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Clinical trials

The purpose of clinical trials is to gain a better understanding of brain tumours to help improve diagnosis and treatment. 

Some trials also seek to control symptoms more effectively and improve quality of life. 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 developing a new treatment or way of managing a condition. It aims to find out:

  • if the new way works
  • if it’s safe
  • what the best way of giving it is
  • whether the new approach is better than the old one.

Before a drug enters a clinical trial, it will have had preclinical testing in the laboratory, often for several years.

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Developing a new treatment

A clinical trial to develop a new treatment has up to 5 phases, each of which can take some time. So it can take 10-15 years or more for a treatment to go from initial design to becoming standard treatment in the clinic.

Not all treatments go through a phase 0 trial.

Phase 0 clinical trials:

  • aim to speed up the development of promising drugs by testing them in people rather than animals, which gives more reliable results.
  • use small, therapeutic doses of a drug or treatment to see what it does in people.
  • use doses too small to treat your tumour, but you’re less likely to have side-effects.

These are usually the earliest trial of a treatment in people, after testing in the laboratory

Phase 1 clinical trials tend to be carried out in specialised clinical research units rather than hospitals. They recruit only a few people and last several months.

Phase 1 clinical trials aim to find out:

  • whether the treatment is safe
  • what’s the right dose to use
  • whether it has any side-effects.

Treatment is:

  • initially given at a low dose to a small group of people, who may be healthy volunteers
  • then, if the first group experienced no serious side-effects, given to another group at a higher dose.
  • This process is repeated with other groups, until the ‘maximum tolerated dose’ or MTD is determined.

Patients receive optimum care and attention while in the unit.

Not all treatments tested in phase 1 will make it to phase 2.

Phase 2 clinical trials use larger groups of patients (up to about 100) and can last for a couple of years.

Phase 2 clinical trials aim to find out:

  • more about the safety and side-effects of the treatment
  • whether the new treatment does what’s hoped (referred to as its efficacy), for example, shrink the tumour, keep it away for longer, make the patient feel better.


  • often compare the new treatment to an existing treatment or to a dummy drug (placebo)
  • may be a randomised trial – people are put into the different treatment groups at random.

Most drugs don’t make it to phase 3 because they turn out to be ineffective, unsafe or have intolerable side-effects in phase 2.

Phase 3 clinical trials often take place in many hospitals across different countries. They are carried out on larger groups (several thousand patients), who are usually ill, and often last for several years.

Phase 3 trials aim to find out:

  • if the new treatment works better than the existing, ‘standard’ treatment
  • whether it produces fewer side-effects.


  • Compare 2 groups of patients with similar characteristics to see if the new treatment is better
    – 1 group has the standard treatment and 1 group has the new treatment.
  • Are often ‘randomised, blind trials’, to make sure the results of the trial are not biased and can be trusted.

Randomised = patients are put into the different treatment groups at random.

Blind = the patient doesn’t know which treatment they are receiving [single blind] or = the patient AND the researcher don’t know which treatment the patient is receiving [double blind].

Many drugs fail at this stage.

If successful, however, the treatment can be put forward to the appropriate authorities for approval to be licensed for use. This approval process can take several years.

Phase 4 trials are conducted after a drug has been shown to be effective and has been licensed to treat an illness.

Phase 4 trials:

  • aim to find out what happens when the drug is given to thousands of people in the general community
  • assess any long-term risks and benefits and any rare side-effects of the drug.

Expanded Access Programmes

Patients who’ve been treated with a promising medicine during a clinical trial and who want to continue treatment after the end of the trial, may be able to do so via an Expanded Access Programme (EAP). In the US, this is called an open-label extension (OLE) study.

If the medicine then goes on to be licensed, you can no longer receive it via the Expanded Access Programme, but you can be prescribed it in the usual way medicines are prescribed.

In an Expanded Access Programme, patients are usually followed up in the same way as patients in a clinical trial.

Dr Packer discusses the impact of molecular understandings on brain tumour clinical trial design and performance, at the ISPNO National Paediatric Information Day, 2016.

How do I find a suitable clinical trial?

Every trial has a strict set of entry criteria that you must fit to be able to take part in the trial. The best way to find a trial is to speak to your healthcare team about trials that may be suitable for you.

You can search for a clinical trial online or by signing up to BRIAN, our trusted online app, and using the Clinical Trials Finder. 

“In the past for the medication I’ve been on, 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.” Stephen

What are the advantages and disadvantages of taking part in a clinical trial? 


  • The new treatment might be better
  • If the treatment is an improvement, you may be one of the first patients to benefit from it
  • You’re more closely observed for changes in your overall health – so these may be picked up and dealt with more quickly
  • Satisfaction from knowing that you’re contributing to advancement of science.

I felt that it was only right for me to help people who would have a brain tumour in the future – so they can receive proven medication that works to control the disease and aid recovery.


  • They can be inconvenient, time consuming and costly due to additional trips to hospital, travelling to treatment centres that are further away and the potential effect on your insurance. 
  • The experimental treatment might not be better than, or even as good as, approved therapies already available. 
  • Unexpected side-effects. (The researchers will monitor you closely while you’re in the trial to keep these to a minimum.) 
  • You might not be receiving the new treatment. (You might be on a placebo.)

Tips from our community

“Our experience was that the NHS seemed to run out of treatment options very quickly. They didn’t suggest any clinical trials for my son, but after we did some research ourselves we found some trials that he would be eligible for.”

“You have to look at all the pros and cons and weigh up what the right choice is for you. Quality of life vs. quantity of life can be hard to think about, but it’s important to consider.”

“You’re entitled to a second opinion so possibly worth seeing if you can see another oncologist before making the decision whether to participate. If they agree that traditional chemo wouldn’t be effective then you’ve nothing to lose really by taking part in the clinical trial.”

“We decided against a clinical trial. There’s no right or wrong decision. I spent every waking hour trawling through the internet to try and find something but in the end I decided to just enjoy the time I have instead.”

By joining one of our our Online Support Communities, you can get more tips about living with or beyond a brain tumour diagnosis from people who truly understand what you’re going through.

Find out more

Frequently asked questions

Traditional clinical trials test only one drug or 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, thereby speeding up the research.

This is particularly useful for fast growing tumours.

Find out more

The trial will go on until one of the following happens:

  • you decide to withdraw – it’s your right to leave the trial at any time, without having to give a reason
  • the trial comes to an end
  • the treatment is clearly failing or there are safety concerns
  • your doctors believe it’s in your best interest to take you off the trial.

This may not be easy. It can mean travelling to and staying near the site of the clinical trial. Sometimes a trial is geared to take patients from other regions. The best thing is to talk to your healthcare team to see if particular arrangements can be made.

The UK has some of the most rigorous patient protection practices in the world. All trials are scrutinised to make sure they meet international, national and local standards of good practice by organisations including (but not limited to):

  • Medicines and Healthcare Products Regulatory Agency (MHRA)
  • Research Ethics Committee
  • clinical trial committees.

No, it would be unusual for patients to be paid for taking part in an oncology trial. 

You can ask your local doctor or nurse if there are any local arrangements they can offer to help with the cost, e.g. access to hospital transport or free/reduced parking.

Clinical trials and brain tumours – Clear Print version (pdf) –

Find out more in the full fact sheet – Clear Print version, designed to RNIB guidelines.

If you have further questions, need to clarify any of the information on this page, or want to find out more about research and clinical trials, please contact our team:
Support and Information Services
0808 800 0004 Free from landlines and mobiles
Phone lines open Mon-Fri, 09:00-17:00

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