A trial looking at a drug called ch14.18/CHO for neuroblastoma in children and young people (GD2)
This trial is looking at different ways of giving a drug called ch14.18/CHO as treatment for neuroblastoma. The trial is supported by Cancer Research UK.
This trial is for children and young people up to and including the age of 21. We use the term 'you' in this summary, but of course if you are a parent, we are referring to your child.
Neuroblastoma develops in specialised nerve cells and most commonly starts in the nerve cells of the adrenal glands. Doctors can use different treatments for neuroblastoma including surgery, chemotherapy, radiotherapy and a stem cell transplant.
But even if neuroblastoma has responded well to treatment, it still tends to come back later, and is difficult to treat successfully. Doctors are looking for new ways to treat neuroblastoma and stop it coming back.
In this trial, they are looking at a drug called ch14.18/CHO, which is a type of biological therapy called a monoclonal antibody. These can seek out cancer cells by looking for particular proteins on the cancer cells..
We know from other trials that this antibody can help people with neuroblastoma, but without a strong painkiller such as morphine, it causes severe pain. Children and young people having this treatment have had to stay in hospital and be given strong painkillers. But we also know from research that giving each dose of ch14.18/CHO more slowly, over a longer period of time, reduces the pain it causes.
In this trial, researchers are looking at different ways of giving the drug over a number of days, to see if this means people could have the treatment more comfortably and not need to be in hospital all the time having strong painkillers. You will need to take painkillers by mouth or use skin patches.
Who can enter
You may be able to enter this trial if
- You have neuroblastoma and have already had at least 1 high dose treatment followed by a stem cell transplant
- Your neuroblastoma responded slowly to the first line of treatment, or your disease came back after treatment but has responded again to further treatment
- You are at least 1 year old but no more than 21 years old
- You are well enough to take part – if you are over 16 years old this means that you are able to care for yourself but may not be able to carry on with all your normal activities or do active work (Karnofsky score of more than 70), for children under 16 it means that you are up and around but may not play as energetically as usual (Lansky score of more than 70)
- You finished any other treatment at least 2 weeks ago and have recovered from any side effects
- You have satisfactory blood test results
- You are willing to use reliable contraception if you are sexually active and there is any chance you or your partner could become pregnant
You cannot enter this trial if you
- Have neuroblastoma that your doctors can see is getting worse at this time
- Have cancer that has spread to your brain or spinal cord (central nervous system) unless this has been successfully treated, scans show that it has not got any worse for at least 4 weeks and you have not needed to take steroids in the last 4 weeks
- Are currently having any other treatment for your neuroblastoma, or you need to have chemotherapy, radiotherapy or surgery to treat symptoms at the time of joining the trial
- Have had major surgery in the last 4 weeks or have not fully recovered from earlier surgery
- Have already had the trial drug or another antibody called ch14.18/SP2/0
- Need to have transfusions of platelets
- Need to take steroids or other drugs that can affect your immune system
- Have any other medical condition that the trial doctors think could affect you taking part
- Are HIV or hepatitis B positive
- Are pregnant or breastfeeding
This is a phase 1/2 trial. Everybody taking part has the monoclonal antibody ch14.18/CHO. You have it slowly though a drip into a thin tube that goes directly into one of the blood vessels in your chest. This is called a central line. If you don’t already have a central line, you will have one put in before you start treatment.
The antibody drip will take 10 days. You have treatment in cycles. The length of the cycle is 32 days. You have up to 5 cycles of treatment all together lasting around 6 months.
As well as having the monoclonal antibody, you also have drugs called aldesleukin and isotretinoin (also known as 13-cis retinoic acid) in each cycle of treatment. The first drug helps the antibody to work better against neuroblastoma. The second drug makes any neuroblastoma cells left in the body less active.
You have aldesleukin as injections under your skin (subcutaneous injection). You have an injection once a day for 5 days before you have the antibody drip and then on the first 5 days that you have the drip.
You take isotretinoin by mouth or through a feeding tube twice a day for 2 weeks, starting shortly after you finish the antibody drip.
Before you start treatment, you see the trial doctors and have some tests. The tests include
- Physical examination
- Blood tests and urine tests
- Heart trace (ECG)
- Heart ultrasound (echocardiogram)
- Chest X-ray
- Tests to see how well your lungs are working (lung function tests)
- A test that looks at the activity in your brain (EEG)
You will also have other scans and a bone marrow test.
During the trial treatment, you have regular blood tests. You have more scans after 2 cycles of treatment. You may also have another bone marrow test.
In the 1st and 2nd cycles of treatment, you stay in hospital to have the antibody drip and the aldesleukin injections. After that, the trial team may talk to you about having part of the treatment at home with very regular visits to the hospital.
When you finish the treatment, you see the trial team once a month for 3 months to have a regular check up. The trial team may ask you to have more tests and scans. They will collect information about how you are getting on from yourself, your parents and other nurses and doctors who know you, for at least 3 years.
As ch14.18/CHO is still quite a new drug, there may be side effects we don’t know about yet. The known side effects include
- Pain – this can be in the back, tummy, arms, legs or other parts of the body
- Numbness and tingling in your hands and feet (peripheral neuropathy)
- Changes in your blood pressure
- Fast heartbeat which may cause pain in your chest
- High temperature (fever)
- Feeling sick
- Low levels of salt in your blood
- Loss of appetite
The side effects of aldesleukin include
- High temperature (fever) and chills
- Flu like symptoms such as headache, tiredness, aches and pains
- Loss of appetite
- Weakness or tiredness (fatigue)
- A drop in your blood pressure
- Rash or itching
- A build up of fluid causing swelling – this can be in your face, neck, arms, tummy or legs
- Changes to the way your liver or kidneys work
- An increase in the number of white cells in your blood
- A drop in the number of red blood cells and platelets causing bleeding problems and tiredness
The side effects of isotretinoin include
- Dryness of the skin around the mouth and nose which can cause bleeding
- Dry, cracked or bleeding lips
- An increased risk of sunburn
- Aches and pains in your joints
- Back pain
- An increase in the amount of fat and calcium in your blood
The trial team will talk to you about all the possible side effects before you agree to join the trial.
Location of trial
This is Cancer Research UK trial number CRUK/11/035.
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