A trial of LEE011 for children and young people with neuroblastoma or a malignant rhabdoid tumour

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 drug called LEE011 as treatment for neuroblastoma and a very rare type of childhood cancer called a malignant rhabdoid tumour or MRT.

This trial is for children and young adults up to the age of 21. We use the term ‘you’, but if you are a parent, we are referring to your child.

LEE011 is a type of biological therapy.  It is a cancer growth blocker. It stops signals that cancer cells use to divide and grow. LEE011 may help people with neuroblastoma or MRT.

The children and young people taking part in this trial have already had treatment for neuroblastoma or MRT Open a glossary item. But their cancer has not responded to treatment, or has come back and their doctors don’t think other treatments are likely to help them.

The aim of the trial is to find the highest safe dose of LEE011 that children and young people can have.

Who can enter

You may be able to enter this trial if you

  • Are at least 12 months old, but no older than 21years
  • Have neuroblastoma or a malignant rhabdoid tumour (MRT) Open a glossary item that has got worse despite having treatment and there is no other treatment that is likely to help you
  • Have satisfactory blood test results
  • Have cancer that can be measured
  • Have recovered from the side effects of earlier cancer treatment
  • Are well enough to take part – if you are over 16 years old this means that you need help to care for yourself, but not all the time (Karnofsky score of more than 50); for children under 16 it means you get dressed and even if you don’t actively play, you take part in quiet play and activities (Lansky score of more than 50)
  • Are willing to use reliable contraception during the trial if you are sexually active and there is any chance you could become pregnant

If you have cancer affecting your brain or spinal cord (your central nervous system) you must have been on a stable dose of steroids for at least a week, with no plans to increase your dose.

You cannot enter this trial if you

  • Have neuroblastoma that is only in your bone marrow Open a glossary item
  • Have any other cancer apart from neuroblastoma or a malignant rhabdoid tumour
  • Have had other anti cancer drugs or experimental drugs in the last 3 weeks (in the last 6 weeks if you had a drug called bevacizumab or one of a group of drugs called nitrosoureas Open a glossary item)
  • Have already had a drug that works in a similar way to LEE011 – the trial team can advise you about this
  • Have had radiotherapy or major surgery in the last 2 weeks
  • Have any problem with your digestive system Open a glossary item that could affect how you absorb the trial drug
  • Have had a stem cell transplant using your own cells in the last 3 months, or have ever had a stem cell transplant using cells from a donor
  • Have certain heart problems or take other medication that can affect your heart – the trial team can advise you about this
  • Take any other medication that affects body proteins called cytochrome P enzymes – your doctor can advise you about this
  • Have any other medical condition or mental illness that the trial team think could affect the results of this trial
  • Are pregnant or breastfeeding

Trial design

This phase 1 trial will recruit about 64 children and young people. The trial is in 2 parts. Everybody joining the trial has LEE011.

In the 1st part of the trial, the researchers are trying to find the best dose of LEE011. The first few patients taking part have a low dose of LEE011. If they don’t have any serious side effects, the next few patients will have a higher dose. And so on, until they find the highest dose you can have without causing bad side effects.

In the 2nd part of the trial, the researchers want to learn more about what happens to LEE011 in your body. Everybody joining this part of the trial has the highest safe dose that was found in part 1.

LEE011 is a capsule that you swallow. If you have difficulty swallowing, you can sprinkle the contents of the capsule on food. Or you can mix them with water and have this via a feeding tube.

You have LEE 011 once a day for 3 weeks out of every 4. As long as you don’t have bad side effects, you can carry on taking LEE011in this way for as long as it helps you.

Hospital visits

You see the trial team and have some tests before you start LEE011. The tests include

  • Physical examination
  • Blood tests
  • Heart trace (ECG Open a glossary item)
  • Heart scan (echocardiogram Open a glossary item)
  • X-rays
  • CT scan or MRI scan

If you have neuroblastoma, you may also have

If you have a malignant rhabdoid tumour, you may also have a lumbar puncture.

The trial team will get a sample of your cancer that was removed when you had surgery or a biopsy Open a glossary item. They will use this to learn more about your cancer and to look for genes and proteins that may help researchers to understand more about LEE011 and why it helps some people more than others.

You go to see the trial team once a week for the first 8 weeks of treatment and then once every 2 weeks after that. You have regular blood tests and scans.

Side effects

As LEE011 is a new drug and hasn’t been given to children before, there may side effects we don’t know about yet. The possible side effects include

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 Andrew Pearson

Supported by

NIHR Clinical Research Network: Cancer

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 11138

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

Cara took part in a clinical trial

A picture of Cara

"I am glad that taking part in a trial might help others on their own cancer journey.”

Last reviewed:

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