"He went through six operations and was placed on a clinical trial so he could try new treatments.”
A study looking at venetoclax for children and young adults with cancer
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
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.
- 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(CNS)
- have had a
monoclonal antibody(MAB) including inotuzumab ozogamicin within 30 days of starting treatment
- have had an immunotherapy treatment called
adoptive cell therapyfor 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 chromosomechange and you were taking a tyrosine kinase inhibitor(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 transplantless than 100 days ago
- had a bone marrow transplant more than a 100 days ago and have active
graft versus host disease(GvHD) or you are still taking medication after the transplant to damp down your immune systemwithin 7days of starting study treatment
- have neuroblastoma and had a type of radiation therapy called MIBG less than 6 weeks ago
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 systemthat affects how well you absorb medication into your body
- had medication that affects the CYP enzymes within 7 days of starting study treatment
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
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 bone marrow test
- small piece of tissue (
biopsy) 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
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.
Depending on your cancer type you might also have one or more of the following:
- scan such as a CT scan or MRI scan
- MIGB scan
- PET-CT scan
- lumbar puncture
- bone marrow test
- a small sample of tissue taken from your cancer (biopsy)
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.
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:
- a drop in the white blood cells causing an increased risk of infection
- a drop in the red blood cells causing tiredness and weakness
- infections such as chest infections, pneumonia and urine infections
- a serious blood infection called sepsis
- diarrhoea or constipation
- feeling or being sick
- changes to the amount of substances such as salts, fat and proteinin the blood
- tumour lysis syndrome
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.
How to join a clinical trial
Dr Quentin Campbell-Hewson