What Happens In Clinical Trial?

Centre for Eye Research Australia

The journey from an idea to a treatment in the clinic can be a long one, often taking years of testing, improvement and collaboration.

It requires the dedication and time of all those involved - including scientists, clinical staff and people with an eye disease that work with us to test and trial new ideas and technology.

It's these people, many of them who volunteer for trials, that help us work towards our mission to change the lives of people who are at risk of eye diseases like glaucoma - which is most associated with developing due to a build up of pressure in the eye.

"It can sometimes be daunting to be part of a clinical trial if you have never been part of one before, but our team is here to assist you every step of the way," says Dr Flora Hui.

Your contribution is what helps us advance our research to find new treatments to prevent vision loss.

Dr Hui co-leads the world-first international clinical trials with Professor Pete Williams into harnessing the potential of nicotinamide - a form of vitamin B3 - to power the cells in the eye and support the current treatments for eye pressure like eye drops and surgery.

"We have known for a long time now that eye pressure is not the final story in glaucoma," says Dr Hui.

People can develop glaucoma even when their eye pressure isn't high to begin with. Even when the eye pressure is lowered, up to a third of people continue to progress towards blindness.

"If lowering eye pressure cured glaucoma or prevented its progression then we would have nothing to research!"

Moving towards a treatment

"There has been a lot of research done to try to understand what other factors may play a role in pressure in the eye - including ageing, poor blood flow and energy supply to the retina," says Dr Hui.

This research includes initial work at CERA that suggested insufficient energy supply and production was a problem in glaucoma.

It showed that mitochondria (the energy source for nerve cells) are not working properly.

Then, in 2017 Professor Williams and his colleagues found that increasing vitamin B3 through a daily oral supplement would protect the pressure on the cells in the eyes of mice.

"This then brought about the first clinical trial we did at CERA which yielded positive results," says Dr Hui.

"This was followed by another positive result from two other independent clinical trials in the United States and South Korea. And now the longer trial is occurring, which is a collaboration between Australia, Singapore, Sweden and the United Kingdom."

What happens in a clinical trial?

For people in this current trial, all that is different to their normal routine is the addition of taking vitamin B3 tablets.

"Participants continue taking their eye pressure lowering treatments because vitamin B3 isn't targeted towards pressure lowering," says Dr Hui.

"We know pressure lowering is important, but our trial is focused on what else we can do on top of it to help people with glaucoma."

Using gold standard clinical measures for glaucoma, trial participants undertake tests for peripheral vision, as well as scans of the optic nerve to measure the thickness of the nerve tissue.

The results of these will enable confirmation of the effectiveness of vitamin B3 over time and are expected to be known in a few years.

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In our latest edition, we share how finding new ways to save a cell's life can prevent vision loss.

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