Clinical Trials

Clinical trials test new ways to prevent, find, treat, or manage health conditions. They help improve care for everyone.

Trials may study things like:

• New medicines or vaccines
• Surgical techniques or medical devices
• Diets or lifestyle changes
• Education programs
• Mental health or behaviour support

Even if a trial doesn’t help one person directly, it can lead to better treatments for others in the future.

Some trials are run by companies that create new drugs or medical devices. These are called Commercially Sponsored Trials. The company—called the sponsor—must be based in Australia, even if the product was developed overseas. The sponsor pays the hospital to run the trial.

Other trials are started by doctors, nurses, or medical researchers. These are called Investigator-Initiated Trials (IITs). They’re usually funded by research grants, medical organisations, or the hospital itself.

Every clinical trial is led by a doctor called the Principal Investigator. They’re supported by a team that can include:

• Doctors and nurses
• Pharmacists and medical scientists
• Imaging and other hospital staff (depending on the trial)

Behind the scenes, there’s a whole support crew—people who handle training, data, legal advice, quality checks, and more. It takes a big team to run a safe and successful trial!

Clinical trials can be different phases, each with specific goals

1. Phase One – Determines if the intervention is safe in humans. This phase involves a small group of participants and focuses on identifying side effects and appropriate dosage levels.

2. Phase Two – Assesses whether the intervention is effective while continuing to evaluate safety. A larger group of participants is involved to further understand the treatment's impact.

3. Phase Three – Confirms the usefulness of the intervention for a wider population. This phase often includes comparisons to existing treatments or placebos and involves hundreds or even thousands of participants.

4. Phase Four – Monitors the intervention post-approval in real-world use. This phase ensures long-term safety and effectiveness once the treatment is available to the general public

Being part of a clinical trial means your child could help improve care for others—and maybe even benefit themselves.

Clinical trials are how we discover better treatments, tests, and ways to care for children and young people with health conditions. Some trials test new medicines or therapies. Others focus on improving quality of life for children and young people with long-term illnesses.

People join trials for different reasons:

• Healthy volunteers want to help others and support medical progress.
• Children with a condition may get access to new treatments and extra care from the trial team.

Clinical trials offer hope—and a chance to make a real difference for the future.

Yes. All clinical trials in Australia must follow strict laws and ethical guidelines to protect participants and ensure the research is done properly.

Before a trial can begin, it must be reviewed and approved by a registered Human Research Ethics Committee (HREC). This ensures the trial meets national standards, including:

• The National Statement on Ethical Conduct in Human Research (by the NHMRC)
• The National Clinical Trials Governance Framework (by the Australian Commission on Safety and Quality in Health Care)

These rules make sure trials are safe, fair, and focused on doing what’s best for patients.

Clinical trials can offer real benefits—but they also come with some risks.

Before joining, you and your child will be given all the information you need to make an informed choice.

✅ Possible Benefits:

• Access to new treatments not yet available to the public
• Care from expert doctors and researchers
• Closer monitoring and personalised care
• A chance to help improve treatments for others in the future

⚠️ Possible Risks:

• The treatment might not work for your child
• There could be side effects—some may be serious
• Extra tests, hospital visits, or time may be needed

In the past, most clinical trials didn’t include children. That means we still don’t know enough about how some medicines, devices, or tests work for children and young people.

Now, more trials are designed just for children and young people—to make sure they get treatments that are safe, effective, and made with their needs in mind.

It depends on the trial. Each clinical trial has specific criteria about who can take part. These are based on what the researchers are studying and are approved by an ethics committee.

Your child may be eligible based on things like:

• Age
• Type or stage of illness
• Medical history
• Current health
• Past treatments
• Other personal factors

Before joining, your child may need some tests to check if the trial is a good fit. These tests also help create a “baseline” to track how your child responds during the trial.

Want to find a clinical trial for your child? Here’s how:

• 🔍 Search online: Visit the Australian Clinical Trials website to look up trials by condition or keyword.
• 🎥 Watch a quick video: Learn how to search for trials with this easy tutorial.
• 🩺 Talk to your child’s doctor: They can help you find trials that might be a good fit.
• 🧬 Check out MCRI: Visit the Murdoch Children’s Research Institute  Research Theme website to see what research is happening and who to contact.

👉 Click here to start searching for clinical trials in Australia

You can take your child out of a clinical trial at any time.
You don’t have to give a reason, and it won’t affect your child’s care or your relationship with their doctor.

If you decide to leave, talk to the research team. They’ll explain what happens next and what will be done with any information already collected about your child.