Clinical trials

Eppendorf pipettesClinical 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 Clinical Trials Information Sheet

mnd clinical trials IN AUSTRALIA

Clinical trials for MND and/or amyotrophic lateral sclerosis (ALS) are currently underway in Australia.
Clinical trial title Description Status Location MNDRIA investment
A Phase 1 single and multiple Dose escalation and pharmacokinetic Study of Cu(II)ATSM administered orally to patients With amyotrophic lateral Sclerosis/motor neuron disease
> more information
The first clinical trial of copper-ATSM (Cu(II)ATSM) as a potential treatment option for MND and ALS. Read a summary of the research that led to the trial Completed Sydney
More than $1.2 million to support 3 projects that aim to develop copper-ATSM as a potential therapeutic
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. Completed  Sydney
This clinical trial was partly supported by MNDRIA
BreatheMND-1 – 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
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. > more information
Recruiting Melbourne This clinical trial is partly supported by MNDRIA
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. Recruiting Sydney 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.  Recruitment completed Adelaide,
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.

FORTITUDE-ALS is currently enrolling participants in both the United States and Canada. Recruitment in Australia commenced July 2018
Actively 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.