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.
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.
If you are considering involvement in a clinical trial, you will first need to speak with your neurologist to assess your eligibility.
Phase 1: Provisionally registered. Not yet commenced or recruiting.
A Phase 1 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple ascending doses of ALS-205 in healthy volunteers.
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.
MNDRA have supported Professor Trent Woodruff in developing this potential treatment.
Phase 3: Active, currently recruiting patients
Location: Trial sites are planned in Sydney, Brisbane and Perth.
A Phase 3 evaluate the efficacy and safety of ravulizumab. 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.
Phase 2/3: Active, currently recruiting patients.
Location: study is being coordinated by Macquarie University, Sydney.
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.
MNDRA has provided more than $1.2 million to support three projects that helped develop copper-ATSM as a potential therapeutic.
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.
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 mid-late 2021, with trial sites soon to be announced.
Both the Phase 2 and 3 trials have been partly supported by MNDRA.
Phase 2a: Active, currently recruiting patients
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.
Phase 3: Active, currently not recruiting.
Location: Sydney, Brisbane, Adelaide, Melbourne, Perth.
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 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.
Phase 2: active, currently recruiting
A Phase 2 study of 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.
Phase 2: Recruitment completed. Data currently being analysed.
TEALS (Tecfidera in amyotrophic lateral sclerosis): Phase 2 clinical trial to investigate the safety, tolerability and effectiveness of Tecfidera.
This Phase 2 clinical trial has aimed to assess if Tecfidera is effective in slowing down disease progression and whether it is safe for use in MND patients.
Read 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): Currently recruiting.
A 3-part study to examine the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of BIIB067 (Tofersen). 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 enrol 183 participants, with 99 in Part C which will constitute the Phase 3 component of the trial.
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.
For a comprehensive list of clinical trials currently underway around the world, please see this great resource hosted by IAMALS.