Clinical trials

Clinical trials are research studies that use human volunteers to test new therapies. After scientists test experimental therapies in the laboratory, those with promising results move to clinical trial to determine whether the therapy is safe and effective for use in humans. A new therapy must successfully pass through a series of phases before ultimately being approved by Health Canada and being made widely available to the Canadian public (learn more in our Clinical Trials FAQs).

In general, ALS clinical trials are therapeutic or observational in nature. Therapeutic clinical trials test potential drug therapies or interventional devices that aim to either slow the progression of the disease or help to manage symptoms. Observational trials aim to learn more about the disease and are essential to understanding, diagnosing and ultimately treating ALS. In many cases people who participate in clinical trials will not benefit from the therapy, but their generous involvement will help to find a successful therapy for those diagnosed in the future. One day, a clinical trial will test a therapeutic that slows the progression of ALS and those involved may directly benefit from taking part.

For more information, please speak with your clinician (preferably at an ALS clinic) and visit, where all legitimate, recognized ALS clinical trials are registered globally. You can also visit the EU Clinical Trials Register and the World Health Organization International Clinical Trials Registry for additional information.

You can learn more about the clinical trials currently being conducted at sites across Canada below.

Current Clinical Trials for ALS in Canada

*NEW* Safety Study of Oral Edaravone Administered in Subjects With ALS

Study Type: Therapeutic (Drug: MT-1186); Phase 3
Status: Recruiting
Sites: Edmonton, Montreal (Neuro), Greenfield Park
Sponsor: Mitsubishi Tanabe Pharma Development America, Inc.

In October 2018, a new treatment for ALS called edaravone (Radicava) was approved by Health Canada. The decision was based on evidence from a previous Phase 3 clinical trial which showed that edaravone helped to slow functional decline in participants who were early in their ALS progression, with milder symptoms and a larger vital capacity (the maximum amount of air a person can expel from their lungs after a maximum inhalation). Edaravone is delivered through intravenous (IV) infusion over several consecutive days, with 14 day breaks in between. In this new Phase 3 clinical trial, researchers will enroll 150 participants to test the safety and effectiveness of a new oral form of edaravone (MT-1186) which can be taken by mouth. The trial is expected to last 48 weeks and all participants will be given the active drug meaning there will be no placebo group. The hope is that the oral form proves to be safe and can be delivered to the brain and spinal cord as effectively as the current IV form. Due to the relatively intensive dosing regimen recommended for edaravone, a treatment option like MT-1186 that can be taken orally could significantly improve quality of life for people living with ALS.

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*NEW* An Efficacy and Safety Study of Ravulizumab in ALS Participants (CHAMPION-ALS)

Study Type: Therapeutic (Drug: Ravulizumab); Phase 3
Status: Recruiting
Sites: Montreal (Neuro), Fredericton
Sponsor: Alexion Pharmaceuticals

Ravulizumab (also referred to as Ultomiris) is a drug that contains an antibody that targets a specific protein within the immune system, called C5, which is thought to play a role in the onset and progression of ALS. Researchers hope that by blocking this protein they may be able to slow disease progression. CHAMPION-ALS is a global Phase 3 clinical trial that will evaluate the safety and effectiveness of ravulizumab in 350 participants with familial (inherited) or sporadic ALS. The treatment will be administered through intravenous (IV) infusion every 8 weeks for a total of 50 weeks. The drug’s effectiveness will be measured primarily using the ALS Functional Rating Score Revised (ALSFRS-R), a 12-item questionnaire that assesses function in certain daily activities. Additional outcomes that will be monitored include changes in muscle strength, breathing parameters, and levels of neurofilament light chain (a biomarker of disease severity). Once the trial is completed all participants will have the opportunity to receive ravulizumab in a two-year open-label extension phase.

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*NEW* Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS (COMBAT-ALS)

Study Type: Therapeutic (Drug: Ibudilast); Phase 2b/3
Status: Recruiting
Sites:  Montreal (Neuro)
Sponsor: MediciNova

Ibudilast (also referred to as MN-166) is an experimental therapy being developed to treat ALS. This broad target drug is thought to reduce the activity of immune cells in the brain, thereby supressing inflammation. It is also believed to promote the production of neurotrophic factors which play a role in the growth and survival of motor neurons. The Phase 2b/3 COMBAT-ALS clinical trial will enroll 230 participants living with ALS and last 12 months. Researchers will monitor participants to ensure that the drug is safe. Researchers will also evaluate the impact of ibudilast on the progression of ALS by evaluating changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) score, as well as muscle strength, quality of life, and respiratory function. Ibudilast will be taken by mouth in combination with a dose of riluzole. Researchers believe the anti-inflammatory and neuroprotective characteristics of ibudilast make it a promising treatment option for ALS.

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A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS)

Study Type: Therapeutic (Drug: AP-101); Phase 1
Status: Recruiting
Sites: London, Toronto, Montreal (Neuro)
Sponsor: AL-S Pharma

Antibodies are proteins that are produced by the immune system to protect the body against foreign invaders like bacteria and viruses, and work by binding to specific proteins on the harmful agents and triggering their removal and/or destruction. In this Phase 1 trial, researchers will be testing the safety of a human antibody (called AP-101) designed to target an ALS-linked protein called SOD1. Evidence suggests that the misfolding of SOD1 in cells may cause a toxic gain of function that leads to neurodegeneration. Researchers are hopeful that targeting this protein may represent a promising strategy for the treatment of ALS. The study is expected to enroll 18 participants who will receive doses of AP-101 by intravenous (IV) infusion.

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Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

Study Type: Therapeutic (Drug: Masitnib); Phase 3
Status: Not yet recruiting
Sites: TBD
Sponsor: AB Science

Masitinib is an oral drug that targets cells within the body that play an important role in the immune system. Previous studies suggest that masitinib may reduce inflammation within the nervous system that is thought to be a factor in the progression of ALS. A previous Phase 2/3 clinical trial showed that over a 48-week period, treatment with masitinib reduced loss of function and increased quality of life. This global Phase 3 clinical trial is intended to confirm the results of the previous Phase 2/3 study and is expected to enroll 495 participants in Canada, the US and Europe.

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An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

Study Type: Therapeutic (BIIB067); Phase 3
Status: Recruiting
Sites: Calgary, Toronto, Montreal (Neuro)
Sponsor: Biogen
Collaborator: Ionis Pharmaceuticals, Inc.

*Due to the specificity of this treatment, this clinical trial is only open to individuals with a SOD1 mutation.   

BIIB067 (also referred to as tofersen) is an antisense oligonucleotide (ASO) that is being studied to treat a familial form of ALS linked to mutations in the SOD1 gene. As a result of mutation, SOD1 is believed to gain a toxic function that is damaging to the nerve cells that control movement. BIIB067 is designed to decrease production of SOD1 which researchers hope will lead to preservation of motor neurons and slowed progression of the disease. This Phase 3 clinical trial will enroll 144 participants with a confirmed SOD1 mutation and will test the safety, tolerability and effectiveness of BIIB067. This study is a continuation of a previous Phase 1/2 clinical trial which showed promising results.

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A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 in Adults With C9ORF72-Associated Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (BIIB078); Phase 1
Status: Active, not recruiting
Sites: Calgary, Edmonton, London, Toronto, Montreal (Neuro & CHUM)
Sponsor: Biogen

*Due to the specificity of this treatment, this clinical trial is only open to individuals with a C9ORF72 mutation.   

BIIB078 is an antisense oligonucleotide (ASO) that is being studied to treat a familial form of ALS linked to mutations in the C9ORF72 gene. Approximately 34% of all familial ALS cases are linked to C9ORF72, making it the most common genetic cause of ALS. Two substances are produced in cells as a result of C9ORF72 mutations, commonly referred to as repeat RNAs and DPR proteins. These substances are thought to contribute to the cellular toxicity that leads to disease. BIIB078 is designed to target repeat RNAs preventing them from being used to create the potentially toxic DPR proteins. This Phase 1 clinical trial will enroll 80 participants with a confirmed C9ORF72 mutation. Researchers will monitor participants to ensure that the drug is safe, determine the appropriate dosage and learn more about how the body breaks down the drug internally.

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Novel MRI Biomarkers for Monitoring Disease Progression in ALS

Study Type: Observational
Status: Recruiting
Sites: Edmonton, Calgary, Toronto, Montreal (Neuro), Quebec City
Sponsor: University of Alberta

The ability to accurately measure people’s brain degeneration may help researchers to find much needed biomarkers that are essential to understanding, diagnosing and ultimately treating ALS. Using advanced brain imaging techniques (magnetic resonance imaging, or MRI), this observational study will monitor over time the degree of change that occurs in the brains of people who are living with ALS. Each study participant will have 3 MRI scans over a period of 8 months, along with neurological and cognitive evaluations. This study is led Dr. Sanjay Kalra and will operate within the Canadian ALS Neuroimaging Consortium (CALSNIC), a cross-Canada imaging network funded by the largest-ever grant provided by the ALS Canada research program.

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NeuroCognitive Communicator: Safety Study (NCC-1701)

Study Type: Therapeutic (Device: NeuroCognitive Communicator)
Status: Not yet recruiting
Sites: Ottawa
Sponsor: Ottawa Hospital Research Institute

The progressive paralysis experienced by people living with ALS can eventually make communicating with others difficult as the ability to gesture and speak lessens with the weakening of the muscles. To help improve quality of life for people affected by ALS, researchers from the Ottawa Hospital Research Institute are testing the safety of a new assistive device that uses brain-computer interface (BCI) technology to help people who have motor impairments to communicate. This device can convert brain signals into single letters on a computer screen, allowing people to spell words simply with their thoughts. The technology requires surgical placement of two sensors into the areas of the brain that support motor and cognitive function. Researchers will be monitoring the two participants to ensure that the procedure is safe, and to assess the ability of this technology to support effective communication and improve quality of life.

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A Clinical Trial of Pimozide in Patients with Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: pimozide); Phase 2
Status: Recruiting
Sites: Calgary, Edmonton, Fredericton, Hamilton, London, Ottawa, Toronto, Greenfield Park, Montreal (CHUM)
Sponsor: University of Calgary
Collaborators: ALS Canada, Brain Canada

Some people believe that the loss of muscle function that occurs in ALS is caused by the muscles and the nerves not being able to communicate anymore. The area in the body where this communication occurs is called the neuromuscular junction. Pimozide is a medication originally used in schizophrenia that has been shown to enhance communication at the neuromuscular junction in laboratory worms, fish and mice. This Phase 2 study will investigate whether treatment with pimozide slows the progression of ALS in humans. Pimozide will be evaluated primarily using the ALS Functional Rating Score Revised (ALSFRS-R), a 12-item questionnaire that assesses function in certain daily activities. This Phase 2 clinical trial is supported by an ALS Canada-Brain Canada Arthur J Hudson Translational Team Grant.

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Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) (ARTFL)

Study Type: Observational
Status: Recruiting
Sites: Vancouver, Toronto
Sponsor: University of California

Frontotemporal dementia (FTD) is present in 15 to 18 per cent of people living with ALS. FTD is the second most common cause of early onset dementia and results in degeneration of the frontal and temporal lobes of the brain, which can cause changes in personality and language difficulties. The Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) project is an observational study with the goal to ultimately discover new biomarkers to better understand disease, improve diagnostic criteria, and identify a large group of potential participants for future clinical trials of new treatment options. Participants in this study include people living with FTD and/or ALS, as well as those living with related frontotemporal lobar degeneration (FTLD) syndromes. On-site evaluations will include medical exams, clinical assessments of cognition and function, questionnaires and surveys, and biological samples.

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Diagnosing Frontotemporal Lobar Degeneration

Study Type: Observational
Status: Recruiting
Sites: Toronto
Sponsor: University Health Network, University of Toronto

Diagnosing neurodegenerative conditions such as FTD or ALS can be difficult, as symptoms and disease progression often differ significantly from person to person. The goal of this observational study is to use a variety of tests to determine the best criteria for diagnosing frontotemporal lobar degeneration (FTLD) syndromes. These tests include brain imaging, skin biopsy, cognitive and functional assessments and biological samples for genetic testing. Researchers hope the results of this study will not only guide FTLD diagnosis but also provide a better understanding of the mechanism of disease and aid in the development of new treatments in the future. These advancements may also prove to be helpful in understanding and ultimately treating ALS due to the close overlap in the pathological and genetic features of ALS and FTD.

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Phenotype, Genotype & Biomarkers in ALS and Related Disorders

Study Type: Observational
Status: Recruiting
Sites: Edmonton
Sponsor: University of Miami

This observational study is recruiting people living with ALS and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD). Biological samples will be collected from study participants with the aim of discovering new biomarkers of disease, and the hope to better understand the links between genetic data (genotype) and observable symptoms (phenotype).

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