MND Australia

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 the following: 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.

The UK's MND Association provides a good overview of the different phases of clinical trials, and why this form of testing is important.

MND Clinical Trials in Australia

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

Phase 1: Complete. Planning for Phase 2 currently underway. 
Location: TBA

A Phase 1 study has been completed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses of ALS-205 in healthy volunteers.

This Phase 1 trial evaluated 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 was to determine if ALS-205 is safe, if it affects the body as expected, and how much is in the blood after dosing.

MNDRA have supported Professor Trent Woodruff in developing this potential treatment.

Learn more about this trial

Official clinical trial information is available at the Australia New Zealand Clinical Trials Registry

Phase 3: Complete. Trial has been discontinued due to lack of efficacy. 

The CHAMPION ALS Study was 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 was sponsored by Alexion Pharmaceuticals.

Learn more about this trial

Official clinical trial information is available on clinicaltrials.gov

Phase 2/3: Complete, results not yet available. 
Location: This study is being coordinated by Macquarie University, Sydney. 

A phase 2/3 clinical trial to evaluate the use of copper-ATSM (CuATSM) for the treatment of MND progression. This trial was administered through Macquarie University in Sydney. This trial followed from the successful completion of a phase 1 clinical trial, which identified a safe dosage.

MNDRA has provided more than $1.2 million to support three projects that helped develop copper-ATSM as a potential therapeutic.

Learn more about this trial

Official clinical trial information is available on clinicaltrials.gov
 

Phase 3: Active, currently recruiting patients

Location: Trial sites in Sydney, Brisbane and Perth

A Phase 3 study to evaluate the efficacy and safety of Reldesemtiv. Reldesemtiv is a fast skeletal muscle troponin activator designed to increase the muscles’ response to weak nerve signals. This is expected to slow the progressive muscle weakness seen in ALS and other neuromuscular diseases. The drug has been developed by Cytokinetics.

Learn more about this trial

Official clinical trial information is available on clinicaltrials.gov

Phase 3: Active, currently recruiting patients.
Location: Gold Coast University Hospital, Queensland.

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.

Learn more about this trial

Official clinical trial information is available at the Australia New Zealand Clinical Trials Registry

Phase 1b/2a: Active, currently recruiting patients. 

Location: Perth, WA

A phase 1b/2a clinical trial of WVE-004 as an investigational treatment for C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and frontotemporal dementia (C9-FTD)

Learn more about this trial

Official clinical trial information is available on clinicaltrials.gov

Phase 2 trial complete. Phase 3 trial has not yet commenced.
Location: Australian sites for Phase 3 trial to be finalised.

Safety and tolerability of antiretroviral (Triumeq) in patients with amyotrophic lateral sclerosis (ALS) (The Lighthouse Project).

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 in 2022, with trial sites soon to be announced. 

Both the Phase 2 and 3 trials have been partly supported by MNDRA.

Learn more about this trial

Official clinical trial information is available at the Australia New Zealand Clinical Trials Registry 

Phase 2: Active, recruiting patients. 

Location: Sydney, Central Coast (NSW), Brisbane, Gold Coast (QLD), Melbourne. 

MERIDIAN is a Phase 2 clinical trial evaluating the effectiveness and safety of the investigational medication, pegcetacoplan, in adults with sporadic MND.

LEARN MORE ABOUT THIS TRIAL.

Official clinical trial information is available on clinicaltrials.gov. 

 

Phase 2a: Active, currently recruiting patients
Location: Brisbane

A Phase 2a trial to determine the safety and tolerability of (Metflex) trimetazidine in ALS.

The MetFlex trial will be conducted in a controlled setting across three hospitals globally. The primary goal is to assess the safety and tolerability of Trimetazidine in patients with ALS. Second to this, the trial 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.

This trial is being conducted by a number of MNDRA funded researchers.

Learn more about this trial

Official clinical trial information is available on clinicaltrials.gov

Phase 3: Phase 3 trial now complete.
Location: Sydney, Brisbane, Adelaide, Melbourne, Perth.

REFALS was a phase 3 study to assess the effects of Oral levosimendan (ODM-109) on Respiratory Function in Patients with ALS. The primary objective of this Phase 3 study was 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 was 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 was to be evaluated, assessing up to 48 weeks of exposure.

Learn more about this trial

Official clinical trial information is available at the Australia New Zealand Clinical Trials Registry 

Phase 2: Phase 2 completed in November 2021. No information currently available on a potential Phase 3 trial. 
Location: Sydney

A Phase 2 study that was designed to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8. 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 bioenergetics cellular reactions, and helping to remove the toxic by-products of cellular metabolism that add to the breakdown of motor neurons in ALS.

Learn more about this trial

Official clinical trial information is available at clinicaltrials.gov

Phase 2: Phase 2 complete. No information on a potential Phase 3 trial. 

TEALS (Tecfidera in amyotrophic lateral sclerosis): Phase 2 clinical trial to investigate the safety, tolerability and effectiveness of Tecfidera.

This Phase 2 clinical trial aimed to assess if Tecfidera is effective in slowing down disease progression and whether it is safe for use in MND patients.

Learn more about this trial

Official clinical trial information is available at the Australia New Zealand Clinical Trials Registry 
For further information on the trial design, read this Medicine journal article 

Phase 3 (equivalent): Closed
Location: Sydney

A three part study to examine the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of BIIB067 (Tofersen). Tofersen is an antisense therapeutic specifically targeted at MND patients carrying the disease-causing SOD1 genetic mutation. In total, the study enrolled approximately 183 participants.

This study was completed in three parts – Parts A, B and C. Parts A and B were completed in January 2019. The results from Part C were announced in October 2021.

Learn more about this trial

Official clinical trial information is available at clinicaltrials.gov


International Alliance Scientific Advisory Council

MND Australia is a member of the International Alliance of ALS/MND Associations. One of the resources of this Alliance is the Scientific Advisory Council (SAC), which is a centralised resource to review and provide global perspectives on scientific and biomedical announcements, information and opportunities relating to ALS/MND. 

Read updates and briefing notes from the SAC

Clinical trials around the world

For a comprehensive list of MND/ALS clinical trials currently underway around the world, please visit IAMALS