View clinical trials related to Motor Neuron Disease.
Filter by:The purpose of the project is to obtain skin and adipose tissue samples from patients with ALS to develop new diagnostic and prognostic markers of the disease. These samples will be obtained when percutaneous endoscopic gastrostomy (PEG) is performed as part of their standard of care. Skin and adipose tissue samples will also be obtained from disease control subjects who require a PEG as part of their standard of care.
The study is being conducted to determine if DPS treatment for people with ALS and hypoventilation is associated with improved survival or diaphragm function. The primary objective of the study is to conduct a multi-center, randomized controlled clinical trial comparing standard of care (control) to diaphragm stimulator treatment with the NeuRx® Diaphragm Pacing System™ (DPS) with respect to survival. The secondary objective of the study is to conduct a multi-center, randomized controlled clinical trial to compare standard of care treatment (control) to DPS in ALS subjects with hypoventilation.
Autologous cell therapy in patients with ALS can stimulate neuroplasticity, modifying the neurodegenerative process and stops the clinical progression of disease.
Background: - Some people have a mutation in the C9ORF72 gene that causes amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). The mutation causes a small piece of DNA to repeat itself thousands of times. The C9ORF gene mutation mostly occurs in families. In those families, some persons have ALS and others have FTD. Occasionally the C9ORF gene mutation occurs in persons without a family history. Researchers want to understand how this gene causes different diseases. They will study how symptoms caused by the C9ORF gene develop and change over time. They will measure symptoms that occur in ALS and in FTD. In particular, they will measure strength, ability to move, thinking, and memory. They will also see if other tests are associated with progression of disease. These tests, called biomarkers, may help detect or measure C9ORF72 disease in the future. Objectives: - To understand how symptoms change over time in people with mutations in a gene called C9ORF72, which causes ALS and FTD. Eligibility: - Adults over age 18 who have this genetic mutation Design: - Participants will have up to 4 in-person visits and 3 telephone interviews over 3 years. Each in-person visit may take place over several days. They may be either inpatient or outpatient visits. - At each visit, participants will undergo a series of brain, language, and behavior tests. These will include: - Magnetic resonance imaging (MRI) of the brain. This uses magnets, radio waves, and computers to produce detailed pictures of the brain. - Collecting spinal fluid. The clinician will make the participant s back numb and then insert a needle to collect fluid. <TAB>- Blood samples will be taken. <TAB>- Participants will be asked to perform several language and movement tests. <TAB>- Small skin samples will be taken on one visit - Between visits, participants will answer questions about their health over the phone 3 times.
A new strength measurement device called Accurate Test of Limb Isometric Strength (ATLIS) was developed to precisely and conveniently measure static limb strength in patients with ALS. The investigators will compare ATLIS data with data from two commonly used ALS outcomes measures, the ALS Functional Rating Scale-Revised (ALSFRS-R) and slow vital capacity (SVC) in a prospective, longitudinal study. All three outcomes measures will be performed on 100 subjects collected preferably at bi-monthly clinic visits during the study period.
This is a multicenter, 15-month study evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale (Revised) (ALSFRS-R) score in up to 33 subjects with Amyotrophic Lateral Sclerosis (ALS).
The primary objective of the trial is to investigate the survival time (the time from randomization until death or end of the trial) compared between control group and experimental group. This is a prospective, multicenter, randomized, stratified, parallel-group, double-blind trial comparing placebo with 1 mg/d rasagiline as add-on therapy to 100 mg riluzole in amyotrophic lateral sclerosis (ALS) in 250 enrolled patients. For entry, the El Escorial Criteria for the diagnosis of ALS will be used. The patients have to be stable on riluzole at least 4 weeks prior to randomization.
This research program will focus on gait initiation and postural control in Amyotrophic Lateral Sclerosis (ALS) patients, by comparing patients with vs without postural instability (but also in comparison to controls), by using a multidisciplinary approach which combines neurophysiological and neuroimaging analyses. After clinical evaluation, two groups of ALS patients, defined upon the feature of postural instability, and one group of healthy subjects (n=25 for each group of patients and 20 for healthy subjects) will be included in the study. The neurophysiological evaluation will be performed through a gait initiation assessment, which will alow us to collect biomechanical and electromyographical data, such as the braking index. The neuroradiological evaluation will include first an fMRI analysis, a study of specific circuits in networks will be performed which will provide the first description of neural network dynamics associated with the preparation and execution of movement in ALS patients. The investigators major research hypothesis is: - By comparing patients with vs without postural instability, but also in comparison to controls, the investigators main research neurophysiological hypothesis is that patients with postural instability will display an impaired braking. - This impaired braking could be partly explained by a dysfunction and/or lesion of the basal ganglia and brain stem structures, corresponding to the investigators main neuroradiological hypothesis.
GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system. Neurological diseases are multisystem, multifactorial, and single target drugs are ineffective. Genervon's Master Regulators play a significant role in embryonic/fetal nervous system development and are potent disease modification drug candidates modulating many pathways including inflammation, apoptotic, and hypoxia. The study drug is an regulatory peptide with a sequence identical to one of the active sites of human Motoneuronotrophic Factor and is manufactured by solid phase synthesis. Pre-clinical research indicates it to be a neuro-protective agent in animal models of ALS, motorneuron diseases, PD, other neuro-degenerative diseases and stroke. GM604 controls and modulates over many known and significant ALS genes with positive effects interactively and dynamically through multiple pathways, and up to twenty-two biological processes, including neuro-protection, neurogenesis, neural development, neuronal signaling, neural transport, and other processes. GM6 is not a cocktail of drugs, but one master regulator peptide drug that functions through multiple pathways. Genervon hypothesized that studying the biomarkers of protein expressions of these ALS genes such as superoxide dismutase 1 (SOD1) and the protein expression of substances such as tau, neurofilament - heavy (NF-H), Cystatin C which were indications of degeneration of neuron in the CSF collected from ALS patients will provide information of the possible GM604's mechanisms of action in treating ALS. 1. This pilot trial is designed to test proof of principle, i.e. determine if a 2-week IV bolus treatment with this agent can (1) change ALS protein expression (target biomarkers and efficacy biomarkers) after treatment (2) have preliminary effects measures of ALS disease clinical progression. Study Objectives are: 1. To test the safety and tolerability of GM604 in a population of ALS patients. 2. To test for changes in ALS biomarkers before and after treatment. 3. To determine preliminary effects of injections of GM604 on measures of ALS disease biomarkers and clinical progression
The purpose of this research is to find out if mexiletine is safe and effective in people with Amyotrophic Lateral Sclerosis (ALS). In this trial, participants will be taking either 300 milligrams per day of mexiletine, 900 milligrams per day of mexiletine or placebo (non-active study drug). The safety and efficacy of these doses will be compared to see if one dose is better than the other.