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 or MND clinic 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 2: Active, currently recruiting patients.
Australian sites: Sydney, Melbourne, Adelaide, Launceston
Ambroxol is a prescription cough medication that is predicted to slow ALS disease progression.
Phase 2: Active, currently recruiting patients.
Australian sites: Brisbane, Gold Coast, Melbourne
A key mechanism that causes motor neuron loss in MND is a process called oxidative stress, that leads to cell damage. Oxidative stress increases the production of a particular enzyme called 15-LO. This medication blocks the action of 15-LO in an attempt to reduce oxidative stress and prevent motor neuron death.
Phase 3: Active, currently recruiting patients.
Australian site: Gold Coast University Hospital, Queensland.
There are anecdotal reports of cannabis slowing disease progression in persons with MND and that cannabis may relieve symptoms of spasticity and pain.
Active, currently recruiting patients.
Australian sites: Adelaide, Brisbane, Melbourne, Perth, Sydney
This study will test whether specific and individualised treatment with non-invasive ventilation (NIV) in combination with a sleep study leads to better outcomes in people with MND
Phase 3: Active, currently recruiting patients.
Australian sites: Brisbane, Sunshine Coast, Adelaide, Launceston, Melbourne, Sydney and Perth.
Triumeq is a drug that is used safely in HIV patients.
The Lighthouse I Project was a trial to test Triumeq in MND patients, 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 (known as Lighthouse II) has now commenced. Both the Phase 2 and 3 Lighthouse Trials trials have been partly supported by MNDRA.
Phase 3: Active and currently recruiting patients, however this trial is only open to people with a specific mutation in the unc13A gene
Australian sites: Sydney, Sunshine Coast, Brisbane, Adelaide, Melbourne, Perth
Previous studies have shown that Lithium Carbonate may be beneficial to some people with motor neurone disease. This trial is testing the effectiveness of lithium carbonate in patients who have a specific mutation in the unc13A gene.
Phase 3 (equivalent): Active and currently recruiting patients, however this trial is open only to people with a known SOD1 genetic mutation
Australian sites: Sydney
Please note that this study is open to those who are not yet showing symptoms of motor neurone disease, but who have a known mutation of the SOD1 gene.
Phase 1: Recruitment has now commenced.
Australian sites: Adelaide, Brisbane, Sydney
Recruitment has commenced in Adelaide and Brisbane. Recruitment at the Sydney site is expected to commence shortly. Please note that there are no Melbourne-based trial sites for this phase of the clinical trial.
Phase 1b: Completed, data currently being analysed.
ALS-205 (also called PMX205), has been designed to interfere with how the body’s immune system attacks the brain. The drug works by inhibiting a molecule involved in inflammation.
Phase 1: Recruitment complete. Phase 2 expected to commence in 2024.
Monepantel is a well-known veterinary drug. It is being trialed in MND patients as it has been shown to reduce protein aggregation in cells. The accumulation of abnormal protein within the motor neurons of the brain is associated with motor neurone disease.
Phase 2: Completed, data currently being analysed.
About half of all patients with ALS experience a change in their energy use that causes their body to consume more energy. This process is known as hypermetabolism and accelerates the spreading of ALS throughout the body. Trimetazidine is thought to be able to slow down the hypermetabolism experienced in ALS patients.
Phase 2: Recruitment for Phase 2 of this trial is now complete.
The aim of the REAL Study (Rho-kinasE for Als Study) is to assess the preliminary safety and efficacy of BRAVYL in patients with ALS. The open label study enrolled 20 patients at up to 10 sites and participants are receiving treatment with BRAVYL for 6 months.
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
