Held by the Association of Clinical Research Professionals (ACRP) on the 20th of May 2019, Clinical Trials Day is an opportunity to recognise the important role that clinical trials play in improving public health. On this day in 1747, James Lind conducted what is now considered the first randomised clinical trial aboard the HMS Salisbury of Britain’s Royal Navy fleet. Acting on a hunch that scurvy was caused by a lack of acid in the body, he tested his theory systematically, finding that seamen who were given citrus fruits experienced ‘the most sudden and good visible effects’. Read on to learn about modern-day clinical trials and use #CTD2019 to share this on social media.

So what is a clinical trial exactly?

Clinical trials are research projects to find out whether a new treatment, procedure or test may work in particular disease populations.

Different types of trials can be

  • treatment studies – such as researching a new treatment for a disease
  • prevention trials – most commonly vaccine studies to test for effectiveness and side effects
  • diagnostic or screening trials – evaluation tests or procedures for the diagnosis and detection of diseases and condition
  • observational studies-observing disease outcomes in patients with a particular condition

Clinical trials are conducted in universities, hospitals, cancer centres and clinics (such as AusTrials).  In 2017 there were 1650 new studies registered with the Australian New Zealand Clinical Trials Registry (ANZCTR). This equates to an average of some 138 new trials per month.

Thinking about joining a clinical trial?

Clinical trials provide many opportunities such as

  • being able to trial new treatments not currently available on the market for your medical condition (for example a new treatment for diabetes if you are currently on maximal available therapies)
  • getting close monitoring, advice, care and support for your condition by a team of experienced doctors and nurses
  • playing a more active role in your health care by getting a better understanding of your condition
  • contributing to ground breaking medical research. This helps us to improve scientific knowledge and help our future generations with their health. Without the generosity of clinical trial participants new treatments would not become readily available for the general population.

A little bit about different types of trials

Phase I

These are usually the first trials to involve humans. They are usually small involving less than 50 people. The main purpose of these studies is to find out how safe a medication is, usually the highest dose that can be given without serious side effects. These are usually carried out in a hospital setting with close monitoring and facilities available in case of a serious effect. About 16% of studies in Australia are of this type.

Phase II

This usually involves more patients. The main purpose is to see if a particular treatment is going to be effective. If it looks like a treatment will be effective with minimal side effects the next step is usually a Phase III trial. About 17% of studies in Australia are of this type.

Phase III

The question here is usually whether a new treatment is just as good, if not better than currently available treatments/care. This is usually the last step before a new treatment is assessed by the US Food and Drug Administration (FDA) and the Therapeutic Goods Administration (TGA) in Australia for approval on the general market. About 39% of studies in Australia are of this type.

Phase IV

These are usually done after the treatment has been approved and is available on the market. These trials are usually looking at whether a treatment offers any additional benefits or long-term side effects. About 8% of studies in Australia are of this type.

Other types exist, such as pre-clinical studies (5%) and others (15%).

How does a clinical trial come about?

Researchers usually put together a plan of what they would like to research, what outcomes they are hoping for and how they would like it to be conducted.

This plan is known as the protocol.  The protocol is a document which details all the instructions for those conducting the study (the study doctors and nurses at the research sites).

For this document to be approved it must be submitted to an ethics review board.  The main roles of the ethics board (Bellberry) is to work out if this is an ethical treatment, ensure the safety of potential participants and decide whether the treatment being offered is as good, if not better than currently available treatments.

What happens in a clinical trial?

  1. Study staff explain the study, including the purpose of the study, what treatment is being trialled and what for, how long it is anticipated to go for and any expected side effects that may occur whilst taking the study medication.
  2. Once the study has been discussed with you in detail and you have had the opportunity to discuss any questions or concerns that you may have, both you and the study doctor sign a consent form. This is a document to say that both you and your doctor agree to participate in the study.
    It is important to be aware that even though you have signed the consent form you are free to withdraw from the study at any time without penalty or prejudice.
  3. Once you have signed the consent form, the doctor and nursing staff can start carrying out procedures for the study. This could include procedures such as a physical examination, collection of vital signs (such as blood pressure, height, weight) blood collection, ECGs (tracing of your heart), and urine testing. These vary depending on the requirements of each study as per the study protocol. The study staff will also make sure you meet the criteria for the particular study. This varies from study to study.
  4. Once study staff are happy that you meet all the criteria then you are randomly assigned to a treatment group. These groups include different doses, formulations and placebo. This is called a randomized controlled double-blind study. What this means is that neither you nor the study staff are aware of which treatment group you are in. The reason for this is that some patients and staff may attribute improvements or worsening of their condition and/or side effects to whether they have medication or placebo. This is called bias.
  5. Visits to the study site are usually at set times. Exceptions would be if you have an unexpected event or illness in which case you will be asked to see the study doctor sooner.
    At these visits you may need to have further procedures such as examinations or blood tests. This is to see if there have been any changes between visits. It is also to maintain the safety of participants during the study. Study staff will also ask if you have had any changes to your medical history or new medications (ie. started a new medication for blood pressure or had to be on antibiotics for an infection). It is important that we gather as much information as possible (even if it may seem unrelated) as this helps to provide more evidence for how effective the study treatment may or may not be.

How do I go about finding a clinical trial that might be suitable for me?

Some good resources include

At AusTrials we conduct a wide range of trials from healthy volunteer studies to specific medical conditions.

Most of our studies involve Phase III trials.  As we are starting new studies all the time you can ask to join our database, even if we don’t have what you are specifically looking for at the time. We regularly send out updates for up-coming studies.