“I think it’s really important that people keep signing up to these type of trials to push research forward.”
A study looking at treating children who have high risk acute lymphoblastic leukaemia with modified immune cells (CD19 TPALL)
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
This study is using gene therapy to treat children with acute lymphoblastic leukaemia (ALL).
The study is for children and young people up to and including the age of 18. We use the term ‘you’ in this summary, but of course if you are a parent, we are referring to your child.
Doctors can treat acute lymphoblastic leukaemia (ALL) with a stem cell transplant using cells taken from someone else (a donor).
After a stem cell transplant, you may have some more immune system cells called
In this study, the researchers will carefully pick out the lymphocytes and change some of them in the laboratory. They want to make them better at attacking leukaemia cells, but less likely to cause GVHD. This is a type of gene therapy and researchers hope it will reduce the risk of ALL coming back after a stem cell transplant.
The children and young people taking part in this trial have a type of ALL that affects the B cells. They will either be having their first stem cell transplant or they have had a stem cell transplant but their leukaemia has come back and they are going to have another one. Whichever situation they are in, their doctors think there is a high risk of the leukaemia coming back after the transplant.
The main aims of the study are to learn more about this type of treatment, how well it works and how safe it is.
Who can enter
You may be able to enter this study if
- You are under 19 and have a type of acute lymphoblastic leukaemia called precursor B cell ALL
- You are going to have a stem cell transplant using cells from a donor, but your doctors think there is a high risk of your leukaemia coming back afterwards
- You are well enough to take part – if you are over 10 years old this means that you can mostly care for yourself (Karnofsky score of more than 60, for children under 10 it means that you are up and around even if not playing as energetically as usual (Lansky score of more than 60)
You cannot enter this study if you
- Have graft versus host disease that needs steroid treatment
- Have problems with your lungs, liver or kidneys
- Have a serious infection
- Are HIV, hepatitis B or hepatitis C positive
- Are pregnant
There may be other reasons why this trial would not be suitable for you – the study team can discuss these with you.
This study will treat 30 children in Europe.
Your doctors will give you a lot more information about having a stem cell transplant. Taking part in the trial will not change your treatment very much, but you may have some extra blood cells (
A few days before you have the lymphocytes, your doctor will give you low dose
When you have the lymphocytes depends on whether you are having your first or second transplant.
If this is your 2nd transplant, you will have the lymphocytes about 2 months after your transplant.
If this is your 1st transplant, you only have the lymphocytes if leukaemia cells come back in your
Results from the first 5 children who took part in the study have shown that the lymphocytes don’t last in the blood. So all children joining the trial now have extra vaccines containing cells that are used to make the lymphocytes. Vaccination with these cells may help the treated lymphocytes to grow and stay in your blood. You have the extra vaccines as injections under your skin 2 days before you have the lymphocytes and then 4 and 8 weeks afterwards.
You will spend some time in hospital to have your stem cell transplant. Your doctors will tell you more about what to expect and we have more information about having a transplant in our cancer treatments section.
When you have the lymphocytes, you need to go back into hospital for a couple of days. You have a small amount of lymphocytes one day and the rest of them the following day. This normally takes about 15 minutes each time.
After you have the lymphocytes, you need to go to hospital regularly during the 1st year and then every 6 months for up to 10 years. But you have a lot of hospital visits, blood tests and bone marrow tests after a stem cell transplant, even if you don’t take part in this study.
The changed lymphocytes may cause allergic reactions such as a high temperature or a rash. But the study team will give you other medication to try to stop this happening.
If the lymphocytes work, they may also target and kill some normal blood cells that help you fight infection. If this happens, you may have drugs called immunoglobulins to help fight infections.
The risk of graft versus host disease is low, but can happen after a stem cell transplant or after having the lymphocytes. If you do get GVHD, you will have treatment for it.
The doctors will talk to you about all the possible side effects of treatment before you agree to join the study..
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.
Professor Persis Amrolia
Children with Leukaemia
Department of Health
Experimental Cancer Medicine Centre (ECMC)
Great Ormond Street Hospital for Children NHS Foundation Trust
Moulton Charitable Foundation
University College London (UCL)