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* 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 diane.mckenna-yasek@umassmed.edu
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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*NEW* 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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Blood-Brain Barrier Opening Using MR-Guided Focused Ultrasound in Patients with Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Device: Blood-Brain Barrier opening with MRI-guided focused ultrasound)
Status: Not yet recruiting
Sites: Toronto
Sponsor: InSightec

Delivering promising treatments to the nervous system is challenging because our bodies have a specialized barrier called the blood-brain barrier that makes it difficult for drugs in the bloodstream to cross into the brain or spinal cord. In this clinical trial, researchers from the Sunnybrook Health Sciences Centre and the University of Toronto are testing the safety and effectiveness of a new technology that helps to deliver treatments to specific regions of the brain in eight people living with ALS. Using image-guided focused ultrasound, the researchers can open small spaces in the blood-brain barrier to let the treatments cross into the brain. If successful, the team hopes to use the technology to deliver promising treatments that target upper motor neurons to enhance treatments that are currently directed at lower motor neurons and are already being tested in clinical trials. This clinical trial is funded by the ALS Canada Research Program.
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FORTITUDE-ALS: A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients with Amyotrophic Lateral Sclerosis (ALS)

Study Type: Therapeutic (Drug: CK-2127107); Phase 2
Status: Recruiting
Sites: Calgary, Hamilton, Toronto, Montreal (Neuro & CHUM), Quebec City
Sponsor: Cytokinetics
Collaborator: Astellas Pharma Inc.

As ALS progresses, people will find breathing more difficult due to their respiratory muscles becoming weaker and/or tiring too quickly. This Phase 2 study is designed to assess the safety and effectiveness of CK-2127107, a drug that activates muscles, at improving breathing in people living with ALS. CK-2127107 helps to increase muscle strength while also prolonging the amount of time before muscles become fatigued. In order to determine whether CK-2127107 is an effective treatment option, the research team will monitor participants’ slow vital capacity (SVC), which measures the volume of air exhaled without forced effort after a full breath. Overall functional decline using ALSFRS-R will also be measured.

CK-2127107 is a next generation of the drug tirasemtiv, which was previously shown to significantly slow SVC decline in a large Phase II clinical trial, but had a significant side effect profile of dizziness and nausea. CK-2127107 is modified to prevent these side effects.
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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 (eight more to come)
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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Safety and Efficacy Study of NP001 in Patients with Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation

Study Type: Therapeutic (Drug: NP001); Phase 2
Status: Recruiting
Sites: Montreal
Sponsor: Neuraltus Pharmaceuticals Inc.

This clinical trial tests NP001, an experimental treatment developed by Neuraltus Pharmaceuticals. NP001 is designed to regulate the body’s inflammatory response, a biological process believed to be disrupted in ALS. This Phase 2 study is designed to test the safety and effectiveness of NP001 in people living with ALS. NP001 is evaluated primarily using the ALS Functional Rating Score Revised (ALSFRS-R), which provides a measure of a person’s ability to complete key functions and daily living activities.
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MRI Biomarkers in ALS

Study Type: Observational
Status: Recruiting
Sites: Calgary, Edmonton, Vancouver, Toronto, Montreal
Sponsor: University of Alberta

The ability to accurately measure a person’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 study participants who are living with ALS. This study 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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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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Single and Multiple Dose Study of BIIB067 (Isis-SOD1Rx) in Adults With Amyotrophic Lateral Sclerosis (ALS)

Study Type: Therapeutic (Drug: BIIB067); Phase 1
Status: Recruiting
Sites: Toronto, Montreal
Sponsor: Biogen
*Due to the specificity of this treatment, this clinical trial is only open to individuals with a SOD1 mutation.

BIIB067 (also referred to as ISIS-SOD1RX) is a drug designed to reduce the production of a protein called superoxide dismutase 1 (SOD1). Mutations in the gene for SOD1 represent the second most common cause of the inherited (familial) form of ALS. As a result of mutation, SOD1 is believed to gain a toxic function that leads to the development of ALS. By reducing the amount of SOD1 in people who are living with ALS and have an SOD1 mutation, scientists may be able to reduce its toxic effect and as a result slow or halt disease progression. The aim of this Phase 1 study is to test the safety of BIIB067, as well as its success in reducing SOD1 levels.
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Methodology Study of Novel Outcome Measures to Assess Progression of ALS

Study Type: Observational
Status: Recruiting
Sites: Toronto, Montreal
Sponsor: Biogen

At the outset of a clinical trial, researchers and clinicians will often use a set of standardized tests to determine the baseline function of study participants. As the study progresses, those same tests can then be used to quantify changes in participants’ functional abilities over time, which is often referred to as an outcome measure and is how researchers monitor disease progression and evaluate the efficacy of a treatment. The goal of this observational study is to develop a new set of outcome measures (or tests) to monitor ALS progression that are more sensitive than the traditionally used Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). Researchers hope that this observational study will find new outcome measures that are reliable, easy to administer and quick to show evidence of change in people living with ALS, allowing for more effective testing of new ALS treatments.
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Non-invasive Ventilation in Amyotrophic Lateral Sclerosis (ALS) using the iVAPS Mode

Study Type: Therapeutic (Device: Non-invasive home ventilation)
Status: Recruiting
Sites: Montreal
Sponsor: Marta Kaminska

Home-based non-invasive ventilation is often used as the first step to treat breathing difficulties in people living with ALS. This study will compare the current standard mode of ventilation, called BiST, with the new, more technologically-sophisticated iVAPS mode. The goal of this study is to determine whether the iVAPS mode is superior to the BiST mode in terms of being more comfortable and easy for people to use, as well as improving people’s symptoms of hypoventilation, compliance (hours used per day) and physiologic parameters (such as daytime and overnight oxygen levels). The hope is that the iVAPS mode will reduce the number of required respiratory therapist interventions and alterations in the ventilator settings compared to the BiST mode.
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