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 ClinicalTrials.gov, 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* A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS) (COURAGE-ALS)

Study: Therapeutic (Drug: Reldesemtiv); Phase 3
Status: Recruiting
Sites: Calgary, Edmonton, Saskatoon, Hamilton, Toronto, Ottawa, Montreal (Neuro & CHUM), Quebec City, Fredericton
Sponsor: Cytokinetics

Reldesemtiv is an oral, muscle-activating drug designed to increase the ability for a muscle to contract with reduced nerve stimulation. The development of this drug follows a previous Phase 2 clinical trial which showed that reldesemtiv may help to slow rates of functional and respiratory decline in people living with ALS. This global Phase 3 clinical trial, called COURAGE-ALS, will enroll approximately 555 participants. In the first phase of the trial, participants will be assigned randomly in a 2:1 ratio to receive either the active drug (reldesemtiv) or a placebo for 24 weeks. In the second phase, all the participants will receive reldesemtiv for another 24 weeks. Researchers will evaluate the impact of reldesemtiv by evaluating changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) score, as well as muscle strength, quality of life, and respiratory function.

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*NEW* A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

Study: Therapeutic (Drug: ION363); Phase 3
Status: Recruiting
Sites: Montreal (Neuro)
Sponsor: Ionis Pharmaceuticals, Inc.

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

Mutations in the FUS gene are a known genetic cause of ALS, with most cases of juvenile-onset ALS linked to this gene. It is believed that these mutations result in the production of abnormal FUS proteins, which are prone to create toxic clumps in cells leading to nerve damage. ION363 is an antisense therapy that targets and binds to FUS messenger RNA (mRNA), the molecule that carries the information required to produce FUS protein in cells. It is delivered into the spinal fluid through a procedure known as an intrathecal injection. Researchers are hopeful that ION363 will lower the levels of abnormal FUS protein in cells and ultimately slow the progression of disease. This Phase 3 clinical trial will enroll approximately 64 participants and be conducted in two-parts. In Part 1, participants will be randomized in a 2:1 ratio to receive either ION363 or placebo (via intrathecal injection) for a period of 29 weeks. In Part 2, all participants will receive ION363 for a period of 77 weeks. The effectiveness of ION363 will be studied using measures of functional impairment, quality of life and survival.

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*NEW* Multiple Doses of AT-1501-A201 in Adults With ALS

Study: Therapeutic (Drug: AT-1501); Phase 2
Status: Recruiting
Sites: Montreal (Neuro)
Sponsor: Eledon Pharmaceuticals

Researchers believe that inflammation within the nervous system may contribute to the progression of ALS. AT-1501 is an antibody therapy designed to bind to a specific protein called CD40 ligand (CD40L). This protein is found on the surface of some immune cells and plays a role in regulating the immune response, which is thought to be overactive in people living with ALS. By binding to CD40L, researchers are hopeful that AT-1501 may be able to reduce inflammation and slow the progression of disease in people living with ALS. This Phase 2 trial will enroll approximately 54 participants living with ALS and is open label, meaning that all participants will receive the active drug via intravenous (IV) infusion for up to 19 weeks. Researchers will monitor participants to ensure that the drug is safe and determine the appropriate dosage.

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*NEW* A Study of BIIB067 When Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation (ATLAS)

Study: Therapeutic (Drug: BIIB067); Phase 3
Status: Recruiting
Sites: Calgary, Toronto, Montreal (Neuro)
Sponsor: Biogen

*Due to the specificity of this treatment, this clinical trial is only open to presymptomatic carriers of a specific subset of SOD1 mutations defined as conferring rapidly progressive ALS

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 voluntary muscles, called motor neurons. Tofersen is designed to decrease production of SOD1 which researchers hope will lead to preservation of motor neurons and slowed progression of the disease. It is delivered into the spinal fluid through a procedure known as an intrathecal injection. Although a previous Phase 3 clinical trial studying tofersen was not able to demonstrate a statistically significant difference in ALSFRS-R score between the active drug and placebo groups over the 6-month study period, treatment with tofersen did show signs of clinical effect across multiple measures. This Phase 3 clinical trial, called ATLAS, is the first of its kind as it will be enrolling participants with a SOD1 mutation who are considered to be presymptomatic (e.g., do not yet show overt signs of the disease), but have an elevated biomarker indicating the sub-clinical triggering of ALS. The goal of this study is to determine whether presymptomatic treatment with tofersen can delay the onset of clinical ALS diagnosis and slow functional decline thereafter. Participants in the study will be treated for up to two years.

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*NEW* A Phase 1 Study to Investigate the Safety and Pharmacokinetics of ABBV-CLS-7262 in Patients With Amyotrophic Lateral Sclerosis

Study: Therapeutic (Drug: ABBV-CLS-7262); Phase 1
Status: Recruiting
Sites: Edmonton, Montreal (Neuro), Fredericton
Sponsor: Calico Life Sciences

The integrated stress response (or ISR) is a signaling pathway that is activated within cells in response to a variety of different stressors, including the build-up of abnormal proteins which is seen in many neurodegenerative diseases, such as ALS. When this happens, a cell will signal via the ISR to stop the production of all proteins, which over prolonged periods of time can be detrimental to the health of a cell. ABBV-CLS-7262 is an oral experimental treatment designed to boost a specific protein, called EIF2b, that plays an important role in restarting protein production in cells and has been shown to have a neuroprotective effect in mouse models of ALS. In this Phase 1 clinical trial, researchers will enroll 30 participants who will be randomly assigned to receive either the active drug (ABBV-CLS-7262) or placebo for 4 weeks, followed by an open-label phase where all participants will receive the active treatment for 44 weeks. 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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*NEW* Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) (FOCUS-C9)

Study: Therapeutic (Drug: WVE-004); Phase 1b/2a
Status: Recruiting
Sites: Montreal (Neuro)
Sponsor: Wave Life Sciences

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

Approximately 25-40% of all familial and 5-10% of all sporadic ALS cases are linked to C9ORF72, making it the most common genetic cause of ALS. As a result of C9ORF72 mutations, toxic substances commonly referred to as DPR proteins are produced within cells. These substances are thought to contribute to the progression of disease. WVE-004 is an antisense oligonucleotide (ASO) designed to bind to specific C9ORF72 messenger RNA (mRNA) molecules and prevent the formation of DPRs while still allowing cells to produce normal C9ORF72 protein. Researchers are hopeful that if WVE-004 is successful at reducing DPR levels in humans, they can slow the progression of disease. This Phase 1b/2a will enroll approximately 42 participants, who will be randomly assigned to receive either the active drug or a placebo treatment. It is delivered into the spinal fluid through a procedure known as an intrathecal injection. 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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*NEW* A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

Study: Therapeutic (Drug: AP-101); Phase 2
Status: Recruiting
Sites: Edmonton, London, 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 2 clinical 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 can cause the protein to take on a toxic gain of function. Researchers are hopeful that targeting this protein may represent a promising strategy for the treatment of ALS. A previous Phase 1 study found the drug to be safe and well-tolerated at the tested doses. In this follow-up study, researchers expect to enroll 63 participants who will be randomly divided to either receive the active drug (AP-101) or placebo intravenously (IV), for 48 weeks. Both individuals with familial, SOD1-ALS and sporadic disease will be eligible to participate. Researchers will monitor participants to ensure that the drug is safe, identify any side effects, determine the appropriate dosage, and learn more about how the body breaks down the drug internally.

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Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)

Study: Therapeutic (Drug: ANX005); Phase 2
Status: Recruiting
Sites: Edmonton, Toronto, Montreal (Neuro & CHUM), Fredericton
Sponsor: Annexon, Inc.

ANX005 is an antibody-based drug that targets a specific molecule, called C1q, within an inflammatory pathway called the complement system. Researchers hope that by inhibiting C1q they may be able to slow functional decline in people living with ALS. This Phase 2 clinical trial is open label with no placebo group, meaning everyone who enrolls in the study will receive the active drug. Approximately 24 participants will receive intravenous (IV) infusion of ANX005 over a 12-week period. Participants will be monitored to ensure that the drug is safe and to learn more about how the body breaks down the drug internally.

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Efficacy and Safety Study of Oral Edaravone Administered in Subjects With ALS

Study: Therapeutic (Drug: MT-1186); Phase 3b
Status: Recruiting
Sites: Edmonton, Saskatoon, Hamilton, London, Toronto, Montreal (Neuro), Greenfield Park, Quebec City, Fredericton
Sponsor: Mitsubishi Tanabe Pharma Development America, Inc.

In October 2018, a new treatment for ALS called edaravone (Radicava) was approved by Health Canada. Edaravone is typically delivered through intravenous (IV) infusion over several consecutive days, with 14 day breaks in between. In this new Phase 3b clinical trial, researchers will compare the effectiveness of two different dosing regimens of a new oral form of edaravone, called MT-1186. The study will enroll approximately 380 participants and last for 48 weeks. The effectiveness of oral edaravone at each dosing regimen will be measured primarily using the ALS Functional Rating Score Revised (ALSFRS-R), as well as certain breathing parameters and a questionnaire designed to evaluate aspects of health considered important by patients.

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Enoxacin for Amyotrophic Lateral Sclerosis (ALS) (REALS-1)

Study: Therapeutic (Drug: Enoxacin); Phase 1b/2
Status: Recruiting
Sites: Montreal (Neuro)
Sponsor: McGill University

This Phase 1b/2 clinical trial will test the safety and tolerability of enoxacin in people living with ALS. Enoxacin is an antibiotic that on a cellular level is known to activate an enzyme called DICER, which in turn can increase the levels of specific substances called microRNA (miRNA) which have been shown to be reduced in people living with ALS. The study will enroll approximately 36 participants who will be treated with enoxacin (taken by mouth) over a 30-day period. 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. This study is being led by Canadian researcher Dr. Angela Genge (Montreal Neurological Institute-Hospital) in collaboration with Dr. Eran Hornstein (Weizmann Institute of Science). They are also the recipients of a 2020 ALS Canada-Brain Canada Discovery Grant which will allow them to will conduct more in-depth analyses to further assess the value of enoxacin in ALS. You can learn more about their exciting work here.

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Radicava® (Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)

Study: Observational
Status: Recruiting
Sites: Calgary, Toronto, Montreal (Neuro)
Sponsor: Mitsubishi Tanabe Pharma America Inc.

Biomarkers that can effectively measure neurodegeneration are much needed and essential to understanding, diagnosing, and ultimately treating ALS. In this observational study, researchers are seeking to identify and measure the levels of specific biomarkers in ALS patients being treated with edaravone (Radicava). The biomarkers of interest are linked to oxidative stress, inflammation, and neuronal and muscle injury. The study will enroll approximately 300 participants who will be followed over a 24-week period. Biological samples (blood and urine) will be collected from study participants for analyses. Participants can also opt in to receive their genetic results for five common mutations associated with ALS, including SOD1, TARDBP, C9ORF72, FUS and VCP.

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A Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 in Participants With Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: BIIB105); Phase 1
Status: Recruiting
Sites: Montreal (Neuro)
Sponsor: Biogen

BIIB105 (also referred to as ION541) is an antisense oligonucleotide (ASO) that is being studied to treat ALS. This is the first antisense treatment that has be designed to treat sporadic, rather than familial, forms of ALS. BIIB105 works to reduce production of a protein called ataxin 2 (ATXN2), which has been shown to impact the behaviour of another ALS-linked protein, TDP-43, that behaves abnormally in 97 per cent of people with ALS. It is hypothesized that lowering ATXN2 levels will help to restore the normal function of TDP-43, and therefore result in a therapeutic benefit. This Phase 1 study will enroll approximately 70 people living with ALS. 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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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: Saskatoon, Hamilton, Toronto, 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.

To learn more, please click here to view a webinar hosted by the study sponsor, MediciNova.

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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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NeuroCognitive Communicator: Safety Study (NCC-1701)

Study Type: Therapeutic (Device: NeuroCognitive Communicator)
Status: 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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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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Safety Study of Oral Edaravone Administered in Subjects With ALS

Study Type: Therapeutic (Drug: MT-1186); Phase 3
Status: Active, not recruiting
Sites: Edmonton, Montreal (Neuro), Greenfield Park, Quebec City
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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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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