View clinical trials related to Amyotrophic Lateral Sclerosis.
Filter by:A disease called Amyotrophic lateral sclerosis (or ALS), which leads to difficulty swallowing, breathing, and movement, has been found to be higher for those serving in the military than in the general population. There are approximately 4,200 Veterans with ALS and roughly 1,000 new cases each year. When doctors attempt to determine the degree to which an ALS patient is suffering from the disease, they apply tests that are "graded" by experts. However, this approach to testing patients may not be very accurate. Researchers aim to use a system called DESIPHER to "listen" to ALS patients and find speech mistakes related to their condition. Researchers believe that, by detecting different types of errors, DESIPHER serves as a new kind of indicator of medical problems such as difficulty breathing or swallowing, without human "grading". This may also lead to a better system for automatically understanding ALS patients' speech.
The first objective is to find some biomarkers, or a profile of biomarkers of ALS to help to diagnosis. The second objective is to better understand the pathogenesis of this disease by the exploration of muscle, blood and satellite cells metabolomes and transcriptomes.
Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease with a poor prognosis that occurs in adults 64 years on average. Its prevalence is 4 to 6/100 000 inhabitants. Swallowing disorders occur during evolution and involve the prognosis of patients in the short term by the association of dysphagia with severe malnutrition, and aspiration. The issue of phoniatric monitoring is to detect early onset of the swallowing disorders to develop strategies for respiratory protection, food adapted to disturbances, and speech therapy. The objective of this study is to compare the tongue force in patients with amyotrophic lateral sclerosis at the time of diagnosis and at the onset of swallowing disorders compared to healthy subjects, with the dynamic palatography device developed in the Laboratoire Parole et Langage (UMR 7309, CNRS-Université Aix-Marseille, Aix-en-Provence), which allows the measurement of the strength and duration of the pression of the tongue on the palate.
This first-in-human, double-blind, placebo-controlled Phase I study will be conducted in participants with amyotrophic lateral sclerosis (ALS) to explore safety, tolerability, and pharmacokinetic (PK) properties of GDC-0134. It will include three components: a Single-Ascending-Dose (SAD) stage, a Multiple-Ascending-Dose (MAD) stage, and an Open-Label Safety Expansion (OSE) stage.
Amyotrophic lateral sclerosis (ALS) is a fatal disease leading to motor neuron degeneration and progressive paralysis. Other studies have revealed defects in skeletal muscle even in absence of motor neuron anomalies, focusing on acetylcholine receptors (AChRs) and supporting the so-called "dying-back" hypothesis. Outcome of this study will be to understand if the endocannabinoid palmitoylethanolamide (PEA) can reduce the rundown of AChRs currents in ALS muscle, and if it can modify ALS patients' clinical and electrophysiological parameters.
The primary objectives of Parts A and B of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending doses of tofersen in adults with ALS and a documented superoxide dismutase 1 (SOD1) mutation. The primary objective of Part C of this study is to evaluate the clinical efficacy of tofersen administered to adults with ALS and a confirmed SOD1 mutation. The secondary objective of Parts A and B of this study is to evaluate the effects of tofersen on levels of total SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, pharmacodynamic (PD), and biomarker effects of tofersen.
The primary objectives of the study are to estimate and rank-order the longitudinal standardized mean changes over 6 months and over 12 months, for a set of outcome measures administered to participants with amyotrophic lateral sclerosis (ALS), in order to identify measures that are more sensitive to disease progression than Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R). The secondary objectives of this study are: To evaluate the test-retest reproducibility of each outcome measure; To determine correlations between 6 and 12-month changes in all exploratory measures with 18 and 24-month changes in ALSFRS-R and survival; To assess correlations between/among the various measures; To obtain biological samples in order to identify molecular correlates to the clinical measures and to further characterize previously identified and novel molecular biomarkers of disease progression for incorporation into future clinical studies.
The project focuses on C9orf72, the most frequent genetic form of frontotemporal dementias (FTD, or frontotemporal lobar degeneration, FTLD) and amyotrophic lateral sclerosis (ALS). FTD is the second commonest cause of degenerative dementia in presenium after Alzheimer's disease. Behavioural and cognitive impairments progressively lead to dementia. ALS produces progressive muscle weakness leading to the death in 2 to 4 years. Since 2006, major discoveries have linked FTLD and ALS: 1. TDP-43 aggregates in neurons and 2. C9orf72 mutations is a major genetic cause in both disorders. Two major pathological subtypes are now defined in FTD, FTD-TDP and FTD-TAU. C9orf72 mutations (associated to FTD-TDP) are the most frequent genetic causes of FTD (15%), FTD-ALS (65%) and ALS (40%). FTD is difficult at an early stage; and no clinical, biological or imaging features can predict the underlying pathology in living patients. Therapeutic perspectives emerged against tau aggregation, progranulin deficit and C9orf72 expansion (antisense). Presymptomatic carriers of genetic FTD would benefit, before onset of symptoms, from these therapeutic that would delay or prevent the disease. At this step, it becomes crucial to develop markers to know how many years before symptoms, does the pathological progress begin, to treat the patients at the most early stage of the disease. Markers are also needed to predict the pathology (FTD-TDP/FTD-tau) in patients that will be eligible for trials targeting specific pathological lesion.
The purpose of the study is to evaluate the safety of combining phospholipids with medicinal plants for treatment of patients with amyotrophic lateral sclerosis (ALS)
The objective is to compare the efficacy and safety of masitinib in combination with riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).