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 in Alberta below:
ALN-SOD-ALS-2351
This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with amyotrophic lateral sclerosis (ALS) who have a mutation in a gene called the superoxide dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is.
The study is looking at several other research questions, including:
The effect the study drug has on specific biomarkers, which are molecules in the blood or in the fluid that surrounds the brain and spinal cord, known as cerebrospinal fluid (CSF)
How much study drug is in the blood and in the CSF, at different times
Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
What effects the study drug has on ALS symptoms
Ultrasound Study
This is a study that can be done during an in-clinic visit. The study is trying to determine whether diaphragm ultrasound at the bedside, which is non-invasive and only takes 5 minutes, can detect diaphragm weakness earlier and more accurately than pulmonary function testing and whether abnormal diaphragm ultrasound predicts which patients need to go on non-invasive ventilation (NIV, such as BiPAP). Researchers are also trying to better understand how diaphragm size and strength change over time in patients with ALS.
Please mention your interest to the Edmonton Clinic Nurse, and this can be scheduled when you come for a visit.
COMBAT-ALS (ibudilast)
A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.
QURALIS (QRL-201)
The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS
Canadian Neuromuscular Disease Registry
The Canadian Neuromuscular Disease Registry (CNDR) is actively recruiting all individuals diagnosed with ALS. The CNDR is a national registry partnering with ALS clinics across the country that strives to improve access to care and therapies for people with ALS and to increase the impacts of research in the Canadian healthcare system. The CNDR works with the community to provide essential clinical data related to people living with ALS for research and planning of future studies.
The CNDR is recruiting individuals using or using Edaravone, Riluzole, or Tofersen. The CNDR is collaborating with clinicians, scientists, patient organizations, and industry on studies to evaluate the safety, tolerability, and effectiveness of ALS therapies to support access to drugs for Canadians who may benefit.
Kennedy’s Disease – Spinal Bulbar Muscular Atrophy (SBMA)
Understanding the prevalence, clinical features, and genetics of spinal-bulbar muscular atrophy in people of Indigenous descent Recently, Edmonton researchers found that SBMA (Kennedy’s Disease) is much more prevalent than expected in people of Indigenous descent residing in western Canada.
SBMA is a genetic condition causing weakness of muscles in the face and limbs, starting in adulthood. Because it is “X-linked” it generally only affects males, but females who are carriers of the mutation may also develop a milder condition. It is believed that female carriers may be more likely to develop symptoms if they have inactivation of one X-chromosome more than the other.
The purpose of this research study is to learn more about the prevalence, clinical features, and genetics of SBMA.
CAPTURE ALS
The Amyotrophic Lateral Sclerosis (ALS) Neuroimaging Research Unit from the Department of Medicine is looking for healthy male control participants between ages 60-80 years to participate in CAPTURE ALS (Comprehensive Analysis Platform to Understand, Remedy, and Eliminate). University of Alberta Ethics ID: Pro00110972.
The aim is to learn more about ALS through observational data and biosamples. Control participants will have 2 visits, 8 months apart. Visits will contain biospecimen collection, MRI scans, and neurocognitive testing. This will take 4-5 hours and participants will also receive a monetary honorarium.
If you’re interested in participating, please contact tanushka@ualberta.ca or call 587-926-4815.
Scientific Studies
Investigators at the University of Alberta are using an innovative technology to understand why motor neurons in the spinal cord die in ALS. Drs. Monica Gorassini and Kelvin Jones are using large electrodes placed over the muscle to isolate the activity of single motor units. The activity of the single motor units in the muscle can tell us how the motor neurons in the spinal cord are behaving. The long-term goal is to use these motor neuron recordings for early diagnosis of ALS. These recordings can also tell us if treatments for ALS are working on the desired target - the motor neurons in the spinal cord.
STAFF CONTACTS
Kelsey Tymkow – tymkow@ualberta.ca 780-492-7690 if you would like to learn more or have any questions or concerns