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Brain Cancer Clinical Trials
Clinical trials are at the heart of progress in brain cancer research — turning promising discoveries into real treatment options. Explore current trials, understand what’s involved, and find out if a clinical trial could be right for you or someone you love.
What are Clinical Trials?
Clinical trials are rigorously designed research studies that aim to answer questions and test the safety and efficacy of new medical interventions (e.g., drugs, surgical techniques, management strategies) in human participants.
A clinical trial builds on years of pre-clinical research. Before a new therapy reaches patients, it must show promise in laboratory studies using cancer cell lines, organoids, or animal models.
These studies assess things like:
- How the treatment works
- Whether it targets the tumour effectively
- What side effects it might cause
Only those interventions that demonstrate a strong scientific rationale and an acceptable safety profile in pre-clinical models progress to clinical trials.
Why participate in a Clinical Trial for Brain Cancer?
Clinical trials give patients access to potentially groundbreaking treatments before they are widely available. They also contribute to the development of future therapies and help researchers understand how to treat brain cancer more effectively. Participation is entirely voluntary, and patients can withdraw at any time.
Ultimately, it’s up to you as to whether you think participating in a clinical trial is right for you, and if you meet the eligibility criteria for a Trial.
If you’re considering a clinical trial, your first step should always be to speak with your oncologist and/or physician. Clinical trials follow specific eligibility criteria, which consist of inclusion and exclusion criteria.
Inclusion criteria specify the characteristics that make someone eligible to join, while exclusion criteria identify factors that disqualify potential participants.
These criteria are crucial for ensuring the safety of participants, maintaining the integrity of research results, and determining the generalisability of findings.
If you’re eligible, you’ll go through an informed consent process, where a doctor explains:
- What the trial involves,
- How your care will be managed,
- Any known risks or procedures,
- And your right to withdraw at any time.
In Australia, participation in research is entirely voluntary and governed by the National Statement on Ethical Conduct in Human Research. Choosing not to take part will not affect your access to standard care.
“Clinical trials are the essential step between research that is promising in the laboratory, and research that has real benefit to people’s lives. Every advance that has been made in brain cancer has been built on the generosity of people who have trusted their doctors and taken part in a clinical trial. I encourage everyone – patients, families, doctors, and researchers – to make a difference for people with brain cancer by considering clinical research as a crucial part of clinical care.”
Prof. Anna Nowak
Medical Oncologist
Clinical Trial Phases
Clinical Trials are phased. The process begins with Phase I trials, which are used to assess the safety, maximum tolerated dose of the trial drug, along with the interactions between drugs, pharmacokinetics (what the body does to the drug) and pharmacodynamics (what the drug does to the body) in human subjects. If a therapy appears tolerable, it progresses to Phase II, where preliminary effectiveness and side effects are assessed in larger groups (hundreds). Finally, Phase III trials involve hundreds to thousands of participants and test the new intervention against the current standard of care. Phase IV clinical trials, also known as post-marketing surveillance trials, occur after a treatment or drug has been approved and is already available to the public. These trials focus on gathering more information about the treatment’s long-term safety and effectiveness in a wider population.
Each phase requires rigorous data collection, ethical oversight, and time, which can take between 6 and 10 years from first-in-human use to regulatory approval. Rushing any process increases the risk of approving treatments that are ineffective or harmful. clinical trials, also known as post-marketing surveillance trials, occur after a treatment or drug has been approved and is already available to the public. These trials focus on gathering more information about the treatment’s long-term safety and effectiveness in a wider population.
Each phase requires rigorous data collection, ethical oversight, and time, which can take between 6 to 10 years from first-in-human use to regulatory approval. Rushing any process increases the risk of approving treatments that are ineffective or harmful.
Purpose
Test a new intervention for the first time
Participants
20–80 people
Key Features
- Determines a safe dosage range
- Identifies side effects
Purpose
Evaluate whether the intervention works as intended (efficacy)
Participants
Several hundred people
Key Features
- Further evaluates safety
- Assesses effectiveness
Purpose
Compare the new intervention to current treatments or placebo
Participants
Several hundred to several thousand
Key Features
- Compares with standard care or placebo
- Monitors adverse effects
- Collects safety data for regulatory approval
Purpose
Monitor real-world use after approval
Participants
General population
Key Features
- Tracks long-term effectiveness and side effects
- May assess use in other conditions or with other therapies
Purpose
Test a new intervention for the first time
Participants
20–80 people
Key Features
- Determines a safe dosage range
- Identifies side effects
Purpose
Evaluate whether the intervention works as intended (efficacy)
Participants
Several hundred people
Key Features
- Further evaluates safety
- Assesses effectiveness
Purpose
Compare the new intervention to current treatments or placebo
Participants
Several hundred to several thousand
Key Features
- Compares with standard care or placebo
- Monitors adverse effects
- Collects safety data for regulatory approval
Purpose
Monitor real-world use after approval
Participants
General population
Key Features
- Tracks long-term effectiveness and side effects
- May assess use in other conditions or with other therapies
| Phase | Purpose | Participants | Key Features |
|---|---|---|---|
Phase I | Test a new intervention for the first time | 20–80 people |
|
Phase II | Evaluate whether the intervention works as intended (efficacy) | Several hundred people |
|
Phase III | Compare the new intervention to current treatments or placebo | Several hundred to several thousand |
|
Phase IV | Monitor real-world use after approval | General population |
|
Find Clinical Trials in your area
Important note: Cure Brain Cancer Foundation provides this information to support general understanding. We do not recruit for or manage clinical trials. Decisions about participation should always be made in consultation with your medical team, who can guide you based on your individual situation.
Brain Cancer Clinical Trials Australia FAQs
What are Adaptive Clinical Trials?
Trials like GBM AGILE and INSIGhT in brain cancer use adaptive platform designs that test multiple therapies simultaneously and adjust in real time based on patient responses. This model is already helping brain cancer research move faster and more efficiently. While these innovations are promising, they are not yet the norm. Most trials still rely on traditional designs, and adaptive models require significant upfront investment in infrastructure, analytics, and regulatory negotiation. Until these new models become more widespread, clinical trials will remain a careful, stepwise process.
Are clinical trials safe?
Clinical trials are carefully designed to protect participants. They follow strict ethical and scientific guidelines and must be approved by ethics committees and regulatory authorities. While there are always risks with new treatments, participant safety is a top priority, and patients are monitored closely throughout the trial.
Why do new cancer treatments take so long to become available to patients?
If you or someone you love has cancer, the wait for new treatments can feel unbearably slow. It’s completely understandable to wonder why promising therapies take so long to reach patients. While there are many systemic challenges, such as funding limitations, difficulties with patient recruitment, and complex regulatory processes, part of the delay is built into the structure of how clinical trials work. These studies are intentionally designed to move in careful, sequential phases to ensure that any new treatment is both effective and safe before it becomes widely available.
Will I receive a placebo instead of real treatment?
In brain cancer trials, placebos are rarely used alone. Most trials compare a new treatment to the current standard of care, meaning all participants receive some form of active treatment. Placebos may be used in combination with standard treatments to test the effect of a new therapy.
Can I still see my own doctor if I join a clinical trial?
Yes. In most cases, you can continue seeing your regular doctor while participating in a clinical trial. The trial team will often work with your healthcare provider to coordinate your care and ensure you’re supported throughout.
What happens if the trial treatment doesn’t work for me?
If a trial treatment isn’t helping or is causing harmful side effects, your care team will discuss other options with you. You always have the right to stop participating in the trial at any time and return to standard treatment options.
What do brain cancer clinical trials test?
Clinical trials for brain cancer may test new treatments, new combinations of therapies or different ways to use existing treatments. This can include treatments used alongside surgery, radiotherapy, chemotherapy or other drug therapies.
Depending on the phase of the study, researchers may look at safety, side effects, outcomes, how a treatment works in the body, whether it shows benefit for patients, and whether it should be studied in a larger group. Every clinical trial has its own aim, eligibility criteria, risks and potential benefits, so it’s important to discuss any trial with your medical team.
Are there different clinical trials for different types of brain cancer?
Yes. Brain cancer clinical trials may focus on different brain tumours, age groups, cancer types or stages of disease. Some trials may be designed for people with glioblastoma, while others may involve children, young adults or adult patients with other types of brain cancer.
Eligibility can depend on many factors, including your diagnosis, tumour type, symptoms, previous treatment, general health and whether the trial is currently recruiting. Your oncologist or treating team can help you understand whether a clinical trial may be relevant to your situation.
Do I have to travel to take part in a clinical trial?
Some trials require visits to specific hospitals or research centres, which might involve travel. Others may be available at your treating hospital. Ask the trial coordinator if travel assistance is available or if remote participation is possible for parts of the study.
Is there a cost to participate in a clinical trial?
In Australia, the cost of the treatment being studied is typically covered by the trial sponsor, and standard medical care is often funded through Medicare or private health insurance. However, it’s important to ask whether there are any out-of-pocket costs, such as travel or accommodation.
Trials like GBM AGILE and INSIGhT in brain cancer use adaptive platform designs that test multiple therapies simultaneously and adjust in real time based on patient responses. This model is already helping brain cancer research move faster and more efficiently. While these innovations are promising, they are not yet the norm. Most trials still rely on traditional designs, and adaptive models require significant upfront investment in infrastructure, analytics, and regulatory negotiation. Until these new models become more widespread, clinical trials will remain a careful, stepwise process.
Clinical trials are carefully designed to protect participants. They follow strict ethical and scientific guidelines and must be approved by ethics committees and regulatory authorities. While there are always risks with new treatments, participant safety is a top priority, and patients are monitored closely throughout the trial.
If you or someone you love has cancer, the wait for new treatments can feel unbearably slow. It’s completely understandable to wonder why promising therapies take so long to reach patients. While there are many systemic challenges, such as funding limitations, difficulties with patient recruitment, and complex regulatory processes, part of the delay is built into the structure of how clinical trials work. These studies are intentionally designed to move in careful, sequential phases to ensure that any new treatment is both effective and safe before it becomes widely available.
In brain cancer trials, placebos are rarely used alone. Most trials compare a new treatment to the current standard of care, meaning all participants receive some form of active treatment. Placebos may be used in combination with standard treatments to test the effect of a new therapy.
Yes. In most cases, you can continue seeing your regular doctor while participating in a clinical trial. The trial team will often work with your healthcare provider to coordinate your care and ensure you’re supported throughout.
If a trial treatment isn’t helping or is causing harmful side effects, your care team will discuss other options with you. You always have the right to stop participating in the trial at any time and return to standard treatment options.
Clinical trials for brain cancer may test new treatments, new combinations of therapies or different ways to use existing treatments. This can include treatments used alongside surgery, radiotherapy, chemotherapy or other drug therapies.
Depending on the phase of the study, researchers may look at safety, side effects, outcomes, how a treatment works in the body, whether it shows benefit for patients, and whether it should be studied in a larger group. Every clinical trial has its own aim, eligibility criteria, risks and potential benefits, so it’s important to discuss any trial with your medical team.
Yes. Brain cancer clinical trials may focus on different brain tumours, age groups, cancer types or stages of disease. Some trials may be designed for people with glioblastoma, while others may involve children, young adults or adult patients with other types of brain cancer.
Eligibility can depend on many factors, including your diagnosis, tumour type, symptoms, previous treatment, general health and whether the trial is currently recruiting. Your oncologist or treating team can help you understand whether a clinical trial may be relevant to your situation.
Some trials require visits to specific hospitals or research centres, which might involve travel. Others may be available at your treating hospital. Ask the trial coordinator if travel assistance is available or if remote participation is possible for parts of the study.
In Australia, the cost of the treatment being studied is typically covered by the trial sponsor, and standard medical care is often funded through Medicare or private health insurance. However, it’s important to ask whether there are any out-of-pocket costs, such as travel or accommodation.
References
- DiMasi, J. A., Grabowski, H. G., & Hansen, R. W. (2016). Innovation in the pharmaceutical industry: New estimates of R&D costs. Journal of Health Economics, 47, 20–33. https://doi.org/10.1016/j.jhealeco.2016.01.012
- Wong, C. H., Siah, K. W., & Lo, A. W. (2019). Estimation of clinical trial success rates and related parameters. Biostatistics, 20(2), 273–286. https://doi.org/10.1093/biostatistics/kxy072
- Alexander, B. M., Trippa, L., Gaffey, S. C., Arrillaga-Romany, I., Taylor, J. W., Wen, P. Y., & Cloughesy, T. F. (2023). Adaptive platform trials in glioblastoma: Lessons from INSIGhT and GBM AGILE. Cancer, 129(6), 930–940. https://doi.org/10.1002/cncr.34850
- DiMasi, J. A., Grabowski, H. G., & Hansen, R. W. (2016). Innovation in the pharmaceutical industry: New estimates of R&D costs. Journal of Health Economics, 47, 20–33. https://doi.org/10.1016/j.jhealeco.2016.01.012
- Wong, C. H., Siah, K. W., & Lo, A. W. (2019). Estimation of clinical trial success rates and related parameters. Biostatistics, 20(2), 273–286. https://doi.org/10.1093/biostatistics/kxy072
- Alexander, B. M., Trippa, L., Gaffey, S. C., Arrillaga-Romany, I., Taylor, J. W., Wen, P. Y., & Cloughesy, T. F. (2023). Adaptive platform trials in glioblastoma: Lessons from INSIGhT and GBM AGILE. Cancer, 129(6), 930–940. https://doi.org/10.1002/cncr.34850