Dialog Box


Clinical Trials

If you have a brain tumour you may consider taking part in a clinical trial. In the past, clinical trials were often seen as a last resort for people who had no other treatment choices. Today, people often have the option of receiving their first cancer treatment in a clinical trial.

If you want to enrol on a clinical trial you should speak to your doctor first. 

Cure Brain Cancer Foundation's vision 

Current treatments for brain cancer do not work for the majority of patients, with only 2 in 10 people surviving for five years. Clinical trials are the best way for patients to access novel treatments, until effective therapies become part of routine care. Cure Brain Cancer's vision is that every person diagnosed with brain cancer in Australia can access new treatments through world-class clinical trials. By working with our international partners to bring these trials here, we are enabling access to novel treatments for patients in Australia at the same time as they are available globally. We are also supporting Australian science to help get promising treatments developed here from the lab into the clinic. 

The information learned from these trials will benefit patients across the world.

Overview of clinical trials

If a new treatment proves effective or more effective than the current standard treatment, trial patients who receive it will be among the first to benefit. Despite this benefit, only 5% of brain cancer patients participate in clinical trials in Australia.* Cure Brain Cancer encourages patients to ask their doctor about the possibility of participating in current trials. 

Clinical trials help improve the diagnosis, treatment and management of people with cancer. People participating in clinical trials also report better health outcomes.

*Canstat: A digest of facts and figures on cancer, Cancers of the brain & central nervous system, Cancer Council Victoria, No. 48, February 2010. Source link.

Phases of clinical trials 

For a treatment to become standard, it must first go through three or four clinical trial phases. 

  • Phase I trials usually involve small groups to test safety, including assessing appropriate dosage and side effects. 
  • Phase II trials usually involve a larger group to build on the results from phase I, further assessing safety but also efficacy.  
  • Phase III trials are intended to demonstrate efficacy of an experimental treatment by determining whether it works better than the standard treatment and can become registered for use in patients. 
  • Phase IV trials occur after a treatment has been made available on the market and monitor efficacy and any side effects occurring over time. Phase IV trials may also examine whether the treatment can be repurposed or used in combination with other treatments.  

Randomisation and standard care

Randomisation is a process used in some clinical trials to prevent bias. Bias occurs when a trial's results are affected by human choices or other factors not related to the treatments being tested. Randomisation helps ensure that unknown factors do not affect trial results.

All phase III and some phase II trials are randomised clinical trials. If you participate in such a trial, you will be assigned by chance (by a computer) to either an experimental group or a control group. Neither you nor your doctor will know which group you are in. 

If you are assigned to the control group, you will get the most widely accepted treatment (standard treatment) for your cancer. If you are assigned to the experimental group, you will get the new treatment being tested. 

Comparing these groups to each other often clearly shows which treatment is more effective or has fewer side effects.

Placebos are almost never used in cancer treatment trials. Even if you are in the control group, you will always receive the best available treatment for your brain tumour.

Taking part in a clinical trial

Researchers create strict eligibility criteria to make sure the people in a study have certain characteristics. The criteria help produce trustworthy results because the participants will have many similarities.Depending on the study, criteria might include:

  • age 
  • type and stage of cancer 
  • symptoms or side effects experienced 
  • length of time since diagnosis or treatment
  • previous medical history 
  • which treatments you have had in the past.  

If you are eligible you’ll need to weigh up the advantages and disadvantages of taking part in a trial. One advantage is accessing programs, medicines or other treatments that are not available outside of the study and may be better than the current standard care. 

Clinical trial safety & eligibility criteria

You may want to know if there are any risks to participating in a clinical trial—will it be safe? Researchers must follow strict guidelines, or duty of care, to make sure studies are as safe as possible.

In clinical trials, requirements must be met in order for a person to be included. These are commonly known as the trial's eligibility criteria and they ensure participants in a trial are like each other in terms of specific factors such as age, type and stage of cancer, general health and previous treatment. When all participants meet the same criteria, it is more likely that the results of the study are caused by the drug / therapy being tested and not by other factors such as chance. [cancer.gov.au]

Clinical trials for childhood brain cancer

About 85 per cent of children who have cancer participate in trials even though there are far fewer children than adults who are diagnosed with cancer. This has led to a great improvement in children’s survival rates because children have been able to access promising treatments, and the evidence for their effectiveness has been obtained quickly. 

Read more about childhood brain cancer here

Zero Childhood Cancer 

Cure Brain Cancer Foundation is excited to be co-funding one of the most exciting childhood cancer research initiatives ever undertaken in Australia, Zero Childhood Cancer. This personalised medicine program involves detailed laboratory analysis of each child’s unique cancer cells, to help identify the drugs most likely to kill their cancer. 

Find out more about Zero Childhood Cancer 

Search for clinical trials for children here and add the tumour type to the search criteria. Make sure you check with your child's medical team about eligibility for specific trials.  

Enrol in a clinical trial 

If you want to join a clinical trial or other study, speak to your doctor first. Most specialists know about current studies and may recommend them to you.

Clinical trials we are helping to fund

GBM AGILE: Adaptive Global Innovative Learning Environment

Lead: Global Coalition for Adaptive Research (GCAR) (2015)

GBM AGILE is a revolutionary new clinical trial which presents a brand new way of testing and developing brain cancer treatments; a world-first global adaptive clinical trial brought about by the biggest collaboration in the history of brain cancer research.



Zero Childhood Cancer: a personalised medicine program 

Lead: Prof Michelle Haber, Children's Cancer Institute Australia, NSW (2015)

Zero Childhood Cancer is a personalised medicine and clinical trial program for children, where researchers and clinicians conduct detailed laboratory analysis of each child’s unique cancer cells, to help identify the drugs most likely to kill their cancer.



VERTU - A phase 2 clinical trial for people with newly diagnosed unmethylated MGMT glioblastoma

Mustafa Khasraw

Lead: A/Prof Mustafa Khasraw, NHMRC Clinical Trials Centre, University of Sydney, NSW (2014 - Present)

A phase 2 clinical trial investigating of adding a new drug to radiotherapy and chemotherapy in patients with newly diagnosed, unmethylated MGMT glioblastoma.



A phase 1 and biodistribution study of an anti-EphA3 antibody in patients with glioblastoma

Hui Gan

Dr Hui Gan, La Trobe University, VIC (2014 - Present)

A phase 1 clinical trial of a new antibody that targets EphA3, which is a cancer protein that makes GBM aggressive and treatment resistant.



Useful links

The Australia Cancer Trials website has been developed by Cancer Australia to provide Australians with consumer-friendly information about current cancer clinical trials. 

Other websites providing information about clinical trials include:

Australian Clinical Trials

Australian New Zealand Clinical Trials Registry


Cancer Trials Australia

Trans-Tasman Radiation Oncology Group

Help Us Bring Trials to Australia
Brain cancer kills more children than any other disease in Australia. Our mission is to increase five-year survival from the current 20% to 50% by 2023 by funding vital research for both adults and children.