Resources
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.
Read the MND Australia’s Position Statement on the development and approval of drugs to treat MND.
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 about Clinical Trials.
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). Current briefing notes produced by the alliance 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: ALS Signal: Clinical Research Dashboard.
Clinical trials for MND and/or amyotrophic lateral sclerosis (ALS) are currently underway in Australia.
Clinical trials for MND and/or amyotrophic lateral sclerosis (ALS) are currently underway in Australia.
Clinical Trial Title
Phase 2/3 Cu(II)ATSM.
Read the official clinical trial information.
Description
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.
Status
Active, currently recruiting patients.
Location
Study being coordinated by Macquarie University. Please contact your neurologist for more information.
MNDRA Investment
More than $1.2 million to support 3 projects that helped develop copper-ATSM as a potential therapeutic
Clinical Trial Title
Environmental risk factors for MND: Macquarie University Neurodegenerative Disease Biobank.
Description
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.
Status
Active, currently recruiting patients.
Location
Study being coordinated by Macquarie University.
Email Susan D’Silva or call
(02) 9812 3741
MNDRA Investment
Clinical Trial Title
EMERALD: Phase 3 study on the safety, tolerability and efficacy of Cannabis Based Medicine Extract.
Description
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.
Status
Active, currently recruiting patients.
Location
Gold Coast University Hospital.
Click here for more information on eligibility and for contact information.
MNDRA Investment
Clinical Trial Title
South Western Sydney Local Health District (SWSLHD) Communication and Assistive Technology (CAT) Clinic Research Study
Description
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.
Status
Active, recruiting patients.
Location
Liverpool Hospital, Sydney.
See here for more information on eligibility and for contact information.
MNDRA Investment
Clinical Trial Title
REFALS: Effects of Oral levosimendan (ODM-109) on Respiratory Function in Patients with ALS.
Description
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.
Status
Active, currently not recruiting.
Location
Sydney, Brisbane, Adelaide, Melbourne, Perth.
MNDRA Investment
Clinical Trial Title
Phase 2 Triumeq – Complete:
Safety and tolerability of antiretroviral (Triumeq) in patients with amyotrophic lateral sclerosis (ALS) (The Lighthouse Project).
Description
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 early in 2020.
Status
Phase 2 trial complete. Phase 3 trial has not yet commenced
Location
Australian sites for Phase 3 trial to be finalised
MNDRA Investment
Both the Phase 2 and 3 trials have been partly supported by MNDRA
Clinical Trial Title
TEALS (Tecfidera in amyotrophic lateral sclerosis) Study:
Phase 2 clinical trial to investigate the safety, tolerability and effectiveness of Tecfidera.
Description
This Phase 2 clinical trial will assess if Tecfidera is effective in slowing down disease progression and whether it is safe in MND patients.
Status
Recruitment completed.
Data currently being analysed
Location
Adelaide, Brisbane, Melbourne,
Sydney, Perth
MNDRA Investment
Clinical Trial Title
VALOR: This 3-part study will examine the efficacy, safety, tolerability, PK and PD of BIIB067 (Tofersen).
Description
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.
Status
Currently recruiting
Location
Sydney
MNDRA Investment
Clinical Trial Title
RESCUE-ALS:
Phase 2 study of the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8.
Description
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.
Status
Active. Currently recruiting.
Location
Sydney
MNDRA Investment
Clinical Trial Title
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.
Description
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. A Phase 1A Safety trial in healthy volunteers planned to commence in March 2020.
Status
Provisionally registered. Not yet commenced or recruiting.
Location
Brisbane
MNDRA Investment
MNDRA have supported Trent Woodruff in developing this potential treatment.
