View clinical trials related to Motor Neuron Disease.
Filter by:The goal of this clinical trial is to evaluate the safety and efficacy of a single intrathecal injection of RJK002 in patients with Amyotrophic Lateral Sclerosis (ALS). The main questions it aims to answer are: - The safety, tolerability, and preliminary efficacy of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS) - The adeno-associated virus (AAV) viral load, changes of biomarkers in serum and cerebrospinal fluid (CSF), and electromyography (EMG) motor unit counts in subjects with ALS treated with a single intrathecal injection of RJK002. Participants will receive a single intrathecal administration of investigational product and a systemic immunomodulatory regimen. There will be 3 cohorts: 6E13 vg/person (6 mL), 1.2E14 vg/person (12 mL), and 2.4E14 vg/person (24 mL). 3 subjects will be enrolled in each dose cohort. The dose level will be escalated sequentially from low to high.
Amyotrophic Lateral Sclerosis (ALS) is the most common motor neuron disease in adults. This longitudinal study involves three cohorts of participants: patients with sporadic or hereditary ALS, asymptomatic individuals carrying pathogenic mutations responsible for ALS, and control subjects. In this study, a skin biopsy and blood sampling will be performed at the initial visit (M0), then at M12 (+/- 2 months) for patients, and at M36 (+/- 12 months) for asymptomatic carriers of pathogenic mutations. The aim of this research is to model ALS pathology using fibroblasts derived from the patients' skin biopsies.
Many people living with neurodegenerative conditions like dementia, motor neuron disease (MND), multiple sclerosis (MS), and Parkinson's disease (PD), suffer from speech problems. Using common digital technologies such as smartphone apps, the investigators can record and analyse speech in detail to provide new information for people living with these conditions, researchers, and healthcare professionals. This study will investigate the use of these digital speech recordings to help diagnose and monitor these conditions. To take part, participants will have either a diagnosis of dementia, motor neuron disease, Parkinson's disease or Multiple Sclerosis, OR they will have no diagnosis of a neurological condition. Researchers will compare people with a diagnosis of a Neurological condition to those without.
This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function. The study consists of 2 periods: a treatment period when participants will receive one of three ARGX-119 doses or placebo and an extension period when all participants will receive the same dose of ARGX-119. Participation in the study will last up to approximately 100 weeks.
The investigators aim to elucidate characteristics of structural and functional brain connectivity in patients with amyotrophic lateral sclerosis (ALS) and pseudobulbar affect (PBA) using diffusion kurtosis imaging (DKI) and magnetoencephalography (MEG).
Amyotrophic lateral sclerosis (ALS) is one of the most lethal neurodegenerative diseases, with most patients dying from respiratory failure 3-5 years after the onset. The purpose of this study is to explore the efficacy and safety of nerve growth factor (NGF) encapsulated with 2-methacryloyloxyethyl phosphorylcholine (MPC) nanocapsules in the treatment of ALS patients.
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.