A trial of treatment for children and adults with rhabdomyosarcoma (FAR-RMS)

Cancer type:

Children's cancers
Sarcoma
Secondary cancers
Soft tissue sarcoma

Status:

Open

Phase:

Other

This trial is looking at new ways of treating rhabdomyosarcoma in children and adults.

Rhabdomyosarcoma is a type of soft tissue sarcoma.

This trial is for children and adults. We use the term ‘you’, but of course if you are a parent, we are referring to your child.

Cancer Research UK supports this trial.

More about this trial

Rhabdomyosarcoma (RMS) mainly affects children but teenagers and adults can also get it. The main treatments include chemotherapy, surgery and radiotherapy. Often you have a combination of these.

Researchers are looking for ways to improve treatment for people with rhabdomyosarcoma. To do this they are looking at whether changes to chemotherapy and radiotherapy can improve treatment outcomes.

The trial is trying to answer a number of questions about the treatment of RMS. These include looking at:

  • new combinations of chemotherapy as initial treatment (induction)
  • the timing, dose and extent of radiotherapy
  • chemotherapy to keep the cancer under control after initial chemotherapy. This is called maintenance treatment.
  • a new combination of drugs for sarcoma that has come back (relapsed) 

The main aims of the trial are to improve treatment outcomes and quality of life.

Who can enter

Please note, there are several treatment groups so the entry conditions for this trial are complex. Each group has specific entry conditions and we haven’t listed them all. 

The trial team check if you are suitable to join the trial and which group you might join. They can tell you more about this. 

The group you join depends on a number of risk factors such as: 

  • whether your sarcoma is newly diagnosed, has come back or got worse
  • where your sarcoma is in the body 
  • if it has spread to lymph nodes Open a glossary item or elsewhere in the body
  • if you have any genetic changes (mutations Open a glossary item) in the sarcoma cells
  • your age

The following bullet points summarise some of the main entry conditions. 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 the trial if you:

  • have rhabdomyosarcoma that is newly diagnosed, has come back or has got worse
  • are well enough to have treatment 
  • have satisfactory blood test results
  • have a sample of tissue available for the trial team to do some tests on 
  • are willing to use reliable contraception if you are at an age where you are sexually active. You must be willing to use this contraception during treatment and for a period of time after if you or your partner could become pregnant.

Who can’t take part

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

  • have had a stem cell transplant Open a glossary item in the past 
  • have an infection that needs treatment 
  • are pregnant or breastfeeding
  • have a medical condition or mental health problem that the team think might affect you taking part in the trial 

As well as the above there are specific entry conditions and exclusion criteria for each treatment group. Speak to your doctor or research nurse if you want to find out more about the entry conditions for this trial.

Trial design

The trial team hope to find 1,250 children and adults to join this trial. 

The team are looking at:

  • initial chemotherapy to get rid of sarcoma (induction treatment)
  • radiotherapy 
  • chemotherapy to get rid of any traces of cancer that are leftover from previous treatment or to keep the sarcoma under control for longer (maintenance treatment)
  • adding regorafenib to chemotherapy for sarcoma that has come back

Your doctor will tell you about the treatment group you might be suitable to join. And how this treatment may differ from standard treatment Open a glossary item. You may be able to join more than 1 part of the trial. 
         
The team tell you when you have treatment and for how long.

We have included a brief summary of each treatment group.

Dose finding group (for newly diagnosed RMS)
This part is for people whose cancer has spread to the lymph nodes or elsewhere in the body. This part of the trial is open to recruitment. 

Researchers think adding a drug called irinotecan to standard chemotherapy might improve treatment. A standard combination of chemotherapy includes the drugs ifosfamide, vincristine and actinomycin D (IVA).

Irinotecan is a chemotherapy drug which is already used to treat many different types of cancer. In this group you have irinotecan alongside IVA chemotherapy. 

The first few people have a low dose of irinotecan. If they don’t have any side effects the next few people have a higher dose and so on until they find the best dose. This is a dose finding study. 

When they find the best dose then the initial chemotherapy groups can open. These are groups CT1a and CT1b. 

Initial chemotherapy – very high risk group (CT1a)
This part of the trial opens when they find the best dose of irinotecan in the dose finding study. This part of the trial isn’t open yet. 

The team are looking at adding irinotecan to standard chemotherapy. A usual treatment for this group is a combination of chemotherapy called IVADo.

You are put into a treatment group at random. You have 1 of the following:

  • ifosfamide, vincristine, actinomycin D and doxorubicin (IVADo) – standard treatment 
  • irinotecan, ifosfamide, vincristine and actinomycin D (IRIVA) – new combination of treatment

The aim is to see which treatment works best. You have 9 cycles of chemotherapy Open a glossary item.

Initial chemotherapy – high risk group (CT1b)
This part of the trial opens when they find the best dose of irinotecan in the dose finding study. This part of the trial isn’t open yet. 

The team are looking at adding irinotecan to standard chemotherapy. A standard treatment for this group is a combination of chemotherapy called IVA. 

You are put into a treatment group at random Open a glossary item. You have 1 of the following:

  • ifosfamide, vincristine, actinomycin D (IVA)
  • irinotecan, ifosfamide, vincristine and actinomycin D (IRIVA)

The aim is to see which treatment works best. You have 9 cycles of  chemotherapy. 

Maintenance chemotherapy – very high risk group (CT2a)
This part of the trial is for people who are having maintenance chemotherapy that includes the drugs vinorelbine and cyclophosphamide. This part of the trial is open to recruitment. 

The team are looking at whether giving maintenance therapy for longer works better. 

Everyone has 12 cycles of chemotherapy as usual. This is standard for this group. You have the drugs vinorelbine and cyclophosphamide (VC). Towards the end of this treatment you are put into a treatment group at random. 

You:

  • either stop maintenance chemotherapy or
  • have 12 extra cycles of chemotherapy Open a glossary item

Maintenance chemotherapy – high risk group (CT2b)
This part of the trial is for people who are having maintenance chemotherapy that includes the drugs vinorelbine and cyclophosphamide. This part of the trial is open to recruitment.

The team are looking at whether giving maintenance therapy for longer works better. 
Everyone has 6 cycles of chemotherapy as usual. This is standard for this group. Towards the end of this treatment you are put into a treatment group at random. You:

  • either stop maintenance chemotherapy or
  • have 6 extra cycles


    

Chemotherapy for sarcoma that has come back (CT3)
This part of the trial is for people whose sarcoma has come back (relapsed) after treatment. And they are now due to have more chemotherapy.

This part of the trial is open to recruitment.

The standard chemotherapy for relapsed sarcoma is a combination of three chemotherapy drugs. They are called vincristine, irinotecan and temozolomide (VIT). The trial team want to see if a newer combination of drugs works better. They think that replacing temozolomide with a drug called regorafenib will improve treatment. 

You are put into a treatment group at random. You have one of the following:

  • vincristine, irinotecan and temozolomide (standard treatment)
  • vincristine, irinotecan and regorafenib

You have up to 12 cycles of treatment.

Radiotherapy for sarcoma that can be removed by surgery (Groups RT1a and RT1b)
This part of the trial is for people who have already had chemotherapy as part of this trial or as part of their standard care.

This part of the trial is open to recruitment.

It is usual to have radiotherapy after surgery for sarcoma. The trial team want to see if radiotherapy before surgery works better. And if a higher dose works better than the standard dose. 

You are put into a treatment group at random. You have 1 of the following:

  • radiotherapy before surgery
  • radiotherapy after surgery


Some people have a higher risk of their sarcoma coming back. If this risk applies to you, you also enter a second randomisation. This determines the dose of chemotherapy you have. You have 1 of the following: 

  • standard dose radiotherapy
  • high dose radiotherapy 

Radiotherapy for sarcoma that can’t be removed by surgery (Group RT1c)
This part of the trial is for people who have already had chemotherapy as part of this trial or as part of their standard care. And whose cancer hasn’t spread elsewhere in the body.

This part of the trial is open to recruitment. 

You are put into a treatment group at random. You have 1 of the following:

  • standard dose radiotherapy
  • high dose radiotherapy

 
Radiotherapy for sarcoma that has spread elsewhere in the body (Group RT2)
This part of the trial is for people who have already had chemotherapy as part of this trial or as part of their standard care.

This part of the trial is open to recruitment. 

Cancer that has spread elsewhere in the body is called metastatic cancer. This part of the trial is looking at which sites in the body to give radiotherapy to.
You are put into a group at random. You have 1 of the following:

  • radiotherapy to where the sarcoma started and lymph nodes with cancer cells in them
  • radiotherapy to where the sarcoma started, lymph nodes with cancer cells and other areas of cancer spread in the body

The researchers want to find out which method of giving radiotherapy works best. 

Quality of life
For some groups the trial team ask you to fill out a questionnaire before you start treatment and at set times during treatment. The questionnaire asks about side effects and how you’ve been feeling. This is called a quality of life study.

Hospital visits

You see a doctor and have some tests before taking part in the trial. These include:

You might need a lumbar puncture if the sarcoma is around your ears, nose or face.

Chemotherapy and how you have it 
How you have chemotherapy depends on the group you join. You have some chemotherapy drugs as a drip into a vein. You might need a central line. This is a long plastic tube that gives the drugs into a large vein, either in your chest or through a vein in your arm. It stays in while you’re having treatment, which may be for a few months.

You may have some chemotherapy drugs as a tablet you take by mouth. 

The team tell you when you have treatment and for how long. You might need to stay overnight for some hospital visits. 

Radiotherapy and how you have it 
Before treatment starts, you have a planning scan. Your radiotherapy team carefully work out how much radiation you need to treat the cancer and exactly where you need it.

You have radiotherapy Monday to Friday. It takes about 10 to 20 minutes each time. Depending on which group you join treatment takes between 4 to 7 weeks. The trial team will tell you how long your treatment takes. 

Targeted treatment and how you have it 

Regorafenib is a type of targeted drug Open a glossary item called a cancer growth blocker. It stops signals that the cancer cells use to divide and grow. It also stops them from forming new blood vessels that they need to divide and grow.  

You have regorafenib is a tablet or granules. Your doctor can tell you how you will have treatment. You have it once a day, everyday. 

Follow up
When you finish treatment you have regular check ups with your doctor. They repeat some of the tests you had when you joined the trial. How often you have check ups depends on the routine follow up care at your hospital. The team will tell you more about this.  

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. 

The most common side effects of chemotherapy include:

The most common side effects of radiotherapy include: 

The symptoms are usually worse at the end of treatment and start to improve a few weeks after treatment has finished. 

Some possible side effects may develop many years after radiotherapy. These include:

  • developing another cancer
  • problems with puberty and fertility
  • other problems in the body for example it may affect the muscles, bones, the heart and lungs 

The possible side effects of regorafenib include:

  • soreness, redness and peeling on palms or soles of feet  
  • skin rash
  • bleeding 
  • problems with wound healing 
  • low levels of the thyroid Open a glossary item hormone. This might cause muscle cramps and  tiredness. 
  • diarrhoea
  • loss of appetite and taste changes
  • voice changes such as hoarseness
  • kidney problems
  • mouth sores and ulcers 

Your doctor will talk to you about all the possible side effects of treatment. 

We have more information about:

Location

Aberdeen
Birmingham
Bristol
Cambridge
Cardiff
Edinburgh
Glasgow
Leeds
Leicester
Liverpool
London
Manchester
Newcastle upon Tyne
Nottingham
Sheffield
Southampton
Sutton

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 Meriel Jenney

Supported by

Cancer Research UK
University of Birmingham

Other information

This is Cancer Research UK trial number CRUK/17/011.

Freephone 0808 800 4040

Last review date

CRUK internal database number:

14954

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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