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

Involvement in a clinical trial
If you are considering involvement in a clinical trial, you will first need to speak with your neurologist to assess your eligibility. 

Scientific Advisory Council
MND Australia is a member of the International Alliance of ALS/MND Association. One of the resources of this Alliance is the Scientific Advisory Council (SAC). Click here to read updates and briefing notes from the SAC.

Clinical trials around the world
For a comprehensive list of clinical trials currently underway around the world, please see this great resource hosted by IAMALS.

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 MNDRA

MetFlex: A Phase 2a trial to determine the safety and tolerability of trimetazidine in ALS.

Official clinical trial information is here

The MetFlex trial will be conducted in a controlled setting across three hospitals globally. Our primary goal is to assess the safety and tolerability of Trimetazidine in patients with ALS. Second to this, we will explore if Trimetazidine can reduce hypermetabolism in patients with ALS. This open-label trial (no placebo) is open to patients with familial or sporadic ALS. Enrolment into the trial is dependent on participants meeting inclusion criteria.

See our FAQ page here
Active, currently recruiting patients Brisbane Project being undertaken by a number of MNDRA funded researchers
CHAMPION ALS Study: Phase 3 evaluate the efficacy and safety of ravulizumab.

Official clinical trial information is here
The CHAMPION ALS Study is a Phase III, double-blind, randomised, placebo-controlled, parallel-group, multicentre study with an open-label extension period, to evaluate the efficacy and safety of ravulizumab (also known as Ultomiris) in adult patients with amyotrophic lateral sclerosis (ALS). The study is sponsored by Alexion Pharmaceuticals.

See our FAQ page here
Active, currently recruiting patients.  Trial sites are planned in Sydney, Brisbane and Perth.

The trial is currently open for recruitment. 
Experiences of genetic testing Participants are being invited to be interviewed by telephone, video conference, email or online text chat/instant messaging if:

- You have MND and have been offered genetic testing for MND genes OR;

- You are a family member of a person with MND who was offered genetic testing for MND genes. 

See our FAQ page for more information
Active, currently recruiting patients and family members of those with MND.  Interview is conducted by telephone, video conference, email or online text chat/instant messaging. Project being undertaken by a researcher who has been previously funded by MNDRA
Phase 2/3 Cu(II)ATSM. 
Official clinical trial information is here
A phase 2/3 clinical trial of copper-ATSM (CuATSM) for the treatment of MND progression is currently recruiting in Australia. This trial will be administered through Macquarie University in Sydney. This trial follows on from the successful completion of a phase 1 clinical trial, which identified a safe dosage.

More information is available on our FAQ page. 
Active, currently recruiting patients.  Study being coordinated by Macquarie University. Please contact your neurologist for more information.  More than $1.2 million to support 3 projects that helped develop copper-ATSM as a potential therapeutic
Environmental risk factors for MND: Macquarie University Neurodegenerative Disease Biobank. 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, currently recruiting patients.  Study being coordinated by Macquarie University. 

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

More information
EMERALD: Phase 3 study on the safety, tolerability and efficacy of Cannabis Based Medicine Extract.

Official clinical trial information is here
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). 
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, currently 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         

More information. 
Liverpool Hospital (Sydney) offers a state of the art Communication and Assistive Technology Clinic for those diagnosed with MND and living within the SWSLHD. 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 eligibilitand for contact information. 
REFALS:Effects of Oral levosimendan (ODM-109) on Respiratory Function in Patients with ALS. 

Official clinical trial information is here

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, currently not recruiting. Sydney, Brisbane, Adelaide, Melbourne, Perth. 

Phase 2 Triumeq - Complete: 
Safety and tolerability of antiretroviral (Triumeq) in patients with amyotrophic lateral sclerosis (ALS) (The Lighthouse Project).

Official clinical trial information is here

The Lighthouse Project was a trial to test an antiretroviral therapy called Triumeq, following research suggesting that a virus may be a cause or trigger for ALS. The phase 2 trial showed positive results and a Phase 3 trial has now been planned. The phase 3 trial will be conducted in Europe, the U.K. and at five sites in Australia. It is hoped this trial will begin mid-late  2021, with trial sites soon to be announced.  Phase 2 trial complete. Phase 3 trial has not yet commenced  Australian sites for Phase 3 trial to be finalised Both the Phase 2 and 3 trials have been partly supported by MNDRA
TEALS (Tecfidera in amyotrophic lateral sclerosis) Study:
Phase 2 clinical trial to investigate the safety, tolerability and effectiveness of Tecfidera.

Official clinical trial information is here
This Phase 2 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.

For further information on the trial design see here.
Recruitment completed.

Data currently being analysed
Adelaide, Brisbane, Melbourne,
Sydney, Perth
VALOR: This 3-part study will examine the efficacy, safety, tolerability, PK and PD of BIIB067 (Tofersen).

Official clinical trial information is here
Tofersen (BIIB067)is an antisense therapeutic specifically targeted at MND patients carrying the disease-causing SOD1 mutation.
This is a 3-part study – Parts A, B and C.
The primary objective of Parts A and B of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of Tofersen in adults with MND. Parts A and B act as Phases 1 and 2 of the clinical trial. Part A is the single ascending dose (SAD) component of the study, and Part B is the multiple ascending dose (MAD) component of the study.
The primary objective of Part C, the fixed dose component of the study of this study, is to evaluate the clinical efficacy of Tofersen administered to adult participants with ALS and confirmed superoxide dismutase 1 (SOD1) mutation. This is equivalent to a Phase 3 study.
Parts A and B were completed on 15-Jan-2019. In total, the study is estimated to enroll 183 participants, with 99 in Part C which will constitute the Phase 3 component of the trial.

For more information please see our FAQ page.
Currently recruiting Sydney  
Phase 2 study of the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8.

Official clinical trial information is here.
This is a Phase 2 trial.
CNM-Au8 is an oral, gold nanocrystal liquid suspension designed to assist cells with the energy they need to function well. It works by supporting bioenergetic cellular reactions, and helping to remove the toxic byproducts of cellular metabolism that add to the breakdown of motor neurons in ALS.

For more information please see our FAQ page.
Active. Currently recruiting. Sydney  
ALS-205: Phase 1 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses of ALS-205 in healthy volunteers.

Official clinical trial information is here.
This Phase 1 trial will test the safety of ALS-205 (also called PMX205), which has been designed to interfere with how the body’s immune system attacks the brain. The purpose of this trial is to determine if ALS-205 is safe, if it affects the body as expected, and how much is in the blood after dosing.  

For more information please see our FAQ page.
Provisionally registered. Not yet commenced or recruiting. Brisbane MNDRA have supported Trent Woodruff in developing this potential treatment.


There are many research projects around Australia which are supported by MNDRA 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. 

Proud member of:

International Alliance of ALS/MND Associations

ACNC Registered Charity


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