A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS) (FORTITUDE-ALS)
Therapeutic Study (Drug: CK-2127107); Phase 2
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.
A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS) (Pimozide2)
University of Calgary
Therapeutic Study (Drug: pimozide); Phase 2
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.
MRI Biomarkers in ALS
University of Alberta
A significant hurdle to finding an effective treatment(s) for ALS has been an inability to accurately measure brain degeneration in humans. Advanced magnetic resonance imaging (MRI) techniques hold promise in this respect, and may assist in aiding diagnosis and the efficient testing of new drugs. Different MRI features of brain degeneration will be measured in a large sample of patients with ALS. The study will operate within the Canadian ALS Neuroimaging Consortium (CALSNIC). CALSNIC is a clinical research platform comprised of ALS clinics with standardized clinical and neuroimaging protocols.
Phenotype, Genotype & Biomarkers in ALS and Related Disorders
University of Miami (Site is in Edmonton)
The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders.
A Registry-Based Clinical Trial of Pimozide in Patients with Neuromuscular Junction Transmission Dysfunction due to ALS
University of Calgary
Active (no longer recruiting)
The only approved treatment to slow the progression of ALS is called Rilutek® (riluzole) which has only a modest effect and has been shown to increase survival by a few months.
Muscular dysfunction present in people with ALS is caused by nerve breakdown and a dysfunction in the communication between the muscles and the nerves. The area where these communications occur is called the neuromuscular junction. Some recent studies have focused on using different medications to enhance communication at the neuromuscular junction with the goal of improving muscle function as a result. This approach is unproven but may help to slow the progression of the disease.
Pimozide is a medication that has been demonstrated to enhance communication at the neuromuscular junction in fish and mice. This study will look at whether Pimozide may help to slow the progression of ALS and how much medication needs to be taken to have an effect.