View clinical trials related to Amyotrophic Lateral Sclerosis.
Filter by:This study will investigate the efficacy, safety, tolerability and pharmacokinetics (PK) of multiple intravenous infusions of NX210c, at two dose levels, in patients with Amyotrophic lateral sclerosis (ALS).
Amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease, affects motor neurons, causing progressive muscle atrophy and weakness. Current treatments are ineffective, with most patients dying within 3-5 years of diagnosis. The disease's exact cause is unclear, but factors such as oxidative stress and protein abnormalities are implicated. Abnormal protein deposits and neurotoxic factors in the brain and spinal cord contribute to ALS pathology. Recent research on the brain's glymphatic-lymphatic system suggests impaired waste clearance may exacerbate ALS. Restoring drainage connections between cervical lymphatic vessels and veins could potentially alleviate neurodegenerative disease progression.
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
This is a single-session, case-control study that incorporates digital tools for assessing speech and motor function in motor neuron disease. Patients with motor neuron disease (including amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), and progressive muscular atrophy (PMA)) and age-matched healthy controls will be enrolled. Subjects will complete a speech and handwriting assessment during the study visit on a tablet computer (BioSensics LLC, Newton, MA). We will explore whether these digital biomarkers are sensitive to functional disease severity as reported by the ALS Functional Rating Scale - Revised (ALFRS-R) [1]. We will also compare assessment data between the patient and control groups.
The proposed study is an Open-Label, Single-Dose Study to Assess the Safety, and Pharmacodynamics (PD) signals of MRG-001 in Patients with Amyotrophic Lateral Sclerosis (ALS). MRG-001 will be administered subcutaneously 3 times per week for 2 weeks. This cycle will be repeated for 3 months. In total, patients are expected to receive 18 injections over the span of 3 months.
This study aims at evaluating efficacy and tolerability of an ultra-high-caloric, fatty diet (UFD) compared to placebo in patients with amyotrophic lateral sclerosis (ALS).
The goal of this clinical trial is to learn about the effect of communicative interaction on verbal communication in people with amyotrophic lateral sclerosis (ALS) and age-matched speakers. The question is, What are the effects of communicative interaction on verbal communication in people with ALS? Participants will read words and sentences while they are in a solo setting and interactive setting.
Amyotrophic lateral sclerosis (ALS) is a progressive neurological disorder characterized by selective death of upper and lower motor neurons, which leads to severe disability and fatal outcomes. One of the major hallmarks of ALS is the denervation of neuromuscular junctions (NMJs), which is one of the earliest events seen in ALS patients and mouse models of ALS. Under healthy conditions, glial cells called Perisynaptic Schwann Cells (PSCs) have a key role in regulating the stability and maintenance of NMJs, but they only participate in NMJ repair once denervation occurs. Denervation and the subsequent decline in synaptic activity triggers a loss of muscarinic acetylcholine receptors (mAChRs) in the PSC, and the resulting decrease in mAChR-mediated gene expression drives the "repair mode" of the PSC. In assessing the NMJ under conditions of ALS, a scarcity of process extensions in PSCs was observed for months prior to disease onset in the superoxide dismutase 1 (SOD1) mouse model of ALS, indicating inadequate glial repair. Collectively, these preclinical findings support the hypothesis that dampening glial mAChRs will restore the anticipated "repair" response of PSCs in the NMJ. Hence, the use of a selective M3 muscarinic receptor antagonist, Darifenacin, as a disease-modifying therapeutic in familial and sporadic ALS could improve NMJ function, resulting in a beneficial impact on the autonomy and quality of life of ALS patients. The purpose of the current Phase 2 trial is therefore to test the safety, tolerability, and pharmacology of Darifenacin in patients with ALS. Specifically, 30 eligible subjects between 18 and 85 years of age will take 7.5 mg of darifenacin or placebo daily (by mouth) for two weeks followed by an increased dose of 15 mg for the next 22 weeks. The trial will evaluate the effects of this medication on several outcome measures including patient safety, physical and neurological function, muscle strength, depression levels, and NMJ innervation of patients with ALS. Detailed clinical assessments will be conducted at regular intervals throughout the study in order to achieve these objectives.
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder that impairs motor neurons, with a life expectancy of 2 to 7 years after diagnosis. ALS manifests as 'spinal' when it primarily affects limbs, or 'bulbar' when it impairs speech and swallowing. The disease progressively weakens all skeletal muscles, causing respiratory issues and increased risk of lung infections due to ineffective coughing. Mechanical cough assistance via In-exsufflation therapy/ mechanical in-exsufflator devie (INEX/MI-E) applies positive and negative airway pressures non-invasively to improve coughing. However, MI-E may fail in some ALS patients due to airway collapse, often related to brainstem muscle dysfunction.Research by Andersen et al. in 2017 highlighted that during MI-E, ALS patients often experience adverse laryngeal movements, which can obstruct airways and reduce the therapy's effectiveness. To combat this, they suggested individualized MI-E settings to minimize airway collapse. Modern MI-E devices, such as the EOVE-70, offer adjustable positive expiratory pressure (PEP) between cycles to potentially enhance airway stability and coughing efficiency. The current study focuses on the impact of PEP during therapy pauses on the peak expiratory flow rate in ALS patients, which could lead to improved therapeutic outcomes.
In response to oxidative stress, cells activate the Nrf-2 pathway, which induces translation of its target genes and corresponding proteins involved in the antioxidant response. This explains the interest in the Nrf-2 pathway in the pathophysiology of Amyotrophic lateral sclerosis (ALS), supported by the results of several studies and the modulatory effect of TDP-43 on the Nrf-2 pathway. Since both TDP-43 and Nrf-2 proteins are present in the peripheral blood mononuclear cells (PBMC) of ALS patients and may be correlated with disease progression, the investigators wish to explore their relationship and their application in the clinic as potential blood biomarkers for ALS.