A trial looking at temozolomide for rhabdomyosarcoma

Cancer type:

Children's cancers
Soft tissue sarcoma




Phase 2

This trial is looking at temozolomide for people with a type of soft tissue sarcoma called rhabdomyosarcoma. The trial is supported by Cancer Research UK.

This trial is for children over 6 months old as well as adults. We use the term ‘you’ in this summary, but of course if you are a parent, we are referring to your child.

Treatment for rhabdomyosarcoma is often successful, but sometimes the sarcoma does not respond to treatment or comes back afterwards.

Doctors can treat rhabdomyosarcoma that has not responded to treatment or has come back with the chemotherapy drugs vincristine and irinotecan. In this trial, they are looking at adding a drug called temozolomide.

Temozolomide is a chemotherapy tablet that doctors use to treat other types of cancer. They want to find out if it can help people with rhabdomyosarcoma.

The aims of the trial are to

  • See if adding temozolomide to irinotecan and vincristine helps people who have rhabdomyosarcoma that did not respond to treatment or has come back
  • Learn more about the side effects

Who can enter

You may be able to enter this trial if you

  • Have rhabdomyosarcoma that did not respond to treatment or has come back
  • Have sarcoma that can be measured on CT scan or MRI scan
  • Are aged between 6 months and 50 years old
  • Are well enough to carry out all your normal activities, apart from heavy physical work (performance status of 0 or 1) or have a Lansky play score of 70% - 100% if you are under 12
  • Have satisfactory blood test results
  • Are willing to use reliable contraception during treatment and for 6 months afterwards if you are sexually active and there is any chance that you or your partner could become pregnant

You cannot enter this trial if you

  • Have had radiotherapy in the last 3 weeks unless it was to control symptoms of the cancer (palliative radiotherapy)
  • Have had irinotecan or temozolomide in the past
  • Have had drugs called vincristine, vinorelbine, vinblastine or cyclophosphamide in the last 2 weeks, or any other chemotherapy that causes a drop in the number of blood cells in the last 3 weeks (6 weeks if you had a drug called a nitrosourea Open a glossary item)
  • Are allergic to the drugs in the trial
  • Have diarrhoea that is causing you a problem
  • Have an infection that cannot be treated
  • Have any other medical problems that the trial doctors think could affect you taking part
  • Are taking any medicine to prevent fits such as phenytoin, phenobarbital, or carbamazepine
  • Are taking certain other drugs such as rifampicin, voriconazole, itraconazole, ketoconazole or aprepitant – the trial doctors can advise you about this
  • Are pregnant or breastfeeding

Trial design

The trial will recruit 80 people in Europe. It is a randomised trial. 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.   

  • People in group A have vincristine and irinotecan
  • People in group B have vincristine, irinotecan and temozolomide

People in both groups have vincristine and irinotecan through a drip into a vein every 3 weeks. Each 3 week period is called a cycle of treatment. People in group B also take temozolomide tablets daily for 5 days every 3 weeks. Everybody taking part has up to 12 cycles of treatment.

If you have a central line (long line), you can have your treatment through this.

Hospital visits

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

While you are having treatment you see the doctor and have blood tests frequently. You have CT scans or MRI scans every 6 weeks.

When you finish treatment, you see the trial team, have a blood test and a CT or MRI scan. You may also have a PET scan and bone marrow test.

You then see the trial team and have a CT or MRI scan

  • Every 3 months for 2 years
  • Every 6 months for 3 years

Side effects

The most common side effects of vincristine are

The most common side effects of irinotecan are

The most common side effects of temozolomide are

There is more information about vincristine, irinotecan and temozolomide in our cancer drugs section.


Newcastle upon Tyne

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 Julia Chisholm

Supported by

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

Other information

This is Cancer Research UK trial number CRUK/11/019.

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 8276

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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