ALS Clinical Trials
Full Name: A Phase 3, Randomized, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis
Description: This is a multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of 1200 mg arimoclomol (400 mg TID) over a 76-week treatment period with patients who have Amyotrophic Lateral Sclerosis (ALS) and, more specifically look to find if arimoclomol extends your ability to breathe on your own, improves survival, functional health and safety as assessed with CAFS (Combined Assessment of Function and Survival). The study visits consists of both in-person and remote visits (telephone calls).
Principal Investigator: Shafeeq Ladha, MD
ALS Post-Mortem Tissue Bank
Full Name: ALS Post-Mortem Tissue Bank
Description: The purpose of this program is to create a postmortem (after death) tissue bank of spinal cord, muscle, and brain tissue samples from both amyotrophic lateral sclerosis (ALS) patients and those without the disease (control subjects). Tissue donation provides an important and vital resource toward the advancement of research in ALS and other neurodegenerative diseases.
The intention of this tissue bank is to support efforts by the research community, enhance collaborations between clinicians and basic scientists, and collate and share information on how to better understand, prevent, diagnose, and treat ALS and other diseases.
Participants in this program must provide consent to have their tissues stored at Barrow Neurological Institute for use in current and future research projects.
Principal Investigator: Robert Bowser, PhD
Sponsor: Target ALS Foundation
Full Name: AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) (CENTAUR)
Description: The CENTAUR trial will be a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and able to slow decline in function as measured by the ALSFRS-R.
The trial will also assess the effects of AMX0035 on muscle strength, vital capacity, and biomarkers of ALS including markers of neuronal death and neuroinflammation.
Sponsor: Amylyx Pharmaceuticals, Inc.
Biofluid Biomarker Addition to Postmortem Tissue Bank
Full Name: Biofluid Biomarker Addition to Postmortem Tissue Bank
Description: The purpose of this study is to collect, process, and store biospecimens and clinical information premortem (before death), and link this information to postmortem (after death) tissue samples for current and future research in amyotrophic lateral sclerosis (ALS), motor neuron diseases, and other medical conditions.
Participants in this study complete at least one sample collection visit, which includes blood collection and assessments that measure motor neuron and cognitive functions. Additional visits and procedures, such as cerebrospinal fluid (CSF) collection, may be completed if the participant chooses to undergo the optional portions of the study.
Principal Investigator: Lyle W. Ostrow, MD, PhD
Site Principal Investigator: Robert Bowser, PhD
Sponsor: ALS Association, Target ALS
Magnetic Resonance Imaging Cytography in Patients with ALS
Full Name: BNI-ALS-002 Magnetic Resonance Imaging Cytography in Patients with ALS
Description: The purpose of this study is to determine whether magnetic resonance imaging (MRI) cytography (a type of noninvasive body scan) is useful in establishing disease severity in individuals with amyotrophic lateral sclerosis (ALS).
MRI cytography will be compared to measures typically taken at clinic visits that measure motor neuron and cognitive functions to see if it may be related to disease status.
In most cases, participants will attend a one-time clinic visit, though the option of two separate study visits may be available.
Principal Investigator: Chad Quarles, PhD
Sponsor: Flinn Foundation
Full Name: Efficacy, Safety and Tolerability of CK-2127107 in Patients With ALS (FORTITUDE-ALS)
Description: The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.
Full Name: Topiramate as a Disease Modifying Therapy for Cryptogenic Sensory Peripheral Neuropathy in Metabolic Syndrome (CSPN)
Description: The TopCSPN study is a multicenter, randomized, parallel-group, double-blind, placebo-controlled, Phase II clinical trial. The purpose of this study is to determine if a target daily dose of 100 milligrams of oral topiramate slows the progression of neuropathy and improves quality of life for adults age 18-80 with cryptogenic sensory peripheral neuropathy (commonly called idiopathic neuropathy), who also have symptoms of metabolic disease (such as high cholesterol, elevated blood sugar, high blood pressure, and abdominal obesity) at screening.
A variety of assessments and questionnaires will be performed during study visits to evaluate if topiramate improves neuropathy symptoms. These study visits will be conducted over a 96-week (24-month) treatment period, with in-person visits occurring every 16 weeks and monthly follow-up phone calls in between visits.
Sponsor: The National Institute of Neurological Disorders and Stroke (NINDS)
Sublingual Disintegrating Riluzole Tablets
Full Name: Expanded Access Protocol for BHV-0223/Zydis SL (Sublingual Disintegrating Riluzole Tablets)
Description: Oral riluzole is FDA-approved for the treatment of amyotrophic lateral sclerosis (ALS). These standard tablets may be difficult for people with ALS to take as directed, because of trouble swallowing pills, the requirement for fasting (not eating) around each dose, or some other reason.
By contrast, when BHV-0223 is placed under the tongue, it rapidly dissolves and does not require swallowing. In addition, because it is absorbed under the tongue the effect of food in the stomach may be less of an issue. Finally, clinical research studies demonstrated that the lower dose of riluzole in BHV-0223 administered under the tongue achieved similar blood levels to a standard oral riluzole tablet. This means that BHV-0223 may pose a lower risk of riluzole associated side-effects, such as liver test abnormalities.
BHV-0223 will be made available in this program at no cost to participants, until the time that it becomes available by prescription.
Principal Investigator: Jeremy Shefner, MD, PhD
Sponsor: Biohaven Pharmaceuticals, Inc.
Jacquelyn Nicolari, RN
Fluid Biomarkers with Deep Phenotyping in Patients with ALS
Full Name: Fluid Biomarkers with Deep Phenotyping in Patients with ALS
Description: This study is being performed to provide researchers with clinical information, combined with a wide range of biospecimens, to help identify biomarkers associated with amyotrophic lateral sclerosis (ALS), and to assess therapeutic targets. Participants will be asked to participate in four clinic visits and three telephone follow-up calls.
At each clinic visit, participants will have blood (including DNA and RNA) collected and will be evaluated with assessment tools focusing on motor neuron and cognitive (memory and thinking) functions.
Of the four visits in this study, three will also involve the collection of cerebrospinal fluid (CSF), and two will involve magnetic resonance imaging (MRI) cytography (a type of noninvasive body scan). Overall, participants may be in the research study for about 18 months.
Principal Investigator: Shafeeq Ladha, MD
Sponsor: ALS Association
Frequency of Genetic Abnormalities in ALS Patients
Full Name: Frequency of Genetic Abnormalities in ALS Patients
Description: This study is being performed to determine how many participants may have genetic forms of amyotrophic lateral sclerosis (ALS). It may also help identify which genes most commonly relate to genetic ALS, with or without a family history of the disease.
Blood samples from subjects with amyotrophic lateral sclerosis will be used to perform genetic tests in a research laboratory. The results of the testing will be used to determine how common genetic ALS is, and if there are any basic features of the disease that can predict a specific gene or genes as the cause of ALS.
Participants will be asked to complete a one-time blood draw at a one-time clinic visit.
Principal Investigator: Shafeeq Ladha, MD
Sponsor: Mayo Clinic Laboratory Department of Neuroscience
Speech Analysis in ALS Patients
Full Name: Speech Analysis in ALS Patients with and without Cognitive Abnormalities
Description: The purpose of this study is to determine whether speech and language measures can be used to detect cognitive changes, such as alterations in memory or thinking, in patients with amyotrophic lateral sclerosis (ALS).
By detecting cognitive changes earlier in the ALS disease course, we may be able to implement more tailored clinical trial designs, as well as provide better clinical care to ALS patients.
There are five visits required for this study. These are scheduled at the same time as regularly scheduled visits to the Barrow ALS Clinic, and occur approximately every 3 months. Participants also complete speech and language assessments at home each week. Overall, participants may be in the research study for about 12 months.
Principal Investigators: Jeremy Shefner, MD, PhD, Julie Liss, PhD, and Visar Berisha, PhD
Sponsor: Dignity Health and Arizona State University Collaborative Strategic Initiatives Program 2018