
"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
This trial is looking at how well selumetinib might work for plexiform neurofibromas.
Plexiform neurofibromas are not cancer but are a growth on the nerves.
This trial is open to people with type 1 (NF1) who have plexiform neurofibromas that are causing symptoms. And you can’t have surgery to remove them.
NF1 is a rare genetic () disease. It can affect many parts of the body including the nervous system. This system is made up of:
When NF1 affects the peripheral nervous system this is plexiform neurofibroma (PN).
PN can continue to grow throughout your life. It grows on and along the nerves. This can cause a lot of pain and affect your daily living. PN can also lead to cancer but this is rare.
Doctors treat PN with surgery. However, this is not always possible. Researchers are looking for other treatments for PN.
Selumetinib is a called a growth blocker. It works by stopping an enzyme that PN cells need to grow. Selumetinib is already used to treat some types of cancer.
Researchers think that selumetinib might help people whose PN can’t be removed by surgery. In this trial half the people have selumetinib and the other half have a dummy drug ().
The aims of this trial are to find out:
The following bullet points are a summary of the entry conditions for this trial. Talk to your doctor or the trial 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 trial if all of the following apply. You:
If you are taking painkilling medication the dose needs to have been the same for the past 28 days before going into the trial.
Who can’t take part
Medical conditions
You cannot join this trial if any of these apply. You:
Other
You cannot join this trial if any of these apply. You are:
This is an international phase 3 trial. The team need 146 people worldwide to take part with 10 people in the UK.
It is a randomised double blind trial. A computer puts you into 1 of 2 groups. Neither you nor your doctor can choose which group you go into. And neither you nor your doctor will know which group you are in.
The trial team will be able to tell your doctor which group you are in if this becomes necessary.
The groups are:
Selumetinib is a capsule. You take the capsules twice a day. You must take them on an empty stomach. That means you can’t have any food or drink, apart from water, for 2 hours before taking the capsules and for 1 hour after. Your doctor tells you how many to take.
The dummy drug is a capsule. You take it twice a day. You must take them on an empty stomach. Your doctor tells you how many to take.
You take the dummy drug for a year. After the year you can start taking selumetinib. During the year if your scans show that your PN is getting worse you might be able to start taking selumetinib sooner.
You should not have certain foods and drinks for 7 days before starting treatment and during. As they can affect how well the body absorbs selumetinib. Your doctor will tell more about this.
You continue treatment as long as it is helping and the side effects aren’t too bad.
e-Diary
You have an e-diary to fill in before starting treatment and during treatment. You fill it in everyday and record any pain you have from the PN and what painkillers you take. During treatment you will also record when and how much of selumetinib or the dummy drug you take.
There are also questions in the e-diary about:
This is a quality of life questionnaire.
You must bring this e-diary with you to every clinic visit.
Optional Interview sub study
A member of the team will phone 3 times during the trial:
They will arrange to phone you at a time that is convenient to you. The questions are about:
The interviewer records the interview and keeps it in a safe and confidential place.
You don’t have to take part in this sub study.
Samples for research
You give several blood samples and urine samples during the trial. Your doctor will tell when and how often.
Researchers use these samples to find out:
You see the doctor for tests before taking part. These include:
You see the doctor:
This is to see how you are and to have the same tests you had at the start.
The trial 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 most common side effects of selumetinib are:
Your doctor will talk to you about the possible side effects before you agree to take part.
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Dr Angela Swampillai
AstraZeneca
Freephone 0808 800 4040
"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”