A trial of anti-GD2 T-Cells for neuroblastoma that is not responding to treatment or has come back

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Children's cancers




Phase 1

This trial is looking at a new treatment using anti-GD2 T-cells. The researchers are taking immune cells from the blood and changing them so they can attack the cancer cells. Cancer Research UK is supporting this trial. 

This 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

Doctors can use different treatments for neuroblastoma including surgery, chemotherapy and radiotherapy.

But sometimes neuroblastoma gets worse despite treatment. And even if it responds well to treatment to begin with, it might come back later on. In this situation, doctors are looking at new ways to treat this cancer.

Anti-GD2 T-cells are made by taking some immune cells (T-cells) from the blood and changing them in the laboratory. They change them by putting a gene into them. You might hear this called CAR T-cell therapy, it is a type of adoptive cell transfer.

T-cells are part of the immune system and help fight infections. Doctors hope the new gene will help the T-cells to recognise and attack the cancer cells.

Results from laboratory studies of anti-GD2 T-cells look promising. This is the first time people have been given these particular cells.

There will be up to 5 treatment groups. Four of the groups will have chemotherapy before they have the cells this is to see if it helps the anti-GD2 T-cells work better.

You usually have chemotherapy to destroy cancer cells. But in this trial the doctors hope that it will reduce the number of other immune cells in the body and so make space for the anti-GD2 T-cells.

The aims of the trial are to find out:

  • if they can make anti-GD2 T-cells in the laboratory and if it is safe to give to people
  • about any side effects and the best way to treat them
  • whether giving chemotherapy first, improves how long the anti-GD2 T-cells survive and helps to increase their number
  • how well and for how long the anti-GD2 T-cells survive inside the body
  • whether anti-GD2 T-cells can shrink the neuroblastoma

Who can enter

The following bullet points list 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.

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

  • have neuroblastoma that has come back or neuroblastoma that isn’t responding to treatment
  • have neuroblastoma which can be assessed using a scan or a bone marrow test
  • are at least 1 year old. Babies between 6 to 12 months may be able to take part in a later stage of the trial
  • are well enough to take part. This means a Karnofsky or Lansky score of at least 60
  • have satisfactory blood test results
  • are willing to use reliable contraception during the trial and for 6 months afterwards if you are sexually active and there is any chance you or your partner could become pregnant. If you are female and have the drug cyclophosphamide or rituximab as part of the trial you must use effective contraception for 12 months after having these drugs

You cannot join this trial if you:

  • have neuroblastoma that has spread to your brain and spinal cord (central nervous system). If you have had neuroblastoma in your central nervous system but have had it removed by surgery and there have been no signs of it coming back in the last 2 months you might be able to take part
  • have neuroblastoma that has spread to your airways or is pressing on your airways
  • have had anti-GD2 antibody treatment in the last 2 weeks. If you have had dinutuximab or other anti-GD2 directed treatment this may be longer, your doctor can advise you
  • are having experimental treatment as part of another clinical trial
  • have had major surgery and not yet recovered
  • have any side effects from previous treatment that are causing you problems
  • are allergic or sensitive to the drug rituximab or there is any other reason why you can’t have this drug
  • have an autoimmune disease and need to have treatment for this that affects your whole body (systemic treatment)
  • are taking or will need to take high dose steroids or any other treatment that can affect how your immune system works
  • have any disease that affects your brain and nervous system (neurological disease) that you have had for a while and is getting worse
  • have an active infection which is not controlled by treatment
  • are hepatitis B, hepatitis C or HIV positive
  • have any other condition that the trial team think could affect you taking part

Trial design

This is a phase 1 trial. The doctors need between 15 to 27 children and young adults to take part.

There are 5 treatment groups. 4 of the treatment groups have now completed. The treatment groups are:

  • group1: anti-GD2 T-cells with no chemotherapy (this group is now complete)
  • group 2: 1 chemotherapy drug (cyclophosphamide) and anti-GD2 T-cells (this group is now complete)
  • group 3: 2 chemotherapy drugs (cyclophosphamide and fludarabine) and anti-GD2 T-cells (this group is now complete)
  • group 4: cyclophosphamide and fludarabine and a higher dose of anti-GD2 T-cells than used in groups 1 to 3 (This group is now complete)
  • group 5: cyclophosphamide and fludarabine and a higher dose of anti-GD2 T-cells than used in groups 1 to 4. This group might not be needed if the anti-GD2 T cells work well in the other groups.

In order to make the anti-GD2 T-cells the doctors need to collect the T-cells from your blood. There are 2 ways they can do this. They can either take a regular blood sample (venepuncture) or they can use a process called leukapheresis (pronounced loo-kah-fur-ee-sis). 

To have leukapheresis you have a catheter (a plastic tube) put into a large vein (usually at the top of your leg). You might have this done under general anaesthetic or local anaesthetic.

You are then connected to a machine by two tubes. One tube removes blood. The blood then passes into the machine and it removes the white blood cells. The rest of your blood cells and normal blood fluid (plasma) then go back into your body through the other tube.

Once the researchers have the T-cells they change them in the lab. This process takes about 2 weeks. They freeze and store them until you need them. You only have anti-GD2 T-cells made from your own T-cells.

Anti-GD2 T-cells with no chemotherapy

You have:

  • fluid in a drip (IV fluids) for 6 hours
  • anti-GD2 T-cells as an injection into the bloodstream (intravenously) over about 5 to 30 minutes
  • fluids as a drip for 24 hours

You also have an anti allergy drug (an antihistamine).

Cyclophosphamide and anti-GD2 T-cells

Once a day for 4 days before you have the anti-GD2 T-cells you have:

  • cyclophosphamide into the bloodstream
  • a  drug called mesna, you have this in a drip into the bloodstream to stop the cyclophosphamide irritating your bladder and causing bleeding
  • extra fluids as a drip (IV fluids) before and after each dose of cyclophosphamide

Then you have anti-GD2 T-cells as described above.

Fludarabine, cyclophosphamide and anti-GD2 T-cells

8 days before you have the anti-GD2 T-cells you have:

  • 1 dose of fludarabine into the bloodstream

Your doctors call this day -8.


  • 4 days of fludarabine followed by cyclophosphamide, mesna and IV fluids (days -7 to -4)

After this you have a break from treatment for 3 days (days -3 to -1). You then have the anti-GD2 T-cells as described above (this is day 0).

In group 5 you might have the anti-GD2 T-cells in 2 seperate doses. The first dose on day 0 and the second dose the next day (day 1). 

You will probably only have 1 dose of anti-GD2 T-cells but you may have another dose at a later time. This depends on a number of things including:

  • how well the anti-GD2 T-cells survived in your body
  • if it got rid of any of your neuroblastoma
  • any side effects you had

Hospital visits

You see the doctors and have some tests before you start treatment. The tests include:

If your neuroblastoma is not suitable for an MIBG SPECT-CT scan you might have a FDG-PET-CT scan or a bone scan instead.

You will be asked if the doctors can take a sample of your neuroblastoma (a tumour biopsy). You do not have to give this sample if you don’t want to. You can still take part in the trial.

You go to hospital for your treatment and then stay for up to 14 days after you have had the anti-GD2 T-cells. You may need to stay in hospital for longer if you have any side effects from the anti-GD2 T-cells.

During the time you are in hospital you have regular blood tests. These are to check your general health and to learn more about how the anti-GD2 T-cells work in the body.

When you leave hospital the doctors continue to see you regularly. You see them for blood tests 18 days after you had the anti-GD2 T-cells.

Then you see them:

  • weekly until 6 weeks after having the anti-GD2 T-cells
  • monthly from 2 months to 6 months after treatment
  • then at 9 months and 1 year  after treatment
  • then once a year until the study finishes

At these visits you might have a physical examination and blood tests. At some appointments you might also have a scan and a bone marrow test.

Your doctor might ask you to give a sample of your neuroblastoma (a tumour biopsy) a month after you have treatment. This is to help the doctors learn more about neuroblastoma and the anti-GD2 T-cell treatment. You do not have to give this sample if you don’t want to. You can still take part in the trial.  

Side effects

As this is the first time people have had these particular anti-GD2 T-cells there may be some unknown side effects.

Your doctors closely monitor you during the time you are in hospital. And they will give you a number to call the hospital straightaway if you are worried about anything once you have gone home.

The possible side effects of anti-GD2 T-cells include:

  • an allergic reaction or an allergic type reaction including high temperature (fever), chills, low blood pressure, a fast pulse, headache, rash, shortness of breath and problems breathing. This reaction could happen immediately or come on over several days.
  • pain, inflammation and tenderness in the places where you have neuroblastoma cells.
  • pain (in other places than where the neuroblastoma cells are).
  • skin sensitivity when exposed to the sun
  • changes to the way your brain and nervous system work. This could cause confusion, drowsiness, being less alert, disorientation, changes in mood and behaviour, fits, and eye problems.

If you have side effects from the anti-GD2 T-cells that don’t get better after treatment with other drugs the doctors might decide to give you a drug called rituximab. Rituximab is a type of drug called a monoclonal antibody.

Anti-GD2 T-cells have a ‘suicide switch’. This means rituximab can recognise the anti-GD2 T-cells and kill them.

The doctors will explain more about this drug and its side effects if you need to have it.

We have information about the side effects of

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

Professor John Anderson

Supported by

Cancer Research UK (Centre for Drug Development)
NIHR Clinical Research Network: Cancer

Other information

This is Cancer Research UK trial number CRUKD/15/001.

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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