A trial looking at treatment for children and young people with neuroblastoma (HR-NBL-I/ESIOP NB 2002 06)

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

Cancer type:

Children's cancers
Neuroblastoma

Status:

Closed

Phase:

Phase 2/3

This trial is looking at treatment for children and young people with a rare type of cancer called neuroblastoma. This trial is supported by Cancer Research UK.

This trial is for children and young people up to and including the age of 20. We use the term 'you' in this summary, but of course if you are a parent, we are referring to your child.

More about this trial

Neuroblastoma develops in specialised nerve cells and most commonly starts in the nerve cells of the adrenal glands Open a glossary item. Children with neuroblastoma usually have a combination of treatments including surgerychemotherapy and radiotherapy.

They may also have a stem cell transplant.

This trial is looking at the treatment of children with ‘high risk’ neuroblastoma. This means that there is a high risk that your cancer will continue to grow or come back (recur) after treatment.

Who can enter

You can enter this trial if you

  • Have stage 4 neuroblastoma, or stage 2 or 3 neuroblastoma with MycN amplification Open a glossary item - your doctor would be able to tell you if this applies to you (children under 1 year old should be stage 2, 3 or 4 with MycN amplification)
  • Are aged between 0 and 20 years old (inclusive)
  • Have satisfactory blood tests
  • Are well enough to take part

You cannot enter this trial if you

  • Have had previous chemotherapy, except your first cycle of chemotherapy
  • Have stage 1 neuroblastoma, or stage 2 or 3 neuroblastoma without MycN amplification Open a glossary item

Trial design

This European trial aims to recruit more than 1,000 patients over 5 to 6 years. There are six different parts to the treatment in this trial.

Before your treatment begins, you have a central line or long line put in. This is also sometimes called a ‘wiggly line’. It makes it easier to give chemotherapy and take blood samples.

Part 1 - Induction chemotherapy

Everyone will have induction chemotherapy to shrink the primary cancer and try to get rid of cancer cells that have spread. When the trial started, everybody taking part had chemotherapy called COJEC. But the trial team are now comparing COJEC with another type of chemotherapy called modified N7 in children who have stage 4 neuroblastoma with MycN amplification Open a glossary item.

This part of the trial is randomised. The people taking part are put into treatment groups by a computer. Neither you nor your doctor will be able to decide which group you are in.

  • One group have COJEC chemotherapy
  • The other group have Modified N7 chemotherapy

Children and young people who have stage 2 or 3 neuroblastoma won’t be randomised. They will all have COJEC.

COJEC includes the drugs

  • Vincristine
  • Caroboplatin,
  • Etoposide,
  • Cisplatin
  • Cyclophosphamide

You have 2 or 3 of these drugs every 10 days. One 10 day course is called a cycle of chemotherapy. You will have 8 cycles of treatment.

Modified N7 includes the drugs

  • Cyclophosphamide
  • Doxorubicin
  • Vincristine
  • Cisplatin
  • Etoposide

Treatment lasts 3 or 4 days every 3 weeks. Every 3 weeks is called a cycle of treatment and you have 5 cycles.

You also have a growth factor Open a glossary item called G-CSF. You have this as an injection just under the skin (subcutaneously). You have these injections daily for several days between chemotherapy cycles to lower the risk of infection.

If your neuroblastoma responds well to treatment, your stem cells Open a glossary item will then be collected and stored. You have more G-CSF to help your bone marrow make extra stem cells in preparation for your stem cell collection.

Part 2 - Surgery

You will have an operation to remove as much of the neuroblastoma as possible.

Part 3 - High dose chemotherapy and stem cell transplant

When you have recovered from surgery, you have high dose chemotherapy Open a glossary item. Earlier in the trial people had either

  • Carboplatin, etoposide and melphalan  OR
  • Busulfan and melphalan

But now, everybody has busulfan and melphalan. You have the chemotherapy through a drip into your central line.

You then have your stem cells Open a glossary item through a drip into your central line.

Part 4 - Radiotherapy

After the stem cell transplant, you have radiotherapy to your operation site. You have this every day from Monday to Friday for about 3 weeks.

Part 5 - Differentiation treatment

After radiotherapy, you have a tablet called ‘13-cis retinoic acid’ or isotretinoin. This is a man made form of vitamin A. You have this tablet twice a day for 2 weeks, followed by 3 weeks with no tablets. You will have this 6 times.

Part 6 – Immunotherapy

At the same time as having differentiation treatment, you have immunotherapy. Everyone taking part is put into 1 of 2 treatment groups at random (randomisation).

  • One group have antibody treatment that targets neuroblastoma cells and triggers the immune system
  • The other group have antibody treatment and another type of immunotherapy called aldesleukin (IL2)

Each 5 week period is called a cycle of treatment. You have antibody treatment through a drip into your central line as a continuous infusion over 10 days. You have it during the 2nd and 3rd week of each 5 week cycle.

If you have aldesleukin, you have this as an injection under the skin. You have this every day for 5 days during the 1st week of every cycle. Then every other day on the days you have antibody treatment.

You have up to 5 cycles of immunotherapy. Your treatment in parts 5 and 6 will take just over 6 months.

In early 2014, the trial team stopped giving aldesleukin (IL2). So people who joined the trial during this time just had the antibody treatment. The trial team have now gone back to having the 2 treatment groups. 

Hospital visits

Your will see a doctor and have various tests before you start treatment. These tests include

  • Taking a sample (biopsy) of your cancer
  • Ultrasound and MRI or CT scan of the cancer
  • Bone marrow test
  • Blood tests
  • Tests to check your heart function
  • Hearing test (audio gram)
  • Urine tests

You may have some of these tests again during and after your treatment.

Your treatment for neuroblastoma is very intensive, whether you take part in this trial or not. You will be in hospital on and off for about a year.

Side effects

The combination of treatments for this type of cancer can cause severe side effects. If you take part, you will have a detailed information sheet explaining the side effects. Below is a brief outline.

The most common side effects of chemotherapy and a stem cell transplant are

The side effects of having an operation include the risk of infection and pain.

The most common side effects of radiotherapy are

The side effects of 13-cis retinoic acid (isotretinoin) are a sore mouth and lips and dry skin.

The side effects of the immunotherapy include

  • Tummy (abdominal) pain
  • Temporary changes to the eyes
  • Allergic reactions

We have more information about

And the side effects of  

The side effects you have will depend on the combination of drugs used. Your doctor will discuss this with you.

The side effects you have will depend on the combination of drugs used. Your doctor will discuss this with you.

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 Martin Elliot

Supported by

Cancer Research UK
Cancer Research UK Children's Cancer Trials Team University of Birmingham
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer

Other information

This is Cancer Research UK trial number CRUK/02/013.

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

239

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

Wendy took part in a new trial studying the possible side effect of hearing loss

A picture of Wendy

"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

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