The purpose of clinical trials for brain tumour patients is to advance understanding of these tumours and to improve diagnosis and treatment. By their definition, clinical trials are experimental, and while the hope is that they will be beneficial, there is no guarantee.
What is a clinical trial?
A clinical trial is an experiment that involves patients in a new way of managing a condition. Trials are vital to establish whether a new approach is better than the old one.
Developing a new treatment
There are normally four phases to developing a new treatment:
After a treatment is tested in the laboratory, it goes into Phase 1 trials. These set out to answer:
whether the treatment is safe
whether it has any side-effects
what is the right dose to use
Initially, the treatment is given at a low dose to a small group of people, who may be health volunteers. If the group experiences no serious side-effects, another group is given the drug at a higher dose. This process is repeated until the 'maximum tolerated dose' or MTD is determined.
In phase 2, the aim is to find out more about the safety and about whether the new treatment does what is hoped (referred to as its 'efficacy'):
Is it any good?
Does it shrink the tumour?
Does it keep the tumour away for longer? ('progression-free survival')
Does it make the patient feel better?
Is the treatment safe and well-tolerated?
This phase uses a larger group of patients (up to about 100) and can last for a couple of years.
Phase 3 looks at whether the new treatment works better than the existing, 'standard' treatment or whether it produces fewer side-effects. This is done by comparing two groups of patients with similar characteristics. Some of the patients receive the standard treatment and some receive the new treatment. The outcome of the two groups is compared to see whether the new treatment is better.
Which treatment patients receive (standard or new) is often decided on a random basis. It is the most successful way of ensuring that the results of the trial are not biased and is known as 'blinding'.
These trials are on larger groups of people (several thousand patients), who are usually ill, and often last a year or more.
Phase 4 trials are conducted when a drug has been shown to be effective and has been licensed to treat an illness. This phase aims to find out what happens when the drug is given to thousands of people in the general community. The aim is to assess any long-term risks and benefits of the drug, and any rare side effects.
What is an adaptive clinical trial?
Traditional clinical trials test only one drug or different aspect of treatment at a time and take several years to produce results - positive or negative.
In an adaptive clinical trial, researchers can add in new drugs or combinations of therapies in response to a patient's improved or deteriorating condition.
Every trial has a set of 'entry criteria' that you must fit to be able to enter. The best way is to speak to your clinician about trials that may be suitable for you. You can also search for a clinical trial matching your criteria on our Find a clinical trialpage.
What are the advantages and disadvantages of taking part in a clinical trial?
The new treatment might benefit you
Satisfaction from knowing that you are contributing to advancement of science
You are more closely observed for changes in your overall health.
They can be inconvenient and time consuming
It might be that the experimental treatment is not better, or even not as good as, approved therapies which are already available
Unexpected side-effects - the researchers will monitor you closely whilst you are in the trial and make every effort to keep these to a minimum.
Watch Stephen's story, of how he took part in a clinical trial.
"In the past for the medication I've been on, Chemotherapy, somebody else has gone through a clinical trial to ensure that drug was safe to use. I thought it was only fair for myself, in a similiar situation, to do this for other people, who in the future will hopefully benefit from the drug I was going to test."
How long do I stay on the trial?
The trial will go on until one of the following:
You decide to withdraw
It is your right to leave the trial at any time you wish without obligation to give a reason.
The trial comes to an end
The treatment is clearly failing or there are safety concerns
Your doctors believe it is in your best interest to take you off the trial
How can I get onto a trial if it is not offered by my hospital?
This may not be easy. It can mean travelling and staying near the site of the clinical trial. Sometimes a trial is geared to taking patients from other regions. The best thing is to discuss your wishes with your health team to see if particular arrangements can be made.
Dr Packer discusses the impact of molecular understandings on brain tumour clinical trial design and performance, at the ISPNO National Paediatric Information Day, 2016.
Page last reviewed: 01/2018 Next review date: 07/2020