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 model of testing is important.
If you are considering involvement in a clinical trial, you will first need to speak with your neurologist to assess your eligibility. Clinical trials with Australian location sites are listed below and are divided into those that are currently recruiting and those that are active, but not currently recruiting.
Phase 1: Complete
Phase 1b: Underway, currently recruiting
Location: Wesley Hospital, Brisbane
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.
The follow-up Phase 1b trial will primarily evaluate the safety and tolerability of a single dose of PMX205 in individuals with ALS. The secondary objectives are: to determine the plasma pharmacokinetics (PK) of PMX205, evaluate PMX205 levels in the cerebrospinal fluid (CSF) after dosing at a single level, and to evaluate PMX205 pharmacodynamic (PD) activity by measuring cytokine levels following C5a challenge in peripheral immune cells. It is important to determine the extent to which PMX205 crosses the blood-brain barrier and enters brain tissue in individuals with ALS.
Eligibility criteria:
MNDRA have supported the early development of this potential treatment.
Official clinical trial information is available at the Australia New Zealand Clinical Trials Registry
Phase 2: Active, currently recruiting patients.
Location: Concord Hospital, Sydney
A phase 2 clinical trial of Ambroxol, a cough medicine, as an investigational treatment for MND.
Eligibility criteria:
Exclusion criteria:
Official clinical trial information is available on Australian New Zealand Clinical Trials Registry (ANZCTR)
Phase 3: Active, currently recruiting patients
Location: Trial sites in Sydney, Brisbane, Adelaide 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.
Eligibility criteria:
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.
Eligibility criteria:
Official clinical trial information is available at the Australia New Zealand Clinical Trials Registry
Active, currently recruiting patients.
Location: Melbourne, Sydney, Adelaide, Brisbane, Perth
This study will test whether specific and individualised titration of NIV in combination with a sleep study (polysomnography) leads to better outcomes in people with MND in a large, multi-centre randomised controlled trial (RCT) across multiple sites.
Eligibility criteria:
Official clinical trial information is available on ClincialTrials.gov
Phase 3: Currently recruiting patients
Location: Australian sites: Brisbane, Adelaide, Launceston, Melbourne, Sydney and Perth.
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 commenced. The phase 3 trial will be conducted in Europe, the U.K. and at five sites in Australia (Brisbane, Adelaide, Launceston, Melbourne, Sydney and Perth).
Eligibility criteria:
Exclusion criteria:
Both the Phase 2 and 3 trials have been partly supported by MNDRA.
Official clinical trial information is available at 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.
Eligibility criteria:
This trial is being conducted by a number of MNDRA funded researchers.
Official clinical trial information is available on clinicaltrials.gov
Phase 2: Active, currently recruiting patients.
Location: Brisbane, Melbourne, Sydney
REAL is a phase 2a open-label study to assess preliminary safety, tolerability, and effect on clinical outcomes as well as neurodegeneration biomarkers, of an oral Rho kinase inhibitor, BRAVYL, in patients with ALS. The study has been sponsored by Woolsey Pharma. Inhibition of the serine/threonine kinase Rho kinase was shown to counteract neurodegenerative processes and to foster neuronal regeneration in different animal models of neurodegenerative disease.
The study will enrol approximately 20 patients, all of whom will be treated with BRAVYL for 24 weeks.
Eligibility criteria:
Exclusion criteria:
Official clinical trial information is available at clinical trails.gov
Phase 3 (equivalent): Open - see information below
Location: Sydney
Biogen initiated the ATLAS study in 2021 to determine if pre-symptomatic treatment of SOD1 mutation carriers may represent more optimal timing of intervention. Given that individuals with SOD1 mutations can be recognised as at-risk of developing ALS through genetic testing prior to onset of symptoms, tofersen represents a tremendous opportunity to determine if treatment in pre-symptomatic individuals could provide a more robust effect on disease progression.
The primary objective of this study is to evaluate the efficacy of BIIB067 when initiated in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability of BIIB067 and to evaluate the effect of BIIB067 on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).
Eligibility criteria:
Official clinical trial information is available at clinicaltrials.gov
Active, no longer recruiting patients.
Location: Brisbane
This study is a First in Human, open label, Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) study to determine the safety and PK characteristics of mEphA4-Fc in healthy volunteers and patients with ALS. Safety and tolerability of mEphA4-Fc assessed from Incidence and severity of adverse events (AEs). Measurements of clinical and lung function endpoints associated with ALS will also be assessed.
Official clinical trial information is available on the Australia New Zealand Clinical Trials Registry (ANZCTR)
Phase 2/3: Complete, results not yet available. The community is keenly waiting to hear about the outcome of this trial but the company (Collaborative Medicinal Development Pty Limited) running the trial have not yet announced the outcome despite repeated requests.
Location: This study was 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.
Official clinical trial information is available on clinicaltrials.gov
Phase 2: Phase 2 completed in November 2021. The treatment was also tested in the Phase 2 Healey Platform trial. The company are currently considering their next stage of development
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.
Official clinical trial information is available at clinicaltrials.gov
Phase 2: Active, no longer 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.
Official clinical trial information is available on clinicaltrials.gov.
Biogen initiated the ATLAS study in 2021 to determine if pre-symptomatic treatment of SOD1 mutation carriers may represent more optimal timing of intervention. Please see the Atlas Preventative Trial for more information.
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.
Official clinical trial information is available at clinicaltrials.gov
Phase 1b/2a: Active, no longer recruiting patients.
Location: Perth, Brisbane and Sydney
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)
Official clinical trial information is available on clinicaltrials.gov
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
For a comprehensive list of MND/ALS clinical trials currently underway around the world, please visit IAMALS
