Clinical Trials Help Medical Research

What is a clinical trial?

People can volunteer to participate in clinical trials. A clinical trial is used to evaluate the safety and effectiveness of a new procedure, medication or device to prevent, diagnose or treat a disease or disorder.

People with inherited retinal diseases may decide to take part in clinical trials. In 2014 three people participated in a proof of concept study for the bionic eye made by Bionic Vision Technologies in Melbourne. They were given implants to test the safety of the bionic eye and for the purpose of study in the laboratory. In 2018, four people commenced trialling a newer version of that bionic eye.
There are four different phases in a clinical trial. The time from a phase one to a phase four trial can take many years.

What are the different phases of a clinical trial (for inherited retinal disease treatment)?

Phase 1
These are the earliest trials in the life of a new drug, device or treatment. They are usually small trials, recruiting up to 30 patients. Phase one trials are conducted to determine the safety of a potential treatment. People recruited to phase one trials often have advanced eye disease. These trials must be completed first as safety is the most important issue to resolve before wider testing is undertaken.

Phase 2
This type of trial tests the potential new treatment in a larger number of volunteers to learn more about how the body responds to the treatment, the optimal dose of the treatment and how the treatment affects a certain eye condition. If the results of phase two trials show that a new treatment may be as good as or better than an existing treatment phase three will begin. Better determining the safety of the treatment is also a large component of phase two testing and sometimes phase one and phase two trials are run at the same time.

Phase 3
Treatments only move into a phase three clinical trial if the first two phases suggest that a substance is safe and might be useful in ways that patients regard as important. Phase three trials are usually much larger than phase one or two,sometimes involving hundreds or thousands of patients in many different environments. Phase three trials are often randomised. This means the researchers will separate participants into two groups; one group will get the new treatment and the other the standard treatment or a placebo (non-active) treatment.

Phase 4
Phase four trials are performed after a drug, device or other treatment has been shown to be effective and has been granted a licence. They are performed in order to understand more about the treatment. They further evaluate its safety and effectiveness in larger numbers of patients, subgroups of patients, and by comparing it to or combining it with other available treatments.

Are there clinical trials ongoing in Australia?

Globally, there are many clinical trials already at phase one and two stages for inherited retinal diseases, especially in the area of gene therapy. The aim of these trials is to determine the best safe dose to give to an individual. If these trials are successful, they may progress to phase three trials that will test the new therapy on larger numbers of people across many countries, potentially including Australia.

To participate in a trial a candidate must meet a set of ‘entry criteria’. In the case of inherited retinal diseases, candidates may have to have an anomaly in a specific gene. Therefore, the work of the Australian Inherited Retinal Disease Register & DNA Bank (AIRDR) is vitally important. Further information on the AIRDR can be found below.

How can I find out more information about clinical trials?

The Australian Government National Health and Medical Research Council are an excellent source of information about clinical trial. Their website provides up to date information about clinical trials. Clinical trials that are currently being conducted worldwide can be found on the database maintained by the US National Institutes of Health and can be searched by condition and trial location.

Clinical trials are extremely important. Doctors, other healthcare professionals and patients require evidence from clinical trials to know which treatments work and are safe. Some patients may benefit from participating in a clinical trial however, there are no guarantees that a study participant will receive any direct benefit from participation in a clinical trial.

By volunteering to participate in clinical trials, participants help advance our knowledge about medical interventions. Clinical trial participants play an important role in the approval of new drugs and devices for treating other patients.

What happens if I agree to enter a trial but then change my mind?

You are free to leave a trial at any time without having to explain why.


The Australian Inherited Retinal Disease Register and DNA Bank

The Australian Inherited Retinal Disease Register and DNA Bank was established as a national resource in 2009. It contains clinical and family history information of over 6,800 participants affected with an IRD. It also contains DNA collected from over 4,900 of those participants.

The results of genetic analysis of that DNA (which may include the specific genetic mutation causing a participant’s IRD) are also recorded in this resource, as they become available. As the molecular mechanisms leading to inherited retinal disease are better understood, novel therapies are being developed which will in time prevent loss of sight and improve visual function.

Ultimately, the AIRDR aims to identify the exact genetic profile of the estimated 12,000 Australians affected by an IRD. If you have a condition such as retinitis pigmentosa, Choroideremia, Leber congenital amaurosis, Usher syndrome, Stargardt disease, cone/rod dystrophy or others, you can get involved by contacting the AIRDR.

The AIRDR’s national register is essential for the progress of genetics research and the enablement of clinical trials. It also strengthens Australia’s position as a potential location for clinical trials.

The early establishment of an affected person’s genetic status is vital if they are to have the opportunity to take full advantage of these emerging therapies. The AIRDR is actively used by clinicians and researchers to identify participants who may be suitable for emerging gene-specific clinical trials, to improve our understanding and treatment of inherited retinal diseases, and to facilitate clinical counselling of patients.

To contact the AIRDR phone: (08) 6457 2866
The website is: