A trial of dabrafenib for children and teenagers who have a solid tumour with a change to a gene called BRAF

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Children's cancers

Status:

Closed

Phase:

Phase 1/2

This trial is looking at a drug called dabrafenib. It is for children and teenagers who have a solid tumour Open a glossary item that has got worse or come back despite having standard treatment . A solid tumour is any cancer apart from leukaemia .

More about this trial

The trial is for children and teenagers up to 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.

In some solid tumours, there is a change (mutation Open a glossary item ) in a gene called BRAF. The BRAF gene makes a protein that affects how cancers divide and grow. Dabrafenib is a drug that blocks the action of the BRAF protein (a BRAF inhibitor).

The aims of this study are to

Find the highest safe dose of dabrafenib that people between the age of 1 year and 18 years can have safely
Learn more about the side effects and what happens to the drug in your body

Who can enter

You may be able to enter this trial if

You are at least 1 year old, but have not yet reached your 18th birthday
Your cancer has got worse or came back despite having at least 1 type of standard treatment – if you have melanoma that can’t be removed with surgery, you can take part even if you haven’t had any other treatment
Your cancer has a specific change to a gene called BRAF – the trial team will need a sample of your tumour to check for this
You are well enough to take part – this means that you need help to care for yourself, but not all the time (Karnofsky score of more than 50), or for younger children it means you get dressed and even if you don’t actively play, you take part in quiet play and activities (Lansky score of more than 50)
You have satisfactory blood test results
You are willing to use reliable contraception if there is any chance you could become pregnant

You cannot enter this trial if you

Have leukaemia
Have had radiotherapy or chemotherapy in the last 3 weeks (6 weeks if you had a drug called a nitrosourea )
Have had an experimental drug in the last 4 weeks (or earlier if there is any chance some of the drug could still be in your body)
Have any cancer other than the solid tumour with the BRAF gene change, or have had another cancer in the past unless it was carcinoma in situ of the cervix or non melanoma skin cancer that was successfully treated, or another cancer that was successfully treated at least 3 years ago
Haven’t recovered from the side effects of other cancer treatment, unless they are very mild
Have had an allergic reaction to a drug similar to dabrafenib, or to anything it contains
Have had a stem cell transplant in the last 3 months
Have had a heart attack or have certain other heart problems
Have a problem with your digestive system that could affect how you absorb the trial drug
Are known to be HIV, hepatitis B or hepatitis C positive
Are pregnant or breastfeeding

As well as the above, you can’t enter the 2nd part of the trial if you have already had dabrafenib or a similar drug.

Trial design

This phase 1/2 trial will recruit nearly 85 children and young people all together. It is in 2 parts. Everybody joining the trial has dabrafenib.

In part 1, the researchers are looking for the highest dose of dabrafenib that people between 1 year and 18 years can have.

The first few patients taking part will have a low dose of dabrafenib. If they don’t have any serious side effects, the next few patients will have a higher dose. And so on, until they find the best dose to give. This is called a dose escalation study. Part 1 has recruited enough patients and is now closed.

In part 2, the researchers want to learn more about the side effects, what happens to dabrafenib in your body, and whether it helps children who have solid tumours with a BRAF gene mutation. Everybody joining this part of the trial will have the highest safe dose found in part 1.

Dabrafenib is a capsule that you take twice a day. If you can’t swallow capsules, you can have the drug as a liquid. The trial team will give you more information about exactly how and when to take the drug.

As long as you don’t have bad side effects, you can carry on having dabrafenib for as long as it helps you.

Hospital visits

You see the trial team and have some tests before you start the trial treatment. The tests include

Physical examination
Heart trace (ECG )
Heart scan (echocardiogram )
Blood tests
Urine test
MRI scan or CT scan

The trial team need a sample of your tumour to check if it has the specific change to the BRAF gene. They will get a sample of tissue that was removed when you had surgery or a biopsy Open a glossary item in the past. But if there isn’t a sample available, you will need to have a new biopsy to be able to take part in the trial.

You go to hospital once a week for the first 4 weeks of treatment. After that, you go every 4 to 12 weeks for the rest of the time you have the trial treatment.

You have regular blood tests throughout the trial. And you will have more heart traces and heart scans. The trial team may ask you to have more CT or MRI scans.

When you stop taking dabrafenib, you see the trial team again and have a physical examination, blood tests, a heart trace and a heart scan. You will also have a CT or MRI scan if you haven’t had one recently.

The researchers may ask you to have another biopsy. But you don’t have to have this biopsy if you don’t want to.

Your doctor may also want you to have your skin examined 2 months after you stop taking dabrafenib.