose of GM-CSF
  • have had cancer treatment that included chemotherapy or immunotherapy Open a glossary item within 3 weeks of the first dose of GM-CSF
  • have had naxitamab in the past 
  • have a major problem with how an organ in your body works. For example your heart or liver. You can join if you have hearing loss, certain changes to blood counts Open a glossary item or acceptable changes to how your kidneys and liver work.

Medical conditions
You cannot join this trial if any of these apply. You:

  • have a serious problem with how your kidneys work 
  • have a very serious infection
  • have had recent treatment to damp down the immune system Open a glossary item. You can join if you had local steroid Open a glossary item treatment. This might have included creams or injections to an area where you had pain.
  • have a heart problem Open a glossary item that would affect you taking part in the trial. Your doctor checks how your heart is working before you can join the trial. 
  • have any other medical condition that would affect you taking part in the trial 

You cannot join this trial if any of these apply. You:

  • are allergic to any of the trial drugs or anything they contain 
  • have had a severe allergic reaction to a drug similar to naxitamab in the past 
  • are pregnant or breastfeeding

Trial design

This phase 2 trial is taking place worldwide. The team need to find up to 122 people including about 5 from the UK. 

You have treatment in cycles Open a glossary item. Each 4 week period is a cycle of treatment. In each cycle you have:

  • low dose GM-CSF injections for the first 5 days
  • high dose GM-CSF injections for the next 5 days. You also have naxitamab 3 times during this week. Each dose is 2 days apart. 

You then have no more treatment until the next cycle begins. 

You have naxitamab as a drip into a vein. It takes about 30 to 60 minutes each time. You will be at the hospital for longer than this. 

You have GM-CSF as an injection under the skin. You have the first dose at the hospital and the rest of the injections at home. Your nurse will show you how to do this. They will give you written instructions and the equipment you need. They’ll ask you to fill in a diary to record the date and time you have the injections. 

You have a scan and bone marrow test Open a glossary item at 6 weeks after starting treatment. For people whose neuroblastoma has gone away completely or a little bit you have 5 more cycles of treatment every 4 weeks. You then have treatment every 8 weeks. 

Everyone else continues to have treatment every 4 weeks. The trial team can let you know how long you have treatment for. This depends on your individual situation. 

Samples for research
The researchers ask you to give some extra blood samples. Where possible, you have these at the same time as your routine blood tests.

They plan to use the samples to:

  • see what happens to naxitamab and GM-CSF in the body 
  • look at genes Open a glossary item to understand more about how treatment works 

You need to give these samples to take part in the trial. 

Quality of life
The trial team ask you to fill out a questionnaire:

  • before you start treatment
  • at set times during treatment
  • after the end of treatment

The questionnaire asks about how you’ve been feeling. This is called a quality of life study.

Hospital visits

You see a doctor and have some tests before you can take part in this trial. These include:

You might also have a PET scan.

You have naxitamab and the first dose of GM-CSF at the hospital on the day care ward. 

You see the doctor for check ups and blood tests regularly during treatment.

Trial scans and bone marrow tests
You have a bone marrow test and trial scan after you started treatment. If the neuroblastoma has gone away completely or a little bit, you have these tests done again at 18 weeks after you started treatment. Everyone else has these tests done again at 14 weeks after starting treatment. 

You have more scans and bone marrow tests during the trial to see how well  treatment is working. The team can tell you more about when you have these. 

Follow up 
You see the trial team about 6 weeks after you finish treatment. After that you see them every 3 months for 3 years. You might see them at a routine hospital visit or they may call you to see how you are getting on.

Depending on how well treatment worked for you, you might have extra trial scans and bone marrow tests.

Side effects

The trial team monitor you during treatment and afterwards. Contact your advice line or tell your doctor or nurse if any side effects are bad or not getting better.

Naxitamab can affect the immune system Open a glossary item. This may cause inflammation Open a glossary item and other reactions in different parts of the body. For many people the inflammation and reactions are not too bad. For some people they can cause serious side effects. 

These side effects could happen during treatment or months after treatment has finished. Rarely, these side effects could be life threatening. Your doctor or nurse can explain what these side effects are, the risk of them happening and what to look out for.

If you have any of these side effects tell your doctor or nurse as soon as possible. You should tell them that you are on or have been on an immunotherapy

Naxitamab is a new drug. So there may be side effects we don’t know about yet. 

The most common side effects of naxitamab we know about so far include:

  • a reaction to the infusion. This can cause low blood pressure, hives or skin rash and itching, trouble breathing, cough or wheezing, fast heartbeat, high temperatures, swelling of the face, lips, mouth, tongue, throat or swelling of the body, feeling or being sick or high blood pressure. 
  • pain caused by the infusion. You have strong pain medication before naxitamab to help with this. You may need more pain medication or icepacks to painful areas. The worst pain happens during the infusion and in many cases, it will go away about 15 minutes after it finishes. 

The most common side effects of GM-CSF include:

The team will talk to you about all the possible side effects of treatment. You will have a chance to ask any questions you may have.



Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Juliet Gray

Supported by

Y-mAbs Therapeutics

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:


Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Around 1 in 5 people take part in clinical trials

3 phases of trials

Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.

Last reviewed:

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