The following information is taken substantially from the Clinical Trials page of the Australian Leukaemia Foundation’s website and from the MPN Advocates Network’s clinical trial page (an international MPN organisation).
The Leukaemia Foundation’s Clinical Trials page has more detail and links to clinical trial sites for Australian patients.
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.
Clinical trials have three phases:
- In Phase I, the new drug or treatment is tested for its safety in humans and the maximum dose will be determined. In general, patients for whom a standard treatment no longer exists are asked to participate in phase 1 trials.
- In a phase II study, the effectiveness and the optimal dose of the new drug are studied. In a phase III study, the effectiveness of the new drug will be compared to the currently defined standard or best available therapy.
- In Phase lll trials larger patient groups will be used to examine whether the new drug is more effective than the current best treatment. After successful phase III trials, drugs can then be submitted for approval by medical authorities such as the European Medicines Agency (EMA), or the Food and Drug Administration (the FDA) in America.
After a drug is launched, the long-term effects may still be monitored, sometimes referred to as postmarketing studies or phase IV studies.
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 would 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?
First, talk to your doctor. They will be able to access an up-to-date listing of clinical trials.
Information about current MPN clinical trials in Australia are available on the clinical trials page of the Leukaemia Foundation website.
Australian clinical trials are recognised internationally for including very high quality patient care.
FAQs about clinical trials
If you are considering participating in a clinical trial these clinical trial FAQs from the MPN Research Foundation in the US are useful.
