Clinical trials

Clinical trials test new treatments and interventions in people to find out if they are safe and/or effective. Clinical trials may test experimental drugs, cells and other biological products, vaccines, medical devices, surgical and other medical treatments and procedures, psychotherapeutic and behavioural therapies, preventive care strategies and educational interventions.

To read MND Australia's Position Statement on the development and approval of drugs to treat MND click here.

For an easy to understand overview of the different phases of clinical trials, and why this form of testing is important, take a look at the MND Association of England, Wales and Northern Ireland Information Site

mnd clinical trials IN AUSTRALIA

Clinical trials for MND and/or amyotrophic lateral sclerosis (ALS) are currently underway in Australia.
Clinical trial title
Status Location MNDRIA
Phase 2/3 Cu(II)ATSM: A phase 2/3 clinical trial of copper-ATSM (CuATSM) for the treatment of MND progression will soon begin in Australia. This trial will be administered through Macquarie University in Sydney. 
More information is available on our FAQ page. 
This trial follows on from the succesful completion of a phase 1 clinical trial, which identified a safe dosage. More information is available here Recruitment will soon begin.  Study being coordinated by Macquarie University. Please contact your neurologist for more information.  More than $1.2 million to support 3 projects that aim to develop copper-ATSM as a potential therapeutic
Environmental risk factors for MND: Scientists at Macquarie University are searching to find the causes of MND. As part of this research, data and biological samples are being sought for the Neurodegenerative Disease Biobank.  This study aims to screen for environmental risk factors and their potential association with MND in Australia, specifically in the agricultural Riverina Region. A number of groups are being sought from the region: patients with an MND diagnosis; people living in the same household as a person diagnosed with MND in the last ten years; or people who have lived in the region for at least ten years in the past, but have since moved away. All participants will be required to provide a urine sample and answer a questionnaire.  Active, recruiting patients and controls.  Study being coordinated by Macquarie University. 

Email Susan D'Silva or call 
(02) 9812 3741

More information
A survey of healthcare professionals on the measurement of physical functioning in ALS/MND and attitudes to the development of technology based measurement and monitoring solutions.  Emerging technologies provide opportunities for development of novel solutions for assessment and remote monitoring of physical functioning in people with ALS/MND. This study is looking at the perspectives of health professionals regarding the potential of new technologies to replace or augment current assessment methods. This study involves an online survey of healthcare professionals working with ALS/MND internationally.  Active, recruiting healthcare professionals.  An international study coordinated by Dr Deirdre Murray at Trinity College in Dublin. 

Click here for more information. 
DEALS (Diet and Exercise in ALS) Study - The effects of active exercise combined with an enriched diet on swallowing, speech function and weight in patients with MND; a randomised trial.
More information. 
This clinical trial is evaluating the effects of diet changes in patients with MND combined with active exercising on swallowing function, speech and weight status. Final stages of recruitment Sydney

Contact Professor Vicki Flood  if you would like to be involved in this trial. 
This clinical trial is partly supported by MNDRIA
EMERALD: A randomised, double-blind, single-centre Phase 3 study on the safety, tolerability and efficacy of Cannabis Based Medicine Extract (CannTrust CBD Oil) in slowing the disease progression in Amyotrophic Lateral Sclerosis or Motor Neurone Disease patients (EMERALD). 
More information
Participants will be assigned by chance to receive either medicinal cannabis or placebo. Treatment will continue for 180 days and participants will be seen every 3 months in the research clinic, with a monthly telephone call in between. See our FAQ page for more information. Active, recruiting patients.  Gold Coast University Hospital.

Click here for more information on eligibility and for contact information. 
South Western Sydney Local Health District (SWSLHD) Communication and Assistive Technology (CAT) Clinic Research­ Study         
Liverpool Hospital (Sydney) offers a state of the art Communication and Assistive Technology Clinic for those diagnosed with MND and living within the SWSLHD. 
More information. 
This project aims to identify if assessment and intervention within the Communication and Assistive Technology Clinic within 12 months of diagnosis improves quality of life, function and decreases carer burden. Participants will complete surveys and interviews in addition to standard care by specialised Occupational and Speech Pathologists. See our FAQ page for more information.  Active, recruiting patients. Liverpool Hospital, Sydney. 

See here for more information on eligibilityy and for contact information. 
Lung volume recruitment in neuromuscular disease: Can ‘breath-stacking’ improve lung function, respiratory symptoms and quality of life for people with neuromuscular disease?
> more information
This clinical trial is comparing breath-stacking breathing exercises to
diaphragmatic breathing exercises in people with neuromuscular disease including people with MND to see which treatment is more effective. See our FAQ page for more information. 
Active, recruiting patients. Melbourne This clinical trial is partly supported by MNDRIA
Effects of Oral levosimendan (ODM-109) on Respiratory Function in Patients with ALS. 

more information
The primary objective of this Phase 3 study is to confirm that levosimendan can significantly improve respiratory function measured by supine slow vital capacity (SVC) in amyotrophic lateral sclerosis (ALS) patients.
The secondary objective is to confirm that levosimendan improves the functionality of subjects, using sleep scales to assess daytime somnolence and sleep quality. In addition, the long-term tolerability and safety of levosimendan in ALS patients will be evaluated, assessing up to 48 weeks of exposure. See our FAQ page for more information. 
Active, recruiting patients. Sydney, Brisbane, Adelaide, Melbourne, Perth. 

Contact your MND clinic or neurologist for more information on participating in this trial. 
Phase 2 Triumeq Complete: 
Safety and tolerability of antiretroviral (Triumeq) in patients with amyotrophic lateral sclerosis (ALS) (The Lighthouse Project)
> more information
The Lighthouse Project is a trial to test an antiretroviral therapy called Triumeq, following research which suggests that a virus may be a cause or trigger for ALS. See our Grants Awarded in 2019 page for information on the upcoming Phase 3 trial of Triumeq.  Phase 2 trial complete.  Sydney, Melbourne This clinical trial is partly supported by MNDRIA
A prospective study to assess the causes and treatment of sleep disordered breathing and respiratory failure in patients with MND.
More information. 
This clinical trial is investigating patterns of abnormal breathing and tailoring targeted treatment to improve breathing, quality of life, sleep and survival in patients with MND. This includes assessing the effect of two different settings of non-invasive ventilation, respiratory support delivered by a mask and machine, by using a new “smart” ventilator. Active, not recruiting Adelaide This clinical trial is partly supported by MNDRIA
TEALS (Tecfidera in amyotrophic lateral sclerosis) Study:
A multi-centre Phase 2 clinical trial to investigate the safety, tolerability and effectiveness of Tecfidera in people with MND. More information. 
This clinical trial will assess if Tecfidera is effective in slowing down disease progression and whether it is safe in MND patients. See our FAQ page for more information.  Recruitment completed Adelaide, Brisbane, Melbourne,
Sydney, Perth
FORTITUDE-ALS is a phase 2 clinical trial which is designed to evaluate whether reldesemtiv (also known as CK-2127107) has an effect on participants with ALS/MND and what kind of side effects may occur.
More information. 
Reldesemtiv is an investigational medicine that is being evaluated as a potential new medicine which could slow the decline of skeletal muscle function in diseases and conditions associated with muscle weakness or fatigue such as ALS/MND. May 2019 Phase 2 results now availailble here Active, not recruiting.  Sydney, Brisbane, Adelaide, Perth.  


There are two drugs being investigated in Australia that look promising in animal studies. They are not ready to be tested in humans.

Following an announcement by Queensland Premier Annastacia Palaszczuk in June 2016, Brisbane-based Patheon Biologics is manufacturing a protein, which will undergo laboratory testing to see if it is suitable for MND clinical trials. A Queensland research team, led by Professor Perry Bartlett from the Queensland Brain Institute and Professor Andrew Boyd from the QIMR Berghofer Medical Research Institute, has found that a protein called EphA4 plays a role in repairing damaged motor neurones. EphA4 appears to impede the recovery of motor neurones following injury. This discovery has led to research to develop an EphA4 inhibitor, which could be effective in treating MND in humans. Preclinical research is underway to better understand the inhibitor before a first-in-human study can begin. The researchers hope to begin a clinical trial in 2019.
Associate Professor Trent Woodruff and colleagues based at The University of Queensland are working on a novel drug “PMX205”, which dampens down inflammation in MND. To date, PMX205 has been tested in rats and mice. PMX205 extends survival, slows disease progression and significantly increases the muscle strength of mice with a SOD1 mutation. Formal preclinical safety and toxicity studies need to be successfully completed before the drug can be tested in humans. This research was partly supported by MNDRIA. For more details of PMX205 research to date click here.


There are many research projects around Australia which are supported by the MNDRIA and involve participation of people with MND. They are not clinical trials. Participation in such studies is generally managed by specialist MND clinics in association with different research institutes. These specialised clinics, listed here, are best placed to link patients to research projects. If you would like to know more, talk to your neurologist about what research opportunities are available in your area.
For more information click here.


There are a number of online databases, or registries, that list clinical trials from across the globe. Information on the trial status, trial design, active sites and key recruitment criteria for each trial can be found. Trials listed may be currently recruiting, have been completed or are pending. Trials may have recruitment sites in multiple countries. Trials are commonly listed on multiple databases.
The databases mentioned below should provide the most up-to-date information on clinical trials. When searching for a trial, you can refine your search results to relevant trials by choosing the Advanced Search option. Enter ‘motor neuron disease’ AND/OR ‘amyotrophic lateral sclerosis’ in the search terms; select ‘Recruiting’ for recruiting status to see trials that are accepting currently patients; and select the correct location, such as ‘Australia’ or ‘Outside the US’ to see accessible sites.
If you want to know whether you may be eligible for a current trial, print off the trial information and talk to your neurologist or another health professional involved in your care such as your general practitioner (GP).

Australian clinical trial databases

Australian Clinical Trials is the database of the National Health and Medical Research Council (NHMRC) of Australia and includes MND studies. It lists all trials with a recruitment site in Australia that are registered on the Australian New Zealand Clinical Trials Registry (ANZCTR) as well as, the database of the United States (US) National Institute of Health (NIH).
Australian Clinical Trials also provides information on how to be part of a clinical trial, explains informed consent and describes the different phases of clinical trials.

International clinical trial databases

The ALS Therapy Development Institute is a research institute in Cambridge, Massachusetts, USA. The institute’s website lists clinical trials specifically for amyotrophic lateral sclerosis (ALS) in North America, South America, Europe, Asia and Australia.

The International Clinical Trials Registry Platform is the World Health Organisation's search portal for clinical trials. The clinical trials listed in the database are collected from a range of sources, including the Australian New Zealand Clinical Trials Registry listed above. is the United States (US) National Institutes of Health's (NIH) database of private and public clinical trials conducted in the US and elsewhere around the world.


BrainStorm’s NurOwn
NurOwn and the treatment approach were developed by BrainStorm Cell Therapeutics, a biotechnology company based in New York. The company research and develop autologous stem cell therapies for debilitating neurodegenerative diseases, including motor neurone disease (MND), Multiple Sclerosis (MS) and Parkinson’s Disease (PD). At this stage, evidence suggests the NurOwn treatment is safe, and has potential benefits for slowing the progression of MND – although the clinical data is currently insufficient to know with certainty. Phase 3 of the trial is currently underway and results will hopefully become available in 2020. Read more about NurOwn on our FAQ page.