Clinical Trials 

 

A Clinical Trial of Pimozide in Patients With Amyotrophic Lateral Sclerosis (ALS) (Pimozide2)

University of Calgary
Therapeutic Study (Drug: pimozide); Phase 2
Not yet open for recruitment (expected Oct. 2017)

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
Observational Study
Currently Recruiting

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)
Observational Study
Currently Recruiting

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.

Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices after Treatment for a Year (VITALITY-ALS)

Cytokinetics
Active (no longer recruiting)

This study is to assess the effect of tirasemtiv versus placebo on respiratory function in patients with ALS.