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Clinical Trials

40% of all cancers spread to the brain

What is a clinical trial?

Clinical trials study how safe and effective new tests and treatments could be when used in a clinical setting (such as a hospital) to detect, manage, treat or prevent brain tumours. They are therefore the leap from laboratories into people: "from bench to bedside”. 

Clinical trials are part of the research process, so there are no guarantees that an experimental treatment will prove to be of benefit.

However, for brain tumour patients the option to join a trial is often seen as an avenue of hope, as well as the chance to be at the forefront of creating new levels of understanding about brain tumours.

Clinical trials are the culmination of years of knowledge gathered as part of basic scientific research in laboratories, such as those funded by Brain Tumour Research: because it is only when you understand a disease that a potential cure can be designed.

There are also other forms of research that you may wish to consider participating in, whether or not you have a brain tumour. These include epidemiological (population-based) research projects that may provide clues about the causes of brain tumours, for example. Unlike clinical trials that offer patients a new test or treatment, epidemiological research is about observing and asking questions, but it is also an invaluable part of finding a cure for brain tumours.

Discover more about participating in other types of research here.

What is an adaptive clinical trial?

Adaptive clinical trials are designed from the beginning to analyse data and take into consideration new research findings whilst the trial is underway. They include an on-going review/adapt element so that pre-specified changes to the trial can be made as and when required.

This means that adaptive trials are different from traditional clinical trials, which are more rigid in their format and need to run to the end of their plan without being changed during that time. Adaptive clinical trials should therefore be able to make better use of resources such as time and money, to respond more quickly to new discoveries, and to provide useful data throughout the time of the trial. It should also make it easier to test combined treatment approaches: for example, a combination of drugs rather than just one at a time.

Results are also highly likely to be analysed based on personalised medicine – in other words, why are some participants responding better than others to the new treatment being tested? If they represent a particular genetic profile within a tumour type, could the new treatment be altered in order to enhance these results? Should patients who are not responding any longer be offered a treatment that isn’t working for them, but may be causing side effects?

Who can take part in clinical trials?

Clinical trials are carefully designed to meet a wide range of various criteria and considerations, based on the previous years of research findings leading to the trial. This is why clinical trials always stipulate strict eligibility criteria; in other words, it is not the case that any brain tumour patient could join any clinical trial looking at the disease.

Eligibility criteria are designed to ensure that the trial has the very best chance of providing meaningful results. For example, a trial could be designed to test the efficacy in humans of a drug that only influences tumours with a certain genetic mutation. If a patient’s tumour cells don’t have that mutation the drug probably won’t be effective, so these patients would be excluded from the trial because it would be unethical to give them something that is unlikely to help.

How to find a clinical trial for brain tumours

Speak to the medical team where the patient is being treated

The medical team will know if there are any suitable clinical trials recruiting at the centre where the patient is being treated, and may also be aware of others for which the patient would be eligible.

To find a suitable clinical trial it is important to know as much as possible about the type of tumour that the patient has: the name, grade and genetic mutations if possible. The medical team will be able to supply all of this information after they have taken a biopsy and analysed the tumour tissue: although they may well be able to identify the tumour name and possibly grade from the results of an MRI scan before any surgery has taken place.

Online listings of UK based clinical trials

You may want to do a general online search using the tumour type name and the country in which you are looking for a clinical trial. However there are also some databases that are well worth exploring:

  • ClinicalTrials.gov is a resource offered by the US National Library of Medicine. It is a searchable database of privately and publicly funded clinical studies conducted around the world, updated on a daily basis.
  • The European Union Clinical Trials Register allows you to search for interventional clinical trials conducted in the European Union (EU) and the European Economic Area (EEA), as well as clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Be Part of Research is the UK based NHS and National Institute for Health Research (NIHR) website that helps you find out about health and social care research that is taking place across the UK. It was originally known as the UK Clinical Trials Gateway and is an excellent place to search for UK based clinical trials for brain tumours.

If you struggle to interpret these databases, you may also wish to contact our member charity brainstrust. They are there on the end of the phone to help you identify any clinical trials that may be appropriate for you to consider.

Understanding Clinical Trials

Clinical trial phases

New treatments are taken through certain stages of clinical trials in order firstly to establish safety, and then effectiveness.

Phase 0 clinical trials: What effect does this treatment have in humans, if any?

Phase 0 trials use only a few patients (usually fewer than 15). The aim is to test what effects the treatment has on people; for example how successfully a drug can cross the blood brain barrier to reach the brain tumour, and how tumour cells respond. This helps to establish if the drug acts as expected and is safe, because this may be the first time that it has moved from the laboratory into humans.

Not every drug is taken into a phase 0 clinical trial as some may have been used in humans before, but in a different health condition. These repurposed drugs often start testing in a phase 1 trial.

Phase 1 clinical trial: Is the new treatment safe?

In phase 1 trials, researchers test a drug or treatment in larger group of approximately 20 - 80 people. The purpose is to learn about safety and identify any side effects. For example, it may be that a phase 1 clinical trial starts with a low dose of a drug, and then the dose is increased to what the researchers hope will be an effective level, whilst side effects are closely monitored.

Phase 2 clinical trial: Does the treatment work?

These trials typically involve 100-300 people. The goal of a phase 2 clinical trial is usually to extend life but it may also be to improve quality of life. For example, the trial may explore whether reducing the dose of an existing treatment such as a drug or radiotherapy has the same clinical benefits for the patient, but with fewer side effects.

In order to enrol this number of patients in a relatively rare disease such as brain tumours, a phase 2 clinical trial is likely to be a collaboration between a number of different treatment centres.

In both phase 2 and phase 3 clinical trials, participants are likely to be randomly assigned to different groups who are then given different treatment regimes, so that the results can be compared in order to establish which is most effective: this is called a “randomised” clinical trial.

A “randomised, controlled” clinical trial is, for example, one in which the “control” group is given the existing standard of care, so that the new treatment can be compared to the best that is currently available.

Phase 3 clinical trial: Is the new treatment better than the current standard treatment?

The new drug or treatment is given to several hundred people to continue to monitor its effectiveness and comparing it with standard or similar treatments, usually using the randomised, controlled clinical trial format.

In a rare disease such as brain tumours, this is likely to be a global collaboration in order to enrol enough patients.

Phase 4 clinical trial: What happens when the treatment is being commonly used?

After a new treatment is approved and becomes widely available in clinics, researchers continue to track its safety and effectiveness in the general population. Some effects only become clear after a longer period of time. For example, a drug may give patients a longer lifespan than the current standard treatment, but does it give them a better quality of life as well? If it delays a tumour from returning, does it give them a longer overall lifespan or not?

Reporting drug and treatment side effects in the UK

In the UK, the Yellow Card Scheme can contribute to a phase 4 clinical trial. It is run by the Medicines and Healthcare products Regulatory Agency (MHRA) in order to track the long-term effects of medicines and medical devices, as well as other health related products such as vaccines and e-cigarettes. If anyone experiences a side effect, they can report it via the Yellow Card Scheme home page.

These reports are evaluated alongside clinical trial data, medical literature and data from international medicines regulators, to identify previously unknown safety issues. If a new side effect is identified, the safety profile of the medicine or treatment is investigated and compared to the side effects of other ways to treat the same condition. The MHRA will then take action if necessary to ensure that treatments are used in a way that minimises risk, while maximising patient benefit.

Discover more about participating in other types of research here.

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