"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
A trial of blinatumomab for children and young people with a certain type of acute lymphoblastic leukaemia
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
This trial is looking at a drug called blinatumomab (also known as MT103). It is for children and teenagers with a type of acute lymphoblastic leukaemia called precursor B cell ALL. Blinatumomab is pronounced blin-a-too-moo-mab.
The trial is for children and young people up to and including the age of 17. We use the term ‘you’ in this summary, but if you are a parent, we are referring to your child.
Doctors usually treat acute lymphoblastic leukaemia (ALL) with chemotherapy. You may also have a stem cell transplant. Unfortunately for some people, these treatments may not work and their leukaemia continues to grow or comes back after treatment. Researchers are looking at a drug called blinatumomab to see if it could help children and young people in this situation.
The researchers have already found the highest safe dose of blinatumomab that people under 18 can have. The aims of this trial are to
- See if blinatumomab helps children and teenagers who have precursor B cell ALL that has not responded to treatment or has come back after treatment
- Find out more about the side effects
Who can enter
You may be able to enter this trial if
- You have a type of acute lymphoblastic leukaemia called precursor B cell ALL
- Your leukaemia did not respond to treatment or it has come back after treatment
bone marrowcontains more than 25% immature cells (blasts)
- You haven’t reached your 18th birthday yet
- You 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 at least 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 at least 50)
- You have satisfactory blood test results
As well as the above, young people who are sexually active must be willing to use reliable contraception during the treatment phase of the trial and for 3 months afterwards if there is any chance they or their partner could become pregnant.
You cannot enter this trial if you
- Have acute graft versus host disease (GVHD) or chronic GVHD if it is very widespread (the trial doctor can advise you about this)
- Take medication that damps down your immune system to prevent or treat GVHD in the 2 weeks before starting blinatumomab
- Are male and have leukaemia cells in your testicles
- Have leukaemia cells in your brain or spinal cord (your central nervous system) unless this has already been successfully treated and hasn’t caused symptoms
- Have had an
autoimmune diseasethat could affect your central nervous system (your doctor can advise you about this)
- Have a stem cell transplant in the 3 months before starting blinatumomab
- Have chemotherapy in the 2 weeks before starting blinatumomab unless this is injected into the fluid in your spine (intrathecal chemotherapy), or you have only a low dose of certain chemotherapy drugs (the trial team can advise you about this)
- Haven’t recovered from the side effects of chemotherapy unless they are only mild
- Have radiotherapy in the 2 weeks before starting blinatumomab
- Have had other treatment that stimulates your immune system (
immunotherapy) in the last 6 weeks, including a monoclonal antibody
- Have had another experimental treatment in the last 4 weeks
- Have already had blinatumomab
- Are known to be very sensitive to drugs made in a similar way to blinatumomab or to anything it contains
- Have any other type of cancer
- Have any other serious medical condition that could affect your taking part
- Are known to have HIV, hepatitis B or hepatitis C
- Are pregnant or breastfeeding
You have blinatumomab for 4 weeks out of every 6. Each 6 week period is called a cycle of treatment. You have up to 5 cycles of treatment.
You have blinatumomab via a small pump which is connected to a bag containing the drug. The pump gives you a continuous dose of blinatumomab over 4 weeks. You go home with the pump attached. You can carry it in a backpack, shoulder bag or attached to your belt.
You can walk around as normal and do most of your usual activities while you are wearing the pump. But there are some things you can’t do, such as go swimming. The trial team will give you more information about things you should not do with the pump attached.
You see the trial team and have some tests before you start treatment. The tests include
You may also have an MRI scan of your head to see if the leukaemia is affecting your brain.
You go to hospital regularly during treatment. You will stay in hospital for the first week of treatment and for the first 2 days of the 2nd cycle of treatment.
In cycles 3 to 5, you may not have to stay in hospital overnight. But you will need to stay there for 8 hours each time you have the pump attached. And you go back for short visits on the next 2 days.
You then go to hospital at least once a week. The trial team will tell you about the tests you need to have at these visits. They include blood tests, bone marrow tests and lumbar punctures. But you won’t have every test at every visit.
The bag of blinatumomab needs to be changed every 4 days. It will be changed at your weekly hospital visits. The change 4 days later may be done at home by a specialist nurse.
When you finish treatment, the trial team will monitor how you are getting on for up to 2 years. This is called the follow up period. You see them about a month after finishing treatment, then every 3 months for the 1st year and every 6 months in the 2nd year. At each visit, you have a physical examination, blood tests and a bone marrow test.
If your leukaemia completely disappears during treatment, but then comes back during the follow up period, you may be able to have 3 more cycles of treatment. The trial doctor will talk to you about this in more detail if they think further treatment may help you.
As blinatumomab is a new drug, there may be side effects we don’t know about yet. In other trials, the most common side effect has been a drop in the number of white blood cells causing an increased risk of infection.
The trial team will talk to you about all the possible side effects before you agree to take part in the trial.
During treatment, you must tell the trial team straight away if you notice any problems or side effects.
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 Ajay Vora
National Institute for Health Research Cancer Research Network (NCRN)