View clinical trials related to Motor Neuron Disease.
Filter by:The purpose of this research is to investigate the validity of a previous clinical trial named EH301, which showed beneficial effects of anti-oxidant therapies in patients with amyotrophic lateral sclerosis (ALS). If validated by this study, providing over-the-counter anti-oxidants would be a simple, low risk, low-cost approach to significantly slow or stop the progression of ALS, for which currently no effective treatment exists. It is currently thought that oxidative stress is a major cause of ALS. The study investigators are therefore planning to expand the original scope of the previous trial by including anti-oxidants at high doses that were not previously used. All of these compounds are considered safe.
BACKGROUND/RATIONALE: The paucity of biomarkers for the diagnosis and monitoring of patients affected by Amyotrophic Lateral Sclerosis (ALS) is one of the greatest concerns in ALS clinics and research. Phenotypic signs, electrophysiological test and clinical scales are currently used for ALS diagnosis and follow up before and after treatments. Nowadays, the diagnosis and differential diagnosis used to discriminate ALS from other comparable neurodegenerative diseases, are time-consuming and complex processes that reduce the time for a prompt intervention. Thus, the scientific community is asked to strive for new, measurable, fast and objective biomarkers for the diagnosis and stratification of patients. Saliva is a complex biofluid composed of bioactive molecules that can be collected by means of a non-invasive procedure. The possibility to simultaneously monitor all the variations in the endocrine, electrolytic and metabolic messengers in saliva has recently suggested its use for the diagnosis of complex diseases, like neurodegenerative diseases, but only limited information are available on the potential of saliva as alternative carrier of ALS biomarkers. OBJECTIVES: The aim of the present project is to optimize an innovative, non-invasive and fast procedure for the ALS onset and for the stratification of ALS patients, taking advantage of the sensitivity of Raman Spectroscopy (RS) and of accessible saliva. Fondazione Don Gnocchi (FDG) preliminary results on a small cohort of subjects demonstrated the feasibility of the methodology and the ability of LABION protocol to obtain a reproducible Raman fingerprint of saliva that can be used for the discrimination of healthy subjects, ALS patients and subjects affected by other types of neurological diseases. METHODS: Starting from FDG preliminary results, the biochemical composition of saliva in patients with diagnosed ALS will be evaluated and statistically compared with the one obtained from age and sex-matched healthy subjects and from patients affected by other neurological diseases (Parkinson's and Alzheimer's diseases). Moreover, an intra-group ALS clustering will be analysed in order to verify a different Raman fingerprint obtained from ALS patients with a bulbar or spinal onset. The collected Raman data will be processed using a multivariate analysis approach through Principal Component Analysis - Linear Discriminant Analysis (PCA-LDA). The classification model will be created using cross-validation and subset validation. Thanks to RS, the overall composition of saliva will be established with minimal sample preparation, providing comprehensive biochemical fingerprint of the sample. In parallel, routine salivary parameters will be measured including viscosity, pH, total protein and carbohydrates concentration, amylase and pepsin, cortisol and Chromogranin A. EXPECTED RESULTS: By the end of this study, the investigators expected to verify the possibility to use the Raman salivary pattern as new promising biomarker for ALS diagnosis and progression to be related with clinical scales for the personalized and fine tuning of the therapeutic approach. The intent of this project is to create a classification model able to: 1. Determine the ALS onset 2. Discriminate the signal obtained from ALS patients from the one collected from other neurodegenerative diseases 3. Stratify ALS patients into bulbar and spinal onset 4. Correlate the Raman data with clinical and paraclinical scales used nowadays for ALS diagnosis and monitoring
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records regarding ALS
There is currently no effective treatment in ALS. Oxidative stress, probably interacting with other neurodegenerative processes, is hypothesized to play a leading role in pathogenesis. These include mechanisms that promote glutamate excitotoxicity, mitochondrial dysfunction and axonal dysfunction. In a transgenic mouse model of fALS that develops a disease with a clinical phenotype similar to ALS, dietary vitamin E supplementation delayed disease onset and slowed progression, although it did not prolong survival. When used as an experimental therapy in human trials, vitamin E did not affect survival significantly, but possibly slowed ALS progression. Two large, prospective epidemiologic studies suggest that longterm use of vitamin E supplements could be inversely associated with risk of ALS or ALS death. In another study, higher baseline serum α-tocopherol was associated with lower subsequent risk of ALS. A modest, non-significant protective effect from supplementation was seen in subjects with baseline serum α-tocopherol levels below median levels. In the current study, we aim to investigate the effects of tocotrienols in patients with ALS, particularly in delaying disease progression as well as assessing its safety profile in this group of patients.
This study is being conducted to help the investigators better understand how the new FDA approved medication Edaravone (also known as Radicava) works in subsets of patients with ALS. The investigators are also trying to understand if there are specific ALS patients, with different presentations of ALS, who might benefit most from this medication. Also, the investigators are following specific biomarkers to determine the optimal treatment duration in patients with different forms of ALS There is no study medication being offered in this trial. Edaravone is prescribed as part of regular care. In this trial we are collecting blood, urine, and spinal fluid samples in ALS patients who are taking Edaravone and ALS patients who are not taking Edaravone to measure certain markers that could indicate why the drug may be working in a specific type of ALS.
Multicenter, randomized, double-blind, placebo controlled study to assess the tolerabilty and efficacy of CuATSM in patients with ALS/MND. Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment.
It is an open label study to evaluate safety, tolerability and brain microglia response in participants with ALS following multiple doses of BLZ945.
A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.
Amyotrophic lateral sclerosis (ALS) is a kind of motor neuron degeneration disorder without effective therapy. This registered cohort study will provide further insights into the clinical course of ALS, and investigate disease-relative risk factors and the genetic background of Chinese ALS patients.
This will be a multistate, multicenter clinical study to determine the efficacy and safety of medical cannabis for a wide variety of chronic medical conditions.