News & FAQs
We are pleased to announce the Australian Parkinson’s Mission clinical trial (APM001) has now commenced, with eight sites across Australia (in NSW, VIC, SA, WA and QLD), opening for participant enrolment and recruitment beginning in the upcoming months. (Please note that some centres have already started screening and recruiting participants while other centres will begin in the near future).
Each site will continue enrolment and recruitment activities for 12 months from the initial start date.
We are busy finalising the necessary requirements in order to move to the first intake stage of the clinical trials, but wanted to give you an update on the progress of the APM to date.
With key partnerships finalised, the first patient visits are scheduled to begin in later in 2020 (once ethics approval is confirmed) and take place over a year. We are planning to have the clinical trial, located in hospitals and clinical centres in NSW, Victoria, South Australia and Western Australia.
Once critical organisational aspects of the trial are finalised, we will be alerting you closer to the intake period so you can discuss your suitability as a participant with one of the APM clinical trial site doctors. .
A novel feature for Trial 1, is that it will assess four repurposed medications against placebo, recruiting 300 patients nationwide. These medications each have different but compelling biochemical evidence for their potential to slow or stop long-term decline in Parkinson's. This trial design will allow the testing of various pathological pathways, such as inflammation and oxidative stress, within one study. The APM researchers will test the target engagement of these pathways, and evaluate disease progression.
The APM strives to transform our understanding of Parkinson’s, identify genetic targets to guide drug development in Australia, and generate the evidence for personalised treatments for patients with Parkinson’s based on each individual’s genetic information.
We are excited to announce that the University of Sydney will join the APM as a partner, joining the Garvan Institute of Medical Research, The Cure Parkinson’s Trust, Shake It Up Australia, Parkinson’s Australia and the Michael J. Fox Foundation. The University of Sydney is one of Australia's leading higher education and research universities and will bring critical expertise to the program.
Frequently asked questions
APM clinical trial enrolment and recruitment
- Male or female aged 25 to 80 (inclusive)
- Diagnosed with Parkinson’s Disease
- Stable on dopaminergic Parkinson’s disease treatment for at least four weeks.
- Participants will undergo whole genome sequencing and analysis to identify the genomic variations contributing to their disease and inform clinicians which patients are most likely to respond to each of the treatments being trialled.
- Receive one of the three experimental treatments for Parkinson’s Disease. These treatments are currently being used for the treatment of other diseases; they are therefore known as repurposed medicines. You will receive either one of the repurposed medicines or a placebo (a placebo is a medication with no active ingredients).
- Undertake a physical examination, measurement of vital signs (heart rate, temperature, and blood pressure), electrocardiogram (ECG) as well as your weight and height.
- Have standard blood tests (e.g. cell counts, blood chemistry, blood sugars, pregnancy test) plus provide a collection of blood, saliva and urine for biomarker and genomic research
- Complete a number of Parkinson’s disease assessments at each visit of the five visits.
- New South Wales – Sydney
- Brain and Mind Centre
- Macquarie University Hospital
- St Vincent’s Sydney
- Southern Neurology
- Victoria – Melbourne
- Western Australia – Perth
- South Australia - Adelaide
- Queensland – Gold Coast
There is no cost associated for the participants of the Australian Parkinson’s Mission. The costs of the trials have been funded through the Federal Government and philanthropic contributions.
Patients participating in the clinical trial will be reimbursed for travel related expenses as approved by a Human Research Ethics Committee.
The APM provides an opportunity for people to be involved in a variety of ways.
Registering your interest is your opportunity to contribute to the APM program.
Subscribe for APM Updates and we'll keep you informed as the program develops.
General clinical trial information
A clinical trial is a research investigation in which people volunteer to trial new treatments, interventions, drugs and tests to find better ways to prevent, screen for, diagnose or treat disease.
By the time a new drug reaches the clinical trial stage, it has already been extensively tested in a laboratory session for side effects and patients are carefully monitored by doctors and nurses during the trial period. However, when testing a new drug, one of the purposes of the trials is to deem whether there are any problems or side effects.
For the first clinical trial, the drugs being trialled for the Australian Parkinson’s Mission are not new drugs, but have been repurposed for this program.
APM therapeutics - repurposed drugs
A repurposed drug is a drug that has already been approved by the Therapeutic Goods Administration (TGA) – a division of the Australian Government’s Department of Health – for one disease. A repurposed drug means that same drug is used again in a clinical trial to see whether it can help people with another disease. Repurposed drugs have already been through clinical trials for another disease, and have been demonstrated as safe in healthy volunteers.
New drug development is a long and costly procedure. From the time a new compound is discovered, it can take upwards of a decade and billions of dollars before it is available on the market. By using drugs that already have passed rigorous safety and toxicology trials, the Australian Parkinson’s Mission aims to significantly cut the amount of time it takes for a potential treatment to move from the laboratory to clinical trials and, ultimately, to the patient.
Off-label drug use is a broad term that refers the unapproved use of an approved drug. Drugs that have been approved by the TGA have been approved for a specific disease or particular uses – any deviation from this is considered off-label use.
When medically appropriate, medical practitioners can, and do, prescribe medication for off-label use. However, just because a drug is safe for a person with one condition, safety cannot be assumed for a person with a different disease or health issue.
Given the chronic nature of Parkinson’s disease, a new drug must be tolerated over an extended period of time, most likely for life, and the safety, tolerability and efficacy of a drug can only be determined after extensive clinical trials.
It is essential these treatments are evaluated for safety in Parkinson’s and that you and your doctor have rigorous scientific evidence on which to base your treatment decisions. This will hopefully avoid potential harmful effects and adverse health outcomes.
APM research - genome and DNA sequencing
Our genome is the complete set of genetic information we inherit from our parents, encoded within 2 metres of DNA packed tightly into each of our cells as chromosomes. A human genome is approximately 6 billion bases, or letters of DNA code. Genomics is the study of the structure and function of the genome of an organism.
DNA sequencing is a laboratory technique used to determine the sequence of units or bases in a DNA molecule. Sequencing methods have changed over time; the machines used by Garvan’s Kinghorn Centre for Clinical Genomics use complex chemistry and high-resolution optics to determine the sequence.
The DNA sequence is a series of letters – As, Cs, Gs, and Ts – that represent the order of base pairs in a person’s DNA. The sequence of a single human genome has approximately 6 billion letters to read and interpret. In a sequencing laboratory, machines break the DNA up into manageable segments and read the order of the DNA bases or letters. Computers are then used to compare the DNA sequence with other sequences to locate the differences or variants.
A cohort is a group of people who share one or more important characteristics. Cohort studies usually focus on a group over time and help researchers learn about how a range of factors affect health and disease. In a genomic cohort, the genome of each individual is sequenced in order to compare it with others, both within in the cohort and beyond.