A study looking at venetoclax for children and young adults with cancer

Cancer type:

Children's cancers

Status:

Open

Phase:

Phase 1

This study is for children and young adults whose cancer has come back after treatment (relapsed) or continued to get worse during or after treatment (refractory).

This trial is for children and young people up to and including the age of 25. We use the term 'you' in this summary, but if you are a parent, we are referring to your child.

More about this trial

Venetoclax is a targeted drug Open a glossary item. It works by blocking a certain protein that cancer cells need to stay alive. 

Doctors in the UK use venetoclax to treat adults with chronic lymphocytic leukaemia (CLL)

Researchers want to find out if it can help children and young adults with cancer when their cancer comes back or has continued to get worse during treatment. 

The aims of the study are to find out:

  • how well venetoclax works on its own and in combination with chemotherapy
  • what the side effects are of having venetoclax is on its own or in combination with chemotherapy
  • what happens to venetoclax in the body 

Who can enter

The following bullet points list the entry conditions for this study. Talk to your doctor or the study 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 this study if all of the following apply. You:

  • have cancer that has come back after treatment (relapsed) or continued to get worse during treatment (refractory) 
  • have satisfactory blood test results 
  • are under 16 years old and might not play as actively as before your cancer but take part in quiet play and activities (Lansky performance score 50 and above) or are over 16 years old and you might need quite a lot of help to care for yourself (Karnofsky performance score 50 and above)
  • are willing to use contraception if you are a woman who is sexually active while having venetoclax and for 30 days after your last venetoclax dose if there is any chance you could become pregnant. If you have chemotherapy with venetoclax your doctor will tell you how long after your last treatment you need to continue using contraception.
  • are willing to use contraception if you are a man who is sexually active while having venetoclax and for 90 days after the last dose if there is any chance your partner could become pregnant. If you have chemotherapy with venetoclax your doctor will tell you how long after your last treatment you need to continue using contraception.  You are also willing not to store your sperm while having treatment.
  • are younger than 25 years old

Please note depending on what type of cancer you have there are other entry conditions that apply for you to join. Your doctor will talk to you about these. 

Who can’t take part

You cannot join this study if any of these apply. 

Cancer related
You:

  • have cancer that started in the brain (primary brain tumour)
  • have cancer that has spread to the brain
  • need to have radiotherapy to your head due to disease of the central nervous system Open a glossary item (CNS)
  • have had a monoclonal antibody Open a glossary item (MAB) including inotuzumab ozogamicin within 30 days of starting treatment
  • have had an immunotherapy treatment called adoptive cell therapy Open a glossary item for example CAR-T cell therapy within 30 days of starting study treatment
  • have any other cancer treatment including blinatumomab within 2 weeks of starting study treatment unless you have acute lymphoblastic leukaemia (ALL) with the Philadelphia chromosome Open a glossary item change and you were taking a tyrosine kinase inhibitor Open a glossary item (TKI) when asked to join the study
  • are taking steroids to treat your cancer unless you have stopped taking them at least 5 days before starting study treatment 
  • are taking hydroxycarbamide (hydroxyurea) unless you can stop taking while in the study
  • had a bone marrow transplant Open a glossary item less than 100 days ago 
  • had a bone marrow transplant more than a 100 days ago and have active graft versus host disease Open a glossary item (GvHD) or you are still taking medication after the transplant to damp down your immune system Open a glossary item within 7days of starting study treatment
  • have neuroblastoma and had a type of radiation therapy called MIBG less than 6 weeks ago

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

  • have hepatitis
  • have an active infection. If you had a COVID-19 test or have been in contact with someone who had COVID-19 or someone who has symptoms of COVID-19 you must have at least 2 tests showing you don't have COVID-19 before joining the trial. 
  • have ongoing moderate to severe side effects from previous treatment
  • have problems with your digestive system Open a glossary item that affects how well you absorb medication into your body
  • had medication that affects the CYP enzymes within 7 days of starting study treatment

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

  • had fruits such as grapefruit, grapefruit juice, Seville oranges including marmalade and star fruit within 3 days of starting study treatment
  • are pregnant or breastfeeding

Trial design

This is an international phase 1 study. The team need 10 children and young people from the UK to join and they need 165 worldwide to join.  

Venetoclax is a tablet. Your doctor will tell you how many venetoclax tablets to take. 

There are 2 types of tablet:

  • tablets you swallow whole
  • dissolvable tablets

You have tablets you swallow whole if you can do so. You take them once a day with a glass of water. You take the tablets within 30 minutes of eating a meal. You cannot chew, crush or breakup the tablets. 

For those who cannot swallow whole tablets you have venetoclax tablets that you dissolve in water, grape juice or apple juice. You take these tablets once a day within 60 minutes of eating a meal. 

You might stay in hospital for 2 or 3 days when you start taking venetoclax. This is so your doctor can monitor you. During this time the doctor increases the dose of venetoclax to find the best dose for you. 

You might have chemotherapy with venetoclax. Depending on your cancer this might start any time after having 3 or 4 doses of venetoclax. You will stay in hospital for at least a day if you start chemotherapy. This is so the doctor can monitor you. 

You continue to have venetoclax as long as it is still working. 

Diary and questionnaire
You have a diary to write down:

  • when you take venetoclax
  • the time of the meals you take it with

For those who are having the tablets that dissolve you fill in a questionnaire during treatment at:

  • 4 weeks
  • 12 weeks
  • 36 weeks

The questions ask:

  • how easy it is to use
  • how long it takes to prepare
  • how easy it is to swallow 
  • did you use water, grape juice or apple juice to dissolve the tablets
  • how does it taste 

Samples for research
What tests you have will depend on your cancer type. The study team will take an additional sample if you have any of these tests:

  • lumbar puncture Open a glossary item
  • bone marrow test Open a glossary item
  • small piece of tissue (biopsy Open a glossary item) taken from your cancer  

The team use these samples:

  • to look for substances in them that might show how well treatment is working
  • to find out what happens to venetoclax in the body

Hospital visits

You see the doctor to have tests before taking part. These tests include:

  • a physical examination
  • blood tests

During treatment you see the doctor often for the first 3 months. You then see then see them every 3 months for the rest of the time you’re having treatment and at the end of treatment. 

After you have finished treatment the study doctor will phone you every 3 months for 5 years. This is to see how you are and if you are having any other treatment. You do not have to agree to this. 

Extra tests

Depending on your cancer type you might also have one or more of the following:

You have these:

  • before starting treatment
  • during treatment
  • and at the end of treatment

Your doctor will tell you which extra tests you have and when you have them. 

Side effects

The study 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 common side effects of venetoclax include:

Your doctor or a member of the study team will talk to you about the possible side effects of venetoclax before you agree to join the study.

Location

London
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 Quentin Campbell-Hewson

Supported by

AbbVie

Freephone 0808 800 4040

Last review date

CRUK internal database number:

17042

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

Rhys was only four years old when he was diagnosed with a brain tumour

A picture of Rhys

"He went through six operations and was placed on a clinical trial so he could try new treatments.”

Last reviewed:

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