Clinical Trials Day is observed annually on 20 May and commemorates the day James Lind started what is often considered the first randomised clinical trial aboard a ship in 1747. Lind’s experiment involved sailors who had scurvy, a disease caused by vitamin C deficiency. He tested various remedies to determine which was most effective in treating it.
This day now serves to recognise the importance of clinical trials in medical research and healthcare advancement. It highlights the contributions of researchers, healthcare professionals and study participants to the development of new treatments and medical interventions.
Clinical trials are essential for evaluating the safety and effectiveness of new medical interventions and improving patient care. The efforts of these individuals in conducting groundbreaking research and their commitment to finding new and innovative treatments are greatly appreciated.
What are clinical trials?
Clinical trials are the essential final step in introducing new medical interventions to the clinic, and they provide hope for millions of people living with chronic illnesses, including MS. Conducted in four separate phases, they’re rigorously monitored and regulated:
- Phase 1 assesses the safety and tolerability (how manageable any side effects are) of an intervention in a small group of healthy volunteers, determining the right dosage and spotting side effects.
- Phase 2 tests the effectiveness in a larger group of people with the disease, gathering initial data on effectiveness and side effects.
- Phase 3 confirms the effectiveness in a larger group with the disease, providing more definitive data and identifying rare side effects.
- Phase 4 focuses on post-approval surveillance, monitoring the intervention’s long-term safety and effectiveness in a larger population.
The number of people needed for each phase can vary depending on the treatment being tested. Clinical trials are important for people with MS because they help determine if new drugs or other interventions are safe and effective.
Participating in clinical research may provide early access to new treatments and improved quality of life for the individual, but it can also benefit many others in the MS community.
PLATYPUS – an adaptive clinical trial for progressive MS
The treatment of progressive MS represents a critical unmet need in the MS landscape. To address this challenge, MS Australia has collaborated with a national group of clinicians and researchers to create a groundbreaking clinical trial platform known as PLATYPUS (PLatform Adaptive Trial for remYelination and neuroProtection in mUltiple Sclerosis).
PLATYPUS, an innovative clinical trial to be conducted in Australia, seeks to assess the potential of repurposed drugs (existing medications for other diseases) in repairing myelin and/or protecting the nerves. Its goal is to slow the progression of disability in individuals with progressive MS.
This adaptive clinical trial will enable the simultaneous evaluation of several potential therapies and will allow interim analyses to gauge drug effectiveness as the trial progresses, rather than waiting till the end, as is the case for traditional clinical trials.
For example, it will allow treatment arms to be dropped if they do not look promising, and new treatment arms to be incorporated throughout the trial. Also, as PLATYPUS will be testing drugs that have been approved for other indications, phase 1 can be skipped.
Together, this means that PLATYPUS will accelerate results compared to traditional clinical trials.
PLATYPUS will be Australia’s first adaptive clinical trial for MS, part of the OCTOPUS clinical trial currently underway in the UK. It will allow us to leverage large-scale and rapid UK recruitment in OCTOPUS to achieve excellent statistical power.
OCTOPUS launched in April 2023, and the Australian planning and rollout are underway, with recruitment expected to begin later in 2024.
PLATYPUS represents a significant leap forward in MS research, offering hope for improved treatments and better outcomes for people living with progressive MS.
If you are interested in being part of PLATYPUS, you can register your interest here.
Clinical Trials Network
MS Australia hosts the Clinical Trials Network website that lists clinical trials and research studies for MS recruiting in Australia and New Zealand, encouraging high-quality clinical MS research and increasing awareness of current trials and research for people with MS.
The clinical trials and research studies underway in Australia and New Zealand for MS are at various stages, with some focused on the safety and effectiveness of new drugs while others are exploring additional interventions, including online treatments for wellbeing and changes in lifestyle.
Visit the MS Australia Clinical Trials Network to find out more about the clinical trials and research studies currently recruiting in Australia and New Zealand.
If you would like to learn more about clinical trials in general, MS Australia has developed an educational series for the community exploring different aspects of clinical trials, called Trial.Smart, which you can take part in.
Gratitude to those involved in clinical trials
Clinical trials are a crucial part of the medical research process, providing the scientific evidence necessary to develop new treatments and interventions that can improve the lives of millions of people.
We give thanks to the MS researchers and volunteers who participate in clinical trials, and we celebrate their contribution to advancing medical knowledge and improving the lives of people living with MS around the world.
