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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.

Clinical trials and coronavirus

Those who are already part of a clinical trial will generally continue to be treated, but many clinical trials are now halting recruitment.

If you're already part of a trial

Although there may need to be some adjustments to protocols, those already on a trial will generally continue to be treated. Of course, safety is the top priority.

If, for any reason, a trial can’t continue or you aren't able to remain part of the trial, you will still be given standard care.

You should continue to talk to your doctors or trial team if you have any concerns. Some trial co-ordinators have suggested that email is better, as these are easier to monitor than phone calls when people are working across different sites and emails create a trail.

I want to take part in a clinical trial

Across the UK, recruitment onto trials is being managed on a site-by-site and case-by-case basis, but most trials are halting recruitment to:

  • redploy staff to deal with Covid-19 on the frontline
  • make research facilities available for tackling Covid-19
  • make sure there is sufficient staff to monitor patient safety for those already on trials
  • limit the contact between people, especially those who are particularly vulnerable.

The decision to halt recruitment is often being taken by the organisations who own the trial, not the unit/site itself.

It's important to remember that not all trials are halting recruitment and there may be very rare exceptions where a doctor can apply for you to join a trial. So, if you're interested in joining a trial you should still discuss it with your doctor to get the latest advice and information.

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.

Phase 0 trials

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.

Phase 1 trials

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.

Phase 2 trial

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.

Phase 3 trial

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

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.

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. 

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.

Read Stephen's story

Stephen Lee, diagnosed with a glioblastoma in 2010

"I didn’t want to know my prognosis - my brother died from the same tumour, so I’d a good idea. My wife Margaret and I tried to remain positive and enjoy whatever time we had.

"I had various treatments, including surgery, then chemotherapy and radiotherapy, and lived as healthy a life as possible.

"Five years later, my tumour was back. I had more surgery and now have epilepsy, plus reduced spatial, concentration and memory skills.

"But I still have a good quality of life and am grateful to people before me who took part in trials that led to the treatment I’ve received.

"Clinical trials are so important to make sure the best possible medication is developed to treat brain tumours.

"In 2015, I took part in a clinical trial to test cannabis-based drug Sativex, taking it alongside my chemo for 21 days in every 28, for a year. It aimed to improve quality of life, which is very important to Margaret and me.

"Now, 10 years on from my diagnosis, we’re still making the most of our time together."

Join one of our Online Support Communities for more stories and tips about coping with a brain tumour diagnosis from people who know what you're going through.


  • 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.)

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

What is an adaptive clinical trial?

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

How long do I stay on a trial?

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.

How can I get onto a trial if it's not offered by my hospital?

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.

How do I know if a clinical trial is safe to enter?

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.

Will I be paid to take part?

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.

Share your experiences and help create change

By taking part in our Improving Brain Tumour Care surveys and sharing your experiences, you can help us improve treatment and care for everyone affected by a brain tumour.

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:

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