What is a Clinical Trial?

Clinical trials are research in action and are an essential part of the health system in Australia and New Zealand. Clinical trials are carefully designed research studies that find out if new treatments are more effective than those currently accepted as the best available standard of care. Clinical trials can look at how well treatments work to control or cure disease, symptoms, side effects, and whether they can improve quality of life.

All new breast cancer treatments and prevention strategies must be rigorously tested through the clinical trials process before they are made widely available to the community.

Why have Clinical Trials?

Clinical trials enable us to find new ways of preventing, diagnosis and treating breast cancer. All treatments currently used in the clinic in Australia and New Zealand, have undergone a clinical trial to ensure efficiency and safety of patients. Some examples of cancer care milestones that have been achieved through clinical trials research are:

Chemotherapy and hormone treatment which are effective for preventing breast cancer recurrence and can save lives.
Mammograms which save lives through early detection of disease.
Surgical procedures such as lumpectomies which enable surgeons to safely remove the cancer whilst preserving the breast.
Drug treatments such as Tamoxifen which can prevent breast cancer in women with an increased risk of developing the disease.
Personalised and targeted treatments which enables a specific cancer type to be targeted with a treatment that can improve patient outcomes substantially.

Are Clinical Trials Safe?

Patient safety is the number one priority for researchers and doctors conducting breast cancer clinical trials in Australia and New Zealand. To ensure patient safety in the conduct of clinical trials, every clinical trial must have a document called a protocol. Clinical trial protocols are written by experienced doctors, and a team of experts in breast cancer treatment, translational research, and clinical trial coordination. All clinical trials conducted by Breast Cancer Trials, are also monitored by the Breast Cancer Trials Scientific Advisory Committee.

The clinical trial protocol outlines the reason for doing the study, who is eligible to participate, the treatment and test that are involved, when these will be done and why. The protocol must be approved by an independent panel of scientists, medical professionals, and consumers, called an ethics committee. The progress of the clinical trial and the safety of clinical trial participants is carefully reviewed and monitored by an independent Data and Safety Monitoring Committee and by the ethics committee responsible for approving the clinical trial.

Before joining a clinical trial, potential participants must understand:

Why the clinical trial is being conducted?
The potential risks and benefits of participating in the clinical trial.
What their involvement would include.

The decision to participate is made based on information provided to the patient by her treating doctor. Written information about the clinical trial will also be provided. The potential participant will be encouraged to go home and read through the information with a trusted person and will be able to ask any questions they have to their doctor before agreeing to participate. Both the doctor and the potential participant must be satisfied that all information about the clinical trial is understood and a statement to this effect is signed by both parties. This process is called ‘obtaining informed consent to participate in a clinical trial.’

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Download our “What are Breast Cancer Clinical Trials” brochure to find out more information on the purpose and benefit of participating in clinical trials research.

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What are the Different Phases of Clinical Trials?

Clinical trials are generally conducted in four phases. Each phase is designed to answer a certain scientific question.

Phase One Trials

Phase one clinical trials are conducted to test a new biomedical intervention for the first time in a small group of people, usually around 20 to 50 people. A phase one trial evaluates the safety and side effects of the new treatment or intervention. A phase one trial is conducted after the new biomedical intervention has been tested extensively in the lab, or another research setting. Phase one trials are not randomised.
Phase Two Trials

If the treatment of intervention is proven successful in phase one, it is then moved to a phase two clinical trial and tested in a larger group of people. A phase two clinical trial aims to see how well the new treatment or intervention works against cancer, and to monitor for side effects.
Phase Three Trials

Phase three clinical trials compare new treatments with the best currently available treatment, also known as standard treatment. This phase of clinical trials can also test standard treatment with a different dose of the same treatment, or a different way of giving the same treatment. Phase three clinical trials study the efficiency of a treatment or intervention in large groups of people, from several hundred to several thousand. It looks at which treatments work best for the disease, how the treatment affects quality of life and helps researchers learn more about the treatment or interventions side effects.
Phase Four Trials

A phase four clinical trial is conducted after the new treatment or intervention has been marketing and approved. A phase four clinical trial aims to learn more about the side effects and safety of a new treatment or intervention, the long-term risks and benefits of the new treatment and how effective the treatment is when used in the general population over a longer period of time.

What are the Different Types of Clinical Trials?

There are four main types of clinical trials conducted in Australia and New Zealand for the treatment and prevention of breast cancer: treatment, screening, prevention clinical trials and quality of life studies.

Treatment Clinical Trials

Treatment clinical trials are designed to find out which treatments are the most effective and to test new treatments, surgical methods, devices and treatment combinations or dosages, to see if they are better at improving outcomes for patients compared to the current standard treatments available.
Screening Clinical Trials

Screening clinical trials are used to evaluate tests or procedures to diagnose and detect diseases or conditions. The aim of a screening clinical trial is to identify the breast cancer before it has caused symptoms, with the hope of offering early treatment for the disease and help to improve patient outcomes. A screening test, like a mammogram, does not usually diagnose the cancer but can show if there is reason for further investigation.
Prevention Clinical Trials

Prevention clinical trials are designed to test new strategies to prevent breast cancer in women who have never had the disease but who are at high risk. They can also be conducted in the general population. These clinical trials test an intervention, like a drug, to see if it can stop a cancer from developing.
Quality of Life Studies

Quality of life studies pay special attention to the breast cancer patient’s feelings about the impact and side effects of treatment. The aim of these assessments is to look for ways to improve the overall experience of the patient who receive these treatments in the future. Breast Cancer Trials has pioneered the measurement of quality of life in patients and carried out this research for more than 40 years.
Observational Studies

Observational studies do not involve interventional treatment, and do not alter a patient’s treatments. In an observational study, researchers take note of information collected during the patient’s standard care and treatment, and from questionaries patients complete during the life of the study, about how they feel during treatment planning and care. The Breast MRI Evaluation study is an example of an observational study.

Within these different types of clinical trials, clinical trials can also be described by how they are conducted. A clinical trial can be described as more than one of the following terms. For example, a clinical trial can be a randomised controlled blind study.

What is a Randomised Control Trial?

Randomised controlled trials are the most rigorous way of determining the safety and efficacy of new treatments. They are necessary to get new treatment approved.
What is a Blind Study?

If a clinical trial is a blind study, it means the clinical trial participants and/or researchers do not know who is receiving the medical intervention or different treatment and who is not. A study may be double blind and/or single blind.

In a double-blind study, the patient and study coordinators do not know what treatment the patient has been given. This is advantages as knowing this information could influence the course and results of the study. These studies could be comparing a new treatment with an existing treatment, a treatment with a placebo or two treatments or doses.

A randomised double-blind placebo-controlled study is considered the ‘gold standard’ for clinical trials.
What is a Placebo Study?

A placebo can often be used in a blind study to help determine if a treatment or prevention strategy is effective. This is because by blindly allocating patients in a trial, investigators can ensure that any possible ‘placebo effect’ can be minimised.

The placebo is a pharmaceutically inert substance or dummy treatment, for example a sugar pull. Using a placebo in a clinical trial helps to prove a new intervention or treatment has a better outcome for patients.

Potential clinical trial participants are always told if a placebo is used in a trial before they agree to participate.
What is an Open-Label Study?

An open label study is a clinical trial in which both the patient and researcher know which drug treatment is being administered. Open-label studies may be used to compare treatments or gather additional information about the long-term effects in the intended patient population.

An open-label study or trial may be randomised and may also be uncontrolled (without a placebo), with all participants receiving the study drug or treatment.

WHAT IS A CLINICAL TRIAL?