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* A Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: Acthar); Phase 2
Status: Recruiting
Sites: Edmonton, Ottawa, Montreal (Neuro & CHUM), Greenfield Park
Sponsor: Mallinckrodt

H.P. Acthar® Gel is an injectable drug that contains ACTH, a hormone normally found within the body that stimulates the release of natural steroids, such as cortisol. It is believed that ACTH may also affect immune cells within the body. This Phase 2 clinical trail will test whether H.P. Acthar® Gel can slow the progression of ALS. Participants will receive daily subcutaneous injections (i.e. injections under the skin) of the drug for a period of 36 weeks. To determine the effectiveness of the drug, researchers will measure participants’ functional decline via telephone calls using the ALS Functional Rating Score Revised (ALSFRS-R). H.P. Acthar® Gel is currently used to treat other conditions, such as lupus and multiple sclerosis, and so researchers are hopeful that there may be a positive effect for people living with ALS as well.

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*NEW* A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: Arimoclomol); Phase 3
Status: Recruiting
Sites: Toronto, Montreal (Neuro), London
Sponsor: Orphazyme

One of the defining characteristics of ALS and other neurodegenerative diseases is that proteins can become misfolded and clump together, potentially damaging nerve cells. Arimoclomol is an oral drug that increases the production of a group of proteins called heat shock proteins (HSPs) that work to prevent protein misfolding in cells. This Phase 3 clinical trial will test whether treatment with Arimoclomol can slow the progression of ALS. Researchers will determine the effectiveness of the drug through a combined assessment of functional decline (measured using the ALSFRS-R) and survival. Researchers will also monitor changes in participant’s slow vital capacity (SVC), a measure of lung function. The study is expected to last 76 weeks, after which participants will have the option to partake in an open-label extension trial.

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REFALS: Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS

Study Type: Therapeutic (Drug: levosimendan); Phase 3
Status: Recruiting
Sites: Calgary, Edmonton, Toronto, Montreal (Neuro & CHUM), Fredericton, Moncton
Sponsor: Orion Corporation, Orion Pharma

This Phase 3 clinical trial will determine whether levosimendan (ODM-109) can improve respiratory function (breathing abilities) in people living with ALS. Levosimendan is an oral drug shown in previous studies to help muscles (such as the diaphragm) contract more easily. This study will enroll approximately 450 people living with ALS. Participants will take the drug daily for a period of 48 weeks. The effectiveness of the treatment will be assessed by monitoring changes in a participant’s slow vital capacity (SVC), a measure of the volume of air exhaled without forced effort after a full breath. Overall functional decline will also be measured using the ALS Functional Rating Score Revised (ALSFRS-R).

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Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

Study Type: Therapeutic (Drug: NurOwn®); Phase 3
Status: Recruiting
Sites: University of Massachusetts Medical School
*Canadians may contact
Sponsor: Brainstorm-Cell Therapeutics
Collaborator: California Institute for Regenerative Medicine

Stem cells are cells in the body that have not yet matured into a specific type of cell with a function (such as skin, muscle, bone, etc.). Because of this, stem cells have the ability to become part of any organ in the body. This special ability has led scientists to believe that stem cells may have the ability to repair and replace tissue within the human body. In this Phase 3 clinical trial, researchers are testing the safety and effectiveness of a new potential ALS treatment called NurOwn®. In this study, a person’s own stem cells are taken from their bone marrow and are then combined with the NurOwn® drug. The drug converts the stem cells into cells that secrete neurotrophic factors (NTFs), substances thought to support motor neuron health and delay their degeneration in ALS. These transformed cells are then injected back into the spinal cord of the person living with ALS (intrathecal injection). Researchers will be monitoring participants to assess the safety and ability of NurOwn® to slow the progression of ALS.

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A Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Participants with Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: GDC-0134); Phase 1
Status: Recruiting
Sites: Montreal (Neuro)
Sponsor: Genentech, Inc.

This Phase 1 clinical trial is designed to test the safety and tolerability of a new oral medicine called GDC-0134 in people living with ALS. GDC-0134 is a drug designed to block the activity of a protein called dual leucine zipper kinase or DLK. DLK is found in nerve cells in the brain and spinal cord and previous studies have shown that activation of DLK may cause these nerve cells to die when they become stressed. Preclinical studies using ALS animal models also revealed that when DLK is blocked it may help to delay motor neuron death. Based on these positive results, GDC-0134 is now being tested as a potential treatment for ALS with approximately 70 people living with ALS to be enrolled in this study. 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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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, 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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