What are clinical trials?
What comes first?
In order to explain clinical trials, we first have to go back to a time before these studies have started. These are called pre-clinical trials.
When scientists discover a promising new drug, device, or treatment (investigational product or IP), they first test it in a laboratory – in vitro testing. This involves testing on isolated, specific cells, like testing an asthma treatment on lung cells in a petri dish.
While this can provide useful data, there are limits to what scientists can learn from this stage. A treatment that works in a petri dish won't necessarily work in living beings!
That’s why the next stage is in vivo testing in living subjects such as animals. This is necessary to gather the data that we need in order to proceed to testing the IP in humans – or what we call clinical trials.
Phase 1, 2, 3, 4 – the clinical trial stages & goals
There are 4 stages of clinical trials, and at Nucleus Network we specialise in phase 1.
Phase 1 clinical trials
We give the IP to a small group of healthy volunteers (on average between 20-100 people). The goal at this stage is to determine the optimal dose for future studies. This is the lowest dose that will produce the desired effect with the smallest chance of adverse events. We want to understand how the human body affects the IP (pharmacokinetics) and in some cases how the IP affects the human body (pharmacodynamics).
We usually start with a very small dose of IP and then work our way up. At each dosage level, we study how much of the drug is absorbed by the body and what effect it has. We’ll also collect safety data, to determine the dose that is most likely to be effective and safe.
Each dosage level is called a cohort, and they are often split into Sentinel & Rest of Cohort (ROC). The sentinel participants will receive the IP (or a placebo) first. Only after their initial results are reviewed do we start dosing the ROC. If there is a safety concern, this limits the number of people who are exposed. You can read more information about our safety measures & track record here.
Phase 2 & 3 clinical trials
During these studies, the IP is given to patients to understand the efficacy of the potential treatment. It will also be compared to existing treatments. It’s not enough to know that the treatment works – it needs to have an advantage over existing treatments. This could mean that it has higher efficacy, it doesn’t need to be administered as often, it has fewer side effects, ...
Testing the efficacy of an IP means that we look at how well the treatment works under ideal and controlled circumstances.
Phase 4 clinical trials
These take place after the new treatment has been approved, and monitor effectiveness in the general population. This might be different from efficacy - a treatment may not work as well when it is more widely administered than during a rigorously controlled clinical trial.
There is also continued monitoring of adverse effects.
Why are these clinical trials so important?
We cannot overstate how vital these trials are. Without clinical trials, no new medical treatments can be developed.
Any medication that you or a loved one require for a medical condition will have gone through a clinical trial at some point.
At Nucleus Network we work on dozens of clinical trials each year, for a wide variety of medical conditions. Psoriasis, breast cancer, and type 2 diabetes are just a few of the medical conditions for which we have helped to develop new treatments.
By participating in a clinical trial today, you play a vital role in the development of the medication that will save or improve people’s lives in the future.
Why are you looking for healthy volunteers?
For the phase 1 trials that we specialise in, we often only allow healthy volunteers. That means people with no medical conditions who are not taking any prescription medication. There are two reasons for this:
- It’s safer: we do everything we can to make our studies as safe as possible, but this is still an investigational product that is not yet fully understood. Maybe it temporarily increases blood pressure. Not an issue for a healthy person, but potentially dangerous for someone who already had elevated blood pressure
- It provides better data: what if a participant has a temporary increase in blood pressure, but they are also taking other medication that can have this effect? Now we don’t know whether it was the IP or the approved medication that caused it. Or the IP could cause a rash, which might not be visible on someone who already has a skin condition.
We have some studies that allow for participants with specific medical conditions if they are well-managed on a stable dose of medication. E.g., if someone has been taking the dose of the same antidepressant for over 6 months, they may be eligible to participate in a clinical trial of a new vaccine.
Isn’t this unfair to people who are taking medication/have medical conditions?
It’s important to remember that the phase 1 studies we do are only the first step towards an IP getting approved. Phase 2 & 3 studies will include different groups of participants, who are taking medication or have other medical conditions. These studies will build on the results of our phase 1 trials, and all contribute to one common goal – advancing medicine & improving lives!
If you have a medical condition and are looking for a trial to participate in, you can find studies here: https://www.anzctr.org.au/
More information
You can find more information about the clinical trial process here:
- TGA: clinical trials in Australia
- FDA: the drug development process
- Australian Clinical Trials: phases of clinical trials
- Cancer.org: types & phases of clinical trials
We are always looking for healthy volunteers to participate in our clinical trials. If you are interested, give us a call on 1800 243 733!