View clinical trials related to Motor Neuron Disease.
Filter by:The primary objective of the study is to evaluate the feasibility of eliciting continuous narrative speech in different neurodegenerative and psychiatric indications, using remote, self-administered speech tasks, as measured by the average length of speech elicitation for each speech task during the first week of self-assessment. Secondary objectives include (1) evaluating the reliability of speech tasks in the remote self-administered setting, as measured by the intra- and inter-subject variance; (2) accessing the adherence of speech tasks in this setting, as measured by the subject average fraction of days during the first week, where at least one task response is submitted; (3) evaluating the feasibility of using speech tasks in the setting of a telemedicine videoconference, as measured by the average length of speech elicited in each group; (4) evaluate whether a set of acoustic and linguistic patterns can detect each indication, compare to either a control group or all other indications, as measured by the area under the receiver operating characteristic curve (AUC), sensitivity, specificity and Cohen's kappa of the relevant binary classifier; (5) evaluating how the performance of such algorithms can be impacted by speaker and environment covariates, as measured by the Kendall rank correlation coefficient of the AUC of each classifier and each of age group, gender and speech-to-reverberation modulation energy ratio.
Amyotrophic lateral sclerosis/ Motor Neurone Disease (ALS/MND) is a rare and invariably fatal neurological disease. ALS/MND has a terribly high burden on patients, family and carers, and carries great socioeconomic burden. Current best treatment options are expensive and attempt to control disease progression and manage symptoms while offering no cure. Better treatments are wanting. Monepantel is a well-known veterinary drug, registered as a livestock wormicide in 39 countries. The industry collaborator, PharmAust Ltd, has found that monepantel shows off-target activity, inhibiting a cellular signaling system controlled by mammalian target of rapamycin (mTOR). This stops cancer growth and reduces protein accumulation in diseased cells. PharmAust has already tested monepantel in humans and pet dogs in Phase I and II anti-cancer clinical trials, respectively, in Australia. Data from these trials show that monepantel treatment associates with an exceptionally high safety profile, mTOR signaling inhibition and anticancer activity. Abnormal protein accumulation within motor neurons of the brain associates with the cause of ALS/MND. Inhibition of the mTOR signaling pathway slows disease progression in certain preclinical models of ALS/MND and is suggested to provide synergy with the ALS/MND standard-of-care drug, riluzole. An alternative mTOR inhibitor, rapamycin, is currently the subject of an ALS/MND clinical trial in humans investigating control of disease progression. Monepantel has a different structure to rapamycin and an apparently better safety profile. This Phase I Clinical Trial hypothesis is that monepantel administration to individuals living with ALS/MND will safely reduce disease associated protein accumulation in motor neurons and provide therapeutic benefit. To test this hypothesis, the safety and tolerability of oral monepantel administration and markers of efficacy will be tested in individuals living with ALS/MND in a dose escalating Phase I/II Clinical Trial. To mitigate risk, only patients with sporadic and certain known familial types of ALS will be eligible. To further mitigate risk, the monepantel starting dose will be reduced a calculated five-fold compared to that already used in human cancer patients and already demonstrated to be safe and effective as an mTOR inhibitor. Dependent upon incremental outcomes, three higher doses may then be tested, each for minimally 28 days with a duration at the optimal dose of at least six months.
Abstract: Context/background: people affected by Amyotrophic Lateral Sclerosis (ALS) see their own life totally disturbed after the diagnosis. This disease also courses, apart from the functional and depressing worsening, with internal damage manifested by a cardio respiratory deterioration. There are not many clinical studies publications about this disease given that is considered a weird illness with short prognosis. Objectives: to examine the effects of the inspiratory muscle training (IMT) on respiratory muscle strength, heart rate variability (HRV), quality of life and mood in patients with ALS. Methods: 20 volunteer patients, male and female, with ALS, bulbar or spinal will take part of the cuasi-experimental study and they will be divided into two groups: an experimental group (n = 10) and a control group (n = 10). The Maximum Inspiratory Pressure (PIM), the HRV, the quality of life and mood will be measured. The participants of experimental group will conduct 30 inspirations per day, 15 in the morning and 15 in the evening, 5 days per week, through 8 weeks. The resistance of the training in the experimental group will be increase acording to the PIM measured at the first visit. During the first week, the resistance will be at 30% of PImax, weeks 2 and 3 at 40%, weeks 4 and 5 at 50% and the last 3 weeks at 60%. After 8 weeks, all participants will fill up again all scales and post training measurements will be taken.
Anti alfa-3 and alfa-7 ganglionic cholinergic receptors (anti-AChRs) antibodies (Abs) plasma removal by plasmapheresis (1,2) acutely improved dysautonomia symptoms in case reports with Pure Autonomic Failure (PAF) (3). We shall assess the prevalence of anti-AChRs Ab and the relationship among Ab titer, cardiovascular autonomic profile and symptoms in neurodegenerative diseases characterized by similar dysautonomia symptoms such as PAF, Amyotrophic Lateral Sclerosis (ALS) and Postural Orthostatic Tachycardia Syndrome (POTS) (4). Ab positive patients will undergo selective immunoabsorption once a week up to achievement of Ab titer lower than 65% of baseline followed by immunosuppressive therapy with prednisone. Both Ab positive and negative groups will undergo anti-AChR Abs, autonomic profile and dysautonomia symptoms assessment, every 4 months up to 3 years. Evidence of correlation among reduced Ab titer and autonomic profile and symptoms improvement may result in new effective therapy.
The study will assess the safety of the drug enoxacin at specific dose levels in adults with ALS.
An open-label, single-center clinical trial to evaluate the safety and efficacy of repeated intrathecal administrations of autologous bone marrow derived mesenchyme stem cells in ALS patients. The study includes 20 subjects (age: 20-70) with definite diagnosis of ALS and ALS-FRS-R score of at least 20 and disease-duration of less than 3 years. The treatment protocol includes four intrathecal injections of MSC, at intervals of 3 months between the injections. The primary endpoints are safety and tolerability. Several efficacy measures are assessed as secondary endpoints.
The purpose of the study is to determine the frequency of mutations in the C9orf72 and SOD1 genes in the incident population of ALS patients followed in the FILSLAN centres
The purpose of this study is to assess the functional mobility and self-reported satisfaction with the Xavier electromyography hands-free wheelchair control system in comparison with a standard joystick.
MetFlex is an investigator led, open-label, single-arm, Phase 2a trial to determine the safety and tolerability of trimetazidine for the treatment of amyotrophic lateral sclerosis/motor neuron disease (ALS/MND).
Our objective in the proposed project is to: (a) operationalize and determine the feasibility and acceptability of a trial where clinic multi-disciplinary clinic (MDC) visits are audio/video recorded and shared with patients with ALS and their caregivers; (b) gather preliminary data examining the impact of routinely adding audio/video recordings of clinic visits to UC on self-management ability and other behavioral, health and health services outcomes at baseline (T0) and other regular interviews from enrollment (T1= 1 Week, T2= 3 Months); and (c) identify factors pertinent to the acceptability of our study protocol and the audio/video recording of visits.