Some useful links to information about clinical trials in Australia are provided below. This page covers:

What is a clinical trial?

A clinical trial is a research study that helps to determine whether a new drug or device is safe and effective. Each study is designed to answer questions and find better ways to screen, diagnose, prevent or treat a disease or condition. They may be sponsored by drug manufacturers, government, and organisations such as the Leukaemia Foundation.

Each trial has a plan that maps out how the trial will be conducted–what will be done, by whom, when and why. This protocol also explains who is eligible to participate in a trial and what is expected of them. If you are eligible, a team of doctors and nurses will manage your care. Trials are held at hospitals and research centres around the country.

What are the benefits of participating in a clinical trial? Should I consider taking part?

Participation in a clinical trial can give patients access to cutting-edge, potentially life-saving and life-enhancing treatments. Patient participation contributes to the advancement of medicine and helps others who share or may develop a myeloproliferative neoplasm.

Before signing up, patients should learn as much as possible about the trial, then discuss options with their doctor. Clinical trials are not right for everyone, nor is every patient able to participate. Before starting any trial, patients should understand what will happen during the study, what is expected, the care that will be received and any costs that you may have to cover. You will then be given a written consent form to sign.

Myelofibrosis patient and clinical trial participant Charlie was interviewed by the Leukaemia Foundation.
Here is her interview as part of a Leukaemia Foundation webinar about ‘Understanding clinical trials’.

Clinical trials usually have four phases (information below taken from australianclinicaltrials.gov.au): 

Phase I clinical trials:

  • test a new intervention for the first time
  • use a small number of people (between 20 and 80)
  • determine a safe dosage range
  • identify side effects.

Phase 2 clinical trials:

  • use a larger group of people, for example several hundred
  • determine efficacy; that is, whether it works as intended
  • further evaluate safety.

Phase 3 clinical trials:

  • use large groups of people, from several hundred to several thousand
  • compare the intervention to other standard interventions, experimental interventions, standard care or a placebo
  • monitor adverse effects
  • collect information that will allow the intervention to be used safely.

Phase 4 clinical trials:

  • happen after an intervention is in use
  • monitor the effectiveness of the approved intervention in the general population
  • collect information about any adverse effects associated with widespread use over longer periods of time
  • may also be used to investigate potential use of the intervention in a different condition, or in combination with other therapies.

What are the risks – isn’t it dangerous to take an experimental drug?

The risks depend on the type of treatment being studied and the health of the individual patient. For some, there could be unpleasant, even serious, side effects. Often these side effects are temporary and end when the treatment stops. There are both known and unknown risks with any trial. Be sure you understand the known risks before you join any study.

Whilst most clinical trials involve some risk, researchers must follow strict scientific guidelines and ethical and legal codes to ensure that you are protected. Studies need to be approved by an Independent Ethics Committee (IEC) or Institutional Review Board (IRB), or an equivalent, depending upon the regulations of the country where the trial is being carried out.

As a participant of a clinical trial, you will receive excellent medical care from a team of doctors, nurses, researchers, social workers and other health professionals who are on hand to manage your condition. The trial’s protocol may require you to visit the study site more often to check in with your study doctor. Plus you may receive more tests and treatments than usual.

How do I find a clinical trial in Australia?

  1. First, always discuss the clinical trial option with your haematologist.
  2. The MPN AA has identified that the following clinical trials are currently available for Australian MPN patients – see our document Australian Clinical Trials as at Nov 2025.
    While we try to keep this up to date, things change. New trials appear and others progress to different stages. For the most up to date information on the trials, follow the links in the document or check out the sources below.
  3. FYI, our document listing current Australian clinical trials for MPN patients was obtained from:

Australian clinical trials are recognised internationally for including very high quality patient care.

Source of information on this page
NB. The background clinical trials information on this page has been taken substantially from the Clinical Trials page of the Australian Leukaemia Foundation’s website and from the Australian Government’s Clinical Trials website.
The Leukaemia Foundation’s Clinical Trials page has even more detail for Australian patients.

 

 

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