Clinical trial title
|
Description
|
Status |
Location |
MNDRA
investment |
CHAMPION ALS Study: Phase 3 evaluate the efficacy and safety of ravulizumab.
Official clinical trial information is here |
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.
See our FAQ page here |
Active, currently recruiting patients. |
Trial sites are planned in Sydney, Brisbane and Perth.
The trial is currently open for recruitment. |
|
Experiences of genetic testing |
Participants are being invited to be interviewed by telephone, video conference, email or online text chat/instant messaging if:
- You have MND and have been offered genetic testing for MND genes OR;
- You are a family member of a person with MND who was offered genetic testing for MND genes.
See our FAQ page for more information. |
Active, currently recruiting patients and family members of those with MND. |
Interview is conducted by telephone, video conference, email or online text chat/instant messaging. |
Project being undertaken by a researcher who has been previously funded by MNDRA |
Phase 2/3 Cu(II)ATSM.
Official clinical trial information is here. |
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.
More information is available on our FAQ page. |
Active, currently recruiting patients. |
Study being coordinated by Macquarie University. Please contact your neurologist for more information. |
More than $1.2 million to support 3 projects that helped develop copper-ATSM as a potential therapeutic |
Environmental risk factors for MND: Macquarie University Neurodegenerative Disease Biobank. |
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. |
Active, currently recruiting patients. |
Study being coordinated by Macquarie University.
Email Susan D'Silva or call
(02) 9812 3741
More information |
|
EMERALD: Phase 3 study on the safety, tolerability and efficacy of Cannabis Based Medicine Extract.
Official clinical trial information is here |
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.
See our FAQ page for more information. |
Active, currently recruiting patients. |
Gold Coast University Hospital.
Click here for more information on eligibility and for contact information. |
|
South Western Sydney Local Health District (SWSLHD) Communication and Assistive Technology (CAT) Clinic Research Study
More information. |
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.
See our FAQ page for more information. |
Active, recruiting patients. |
Liverpool Hospital, Sydney.
See here for more information on eligibility and for contact information. |
|
REFALS:Effects of Oral levosimendan (ODM-109) on Respiratory Function in Patients with ALS.
Official clinical trial information is here
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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.
See our FAQ page for more information. |
Active, currently not recruiting. |
Sydney, Brisbane, Adelaide, Melbourne, Perth.
|
|
Phase 2 Triumeq - Complete:
Safety and tolerability of antiretroviral (Triumeq) in patients with amyotrophic lateral sclerosis (ALS) (The Lighthouse Project).
Official clinical trial information is here
|
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. |
Phase 2 trial complete. Phase 3 trial has not yet commenced |
Australian sites for Phase 3 trial to be finalised |
Both the Phase 2 and 3 trials have been partly supported by MNDRA |
TEALS (Tecfidera in amyotrophic lateral sclerosis) Study:
Phase 2 clinical trial to investigate the safety, tolerability and effectiveness of Tecfidera.
Official clinical trial information is here |
This Phase 2 clinical trial will assess if Tecfidera is effective in slowing down disease progression and whether it is safe in MND patients.
See our FAQ page for more information.
For further information on the trial design see here. |
Recruitment completed.
Data currently being analysed |
Adelaide, Brisbane, Melbourne,
Sydney, Perth |
|
VALOR: This 3-part study will examine the efficacy, safety, tolerability, PK and PD of BIIB067 (Tofersen).
Official clinical trial information is here |
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.
For more information please see our FAQ page. |
Currently recruiting |
Sydney |
|
RESCUE-ALS:
Phase 2 study of the efficacy, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8.
Official clinical trial information is here.
|
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.
For more information please see our FAQ page. |
Active. Currently recruiting. |
Sydney |
|
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.
Official clinical trial information is here. |
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.
For more information please see our FAQ page. |
Provisionally registered. Not yet commenced or recruiting. |
Brisbane |
MNDRA have supported Trent Woodruff in developing this potential treatment. |