The APM will be available for all people with Parkinson's -- those who have been newly diagnosed, and those have been living with the disease.
- Patient recruitment will begin mid 2019 in Qld, Vic and NSW, and then roll out to other states.
- We will begin with capital city centres and extend to regional Australia where possible.
Please fill in our form to register your interest, or you can email us on info@TheAPM.org.au and we'll add you to our mailing list. We'll keep you informed on key dates and 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.
The Australian Parkinson’s Mission will involve many hundreds of people with Parkinson’s, each of whom will get their genome – their entire DNA – sequenced to determine the Parkinson’s disease subtype that they have.
The first clinical trial is a Phase II study which aims to establish the efficacy of four investigational products individually in slowing progression of PD and maintaining this effect even after the investigational product is discontinued for 12 weeks to eliminate the possibility of an unexpected symptomatic response rather than disease modifying benefit.
Initially, 300 people with moderate severity Parkinson’s , on a stable dose of usual Parkinson’s medication [L-dopa, dopamine agonists, catechol-O-methyl transferase (COMT) inhibitors monoamine oxidase (MAO) inhibitors and/or amantadine], will be randomized to one of the four investigational products or placebo.
Participants will be evaluated for safety and tolerability following initiation of the treatment with the investigational product or placebo. Efficacy as well as safety measures will be evaluated, and scheduled assessments will be conducted at 12, 30 and 48 weeks of treatment with the investigational product for comparison with those scores obtained at baseline.
Investigational product treatment will cease at 48 weeks where the final assessments will be made to determine the effect while on the investigational product. An additional assessment will occur at week 60 (following a 12-week washout of investigational product) where all evaluations will be repeated to determine if the efficacy has been maintained after the investigational product has been withdrawn.
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.
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.
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.
The APM website will be updated with clinical trial details including when recruitment will start, which sites across Australia have been chosen to participate and how to become involved.